And just like that, Santhera’s ambitions to commercialize its lead Duchenne muscular dystrophy (DMD) drug in the U.S. have vanished behind a lucrative cloak.
The Swiss biotech is selling off North American commercialization rights for registrational-stage vamorolone to Catalyst for up to $231 million, including $90 million upfront, according to an announcement Tuesday. Access to the U.S., Mexico and Canada are included in the deal.
The FDA in January accepted an approval application for the med to treat DMD and plans to decide its fate by late October.
It’s a bittersweet pill to swallow for Santhera, which can now afford to pay off all short-term debt to Highbridge but must watch any future revenue flow into another company’s pocket. Should vamorolone be approved by the FDA later this year, Santhera will net $10 million in additional revenue in milestone payments. There are also more than $100 million in sales-based milestone payments on the table.
“As part of this agreement, Santhera benefits from upfront, milestone and royalty payments which allows us to focus on the commercial roll-out of vamorolone in DMD and future other indications in Europe,” said Santhera CEO Dario Eklund in a press release.
The new funds will extend Santhera's runway into the first quarter of 2025 and allow it to pay off exchangeable notes from Highbridge, according to the announcement.
Santhera will focus resources on commercializing vamorolone in six critical European markets—France, U.K., Spain, Germany, Italy and Benelux—and will seek partners everywhere else. Catalyst has been granted the right of first negotiation to offer up commercialization rights to these additional markets plus Japan.
Santhera expects to hear a recommendation from European health advisers in the third quarter of this year with a full decision from the European Medicines Agency by the end of 2023. The treatment is also under review in the UK.
Catalyst marks the fourth company to take the vamorolone baton, emblematic of both the treatment’s potential and its debatable market opportunity in a field dominated by Sarepta Therapeutics. The drug originated with Reveragen before Actelion (now Idorsia) picked up rights that were ultimately handed over to Santhera.
The DMD indication is vamorolone’s prized direction but it’s also being developed to treat Becker muscular dystrophy and act as an alternative to steroids in multiple rare pediatric diseases. Catalyst and Santhera will team up to jointly develop any additional indications beyond DMD.