Sanofi bets $750M to enter glycogen-lined Maze deal with lead asset waiting in the middle

Sanofi is offering up to $750 million in upfront cash and biobucks for Maze Therapeutics’ glycogen synthase 1 (GYS1) program, including a lead asset that’s recently completed a phase 1 trial for patients with Pompe disease.

The new licensing deal for MZE001 adds another clinical-stage rare disease med to Sanofi's pipeline, a therapeutic area that currently makes up about 15% of the pharma’s pipeline. Maze in exchange will receive $150 million in upfront cash and equity plus up to $600 million in milestone payments. Executives for Maze wouldn’t elaborate on the deal structure, including how the upfront payment breaks down or when the smaller biotech is set to earn its first milestone payment. 

Maze recently reported updated data from a phase 1 trial of MZE001 in healthy volunteers and found the med to be well tolerated up to 720 mg administered twice daily. Maze also found that the med lowered blood and muscle glycogen levels, potential biomarkers for therapeutic impact against Pompe disease. Sanofi will be responsible for launching a phase 2 trial for the med, which Maze had previously teased would happen later this year. 

Although MZE001 is the main attraction, Sanofi also receives any intellectual property or “know-how” around other indications or GYS1 backup programs. Maze Chief Medical Officer Harold Bernstein, M.D., Ph.D., said in a written response to questions that once the deal closes, it will be up to Sanofi to decide whether to expand MZE001’s use.

The deal has spurred a recalibration of Maze's pipeline, with "near-term resources" aimed at the company's common disease programs, according to Bernstein, namely two treatments being developed for chronic kidney disease. The asset furthest along targets APOL1 and is set to enter the clinic before the end of the year. A second CKD med with an undisclosed target should reach human trials in 2024. 

Bernstein says that Maze's treatment for phenylketonuria is "off the pipeline for now" while the common disease meds take priority but is also "still in play." Maze has two other preclinical rare disease meds aimed at ALS, one of which was slated to have nonhuman primate data before the end of 2022, although the company has yet to disclose any.