MeiraGTx Holdings PLC (NASDAQ: MGTX) has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia (RIX). This designation recognizes the preliminary clinical evidence of the potential benefit of AAV2-hAQP1 as a one-time treatment for the condition.
The company's phase 1 AQUAx clinical trial data, presented at the American Academy of Oral Medicine (AAOM) 2024 annual meeting, demonstrated that treatment with AAV2-hAQP1 resulted in significant improvements across three different patient-reported outcomes and in saliva production, with no treatment-related serious adverse events or dose-limiting toxicities reported.
The phase 2 AQUAx2 study (NCT05926765) continues to enroll and dose participants at multiple sites in the U.S., Canada, and the U.K. The company has gained alignment with the FDA on requirements for the ongoing phase 2 AQUAx2 clinical trial to be considered a pivotal trial in support of a potential biologics license application (BLA) filing.
Additionally, AAV2-hAQP1 was previously granted orphan drug designation by the FDA.
Grade 2/3 RIX is a severely debilitating consequence of radiation treatment for head and neck cancer, affecting approximately 30-40% of all patients treated with radiation for this type of cancer. There are currently no treatment options available for this condition.
MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. The company has internal plasmid production for GMP and two GMP viral vector production facilities, as well as an in-house quality control hub for stability and release, all fit for IND through commercial supply.
In addition, MeiraGTx has developed a proprietary manufacturing platform with leading yield and quality aspects and commercial readiness, core capabilities in viral vector design and optimization, and a transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules.
The company is focusing the riboswitch platform on the delivery of metabolic peptides, including GLP-1, GIP, glucagon, and PYY, using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. MeiraGTx aims to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high. The market has reacted to these announcements by moving the company's shares 6.5% to a price of $6.54. If you want to know more, read the company's complete 8-K report here.
