On September 30, 2021 Merck, a leading science and technology company, reported a mutual decision with GSK to terminate their agreement on bintrafusp alfa, effective September 30, 2021 (Press release, Merck & Co, SEP 30, 2021, View Source [SID1234590583]).

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The decision is based on the clinical trial data generated to date, most notably the previously reported results from the INTR@PID Lung 037 study, which did not replicate the encouraging data observed in earlier studies. Based on the data generated during the agreement, no milestone payments were made by GSK and no future milestone obligations remain.

The INTR@PID clinical program sought to validate the potential of the novel mechanism of simultaneously blocking TGF-ß and PD-L1, with the ambition to improve outcomes for patients with difficult-to-treat cancers. Given the extent of the clinical program, Merck will deepen its scientific leadership in the field and interrogate the data leveraging the power of advanced analytics. The important insights this program has yielded about the biology of TGF-β will inform the collective understanding of this pathway.

Merck is a science-led organization dedicated to delivering transformative medicines with the goal of making a meaningful difference in the lives of people affected by cancer. Our oncology research efforts, comprised of 10 ongoing development programs, aim to leverage our synergistic portfolio in oncogenic pathways, immuno-oncology, and DNA Damage Response (DDR) to tackle challenging tumor types in gastrointestinal, genitourinary, and head and neck and thoracic cancers. Learn more at View Source

All Merck Press Releases are distributed by e-mail at the same time they become available on the Merck Website. Please go to www.merckgroup.com/subscribe to register online, change your selection or discontinue this service.

On September 30, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that Manuel Litchman, M.D., President and Chief Executive Officer, and members of the Mustang Leadership Team will participate in a fireside chat at Chardan’s Virtual 5th Annual Genetic Medicines Conference, taking place on Tuesday, October 5, 2021, at 8:00 a.m. ET (Press release, Mustang Bio, SEP 30, 2021, View Source [SID1234590581]). The company will also participate in one-on-one meetings during the conference. A live webcast of the company’s chat will be available on the Events page of the Investor Relations section of Mustang’s website, www.mustangbio.com, for approximately 30 days after the meeting.

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On September 30, 2021 Reata Pharmaceuticals, Inc. (Nasdaq: RETA) ("Reata," the "Company," or "we"), a clinical-stage biopharmaceutical company, reported that it has completed its pre-New Drug Application ("NDA") meeting with the United States Food and Drug Administration ("FDA") for omaveloxolone for the treatment of patients with Friedreich’s ataxia and reaffirmed its plan to submit an NDA in the first quarter of 2022 (Press release, Reata Pharmaceuticals, SEP 30, 2021, View Source [SID1234590580]).

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The purpose of the pre-NDA meeting was to discuss the content of Reata’s planned NDA submission. We plan to submit the NDA seeking standard approval for omaveloxolone for the treatment of Friedrich’s ataxia. We are not planning to conduct a second pre-approval clinical study prior to the submission. The FDA indicated that the appropriate approval pathway would be a matter of review after submission of the NDA. In response to our questions about the contents of the filing and because of the seriousness of the indication, the FDA exercised its discretion subject to review to permit us to submit the results of certain nonclinical and clinical studies after approval.

"We are pleased with the outcome of our recent pre-NDA meeting and that we have a path to submit our NDA in the first quarter of 2022," said Warren Huff, Reata’s President and Chief Executive Officer. "Friedreich’s ataxia is a severe, ultra-rare disease that affects approximately 5,000 patients in the United States. We remain committed to our goal of working with the FDA to secure regulatory approval for omaveloxolone as quickly as possible for patients with this devastating disease."

"Omaveloxolone could be the first drug approved for the treatment of Friedreich’s ataxia—actually the first drug approved for any ataxia," said Dr. Susan Perlman, MD, Professor, Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA. "The MOXIe Part 2 study with omaveloxolone is the first to demonstrate a significant improvement in neurological function in patients with Friedreich’s ataxia. While not a cure, if approved, Friedreich’s ataxia would finally become a treatable disease, something the Friedreich’s ataxia community has been working towards for a long time."

About Friedreich’s Ataxia

Friedreich’s ataxia is a rare, inherited, life-shortening, debilitating, and degenerative neuromuscular disorder, which is normally diagnosed during adolescence. Friedreich’s ataxia is caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin. Pathogenic repeat expansions can lead to impaired transcription and reduced frataxin expression, which can result in mitochondrial iron overload and poor cellular iron regulation, increased sensitivity to oxidative stress, and impaired mitochondrial ATP production. Patients with Friedreich’s ataxia experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly resulting in motor incapacitation with patients requiring a wheelchair by their teens or early 20s. Patients with Friedreich’s ataxia may also experience visual impairment, hearing loss, diabetes, and cardiomyopathy. Based on literature and proprietary research, we believe Friedreich’s ataxia affects approximately 5,000 children and adults in the United States and 22,000 individuals globally. There are currently no approved therapies for the treatment of patients with Friedreich’s ataxia.

About Omaveloxolone

Omaveloxolone is an investigational, oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA has granted Orphan Drug designation to omaveloxolone for the treatment of Friedreich’s ataxia. The European Commission has granted Orphan Drug designation in Europe to omaveloxolone for the treatment of Friedreich’s ataxia.

On September 30, 2021 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), a company developing new drugs for the treatment of cancer and fibrosis, reported that publication of a key paper from the Garvan Institute of Medical Research ("Garvan") (Press release, Amplia Therapeutics, SEP 30, 2021, View Source;[email protected] [SID1234590579]). The paper, which describes the fundamental biology underpinning Amplia’s planned Phase 2 clinical trial in pancreatic cancer patients, further highlightsthe potential benefits of using a focal adhesion kinase (FAK) inhibitor prior to administration of standard chemotherapy.

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Entitled "Intravital imaging technology guides FAK-mediated priming in pancreatic cancer precision medicine according to Merlin status" the paper has been published in the high impact peer-reviewed journal Science Advances. 1 Professor Paul Timpson, a leading researcher in FAK biology at Garvan and a member of Amplia’s Scientific Advisory Board, led the research program which has shown that in mice that have been implanted with human pancreatic cancer tissue, pre-treatment with a FAK inhibitor (‘priming’) increased the responsiveness of the cancer to subsequently administered gemcitabine/Abraxane chemotherapy. Furthermore, FAK-priming reduced the metastatic spread of tumour cells to secondary sites such as the liver.

"There have been several publications over the last two years that have highlighted the potential of FAK inhibitors in pancreatic cancer, including their ability to work synergistically with chemotherapy agents" said John Lambert, CEO of Amplia: "This latest study from our collaborators at the Garvan Institute is particularly exciting as its replicates the approach that we are taking to treat first line pancreatic cancer patients in our recently announced Phase 2 clinical trial. We believe that making an established standard of care, namely chemotherapy with gemcitabine/Abraxane, more effective offers a very promising approach for improving the outcomes for these patients".

On September 30, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported two poster presentations at upcoming scientific conferences scheduled to take place in October 2021 (Press release, Phio Pharmaceuticals, SEP 30, 2021, View Source [SID1234590578]). These presentations include an abstract being presented by the Phio team at the AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), which is being held from October 7-10, 2021; and a poster presentation of data from a study using INTASYL by the Company’s collaborators at Helmholtz Zentrum München (HMGU) at the 8th Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference (ITOC8), which is being held from Oct. 8–9, 2021.

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Details are as follows:

Event: AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper)
Title: Targeting BRD4 in T cells with self-delivering RNAi PH-894 for immunotherapy
Authors: Melissa Maxwell, et al.
Date: October 7-10, 2021

An archived version of the Phio presentation will be made available on the "Investors – Events and Presentations" section of the Company’s website (click here).

Event: 8th Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference (ITOC8)
Title: Targeting Diaclyglycerol Kinase alpha and zeta by self-delivering RNAi to optimize T lymphocytes for adoptive therapy of solid tumors
Authors: Anna Herbstritt, et al.
Abstract Number: P01.03
Date: October 8-9, 2021