Maze Therapeutics and Sanofi sign exclusive worldwide license agreement for Pompe disease treatment

Betsy Goodfellow | May 3, 2023 | News story | Sales and Marketing License Agreement, Pompe disease, Rare Diseases, Sanofi, maze therapeutics, treatment 

Maze Therapeutics and Sanofi have announced that they have signed an exclusive worldwide license agreement for Maze’s gluogen synthase 1 (GYS1) programme, including its clinical candidate MZE001, a treatment for Pompe disease that is currently in development.

MZE001 was both designed and developed by Maze and acts as an oral GYS1 inhibitor aiming to treat Pompe disease ‒ and any other potential indications ‒ by limiting the disease-causing glycogen accumulation.

Under the terms of this agreement, Maze will receive a $150m payment made up of an upfront payment and future equity investment for the rights to further develop and commercialise MZE001. Maze will be eligible to receive up to $600m in potential development, regulatory and sales milestones as well as royalties on sales if MZE001 is successfully commercialised.

Jason Coloma, PhD, chief executive officer of Maze, commented: “People with Pompe disease continue to need additional treatment options for this life-threatening condition. Sanofi is a leading global healthcare company with deep experience working with this community and the ideal partner to continue the advancement of MZE001. This agreement provides important validation for the potential of our Compass platform to elucidate novel, genetic insights that inform the discovery and advancement of new medicines. We believe the applicability of these insights is broad, and as we look ahead, Maze will focus on more common disorders, where ultimately, we may best apply our expertise and resources to offer the biggest impact for patients. We are thankful for this partnership and are excited to enter the next phase of Maze’s ambitious mission to transform the lives of patients through genetically informed therapies.”

Karin Knobe, global head of Clinical Development Rare Diseases and Rare Blood Disorders at Sanofi, added: “Improving the lives of people with Pompe disease is a key focus for Sanofi, and we believe MZE001 could be an important addition to the patient treatment paradigm. MZE001 has demonstrated meaningful preclinical proof of concept by inhibiting GYS1, a validated, genetic driver of Pompe disease. We are pleased to enter this agreement with Maze and look forward to continuing the advancement of this programme.”

Betsy Goodfellow

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