PHARMA TECH

Saama Launches Active Safety Analytics for Pharma (ASAP), Co-built With Gilead to Transform Safety Surveillance

Saama Technologies, Inc. | October 27, 2020

Saama Technologies, Inc. (“Saama”), the AI clinical analytics platform company, announced today the launch of the new Active Safety Analytics for Pharma (ASAP) product. ASAP is the first validated pharmacovigilance solution to leverage the U.S. Food and Drug Administration’s (FDA) Sentinel Common Data Model and the TreeScan methodology for detecting safety signals. ASAP was co-built with Gilead Sciences, a research-based biopharmaceutical company that discovers, develops and commercializes innovative medicines in areas of unmet medical need. Saama and Gilead partnered to develop ASAP, with scientific advice from the Reagan-Udall Foundation for the FDA, and accelerate the life sciences industry’s paradigm shift to adopt active safety analytics.

Spotlight

As pharma companies expand, they are looking more and more to biologics for their next potential blockbusters. However, this class of product ranging from well-established large molecule drugs to truly novel therapies poses major challenges because of their scientific complexity and sophisticated development requirements. Furthermore, expanding the drug pipeline isn’t the only growth strategy most companies are pursuing: They are also planning to expand geographically and expect to face various risks doing so, including unfamiliar regulatory environments, shifts in pricing and customers’ ability to pay.

Spotlight

As pharma companies expand, they are looking more and more to biologics for their next potential blockbusters. However, this class of product ranging from well-established large molecule drugs to truly novel therapies poses major challenges because of their scientific complexity and sophisticated development requirements. Furthermore, expanding the drug pipeline isn’t the only growth strategy most companies are pursuing: They are also planning to expand geographically and expect to face various risks doing so, including unfamiliar regulatory environments, shifts in pricing and customers’ ability to pay.

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PHARMACY MARKET

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.”

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PHARMACY MARKET

Ascentage Pharma Announces its 9th Orphan Drug Designation from the US FDA in 2020

Ascentage Pharma | January 05, 2021

Ascentage Pharma (6855.HK), an internationally engaged, clinical-stage biotechnology organization occupied with creating novel treatments for malignancies, persistent hepatitis B (CHB), and age-related sicknesses, today reported that the US Food and Drug Administration (FDA) has allowed an Orphan Drug Designation (ODD) to the organization's novel Bcl-2 inhibitor, APG-2575, for the therapy of patients with intense myeloid leukemia (AML). This is the fourth ODD allowed to APG-2575 by the US FDA, following the past ODDs for the therapy of Waldenström macroglobulinemia (WM), ongoing lymphocytic leukemia (CLL), and numerous myeloma (MM). Until this point, four of Ascentage Pharma's investigational drug up-and-comers have been allowed an aggregate of nine ODDs by the US FDA, a record number for any Chinese biopharmaceutical organization. AML is an exceptionally heterogenous hematologic threat that is more normal in the old populace with a middle age at analysis of 68 years1. The latest information from the Surveillance, Epidemiology, and End Results Program (SEER) of the US National Cancer Institute (NCI) assessed 19,940 new instances of AML and 11,180 passings from this infection in the United States in 2020. Notwithstanding the critical advances in therapeutics as of late, the 5-year endurance pace of AML stays at 25%–30%, which actually speaks to a huge neglected clinical requirement for treatments with more solid viability and a superior wellbeing profile. The expression "vagrant medications" alludes to drug items produced for the counteraction, finding, and treatment of uncommon illnesses or conditions. In the United States, a vagrant sign is characterized as a sickness or condition with a pervasiveness of under 200,000 patients in the nation. Since the Orphan Drug Act was passed in 1983, the US government has given motivators and strategy backing to support advancement of vagrant medications. Treatments conceded ODDs by the FDA are equipped for different advancement motivators, including a tax break on consumptions brought about in clinical examinations, a waiver of the New Drug Application (NDA) charge, conceivable exploration award granted by the FDA, and above all, 7 years of US market restrictiveness upon endorsement.

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PHARMA TECH

Hologic Launches Panther Trax for High Volume Molecular Testing

Hologic | December 24, 2021

Hologicannounced that it has completed regulatory filings needed to make Panther Trax® available in a number of countries and regions. Launch of Panther Trax represents the latest addition to the Panther® Scalable Solutions portfolio of products, offering the ultimate in lab automation by physically linking multiple Panther instruments together into a single, powerful workcell that allows labs to increase testing volumes without increasing staff. Panther Trax is now listed as a Class I medical device with the United States Food and Drug Administration, CE-marked for diagnostic use in Europe, and will also be commercially available in Canada, Australia and New Zealand. “Clinical laboratories have performed heroically, meeting unprecedented demand for close to two years now due to the COVID-19 pandemic. It is the job of manufacturers like Hologic to continue to innovate and enable our lab partners to accomplish more with less, ensuring they manage their workloads while responding to further potential surges in COVID testing.” Kevin Thornal, president, Diagnostic Solutions Division at Hologic The Panther system, initially launched in Europe in 2010, offers random access and full automation for molecular testing. It provides a broad assay menu that includes tests for women’s health, sexually transmitted infections, respiratory health, viral load and COVID-19 testing, as well as Open Access™ functionality for laboratory developed tests. This broad menu enables labs to consolidate molecular testing onto a single platform. The Panther system serves as the foundation for optional add-ons including: Panther® Fusion, which launched in 2017 and provides additional IVD menu and the Open Access functionality to run LDTs; Panther® Plus, which gives labs greater flexibility, walk-away time and throughput; and Panther® Link, which virtually connects multiple instruments to share data and reagents while providing a centralized dashboard to monitor all instruments. These scalable solutions address the needs of today’s laboratories, allowing them to increase operational capacity and testing volumes at their own pace, while building on the flexibility, and streamlined user experience they require. With Panther Trax, labs can customize configuration to meet space constraints and minimize facility costs. Taken together, the suite of Panther Scalable Solutions offered by Hologic significantly expands upon the proven performance and reliability of the foundational Panther system. About the Panther System The Panther system for molecular diagnostics is a best-in-class fully automated, sample-to-result platform with adaptable workflow options and consolidated testing menu. The Panther Fusion system provides an expanded in-vitro diagnostics menu, as well as Open Access functionality to run LDTs. Hologic’s Panther and Panther Fusion systems now offer 19 FDA-cleared assays and up to 22 assays outside the U.S. depending on the region. This high-throughput molecular diagnostic platform combines comprehensive sexual health, cervical health, viral load, respiratory testing and open channel functionality on a fully automated system. About Hologic Hologic, Inc. is an innovative medical technology company primarily focused on improving women’s health and well-being through early detection and treatment.

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