Raya Therapeutic Announces Favorable Clinical Results for Fasudil (RT1968) in Patients With ALS

MONTREAL, June 17, 2024 /PRNewswire/ – Raya Therapeutic Inc., (“Raya”), a mission-driven company focused on the treatment of ALS and other neurodegenerative diseases, is pleased to announce favorable clinical data for its lead compound, fasudil (RT1968, for the treatment of Amyotrophic Lateral Sclerosis (ALS)), as disclosed in a conference abstract for the ENCALS meeting by investigators of the ROCK-ALS phase 2 clinical trial.

As noted in the conference abstract, the Phase 2 ROCK-ALS study was a randomized, double-blind, placebo-controlled trial that enrolled 120 patients at sites across the country. Germany, FranceAnd Swiss. Subjects were randomly assigned (1:1:1) to receive 30 mg fasudil, 60 mg fasudil, or placebo intravenously (IV) for 20 days of treatment. Assessments were carried out 45, 90 and 180 days after the start of treatment. The primary endpoints were tolerability and safety, and secondary endpoints included, among others, the motor unit number index (MUNIX), which estimates the number of motor neurons innervating a muscle, and the slow vital capacity (SVC), which is a measure of respiratory function.

In the intention-to-treat population (118 participants), safety and tolerability were not significantly different between groups. In secondary endpoints, MUNIX showed a significantly reduced decline with Fasudil 60 mg at 26 and 90 days, and with Fasudil 30 mg at 90 days after treatment initiation. Additionally, there was a directional difference in slower loss of SVC favoring treatment with fasudil 60 mg at day 26, which was statistically significant in a post hoc analysis of subgroups of women at all time points. , as noted in the conference summary.

The authors, who include the principal investigators of this investigator-initiated trial, conclude the following regarding the results of this trial in the abstract: “Treatment with Fasudil was well tolerated and safe in ALS patients. MUNIX assessment suggests slower loss of motor units in patients treated with fasudil compared to placebo, suggesting a benefit of fasudil to be explored in further clinical trials“.

These preliminary results were shared in the conference abstract and have not yet been peer-reviewed.

“We are excited by the encouraging data from the ROCK-ALS fasudil trial and remain committed to our mission to develop breakthrough therapies for ALS and other neurodegenerative disorders. » said Anjan Aralihalli, Chairman and Founder of Raya.

Based on these encouraging results, Raya will now test fasudil in further studies in a larger cohort of patients to further evaluate the compound’s effectiveness in ALS. Additionally, Raya reformulated fasudil into a new formulation to improve ease of use, particularly for the approximately 80% of ALS patients who experience dysphagia.

Anjan Aralihalli continues: “With a comprehensive combination strategy on the horizon, as well as plans to initiate clinical trials next year for monotherapies and combination therapies, we are optimistic about the future prospects of fasudil and overall of our pipeline. We are especially grateful to our licensor, University Medical. Center Göttingen (UMG), for their essential support and collaboration as lead sponsor of this phase 2 study. Their commitment and support have been instrumental in the advancement of fasudil for ALS patients.

For more information about Raya and its innovative pipeline, visit www.rayatherapeutic.com or LinkedIn

Members of the Raya management team will attend the ENCALS annual meeting in Stockholm, Sweden from June 17 to 20. https://www.encals.eu/meetings/stockholm/

About Raya

Raya is a company whose mission is focused on the treatment of ALS, leveraging the latest techniques for the selection and development of disease-modifying therapies. The company has a robust pipeline of five distinct clinical-stage compounds, each targeting different pathways involved in the motor neuron degeneration seen in ALS patients. The compounds were licensed following a rigorous selection process based on biological plausibility, clinical target engagement and functional clinical effects reflecting efficacy. This diverse approach could enable the development of combination therapies that could have a significant impact on disease progression. To develop additional combination therapies, Raya entered into a strategic research collaboration with argenx last summer (https://www.prnewswire.com/in/news-releases/raya-therapeutic-announces-early-stage-rd -collaboration-with-argenx-301874999.html). This partnership explores the synergistic effects of their combination drug candidates in hopes of developing more effective treatment options for patients with ALS. Raya also benefits from the support of global experts and will leverage a unique, patient-friendly trial design, offering new hope in the fight against ALS.

About Fasudil

Fasudil is a small molecule inhibitor of rho-associated kinase (ROCK) and is approved for the treatment of subarachnoid hemorrhage in a few Asian countries. In preclinical studies, fasudil attenuates neurodegeneration, modulates neuroinflammation, and promotes axonal regeneration.

About UMG (University Medical Center Göttingen)

The UMG located in Göttingen, Lower Saxony, Germany is one of the leading academic medical institutions in Germany. It includes a medical school engaged in research and teaching, a teaching hospital providing patient care and research enabling the rapid transfer of innovative therapeutic options into patient care and education.

SOURCE Raya Therapeutics Inc.