Shionogi and Maze Therapeutics Announce Global License for Pompe Disease Treatment MZE001

Shionogi & Co., Ltd., based in Osaka, Japan, and Maze Therapeutics, Inc., located in South San Francisco, California, have finalized an exclusive worldwide licensing agreement concerning MZE001. This investigational oral glycogen synthase 1 (GYS1) inhibitor aims to tackle Pompe disease by preventing the harmful accumulation of glycogen in muscle tissues.

Pompe disease is a rare genetic disorder caused by mutations in the gene responsible for acid alpha-glucosidase (GAA). These mutations lead to the buildup of glycogen in various muscle tissues, including skeletal, respiratory, and cardiac muscles, resulting in progressive muscle weakness and respiratory issues.

As per the agreement, Shionogi procures exclusive global rights to MZE001, along with associated programs and intellectual property. Shionogi will make an initial payment of $150 million to Maze, which could also receive additional milestone payments contingent on developmental, regulatory, and commercial milestones. Further, Maze will benefit from tiered royalties on future net sales of the product. This transaction has been completed following the expiration of the 30-day waiting period mandated by the United States Hart-Scott-Rodino (HSR) Act.

Shionogi’s CEO, Isao Teshirogi, Ph.D., remarked that the agreement aligns well with Shionogi's strategic goals, particularly its objective to develop innovative treatments targeting unmet medical needs. The acquisition is part of Shionogi's expanded pipeline initiatives outlined in its Medium-Term Business Plan STS2030 Revision. Teshirogi expressed enthusiasm about the potential of MZE001, highlighting its promise as a differentiated and innovative solution that could be developed both as a standalone treatment and an adjunct to existing enzyme replacement therapies.

MZE001 is a small molecule that specifically inhibits GYS1, an enzyme crucial in glycogen synthesis. By targeting this enzyme, MZE001 aims to reduce glycogen accumulation in muscle tissues. Early Phase 1 study results indicate that MZE001 could potentially become the first oral treatment for Pompe disease. It is being explored both as a monotherapy and as an add-on to enzyme replacement therapies, which are currently the standard treatment for Pompe disease.

Jason Coloma, Ph.D., CEO of Maze, expressed confidence in Shionogi's commitment to advancing and commercializing MZE001, recognizing Shionogi’s history of developing and delivering innovative treatments globally. Coloma believes that with Shionogi as a partner, MZE001 can progress through clinical trials and reach patients suffering from this severe condition more rapidly.

In 2022, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to MZE001. This designation is awarded to treatments targeting rare diseases and comes with incentives such as tax credits, grants, waivers of certain administrative fees, and seven years of market exclusivity post-approval.

Pompe Disease is a rare congenital metabolic disorder marked by a deficiency of the enzyme needed for glycogen breakdown within cells. This deficiency leads to the accumulation of glycogen, particularly in muscle cells, causing symptoms like muscle weakness and delayed growth. In Japan, Pompe disease is recognized as a "specified rare disease" and a "specific pediatric disease," which qualifies it for various medical expense assistance programs and other support measures.

Maze Therapeutics leverages human genetics to develop therapies for various diseases, including chronic kidney disease. The company utilizes a proprietary platform, Maze Compass PlatformTM, to integrate genetic variant functionalization into drug development. Maze is building a broad portfolio of programs, both independently and through partnerships.

Shionogi & Co., Ltd. is a prominent global pharmaceutical company focused on discovering and developing medicines for various conditions, including infectious diseases and central nervous system disorders. The company’s research pipeline spans multiple therapeutic areas, including infectious disease, pain/CNS, metabolic disorders, rare diseases, oncology, and stroke.