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Covid 19 Pandemic: Parkinsons disease treatment Market Size, Share & Trends Analysis Report By Product, By Technology, By Application, By End Use,…

Global Parkinsons disease treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Parkinsons disease treatment Market Size Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

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Global Parkinsons disease treatment Market: Global Size, Trends, Competitive, Historical & Forecast Analysis, 2018-2024Scope of Global Parkinsons disease treatment Market Reports

Parkinsons disease is a neuro degenerative disorder in which parts of the brain become progressively damaged over many years. It is a type of movement disorder. It occurs when nerve tissue unable to produce a chemical named Dopamine which is used to send signals that help you move your body. Parkinsons disease usually developed for aged people i.e. above 60 years, but it can also start earlier. It is more common in men than in women. The cause of Parkinsons disease is unknown, but researchers think that both genetic and environmental factors are involved. Primary symptoms include tremors, Stiffness, Slowness, impaired balance, mild memory & Thinking problem and shuffling gait. Secondary symptoms include Anxiety, Depression, Fatigue, Low blood pressure, muscle cramp, Speech & communication problem, skin & Dental Problem and Dizziness, and Dementia. Risk of Parkinsons disease increases with age and the disease mainly affects person over 50 years of age. The development of Parkinsons disease and the degree of destruction differ from person to person. Many people with Parkinsons disease live long productive lives, whereas others become disabled much more quickly. As symptoms worsen, it may become difficult to walk, talk, and complete simple tasks. Malnutrition and weight maintenance is common problem for people with Parkinsons disease. There is no test to diagnose Parkinsons disease with certainty. Treatment options include medication or surgical treatment. Combination of Levodopa with carbidopa is considered as a gold standard for symptomatic treatment of Parkinsons disease. Surgical treatment options include deep brain stimulation and Carbidopa/levodopa enteral suspension therapy but surgical therapy is effective only for patients who have previously responded to Levodopa therapy. Commercially available drugs for treatment of Parkinsons disease are Sinemet, Safinamide, Rotigotine, Ropinirole, Pramiprexol, Amantidine, Cycloset, Parlodel (Bromocriptine Mesylate), Rytary, Azilect, Northera, Stalevo, Comtess/Comtan and others. More than past decade, huge improvement has been made in the treatment of Parkinsons disease such as identification of new therapeutic targets through genetic research, understanding the common mutations that contribute to Parkinsons disease etc.

Global Parkinsons diseases Treatment Market report is segmented on the basis of Medication, Medical devices, End user, Distribution Channels and Geography. Based on Medication Global Parkinsons disease Treatment Market is classified into Dopamine Precursors, Peripheral Decarboxylase Inhibitors, COMT (Catechol-O-methyltransferase) Inhibitors, MAO (Monoamine Oxidase), Inhibitors and Others (anticholinergics, antihistaminics etc.)

On the basis of Medical Devices Global Parkinsons disease Treatment Market is classified into Deep Brain Stimulation (DBS) Devices, Carbidopa/Levodopa Enteral Suspension (Duopa) delivery devices and others. On the basis of End User Global Parkinsons disease Treatment Market is classified into Hospitals, Homecare setting, Clinics and Others. On the basis of Distribution Channel Global Parkinsons disease Drug Market is classified into Retail Pharmacies, Online Pharmacies, Hospital Pharmacies and Drug Stores.

The regions covered in Global Parkinsons diseases Treatment Market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, Global Melanoma Drug Market sub divided in to U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for Global Parkinsons disease treatment Market Reports

Global Parkinsons disease Treatment Market reports cover prominent players like Teva Pharmaceuticals, AstraZeneca, Novartis AG, Boehringer Ingelheim, GlaxoSmithKline Plc., Salix Pharmaceuticals, mpax Laboratories, Orion Corporation, Mylan N.V., Cipla Inc., Daiichi Sankyo, Apotex Inc, Impax Laboratories, Lundbeck, Sun Pharma, Wockhardt ,UCB, Valeant Pharmaceuticals Acadia, Abbvie, Zydus Cadilla, Strides, 1 A Pharma, Upsher-Smith, Intas, US World Meds, Dr. Reddys, Medtronic plc, Biotie Therapies Corp., Adamas Pharmaceuticals Inc., Akorn Inc., Astellas Pharma Inc., Desitin Arzneimittel GmbH, Endo International plc, Kyowa Hakko Kirin, Newron Pharmaceuticals and Merck.

Global Parkinsons disease treatment Market Dynamics

Global Parkinsons disease Treatment Market is mainly driven by growing prevalence of Parkinsons disease and government funding for research on Parkinsons disease. The increase in number of geriatric population and technological advancements such as combination therapies for prolong action of continuous dopaminergic stimulation drugs, gene therapy, neural transplantation are few other causes which are possible to boost the Parkinsons disease Treatment market. However some restraints like availability of alternative treatments is expected to hold back market and expiry of patents for several drugs such as Stalevo, Azilect, Rytary, Comtan etc. can cause the termination of Parkinsons disease Treatment market. High cost of treatment such as 14.4 Billion USD and lack of expertise for early diagnosis is likely to restrict the market growth.

Also lack of novel and successful therapies in the market to reduce the risk of mortality restraints for this market. The opportunities for Global Parkinsons disease Treatment market includes upcoming technologies like surface EMG (Electromyography) sensors, Pulse oximetry sensors, development in personalized medicine along with huge investment in anti-Parkinsons drugs research will boost the global Parkinsons disease Treatment market. Also development of novel drugs and combination therapy with less side effects and better survival rates is expected to increase the global Parkinsons disease Treatment market.

Global Parkinsons disease treatment Market Regional Analysis

Europe is likely to achieve the largest share of the global Parkinsons disease treatment market, which is expected to be followed by North America with a sizeable market share. High knowledge about the treatment, increase in expenditure for Research & Development, favorable medicinal reimbursement regulations and policies, the high occurrence of Parkinsons disease, the rapidly growing geriatric population and potential medical pipeline products are likely to contribute to the major share of Europe in Global Parkinsons disease treatment market. The global Parkinsons disease treatment market in the Asia Pacific is anticipated to develop at a profitable rate. The market share of the global Parkinsons disease treatment market is to be expected to grasp by China and Japan in the Asia Pacific, due to the large population of elderly people who are affected with Parkinsons disease.

Furthermore, increase in awareness of disease and government initiatives for improving health care facilities are expected to boost the regional market to a certain extent.

Key Benefits for Global Parkinsons disease treatment Market Reports

Global Parkinsons disease Treatment Market report covers in depth historical and forecast analysis.Global Parkinsons disease Treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Global market sale (K Units), Global market Drivers, Market Restraints, Market opportunities, Competitive Analysis, Regional and Country Level.Global Parkinsons disease Treatment Market report helps to identify opportunities in market place.Global Parkinsons disease Treatment Market report covers extensive analysis of emerging trends and competitive landscape.Global Parkinsons disease treatment Market Segmentation

Global Parkinsons disease Treatment Market: By Medication Analysis

Dopamine PrecursorPeripheral Decarboxylase InhibitorsCOMT (Catechol-O-methyltransferase) InhibitorsMAO(monoamine oxidase) InhibitorsOthers (anticholinergics, antihistaminics etc.)Global Parkinsons disease Treatment Market: By Medical Devices Analysis

Deep Brain Stimulation (DBS) DevicesCarbidopa/Levodopa Enteral Suspension (Duopa) delivery devicesOthersGlobal Parkinsons disease Treatment Market: By End User Analysis

HospitalsClinicsHomecare settingOthersGlobal Parkinsons disease Treatment Market: By Distribution Channel

Retail PharmaciesOnline PharmaciesHospital PharmaciesDrug StoreGlobal Parkinsons disease Treatment Market: By Regional & Country Analysis

North AmericaU.S.MexicoCanadaEuropeUKFranceGermanyItalyAsia PacificChinaJapanIndiaSoutheast AsiaLatin AmericaBrazilThe Middle East and AfricaGCCAfricaRest of Middle East and AfricaNeed a PDF of the global market report? Visit:

Table of Content:

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the Global Parkinsons disease treatment Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the Worldwide Global Parkinsons disease treatment Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the Global Parkinsons disease treatment Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the Global Parkinsons disease treatment Market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the Global Parkinsons disease treatment Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This section of the research study shows how different end-user/application segments contribute to the Global Parkinsons disease treatment Market.

Market Forecast: Here, the report offers a complete forecast of the Global Parkinsons disease treatment Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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Covid 19 Pandemic: Parkinsons disease treatment Market Size, Share & Trends Analysis Report By Product, By Technology, By Application, By End Use,...

Hofseth Biocare ASA: Two-year research collaboration contract signed with Stanford on the use of Salmon Protein Hydrolysate peptides for the treatment…

Hofseth BioCare ASA (HBC) has signed a two-year research agreement with Stanford University School of Medicine, CA, USA to further investigate the gastro-intestinal (GI)-protective properties of HBCs Salmon Protein Hydrolysate Peptides (SPH) in children and adults with GI tract disorders. The research agreement follows recent findings of SPHs reduction of GI injury in both in vitro and in vivo pre-clinical models that reflect the underlying disease process of necrotizing enterocolitis (NEC) and ulcerative colitis / inflammatory bowel disease (IBD).

The focus of the research collaboration is to further elucidate how SPH reduces or prevents intestinal inflammation in preclinical models of NEC and IBD and thereby helps to restore GI barrier function. In the second year of the project, it is anticipated that this work may form the basis for proceeding to a pilot study to assess SPH as an adjunctive treatment for the maintenance of remission in human subjects with mild to moderate ulcerative colitis.

Prior research undertaken by HBC and Stanford has demonstrated that gene systems that protect the GI tract against oxidative stress and inflammation are upregulated in human GI cells exposed to SPH. In proprietary animal models of GI inflammation, Stanfords work has shown SPH to markedly reduce gut inflammation. Oxidative stress is implicated as an important underlying driver of many digestive diseases, including NEC and IBD.

IBD is a chronic disease that can affect both physical health and quality of life (QoL). Symptoms include crampy abdominal pain, diarrhoea and weight loss. Ongoing blood loss from the bowel can result in anaemia further exacerbating the impact on QoL with anaemia symptoms including fatigue and reduced exercise capacity. Long term effects of bowel inflammation include an increased risk of colon cancer. There are more than 70,000 new cases of IBD diagnosed each year in the US alone. Steroids and biologic therapies are commonly used treatments. Whilst these can provide significant benefit and are generally well tolerated, they are associated with side effects such as osteoporosis and diabetes with long term steroid use and serious infection with biologic therapy. Furthermore, a significant number of patients do not attain full remission and continue to suffer from the effects of IBD. There is therefore a clear need for further, well tolerated treatments which can help to attain and maintain remission for these patients.

For further information, please contact:James Berger, Head of Investor Relations & Strategy of Hofseth BioCare ASAPhone: +41 79 950 1034E-mail:

Dr. Bomi Framroze, Chief Science Officer at Hofseth BioCare ASAPhone: +1 650 283 1196E-mail:

About Hofseth BioCare ASA:HBC is a Norwegian biotech company that develops high-value ingredients and finished products. Ingredients are further developed into discovery and pre-clinical studies in multiple clinics and university research labs in several countries. Lead clinical and pre-clinical candidates are development towards a Gastro-Intestinal (GI) Protective Medical Food, Medical Food to help treat age-related Sarcopenia, and future phase 1 studies on treatment of Anemia and NEC-IBS with Salmon Protein Hydrolysate fractions.The company is founded on the core values of sustainability, traceability and optimal utilization of natural resources. Through an innovative hydrolysis technology, HBC can preserve the quality of lipids, proteins and calcium from fresh salmon off-cuts. Hofseth BioCare's headquarters are in lesund, Norway with branches in Oslo, London, Zrich, Chicago, Mumbai, Menlo Park and Tokyo.HBC is listed on Oslo Stock Exchange Axess list with ticker "HBC". More information about Hofseth BioCare at and

This information is subject to the disclosure requirements pursuant to Section 5-12 the Norwegian Securities Trading Act

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Hofseth Biocare ASA: Two-year research collaboration contract signed with Stanford on the use of Salmon Protein Hydrolysate peptides for the treatment...

Edited Transcript of FLXN earnings conference call or presentation 7-May-20 8:30pm GMT – Yahoo Finance

BURLINGTON May 8, 2020 (Thomson StreetEvents) -- Edited Transcript of Flexion Therapeutics Inc earnings conference call or presentation Thursday, May 7, 2020 at 8:30:00pm GMT

* David A. Arkowitz

Flexion Therapeutics, Inc. - CFO

Flexion Therapeutics, Inc. - Chief Commercial Officer

* Michael D. Clayman

Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director

Flexion Therapeutics, Inc. - VP of Corporate Communications & IR

* Randall S. Stanicky

RBC Capital Markets, Research Division - MD of Global Equity Research & Lead Analyst

Scott Young, Flexion Therapeutics, Inc. - VP of Corporate Communications & IR [1]

Good afternoon. This is Scott Young, Vice President of Corporate Communications and Investor Relations.

Before we begin, I'd call your attention to the metrics slides that we will discuss in today's presentation. Those slides can be viewed directly via the webcast or under the Investors tab on In addition, the press release we issued this afternoon and an archive of this conference call can also be found there.

Today's conference call will be led by Flexion's Chief Executive Officer, Dr. Michael Clayman; and he is joined by David Arkowitz, Chief Financial Officer; and Melissa Layman, Chief Commercial Officer.

On today's call, we will be making forward-looking statements that include commercial, financial, clinical and regulatory projections. Statements relating to future financial or business performance, conditions or strategies and other business matters, including expectations regarding net sales, operating expenses, cash utilization, clinical, regulatory and commercial developments and anticipated milestones are forward-looking statements within the meaning of the Private Securities Litigation Reform Act. Flexion cautions that these forward-looking statements are subject to various assumptions, risks and uncertainties which change over time. Additional information on the factors and risks that could affect Flexion's business, financial conditions and results of operations are contained in Flexion's Form 10-Q for the quarter ended March 31, 2020, filed with the SEC today and other filings which are available at as well as Flexion's website. These forward-looking statements speak only as of the date of this call and Flexion assumes no duty to update such statements.

I will now turn the call over to Flexion's CEO, Mike Clayman.

Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [2]

Thank you, Scott, and thank you all for joining us today.

It is clear that in the only 8 weeks since our fourth quarter earnings call, the world has changed dramatically as a result of COVID-19. On today's call, we'll talk about what we've been seeing in doing both commercially and operationally during the unprecedented public health crisis, but we'll begin with a focus on our first quarter performance and provide color on why we remain enthusiastically bullish about ZILRETTA's future.

Today, we reported net ZILRETTA sales of $20.1 million for the first quarter of 2020. We had previously indicated that we believed our Q1 sales would be roughly flat versus Q4 sales of $23.7 million, and we were on track to deliver sales in this range prior to the COVID-19 outbreak. We also said that at that point, it was simply impossible to predict how the outbreak would evolve or what the effects of more aggressive social distancing or other containment efforts might have on patients or practices. Since then, the picture has become much clearer, and we can speak now to the effects of the pandemic on ZILRETTA sales and later in the call, on our development activities.

Beginning in late Q1, we saw most practices substantially reduce patient visits or even suspend these altogether. And as a result, utilization of ZILRETTA dropped significantly. We believe it will take time to get to the new normal and the cadence of the recovery will vary practice by practice, region by region and even person by person. Thus far, we have seen decreased demand for ZILRETTA in the second quarter and believe COVID-19 will adversely impact ZILRETTA revenue for the remainder of the year.

That said, we do take note of a number of external circumstances that have the potential to increase demand for ZILRETTA over time. These all relate to the delay, postponement or cancellation of total knee arthroplasties or TKAs, and the intense medical need to provide rapid, substantial, durable non-opioid analgesia for patients whose knee pain drove them to consider TKA in the first place. Three factors contribute to this dynamic and these were all brought to our attention by orthopedic key opinion leaders.

First, while elective surgeries are beginning to happen around the country, in most cases, there will be a gradual return to full capacity, and many TKAs will be further delayed. Second, particularly for those of Medicare age, who are all considered in the vulnerable COVID-19 population, there may be reluctance to enter a hospital to have surgery until these patients have greater confidence they can avoid infectious risk. Finally, for those who are younger than 65 and were previously employed, many may prioritize income generation over surgery and rehab.

Recent survey data indicate that almost 50% of orthopedic surgeons expect that procedure volume will not be back to normal until some time in 2021 and 63% expect anywhere between 25% and 75% of their patients will continue to delay their surgery for fear of COVID-19.

Of course, Flexion is adapting, too. Since instructing our employees to work from home in mid-March, our MBMs have adopted various technologies to engage in meaningful conversations with prescribing physicians. Melissa can provide more color on what we are seeing in the field, but since health care practitioners have more time on their hands, our MBMs have experienced greater access to prescribers who have historically been very difficult to see, and their response to the ZILRETTA clinical data has been highly encouraging. And even with COVID-19, we continue to hear stories of patients who have received unprecedented OA knee pain relief from ZILRETTA.

The bottom line is that our confidence in ZILRETTA's clinical performance and long-term potential is undiminished. We are not the only company to believe that ZILRETTA holds tremendous potential for millions of patients with OA knee pain. In April, in the midst of the pandemic, we announced that we entered into an exclusive license agreement with HK Tainuo and Jiangsu Tainuo, a subsidiary of CSPC, a leading Chinese biopharmaceutical company. Under the terms of the agreement, we are entitled to receive an upfront payment of $10 million and up to $32.5 million in development, regulatory and commercial sales milestone payments. And Flexion will be solely responsible for the manufacturing and supply of ZILRETTA for all clinical and commercial activities.

While our confidence in ZILRETTA is resolute, it is impossible for us to predict how long the pandemic will affect sales. And as we announced in our press release this afternoon, we have taken meaningful actions to reduce our operating expenses in the near term. David will discuss these steps in more detail, but we are committed to ensuring that we come out of the pandemic in a strong financial position.

Regarding clinical development, recognizing that many clinical trial sites had shut down, wanting to ensure the safety of our research participants and in consideration of guidance issued by the FDA, in early April we announced that we would temporarily suspend our Phase II clinical trial evaluating the efficacy of ZILRETTA in patients with shoulder OA or adhesive capsulitis and also our Phase I clinical trial evaluating the safety and tolerability of FX201, our intra-articular gene therapy candidate for OA.

Subsequently, we made the strategic decision to discontinue our Phase II trial investigating ZILRETTA in shoulder OA and in adhesive capsulitis. Given the small number of patients enrolled in that trial, the uncertainty of when we would be able to restart it, as well as the costs required to maintain the study in an inactive status, we believe terminating the trial is the most responsible action at this time. Moving forward, we will look to leverage our learnings from the study and potentially use these to refine the trial design to advance ZILRETTA in these indications.

We also aim to reinitiate the FX201 single ascending dose trial and recommence enrollment when conditions and staffing at our trial sites safely permit the return of patients.

Turning to FX301, our NaV1.7 inhibitor formulated within a thermosensitive hydrogel for administration as a peripheral nerve block for controlled postoperative pain, we continue to advance this product candidate. FX301 is a liquid at room temperature and gels following injection at body temperature to create a controlled release depot that can provide persistent therapeutic concentrations of drug locally at the selected nerve. We recently unveiled new preclinical data in a virtual poster presentation now available on the American Society of Regional Anesthesia and Acute Pain Medicine website. These data show that compared to liposomal bupivacaine and placebo, FX301 provided sustained postoperative analgesic effect with no impairment in motor function. In addition, high local concentrations of funapide, the active ingredient in FX301, were measured at the site of administration for the duration of the study, consistent with the creation of the depot providing controlled drug release.

While it is still early days for the program, these findings support our belief that unlike typical local anesthetics, the selective pharmacology of FX301 has the potential to deliver substantial and persistent pain relief while preserving motor function which could enable earlier rehabilitation following musculoskeletal surgery and potentially more rapid discharge from the hospital. We are completing GLP tox studies for FX301, and we remain on track to initiate clinical trials in 2021.

Before I turn it over to Melissa, I would like to make just a few final comments. While there are many uncertainties about the shape and pace of the recovery, there are some key facts we know right now. We know that there are millions of patients for whom ZILRETTA can provide substantial and extended relief from OA knee pain. We know that at least many tens of thousands of elective knee replacements have been deferred due to COVID-19 and those patients will need effective pain relief while they wait for surgery. We know that our commercial organization is poised for the recovery at whatever pace or shape it takes. Finally, we know that everyone at Flexion stands ready to adapt to any circumstances we face, and we remain confident in our future prospects.

At this point, I would like to turn it over to Melissa to discuss our commercial operations.


Melissa Layman, Flexion Therapeutics, Inc. - Chief Commercial Officer [3]


Thank you, Mike.

I've been with Flexion now for just under 2 months, my first day marked by our Q4 earnings call and my second day by the work-from-home directive resulting from the rise of the COVID-19 pandemic. Despite having yet to set foot in the field, I've met virtually with dozens of physicians and members of our sales organization. Each of these meetings has only confirmed what I believed about ZILRETTA before joining the team and bolstered my perceptions of the Flexion organization, particularly in light of our response to COVID-19 and the enhanced role I know ZILRETTA can play in effective, durable pain management for patients all along the OA knee pain treatment continuum.

Mike discussed some of what we have experienced in the field during recent weeks. I'll provide some additional color on how we've been engaging with prescribers during the crisis and how we plan to emerge from the crisis, restore ZILRETTA's prior sales trajectory and continue to grow from there. Before I talk about what we are seeing now, I'd like to start by walking through the commercial metrics for the first quarter.

As Mike mentioned, we were pleased with the way the first quarter was progressing prior to the onset of COVID-19 and believe ZILRETTA was tracking to meet our expectations. You can see in Slide 2 that we recorded ZILRETTA net sales of $20.1 million, reflecting the pandemic's material impact on March sales. We expanded our list of target accounts in the fourth quarter to 5,000 and by March 31, 2020, our reach had extended to almost all of them. And by the end of the first quarter, 3,672 accounts had purchased ZILRETTA, which was an increase of 184 purchasing accounts compared to the fourth quarter of 2019. As of the end of March, 2,832 or 77% of accounts had reordered ZILRETTA. Notably, this increase in purchasers occurred off a customer base that continued to grow even in light of the pandemic.

On Slide 3, you can see how our sales have progressed since launch. And as we've mentioned, we were pleased with the performance in the first part of Q1 prior to the onset of COVID-19, when we were on track to deliver sales in line with fourth quarter of last year, consistent with our previous guidance.

The next 3 slides reflect ZILRETTA purchasing patterns across our growing account base comprised of practices, clinics and hospitals of various sizes and potential.

Slide 4 describes the cumulative number of accounts that have purchased ZILRETTA since launch as well as the distribution of quarterly purchase volumes across our account base. In Q1, the trend continued with a growing number of accounts purchasing within and across the purchase break of 1 to 10 units, 11 to 50 units and 51 or more units with significant growth to 916 accounts in the 51-plus category.

Slide 5 flips the perspective, looking at the cumulative distribution of ZILRETTA purchases by accounts. For example, those 916 accounts in the 51-plus purchase group shown on the previous slide have been responsible for 177,664 of the total units purchased since launch and roughly 35,000 more units than were purchased from this purchase volume group in Q4 2019. This clearly illustrates that purchasing accounts continue to move through the ZILRETTA utilization continuum described on previous calls.

As we've also previously highlighted, even our largest accounts remain underpenetrated, and we continue to believe that there is tremendous opportunity to increase ZILRETTA utilization within and across each of these groups.

It is also important to point out that while total ZILRETTA purchases in the first quarter were approximately 36,500 units, which is slightly lower than the 37,500 units purchased in the fourth quarter, through the middle of March, we were pleased with our progress and believe we were well on track to meet or exceed the fourth quarter purchase volume until the effects of COVID-19 began to impact our business and ZILRETTA purchases dropped precipitously.

In our final slide, #6, we break out ZILRETTA purchasing by new and existing accounts. In Q1, new accounts purchased 12,504 units, which further illustrates our strong start to 2020 prior to the onset of COVID-19 across the U.S.

As Mike mentioned, COVID-19 has had significant impact on our customers. Throughout the COVID-19 impacted window, overall clinician access to ZILRETTA-eligible patients has been limited, variable and practice dependent. Some practices have shut down in-person patient visits altogether, while others are open to high priority critical or acute cases while implementing social distancing measures. While the circumstances vary, one common denominator has been the presence and tenacity of our sales force. While not a replacement for in-person exchanges, our reps have been successful in targeted remote detailing, in some cases gaining access to prescribers that exceeds pre-COVID-19 levels. They have effectively employed various audiovisual technologies to engage with both existing and new accounts to discuss the important role ZILRETTA can play in the OA treatment paradigm, including for those patients who are awaiting or opt to decline surgery once COVID-19 restrictions are lifted.

To better understand the current impact of COVID-19 on orthopedic practice and what the recovery might look like, we held a series of focus groups with specialists from across the nation. The insight from those exchanges support our assumptions that the return to new normalized practice patient flow will be gradual and driven by ongoing social distancing requirements and safety requirements as well as patients' comfort in returning to their doctors' offices. However, we also see the potential for increased use of ZILRETTA in a wider variety of patient types as both existing and new prescribers look to manage the growing backlog of surgical candidates, including those previously scheduled for TKR who, for various COVID-19-related reasons, choose to defer treatment in favor of less invasive options like ZILRETTA.

As Mike aptly described, we cannot predict the timing or shape of the recovery. But as it occurs, our commercial team will be there to support prescribers and ensure they understand the even more important role ZILRETTA can play in effectively managing their patients' OA knee pain prior to or in avoidance of surgery.

With that, I'll now turn it over to David for the financial update.


David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [4]


Thank you, Melissa.

Before I provide an overview of our financial performance in the first quarter, I'd like to share some details on our fiscal response to the economic and business disruption caused by COVID-19.

As Mike mentioned and as we described in our first quarter earnings press release, we undertook a series of prudent and disciplined steps to reduce expenses across the organization in order to enhance our financial flexibility and liquidity. In total, we believe these steps will deliver between $43 million and $53 million in savings this year. As a result, our full year 2020 total operating expenses, which include the cost of sales, research and development, and selling, general and administrative expenses, are expected to be in the range of $167 million to $177 million.

We undertook cost savings initiatives across every area of the business, but the majority of the savings will be realized through the following: hiring and travel freezes, suspension and/or termination of active clinical trials, elimination of live presence at medical and industry conferences, reductions in in-person physician speaker programs and reductions in market research and select marketing programs and materials as well as elimination of nonessential operating expenses.

In addition, given the impact of COVID-19 and the uncertainty surrounding ZILRETTA sales for the remainder of this year, we are temporarily suspending manufacturing activities for ZILRETTA to avoid excessive inventory buildup and to reduce costs. We believe our current inventory of finished goods will be sufficient to meet demand for ZILRETTA through at least the remainder of this year. And unlike typical contract manufacturing agreements, our condominium model at Patheon provides us with the ability to scale up production based on market dynamics. And once we determine that additional supply will be needed, we can reinitiate manufacturing.

With respect to our first quarter financial performance, we reported a net loss of $36.8 million for the first quarter of 2020 compared to a net loss of $41.5 million for the same period of 2019.

Net sales of ZILRETTA were $20.1 million and $10.6 million for the 3 months ended March 31, 2020 and 2019, respectively. Cost of sales were $2.3 million and $1.8 million for the 3 months ended March 31, 2020 and 2019, respectively. The first quarter 2020 net sales reflect a gross to net reduction of 11%. The gross to net reduction is primarily comprised of distributor and service fees, returns reserve, health care provider rebates and mandatory government discounts and rebates, such as Medicaid, 340B institutions, Veterans Administration and Department of Defense. The rebates to health care providers are variable based on the volume of product purchased and contribute 2% of the first quarter total gross to net reduction of 11%.

As Melissa mentioned, starting in the middle of March, purchases of ZILRETTA by accounts, meaning physician practices, clinics and hospitals, dropped materially due to COVID-19. As a result, the inventory levels held by our distributors at the end of the first quarter were higher than our target level of 1 to 3 weeks. While purchases of ZILRETTA by accounts continue at a decreased rate, it will take time for our distributors to work through their current inventory. Since we recognize revenue upon sales to our distributors, a combination of reduced purchases in the second quarter by account and higher-than-normal distributor inventory levels have the potential to particularly impact our second quarter revenue negatively.

Research and development expenses were $21.1 million and $15.4 million for the 3 months ended March 31, 2020 and 2019, respectively. The increase in research and development expenses of $5.7 million was primarily due to an increase of $2.2 million in salary and other employee-related costs for additional headcount and stock compensation expense; an increase in expenses related to FX201, including the $2.5 million milestone to GeneQuine for dosing the first human patient in the Phase I clinical trial; and an increase in other portfolio expenses primarily related to a $0.5 million milestone to Xenon Pharmaceuticals, offset by a decrease of $0.5 million in development expenses for ZILRETTA due to lower clinical trial expenses during the period.

Selling, general and administrative expenses were $29.3 million and $32.2 million for the 3 months ended March 31, 2020 and 2019, respectively. Selling expenses were $20.5 million and $23.8 million for the 3 months ended March 31, 2020 and 2019, respectively. The year-over-year decrease in selling expenses of $3.3 million was primarily due to a reduction in physician and patient marketing activities. General and administrative expenses were $8.8 million and $8.4 million for the 3 months ended March 31, 2020 and 2019, respectively, which represents an increase of $0.4 million.

Interest expense was $0.4 million and $1 million for the 3 months ended March 31, 2020 and 2019, respectively. Interest expense was $4.7 million and $3.9 million for the 3 months ended March 31, 2020 and 2019, respectively.

As of March 31, 2020, we had approximately $125.2 million in cash, cash equivalents and marketable securities compared with $136.7 million as of December 31, 2019.

At this point in time, we cannot forecast with any precision the duration of the pandemic or its full impact on purchasing patterns or utilization of ZILRETTA. Therefore, it would be imprudent for us to provide specific guidance with respect to our future sales of ZILRETTA or our cash runway. We will continue to review and reassess our assumptions and provide additional color when possible. As Mike mentioned, our confidence in ZILRETTA is as strong as ever, and we will take the steps necessary to ensure we are well positioned from a financial perspective to maximize ZILRETTA's value in a post-COVID-19 environment.

At this point, I would ask the operator to please open the line for questions.


Questions and Answers


Operator [1]


(Operator Instructions) Our first question or comment comes from the line of Randall Stanicky from RBC Capital Markets.


Randall S. Stanicky, RBC Capital Markets, Research Division - MD of Global Equity Research & Lead Analyst [2]


Great. Do you have a sense from any of the regions or the large practices that have reopened as to what their ordering patterns look like? Because presumably, that would give you a sense for at least physician or practice thinking around any pent-up demand and then where they intend to focus on from a procedure perspective kind of channel inventory aside. And then I have a follow-up.


Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [3]


Melissa, do you want to take that?


Melissa Layman, Flexion Therapeutics, Inc. - Chief Commercial Officer [4]


Yes. I think that's me. We have been sort of following the basis for which practices have been reopening and it is not geographically specific. We believe that the return to normal patient volume is going to vary by region, by practice and even by patient. And the impact to existing office-based patient flow that we've seen don't necessarily correlate to the areas where COVID-19 has been more prevalent.


Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [5]


And just building on Melissa. I mean, Randall, I just think it's too early for us to declare that we're seeing patterns. We're seeing some accounts order substantial quantities, other accounts ordering lesser amounts, but it's far from us having an update and then say, we have clarity on ordering pattern.


Randall S. Stanicky, RBC Capital Markets, Research Division - MD of Global Equity Research & Lead Analyst [6]


Let me ask a follow-up. I mean, during this shutdown period, which in some regions, regions like New York is probably going to continue, have you guys been able to alter your outreach via DTC? I mean, you have captive patients who are homebound, who are probably in pain, maybe thinking about the timing of elective surgeries. Have you figured out a way to access those patients to get ZILRETTA as an option in front of them?


Melissa Layman, Flexion Therapeutics, Inc. - Chief Commercial Officer [7]


So with regard to accessing patients specifically, our efforts haven't been focused there during this period. They have been focused specifically with our health care providers. Our sales force has been working from home since the middle of March, and they have been fully audiovisual equipped to continue to engage physicians remotely. And in fact, many of their conversations with our users and some of our nonusers have focused around that very point, which is that with surgeries having been rescheduled, postponed, canceled altogether, the wait times to get to those surgeries are mounting and that ZILRETTA offers an alternative to managing the intractable pain that these patients are continuing to suffer with and therefore is being more widely considered by our customers as that bridging tool that they can use until those patients are able to be put back on to the surgical schedule.


Operator [8]

See the article here:
Edited Transcript of FLXN earnings conference call or presentation 7-May-20 8:30pm GMT - Yahoo Finance

The medicine of music: Tim Vallillee turns to song as therapy for cystic fibrosis and for life –


For Tim Vallillee, music is medicine.

Something he can reach out to when hes hurting and when hes not. Something he can tap into for his physical health and his soul.

Something, most importantly,he can share with others.

Ive always felt that the fact I sing so much has been added therapy for me, says Vallillee, 52, who has cystic fibrosis.

Vallillee grew up in Yarmouth and lives in the Valley with his wife Agatha and son Isaiah. When COVID-19 came into play, he lost his gigs as venues were no longer open to the public.

When theyre taken away your musical brain says, this really sucks. But my CF brain says I cant do my thing, how am I going to tell how Im feeling?

Singing is a barometer that I use for the level of my health, he explains, likening it to an early warning device that lets him know how hes feeling inside especially when hes reaching for those high notes.

Ive got a pretty powerful voice but you cant do that without air in your lungs, he says. If that air is not in there it shows in my voice. It can be very subtle, but I notice it. I know something is brewing.

But where theres a will, theres a way.

Vallillee has been performing and sharing music Thursday evenings via Facebook live on his personal social media page. For his first session he put his iPhone in a tripod, sat on his bed and played for a couple of hours. Its now a highlight of his week.

Its great because I get to do what I like to do and its all part of my life therapy to have music in my life.

And hes happy to have that connection with others again.

In addition to Facebook you can also hear his music on YouTube. He also has a website to connect with others

Vallillee learned to play the guitar when he was 18. But it was something that happened when he was 16 that chartered his musical course.

My dad had a massive heart attack and when he was in the hospital . . . I promised my dad that if he wouldnt die I would learn to play an instrument, he says. He always wanted one of his four kids to learn an instrument. He always joked the best thing we could play was the record player.

His father recovered and a couple of years later reminded Vallillee of his promise. So the son grabbed a guitar and taught himself to play.

He ended up playing in many bands with friends. One of his first gigs was a variety show during Yarmouth high schools winter carnival in the 1980s.

Vallillee turns to music to express himself when hes feeling happy, sad, joyful, fearful sometimes bored and, more recently, heartbroken. His most recent original song is called Home To Me.

Its a song he wrote about what a special place Nova Scotia is. He wrote the song following the April mass shooting tragedy in the province. In the lyrics he picked out places and experiences in Nova Scotiathat he connects with and hopes others will as well.

I tried to encompass as many people as I could from Yarmouth to Cape Breton, he says.

Vallillee didnt want the song to just be about healing right now but a song people can listen to at any point in their life, whether next week, next month, or next year, and it will make them feel good.

Folks from this part of the country, are different from all the rest. We've got a special something deep down in our chest.

Nova Scotia is where I roam...Nova Scotia, that's my home. Nova Scotia is where I'll be ... Nova Scotia, is home to me.

Vallillee'swife, Agatha Bourassa, says not only is music where her husband shines, its a gift he gives to others.

This great song idea came about like every one of Tims songs have over the past two decades. Nobody really knows how he writes but he sparks an idea. Sometimes he pulls off on the side of the road and all of a sudden he regurgitates a song, she says. We just lived through this horrific tragedy in Nova Scotia and maybe no one feels like celebrating but Tim truly wrote that song to try and uplift every Nova Scotian. Lets celebrate life because every breath is worth it.

Indeed. Breaths are something those with cystic fibrosis cant take for granted. Which makes life challenging during the COVID-19 pandemic. People with immune deficiencies and underlying health issues are especially at risk.

Still, Vallillee isnt doing anything drastically different now than he does in normal times. Thats because, in a sense, hes always living in a COVID reality.

I used to say Im one bug away from getting sick and ending up in the hospital and dying in a week Ive always lived that perspective, he says, adding those with CF are very used to avoiding germs.

He calls himself a germ ninja.

Its not like I normally go around in a mask, but I will in a hospital. And Im vigilantabout always trying to wash my hands and avoid germs.

My wife, God love her, if she hears anybody coughing or sneezing in my direction even if were sitting a movie theatre and she hears somebody cough behind or near us its like weve got to move, he says. Shes seen me knock on deaths door quite a number of times. Thankfully we survived that.

Fortunately for Vallillee and his family, his health has been good in recent years. He hasnt been in the hospital in abouteight years and he attributes this to Kalydeco, a new medication he received six years ago. Its been a game-changer.

It basically goes in and changes CF genes inside my body on a genetic level. Because of that my lung function jumped about 20 per cent when I first got on it, he says. It virtually saved my life. I havent been in the hospital since. Ive had a couple of rounds of being sick, but never to the point that I was hospitalized.

The health of others with cystic fibrosis is also never far from Vallillees thoughts. Hes been promoting an online House of Commons petition aimed at helping Canadians with cystic fibrosis, cancer and other life-threatening diseases to have access to medicines and clinical trials that could save, prolong and/or improve their lives.

And then there is fundraising that has always been a special cause for Vallillees family, especially during May, which is Cystic Fibrosis Month.

COVID-19 will have an impact in terms of preventing people from coming together physically in large numbers to fundraise. In some areas that held traditional walks each May, virtual walks are being held instead.

In other words, COVID wont silence efforts in more ways than one.

On Saturday, May 16, from noonto 12 a.m. he and others will take part in an event called the 12-Hour Sing-A-Thon for Cystic Fibrosis via Facebook live. Darrin Harvey of 89.3 K-Rock will the celebrity host for the kickoff that day and Vallillee and Eben Higgins (both living with CF) will perform and also present numerous East Coast musicians from the region. The goal is to entertain, raise awareness and fundraise. Information about 12-Hour Sing-A-Thon for Cystic Fibrosis can be found on Facebook.

Vallillee is the second oldest person that he knows of in Nova Scotia living with cystic fibrosis.

Every cystic has this timeline, but we dont know what the timeline is, its all dependent on how healthy we are, if we take care of ourselves, if we get exposed to some bug, he says.

But Vallillee doesnt focus on getting sick. Instead, his focus is always on staying well.

And thats where his songs help.

Its the medicine of music, he says.

And he hopes its contagious.

Cystic fibrosis (CF) is the most common fatal genetic disease affecting Canadian children and young adults. At present, there is no cure.

CF causes various effects on the body, but mainly affects the digestive system and lungs. The degree of CF severity differs from person to person, however, the persistence and ongoing infection in the lungs, with the destruction of lungs and loss of lung function, will eventually lead to death in the majority of people with CF.

Typical complications caused by cystic fibrosis are:

It is estimatedone in every 3,600 children born in Canada has CF. More than 4,300 Canadian children, adolescents, and adults with cystic fibrosis attend specialized CF clinics.

Visit the Cystic Fibrosis Canada website to learn more and to see how you can donate.

You can also visit the Cystic Fibrosis Atlantic Canada website

CF is a multi-system disorder that produces a variety of symptoms including:

CF is a genetic disease that occurs when a child inherits two defective copies of the gene responsible for cystic fibrosis, one from each parent. Approximately, one in 25 Canadians carry one defective copy of the CF gene. Carriers do not have CF, nor do they exhibit any of the related symptoms.

When two CF carriers have a child, there is a 25 percent chance that the child will be born with CF. There is also a 50 per cent chance that the child will be a carrier, and a 25 per cent chance that the child will not be a carrier, nor have CF.


The medicine of music: Tim Vallillee turns to song as therapy for cystic fibrosis and for life -

Edited Transcript of CLVS earnings conference call or presentation 5-May-20 8:30pm GMT – Yahoo Finance

Boulder May 6, 2020 (Thomson StreetEvents) -- Edited Transcript of Clovis Oncology Inc earnings conference call or presentation Tuesday, May 5, 2020 at 8:30:00pm GMT

Clovis Oncology, Inc. - VP of IR

* Daniel W. Muehl

Clovis Oncology, Inc. - Executive VP & CFO

* Patrick J. Mahaffy

Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director

SVB Leerink LLC, Research Division - MD of Targeted Oncology & Senior Research Analyst

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

* Kennen B. MacKay

RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research

Thank you for standing by, and welcome to the Clovis Oncology First Quarter 2020 Financial Results Conference Call. (Operator Instructions) Please be advised that today's conference is being recorded. (Operator Instructions) I'd now like to hand the conference over to your speaker today, Anna Sussman, Vice President of Investor Relations. Thank you. Please go ahead.

Anna Sussman, Clovis Oncology, Inc. - VP of IR [2]

Thanks, Jessie. Good afternoon, everyone. Welcome to the Clovis Oncology First Quarter 2020 Conference Call. Thank you for joining us. You've likely seen this afternoon's news release. If not, it's available on our website. As a reminder, this conference call is being recorded and webcast. Remarks may be accessed live on our website during the call and will be available in our archive for the next several weeks.

Today's agenda includes the following: Pat Mahaffy, our President and CEO, will discuss the key components and highlights of today's corporate update, including commentary about any potential impact related to COVID-19; then Dan Muehl, Clovis' Chief Financial Officer, will cover the quarter's financial results in greater detail; Pat will make a few closing remarks; and then we'll open the call for Q&A, during which time, Lindsey Rolfe, our Chief Medical Officer, will also be available to answer questions.

Before we begin, please note that during today's conference call, we may make forward-looking statements within the means of the federal securities laws, including statements concerning our financial outlook and expected business plans. All of these statements are subject to risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Our actual results could differ materially due to a number of factors, including the extent and duration of the effects of the COVID-19 pandemic. Please refer to our recent filings with the SEC for a full review of the risks and uncertainties associated with our business. Forward-looking statements speak only as of the date on which they are made, and Clovis undertakes no obligation to update or revise any forward-looking statements.

Now I'll turn the call over to Pat Mahaffy.

Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [3]


Thanks, Anna. Welcome, everybody. Appreciate you taking the time today. As we all know, the world has changed so much since our last quarterly update in late February. It's a complicated time for all of you, I know. Health care professionals have been and remain on the front lines of this global pandemic and I'd like to acknowledge the contributions of health care workers around the world, putting their lives on the line to care for those affected by COVID-19.

Closer to home, we'd also like to recognize the tremendous effort being made by our investigators and prescribers to maintain enrollment and safely manage ongoing patients in our clinical trials and for continuing to prescribe and manage Rubraca commercial patients during this period of significant upheaval to their clinics and practices.

I'll use our time today to discuss the highlights of the topics you've come to expect on our quarterly calls and focus on providing additional color on how we are navigating the COVID-19 world here at Clovis, and our view of how COVID-19 may affect oncology treatment going forward, and then we'll open it up for Q&A with Dan, Lindsey and myself.

Let's begin with a commercial update for Rubraca. I'm pleased to report that we had a very encouraging first quarter. Our global net revenue was $42.6 million. This represents an 8% sequential increase from Q4 2019 and 29% increase over Q1 2019. This was our best quarter of sales to date, despite the fact that reps had to begin staying home beginning in mid-March in the United States and could no longer call on health care providers in person for the last few weeks of the quarter. Also, our European launches in Italy, Spain and France are all occurring in an environment in which our field-based personnel have not been allowed to visit hospitals or clinics beginning in late February and are therefore also working from home.

Given these circumstances, we are very pleased with our sales growth in the first quarter. And now I'll share why we believe that Rubraca is well positioned as an oncology treatment option in the current acute COVID-19 era, and in the chronic COVID environment that is sure to follow. This period has been very disruptive for hospitals, clinics and patients as health care professionals are redirected, broadly described elective procedures are delayed and health care facilities are converted to support COVID-19 treatment efforts. We do believe that oncology will be among the first health care specialty to return to some normalcy so that likely means adapting to a new normal in a chronic COVID-19 world, one in which there is a focus on minimizing clinical visits to avoid risk to patients, especially cancer patients and other patients with known comorbidities.

It is also clear that cancer patients will need to be diagnosed and treated, given the evident risk in not actively managing their disease. We believe that Rubraca, a convenient oral therapy has significant advantages as a maintenance option in the recurrent ovarian cancer setting in an environment in, as I described, physicians are trying to reduce patient visits to their clinics. Unlike Avastin as a maintenance option that requires frequent infusions and weekly monitoring for hypertension, a known risk factor for COVID-19, Rubraca is an oral agent and is taken at home and only requires monthly routine monitor. Unlike observation, which on average leads to disease progression and requires a return to immunosuppressive chemotherapy after approximately 5 months, Rubraca has been shown to extend progression-free survival and therefore, subsequent chemotherapy, on average, nearly 14 months by independent assessment, nearly 3x longer than placebo. In fact, observation is an invitation to infusion. And unlike ZEJULA, which requires weekly blood monitoring for the first month, which obviously requires weekly visits to the clinic or a laboratory, Rubraca requires only monthly routine monitoring. As you can see, Rubraca offers numerous potential advantages in a chronic COVID-19 world, and we have already introduced a variety of new digital materials for our now home-based field personnel we use to engage with hospitals, clinics, doctors and pharmacies.

While we may see some near-term impact on revenues as physicians adapt their practices to COVID-19, we believe these advantages will remain over the course of this year and future years and as we all know, COVID-19 is not likely going away in the near term.

In addition to seeking to establish Rubraca as the maintenance treatment option of choice in recurrent ovarian cancer, we also look forward to the potential launch in the United States of Rubraca in advanced mutant BRCA prostate cancer, and that brings us to our most near-term development and regulatory program in this setting.

In November 2019, we submitted our planned supplemental new drug application, or sNDA for Rubraca as a monotherapy treatment of adult patients with BRCA1/2 mutant recurrent, metastatic CRPC. The FDA filing was based on data from the TRITON2 clinical program in advanced prostate cancer. In the U.S., by the way, approximately 12% of men with metastatic CRPC have a mutation of BRCA1/2 in their tumor.

In January 2020, we announced that the FDA accepted our sNDA for Rubraca and granted priority review status to the application with the PDUFA date of May 15, 2020. Based on our interactions with the FDA, we have no reason to expect any delay to our May 15 PDUFA date. We think that Rubraca represents an important hormone-free and chemotherapy-free option for men with metastatic CRPC and a BRCA1/2 mutation. Recall that we've previously reported at ESMO last fall, a confirmed objective response rate of 44% by investigator and a confirmed PSA response of 52%. The safety data for men with CRPC were consistent with prior safety reports for patients with ovarian cancer and other solid tumors.

We've been engaged by -- encouraged by our interactions with both the medical oncology and urology communities about the potential for Rubraca to address the unmet medical need in recurrent metastatic CRPC. We are actively engaged in launch preparations, including sales force training that was completed in early March, and we will be ready to launch upon approval. Obviously, this will be among the first group of oncology launches that we'll incur entirely or almost entirely virtually. And we have taken considerable effort to prepare for this virtual launch. Our field sales team is prepared to initiate Zoom-based sales calls with prescribers, and will leverage learnings accumulated through their virtual selling efforts in the ovarian cancer setting since mid-March. All launch collateral for the sales team has been digitized to ensure they have the ability to utilize resources in virtual interactions. The promotional national broadcast has been fully converted to a virtual streaming program enabling HCPs to watch from any computer or iPad or any device in their office or home. Additional broadcast times have been added to ensure flexibility across all U.S. time zones. Program registration will be aided through targeted online advertising that will commence the day of approval. Media and advertising efforts have been weighted toward digital programming versus print to maximize impact and effectiveness of resources invested.

So to be clear, we will be ready to launch in prostate even in this new environment. Let me turn now to the clinical pipeline for Rubraca and lucitanib as well as our ongoing plans for FAP-2286. To begin, we are adhering to the regulatory guidance that FDA and other agencies have provided regarding clinical trial conduct during COVID-19, and our clinical teams are working closely with investigators to assure the safety of trial participants and investigators while maintaining compliance with good clinical practice and minimizing risk to the integrity of our trials.

While we did not see any material disruption to our clinical trials as a result of COVID-19 during the first quarter, it is possible that near-term effects may begin to emerge across different aspects of our clinical trial programs. For example, new patient recruitment in certain clinical studies may be affected, and the conduct of clinical trials may vary by geography as some regions are more adversely affected. I will note that we continue to anticipate completing enrollment in our largest study, the ATHENA frontline maintenance study before the end of this quarter. The LODESTAR study, our Phase II pan-tumor study to evaluate Rubraca in homologous recombination repair genes across tumor types continues to enroll patients. The study will evaluate Rubraca in patients with recurrent solid tumors associated with the deleterious homologous recombination repair or HRR gene mutation. Based on our interactions with FDA, this study may be registration-enabling for a targeted gene and tumor-agnostic label. If enrollment continues as planned, we could potentially file for approval in 2021.

Next, I'd like to briefly highlight our combination studies with BMS for both Rubraca and lucitanib, and then discuss our newest compound, 2286. We remain enthusiastic about our ongoing clinical collaboration with Bristol-Myers Squibb and I'll take a moment to review certain of our combination studies for both Rubraca and lucitanib with nivolumab. I'll begin with the Rubraca combinations.

FRACTION-GC is a BMS-sponsored multi-arm Phase II study evaluating the combinations of each of Opdivo and Yervoy with Rubraca as well as Opdivo, Yervoy and Rubraca in combination for the treatment of advanced gastric cancer. This is the first sponsored study to explore this triplet combination, and it is currently enrolling patients into the safety lead-in portion of the study. The Clovis-sponsored Phase III ATHENA trial in first-line maintenance for advanced ovarian cancer continues to enroll well, despite the COVID-19 environment. And as I noted, we continue to anticipate completing enrollment in this 1,000 patient study in the second quarter of 2020.

With ATHENA, we believe we are uniquely positioned to evaluate Rubraca in terms of 2 outcomes as monotherapy versus placebo in the first-line maintenance setting in the HRD population, inclusive of BRCA and in the all-comers or intent-to-treat population as well as any potential advantage of the combination of Rubraca and Opdivo in the same patient populations. ATHENA is the first frontline switch maintenance study designed to show both PARP monotherapy and PARP/PD-1 combination therapy in one study design. I'll take a moment to remind you of the statistical analysis planned for ATHENA. First, expected in the second half of next year, we will see the results of Rubraca monotherapy versus placebo in all study populations. And then probably a year or more later, we will see the results of Rubraca plus Opdivo versus Rubraca in all study populations. In each of these analyses, we will first evaluate outcomes in the HRD population, including BRCA, and then step down to the entire intent-to-treat population.

To wrap up Rubraca and move to lucitanib, I'll described SEASTAR, our Clovis-sponsored Phase Ib/II study that includes multiple single-arm Rubraca combination studies, including the combination of Rubraca with sacituzumab govitecan, now known as Trodelvy for the treatment of advanced metastatic triple-negative breast cancer, relapsed platinum-resistant ovarian cancer and metastatic urothelial cancers. A separate arm of SEASTAR includes the combination of Rubraca with lucitanib in advanced solid tumors, which is currently in the dose-finding Phase Ib portion of the study.

Lucitanib, of course, is our investigational inhibitor of tyrosine kinases, including vascular endothelial growth factor receptors 1 through 3, platelet-derived growth factor receptors alpha and beta and fibroblast growth factor receptors 1 through 3. In February 2019, we and Bristol-Myers Squibb expanded our clinical collaboration to include planned combinations of Opdivo with lucitanib. The Clovis-sponsored LIO-1 study is a Phase Ib/II study evaluating lucitanib in combination with Opdivo. LIO-1 is now enrolling patients with advanced solid tumors in the Phase Ib portion of the study. We anticipate submitting abstracts for presentations at a medical meeting in the fall of 2020.

Lastly, the BMS-sponsored CheckMate 79X study is a Phase I/II study evaluating multiple combinations with Opdivo including an arm in combination with lucitanib in patients with second-line non-small cell lung cancer. Start-up activities for the CheckMate 79X study are proceeding for regulatory guidelines for clinical trial conduct during COVID-19.

We remain very enthusiastic about our peptide-targeted radiopharmaceutical therapy program, and in particular, our lead compound, FAP-2286. FAP is highly expressed in cancer-associated fibroblast or CAFs, which are found in the majority of cancer types, potentially making it a suitable target across a wide array of solid tumors. It is highly expressed in many epithelial cancers, including more than 90% of breast, lung, colorectal and pancreatic carcinomas. Recent preclinical data in animal models, which we expect will be reported at an upcoming medical meeting, has only increased our optimism around this program.

In addition, we and 3BP are collaborating on a discovery program directed at 3 additional targets for radionuclide therapy, to which we have global rights. We've regone to this program for many reasons, including, of course, the opportunity to be a leader in the emerging field of targeted radiotherapy for the treatment of solid tumors. In this case, we have the opportunity to be the first to clinically develop an FAP-targeted radionuclide, and we are also enthusiastic about the targets of the subject of our planned -- or our ongoing discovery collaboration.

Clovis currently plans to submit an investigational new drug or IND application for FAP-2286 in the second half of 2020, followed by a Phase I study to determine the dose and tolerability of the FAP-targeting therapeutic agent with expansion cohorts planned in multiple tumor types as part of the global development program. Thus far, in radiotherapeutic development, physicians have used an imaging agent to identify patients with the appropriate level of tumor target, in our case, FAP. We are exploring opportunities to generate imaging data for FAP-2286, potentially even before our IND is submitted. Not only would this information be useful to gain additional experience with FAP-2286 and better understand the characteristics of FAP expression in multiple tumor types, but further will allow us to collaborate with other academic institutions eager to explore the potential of FAP-2286 as an imaging and as a treatment modality.

And with that, I'll turn the call over to Dan to discuss first quarter 2020 financial results.


Daniel W. Muehl, Clovis Oncology, Inc. - Executive VP & CFO [4]


Thanks, Pat, and hello, everyone. We reported net product revenue for Rubraca of $42.6 million for Q1 2020, which included U.S. net product revenue of $39.3 million and ex-U. S. net product revenue of $3.3 million. This represents a sequential increase of 8% over Q4 2019 net revenue of $39.3 million and a 29% increase over Q1 2019 net product revenue of $33.1 million. U.S. net product revenue was $39.3 million for the first quarter, up 9% from $36.1 million reported in Q4 2019 and up 23% from the $31.9 million reported in Q1 2019. The supply of free drug distributed to eligible patients in the U.S. through the Rubraca Patient Assistance Program for Q1 2020 was 12% of overall commercial supply compared to 18% in Q4 2018. This represented $5.6 million in commercial value for Q1 2020 compared to $8 million in Q4 2019. We can't yet predict the impact of COVID-19 and related unemployment on cap utilization over the remainder of 2020.

Ex-U. S. net product revenue was $3.3 million for the first quarter of 2020, which represents a slight increase over the $3.2 million reported for Q4 2019 and the $1.2 million reported in the first partial quarter of ex-U. S. sales in Q1 2019. We launched Rubraca in France and Spain during March 2020, so we only expected a small contribution in Q1 for those countries. We have now recorded product revenue in each of Germany, United Kingdom, Italy, France and Spain, and we expect to launch into additional smaller European markets over time.

Gross to net adjustments totaled 22.6% in Q1 2020 compared to 17.4% in Q4 2019. The sequential increase in gross to net adjustments reflects primarily an increase in the U.S. contracting and government-related programs and the impact of growing European sales that generally have higher GTN rates. We expect gross to net adjustments to remain in this low 20% range, depending on revenue and distribution mix for the U.S. and Europe. The number of weeks in distributor inventory was flat at the end of Q1 versus Q4, so there was no buildup of inventory as a result -- as a reaction to COVID-19.

At this point in time, we have no issues with either drug supply or distribution of drug to the patient. We have described product supply costs as a meaningful part of our cash spend over the last couple of years as we transition to a new manufacturing facility, so we are in a favorable position for some time to come.

Turning now to a discussion of cash. As of March 31, we had $228.4 million in cash, cash equivalents and available for sale securities. In January 2020, the company repurchased $123.4 million aggregate principal amount of its 4.5% convertible senior notes due 2024 that were initially issued in August 2019. In April 2020, the company exchanged approximately $36 million in aggregate principal amount of its 4.5% convertible senior notes due 2024 in exchange for approximately $32.8 million in aggregate principal of 2021 notes held by such holder. In May 2020, a holder of the 4.5% convertible notes due 2024, converted $24.3 million par value of notes into approximately 3.3 million shares of common stock per the standard terms of the indenture. Following these transactions, approximately $64.4 million aggregate principal amount of these 2021 notes remain outstanding and approximately $150.6 million in aggregate principal amount of these 2024 notes remain outstanding. Additionally, the company has $300 million aggregate principal amount outstanding of its 1.25% convertible notes due 2025.

As a result of the transactions noted above, the company has reduced its total outstanding convertible debt by $145.1 million in outstanding principal amount from December 31, 2019, through May 5, 2020. And as of March 31, we had drawn approximately $50 million under the TPG ATHENA clinical trial financing and had up to $125 million available to draw under the agreement to fund the expenses of the ATHENA trial through Q3 2022.

Based on the company's anticipated revenues, spending, available financing sources and existing cash, cash equivalents and available for sale securities, we believe we have sufficient cash, cash equivalents and available for sale securities to fund our operating plan into the second half of 2021. This does not include any cash repayment that may be required to pay off unless we refinance earlier the remaining $64.4 million aggregate principal amount of the 2.5% convertible notes at their maturity in September 2021. While we did not see an impact in Q1 on our revenues, the effects of COVID-19 on our future sales are difficult to assess or predict, and we may see some near-term impact on revenues related to COVID-19. Net cash used in operating activities was $82.5 million for Q1 2020 compared to $98.5 million for Q1 2019. In addition, borrowings under the TPG ATHENA financing provided $15.6 million in cash in Q1 2020, reducing net cash utilized in operating activities to $66.9 million during the quarter. Net cash used in operating activities for Q1 2020 included product supply costs of $12.4 million and once-a-year annual incentive compensation payment. We expect product supply costs will be significantly reduced from this first quarter level for the remainder of 2020 and at least the first half of 2021. We also expect significantly lower cash burn in the second half of 2020, assuming achievement of our planned revenues over that time frame.

We reported a net loss for Q1 2020 of $99.3 million or $1.39 per share compared to a net loss for the first quarter of 2019 of $86.4 million or $1.63 per share. Net loss for Q1 2020 included share-based compensation expense of $13 million compared to $13.6 million for Q1 2019. Research and development expenses totaled $68.2 million for Q1 2020 compared to $62 million for the first quarter of 2019. The increase is primarily due to higher research and development costs for Rubraca clinical trials. We expect research and development expenses to be lower in the full year 2021 compared to 2020.

Selling, general and administrative expenses totaled $42.6 million for Q1 2020 compared to $47.8 million for the comparable periods in 2019. Selling, general and administrative expenses decreased during the first quarter of 2020, primarily due to decreased commercialization expenses for Rubraca in the U.S. and Europe. We expect savings in selling, general and administrative expenses as a result of the COVID-19 situation globally.

Lastly, we continue to explore ways to improve our balance sheet and capital structure and extend our cash balance beyond the second half of 2021. As noted, we expect our R&D expenses to decrease in 2021 compared to 2020. SG&A expenses should be lower in the upcoming months, and we expect they will be in line with the Q1 2020 levels through 2021. Our inventory purchases and other nonrecurring milestone payment expenses will significantly decrease through 2021, and we anticipate planned revenues to increase with growth in all geographies and with our anticipated prostate indication approval and launch in the U.S. All of these factors should contribute to a reduction in quarterly cash burn into and through 2021. Back to you, Pat.


Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [5]


Thanks, Dan. In summary, we're pleased with our progress in the first quarter, and we believe that Rubraca is well positioned as a maintenance therapy of choice for recurrent ovarian cancer patients in the acute and in the coming chronic COVID-19 environment. Physicians will continue to seek to reduce patient visits to their clinics, and Rubraca offers certain advantages to achieve this goal. Rubraca is an oral agent delivered to and taken at home. Rubraca has been shown to extend progression-free survival by independent assessment by nearly 14 months on average compared to placebo or observation, which has shown PFS of only 5 months on average. And Rubraca requires only monthly routine monitoring, thus limiting patient visits to the clinic. We believe these equalities offer a compelling argument for clinicians to consider Rubraca in the maintenance setting for recurrent ovarian cancer. And soon, we hope to offer a new therapeutic option for BRCA-mutant recurrent, metastatic castrate-resistant prostate cancer patients in the U.S. as well.

We remain focused on managing our net cash utilized operations and improving our balance sheet through convertible debt and other transactions such as the transactions which occurred in January, April and May of this year. And last, but certainly not least, I'd like to acknowledge our employees, all of whom have been working from home since mid-March, and I am grateful for their ongoing commitment to support patients, health care providers and each other during this challenging and unprecedented time.

And with that, we're happy to answer any questions you may have.


Questions and Answers


Operator [1]


(Operator Instructions) Your first question comes from Kennen MacKay with RBC Capital Markets.


Kennen B. MacKay, RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research [2]


Congrats on the operational progress despite the pandemic. Pat, it seems like maybe you really have had some tailwinds from the COVID pandemic going on, obviously, arising from some of the decreased toxicity on the myeloid department. Can you maybe talk about how this could read through to prostate cancer, given some of the alternative agents and the chemotherapies that are out there have maybe even more toxicity than the PARP class.


Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [3]


Yes. As you're aware, both ASCO and FDA have encouraged physicians to consider oral therapeutics as they consider treatment options for patients. And obviously, we hope and believe that, that will continue to accrue to our advantages. I discussed not only in the ovarian cancer setting versus certain alternative infusion based products, but versus immunosuppressive chemotherapy in prostate cancer.


Operator [4]


Your next question comes from Gena Wang with Barclays.


Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [5]


Maybe first one is, any geographic differences in terms of a COVID-19 impact regarding launch? And also, second question is regarding the prostate cancer. Should we actually expect any revenue in second quarter?


Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [6]


Yes. So first, as the geographic differences regarding the launch, yes. We -- it's pretty evident that, for instance, the New York metropolitan area has been hit pretty hard. And I think we probably did see an impact on sales, at least new patient starts in New York, maybe even during the quarter. As to the prostate cancer launch, there will likely be some hotspots, where distractions to the health care system occur and could temporarily impact on prescribing. I will say that with a PDUFA date on May 15 and being prepared to launch on or before May 15, we absolutely would expect to see sales in prostate cancer in the second quarter. We'll have 6 weeks of sales.


Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [7]


Okay. That's very helpful. If I may just squeeze one more question. Any thoughts on ZEJULA approval in the first-line ovarian cancer? And then, how would that impact the competitive landscape and your ATHENA trial readout?


Patrick J. Mahaffy, Clovis Oncology, Inc. - Co-Founder, CEO, President & Executive Director [8]


So one, it was totally expected. And so it came as expected and are approved. It is not going to have any impact on our ATHENA readout. The trial is almost fully enrolled, so it will have no impact on enrollment, obviously. And in fact, we aren't even enrolling in the United States. We've started shutting down country by country, certain areas, and we've already shut down enrollment in the U.S. So it will have no impact on the timing of our readout for ATHENA.


Operator [9]


Your next question comes from Michael Schmidt with Guggenheim Securities.


Yige Guo, Guggenheim Securities, LLC, Research Division - Associate [10]

Read more:
Edited Transcript of CLVS earnings conference call or presentation 5-May-20 8:30pm GMT - Yahoo Finance

Edited Transcript of BMRN earnings conference call or presentation 29-Apr-20 8:15pm GMT – Yahoo Finance

NOVATO Apr 30, 2020 (Thomson StreetEvents) -- Edited Transcript of Biomarin Pharmaceutical Inc earnings conference call or presentation Wednesday, April 29, 2020 at 8:15:00pm GMT

* Brian R. Mueller

BioMarin Pharmaceutical Inc. - Senior VP of Finance, Acting CFO & CAO

* Henry J. Fuchs

BioMarin Pharmaceutical Inc. - President of Worldwide Research & Development

BioMarin Pharmaceutical Inc. - Chairman & CEO

BioMarin Pharmaceutical Inc. - Executive VP & Chief Commercial Officer

* Robert A. Baffi

BioMarin Pharmaceutical Inc. - President of Global Manufacturing & Technical Operations

BioMarin Pharmaceutical Inc. - VP of IR

Evercore ISI Institutional Equities, Research Division - Senior MD & Equity Analyst

* Kennen B. MacKay

RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research

* Peter B. Kim

* Philip M. Nadeau

William Blair & Company L.L.C., Research Division - Co-Group Head of Biopharma Equity Research

Sanford C. Bernstein & Co., LLC., Research Division - VP

Welcome to the BioMarin First Quarter 2020 Financial Results Conference Call. Hosting the conference call today from BioMarin is Traci McCarty, Vice President of Investor Relations. Please go ahead, Traci.

Traci McCarty, BioMarin Pharmaceutical Inc. - VP of IR [2]

Thank you, May, and thank you, everyone, for joining us today. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin, including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development.

Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.

On the call remotely from BioMarin management today are J.J. Bienaim, Chairman and Chief Executive Officer; Jeff Ajer, Executive Vice President, Chief Commercial Officer; Robert Baffi, President, Global Manufacturing and Technical Operations; Hank Fuchs, President, Worldwide Research and Development; and Brian Mueller, acting Chief Financial Officer. We hope to keep this call to 1 hour and also give everyone the opportunity to ask a question today, so we request that you limit yourself to one during the Q&A portion of the call. Thank you for your understanding. I will now turn the call over to our Chairman and CEO, J.J. Bienaim.


Jean-Jacques Bienaim, BioMarin Pharmaceutical Inc. - Chairman & CEO [3]


Thank you, Traci. Good afternoon, and thank you for joining us on today's call. We hope you and your families are healthy and managing through these unusual circumstances brought about by the COVID-19 virus. So these are unprecedented times, but the essential nature of our medicines to the patients who need them has enabled BioMarin to weather the challenge of COVID-19 quite well. Equally as important, I want to underscore the extraordinary dedication of our employees who have kept operations running smoothly in order to maintain access to our therapies around the world.

Our first quarter record results of $502 million of total revenues or 25% growth over last year, the testament to the importance of our therapies and our diversified product base and commercial footprint. Due in part to the sale of Firdapse, GAAP net income in the first quarter was $81.4 million, exceeding our current full year guidance range of $20 million to $80 million.

In the first quarter, we experienced minimal interruptions due to COVID-19, but we do anticipate the potential for more meaningful business disruptions for the remainder of 2020 due to the pandemic. As a result, we have chosen to reduce our full year total revenue guidance by around 5% or a total of $100 million, while maintaining both GAAP and non-GAAP income fully estimated provided earlier this year. Despite potential near-term impacts to our commercial business on COVID-19, our next blockbusters, BMN 270, valoctocogene roxaparvovec, (inaudible) valrox for hemophilia A and vosoritide for achondroplasia continuing to advance, and Jeff will reveal our recently approved brand name for BMN 270 in a moment.

Briefly on vosoritide for achondroplasia. In the quarter, we announced that based on recent meetings, a successful meeting with health authorities in the U.S. and Europe, we plan to submit marketing applications to the FDA and EMA in the third quarter of this year. If approved, vosoritide will be the first medicine for the treatment of achondroplasia in the U.S. and Europe. So we are delighted that this potential therapy proceeds a step closer to regulatory.

In conclusion, BioMarin employees have risen to the evolving challenges of the COVID-19 pandemic, demonstrating a high level of commitment and dedication to the patients we serve. The underlying fundamentals of our business remains strong and our manufacturing and supply chain resilience. We have built a durable base business with essential medicines transition the pipeline to address larger rare indications, diversified risk and positions ourselves for substantial success in both the near term and the long term. We are confident in our ability to manage through this ongoing global health crisis, while staying grounded in our long-term strategy for success.

I'd like to say a few words about Robert Baffi, who has made tremendous contributions to the organization over the last 20 years. During this time at BioMarin, he has manufactured the most complex biological products in the world and visits the most advanced commercial scale gene therapy manufacturing capability. His leadership, technical expertise, foresight and dedication has played a key role in where we stand today, and we want to acknowledge his many contributions. Thank you, Robert. And we are pleased that he will remain with BioMarin through the review of BMN 270 valrox and the vosoritide marketing applications to ensure manufacturing continuity as the successor, Greg Guyer, begins his journey with us in May, coming from Bristol-Myers Squibb.

Thank you all for your continued support. And now I would like to turn the call over to Robert to say a few words. Robert?


Robert A. Baffi, BioMarin Pharmaceutical Inc. - President of Global Manufacturing & Technical Operations [4]


Thank you, J.J. Innovation has always been at the core of BioMarin's success. During my 20 years, as the Head of Technical Operations, and has instill in the company a few guiding principles to foster a culture of innovation: first, let science inform and lead decision-making; second, let compliance focus our efforts on patient safety and clinical outcomes; and third, let ingenuity create adaptivity and resiliency in our approach to drug development. These 3 tenants, infused with the talents of the most dedicated people I've ever worked with, have consistently enabled us to take research ideas rapidly through development, navigating the complexity of the regulatory approval process in a highly effective and differentiated manner to meet the needs of patients.

BioMarin's leadership team has shared and supported the vision for creating a fully integrated company with technical operations, powers clinical studies and commercial demand, and is an integral component of strategic technology development paradigms for assuring the timely delivery of an uninterrupted supply of product.

Furthermore, innovative and appropriately implemented CMC strategies linked to a faster clinical design allows for rapid development and high success rates that benefit both patients and shareholders alike. As a company, we are going through multiple transitions simultaneously, challenging in some ways, invigorating in others. Our transition to profitability this year provides the resources to develop more innovative therapies. Our transition to gene therapy product leverages our clinical manufacturing and commercial capabilities and place us squarely at the forefront of the emerging technological advancement in precision medicine. Our transition and technical operation leadership provides the opportunity to build an innovative approach to drug development to fuel our growth.

When I first saw that to Dr. Greg Guyer's CV, I could not help but be impressed with the scope of his responsibility and the experiential variety and diversity of his career. In many ways, while a different journey, it shared a lot of commonality with my own and that is not to let us vote to BioMarin. I am confident and committed that the transition in technical operations at BioMarin from me to Greg will build on the legacy of science, compliance and ingenuity, for our patients that will benefit from the products that will emerge from our efforts, for our employees and their careers, and for our shareholders as we become profitable.

In terms of licensure of our gene therapy manufacturing facility in support of BMN 270 approval, I'm pleased to share that the Health Products Regulatory Authority of Ireland conducted, on behalf of the European Medicines Agency, a pre-approval inspection in Q1. This inspection involves a detailed review of the facility, equipment, process, and analytical studies and relevant documentations generated in support of validation, production and testing. Following this inspection, a CGMP certification was granted, allowing for commercial production and distribution of BMN 270 in the EU when the product is approved.

At present, the inspection of the facility by FDA is expected to be completed during Q2, allowing full licensure in the U.S. of the facility consistent with the August 21 PDUFA action date. We have more than 400 doses of commercial BMN 270 ready for potential launch later this year, and remain very enthusiastic about the prospects for introducing the first gene therapy product for a bleeding disorder to the hemophilia community as soon as possible.

I thank you for your support throughout my time at BioMarin. And now I'd like to turn the call over to Jeff to discuss the commercial business update. Jeff?


Jeffrey Robert Ajer, BioMarin Pharmaceutical Inc. - Executive VP & Chief Commercial Officer [5]


Thank you, Robert. As we begin 2020, I'm very pleased with the team's performance across all brands and all regions during the quarter. As J.J. mentioned, we achieved our highest quarterly revenue on record, with total revenues of $502 million in the first quarter with net product revenues marketed by BioMarin, up 24% to $433 million. This achievement reflects the fundamental strength and growth of our business despite near-term challenges related to COVID-19, which I will address in a moment.

On to results in the quarter and starting with Palynziq. In the U.S., the trend of increasing revenue based on a steadily growing base of patients on commercial therapy, including progression from induction and titration to daily maintenance dosing, continued in Q1. In the early part of the quarter, we did experience a seasonal slowing of new patient enrollments and patient starts, somewhat mirroring our historical experience with Kuvan in the United States. We are reporting $35 million in Palynziq revenue for the first quarter, with the majority of that revenue coming from the U.S.

In Europe, in the first quarter, multiple clinics across Germany continued to actively treat patients with Palynziq and early uptake signals are encouraging. During the quarter, we made significant progress in Germany, adding clinics that now have some experience prescribing Palynziq and managing patients through the induction and titration phase to daily maintenance dosing. As the number of commercial patients in Germany steadily grows, we anticipate meaningful revenue contribution from the EU starting this year. We anticipate finalizing price and reimbursement negotiations in Germany by mid this year, an important step toward getting price and reimbursement approvals in other high priority European markets.

Kuvan contributed $122 million in revenues in the quarter or 14% growth year-over-year, with most of that growth coming from the United States. Vimizim revenues grew 9% year-over-year, contributing $137 million in the first quarter, driven by an 11% increase in patients year-over-year. This is reflective of the continued anticipated growth potential we expect for Vimizim.

Turning to Naglazyme. Revenues totaled $114 million, a 32% year-over-year growth for the well-established brand. As with Vimizim, the impact from uneven large order patterns makes the quarterly comparison difficult. The number of commercial patients on Naglazyme grew by 6% in the past year, and is indicative of the ongoing growth potential for this brand, nearly 15 years since being approved.

And finally, Brineura contributed $24 million in net product revenues, which represented 97% year-over-year growth. These revenues were essentially flat over Q4, and that was driven by a modest year-end inventory build in the EMEA region in Q4. Importantly, the growth in Brineura revenues compared to prior year reflects an underlying growth of 86% in commercial patients. We are seeing a net increase in patients benefiting from Brineura treatment due to the success of our disease awareness and patient identification programs.

Taken together, we are pleased with first quarter results and demand for our products. And while we experienced minimal financial impact in the first quarter due to COVID-19, we anticipate the potential for a higher degree of impact during the remainder of 2020 as disruptions of day-to-day operations of clinics and hospitals flow through our business.

Our global commercial teams will continue to adjust to implement innovative approaches to engage with clinics and patients to ensure continuity of access to our medicines. Where possible, we are supporting home infusion efforts to help mitigate impact. However, some COVID-19 disruption, the new patient starts as well as to ongoing infusion center visits from existing patients are expected to continue. As a result, we are reducing total revenue guidance by 5% at the midpoint to between $1,850 million to $1,950 million for the full year 2020. The vast majority of today's updated total revenue guidance reflects adjustments to in line brands, including Vimizim, Naglazyme and Palynziq. And assumes our business will return to normalized demand patterns in the second half of 2020. Although we did not give BMN 270 2020 revenue guidance in February, our 2020 total revenue guidance did assume some contribution from BMN 270 in Europe.

And now I'd like to end my remarks with an update on our hemophilia gene therapy program and introduce you to the intended brand name, ROCTAVIAN. In previous calls, you've heard references to valrox, which was an abbreviated form of our INN or international nonproprietary name, valoctocogene roxaparvovec or alternatively, our program identifier, BMN 270. Both the FDA and the EMA have accepted ROCTAVIAN as our brand name, and we look forward to adopting ROCTAVIAN as we get closer to launch. In the meantime, we will cease to use valrox, so as to not confuse it with our intended brand name.

Other key launch readiness activities have continued to progress. We have essentially built out the commercial team in the United States, and have added key individuals to support priority in markets in the EU. The majority of these new employees have substantial and diverse experience in hemophilia. Our brand campaigns also continue to develop as anticipated and teams have pivoted to virtual and digital platforms, allowing for ongoing engagement with the marketplace in lieu of face-to-face interactions. Obviously, certain activities are more amenable to virtual engagements than others, and where COVID-19 is challenging us most in the short-term is with gene therapy educational programs and site readiness.

Fortunately, with the team already on board, we anticipate being well prepared to launch if we receive regulatory approvals. In the meantime, we have recently finished some very positive pricing research, which validated payer willingness to embrace ROCTAVIAN with the current data set, and we'll look forward to providing you with updates on our pricing at launch.

Thank you for your attention, and I will now turn the call over to Hank to provide an R&D update. Hank?


Henry J. Fuchs, BioMarin Pharmaceutical Inc. - President of Worldwide Research & Development [6]


Thanks, Jeff. I'd also like to echo J.J.'s expression of deep and heartfelt appreciation for the time Robert has spent with us. And also to welcome Greg Guyer, to the organization where the secret sauce is bottled.

The R&D organization is delighted that our next significant product opportunities continue to progress, particularly under the circumstances brought about by COVID-19. The ability to come to work mostly virtually and focus on the advancement of our innovative products to date, ROCTAVIAN and vosoritide has been especially gratifying and a welcome distraction from the ongoing pandemic. I want to acknowledge and thank our teams for their commitment and contributions during these challenging times. I have been impressed by your flexibility and your ability to keep the story going while we are dealing with the pandemic.

Starting with ROCTAVIAN, and with a strong and memorable brand name, congratulations, Jeff, the FDA is committed to meet the August 21 PDUFA action date. In Europe, our marketing authorization application filing remains on accelerated assessment at this time. However, the review procedure is to be extended by at least 3 months due to COVID-19 delays. Further, as is the case with most filings that initially receive accelerated assessment, we believe there is a high possibility that our M&A will revert to a standard review procedure from accelerated assessment. Based on these assumptions, we expect the CHMP opinion by late '20 or early '21.

We continue to plan to share our 3 -- our 4-year update of the 16, 13 vector genome per kilo dose as well as the 3-year update on the 40, 13 vector genome per kilo dose in the middle of the year, but the form is as yet to be defined given the changing environment for medical meetings. We have a data analysis plan in place. We'll move forward with business as usual, but the venue and method for providing the update is still fluid at this point. So thanks for bearing with us.

Importantly, we do not expect COVID-19 to impact the time lines for completion of the ROCTAVIAN Phase III trial. Enrollment was completed in November of last year, and one of the benefits of being a onetime treatment is that patients do not need to receive therapy on a chronic basis. We're also confident that the integrity of the ongoing data collection for the study -- for this pivotal study is being sufficiently maintained as home health care solutions align nicely with the collection of the primary endpoint annualized bleed rate data.

Turning now to vosoritide for the treatment of achondroplasia. As J.J. mentioned, we plan to submit a global marketing applications in the third quarter of this year. Our multipronged development program, including a long-term Phase II clinical results in 5 to 18-year-old children, comprehensive natural history data, the ongoing study of newborns through 5 years and the highly statistically significant placebo-controlled Phase III trial makes for a very comprehensive data package spanning more than 5 years of treatment with children with achondroplasia. Again, we're the beneficiary of fortunate timing and that our pivotal submission data read out prior to the pandemic, and now much of the work can be concluded remotely. If approved, vosoritide would be the first and only medicine designated for the treatment of achondroplasia in the U.S. and in European Union.

We continue to look forward to publishing the full data from the Phase III study later this year, and we're pleased to let you know that our late-breaker has been accepted in an upcoming medical Congress. The presentation will include 1 year growth velocity, height Z scores, body proportionality, safety and subgroup analyses. So stay tuned for more specifics as to when and where those data will appear.

The Phase II study of vosoritide in 0 to 5 year olds, referred to as study 206, is proceeding well, and we are very pleased that safety data from children ages 6 months to 5 years participating in that study will be available as part of our registration package. We're grateful that the timing of key studies has aligned well with our pre-COVID-19 plans.

Moving to BMN 307, our investigational gene therapy for phenylketonuria. We're continuing to prepare new sites to open in order to enroll patients when it is safely do so given the COVID-19 circumstances. We're excited about the prospect of BMN 307 as it represents a third treatment for phenylketonuria in our PKU franchise and a second gene therapy development program, leveraging our learnings and capabilities from ROCTAVIAN. Currently, we expect the study to start later -- we expect to start the study later in 2020.

The R&D organization is energized by the opportunities before us in 2020, with both ROCTAVIAN for severe hemophilia A and vosoritide for children with achondroplasia advancing towards potential approvals. We are hopeful that these innovative treatments will be available in the very near future. We look forward to updating you on our progress over the coming quarters, and thank you for your continued support.

And I'll now turn the call over to Brian to review the financials.


Brian R. Mueller, BioMarin Pharmaceutical Inc. - Senior VP of Finance, Acting CFO & CAO [7]


Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the first quarter of 2020, and as usual, our comprehensive report on the quarter will be available in our upcoming Form 10-Q, which we are on track to file over the next couple of days.

As Jeff mentioned, we are experiencing some modest impacts from the COVID-19 pandemic and as a result, we have updated full year total revenue guidance to between $1.85 billion to $1.95 billion. As Jeff noted,our updated revenue guidance is based on the assumption that our business will return to normalized demand patterns in the second half of the year.

Importantly, while we lowered our revenue guidance due to the impact of COVID-19 on our commercial business, we were able to analyze our 2020 spending projections and make adjustment that allowed us to maintain our prior GAAP and non-GAAP income guidance despite the lower revenue.

Moving to operating expenses. R&D expense for the first quarter of 2020 was $142 million and lower compared to R&D expense for the first quarter of 2019 of $184 million, mostly due to less R&D activity for ROCTAVIAN, given its late stage of development as well as Palynziq following its approvals in the U.S. and Europe.

SG&A expense for the first quarter of '20 was $187 million, which was higher than SG&A expense for the first quarter of 2019 of $162 million. The year-over-year increase was expected with the single largest driver, being the commercial preparation for the launch of ROCTAVIAN and the continued global launch of Palynziq. We also incurred some unpredicted foreign currency exchange losses during the month of March as the COVID pandemic negatively affected some of our assets denominated and some of the more volatile global currencies.

During the bottom line results, we reported GAAP net income of $81 million in the first quarter of 2020, compared to a GAAP net loss of $56.5 million in the first quarter of 2019. The improvement in GAAP income was primarily due to higher revenue, lower R&D expenses and the gain on the sale of the deferred tax assets. With higher revenues and lower R&D expenses, non-GAAP income of $117 million in the first quarter of 2020 grew substantially as compared to Q1 2019 non-GAAP income of $25 million. Both of these first quarter 2020 bottom line results gives us a great start towards achieving our 2020 goals of GAAP net income on an annual basis for the first time in the company's history, a considerable growth in non-GAAP income.

I'd also like to touch on the potential tax benefit that we mentioned last quarter that may be recognized in the second half of this year. Our current 2020 GAAP net income guidance of between $20 million to $80 million, excludes the potential impact of intra-entity intangible asset transfers between BioMarin entities. If these intangible asset transfers occur, we estimate that the tax effect could result in a onetime noncash income tax benefit of greater than $500 million. As I mentioned previously, you may have seen similar transactions completed by some of our larger peers in recent quarters.

Speaking to total cash and investments, we ended the first quarter of 2020 with $1.15 billion compared to $1.17 billion at the end of December 2019. The modest decrease in total cash and investments during Q1 2020 was largely due to some timing of operating cash flows. However, the significant improvement over the first quarter of 2019 where total cash and investments decreased by $105 million. This valid cash position, coupled with vibrant business fundamentals, put us in good standing to manage through the continued uncertainty related to COVID-19.

In closing, the stronger performance of the business during the first quarter of 2020, plus our positive financial outlook for the rest of the year, indicate that 2020 should be a transformational year for the company. And the prospects of value to come from ROCTAVIAN and vosoritide, if they are approved commercially, give us an enthusiasm about our future.

Thank you for your support, and we will now open the call to your questions. Operator?


Questions and Answers


Operator [1]


(Operator Instructions) Our first question is from the line of Robyn Karnauskas from Suntrust.


Robyn Kay Shelton Karnauskas, SunTrust Robinson Humphrey, Inc., Research Division - Research Analyst [2]


And just first off, congratulations to Greg. But to Rob, it was lovely working with you. I think you brought a breath of fresh air to working with management teams in all of my buyers (inaudible). So thank you so much. I learned a lot. I guess I'm going to start-up with some questions on ROCTAVIAN, and I hope I get that correctly. So first of all, what gives you confidence that in the United States, that there won't be any more delays? People ask me this nonstop. And then when you talk about assuming normal operations go -- resume in the second half, is that in the beginning of the second half? Do you have a time line for that? If it goes into fourth quarter, could we see further delays?

And the third question is, what are you hearing as far as like people willing to have gene therapy procedures done in the COVID environment as early as fourth quarter? Are people open to it? Is it separate from the hospitals? Or what are you hearing from the ground?


Jean-Jacques Bienaim, BioMarin Pharmaceutical Inc. - Chairman & CEO [3]


Okay. Hi. (inaudible) do you want to answer the question on the no delays with the FDA? Hank?


View original post here:
Edited Transcript of BMRN earnings conference call or presentation 29-Apr-20 8:15pm GMT - Yahoo Finance

Immunotherapy Before Surgery Could Advance Care of an Aggressive Form of Skin Cancer – Newswise

Newswise In what is believed to be a first-of-its-kind study to evaluate the safety of a type of immunotherapy before surgery in patients with an aggressive form of skin cancer, researchers report that the treatment eliminated pathologic evidence of cancer in nearly half of the study participants undergoing surgery. In patients whose tumors respond, this treatment approach offers the potential to reduce the extent of surgery and may also slow or eliminate tumor relapses that often occur after surgery.

A report on the Merkel cell cancer trial, directed by investigators at the Johns Hopkins Bloomberg~Kimmel Institute for Cancer Immunotherapy and the Johns Hopkins Kimmel Cancer Center in collaboration with the University of Washington Seattle Cancer Care Alliance and 10 other medical centers across the U.S. and Europe, will be published April 23 in the Journal of Clinical Oncology. An additional review article by Bloomberg~Kimmel Institute experts, published Jan. 31 in the journal Science, also points to the potential strategic value of giving immune checkpoint inhibitor therapy earlier in the course of cancer treatment.

Merkel cell carcinoma is classified as an orphan disease by the National Cancer Institute, diagnosed in approximately 2,000 people annually in the U.S. It typically appears as a red, blue or flesh-colored lump on the skin in older people and those who have suppressed immune systems. About 80% of Merkel cell carcinomas are caused by a virus called the Merkel cell polyomavirus. The remaining cases are linked to sun or other ultraviolet light exposure and unknown factors. Merkel cell carcinoma can spread to the lymphatic system and other organs. Surgery, radiation therapy and chemotherapy have been the mainstays of treatment. However, for Merkel cell carcinomas advancing beyond the point of surgery, there were previously no treatment options that could prolong survival. Recently, drugs blocking the immune checkpoints PD-1 and PD-L1 were shown to be effective in advanced inoperable Merkel cell carcinomas, and were approved by the U.S. Food and Drug Administration in this treatment setting.

Merkel cell carcinoma appears to respond very rapidly to anti-PD-1 immunotherapy in some patients with advanced disease, says lead study author Suzanne Topalian, M.D., associate director of the Bloomberg~Kimmel Institute for Cancer Immunotherapy. This led us to test whether anti-PD-1 could be effective if given for a brief period before surgery, as so-called neoadjuvant therapy. Using this approach, we found that patients who had substantial tumor regressions on CT scans or in pathology studies of surgically removed tumor specimens had extended cancer recurrence-free survival that was statistically significant. Radiographic and pathologic tumor regressions following neoadjuvant anti-PD-1 therapy are therefore potential new, early markers that will help us predict what a patients long-term outcome will be. This is critical information for oncologists planning treatment strategies for their patients.

Nivolumab, the immunotherapy drug used in this study, works against cancers including Merkel cell carcinoma by blocking PD-1, a molecule on the surface of immune cells that suppresses immune responses. Cancer cells often manipulate PD-1 by expressing its partner molecule PD-L1, sending a stop signal to the immune system. Blocking that signal with a checkpoint inhibitor such as nivolumab initiates a go signal, unleashing immune cells to attack cancer cells.

In the phase I/II trial of nivolumab in virus-associated cancers called CheckMate 358, patients with operable Merkel cell cancers received 240 mg of the anti-PD-1 drug intravenously on days one and 15 of the study, with surgery planned for day 29.

The trial was designed primarily to assess the safety and tolerability of nivolumab in this treatment setting. Investigators also assessed tumor regression using CT and MRI scans; studied the presence of cancer cells microscopically in surgically removed tumors; and studied pretreatment tumor biopsies to measure the presence of the polyomavirus causing Merkel cell carcinoma, the mutational burden (the quantity of gene mutations found in a tumor) and expression of the PD-L1 protein.

Overall, 39 patients with stage IIA-IV Merkel cell cancer (locally advanced or having spread to lymph nodes or internal organs) received at least one dose of nivolumab between January 2016 and March 2019. Among 36 patients who underwent surgery, 17 (47%) achieved a pathologic complete response, meaning that there were no live tumor cells anywhere in the surgical tissue. Among 33 patients undergoing surgery who also had imaging scans, 18 (54.5%) had radiographic tumor reductions of at least 30%. Each of these findings correlated significantly with prolonged recurrence-free survival. Patients were followed for a median of 20 months.

These rates of pathologic and radiographic tumor regression after a brief four-week period of nivolumab therapy are very high compared to other cancer types in which anti-PD-1 treatment has been tried before surgery, Topalian says. For instance, in lung cancer, the published rate of complete pathologic response after neoadjuvant anti-PD-1 monotherapy is 15%, and in melanoma it is 19-25%. Furthermore, substantial radiographic tumor regression is not common within such a brief treatment period in other cancer types.

Overall, among 36 patients who underwent surgery, recurrence-free survival rates were 77.5% at 12 months and 68.5% at 24 months after surgery. However, those with a complete pathologic response had a recurrence-free survival of 100% at 12 months and 88.9% at 24 months, compared with those without a complete pathologic response, who had recurrence-free survivals of 59.6% and 52.2% at 12 and 24 months, respectively. Similarly, patients with substantial radiographic tumor regressions before surgery experienced prolonged recurrence-free survival, compared with the other patients in the study. These effects of neoadjuvant nivolumab on recurrence-free survival in some patients appear to offer an advantage compared to historical reports of conventional care.

Three of 39 patients (7.7%) did not undergo surgery, one because of tumor progression, and two because of adverse effects from nivolumab. Treatment-related adverse events occurred in 18 of 39 patients (46.2%) and most commonly included skin rashes. Three patients (7.7%) had a severe adverse event, including immune-related colitis. The characteristics of adverse events were similar to those previously reported for anti-PD-1 drugs in patients with other cancer types.

Topalian cautions that the study was a relatively small one, and did not have a control group for comparison. All patients received the same treatment. However, she says, We think these findings provide a rationale to conduct larger trials of neoadjuvant anti-PD-1 therapy in Merkel cell carcinoma, and have the potential to be practice-changing.

To our knowledge, this is the first attempt to look at the role of anti-PD-1 therapy before surgery in patients with Merkel cell carcinoma who are candidates for complete surgical removal of their tumor, Topalian says. We know that, historically, many of these patients would subsequently relapse after standard surgical and postoperative treatments. Even if we think were removing all of the existing tumor at the time of surgery, in many patients the tumor has already spread to other parts of the body, at microscopic sites of metastasis that are too tiny to be detected with scans.

Investigators from the Bloomberg~Kimmel Institute co-led this study with researchers from the University of Washington Seattle Cancer Care Alliance, in collaboration with 10 other medical centers in the U.S. and Europe. The Bloomberg~Kimmel Institute team included Topalian; William Sharfman, M.D.; Julie Stein, M.D.; Elizabeth Engle, M.S.; and Janis Taube, M.D., M.Sc.

Separately, in a review paper published in Science by Topalian and Johns Hopkins colleagues Taube and Drew M. Pardoll, M.D., Ph.D., director of the Bloomberg~Kimmel Institute for Cancer Immunotherapy, the researchers summarized scientific and medical knowledge about the use of immune checkpoint blockers before cancer surgery. This includes the first published report of neoadjuvant PD-1 pathway blocking antibodies, tested in non-small cell lung cancer at Johns Hopkins and described in the New England Journal of Medicine in 2018, and additional studies of immune checkpoint blockers in melanoma, bladder cancer and brain cancer from other research groups. There are many more neoadjuvant anti-PD-1 studies maturing now in other cancer types including breast and head and neck cancers, Topalian says.

When people talk about preventing cancer, they generally mean preventing cancer from forming, she says. However, these neoadjuvant immunotherapy studies speak to the possibility of preventing early-stage cancers from becoming end-stage. We think this is a very valuable approach. By using immune checkpoint blockers before definitive surgery for cancer, it may be possible in some patients to prevent to the disease from progressing to an inoperable stage. We look forward to a lot more information to come in this area.

Preliminary results of the Merkel cell carcinoma study were presented at the American Society of Clinical Oncology meeting in 2018. Other centers participating in that trial were the Levine Cancer Institute, Atrium Health, of Charlotte, N.C.; Winship Cancer Institute of Emory University, Atlanta; Universit de Paris, Saint Louis Hospital, Paris; Institut Claudius Regaud, Toulouse, France; Memorial Sloan Kettering Cancer Center, New York; H. Lee Moffitt Cancer Center and Research Institute, Tampa, Fla.; SLK-Clinics, MOLIT Institute, Heilbronn, Germany; University of Pittsburgh Medical Center Hillman Cancer Center; University Medical Center Utrecht, Cancer Center, the Netherlands; and the University of Michigan Comprehensive Cancer Center, Ann Arbor, Mich. Bristol Myers Squibb, which sponsored the trial, also had co-authors.

The work was supported by Bristol Myers Squibb and ONO Pharmaceutical Company Limited. Some of the scientific correlative work conducted at Johns Hopkins was supported by The Mark Foundation for Cancer Research and National Cancer Institute R01 grant CA142779. Authors received no financial support or compensation for publication of the study.

The Science review was supported by the Johns Hopkins Bloomberg~Kimmel Institute for Cancer Immunotherapy, the National Cancer Institute (R01 CA142779), the Cancer Research Institute/Stand Up To Cancer-Immunology Translational Cancer Research Grant, Bristol Myers Squibb, the Barney Family Foundation, Moving for Melanoma of Delaware, the Laverna Hahn Charitable Trust, the Melanoma Research Alliance, the Harry J. Lloyd Charitable Trust, the Emerson Collective Foundation, and the Mark Foundation for Cancer Research.

Topalian reported stock and other ownership interests for herself or an immediate family member from Aduro Biotech, DNAtrix, Dragonfly Therapeutics, Ervaxx, Five Prime Therapeutics, RAPT Therapeutics, Potenza Therapeutics, Tizona Therapeutics, Trieza Therapeutics and WindMIL; consulting or advisory roles with Amgen, Compugen, DNAtrix, Dragonfly Therapeutics, Dynavax, Ervaxx, Five Prime Therapeutics, RAPT Therapeutucs, Immunocore, Immunomic Therapeutics, Janssen Oncology, MedImmune, Merck, Tizona Therapeutics and WindMIL; research funding from Bristol Myers Squibb, Compugen and Potenza Therapeutics; travel, accommodations and expenses from Bristol Myers Squibb, Dragonfly Therapeutics, Five Prime Therapeutics and Merck; and patent royalties from Aduro Biotech, Arbor Pharmaceuticals, Bristol Myers Squibb, Immunomic Therapeutics, NexImmune, and WindMIL. These relationships are being managed by The Johns Hopkins University in accordance with its conflict of interest policies.

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Immunotherapy Before Surgery Could Advance Care of an Aggressive Form of Skin Cancer - Newswise

Study provides new insight into the neural circuitry underlying irritability and anxiety in youths – PsyPost

New neuroimaging research published in JAMA Psychiatry helps to untangle the links between irritability, anxiety, and brain functioning in youths. The findings suggest that irritability and anxiety have interactive, rather than additive, effects when processing negative social information.

I am a practicing child and adolescent psychiatrist. Over the years, I have met with families struggling with all types of anxiety and irritability, explained study author Joel Stoddard of the University of Colorado and Childrens Hospital Colorado.

When I was early in my career, I began to appreciate that even though anxiety and irritability seem like straightforward symptoms, their presentation is often intermingled. We simply dont know why they co-occur or conversely why a child might have one symptom but not the other.

Moreover, there was very little work looking at both symptoms together, especially without a focus on traditional mental health diagnoses. At the time this study started, mental health research was moving away from traditional diagnoses because most arent well grounded in biology. Though the transition has been difficult, it also gave clinical scientists room to restart simply, said Stoddard, who also leads the Emotion and Development Lab.

In this study we examined symptoms in a group of children diagnosed with clinically significant affective issues. Are these symptoms related to how the basic emotion recognition circuitry responds to facial expressions?

The researchers used functional magnetic resonance imaging to examine the brain activity of 115 youths aged 8 to 17 years as they completed a face emotion processing task. About 90 of the participants had been diagnosed with anxiety, disruptive mood dysregulation, and/or attention-deficit/hyperactivity disorders. The youths and their parents also completed assessments of the participants irritability and anxiety.

Stoddard and his colleagues found that high levels of both anxiety and irritability were associated with decreased connectivity between the amygdala and medial prefrontal cortex when viewing angry facial expressions. High levels of anxiety but low levels of irritability, on the other hand, were associated with increased connectivity in this brain circuit. The amygdala is known to be important for responses to threatening situations, while the medial prefrontal cortex is associated with decision-making processes.

This is a demonstration that the response of amygdala-centered brain networks to emotional expressions differs by anxiety and irritability in children who we might very well see in the clinic, Stoddard told PsyPost.

Had we looked at either symptom alone, we might have missed how they respond differently. For example, we observed that changes in the prefrontal-amygdala network during viewing angry faces differ by an individuals degree of anxiety or irritability. These differences offer important clues about the neural function youth who may express anxiety, irritability, or both. But these are clues, I dont think much more should be taken from this study by itself.

Increased irritability by itself, meanwhile, was associated with increased activity in several brain regions in response to angry faces and happy faces compared to fearful faces.

But as with all research, the study includes some limitations.

This study used functional magnetic imaging in children with serious emotional issues, these facts are the source of strengths and weaknesses. Clearly major strengths are that we examined the clinical population directly and used a noninvasive, largely safe method of measuring localized brain activity. However, it is very difficult to get a good brain signal from youngsters because they move, causing a blurry image (especially for network imaging), or get tired of the task (so their brain responses may change), Stoddard explained.

Also, we could not ethically ask the participants to stop their medications or pause therapy risking harm. Also, if we did so, we might be studying the effects of discontinuation. On the other hand, if we only invited kids who didnt need treatment, we might not study a group of youth who represent the conditions we see in clinics. We also didnt focus on anxiety and irritability in some conditions like bipolar disorder or active posttraumatic stress or in different cultural contexts. Though there are caveats with each aspect of the study, it represents an interesting result and considerable effort by participants and experts.

This type of work is critical to build a strong foundation for understanding the brain mechanisms. It is part of a growing body of literature that is revealing how brain networks function differently by development, experience, or clinical symptoms. I personally find this emerging literature satisfying, because I see complicated clinical pictures. Some stories are simpler, such as dysfunction in a single gene or brain region. These occasionally explain a lot for rare conditions, but rarely much for common conditions, Stoddard added.

This work was a team effort. I contributed to it as part of my research training fellowship at the National Institutes of Mental Health and as an assistant professor at the University of Colorado, School of Medicine, Department of Psychiatry, Division of Child and Adolescent Psychiatry. I continue to have that latter title and am an outpatient psychiatrist at the Childrens Hospital Colorado. Im supported by all those organizations to study pediatric anxiety and irritability. The content is solely my responsibility and does not necessarily represent the official views of the National Institutes of Health, the University of Colorado, or Childrens Hospital Colorado.

The study, Association of Irritability and Anxiety With the Neural Mechanisms of Implicit Face Emotion Processing in Youths With Psychopathology, was authored by Joel Stoddard, Wan-Ling Tseng, Pilyoung Kim, Gang Chen, Jennifer Yi, Laura Donahue, Melissa A. Brotman, Kenneth E. Towbin, Daniel S. Pine, and Ellen Leibenluft.

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Study provides new insight into the neural circuitry underlying irritability and anxiety in youths - PsyPost

Cell Harvesting Market Industry Trends and Forecast to 2025 | PerkinElmer Inc, Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical – Bandera County…

Global Cell Harvesting Market,By Type (Manual Cell Harvesters and Automated Cell Harvesters), By Application (Biopharmaceutical Application, Stem Cell Research and other Applications), By End Users (Hospitals, Ambulatory Centers, Clinics, Community Healthcare, Others), By Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2025

Market Analysis:Global Cell Harvesting Market

The Global Cell Harvesting Market is expected to reach USD 387.9 Million by 2025, from USD 196.9 Million in 2017 growing at a CAGR of 8.9% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2017, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

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Market Definition:Global Cell Harvesting Market

This market report defines the market trends and forecast the upcoming opportunities and threats of the cell harvesting market in the next 8 years. Cell harvesting is extracting the cells either from bone marrow and peripheral blood cells and culturing the cells in the culture dish containing nutrient media. Cell harvesting is used in the cell therapy as well as in gene therapy. University of California developed a cure for bubble baby disease for new born babies by using the cell harvesting in stem cells and gene therapy. Moreover, Asterias developed the stem cell therapy to regain the upper body motor function. University of California, Irvine developed the stem cell therapy to destroy the breast cancer cells.. Now a days cell harvesting is also used in the animal research and development. Cell Harvesting is also used in may research labs for in-Vitro testing. In September 2016, Terumo BCT collaborated with Cognate Bioservices for developing the immunotherapies and other related products like cell therapy products. These innovations in the cell harvesting market is notably attributing towards its increasing demand at the global pace. Further, its demand is likely to gain momentum over the forecast period.

Major Market Drivers and Restraints:

Market Segmentation:Global Cell Harvesting Market

Competitive Analysis:Global Cell Harvesting Market

The global cell harvesting market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cell harvesting market for global, Europe, North America, Asia Pacific and South America.

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Major Market competitors/players:Global Cell Harvesting Market

Some of the major players operating in the global cell harvesting market are PerkinElmer Inc, Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical, Argos Technologies, SP Industries, Teleflex Incorporated, Arthrex, Inc, Thomas Scientific, Brand GMBH, Brandel, Cox Scientific, Connectorate, Scinomix, Adstec.

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Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analyzed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more pleaseRequest an Analyst Callor can drop down your inquiry.

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Cell Harvesting Market Industry Trends and Forecast to 2025 | PerkinElmer Inc, Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical - Bandera County...

Muscular Dystrophy Association Announces Formation of Strategic Medical Advisory Team of Experts in Neuromuscular Care and Research – BioSpace

NEW YORK, April 2, 2020 /PRNewswire/ --TheMuscular Dystrophy Association(MDA) announced today the formation of its formal Medical Advisory Team who provide MDA with strategic guidance on issues that impact research and clinical care for people living with muscular dystrophy, amyotrophic lateral sclerosis (ALS) and related neuromuscular disorders more than 43 diseases. Each of the team members is a leading clinician at an MDA Care Center and is an internationally known expert in muscular dystrophy, ALS and related neuromuscular diseases. The MDA Care Center network spans over 150 locations at the nation's top medical institutions.

"We believe that engaging this team of world-class experts across neuromuscular diseases will enable MDA to lead the way in speeding the development of groundbreaking therapies and treatment paradigms that will transform the lives of those we serve," says Lynn O'Connor Vos, President and CEO of MDA. "These physicians are pillars of the neuromuscular disease community, each with focused expertise in specific diseases. Together, they will provide critical advice to MDA on our innovations in science and care programs and help ensure that we are making the maximum impact as the landscape of neuromuscular research, care and treatments evolve."

MDA's medical advisors will help lead the effort, along with more than 2,000 health care providers, to establish new clinical trials geared toward integration of MDA's innovative MOVR technology hub, which allows clinicians and researchers to share a wealth of disease-related information, establish professional protocols, and accelerate innovation.

Tonight, Dr. Barry Byrne, who will serve as chief medical advisor for the team, is joining MDA's EVP, Chief Advocacy & Care Services Officer, Kristin Stephenson for an MDA Facebook Live conversation about the precautions and best practices needed to protect the neuromuscular community in light of COVID-19. The event, scheduled for Thursday, April 2 from 6:00 to 6:30 pm EDT is accessible at The recording will be available for viewing after the event on MDA's COVID-19 resource page. The conversation will feature a live Q&A answering questions from people living with muscular dystrophy, ALS and related neuromuscular diseases including MDA families, and aims to provide the answers to questions regarding care duringthese uncertain times, and will cover topics related to preparedness, community impact, telemedicine and MDA's Care Center network of over 150 multidisciplinary care teams at top medical institutions nationwide.

Barry J. Byrne, MD, PhD, is an ardent supporter of newborn screening and has been an innovator and early adopter of new FDA-approved therapies that have the potential to alter the course of some neuromuscular disorders. He was the first physician to administer an approved gene therapy to a neuromuscular patient in the U.S.

"We are just beginning to realize the impact of the current revolution in the treatment of neuromuscular disorders. Newborn screening and access to newly-approved therapies are changing patients' lives when diagnosed with neuromuscular diseases; the MDA Care Center network is ready to provide these innovative treatments," says Dr. Byrne. "Disease-modifying therapies are no longer a thing of the future. There is an urgent need to bring innovative care, cutting-edge clinical research and new breakthrough treatments to the community."

Dr. Byrne will continue in his role as the associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida College of Medicine as he assumes this new position. He is also director of the MDA Care Center at the University of Florida.

Matthew B Harms, MD, will serve as a medical expert on ALS and other neuromuscular disorders. He is active in clinical research. Dr. Harms is associate professor of neurology at Columbia University's Vagelos College of Physicians and Surgeonsand serves at MDA's ALS Care Center at Columbia University. Dr. Harms received MDA's Diamond Award for his work directing an international effort with whole genome and transcriptome sequencing to bring precision medicine to ALS treatments.

John W. Day, MD, PhD, completes the Medical Advisory Team. Dr. Day is professor of neurology and pediatrics and director, Division of Neuromuscular Medicine at Stanford University. He directs Stanford's MDA Care Centers, which uniquely integrate the Lucile Packard Children's Hospital Pediatric and Transitional Neuromuscular Clinic with the Stanford Hospital Neuroscience Health Center's Neuromuscular and ALS Research and Clinic.The comprehensive team of investigators and clinicians in the Stanford Neuromuscular Program have helped develop novel gene modification and gene replacement treatments for spinal muscular atrophy, muscular dystrophy and ALS, and have spearheaded development of centralized data hubs like MDA's MOVR for neuromuscular disease.

About Barry J. Byrne, MD, PhD

Dr. Byrne is a clinician scientist who is studying a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function. His group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disorder caused by an excess of a type of sugar (glycogen) in certain muscles. The research team has pioneered the use of adeno-associated virus (AAV) mediated gene therapy to restore heart and skeletal muscle function in Duchenne muscular dystrophy, Pompe, Friedrich's ataxia and other neuromuscular diseases. His group at the Powell Center has also established a series of new methods for large-scale AAV manufacturing to enable access for a wide variety of conditions.

About Matthew B. Harms, MD

Dr. Harms' post-doctoral and faculty work in neurogenetics led to the discovery of genes for dominant spinal muscular atrophy and limb-girdle muscular dystrophy type 1D. He sees patients in the Eleanor and Lou Gehrig ALS Center, the Adult Muscular Dystrophy Association Clinic, the Pediatric Muscular Dystrophy Association Clinic, and until recently, the ALS Clinic of the Bronx VA Hospital. His laboratory straddles Columbia's Motor Neuron Center and the Institute for Genomic Medicine, with a focus on generating, integrating, and analyzing clinical, genomic and transcriptomic datasets for amyotrophic lateral sclerosis and other neurological disorders.

About John W. Day, MD, PhD

Dr. Day has combined his expertise in synaptic physiology, genetics and neuromuscular medicine to help define the molecular mechanisms underlying myotonic dystrophy and other muscular dystrophies, neuropathies and ataxias. Under his leadership, the Stanford Neuromuscular Program is dedicated to the elucidation and treatment of neuromuscular diseases, integrating clinical care and clinical research with Stanford's basic science and translational programs. All Stanford pediatric and adult neuromuscular patients are asked to participate in research and are followed over time with functional evaluations. The Stanford Neuromuscular Program mission aligns fully with the goals of the MDA: to diagnose, investigate and characterize neuromuscular disorders precisely and completely; to develop novel treatments for neuromuscular disease; to incorporate novel treatments into the comprehensive care of patients with neuromuscular disorders; to advocate and support patients and families affected by neuromuscular disease so they can live life as fully and independently as possible; and to train the next generation of experts in neuromuscular diagnosis, care and research.

About MDA

MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than$1 billionsince our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best in class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. For more information

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Muscular Dystrophy Association Announces Formation of Strategic Medical Advisory Team of Experts in Neuromuscular Care and Research - BioSpace