Shionogi has concluded an exclusive global licence agreement for Maze Therapeutics’s MZE001, an oral glycogen synthase 1 (GYS1) inhibitor to treat Pompe disease.
The agreement grants Shionogi the sole rights to MZE001 and related programmes as well as intellectual property globally.
Maze is entitled to receive $150m in upfront payment from Shionogi.
Maze will receive milestone payments contingent on the progress of the development, regulatory approvals and commercial success.
The company will also earn tiered royalties on future net sales of the therapeutic candidate.
The 30-day waiting period under the Hart-Scott-Rodino Act in the US has now lapsed, marking the completion of the deal.
MZE001 works by reducing the accumulation of disease-causing glycogen. It hinders GYS1, an enzyme responsible for glycogen synthesis. This inhibition reduces glycogen accumulation in muscles, a key factor in Pompe disease.
Phase I clinical trial results indicated that MZE001 could become the first oral therapy option.
Its potential extends to both a standalone treatment and an adjunctive therapy alongside enzyme replacement, which is currently the standard care.
The US Food and Drug Administration awarded orphan drug designation to MZE001 in 2022.
Shionogi CEO Isao Teshirogi stated: “This agreement is a strong strategic fit for Shionogi. It will help meaningfully advance our commitment to developing 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 mono and add-on therapy to enzyme replacement therapies.”
In June 2023, Shionogi entered a definitive agreement to acquire Qpex Biopharma for $140m.
