Sanofi In-Licenses Pompe Disease Therapy From Privately-Held Maze Therapeutics For $150M Upfront

Maze Therapeutics has signed an exclusive worldwide license agreement with Sanofi SA SNY for Maze's glycogen synthase 1 (GYS1) program, including clinical candidate MZE001, which is currently in development for the treatment of Pompe disease and other potential indications. 

MZE001, designed and developed by Maze, is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen accumulation.

Maze has advanced MZE001 through Phase 1 development and recently reported results of its study in healthy volunteers. 

Also Read: Sanofi Posts Upbeat Q1 Earnings, Sales Buoyed By Blockbuster Dupixent Sales.

In the first-in-human, double-blind, placebo-controlled, single, and multiple ascending dose clinical trial, MZE001 was well-tolerated at doses up to 720 mg twice daily. 

Response to MZE001 was evaluated in patients using a novel biomarker, peripheral blood mononuclear cell (PBMC) glycogen. It demonstrated exposure-dependent PBMC glycogen reduction across dose levels ten days after administration, confirming target engagement with GYS1. 

These results were further confirmed in a muscle biopsy cohort that showed equivalent reductions in muscle glycogen with MZE001.

Under the terms of the agreement, Maze will receive a $150 million payment consisting of both upfront cash and future equity investment. 

Maze will be eligible to receive up to an additional approximately $600 million in potential milestone payments and sales-based royalties.

Price Action: SNY shares closed at $53.65 on Friday.