Biotech Startup Maze Therapeutics Strikes $750 Million Deal With Sanofi

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Maze Therapeutics Inc. has raised about $400 million in venture capital since its 2019 launch on the promise of a platform for using genetic insights to develop new drugs. Now, the biotechnology startup is making good on that potential.

Maze could earn $750 million through a deal to license out a potential rare-disease drug to French drugmaker Sanofi SA, the South San Francisco, Calif.-based startup said Monday. Maze will receive $150 million in cash and an equity investment from Sanofi, and could earn about $600 million more in success-based payments, plus royalties if the treatment for the genetic disorder Pompe disease is successfully brought to market.

The startup, whose investors include Third Rock Ventures and ARCH Venture Partners, now has cash to last through 2025, Chief Executive Jason Coloma said.

Maze said it now plans to focus on treatments for more common diseases that are advancing toward clinical trials, including drugs for chronic kidney disease.

Pompe disease occurs because of mutations in a gene for an enzyme, acid alpha-glucosidase, needed to break down glycogen, a stored form of sugar used for energy, according to the National Institute of Neurological Disorders and Stroke.

The mutations reduce or eliminate the enzyme, leading to a buildup of glycogen that damages skeletal and heart muscles, according to the institute.

Enzyme-replacement therapy through infusions, such as Sanofi’s Nexviazyme, can treat Pompe disease. Maze’s drug is taken orally and has gone through initial, Phase I clinical trials. The product could be an important addition to treatment options, Sanofi said.

Maze’s drug is designed to stop the buildup of glycogen in the muscles of Pompe patients. The company used its technology platform to answer the question of what would happen if Maze could slow glycogen production in muscle tissues, said Harold Bernstein, president, research and development and chief medical officer, adding that preclinical genetic analysis gave the company confidence it would be safe to do so. The drug was well-tolerated in the Phase I study in healthy volunteers, Maze said.  

The Pompe drug illustrates how Maze can use its platform for new potential therapies, Dr. Bernstein said. But Maze can best apply its expertise and resources, and make the biggest impact for patients, by focusing on conditions such as APOL1-mediated chronic kidney disease, a kidney disease caused by mutations in the APOL1 gene, he added.

Maze expects to start clinical trials of its drug for APOL1-mediated chronic kidney disease around year-end and expects its second program in chronic kidney disease to enter clinical trials in 2024, Dr. Bernstein said. 

Write to Brian Gormley at brian.gormley@wsj.com