Shionogi & Co and Maze Therapeutics have announced an exclusive worldwide licensing agreement for an investigational Pompe disease therapy.
Estimated to affect about one in 40,000 people in the US, Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase, resulting in the accumulation of glycogen in cells throughout the body.
The disease causes a range of symptoms, including progressive muscle weakness, respiratory compromise and delayed growth.
Shionogi has acquired exclusive worldwide rights for Maze’s MZE001, an oral glycogen synthase 1 inhibitor that aims to address Pompe disease by limiting glycogen buildup, as well as related programmes and intellectual property.
The candidate has already been granted Orphan Drug Designation by the US Food and Drug Administration and has demonstrated potential in an early-stage trial to be the first oral therapy for Pompe disease, according to Shionogi, adding that it could be used as both a monotherapy and an add-on to enzyme replacement, the current standard of care.
In exchange, Maze will receive an upfront fee of $150m and will be eligible for undisclosed milestone payments based on development, regulatory and commercial achievements, plus tiered royalties on future net sales.
Isao Teshirogi, chief executive officer of Shionogi, said: “This agreement is a strong strategic fit for 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.”
Also commenting on the deal, Maze’s chief executive officer, Jason Coloma, said: “Shionogi is committed to advancing and commercialising MZE001 because [it understands] the potential this therapy has for patients and the unmet medical needs it could address.
Coloma added that the company is “confident” that Shionogi is the right partner to continue to advance MZE001 through clinical trials so that it reaches Pompe disease patients “as soon as possible”.
The agreement comes just one day after Shionogi announced a collaboration with Nagasaki University, the National Institute of Infectious Diseases and Medicines for Malaria Venture to create new prevention drugs for malaria.