Future Thoughts and Reason for Excitement in ALS: Timothy Miller, MD, PhD


The codirector of the ALS Center at Washington University School of Medicine detailed the reasons for hope within the ALS space and the promising innovations in the coming future. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

"These trials don’t go without huge input and commitment from the participants and of course, their caregivers, and the clinical trial sites. I have the privilege of speaking to you about the trial as the lead author of this paper, but this is a team effort and includes a large group of people from multiple different organizations and the participants."

For more than 2 decades, the only approved therapies for patients with amyotrophic lateral sclerosis (ALS) that treated disease progression were riluzole (Rilutek) and edaravone (Radicava; MT Pharma). Despite this, there have been increased efforts within the field in recent years, which led to the most recent approval of AMX0035 (Relyvrio; Amylyx Pharmaceuticals) for patients with sporadic forms of the disease. On the heels of that approval, another agent, tofersen (Biogen), looks to become the first approved therapy for SOD1-mutated mediated forms of ALS.

The antisense oligonucleotide agent is currently in review by the FDA, with a decision expected to come in early Q2 2023. Part of the basis for the new drug application of tofersen was data from the phase 3 VALOR study (NCT02623699) and its open label extension, which were led by Timothy Miller, MD, PhD. VALOR, a large-scale study that included 108 participants with SOD1 ALS, showcased tofersen’s impact on neurofilament light and SOD1 protein. Although it failed to meet its primary end point of statistically significant change from baseline in Revised ALS Functional Rating Scale after 28 weeks of treatment (difference, 1.2 points; = .97), it continued to demonstrate positive effects across multiple secondary and exploratory end points that were observed over a 12-month treatment period.

Miller, the codirector of the ALS Center at Washington University School of Medicine in St. Louis, recently sat down to discuss where drug development is trending, and the reasons to be positive. Additionally, he provided perspective on the progress made within the field, the role of tofersen, and how patients have become a driving force in research.

1. Miller TM, Cudkowicz ME, Genge A, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387:1099-1110. doi:10.1056/NEJMoa2204705.
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