Mid-stage data for 4D Molecular Therapeutics’ wet AMD drug 4D-150 show improvements in visual acuity, reduced injection rate and encouraging safety data.
4D Molecular Therapeutics revealed positive Phase II data Wednesday for its wet age-related macular degeneration candidate at the American Society of Retina Specialists (ASRS) annual scientific meeting.
The latest data from the Phase II PRISM study show the gene therapy, 4D-150, to be safe and well-tolerated, with no serious adverse events, 4DMT reported. The trial’s objective is to determine whether 4D-150 can decrease the number of times patients need injections of Regeneron’s Eylea, which are typically given every four or eight weeks. In this, the candidate was successful, as 77% of 4D-150-treated patients were injection free after 24 weeks; 93% of patients received either zero or one additional injection.
Improvements were also found in the best corrected visual acuity metric at the planned Phase III dosage, 3E10 vg/eye, with an increase of 4.2 letters from the baseline at week 24. This dosage level also elicited sustained greater anatomic control without fluctuations.
4D-150 uses 4DMT’s intravitreal vector, known as R100, and a transgene cassette.
The candidate also showed a solid safety profile as treatment with 3E10 vg/eye did not cause anterior chamber inflammation, and no significant vitreous inflammation was found.
Ophthalmologists have said that safety is the priority for wet AMD treatment, especially with gene therapies, given the risk of blindness arising from significant intraocular inflammation, Jefferies noted in an analyst report. The report called safety one of 4D-150’s “key differentiators.”
The data readout sent 4DMT’s stock price up over 9% premarket Wednesday.
Wednesday’s announcement follows another positive data readout for 4D-150. In February, topline results from the PRISM study showed the investigational gene therapy cut annual rates of Eylea injections by 85% and 89% at the low and high doses, respectively.
“The data presented on 4D-150 continue to show its promise as a potentially safe, routine and one-time intravitreal treatment with the long-term objective to preserve vision for millions of wet AMD patients, regardless of their treatment burden or disease severity,” said 4DMT Chief Medical Officer Robert Kim in a statement.
Kim added that 4DMT expects additional data readouts from its SPECTRA study of 4D-150 in diabetic macular edema in Q4, 2024. Phase III clinical trial alignments are ongoing with the FDA and the EMA and the final trial design is expected in Sept. 2024, according to the press release. The trial is slated to begin in the first quarter of 2025.
