4D Molecular Therapeutics, a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, announced FDA clearance of the Investigational New Drug Application (IND) for 4D-150, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with Diabetic Macular Edema (DME).
The Phase 2 SPECTRA clinical trial will assess 4D-150 in patients with DME. The study design consists of a Dose Confirmation stage followed by a masked Dose Expansion stage, in which patients will be randomized to receive a single intravitreal injection at one of two dose levels of 4D-150 or aflibercept in a 1:1:1 ratio (n=54 patients). The doses to be evaluated in DME are anticipated to be between 6E9 to 3E10 vg/eye. The IND clearance enables the initiation of SPECTRA clinical study sites, and 4DMT expects to begin enrollment in the third quarter of 2023.
Initial Cohort 1 data (n=5) from the Phase 1 portion of the Phase 1/2 PRISM clinical trial with 4D-150 for wet age-related macular degeneration (wet AMD) demonstrated a reduction in annualized anti-VEGF injection rate by over 95%, further validating the potential of our intravitreal R100 vector for other large market eye diseases such as geographic atrophy.
On January 9, 2023, 4DMT disclosed that the Company had initiated the randomized Phase 2 portion of the Phase 1/2 PRISM clinical trial for 4D-150 in patients with wet AMD. This portion of the trial is now enrolling patients. In addition, the Company intends to present interim data for dose Cohorts 1, 2, & 3 (n=15) at the 2023 ARVO Annual Meeting taking place April 23-27, 2023; at the time of the ARVO data presentation, all patients are predicted to have at least six months of follow-up following 4D-150 treatment.
“This is the sixth US IND submitted by 4DMT, and all six have been cleared by the FDA. The robustness and efficiency of our product design and development engine are based on sustained excellence by our manufacturing, preclinical, clinical, and regulatory teams,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “We are excited to have started enrolling the Phase 2 portion of our PRISM trial in wet AMD, and to evaluate the potential of 4D-150 in patients with DME who also require frequent anti-VEGF injections and have a high treatment burden.”