4DMT's gene therapy decreased reliance on standard injections for age-related vision loss

4D Molecular Therapeutics has released promising mid-stage trial data for its lead gene therapy candidate, 4D-150, which aims to reduce the need for annual injections in patients suffering from age-related blindness. The company disclosed on Wednesday that in a Phase 2b trial involving 30 patients, 70% did not require an annualized injection of Regeneron’s Eylea. Furthermore, there was an 89% reduction in annualized injections over 52 weeks, with 80% of patients needing zero or just one injection.

The results were even more promising in a 15-person group of recently diagnosed wet age-related macular degeneration patients. Similar success was noted in a Phase 1/2 trial involving more severe cases, which showed an 83% reduction in annual injections. Over half of these patients required zero or one injection through 52 weeks, and 44% were entirely injection-free.

CEO David Kirn emphasized that these results provide substantial evidence that 4D-150's intravitreal injection method, which is administered directly into the eye, is as effective as other anti-VEGF treatments that require the more invasive subretinal injections. Intravitreal injections are simpler to perform as they do not require surgery, unlike subretinal injections.

Despite the encouraging data, the company's shares dropped by 22% when the market opened on Thursday. On a positive note, the treatment did not introduce any new side effects and aligned with the safety profile of existing anti-VEGF options. About 3% of patients experienced intraocular inflammation, and nearly all patients completed their steroid prophylaxis taper on schedule.

Looking ahead, the company is gearing up for pivotal trials in 2025. The first of these U.S.-based studies is scheduled to start in the first quarter of the next year. According to Kirn, the company has already aligned with the FDA and is finalizing study details with European regulators, aiming to commence the European trial a few months after the U.S. one.

4D Molecular Therapeutics ended June with $578 million in funds and has no immediate plans for additional public offerings. Kirn stated that the current financial runway is sufficient through the first Phase 3 readout. He also mentioned that the company expects to continue advancing its earlier-stage candidates while focusing on the late-stage development of 4D-150. The company has two other clinical-stage ophthalmology candidates in its pipeline, along with one that has received IND clearance. Additionally, 4DMT has clinical-stage candidates in both its pulmonary and cardiology divisions.

Kirn expressed confidence in the company’s strategic direction, stating, "We’re very rapidly shifting from a diversified company to what we hope to be the leader in large-market ophthalmology for genetic medicine."