4D Molecular Therapeutics’ gene therapy cuts Eylea use in wet AMD patients, biotech divulges plans for pivotal studies

4D Molecular Therapeutics said people with wet age-related macular degeneration (AMD) who received the company’s gene therapy as part of a clinical trial needed fewer injections of Eylea, a staple treatment for the eye disease.

In the Phase II PRISM study, 51 patients with wet AMD received either 4D’s gene therapy or their usual injections of Eylea, a blockbuster medicine marketed by Regeneron. Over 24 weeks, 63% of the patients who received a high dose of the gene therapy, dubbed 4D-150, and 50% of those who received the low dose did not need supplemental Eylea injections.

4DMT’s shares $FDMT jumped over 60% in premarket trading Monday.

Wet AMD is a disease related to aging that occurs when the eye’s “straight-ahead” vision becomes blurry. The patients enrolled in this study were people with very high disease activity, the company said. “These are patients who normally are excluded from other wet AMD studies,” 4DMT CEO David Kirn told Endpoints News.

In addition to the participants who didn’t need injections, four patients in the high-dose group and eight in the low-dose group only required one Eylea injection over the 24-week period. There were approximately 20 patients in each group. The results translated to 89% and 85% reductions in yearly injection rates for the high- and low-dose arms, respectively.

The data were revealed Saturday at the Angiogenesis, Exudation, and Degeneration annual conference.

BMO Capital analyst Kostas Biliouris said that investors believe a successful high-dose outcome would be an 85% reduction in injections, with between 60% to 65% of patients being injection-free. The high-dose group hit 63% on the injection-free measure. Out of 20 patients, 12 were injection-free and one patient was excluded for stopping treatment early for reasons unrelated to treatment.

Kirn said the gene therapy could also lead to less oscillation in the condition of the eyes compared to the chronic injections. “This is not just about improving patients’ quality of life by dramatically reducing their need for future injections,” Kirn said. “It’s also about preserving visual acuity.”

Next up for 4DMT is a Phase III trial with the high dose that is expected to begin early next year. The biotech is planning it as a non-inferiority trial versus Eylea, which is expected to lose exclusivity later this year, as part of a package it can take to the FDA for approval.

It’s also studying the therapy in diabetic macular edema, as well as in patients who have a wider range of disease severity. That portion of the mid-stage study has completed enrollment, the biotech said Saturday.

AbbVie and Regenxbio are also partnered on an experimental gene therapy for wet AMD and diabetic macular edema. And in July, Astellas penned a deal with 4DMT for $20 million upfront to use its viral vector to develop a therapy for an undisclosed rare monogenic eye disease.

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