Cyclerion Therapeutics Reports Third Quarter 2021 Financial Results and Corporate Update
First patients enrolled in study in Cognitive Impairment Associated with Schizophrenia (CIAS)
Patient screening underway in study in Alzheimer’s disease with vascular pathology (ADv)
Enrollment ongoing in study in Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS); Topline clinical results expected in H1 2022
“Cyclerion has made pipeline progress with three ongoing clinical studies related to CY6463, our lead investigational candidate. CY6463 development program is supported by completed clinical studies including a translational pharmacology study that demonstrated efficient entry into the brain and positive effects on multiple measures of brain neurophysiology associated with cognition. Our strategy is to pursue small concurrent signal-seeking studies to evaluate CY6463 in multiple CNS diseases where effective treatment options are lacking today. We plan to review the clinical data from our ongoing exploratory studies to determine how to optimally advance to later stages of development,” said
“We are pleased to have recently initiated enrollment in our CY6463 clinical study in patients with Cognitive Impairment Associated with Schizophrenia (CIAS), a debilitating aspect of schizophrenia that affects over two million patients in the
Recent Program and Business Updates
Cyclerion continues to develop CY6463 with signal-seeking studies in CIAS, ADv, and MELAS:
- The CIAS trial (NCT04972227) is a randomized, placebo-controlled, multiple-ascending dose study of oral once-daily CY6463 in up to 60 adults aged 18-50 with schizophrenia. The study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics over 14 days of dosing and includes a broad battery of EEG-based tests and computerized battery of cognitive tests. Clinical sites are actively recruiting, and the first participants have been enrolled into the study.
- The ADv trial (NCT04798989) is a randomized placebo-controlled study of oral CY6463 in approximately 30 adults older than 65 years of age to evaluate safety, tolerability, and pharmacodynamic effects (EEG, MRI, CSF biomarkers, cognition). CY6463 will be administered once-daily for 12 weeks. Participants must have confirmed AD pathology as assessed by PET and/or CSF biomarkers, cardiovascular risk factors, as well as mild-moderate subcortical small-vessel disease as assessed by MRI. The study is active and patient screening is underway.
- The MELAS trial (NCT04475549) is an open-label, single-arm study in up to 20 participants aged 18 or older to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics of oral CY6463 in MELAS. CY6463 will be administered once-daily for up to 29 days. Enrollment in the MELAS study has been slowed by several factors, including COVID-19 and disease-specific operational challenges. Cyclerion has implemented measures to improve enrollment and will continue to work with the participating centers of excellence to enroll participants through the end of 2021. Clinical data are expected in H1 2022.
In September, Cyclerion announced a publication in the
In September, Cyclerion presented at two investor conferences:
Cyclerion will participate in the upcoming
Third Quarter 2021 Financial Results
- Cash Position: Cash, cash equivalents, and restricted cash balance on
September 30, 2021was approximately $63 million, as compared to approximately $70 millionon June 30, 2021. The Company’s operating discipline, including workforce and leased space reductions, decreased cash burn rate in 2021 compared to 2020.
- Research & Development Expenses: R&D expenses were approximately
$7.0 millionfor the third quarter of 2021, as compared to approximately $13.7 millionfor the third quarter of 2020. The decrease of approximately $6.7 millionwas driven by a decrease of approximately $3.9 millionin salaries and other employee-related expenses, a decrease of approximately $2.8 millionof facilities and operating costs and a decrease of $2.0 millionrelated to completion of the STRONG-SCD study in October 2020, a decrease of $0.5 millionin discovery, offset by increases of $1.5 millionassociated with CY6463 clinical trials (MELAS, CIAS, and Adv) and $1.0 millionfor CY3018 preclinical development costs.
- General and Administrative Expenses: G&A expenses were approximately
$4.6 millionfor the third quarter of 2021, as compared to approximately $8.0 millionfor the third quarter of 2020. The decrease of $3.4 millionwas driven by a decrease of approximately $1.4 millionin salaries and other employee-related expenses, a decrease of approximately $1.5 millionin fees associated with 2020 company financing, and a decrease of approximately $0.5 millionin facilities and operating costs.
- Net Loss: Net loss was approximately
$11.3 millionfor the third quarter of 2021, as compared to $18.8 millionfor the third quarter of 2020.
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 for the treatment of serious CNS diseases.
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. 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
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Source: Cyclerion Therapeutics, Inc.