which seeks to prevent clinical manifestation of ALS in pre-symptomatic patients diagnosed using SOD1 and filament levels. While we could see a path to approval for the drug, either with continued follow-up from the Valor study or with data from Atlas, we continue to see failure as slightly more likely. Biogen’s broad neurology portfolio and pipeline as warranting a wide moat and Ionis’ antisense technology supporting a narrow moat.
Comapany’s Future Outlook
The Valor study focuses on a small subset of ALS patients: those with the SOD1 mutation, who compose roughly 2% of ALS cases globally. Biogen and Ionis are also studying several other potential ALS drugs that are in earlier stages of development, including BIIB078, in phase 1/2 in patients with the C9Orf mutation (7% of cases, initial data expected in 2022). Biogen and Ionis are moving additional therapies for familial and sporadic (nonfamilial) forms of ALS into testing; for example, a phase 1 study of ataxin-2-targeting ION541/BIIB105 in sporadic ALS (which could address more than 75% of the broader ALS population) started in September 2020.
Ionis is independently testing ION363 in patients with the FUS mutation (even rarer than SOD1), with phase 3 data expected in 2024. In cardiometabolic diseases, Ionis has several programs in late-stage studies, including the wholly owned APOCIII program (data in 2023, 2024), and Novartis-partnered Lp(a) program (2024 data). Ionis is also poised to enter phase 3 for its PKK-targeting therapy in hereditary angioedema, a competitive niche indication where Ionis has potential to be best in class.
Company Profile
Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy. Ionis subsequently brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019).
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