MeiraGTx receives FDA designations, positive data for potential therapies, and reports financial results.

From GlobeNewswire: 2024-11-13 08:00:00

MeiraGTx Holdings plc received 3 Rare Pediatric Disease Designations from the FDA for potential therapies for rare inherited retinopathies, including AAV-AIPL1. They also agreed on a pathway with MHRA for Marketing Authorization Application for AAV-AIPL1 without further clinical studies. Positive data from a study of AAV-GAD for Parkinson’s disease was announced. MeiraGTx reported financial and operational results for the third quarter of 2024 and announced plans for expedited approval pathways for their therapies.

The primary objective of the AAV-GAD study was met, showing safety and significant improvements in key efficacy endpoints at 26 weeks. AAV-GAD was safe and well tolerated, with a statistically significant 18-point improvement in UPDRS Part 3 score in the high dose group. Significant improvements in PDQ-39 scores were also observed.

MeiraGTx received Rare Pediatric Disease Designations for AAV8-RK-AIPL1, AAV8-RK-BBS10, and AAV5-RDH12 programs. These designations are granted to drugs intended to treat rare orphan diseases affecting children. MeiraGTx’s AAV8-RK-AIPL1 therapy for Leber congenital amaurosis demonstrated meaningful responses in all children treated.

MeiraGTx also announced positive data from a randomized clinical study of AAV-GAD for Parkinson’s disease. The study showed significant improvements in key efficacy endpoints, including UPDRS scores and quality of life measures. The Company is engaging with global regulatory agencies to initiate a Phase 3 registrational study for AAV-GAD.

MeiraGTx’s AAV8-RK-AIPL1 program for Leber congenital amaurosis was granted an Innovation Passport designation by the U.K. ILAP Steering Group. The program has shown promising results in children treated with the therapy, leading to plans for Marketing Authorization Application submissions in the UK and discussions with the FDA for approval in the US.

MeiraGTx’s financial results for the third quarter of 2024 showed service revenue of $10.9 million and total revenue of $10.9 million. Operating expenses, including cost of service revenue, general and administrative expenses, and research and development expenses, totaled $50.9 million. The Company reported a net loss of $39.3 million for the quarter.

MeiraGTx’s balance sheet as of September 30, 2024, showed cash and cash equivalents of $122.9 million and total assets of $299.5 million. Total liabilities were $203.8 million, and shareholders’ equity was $95.7 million. The Company believes it has sufficient capital to fund operating expenses and capital requirements into the second quarter of 2026, excluding potential milestone payments.



Read more at GlobeNewswire:: MeiraGTx Announces Third Quarter 2024 Financial and