Mitochondrial encephalomyopathy

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

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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 CY6463 Clinical Pipeline Progress and Second Quarter 2022 Financial Results

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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)

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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

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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

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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

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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.

Cyclerion Therapeutics to Share CY6463 MELAS Topline Study Results, CHOP to Present CY6463 Preclinical Data at UMDF Mitochondrial Medicine 2022 Symposium

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Monday, June 6, 2022
Cognitive deficit, Biomarker, Pediatrics, Disorder, CYCN, Cognition, Research, Government budget balance, Physiology, Mitochondrial encephalomyopathy, SGC, UMDF, Pharming, LinkedIn, CIAS, ADV, Perelman School of Medicine at the University of Pennsylvania, Nasdaq, Alzheimer's disease, Guanylate cyclase, University, Pharmacology, NO, Therapy, Zebrafish, GLOBE, Program, Trial of the century, Genetics, Twitter, CGMP, Department, CNS, Clinical research, MELAS, CHOP, Nitric oxide, Medical imaging, Dietary supplement, Schizophrenia

CAMBRIDGE, Mass., June 06, 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 participation in the United Mitochondrial Disease Foundation (UMDF) Mitochondrial Medicine 2022 Symposium taking place June 8-11, 2022, in Phoenix, Arizona. Chad Glasser, Pharm.D., Director of Clinical Research at Cyclerion Therapeutics, will share topline Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS) clinical study data. The company also plans to issue a press release summarizing study results. In addition, Leonard Burg, Ph.D., from Dr. Marni Falk’s research laboratory in the Mitochondrial Medicine Frontier Program in the Division of Human Genetics in the Department of Pediatrics at Children’s Hospital of Philadelphia (CHOP) and University of Pennsylvania Perelman School of Medicine, will present a poster on preclinical data from a series of studies demonstrating functional improvement in zebrafish mitochondrial disease models treated with CY6463.

Key Points: 
  • Chad Glasser, Pharm.D., Director of Clinical Research atCyclerion Therapeutics, will share topline Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS) clinical study data.
  • The company also plans to issue a press release summarizing study results.
  • Cyclerion is advancing novel, first-in-class, CNS-penetrant, sGC stimulators that modulate a key node in a fundamental CNS signaling pathway.
  • For more information about Cyclerion, please visit https://www.cyclerion.com/ and follow us on Twitter (@Cyclerion) and LinkedIn ( www.linkedin.com/company/cyclerion ).

Precision BioSciences Announces Preclinical Data Showcasing Premier In Vivo Gene Editing Capabilities at American Society of Gene & Cell Therapy Annual Meeting

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Monday, May 16, 2022
Science, Biotechnology, Research, Pharmaceutical, Health, Infectious Diseases, Genetics, Clinical Trials, RNA transfection, HBV, Lactic acidosis, Washington Convention Center, Liver, Mitochondrial encephalomyopathy, CccDNA, Private Securities Litigation Reform Act, Animal, Mitochondrion, CAR, SEC, LNP, HAO1, Annual report, Serum, Investor, COVID-19, WT, Research, Mouse, MELAS, Growth, Achievement, DNA, Genome, Protein engineering, PH1, Infection, Insurance, PCSK9, Abstract (summary), Gene editing, Hepatitis B, Adult, Ownership, Organic acid, CTA, List, Walter E. Washington Convention Center, Hepatitis B virus, Therapy, Cell, NHP, A-DNA, PHH, Nasdaq, Ornithine transcarbamylase deficiency, Patient, Poster, OTC, Society, Privacy, HAO, Gene, AAV, Intellectual property, Forward-looking statement, Compliance, Walter, Re Recher's Will Trusts, Safety, Technology, SEC filing, Infant, Hepatocyte, Hepatitis, Mutation, Vaccine, D.C, HBsAg, IND

Data from this preclinical study demonstrate Precisions gene editing approach designed to eliminate hepatitis B virus (HBV).

Key Points: 
  • Data from this preclinical study demonstrate Precisions gene editing approach designed to eliminate hepatitis B virus (HBV).
  • These data suggest that LNP-delivered ARCUS mRNA is a promising approach and potential functional cure for chronic hepatitis B.
  • Precision will continue developing its PBGENE-HBV product candidate using LNP delivery and expects to submit an IND/CTA in 2024.
  • Preclinical data presented in this poster demonstrate Precisions gene editing approach to shift mitochondrial DNA (mtDNA) heteroplasmy for the m.3243A>G mtDNA mutation.

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

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Wednesday, May 4, 2022
Disorder, Alzheimer's disease, Brain, MRI, Twitter, Nasdaq, Cognitive deficit, Pharmacokinetics, GLOBE, Development, CNS, Government budget balance, Company, Securities Exchange Act of 1934, Securities Act of 1933, Biomarker, Safety, Cognition, CSF, CGMP, Nitric oxide, Neurophysiology, Pharmacology, CIAS, Administration, CYCN, Maritime Transportation Security Act of 2002, Guanylate cyclase, ADV, Mitochondrial encephalomyopathy, Ageing, EEG, LinkedIn, NO, Physiology, SEC, Neuropharmacology, SGC, Research, Pathology, State Administrative Expenses, Therapy, PET, Medical imaging, Pharmaceutical industry, Schizophrenia, Q2, MELAS

CAMBRIDGE, Mass., May 04, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function, announced today clinical development progress for CY6463, its lead program, and first quarter 2022 financial results.

Key Points: 
  • We have recently closed enrollment on our CY6463 studies in MELAS and CIAS and look forward to obtaining topline clinical data from these exploratory studies in Q2 and Q3 2022, respectively.
  • We have also continued to enroll our CY6463 study in Alzheimers Disease with vascular pathology, while efficiently driving Cyclerions earlier stage research programs forward.
  • The MELAS trial ( NCT04475549 ) is an open-label, single-arm study of oral, once-daily CY6463 in adults aged 18 or older with MELAS.
  • Net Loss: Net loss was approximately $13.0 million for the first quarter of 2022, as compared to $13.4 million for the first quarter of 2021.