Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage
biopharmaceutical company on a mission to develop treatments that
restore cognitive function, announced today clinical development
updates for CY6463, its lead program, and additional corporate
progress.
“We are developing CY6463 as a potentially
transformative medicine for cognitive impairment associated with
certain CNS diseases that are lacking effective therapies today.
The development strategy for this lead program is guided by a
robust neuroinnovation engine, designed to identify the patient
populations mostly likely to benefit from our treatments,” said
Peter Hecht, Ph.D., Chief Executive Officer of Cyclerion
Therapeutics.
Dr. Hecht continued: “We continue to execute on
our strategic priority to evaluate the clinical promise of CY6463
in multiple ongoing exploratory studies in patient populations that
we believe may be well-suited for the drug candidate’s mechanism of
action. We have recently closed enrollment on the MELAS study, and
we look forward to sharing clinical results in Q2 2022. We also
continue to progress our other ongoing CY6463 exploratory studies,
including in CIAS, and our recently initiated ADv study. Patient
recruitment rates in the CIAS study have been encouraging and we
expect topline data in H2 2022. While we drive these studies to
data readouts, we plan to advance our earlier stage CNS efforts,
and also explore additional external opportunities for value
creation.”
Clinical Pipeline Updates
Mitochondrial Encephalomyopathy, Lactic
Acidosis, and Stroke-like episodes (MELAS)
The Phase 2a MELAS trial (NCT04475549) is an
open-label, single-arm study of oral, once-daily CY6463 in adults
aged 18 or older with MELAS. The study includes measures of safety,
tolerability, pharmacokinetics, and exploratory pharmacodynamic
effects, including MRI and various disease-relevant biomarkers.
Study enrollment has closed and topline data are expected in Q2
2022.
Cognitive Impairment Associated with
Schizophrenia (CIAS)
The Phase 1 CIAS trial (NCT04972227) is a
randomized, placebo-controlled, multiple-ascending-dose study of
oral, once-daily CY6463 in adults aged 18-50 diagnosed with
schizophrenia. The study includes measures of safety, tolerability,
pharmacokinetics, and pharmacodynamics, including a broad battery
of EEG-based assessments and a computerized battery of cognitive
performance tests. Clinical sites are actively enrolling study
participants and topline data are expected in H2 2022.
Alzheimer’s disease with vascular pathology
(ADv)
The Phase 2a ADv trial (NCT04798989) is a
randomized, placebo-controlled study of oral, once-daily CY6463
over a twelve-week dosing period. Study participants must have
confirmed Alzheimer’s disease pathology as assessed by PET or CSF
biomarkers, cardiovascular risk factors, as well as
mild-to-moderate subcortical small-vessel disease as assessed by
MRI. The study will evaluate safety, tolerability, and
pharmacokinetics as well as explore the impact of CY6463 on various
disease-relevant pharmacodynamic biomarkers (e.g., EEG, MRI,
neuroinflammatory biomarkers) and cognitive performance. The ADv
study has initiated, and enrollment is ongoing.
Collaborations
- Cyclerion and Ariana Pharma
announced an artificial intelligence-driven, precision medicine
collaboration to identify patient-selection biomarkers in
neurological and neuropsychiatric diseases associated with
cognitive impairment. The collaboration aims to guide and
accelerate the clinical development of Cyclerion’s investigational
therapeutics.
Leadership Additions
- Bruce Kinon, MD has joined
Cyclerion as Vice President, Clinical Development and is leading
the ongoing CY6463 clinical efforts. Dr. Kinon has been a clinical
leader in both academic research and the pharmaceutical industry,
at Eli Lilly and Company and Lundbeck Pharmaceuticals LLC, for the
development of innovative drug treatments for neuropsychiatric
disorders and their effective delivery into clinical practice. He
received his M.D. and psychiatry training at the New York
University-Bellevue Hospital Medical Center in New York City.
Financial Position
- Cash, cash equivalents, and
restricted cash balance on December 31, 2021 was approximately $54
million, as compared to approximately $63 million on September 30,
2021.
- Research and development expenses
were approximately $37.6 million for the full year 2021, as
compared to approximately $56.4 million for the full year 2020. The
decrease of approximately $18.8 million was driven by decreases of
approximately $16.1 million in salaries and other employee-related
expenses and approximately $9.2 million of facilities and operating
costs, partially offset by increases of approximately $4.2 million
related to a non-cash write-off of leasehold improvements and
approximately $2.3 million in CY3018 external research costs.
- General and administrative expenses
were approximately $20.6 million for the full year 2021, as
compared to approximately $28.8 million for the full year 2020. The
decrease of approximately $8.2 million was driven by decreases of
approximately 5.4 million in salaries and other employee-related
expenses, approximately $2.0 million in facilities and operating
costs, and approximately $2.9 million in outside professional and
corporate expenses, partially offset by an increase of
approximately $2.1 million related to a non-cash write-off of
leasehold improvements.
- Net Loss: Net loss was
approximately $51.6 million for the full year 2021, as compared to
$77.8 million for the full year 2020.
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 Cyclerion
Therapeutics
Cyclerion Therapeutics is a clinical-stage
biopharmaceutical company on a mission to develop treatments that
restore cognitive function. Cyclerion is advancing novel,
first-in-class, CNS-penetrant, sGC stimulators that modulate a key
node in a fundamental CNS signaling pathway. The multidimensional
pharmacology elicited by the stimulation of sGC has the potential
to impact a broad range of CNS diseases. The most advanced
compound, 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),
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 Statement
This 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, including statements about the anticipated timing of
release of topline results of our clinical trials; the progression
of our clinical programs; and the business and operations of the
Company. 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 2021 Form 10-K filed on February
24, 2022, and our subsequent SEC filings. 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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