4DMT will look to begin the Phase II dose expansion stage in its AEROW clinical trial after the biopharma reported positive interim data for its gene therapy 4D710.

4DMT is evaluating its asset 4D-710 – an AAV vector combined with a CFTR transgene – in patients with cystic fibrosis (CF). Patients eligible for the therapy are those who are ineligible for or intolerant of CFTR modulator therapy.

CF is an inherited progressive disease caused by variants in the CFTR gene. It affects the lungs, pancreas, and other organs. Nearly 40,000 people in the US live with the disease.

The company enrolled ten participants in its Phase I dose exploration stage. All participants had impairment of percent predicted forced expiratory volume (ppFEV1) in one second, a measure of lung function.

After receiving a single aerosolised dose of 4D-710, patients in the lower and middle dose cohorts had no related adverse events and no dose-limiting toxicities. At the highest dose, there was complete resolution of a previously reported serious adverse event.

Lung biopsy results demonstrated widespread CFTR transgene RNA and protein expression, but none more so than the next highest dose. 4DMT stated that the highest dose – 2E15 vg – will not be further evaluated and 1E15 vg was selected as the highest dose for dose expansion. Enrolment is slated to begin in the second half of this year.

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4DMT’s head of pulmonology Alan Cohen said: “Based on the emerging favourable safety and clinical activity profile, we look forward to beginning our Phase II dose expansion stage in participants with mild to moderate lung function impairment, to confirm the clinical activity of the 1E15 vg dose.”

4DMT stated that all participants in the 2E15 vg and 1E15 vg cohorts had stable or improved ppFEV1 at 12 months.

Lead investigator of the AEROW trial Jennifer Taylor-Cousar said: “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.”

“The consistent widespread 4D-710–mediated CFTR expression and initial clinical activity in Phase I 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.”

4DMT secured rare paediatric designation for the gene therapy in January 2024. If 4D-710 is eventually approved by the US Food & Drug Administration (FDA), 4DMT will be eligible to receive a priority review voucher. Recipients can redeem priority review vouchers themselves for any chosen drug or can instead sell them to other pharma companies. Cutting four months off FDA review time, vouchers currently sell for approximately $100m.

4DMT enjoyed clinical success earlier this year in another area of its portfolio. The company reported positive data for its gene therapy 4D-150 in wet age-related macular degeneration, with a Phase III trial slated to start early next year.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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