Entrada Therapeutics can restart the development of its Duchenne muscular dystrophy (DMD) treatment, ENTR-601-44, in the US as the Food and Drug Administration (FDA) has lifted the clinical hold on the candidate.
The US regulatory agency placed a hold on the therapy back in December 2022 while it asked the biotech to gather “additional information.” Entrada did not disclose many details on the reason behind the hold. In November 2023, it noted that despite meeting the FDA’s request for the data, it “declined to lift the clinical hold”.
Entrada’s stock was up by 8.09% at pre-market trading today, compared to the market close yesterday (24 February). The company’s market cap currently stands at $480.8 million.
Entrada’s plans for ENTR-601-44
Earlier this month, the company released plans to start a multiple ascending dose (MAD), randomized, placebo-controlled Phase I/II ELEVATE-44-201 trial evaluating ENTR-601-44 in DMD in the UK. Entrada has received clearance from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee to start the trial.
The study is expected to kick off in Q2 this year and will enroll up to 32 non-ambulatory and ambulatory adult patients with DMD who are exon 44 skipping amenable. Entrada also plans to “evaluate target engagement as measured by exon skipping and dystrophin production, and pharmacokinetics” as part of the trial.
“Given the strength of our safety and target engagement data from our Phase I 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,” said Dipal Doshi, CEO of Entrada Therapeutics in a press release.
Adding that: “ELEVATE-44-102 will provide clinical experience from this important population for our growing data package in support of what we believe will be a best-in-class therapy.”
In the US, Entrada plans to start a Phase Ib study, which is expected to start in the first half of 2026. The trial will evaluate ENTR-601-44’s efficacy and safety in adults with a confirmed mutation in the DMD gene amenable to exon 44 skipping.
In the two years ENTR-601-44’s development was stuck due to clinical hold, DMD space has seen multiple advancements. In 2023, The US FDA approved a gene therapy for DMD, Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl). The therapy has since expanded its US label to include individuals with DMD with a confirmed mutation in the DMD gene who are at least 4 years of age.
