Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage
biopharmaceutical company on a mission to develop treatments that
restore cognitive function, will participate in a webinar hosted by
the United Mitochondrial Disease Foundation (UMDF) on Tuesday, June
28th at 8 a.m. EDT with a live Q&A. The Company will provide an
update on positive topline clinical data from the Phase 2a study of
CY6463, a first-in-class CNS-penetrant sGC stimulator, in patients
with Mitochondrial Encephalomyopathy, Lactic Acidosis and
Stroke-like episodes (MELAS). For more information about the
webinar, please visit the resources page of the UMDF website.
“UMDF welcomes every opportunity to lift up the
voices of MELAS patients and their loved ones, researchers and
medical experts who know the most about this debilitating
mitochondrial disease, which severely impacts multiple organs,
including the CNS,” said Brian Harman, President and Chief
Executive Officer of UMDF. “We are encouraged by these early CY6463
study data and look forward to discussing study findings and their
potential impact for people living with MELAS.”
Mitochondrial disease clinician-researcher and
investigator in the MELAS study, Amel Karaa, M.D., Assistant
Professor and Director of the Mitochondrial Disease Program and
Lysosomal Disorders Program at Harvard Medical School and
Massachusetts General Hospital, and 2013 UMDF fellow, will discuss
the implications and potential future impact of these data on
patients with MELAS. Dr. Karaa is a Board-certified medical
geneticist who has dedicated her professional life to treating
patients with complex medical conditions and established a
specialized program evaluating hundreds of national and
international patients with mitochondrial and lysosomal
disorders.
“These highly encouraging data provide
additional support for the therapeutic potential of CY6463 in
MELAS,” said Peter Hecht, Ph.D., Chief Executive Officer of
Cyclerion. “We are excited to learn more about the potential impact
of CY6463 in patients who are in desperate need of new therapeutic
options.”
Webinar Information For more
information about the webinar, please visit the UMDF website. The
live event can be accessed by visiting the investors' section of
the Cyclerion website
at https://ir.cyclerion.com/news-events/event-calendar. An
archived replay will also be available on both the UMDF and
Cyclerion websites.
About CY6463
CY6463 is the first CNS-penetrant sGC stimulator to be developed
as a symptomatic and potentially disease-modifying therapy for
serious CNS diseases. The nitric oxide (NO)-soluble guanylate
cyclase (sGC)-cyclic guanosine monophosphate (cGMP) signaling
pathway is a fundamental mechanism that precisely controls key
aspects of physiology throughout the body. In the CNS, the
NO-sGC-cGMP pathway regulates diverse and critical biological
functions including neuronal function, neuroinflammation, cellular
bioenergetics, and vascular dynamics. Although it has been
successfully targeted with several drugs in the periphery, this
mechanism has yet to be fully leveraged therapeutically in the CNS,
where impaired NO-sGC-cGMP signaling is believed to play an
important role in the pathogenesis of many neurodegenerative and
neuropsychiatric diseases and other disorders associated with
cognitive impairment. As an sGC stimulator, CY6463 acts as a
positive allosteric modulator to sensitize the sGC enzyme to NO,
increase the production of cGMP, and thereby amplify endogenous NO
signaling. By compensating for deficient NO-sGC-cGMP signaling,
CY6463 and other sGC stimulators may have broad therapeutic
potential as a treatment to improve cognition and function in
people with serious CNS diseases.
About the Study of CY6463 in MELAS
The Phase 2a study was an open-label, single-arm study of oral,
once-daily CY6463 in eight adults with MELAS. The primary objective
of the study was to assess the safety and tolerability of a 15
milligram, once-daily, oral dose of CY6463 over 29 days. The
secondary objectives included pharmacokinetics and exploratory
pharmacodynamic effects, with the goal of identifying which
biomarkers to carry forward into additional studies.
About MELAS
Mitochondrial Encephalomyopathy, Lactic Acidosis, and
Stroke-like episodes (MELAS) is one of the most complex orphan
diseases affecting multiple organ systems, including the CNS, with
different degrees of severity, and no approved therapies. MELAS is
caused by some of the most common mitochondrial DNA mutations
affecting the mitochondrial tRNA, and results in large clusters of
familial cases of primary mitochondrial diseases (PMD). It is
estimated that about 1 in 4,300 individuals has a mitochondrial
disease, and ~80% of individuals with mitochondrial disease have
CNS symptoms. The unmet need in MELAS is immense, symptoms can
affect virtually any organ and cause intense fatigue, muscle
weakness, and pain in addition to neurological
manifestations. Life expectancy is estimated at ~17 years from
onset of CNS symptoms. The disease impedes the individual’s
ability to live independently, leads to social isolation, and
overall reduced quality of life.
About Cyclerion
Therapeutics Cyclerion Therapeutics is a
clinical-stage biopharmaceutical company on a mission to develop
treatments that restore cognitive function. Cyclerion’s lead
molecule is CY6463, a novel, first-in-class, CNS-penetrant, sGC
stimulator that modulates a key node in a fundamental CNS signaling
network. The multidimensional pharmacology elicited by the
stimulation of sGC has the potential to impact a broad range of CNS
diseases. CY6463 has shown rapid improvement in biomarkers
associated with cognitive function and is currently in clinical
development for Alzheimer's Disease with Vascular pathology (ADv)
and Mitochondrial Encephalomyopathy, Lactic Acidosis and
Stroke-like episodes (MELAS) and Cognitive Impairment Associated
with Schizophrenia (CIAS). Cyclerion is also advancing CY3018, a
next generation sGC stimulator.
For more information about Cyclerion, please
visit https://www.cyclerion.com/ and follow us on Twitter
(@Cyclerion) and LinkedIn (www.linkedin.com/company/cyclerion).
Forward Looking StatementThis press release
contains forward-looking statements within the meaning of Section
27A of the Securities Act of 1933, as amended, and Section 21E of
the Securities Exchange Act of 1934, as amended. Our
forward-looking statements are based on current beliefs and
expectations of our management team that involve risks, potential
changes in circumstances, assumptions, and uncertainties. We may,
in some cases use terms such as “predicts,” “believes,”
“potential,” “continue,” “anticipates,” “estimates,” “expects,”
“plans,” “intends,” “may,” “could,” “might,” “likely,” “will,”
“should” or other words that convey uncertainty of the future
events or outcomes to identify these forward-looking statements.
Each forward-looking statement is subject to risks and
uncertainties that could cause actual results to differ materially
from those expressed or implied in such statement. Applicable risks
and uncertainties include the risks listed under the heading “Risk
Factors” and elsewhere in our 2020 Form 10-K filed on February
25, 2021. Investors are cautioned not to place undue reliance on
these forward-looking statements. These forward-looking statements
(except as otherwise noted) speak only as of the date of this press
release, and Cyclerion undertakes no obligation to update these
forward-looking statements, except as required by law.
InvestorsCarlo Tanzi, Ph.D.Kendall Investor
Relationsctanzi@kendallir.com
MediaAmanda SellersVerge Scientific
Communicationsasellers@vergescientific.com
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