Sanofi bets on Maze's oral GYS1 inhibitor MZE001 for Pompe disease
01 May 2023
Phase 1License out/inClinical ResultPhase 2
Sanofi has signed a licensing agreement for Maze Therapeutics' GYS1 programme, including its MZE001 candidate currently in development for Pompe disease and other glycogen storage disorders. "We believe MZE001 could be an important addition to the patient treatment paradigm," said Karin Knobe, Sanofi's global head of clinical development for rare diseases and rare blood disorders, in a statement Monday, adding "MZE001 has demonstrated meaningful preclinical proof-of-concept by inhibiting GYS1, a validated, genetic driver of Pompe disease."
The drug is designed to work by limiting disease-causing glycogen accumulation. Maze has advanced MZE001 through Phase I, and recently reported results of its first-in-human study in healthy volunteers. Response to MZE001 was evaluated using peripheral blood mononuclear cell (PBMC) glycogen. According to Maze, results demonstrated target engagement with GYS1, with exposure-dependent reductions in PBMC glycogen across dose levels 10 days after administration. The company said these findings were confirmed in a muscle biopsy cohort that showed equivalent reductions in muscle glycogen with MZE001. The compound was also well-tolerated at doses up to 720mg twice daily, it added.
Up to $600 million milestones
Under the agreed terms, Maze will receive a $150-million payment consisting of both upfront cash and future equity investment for rights to further develop and commercialise MZE001, as well as an exclusive license to related GYS1-targeting back-up programmes and intellectual property. Maze could also pocket up to an additional $600 million in potential development, regulatory and sales milestones, as well as "meaningful royalties" on potential sales.
The company, which launched in 2019, secured $190 million in financing last year from the likes of Matrix Capital, General Catalyst and a16z Bio+Health, among others, to build out its platform to discover and drug genetic modifiers. "This agreement provides important validation for the potential of our Compass platform to elucidate novel, genetic insights that inform the discovery and advancement of new medicines," remarked CEO Jason Coloma. "We believe the applicability of these insights is broad, and as we look ahead, Maze will focus on more common disorders, where ultimately, we may best apply our expertise and resources to offer the biggest impact for patients," he added.
MZE001 is Maze's most advanced candidate, which it describes as "Phase II ready." According to its website, the company is also looking to advance a small-molecule APOL1-targeting therapy into the clinic by year end for chronic kidney disease.
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