- Perpetual partners Biogen Inc. and Ionis Pharmaceuticals Inc. have entered another research collaboration that aims to their portfolio of spinal muscular atrophy (SMA) treatments beyond Spinraza.
- The collaboration will focus on identifying antisense oligonucleotide drug candidates for the rare muscular disease, with Ionis responsible for leading and funding the earliest stages of drug development. Once the California-based biotech has discovered a potential candidate, Biogen takes over. The ultimate goal of the collaboration is to raise up two such candidates, according to Ionis' Wade Walke, vice president or corporate communications and investor relations.
- In addition to a $25 million upfront payment, Ionis may also receive milestone payments and tiered royalties on net sales of any drug that comes to market from the collaboration. While the companies didn't put a figure to those payments, Walke told BioPharma Dive the royalties go up to the mid-20% range.
Though there have been a few bumps, Spinraza (nusinersen)'s first year on the market has been fairly positive. The drug had a better-than-expected rollout during the first quarter, and continued to rake in money over the coming months. As of Sept. 30, its revenues totaled $521 million — putting it well on the way to blockbuster status.
But Biogen and Ionis aren't resting on their laurels, possibly because competitors are gaining ground. This summer, PTC Therapeutics Inc. and Roche AG reported positive early data from the first part of a Phase 2 trial testing their drug RG7916 in patients with type 2/3 SMA. The data showed patients treated with RG7916 had a dose-dependent increase of about 400% in the ratio of full length survival motor neuron (SMN) 2 protein to delta7 mRNA. In SMA, genetic mutations lead to decreased production of SMN protein.
Ionis and Biogen have been working together on antisense therapeutics since 2012. A deal inked in December of that year had Biogen paying $30 million upfront in exchange for development of up to three gene targets. Less than 12 months later, the companies entered into a six-year research collaboration centered on discovering, developing and commercializing antisense or other therapeutics for neurological disorders.
So far, Biogen has licensed or taken an option to license seven drugs from its various collaborations with Ionis: Sprinraza, IONIS-SOD1 Rx, IONIS-MAPT Rx, IONIS-BIIB5 Rx, IONIS-BIIB6 Rx, IONIS-BIIB7Rx, and IONIS-BIIB8Rx.
Ionis’ Walke noted that, unlike in previous deals, this latest collaboration doesn’t task his company with conducting toxicology studies for Innovative New Drug (IND) applications. Biogen is picking up that work, yet will still be required to pay a licensing fee after IND-toxicology trials, according to Walke.
The new deal could also be a helpful lift to Ionis’ financials, as it offers larger royalties on any would-be product. With Spinraza, Ionis is eligible for royalties in the mid-teens, whereas it could fetch tiered royalties in the mid-20s on drugs brought to market through this latest collaboration. From Jan. 1 to the end of the third quarter, Spinraza royalties accounted $60 million of revenue for Ionis, or a little less than one-fifth of the company's total revenue for the period.
"We hadn't validated as much the technology is the CNS space as we have now with Spinraza," Walke said, adding that Ionis’ antisense platform is “much more valuable today than it was six or seven years ago."