4DMT’S gene therapy for AMD clears Phase II, heads to late-stage studies
4D Molecular Therapeutics announced on Wednesday that its investigational gene therapy 4D-150 demonstrated durable clinical activity in the Phase I/II PRISM study of patients with wet age-related macular degeneration.
In a 30-patient cohort, 4D-150 cut the need for standard-of-care injections by 89% through 52 weeks of follow-up, and 70% of these patients were injection-free, according to 4DMT’s announcement. In another cohort of 27 patients with severe disease, the need for standard-of-care treatment dropped by 83% and 44% were injection-free.
The gene therapy also resulted in sustained benefits in terms of central subfield thickness and mean best-corrected visual acuity.
In terms of safety, PRISM found 4D-150 to be well-tolerated. The frequency for of intraocular inflammation (IOI) was consistent with approved anti-VEGF therapies, which are currently widely used to treat age-related macular degeneration (AMD). Overall, 4DMT documented two IOI episodes in all 71 wet AMD patients enrolled, yielding an incidence rate of 2.8%.
There were no cases of retinal artery occlusions, hypotony, vasculitis, choroidal effusions and endophthalmitis related to the gene therapy.
4DMT CEO David Kirn in a statement called 4D-150 a “potentially paradigm shifting product candidate,” noting that PRISM’s findings highlight “clear reduction in overall treatment burden and potential multiyear clinical benefit in previously treated patients.” The California-based biotech will continue to invest in 4D-150’s development, Kirn said, and push it into late-stage studies.
