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BREAKING: Baby body parts criminal trial to proceed against Daleiden with possible jail time – Lifesite

PETITION: Tell Attorney General Barr to PROSECUTE Planned Parenthood's alleged sale of baby body parts. Click here.

SAN FRANCISO, California, December 6, 2019 (LifeSiteNews) -- A San Francisco judge ruled today that pro-life investigators David Daleiden and Sandra Merritt will stand trial on nine felony counts of secretly recording conversations as part of their expos of Planned Parenthoods illegal harvesting and trafficking of aborted baby body parts.

In a ruling released today, Judge Christopher C. Hite of the San Francisco Superior Court dropped five counts against the Center for Medical Progress (CMP) pro-life investigators. He ruled, however, that there was sufficient evidence from the preliminary hearing to try Daleiden and Merritt on nine counts of breaking Californias anti-eavesdropping law during their undercover investigations.

Daleiden, 30, and Merritt, 64, could face jail time if convicted.

Thomas More Society, which represents Daleiden, tweeted:

Daleiden tweeted that the case is falling apart.

Former California Attorney General Kamala Harris concocted this bogus, biased prosecution with her Planned Parenthood backers against undercover video recording, and now their case is falling apart as the facts about Planned Parenthoods criminal organ trafficking are revealed in the courtroom, Daleiden said in a tweeted statement.

The remaining charges under the California video recording lawthe first and only time it has ever been used against undercover news gathererswill fall for the same reasons that five charges were dismissed today: these were public conversations easily overheard by third parties. The real criminals are the Planned Parenthood leadership who sold fetal-body parts from late-term abortions and weaponized the justice system to try to cover it up.

Daleiden and Merritt must now appear at California's Superior Court for instruction and arraignment on January 30.

Daleiden, Merritt and others secretly recorded gatherings of abortionists and organ harvesting companies, and CMP groundbreaking undercover videos released in July 2015 showed Planned Parenthood executives callously discussing how to dismember babies to procure intact organs and haggling over fees of aborted baby parts. The videos shocked the country and led to congressional and senate investigations into Planned Parenthood.

The abortion giant retaliated by suing the pro-life investigators.

A jury ruled November 15 that Daleiden, Merritt, Albin Rhomberg, and Troy Newman of Operation Rescue must pay Planned Parenthood $2.3 million in compensatory and punitive damages and lawyers fees for secretly recording Planned Parenthood and National Abortion Federation conferences in 2014 and 2015.

The pro-lifers are appealing the civil ruling.

Live Actions Lila Rose called Judge Hite's ruling today on the criminal prosecution an abuse of power.

Today's ruling proves unfounded and outrageous charges against two pro-life journalists who exposed the harvesting and trafficking of innocent babies. This is a gross abuse of power and a violation of the First Amendment, she wrote on Twitter.

Animal activist groups went undercover at a duck farm & former CA AG @KamalaHarris used their expose to try to ban foie gras. But when pro-life reporters exposed @PPFA execs haggling over baby parts, the reporters were prosecuted & the abortionists protected, she added in another Tweet.

Harris initiated the investigation of Daleiden and Merritt in 2016, and current Democrat Attorney General Xavier Beccera announced the charges at a press conference in March 2017.

Testimony and evidence presented at the preliminary hearing in September exposed this completely bogus, sham, politically motivated case for the farce that it is, Daleiden told LifeSiteNews in an earlier interview.

Documents from the case now on public record show Harris had an in-person meeting at the attorney generals meeting in Los Angeles with no less than six top-level Planned Parenthood of California executives, he said.

Daleiden added that the meeting took place a couple of weeks before Harris ordered Department of Justice agents to raid his Orange County apartment in April 2016 and seize all his recording materials, including unpublished source documents of his undercover investigation.

We have the minutes in an email from the meeting that Kamala had with the Planned Parenthood leadership, and in that meeting they discussed both Planned Parenthoods political agenda in the state of California, and they also discussed the attorney generals investigation of me and of CMP, Daleiden said.

PETITION: Tell Attorney General Barr to PROSECUTE Planned Parenthood's alleged sale of baby body parts. Click here.

Daleiden and Merritt are claiming a Section 633.5 defense that allows covert recording of confidential communications when done to collect evidence of violent crimes.

Defense is also arguing that the law does not consider confidential any conversation that can reasonably be expected to be overheard.

The two-week preliminary hearing also saw high-level Planned Parenthood abortionists along with the CEO of baby parts harvesting company StemExpress dodging questions on their gruesome business under cross-examination by pro-life defense lawyers.

It was the venue for Daleidens first-ever public testimony about why he embarked on a 30-month undercover sting operation into Planned Parenthood trafficking in aborted baby parts and how he penetrated the networks of those involved in the grisly and illegal business.

It included a cross-examination of investigating agent for the Department of Justice Brian Cardwell that showed he did next to nothing to find out if those accusing Daleiden and Merritt of illegally taping them were telling the truth or understood confidentiality as defined in the law.

It featured defense testimony by one of the United States longest-practicing abortionists Dr. Forrest Smith. He testified that it is almost certain that some of the abortionists featured in the undercover videos deliberately altered abortion procedures in a way that both led to the birth of living babies with beating hearts and put women at risk. The goal in such abortions would be to obtain fresher, more intact organs.

It also included testimony by stem cell expert Dr. Theresa Deisher that the human fetal hearts used in a 2012 Stanford University study and supplied by StemExpress had to be harvested while the babies were still alive.

The really shocking and troubling and clarifying truth that was exposed in the preliminary hearing is that its very, very clear that the only crimes that were committed in the course of the undercover videotaping that Center for Medical Progress did were the crimes committed by the Planned Parenthood abortion doctors, and by the StemExpress body parts harvesters who were forced to testify under oath these past two weeks, Daleiden told LifeSiteNews at that time.

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BREAKING: Baby body parts criminal trial to proceed against Daleiden with possible jail time - Lifesite

Jasper Therapeutics launches out of Stanford with new approach to stem cell treatment – Endpoints News

The first girl in the trial came in with chronic diarrhea and the immune system of an untreated HIV patient. Born with a rare genetic disease that impeded her ability to make B and T cells, she had once been given a stem cell transplant but it didnt take. Back in the hospital, she was injected with a new experimental antibody and then given a new stem cell transplant. Soon, she gained weight. The diarrhea stopped.

She has normal T cells now, Judith Shizuru, the Stanford scientist who pioneered the antibody, told Endpoints News. Shes in school.

Its the kind of medical story to launch a biotech around, and thats what Shizuruis doing. Today, her company Jasper Therapeutics is emerging out of stealth-mode with $35 million in Series A funding led by Abingworth and Qiming, a molecule from Amgen, and a Phase I trial set for its first readout on Monday at ASH.

Jasper is broadly aimed at making stem cell transplants safer, more accessible and more effective by using antibodies as conditioning agents. Theseagents clear out bone marrow to make room for the new stem cells to graft onto the body.

Their Phase I uses a naked antibody called JSP191 to help patients with severe combined autoimmune deficiency receive stem cell transplants the only possible cure for the life-threatening disease but such transplants are used in a wide variety of conditions and Jasper has broader aims. Those include other autoimmune diseases, acute myeloid leukemia and cell-directed gene therapy.

Theres a significant amount of progress being made in gene therapy, interim CEO William Lis told Endpoints, but no progress being made in a conditioning agent that will help graft gene therapy.

Shizuru path to the new antibody was long and fortuitous. In 1987, Arl Arzst, the legendary ad executive and president of Proctor and Gamble international flew in on a recruiting trip for Stanford business students. There he visited Shizuru, a young biologyPhD candidate, because he knew her roommate. Arzsts daughter had diabetes and as Shizuru explained the work she was doing on pancreatic islet cell transplants, he told her to come to Europe.

Shizuru had never been to Europe, but there Arszt introduced her to Ken Farber and the other founders of the Juvenile Diabetes Foundation (now the JDRF). The founders struck a years-long correspondence and encouraged Shizuru to go to medical school, where she decided that if scientists were ever going to develop transplants that didnt trigger an immune response, it would be through stem cell work. She continued her work at the Irv Weissman Stanford regenerative lab, where eventually a graduate student made a discovery that piqued her interest.

To put new stem cells in, you have to get the old stem cells out. Thats not always easy. The cells sit inthese pockets in the bone marrow, and theyre pretty comfortable there. Doctors have to force them out, often using chemotherapy or radiation, which damage DNA and cause severe side effects. The costs sometimes outweigh the benefits.

There are diseases were not treating because its too dangerous, Shizuru said. And the kids were treating, theyre so, so fragile.

The grad student had shown in mice that antibodies could be used to deplete the stem cells and potentially eliminate the need for chemotherapy or radiation. Shizuru and her team began looking to see if anyone had developed a human version of the antibody, CD117. It turned out Amgen had already developed a version of this antibody for a different use. It also turned out she had a former postdoc and a former advisor who worked there. They began a collaboration.

We set out to cross the valley of death, Shizuru said, using an industry slang term for the jump from animal models to human uses.

After making a variety of tweaks to the treatment, they published a paper inScience Translational Medicine in 2016showing the antibodies created a 10,000 fold reduction in the number of stem cells in mice.

The same year, they began a clinical trial on 90 SCID patients. These patients had received stem cell transplants when they were very young but hadnt been given chemo or radiation for fear the side effects would be too severe. The original transplants boosted their numberof immune cells, but without chemo or radiation, the stem cells dont graft into those pockets and the body wont continue producing T cells. Without those, they are extraordinarily prone to infection. Many pass away before age 2.

The hope is that the antibodies allowed the stem cells to graft, and the preliminary answer to that question will be out on Monday. For the first girl in the trial, life has improved but questions about how long her body will make immune cells remain. Still, for that girl and others, Shizuru is confident.

We see there is stem cell engraftment, Shurizi said. They are actually making new T cells.

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Jasper Therapeutics launches out of Stanford with new approach to stem cell treatment - Endpoints News

MaaT Pharma Announces the Presentation of Positive Data with Its Lead Microbiome Biotherapeutic in Intestinal-Predominant Acute…

MaaT Pharmas full-ecosystem microbiota biotherapeutic provided positive impact with satisfactory safety profile in eight patients with advanced stages of GI aGvHD

MaaT Pharma announced today that leading hemato-oncological experts presented clinical data on the compassionate use of MaaT Pharmas lead full-ecosystem microbiome restoration biotherapeutic, MaaT013. The data included eight patients that developed gastrointestinal-predominant, acute Graft-versus-Host-Disease (GI aGvHD) after receiving an allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) to treat their hematologic malignancies. All patients were positively impacted by the MaaT013 treatment, with three patients achieving complete response. GvHD, a condition where the transplant donors immune cells attack the patients tissues, is one of the most serious complications of allo-HSCT, and its acute GI form is fatal in most cases. MaaT013 features a consistently high diversity and richness of microbial species in their natural environment. It aims to restore the symbiotic relationship between microbes in the gut and the immune system of the patient to correct the responsiveness and tolerance (homeostasis) of immune functions and thereby contain GI GvHD. The results were presented in a poster presentation on December 7, 2019 during the 61st American Society of Hematology (ASH) Annual Meeting and Exposition held in Orlando, Florida.

"The GI aGvHD patients who were treated with MaaT013 had a very poor prognosis with no other therapeutic options. The results following MaaT013 administration showed a positive impact on all patients," commented Professor Mohamad Mohty, MD, PhD, Head of the Hematology and Cellular Therapy Department at Sorbonne University, Saint Antoine Hospital in Paris. "The most impressive results were seen in those patients who achieved a complete response and who were able to taper and stop using steroids and other immunosuppressants without relapse of gastrointestinal symptoms."

In the presented evaluation, eight patients with a median age of 67 were treated for classical aGvHD, late-onset aGvHD or aGvHD with overlap syndrome that were either steroid-resistant or steroid-dependent following stem cell transplantation. These patients had previously been treated with and failed up to five lines of systemic therapy for aGvHD. Each patient received at least one and up to three doses of MaaT013 and treatment response was evaluated seven days after each administration and on day 28 after the first dose. Based on the best response to the treatment, all eight patients experienced at least a partial response with three patients achieving complete response, two patients with very good partial response and three patients with partial response. Overall, the data demonstrated that reintroduction of a full-ecosystem microbiota provided therapeutic effect and was tolerated in a satisfactory manner in these patients.

Herv Affagard, Co-founder and CEO of MaaT Pharma added, "We provided our cGMP-manufactured lead biologic drug, MaaT013, to hospitals as part of a compassionate use program to give GI GvHD patients a therapeutic option where there are no other available treatments after steroids and additional lines of treatment. These findings indicate that reestablishing the gut microbiome improved outcomes in these patients."

Moreover, MaaT Pharma is currently conducting the HERACLES Phase II clinical trial (NCT03359980) to evaluate the safety and efficacy of MaaT013 in steroid-refractory, GI aGvHD patients, with more than half of the patients enrolled.

To date, a total of 46 patients with GI GvHD have been treated with MaaT013, including patients under compassionate use and patients enrolled in the Phase II clinical trial. MaaT Pharma is actively developing an oral formulation of MaaT013 (a capsule, MaaT033) to provide easier administration for patients while delivering a similar effect of regenerating the microbial ecosystem with the goal of restoring immune homeostasis in the gut.

The poster can be viewed on the companys website under "News".

About HERACLES

The HERACLES study is a multi-center, single-arm, open-label study, enrolling 32 patients to evaluate the efficacy and safety of MaaT Pharmas lead microbiome restoration drug candidate, MaaT013, in steroid-resistant, gastrointestinal-predominant aGvHD patients. Acute GvHD is a serious, often fatal syndrome typically involving the gut, skin, and liver. Treatments up to now focused largely on suppressing the immune reaction that is induced by the donor cells derived from the hematopoietic stem cell graft reacting against the host. These strategies have remained clinically unsuccessful in most cases, with mortality rates around 80% after twelve months in steroid-resistant cases. Patients with hematological malignancies receive multiple courses of chemotherapy, antibiotics, and ultimately conditioning before HSCT, which are known to severely impact the gut microbial composition.

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About MaaT013

MaaT013 is the first full-ecosystem, off-the-shelf, reproducible, enema formulation manufactured using MaaT Pharmas integrated Microbiome Restoration Biotherapeutic (MMRB) platform. The product has a stability of up to 24 months and is characterized by a high diversity and consistent richness of microbial species derived from pooled healthy donors and manufactured at the companys centralized European cGMP production facility. MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and is already being administered in compassionate use.

About MaaT Pharma

MaaT Pharma, a clinical stage company, has established the most complete approach to restoring patient-microbiome symbiosis to improve survival outcomes in life-threatening diseases. Committed to treating blood cancers and Graft-versus-Host-Disease, a serious complication of allogeneic stem cell transplantation, MaaT Pharma has already achieved proof of concept in acute myeloid leukemia patients. Supporting the further expansion of our pipeline into improving outcomes of immunotherapy in solid tumors, we have built a powerful discovery and analysis platform, GutPrint, to evaluate drug candidates, determine novel disease targets and identify biomarkers for microbiome-related conditions. Our biotherapeutics are produced under the strictest cGMP manufacturing and quality control process to safely deliver the full diversity and functionality of the microbiome. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to spearhead microbiome treatment integration into clinical practice.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191207005042/en/

Contacts

For MaaT Pharma Herv Affagard, CEOPhone: +33 4 2829 1400E-Mail: haffagard@maat-pharma.com

Media Requests for MaaT Pharma Dr. Stephanie May or Dr. Jacob VergheseTrophic CommunicationsPhone: +49 89 23 88 77 30 or +49 171 185 56 82E-Mail: may@trophic.eu or verghese@trophic.eu

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MaaT Pharma Announces the Presentation of Positive Data with Its Lead Microbiome Biotherapeutic in Intestinal-Predominant Acute...

Autolus Therapeutics Announces New Data Showcasing Clinical Progress of Programmed T Cell Therapy Pipeline in Blood Cancers – GlobeNewswire

AUTO1 shows 87% MRD negative complete response in adult patients with r/r ALL, with no severe cytokine release syndrome

Data presented at 61st American Society of Hematology Annual Meeting form basis for advancement of AUTO1 into pivotal clinical trial in adult ALL

Investor call to be held December 9 at 8:30 am ET / 1:30 pm GMT to review data

LONDON, Dec. 07, 2019 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc(Nasdaq: AUTL) announced today new data highlighting progress on its next-generation programmed T cell therapies to treat patients with acute lymphoblastic leukemia (ALL) and adults with relapsed/refractory diffuse large B cell lymphoma (DLBCL). The data were presented in oral presentations at the 61stAmerican Society of Hematology(ASH) Annual Meeting and Exposition inOrlando, FL. Additional data on pediatric patients with ALL will be presented on December 8.

The data on AUTO1 presented at this years ASH meeting demonstrate the favorable safety profile and high level of clinical activity of AUTO1 in both adults and pediatric patients with ALL, and we look forward to initiation of the pivotal program in adult ALL in the first half of 2020, said Dr. Christian Itin, chairman and chief executive officer of Autolus.

Acute Lymphoblastic Leukemia Data Presented

Title: AUTO1 A novel fast off CD19CAR delivers durable remissions and prolonged CAR T cell persistence with low CRS or neurotoxicity in adult ALL (Abstract # 226)

Updated results for ALLCAR19, the Phase 1 trial evaluating AUTO1 in adults with recurrent/refractory ALL, were presented by Dr. Claire Roddie MB, PhD, FRCPath, honorary senior lecturer,Cancer Institute, University College London (UCL), in an oral presentation. The trial is designed to assess the primary endpoints of safety ( Grade 3 toxicity) and feasibility of product generation, as well as other secondary endpoints, including efficacy. The trial enrolled patients with a high tumor burden (44% had 50% BM blasts), who were considered high-risk for experiencing cytokine release syndrome (CRS). Product was manufactured for 19 patients; product for 13 of those patients was manufactured using a semi-automated closed process, which will be used for commercial supply.

As of the data cut-off date of November 25, 16 patients had received at least one dose of AUTO1. AUTO1 was well tolerated, with no patients experiencing Grade 3 CRS, and 3 of 16 patients (19%), who had high leukemia burden, experiencing Grade 3 neurotoxicity that resolved swiftly with steroids.

Of 15 patients evaluable for efficacy, 13 (87%) achieved MRD negative CR at 1 month and all patients had ongoing CAR T cell persistence at last follow up. CD19-negative relapse occurred in 22% (2 of 15) patients. In the patients dosed with AUTO1 manufactured in the closed process, 9 of 9 (100%) achieved MRD negative CR at 1 month and 6 months event free survival, and overall survival in this cohort was 100%.

Adult ALL patients, who face a median survival of less than one year after their ALL recurs or relapses, have a significant need for a CAR T cell therapy that is highly active, safe and is a standalone therapy not requiring a stem cell transplant, said Dr. Hagop M. Kantarjian, Chair of the Department of Leukemia at The University of Texas MD Anderson Cancer Center.

The novel CD 19 CAR-T therapy, AUTO1, is potentially transformative as a standalone curative option for patients with r/r ALL, especially in adults, given its favorable safety profile, said Dr. Max Topp associate professor of Internal Medicine, Hematology and Oncology at the University of Wuerzburg.

Title: Therapy of pediatric B-ALL with a lower affinity CD19 CAR leads to enhanced expansion and prolonged CAR T cell persistence in patients with low bone marrow tumor burden, and is associated with a favorable toxicity profile (Abstract # 225)

Dr. Sara Ghorashian, honorary senior lecturer, Great Ormond Street Institute of Child Health, University College London, presented updated data from the phase 1 CARPALL study of AUTO1 in pediatric ALL patients with low bone marrow tumor burden. The trial is intended to assess the primary endpoints of safety and proportion of patients in molecular complete remission at 1 month. The study recruited a total of 25 patients and stratified them into 2 cohorts. Fourteen patients were treated in cohort 1, which utilized a manual manufacturing process; product was unable to be generated in 3 patients. Median follow-up was 27 months in cohort 1. Seven patients were treated in cohort 2, which utilized the semi-automated closed manufacturing process, which will be used for commercial supply. The aim of cohort 2 was to demonstrate feasibility of manufacture at scale. Product was generated for 100% of patients. Median follow-up was 7 months in cohort 2.

AUTO1 was well-tolerated overall, with no patients experiencing Grade 3 CRS and 1 of 21 (5%) experiencing Grade 4 neurotoxicity, which was considered unrelated to CAR T therapy.

Nineteen of 21 treated patients (90%) achieved molecular complete remission at 1 month post infusion. Consistent with pre-clinical data, CAR T cell expansion was excellent and detectable by flow in a number of patients up to 36 months. Persistence was noted in 15 of 21 patients at last follow-up, up to 36 months. In cohort 2, 100% of patients achieved molecular complete remission at 1 month post infusion.

In the 14 patients in cohort 1, the overall survival at 6 months was 86% and at 12 months was 71%; event free survival (EFS) at 6 months was 71% and at 12 months was 54%. The patients in cohort 2 are not yet evaluable for these parameters. Overall, nine patients relapsed; 5 of 8 evaluable relapses were due to loss of CD19 antigen on the tumor cells.

Title: Clonal dynamics of early responder and long-term surviving CAR-T cells in humans (Abstract # 52)

Dr. Luca Biasco, senior research associate at University College London, presented a detailed analysis of CAR T products, and insertion site analysis from the CARPALL phase 1 patients. This analysis revealed highly polyclonal engraftment, even at very late time-points. Dr. Biasco hypothesized that the propensity for high level polyclonal long-term engraftment was due to favorable phenotype of the CAR T product and the binding kinetic of the receptor.

Diffuse Large B-cell Lymphoma Data Presented

Title: Phase 1/2 study of AUTO3, the first bicistronic chimeric antigen receptor (CAR) targeting CD19 and CD22 followed by an anti-PD1 in patients with relapsed/refractory (r/r) Diffuse Large B Cell Lymphoma (DLBCL): Results of cohort 1 and 2 of the ALEXANDER study (Abstract # 246)

Dr. Kirit Ardeshna, consultant hematologist, Department of Hematology, University College London Hospital NHS Foundation Trust, presented updated data from the ALEXANDER Phase 1/2 study of AUTO3, the first bicistronic CAR T targeting CD19 and CD22 followed by an anti-PD1, in diffuse large B cell lymphoma (DLBCL). 16 patients were treated, and fourteen patients were evaluable at one month. AUTO3 was well-tolerated, with no patients experiencing Grade 3 CRS with primary treatment, and 1 of 14 experiencing Grade 3 neurotoxicity that resolved swiftly with steroids. Five of 14 had a complete response, with 4 of 5 complete responses ongoing, the longest at 18 months.

DLBCL is an aggressive and rapidly progressing cancer, and early response is critical to ensuring positive outcomes for these patients. These early data show the promise of AUTO3 in DLBCL, and we expect to advance AUTO3 to a decision point in relapsed/refractory DLBCL by the middle of next year, said Dr. Christian Itin, chairman and chief executive officer of Autolus. In addition, we look forward to presenting the data from the AMELIA trial of AUTO3 in pediatric ALL during poster sessions on Sunday, December 8, 6:00 8:00 PM ET.

Investor call to review data on Monday, December 9

Autolus management will host an investor conference call on Monday, December 9, at 8:30 a.m. EDT/ 1:30pm GMT, to review the data presented at ASH.

To listen to the webcast and view the accompanying slide presentation, please go to:https://www.autolus.com/investor-relations/news-and-events/events.

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 9796038. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 9796038.

About AUTO1

AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety - while maintaining similar levels of efficacy - compared to current CD19 CAR T cell therapies.Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the T cells' abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a license agreement under which Autolus acquired global rights fromUCL Business plc(UCLB), the technology-transfer company of UCL, to develop and commercialize AUTO1 for the treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL.

About AUTO3

AUTO3 is a programmed T cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that have each been independently optimized for single target activity. By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies. AUTO3 is currently being tested in pediatric ALL in the AMELIA clinical trial and in diffuse large B cell lymphoma in the ALEXANDER clinical trial.

AboutAutolus Therapeutics plc

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information please visit http://www.autolus.com.

Forward-Looking Statement

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus financial condition and results of operations, as well as statements regarding the anticipated development of Autolus product candidates, including its intentions regarding the timing for providing further updates on the development of its product candidates, and the sufficiency of its cash resources. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed on November 23, 2018 as well as discussions of potential risks, uncertainties, and other important factors in Autolus' future filings with the Securities and Exchange Commission from time to time. All information in this press release is as of the date of the release, and the company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

Investor and media contact: Silvia TaylorVice President, Corporate Affairs and Communications Autolus+1-240-801-3850s.taylor@autolus.com

UK:Julia Wilson+44 (0) 7818 430877j.wilson@autolus.com

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Autolus Therapeutics Announces New Data Showcasing Clinical Progress of Programmed T Cell Therapy Pipeline in Blood Cancers - GlobeNewswire

Sickle Cell Anemia Patient Becomes First Person in the US to Have Her Genes Edited With CRISPR – Interesting Engineering

Last week, a woman namedVictoria Gray became the first person in the U.S. to have her cells edited with CRISPR. The41-year-old patient was sufferingfromsickle cell anemia.

RELATED:FIRST HUMAN TRIAL USING CRISPR GENE-EDITING IN US BEGINS

The condition, caused by a genetic mutation that messes with the shape of red blood cells, causes havoc on patients, and to make things even worse, the options for treatment are very limited and ineffective. The only current treatment for sickle cell anemia patients is a donor transplant that works for just 10% of patients, but all that is about to change.

It was clear that analternative, much more effectivesolutionwas desperately needed. After much consideration, doctors believed that editing cells extracted from a patient's own bone marrow could restore effective red blood cell creation, and this is exactly the operation they attempted on Gray.

The doctors used CRISPR to tweak Gray's bone marrow DNA to turn on a specific protein that would allow proper red blood cell generation. The operation makes Gray the first person in the U.S. to undergo a CRISPR editing procedure and the second globally.

The treatment comes from observations made back in the 1940s.In 1941 a pediatrician named Jane Watson noticed that babies with sickle cell didnt have symptoms until 6 months to 1 year of age, Vivien Sheehan, a hematologist at Baylor University told Popular Science.

The pediatrician also discovered that these infants produced fetal hemoglobin for much longer periods than healthy babies.Following Watson's observations, the research since then has indicated that increasing fetal hemoglobin could provide an effective treatment for the disease.

Now, CRISPR may just make that treatment viable. But before we get too excited, it should be noted that the strategy comes with several risks.

In order for the edited cells to be inserted back into the patients bone marrow, other stem cells need to be deactivated. Otherwise, there is the chance the unedited stem cells may continue to produce sickled red blood cells very fast, outpacing the edited cells' production of healthy cells.

Now researchers say they need to follow Gray's progress for at least 15 years to rule out any other potential dangers of the procedure. Still, for those 90% suffering with sickle cell anemia that don't respond well to current treatment, the procedure, if successful, would offer the much-needed lifeline they've been hoping for.

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Sickle Cell Anemia Patient Becomes First Person in the US to Have Her Genes Edited With CRISPR - Interesting Engineering

Ask the Doc! Check-ups and Stem Cell – Clay Co N.C. – Fetchyournews.com

Ask the Doc! Check-ups and Stem Cell Health December 6, 2019 , by Destyne Riblett

Dr. William Whaley answers your questions about getting family members to go for check-ups and one family asking about stem cells in cancer treatment.

Previous Article Three arrested for maintaining an alleged drug house

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Ask the Doc! Check-ups and Stem Cell - Clay Co N.C. - Fetchyournews.com

Teen diagnosed with cancer again just months after being told he beat disease – Mirror Online

A teenage boy has been diagnosed with an aggressive cancer again just four months after a doctor told him he had beaten the devastating disease.

There is just a 10 per cent chance that chemotherapy will work this time for 17-year-old Jacob Fradgley, who is "wasting away", his heartbroken mum said.

The teen, from Skegby in Nottinghamshire, originally found out that he had metastatic Ewing sarcoma, a rare form of bone cancer, when he was just 15 in May 2018.

After a "whirlwind" year of treatment, including 19 rounds of chemotherapy and 56 sessions of radiation therapy, Jacob was classed as cancer free in July, his mum, Sammy Fradgley, 34, told NottinghamshireLive.

But they were given the heartbreaking news that his cancer had returned after his mum found a lump on his head just weeks before Christmas.

Jacob was diagnosed with a relapse of metatistic ewing sarcoma, which affects bones and bone tissue.

Only 30 children a year are diagnosed in the UK.

Miss Fradgley has launched a campaign to fulfil some of her son's wishes - including meeting ex-soldier Ant Middleton SAS: Who Dares Wins - after doctors told her that if there was anything he wanted to do he should "do it now".

After his mum found the lump, Jacob was taken to Queen's Medical Centre in Nottingham for a CT scan.

He underwent surgery to remove the tumour in his head a week later.

Doctors then found more tumours in his head, hip and knees.

Miss Fradgley, a senior carer, said: "It took me two weeks to accept it.

"There's not a lot out there to treat a relapse, and we're just getting further away from solutions.

"There's around a 10 per cent chance the chemotherapy will work this time.

"You never think it's going to come back but I've just got to support him now."

Jacob had his final round of radiation therapy in January and his last round of chemo on New Year's Eve.

In the following months he took medication to prevent the cancer returning.

His mum said: "He was able to go to his prom, he was going to cadets and studying Public Services at Vision West Notts College.

"But three weeks ago I was touching his hair, and felt the lump. It all just happened so quick.

"Now its more or less the same thing. It's so aggressive and is known for coming back."

She added: "It has upset him, that he has to go through it all again.

"He has to have six rounds of chemotherapy and the doctors also want to harvest stem cells. If the chemo works, he may be able to go for stem cell treatment."

Jacob's consultant has told her that if there was something he wanted to do he should "do it now".

She has launched a JustGiving campaign to raise enough money so Jacob can create memories with his six-year-old sister Leah.

It has raised more than 2,300 so far.

Miss Fradgley said: "I just don't want to say no. I want to give him everything he wants.

"It's so hard to just watch him wasting away in front of me, but I am trying to do everything I can for him.

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"He does still smile, but I am also there when he gets upset.

"His biggest aspiration is to drive a tank, meet Ant Middleton - anything to do with the army.

"His favourite colour is red so I have also started a 'Going Red for Jacob' campaign and I plan to make red bands with 'Team Fradgley' so everyone can support him and donate."

Barbera White, founder of the When You Wish Upon a Star charity, which is supporting the family, said: "I just wanted mum to know if there's anything we can do, we will try.

"I am trying to arrange a wish for him and I am currently working on something that I know he wants.

"Even after 30 years of the charity, I keep in touch with the families I meet, and we all want to help create some memories for Jacob."

Last December, the charity gave Jacob, his mum and his sister a trip to Lapland as he battled cancer.

At the time, he was looking forward to spending Christmas out of hospital and said the Lapland trip was a chance to "be together like a normal family".

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Teen diagnosed with cancer again just months after being told he beat disease - Mirror Online

Living with cancer: ‘Exercising during my chemotherapy treatment worked wonders’ – inews

NewsReal LifeCathal Morrow, 54, talks about the positive effects running and gym sessions had on him both physically and mentally

Friday, 6th December 2019, 5:33 pm

As part of a new series calledLiving with cancer,iis sharing people's stories about how the disease affects their life and relationships, both practically and emotionally.

Cathal Morrow knew that he could have easily have slumped into a depression after hearing the dreaded C word.

When he began his three month chemotherapy treatment, it knocked him for six. "The first 10 days I lay in bed watching Netflix. I felt rotten, exhausted and sick. I felt like I'd been hit by a bus. I thought oh gosh, I've got three months of this."

Then his youngest son had what he calls a post-traumatic stress disorder (PTSD) episode, and Cathal had to physically restrain him. It made him realise he was stronger than he had realised. And so, from then, he forced himself to turn off the telly and go outside a trip to the shops or a stroll around the park.

"I was knackered afterwards, but it felt good," he said. Having quit smoking when he was diagnosed with T-Cell Lymphoma, a type of blood cancer, he decided to go to the gym. It felt good, so the next day he went for a run, and managed 6 km (3.7 miles), and the next day pounded the pavements again.

Cathal had run a marathon a decade ago, but had stopped running regularly. The 54-year-old soon built up his fitness and took on a half marathon (21 km/13.1 miles) while still having chemo.

Exercise can help general health, and there's been a recent paradigm shift with regards to the role it plays in cancer patients. Last year, leading experts on cancer called for exercise to be prescribed to all cancer patients, alongside traditional treatment, and say not to do so would be harmful.

If the effects of exercise could be encapsulated in a pill, it would be prescribed to every cancer patient worldwide and viewed as a major breakthrough in cancer treatment, Professor Prue Cormie from the Australian Catholic University wrote on The Conversation.

'It stops me from becoming a miserable sod'

Cathal began exercising five times a week, and he says it helped empower him to move out of a more passive patient role, and improved not just his well-being but his attitude, too.

"It would be easy to think, I'm sick, I'm having treatment, so I must wrap myself up in cotton wool. Doing the half-marathon, I was shouting 'Fuck cancer' as I hauled myself up a gruesome hill. It was my way of sticking my finger up to the disease. It worked wonders.

"Running helped me manage my fear of dying. There's no two ways about it, it's terrifying to be told you have cancer. I had a good cry, then I thought right, I'm beating this. Exercise was a way to channel my frustration, at times rage.

"It gave me something to look forward to almost every day, something very immediate to focus on that I could achieve. It made me feel at least partly in control of my health.

"It's also had a positive effect on my two sons. They saw I was fighting cancer, that I refuse to give in. It stops me from becoming a miserable sod, because I dont need to fake positivity."

Rest is 'counterproductive'

Macmillan Cancer Support also say that traditional advice for combating fatigue centering on energy conservation is "counterproductive", since "excessive rest worsens treatment-related loss of physical function".

'Studies have found exercise during treatment can actually change the tumor microenvironment and trigger stronger anti-tumor activity'

Mayo Clinic experts

The charity states: "This can lead to a vicious cycle of accumulating fatigue and deteriorating function. A promising body of evidence indicates that an appropriate balance of physical activity alongside rest during treatment periods helps to control fatigue and maintain physical function."

Experts at the Mayo Clinic say as well as reducing depression and anxiety, exercise can reduce pain. They point out "studies have found that exercise during treatment can actually change the tumor microenvironment and trigger stronger anti-tumor activity in your immune system". And animal studies have found that exercise can lead to tumor reduction in rodents.

Physical activity also helps you manage your weight, which is an important cancer risk factor and has been linked to higher risk of cancer recurrence.

'Balance is key'

Cathal, from Wimbledon, London, says exercise made him feel better prepared physically to be able to handle the treatment. His doctor approved of him getting active, but warned him not to overdo it.

Cathal, who works in PR and as a writer, underwent stem cell treatment last November, and has since gone into remission, although he still struggles with fatigue.

"My doctor is fully supportive of me exercising my way through chemo although she cautioned me not to 'go mental'. I probably did a bit. I just love pushing my body past what I think is possible. It's felt fantastic feeling the fittest I've been in a decade.

"But I think it's also important to listen to you body, balance is key. I aimed to exercise five times a week but if I felt rubbish I'd rest. Same goes for the emotional side. Sometimes you have to succumb to not feeling very positive. Some days I feel too crap to do anything and spend a few days in bed."

Cancer Research says how much and how intense a cancer patient should exercise will depend on the individual, and advises people check with their doctor beforehand. You can read their guidelines about exercising when you have cancer here.

Do you have a personal story? Email claudia.tanner@inews.co.uk

View original post here:
Living with cancer: 'Exercising during my chemotherapy treatment worked wonders' - inews

The future of Ipsen’s new $1.3B drug is in doubt as safety fears force the FDA to slam the brakes on late-stage studies – Endpoints News

Having made its marketing pitch to the EU regulator, Novartis on Thursday unveiled positive pivotal study data supporting the use of its inhaled asthma treatment.

The therapy, QMF149, consists of the long-acting beta-agonist, or LABA, called indacaterol acetate and the corticosteroid mometasone furoate. In the 2,216-patient, 52-week PALLADIUM study, asthma patients either received a medium or high dose of the Novartis therapy (150/160 g; 150/320 g) or mometasone furoate (MF) alone.

Read More

The rest is here:
The future of Ipsen's new $1.3B drug is in doubt as safety fears force the FDA to slam the brakes on late-stage studies - Endpoints News

Takeda to Present Results from the Phase 3 TOURMALINE-AL1 Trial of NINLARO in Patients with Amyloidosis – Business Wire

CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) today announced that results of the TOURMALINE-AL1 trial will be presented during an oral session at the 61st American Society of Hematology (ASH) annual meeting on Saturday, December 7, 2019 in Orlando, Florida. TOURMALINE-AL1 is a Phase 3, randomized clinical trial evaluating the effect of NINLAROTM (ixazomib) in combination with dexamethasone in patients with relapsed or refractory systemic light-chain (AL) amyloidosis.

The TOURMALINE-AL1 trial did not meet the first of the two primary endpoints of significant improvement in overall hematologic response, as reported in June 2019. Hematologic responses were seen in 53% versus 51% of patients receiving NINLARO plus dexamethasone versus physicians choice (odds ratio 1.10 [95% CI 0.60-2.01], p=0.762) as assessed by an Adjudication Committee (AC). The second primary endpoint of two-year vital organ deterioration or death was not mature at the time of analysis. Other endpoints studied including vital organ progression free survival (PFS), hematologic PFS, time to treatment failure and time to subsequent therapy were numerically higher in the NINLARO plus dexamethasone arm compared to the physicians choice arm. Takeda is committed to making data available to researchers to continue investigation of this disease. NINLARO is not approved as a treatment for AL amyloidosis.

AL amyloidosis is a rare condition, for which prognosis and patient outcomes are poor. Current treatments are often retrofitted from therapies used for multiple myeloma, said Angela Dispenzieri, MD, Mayo Clinic, and the trials principal investigator and lead author. For a Phase 3 study that did not meet its primary endpoint, this trial provides interesting information for this community and for future studies. Ongoing research and development to investigate potential treatment options for this underserved patient population is critical.

We look forward to the opportunity to share the data from the TOURMALINE-AL1 trial, said Phil Rowlands, Head of Oncology Clinical Research and Development, Takeda. We are confident that sharing our findings with the community will help encourage conversations around the need for continued research to address the needs that remain in this patient population.

There are serious unmet needs for people living with amyloidosis. AL amyloidosis is a progressive and fatal disease; many patients are diagnosed late, significantly impacting life expectancy. The challenges associated with developing drugs for this disease make continued research and development for treatment critical, said Isabelle Lousada, Founder and CEO of the Amyloidosis Research Consortium. The data from TOURMALINE-AL1 provide valuable insights to researchers as they select endpoints for future amyloidosis studies, and knowledge that will provide context in future drug reviews and approvals, ultimately aiding in providing treatment options for patients.

Primary Results from the Phase 3 TOURMALINE-AL1 Trial of Ixazomib-Dexamethasone Versus Physicians Choice of Therapy in Patients (Pts) with Relapsed/Refractory Primary Systemic AL Amyloidosis (RRAL). Saturday, December 7, 9:30 a.m., Orange County Convention Center, Hall E1.

Key findings, to be presented by Dr. Angela Dispenzieri, include:

About the TOURMALINE-AL1 Trial

TOURMALINE-AL1 (NCT01659658) is an international, randomized, controlled, open-label, multicenter, Phase 3 study, designed to determine whether NINLAROTM (ixazomib) in combination with dexamethasone improves hematologic response, two-year vital organ (heart or kidney) deterioration and mortality rate versus a physician's choice of a chemotherapy regimen in participants diagnosed with relapsed or refractory systemic light chain (AL) amyloidosis. 168 patients were enrolled and randomly selected to receive either NINLARO plus dexamethasone, or physicians choice of the following: dexamethasone plus melphalan; dexamethasone plus cyclophosphamide; dexamethasone plus thalidomide; dexamethasone plus lenalidomide; or dexamethasone alone. The discontinuation of the TOURMALINE-AL1 trial was announced in June 2019. For more information, please visit https://clinicaltrials.gov/ct2/show/NCT01659658.

About AL Amyloidosis

Primary AL amyloidosis is a condition that falls under the umbrella of plasma cell dyscrasias. AL amyloidosis arises from a clonal plasma cell that produces abnormal immunoglobulin light-chain fragments. These misfolded light-chains form insoluble fibrils that aggregate as amyloid deposits in organs and tissues throughout the body, ultimately leading to organ dysfunction and death. The most common organs affected are the kidneys, heart, liver, and autonomic or peripheral nerves.

There are currently no treatments approved for the treatment of AL amyloidosis.

About NINLAROTM (ixazomib) capsules

NINLARO (ixazomib) is an oral proteasome inhibitor which is being studied across the continuum of multiple myeloma treatment settings. NINLARO was first approved by the U.S. Food and Drug Administration (FDA) in November 2015 and is indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy. NINLARO is currently approved in more than 60 countries, including the United States, Japan and in the European Union, with more than 10 regulatory filings currently under review. It was the first oral proteasome inhibitor to enter Phase 3 clinical trials and to receive approval.

The comprehensive ixazomib clinical development program, TOURMALINE, includes several ongoing pivotal trials, which together are investigating major multiple myeloma patient populations:

In addition to the TOURMALINE program, ixazomib is being evaluated in multiple therapeutic combinations for various patient populations in investigator initiated studies globally.

NINLARO (ixazomib) capsules: Global Important Safety Information

SPECIAL WARNINGS AND PRECAUTIONSThrombocytopenia has been reported with NINLARO (28 percent vs. 14 percent in the NINLARO and placebo regimens, respectively) with platelet nadirs typically occurring between Days 14-21 of each 28-day cycle and recovery to baseline by the start of the next cycle. It did not result in an increase in hemorrhagic events or platelet transfusions. Monitor platelet counts at least monthly during treatment with NINLARO and consider more frequent monitoring during the first three cycles. Manage with dose modifications and platelet transfusions as per standard medical guidelines.

Gastrointestinal toxicities have been reported in the NINLARO and placebo regimens respectively, such as diarrhea (42 percent vs. 36 percent), constipation (34 percent vs. 25 percent), nausea (26 percent vs. 21 percent), and vomiting (22 percent vs. 11 percent), occasionally requiring use of antiemetic and anti-diarrheal medications, and supportive care.

Peripheral neuropathy was reported with NINLARO (28 percent vs. 21 percent in the NINLARO and placebo regimens, respectively). The most commonly reported reaction was peripheral sensory neuropathy (19 percent and 14 percent in the NINLARO and placebo regimens, respectively). Peripheral motor neuropathy was not commonly reported in either regimen (< 1 percent). Monitor patients for symptoms of peripheral neuropathy and adjust dosing as needed.

Peripheral edema was reported with NINLARO (25 percent vs. 18 percent in the NINLARO and placebo regimens, respectively). Evaluate patients for underlying causes and provide supportive care, as necessary. Adjust the dose of dexamethasone per its prescribing information or the dose of NINLARO for severe symptoms.

Cutaneous reactions occurred in 19 percent of patients in the NINLARO regimen compared to 11 percent of patients in the placebo regimen. The most common type of rash reported in both regimens was maculo-papular and macular rash. Manage rash with supportive care, dose modification or discontinuation.

Hepatotoxicity, drug-induced liver injury, hepatocellular injury, hepatic steatosis, and hepatitis cholestatic have been uncommonly reported with NINLARO. Monitor hepatic enzymes regularly and adjust dose for Grade 3 or 4 symptoms.

Pregnancy- NINLARO can cause fetal harm. Advise male and female patients of reproductive potential to use contraceptive measures during treatment and for an additional 90 days after the final dose of NINLARO. Women of childbearing potential should avoid becoming pregnant while taking NINLARO due to potential hazard to the fetus. Women using hormonal contraceptives should use an additional barrier method of contraception.

Lactation- It is not known whether NINLARO or its metabolites are excreted in human milk. There could be potential adverse events in nursing infants and therefore breastfeeding should be discontinued.

SPECIAL PATIENT POPULATIONSHepatic Impairment: Reduce the NINLARO starting dose to 3 mg in patients with moderate or severe hepatic impairment.

Renal Impairment: Reduce the NINLARO starting dose to 3 mg in patients with severe renal impairment or end-stage renal disease (ESRD) requiring dialysis. NINLARO is not dialyzable and, therefore, can be administered without regard to the timing of dialysis.

DRUG INTERACTIONSCo-administration of strong CYP3A inducers with NINLARO is not recommended.

ADVERSE REACTIONSThe most frequently reported adverse reactions ( 20 percent) in the NINLARO regimen, and greater than in the placebo regimen, were diarrhea (42 percent vs. 36 percent), constipation (34 percent vs. 25 percent), thrombocytopenia (28 percent vs. 14 percent), peripheral neuropathy (28 percent vs. 21 percent), nausea (26 percent vs. 21 percent), peripheral edema (25 percent vs. 18 percent), vomiting (22 percent vs. 11 percent), and back pain (21 percent vs. 16 percent). Serious adverse reactions reported in 2 percent of patients included thrombocytopenia (2 percent) and diarrhea (2 percent). For each adverse reaction, one or more of the three drugs was discontinued in 1percent of patients in the NINLARO regimen.

For European Union Summary of Product Characteristics: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-Product_Information/human/003844/WC500217620.pdf For US Prescribing Information: https://www.ninlarohcp.com/pdf/prescribing-information.pdf For Canada Product Monograph: http://www.takedacanada.com/ninlaropm

About Takeda Pharmaceutical CompanyTakeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries.

For more information, visit https://www.takeda.com

Important Notice

For the purposes of this notice, press release means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (Takeda) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, Takeda is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words we, us and our are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as targets, plans, believes, hopes, continues, expects, aims, intends, ensures, will, may, should, would, could anticipates, estimates, projects or similar expressions or the negative thereof. Forward-looking statements in this document are based on Takedas estimates and assumptions only as of the date hereof. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takedas global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the timing and impact of post-merger integration efforts with acquired companies; and the ability to divest assets that are not core to Takedas operations and the timing of any such divestment(s), any of which may cause Takedas actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takedas results, performance, achievements, or financial position, see Item 3. Key InformationD. Risk Factors in Takedas most recent Annual Report on Form 20-F and Takedas other reports filed with the U.S. Securities and Exchange Commission, available on Takedas website at: https://www.takeda.com/investors/reports/sec-filings/ or at http://www.sec.gov. Future results, performance, achievements or financial position of Takeda could differ materially from those expressed in or implied by the forward-looking statements. Persons receiving this press release should not rely unduly on any forward-looking statements. Takeda undertakes no obligation to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takedas future results.

Link:
Takeda to Present Results from the Phase 3 TOURMALINE-AL1 Trial of NINLARO in Patients with Amyloidosis - Business Wire