LONDON and NEW YORK, Feb. 21, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the publication of results from the first-in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy. The data were published in The Lancet in a paper titled, "Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study". This paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally with rAAV8.hRKp.AIPL1 demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.
MeiraGTx Holdings plc is advancing AAV-AIPL1 for AIPL1-associated Leber congenital amaurosis 4, with a near-term BLA filing in the UK under Exceptional Circumstance possible. Bota-vec, developed with Janssen, could see a BLA filing for X-linked Retinitis Pigmentosa in 2025. Initiation of phase 3 study, using AAV-GAD for the treatment of patients with Parkinson's Disease, expected in 2025.
MeiraGTx Holdings plc has rallied over 40% following promising Parkinson's disease data, with significant potential in its Riboswitch platform for gene expression control. The company's diverse pipeline includes gene therapies for salivary gland conditions, neurological disorders, and inherited retinal diseases, with multiple BLAs expected from 2024-2027. Recent strategic deals with J&J and Sanofi have bolstered MeiraGTx's financial position, providing a runway into 2Q26 and highlighting strong investor confidence.
MeiraGTx Holdings PLC (MGTX) came out with a quarterly loss of $0.54 per share versus the Zacks Consensus Estimate of a loss of $0.47. This compares to loss of $0.74 per share a year ago.
MeiraGTx stock rises 15% as gene therapy candidate betters sham treatment in Parkinson's disease patients.
MeiraGTx (MGTX) was a big mover last session on higher-than-average trading volume. The latest trend in earnings estimate revisions might not help the stock continue moving higher in the near term.
MeiraGTx Holdings plc's GAD gene therapy for Parkinson's Disease shows promising phase 1/2 results, with significant improvements in UPDRS Part 3 and PDQ-39 scores. The company plans to discuss phase 3 study designs with regulatory agencies in Q4 2024, potentially initiating late-stage studies in 2025. MeiraGTx secured $50 million in funding, including a $30 million equity investment from Sanofi, enhancing its financial stability for future developments.
Results indicate a potent and effective locally delivered AAV-BDNF gene therapy for the treatment of obesity caused by MC4R deficiency Results indicate a potent and effective locally delivered AAV-BDNF gene therapy for the treatment of obesity caused by MC4R deficiency
MeiraGTx Holdings PLC (MGTX) came out with a quarterly loss of $0.76 per share versus the Zacks Consensus Estimate of a loss of $0.31. This compares to loss of $0.53 per share a year ago.
- Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented at the American Academy of Oral Medicine 2024 annual meeting (AAOM) showed meaningful improvements in patient-reported outcomes and saliva production with AAV2-hAQP1 treatment