Shionogi acquires rights to Maze Pompe drug

Maze Therapeutics and Japan-based Shionogi have formed an exclusive licensing agreement involving MZE001, an oral inhibitor of the enzyme glycogen synthase 1 (GYS1), designed to treat Pompe disease. Pompe disease is a rare genetic disorder that primarily affects skeletal and cardiac muscles, leading to severe muscle weakness due to the accumulation of glycogen.

Under this agreement, Shionogi has secured global rights to develop, manufacture, and commercialize MZE001, along with associated programs and intellectual property. As part of the deal, Shionogi will make an upfront payment of $150 million to Maze, with provisions for additional milestone payments and royalties from future sales.

MZE001 works by inhibiting the GYS1 enzyme, potentially reducing the progression of Pompe disease. This innovative approach could make MZE001 the first oral therapy for the condition, providing an alternative to existing enzyme replacement therapies, which are the current standard of care.

Pompe disease results from a deficiency in the enzyme acid alpha-glucosidase (GAA), leading to excessive glycogen buildup in various tissues. This excessive glycogen particularly affects muscle tissues and can cause debilitating muscle weakness. By targeting GYS1, MZE001 aims to address this buildup and alleviate the symptoms of the disease.

Previously, Sanofi had shown interest in licensing MZE001. In May of the previous year, Sanofi proposed a $755 million agreement for the drug. However, this deal was terminated in December after the Federal Trade Commission (FTC) intervened, arguing that the acquisition would stifle competition and maintain Sanofi's monopolistic hold on the Pompe disease therapy market. While Sanofi disagreed with the FTC's stance, the company chose to abandon the acquisition to avoid prolonged legal battles, concluding that the extended litigation would not be beneficial for patients.

MZE001 is currently in phase 1 clinical trials, and its development is being closely watched within the medical and pharmaceutical communities. If successful, this oral treatment could significantly improve the quality of life for patients with Pompe disease by offering a more convenient and potentially more effective treatment option compared to enzyme replacement therapies, which often require intravenous administration.

The partnership between Maze Therapeutics and Shionogi is a significant step forward in the fight against Pompe disease. The agreement not only provides Maze with substantial financial backing but also leverages Shionogi's expertise in drug development and commercialization, potentially accelerating the availability of this promising treatment to patients worldwide.

This deal highlights the ongoing efforts in the biopharmaceutical industry to develop innovative treatments for rare genetic disorders, which often have limited therapeutic options. The success of MZE001 could pave the way for further advancements in the treatment of Pompe disease and similar conditions, underscoring the importance of continued research and collaboration in this field.