On 30 June 2024 at the 10th Congress of the European Academy of Neurology (EAN) 2024, during an e-presentation session focusing on motor neuron diseases, Prilenia Therapeutics presented results from the Phase II Healey ALS platform trial (NCT04297683) evaluating its oral sigma-1 receptor (S1R) activator, pridopidine, in amyotrophic lateral sclerosis (ALS) patients.

According to GlobalData’s Amyotrophic Lateral Sclerosis: Opportunity Assessment and Forecast – Update to 2029 report, developing disease-modifying treatments for this rare and aggressive disease has proved challenging. The high failure rate in ALS clinical trials is often attributed to the disease’s unclear aetiology and complex pathophysiology, resulting in a persistent unmet need for effective treatments. However, the ALS pipeline looks promising and is likely to change up to 2028 with 12 drugs in active mid to late-stage development (Phases II–III) with varying mechanisms of action.

Shared infrastructure and common protocol allow sharing of placebo data

The Healey ALS platform trial aims to accelerate the path to approval for ALS drugs by testing drugs simultaneously. Given the challenges in developing treatments for this disease, the platform trial is anticipated to find an effective therapy more quickly, with fewer total participants and fewer participants on placebo. Participants have an equal chance of being randomised to any active treatment regimen at the time of their screening. Once randomised to a regimen, participants are again randomised, this time to active drug versus placebo at a 3:1 ratio. The shared infrastructure and common protocol allow for the sharing of placebo data. New regimens are continuously added to the platform trial as new investigational products become available.

The results presented at EAN 2024 showed significant benefits in definite, probable and early (less than 18 months) ALS patients in key Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) metrics. At 24 weeks follow-up, the presented data demonstrated that pridopidine slowed disease progression by 32% (p=0.03) as defined by ALSFRS-R total score, with a 62% slowing of respiratory decline (p=0.03), an 87% slowing of decline in dyspnea (p=0.04), and a further slowing of the decline of two quantitative speech characteristics, articulation (by 93%) and speaking rates (by 65%). However, outside of the pre-specified and additional analyses, primary and secondary key analyses in the total ALS population showed no benefit from treatment with pridopidine.

Furthermore, a survival analysis of the definite, probable and early (less than 18 months) ALS patients demonstrated that approximately 10 months post-first dose, pridopidine showed a survival probability of 85% versus 50% with placebo, and an approximate doubling of survival time (from approximately 300 days to 600 days) was observed in this subgroup. The results support the positive opinion of key opinion leaders (KOLs) previously interviewed by GlobalData, who highlighted the potential positive effects of the mechanism of the S1R agonist on speech and bulbar function. KOLs believe pridopidine is likely to be positioned as adjunctive therapy in ALS patients who have bulbar onset or worse bulbar dysfunction, representing around one-quarter of all ALS patients.

Pridopidine may account for $11.1 million in sales by 2029

Beyond the Healey trial, Prilenia also enrolled its first patient into the National Institutes of Health (NIH)-sponsored Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) expanded access protocol (EAP2) (NCT06069934) at the end of March 2024. The EAP2 is expected to provide expanded access to up to 24 months of pridopidine treatment to 200 ALS patients based in the US with longer than 24 months duration of disease onset.

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GlobalData forecasts that shared infrastructure and common protocol allow for the sharing of placebo data. in the eight major markets (8MM: Canada, France, Germany, Italy, Japan, Spain, the UK and the US). GlobalData expects that 12 pipeline products will launch by 2029, contributing $375.3 million to total sales. If Pridopidine demonstrates statistically significant efficacy results in pivotal Phase III trials, GlobalData anticipates that it will account for $11.1 million in sales in 2029, as the potential add-on drug of choice for bulbar onset ALS patients. 

Prilenia is planning to use pre-specified and additional analysis data from the Healey ALS Platform Trial to inform its pivotal Phase III trial to further evaluate the efficacy and safety of pridopidine beyond the ongoing EAP2. If the Phase III outcomes follow the positive outcomes from the Healey study, they will pave the way for pridopidine to become a key part of the treatment landscape for ALS patients with the bulbar onset form of the disease.