4D Molecular Therapeutics, a clinical-stage genetic medicines company, has announced promising topline interim results from Part 1 of the SPECTRA clinical trial evaluating 4D-150, a novel treatment for diabetic macular edema (DME).

The trial, designed to assess safety, tolerability, and identify the optimal dose level for further evaluation, enrolled 22 patients across three dose levels: 3E10 vg/eye (n=9), 1E10 vg/eye (n=12) and 5E9 vg/eye (n=1). One patient in the 1E10 vg/eye arm discontinued participation due to an unrelated death before completing post-baseline assessments.

The safety profile of 4D-150 was highly favourable, with no intraocular inflammation reported at any time. Patients successfully completed a 16-week topical corticosteroid taper and remained steroid-free thereafter. No cases of hypotony, endophthalmitis, vasculitis, choroidal effusions or retinal artery occlusions were observed.

Efficacy results through 32 weeks in the 3E10 vg/eye dose arm showed sustained improvements. The average gain in best corrected visual acuity (BCVA) was +8.4 letters, while central subfield thickness (CST), as measured by optical coherence tomography (OCT), was reduced by -194 µm. The need for supplemental injections was significantly lower in the 3E10 vg/eye arm compared to the 1E10 vg/eye arm and projected on-label aflibercept treatment. Patients in the 3E10 vg/eye group required an average of 0.6 injections per patient, compared to 1.4 in the 1E10 vg/eye group and 4.0 in the projected aflibercept 2mg Q8W group. Furthermore, five out of nine patients in the 3E10 vg/eye arm were injection-free, compared to two out of ten in the 1E10 vg/eye arm.

The 3E10 vg/eye dose has been selected for phase III evaluation. A 52-week interim update is anticipated at a scientific conference in mid-2025. Based on data from the SPECTRA and PRISM trials, the US. Food and Drug Administration (FDA) has agreed that a single phase III trial, combined with data from two planned phase III trials in the 4FRONT wet AMD program, will be sufficient for a Biologics License Application (BLA) submission for 4D-150 in DME.

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Disclosures: This article was created by the touchOPHTHALMOLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.