Raya Therapeutic Reports Positive Clinical Outcomes of Fasudil (RT1968) in ALS Patients

MONTREAL, June 17, 2024 - Raya Therapeutic Inc. ("Raya"), a company dedicated to combating ALS and other neurodegenerative diseases, has announced promising clinical data for its lead compound, fasudil (RT1968), in the treatment of Amyotrophic Lateral Sclerosis (ALS). These findings were presented in a conference abstract at the ENCALS meeting, showcasing the results of the ROCK-ALS Phase 2 clinical trial.

The ROCK-ALS Phase 2 trial was a randomized, double-blind, placebo-controlled study conducted across sites in Germany, France, and Switzerland. It involved 120 patients who were randomly assigned to receive either 30 mg of fasudil, 60 mg of fasudil, or a placebo intravenously over 20 treatment days. Patients were assessed at intervals of 45, 90, and 180 days after the start of the treatment. The primary endpoints were to evaluate the tolerability and safety of the drug. Secondary endpoints included assessments like the motor unit number index (MUNIX) and slow vital capacity (SVC), which measure respiratory function.

In the study's intention-to-treat population of 118 participants, no significant differences in safety and tolerability were observed among the groups. However, secondary outcomes revealed that fasudil 60 mg significantly reduced the decline in MUNIX at 26 and 90 days, and fasudil 30 mg showed similar effects at 90 days post-treatment initiation. Additionally, there was a noticeable slower loss of SVC favoring the 60 mg fasudil treatment at day 26. This effect was statistically significant in a post-hoc subgroup analysis of female participants at all time points.

The principal investigators of this trial concluded that fasudil treatment was well tolerated and safe for ALS patients. The MUNIX evaluation indicated a slower loss of motor units in patients treated with fasudil compared to those given a placebo, suggesting a potential therapeutic benefit that warrants further exploration in clinical trials.

These preliminary results have not yet been peer-reviewed but were shared in the conference abstract. Anjan Aralihalli, President and founder of Raya, expressed excitement about the data, emphasizing the company's commitment to developing groundbreaking therapies for ALS and other neurodegenerative disorders.

Encouraged by these findings, Raya plans to conduct more extensive studies with a larger patient group to further investigate fasudil's efficacy in ALS. Additionally, Raya has reformulated fasudil into a new form to improve its usability, particularly for the approximately 80% of ALS patients who suffer from dysphagia.

Anjan Aralihalli highlighted the strategic plans for future clinical trials for both single and combination therapies, expressing optimism about fasudil's future in Raya's pipeline. He also acknowledged the critical support from University Medical Center Göttingen (UMG), the principal sponsor of the Phase 2 study, for their pivotal role in advancing fasudil for ALS patients.

Raya is a company focused on ALS treatment, utilizing advanced techniques to develop disease-modifying therapies. Their pipeline includes five clinical-stage compounds targeting different pathways involved in motor neuron degeneration in ALS patients. Raya's diversified approach aims to develop combination therapies that could significantly impact disease progression. Last summer, Raya entered a strategic research collaboration with argenx to explore synergistic effects of their combined drug candidates, aiming to develop more effective treatment options for ALS patients. Supported by global experts, Raya plans to leverage a patient-friendly platform trial design to offer new hope in the fight against ALS.

Fasudil, a small molecule inhibitor of the rho-associated kinase (ROCK), is approved for treating subarachnoid hemorrhage in some Asian countries. Preclinical studies have shown that fasudil can attenuate neurodegeneration, modulate neuroinflammation, and promote axonal regeneration.

The University Medical Center Göttingen (UMG) in Germany is a leading university medical facility engaged in research, teaching, and patient care, facilitating the swift transfer of innovative therapy options into clinical practice.

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