Sanofi, the French pharmaceutical giant, has dropped plans to license an experimental drug for Pompe disease from Maze Therapeutics, following a Federal Trade Commission (FTC) challenge of the transaction on antitrust grounds. The FTC’s administrative complaint filed on Monday framed Sanofi’s deal for Maze’s drug candidate as a large corporation’s attempt to sideline a smaller competitor.
This move comes in the wake of Sanofi’s marketing of Lumizyme and Nexviazyme for the disorder. Before this year, these were the only available treatments for Pompe disease. Both Sanofi and South San Francisco-based Maze contest the FTC’s stance. Following the FTC’s action, Sanofi has decided to terminate the licensing agreement, considering any dispute would only delay the process and not serve the best interest of patients.
Maze is now reviewing its legal and business options.
Pompe disease is caused by mutations to the acid alpha glucosidase (GAA) gene, leading to the accumulation of glycogen, a form of glucose, in skeletal, lung and cardiac muscle tissues. Left untreated, this results in a progressive worsening of muscle weakness, respiratory problems, and can prove fatal. The countermeasure for many inherited enzyme deficiencies like Pompe is enzyme replacement therapy. Being the first product for Pompe treatment, Sanofi’s Lumizyme offers patients an engineered version of GAA and costs more than $750,000 annually.
The Maze’s drug candidate MZE001, contrary to replacing the GAA, aims to slow glycogen production by blocking glycogen synthase, an enzyme responsible for producing glycogen. A twice-daily pill, MZE001, is not only more convenient for patients needing regular infusions but also less expensive to make than Sanofi’s engineered enzymes.
However, the FTC contends that Sanofi’s licensing agreement for MZE001—composed of $150 million up front and up to $600 million in milestone payments—only serves to eliminate competition. It even considers this as a case of a monopolist trying to eliminate a nascent threat. The publicly visible parts of the 17-page complaint suggest that the agreement was deliberately aimed to keep MZE001 out of reach from rival pharmaceutical companies. The FTC emphasizes that the agreement violates the anti-monopoly provisions of Section 2 of the Sherman Act.
While Sanofi saw a 7.3% annual growth in its revenue for Pompe franchise, amounting to over 1 billion euros in 2022, it attributes the increase mainly to the launch of Nexviazyme in the U.S., Europe, and Japan. On the contrary, sales of Lumizyme/Myozyme dipped due to eligible Pompe patients switching to Nexviazyme, as per the company’s annual report.
Following Sanofi’s withdrawal from developing MZE001, Maze, a privately held biotech, will need to finance development of this molecule and other drugs in its pipeline. With biotech IPOs largely closed off currently, regardless of Sanofi’s $20 million equity investment in Maze upon its initial public offering, the financial future of Maze’s pipeline looks somewhat uncertain.
The FTC’s evidentiary hearing is set for May 15, 2024. Meanwhile, the status of MZE001, which was ready for phase 2 testing, remains uncertain as long as the case is ongoing.