EMERYVILLE, Calif. — 4D Molecular Therapeutics, 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.”
About Cystic Fibrosis Lung Disease and 4D-710
Cystic fibrosis is an inherited, progressive disease caused by mutations in the CFTR gene. It affects the lungs, pancreas, and other organs. According to the CF Foundation, nearly 40,000 people in the United States and more than 105,000 people worldwide are living with cystic fibrosis, with approximately 1,000 new cases of cystic fibrosis diagnosed in the United States each year. Lung disease is the leading cause of morbidity and mortality in people with cystic fibrosis. Cystic fibrosis causes impaired lung function, inflammation and bronchiectasis and is commonly associated with persistent lung infections and repeated exacerbations due to the inability to clear thickened mucus from the lungs. People with cystic fibrosis require lifelong treatment with multiple daily medications. The complications of the disease result in progressive loss of lung function, increasing need for IV antibiotics and hospitalizations, ultimately leading to end-stage respiratory failure.
4D-710 is comprised of our targeted and evolved next generation vector, A101, and a codon-optimized CFTR∆R transgene. 4D-710 has the potential to treat a broad range of people with cystic fibrosis, independent of the specific CFTR mutation, and is designed for aerosol delivery to achieve CFTR expression within lung airway epithelial cells. 4D-710 is being initially developed for the approximately 15% of people whose disease is not amenable to existing CFTR modulator medicines (based on variant-eligibility and/or drug intolerance) targeting the CFTR protein. In people with CFTR mutations whose disease is amenable to modulator medicines, the improvement in lung function is incomplete and is variable. We therefore expect to potentially develop 4D-710 in this broader population, as a single agent and/or in combination with CFTR modulator small molecule medicines.
About 4DMT
4DMT is a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our product design, development, and manufacturing engine helps us efficiently create and advance our diverse product pipeline with the goal of revolutionizing medicine with potential curative therapies for millions of patients. Currently, 4DMT is advancing five clinical-stage and two preclinical product candidates, each tailored to address rare and large market diseases in ophthalmology, pulmonology, and cardiology. In addition, 4DMT is also advancing programs in CNS through a gene editing partnership. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.
All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied.
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