LONDON and NEW YORK - MeiraGTx Holdings plc (NASDAQ:MGTX), a clinical stage genetic medicines company with a market capitalization of approximately $480 million, announced today that its gene therapy candidate AAV2-hAQP1 has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of Grade 2/3 radiation-induced xerostomia (RIX), a severe side effect of radiation therapy in head and neck cancer patients. According to InvestingPro data, the company generated revenue of $13.93 million in the last twelve months while maintaining a healthy liquidity position with a current ratio of 2.34.
The RMAT designation is a significant regulatory milestone that reflects the preliminary clinical evidence suggesting AAV2-hAQP1's potential as a one-time treatment for RIX. It is a condition with no current treatment options, affecting a significant portion of the over 170,000 patients in the U.S. who have undergone radiation for head and neck cancer, with an additional 15,000 patients diagnosed annually. This development has contributed to MGTX's strong market performance, with the stock showing a 28.7% gain over the past six months.
AAV2-hAQP1's clinical promise was supported by data from the Phase 1 AQUAx clinical trial, presented at the American Academy of Oral Medicine's annual meeting in April 2024. The trial showed significant patient-reported improvements and increased saliva production, without serious adverse events or dose-limiting toxicities.
The RMAT designation, part of the 21st Century Cures Act, is designed to expedite the development and review process for promising regenerative medicine therapies. It includes benefits similar to Fast Track and Breakthrough Therapy designations, such as increased FDA interaction, rolling review, and potential for Priority Review of the Biologics License Application (BLA).
MeiraGTx's ongoing Phase 2 AQUAx2 study, a randomized, double-blind, placebo-controlled trial, continues to enroll participants in the U.S., Canada, and the U.K., and is aligned with FDA requirements to potentially support a future BLA filing.
The company, with in-house manufacturing facilities, aims to leverage the RMAT designation to accelerate the development pathway for AAV2-hAQP1. This includes frequent regulatory interactions and comprehensive discussions on the product development program, clinical trials, and manufacturing strategy. While InvestingPro analysis indicates the company is currently burning through cash with net losses, it maintains moderate debt levels and strong liquid assets. For deeper insights into MGTX's financial health and growth prospects, including 8 additional ProTips and comprehensive valuation metrics, investors can access the detailed Pro Research Report available on InvestingPro.
The information in this article is based on a press release statement.
In other recent news, MeiraGTx Holdings has reported encouraging results from its latest clinical trial, MGT-GAD-025, focused on treating Parkinson’s disease. The study demonstrated significant improvements in key efficacy endpoints for patients with idiopathic Parkinson's disease. The company plans to engage with regulators in the US, Europe, and Japan with the aim of initiating a Phase 3 study.
Financial analysts have also weighed in on MeiraGTx's prospects. Piper Sandler maintained its Overweight rating on the company's shares, noting potential near-term catalysts and an anticipated milestone payment of $285 million from a partnership with Johnson & Johnson. RBC Capital adjusted its price target for MeiraGTx to $9.00, while maintaining an Outperform rating.
MeiraGTx has also announced a public offering of 12.5 million ordinary shares at $4 each, with the intention to generate $50 million for general corporate purposes, including research and development expenses and clinical trial costs. Chardan Capital Markets initiated coverage on MeiraGTx with a Buy rating, expressing confidence in the company's gene therapy pipeline. These are among the recent developments that underscore the ongoing financial and research activities of MeiraGTx.
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