4D Molecular Therapeutics receives RMAT designation for 4D-150 for the treatment of diabetic macular edema

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4D Molecular Therapeutics (4DMT) announced that the US Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to 4D-150 for the treatment of diabetic macular edema (DME).¹

David Kirn, MD, co-founder and CEO of 4DMT commented on the announcement in a press release.¹

“This milestone validates the potential of 4D-150 to address the significant unmet needs of patients with DME, a second large market retinal vascular disease indication after wet age-related macular degeneration (wet AMD),” said Kirn. “The RMAT designation is based on the review of our results to-date from our ongoing 4D-150 SPECTRA DME study, underscoring the potential of 4D-150 to sustain visual acuity improvements while dramatically reducing treatment burden for patients. This designation in DME follows the RMAT designation granted for 4D-150 in wet AMD, and to our knowledge, 4D-150 is the first investigational medicine to be granted the designation in both indications.”

RMAT designation was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, allowing for early, close and frequent interactions with the FDA with the goal of expediting drug development.

4D-150 is a potential backbone therapy that is designed to provide multiyear sustained delivery of anti-VEGF (aflibercept and anti–VEGF-C) to be targeted to the retina with a single, well-tolerated intravitreal injection. 4D-150 is being developed for both DME and AMD.²

At the beginning of the year, 4DMT announced² a strategically focused pipeline in addition to updated phase 3 4FRONT program plans, initial 4FRONT guidance, and resulting updated cash runway guidance.

Positive 32-week interim data from the ongoing SPECTRA part 1 trial was also announced in January 2025. The trial found that 4D-150 continues to be well tolerated with no intraocular inflammation observed at any time point or dose level. The 3E10-vg/eye dosage demonstrated a sustained gain of best corrected visual acuity (BCVA) of +8.4 letters and a reduction of central subfield thickness (CST) of –194 from baseline through week 32. The single phase 3 clinical trial was found to be acceptable for the basis of a biologics license application submission for 4D-150 in DME based on data from the SPECTRA and PRISM trials to date.

References:

1. 4DMT Announces RMAT Designation Granted by FDA for 4D-150 for DME. News release. 4DMT. May 1, 2025. Accessed May 1, 2025. https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4dmt-announces-rmat-designation-granted-fda-4d-150-dme

2. Joy J. 4DMT refocuses pipeline to prioritize 4D-150 trials in wet AMD and DME. Ophthalmology Times. January 13, 2025. Accessed May 1, 2025. https://www.ophthalmologytimes.com/view/4dmt-refocuses-pipeline-to-prioritize-4d-150-trials-in-wet-amd-and-dme