Maze gets new Pompe partner after FTC blocks Sanofi deal

Five months after Sanofi backed away from a proposed deal with Maze Therapeutics on a Pompe disease therapy after an antitrust challenge, Shionogi has snapped up the drug.

The Japanese pharma group has licensed Maze’s MZE001 for an upfront fee of $150 million and additional undisclosed milestone payments, adding to its pipeline of rare disease therapies.

Sanofi also said it was prepared to stump up $150 million upfront in its deal, first disclosed a year ago, with another $600 million at the back end. A few months later, however, the US Federal Trade Commission launched a lawsuit seeking to block the deal and the drugmaker abandoned its attempt.

The FTC was concerned about Sanofi’s strong position in Pompe disease with drugs like Nexviazyme/Nexviadyme (avalglucosidase alfa) and Myozyme/Lumizyme (alglucosidase alfa), which together generated $1.21 billion in sales last year.

That gave Shionogi the chance to get its hands on MZE001, which is vying to become the first oral therapy for Pompe disease, a glycogen storage disorder caused by a deficiency in the enzyme acid alfa glucosidase (GAA) that results in progressive weakness in the heart and skeletal muscles.

Current therapies are based on recombinant forms of the missing enzyme and have to be administered by intravenous infusion.

Glycogen synthase 1 (GYS1) inhibitor MZE001 works differently, inhibiting the formation of glycogen, which has to be broken down in the body by GAA to prevent toxic effects. According to Maze, that provides a more complete blockade of glycogen accumulation that should translate to improved efficacy.

The company has completed a phase 1 trial of the drug and has said the results suggest it has the potential to be used both as a monotherapy option and as an add-on therapy with enzyme replacement therapies.

“Shionogi is committed to advancing and commercializing MZE001 because they understand the potential this therapy has for patients and the unmet medical needs it could address,” said Jason Coloma, Maze’s chief executive.

“Shionogi has a track record of developing and delivering innovative medicines to patients worldwide, and we’re confident they are the right partner to continue to advance MZE001 through clinical trials so that it may reach patients with this life-threatening condition as soon as possible.”