- Maze Therapeutics is licensing its treatment for Pompe disease, a genetic disorder that weakens muscles, to Sanofi for $150 million in cash and equity investment.
- In a deal announced Monday, Sanofi will acquire rights to develop and commercialize Maze’s drug candidate MZE001, which is slated to begin a Phase 2 study later this year.
- Maze has raised $381 million in private financing since launching in 2019. Flush with new cash, the company will accelerate its research into more common conditions like chronic kidney disease.
The company announced Phase 1 results for MZE001 in February, showing the drug reduced levels of glycogen, the stored form of glucose that builds up in the skeletal, respiratory and cardiac muscle tissues of people with Pompe.
An inherited metabolic disorder, Pompe affects approximately 5,000 to 10,000 people globally, according to Maze. Many are children when they’re diagnosed, though a late-onset form of the disease can be diagnosed at any age. The disorder is caused by a mutation in the GAA gene, which contains blueprints for an enzyme that breaks down glycogen into glucose.
Enzyme replacement therapies, like Sanofi’s Lumizyme and Nexviazyme, reintroduce an artificial version of the missing enzyme and are standard treatment. They can be life-saving and slow the disease’s progression, but their benefits can wane and they don’t ease all symptoms.
MZE001 is meant to limit glycogen production by blocking an enzyme called GYS1, which is involved in glycogen production. By lowering GYS1 activity, Maze aims to reduce how much glycogen accumulates in muscle.
Maze’s deal with Sanofi also gives the French pharmaceutical company exclusive rights to related GYS1-targeting programs and intellectual property. Maze could receive up to $600 million more if certain development, commercial and regulatory milestones are met.
“Sanofi is a leading global healthcare company with deep experience working with this community and an ideal partner to continue the advancement of MZE001 to patients,” said Jason Coloma, Maze’s CEO, in an email to BioPharma Dive.
The money from Sanofi will go toward developing Maze’s other drug candidates, which are focused on more common diseases, said Harold Bernstein, Maze’s chief medical officer.
The South San Francisco-based company has two research programs for chronic kidney disease in development. One targets a mutation in the APOL1 gene and could start a clinical trial by the end of 2023, while the other is aimed at an undisclosed target and is slated to enter human studies by mid-2024, Bernstein said.
“With our current runway projected through 2025 once the deal closes, we are fortunate to have been, and remain, well capitalized with a strong shareholder base to weather various external market conditions,” Bernstein said.
Sanofi has announced a handful of acquisitions and partnerships over the past year to shore up its drug pipeline. In March, the drugmaker bought Provention Bio’s Type 1 diabetes drug in a $2.9 billion deal and, months prior in September, announced a research collaboration with gene editing company Scribe Therapeutics.
Several other drugmakers have tried to target Pompe disease in new ways, but have hit setbacks. The Food and Drug Administration briefly halted testing of Astellas Pharma's gene therapy over safety concerns, but lifted the hold in October. U.S. regulators also delayed their review of Amicus Therapeutics’ treatment due to delays in inspecting the company’s facilities in China.