# EDGAR Filing Document

**Accession Number:** 0001689375
**File Stem:** 0001689375-26-000027
**Filing Date:** 2026-5
**Character Count:** 43876
**Document Hash:** c6a9513711e7047209355fc80402225c
**Contains OCR:** False
**Source Format:** 

## Filing Content

## Filing Summary
**0001689375-26-000027.hdr.sgml**: 20260507

**ACCESSION NUMBER**: 0001689375-26-000027

**CONFORMED SUBMISSION TYPE**: 8-K

**PUBLIC DOCUMENT COUNT**: 18

**CONFORMED PERIOD OF REPORT**: 20260507

**ITEM INFORMATION**: Results of Operations and Financial Condition

**ITEM INFORMATION**: Regulation FD Disclosure

**ITEM INFORMATION**: Financial Statements and Exhibits

**FILED AS OF DATE**: 20260507

**DATE AS OF CHANGE**: 20260507

**FILER**: 

**COMPANY DATA:**
- **COMPANY CONFORMED NAME:** Entrada Therapeutics, Inc.
- **CENTRAL INDEX KEY:** 0001689375
- **STANDARD INDUSTRIAL CLASSIFICATION:** PHARMACEUTICAL PREPARATIONS [2834]
- **ORGANIZATION NAME:** 03 Life Sciences
- **EIN:** 813983399
- **STATE OF INCORPORATION:** DE
- **FISCAL YEAR END:** 1231

**FILING VALUES:**
- **FORM TYPE:** 8-K
- **SEC ACT:** 1934 Act
- **SEC FILE NUMBER:** 001-40969
- **FILM NUMBER:** 26950835

**BUSINESS ADDRESS:**
- **STREET 1:** ONE DESIGN CENTER PLACE
- **STREET 2:** SUITE 17-500
- **CITY:** BOSTON
- **STATE:** MA
- **ZIP:** 02210
- **BUSINESS PHONE:** 857-305-1825

**MAIL ADDRESS:**
- **STREET 1:** ONE DESIGN CENTER PLACE
- **STREET 2:** SUITE 17-500
- **CITY:** BOSTON
- **STATE:** MA
- **ZIP:** 02210

**FORMER COMPANY:**
- **FORMER CONFORMED NAME:** CycloPorters, Inc.
- **DATE OF NAME CHANGE:** 20161104

?xml version='1.0' encoding='ASCII'? trda-20260507

**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**

**ENTRADA THERAPEUTICS, INC.**

**(Exact name of registrant as specified in its charter)**

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| | | |
|:---|:---|:---|
| **Delaware** | **001-40969** | **81-3983399** |
| **(State or other jurisdiction<br>of incorporation)** | **(Commission<br>File Number)** | **(I.R.S. Employer<br>Identification No.)** |

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| | |
|:---|:---|
| **One Design Center Place** | |
| **Suite 17-500** | |
| **Boston, MA** | **02210** |
| (Address of principal executive offices) | (Zip Code) |

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**Registrant's telephone number, including area code: (857) 520-9158**

**Not Applicable**

**(Former name or former address, if changed since last report)**

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

□ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

□ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

□ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

□ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

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Securities registered pursuant to Section 12(b) of the Act:

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| | | |
|:---|:---|:---|
| **Title of each class** | **Trading<br>Symbol(s)** | **Name of each exchange<br>on which registered** |
| **Common Stock, $0.0001 par value per share** | **TRDA** | **The Nasdaq Global Market** |

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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 ⌧

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. □

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**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&nbsp;&nbsp;&nbsp;&nbsp;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:

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| | |
|:---|:---|
| 99.1 | <u>[Press Release issued by Entrada Therapeutics, Inc. on](entradatherapeuticsreports.htm)[May 7](entradatherapeuticsreports.htm)[, 2026](entradatherapeuticsreports.htm)</u> |
| 99.2 | <u>[Press Release issued by Entrada Therapeutics, Inc. on May 7, 2026](entradatherapeuticscohort1.htm)</u> |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). |

---

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**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.

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| | | |
|:---|:---|:---|
| | Entrada Therapeutics, Inc. | Entrada Therapeutics, Inc. |
| Date: May 7, 2026 | Date: May 7, 2026 | /s/ Dipal Doshi |
| | | Dipal Doshi |
| | | Chief Executive Officer |

---

## Exhibit 99.1

![image_0a.jpg](image_0a.jpg)

**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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• **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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• **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

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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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• **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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• **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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• **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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• **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. <br>

***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**

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• H.C. Wainwright 4<sup>th</sup> Annual BioConnect Investor Conference, New York, NY on May 19, 2026

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• 2026 Jefferies Global Healthcare Conference, New York, NY on June 3, 2026

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Goldman Sachs 47<sup>th</sup> 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.

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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**<br>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**

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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

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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.

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**ENTRADA THERAPEUTICS, INC.**

**Condensed Consolidated Statements of Operations (Unaudited)**

**(In thousands, except share and per share amounts)**

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| | | |
|:---|:---|:---|
| | **Three Months Ended March 31,** | **Three Months Ended March 31,** |
| | **2026** | **2025** |
| Collaboration revenue | $875 | $20558 |
| Operating expenses: |  |  |
| &nbsp;&nbsp;&nbsp;Research and development | 33054 | 32074 |
| &nbsp;&nbsp;&nbsp;General and administrative | 10124 | 10274 |
| &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;Total operating expenses | 43178 | 42348 |
| Loss from operations | (42303) | (21790) |
| Other income: |  |  |
| &nbsp;&nbsp;&nbsp;Interest and other income | 2624 | 4441 |
| &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;Total other income | 2624 | 4441 |
| Loss before provision for income taxes | (39679) | (17349) |
| &nbsp;&nbsp;&nbsp;Provision for income taxes | 38 |  |
| Net loss | $(39717) | $(17349) |
| Net loss per share, basic and diluted | $(0.95) | $(0.42) |
| Weighted-average common shares outstanding, basic and diluted | 41836275 | 41073732 |

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**ENTRADA THERAPEUTICS, INC.**

**Condensed Consolidated Balance Sheet Data (Unaudited)**

**(In thousands)**

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| | | |
|:---|:---|:---|
| | **March 31,** | **December 31,** |
| | **2026** | **2025** |
| Cash, cash equivalents and marketable securities | $254859 | $295698 |
| Total assets | $335518 | $377378 |
| Total liabilities | $64664 | $71245 |
| Total stockholders' equity | $270854 | $306133 |

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**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

## Exhibit 99.2

![image_3a.jpg](image_3a.jpg)

**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.

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![image_3a.jpg](image_3a.jpg)

***Table 1: Demographics and baseline characteristics***

![a5726datapr-image1a.jpg](a5726datapr-image1a.jpg)

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

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![image_3a.jpg](image_3a.jpg)

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** 

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Favorable safety and tolerability with ENTR-601-44 at the 6 mg/kg dose.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• All treatment emergent adverse events (TEAEs) were mild to moderate.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• No reported SAEs and no AEs leading to discontinuation from the study.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• The most common AE was headache.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Markers of kidney function including eGFR, Cystatin C and magnesium were within normal ranges and comparable to placebo.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• 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***

![a5726datapr-image2a.jpg](a5726datapr-image2a.jpg)

***Table 3: Renal markers were within normal range and comparable to placebo***

***(Participant-level data)***

![a5726datapr-image3a.jpg](a5726datapr-image3a.jpg)

**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

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![image_3a.jpg](image_3a.jpg)

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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• 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.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Demonstrated 2.36% increase in dystrophin over 4.00% baseline in treated participants.

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• 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.

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**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.

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**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.

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**Investor and Media Contact&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;Patient Advocacy Contact**

Karla MacDonald &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;Sarah Friedhoff

Chief Corporate Affairs Officer &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;Head of Patient Advocacy

kmacdonald@entradatx.com &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;patientadvocacy@entradatx.com

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