Sanofi has signed an exclusive worldwide licensing agreement with Maze Therapeutics for its glycogen synthase 1 (GYS1) programme, including a lead candidate for Pompe disease and other glycogen storage disorders.
Under the terms of the deal, Sanofi will pay the Californian biotech $150m in upfront cash and equity, with Maze also eligible for an additional $600m in potential development, regulatory and sales milestones plus royalties.
In exchange, Sanofi will gain the rights to further develop and commercialise MZE001 and have an exclusive licence to related GYS1-targeting back-up programmes and intellectual property.
Pompe disease is a rare genetic disorder that causes progressive weakness to the heart and skeletal muscles.
The condition is caused by mutations in the gene coding for acid alpha-glucosidase (GAA), which the body uses to break down glycogen.
MZE001, designed and developed by Maze, is an oral GYS1 inhibitor that aims to address Pompe disease by limiting glycogen accumulation.
The candidate has recently cleared its first phase 1 clinical trial, with phase 2 development due to start later this year.
Karin Knobe, global head of clinical development rare diseases and rare blood disorders at Sanofi, said: “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.”
Jason Coloma, chief executive officer of Maze, said: “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.”
In February, Sanofi announced that its long-term enzyme replacement therapy will now be available on the NHS to patients with Pompe disease, following a recommendation from the National Institute for Health and Care Excellence last year.
The company’s latest enzyme replacement therapy, Nexviadyme (avalglucosidase alfa), showed a 2.89% improvement from baseline, compared to 0.46% with its own Myozyme (alglucosidase alfa), in forced vital capacity percent-predicted, a key measure of respiratory function and the phase 3 COMET study’s primary endpoint.