Entrada Therapeutics Inc. (Nasdaq: TRDA) reported initial Phase 1/2 results for ENTR-601-45, an investigational genetic medicine being developed for people with Duchenne muscular dystrophy who are amenable to exon 45 skipping.
The October 14 results came from the first cohort of the ELEVATE-45-201 study. Eight ambulatory participants between four and 20 years old were randomized 3:1 to receive ENTR-601-45 or placebo. Participants received three intravenous doses of 5 mg/kg, administered once every six weeks.
Entrada said the cohort met its primary safety and tolerability objectives. Headache was the most common treatment-emergent adverse event, with five events reported across two participants. One participant experienced two serious treatment-emergent adverse events—vomiting and fever—which subsequently resolved.
The company reported no treatment discontinuations, hypomagnesemia or identified renal-safety concerns. All eight participants entered the study’s open-label Phase 2 portion.
Biomarker results showed a mean 0.10% increase from baseline in dystrophin production among six treated participants, compared with a mean 0.02% decrease in the placebo group. The company also reported a mean 0.15% increase in exon skipping among five evaluable treated participants, compared with a 0.04% decrease for placebo.
No functional benefit was expected or observed after three doses at the initial 5 mg/kg level. The results therefore provide early safety and biomarker information but do not establish that ENTR-601-45 improves strength, mobility or other clinical outcomes. The treated group contained only six participants, and the dystrophin analysis used a nominal p-value without adjustment for multiple comparisons.
“Establishing a favorable safety profile in Cohort 1 was our main objective,” President of Research and Development Natarajan Sethuraman said.
ENTR-601-45 combines an exon-skipping oligonucleotide with Entrada’s Endosomal Escape Vehicle technology. It is designed to restore the messenger RNA reading frame and allow production of a shortened but potentially functional form of dystrophin. Entrada estimates that approximately 9% of the global Duchenne population may be amenable to exon 45 skipping.
Enrollment in the study’s second cohort is complete, with participants receiving 10 mg/kg. Entrada expects to report those results during the first half of 2027. A third cohort is planned to evaluate doses of up to 15 mg/kg.
Entrada reported $223 million in cash, cash equivalents and marketable securities as of June 30, 2026, down from $295.7 million at the end of 2025. The company recorded a second-quarter net loss of approximately $42.8 million and expects its existing capital to fund operations into the third quarter of 2027.
Upcoming Catalysts
- ELEVATE-45-201 Cohort 2 data during the first half of 2027
- Subsequent results from the planned Cohort 3 dose escalation
- Longer-term safety and efficacy data from the open-label portion
- Additional clinical updates from Entrada’s broader Duchenne pipeline
Sources
- Entrada Therapeutics announces ELEVATE-45-201 Cohort 1 results — October 14, 2026
- Entrada Therapeutics Form 10-Q for the quarter ended June 30, 2026
- Entrada Therapeutics Duchenne muscular dystrophy program
Editorial Disclosure
This article is based entirely on publicly available information, including company announcements and SEC filings. Securities discussed or referenced include Entrada Therapeutics Inc. (Nasdaq: TRDA). Biotech Stocks Daily has not received compensation from Entrada Therapeutics, its management, investor relations representatives or any third party for this specific article. Biotech Stocks Daily may have current or past paid business relationships with other companies, which do not influence the content or conclusions of this article. No staff member or principal of Biotech Stocks Daily holds a position in any security mentioned at the time of publication.
Confirmed sources include Entrada Therapeutics’ October 14, 2026 clinical announcement, its Form 10-Q for the quarter ended June 30, 2026 and publicly available information concerning its Duchenne muscular dystrophy programs. Information regarding ENTR-601-45’s mechanism, potential benefits and anticipated development timelines originated primarily from Entrada and has not been independently validated by Biotech Stocks Daily.
ENTR-601-45 remains investigational and has not received regulatory approval. The disclosed results came from eight participants at the initial dose level, including six who received ENTR-601-45. No functional benefit was observed. Biomarker changes may not translate into clinical improvement, while safety findings or results from higher-dose and longer-duration cohorts may differ materially.
Financial information reflects Entrada’s position as of June 30, 2026. The company has incurred recurring operating losses and used approximately $74.2 million in operating cash during the first six months of 2026. Its projected cash runway into the third quarter of 2027 is a forward-looking estimate that depends on its operating plans and other assumptions.
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