Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA lifted a more than two-year-long hold.
Dubbed ENTR-601-44, the endosomal escape vehicle-conjugated phosphorodiamidate morpholino oligomer was placed on hold by the agency back in December 2022 while it asked the biotech to gather “additional information.” Entrada subsequently submitted another data package to the FDA, but the biotech revealed in November 2023 that this wasn't enough to persuade the agency to change its stance.
Now, the ordeal is finally over, with Entrada confirming this morning it has received the green light from the FDA to launch the ELEVATE-44-102 study.
This phase 1b study, which is expected to kick off in the first half of 2026, will assess ENTR-601-44 in adults with a confirmed mutation in the DMD gene amenable to exon 44 skipping.
The company has managed to trial the drug in healthy volunteers in a phase 1 study in the U.K. Regardless of the FDA’s decision, a study in DMD patients had already been scheduled to launch in that country in the second quarter of this year.
“Given the strength of our safety and target engagement data from our phase 1 clinical study and the profound unmet need in adults living with Duchenne, we are pleased to have obtained FDA clearance for the ELEVATE-44-102 study,” Entrada CEO Dipal Doshi said in a Feb. 24 release.
“The study will help assess the potential of ENTR-601-44 in both non-ambulatory and ambulatory adult patients who are unfortunately often left out of clinical studies due to the advanced stage of their disease,” Doshi added.
While ENTR-601-44 was stuck in regulatory limbo in the U.S., Sarepta secured FDA approval for its DMD gene therapy Elevidys in 2023. RegenXBio is also advancing its own gene therapy prospect into late-stage testing.
Elsewhere in the DMD space, daily corticosteroids from PTC Therapeutics and Santhera are approved by the FDA to treat patients 2 and older. Last year, Italfarmaco's Duzyvat became the first FDA-approved nonsteroidal drug for DMD patients 6 and older. Only this month, Cumberland Pharmaceuticals’ active thromboxane receptor antagonist scored a phase 2 win in DMD, and a number of other biotechs are advancing in the space.