4D Molecular Therapeutics has posted 60-week data on a gene therapy, providing evidence of durability in diabetic macular edema (DME) and sending its stock up more than 40% in premarket trading.
The phase 2 trial is assessing single doses of 4D-150, a gene therapy that encodes aflibercept and a sequence that inhibits expression of VEGF-C. Aflibercept, an anti-VEGF drug that Regeneron markets as Eylea, is the active control in the study. 4DMT designed 4D-150 to provide sustained delivery of anti-VEGF agents, freeing patients from burdensome injections and potentially improving outcomes.
Researchers shared 60-week results from the trial at the Annual American Society of Retina Specialists annual scientific meeting. Among the nine patients who received the phase 3 dose, 4DMT reported a gain of best corrected visual acuity of 9.7 letters, compared to 8.4 letters in the 32-week cut of the data.
The biotech tracked a 78% reduction in supplemental injections in people who received the phase 3 dose compared to projected on-label use of aflibercept 2 mg. 4DMT saw an 86% reduction in injection burden after 32 weeks. After 60 weeks, participants on the phase 3 dose had 58% fewer injections than their peers on the lower doses of 4D-150. Four of the nine patients on the phase 3 dose were injection free.
Shares in 4DMT rose 42% to $6.30 in premarket trading Friday in the wake of the readout.
4DMT said the therapy continues to be well tolerated, with no intraocular inflammation at any time point and no subjects needing modification to the topical corticosteroid regimen. No cases of the eye conditions hypotony, endophthalmitis, vasculitis, choroidal effusions or retinal artery occlusion were seen. Mean intraocular pressure was within the normal limits.
The biotech provided a regulatory update, revealing that the European Medicines Agency has followed the FDA in agreeing that a single phase 3 trial can support approval in DME. 4DMT will also have data from two phase 2 trials, including one in DME, and two planned phase 3 studies in wet age-related macular degeneration.