MeiraGTx Holdings plc develops clinical-stage genetic medicines for diseases with high unmet need, with recurring updates centered on AAV gene therapy programs, ocular disease, radiation-induced xerostomia, and regulated gene-expression technology. Company news includes clinical disclosures for AAV-hAQP1 and AAV2-hAQP1 in grade 2/3 radiation-induced xerostomia, ophthalmology programs such as AAV-AIPL1 for Leber congenital amaurosis 4, and the acquired bota-vec program for X-linked retinitis pigmentosa.
Updates also cover strategic collaborations and license agreements, in-house manufacturing capabilities for genetic medicines, riboswitch platform development, operating results, financing arrangements, shareholder voting matters, and capital-structure disclosures tied to MGTX ordinary shares.
MeiraGTx Holdings plc (MGTX) reported key advancements in its clinical pipeline, including the initiation of Phase 3 trials for botaretigene sparoparvovec in collaboration with Janssen Pharmaceuticals, resulting in a $30 million milestone payment. The company also shared positive preliminary results from the Phase 1 AQUAx trial for AAV-hAQP1, aimed at treating radiation-induced xerostomia, where 6 of 7 patients showed symptom improvement. As of December 31, 2021, MeiraGTx had cash reserves of $137.7 million, ensuring operational funding through mid-2023. However, net losses increased to $79.6 million.
MeiraGTx has announced the receipt of a $30 million milestone payment from Janssen Pharmaceuticals for achieving a clinical milestone in the Phase 3 Lumeos trial of botaretigene sparoparvovec (AAV-RPGR), an investigational gene therapy targeting X-linked retinitis pigmentosa (XLRP). The trial is now actively dosing patients, and the company is also eligible for additional milestone payments related to other gene therapy programs in collaboration with Janssen. XLRP is a severe retinal disease, and this therapy aims to improve vision outcomes for patients.
On December 15, 2021, MeiraGTx Holdings plc (NASDAQ: MGTX) will conduct a virtual R&D Day showcasing its innovative gene regulation platforms. The event, led by CEO Alexandria Forbes, will cover topics such as promoter engineering and synthetic riboswitch technology. Participants can register for the event online and access a live webcast, with a replay available on the company's investor page. MeiraGTx aims to transform gene therapy with its proprietary platforms that promise precise gene expression control, targeting ocular, neurodegenerative diseases, and xerostomia.
MeiraGTx Holdings (MGTX) reported promising preliminary data from its Phase 1 AQUAx trial of AAV-hAQP1, aimed at treating grade 2/3 radiation-induced xerostomia (RIX). The results indicate that 6 out of 7 participants showed clinically meaningful improvement in xerostomia symptoms at 90 days. One participant maintained improvement at 24 months. AAV-hAQP1 demonstrated good safety and tolerability. The company plans to initiate a Phase 2 study in late 2022. RIX affects about 170,000 patients in the U.S., revealing a significant unmet medical need.
MeiraGTx Holdings plc (NASDAQ: MGTX) announced a conference call on December 7, 2021, to discuss updates on its Phase 1 AQUAx clinical trial targeting radiation-induced xerostomia. Additionally, a virtual R&D Day will occur on December 15, 2021, focusing on their gene regulation platforms. The AQUAx trial evaluates AAV-hAQP1's safety and its efficacy in improving salivary output in patients post-radiation therapy. The company is pioneering riboswitch gene regulation technology, aiming to address unmet medical needs across multiple areas, including ocular and neurodegenerative diseases.
MeiraGTx Holdings plc (Nasdaq: MGTX) reported its Q3 2021 financial results, revealing a license revenue of $6.9 million, up from $5.1 million in Q3 2020. The net loss for the quarter was $25.9 million, or $0.59 per share, compared to a loss of $6.4 million last year. The company is progressing its Phase 3 Lumeos Trial and the AQUAx Phase 1 study, while also presenting advancements in its riboswitch gene regulation platform. Despite increased R&D expenses of $21.6 million, MeiraGTx has $143.6 million in cash, sufficient for operations into Q2 2023.
MeiraGTx Holdings (NASDAQ: MGTX), a clinical stage gene therapy company, announced participation in upcoming virtual investor conferences. Key presentations include:
- Barclays Gene Editing & Gene Therapy Summit on November 15, 2021, at 2:00 p.m. ET.
- 33rd Annual Piper Sandler Healthcare Conference available from November 22 to December 2, 2021.
- Evercore ISI 4th Annual HealthCONx on November 30, 2021, at 10:05 a.m. ET.
Webcasts of these events will be accessible on their Investors page for 30 days post-presentation.
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MeiraGTx Holdings (Nasdaq: MGTX) announced that CEO Alexandria Forbes, Ph.D., will present at the virtual Chardan 5th Annual Genetic Medicines Conference on October 5, 2021, at 10:30 a.m. ET. Additionally, Dr. Forbes will participate in a panel titled “The Next Big Thing in Vector Gene Therapy—Non-traditional Vectors and Gene Regulation” at 9:30 a.m. ET on the same day.
A live webcast of the presentation will be available on the company's investor page for 30 days.
MeiraGTx is focused on gene therapy for inherited retinal diseases, neurodegenerative diseases, and xerostomia.
MeiraGTx Holdings plc (MGTX) announced new data from its Phase 1/2 trial regarding AAV5-RPGR, a gene therapy for X-linked retinitis pigmentosa (XLRP). Results show that 12 months post-treatment, retinal function improvement was observed, reversing disease progression in treated subjects. The therapy, developed in collaboration with Janssen Pharmaceuticals, was presented at the EURETINA 2021 Virtual Meeting. The company is also preparing for the pivotal Phase 3 Lumeos clinical trial. AAV5-RPGR has received Fast Track and Orphan Drug designations from the FDA.