Cyclerion Therapeutics Receives U.S. FDA Orphan Drug Designation for Zagociguat for the Treatment of Mitochondrial Diseases
March 27 2023 - 07:00AM
Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage
biopharmaceutical company on a mission to develop treatments for
serious diseases, today announced that the U.S. Food and Drug
Administration (FDA) has granted orphan drug designation to
zagociguat (previously CY6463) for the treatment of mitochondrial
diseases.
Zagociguat is the first CNS-penetrant sGC
stimulator to be developed as a symptomatic and potentially
disease-modifying therapy for serious diseases that involve the
CNS. In an open-label, 29-day study in patients with MELAS*,
zagociguat treatment was associated with improvements in multiple
disease-relevant biomarkers: mitochondrial function, inflammation,
cerebral blood flow, functional brain connectivity, and visually
evoked brain activation. These data coupled with data from
preclinical studies in cells from mitochondrial disease patients
and in zebrafish disease models support the potential of zagociguat
as a treatment for MELAS/mitochondrial diseases.
“Orphan drug designation underscores the FDA’s
recognition of zagociguat’s potential promise as a first-ever
therapy for patients with MELAS, a rare, genetic mitochondrial
disease,” said Peter Hecht, Ph.D., Chief Executive Officer of
Cyclerion. “Cyclerion is working expeditiously to advance this
potential treatment to help address the immense unmet needs of
patients with MELAS, a patient population in desperate need of
therapies.”
The FDA's Orphan Drug Designation program
provides orphan status to drugs defined as those intended for the
treatment, diagnosis or prevention of rare diseases that affect
fewer than 200,000 people in the United States. Orphan drug
designation qualifies the sponsor of the drug for certain
development incentives, including tax credits for qualified
clinical testing, prescription drug user fee exemptions, and
seven-year marketing exclusivity upon FDA approval.
About MELAS
MELAS is a complex orphan disease affecting
multiple organ systems, including the CNS, with different degrees
of severity, and no approved therapies. MELAS, one of the most
common primary mitochondrial diseases (PMDs), is caused by
mitochondrial DNA mutations resulting in large clusters of familial
cases. 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, including stroke-like episodes, encephalomyopathy,
seizures, and headaches. Life expectancy is estimated at ~17 years
from onset of CNS symptoms. The disease impedes the individual’s
ability to live independently and leads to social isolation and
overall reduced quality of life.
About Zagociguat
Zagociguat is the first CNS-penetrant sGC
stimulator to be developed as a symptomatic and potentially
disease-modifying therapy for serious diseases that involve the
CNS. 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 mitochondrial
function, neuronal function, inflammation, 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. 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 may have broad therapeutic
potential as a treatment to improve cognition and function in
people with serious diseases that involve the CNS, including
mitochondrial diseases.
About Cyclerion
Therapeutics
Cyclerion Therapeutics is a clinical-stage
biopharmaceutical company on a mission to develop treatments for
serious diseases. Cyclerion’s portfolio includes novel sGC
stimulators that modulate a key node in a fundamental signaling
network in both the CNS and the periphery. The multidimensional
pharmacology elicited by the stimulation of sGC has the potential
to impact a broad range of diseases. Zagociguat is a CNS-penetrant
sGC stimulator that has shown rapid improvements across a range of
endpoints reflecting multiple domains of disease activity,
including mitochondrial disease-associated biomarkers. CY3018 is a
CNS-targeted sGC stimulator in preclinical development that
preferentially localizes to the brain and has a pharmacology
profile that suggests its potential for the treatment of
neuropsychiatric diseases and disorders. Praliciguat is a systemic
sGC stimulator that is licensed to Akebia and being advanced in
rare kidney disease. Olinciguat is a vascular sGC stimulator that
the Company intends to out-license for cardiovascular diseases. 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 Statement
Certain matters discussed in this press release are
“forward-looking statements”. We may, in some cases, use terms such
as “predicts,” “believes,” “potential,” “continue,” “estimates,”
“anticipates,” “expects,” “plans,” “intends,” “may,” “could,”
“might,” “will,” “should”, “positive” or other words that convey
uncertainty of future events or outcomes to identify these
forward-looking statements. The forward-looking statements include
risks and uncertainties, including, but not limited to, being able
to complete clinical studies for zagociguat for the treatment of
mitochondrial diseases, including submitting additional regulatory
applications in other countries; ability to demonstrate
effectiveness of zagociguat in treating mitochondrial disease in
patients; ability to maintain and expand related intellectual
property portfolio; and statements regarding the timing of
regulatory filings regarding development programs. The factors
discussed herein could cause actual results and developments to be
materially different from those expressed in or implied by such
statements. The forward-looking statements are made only as of the
date of this press release and the Company undertakes no obligation
to publicly update such forward-looking statements to reflect
subsequent events or circumstance.
* MELAS (Mitochondrial Encephalopathy, Lactic
Acidosis, and Stroke-like episodes syndrome)
Investors and Media
Inquiries
Cyclerion Investor RelationsPhone:
857-327-8778Email: info@cyclerion.com
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