On May 24, 2022 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported positive topline results from the DeFi trial, a double-blind, placebo-controlled Phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with progressing desmoid tumors (Press release, SpringWorks Therapeutics, MAY 24, 2022, View Source [SID1234614978]).

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The DeFi trial met its primary endpoint of improving progression-free survival (PFS), demonstrating a statistically significant improvement for nirogacestat over placebo, with a 71% reduction in the risk of disease progression (hazard ratio (HR) = 0.29 (95% CI: 0.15, 0.55); p < 0.001). In addition, the trial met all key secondary endpoints, with nirogacestat demonstrating statistically significant improvements as compared to placebo in objective response rate (ORR) and patient-reported outcomes (PROs). Nirogacestat was generally well tolerated with a manageable safety profile. The majority of women of childbearing potential had adverse events consistent with ovarian dysfunction. Other adverse events were generally consistent with previously reported data.

Additional data are expected to be presented at an upcoming medical conference in the second half of 2022 and SpringWorks plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2022.

"Desmoid tumors are aggressive soft-tissue tumors that can lead to severe negative outcomes for patients, including long-lasting pain, disfigurement, and amputation. In rare cases, when vital organs are impacted, desmoid tumors can also be life-threatening," said Saqib Islam, Chief Executive Officer of SpringWorks. "Today’s announcement represents a significant milestone towards our goal of bringing the first approved therapy to the desmoid tumor community. We look forward to sharing the DeFi trial data with the FDA and to presenting detailed study results at a medical meeting later this year."

Nirogacestat has received Orphan Drug Designation from the FDA for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

About the DeFi Trial

DeFi (NCT03785964) is an ongoing, global, randomized (1:1), double-blind, placebo-controlled Phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The study randomized 142 patients to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by >20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to the first dose of study treatment. The primary endpoint is progression-free survival, as assessed by blinded independent central review. Secondary and exploratory endpoints include safety and tolerability measures, objective response rate (ORR), duration of response, changes in tumor volume assessed by magnetic resonance imaging (MRI), and changes in patient-reported outcomes (PROs).

About Desmoid Tumors

Desmoid tumors are rare, aggressive, locally invasive, and potentially morbid tumors of the soft tissues.1,2 While they do not metastasize, desmoid tumors are associated with a high rate of recurrence.2,3,4 Sometimes referred to as aggressive fibromatosis, or desmoid fibromatosis, these soft tissue tumors can be serious, debilitating, and, in rare cases when vital organs are impacted, they can be life-threatening.2,5

Desmoid tumors are most commonly diagnosed in patients between the ages of 20 to 44 years, with a two-to-three times higher prevalence in females.4,6,7 It is estimated that there are 1,000-1,650 new cases diagnosed per year in the United States.7,8

Historically, desmoid tumors were treated with surgical resection, but this approach has become less favored due to a high recurrence rate after surgery.1,4,9 There are currently no FDA-approved therapies for the treatment of desmoid tumors.

About Nirogacestat

Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

In addition, gamma secretase has been shown to directly cleave membrane-bound B cell maturation antigen (BCMA), resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat’s ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has eight collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies, three bispecific antibodies and a monoclonal antibody. SpringWorks has also formed research collaborations with Fred Hutchinson Cancer Research Center and Dana-Farber Cancer Institute to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA-directed therapies using a variety of preclinical multiple myeloma models.

Conference Call Details

SpringWorks will host a conference call and webcast today, Tuesday, May 24, 2022, at 8:30 a.m. Eastern Time to discuss the DeFi trial data. Participants can listen to the call by dialing +1 (844) 946-0285 (domestic) or +1 (602) 585-9676 (international) and providing the conference ID 2268304. A live webcast presentation can be accessed through the Investors & Media section of the Company’s website at View Source A replay of the webcast will be available on the SpringWorks website for a limited time following the event.

On May 24, 2022 Genexine (KOSDAQ: 095700) a publicly traded, clinical stage biopharmaceutical company committed to the discovery and development of novel biologics for the treatment of unmet medical needs, reported the appointment of Neil Warma as its President and Chief Executive Officer (Press release, Genexine, MAY 24, 2022, View Source [SID1234614998]). Mr. Warma who has over 25 years of experience as a global entrepreneur, company builder and successful CEO will lead Genexine onto the world stage with a focus on advancing Genexine’s key products to market, expanding its pipeline and focusing on global drug development.

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Mr. Warma has been a successful healthcare entrepreneur for over 25 years having led and managed numerous biotechnology and pharmaceutical companies across the globe. From 2019-2022, Mr. Warma was the General Manager of I-Mab Biopharma U.S., (Nasdaq:IMAB) with offices in Shanghai, Beijing, Hangzhou, Guangzhou, Lishui and Hong Kong in China, and Maryland and San Diego, in the U.S., where he was a member of the executive team responsible for the Company’s expansion onto the global stage and for establishing I-Mab’s state-of-the-art research facilities in San Diego, California. From 2008-2017, Mr. Warma was President, CEO and Director of Opexa Therapeutics, Inc. (Nasdaq:OPXA), a publicly traded biopharmaceutical company developing novel cellular therapies for the treatment of autoimmune diseases, where he orchestrated global deals with Novartis and Merck Serono. From 2004-2007, he was President, CEO and Director of Viron Therapeutics, a private biotechnology company developing novel protein-based therapeutics for cardiovascular disease and transplantation. In 2000, Mr. Warma co-founded and later sold MedExact, a health-technology company dedicated to providing an interface between physicians and pharmaceutical companies. From 1992-2000, Mr. Warma held several key senior management roles at Novartis Pharmaceuticals at its corporate headquarters in Basel, Switzerland, in Pharma Policy and Global Marketing. Mr. Warma obtained an honors Bachelor of Science degree specializing in neuroscience from the University of Toronto and an International MBA from the Schulich School of Business at York University in Toronto. Mr. Warma currently serves on the Board of ProMIS Neurosciences (TSX:PMN) and the Biotechnology Innovation Organization (BIO). Mr. Warma has also been a member of the Board of Directors of Genexine since March 2021 and will continue to serve on the Board as an Executive Director.

"I am deeply honored to have been selected as Genexine’s new CEO by the Board of Directors," said Mr. Warma. "The Company has built a strong scientific foundation based on novel science and differentiated technology over several years. My focus will be to commercialize existing late stage products and further expand Genexine’s pipeline of life-saving products with a focus on first and best-in-class products. We will not only leverage our strength and position as a leading Korean biopharmaceutical company but will also expand our reach into the U.S. and Europe to increase our shareholder base and access new technology and top talent. Genexine has an exciting pipeline and with its global partners is conducting over 20 clinical trials, several in Phase 3 registrational trials. We expect to file several important BLAs in the next 1-3 years and commercialize our first products shortly thereafter."

"My goal is to introduce the biotech world to the top-class technology we have developed in Korea such that we can leverage our clinical data in the US and EU to reduce our development costs and speed the delivery of our products to the patient. We will also look to secure partnerships with global multinational pharmaceutical partners to share costs and leverage their substantial expertise. We are excited to be reporting some near term milestones coming up over the next several months with the planned announcement of Phase 1b/2 clinical data in our potential blockbuster product, GX-I7, the long-acting recombinant Interleukin-7 protein and Phase 2 data in our first-in-class DNA cancer vaccine being studied in cervical and head and neck cancers. These near term milestones should demonstrate our ability to execute and advance our products towards the market and, ultimately, to the patient," added Mr. Warma.

On May 24, 2022 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme") reported the successful completion of its acquisition of Antares Pharma, Inc. ("Antares") (Press release, Halozyme, MAY 24, 2022, View Source [SID1234614979]).

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The tender offer for all of the outstanding shares of Antares common stock expired as scheduled at one minute after 11:59 p.m., Eastern Time, on Monday, May 23, 2022. The minimum tender condition and all of the other conditions to the offer have been satisfied and on May 24, 2022, Halozyme accepted for payment and will promptly pay for all shares validly tendered and not validly withdrawn.

Following its acceptance of the tendered shares, Halozyme completed its acquisition of Antares through the merger of a wholly owned subsidiary of Halozyme with and into Antares, pursuant to Section 251(h) of the General Corporation Law of the State of Delaware, with Antares continuing as the surviving corporation and becoming a wholly owned subsidiary of Halozyme.

In connection with the merger, all Antares shares not validly tendered in the tender offer have been converted into the right to receive the same $5.60 per share in cash, without interest thereon and net of any applicable withholding taxes, that would have been paid had such shares been validly tendered in the tender offer. As of May 24, 2022, Antares common stock will cease to be traded on the NASDAQ Global Select Stock Market.

BofA Securities, Inc. and Wells Fargo Securities LLC acted as financial advisors to Halozyme and Weil, Gotshal & Manges LLP acted as legal advisor. Jefferies LLC acted as financial advisor to Antares and Skadden, Arps, Slate, Meagher & Flom LLP acted as legal advisor.

On May 24, 2022 Genexine (KOSDAQ: 095700), a publicly traded, clinical stage biopharmaceutical company committed to the discovery and development of novel biologics for the treatment of unmet medical needs, reported that Neil Warma, CEO of Genexine will be presenting virtually at the H.C. Wainwright Global Investment Conference being held on May 23-26, 2022 (Press release, Genexine, MAY 24, 2022, View Source [SID1234614999]).

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The presentation will provide an overview of Genexine’s pipeline, business operations and corporate achievements, as well as discuss upcoming milestones with the Company’s lead assets, including GX-I7 (a first-in-class long-acting recombinant interleukin 7, in development for TNBC and GBM) and GX-188 (a first-in-class DNA vaccine, in development for cervical and head and neck cancers).

Details of the presentation are as follows:

Event: H.C. Wainwright Global Investment Conference
Date & Time: On-demand, beginning 7.00 a.m. ET, Tuesday May 24, 2022
Webcast Link: Genexine_Wainwright_investor_conf.

A replay of the presentation will be available for 90 days at the link above or by visiting www.genexine.com. Registered investors will be able to schedule a one-on-one meeting via the conference portal.

On May 24, 2022 MaaT Pharma (EURONEXT: MAAT – the "Company"), a French clinical-stage biotech and a pioneer in the development of Microbiome Ecosystem Therapies dedicated to improving survival outcomes for patients with cancer reported that it will host its first R&D Day for analysts and investors to be held virtually on Tuesday, June 7th, 2022 from 4:00 pm CEST (10:00 am EST) to 6:00 pm CEST (12:00 pm EST) (Press release, MaaT Pharma, MAY 24, 2022, View Source [SID1234614981]).

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MaaT Pharma’s speakers will discuss the microbiome potential in cancer therapy and the latest clinical results of MaaT013 and MaaT033. They will also present the Company’s innovative proprietary drug discovery platform, the next generation of products MaaT03X and the construction of Europe’s largest cGMP manufacturing facility for Microbiome Ecosystem Therapies.

In addition to the presentations by MaaT Pharma’s senior team, the R&D Day will feature talks from world renowned scientists and physicians including:

Joël Doré – Research Director, Director of INRAE and Scientific Advisor at MaaT Pharma, France
Ernst Holler, M.D. – Senior Professor on Clinical and Experimental Allo-HSCT, Department of Internal Medicine, University Hospital Center Regensburg, Germany
Florent Malard, M.D. – Professor of Hematology, Saint Antoine Hospital (AP-HP) and Sorbonne University
Mohamad Mohty, M.D. – Professor, Sorbonne University and Head of the Clinical Hematology and Cellular Therapy Department, Saint-Antoine Hospital (AP-HP), France
Hassane Zarour, M.D. – Professor of Medicine, Immunology and Dermatology, University of Pittsburg, James and Frances McGlothlin Chair in Melanoma Immunotherapy Research
"MaaT Pharma’s inaugural R&D Day is an opportunity to bring together world-renowned scientists and our talented internal team to share MaaT Pharma’s innovative research and highlight our unique positioning in the microbiome space," said Hervé Affagard, Chief Executive Officer and Co-Founder of MaaT Pharma. "Since our successful IPO in November 2021, we have delivered on the key milestones we had defined and we continue to progress, with the ambition to leverage the microbiome for the benefit of millions of patients fighting cancer."

A question-and-answer session will follow the presentations. Please note that all the presentations are in English with French subtitles. The webcast will be made available on the Company’s website after the event.