Valemetostat New Drug Application Submitted in Japan for Treatment of Patients with Adult T-Cell Leukemia/Lymphoma

Daiichi Sankyo | December 29, 2021

Daiichi Sankyo Company, Limited announced that it has submitted a New Drug Application to Japan’s Ministry of Health, Labour and Welfare for valemetostat, a potential first-in-class dual inhibitor of EZH1 and EZH2, for the treatment of patients with relapsed/refractory adult T-cell leukemia/lymphoma.

ATL is a rare and aggressive type of peripheral T-cell lymphoma that occurs with greater frequency in parts of Japan and other regions.1,2 Patients with ATL face a poor prognosis with current therapies.3 Nearly 90% of patients relapse after completing intensive first-line treatment, at which point there are few options available.1,4

The Japan NDA submission of valemetostat is based on pivotal phase 2 study results in Japanese patients with three aggressive subtypes of relapsed/refractory ATL, recently presented at the 2021 American Society of Hematology (ASH) Annual Meeting. Valemetostat previously received Orphan Drug designation from the Japan MHLW for treatment of patients with relapsed/refractory ATL.

Valemetostat would potentially be the first dual inhibitor of EZH1 and EZH2 to be approved anywhere in the world and could provide a new type of targeted therapy option for patients with relapsed/refractory ATL, which represents one of the most significant unmet medical needs in Japan. Valemetostat is the fifth innovative oncology medicine from our pipeline to be submitted for regulatory approval in Japan in the past three years.”

 Wataru Takasaki, PhD, Executive Officer, Head of R&D Division in Japan, Daiichi Sankyo

About Adult T-Cell Leukemia/Lymphoma
Adult T-cell leukemia/lymphoma is a rare and aggressive type of peripheral T-cell lymphoma that is caused by human T-cell lymphotropic virus type 1.1 More than 3,000 new cases of ATL are diagnosed each year worldwide.5 ATL occurs with greater frequency in regions where the HTLV-1 virus is endemic including southwest Japan, Central and South America and central Australia.3 Cases are also observed in North America and Europe, and incidence of ATL is rising in non-endemic areas.3 In Japan, there are approximately 1,000 new ATL cases and over 1,000 deaths due to ATL annually.6

ATL has the poorest prognosis compared to other types of PTCL, with a five-year overall survival rate of about 14%.7 A median survival time of approximately eight months (252 days) was reported for patients in Japan with the most common acute ATL subtype.5

Treatment of ATL is based on subtype and consists primarily of intensive multi-drug chemotherapy regimens.3 Nearly 90% of patients relapse after completing intensive first-line treatment, at which point there are few options available.1,4 Additional therapies are needed to improve the prognosis of ATL in Japan and worldwide.1,3

About Valemetostat
Valemetostat is a potential first-in-class dual inhibitor of EZH1 and EZH2 currently in clinical development in the Alpha portfolio of Daiichi Sankyo. A potent and selective small molecule inhibitor, valemetostat is designed to counter epigenetic dysregulation by targeting both the EZH1 and EZH2 enzymes.8

The valemetostat development program includes VALENTINE-PTCL01, a global pivotal phase 2 trial in patients with relapsed/refractory PTCL and ATL; a pivotal phase 2 trial in patients with relapsed or refractory ATL in Japan; and, a phase 1 study in patients with relapsed/refractory NHL in the U.S. and Japan. Valemetostat received ODD from the U.S. Food & Drug Administration for the treatment of PTCL in December 2021, ODD from the Japan MHLW for the treatment of relapsed/refractory ATL in November 2021 and SAKIGAKE Designation from the Japan MHLW for the treatment of adult patients with relapsed/refractory PTCL in April 2019.

Valemetostat is an investigational medicine that has not been approved for any indication in any country. Safety and efficacy have not been established.

About the Pivotal Phase 2 Study
The pivotal, open-label, multi-center, single-arm phase 2 study evaluated efficacy and safety of valemetostat as monotherapy in patients with relapsed/refractory ATL who were previously treated with mogamulizumab or at least one systemic chemotherapy in case of intolerance/ contraindication for mogamulizumab and with no history of allogenic hematopoietic stem cell transplant.

The primary endpoint is objective response rate assessed by independent efficacy assessment committee. Secondary endpoints include investigator-assessed ORR, best response in tumor lesions, complete remission rate, tumor control rate, time to response, duration of response, progression-free survival, overall survival and safety. A total of 25 patients were enrolled in the study in Japan. 

About Daiichi Sankyo Oncology
The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025.

About Daiichi Sankyo
Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.” 


The reason that large pharmaceutical companies have not been able to do what we’ve done is because they have adopted a very narrow approach to treating osteoarthritis (OA). Big Pharma has tried to imitate the successful discovery of a treatment for rheumatoid arthritis (RA) by looking for a single cause of OA.


The reason that large pharmaceutical companies have not been able to do what we’ve done is because they have adopted a very narrow approach to treating osteoarthritis (OA). Big Pharma has tried to imitate the successful discovery of a treatment for rheumatoid arthritis (RA) by looking for a single cause of OA.

Related News


WuXi ATU Announces Licensing Agreement with Janssen for TESSA™ Technology

WuXi AppTec | August 12, 2022

WuXi Advanced Therapies a wholly owned subsidiary of WuXi AppTec, announced a licensing agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson. Under this agreement, WuXi ATU will license to Janssen its TESSA™ technology, a high-performance system that can produce 10 times more adeno-associated viral vectors than traditional AAV manufacturing systems. Janssen will also have access to work on WuXi ATU's proprietary clonal suspension HEK293 cell line. This agreement was facilitated by Johnson & Johnson Innovation. AAV vectors are commonly used for the delivery of gene therapies to patients due to their ability to transduce numerous cell and tissue types. WuXi ATU's TESSA™ technology responds to industry demand for large scale AAV manufacturing by producing higher quality AAV particles more efficiently. WuXi ATU has also successfully scaled up the TESSA™ technology to 200L; this achieved a 10-fold higher yield and a significantly higher percentage of full AAV capsids, greatly reducing overall AAV production costs compared to traditional plasmid-based AAV production systems. "We are honored that Janssen selected WuXi ATU's TESSA™ technology. We remain committed to improving the TESSA™ platform to produce faster and more cost-effective AAV products for patients." Dr. David Chang, Chief Executive Officer of WuXi Advanced Therapies As a Contract Testing, Development and Manufacturing Organization with global operations, WuXi ATU will continue to enhance its capability and capacity to help customers develop and deliver life-changing cell and gene therapies faster for patients in need. About WuXi Advanced Therapies (WuXi ATU) As the advanced therapies business unit of WuXi AppTec, WuXi Advanced Therapies is a Contract Testing, Development and Manufacturing Organization (CTDMO) that offers integrated platforms to transform the discovery, development, testing, manufacturing, and commercialization of cell and gene therapies. Our services and solutions accelerate time to market and support customer programs around the world. About WuXi AppTec As a global company with operations across Asia, Europe, and North America, WuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable global pharmaceutical and healthcare industry to advance discoveries and deliver groundbreaking treatments to patients. Through its unique business models, WuXi AppTec's integrated, end-to-end services include chemistry drug CRDMO (Contract Research, Development and Manufacturing Organization), biology discovery, preclinical testing and clinical research services, cell and gene therapies CTDMO (Contract Testing, Development and Manufacturing Organization), helping customers improve the productivity of advancing healthcare products through cost-effective and efficient solutions. WuXi AppTec received AA ESG rating from MSCI in 2021 and its open-access platform is enabling more than 5,850 collaborators from over 30 countries to improve the health of those in need – and to realize the vision that "every drug can be made and every disease can be treated."

Read More


BlackRock and QLS Advisors Partner Through BlackRock Systematic to Invest in Biomedical Innovation

BlackRock and QLS Advisors | July 19, 2022

BlackRock and QLS Advisors LLC, a life sciences technology and advisory firm, have announced a partnership through BlackRock Systematic to develop data-driven investment solutions to help accelerate biomedical innovation. The partnership will leverage the firms’ combined Research and Development and investment experience, directing funding toward public and private companies that have the potential to create transformative therapies while seeking to earn attractive investment returns for clients. Biomedicine is at an inflection point: breakthrough therapies—with the potential to extend longevity and improve quality of life—are emerging at an ever-increasing pace. Funding these therapeutic development programs has the potential to deliver attractive investment returns, but as biomedicine has grown more complex, so have the risks to investors. QLS has developed proprietary technology for estimating the probability of success of clinical trials, a key driver of financial performance, and systematically incorporating these forecasts into fundamental valuation models and portfolio construction algorithms. Their approach applies machine learning techniques to a multitude of predictive factors including therapeutic characteristics, clinical trial design features, disease-specific characteristics, and drug company track records. This methodology, coupled with BlackRock Systematic’s global platform, proprietary analytics, and more than 35 years1 of data-driven investment experience, presents a differentiated approach to investing in life sciences companies. “BlackRock’s partnership with QLS applies sophisticated quantitative investment technologies to the intersection of finance, academia, and life sciences. QLS brings an industry-leading approach to investing in life sciences that complements the work that BlackRock Systematic is doing in our actively managed portfolios. We expect this will create compelling, differentiated solutions that can deliver attractive returns for our clients while providing funding for life-changing therapies.” Rich Kushel, Head of the Portfolio Management Group BlackRock Andrew Lo, QLS Co-founder and Professor of Finance at the MIT Sloan School of Management commented: “We founded QLS to improve the way financial resources are allocated to emerging therapies, with the goal of lowering the cost of capital in this important sector. With BlackRock, we gain a partner who is a recognized leader in data-driven investing and shares our passion for delivering investment returns with positive outcomes for society. We’re thrilled and honored to join forces with a firm that has such a strong and trusted global brand, broad network, and access to world-class investment resources.” BlackRock and QLS have also assembled a Scientific Advisory Council comprising renowned academics, medical doctors, and inventors. The Council will support sourcing, due diligence, and value creation through the members’ vast networks and decades of experience as industry leaders in their respective fields. The Council is expected to include Dr. Robert Langer, Institute Professor, MIT Department of Chemical Engineering, and founder of over 40 biotechnology companies including Moderna. Ms. Lita Nelsen, retired director of the MIT Technology Licensing Office; now consultant in technology licensing. Dr. Larry Norton, Senior Vice President, Norna S. Sarofim Chair in Clinical Oncology and Medical Director of the Evelyn H. Lauder Breast Center at Memorial Sloan Kettering Cancer Center, Past President of the American Society of Clinical Oncology, ASCO David Karnofsky Awardee, FASCO, FAACR. Dr. Richard H. Scheller, Chairman of Research and Development BridgeBio. Board of Directors BridgeBio, Alector, 23andMe, DICE and others. Former Executive Vice President Research and Early Development Genentech and former HHMI Investigator and Professor Stanford University Medical School. Dr. Phillip A. Sharp, Institute Professor, MIT Department of Biology, geneticist and molecular biologist who co-discovered RNA splicing, co-founded Biogen and Alnylam, and the recipient of the 1993 Nobel Prize in Physiology or Medicine. About BlackRock Systematic BlackRock’s systematic investment platform brings together BlackRock’s community of systematic investors across equity, fixed income, and factor-based strategies. Systematic portfolios combine cutting-edge technology, scientific research, and human insight in the relentless pursuit of investment performance. With over 35 years of experience and nearly 200 professionals across the globe managing over US$200 bn in client assets as of March 31, 2022, BlackRock Systematic partners with clients to deliver quantitative alpha-seeking and factor-based investment solutions for the whole portfolio. About QLS Advisors LLC QLS Advisors LLC is a life sciences technology and advisory company based in Cambridge, MA, dedicated to fostering biomedical innovation. QLS employs a unique blend of fundamental and quantitative tools to help clients manage risk, assess reward, and develop investment and financing strategies for portfolios of healthcare-related assets.

Read More


Cosán and Genomind Partner to Help Prevent Adverse Prescription Drug Events for Older Adults

Cosán and Genomind | July 20, 2022

Cosán, a provider of technology-driven preventative care services for older adults and the physicians that support them, and Genomind, a leading precision healthcare company, have partnered to fill an existing gap in aging adult care management by providing Cosán’s network of physicians with Genomind’s integrated precision medication management platform. The platform will provide insights that will help Cosán to achieve the best outcomes for their patient population, prevent adverse drug events and reduce avoidable costs of medication mismanagement. With over 40% of U.S. adults 65 years of age and older taking five or more prescription medicines, aging populations are at high-risk for adverse drug events. Through this partnership, Genomind’s analytic capabilities will provide Cosán with risk stratification insights, enabling Cosán clinicians to target individuals that are most likely to benefit from medication reviews and pharmacogenetictesting, also known as drug-gene testing. Genomind’s PGx test adds a layer of precision medicine that leverages genetics to help providers select appropriate medications and dosing. Equipped with advanced pharmacogenetic screening, clinical concierge services and access to expert pharmacists, Cosán’s care teams can then incorporate individualized insights into each patient’s chronic care management plan so medications are administered safely. “This partnership brings a new level of pharmacy and genetic expertise to the thousands of providers and the patients they support, marking yet another stride in the delivery of effective preventative care management services. These unique drug analytic and genetic testing capabilities provide Cosán’s network of physicians and care coordinators with another tool designed to help eliminate avoidable drug risks and improve overall care quality for vulnerable patients receiving care from their homes.” David Hunt, Founder & President at Cosán According to the CDC, adverse drug events cause approximately 1.3 million emergency department visits each year, and older adults are nearly seven times more likely to end up hospitalized after such events than younger persons. Many of these adverse events are caused by just a few drugs. This new platform will help physicians develop personalized therapeutic approaches for patients based on their individual genetic makeup and are designed to improve patient and physician satisfaction and reduce medical expenses. With the recent expansions by Medicare to cover PGx testing in certain circumstances, this population is particularly suited to dramatically benefit from this platform. “The prevalence and cost of medication mismanagement is unacceptable, costing thousands of lives and billions of dollars annually. We’re thrilled to join Cosán in helping to improve care management for older adults, who are at high risk for these adverse drug events,” said Cynthia Kilroy, Co-CEO, Genomind. About Cosán Group Cosán Group, established in 2015, is an industry-leading healthcare organization creating new pathways to modern aging with technology-driven preventative care services, offering concierge home care for older adults. Early market exposure in the delivery of technology and services to support the Chronic Care Management (CCM) program, and Behavioral Health Integration (BHI) programs with Remote Patient Monitoring (RPM) has propelled Cosán to deliver a practice and patient-centric approach to remote care coordination. About Genomind Genomind is a leading precision health company focused on optimizing the treatment experience for individuals and healthcare providers through actionable genetic insights and innovative health technology. Genomind’s platform integrates precision health technology and pharmacogenetic testing on an individual and population level beyond mental health. Built on the foundation of compassionate care and clinical expertise, Genomind is empowering a more targeted and personalized approach to medication management and supporting providers in a new standard of care.

Read More