1. 4DMT reports positive interim data for 4D-710 in cystic fibrosis treatment. 2. 4D-710 shows potential as a durable, redosable, disease-modifying genetic medicine. 3. Phase 2 dosage was selected based on safety and efficacy data evaluation. 4. 4DMT aims to complete Phase 2 enrollment in H1 2026. 5. Cystic fibrosis affects over 40,000 individuals in the U.S., creating significant unmet need.
FAQ
Why Bullish?
The announcement of promising clinical data can increase investor confidence and attract interest, similar to historical spikes seen in biotech stocks after positive trial results. For example, stocks like CRSP (CRISPR Therapeutics) have previously surged following successful trial news.
How important is it?
The article highlights significant clinical progress in a critical therapeutic area for FDMT, illustrating the company’s growth potential and strategic position. Such developments tend to have substantial ramifications for stock valuation.
Why Long Term?
This trial data could establish a sustained market position for FDMT, akin to success seen in other rare disease therapies which command premium pricing and demand.
4D Molecular Therapeutics (FDMT) Announces Positive Interim Clinical Data for 4D-710 in Cystic Fibrosis Treatment
EMERYVILLE, Calif., December 17, 2025 (GLOBE NEWSWIRE) — 4D Molecular Therapeutics (Nasdaq: FDMT), a leading biotechnology company, has released promising interim clinical data from its Phase 1 AEROW clinical trial for 4D-710, a novel treatment for cystic fibrosis (CF) lung disease. The findings suggest the therapy's potential as a durable and disease-modifying solution for patients with high unmet needs.
Highlights of the AEROW Trial
The interim results highlight several key metrics regarding the safety and efficacy of 4D-710. The emerging data emphasized:
- Positive lung function activity assessed through ppFEV1 and LCI2.5 measurements.
- Durable CFTR transgene expression maintained within the therapeutic range for over a year.
- Data supporting the ongoing selection of 4D-710 as a potential redosable genetic therapy.
Clinical Insights from Key Opinion Leaders
Dr. Jennifer L. Taylor-Cousar, Co-Director of the Adult Cystic Fibrosis Program at National Jewish Health, commented: “The preliminary data from the AEROW trial are highly encouraging. The selected Phase 2 dose of 4D-710 was well tolerated and indicated clinically meaningful improvements across various metrics of lung function and pulmonary symptoms.”
Dr. Felix Ratjen, a paediatrics expert at the University of Toronto, noted the significance of LCI2.5 as a measure to evaluate lung disease progression, stating: “LCI2.5 offers a more comprehensive view of lung health compared to ppFEV1, particularly in detecting early changes in disease impact.”
Results and Expected Milestones
The data cutoff for this interim analysis was December 1, 2025, which revealed the following:
- 16 participants with CF lung disease were enrolled across four dosing cohorts.
- No new safety concerns were raised since previous updates on higher-dose cohorts, indicating a well-tolerated treatment.
- Mild and transient adverse events from 4D-710 that resolved within two months.
- The selected Phase 2 dose of 2.5E14 vg demonstrated clinically meaningful outcomes in all endpoints evaluated.
Next steps include completing the enrollment of the AEROW Phase 2 Dose-Expansion cohort, targeting six participants in the first half of 2026, followed by a program update in the second half of 2026.
About Cystic Fibrosis and 4D-710
Cystic fibrosis (CF) is a progressive genetic disorder caused by mutations in the CFTR gene, affecting nearly 40,000 individuals in the U.S. and over 105,000 worldwide. The disease primarily leads to severe lung complications and a significant treatment burden for patients.
4D-710 aims to address the root cause of CF by utilizing an evolved aerosolized AAV vector to enhance airway function and improve patients' quality of life, regardless of their specific CFTR mutation. This innovative approach aligns with the increasing demand for variant-agnostic therapies in the field.
About 4DMT
4D Molecular Therapeutics (FDMT) is a pioneering biotechnology firm dedicated to revolutionizing treatment paradigms with durable and targeted therapeutics. Alongside 4D-710, the company focuses on developing therapies to address blinding retinal vascular diseases, further positioning itself as a leader in innovative treatment solutions.