Shionogi & Co., Ltd. and Maze Therapeutics, Inc. announced the companies have completed an exclusive worldwide license agreement for the rights to MZE001, an investigational oral glycogen synthase 1 (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup.
Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA), which can lead to the buildup of glycogen in skeletal muscle, respiratory muscle and cardiac muscle tissues resulting in progressive weakness and respiratory compromise.
Under the terms of the agreement, Shionogi has acquired exclusive worldwide rights for MZE001 as well as related programs and intellectual property. Shionogi will pay an upfront fee of $150 million, and Maze will be eligible for milestone payments based on development, regulatory and commercial achievements plus tiered royalties based upon future net sales. The required 30-day waiting period outlined in United States Hart-Scott-Rodino (HSR) Act has expired, and the transaction is completed.
“This agreement is a strong strategic fit for Shionogi. It will help meaningfully advance our commitment to develop innovative medicines that address unmet medical needs and complement Shionogi’s rapidly expanding pipeline in the focus areas designated in our Medium-Term Business Plan STS2030 Revision,” said Isao Teshirogi, Ph.D., CEO of Shionogi. “The science behind MZE001 is differentiated and promising, and we look forward to developing the compound as both mono and add-on therapy to enzyme replacement therapies.”
MZE001 is a small molecule and specific inhibitor of GYS1, an enzyme involved in glycogen synthesis. It reduces the glycogen concentration in muscles by inhibiting this enzyme, and the results from the Phase 1 study of MZE001 suggest that it has the potential to be the first oral therapy for the treatment of Pompe disease. MZE001 has the potential to be used both as a monotherapy option and as an add-on therapy with enzyme replacement, the current standard of care, to enhance the treatment of patients with Pompe disease.
“Shionogi is committed to advancing and commercializing MZE001 because they understand the potential this therapy has for patients and the unmet medical needs it could address,” said Jason Coloma, Ph.D., CEO of Maze. “Shionogi has a track record of developing and delivering innovative medicines to patients worldwide, and we’re confident they are the right partner to continue to advance MZE001 through clinical trials so that it may reach patients with this life-threatening condition as soon as possible.”
In 2022, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to MZE001. The FDA grants Orphan Drug designation to prevent, diagnose or treat a rare disease or condition. Under the FDA’s Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval.