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Shionogi and Maze Therapeutics announce worldwide exclusive licensing agreement for novel Pompe disease candidate

James Spargo | May 10, 2024 | News story | Sales and Marketing Pompe disease, Rare Diseases, Shionogi, licensing agreement, maze therapeutics 

Japanese pharmaceutical company Shionogi and US-based Maze Therapeutics have finalised an exclusive worldwide license agreement for MZE001, a novel therapeutic candidate for the treatment of Pompe disease.

MZE001 is an investigational oral glycogen synthase 1 (GYS1) inhibitor which aims to treat Pompe disease – a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA) – by limited disease-causing glycogen buildup. This build-up can cause progressive weakness and respiratory problems in the skeletal, respiratory and cardiac muscle tissues.

Under the agreement, Shionogi will pay $150m upfront for the exclusive worldwide rights for MZE001, as well as related programmes and intellectual property. Maze will be eligible for developmental, regulatory and commercial milestone payments, plus tiered royalties based on future net sales.

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Isao Teshirogi PhD, CEO of Shionogi, said, “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. The science behind MZE001 is differentiated and promising, and we look forward to developing the compound as both monotherapy and add-on therapy to enzyme replacement therapies.”

Jason Coloma, CEO of Maze, added: “Shionogi is committed to advancing and commercialising MZE001 because they understand the potential this therapy has for patients and the unmet medical needs it could address. 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.”

James Spargo

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