4D Molecular Therapeutics is axing two clinical programs and cutting off funds for three other assets, with resources to be pivoted toward launching late-stage studies for the biotech's eye disease gene therapy, 4D-150.
The genetic medicines biotech is discarding a choroideremia program assessing a gene therapy dubbed 4D-110, according to a Jan. 10 release. Choroideremia is a rare eye disease that primarily impacts men.
The therapy had initially pulled in support from Roche, but the Big Pharma lost interest in 2021, removing funds for an early-stage clinical trial for the asset and handing rights to the therapy back to 4DMT. That phase 1 study was slated to conclude mid-2024, according to ClinicalTrials.gov.
The company is also tossing another rare disease clinical program, this one in X-linked retinitis pigmentosa, a genetic condition that causes blindness in men. That gene therapy, known as 4D-125, was being studied in a phase 1/2 trial that enrolled 21 patients and was expected to read out next year, according to ClinicalTrials.gov.
In addition, 4DMT is cutting off further investment for three more programs, pending additional financing or partnerships.
Those include an ongoing phase 1/2 trial assessing 4D-310 for Fabry disease-related cardiomyopathy. Back in 2023, the study was hit with an FDA hold when three cases of a kidney condition called atypical hemolytic uremic syndrome occurred in the first six patients dosed. The hold was later lifted in August 2024.
The other two programs subject to the money shutoff are both preclinical: 4D-175, in geographic atrophy, and 4D-725, in alpha-1-antitrypsin deficiency lung disease.
4DMT has also decided not to invest anything into new preclinical candidates for the time being. The company said it’s currently exploring partnership prospects and other financing options.
The reorganization is expected to extend the California biotech’s runway into 2028. As of Dec. 31, the company had $506 million in cash and marketable securities.
Those funds will be used mostly to fuel pre-commercialization activities and collect 52-week data from two phase 3 clinical trials in wet age-related macular degeneration, or wet AMD, for 4D-150. The gene therapy uses an adeno-associated virus vector to deliver transgenes encoding aflibercept, the molecule that Regeneron sells as Eylea, and interferes with RNA to inhibit VEGF-C.
The money will also be used to push 4D-150 into late-stage testing for diabetic macular edema. In a phase 2 trial, the drug was found to be well tolerated among 21 patients, according to interim results also shared Jan. 10.
Additionally, the study helped the biotech determine a phase 3 dose level: 3E10 vg/eye. Patients within that dosing arm during the midstage trial were linked to a sustained gain of best corrected visual acuity of 8.4 letters, according to 4DMT.
The company will be presenting the 32-week results in a webcast on Feb. 10, and also expects to share a 52-week interim data update at a midyear scientific conference.