- Ionis Pharmaceuticals, a California biotech developing drugs with RNA-regulating technology, said Thursday that Biogen agreed to license its experimental therapy for Alzheimer's disease.
- The therapy is in Phase 1 testing for its ability to target tau, a protein that has become a main focus area for Alzheimer's drug research. Biogen is paying $45 million upfront for the license, and could give Ionis up to $155 million more if certain milestones are hit.
- Meanwhile, an Ionis spinout named Akcea Therapeutics announced that Novartis decided against pursuing rights to an experimental RNA treatment for high triglyceride levels. Novartis opted in on another Akcea drug earlier this year and "remains a strategic partner," according to Akcea's interim CEO. The biotech expects to have Phase 2 data next year on the treatment Novartis isn't licensing.
It's been a challenging year for Ionis and Akcea. The former's blockbuster drug Spinraza (nusinersen), which was co-developed with Biogen, faces new competition from Novartis and Roche. The latter has seen investor confidence waver following the surprise exit of its CEO, president and chief operations officer in September.
Additionally, Ionis and Akcea's rare disease drug Tegsedi (inotersen) is at risk of getting edged out by rival products from Pfizer and Alnylam Pharmaceuticals just a year into its launch.
Novartis pulling back from Akcea's therapy for high triglyceride levels creates another headache, though the biotech said it will continue to develop it as a treatment for a rare condition known as familial chylomicronemia syndrome. Had Novartis decided to license the drug, it would have meant a $150 million payment to Akcea.
Paul Matteis, an analyst at Stifel, noted how the drug could have broad applications for cardiovascular diseases, but it's "probably unrealistic" that Ionis and Akcea can develop or launch the drug for such large indications. He also guessed that Novartis, fresh off a nearly $10 billion bid for The Medicines Co., is already full on cardiovascular drugs for the time being.
It's not all bad news for Ionis and its spinout, however. Novartis sees value in the companies' technology platforms as exemplified by its decision in February to license a different candidate. Biogen clearly does too, having inked three separate deals with Ionis since 2012.
The new licensure is part of a partnership between Biogen and Ionis that gives the big biotech an option to license up to three candidates before they complete a Phase 2 study. Biogen will now be responsible for further development of Ionis' tau-targeting therapy.
"Overall, we think the target here is interesting ... though relative to other Alzheimer's assets in development, we do think the threshold for clinically meaningful benefit here is higher given intrathecal dosing," Matteis wrote in a Dec. 19 note.
Phase 1/2 data on this Alzheimer's drug should become available in early 2020, according to Ionis. That's around the time Biogen said it intends to submit its closely watched Alzheimer's treatment aducanumab for U.S. approval. While Biogen has signaled that regulators seem onboard with the submission, analysts and doctors are skeptical about the complicated and divergent results the drug showed in clinical testing.
Ionis shares were down about 1.5% in late morning trading Thursday, while Akcea's were mostly unchanged.