AMT-260 / uniQure, REGENXBIO - LARVOL DELTA

uniQure Announces First Quarter 2025 Financial Results and Highlights of Recent Company Progress (GlobeNewswire) - "AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE) – The Company continues to screen patients across 12 clinical sites, with plans to activate additional centers in the second half of the year. The Company expects to present initial safety and exploratory efficacy data from the first treated patient at the Epilepsy Therapies & Diagnostics Development Symposium on Thursday, May 29, 2025....AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – In January 2025, the Company announced a favorable recommendation from the IDMC based on the review of initial safety data from the first study cohort in the Phase I/II EPISOD1 study. The Company has completed enrollment in a second dose cohort and expects to initiate enrollment in the third dose cohort in the third quarter of 2025. The Company expects to present initial data from the study in the first half of 2026."

P1/2 data • Amyotrophic Lateral Sclerosis • Epilepsy

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

Preclinical Efficacy and Safety Assessment of AMT-260, a Novel AAV9-Dual MicroRNA-Based Vector Targeting GRIK2 for the Treatment of Temporal Lobe Epilepsy (AES 2023) - " We administered the AAV9-vector carrying two small interfering RNAs targeting GRIK2 (AMT-260) bilaterally into the hippocampus of a mouse model for TLE (pilocarpine) and into non-human primates (NHP). With the preclinical efficacy in pharmacology models and a good safety profile, AMT-260 will proceed into clinical testing for the treatment of patients with refractory mesial temporal lobe epilepsy."

Preclinical • CNS Disorders • Epilepsy • GRIK2

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: uniQure France SAS | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • MRI • CNS Disorders • Epilepsy • Gene Therapies

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: uniQure France SAS

Gene therapy • MRI • New P1/2 trial • CNS Disorders • Epilepsy • Gene Therapies

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-260 Gene Therapy for Refractory Mesial Temporal Lobe Epilepsy (GlobeNewswire) - "uniQure N.V...announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-260, the Company’s gene therapy candidate for refractory mesial temporal lobe epilepsy (MTLE). AMT-260 comprises an AAV9 vector that locally delivers two engineered miRNAs designed to degrade the GRIK2 gene and suppress the aberrant expression of glutamate receptor subtype GLUK2 that is believed to trigger seizures in patients with refractory MTLE....The clinical trial is expected to begin patient screening in the fourth quarter of 2023."

IND • New P1/2 trial • CNS Disorders • Epilepsy

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

Preclinical Proof-of-Concept of AMT-260, a Novel AAV9-Dual microRNA-Based Vector Targeting GRIK2 for the Treatment of Temporal Lobe Epilepsy (ASGCT 2023) - "The vector was tested in the pilocarpine mouse model for TLE and using hippocampal organotypic slices from patients with TLE. No adverse findings were reported in the NHP. With the preclinical efficacy in TLE and a good safety profile, AMT-260 will proceed into clinical testing for the treatment of patients with refractory mesial temporal lobe epilepsy."

Preclinical • CNS Disorders • Epilepsy • GRIK2 • Napsin A

"$QURE #ASGCT23 Preclinical proof-of-concept of AMT-260, a novel AAV9-dual microRNA-based vector targeting GRIK2 for the treatment of temporal lobe epilepsy #TLE" (@floszcrxl)

Preclinical • CNS Disorders • Epilepsy • GRIK2

Preclinical Proof-of-concept of a Novel AAV9-microRNA based Targeting of GRIK2 for the Treatment of Temporal Lobe Epilepsy (AAN 2023) - "The vector has been tested in a pilocarpine mouse model for TLE and assessed for safety in non-human primates (NHP). Convection enhanced, MRI guided intrahippocampal delivery in NHP resulted in local high copies of vector and miRNA expression with substantial knockdown of GRIK2, but with no adverse findings and limited peripheral exposure to the vector. Conclusions AMT-260 shows an excellent preclinical and safety profile and will proceed into clinical testing to treat patients with refractory mesial temporal lobe epilepsy."

Preclinical • CNS Disorders • Epilepsy • GRIK2

uniQure Announces 2022 Financial Results and Highlights Recent Company Progress (BioSpace) - "The Company expects to submit an investigational new drug application and initiate a Phase I/II clinical study of AMT-260 in the second half of 2023....R&D expenses: Research and development expenses were $197.6 million for the year ended December 31, 2022, compared to $143.5 million during the same period in 2021. The increase was primarily related to advancing the clinical development of the Company’s Huntington’s disease gene therapy program, the preclinical development of the temporal lobe epilepsy program (AMT-260)..."

New P1/2 trial • CNS Disorders • Epilepsy

uniQure Highlights Therapeutic Potential of AMT-260 in Refractory Temporal Lobe Epilepsy (rTLE) at Virtual Research & Development Event (GlobeNewswire) - "uniQure N.V...hosted a virtual investor event focused on AMT-260, an AAV gene therapy for refractory temporal lobe epilepsy (rTLE) and other focal epilepsies....The presentation highlighted preclinical data that supports the safety and tolerability of AMT-260 and plans for its clinical development, as well as uniQure’s miQURE™ and linQURE™ technology platforms that allow the Company to use miRNAs to safely reduce the expression of genes in the brain....'We look forward to continuing to work toward submitting an investigational new drug application for AMT-260 and advancing it into the clinic in 2023'....uniQure is planning to conduct a Phase I/II study of AMT-260 starting in 2023."

IND • New P1/2 trial • CNS Disorders • Epilepsy

uniQure Announces Third Quarter 2022 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "AMT-260 for the treatment of refractory temporal lobe epilepsy (rTLE) – In July 2022, the Company initiated an IND-enabling GLP toxicology study in non-human primates to support an IND submission expected in 2023....R&D expenses: Research and development expenses were $48.1 million for the three months ended September 30, 2022, compared to $36.4 million during the same period in 2021. The increase was primarily related to an increase in the fair value of contingent consideration associated with the acquisition of Corlieve Therapeutics SAS, the preclinical development of temporal lobe epilepsy (AMT-260), an increase in facility-related expenses and an increase in disposable costs."

Commercial • IND • CNS Disorders • Epilepsy

uniQure Announces First Quarter 2022 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "'We continue to advance our pipeline of earlier-stage gene therapy candidates and are pleased to have recently initiated a dose-ranging IND-enabling study for AMT-260 in refractory temporal lobe epilepsy.'"

Clinical • CNS Disorders • Epilepsy

uniQure Announces 2021 Financial Results and Highlights Recent Company Progress (BioSpace) - "Refractory Temporal Lobe Epilepsy (TLE) - The Company expects to initiate a GLP toxicology study in non-human primates in the second half of 2022, which is expected to support an IND submission in 2023."

IND • Preclinical • CNS Disorders • Epilepsy

uniQure to Participate in Upcoming Industry Conferences in November (GlobeNewswire) - "uniQure N.V...announced its participation in the following upcoming investor and scientific conferences:...Virtual Neuroscience 2021 – 50th Annual Meeting, November 8 - 11, 2021."

Clinical data • CNS Disorders • Epilepsy

uniQure Announces Presentations at the Annual Meeting of The European Society of Gene and Cell Therapy (ESGCT) (GlobeNewswire) - "uniQure N.V...announced that four data presentations, including one oral presentation, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) 28th Annual Meeting taking place virtually today through October 22, 2021...AMT-260, the Company’s investigational AAV gene therapy for temporal lobe epilepsy, efficiently reduces the expression of GluK2 in cortical neurons, reduces epileptiform activity and hyperlocomotion in a preclinical model of epilepsy and blocks epileptiform discharges in organotypic slices from patients with TLE."

Preclinical • CNS Disorders • Epilepsy

uniQure to Acquire Corlieve Therapeutics and Advance its Gene Therapy Program to Treat Temporal Lobe Epilepsy (TLE) (GlobeNewswire) - "Preclinical Proof-of-Concept Data from Disease Model and from Resected Hippocampi from Refractory TLE Patients Demonstrate Clear Suppression of Chronic Spontaneous Epileptic Seizures...uniQure N.V...announced it has entered into a definitive agreement to acquire Corlieve Therapeutics and its lead program, which will be known as AMT-260, to treat temporal lobe epilepsy, the most common form of focal epilepsy."

M&A • Preclinical • CNS Disorders • Epilepsy