Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Strike Exclusive Global Licensing Deal for MZE001, a Promising New Treatment for Pompe Disease

Shionogi & Co., Ltd. (based in Osaka, Japan; CEO: Isao Teshirogi, Ph.D.) and Maze Therapeutics, Inc. (headquartered in South San Francisco, California, USA; CEO: Jason Coloma, Ph.D.) have finalized an exclusive worldwide licensing agreement for MZE001. This novel therapeutic candidate targets Pompe disease by inhibiting glycogen synthase 1 (GYS1), thus curtailing the accumulation of disease-causing glycogen.

Pompe disease is a rare genetic disorder arising from mutations in the acid alpha-glucosidase (GAA) gene, leading to the excessive buildup of glycogen in various muscle tissues, resulting in progressive weakness and respiratory complications.

Per the agreement, Shionogi gains exclusive global rights to MZE001 along with associated programs and intellectual property. The deal involves an upfront payment of $150 million to Maze, with additional milestone payments contingent on developmental, regulatory, and commercial milestones, alongside tiered royalties based on future sales. The completion of the transaction follows the expiration of the mandated 30-day waiting period stipulated by the United States Hart-Scott-Rodino (HSR) Act.

Isao Teshirogi, Ph.D., Shionogi’s CEO, expressed optimism about the strategic alignment of the agreement with Shionogi’s commitment to addressing medical needs. He underscored the potential of MZE001’s science in advancing treatment options for Pompe disease, anticipating its development both as standalone therapy and in conjunction with enzyme replacement therapies.

MZE001, a small molecule GYS1 inhibitor, has shown promise in reducing glycogen levels in muscles based on Phase 1 trial results. It holds potential as the first oral therapy for Pompe disease, offering prospects for use as monotherapy or alongside existing treatments to improve patient outcomes.

Jason Coloma, Ph.D., CEO of Maze, emphasized Shionogi’s dedication to advancing MZE001, citing their track record in developing and delivering innovative medicines globally. He expressed confidence in Shionogi’s capability to propel MZE001 through clinical trials, bringing it closer to patients grappling with this debilitating condition.

In 2022, MZE001 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), a recognition aimed at facilitating the development of therapies for rare diseases. This designation grants various incentives, including tax credits, grants, fee waivers for clinical trials, and seven years of market exclusivity post-approval.

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