4DMT Receives Rare Pediatric Disease Designation from FDA for Aerosolized 4D-710 for Treatment of Cystic Fibrosis Lung Disease

• 4D-710 has demonstrated promising, reproducible, CFTR expression significantly above normal levels for seven patients across Cohorts 1 & 2 (1E15-2E15 vg) and durable improvement or stabilization of quality of life & pulmonary function for three patients through 12 months in Cohort 1
• Given above normal CFTR transgene expression (~400% of normal lung levels), dose exploration continues with evaluation of lower doses (Cohort 3 dose 5E14 vg)
• Interim data update from Phase 1/2 AEROW clinical trial expected in mid-2024, and pivotal trial planning update expected in Q1
  

EMERYVILLE, Calif., Jan. 23, 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 that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for the Company’s product candidate aerosolized 4D-710 for treatment of cystic fibrosis (CF) lung disease. 4D-710 is being evaluated in the Phase 1/2 AEROW clinical trial in people with CF who are not eligible for, or cannot tolerate any of, the currently approved CFTR modulators.

RPDD may be granted to investigational drugs and biologics designed to address serious or life-threatening diseases primarily affecting individuals 18 years of age and under, and meeting the definition of “rare disease or condition” (affects less than 200,000 individuals in the U.S.). If the drug or biologic is approved for marketing, 4DMT may qualify for a priority review voucher that may be transferred or sold to another sponsor.

“The Rare Pediatric Disease Designation is a very important regulatory milestone in our development path for 4D-710 and highlights the urgent need for novel therapeutic options for people living with CF lung disease, including children, especially those who are not eligible for currently available disease modifying therapies,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “Based on the CFTR expression levels and clinical activity seen to-date, we are excited about the potential for 4D-710 to transform the lives of people with CF with an effective and durable treatment option. We continue to enroll our AEROW clinical trial and work with the CF Foundation and regulators to identify the most efficient path to advance this therapy, with preliminary feedback expected this quarter.”