Maze Therapeutics and Japan-based Shionogi have entered into an exclusive licensing agreement for MZE001, an oral GYS1 inhibitor aimed at treating Pompe disease, a rare genetic disorder.
Under the agreement, Shionogi will gain global rights to develop, manufacture, and commercialize MZE001, alongside related programs and intellectual property. The deal includes an upfront payment of $150 million from Shionogi to Maze, with additional milestone payments and royalties on future sales.
MZE001 targets this buildup by inhibiting the glycogen synthase 1 (GYS1) enzyme, potentially reducing disease progression. This treatment, currently in phase 1 trials, could become the first oral therapy for Pompe disease, offering an alternative over existing enzyme replacement therapies. Pompe disease leads to severe muscle weakness due to an accumulation of glycogen, primarily affecting skeletal and cardiac muscles.
It's the same drug that Sanofi attempted to license last May, in a $755 million deal. However, the deal fell through in December after the FTC moved to block the acquisition. The FTC argued that the acquisition would eliminate a potential competitor and sustain Sanofi's monopoly in the Pompe disease therapy market. Despite disagreeing with the FTC's allegations, Sanofi decided to terminate the deal to avoid prolonged litigation, concluding that continuing the deal would not benefit patients.