Pictured: Handshake over a deal/iStock, PeopleImages

Biopharma’s biggest royalties buyer is upping its stake in a popular spinal muscular atrophy drug. Royalty Pharma announced Thursday a $1 billion upfront payment to beleaguered PTC Therapeutics for additional royalties in Evrysdi, licensed and marketed by Roche. 

Royalty initially bought into Evrysdi in July 2020, just before its approval by the FDA. The drug has already been used to treat over 11,000 patients worldwide. Spinal muscular atrophy (SMA) is a rare and often fatal genetic disease that typically presents in infants and young children.  

The deal increases Royalty’s ownership of the Evrysdi royalty from 43% to 81%, equating to 13% of the royalties Roche pays on worldwide net product sales. Roche reported $801.4 million in sales for the first half of 2023. GlobalData predicts sales to rise to just under $3 billion in 2029. 

In Thursday’s announced deal, PTC has the option to sell the remainder of the Evrysdi royalty to Royalty for up to $500 million until the end of 2025. Otherwise, Royalty has three months after that to buy half of the retained royalties for up to $250 million. 

The billion-dollar boost to PTC Therapeutics is a much needed one. The biopharma has suffered setbacks of late as its investigational drug vatiquinone missed the mark in two different indications— mitochondrial disease-associated seizures and Friedrich’s ataxia.  

PTC is also scrambling to keep its Duchenne muscular dystrophy therapy on the market in Europe after the EMA refused to convert Translarna’s conditional marketing authorization to a full approval. The company announced it will be requesting a re-examination, as it slashed its workforce by around 25% and reprioritizes even further.  

The oral Evrysdi therapy is priced by patient weight—around $100,000 for an infant with a cap of $340,000 per year. The cost is substantially less than that of its gene therapy competitors, Biogen’s Spinraza and Novartis’ Zolgensma.  

Spinraza is also in Royalty’s portfolio since Ionis sold a portion of its interest to the biopharma funder in January 2023. Royalty paid $500 million upfront for the royalties to both Spinraza and Novartis’ pelacarsen, a potential treatment in development for reducing cardiovascular disease risk.  

In August 2023, Royalty dropped $300 million for royalties to Ferring Pharmaceuticals already-approved gene therapy for bladder cancer, Adstiladrin.