Entrada Therapeutics (NASDAQ: TRDA) posts Q1 2026 results and promising Duchenne data
Filing Impact
Filing Sentiment
Form Type
8-K
Rhea-AI Filing Summary
Entrada Therapeutics reported first quarter 2026 results and detailed encouraging early data for its Duchenne muscular dystrophy programs. Cash, cash equivalents and marketable securities were $254.9 million as of March 31, 2026, and the company expects its cash runway to extend into the third quarter of 2027.
In the Phase 1/2 ELEVATE-44-201 study, Cohort 1 participants receiving ENTR-601-44 at 6 mg/kg showed favorable safety and tolerability, with no serious adverse events or discontinuations and kidney function markers remaining normal. Treated participants had a 2.36% increase in dystrophin over a 4.00% baseline and a 2.31% increase in exon skipping over a 2.66% baseline.
The study also showed a statistically significant improvement in Time to Rise velocity versus placebo, more than three times the minimal clinically important difference, suggesting early functional benefit. Financially, collaboration revenue declined to $0.9 million from $20.6 million a year earlier, while R&D expenses were $33.1 million and net loss widened to $39.7 million, or $0.95 per share.
Positive
- Strong early ENTR-601-44 signal: Cohort 1 in the Phase 1/2 ELEVATE-44-201 study showed favorable safety, no serious adverse events or discontinuations, a 2.36% dystrophin increase over 4.00% baseline, and statistically significant Time to Rise velocity improvement exceeding three times the minimal clinically important difference.
- Solid cash runway: Cash, cash equivalents and marketable securities of $254.9 million as of March 31, 2026 are expected to fund operations into the third quarter of 2027, supporting multiple upcoming Duchenne and ocular program milestones.
Negative
- Revenue decline and higher losses: Collaboration revenue fell to $0.9 million from $20.6 million in the prior-year quarter, while net loss more than doubled to $39.7 million, reflecting reduced collaboration contribution and continued R&D spending.
Insights
Early ENTR-601-44 data show clean safety and meaningful functional signals.
Entrada Therapeutics reported positive topline Cohort 1 results from its Phase 1/2 ELEVATE-44-201 trial in exon 44–amenable Duchenne muscular dystrophy. ENTR-601-44 at 6 mg/kg achieved the primary safety objective, with no serious adverse events, no discontinuations and kidney markers comparable to placebo.
Treated participants showed a 2.36% increase in dystrophin over a 4.00% baseline and a 2.31% increase in exon skipping over a 2.66% baseline. Importantly, Time to Rise velocity improved significantly versus placebo and exceeded three times the minimal clinically important difference, indicating clinically meaningful early functional benefit in this small cohort.
All eight participants have moved into the open-label Phase 2 portion at 6 mg/kg, while Cohort 2 at 12 mg/kg has begun dosing with data expected by year-end 2026. Updated pharmacokinetic modeling, supported by juvenile nonhuman primate data, underpins expectations for higher exposure and dystrophin levels at increased doses, though confirmation will depend on forthcoming Cohort 2 and Cohort 3 readouts.
Pipeline progress offsets weaker collaboration revenue and wider losses.
For Q1 2026, Entrada reported collaboration revenue of $0.9 million, sharply lower than $20.6 million a year earlier, mainly because collaboration work on VX-670 substantially completed in early 2025. Research and development expenses edged up to $33.1 million, reflecting continued investment in Duchenne programs.
General and administrative expenses were stable at $10.1 million. Net loss widened to $39.7 million from $17.3 million, or $0.95 per share versus $0.42. Despite higher losses, the balance sheet remains solid with $254.9 million in cash, cash equivalents and marketable securities and total stockholders’ equity of $270.9 million. Management believes this cash will fund operations into the third quarter of 2027.
Taken together, the quarter combines a materially reduced revenue contribution from the Vertex collaboration with higher operating loss, but also delivers important clinical de-risking for the lead Duchenne asset. Subsequent quarters will show how spending and potential future collaboration income evolve alongside key data milestones in 2026.
8-K Event Classification
3 items: 2.02, 7.01, 9.01
3 items
Item 2.02
Results of Operations and Financial Condition
Financial
Disclosure of earnings results, typically an earnings press release or preliminary financials.
Item 7.01
Regulation FD Disclosure
Disclosure
Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, and exhibit attachments filed with this report.
Key Figures
Cash, cash equivalents and marketable securities: $254.9M
Collaboration revenue: $0.9M
Research and development expenses: $33.1M
+5 more
8 metrics
Cash, cash equivalents and marketable securities
$254.9M
As of March 31, 2026; expected to fund operations into Q3 2027
Collaboration revenue
$0.9M
Three months ended March 31, 2026; down from $20.6M in 2025
Research and development expenses
$33.1M
Three months ended March 31, 2026; primarily DMD program costs
Net loss
$39.7M
Three months ended March 31, 2026; vs. $17.3M in 2025
Net loss per share
$0.95
Basic and diluted, Q1 2026; weighted‑average 41.8M shares
Dystrophin increase
2.36% over 4.00% baseline
ENTR-601-44 treated participants, ELEVATE-44-201 Cohort 1
Exon skipping increase
2.31% over 2.66% baseline
ENTR-601-44 treated participants, ELEVATE-44-201 Cohort 1
Total stockholders’ equity
$270.9M
As of March 31, 2026
Key Terms
Time to Rise velocity, multiple ascending dose, Endosomal Escape Vehicle, exon 44 skipping amenable, +2 more
6 terms
Time to Rise velocity medical
"statistically significant improvement in Time to Rise (TTR) velocity in the majority of participants"
multiple ascending dose medical
"global Phase 1/2 multiple ascending dose (MAD) portion of the clinical study of ENTR-601-44"
A multiple ascending dose is a method used in testing new medicines where small groups of people receive gradually larger amounts of the drug over time. This approach helps researchers find the safest and most effective dose without causing too many side effects. For investors, it signals ongoing steps in drug development that can impact a company's potential success or approval prospects.
Endosomal Escape Vehicle medical
"ENTR-601-44 is a proprietary Endosomal Escape Vehicle (EEV™)-conjugated oligonucleotide"
exon 44 skipping amenable medical
"participants living with DMD who are amenable to exon 44 skipping"
collaboration revenue financial
"Collaboration revenue was $0.9 million for the first quarter of 2026"
Payments a company receives from business partners under joint development, commercialization, or licensing agreements—often including upfront fees, milestone payments and a share of product sales—earned as part of a formal collaboration. Investors care because this income can validate a company’s technology, boost cash flow and reduce the cost of bringing products to market, but it is often lumpy and contingent on future milestones and partner performance, so it affects revenue predictability and valuation.
Rare Pediatric Disease Designation regulatory
"the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to ENTR-601-44"
A rare pediatric disease designation is an official regulatory status given to a drug or therapy that targets a serious or life‑threatening condition primarily affecting children and is uncommon in the population. It matters to investors because the status often brings financial and development perks — such as tax credits, reduced fees, faster review and periods of market protection — which can lower costs, speed approval and improve the commercial outlook; think of it as a VIP pass that makes bringing a scarce, child‑focused treatment to market easier and potentially more profitable.
Earnings Snapshot
Collaboration revenue: $0.9M
Q1 2026
Collaboration revenue
$0.9M
Net loss
$39.7M
Net loss per share
$0.95
Cash, cash equivalents and marketable securities
$254.9M
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): May 7, 2026
(Exact name of registrant as specified in its charter)
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company x
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o
Item 2.02 Results of Operations and Financial Condition.
On May 7, 2026, Entrada Therapeutics, Inc. (the “Company”) announced its financial results for the quarter ended March 31, 2026 and other corporate updates. A copy of the press release in connection with the announcement is being furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in Item 2.02 of this Current Report on Form 8-K (including Exhibit 99.1 attached hereto) is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 7.01 Regulation FD Disclosure.
On May 7, 2026, the Company issued a press release titled “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.” A copy of the press release is furnished hereto as Exhibit 99.2 and incorporated herein by reference.
The information in Item 7.01 of this Current Report on Form 8-K, including the accompanying Exhibit 99.2, is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Exchange Act or otherwise subject to the liability of such section, nor shall it be deemed incorporated by reference in any filing under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d)Exhibits.
The following exhibit relating to Item 2.02 of this Form 8-K shall be deemed to be furnished and not filed:
99.1 | Press Release issued by Entrada Therapeutics, Inc. on May 7, 2026 | ||||
| 99.2 | Press Release issued by Entrada Therapeutics, Inc. on May 7, 2026 | ||||
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). | ||||
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Entrada Therapeutics, Inc. | ||||||||
| Date: May 7, 2026 | /s/ Dipal Doshi | |||||||
| Dipal Doshi | ||||||||
| Chief Executive Officer | ||||||||
Entrada Therapeutics Reports First Quarter 2026 Financial Results
-- Announced positive ELEVATE-44-201 Cohort 1 topline results in Duchenne muscular dystrophy showing favorable safety, tolerability and early functional benefit --
-- Company on track to report ELEVATE-45-201 Cohort 1 data in mid-2026, as well as ELEVATE-44-201 open-label period and Cohort 2 data by year-end 2026 --
-- Cash runway expected into Q3 2027 with $255 million in cash, cash equivalents and marketable securities as of March 31, 2026 --
-- Entrada to host investor webcast and conference call today, Thursday, May 7, at 8:30 a.m. ET --
BOSTON, May 7, 2026 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA) today reported financial results for the first quarter ended March 31, 2026, and highlighted recent business updates.
“With the recently announced positive data from Cohort 1 of our ELEVATE-44-201 clinical study, this year has already delivered a significant clinical inflection point. Establishing that ENTR-601-44 demonstrated not only favorable safety and tolerability, but early and differentiated functional benefits at 6 mg/kg, is a clear milestone for the program as well as Entrada’s neuromuscular pipeline,” said Dipal Doshi, Chief Executive Officer at Entrada Therapeutics. “With cash runway into the third quarter of 2027, we are well positioned to achieve additional clinical inflection points throughout the year, including data from the first participant cohort of the ELEVATE-45-201 study, as well as the open-label and second cohort of the ELEVATE-44-201 study. The Company is also carefully evaluating the optimal timing for initiating the planned clinical studies of ENTR-601-50 and ENTR-601-51.”
Recent Corporate Highlights
Clinical-Stage Development Pipeline: Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy (DMD) in the U.K., EU and U.S., complementing the ongoing clinical progress of its myotonic dystrophy type 1 (DM1) partnership (VX-670) with Vertex.
•ELEVATE-44-201: Announced positive topline results from Cohort 1 in the global Phase 1/2 multiple ascending dose (MAD) portion of the clinical study of ENTR-601-44 in ambulatory participants living with DMD who are amenable to exon 44 skipping. Study participants in Cohort 1 received three doses of 6 mg/kg of ENTR-601-44, the lead investigational product in Entrada’s DMD franchise, or placebo. Topline results demonstrated meaningful and potentially differentiated early functional benefits including statistically significant improvement in Time to Rise (TTR) velocity in the majority of participants treated with ENTR-601-44. Results also demonstrated a favorable safety and tolerability profile, all adverse events (AEs) were mild or moderate, there were no reported serious adverse events (SAEs), and no AEs leading to discontinuation from the study. Plasma markers for kidney function were normal. The Company is on track to report data from the Cohort 1 open-label period and Cohort 2 (12 mg/kg) MAD by year-end 2026, with data from Cohort 3 MAD (up to 18 mg/kg) to follow.
•ELEVATE-44-102: The Company believes this clinical study, in the underserved adult patient population with advanced disease, would be best to initiate at the highest advisable starting
dose. Following a review of safety, pharmacokinetic and pharmacodynamic data from Cohort 1 of the ELEVATE-44-201 study in the U.K. and EU, the Company plans to re-engage with the FDA to discuss increasing the planned doses in this clinical study. As such, the Company will provide an update on clinical study design and timing following interactions with the FDA.
•ELEVATE-45-201: Completed enrollment and initiated dosing in Cohort 1 of the global Phase 1/2 MAD clinical study of ENTR-601-45 in ambulatory participants living with DMD who are amenable to exon 45 skipping. The Company is on track to report data from Cohort 1 (5 mg/kg) in mid-2026, with data from Cohort 2 and Cohort 3 (up to 10 mg/kg and 15 mg/kg, respectively) to follow.
•ELEVATE-50-201: The Company received regulatory authorization from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee to initiate a Phase 1/2 MAD clinical study of ENTR-601-50 in ambulatory participants living with DMD who are amenable to exon 50 skipping. The Company expects to submit additional regulatory applications and obtain authorization in the EU following a review of data from the ongoing studies of its lead programs.
•ENTR-601-51: The Company has completed Clinical Trial Authorization (CTA)-enabling studies for people living with DMD who are amenable to exon 51 skipping, which is applicable to the largest sub-population of exon skipping amenable patients. The Company expects to submit regulatory applications and obtain authorization following a review of data from the ongoing studies of its lead programs.
•VX-670: Vertex continues to enroll and dose the MAD portion of the GALILEO global Phase 1/2 clinical study of VX-670 in people with DM1. The study assesses both safety and efficacy and Vertex is on track to share results during the second half of 2026.
Expanding Preclinical Pipeline: The Company has generated compelling preclinical data from programs focused on ocular and metabolic diseases. The pipeline includes the advancement of two novel oligonucleotide-based programs for the potential treatment of inherited retinal diseases, where there exists high unmet need. The first ocular candidate, ENTR-801, for the potential treatment of Usher syndrome type 2A (USH2A) was announced in December 2025. The Company plans to announce a second clinical candidate in ocular disease in the second half of 2026 and will provide additional details on its clinical development strategy at that time.
Upcoming Investor Conferences
•H.C. Wainwright 4th Annual BioConnect Investor Conference, New York, NY on May 19, 2026
•2026 Jefferies Global Healthcare Conference, New York, NY on June 3, 2026
•Goldman Sachs 47th Annual Global Healthcare Conference 2026, Miami Beach, FL on June 8, 2026
Investor Webcast and Conference Call Information
Entrada Therapeutics will host an investor webcast and conference call today, Thursday, May 7, 2026, at 8:30 a.m. ET to discuss financial results for the first quarter ended March 31, 2026, recent business updates and topline results from Cohort 1 of the Phase 1/2 ELEVATE-44-201 study. 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 (https://edge.media-server.com/mmc/p/dpiu2bfu/). The webcast will be archived and available for replay on the Entrada Therapeutics website for 90 days following the call.
Patients and Their Care Partners
Patients and their care partners are a critical part of our community, and we are committed to keeping them informed and connected. To receive community updates in real time and read today’s update, please visit www.entradatx.com/patients.
First Quarter 2026 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $254.9 million as of March 31, 2026, compared to $295.7 million as of December 31, 2025. The decrease was primarily driven by cash used to fund operations. Based on current operating plans, the Company believes that its cash, cash equivalents and marketable securities as of March 31, 2026 will be sufficient to fund its operations into the third quarter of 2027.
Collaboration Revenue: Collaboration revenue was $0.9 million for the first quarter of 2026, compared to $20.6 million for the same period in 2025. This decrease is primarily attributable to the substantial completion of the collaboration research plan activities associated with VX-670 during the first quarter of 2025.
Research & Development (R&D) Expenses: R&D expenses were $33.1 million for the first quarter of 2026, compared to $32.1 million for the same period in 2025. The increase was primarily driven by additional costs incurred related to the Company’s DMD programs.
General & Administrative (G&A) Expenses: G&A expenses were $10.1 million for the first quarter of 2026, compared to $10.3 million for the same period in 2025. The decrease was primarily driven by fewer professional services costs incurred.
Net Loss: Net loss was $39.7 million for the first quarter of 2026, compared to $17.3 million for the same period in 2025.
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.
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.
For more information about Entrada, please visit our website, www.entradatx.com, and follow us on LinkedIn.
Forward-Looking Statements
This press release contains express and implied forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Entrada’s strategy, future operations, prospects and plans, objectives of management, the validation and differentiation of Entrada’s approach and EEV platform and its ability to provide a potential treatment for patients, expectations regarding Entrada’s Phase 1/2 MAD clinical study of ENTR-601-44, including the timing of data from the Cohort 1 open-label period and Cohort 2 by year-end 2026 with data from Cohort 3 to follow, expectations regarding the initiation of the planned ELEVATE-44-102 study in the U.S., including plans to re-engage with the FDA to discuss increasing planned doses, the ability to recruit for and complete the global Phase 2 clinical studies of ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51, the potential therapeutic benefits of Entrada’s EEV product candidates, including the potential for ENTR-601-44 to be a transformative treatment option, the potential of TTR velocity data observed in Cohort 1 to predict early functional benefit, the potential for a deepening of functional responses, continued functional benefit and higher dystrophin levels with increase in plasma exposure during the Cohort 1 open-label Phase 2 portion of the study, the potential for further enhanced muscle function and a meaningful increase in dystrophin in Cohort 2, expectations regarding Entrada's Phase 1/2 MAD clinical study of ENTR-601-45, including the timing of data from Cohort 1 in mid-2026, with data from Cohort 2 and Cohort 3 to follow, expectations regarding regulatory filings in the EU for the planned Phase 1/2 MAD clinical study of ENTR-601-50, expectations regarding regulatory filings for the ENTR-601-51 program, the potential therapeutic benefits of Entrada’s EEV product candidates and the ability to advance therapeutic candidates in indications beyond neuromuscular disease, including but not limited to ocular disease, expectations regarding the timing of nomination of a second clinical candidate for ocular disease in the second half of 2026, and the continued development and advancement of ENTR-601-44, ENTR-601-45, ENTR-601-50, and ENTR-601-51 for the treatment of DMD and ENTR-801 for the potential treatment of Usher syndrome type 2A and the partnered product candidate VX-670 for the potential treatment of DM1, expectations regarding the progress and success of Entrada’s collaboration with Vertex, including the timing of results from the MAD portion of the global Phase 1/2 study of the VX-670 program in the second half of 2026, the ability to continue to expand and develop additional therapeutic programs and modalities, including further exon skipping programs, and the sufficiency of its cash resources into the third quarter of 2027, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Entrada may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities and the initiation and completion of preclinical studies and clinical studies; uncertainties as to the availability and timing of results from preclinical and clinical studies; the timing of and Entrada’s ability to submit and obtain regulatory clearance and initiate clinical studies; whether results from preclinical studies or clinical studies will be predictive of the results of later preclinical studies and clinical studies; whether Entrada’s cash resources will be sufficient to fund the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Entrada’s filings with the Securities and Exchange Commission (SEC), including the Company’s most recent Form 10-K and in subsequent filings Entrada may make with the SEC. In addition, the forward-looking statements included in this press release represent Entrada’s views as of the date of this press release. Entrada anticipates that
subsequent events and developments will cause its views to change. However, while Entrada may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Entrada’s views as of any date subsequent to the date of this press release.
ENTRADA THERAPEUTICS, INC.
Condensed Consolidated Statements of Operations (Unaudited)
(In thousands, except share and per share amounts)
| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Collaboration revenue | $ | 875 | $ | 20,558 | |||||||
| Operating expenses: | |||||||||||
| Research and development | 33,054 | 32,074 | |||||||||
| General and administrative | 10,124 | 10,274 | |||||||||
| Total operating expenses | 43,178 | 42,348 | |||||||||
| Loss from operations | (42,303) | (21,790) | |||||||||
| Other income: | |||||||||||
| Interest and other income | 2,624 | 4,441 | |||||||||
| Total other income | 2,624 | 4,441 | |||||||||
| Loss before provision for income taxes | (39,679) | (17,349) | |||||||||
| Provision for income taxes | 38 | — | |||||||||
| Net loss | $ | (39,717) | $ | (17,349) | |||||||
| Net loss per share, basic and diluted | $ | (0.95) | $ | (0.42) | |||||||
Weighted‑average common shares outstanding, basic and diluted | 41,836,275 | 41,073,732 | |||||||||
ENTRADA THERAPEUTICS, INC.
Condensed Consolidated Balance Sheet Data (Unaudited)
(In thousands)
| March 31, | December 31, | ||||||||||
| 2026 | 2025 | ||||||||||
| Cash, cash equivalents and marketable securities | $ | 254,859 | $ | 295,698 | |||||||
| Total assets | $ | 335,518 | $ | 377,378 | |||||||
| Total liabilities | $ | 64,664 | $ | 71,245 | |||||||
| Total stockholders’ equity | $ | 270,854 | $ | 306,133 | |||||||
Investor Contact
Karla MacDonald
Chief Corporate Affairs Officer
kmacdonald@entradatx.com
Patient Advocacy Contact
Sarah Friedhoff
Head of Patient Advocacy
patientadvocacy@entradatx.com
Media Contact
Megan Prock McGrath
CTD Comms, LLC
megan@ctdcomms.com
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
-- Achieved the primary objective with favorable safety and tolerability, no discontinuations and no serious adverse events --
-- Markers of kidney function via eGFR, Cystatin C and magnesium were all within normal ranges and comparable to placebo --
-- 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 --
-- 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 --
-- Statistically significant and potentially differentiated improvement in treated participants versus placebo in Time to Rise velocity, a clinically validated functional measurement --
-- Company’s 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 --
-- 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 --
-- Entrada to host investor webcast and conference call today, Thursday, May 7, at 8:30 a.m. ET --
BOSTON, May 7, 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 DMD 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’s Duchenne muscular dystrophy (DMD) franchise, or placebo. Muscle biopsies were performed at the time of screening and six weeks after the last dose.
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.
Table 1: Demographics and baseline characteristics
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.
“The 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’s TTR velocity data are compelling and we believe differentiated,” said Dipal Doshi, Chief Executive Officer at Entrada Therapeutics. “We 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.”
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.
Natarajan Sethuraman, PhD, President of R&D at Entrada Therapeutics, said, “We 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.
Dr. Sethuraman further commented, “Safety 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.”
Highlights from the topline results of Cohort 1 ELEVATE-44-201 include:
Safety and tolerability
•Favorable safety and tolerability with ENTR-601-44 at the 6 mg/kg dose.
•All treatment emergent adverse events (TEAEs) were mild to moderate.
•No reported SAEs and no AEs leading to discontinuation from the study.
•The most common AE was headache.
•Markers of kidney function including eGFR, Cystatin C and magnesium were within normal ranges and comparable to placebo.
•There were no discontinuations and all eight Cohort 1 participants have transitioned to the open-label portion of the study.
Table 2: Adverse events: All TEAEs were mild to moderate
Table 3: Renal markers were within normal range and comparable to placebo
(Participant-level data)
Pharmacokinetics
Consistent with the recently received data in juvenile NHPs, the Company observed a lower-than-expected plasma Cmax and AUC (area under the curve) in pediatric DMD participants when compared
with that seen in healthy adult volunteers and the adult NHPs. The levels observed in Cohort 1 were in line with the exposures observed in juvenile NHPs, thus providing confidence on future modeling of exposure. Updated modeling, following review of the juvenile NHP data, suggest that the AUC will significantly increase in Cohort 2, resulting in higher muscle concentration, exon skipping and dystrophin production. The DMD community at large continues to learn about the biology of Duchenne and the relationship between dystrophin and functional benefit. Despite the drug plasma concentration and dystrophin levels, the Company obtained earlier-than-expected functional responses that were both statistically significant and clinically meaningful.
Efficacy and functional improvement
Mean change in TTR velocity is a robust, low variability measure which carries the largest absolute and proportional annual signal and is used as an early prognostic factor for disease progression and loss of ambulation. Cohort 1 results demonstrated a statistically significant improvement in mean TTR velocity in treated versus placebo participants (p<.05, post hoc analysis) which was 3.5 times higher than the minimal clinically important difference (MCID) threshold of 0.023, suggesting ENTR-601-44 is potentially changing the trajectory of the disease.
Positive change in TTR velocity was seen across the majority of participants, irrespective of their severity of disease or age, which likely supports that Cohort 1’s functional benefit represents a true drug-related effect.
Further, the end of Cohort 1 dystrophin levels correlated with the end of Cohort 1 TTR velocity improvement, suggesting that dystrophin production in both damaged muscle fibers and activated satellite cells may have crossed a critical threshold for functional improvement.
Importantly, a majority of participants on treatment achieved functional benefit.
•In ELEVATE-44-201 Cohort 1, a statistically significant change from baseline in TTR velocity was observed:
oMean change in TTR velocity versus placebo of 0.115.
oMean change in TTR velocity for the treatment group of 0.08.
•Demonstrated 2.36% increase in dystrophin over 4.00% baseline in treated participants.
•Demonstrated 2.31% increase in exon skipping over 2.66% baseline in treated participants.
“The topline results from Cohort 1 of the ELEVATE-44-201 study are promising. Individuals with Duchenne are in urgent need of new treatments that can provide functional improvements while also offering a more targeted treatment option,” said Dr. Laurent Servais, Professor of Paediatric Neuromuscular Diseases at the University of Oxford and principal investigator in the ELEVATE-44-201 clinical study. “For those living with Duchenne, a therapy that could lead to both near- and long-term improvements in functional outcomes would be an important breakthrough. I am excited to be a part of this clinical study and look forward to the data from Cohort 2.”
Investor Webcast and Conference Call Information
Entrada Therapeutics will host an investor webcast and conference call today, Thursday, May 7, 2026, at 8:30 a.m. ET to discuss the topline results from Cohort 1 of the Phase 1/2 ELEVATE-44-201 study. 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 (https://edge.media-server.com/mmc/p/dpiu2bfu/). The webcast will be archived and available for replay on the Entrada Therapeutics website for 90 days following the call.
Patients and Their Care Partners
Patients and their care partners are a critical part of our community, and we are committed to keeping them informed and connected. To receive community updates in real time and read today’s update, please visit www.entradatx.com/patients.
About the ELEVATE-44-201 Phase 1/2 Study
ELEVATE-44-201 is a global, two-part, randomized, double-blind, placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in ambulatory participants ages four to 20 with Duchenne who are exon 44 skipping amenable. The multiple ascending dose (MAD) Part A portion of the study is evaluating the safety, pharmacokinetics, pharmacodynamics and functional parameters following intravenous administration of ENTR-601-44 to study participants in three cohorts at sites in the U.K. and EU. The Cohort 1 MAD portion of the study enrolled eight participants ages six to 17 with Duchenne. They were randomized 3:1 to receive ENTR-601-44 at a dose of 6 mg/kg or placebo, administered intravenously. During this double-blind period, doses were administered on days one, 43 and 85, and muscle biopsies were performed at the time of screening and at six weeks after the last dose. Following the initial three doses administered in Part A, all participants continued into the Phase 2, open-label portion in which the safety and efficacy of ENTR-601-44 are evaluated over a longer period of time.
About ENTR-601-44
ENTR-601-44 is a proprietary Endosomal Escape Vehicle (EEV™)-conjugated oligonucleotide that has a sequence designed and optimized for people with a confirmed mutation in the DMD gene that is amenable to exon 44 skipping, which comprises approximately eight percent of the Duchenne patient population globally. ENTR-601-44 is designed to address the underlying cause of Duchenne, facilitating production of functional dystrophin from the endogenous (naturally occurring) DMD gene.
In December 2025, the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to ENTR-601-44.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare disease caused by mutations in the DMD gene, which encodes for the dystrophin protein. These mutations lead to inadequate dystrophin production. Dystrophin is essential to maintaining the structural integrity and function of muscle cells. Lack of functional dystrophin leads to progressive loss of muscle strength, impacting mobility and causing heart or respiratory complications that contribute to high mortality rates. An estimated 41,000 people in the U.S. and Europe are living with Duchenne. Of those, 14,000 are amenable to exon 44, 45, 50 and 51 skipping.
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.
For more information about Entrada, please visit our website, www.entradatx.com, and follow us on LinkedIn.
Forward-Looking Statements
This press release contains express and implied forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Entrada’s strategy, future operations, prospects and plans, objectives of management, the validation and differentiation of Entrada’s approach and EEV platform and its ability to provide a potential treatment for patients, the timing of data from Entrada’s Phase 1/2 MAD clinical study of ENTR-601-44, including the Cohort 1 open-label study and Cohort 2 MAD by year-end 2026 with data from Cohort 3 to follow, the ability to recruit for and complete the global Phase 2 clinical study for ENTR-601-44, the potential of TTR velocity data observed in Cohort 1 to predict clinically meaningful and potentially differentiated early functional benefits, expectations regarding the Cohort 1 open-label portion of ENTR-601-44, expectations regarding Cohort 2 of ENTR-601-44, including the potential for higher plasma concentrations with a significant increase in plasma exposure, higher muscle concentrations, exon skipping, dystrophin production and substantially higher dystrophin levels, a deepening of functional responses, the potential for further enhanced muscle function, and continued functional benefit,, expectations regarding planned Cohort 3 of Entrada’s ELEVATE-44-201 study, the potential therapeutic benefits of Entrada’s EEV product candidates, including the potential for ENTR-601-44 to be a transformative treatment option, the continued development and advancement of ENTR-601-44, ENTR-601-45, ENTR-601-50, and ENTR-601-51 for the treatment of DMD and the partnered product candidate VX-670 for the potential treatment of DM1, the ability to continue to expand and develop additional therapeutic programs and modalities, including further exon skipping programs and the potential treatment of neuromuscular and inherited retinal diseases, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Entrada may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities and the initiation and completion of preclinical studies and clinical studies; uncertainties as to the availability and timing of results from preclinical and clinical studies; the timing of and Entrada’s ability to submit and obtain regulatory clearance and initiate clinical studies; whether results from preclinical studies or clinical studies will be predictive of the results of later preclinical studies and clinical studies; whether Entrada’s cash resources will be sufficient to fund the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Entrada’s filings with the Securities and Exchange Commission (SEC), including the Company’s most recent Form 10-K and in subsequent filings Entrada may make with the SEC. In addition, the forward-looking statements included in this press release represent Entrada’s views as of the date of this press release. Entrada anticipates that subsequent events and developments will cause its views to change. However, while Entrada may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Entrada’s views as of any date subsequent to the date of this press release.
Investor and Media Contact Patient Advocacy Contact
Karla MacDonald Sarah Friedhoff
Chief Corporate Affairs Officer Head of Patient Advocacy
kmacdonald@entradatx.com patientadvocacy@entradatx.com
FAQ
How did Entrada Therapeutics (TRDA) perform financially in Q1 2026?
Entrada reported collaboration revenue of $0.9 million and a net loss of $39.7 million in Q1 2026. Research and development expenses were $33.1 million and general and administrative expenses were $10.1 million, leading to a basic and diluted net loss per share of $0.95.
What are the key results from Entrada Therapeutics’ ELEVATE-44-201 Cohort 1 study?
Cohort 1 of ELEVATE-44-201 showed favorable safety with no serious adverse events or discontinuations. Treated participants had a 2.36% dystrophin increase over 4.00% baseline and a 2.31% exon skipping increase over 2.66% baseline, alongside statistically significant improvement in Time to Rise velocity versus placebo.
What is Entrada Therapeutics’ cash position and runway as of March 31, 2026?
Entrada held $254.9 million in cash, cash equivalents and marketable securities as of March 31, 2026. Management believes this balance will be sufficient to fund operations into the third quarter of 2027, supporting ongoing Duchenne, ocular and collaboration programs.
How did Entrada’s collaboration revenue change compared with Q1 2025?
Collaboration revenue declined to $0.9 million in Q1 2026 from $20.6 million in Q1 2025. The company attributes this drop primarily to the substantial completion of collaboration research plan activities associated with VX-670 during the first quarter of 2025.
What functional benefits were observed with ENTR-601-44 in Duchenne muscular dystrophy?
ENTR-601-44 produced a statistically significant improvement in Time to Rise velocity versus placebo, more than 3.5 times the minimal clinically important difference threshold. This clinically validated functional measure suggests early, potentially meaningful functional benefit in ambulatory participants with exon 44–amenable Duchenne muscular dystrophy.
What upcoming clinical milestones has Entrada Therapeutics highlighted for 2026?
The company expects ELEVATE-45-201 Cohort 1 data in mid‑2026 and ELEVATE-44-201 Cohort 1 open‑label and Cohort 2 data by year-end 2026. Vertex also plans to share results from the MAD portion of the VX‑670 Phase 1/2 study in the second half of 2026.