Entrada Therapeutics reported early Phase 1/2 results for its Duchenne muscular dystrophy (DMD) candidate ENTR-601-44, showing a 2.36% increase in normalized dystrophin levels in muscle tissue six weeks after the last dose in the first patient cohort of the ELEVATE-44-201 study. The result fell below prior expectations from analysts and company executives, who had anticipated double-digit dystrophin increases.
The data were released on Thursday and triggered a sharp decline in the Boston-based biotech company’s stock price. Shares dropped by around 50% before the opening bell, with the stock later falling as much as 59% from the previous day’s close.
ENTR-601-44 is an oligonucleotide therapy designed to target exon 44 in dystrophin pre-mRNA. The treatment is intended to enable cells to skip the defective exon during RNA splicing, resulting in the production of a shorter but functional dystrophin protein. Duchenne muscular dystrophy is characterized by progressive muscle degeneration caused by a lack of functional dystrophin protein, with complications commonly affecting the heart and lungs.
Analysts reacted negatively to the readout, noting that the dystrophin increase was below forecasts and lower than previously reported data from competitor programs. Oppenheimer analysts described the outcome as “disappointing,” while William Blair analysts said the result missed both their base-case projection of 10% dystrophin restoration and management expectations.
Analysts compared the data with results previously reported for Avidity Biosciences’ delpacibart zotadirsen, also known as del-zota. Avidity previously reported an approximately 25% increase in dystrophin production after one year of treatment in a September 2025 update. No direct comparative studies between the therapies have been conducted.
At the J.P. Morgan Healthcare Conference earlier this year, Entrada CEO Dipal Doshi had outlined the company’s expectations for the program. “We expect to see double-digit dystrophin production, which is really important,” Doshi said at the time.
Despite the lower-than-expected dystrophin findings, analysts highlighted positive functional data from the study. Entrada reported an average improvement of 0.08 in time-to-rise (TTR) velocity, a measure of physical performance used to monitor disease progression and potential loss of ambulation in DMD patients. Oppenheimer analysts described the improvement as clinically significant and said it was larger than competitors’, suggesting a potential clinical benefit.
The company also reported a favorable safety profile for the 6-mg/kg dose evaluated in the first cohort. No serious adverse events or treatment discontinuations were recorded. Oppenheimer analysts said the clean safety profile allows for dose escalation to 12-18 mg/kg that can ultimately deliver a competitive profile.
Entrada attributed the limited dystrophin response to lower-than-expected plasma exposure in juvenile DMD patients. According to the company, exposure levels were roughly 50% lower than those observed in healthy adults and nonhuman primates. CEO Dipal Doshi stated that more recent data from juvenile nonhuman primates revealed a similar pattern.
Natarajan Sethuraman, president of research and development at Entrada, said during a conference call that juvenile nonhuman primate data demonstrated “a steep, nonlinear exon skipping response” at higher plasma concentrations, suggesting that higher doses could produce disproportionately greater exon skipping activity.
The ELEVATE-44-201 trial is continuing into a second cohort using a 12-mg/kg dose of ENTR-601-44, with dosing already underway. Entrada expects to report data from that group by the end of the year. The company also plans to evaluate a third dosing cohort as development of the therapy continues.
Entrada Therapeutics has shared promising early clinical data from its Duchenne muscular dystrophy (DMD) program, with Entrada reporting a 2.36% increase in dystrophin production among trial participants. The findings represent an important milestone for Entrada as the company advances innovative therapies targeting rare neuromuscular diseases.
Overview of the Entrada DMD Trial
The early-stage clinical trial evaluated Entrada’s investigational therapy in patients with Duchenne muscular dystrophy, a severe genetic disorder characterized by progressive muscle degeneration caused by the absence of functional dystrophin protein.
According to the reported data, Entrada observed measurable dystrophin restoration following treatment, suggesting the therapy may help address the underlying genetic cause of the disease.
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
