TRDA News & Events

Entrada Therapeutics announced topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 clinical study. ELEVATE-44-201 is a clinical study of ENTR-601-44 in ambulatory participants ages four to 20 with a confirmed mutation in the DMD gene amenable to exon 44 skipping. Study participants in Cohort 1 were randomized 3:1 to receive three doses of 6 mg/kg of ENTR-601-44, the lead investigational product in Entrada's Duchenne muscular dystrophy franchise, or placebo. Muscle biopsies were performed at the time of screening and six weeks after the last dose. The average age of treated participants in the Cohort 1 study was 9.3 years old with a mean age of disease onset of 2.2 years. Per protocol, all participants were ambulatory and all were on a stable dose of steroids. Baseline dystrophin in both the placebo and treatment population was also lower than that reported in competitive exon 44 skipping clinical studies. This is also notable, as treatment response generally correlates with higher baseline dystrophin levels. The results demonstrated a favorable safety and tolerability profile with no reported serious adverse events (SAEs) and no adverse events leading to discontinuation from the study. Markers of kidney function were normal. All study participants in Cohort 1 have now progressed to the open-label, Phase 2 portion of the study, where they will receive six additional doses of 6 mg/kg of ENTR-601-44. Additional study participants are now being dosed in Cohort 2, in which they will receive three doses of 12 mg/kg of ENTR-601-44 or placebo. The Company expects to report results from the Cohort 1 open-label study and Cohort 2 MAD study by year-end 2026, with data from Cohort 3 to follow. Favorable safety and tolerability with ENTR-601-44 at the 6 mg/kg dose. All treatment emergent adverse events were mild to moderate. No reported SAEs and no AEs leading to discontinuation from the study. The most common AE was headache. Markers of kidney function including eGFR, Cystatin C and magnesium were within normal ranges and comparable to placebo. There were no discontinuations and all eight Cohort 1 participants have transitioned to the open-label portion of the study.

Reports Q1 revenue $875,000, consensus $2.92M. "With the recently announced positive data from Cohort 1 of our ELEVATE-44-201 clinical study, this year has already delivered a significant clinical inflection point. Establishing that ENTR-601-44 demonstrated not only favorable safety and tolerability, but early and differentiated functional benefits at 6 mg/kg, is a clear milestone for the program as well as Entrada's neuromuscular pipeline," said Dipal Doshi, Chief Executive Officer at Entrada Therapeutics. "With cash runway into the third quarter of 2027, we are well positioned to achieve additional clinical inflection points throughout the year, including data from the first participant cohort of the ELEVATE-45-201 study, as well as the open-label and second cohort of the ELEVATE-44-201 study. The Company is also carefully evaluating the optimal timing for initiating the planned clinical studies of ENTR-601-50 and ENTR-601-51."