MONTREAL, June 17,
2024 /CNW/ - Raya Therapeutic Inc., ("Raya") a
mission-driven company focused on the treatment of ALS and other
neurodegenerative diseases, is pleased to announce supportive
clinical data for its lead compound, fasudil (RT1968, in the
treatment of Amyotrophic Lateral Sclerosis (ALS)), as disclosed in
a conference abstract for the ENCALS meeting by the investigators
of the ROCK-ALS Phase 2 clinical trial.
As shared in the conference abstract, the ROCK-ALS
Phase 2 study was a randomized, double-blind,
placebo-controlled trial that enrolled 120 patients across sites in
Germany, France, and Switzerland. Subjects were randomly assigned
(1:1:1) to receive 30 mg of fasudil, 60 mg of fasudil, or
placebo intravenously (iv) 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),
which estimates how many motor neurons innervate a muscle, and 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 the secondary outcomes, MUNIX showed a
significantly reduced decline with Fasudil 60 mg at 26 and 90
days, and for Fasudil 30 mg at 90 days after treatment
initiation. In addition, there was a directional difference in a
slower loss of SVC favoring the 60 mg 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.
The authors, who include the principal investigators of this
investigator-initiated trial, conclude the following regarding the
results of this trial in the abstract: "Fasudil treatment was
well tolerated and safe in ALS patients. MUNIX
evaluation suggests a slower loss of motor units in fasudil-treated
patients 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and have not yet been peer-reviewed.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
trial of fasudil and remain steadfast in our mission to develop
breakthrough therapies for ALS and other neurodegenerative
disorders," said Anjan Aralihalli, President and founder of
Raya.
Based on these encouraging findings, Raya will now test fasudil
in more extensive studies with a larger patient cohort to further
assess the compound's efficacy in ALS. In addition, Raya has
reformulated fasudil into a novel formulation to enhance ease of
use, especially for the approximately 80% of ALS patients that
suffer from dysphagia.
Anjan Aralihalli continues: "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. We are especially grateful to our licensor, University
Medical Center Göttingen (UMG), for their pivotal support and
collaboration as the principal sponsor of this Phase 2 study.
Their commitment and support have been instrumental in
advancing 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 be attending the ENCALS
Annual Meeting in Stockholm,
Sweden from June 17-20.
https://www.encals.eu/meetings/stockholm/
About Raya
Raya is a mission-driven company 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 that each
target different pathways involved in motor neuron degeneration
seen in ALS patients. The compounds were in-licensed following a
rigorous selection process based on biological plausibility,
clinical target engagement and functional clinical effects
reflective of efficacy. This diversified approach may enable the
development of combination therapies which may have a significant
impact on disease progression. To develop further 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 synergistic effects of their combined
drug candidates in the hope of developing more effective treatment
options for ALS patients. Raya is further supported by global
experts and will leverage a unique patient-friendly platform trial
design offering new hope in the fight against ALS.
About Fasudil
Fasudil is a small molecule inhibitor of the 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 fosters axonal regeneration.
About UMG (University Medical
Center Göttingen)
The UMG located in Göttingen, Lower Saxony, Germany is one of the leading university
medical facilities in Germany. It
is comprised of a Medical Faculty engaged in research and teaching,
a University Hospital providing patient care and Research allowing
for the swift transfer of innovative therapy options into patient
care and education.
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SOURCE Raya Therapeutic Inc.