EMERYVILLE, CA – 4D Molecular Therapeutics, Inc. (NASDAQ:FDMT), a biotechnology company specializing in gene therapy products, announced encouraging interim results from its Phase 2 clinical trial of 4D-150 for wet age-related macular degeneration (wet AMD (NASDAQ:AMD)). These results were presented today at the American Society of Retina Specialists Annual Scientific Meeting in Stockholm, Sweden.
The trial, known as PRISM, is evaluating the safety and efficacy of 4D-150, a gene therapy product administered via intravitreal injection. The Population Extension cohort of the trial included 45 patients with varying degrees of disease activity. Patients were divided into two dosage arms, with 30 receiving the planned Phase 3 dose of 3E10 vg/eye and 15 receiving a lower dose of 1E10 vg/eye.
As of the most recent data cutoff on June 24, 2024, the interim analysis showed that 4D-150 was safe and well-tolerated at both doses. No serious adverse events or significant inflammation were reported. Additionally, the treatment demonstrated a substantial reduction in the need for anti-VEGF injections, which are the current standard of care for wet AMD.
Patients in the planned Phase 3 dose arm showed an 89% reduction in the mean annualized injection rate, with 77% remaining injection-free. The low-dose arm also exhibited a 91% reduction with 60% of patients not requiring additional injections.
The 24-week landmark analysis also revealed improvements in visual acuity, with patients in the higher dose arm experiencing an average improvement of +4.2 ETDRS letters from baseline. Retinal anatomy outcomes were favorable, with sustained and greater control over retinal thickness, especially in patients who did not require supplemental injections.
Long-term follow-up results from the Phase 1 trial of 4D-150 were also positive, with all three patients treated with the higher dose remaining injection-free for approximately 2 to 2.5 years. Their visual acuity and retinal thickness remained stable or improved from baseline.
4D Molecular Therapeutics is working with regulatory agencies in the U.S. and Europe to finalize the design of its Phase 3 clinical trial for 4D-150, with an expected announcement in September 2024 and trial commencement in the first quarter of 2025. Further results from the ongoing trials for both wet AMD and diabetic macular edema (DME) are anticipated in the coming months.
The information is based on a press release statement and the company's SEC filing.
In other recent news, 4D Molecular Therapeutics has been the subject of various analyst reports, following significant upcoming events and strategic developments.
RBC Capital Markets has reiterated its Outperform rating on the biotechnology company, highlighting the potential for improved outcomes in the wet Age-related Macular Degeneration (wetAMD) population and a discernible treatment effect for Cystic Fibrosis (CF). They also anticipate a comprehensive analysis of the Diabetic Macular Edema (DME) program in the fourth quarter of 2024 and the introduction of a new pipeline program for Geographic Atrophy (GA).
In addition, BMO Capital Markets Corp. has lowered its price target for 4D Molecular Therapeutics, while maintaining an "Outperform" rating, due to the FDA's requirement for a Phase III randomized trial for the company's cystic fibrosis therapy. Meanwhile, Barclays (LON:BARC) Capital Inc. initiated coverage on the company with an "Overweight" rating, expressing confidence in the wetAMD program and its product candidate 4D-150.
These recent developments come as the company continues to make progress in its clinical trials, with approximately $600 million in cash and equivalents as of Q4 2023, positioning the company to sustain its R&D efforts into the first half of 2027.
These are the latest updates in the company's recent developments, providing investors with crucial insights into the company's progress and potential.
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