CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001 at American Society of Hematology Annual…

December 05, 2020 12:30 ET | Source: CRISPR Therapeutics AG

- Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion -

- Sickle cell disease: All three patients were free of vaso-occlusive crises with 3 to 15 months of follow-up after CTX001 infusion -

- Nineteen patients have been dosed with CTX001 across both programs -

- The New England Journal of Medicine publishes CTX001 manuscript containing the first report of investigational use of CRISPR/Cas9-based gene editing to treat inherited diseases in humans -

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 05, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, that show a consistent and sustained response to treatment. All seven patients with transfusion-dependent beta thalassemia (TDT), including three who have either a severe or b0/b0 genotype, were transfusion independent at last follow-up and all three patients with sickle cell disease (SCD) were free of vaso-occlusive crises (VOCs) from CTX001 infusion through last follow-up. These data will be presented during the Scientific Plenary at the annual ASH Meeting and Exposition on December 6, 2020. A summary of the results from the CLIMB-111 and CLIMB-121 Phase 1/2 clinical studies is provided below.

The companies also announced that The New England Journal of Medicine (NEJM) has published an independently peer-reviewed article entitled CRISPR-Cas9 Gene Editing for Sickle Cell Disease and Thalassemia. The article includes detailed information on the first patient with TDT enrolled in CLIMB-111 and the first patient with severe SCD enrolled in CLIMB-121, at 18 and 15 months of follow-up, respectively.

CTX001 is being investigated in these two ongoing Phase 1/2 clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

We are pleased with the data presented at ASH, which demonstrate potential benefit and durability among a larger population of patients with transfusion-dependent beta thalassemia and sickle cell disease, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. Additionally, the NEJM case study is the first peer-reviewed journal publication for our CRISPR/Cas9 gene therapy, CTX001. Together this is further validation of the potential of CTX001 to become a best-in-class therapy. We plan to continue the rapid advancement of our clinical trials to bring these much-needed therapies to patients.

These are the first published results from CRISPR/Cas9 therapy in people with a genetic disease and represent an important milestone in medicine and for our collaboration with CRISPR Therapeutics. Most importantly, these data represent a critical step in our effort to bring transformative and potentially curative therapies to patients, said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. With clinical proof-of-concept for both beta thalassemia and sickle cell disease and 19 patients dosed, we look forward to continued efforts to bring our investigational treatment to patients living with TDT and SCD as quickly as we can.

Our vision with this approach is to use the patients own stem cells to provide a transformative treatment for these diseases, something almost unimaginable a few years ago, said Dr. Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center. With these data in 10 patients, we can see the potential to fulfill this vision. With more data and longer duration of follow-up, we will hopefully confirm that we have a durable therapy that may transform the lives of many patients.

CLIMB-111 Trial in TDT: Updated Results A total of 13 patients with TDT have been dosed with CTX001, including eight additional patients since the last update in June 2020.

The seven patients with TDT reported at ASH are patients who had reached at least three months of follow-up after CTX001 dosing and therefore could be assessed for initial safety and efficacy. All seven patients showed a similar pattern of response, with rapid and sustained increases in total hemoglobin, fetal hemoglobin and transfusion independence at last analysis.

All seven patients were transfusion independent with follow-up ranging from three to 18 months after CTX001 infusion, with normal to near normal total hemoglobin levels at last visit, including total hemoglobin from 9.7 to 14.1 g/dL and fetal hemoglobin from 40.9% to 97.7%.

Bone marrow allelic editing data collected from four patients with six months of follow-up and from one patient with 12 months of follow-up after CTX001 infusion demonstrated a durable effect.

The safety data from all seven patients were generally consistent with an autologous stem cell transplant and myeloablative conditioning. There were four serious adverse events (SAEs) considered related or possibly related to CTX001 reported in one patient: headache, hemophagocytic lymphohistiocytosis (HLH), acute respiratory distress syndrome and idiopathic pneumonia syndrome. All four SAEs occurred in the context of HLH and have resolved. The majority of non-serious adverse events were considered mild to moderate.

CLIMB-121 Trial in Severe SCD: Updated Results A total of six patients with SCD have been dosed with CTX001, including four additional patients since the last update in June 2020.

The three patients reported at ASH are patients who had reached at least three months of follow-up after CTX001 dosing and therefore could be assessed for initial safety and efficacy. All three patients showed a similar pattern of response, with rapid and sustained increases in total hemoglobin and fetal hemoglobin, as well as elimination of VOCs through last analysis.

All three patients remained VOC-free with follow-up ranging from three to 15 months after CTX001 infusion and had hemoglobin levels in the normal to near normal range at last visit, including total hemoglobin from 11.5 to 13.2 g/dL and fetal hemoglobin levels from 31.3% to 48.0%.

Bone marrow allelic editing data collected from one patient with six months of follow-up and from one patient with 12 months of follow-up after CTX001 infusion demonstrated a durable effect.

The safety data from all three patients were generally consistent with an autologous stem cell transplant and myeloablative conditioning. There were no SAEs considered related to CTX001, and the majority of non-serious adverse events were considered mild to moderate.

About CTX001 CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-111 The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121 The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These Trials Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex Collaboration CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR Therapeutics CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics various clinical programs including CTX001; (ii) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that are being presented in this press release, at the annual ASH Meeting and Exposition, and in the NEJM article; (iii) the expected benefits of CRISPR Therapeutics collaborations; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain, are neither promises nor guarantees and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: that preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of final or future trial results; that CTX001 clinical trial results may not be favorable or may not support registration or further development; potential impacts due to the coronavirus pandemic, such as to the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR THERAPEUTICS word mark and design logo and CTX001 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Samarth Kulkarni, Dr. Reshma Kewalramani and Dr. Haydar Frangoul in this press release and statements regarding the expectations and plans to present data at the annual ASH Meeting and Exposition, the development, including expected timeline for development, updated data on patients treated to date and new data on additional patients, and the potential benefits and curative therapy of CTX001, our plans and expectations for our clinical trials and clinical trial sites, including statements regarding patient enrollment, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy, or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials and clinical trial sites and the clinical trials and clinical trial sites of our collaborators, including patient enrollment, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact: Susan Kim, +1 617-307-7503 susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact: Rachel Eides WCG on behalf of CRISPR +1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals Incorporated Investors: Michael Partridge, +1 617-341-6108 or Zach Barber, +1 617-341-6470 or Brenda Eustace, +1 617-341-6187

Media: mediainfo@vrtx.com or U.S.: +1 617-341-6992 or Heather Nichols: +1 617-839-3607 or International: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001 at American Society of Hematology Annual...

Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease – Albany Times Union

Mo Ebrahimkhani,University of Pittsburgh

Dec. 7, 2020Updated: Dec. 7, 2020 2:02p.m.

(The Conversation is an independent and nonprofit source of news, analysis and commentary from academic experts.)

(THE CONVERSATION) Takeaways

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Scientists have made progress growing human liver in the lab.

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The challenge has been to direct stems cells to grow into a mature, functioning adult organ.

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This study shows that stem cells can be programmed, using genetic engineering, to grow from immature cells into mature tissue.

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When a tiny lab-grown liver was transplanted into mice with liver disease, it extended the lives of the sick animals.

Imagine if researchers could program stem cells, which have the potential to grow into all cell types in the body, so that they could generate an entire human organ. This would allow scientists to manufacture tissues for testing drugs and reduce the demand for transplant organs by having new ones grown directly from a patients cells.

Im a researcher working in this new field called synthetic biology focused on creating new biological parts and redesigning existing biological systems. In a new paper, my colleagues and I showed progress in one of the key challenges with lab-grown organs figuring out the genes necessary to produce the variety of mature cells needed to construct a functioning liver.

Induced pluripotent stem cells, a subgroup of stem cells, are capable of producing cells that can build entire organs in the human body. But they can do this job only if they receive the right quantity of growth signals at the right time from their environment. If this happens, they eventually give rise to different cell types that can assemble and mature in the form of human organs and tissues.

The tissues researchers generate from pluripotent stem cells can provide a unique source for personalized medicine from transplantation to novel drug discovery.

But unfortunately, synthetic tissues from stem cells are not always suitable for transplant or drug testing because they contain unwanted cells from other tissues, or lack the tissue maturity and a complete network of blood vessels necessary for bringing oxygen and nutrients needed to nurture an organ. That is why having a framework to assess whether these lab-grown cells and tissues are doing their job, and how to make them more like human organs, is critical.

Inspired by this challenge, I was determined to establish a synthetic biology method to read and write, or program, tissue development. I am trying to do this using the genetic language of stem cells, similar to what is used by nature to form human organs.

Tissues and organs made by genetic designs

I am a researcher specializing in synthetic biology and biological engineering at the Pittsburgh Liver Research Center and McGowan Institute for Regenerative Medicine, where the goals are to use engineering approaches to analyze and build novel biological systems and solve human health problems. My lab combines synthetic biology and regenerative medicine in a new field that strives to replace, regrow or repair diseased organs or tissues.

I chose to focus on growing new human livers because this organ is vital for controlling most levels of chemicals like proteins or sugar in the blood. The liver also breaks down harmful chemicals and metabolizes many drugs in our body. But the liver tissue is also vulnerable and can be damaged and destroyed by many diseases, such as hepatitis or fatty liver disease. There is a shortage of donor organs, which limits liver transplantation.

To make synthetic organs and tissues, scientists need to be able to control stem cells so that they can form into different types of cells, such as liver cells and blood vessel cells. The goal is to mature these stem cells into miniorgans, or organoids, containing blood vessels and the correct adult cell types that would be found in a natural organ.

One way to orchestrate maturation of synthetic tissues is to determine the list of genes needed to induce a group of stem cells to grow, mature and evolve into a complete and functioning organ. To derive this list I worked with Patrick Cahan and Samira Kiani to first use computational analysis to identify genes involved in transforming a group of stem cells into a mature functioning liver. Then our team led by two of my students Jeremy Velazquez and Ryan LeGraw used genetic engineering to alter specific genes we had identified and used them to help build and mature human liver tissues from stem cells.

The tissue is grown from a layer of genetically engineered stem cells in a petri dish. The function of genetic programs together with nutrients is to orchestrate formation of liver organoids over the course of 15 to 17 days.

Liver in a dish

I and my colleagues first compared the active genes in fetal liver organoids we had grown in the lab with those in adult human livers using a computational analysis to get a list of genes needed for driving fetal liver organoids to mature into adult organs.

We then used genetic engineering to tweak genes and the resulting proteins that the stem cells needed to mature further toward an adult liver. In the course of about 17 days we generated tiny several millimeters in width but more mature liver tissues with a range of cells typically found in livers in the third trimester of human pregnancies.

Like a mature human liver, these synthetic livers were able to store, synthesize and metabolize nutrients. Though our lab-grown livers were small, we are hopeful that we can scale them up in the future. While they share many similar features with adult livers, they arent perfect and our team still has work to do. For example, we still need to improve the capacity of the liver tissue to metabolize a variety of drugs. We also need to make it safer and more efficacious for eventual application in humans.

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Our study demonstrates the ability of these lab livers to mature and develop a functional network of blood vessels in just two and a half weeks. We believe this approach can pave the path for the manufacture of other organs with vasculature via genetic programming.

The liver organoids provide several key features of an adult human liver such as production of key blood proteins and regulation of bile a chemical important for digestion of food.

When we implanted the lab-grown liver tissues into mice suffering from liver disease, it increased the life span. We named our organoids designer organoids, as they are generated via a genetic design.

This article is republished from The Conversation under a Creative Commons license. Read the original article here: https://theconversation.com/genetic-engineering-transformed-stem-cells-into-working-mini-livers-that-extended-the-life-of-mice-with-liver-disease-151089.

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Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease - Albany Times Union

Researchers Trace the Origin of Blood Cancer to Early Childhood, Decades before Diagnosis – PRNewswire

WASHINGTON, Dec. 8, 2020 /PRNewswire/ --Genetic mutations linked with cancer can occur during childhood or even before birth and proliferate in the body for many years before causing cancer symptoms, according to a new study. The study, which traced the genetic origins of a blood cancer in 10 individuals, suggests there may be untapped opportunities to detect cancer warning signs much earlier and potentially intervene to prevent or slow cancer development.

"Our preliminary findings show these cancer driver mutations were often acquired in childhood, many decades before the cancer diagnosis," said senior study authorJyoti Nangalia, MD,of the Wellcome Sanger Institute and University of Cambridge. "Our results finally answer the common question posed by patients, 'How long has this cancer been growing?' as we were able to study how these particular cancers developed over the entire lifetime of individual patients."

The researchers analyzed bone marrow and blood samples from 10 people with Philadelphia-negative myeloproliferative neoplasms, a type of cancer that causes stem cells in the bone marrow to produce too many blood cells. In the majority of patients, this cancer is driven by a genetic mutation called JAK2V617F. By assessing JAK2V617F, other cancer-linked mutations and hundreds of thousands of other mutations that a person naturally acquires throughout life, the researchers were able to trace the ancestry of different blood cells and estimate the time at which each patient acquired JAK2V617F and other important mutations.

They determined that, in these 10 patients, the first cancer-linked mutations emerged as early as a few weeks after the start of life and up to the first decade of childhood despite clinical disease presenting many decades later in life.

"We were not expecting this," said Dr. Nangalia. "In fact, in one patient, the JAK2 mutation was acquired more than 50 years before their diagnosis."

While it is often assumed that most cancers are diagnosed within a few years of their emergence, the findings point to a more gradual, lifelong process in which a single cell acquires a cancer-linked mutation early in life and then slowly grows over decades, ultimately leading to cancer.

"Some of these cancer-linked mutations are found in healthy individuals as we get older, suggesting that aging causes them," said Dr. Nangalia. "However, aging per se doesn't drive such growth it simply takes a long time for the clones to grow." Sometimes, the growing clones pick up additional cancer-linked mutations along the way, accelerating their growth, researchers found.

"For these patients, we calculated how many of these cancer clones would have been present in the past, and our results suggest that these clones may have been detectable up to 10 to 40 years before diagnosis," said Dr. Nangalia. "In addition to detecting the mutations, the rate at which the mutated clones grew was also very important in determining whether, and when, cancer develops." The findings suggest that genetic testing could help identify people at risk for cancer much earlier than current methods allow, according to researchers.

The next steps would be to understand the factors that influence the different rates of cancer growth and determine whether there could be ways to intervene and slow the growth of cells with cancer-linked mutations. The researchers say their method for pinpointing the origin of this blood cancer could also be applied to other mutations and other blood cancers. "Understanding the timelines of development of different cancers is critical for efforts aimed at early cancer detection and prevention," said Dr. Nangalia.

Jyoti Nangalia, MBBChir,Wellcome Sanger Institute and University of Cambridge, will present this study during the Late-Breaking Abstracts session on Tuesday, December 8 at 7:00 a.m. Pacific time on the ASH annual meeting virtual platform.

For the complete annual meeting program and abstracts, visit http://www.hematology.org/annual-meeting. Follow ASH and #ASH20 on Twitter, Instagram, LinkedIn, and Facebook for the most up-to-date information about the 2020 ASH Annual Meeting.

The American Society of Hematology (ASH) (www.hematology.org) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open-access journal.

SOURCE American Society of Hematology

http://www.hematology.org

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Researchers Trace the Origin of Blood Cancer to Early Childhood, Decades before Diagnosis - PRNewswire

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Jasper Therapeutics Announces Data from First Transplant-naive Patient in Phase 1 Clinical Trial of JSP191 as Conditioning Agent in Patients with SCID…

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced clinical data from its ongoing multicenter Phase 1 clinical trial of JSP191, a first-in-class anti-CD117 monoclonal antibody, in patients with severe combined immune deficiency (SCID). The trial is evaluating JSP191 as a conditioning agent to enable stem cell transplantation in patients with SCID who are either transplant-naive or who received a prior stem cell transplant with a poor outcome.

Data from the first transplant-nave SCID patient in the Phase 1 trial, a 6-month-old infant, showed that a single dose of JSP191 administered prior to stem cell transplant was effective in establishing sustained donor chimerism followed by development of B, T and NK immune cells. No treatment-related adverse events were reported. The data were presented by primary investigator Rajni Agrawal-Hashmi, M.D., of Stanford University, at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition.

We have previously shown that JSP191 can be successfully used as a single conditioning agent in SCID patients who had failed a previous transplant, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. This new data presented at ASH 2020 showing success in an infant with SCID undergoing a first transplant provides proof of concept of the safety and efficacy of the use of JSP191 as an alternative to genotoxic chemotherapies currently used to deplete stem cells prior to transplant.

Hematopoietic cell transplantation offers the only curative therapy for SCID, a severe genetic immune disorder that leaves patients without a functioning immune system. With this approach, standard-of-care chemotherapeutic conditioning regimens are given prior to transplant to reduce the number of blood stem cells in the bone marrow to make space for donor blood stem cells to engraft and cure the patient. JSP191 is designed to replace the need for chemotherapeutic conditioning agents, which are DNA-damaging and highly toxic.

Dr. Heller added, With our Phase 1 trials in SCID and hematologic disorders underway, we are planning to expand the development of JSP191 into additional indications, such as gene therapies, autoimmune diseases, Fanconis anemia and other rare disorders that can be cured by stem cell transplant.

The open-label, multicenter Phase 1 study is evaluating the safety, tolerability and efficacy of JSP191 as a conditioning agent in patients with SCID undergoing first or repeat hematopoietic cell transplantation. Up to three different doses of JSP191 are being assessed for dose-limiting toxicities. The trial is currently open for enrollment at Stanford University, the University of California, San Francisco, Memorial Sloan Kettering Cancer Center, the University of California, Los Angeles, and Cincinnati Childrens Hospital. Additional clinical trial sites in the United States will initiate enrollment in the coming weeks.

About SCID

Severe combined immune deficiency (SCID) is a group of rare disorders caused by mutations in genes involved in the development and function of infection-fighting immune cells. Infants with SCID appear healthy at birth but are highly susceptible to severe infections. The condition is fatal, usually within the first year or two of life, unless infants receive immune-restoring treatments, such as transplants of blood-forming stem cells, gene therapy or enzyme therapy.

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. JSP191 is also being evaluated in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191). Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

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Jasper Therapeutics Announces Data from First Transplant-naive Patient in Phase 1 Clinical Trial of JSP191 as Conditioning Agent in Patients with SCID...

After boosting gene therapy focus, Bayer signs up to new cell therapy pact with Atara – FierceBiotech

German pharma Bayer is moving into cell therapy R&D with its latest pact focusing on tough-to-treat lung cancers with Atara Biotherapeutics.

Bayer has been moving deeply into gene therapies over the past few years, but it's now entering the CAR-T research space in its Atara deal. CAR-T has been focused heavily on blood cancers, where there have been some amazing successes (but also serious safety worries), but this pact is focusing on a tougher area for cell therapy, namely solid tumors.

The deal focuses on off-the-shelf T-cell immunotherapy ATA3271 for high mesothelin-expressing tumors, with a focus on more rare and tough-to-treat high mesothelin-expressing tumors such as malignant pleural mesothelioma and non-small cell lung cancer.

At Parexel, we get the right people to you, so you can get the right treatment to your patients. Our functional service provider (FSP) outsourcing has evolved with biopharmas changing needs, so sponsors can efficiently manage operations through access to experts, resource flexibility, and reduced labor costs. Our latest whitepaper discusses the current FSP models and provides expert tips for choosing the right one.

The financials break down like this: Atara gets $60 million upfront, with up to $610 million on the table in biobucks. The biotech will lead IND-enabling studies and process development for ATA3271, while Bayer will be responsible for submitting the IND and then will take over R&D and sales work.

Atara will continue to be responsible for the ongoing ATA2271 phase 1 study, for which an IND filing has been accepted and a test already started.

RELATED: Bayer creates cell and gene therapy platform to support partners

For a limited time, Bayer also has a nonexclusive right to negotiate a license for additional Atara CAR-T product candidates.

This transaction is a fundamental element of Bayers new Cell & Gene Therapy strategy. It strengthens our development portfolio through allogeneic cell therapies and consolidates our emerging leadership in the field, said Wolfram Carius, head of Bayers Cell & Gene Therapy Unit.

We look forward to collaborating with Atara to develop off-the-shelf CAR T-cell therapies for patients with difficult-to-treat cancers.

Germanys Bayer has moved into cell and gene therapies on multiple fronts in recent years, buying up induced pluripotent stem cell specialist BlueRock Therapeutics and adeno-associated virus gene therapy player Asklepios BioPharmaceutical while investing in a clutch of other biotechs. The deals have given Bayer a pipeline of five advanced assets and more than 15 preclinical prospects.

It has backed other CAR-T players over the years, including CAR alternative player Triumvira and Century Therapeutics, but the Atara deal takes it further into directly working on a cell therapy.

This exciting collaboration between Atara and Bayer will accelerate the development of mesothelin-targeted CAR T-cell therapies for multiple solid tumors and helps us advance the power of our allogeneic cell therapy platform to patients as quickly as possible, added Pascal Touchon, president and CEO of Atara.

Bayers proven track record in oncology global development and commercialization, and growing presence in cell and gene therapy, enhances Ataras capabilities and complements our leading allogeneic T-cell platform.

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Data published in Cancer Research show Secarna Pharmaceuticals’ LNAplus(TM) ASOs targeting metadherin significantly slow progression and metastasis of…

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during the forecast period. The growth of this market is majorly driven by the growing awareness about the benefits of cell culture-based vaccines, increasing demand for monoclonal antibodies (mAbs), funding for cell-based research, growing preference for single-use technologies, and the launch of advanced cell culture products.New York, Dec. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell Culture Market by Product, Application, End User - Global Forecast to 2025" - https://www.reportlinker.com/p03665912/?utm_source=GNW On the other hand, the high cost of cell biology research and the lack of proper infrastructure for cell-based research activities are the major factors restraining this markets growth.Based on product, the consumables segment holds the largest market share during the forecast period.Based on product, the cell culture market is segmented into equipment and consumables.The consumables segment accounted for the largest market share in 2019 and is expected to register the highest CAGR during the forecast period.The dominant share and high growth of the consumables segment can be attributed to the repeated purchase of consumables and increased funding for cell-based research.Based onapplication, thebiopharmaceutical productionsegment is expected to register the highest CAGR during the forecast period.Based on application, the cell culture market is categorized into biopharmaceutical production, stem cell research, diagnostics, drug screening & development, tissue engineering and regenerative medicine, and other applications. The biopharmaceutical production application segment is expected to register the highest CAGR during the forecast period.The high growth of this segment is attributed to the commercial expansion of major pharmaceutical companies, growing regulatory approvals for the production of cell culture-based vaccines, and the increasing demand for monoclonal antibodies (mAbs).Pharmaceutical &Biotechnology companies end user segment is expected to grow at the highest CAGR in the cell culture market during the forecast periodBased on end users, the cell culture market is segmented into pharmaceutical & biotechnology companies, academic & research institutes, hospitals and diagnostic centers, and cell banks. The pharmaceutical & biotechnology companies segment is expected to witness the highest growth during the forecast period. The growing use of single-use technologies, the increasing number of regulatory approvals for cell culture-based vaccines, and the presence of a large number of pharmaceutical players in this market are some of the factors driving the cell culture market for this end user segmentNorth America is expected to account for the largest share of the cell culture market in 2019In 2019, North America accounted for the largest share of the cell culture market, followed by Europe, the Asia Pacific, Latin America, and the Middle East & Africa. The large share of this market segment can be attributed to thegrowing regulatory approvals for cell culture-based vaccines, technological advancements, and growth in the biotechnology & pharmaceutical industries in the region are the key factors driving the growth of the cell culture market in North AmericaBreak of primary participants was as mentioned below: By Company Type Tier 136%, Tier 245%, and Tier 319% By Designation C-level33%, Director-level40%, Others27% By Region North America36%, Europe28%, AsiaPacific19%, Latin America-9%, Middle East and Africa8%Key players in the cell culturemarketThe key players operating in the cell culturemarketincludeThermo Fisher Scientific (US), Merck KGaA (Germany), GE Healthcare (US), Danaher Corporation (US), Lonza Group AG (Switzerland), Becton, Dickinson and Company (US), Corning Incorporated (US), Eppendorf (Germany), HiMedia Laboratories (India), Sartorius AG (Germany), PromoCell GmbH (Germany), FUJIFILM Irvine Scientific (US), InvivoGen (US), CellGenix GmbH (Germany), SeraCare Life Sciences Incorporation (US), Miltenyi Biotec (Germany), STEMCELL Technologies, Inc. (Canada), Solida Biotech GmBH (Germany), Caisson Labs Inc. (US), Cellexus Ltd, (UK), PIERRE GURIN (France), Applikon Biotechnology BV (Netherlands), WISENT Inc. (Canada), Koh Jin-Bio Co., Ltd. (Japan), PAN-Biotech GmbH (Germany), and Infors AG (Switzerland).Research Coverage:The report analyzes the cell culturemarketand aims at estimating the market size and future growth potential of this market based on various segments such as product, application, end user, andregion.The report also includes aproduct portfolio matrix of various cell cultureproductsavailable in the market.The report also providesa competitive analysis of the key players in this market, along with their company profiles, product offerings, and key market strategies.Reasons to Buy the ReportThe report will enrich established firms as well as new entrants/smaller firms to gauge the pulse of the market, which in turn would helpthem, garner a more significant share of the market. Firms purchasing the report could use one or any combination of the below-mentioned strategies tostrengthen their position in the market.This report provides insights into the following pointers: Market Penetration: Comprehensive information on product portfolios offered by the top players in the global cell culturemarket. The report analyzes this marketby product, application, and end user Product Enhancement/Innovation: Detailed insights on upcoming trends and productlaunches in the global cell culturemarket Market Development: Comprehensive information on the lucrative emerging markets by product, application, and end user Market Diversification: Exhaustive information about new products or product enhancements, growing geographies, recent developments, and investments in theglobal cell culturemarket Competitive Assessment: In-depth assessment of market shares, growth strategies, product offerings, competitive leadership mapping, and capabilities of leading players in theglobal cell culturemarket.Read the full report: https://www.reportlinker.com/p03665912/?utm_source=GNWAbout ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.__________________________ CONTACT: Clare: clare@reportlinker.com US: (339)-368-6001 Intl: +1 339-368-6001

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Data published in Cancer Research show Secarna Pharmaceuticals' LNAplus(TM) ASOs targeting metadherin significantly slow progression and metastasis of...

Europe Tissue Engineering Market Forecast to 2027 – COVID-19 Impact and Regional Analysis by Material Type, Applications, and Country -…

DUBLIN--(BUSINESS WIRE)--The "Europe Tissue Engineering Market Forecast to 2027 - COVID-19 Impact and Regional Analysis by Material Type, Applications, and Country" report has been added to ResearchAndMarkets.com's offering.

The Europe tissue engineering market is expected to reach US$ 7,368.93 million by 2027 from US$ 2,798.86 million in 2019; it is estimated to grow at a CAGR of 13.2% during 2020-2027.

The market growth is primarily attributed to the increasing incidences of chronic diseases, road accidents, and trauma injuries, and technological advancements in 3D tissue engineering techniques. High cost associated to the tissue engineering process is one of the major factors restraining the growth of the market. Additionally, increasing financial contributions by government and private sector are likely to fuel the growth of the Europe tissue engineering market during the forecast period.

Tissue engineering is a blend of material methods and cellular activities. This approach involves the use of physicochemical and biochemical attributes of humans to replace the biological tissues and strengthen them. It is an innovative technology that works either separately or in conjunction with scaffolds, stem cells, regenerative medicine, and growth factors or negotiators. The process utilizes molecular and cellular processes in combination with the principles of material engineering to surgically repair and restore tissue.

The tissue engineering market in Europe is estimated to grow at a significant CAGR during the forecast period, and the growth is driven by the increase in research activities, growing demand for organ transplants, escalating number of initiatives by market players for expanding their presence in the region, and higher adoption of stem cell research in several European countries.

In the Europe, due to an increasing number of COVID-19 patients, healthcare professionals and leading organizations are rechanneling the flow of healthcare resources from R&D to primary care, which is slowing down the process of innovation. Further, the pandemic is also hindering the conduct of clinical trials and drug development, and the operations of diagnostic industry in Europe.

For instance, Stryker Corporation, a well-known player in the tissue engineering industry, has diverted operations to manufacture COVID-19 diagnostics and PPE kits. Moreover, according to a recent survey published by Medscape in July 2020, substantial disruption has been witnessed in routine research activities that include tissue engineering and regenerative medicines as a result of the COVID-19 pandemic. The rapid increase in the number of the infected patients in the Italy and Spain is likely to result in the slowdown of the market growth in the near future.

In 2019, the biologically derived material segment accounted for the largest share of the Europe tissue engineering market. The growth of the market for this segment is attributed to the rising adoption of biomaterials due to their natural regenerative potential to restore tissue functioning and ability to facilitate the on demand release of chemokines with the procedure. Further, the synthetic material segment is likely to register the highest CAGR in the market during the forecast period.

Key Topics Covered:

1. Introduction

1.1 Scope of the Study

1.2 Report Guidance

1.3 Market Segmentation

2. Europe Tissue engineering Market - Key Takeaways

3. Research Methodology

4. Europe Tissue engineering Market - Market Landscape

4.1 Overview

4.2 PEST Analysis

4.3 Expert Opinion

5. Europe Tissue engineering Market - Key Market Dynamics

5.1 Key Market Drivers

5.1.1 Increasing Number of Road Accidents and Trauma Injuries, and Elevating Incidence of Chronic Diseases

5.1.2 Technological Advancements in the Field of 3D Tissue engineering

5.1.3 Government and Private sector funding

5.2 Key Market Restraints

5.2.1 High Cost associated with tissue engineering

5.3 Impact Analysis

6. Tissue engineering Market - Europe Analysis

6.1 Europe Tissue engineering Market Revenue Forecasts and Analysis

7. Europe Tissue engineering Market Analysis - By Material Type

7.1 Overview

7.2 Europe Tissue engineering Market, By Material Type 2019-2027 (%)

7.2.1 Europe Tissue engineering Market Material Type Segment Revenue and Forecasts to 2027, By Material Type (US$ Mn)

7.3 Biologically Derived Material

7.4 Synthetic Material

7.5 Other

8. Europe Tissue engineering Market Analysis - By Application

8.1 Overview

8.2 Europe Tissue engineering Market, By Application 2019-2027 (%)

8.2.1 Europe Tissue engineering Market Revenue and Forecasts to 2027, By Application (US$ Mn)

8.3 Orthopedic, Musculoskeletal and Spine

8.3.1 Overview

8.3.2 Europe Orthopedic, Musculoskeletal and Spine Market Revenue and Forecasts to 2027 (US$ Mn)

8.4 Skin

8.5 Cardiology and Vascular

8.6 Neurology

8.7 Others

9. Europe Tissue engineering Market Revenue and Forecasts To 2027 - Regional Analysis

10. Impact of COVID-19 Pandemic on Europe Tissue Engineering Market

10.1 Europe: Impact Assessment of COVID-19 Pandemic

11. Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/ppygkp

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Allogene Therapeutics Presents Preclinical Data on ALLO-316 in Acute Myeloid Leukemia at the 62nd Meeting of the American Society of Hematology -…

December 06, 2020 10:00 ET | Source: Allogene Therapeutics, Inc.

SOUTH SAN FRANCISCO, Calif., Dec. 06, 2020 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, today announced preclinical findings of ALLO-316, an AlloCAR T therapy targeting CD70, in models of acute myeloid leukemia (AML). Data were presented in a poster session today at the 62nd Annual Meeting of the American Society of Hematology.

The Company also announced that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for a Phase 1 trial of ALLO-316 for patients with advanced or metastatic clear cell renal cell carcinoma (RCC). The Companys first solid tumor trial is expected to begin enrolling patients in 2021.

We are very excited about the potential of ALLO-316, our fourth AlloCAR T investigational therapy, to treat patients with CD70 expressing malignancies across both hematologic and solid tumor indications, said Rafael Amado, M.D., Executive Vice President of Research & Development and Chief Medical Officer of Allogene. These preclinical results in AML, coupled with previous findings of ALLO-316 in RCC presented at the American Association for Cancer Research Annual Meeting in 2019, reinforce our belief that CD70 may become one of the more important targets across a broad spectrum of cancers.

CD70 is expressed in a number of malignancies ranging from solid tumors such as RCC, lung cancer and glioblastoma to hematologic cancers including AML, diffuse large B-cell lymphoma, multiple myeloma, and chronic lymphocytic leukemia.

In the preclinical studies presented at ASH, CD70 expression was detected on AML cell lines and primary AML samples from patients. No expression of CD70 was identified in hematopoietic stem cells. ALLO-316 demonstrated the ability to mediate efficient killing of leukemic cells in multiple models. This killing activity was specific to CD70 expression on the target cells as ALLO-316 did not kill AML cell lines in which CD70 was knocked out. The preclinical studies also showed that ALLO-316 can mask CD70 on the surface of CAR T cells thereby preventing fratercide and allowing scaled manufacturing of AlloCAR T cells.

AboutAllogene Therapeutics Allogene Therapeutics, with headquarters in South San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T) therapies for cancer. Led by a management team with significant experience in cell therapy, Allogene is developing a pipeline of off-the-shelf CAR T cell therapy candidates with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients. For more information, please visit http://www.allogene.com, and follow @AllogeneTx on Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: timing and ability to progress a clinical trial of ALLO-316 in RCC; ability to manufacture ALLO-316; and the potential benefits of ALLO-316 and AlloCAR T therapy. Various factors may cause differences between Allogenes expectations and actual results as discussed in greater detail in Allogenes filings with the SEC, including without limitation in its Form 10-Q for the quarter ended September 30, 2020. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

AlloCAR T is a trademark ofAllogene Therapeutics, Inc.

ALLO-316 utilizes TALEN gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at CD70 and holds all global development and commercial rights for this investigational candidate.

Allogene Media/Investor Contact: Christine Cassiano Chief Communications Officer (714) 552-0326 Christine.Cassiano@allogene.com

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Allogene Therapeutics Presents Preclinical Data on ALLO-316 in Acute Myeloid Leukemia at the 62nd Meeting of the American Society of Hematology -...

Data Evaluating Tafasitamab with and without Lenalidomide in Combination with R-CHOP in Patients with DLBCL Presented at ASH 2020 – Business Wire

WILMINGTON, Del. & PLANEGG/MUNICH, Germany--(BUSINESS WIRE)--Incyte (Nasdaq:INCY) and MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ:MOR) announce that preliminary data from firstMIND, the ongoing Phase 1b, open-label, randomized study on the safety and efficacy of tafasitamab or tafasitamab plus lenalidomide in addition to R-CHOP for patients with newly diagnosed diffuse large B-cell lymphoma (DLBCL) were presented today during the 62nd American Society of Hematology Annual Meeting & Exposition (ASH). Additionally, a long-term subgroup analysis of the L-MIND study investigating tafasitamab combined with lenalidomide in patients with relapsed or refractory DLBCL was also presented at ASH.

The preliminary results of firstMIND indicate that tafasitamab plus lenalidomide in addition to R-CHOP shows an acceptable tolerability profile. Toxicities appear to be similar to what is expected with R-CHOP alone or in combination with lenalidomide. Serious or severe neutropenia and thrombocytopenia events (grade 3 or higher) were more frequent in the tafasitamab plus lenalidomide arm. The incidence of febrile neutropenia was comparable between both arms and the average relative dose intensity of R-CHOP was maintained in both arms. Interim response assessments after three cycles were available for 45 patients. In both arms combined, 41/45 (91.1%) of patients had an objective response as per Lugano 20141.

The preliminary data from this ongoing study in first-line DLBCL warrant further investigation. To that end, MorphoSys and Incyte plan to initiate frontMIND, a Phase 3 trial evaluating tafasitamab plus lenalidomide in combination with R-CHOP compared to R-CHOP alone as first-line treatment for patients with newly diagnosed DLBCL.

The initial results of the firstMIND study, shared today at ASH, as well as the long-term analyses from L-MIND, underscore the potential of tafasitamab as a combination therapeutic for patients with DLBCL, where there remains a significant unmet need. Along with our partners at MorphoSys, we are pleased to be moving forward with the initiation of a Phase 3 study in 2021, said Steven Stein, M.D., Chief Medical Officer at Incyte.

The preliminary firstMIND study results mark another important step as we explore the potential of tafasitamab as a backbone therapy, said Dr. Malte Peters, Chief Research and Development Officer at MorphoSys. Given the data available to date, including data from the L-MIND study, we believe that the mechanism of action, efficacy and safety profile of tafasitamab have the potential to make it a preferred combination partner as we seek to transform the standard of care in DLBCL. We are committed to developing innovative therapies to battle this aggressive disease for the benefit of patients with DLBCL, and look forward to beginning the planned frontMIND in the first half of 2021.

In addition to the firstMIND data presented today, the long-term L-MIND analyses showed that treatment with tafasitamab plus lenalidomide resulted in durable responses after 2 years of follow-up. At the time of analysis, patients with complete responses (CR) continued to experience durable treatment responses, including long duration of response (DoR) and overall survival (OS). The data also showed that tafasitamab plus lenalidomide taken for 12 cycles, followed by tafasitamab until progression, did not result in any unexpected safety signals2.

In July 2020, the FDA approved Monjuvi (tafasitamab-cxix), a humanized Fc-modified cytolytic CD19-targeting monoclonal antibody, in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s)3.

The FDA decision represented the first approval of a second-line treatment for adult patients with DLBCL who progressed during or after first-line therapy.

About Diffuse Large B-cell Lymphoma (DLBCL)

DLBCL is the most common type of non-Hodgkin lymphoma in adults worldwide4, characterized by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs. It is an aggressive disease with about one in three patients not responding to initial therapy or relapsing thereafter5. In the United States each year, approximately 10,000 patients are diagnosed with relapsed or refractory DLBCL who are not eligible for autologous stem cell transplant (ASCT)6,7,8.

About firstMIND

The firstMIND (NCT04134936) trial is a Phase 1b, randomized study of tafasitamab + R-CHOP (Arm A) or tafasitamab + lenalidomide + R-CHOP (Arm B) in patients with newly diagnosed diffuse large B-cell lymphoma (DLBCL). The study includes a safety run-in phase and a main phase. In the safety run-in phase, 24 patients were enrolled. The primary objective is to assess safety; secondary objectives include objective response rate, PET negative complete response (PET-CR) rate at end of treatment, progression-free survival, event-free survival, long-term safety, pharmacokinetics and immunogenicity of tafasitamab.

About Tafasitamab

Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi is a registered trademark of MorphoSys AG.

XmAb is a registered trademark of Xencor, Inc.

Important Safety Information

What are the possible side effects of MONJUVI?

MONJUVI may cause serious side effects, including:

The most common side effects of MONJUVI include:

These are not all the possible side effects of MONJUVI.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before you receive MONJUVI, tell your healthcare provider about all your medical conditions, including if you:

You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation.

Tell your healthcare provider about all the medications you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Please see the full Prescribing Information for Monjuvi, including Patient Information, for additional Important Safety Information.

About Incyte

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

About MorphoSys

MorphoSys (FSE & NASDAQ: MOR) is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of exceptional, innovative therapies for patients suffering from serious diseases. The focus is on cancer. Based on its leading expertise in antibody, protein and peptide technologies, MorphoSys, together with its partners, has developed and contributed to the development of more than 100 product candidates, of which 27 are currently in clinical development. In 2017, Tremfya, developed by Janssen Research & Development, LLC and marketed by Janssen Biotech, Inc., for the treatment of plaque psoriasis, became the first drug based on MorphoSys antibody technology to receive regulatory approval. In July 2020, the U.S. Food and Drug Administration (FDA) granted accelerated approval of MorphoSys proprietary product Monjuvi (tafasitamab-cxix) in combination with lenalidomide in patients with a certain type of lymphoma.

Headquartered near Munich, Germany, the MorphoSys group, including the fully owned U.S. subsidiary MorphoSys US Inc., has ~500 employees. More information at http://www.morphosys.com or http://www.morphosys-us.com.

Monjuvi is a registered trademark of MorphoSys AG.

Tremfya is a registered trademark of Janssen Biotech, Inc.

Incyte Forward-Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release - including statements about: plans to initiate frontMIND, a Phase 3 trial evaluating tafasitamab plus lenalidomide in combination with R-CHOP compared to R-CHOP alone as first-line treatment for patients with newly diagnosed DLBC; whether the mechanism of action, efficacy and safety profile of tafasitamab have the potential to make it a preferred or ideal combination partner in the treatment of DLBCL and, whether it will change or become the standard of care for the treatment of DLBCL; whether and when, if ever, confirmatory trials of tafasitamab will result in the conditional FDA approval of tafasitamab in the conditionally approved indication described above becoming a final approval; whether and when, if ever, the EMA will approve the filed MAA for tafasitamab; and additional development of tafasitamab, including in B-cell malignancies - contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on the Incytes current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA or the EMA; clinical and commercial supply of products in development or being commercialized; Incytes dependence on its relationships with its collaboration partners; the efficacy or safety of Incytes products and the products of its collaboration partners; the acceptance of Incytes products and the products of its collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in Incytes reports filed with the Securities and Exchange Commission, including its quarterly report on Form 10-Q for the quarter ended September 30, 2020. Incyte disclaims any intent or obligation to update these forward-looking statements.

MorphoSys Forward-Looking Statements

This communication contains certain forward-looking statements concerning the MorphoSys group of companies, including the expectations regarding Monjuvis ability to treat patients with relapsed or refractory diffuse large B-cell lymphoma, the further clinical development of tafasitamab-cxix, including ongoing confirmatory trials, additional interactions with regulatory authorities and expectations regarding future regulatory filings and possible additional approvals for tafasitamab-cxix as well as the commercial performance of Monjuvi. The words anticipate, believe, estimate, expect, intend, may, plan, predict, project, would, could, potential, possible, hope and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The forward-looking statements contained herein represent the judgment of MorphoSys as of the date of this release and involve known and unknown risks and uncertainties, which might cause the actual results, financial condition and liquidity, performance or achievements of MorphoSys, or industry results, to be materially different from any historic or future results, financial conditions and liquidity, performance or achievements expressed or implied by such forward-looking statements. In addition, even if MorphoSys' results, performance, financial condition and liquidity, and the development of the industry in which it operates are consistent with such forward-looking statements, they may not be predictive of results or developments in future periods. Among the factors that may result in differences are MorphoSys' expectations regarding risks and uncertainties related to the impact of the COVID-19 pandemic to MorphoSys business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products, the global collaboration and license agreement for tafasitamab, the further clinical development of tafasitamab, including ongoing confirmatory trials, and MorphoSys ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials, additional interactions with regulatory authorities and expectations regarding future regulatory filings and possible additional approvals for tafasitamab-cxix as well as the commercial performance of Monjuvi, MorphoSys' reliance on collaborations with third parties, estimating the commercial potential of its development programs and other risks indicated in the risk factors included in MorphoSys Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. MorphoSys expressly disclaims any obligation to update any such forward-looking statements in this document to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements, unless specifically required by law or regulation.

1 Belada D, M.D., Ph.D., et al. A Phase 1b, Open-label, Randomized Study to Assess Safety and Preliminary Efficacy of Tafasitamab (MOR208) or Tafasitamab + Lenalidomide in Addition to R-CHOP in Patients with Newly Diagnosed Diffuse Large B-Cell Lymphoma: Analysis of the Safety Run-In Phase. 62nd American Society of Hematology Annual Meeting & Exposition (ASH). Abstract #3028.

2 Maddocks KJ, M.D., et al. Long-Term Subgroup Analyses from L-MIND, a Phase 2 Study of Tafasitamab (MOR208) Combined with Lenalidomide in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma. 62nd American Society of Hematology Annual Meeting & Exposition (ASH). Abstract #3021.

3 Monjuvi (tafasitamab-cxix) Prescribing Information. Boston, MA, MorphoSys.

4 Sarkozy C, et al. Management of relapsed/refractory DLBCL. Best Practice Research & Clinical Haematology. 2018 31:20916. doi.org/10.1016/j.beha.2018.07.014.

5 Skrabek P, et al. Emerging therapies for the treatment of relapsed or refractory diffuse large B cell lymphoma. Current Oncology. 2019 26(4): 253265. doi.org/10.3747/co.26.5421.

6 DRG Epidemiology data.

7 Kantar Market Research (TPP testing 2018).

8 Friedberg, Jonathan W. Relapsed/Refractory Diffuse Large B-Cell Lymphoma. Hematology Am Soc Hematol Educ Program 2011; 2011:498-505. doi: 10.1182/asheducation-2011.1.498.

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Data Evaluating Tafasitamab with and without Lenalidomide in Combination with R-CHOP in Patients with DLBCL Presented at ASH 2020 - Business Wire