4DMT Presents Positive Interim Data from Aerosolized 4D-710 Phase 1/2 AEROW Clinical Trial in Patients with Cystic Fibrosis at the ECFS 46th Annual Meeting

• Reported data from cohort 1 (n=3, 1E15 vg dose) of cystic fibrosis modulator treatment ineligible/intolerant participants with 9-12 months follow-up
• Quality of life outcomes measured by Cystic Fibrosis Questionnaire-Revised respiratory symptom score (CFQ-R-R) meaningfully improved for all three participants
• Percent predicted forced expiratory volume in one second (ppFEV₁) meaningfully improved in participant with moderate ppFEV₁ impairment at baseline; ppFEV₁ maintained stable in participants with normal or mild impairment at baseline

• Bronchoscopy sample results demonstrated widespread and consistent expression of the cystic fibrosis transmembrane conductance regulator (CFTR) transgene protein in 92-99% of lung airway cells, at levels significantly above normal control lung tissues
• Dose selection and initiation of Phase 2 Dose Expansion stage expected in H2 2023; additional interim data including for cohort 2 participants (2E15 vg dose) expected at the North American Cystic Fibrosis Conference in November 2023
• Company to host live webcast today at 4:30 p.m. ET with cystic fibrosis specialist Dr. Jennifer L. Taylor-Cousar

Excerpt from the Press Release:

EMERYVILLE, Calif., June 07, 2023 (GLOBE NEWSWIRE) — 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT, or the Company), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, today announced positive interim clinical data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis lung disease. 4D-710 is comprised of our targeted and evolved vector, A101, and a codon-optimized CFTR∆R transgene and is designed for aerosol delivery to achieve CFTR expression within lung airway epithelial cells. In this clinical update, participants ineligible for (n=2) or intolerant of (n=1) existing CFTR modulators were dosed with 4D-710 in cohort 1 (dose 1E15 vg) and showed meaningful improvement in cystic fibrosis-related quality of life measured by CFQ-R-R and improved or stabilized ppFEV₁. Interim data from the study will be presented in an oral presentation titled, “AAV mediated gene therapy for cystic fibrosis: Interim results from a Phase 1/2 clinical trial,” at the European Cystic Fibrosis Society (ECFS) 46th Annual Meeting held in Vienna, Austria on Thursday, June 8 at 5:00 p.m. CET (11:00 a.m. ET).

“This evidence of tolerability, high level CFTR protein expression and early clinical activity in three participants is promising for the ongoing development of this aerosolized product candidate for cystic fibrosis lung disease,” said Jennifer L. Taylor-Cousar, MD, MSCS, Professor, Departments of Medicine and Pediatrics, and Co-Director, Adult Cystic Fibrosis Program, Director, Cystic Fibrosis Foundation Therapeutics Development Center, National Jewish Health, “I’m highly encouraged that the robust and consistent transduction observed is translating into improvements in quality of life and stabilization or improvement in the pulmonary function in these individuals with cystic fibrosis who otherwise do not have disease modifying therapy available to them. Historical data suggests that on average these measures would have declined without treatment in this population.”

“The widespread CFTR transgene expression demonstrated in all lung samples from participants in cohort one is a critical first step for the development of 4D-710, especially given the signals of clinical activity in this CF population with the highest unmet medical need,” said David Kirn, MD, Co-founder and Chief Executive Officer of 4DMT.

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