AAV-GAD gene therapy / MeiraGTx - LARVOL DELTA

Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=13 | Active, not recruiting | Sponsor: MeiraGTx, LLC | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

AAV-GAD for the Treatment of Parkinson’s Disease (The Manila Times) - "The Company is currently engaging with clinical trial sites globally and expects to initiate the Phase 3 study of AAV-GAD in the coming months."

New P3 trial • Parkinson's Disease

A compact GAD67 promoter enables inhibitory neuron-targeted AAV gene therapy for seizure suppression. (PubMed, Mol Ther) - "Biochemical analyses confirmed increased GABA levels in the cortex and hippocampus, linking functional improvements to enhanced inhibitory neurotransmitter synthesis. Together, these findings establish the cmGAD67 promoter as a versatile platform for inhibitory neuron-targeted AAV gene delivery and identify AAV-GAD65 as a promising strategy for seizure control and disorders associated with E/I imbalance."

Journal • CNS Disorders • Epilepsy • Gene Therapies • Mood Disorders • Psychiatry

AAV-GAD for the Treatment of Parkinson’s Disease (The Manila Times) - "The Company is currently engaging with clinical trial sites globally and expects to initiate the Phase 3 study of AAV-GAD in the coming months."

New P3 trial • Parkinson's Disease

Gene therapy for Parkinson's disease: current landscape, translational challenges, and future directions. (PubMed, Expert Rev Neurother) - "The authors summarize clinical translation with emphasis on trials targeting dopamine synthesis (AAV2 AADC, ProSavin/AXO Lenti PD), neurotrophic factors (AAV2 GDNF, AAV2 NRTN), pathogenic variants (AAV9 GBA1/PR001; LRRK2 RNAi; emerging CRISPR/PINK1 strategies), and circuit modulation (AAV GAD), correlating mechanistic ration. Persistent challenges include limited vector biodistribution, reduced retrograde transport in advanced disease, immune variability, and surgical infrastructure requirements. Overcoming these via engineered capsids, delivery optimization, and validated biomarkers will enable precision, stage‑specific interventions."

Journal • Review • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease • GBA1 • LRRK2

MeiraGTx Reports First Quarter 2025 Financial and Operational Results (GlobeNewswire) - "AAV-GAD for the Treatment of Parkinson’s Disease: The Company plans to initiate a Phase 3 study of AAV-GAD in the second half of 2025 and will continue to work with the FDA to expedite the development of the program supported by MeiraGTx’s end-to-end in-house manufacturing capabilities."

New P3 trial • Parkinson's Disease

AAV-GAD for the Treatment of Parkinson’s Disease (GlobeNewswire) - "The Company plans to initiate a Phase 3 study of AAV-GAD in the second half of 2025 and will continue to work with the FDA to expedite the development of the program supported by MeiraGTx’s end-to-end in-house manufacturing capabilities."

New P3 trial • Parkinson's Disease

MeiraGTx Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for AAV-GAD for the Treatment of Parkinson’s Disease (GlobeNewswire) - "MeiraGTx Holdings plc...announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for the treatment of Parkinson’s disease not adequately controlled with anti-Parkinsonian medications....This RMAT was awarded following the presentation to the FDA of positive data from 3 clinical studies demonstrating the benefit of AAV-GAD when administered in a one-time stereotactic infusion to the subthalamic nucleus in the brain. A Phase 1 dose escalating clinical study (n=12) was conducted, followed by a double-blind, sham-controlled Phase 2 study (n=45) and a second randomized, double-blind, sham-controlled dose ranging clinical bridging study (n=14)."

FDA event • Parkinson's Disease

MeiraGTx shares jump on $200M deal with Hologen AI for Parkinson's therapy (Firstwordpharma Press Release) - "...on Thursday following an announcement that the company will partner with Hologen AI to leverage its multimodal generative AI (genAI) models to accelerate Phase III development of the biotech's AAV-GAD gene therapy for Parkinson’s disease...MeiraGTx will receive an upfront cash payment of $200 million from Hologen, and together, the partners will launch a joint venture called Hologen Neuro AI. The genAI firm will also contribute $230 million in funding for the JV, which will shepherd AAV-GAD through development to commercialisation. The capital will help support other early-stage clinical programmes targeting the central nervous system (CNS), such as AAV-BDNF for genetic obesity...MeiraGTx will retain 30% ownership in Hologen Neuro AI and take the lead on all clinical development and manufacturing. It will form exclusive clinical and commercial manufacturing supply agreements with the JV."

Licensing / partnership • Obesity • Parkinson's Disease

MeiraGTx Announces Positive Data from Randomized, Sham-controlled Clinical Bridging Study of AAV-GAD for the Treatment of Parkinson’s Disease (GlobeNewswire) - P1/2 | N=14 | NCT05603312 | Sponsor: MeiraGTx, LLC | "AAV-GAD was safe and well tolerated, with no serious adverse events (SAEs) related to AAV-GAD treatment. At Week 26, a statistically significant 18-point average improvement from baseline in UPDRS Part 3 'off' medication score was demonstrated in the high dose group (p=0.03), with no significant change in the sham or low dose groups...In the high dose AAV-GAD group, the PDQ-39 score improved by 8 points from baseline (p=0.02), the low dose group improved by 6 points from baseline (p=0.04), while the 0.2 point worsening in the sham surgery group was not statistically significant."

P1/2 data • CNS Disorders • Parkinson's Disease

A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants (clinicaltrials.gov) - P1/2 | N=14 | Completed | Sponsor: MeiraGTx, LLC | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=14 | Enrolling by invitation | Sponsor: MeiraGTx, LLC | Trial completion date: May 2028 ➔ Oct 2029 | Trial primary completion date: May 2028 ➔ Oct 2029

Trial completion date • Trial primary completion date • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants (clinicaltrials.gov) - P1/2 | N=14 | Active, not recruiting | Sponsor: MeiraGTx, LLC | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2024 ➔ Aug 2024 | Trial primary completion date: Mar 2024 ➔ Aug 2024

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

MeiraGTx Announces Third Quarter 2023 Financial and Operational Results (Yahoo Finance) - "The objective of the AAV-GAD trial (NCT05603312) is to evaluate the safety and tolerability of AAV-GAD when delivered to the subthalamic nucleus (STN) of patients with Parkinson's disease. Completion of enrollment is anticipated in the fourth quarter of 2023."

Enrollment status • CNS Disorders • Parkinson's Disease

Chemogenetic Manipulation of Dopaminergic Activity in Subthalamic Nucleus Compares Favorably to AAV-GAD in Animal Model of Parkinsons Disease (CNS Mission 2023) - No abstract available

Preclinical • CNS Disorders • Movement Disorders • Parkinson's Disease

Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=14 | Enrolling by invitation | Sponsor: MeiraGTx, LLC

New P1/2 trial • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

MeiraGTx Reports Fourth Quarter and Full Year 2022 Financial and Operational Results (GlobeNewswire) - “AAV-GAD for the Treatment of Parkinson’s Disease: The Company is now dosing patients in the AAV-GAD clinical trial under a new IND using material manufactured in its cGMP facility in London, United Kingdom using MeiraGTx’s proprietary production process….The objective of the AAV-GAD trial (NCT05603312) is to evaluate the safety and tolerability of AAV-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's disease. Completion of enrollment is anticipated by the third quarter of 2023.”

Enrollment status • CNS Disorders • Parkinson's Disease

A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants (clinicaltrials.gov) - P1/2 | N=14 | Recruiting | Sponsor: MeiraGTx, LLC | Trial completion date: Sep 2023 ➔ Mar 2024 | Trial primary completion date: Sep 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

MeiraGTx Reports Third Quarter 2022 Financial and Operational Results and Receives $25 Million Investment from Johnson & Johnson Innovation - JJDC, Inc. (Yahoo News) - "The Company is now dosing patients in the AAV-GAD Phase 1 study under a new IND using material manufactured in its cGMP facility in London, United Kingdom. The objective of the AAV-GAD trial...is to evaluate the safety and tolerability of AAV-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's disease."

Trial status • CNS Disorders • Parkinson's Disease

A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: MeiraGTx, LLC

New P1/2 trial • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

MeiraGTx Reports Second Quarter 2022 Financial and Operational Results (GlobeNewswire) - "MeiraGTx Holdings plc...announced financial results for the second quarter ended June 30, 2022, and provided an update on recent progress....The Company expects to initiate enrollment in its study of AAV-GAD during the second half of 2022 with material manufactured in its cGMP facility in London, United Kingdom."

Commercial • Trial initiation date • CNS Disorders • Parkinson's Disease

MeiraGTx Reports First Quarter 2022 Financial and Operational Results (GlobeNewswire) - "MeiraGTx Holdings plc...today announced financial results for the first quarter ended March 31, 2022 and provided an update on recent progress....The Company is filing an Investigational New Drug (IND) application in May 2022 with material manufactured in its cGMP facility in London, United Kingdom."

IND • CNS Disorders • Parkinson's Disease

MeiraGTx Reports Fourth Quarter and Full Year 2021 Financial and Operational Results (GlobeNewswire) - "MeiraGTx Holdings plc...today announced financial and operational results for the fourth quarter and full-year ended December 31, 2021, and provided a corporate update....AAV-GAD for the Treatment of Parkinson’s Disease...MeiraGTx anticipates filing an Investigational New Drug application (IND) in the coming weeks, with material that has been manufactured with our proprietary manufacturing process at the MeiraGTx cGMP manufacturing facility in London."

IND • CNS Disorders • Parkinson's Disease

MeiraGTx Reports Third Quarter 2021 Financial and Operational Results (GlobeNewswire) - “Anticipated Upcoming Milestones and Corporate Activities: File an Investigational New Drug (IND) application for AAV-GAD for the treatment of Parkinson’s Disease during the fourth quarter of 2021.”

IND • CNS Disorders • Parkinson's Disease

MeiraGTx Reports First Quarter 2021 Financial and Operational Results (GlobeNewswire) - "AAV-GAD for the Treatment of Parkinson’s Disease: The Company expects to file an Investigational New Drug (IND) application in the third quarter of 2021 with material manufactured from its cGMP facility in London."

IND • CNS Disorders • Parkinson's Disease