ARDSLEY, N.Y., Dec. 17,
2019 /PRNewswire/ -- ARMGO Pharma, Inc., a clinical stage
biopharmaceutical company advancing a novel class of small molecule
drugs known as Rycals®, announced today the start of a
clinical trial using its Rycal ARM210 (also known as S48168), for
the treatment for patients with Ryanodine Receptor Type 1 Related
Myopathies (RYR1-RM). The trial is being performed in collaboration
with the National Institute of Neurological Disorders and Stroke
(NINDS) and the National Institute of Nursing Research (NINR) at
the National Institutes of Health (NIH). In 2018, the FDA granted
orphan drug designation to ARMGO for ARM210 as a potential
treatment for patients with RYR1-RM.
ARM210 is a potential disease modifying therapy for genetic
diseases, which targets the Ryanodine Receptor calcium channel
(RyR), an intracellular calcium-release channel that becomes leaky
in these and other diseases. Intracellular calcium leaks via mutant
RyR1 channels impair muscle contraction leading to muscle weakness
and loss of function, and activate toxic pathways that damage
muscle, causing the symptoms in RYR1-RM.
ARM210 is a small molecule that binds to leaky RyR channels and
repairs the leak, as demonstrated in vitro in muscle
biopsies from RYR1-RM patients. The unique mechanism of action of
ARM210 makes it an ideal potential disease modifying therapy for
RYR1-RM. Muscle biopsies of the patients in this trial have been
previously shown to respond biochemically to ARM210 in
vitro. This trial will evaluate the safety and explore the
biochemical effect of oral administration of ARM210 in these same
patients.
"This trial represents an important achievement for the
Rycal® ARM210 program as we hope to provide effective
treatment for patients with RYR1-RM, for which there are no
approved therapies," said Gene
Marcantonio MD PhD, President and Chief Medical Officer of
ARMGO Pharma. "We are looking forward to continuing the progress of
the ARM210 clinical program, building on success in RYR1-RM
patients to address other genetic mutations of RYR 1 and 2."
Further information can be found at https://clinicaltrials.gov/
Identifier: NCT04141670.
About ARMGO Pharma
ARMGO Pharma, Inc., is a privately held biopharmaceutical
company dedicated to applying targeted mechanism-based science to
the development of novel small-molecule therapeutics to treat
cardiac, musculoskeletal, and neurological disorders characterized
by leaky Ryanodine Receptor (RyR) calcium channels. Leaky RyRs are
caused by genetic mutations as well as post-translational
modifications of RyR channels. The company's proprietary drugs,
known as Rycals®, are a new class of oral agents that
repair calcium leaks through the RyR. ARMGO Pharma has been awarded
an exclusive, worldwide license from Columbia
University for its RyR technology based on the research of
founding scientist Andrew R. Marks,
M.D. The ARM210 program is supported through a Bench to Bedside
grant from the NIH (NNS19005001), and a research collaboration and
financial support from Les Laboratoires Servier.
For more information, please visit www.armgo.com
ARMGO Pharma Inc.
PMB #260, 923 Saw Mill River Road
Ardsley, NY 10502
info@armgo.com
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SOURCE ARMGO Pharma, Inc.