FDMT - 4DMT Presents Positive Interim Data from Phase 1/2 AEROW Clinical Trial of Aerosolized 4D-710 for Modulator-Ineligible/-Intolerant Cystic Fibrosis at 47th European Cystic Fibrosis Conference | Benzinga

• Clinically meaningful improvements in ppFEV₁ at 12 months observed in 2 of 3 participants with mild to moderate baseline lung function impairment (ppFEV₁ 50-80%) and >6 months follow up

• Aerosolized 4D-710 was well tolerated at doses up to 1E15 vg (n=6)
  
• Dose-dependent and widespread 4D-710-mediated CFTR transgene RNA and protein expression observed in all lung biopsies from all participants evaluated to date
• Pre-existing AAV immunity cross-reactive with A101 did not affect transgene expression, biological activity or safety
  
• 1E15 vg dose cleared for evaluation in Phase 2 Dose Expansion stage; enrollment expected to begin in H2 2024 and next interim data update expected in mid-2025
  
• Company to host live webcast today at 8:00 a.m. ET with Dr. Jennifer L. Taylor-Cousar, lead Principal Investigator in the AEROW clinical trial

EMERYVILLE, Calif., June 06, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:FDMT, 4DMT or the Company)), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced positive interim clinical data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis (CF) lung disease. Interim data from the study will be presented by Jennifer L. Taylor-Cousar, M.D., MSCS, in an oral presentation titled, "CFTR transgene expression in airway epithelial cells following aerosolized administration of the AAV-based gene therapy 4D-710 to adults with cystic fibrosis lung disease," at the 47^th European Cystic Fibrosis Conference held in Glasgow, UK on Thursday, June 6 at 5:00 p.m. BST.

"We are pleased with the widespread CFTR transgene and protein expression in airway cells from all participants at all doses in the AEROW clinical trial," said Jennifer L. Taylor-Cousar, M.D., MSCS, Professor, Departments of Medicine and Pediatrics, and Co-Director, Adult Cystic Fibrosis Program, Director, Cystic Fibrosis Therapeutics Development Center, National Jewish Health and lead Principal Investigator in the AEROW clinical trial. "The consistent widespread 4D-710–mediated CFTR expression and initial clinical activity in Phase 1 are encouraging and suggest that 4D-710 has the potential to be the first treatment option for those living with cystic fibrosis who do not currently benefit from available disease-modifying therapies."

"The clinical data to date from the AEROW clinical trial continues to strongly support the advancement of 4D-710 to the next stage of development for CF lung disease," said Alan H. Cohen, M.D., SVP, Therapeutic Area Head of Pulmonology of 4DMT. "Based on the emerging favorable safety and clinical activity profile, we look forward to beginning our Phase 2 Dose Expansion stage in participants with mild to moderate lung function impairment, to confirm the clinical activity of the 1E15 vg dose."

"The interim data we shared today from the AEROW clinical trial continues to showcase the breadth and depth of our platform, product engine and pipeline," said David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT. "4D-710 is well positioned as the lead program in our pulmonology portfolio as we move into mid to late-stage development. These clinical results also support continued use of our next-generation A101 pulmonary vector for large-market lung diseases such as alpha-1 antitrypsin deficiency lung disease, for which we are developing 4D-725, an aerosolized genetic medicine currently in IND-enabling preclinical studies."

AEROW Clinical Trial Phase 1 Dose Exploration Stage Background & Summary

• 10 participants with CF lung disease who are ineligible for or intolerant of CFTR modulator therapy have been enrolled in the Phase 1 Dose Exploration stage across four dose cohorts:
  
  • 2E15 vg Cohort: n=4
  • 1E15 vg Cohort: n=3
  • 5E14 vg Cohort: n=2
  • 2.5E14 vg Cohort: n=1
• Participants enrolled in the Phase 1 Dose Exploration stage had a broad range of baseline ppFEV₁ impairment ranging ...

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