Uploaded March 2026 | Updated September 2026, 2 weeks ago
Welcome to another compelling episode of #ExpertTalk by EverythingALS.
* This presentation is intended for the US audience.
In this session, we are joined by Nil Confer, PhD, Executive Medical Director at Prilenia Therapeutics, and Sabrina Paganoni, MD, PhD, Harvard Medical School and Massachusetts General Hospital, to discuss the PREVAiLS Study, a Phase 3 clinical trial investigating pridopidine as a potential treatment for ALS.
Dr. Confer introduces the science behind pridopidine, an investigational therapy that activates the sigma-1 receptor, a cellular pathway believed to play an important role in neuronal health and neuroprotection. He explains how this receptor helps regulate cellular stress responses, protein folding, and mitochondrial function — all processes that can be disrupted in neurodegenerative diseases like ALS.
Dr. Paganoni then reviews findings from the HEALEY ALS Platform Trial (Regimen D), where pridopidine was previously studied. While the primary endpoints were not met in the overall population, exploratory analyses suggested potential benefits in people with early-stage ALS (within 18 months of symptom onset) and definite or probable ALS diagnoses, particularly in respiratory and bulbar function measures such as speech.
These insights informed the design of the global Phase 3 PREVAiLS trial, which will enroll approximately 500 participants and evaluate functional outcomes over a 48-week placebo-controlled period followed by an open-label extension.
In this talk, we cover:
• What pridopidine is and how it targets the sigma-1 receptor
• Why this cellular pathway may be important for motor neuron protection
• Key findings from the HEALEY ALS Platform Trial
• How those results shaped the design of the PREVAiLS Phase 3 study
• Eligibility criteria and what participation in the trial involves
• The importance of global collaboration in advancing ALS therapies
Clinical trials like PREVAiLS are a critical step in the effort to develop new treatment options for ALS. By bringing together researchers, clinicians, and the ALS community, studies like this help move promising therapies through the development pipeline and closer to potential approval.
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Follow EverythingALS:
► Website: everythingals.org
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#ExpertTalk #EverythingALS #ALSResearch #ALSCommunity #NeurodegenerativeDisease #ALSScience #ALSAdvocacy #MotorNeuronDisease #ALSAwareness #ALSUpdates #ALSExperts #CollaborativeScience
🔔 If you are passionate about advancing ALS research and understanding, like, subscribe, and turn on notifications.
Sharing this episode helps expand awareness and keeps important scientific conversations moving forward.
Welcome to another compelling episode of #ExpertTalk by EverythingALS.
* This presentation is intended for the US audience.
In this session, we are joined by Nil Confer, PhD, Executive Medical Director at Prilenia Therapeutics, and Sabrina Paganoni, MD, PhD, Harvard Medical School and Massachusetts General Hospital, to discuss the PREVAiLS Study, a Phase 3 clinical trial investigating pridopidine as a potential treatment for ALS.
Dr. Confer introduces the science behind pridopidine, an investigational therapy that activates the sigma-1 receptor, a cellular pathway believed to play an important role in neuronal health and neuroprotection. He explains how this receptor helps regulate cellular stress responses, protein folding, and mitochondrial function — all processes that can be disrupted in neurodegenerative diseases like ALS.
Dr. Paganoni then reviews findings from the HEALEY ALS Platform Trial (Regimen D), where pridopidine was previously studied. While the primary endpoints were not met in the overall population, exploratory analyses suggested potential benefits in people with early-stage ALS (within 18 months of symptom onset) and definite or probable ALS diagnoses, particularly in respiratory and bulbar function measures such as speech.
These insights informed the design of the global Phase 3 PREVAiLS trial, which will enroll approximately 500 participants and evaluate functional outcomes over a 48-week placebo-controlled period followed by an open-label extension.
In this talk, we cover:
• What pridopidine is and how it targets the sigma-1 receptor
• Why this cellular pathway may be important for motor neuron protection
• Key findings from the HEALEY ALS Platform Trial
• How those results shaped the design of the PREVAiLS Phase 3 study
• Eligibility criteria and what participation in the trial involves
• The importance of global collaboration in advancing ALS therapies
Clinical trials like PREVAiLS are a critical step in the effort to develop new treatment options for ALS. By bringing together researchers, clinicians, and the ALS community, studies like this help move promising therapies through the development pipeline and closer to potential approval.
📲 Download the EverythingALS App
• Google Play: bit.ly/4nXqIYy
• App Store: bit.ly/42tPBmh
Follow EverythingALS:
► Website: everythingals.org
► X: https://x.com/everything_als
► LinkedIn: linkedin.com/everythingals
► Facebook: facebook.com/EverythingALS
► Subscribe for more Expert Talks: youtube.com/c/everythingals
#ExpertTalk #EverythingALS #ALSResearch #ALSCommunity #NeurodegenerativeDisease #ALSScience #ALSAdvocacy #MotorNeuronDisease #ALSAwareness #ALSUpdates #ALSExperts #CollaborativeScience
🔔 If you are passionate about advancing ALS research and understanding, like, subscribe, and turn on notifications.
Sharing this episode helps expand awareness and keeps important scientific conversations moving forward.
