{"id":959569,"date":"2026-05-07T07:16:04","date_gmt":"2026-05-07T11:16:04","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/entrada-therapeutics-announces-positive-topline-results-from-cohort-1-of-participants-with-duchenne-muscular-dystrophy-treated-with-entr-601-44-in-phase-1-2-elevate-44-201-study\/"},"modified":"2026-05-07T07:16:04","modified_gmt":"2026-05-07T11:16:04","slug":"entrada-therapeutics-announces-positive-topline-results-from-cohort-1-of-participants-with-duchenne-muscular-dystrophy-treated-with-entr-601-44-in-phase-1-2-elevate-44-201-study","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/entrada-therapeutics-announces-positive-topline-results-from-cohort-1-of-participants-with-duchenne-muscular-dystrophy-treated-with-entr-601-44-in-phase-1-2-elevate-44-201-study\/","title":{"rendered":"Entrada Therapeutics Announces Positive Topline Results from Cohort 1 of Participants with Duchenne Muscular Dystrophy Treated with ENTR-601-44 in Phase 1\/2 ELEVATE-44-201 Study"},"content":{"rendered":"
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\n — Achieved the primary objective with favorable safety and tolerability, no discontinuations and no serious adverse events —<\/em>\n <\/p>\n

\n — Markers of kidney function via eGFR, Cystatin C and magnesium were all within normal ranges and comparable to placebo —<\/em>\n <\/p>\n

\n — Observed lower plasma exposure in Cohort 1 participants who are all between six and 17 years of age when compared with healthy adult volunteers; A similar trend was seen between recently received juvenile and adult NHP PK data — <\/em>\n <\/p>\n

\n — Consequently, Cohort 1 demonstrated an increase of 2.36% in dystrophin over a baseline of 4.00% and an increase of 2.31% in exon skipping over a baseline of 2.66% in treated participants —<\/em>\n <\/p>\n

\n — Statistically significant and potentially differentiated improvement in treated participants versus placebo in Time to Rise velocity, a clinically validated functional measurement —<\/em>\n <\/p>\n

\n — Company\u2019s updated PK modeling predicts Cohort 2, building upon Cohort 1 data and combined with the recently received juvenile NHP data, will result in a significant increase of plasma AUC and substantially higher dystrophin levels with continued benefit in muscle function —<\/em>\n <\/p>\n

\n — Company has initiated dosing of ELEVATE-44-201 Cohort 2 at the increased dose of 12 mg\/kg and is on track to report data by year-end 2026 —<\/em>\n <\/p>\n

\n — Entrada to host investor webcast and conference call today, Thursday, May 7, at 8:30 a.m. ET — <\/em>\n <\/p>\n

BOSTON, May 07, 2026 (GLOBE NEWSWIRE) — Entrada Therapeutics, Inc. (Nasdaq: TRDA) today announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose (MAD) 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\u00a0DMD<\/em> 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\u2019s Duchenne muscular dystrophy (DMD) franchise, or placebo. Muscle biopsies were performed at the time of screening and six weeks after the last dose.<\/p>\n

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.<\/p>\n

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\n Table 1: Demographics and baseline characteristics<\/em>
\n <\/strong>\n <\/p>\n

\n \n <\/p>\n

The results demonstrated a favorable safety and tolerability profile with no reported serious adverse events (SAEs) and no adverse events (AEs) leading to discontinuation from the study. Markers of kidney function were normal.<\/p>\n

\u201cThe 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 velocity across participants treated with ENTR-601-44. TTR velocity is an approvable clinical endpoint in Phase 3 studies and importantly, ENTR-601-44\u2019s TTR velocity data are compelling and we believe differentiated,\u201d said Dipal Doshi, Chief Executive Officer at Entrada Therapeutics. \u201cWe 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 (NHP) 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.\u201d<\/p>\n

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 (up to 18 mg\/kg) to follow.<\/p>\n

Natarajan Sethuraman, PhD, President of R&D at Entrada Therapeutics, said, \u201cWe believe we have a highly differentiated delivery mechanism, including the ability to access quiescent satellite cells. Access to satellite cells enables the repair of existing muscle fibers and importantly, the formation of new healthy fibers. These drivers are emerging as a potentially significant competitive differentiator and may explain why the dystrophin levels in Cohort 1 were sufficient to improve TTR velocity.\u201d<\/p>\n

Dr. Sethuraman further commented, \u201cSafety and functional benefit are at the forefront of consideration for patients. Our Cohort 1 data show that ENTR-601-44 is safe at the 6 mg\/kg dose and is promoting early functional benefit which represent an important point of differentiation versus other approved and investigational approaches. We look forward to seeing the results from our Cohort 1 open-label and Cohort 2 MAD study later this year.\u201d<\/p>\n

\n Highlights from the topline results of Cohort 1 ELEVATE-44-201 include:<\/strong>\n <\/p>\n

\n Safety and tolerability<\/strong>\n <\/p>\n