Entrada crashes as Duchenne therapy comes in 'below expectations' in early study. May 7, 2026 | Entrada Therapeutics saw a 2.36% dystrophin increase in patients treated with its oligonucleotide asset - a number that fell short of the company's prior guidance of a double-digit improvement. Entrada Therapeutics' investigational oligonucleotide increased dystrophin concentrations in a Phase 1/2 Duchenne muscular dystrophy but not to the degree that analysts or the company had been expecting. In the first dosing cohort of the Phase 1/2 ELEVATE-44-201 trial, ENTR-601-44 elicited a 2.36% increase in normalized dystrophin levels in muscle tissue six weeks after the last dose, according to a Thursday release - well short short of Entrada's prior guidance of a double-digit jump. Shares of the Boston-based biotech plummeted 50% before the opening bell on Thursday, hitting $8.01 apiece. Oppenheimer analysts called ENTR-601-44's performance "disappointing," noting that the result puts Entrada's dystrophin benefit lower than that of competitor Avidity Biosciences. In a Thursday morning investor note, the firm pointed to a September 2025 readout in which Avidity reported an approximately 25% increase in the protein for its own oligonucleotide therapy del-zota after one year of treatment. Importantly, no trials directly comparing the two have been conducted. William Blair had a similar assessment of Entrada's readout. "Measurements of dystrophin protein expression after ENTR-601-44 treatment, the main efficacy-based endpoint of focus for investors, showed a 2.36% increase from baseline, which missed our base case of 10% and also management's expectations," the analysts wrote in a Thursday note. On a more positive note, Oppenheimer homed in on ENTR-601-44's effects on time-to-rise (TTR) velocity, a robust measure of functional performance that, according to Entrada, is "used as an early prognostic factor for disease progression and loss of ambulation." Thursday's readout demonstrated an average TTR improvement of 0.08 in patients treated with Entrada's molecule. Oppenheimer called this magnitude of effect "clinically significant" and noted it is "larger than competitors, suggesting a potential clinical benefit." As for safety, Entrada touted a favorable overall profile for a 6-mg/kg dose of ENTR-601-44, with no serious side effects or adverse events triggering discontinuation. Oppenheimer found the "clean" safety profile encouraging, explaining that it "allows for dose escalation to 12-18mg/kg that can ultimately deliver a competitive profile." With Thursday's data, ELEVATE-44-201 will continue on to its second patient cohort, which will receive a 12-mg/kg dose of the drug. Dosing for this leg of the study has started, with data expected by year-end. ENTR-601-44 is an oligonucleotide therapy that binds to exon 44 in the dystrophin pre-mRNA, allowing the cell's splicing machinery to skip over the faulty exon, according to Entrada's website. This ultimately results in a shorter but functional dystrophin protein, helping maintain the function and integrity of muscle cells, which in patients with Duchenne muscular dystrophy (DMD) otherwise undergo progressive degeneration. Common complications of the disease include heart and lung problems, often leading to death. Sarepta Therapeutics' Amondys 45 and Vyondys 53 work similarly to ENTR-601-44, removing faulty exons from the final mRNA transcript and facilitating the expression of truncated but functional dystrophin. In November last year, the drugs failed their confirmatory Phase 3 study, unable to elicit significant motor improvements in patients over placebo. Sarepta has nevertheless submitted supplemental applications to the FDA for both drugs. Vyondys 53 was first approved in 2019 and Amondys 45 in 2021, both under the agency's accelerated pathway.

Entrada Therapeutics reported positive topline results from Cohort 1 of its ELEVATE-44-201 study in Duchenne muscular dystrophy, showing favourable safety and tolerability alongside early functional benefits. The study of ENTR-601-44 at 6 mg/kg demonstrated statistically significant improvement in Time to Rise velocity, with all adverse events classified as mild or moderate. The company reported cash, cash equivalents and marketable securities of $255 million as of 31 March 2026, providing runway into the third quarter of 2027. Entrada plans to report ELEVATE-45-201 Cohort 1 data in mid-2026 and ELEVATE-44-201 open-label period and Cohort 2 data by year-end 2026. Net loss for the first quarter was $39.7 million, compared to $17.3 million in the same period last year.

Entrada Therapeutics to announce topline results from Cohort 1 of participants with Duchenne muscular dystrophy treated with ENTR-601-44 in Phase 1/2 ELEVATE-44-201 study on May 7, 2026. BOSTON, May 06, 2026 (GLOBE NEWSWIRE) - Entrada Therapeutics, Inc. (Nasdaq: TRDA) will announce topline results 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 on Thursday, May 7, 2026. The Company will host an investor webcast and conference call at 8:30 a.m. ET to discuss these clinical results. The webcast can be accessed by visiting the Investor Relations section of the Company's website at www.entradatx.com. Analysts planning to participate during the Q&A portion of the live call can join the conference call at the audio-conferencing link here. The webcast will be archived and available for replay on the Entrada Therapeutics website for 90 days following the call. About Entrada Therapeutics Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of genetic medicines that engage intracellular targets that have long been considered inaccessible. Through proprietary, versatile and modular approaches, Entrada is advancing a robust development portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others. The Company's lead oligonucleotide programs are in development for the potential treatment of people living with Duchenne muscular dystrophy who are exon 44, 45, 50 and 51 skipping amenable. Entrada has partnered to develop a clinical-stage program, VX-670, for myotonic dystrophy type 1. Investor Contact Karla MacDonald Chief Corporate Affairs Officer [email protected] Media Contact Megan Prock McGrath CTD Comms, LLC [email protected]