Meiragtx Holdings Plc Stock Price, News & Analysis

MeiraGTx announced groundbreaking results published in The Lancet for their gene therapy treatment rAAV8.hRKp.AIPL1 targeting Leber Congenital Amaurosis 4 (LCA4), a severe form of inherited blindness. The clinical study demonstrated remarkable efficacy in all 11 treated children who were legally blind at birth.

The first cohort of 4 children received unilateral treatment, showing improved visual acuity from light perception to 0.9 LogMAR. A second cohort of 7 children received bilateral treatment, with all participants showing substantial benefits. The treatment demonstrated protection against progressive retinal degeneration without serious adverse effects.

Following these exceptional results, MeiraGTx plans to submit a Marketing Authorization Application to the MHRA under exceptional circumstances, with no additional clinical data required. The company is also engaging with the FDA for expedited approval in the United States. The treatment has received orphan drug and rare pediatric disease designations from the FDA and orphan designation from the European Commission.

MeiraGTx (NASDAQ: MGTX) has received Rare Pediatric Disease Designation (RPDD) from the FDA for its AAV8-RK-RetGC program, targeting Leber congenital amaurosis due to GUCY2D mutations (LCA1). This marks the company's fourth RPDD in three months, following designations for treatments of LCA4 retinal dystrophy, Bardet-Biedl syndrome, and RDH12 associated retinal dystrophy.

The RPDD program applies to diseases affecting fewer than 200,000 U.S. patients, primarily under 18 years old. If approved, MeiraGTx may receive a Priority Review Voucher (PRV), which can be used or sold, with recent PRVs selling for between $100-158 million. The company plans to leverage its manufacturing infrastructure and Specials License to accelerate the development of these treatments for childhood blinding conditions.

MeiraGTx has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AAV2-hAQP1, targeting Grade 2/3 radiation-induced xerostomia (RIX). This designation, granted to less than half of applicants, includes benefits of Fast Track and Breakthrough Therapy designations, enabling increased FDA interaction and potential accelerated approval.

The therapy showed promising results in the Phase 1 AQUAx clinical trial, demonstrating significant improvements in patient-reported outcomes and saliva production, with no treatment-related serious adverse events. The ongoing Phase 2 AQUAx2 study is a randomized, double-blind, placebo-controlled trial across multiple sites in the U.S., Canada, and U.K., which could potentially support a BLA filing.

MeiraGTx announced significant Q3 2024 developments, including three FDA Rare Pediatric Disease Designations for inherited retinal therapies. The company reported positive data from its AAV-GAD Parkinson's disease study, showing significant improvements in key efficacy endpoints. The MHRA agreed to review a Marketing Authorization Application for AAV-AIPL1 without further clinical studies. Financial highlights include $122.9 million in cash and equivalents, with funding projected into Q2 2026. Q3 service revenue was $10.9 million, with a net loss of $39.3 million ($0.55 per share), compared to $44.3 million ($0.74 per share) in Q3 2023.

MeiraGTx Holdings plc (Nasdaq: MGTX) announced five poster presentations at the European Society of Gene and Cell Therapy (ESGCT) 2024 Annual Congress in Rome, Italy. The posters highlight the company's advancements in genetic medicine and cell therapy platforms:

1. Optimized AAV manufacturing platform yielding high titers and improved product quality.
2. Novel AI-designed promoters for rod photoreceptor-specific gene expression.
3. Massively parallel screening to identify potent and tissue-specific promoters.
4. AAV-mediated gene therapy for Bardet-Biedl Syndrome 10, showing efficacy in mouse models.
5. Riboswitch-regulated gene and cell therapy allowing controlled transgene expression.

These presentations showcase MeiraGTx's progress in developing innovative genetic medicines and cell therapies for various applications, including ocular diseases and cancer treatment.

MeiraGTx Holdings plc (Nasdaq: MGTX) announced positive top-line data from its clinical bridging study of AAV-GAD for the treatment of Parkinson's disease. The study, MGT-GAD-025, was a 6-month, three-arm, randomized, double-blind, sham-controlled trial. Key findings include:

1. The primary objective of safety and tolerability was met, with no serious adverse events related to AAV-GAD treatment.

2. The high dose group showed a statistically significant 18-point average improvement in UPDRS Part 3 'off' medication score at Week 26 (p=0.03).

3. Both high and low dose groups demonstrated significant improvements in the PDQ-39 quality of life score at Week 26.

4. A dose response was observed in the PDQ-39 score, with 100% of high dose participants reporting improvement.

The company views these results as encouraging for the potential of AAV-GAD as a disease-modifying treatment for Parkinson's disease.

MeiraGTx Holdings plc (Nasdaq: MGTX) announced a $50 million offering of ordinary shares led by Sanofi and reported Q2 2024 financial results. Key highlights include:

1. Positive data from Phase 1 AQUAx study in radiation-induced xerostomia presented at AAOM 2024.

2. Innovation Passport Designation awarded for AAV8-RK-AIPL1 treatment of LCA4.

3. Enrollment continues in pivotal Phase 2 AQUAx2 clinical trial for xerostomia.

4. Anticipating results from AAV-GAD Parkinson's disease study in Q4 2024.

5. Expecting data from Phase 3 LUMEOS trial of bota-vec for XLRP in 2024.

6. Cash and equivalents of $100 million as of June 30, 2024.

7. Q2 2024 net loss of $48.6 million, or $0.76 per share.

MeiraGTx Holdings plc (Nasdaq: MGTX), a clinical stage genetic medicine company, has announced the pricing of an underwritten offering of 12,500,000 ordinary shares at $4.00 per share. The offering, expected to close on August 13, 2024, aims to raise $50 million in gross proceeds. Sanofi led the financing with a $30 million equity investment, joined by Perceptive Advisors and other institutional healthcare funds. BofA Securities is acting as the sole underwriter. The offering is made pursuant to an effective shelf registration statement filed with the SEC on December 21, 2023.

MeiraGTx reported positive data from the Phase 1 AQUAx study, received a $50 million milestone for the Phase 3 LUMEOS clinical trial, and remains on track with clinical development for various programs. The company highlighted significant progress in gene therapy programs for xerostomia and Parkinson's disease. The riboswitch platform shows promising results in metabolic disease models. Financially, the company had cash and cash equivalents of $120.3 million as of March 31, 2024, and anticipates having sufficient capital until the first quarter of 2026.