4D Molecular Therapeutics presented new Phase 2 data for its wet AMD gene therapy, showing a durable response in a broad patient population, as it prepares to enter Phase 3 testing early next year.
The company’s goal is to prove whether its gene therapy, 4D-150, can reduce the frequency that wet AMD patients need to receive injections of Regeneron’s Eylea, which is typically given every four or eight weeks. In the expanded Phase 2 dataset shared on Wednesday, 4D-150 allowed 77% of 30 patients to become injection-free after 24 weeks.
Researchers presented the data at the American Society of Retina Specialists’ annual meeting in Stockholm. Each of the 30 patients received the dose that 4DMT plans to take into the Phase 3 trial. An additional 15 patients received a lower dose, with 60% of those patients injection-free after 24 weeks.
4DMT CEO David Kirn told Endpoints News that a one-time gene therapy appeals to patients for two reasons: Their eyes eventually develop antibodies to aflibercept — Eylea’s scientific name — and they grow weary of having needles frequently injected into their eyes.
“The beauty of gene therapy is it should be one-and-done,” Kirn said. “With a single treatment, you should be covered for the rest of your life if it’s a stable tissue like the retina.”
4DMT shares $FDMT rose roughly 8% premarket Wednesday, but fell about 24% in the hours after the opening bell.
The results build on data that 4DMT presented earlier this year for previously treated patients with severe wet AMD. That data showed that 63% of the 20 individuals who received the high dose did not need further injections after 24 weeks. Additionally, 50% of the 20 patients on the low dose also remained injection-free.
The new data presented in Stockholm come from a much broader patient population, Kirn said, raising his “conviction” for a Phase 3 success. “The lawyers say we can’t say ‘de-risk,’” he quipped.
Other figures released Wednesday included a reduced annualized injection rate — patients in the high-dose arm saw an 89% reduction — and improved visual acuity scores. Those who took the high dose improved by an average of 5.7 letters compared to the low dose when reading letters on an eye chart.
4DMT expects visual acuity improvement to be the primary endpoint in its pivotal trial, which will test 4D-150’s non-inferiority over Eylea, the standard of care. 4DMT plans to provide another update in September about the final Phase 3 design.
Ahead of the readout, Jefferies analyst Kelly Shi told investors last week that the Phase 2 data would be considered a success if the annualized injection rate maintained the 89% figure from February and if more than 70% of patients were injection-free. Shi also said stabilization of a biomarker in the retina called CST would be important.
4DMT reported that patients who did not need further injections saw CST levels improve over baseline compared to the overall patient population.
Editor’s note: This story has been updated to include 4DMT’s premarket stock movement, and again after the market opened.