The company — in partnership with Biogen (BIIB) — received a positive vote from the FDA Peripheral and Central Nervous System Drugs Advisory Committee meeting on tofersen, an investigational antisense medicine for the treatment of people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
SOD1-ALS is a rare genetic form of ALS that leads to the loss of everyday functions and ultimately death. The committee voted unanimously 9 to 0 for consideration of a potential accelerated approval. The second question on the substantial evidence of the effectiveness of tofersen in the treatment of patients, the committee voted 3 (yes), 5 (no) and 1 (abstain) for consideration of a potential traditional approval.
Additionally, the committee discussed both of these topics and reached consensus that the benefit-risk profile was favorable based on the review of the totality of data for tofersen in people with SOD1-ALS. In our view, with only 330 patients in the U.S. the FDA will grant a conditional approval. The PDUFA action date is April 25, 2023.
IONS — in partnership with AstraZeneca (AZN) — also announced positive topline results from the 66-week analysis of the Phase 3 NEURO-TTRansform study of eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), a debilitating and potentially fatal disease that leads to peripheral nerve damage and motor disability.
The companies are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the U.S. and plan to seek regulatory approval in Europe and other parts of the world. Earlier this month, the FDA accepted the NDA for eplontersen for the treatment of ATTRv-PN with a PDUFA action date of Dec. 22, 2023.
Finally, IONS reported that its partner Biogen presented new Phase 1b clinical data showing that IONIS-MAPTRx (BIIB080) reduced soluble tau protein in cerebrospinal fluid (CSF) in a dose-dependent and sustained manner in patients with early-stage Alzheimer’s disease (AD). Phase 2 CELIA study of IONIS-MAPTRx is in progress and currently recruiting participants in the U.S.
Biogen has been an excellent partner with their strong focus on neuro disease and their sales force has helped turn Spinrazza into a huge success. In my opinion, IONS would be a very clean fit for Biogen if they choose to pull the trigger and acquire IONS. Our best guess is that BIIB already has a plan in place and could make the deal at any time.