Shionogi Secures Global Rights to Maze Therapeutics’ MZE001, a Potential First-in-Class Oral Therapy for Pompe Disease, with $150M Upfront

May 10, 2024 15:53 CST Updated 15:53

Maze Therapeutics

Developer of Therapeutic Drugs for Genetic Diseases

▎Edited by the WuXi AppTec content team

Today (May 10),Shionogi & Co., Ltd.（Shionogi）AnnouncementEnter into an exclusive license agreement with Maze Therapeutics.ShionogiObtainMZE001 developed by the latterofGlobal exclusive rights, andWill Pay$150 million upfront payment，MazeCompanyWill be eligible for milestone payments based on development, regulatory, and commercialization achievements.。MZE001 is an investigationalOralPompe Disease Therapy, which had already achieved positive results in the Phase 1 clinical trial.According to Shionogi's press release, MZE001 has the potential to become the first oral medication for treating Pompe disease.

Pompe disease is a hereditary lysosomal storage disorder caused by the deficiency of acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues, which is why it is also referred to as Glycogen Storage Disease Type II. This condition is a rare and often fatal disorder that results in skeletal and cardiac muscle dysfunction. In severely affected patients with complete or near-complete absence of GAA, symptoms typically manifest within a few months after birth.Most infant patients will die from heart or respiratory-related complications before their first birthday.Currently, enzyme replacement therapy is the standard treatment option for Pompe disease, but there are still unmet clinical needs.

MZE001 isSmall Molecule SpecificityGlycogen Synthase 1 (GYS1) Inhibitor,It reduces the glycogen concentration in muscles by inhibiting this enzyme.According to the Shionogi press release, MZE001 has the potential to be used as both a standalone treatment option and an adjunctive therapy to enzyme replacement, enhancing the treatment of patients with Pompe disease. In 2022, the U.S. FDA granted MZE001 orphan drug designation.

In March 2023, Maze Therapeutics announced positive results from the Phase 1 clinical trial of MZE001 conducted in healthy subjects. In the first-in-human, double-blind, placebo-controlled, single and multiple ascending dose clinical trial, MZE001 was well-tolerated at doses up to 720 mg twice daily. Additionally, researchers used a novel biomarker—peripheral blood mononuclear cell (PBMC) glycogen—to evaluate patients' response to MZE001.After 10 days of dosing, PBMC glycogen showed exposure-dependent reduction at all dose levels, confirming the targeted binding of MZE001 to GYS1.

Shionogi is a Japanese pharmaceutical company that has discovered and developed novel antibiotics, anti-HIV, and influenza drugs. The company's global R&D pipeline includes treatments for infectious diseases, pain/CNS, metabolic disorders, rare diseases, oncology, and stroke.

Maze Therapeutics is a biopharmaceutical company focused on genetic therapies for common diseases such as chronic kidney disease. Notably, in September 2023, Maze Therapeutics was named one of the "Most Promising Emerging Biotech Companies" by the renowned industry media Endpoints.List。

References:

[1]Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Announce Exclusive Worldwide License Agreement for MZE001, a Novel Therapeutic Candidate for the Treatment of Pompe Disease. Retrieved May 9, 2024, from https://www.shionogi.com/global/en/news/2024/05/e20240510.html

[2] Maze Therapeutics Announces Positive Phase 1 Results from First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease. Retrieved February 28, 2023, from https://www.businesswire.com/news/home/20230227005174/en

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