Pharma Frontiers: Daily Digest of Global Pharmaceutical News – Oct 11

1.Bayer’s First-in-Class Therapy NDA Accepted by FDA

On October 9, Bayer announced that the U.S. FDA has accepted the New Drug Application (NDA) for its dual NK-1/NK-3 receptor antagonist, Elinzanetant, for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause, commonly known as hot flashes. This application is based on positive results from the Phase III OASIS 1, OASIS 2, and OASIS 3 studies, which evaluated the efficacy and safety of Elinzanetant compared to placebo for menopausal VMS. The results demonstrated that Elinzanetant (120 mg, oral, once daily) significantly reduced the frequency and severity of moderate to severe VMS and exhibited a favorable safety profile. Elinzanetant is the first dual NK-1 and NK-3 receptor antagonist, working by modulating a group of estrogen-sensitive neurons (KNDy neurons) in the hypothalamus of the brain. In addition to the OASIS program, Bayer is conducting the NIRVANA study (NCT06112756) to assess Elinzanetant's effectiveness in treating menopause-related sleep disturbances.

2.Kymera's Oral STAT6 Degrader Receives IND Approval from U.S. FDA

On October 9, Kymera Therapeutics announced that its Investigational New Drug (IND) application for KT-621 has been approved by the U.S. FDA. KT-621 is a potent and selective oral STAT6 degrader. The Phase I trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending doses of KT-621 compared to placebo. The company expects to initiate the Phase I clinical trial in healthy volunteers in October 2024, with data from the trial anticipated in the first half of 2025. KT-621 is a heterobifunctional degrader (PROTAC) targeting STAT6, being developed for conditions such as atopic dermatitis (AD), asthma, and chronic obstructive pulmonary disease (COPD). Preclinical studies have shown that KT-621 exhibits similar activity to dupilumab and demonstrates good tolerability in various preclinical models of TH2-driven diseases.

3.CARsgen Therapeutics Submits Request to FDA to Lift Clinical Hold on CAR-T Therapies

On October 9, CARsgen Therapeutics announced that its manufacturing facility in Durham, North Carolina, has successfully passed a reinspection by the U.S. FDA, with no issues identified and no Form 483 issued. On October 4, 2024, CARsgen submitted a complete response to the FDA requesting the lifting of the clinical hold on its CAR-T therapies: Zevorcabtagene Autoleucel Injection, Satricabtagene Autoleucel Injection, and CT071. The FDA is expected to provide an official response to the company’s application within 30 calendar days. In December 2023, CARsgen’s U.S. subsidiary, CARsgen Therapeutics Corporation, received notification from the FDA to pause clinical trials for its CAR-T cell therapy products, CT053, CT041, and CT071, until the FDA completed an inspection and review of the Durham facility.

4.Eli Lilly Enters New Collaboration with AI Drug Discovery Company

On October 9, AI-driven drug discovery company Insitro announced that it has signed three strategic agreements with Eli Lilly to jointly advance potential new treatments for metabolic diseases, including metabolic dysfunction-associated steatotic liver disease (MASLD). Insitro has developed two siRNA molecules aimed at treating diseases such as MASLD, but these siRNA molecules require delivery vehicles to reach the liver. Under the first two agreements, Lilly will provide Insitro with an option to license its GalNAc (N-acetylgalactosamine) delivery technology, which enables liver-targeted delivery. In the third agreement, Insitro and Lilly will collaborate to discover and develop an antibody targeting a third novel target for metabolic diseases. As part of this collaboration, the two companies will jointly conduct early preclinical development activities up to the point of drug candidate nomination, after which Insitro will be responsible for all remaining development and commercialization.

5.Johnson & Johnson Completes Acquisition of Atrial Shunt Manufacturer V-Wave

On October 9, Johnson & Johnson announced the completion of its acquisition of V-Wave, a manufacturer of atrial shunt devices. Under the terms of the agreement, Johnson & Johnson will make an upfront payment of $600 million to V-Wave. In addition to the upfront payment, J&J will pay potential regulatory and commercial milestone payments, bringing the total transaction value to approximately $1.7 billion. Founded in 2009, V-Wave focuses on developing percutaneous implant technologies for patients with chronic heart failure. Its core product, the Ventura Interatrial Shunt, aims to alleviate symptoms and improve quality of life in heart failure patients. The final acquisition agreement was reached on August 20.

6.$105 Million Series B Funding to Develop Gene Therapy for Kidney Diseases

On October 9, gene therapy company Purespring Therapeutics announced the completion of a £80 million ($105 million) Series B funding round. The oversubscribed round was led by Sofinnova Partners, with participation from Gilde Healthcare, Forbion, British Patient Capital, and founding investor Syncona Limited. Purespring, based on the work of Professor Moin Saleem, a pediatric nephrology expert at the University of Bristol, is the first company to successfully treat kidney disease models by directly targeting podocytes, a type of cell implicated in about 60% of kidney diseases, using its proprietary adeno-associated virus (AAV) gene therapy platform. The company is currently developing a range of projects, including its lead candidate, PS-002, for IgA nephropathy (IgAN) and other complement-mediated kidney diseases, a project targeting nephrotic syndrome, and an undisclosed glomerular disease program.

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