Why Is 4D Molecular Therapeutics Stock Trading Lower On Thursday? Genetic Disease-Focused Study Data Released

4D Molecular Therapeutics FDMT released data from a clinical trial for cystic fibrosis lung disease.

What Happened: The company unveiled results from the 4D-710 Phase 1/2 AEROW study:

• At high dose – 2E15 vg (n=4), complete resolution of previously reported serious adverse event (SAE) was observed; ppFEV1 in this participant improved by 6% from baseline to month 12.
• Lung biopsy results: No evidence of inflammation or toxicity from histological analysis of tissue samples
• Widespread expression of CFTR protein compared to normal (non-CF) lung samples and no increase vs. 1E15 vg dose
• There is evidence of consistent CFTR protein expression in all major airway epithelial cell types and interstitial tissue cells; interstitial CFTR expression was not detected in normal lung control samples.

Lower Doses – 2.5E14 to 1E15 vg (n=6): 4D-710 was well tolerated, with no 4D–710–related adverse events after administration, no dose-limiting toxicities, and no SAEs.

Related: 4D Molecular Therapeutics Outlines Pivotal Development Plans For Cystic Fibrosis, Rare Inherited Disease.

Based on all available data for 2E15 and 1E15 vg dose level participants, 1E15 vg was selected as the highest dose for Dose Expansion; 2E15 vg dose will not be further evaluated.

Biomarker analyses demonstrated robust, consistent and widespread CFTRDR transgene mRNA and CFTR protein expression throughout all lung biopsy samples from all participants at all four dose levels.

All participants in 2E15 vg and 1E15 vg Cohorts had at least 12 months of follow-up and had generally stable or improved in ppFEV1 at 12 months.

Two of three participants with baseline mild to moderate lung function impairment (ppFEV1 40-80%) showed clinically meaningful improvement in ppFEV1 at 12 months.

The company expects to share interim data updates from the AEROW clinical trial in mid-2025.

Following Phase 2 data, Phase 3 initiation is expected in H2 2025.

Price Action: FDMT shares are down 14.8% at $23.23 at last check Thursday.

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