Rgenta Therapeutics Announces Strategic Alliance with GSK to Develop RNA-Targeted Small Molecule Splice Modulators

Rgenta to receive a cash upfront and pre-option milestone payments

Rgenta has the potential to receive milestone payments, royalties, and a future equity investment

WOBURN, Mass., Dec. 4, 2024 /PRNewswire/ -- Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA for oncology and neurological disorders, announced today that it has entered into 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 splice modulators for multiple disease areas including oncology.

"We are excited about Rgenta's differentiated approach to discover and develop oral small molecule splice modulators for high-value targets," said Christopher Austin, M.D., SVP Research Technologies at GSK. "We are dedicated to partnering with leading companies to complement our existing expertise in RNA-targeting medicines and look forward to advancing this promising modality to more patients with difficult-to-treat diseases."

"We are thrilled to have GSK, a leading biopharma company, as our partner. This alliance further validates the potential of Rgenta's small molecule RNA-targeting drug discovery platform," said Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta. "Together we have the potential to accelerate the development of a new class of medicines that can provide new therapeutic options for patients."

Under the terms of the agreement, Rgenta will receive up to $46 million in a cash upfront and pre-option milestone payments. Rgenta has the potential to receive up to nearly $500 million per target in option exercise, research, development, regulatory, and commercial milestone payments plus tiered royalties and a future equity investment. GSK also has an option to expand the alliance to include additional targets. Under the alliance, Rgenta will use its proprietary discovery platform to develop novel oral RNA-targeting small molecule splice modulators against multiple targets nominated by GSK for development. Following GSK's exercise of its options, GSK will be responsible for further development and commercialization of any drug candidates that arise from the alliance.

Travis Wager, Ph.D. co-founder, president and chief scientific officer of Rgenta added, "We are excited to partner with GSK, whose science-driven philosophy and successful track record of harnessing scientific innovation to benefit patients aligns perfectly with our mission. This alliance additionally validates our discovery platform which has already led to our first clinical stage asset, RGT-61159, an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB."

About Rgenta Therapeutics
Rgenta Therapeutics is a clinical-stage biotechnology company developing a pipeline of oral RNA-targeting small molecule medicines with an initial focus on oncology and neurological disorders. Rgenta's lead program RGT-61159 is an orally available small molecule designed to modulate splicing of the transcription factor MYB, a known oncogene, and is being studied in a Phase 1a/b study for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML).  Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs. Our unique approach and lead programs are focused on unlocking the therapeutic potential of historically undruggable targets in human diseases. Learn more at: http://www.rgentatx.com.

Contacts
Investors:
Sylvia Wheeler
Wheelhouse Life Science Advisors
[email protected]

Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
[email protected]

Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
[email protected]

SOURCE Rgenta Therapeutics