Cyclerion Announces CY6463 Clinical Pipeline Progress and First Quarter 2022 Financial Results
Characterizing the novel neuropharmacology of CY6463’s NO-sGC-cGMP signal modulation in an integrated clinical strategy currently involving three ongoing, exploratory, biomarker-rich, signal seeking studies:
- Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) study enrollment closed; topline data expected in Q2 2022
- Cognitive Impairment Associated with Schizophrenia (CIAS) study enrollment closed; topline data expected in Q3 2022
- Alzheimer’s Disease with vascular pathology (ADv) study enrollment ongoing
“We are building on three previously reported clinical studies that demonstrated a favorable safety and tolerability profile for CY6463 with exposure in the brain that led to neurophysiology changes associated with cognition, by continuing to characterize the novel neuropharmacology of NO-sGC-cGMP signal modulation and advancing CY6463 toward data readouts in three exploratory clinical studies in CNS diseases associated with cognitive impairment.” said
Clinical Pipeline Updates
Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS)
The 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 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. Study enrollment has closed, and topline data are expected in Q3 2022.
Alzheimer’s disease with vascular pathology (ADv)
The 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 is active, and enrollment is ongoing.
First Quarter 2021 Financial Results
- Cash Position: Cash, cash equivalents, and restricted cash balance on
Mar. 31, 2022was approximately $41 million, as compared to approximately $54 millionon Dec. 31, 2021.
- Research & Development Expenses: R&D expenses were approximately
$9.7 millionfor the first quarter of 2022, as compared to approximately $8.0 millionfor the first quarter of 2021. The increase of approximately $1.7 millionwas driven by increases in external research costs, partially offset by decreases in employee-related expenses and facilities and operating costs.
- General and Administrative Expenses: G&A expenses were approximately
$4 millionfor the first quarter of 2022, as compared to approximately $5.4 millionfor the first quarter of 2021. The decrease of $1.4 millionwas driven by decreases in employee-related expenses and facilities and operating costs.
- Net Loss: Net loss was approximately
$13.0 millionfor the first quarter of 2022, as compared to $13.4 millionfor the first quarter of 2021.
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.
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
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Source: Cyclerion Therapeutics, Inc.