Entrada Therapeutics (NASDAQ: TRDA) has announced positive 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 of ENTR-601-44 in ambulatory participants ages four to 20 with a confirmed mutation in the DMD gene amenable to exon 44 skipping.
According to Entrada, the results demonstrated a favorable safety and tolerability profile with no reported serious adverse events and no adverse events leading to discontinuation from the study. Markers of kidney function were normal.
In a statement, Dipal Doshi, CEO at Entrada, commented, “The first dosing cohort readout from ELEVATE-44-201 is a major step forward, showing that ENTR-601-44 has a strong safety profile and is driving important, clinically meaningful and potentially differentiated early functional benefits. We were very encouraged to see that the Cohort 1 data delivered statistically significant improvement in Time to Rise (TTR) velocity across participants treated with ENTR-601-44. TTR velocity is an approvable clinical endpoint in Phase 3 studies and importantly, ENTR-601-44’s TTR velocity data are compelling and we believe differentiated.”
He added, “We were initially surprised to see a significant difference in the pharmacokinetics between juveniles and adults; however, the consistency seen between our recently received juvenile nonhuman primate data and the Cohort 1 participant data explains these differences. This clear explanation gives us confidence that we will see higher plasma exposure, which we expect will drive continued functional responses in Cohort 2. We are incredibly grateful to those living with Duchenne, their care partners and the study investigators and personnel who are taking part in our clinical study.”
