FRANCE — Sanofi and Maze Therapeutics have signed an agreement for the exclusive worldwide license of Maze’s glycogen synthase 1 (GYS1) program and oral substrate reduction therapy, MZE001, to treat Pompe disease.
MZE001 is being developed for other potential indications as well. The drug is a small molecule and is formulated into a twice-daily pill, which offers a more convenient alternative to infused enzyme replacement therapies.
The therapy works by blocking glycogen synthase, which is an enzyme key to glycogen production.
The drug has been clinically validated, and Sanofi will acquire it for US$150 million, which includes upfront cash and a future equity investment in Maze.
The biotech could also receive up to US$600 million in milestone payments depending on the drug’s progress in Sanofi’s hands.
Pompe is a genetic disorder that leads to progressively worsening muscle weakness and breathing problems due to mutations in the gene that codes for acid alpha-glucosidase (GAA), an enzyme needed to break down glycogen in muscles.
Enzyme replacement therapy (ERT) is the standard treatment for Pompe disease, involving the administration of engineered versions of the missing enzyme intravenously.
Sanofi has two therapies for the disease, including enzyme replacement therapy.
Sanofi’s Lumizyme, which has been available for over ten years, is a popular ERT treatment for Pompe disease.
In Europe, the product is marketed under the name Myozyme and generated more than €1 billion (about US$1.1 billion) in revenue last year.
However, Lumizyme has some limitations, such as poor uptake into cells and a short half-life, which has spurred Sanofi to develop a second-generation enzyme replacement therapy called Nexviazyme.
Nexviazyme is an engineered enzyme that targets a receptor, which enhances its uptake into cells, allowing it to clear glycogen buildup.
The US FDA approved Nexviazyme in 2021, and it received European approval last year, where it is sold as Nexviadyme.
However, MZE001 offers advantages over both products as it can be formulated into a twice-daily pill, offering patients a more convenient alternative to infused enzyme replacement therapies.
The drug has been discovered with Compass, a platform technology that yields genetic insights that inform Maze’s drug discovery research.
In a Phase 1 test in healthy volunteers, single and multiple ascending doses of the Maze drug were well tolerated, and reductions in glycogen levels were seen across dose levels.
This confirmed that the drug engaged with its enzyme target. The results were presented in February during WORLDSymposium, the annual lysosomal diseases conference.
With the deal heading to Sanofi, Maze can focus on its next most-advanced program, a drug for APOL1-mediated chronic kidney disease.
Maze is developing antibody drugs for eye disease via Broadwing Bio, a joint venture formed in 2020 with Alloy Therapeutics.
The company will focus on more common genetically linked disorders where Maze could have a bigger impact for patients.
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