Zagociguat for MELAS
phase 2drug program · high · Mon Aug 18 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Develop zagociguat as an oral treatment for MELAS, a rare mitochondrial disease with no effective treatments, and evaluate long-term safety and tolerability in patients who completed the PRIZM lead-in study.
Oral, once-daily, first-in-class, brain-penetrant soluble guanylate cyclase stimulator; clinical studies include a Phase 2a MELAS study, PRIZM lead-in study TIS6463-203, and open-label extension TIS6463-204 at 15 mg daily.
Open-label extension safety study TIS6463-204 for MELAS patients who completed TIS6463-203 PRIZM.
MELAS Patient Voices Interview Study
exploratoryresearch program · high · Wed Jan 01 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Characterize the patient and clinician perspective on MELAS signs, symptoms, and health-related quality-of-life impacts to inform clinical trial outcome measures.
Virtual semi-structured interviews with five clinicians and 16 adults with confirmed pathogenic variant and clinical diagnosis of MELAS; interview transcripts were coded and analyzed for concept frequency, clarification, and saturation.
Publication of patient- and clinician-reported MELAS symptom and impact findings in 2025.
Patients with MELAS could self-report symptoms and impacts. Fatigue, cognitive impairment, hearing loss, exercise intolerance, and memory problems were frequently reported, supporting fatigue and cognitive function as key clinical trial outcome measures.
CY6463 for Alzheimer's Disease with Vascular Pathology
undiscloseddrug program · medium · Tue Nov 02 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate CY6463 in participants aged 60 years or older with Alzheimer's disease and common cardiovascular risk factors.
Clinical study comparing investigational CY6463 with placebo to assess safety, tolerability, and pharmacokinetics.
Clinical trial record published for study in Alzheimer's disease with vascular pathology.
CY6463 for Stable Schizophrenia
undiscloseddrug program · medium · Fri Sep 10 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Assess CY6463 in participants with stable schizophrenia who are on stable antipsychotic medication.
Clinical study evaluating safety and tolerability of CY6463 in stable schizophrenia participants.
Clinical trial record published for stable schizophrenia safety study.
