Cohort 1 data de-risks the safety profile at the starting dose and provides proof-of-mechanism with dystrophin production. The key catalyst is Cohort 2 data in H1 2027 at 2x the dose, where the company expects significantly higher dystrophin expression. Monitor for any IND/CTA updates and the ELEVATE-44-201 IDMC recommendation to proceed to 18 mg/kg as a read-across for the platform.
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Executive Summary
Entrada Therapeutics announced positive topline data from Cohort 1 of the Phase 1/2 ELEVATE-45-201 study of ENTR-601-45 in Duchenne muscular dystrophy patients amenable to exon 45 skipping. The 5 mg/kg dose met primary safety/tolerability objectives and showed a 0.10% mean increase in dystrophin production (nominal p=0.03). Cohort 2 (10 mg/kg) enrollment is complete with data expected in H1 2027.
Key Facts
- Cohort 1 of ELEVATE-45-201 met primary objectives: favorable safety and tolerability at 5 mg/kg
- Mean dystrophin increase of 0.10% from baseline in treated participants (n=6, nominal p=0.03) vs 0.02% decrease on placebo
- Mean exon skipping increase of 0.15% from baseline in treated participants (n=5) vs 0.04% decrease on placebo
- No study discontinuations; all participants enrolled in open-label extension
- No hypomagnesemia or renal safety concerns; renal markers within normal ranges
- Plasma exposures consistent with juvenile NHP predictions; company expects significant dystrophin increase at higher doses
- Cohort 2 (10 mg/kg) enrollment complete; data expected H1 2027; Cohort 3 up to 15 mg/kg to follow
- ENTR-601-45 targets ~9% of DMD patient population globally
Financial Impact
No financial data in filing. Clinical-stage biotech with no approved products or reported revenue.
Risk Factors
- Dystrophin increase of 0.10% is very low absolute level; functional benefit remains unproven
- No functional benefit observed or expected at this dose; efficacy entirely dependent on higher dose cohorts
- Small sample size (n=6 treated, n=2 placebo) limits statistical reliability
- Clinical-stage biotech with no approved products; significant cash burn risk
- Competing exon-skipping therapies (e.g., Sarepta's Casimersen) already approved
Investment Themes
Documents Analyzed
This report is based on 5 SEC documents filed with EDGAR.
| Document | Accession Number |
|---|---|
| 8-K Filing (Primary) | 0001689375-26-000057 |
| Document: entradatherapeuticselevate.htm | 0001689375-26-000057 |
| Document: 0001689375-26-000057-index-headers.html | 0001689375-26-000057 |
| Document: 0001689375-26-000057-index.html | 0001689375-26-000057 |
| Document: 0001689375-26-000057.txt | 0001689375-26-000057 |
Filters
| Type | Now | ||||
|---|---|---|---|---|---|
Oct 14, 2026 13d ago | 8-K | $4.55 | awaiting T+60 | awaiting T+60 | $4.41 (−3.08%) |
Sep 9, 2026 6w ago | Insider Cluster | $6.42 | awaiting T+60 | awaiting T+60 | $4.41 (+31.31%) |
Sep 3, 2026 7w ago | Insider Cluster | $7.13 | awaiting T+60 | awaiting T+60 | $4.41 (−38.15%) |
Aug 31, 2026 8w ago | Insider Cluster | $7.07 | awaiting T+60 | awaiting T+60 | $4.41 (−37.62%) |
Aug 31, 2026 8w ago | Insider Cluster | $7.07 | awaiting T+60 | awaiting T+60 | $4.41 (−37.62%) |
Aug 31, 2026 8w ago | Insider Cluster | $7.07 | awaiting T+60 | awaiting T+60 | $4.41 (−37.62%) |
Aug 26, 2026 8w ago | Insider Cluster | $7.27 | awaiting T+60 | awaiting T+60 | $4.41 (−39.34%) |
Aug 10, 2026 11w ago | Insider Cluster | $7.04 | awaiting T+60 | awaiting T+60 | $4.41 (−37.36%) |
Aug 5, 2026 11w ago | 8-K | $7.06 | awaiting T+60 | awaiting T+60 | $4.41 (−37.54%) |
Aug 3, 2026 12w ago | Insider Cluster | $7.24 | awaiting T+60 | awaiting T+60 | $4.41 (−39.09%) |
US Market Status
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