A year after Biogen $BIIB and Ionis $IONS nailed down an approval for Spinraza to treat spinal muscular atrophy, launching one of the most expensive drugs now on the market, the partners are coming back together to see if they can one-up themselves. And they’re doing it as a promising gene therapy makes its way to a possible near-term meet up with regulators.
Ionis bagged a $25 million upfront for the deal, which puts them back in the lab using antisense tech to identify new therapies for SMA. There are also unspecified development and regulatory milestones built into the deal, but the partners aren’t discussing that right now.
This time around, though, the partners aren’t alone in the clinic. They’re in a very real development race.
Shares of Biogen and Ionis were dinged a few days ago after rival AveXis $AVXS announced that the FDA had green-lighted a Phase I study for their gene therapy for SMA type 2. About 6 weeks ago AveXis reported out data from a small Phase I study of 15 patients treated with their gene therapy for SMA. All of the patients were able to reach 20 months without the need for constant respiratory support, or dying. And many achieved improvements in motor symptoms, speaking to the potential — though still unproven — of this as a once-and-done therapy for a rare but dire ailment.
“We are pleased to extend our collaboration with our valued colleagues at Ionis, which we believe complements our ongoing efforts to enhance and build a portfolio of treatments for SMA,” said Michel Vounatsos, chief executive officer at Biogen, in a statement. “Consistent with our commitment to individuals with SMA and their families, our aim is to increase therapeutic options available for this devastating neuromuscular disease.”
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