GSK and Rgenta Therapeutics Forge Strategic Alliance to Develop Multiple RNA-Targeted Small Molecule Splicing Modulators with Potential Deal Value Exceeding $500 Million per Target

Dec 04, 2024 21:20 CST Updated 21:20

GSK

Pharmaceutical R&D Manufacturer

Rgenta

New Drug Developer

December 4,Woburn, Massachusetts, Rgenta Therapeutics, Inc., a clinical-stage biotechnology company pioneering the development of a new class of orally available small molecule RNA-targeted drugs for oncology and neurological disorders, announced a multi-year, multi-target strategic research alliance with GSK. The alliance aims to advance the discovery and development of novel RNA-targeted small molecule splicing modulators across various disease areas, including oncology.Rgenta completed its seed financing round (USD 20 million) in 2020, seed+ financing round (USD 18 million) in 2021, and a USD 52 million Series A financing round in 2022. Key investors include AstraZeneca and CICC.KIP Capital, Delos Capital,BI Venture Fund (BIVF), Matrix Partners, Lilly Asia Ventures, Vivo Capital, Kaitai Capital, Legend Star, etc.（New Frontier! $90 Million Financing to Develop Small Molecule RNA-Targeted Drugs）

According to the terms of the agreement, Rgenta will receive up to$46 millionCash prepayment and pre-option milestone payments. Rgenta is potentially eligible to receive up to per target in option exercise, research, development, regulatory, and commercial milestone payments, as well as tiered royalties.Nearly $500 Million in RevenueAnd future equity investment. GSK can also choose to expand the alliance to include other targets. Under the alliance, Rgenta will use its proprietary discovery platform to develop novel oral RNA-targeted small molecule splicing modulators for multiple targets nominated by GSK. After GSK exercises its option, GSK will be responsible for the further development and commercialization of any candidate drugs generated by the alliance.

Due to the structural characteristics of RNA and the complexity of related regulatory mechanisms, the development of small molecules targeting RNA has a high threshold. Focusing on traditionally undruggable targets, Rgenta is dedicated to developing innovative small molecule drugs that target RNA. Its integrated discovery platform identifies regulatory sites in RNA suitable for selective modulation by small molecules by analyzing vast amounts of human genomic data, rapidly screening small molecule drugs targeting RNA to modulate protein expression or alter protein function.

Christopher Austin, M.D., Senior Vice President of Research Technology at GSK, said:"We are excited about Rgenta's differentiated approach to discovering and developing oral small molecule splicing modulators for high-value targets. We are committed to collaborating with leading companies to complement our existing expertise in RNA-targeted therapeutics, and look forward to advancing this promising drug modality to benefit more patients with difficult-to-treat diseases."

Rgenta Co-founder and CEO Simon Xi (Hualin Xi) PhD said:"We are delighted to have leading biopharmaceutical companiesGlaxoSmithKline PLC."As our partner, this alliance further validates the potential of Rgenta's small molecule RNA-targeting drug discovery platform. Together, we have the potential to accelerate the development of a new class of drugs and provide patients with new treatment options."

Travis Wager, Ph.D., Co-founder, President, and Chief Scientific Officer of Rgenta, stated:"We are thrilled to partner with GSK, whose science-driven philosophy and proven track record of leveraging scientific innovation for patient benefit align perfectly with our mission. This alliance also validates our discovery platform, which has already led to our first clinical-stage asset, RGT-61159, an orally administered small molecule specifically designed to modulate the splicing of the transcription factor MYB."

August 2021,Rgenta Therapeutics, Inc. and Lundbeck Reach Collaboration, to collaboratively develop small molecule RNA modulators for neurological diseases. Rgenta will receive upfront and near-term payments of up to $10 million from Lundbeck and could potentially obtain clinical and commercial milestone payments of up to $100 million for the initial target-based program. Additionally, Rgenta is eligible to receive tiered royalties on global net sales of the collaboration products. Lundbeck and Rgenta may choose to expand their partnership to include other brain disease targets under consideration.

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