On September 26, 2023 ABM Therapeutics reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the investigation of ABM-1310 for the treatment of Glioblastoma (GBM) patients carrying BRAF V600E mutation, following the Orphan Drug Designation for ABM-1310 to treat malignant gliomas including GBM received in July (Press release, ABM Therapeutics, SEP 26, 2023, View Source [SID1234635427]).

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The FDA grants Fast Track Designation to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need. Fast Track Designation is intended to bring promising medicines to patients sooner.

ABM-1310 is an investigational therapeutic candidate currently undergoing clinical development for BRAF V600E mutant solid tumors. The Fast Track Designation paves the potential path of ABM-1310 to have a significant impact on patients with BRAF V600E mutant Glioblastoma. The Fast Track Designation also reinforces ABM’s commitment to advancing this target therapy for Glioblastoma patients. ABM is ready to work closely with the FDA to expedite its development.

"We are very grateful to the FDA for recognizing the potential of our novel next-generation investigational drug ABM-1310 to help patients with brain tumors," said Zane Yang, M.D., CMO of ABM Therapeutics. "This offers ABM an interactive collaboration with the FDA to ensure ABM-1310 clinical development expeditiously with the highest standards of safety and quality."

ABM Therapeutics acknowledges the contributions of our team, partners, and investors who have played a vital role in reaching this milestone. Our mission remains to focus on developing innovative therapies for patients.

On September 26, 2023 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to IDEAYA’s development program investigating IDE161, a potent and selective inhibitor of poly (ADP-ribose) glycohydrolase (PARG), for the treatment of adult patients having advanced or metastatic ovarian cancer with germline or somatic BRCA 1/2 mutations who are platinum resistant and have received prior antiangiogenic and poly (ADP-ribose) polymerase (PARP) inhibitor therapies (Press release, Ideaya Biosciences, SEP 26, 2023, View Source [SID1234635426]).

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"We are extremely pleased to receive the U.S. FDA Fast Track designation for IDE161 based on the FDA’s review of preclinical and emerging clinical efficacy and tolerability data. We recently reported preliminary clinical proof-of-concept with expansion into priority HRD+ solid tumor indications in our Phase 1 clinical trial. The Fast Track designation has been provided for platinum-resistant BRCA1/2 mutant advanced or metastatic ovarian cancer, which represents a serious condition, and acknowledges the potential for IDE161 to treat this indication," said Dr. Darrin Beaupre, Chief Medical Officer at IDEAYA Biosciences.

Fast Track is a U.S. FDA process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Under the Fast Track designation, the IDE161 development program in BRCA1/2m ovarian cancer, as specified in the Fast Track designation, is eligible for various expedited regulatory review processes, including generally more frequent FDA interactions (e.g., meetings, written communications), potential eligibility for rolling review of a New Drug Application (NDA) and potential accelerated approval and priority review of an NDA.

IDEAYA’s Phase 1 first-in-human clinical trial is evaluating the safety, tolerability, pharmacokinetic and pharmacodynamic properties and preliminary efficacy of IDE161 in patients having solid tumors with homologous recombination deficiency (HRD). Early clinical data from the dose escalation cohorts showed preliminary tumor shrinkage in multiple patients having solid tumors with HRD, including a BRCA 1/2m endometrial cancer subject. These data supported expansion into priority tumor indications in parallel with continuing evaluation of the optimal move-forward dose for Phase 2 expansion.

The expansion portion of the Phase 1 trial will include patients having HRD+ associated breast cancer and ovarian cancer, as well as a basket of other selected solid tumors. The breast cancer focus is on estrogen receptor positive (ER+), human epidermal growth factor receptor 2 negative (Her2-) tumors with HRD, which represent approximately 10% to 14% of breast cancer patients. Ovarian cancer tumors with HRD represent approximately 50% of ovarian cancer patients.

IDEAYA owns or controls all commercial rights in IDE161, subject to certain economic obligations under its exclusive, worldwide license with Cancer Research UK and University of Manchester.

On September 26, 2023 Kyverna Therapeutics ("Kyverna"), a clinical-stage cell therapy company with the mission of engineering a new class of therapies for serious autoimmune diseases and ElevateBio, LLC ("ElevateBio"), a technology-driven company focused on powering transformative cell and gene therapies, reported a partnership to advance process development and manufacturing to produce industry-leading Ingenui-T-derived chimeric antigen receptor (CAR) T-cell therapies (Press release, ElevateBio, SEP 26, 2023, View Source [SID1234635425]). Ingenui-T is an optimized autologous manufacturing platform developed by Kyverna to specifically address the needs of patients with autoimmune diseases, enabling a lower cost of goods and an improved patient experience.

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ElevateBio and Kyverna will implement Kyverna’s Ingenui-T platform into their process development and cell product manufacturing efforts at ElevateBio BaseCamp. BaseCamp is ElevateBio’s end-to-end genetic medicine current Good Manufacturing Practice (cGMP) manufacturing and process development business with capabilities for research, clinical, and commercial cell and gene therapies.

Building and improving on the learnings from CAR T-cell therapy in oncology, Kyverna is seeking to revolutionize the way patients with autoimmune diseases are treated. "We are excited by the potential to not only provide dramatic benefit to patients by eliminating the underlying disease pathology using CAR T-cell therapy, but also to bring innovation to other aspects of the patient journey," said Karen Walker, chief technology officer at Kyverna. "Together with ElevateBio, we aim to deliver radical benefits to patients in less time, with lower impact, and at substantially reduced cost."

"At ElevateBio, we have combined multiple next-generation technology platforms with industry-leading expertise to transform the current cell and gene therapy development paradigm," said Michael Paglia, chief operating officer at ElevateBio BaseCamp. "We are delighted to build a long-term relationship with Kyverna to optimize manufacturing processes and accelerate the development of their therapies."

About KYV-101
KYV-101 is an autologous version of a novel, fully human clinical-stage anti-CD19 chimeric antigen receptor (CAR) T-cell construct with properties well suited for use in B cell-driven autoimmune diseases, such as lupus nephritis and other B-cell driven autoimmune diseases. In a 20-patient Phase 1/2 study in oncology, expected anti-lymphoma activity was associated with a significant reduction of cytokines released that translated into a strong reduction of cytokine-driven side effects, such as the rate of immune effector cells-associated neurotoxicity syndrome (ICANS)1. The fully human anti-CD19 CAR also translated into reduced immunogenicity that favorably impacted cell persistence at one month. Kyverna recognized that these properties singled out KYV-101 as a product ideally poised for use in autoimmune disease patients, and the company obtained exclusive, worldwide licenses from the National Institutes of Health (NIH) to use this CD19 construct in both autologous and allogeneic CAR T-cell therapies.

About Ingenui-T
Ingenui-T is Kyverna’s proprietary cost-efficient autologous CAR T-cell therapy manufacturing process, designed specifically to meet the needs of autoimmune disease patients. The process incorporates components to enhance the patient experience, to accelerate product availability, as well as to reduce cost of goods associated with personalized CAR T-cell therapy manufacturing.

On September 26, 2023 Cantex Pharmaceuticals, Inc., a clinical stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed, and Allegheny Health Network (AHN), an academic healthcare system serving the diverse greater western Pennsylvania community that provides pioneering medical research and education programs focused on transforming healthcare, reported the initiation of a Phase 1/2 study to assess the safety and efficacy of azeliragon in patients refractory to first-line treatment of metastatic pancreatic cancer (Press release, Cantex, SEP 26, 2023, View Source [SID1234635424]).

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"We are extremely pleased to announce the initiation and patient enrollment at Allegheny Health Network, a world-class provider of cancer treatment, of this important Phase 1/2 clinical study to evaluate the safety and efficacy of azeliragon in patients refractory to first-line treatment of metastatic pancreatic cancer," said Stephen G. Marcus, M.D., Cantex’s Chief Executive Officer.

"Azeliragon inhibits activation of a receptor known as ‘RAGE’ on the surface of cancer and cells in the tumor microenvironment. RAGE has been implicated in the growth and spread of pancreatic and other cancers, their resistance to chemotherapy, and complications stemming from pancreatic and other cancers and related treatments. With this study, we seek to slow the growth and spread of pancreatic cancer while alleviating some of its disabling effects," concluded Dr. Marcus.

"Through the initiation of this Phase 1/2 study at AHN, we seek to explore the safety and efficacy of azeliragon in pancreatic cancer patients who are no longer responding to other treatments. Targeting RAGE with azeliragon, a once-daily oral medication well tolerated in previous studies, greatly merits a clinical trial in this setting," said Nathan Bahary, M.D., Academic Chief of Medical Oncology at Allegheny Health Network Cancer Institute. Amongst his various appointments, Dr. Bahary is a member of the National Cancer Institute (NCI) Pancreatic Task Force, and the NCI’s Eastern Cooperative Oncology Group (ECOG) where he serves on the ECOG GI Steering Committee.

About Azeliragon
Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer’s disease by vTv Therapeutics Inc. (NASDAQ: VTVT) from whom Cantex licensed worldwide rights to azeliragon. Clinical safety data from these trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated.

Cantex also has ongoing Phase 2 clinical trials in newly diagnosed glioblastoma, neoadjuvant therapy of breast cancer, brain metastases in combination with stereotactic radiosurgery, and an ongoing Phase 2/3 clinical trial in hospitalized COVID-19 patients to prevent acute kidney injury. These trials are based on azeliragon’s robust pre-clinical data as well as its extensive clinical safety information from randomized placebo-controlled clinical trials.

On September 26, 2023 SK Biopharmaceuticals, a global biotech focused on the research, development and commercialization of treatments for disorders of the central nervous system (CNS) and oncology, and its U.S. R&D subsidiary, Proteovant Therapeutics, reported that they will present data on the discovery and characterization of a selective IKZF2 molecular glue degrader with best-in-class potential, and on MOPED, a novel platform for the discovery of molecular glues, at the 20th Annual Discovery on Target Conference being held in Boston, September 25-28, 2023 (Press release, SK biopharmaceuticals, SEP 26, 2023, https://www.prnewswire.com/news-releases/sk-biopharmaceuticals-proteovant-therapeutics-presents-preclinical-data-on-ikzf2-protein-degrader-program-and-moped-molecular-glue-screening-platform-at-the-20th-annual-discovery-on-target-conference-301938441.html [SID1234635422]).

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These presentations are the first in a series in which the companies will unveil exciting results from its targeted protein degradation (TPD) studies and MOPEDTM Molecular Glue Screening Platform. TPD harnesses the body’s natural protein disposal system and offers the potential to develop new medicines targeting historically difficult-to-drug proteins that play an important role in causing serious diseases. SK Biopharmaceuticals and Proteovant Therapeutics are discovering and developing potential best-in-class and first-in-class degraders to engage previously undruggable targets.

"We are excited to share new data on our IKZF2 molecular glue degrader that demonstrates the potential to combine with immune checkpoint therapies for the treatment of cancer," said Donghoon Lee, President and CEO of SK Biopharmaceuticals and SK Life Science. "We are also pleased to present data showing how our MOPED platform can help find molecular glues with the potential to destroy malfunctioning proteins and kill cancer cells or inhibit their growth."

Discovery on Target Meeting Presentations:

Title: MOPED: A Novel Platform for the Discovery of Molecular Glues
Presenter: Corey Strickland, Ph.D.
Date/Time: Wednesday, September 27; 9:30 – 10:00 AM
Title: Discovery and Characterization of an IKZF2 Selective Molecular Glue Degrader with Best-in-Class Potential
Presenter: Courtney G. Havens, Ph.D.
Date/Time: Thursday, September 28; 2:50 – 3:20 PM