AMT-191 / uniQure - LARVOL DELTA

uniQure Announces 2024 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "A separate Type B meeting to discuss the pivotal statistical analysis plan for the BLA is anticipated to take place in the second quarter of 2025...the Company expects to provide a regulatory update in the second quarter of 2025, including the expected timing of a potential BLA submission. In February 2025, the Company completed enrollment of all 12 patients in the third cohort investigating an optimized immunosuppression regimen. The Company expects to provide an initial safety update on the third cohort in the second quarter of 2025. In the third quarter of 2025, the Company expects to present data from its ongoing Phase I/II studies of AMT-130...AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE)...The Company expects to present initial data from the study in the first half of 2026...AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS)...expects to present initial data from the study in the first half of 2026."

FDA event • FDA filing • P1/2 data • Amyotrophic Lateral Sclerosis • Epilepsy • Huntington's Disease

Extended characterization of AMT-191, an AAV gene therapy for Fabry disease (ESGCT 2024) - No abstract available

Gene therapy • Fabry Disease • Gene Therapies • Genetic Disorders

uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease (GlobeNewswire) - "uniQure...today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease....This designation supports our Phase I/IIa clinical trial and we look forward to rapidly generating clinical proof-of-concept data and providing initial data in 2025."

Orphan drug • P1/2 data • Fabry Disease

AMT-191-01: A PHASE 1/2 STUDY TO EVALUATE THE SAFETY, OF AN AAV5-BASED GENE THERAPY APPROACH IN CLASSIC FABRY DISEASE (SSIEM 2024) - P1/2 | "Discussion/Conclusion The Phase 1/2 AMT-191-01 clinical trial aims to evaluate the safety, PK/PD and exploratory efficacy of AMT-191. Palavras-chave : Fabry Disease, AMT-191, AAV5-based gene therapy, NCT06270316, uniQure"

Clinical • Gene therapy • P1/2 data • Fabry Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders

CT-AMT-191-01: Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: UniQure Biopharma B.V. | Not yet recruiting ➔ Recruiting

Enrollment open • Fabry Disease • Genetic Disorders

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: UniQure Biopharma B.V.

New P1/2 trial • Fabry Disease • Genetic Disorders

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-191 Gene Therapy for Fabry Disease (GlobeNewswire) - "uniQure N.V...announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-191, the Company’s gene therapy candidate for Fabry disease. AMT-191 comprises an AAV5 vector that delivers an α-galactosidase A (GLA) transgene designed to target the liver and produce the deficient GLA protein....'We have designed the Phase I/II study to provide dose-ranging biomarker data as rapidly and cost-effectively as possible, and we look forward to enrolling our first patient in the first half of 2024.'"

IND • New P1/2 trial • Fabry Disease • Genetic Disorders

uniQure Announces First Quarter 2023 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "Expanding the Pipeline and Progress Towards Investigational New Drug (IND) Applications: AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis...The Company expects to initiate a Phase I/II clinical study of AMT-162 in the second half of 2023; AMT-260 for the treatment of refractory temporal lobe epilepsy (rTLE) – In the third quarter of 2022, the Company initiated a GLP toxicology study of AMT-260 in non-human primates. The Company expects to submit an investigational new drug (IND) application and initiate a Phase I/II clinical study of AMT-260 in the second half of 2023; AMT-191 for the treatment of Fabry disease – In the third quarter of 2022, the Company initiated a GLP toxicology study of AMT-191 in non-human primates which is expected to support an IND submission in 2023."

IND • New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Epilepsy • Fabry Disease • Genetic Disorders