Sanofi has two therapies for the uncommon dysfunction Pompe illness, one among which is a comparatively current entry into the market. However a drug candidate from Maze Therapeutics would provide benefits over each merchandise, and now that the molecule has some early validating scientific information, the pharmaceutical big is committing $150 million to accumulate it.
In response to phrases of the agreement introduced Monday, the cost consists of each upfront money and a future fairness funding in South San Francisco-based Maze. The breakdown of that cost was not additional specified. The biotech might additionally obtain as much as $600 million in milestone funds relying on the drug’s progress in Sanofi’s fingers.
Pompe is an inherited dysfunction attributable to mutations to the gene that codes for acid alpha glucosidase (GAA), an enzyme wanted to interrupt down glycogen in muscle mass. With out sufficient GAA, glycogen ranges construct up in skeletal, respiratory, and cardiac muscle tissues. Sufferers expertise progressively worsening muscle weak spot and respiratory issues.
Normal Pompe remedy consists of enzyme substitute remedy during which engineered variations of the important thing enzyme are administered intravenously. Sanofi’s Lumizyme has been obtainable for greater than a decade. The product, which is marketed in Europe below the title Myozyme, accounted for greater than €1 billion (about $1.1 billion) in income final yr.
Sanofi’s second Pompe remedy is an enzyme substitute successor to Lumizyme. Nexviazyme is designed to focus on a receptor that will increase its uptake into cells, which is meant to enhance the engineered enzyme’s capacity to clear glycogen. The FDA accepted Nexviazyme in 2021. It was accepted in Europe final yr, the place it’s marketed as Nexviadyme.
Maze found MZE001 with Compass, a platform expertise that yields genetic insights that inform the biotech’s drug discovery analysis. MZE001 works by blocking glycogen synthase, an enzyme key to glycogen manufacturing. As a small molecule, the drug may be formulated right into a twice-daily tablet, providing sufferers a extra handy different to infused enzyme substitute therapies.
In a Part 1 check in wholesome volunteers, single and a number of ascending doses of the Maze drug had been properly tolerated. Outcomes additionally confirmed reductions in ranges of glycogen within the blood throughout dose ranges, which the corporate mentioned confirmed that the drug engaged with its enzyme goal. These outcomes had been confirmed in a bunch that obtained muscle biopsies, which confirmed equal reductions in muscle glycogen. The outcomes had been offered in February throughout WORLDSymposium, the annual lysosomal illnesses convention.
“Enhancing the lives of individuals with Pompe illness is a key focus for Sanofi, and we imagine MZE001 might be an necessary addition to the affected person remedy paradigm,” Karin Knobe, Sanofi’s international head of scientific improvement uncommon illnesses and uncommon blood issues, mentioned in a ready assertion.
With MZE001 heading to Sanofi, Maze can flip its focus to its subsequent most-advanced program, a drug for APOL1-mediated power kidney illness. APOL1 is a gene that raises the kidney illness threat in individuals of African descent who carry specific gene variants. Such a kidney illness impacts an estimated a million individuals within the U.S. A scientific trial is deliberate to start within the second half of this yr. Maze has one other power kidney illness program that addresses an undisclosed goal. Scientific testing is deliberate for 2024.
In an e-mail, Maze President Jason Coloma and Harold Bernstein, president of R&D and chief medical officer, mentioned the applicability of the insights from Compass is broad. Wanting forward, the corporate will give attention to extra widespread genetically linked issues the place Maze might have a much bigger affect for sufferers.
Maze is creating antibody medication for eye illness through Broadwing Bio, a three way partnership shaped in 2020 with Alloy Therapeutics. Coloma and Bernstein added that the corporate is repeatedly evaluating numerous enterprise improvement alternatives with outdoors companions. The Sanofi licensing deal was the results of such ongoing dialogue. Coloma and Bernstein mentioned Maze has sufficient capital to help the corporate by way of 2025.
Photograph: Nathan Laine/Bloomberg, through Getty Photos