Sanofi will pay $80 million upfront to snag the ex-US license to Fulcrum Therapeutics’ rare neuromuscular disease treatment candidate, which will have Phase 3 data in the fourth quarter and potential drug approval filings in the first half of 2025.
Alex SapirThe oral small molecule, named losmapimod, is in final testing for facioscapulohumeral muscular dystrophy, a condition that impacts about 30,000 people in the US and roughly one million worldwide, Fulcrum CEO Alex Sapir said in an interview.
There are no FDA-approved treatments for the condition, and Fulcrum believes losmapimod is about three to four years ahead of other drugs in development. Avidity Biosciences, Arrowhead Pharmaceuticals, Roche and others are in the clinic.
Fulcrum is not Sanofi’s first partner for the progressive disease, which is the second most common type of muscular dystrophy. The Paris-based pharma licensed a preclinical antibody-RNA conjugate known as MC-DX4 from the small biotech miRecule in 2022. That deal featured about $30 million in upfront and near-term payments and up to $400 million in biobucks.
Boston-area Fulcrum will commercialize the drug on its own stateside, with the help of an external CDMO, Sapir said. Fulcrum will supply the drug to Sanofi, which will have the right to market it elsewhere, including in large markets like Europe and Japan.
“If you look at what Sanofi has been able to do with their neuromuscular franchise,” including Fabry disease treatment Fabrazyme, Sapir said, “that’s a $3 billion franchise globally, and they’ve got neuromuscular presence in over 100 markets outside the US.”
Sanofi has been beefing up its work in areas outside of oncology, where it has recently shed programs from a few acquisitions and pacts as it explores splitting its consumer health business, a move that other pharma giants have also taken in recent years. Last week, the French pharma said it would co-commercialize Novavax’s Covid-19 vaccine and also work on vaccines for other viruses with the biotech.
For Fulcrum, the biobuck package is worth about $975 million when factoring in Phase 3 data, regulatory approvals and sales milestones. Sanofi can also be on the hook to pay tiered royalties “starting in the low-teens,” the companies said.
The $80 million upfront will push Fulcrum’s runway from prior guidance of “into 2026” to 2027, Sapir told Endpoints News. Sanofi and Fulcrum will also split further development costs of the small molecule, which will equate to about $25 million for each company over the next “couple of years,” the CEO added.
Sapir said Fulcrum spoke with a number of different companies before landing on Sanofi. The goal was always to link up with an ex-US partner, rather than do a whole company exit.
“We brought this so far that to entertain an acquisition I don’t think would be in the best interest of shareholders, especially at the value that we’re trading at today,” Sapir said. He came to the biotech around this time last year, after serving as CEO at ReViral, acquired by Pfizer in 2022.
While Sapir has led marketing efforts in the past at drugmakers like United Therapeutics, he said Fulcrum will soon hire a chief commercial officer to build out that team. The company anticipates a 2026 launch in the US if losmapimod is approved.