MELAS

Cyclerion Reports Corporate Update and Full Year 2022 Financial Results

Retrieved on: 
Wednesday, March 22, 2023
MELAS, CYCN, Brain, Neuropsychiatric Disease and Treatment, Patient, Mitochondrial disease, Research, FDA, Therapy, Pharmaceutical industry, Cryptocurrency

More recently, Cyclerion filed a request with the FDA for Orphan Drug Designation and manufactured drug product to support the Phase 2b study.

Key Points: 
  • More recently, Cyclerion filed a request with the FDA for Orphan Drug Designation and manufactured drug product to support the Phase 2b study.
  • Cash, cash equivalents, and restricted cash balance on December 31, 2022 was approximately $13.4 million, as compared to approximately $20.4 million on September 30, 2022.
  • Research and development expenses were approximately $31.5 million for the full year 2022, as compared to approximately $37.6 million for the full year 2021.
  • Net Loss: Net loss was approximately $44.1 million for the full year 2022, as compared to $51.6 million for the full year 2021.

Cyclerion Concludes Unsolicited Proposal Is Not in The Best Interest of The Company

Retrieved on: 
Tuesday, November 22, 2022
Food, Pharmacology, Akebia, Therapy, Biomarker, Food and Drug Administration, CYCN, CNS, Independent Board for Presbyterian Foreign Missions, Degenerative disease, Research, MELAS, SGC, Safety, Pharmaceutical industry

After consultation with its legal and financial advisors, the Independent Board has unanimously concluded that the proposal is not in the best interest of the Company.

Key Points: 
  • After consultation with its legal and financial advisors, the Independent Board has unanimously concluded that the proposal is not in the best interest of the Company.
  • Cyclerion Therapeutics is a clinical-stage biopharmaceutical company on a mission to develop treatments for mitochondrial diseases, including MELAS.
  • Cyclerions lead molecule is CY6463, a novel, first-in-class, CNS-penetrant sGC stimulator that modulates a key node in a fundamental signaling network.
  • For more information about Cyclerion, please visit https://www.cyclerion.com/ and follow us on Twitter ( @Cyclerion ) and LinkedIn ( www.linkedin.com/company/cyclerion ).

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022
Human voice, Deafness, MT-TL1, Learning, Mitochondrial disease, Cell, Cognition, Enzyme, Clinician, Natural history study, Mitochondrial encephalomyopathy, RAND, Leigh syndrome, Conference, Neuro, Pain, Degenerative disease, Fatigue, Exercise, CFQ, Growth, BDI, DNA, Life, Phase, Lactic acidosis, Global health, Jungian cognitive functions, Diabetes, Research, Severe cognitive impairment, Safety, Gross, Pharmacokinetics, Mutation, Heart failure, Beck, Mitochondrion, Muscle weakness, Government, Point mutation, Collection, Quality of life, Beck Depression Inventory, IIB, Pharmaceutical industry, Medical imaging, Medical device, Patient, MELAS, MIDD

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022
Human voice, Deafness, MT-TL1, Learning, Mitochondrial disease, Cell, Cognition, Enzyme, Clinician, Natural history study, Mitochondrial encephalomyopathy, RAND, Leigh syndrome, Conference, Neuro, Pain, Degenerative disease, Fatigue, Exercise, CFQ, Growth, BDI, DNA, Life, Phase, Lactic acidosis, Global health, Jungian cognitive functions, Diabetes, Research, Severe cognitive impairment, Safety, Gross, Pharmacokinetics, Mutation, Heart failure, Beck, Mitochondrion, Muscle weakness, Government, Point mutation, Collection, Quality of life, Beck Depression Inventory, IIB, Pharmaceutical industry, Medical imaging, Medical device, Patient, MELAS, MIDD

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

Cyclerion Announces Mitochondrial Disease-Focused Corporate Strategy

Retrieved on: 
Thursday, October 6, 2022
Quality of life, LinkedIn, Pharmacology, Guanylate cyclase, Müllerian agenesis, Mitochondrial disease, Nitric oxide, Degenerative disease, Fatigue, Therapy, Social isolation, Population, CGMP, Cognition, Muscle weakness, Patient, Neuropsychiatric Disease and Treatment, ADV, NO, Research, Twitter, Program, Cell, Corporation, Seizure, Inflammation, Partnership, Nasdaq, Food, Trial of the century, Company, Cognitive deficit, Food and Drug Administration, Pain, IP, Corporate development, Safety, SGC, CNS, Physiology, GLOBE, Alzheimer's disease, DNA, CYCN, FDA, Kidney, CIAS, Biomarker, Encephalopathy, Pharmaceutical industry, Medical imaging, MELAS, Schizophrenia, Akebia

CAMBRIDGE, Mass., Oct. 06, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN) today announced its mitochondrial disease-focused corporate strategic plan. Propelled by the positive data from the recently completed CY6463 Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS) clinical study, the Company believes it has a unique opportunity to deliver the first-ever approved medicine for patients suffering from rare mitochondrial diseases, a family of debilitating, progressive, and ultimately fatal genetic diseases.

Key Points: 
  • Recent positive MELAS clinical study data drive urgency to deliver potential first-ever therapy for patients with rare, genetic mitochondrial diseases
    CAMBRIDGE, Mass., Oct. 06, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN) today announced its mitochondrial disease-focused corporate strategic plan.
  • Organization: The Companys workforce is being tailored to the mitochondrial disease-focused strategy, leading to a reduction of approximately 45%, to 16 full-time employees.
  • Cyclerion Therapeutics is a clinical-stage biopharmaceutical company on a mission to develop treatments for mitochondrial diseases, including MELAS.
  • For more information about Cyclerion, please visit https://www.cyclerion.com/ and follow us on Twitter ( @Cyclerion ) and LinkedIn ( www.linkedin.com/company/cyclerion ).

Cyclerion Announces CY6463 Clinical Pipeline Progress and Second Quarter 2022 Financial Results

Retrieved on: 
Tuesday, August 9, 2022
Trial of the century, GLOBE, Mitochondrial encephalomyopathy, Harvard University, Board, Research, Patient, Pharmacology, AES, MIT, Board of directors, Development, MRI, Food, Nitric oxide, Disease, CSF, Government budget balance, CNS, Guanylate cyclase, PET, Șaeș, Safety, Pathology, Nasdaq, Alzheimer's disease, NO, Pharmacokinetics, ADV, NASA Distinguished Service Medal, EEG, Disorder, Broad Institute, Janssen Pharmaceuticals, CIAS, Therapy, Harald, Physiology, SGC, Food and Drug Administration, FDA, State Administrative Expenses, Biomarker, Cognitive deficit, Cognition, CGMP, UMDF, Company, Twitter, CYCN, LinkedIn, Administration, Pharmaceutical industry, Medical imaging, MELAS, Schizophrenia

CAMBRIDGE, Mass., Aug. 09, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function, today announced second quarter 2022 financial results and a business update.

Key Points: 
  • Dr. Hecht continued: The promising clinical results we have seen to date strongly support the advancement of CY6463, a first-in-class, CNS-penetrant sGC stimulator.
  • Research & Development Expenses: R&D expenses were approximately $10.2 million for the second quarter of 2022, as compared to approximately $12.1 million for the second quarter of 2021.
  • General and Administrative Expenses: G&A expenses were approximately $3.5 million for the second quarter of 2022, as compared to approximately $6.2 million for the second quarter of 2021.
  • Net Loss: Net loss was approximately $13.4 million for the second quarter of 2022, as compared to $16.2 million for the second quarter of 2021.

Cyclerion Therapeutics Announces Positive Topline Clinical Data for CY6463 in Patients with Cognitive Impairment Associated with Schizophrenia (CIAS)

Retrieved on: 
Thursday, July 28, 2022
Food and Drug Administration, Cognitive deficit, ERP, Ageing, CNS, Safety, Disorder, Broad Institute, MELAS, CIAS, Company, LinkedIn, Șaeș, Quantitative electroencephalography, Population, Government budget balance, CYCN, Delusion, Alzheimer's disease, Neuropsychiatry, NO, Research, Biomarker, Nitric oxide, Stanley Center for Psychiatric Research at Broad Institute, ADV, AES, Guanylate cyclase, SGC, Cognition, GLOBE, Pharmacology, EEG, CGMP, Twitter, Food, Nasdaq, Mitochondrial encephalomyopathy, Therapy, Plasma, Trial of the century, Hallucination, Psychiatry, Movement, EDT, Inflammation, Drug development, Patient, Pharmacokinetics, Physiology, Dietary supplement, Pharmaceutical industry, Medical device, Online shopping, Schizophrenia

CAMBRIDGE, Mass., July 28, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function, today announced positive topline data from its clinical study of CY6463 for the treatment of Cognitive Impairment Associated with Schizophrenia (CIAS) in individuals with stable schizophrenia on a stable, single, atypical antipsychotic regimen. Data from the 14-day, double-blind, randomized, placebo-controlled, multiple-ascending-dose study demonstrate that once-daily CY6463 was safe and well tolerated, with no reports of serious adverse events (SAEs), severe adverse events (AEs), or treatment discontinuation due to AEs. Study data demonstrate a strong effect on cognitive performance after two weeks of 15mg once-daily dosing. A broad positive movement on inflammatory biomarkers was also observed. These signals on exploratory endpoints provide further evidence of the pro-cognitive and anti-inflammatory effects of CY6463 observed in preclinical studies and prior clinical trials.

Key Points: 
  • I am encouraged by the promising cognition signals observed after only two weeks of CY6463 dosing in patients with stable schizophrenia.
  • These data demonstrate the therapeutic potential of amplifying sGC signaling in the CNS, including positive effects on cognition and inflammation, and support further development of CY6463 in diseases characterized by cognitive impairment.
  • The clinical study enrolled 48 participants with stable schizophrenia with no more than moderate positive symptoms and on a stable, single, atypical antipsychotic regimen.
  • Cognitive impairment is a core, debilitating, and untreated symptom of schizophrenia, with nearly all patients suffering from some cognitive deficits.

Cyclerion Therapeutics to Discuss CY6463 MELAS Clinical Data at a Webinar Hosted by United Mitochondrial Disease Foundation

Retrieved on: 
Wednesday, June 22, 2022
Securities Exchange Act of 1934, CNS, Social isolation, UMDF, NO, Fatigue, RNA transfection, Cognition, Safety, Alzheimer's disease, Harvard Medical School, CIAS, PMD, CYCN, Company, Cognitive deficit, Pharmacokinetics, ADV, LinkedIn, Mitochondrial encephalomyopathy, Mitochondrial disease, Massachusetts General Hospital, Guanylate cyclase, Securities Act of 1933, Disease, SGC, DNA, Government budget balance, Physiology, Pain, EDT, Drug Quality and Security Act, Quality of life, Biomarker, Pharmacology, Nasdaq, Twitter, Webcast, Patient, Board, Assistant, CGMP, Muscle weakness, Nitric oxide, Disorder, Life expectancy, Therapy, GLOBE, Pharmaceutical industry, Medical device, Dietary supplement, Schizophrenia, MELAS

CAMBRIDGE, Mass., June 22, 2022 (GLOBE NEWSWIRE) -- 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.

Key Points: 
  • 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).
  • 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.
  • 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.
  • The Phase 2a study was an open-label, single-arm study of oral, once-daily CY6463 in eight adults with MELAS.

Cyclerion Therapeutics Announces CY6463 Data Demonstrating Improved Cellular Energetics in Preclinical Models of Mitochondrial Disease

Retrieved on: 
Friday, June 17, 2022
CNS, NO, Food and Drug Administration, Poster, Cognition, Inflammation, CIAS, International Conference on Emerging Infectious Diseases, GFAP, CYCN, Company, Biology, Cognitive deficit, ADV, Research, Data, LinkedIn, Mitochondrial disease, Mitochondrial encephalomyopathy, Guanylate cyclase, SGC, Government budget balance, Physiology, Biomarker, Pharmacology, Nasdaq, Twitter, Food, Patient, Alzheimer's disease, CGMP, Conference, Nitric oxide, Disorder, Therapy, GLOBE, Medical device, Pharmaceutical industry, Schizophrenia, MELAS

CAMBRIDGE, Mass., June 17, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function, today announced research from preclinical studies demonstrating treatment with its lead soluble guanylate cyclase (sGC) stimulator, CY6463, was associated with improved cellular energetics and reduced inflammation in preclinical models of mitochondrial disease. The data will be presented today at the 10th International Conference on cGMP, taking place June 17-19, 2022, in Augsburg, Germany.

Key Points: 
  • CAMBRIDGE, Mass., June 17, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function, today announced research from preclinical studies demonstrating treatment with its lead soluble guanylate cyclase (sGC) stimulator, CY6463, was associated with improved cellular energetics and reduced inflammation in preclinical models of mitochondrial disease.
  • The data will be presented today at the 10th International Conference on cGMP, taking place June 17-19, 2022, in Augsburg, Germany.
  • Were excited to share results highlighting the beneficial impact of sGC stimulation in multiple preclinical models of mitochondrial disease, said Juli Jones, Ph.D, Head of Disease Biology at Cyclerion Therapeutics.
  • Data will be presented by Emmanuel Buys, Ph.D., Head of Network Excellence at Cyclerion Therapeutics in a poster titled, CY6463, a CNS-penetrant sGC stimulator, elicits benefits in preclinical models of mitochondrial complex 1 deficiency during todays poster session.

Cyclerion Therapeutics Announces Positive Topline Clinical Data for CY6463 in MELAS Patients at UMDF Mitochondrial Medicine 2022 Symposium

Retrieved on: 
Friday, June 10, 2022
Biomarker, Company, Government budget balance, Plasma, Inflammation, Pain, Â, Citrulline, Food and Drug Administration, CGMP, Clinical research, Disorder, Șaeș, PMD, SGC, Food, Pharming, Pharmacokinetics, Ageing, Alzheimer's disease, Trial of the century, Social isolation, Quality of life, Life expectancy, CNS, Nitric oxide, CYCN, AES, Cognition, Clinical trial, CIAS, RNA transfection, Guanylate cyclase, Cerebrospinal fluid, Patient, Chronic fatigue, Muscle weakness, Mutation, Cognitive deficit, PK, CSF, Research, Physiology, Mitochondrial encephalomyopathy, UMDF, LinkedIn, Disease, ADV, Nasdaq, Skeletal muscle, Pharmacology, NO, Therapy, GLOBE, PGIC, Twitter, Mitochondrial disease, Safety, Medical imaging, Dietary supplement, Pharmaceutical industry, Schizophrenia, MELAS

CAMBRIDGE, Mass., June 10, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function, today announced positive topline data in its signal-seeking clinical study of CY6463, for the potential treatment of Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS). Chad Glasser, Pharm.D., Director of Clinical Research at Cyclerion Therapeutics, will present results from this clinical study today during the Clinical Trial Updates Panel at the United Mitochondrial Disease Foundation (UMDF) Mitochondrial Medicine 2022 Symposium, taking place June 8-11, 2022, in Phoenix, Arizona.

Key Points: 
  • Chad Glasser, Pharm.D., Director of Clinical Research at Cyclerion Therapeutics, will present results from this clinical study today during the Clinical Trial Updates Panel at the United Mitochondrial Disease Foundation (UMDF) Mitochondrial Medicine 2022 Symposium , taking place June 8-11, 2022, in Phoenix, Arizona.
  • The positive data from this study further support the potential of CY6463, the first and only CNS-penetrant sGC stimulator in clinical development, to provide therapeutic benefit to people living with MELAS.
  • We are excited by the strength of these data and consistency across disease domains, which support the further advancement of CY6463 as a potential treatment option.
  • A video presentation of the topline data is available on the Investor page of the Cyclerion website.