Sanofi Abandons $750M Deal for Maze Therapeutics' Pompe Disease Candidate Amid FTC Antitrust Pressure

Dec 13, 2023 12:02 CST Updated 12:02

Maze Therapeutics

Developer of Therapeutic Drugs for Genetic Diseases

Sanofi

Pharmaceutical R&D Developer

Since the acquisition of Genzyme in 2011, Sanofi has arguably maintained an absolute leading position in the field of rare disease drugs. However, once it involves the acquisition of rare disease drug companies or licensing agreements, potential monopoly issues inevitably come to the forefront.

Recently (December 12), Sanofi announced that due to the antitrust pressure from the Federal Trade Commission (FTC), it will abandon the licensing agreement with Maze Therapeutics, a company specializing in Pompe disease research and development.

Collaboration partner Maze Therapeutics reached a cooperation agreement with Sanofi in May this year. Sanofi will obtain the development and commercialization rights to Maze's Pompe disease candidate drug MZE001, which was originally scheduled to advance to Phase II clinical trials later this year. The upfront payment is $150 million, with milestone payments of $600 million.

The reason why this case involves monopoly lies in the promising prospects of the drug MZE001 in Pompe disease treatment and the sales of Sanofi's own Pompe disease therapy.

The Market Competition for Pompe Disease Treatment Drugs

Pompe disease is an autosomal recessive genetic disorder. A mutation in the gene encoding acid α-glucosidase (GAA) on chromosome 17 results in a deficiency of this enzyme, leading to an inability to break down glycogen. This causes symptoms such as muscle weakness and cardiomegaly.

Sanofi itself owns two enzyme replacement therapy pipelines for Pompe disease, holding a monopolistic position in the Pompe disease market. The first-generation product Lumizyme (alglucosidase alfa) and the second-generation product Nexviazyme (avalglucosidase alfa-ngpt), both of which require intravenous injections administered once every 1-2 weeks.

And this year, this monopolistic position has started to be challenged.

Amicus Therapeutics' Combination Therapy Pombiliti + Opfolda Approved for Marketing by European and American Regulatory Authorities This Year, Starting to Challenge Sanofi's Position.

It is worth mentioning that the founder of Amicus Therapeutics, John Crowley, isElectricityThe prototype of the movie "Extraordinary Measures," who founded a company to develop drugs after his child was diagnosed with Pompe disease, relied on WuXi Biologics for the initial development and subsequent commercialization of Pombiliti.Since John Crowley is also a former VP of Genzyme, it can be said that the current market competition will be an internal battle within the old Genzyme.

In terms of efficacy comparison, the combination of Pombiliti + Opfolda is noteworthy. Pombiliti is also an enzyme replacement therapy, while Opfolda is an enzyme stabilizer that reduces enzyme inactivation. The efficacy of this combination has been well-proven previously and outperformed Sanofi's Lumizyme in head-to-head clinical trials.

Therefore, facing challenges, Sanofi urgently needs a drug to address the current pain points of enzyme replacement therapy.

MZE001 is an oral GYS1 inhibitor that can selectively inhibit GYS1 to limit pathogenic glycogen accumulation. Since enzyme replacement therapies all require intravenous injection,Patients with Pompe disease already have mobility issues, so enzyme replacement therapy via intravenous injection poses a significant challenge to the convenience for patients.MZE001, on the other hand, only requires oral administration twice a day.Therefore, if Sanofi obtains MZE001, it may be able to crush its competitors.

Although the deal would indeed be very lucrative if completed, from the perspective of the amount involved in this transaction, it would not be worthwhile for Sanofi to stubbornly fight against the FTC.Since the sales of drugs for rare diseases like Pompe disease do have a certain ceiling, the $150 million upfront payment and $600 million in milestone payments align quite well with this ceiling. The annual sales of Lumizyme and Nexviazyme might barely qualify as blockbuster drugs with annual revenues reaching one billion dollars.

Summary

Overall, this antitrust case has indeed made Sanofi feel that being entangled in related litigation is not worthwhile. It also shows that even in some smaller disease markets, there may still be potential antitrust issues. Additionally, due to Sanofi abandoning the agreement, Maze Therapeutics' cash flow could face problems. If the deal falls through, Maze Therapeutics' operational plan may not be sustainable until 2025.

Reference Source:

https://www.sanofi.com/en/media-room/press-releases/2023/2023-12-11-21-08-20-2794272

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