The FDA lifted an 18-month clinical hold on 4D Molecular Therapeutics’ cardiomyopathy gene therapy for Fabry disease.
The biotech said Thursday it expects to resume enrollment in the Phase 1/2 INGLAXA study of 4D-310 before the end of the year.
The Emeryville, CA-based company announced the hold in February 2023, a month after pausing enrollment in the trial after three patients on the experimental gene therapy experienced kidney issues. The cases were resolved within four weeks.
At the time of the hold, 4DMT CEO David Kirn said the company believed the hold was “an administrative step [and] has no impact on our plans or timing for the 4D-310 program.”
Jefferies analyst Kelly Shi wrote in a note on Thursday that 4DMT management indicated the initial focus will be on ex-US sites so they can “collect cardiac biopsies” and “confirm the expected improved safety of rituximab & sirolimus (R/S) regimen + amended protocol to prescreen patients for complement activation.”
Several drugmakers have approved Fabry medicines, including Sanofi, Chiesi and Amicus. Sangamo, uniQure and others are developing gene therapies for the disease. Other companies like Freeline and Avrobio have scrapped efforts. Meanwhile, earlier this year, Walking Fish Therapeutics shut down months before planning to request FDA clearance for a trial in Fabry disease.
Earlier this summer, Kirn said that the “primary value driver” in 4DMT’s pipeline is its ophthalmology programs, according to an AlphaSense transcript of a presentation at the Goldman Sachs healthcare conference. The company is investigating medicines for wet AMD, diabetic macular edema, X-linked retinitis pigmentosa, choroideremia, geographic atrophy and other indications. It also has an ophthalmology deal with Astellas. Beyond retinal diseases and Fabry, it’s working on candidates for cystic fibrosis, A1AT deficiency and ALS.
Jefferies analysts estimate that 4D-310 could receive approval in the US in 2030 and in the EU in 2031. They predict peak worldwide sales of $172 million in 2035 if the drug is approved.