Pridopidine’s Impact on ALS, Assessing Results From HEALEY ALS Platform Trial: Melanie Leitner, PhD


The consultant and founder of Accelerating NeuroVentures talked about the pridopidine arm from the HEALEY ALS Platform trial, which showed significant benefits in speech measures. [WATCH TIME: 5 minutes]

WATCH TIME: 5 minutes

"The HEALEY platform trial offers an exciting opportunity to test multiple compounds simultaneously, benefiting both patients and researchers in the ALS community. The perpetual nature of the platform trial accelerates the research process, allowing for quicker study initiation and customization of treatment regimens."

The primary goal of the Healey ALS Platform Trial (NCT04297683) is to compare the efficacy of emerging therapies to treat amyotrophic lateral sclerosis (ALS) while using a shared placebo group to help reduce recruitment and improve efficiency. The trial was created in collaboration with the FDA to ensure that positive efficacy and safety results would provide evidence to assist with facilitating regulatory approval of novel agents.1

Recently, at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27, in Boston, Massachusetts, results for primary, secondary, and exploratory endpoints from 4 of the regimens were reported by lead author Sabrina Paganoni, MD, PhD, an associate professor at Harvard Medical School, during the clinical trials plenary session.2Speakers in the session covered landmark clinical trials and how the latest results will impact patient care.

After the meeting, Melanie Leitner, PhD, program director for regimen D of the platform trial, sat down in an interview with NeurologyLive® to talk about how the trial’s patient-friendly format enhances the likelihood of patients receiving active compounds compared with traditional clinical trials. She also spoke about the primary findings of regimen D, a substudy (NCT04615923) assessing pridopidine (Preilenia), an oral agent currently in development that activates sigma-1 receptor. Leitner, consultant and founder of Accelerating NeuroVentures, discussed the therapy’s impact on speech measures and quality of life, as well as the next steps following the completion of the study. In addition, she explained how the platform trial will continue to evolve and contribute to ALS research.

Click here for more coverage on AAN 2023.

1. Paganoni S, Berry JD, Quintana M, et al. Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development. Ann Neurol. 2022;91(2):165-175. doi:10.1002/ana.26285
2. Paganoni S, Berry J, Quintana M. Results from the first four regimens of the HEALEY ALS Platform Trial. Presented at: 2023 AAN Annual Meeting; April 22-27, Boston, Massachusetts. Clinical Trials Plenary Session.
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