Raya Therapeutics announced that its lead pipeline drug, fasudil, was well tolerated and safe, with early signs of efficacy in patients with ALS in a Phase II trial.

The San Diego-based biotech company announced the Phase II results, previously called RT1968, in an abstract for the European Network for the Cure of ALS (ENCALS) meeting in Stockholm, Sweden, on 17–20 June 2024.

The investigator-led, Phase II trial (NCT03792490) was a randomised, double-blind, placebo-controlled trial that enrolled 120 patients across sites in Germany, France and Switzerland. The trial was led by the University Medical Center Göttingen.

Patients either received 30mg fasudil, 60mg fasudil or a placebo over 20 treatment days. Assessments were performed at 45, 90 and 180 days after treatment initiation.

Primary endpoints were tolerability and safety, and secondary endpoints included, among others, motor unit number index (MUNIX) and slow vital capacity (SVC).

Safety and tolerability were not significantly different between groups. For the secondary endpoints, the 60mg fasudil group showed a significant decline in MUNIX at 26 days and 90 days, whilst the 30mg fasudil group demonstrated a significant reduction 90 days after treatment initiation.

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There was also a directional difference in a slower loss of SVC favouring the 60mg of fasudil treatment at day 26, which was statistically significant in a post-hoc subgroup analysis of females at all time points, as stated in the conference abstract.

These preliminary results were shared in the conference abstract and have not yet been peer-reviewed.

Based on the Phase II data, Raya will now test fasudil in more extensive studies with a larger patient cohort to further assess the compound’s efficacy in ALS.

In a 17 June press release, Anjan Aralihalli, president and founder of Raya said: “With a comprehensive combination strategy on the horizon, along with plans to initiate clinical trials next year for both mono and combination therapies, we are optimistic about the future prospects of fasudil and our entire pipeline.”

The ALS landscape

In April 2024, Amylyx Pharmaceuticals pulled its lead drug for ALS, Relyvrio (sodium phenylbutyrate/taurursodiol), from the market after a Phase III trial did not meet its primary endpoint.

There are currently only two drugs on the market to treat ALS: Sanofi’s Rilutek (riluzole) and Mitsubishi’s Radicava (edaravone).

There is therefore an unmet need for more novel candidates to come through the pipeline.