Entrada Therapeutics Inc.

05/07/2026 | Press release | Distributed by Public on 05/07/2026 05:15

Quarterly Report for Quarter Ending March 31, 2026 (Form 10-Q)

MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
You should read the following discussion and analysis of our financial condition and results of operations together with the condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q ("Quarterly Report") and the audited financial information and the notes thereto included in the Company's Annual Report on Form 10-K, which was filed with the Securities and Exchange Commission (the "SEC") on February 26, 2026 ("Annual Report"). Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the "Cautionary Note Regarding Forward-Looking Statements" and "Risk Factors" section of this Quarterly Report, our actual results could differ materially from the results described in, or implied by, the forward-looking statements contained in the following discussion and analysis. You should carefully read the "Cautionary Note Regarding Forward-Looking Statements" and "Risk Factors" sections of this Quarterly Report to gain an understanding of the important factors that could cause actual results to differ materially from our forward-looking statements contained in the following discussion and analysis.
Overview
We are a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engx`age intracellular targets that have long been considered inaccessible. Through proprietary, versatile and modular approaches, we are advancing a robust development portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others. In 2026, we expect to focus on progressing our ENTR-601-44 and ENTR-601-45 clinical trials. In addition, our VX-670 partnership with Vertex Pharmaceuticals Incorporated ("Vertex") continues to progress, with Vertex on track to share results during the second half of 2026. In addition to the ENTR-601-44 Cohort 1 results reported in this Quarterly Report, we anticipate reporting additional ENTR-601-44 Cohort 1 Open Label data and topline results from the second cohort of our ENTR-601-44-201 trial as well as our ENTR-601-45 Cohort 1 data during 2026. As of March 31, 2026, we had cash, cash equivalents and marketable securities of $254.9 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of March 31, 2026 will be sufficient to fund our operations into the third quarter of 2027.
Clinical-Stage Development Pipeline:
Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy ("DMD") in the United Kingdom ("UK"), European Union ("EU") and United States ("U.S."). In 2026, we expect to have four clinical-stage programs in our DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51, which has completed IND-enabling studies but has not yet filed a regulatory application). When combined, we estimate that there are over 11,500 patients in the U.S. and Europe that carry mutations amenable to Entrada's current exon skipping programs. Complementing the ongoing clinical progress of the DMD franchise is the myotonic dystrophy type 1 ("DM1") partnership with Vertex ("VX-670"). Each of these programs utilize the same endosomal escape vehicle, and as such, we anticipate initial data readouts from any one of the candidate clinical trials to provide critical insights for the rest.
ELEVATE-44-201: The Company completed Cohort 1 of the global Phase 1/2 multiple ascending dose ("MAD") portion of the clinical study of ENTR-601-44 in ambulatory patients living with DMD who are amenable to exon 44 skipping, and those patients transitioned to the 6 dose open label portion of the trial. Cohort 1 of the study met its primary goal of favorable safety and tolerability, while also demonstrating (post hoc analysis) significant improvements in functional benefit as measured by change in Time to Rise ("TTR") velocity from baseline, versus minimal clinically important difference ("MCID"). Dosing every 6 weeks of all 8 patients is ongoing in the open-label portion of the study. In February 2026, an independent Data Monitoring Committee (DMC) reviewed the data to date from the eight patients enrolled in Cohort 1 and recommended initiation of Cohort 2 at the increased dose of 12 mg/kg without protocol modification, and Cohort 2 is continuing to enroll. Data from the Cohort 1 open label portion of the study is expected by year-end, and we expect topline results from Cohort 2 (12 mg/kg) by the end of 2026. Data from Cohort 3 (up to 18 mg/kg) is to follow if needed. Based on a combination of non-clinical data, healthy normal volunteer data and Cohort 1 patient data, we anticipate seeing an increase in dystrophin as we increase dosing amounts in the second and third cohorts. We also intend to open an expansion cohort to increase the number of participants treated in the ELEVATE-44-201 study, as this study has been designed to support an accelerated approval in the U.S. Additionally, in December 2025, the U.S. Food and Drug Administration ("FDA") granted Rare Pediatric Disease Designation to ENTR-601-44.
ELEVATE-44-102: The Company believes this clinical trial, in the underserved adult patient population, would be best to initiate at the highest advisable starting dose in patients with advanced disease. Following a review of safety, pharmacokinetic and pharmacodynamic data from Cohort 1 of the ELEVATE-44-201 study in the U.K and EU, we may have an opportunity to re-engage with the FDA to discuss increasing the planned doses in this clinical trial. As such, the Company will provide an update on clinical study design and timing following interactions with the FDA.
ELEVATE-45-201: The Company completed enrollment and initiated patient dosing in Cohort 1 of the global Phase 1/2 MAD portion of the clinical study of ENTR-601-45 in ambulatory patients 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) to follow. The proposed ELEVATE-45-201 clinical trial design and dosing regimen is similar to ELEVATE-44-201, incorporating a MAD Phase 1 portion, a 6 dose open label Phase 2 portion and an expansion cohort. We expect ENTR-601-45 to be both best in class and to be the first PMO-conjugate to generate clinically meaningful data in a population where only low single digit competitive dystrophin production has been observed to date.
ELEVATE-50-201: The Company received regulatory authorization from the UK'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 patients living with DMD who are amenable to exon 50 skipping. We expect to submit additional regulatory applications and obtain authorization in the EU for ENTR-601-50 following a review of data from the ongoing trials of our lead programs. We are evaluating a variety of options to optimize clinical study execution including pursuing a platform or basket based approach, should either pathway become available..
ENTR-601-51: The Company has completed 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. We are reviewing our global regulatory strategy of the program to determine how best to accelerate time to full, as opposed to accelerated, approval. We expect to provide more specific timeline guidance as the new strategy is finalized.
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 advanced two ocular programs into lead optimization for the potential treatment of inherited retinal diseases. Both programs are novel oligonucleotide-based therapeutics with the potential to address areas of high unmet need. In December 2025, Entrada announced its first ocular clinical candidate, ENTR-801, for the potential treatment of Usher syndrome type 2A ("USH2A"). The Company plans to announce a second clinical candidate in ocular diseases in the second half of 2026 and continues to explore novel targets to address both rare and more common retinal conditions. From a strategic point of view, progress in a new therapeutic area enables portfolio diversification in the form of tissue type, route of administration and regulatory pathway. At the same time, however, as in our neuromuscular franchise, initial targets are selected based on well-understood biology, significant unmet need, translational clarity and the potential to differentiate based on our proprietary technologies and capabilities.
ENTR-801: The Company's first ocular candidate is an optimized, proprietary oligonucleotide-based therapy for the potential treatment of a subgroup of patients with USH2A, who are amenable to exon 13 skipping. The clinical candidate was designed to restore functional usherin protein production with the goal of preserving photoreceptors (the light-sensing cells in the eye) to stabilize the overall retinal architecture and preserve function. ENTR-801 was selected from a library of 200 sequences based on its robust exon skipping and usherin protein production, as well as initial safety data in multiple animal models.
We continue to believe that the robust supporting data and ongoing progress of our growing portfolio of clinical and preclinical candidates has the potential to make a significant difference in the lives of patients.
ENTR-601-44-201 (ELEVATE-44-201) Phase 1/2 Cohort 1 Results
Clinical Trial Overview
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 twenty with DMD who
are exon 44 skipping amenable. The multiple ascending dose 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 MAD portion of the study enrolled eight participants with DMD in Cohort 1. 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.
Summary of Demographics and Baseline Patient Characteristics
The average age of treated participants in Cohort 1 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 trials. This is notable as treatment response is generally considered to correlate with higher baseline dystrophin levels.
Safety and Tolerability Data
Cohort 1 achieved its primary endpoint, demonstrating a generally favorable safety and tolerability profile. All treatment emergent adverse events (TEAEs) were mild to moderate. There were no discontinuations and no serious adverse events reported. The most common adverse event (AE) seen was headache.
Importantly, clinically relevant markers of kidney function including eGFR, Cystatin-C and magnesium were all within normal ranges and treated subjects were comparable to placebo. Participant level data is presented below.
We believe these results to be encouraging, given that the kidney has historically been the organ of toxicity concern for PMO-based oligonucleotides and peptide conjugates in particular, and because similarly favorable safety was observed at 6 mg/kg in the Phase 1 healthy normal volunteer trial disclosed in 2024.
Biodistribution and Biomarkers
Lower than expected plasma Cmax and AUC (area under the curve) was observed in the ENTR-601-44-201 pediatric DMD patient study when compared with our prior ENTR-601-44-101 Phase 1 healthy adult volunteer study. Our current understanding is that this difference is due to a combination of age and disease status, and that higher doses of drug may result in higher levels of drug concentration in circulation. This lower than expected drug concentration, along with lower than expected baseline dystrophin value and a high number of patients with multi-exon deletions or more complex arrangements other than exon 45 deletion mutation may have impacted initial biomarker responses. There was a demonstrated mean increase of 2.36% in dystrophin over a baseline of 4.00% (total mean 6.36% dystrophin) and a demonstrated mean increase of 2.31% in exon skipping over baseline of 2.66% (total mean of 4.97%) in treated patients. Importantly, however, even at this first dose with lower than expected exposures, there was a correlation observed between exon skipping, dystrophin production and functional improvement.
Functional Improvement
Meaningful and potentially differentiated early functional benefit was observed in Cohort 1. Results from these initial patients demonstrated a statistically significant improvement in mean TTR velocity in treated versus placebo patients (p<.05, post hoc analysis). Functional data is the ultimate goal for a DMD therapeutic, and we are pleased to have observed this benefit at its starting dose of 6 mg/kg.
"Time To Rise" (referred to as TTSTAND, TTR, and TRF in the literature) is standardized procedure and double-blind evaluative process conducted under GCP conditions and is expressed as the number of seconds taken to rise from a supine position without assistance to standing upright. The clinical evaluators (CEs) are physiotherapists with experience evaluating neuromuscular patients, trained as specified in the protocol, and as detailed in the Evaluator Manual. All CE assessments are videotaped and videos are reviewed by a Master Physiotherapist to ensure that the tests are performed correctly and to remove inter site evaluator variability. TTR declines rapidly over time in patients with DMD and has been previously shown to be an early prognostic factor for disease progression and loss of ambulation.
TTR velocity is calculated as 1/TTR, expressed as rises/second. The measure is designed to reduce the impact of outliers and imputed data. The calculation handles the "unable to perform problem" if a patient cannot rise from the floor unassisted by scoring that observation at zero avoiding arbitrary imputation. It dampens clinically meaningless scoring noise between visits and it compresses the long tail seen in readings and produces a distribution that's much closer to normal, which matters for parametric statistics.
TTR velocity is increasingly preferred as an approvable clinical endpoint in Phase 3 trials over alternatives, given its sensitivity, lower variability and limited outlier bias. There are several completed or ongoing Phase 3 clinical trials in DMD using this as the primary endpoint or as one of several primary endpoints. Currently, steroids are standard of care in DMD, because of their proven ability to impact function. As a reference, vamorolone (Santhera Therapeutics) is a corticosteroid approved in 2023 and indicated for the treatment of DMD in patients 2 years of age and older. In the vamorolone registrational trial, the primary endpoint was the change from baseline to Week 24 in TTR velocity at 6 mg/kg/day compared to placebo. In that study, the mean change from baseline was reported as 0.048 (rises/second), and this remains the highest number published to date in a registrational trial.
Functional Improvement Results
In ELEVATE-44-201 Cohort 1, a statistically significant change from baseline in TTR velocity was observed:
•Mean change in TTR velocity versus placebo of 0.115
•Mean change in TTR velocity in the treatment group of 0.08
The mean change in TTR velocity in the study was 3.5 times higher than the MCID threshold (post hoc analysis) of 0.023 (literature derived), suggesting that ENTR-601-44 has the potential to change the trajectory of the disease at the lowest dose tested in the Phase 1/2 study.
Importantly, the change in TTR velocity was consistent and seen across the majority of patients, irrespective of their severity of disease. In addition, there was no observed correlation between age and TTR velocity, which suggests that Cohort 1's functional benefit represents a true drug related effect. Finally, the end of Cohort 1 dystrophin levels correlated with the end of Cohort 1 TTR velocity improvement suggesting that dystrophin production may have crossed a critical threshold for functional improvement. Positive trends were seen in the treated patients' 10-meter walk/run assessments.
Discussion of Functional Improvement Results
An ideal treatment for DMD is regenerative - replacing damaged, dystrophic muscle with healthy muscle; This requires satellite cell correction which then promotes asymmetric differentiation.
The DMD challenge: Damage and impaired repair
•A lack of dystrophin not only leaves mature tissue prone to damage but also inhibits the body's repair mechanism; This "double hit" results in an inevitable decline in function as damaged tissues fail and are replaced with fat and fibrosis
•Satellite (stem) cells are responsible for muscle regeneration and a lack of dystrophin inhibits the proliferation and differentiation of these progenitor cells which in turn limits muscle repair
The significant unmet need in DMD: Muscle repair
•Competitive treatments target mature, damaged cells to slow decline but may not significantly or substantially promote repair via enhanced satellite cell proliferation and asymmetric division
•Biomarkers (Dystrophin, CK) will improve as damaged tissue is protected, but functional benefit is likely to come back slowly as the repair mechanism is still impaired
•Transferrin receptors are not expressed on quiescent satellite cells, preventing transferrin receptor mediated antibody uptake
•AAV-enabled gene therapies lack the ability to efficiently reach satellite cells, which limits response durability
We believe ENTR-601-44's ability to access the satellite cells is emerging as a competitive differentiator.
Upregulated dystrophin may be enabling the asymmetric division of satellite cells, which could in turn be responsible for the functional data signals seen at the starting dose of 6 mg/kg. DMD progression is driven by both muscle breakdown and impaired regeneration. It has been shown that satellite cells lacking dystrophin have an impaired ability to effectively proliferate, asymmetrically divide and differentiate when compared to healthy stem cells. We believe that enhanced satellite cell activity could result in the regeneration of healthy fibers and the stabilization of the overall muscle, which in turn would be expected to manifest in greater strength as measured by TTR velocity. Therefore we believe that dystrophin levels may have increased enough to cross a threshold and impact satellite cell proliferation and differentiation - enhancing the body's muscle repair mechanisms. If correct, at low drug candidate doses, as damaged muscle is replaced by healthy muscle tissue, the overall stability and strength of the muscle is expected to improve, potentially preceding traditional biomarkers such as creatine kinase (CK) that would be more sensitive to the stabilization of damaged muscle cells specifically. Over time, and at higher doses, those biomarkers would potentially then improve as damaged tissue is replaced by new tissue and as remaining damaged tissue is stabilized. The results observed from this initial dose level of ENTR-601-44, with room to go up to 18 mg/kg in later cohorts, may explain why improved TTR velocity was observed at the dystrophin levels reported in Cohort 1.
Clinical Trial Progress
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 placebo or three doses of 12 mg/kg of ENTR-601-44. We expect topline results from Cohort 2 by the end of 2026.
Components of Our Results of Operations
Revenue
Substantially all of our revenue to date has been derived from the Vertex Agreement. We do not expect to generate any revenue from the sale of products unless and until such time that our product candidates have advanced through clinical development and regulatory approval, if ever. If our development efforts for our therapeutic candidates are successful and result in regulatory approval or we successfully enter into collaboration or license arrangements with third parties, we may generate revenue in the future from product sales, payments from collaboration or license arrangements including those that we may enter into with third parties, or any combination thereof.
Operating Expenses
Research and Development Expenses
Research and development expenses consist primarily of costs incurred for our research activities, including our discovery efforts, and the development of our programs. These expenses include:
•personnel-related expenses, including salaries, related benefits, and stock-based compensation expense for individuals engaged in research and development functions;
•expenses incurred in connection with our research programs and development of our therapeutic candidates, including those incurred under agreements with third parties, such as consultants, contractors, and contract research organizations ("CROs") to conduct preclinical studies and clinical trials;
•the cost of developing and validating our manufacturing process for use in our preclinical studies and clinical trials, including the cost of raw materials used in our research and development activities, and engaging with third party contract manufacturing organizations ("CMOs");
•costs incurred in connection with the performance of research and development activities under the Vertex Agreement;
•the cost of laboratory supplies and research materials;
•the costs of payments made under third-party licensing agreements and related future payments should certain development and regulatory milestones be achieved; and
•facilities, depreciation and other direct and allocated expenses, including rent and other operating costs, incurred as a result of our research and development activities.
We expense research and development costs as incurred. Non-refundable advance payments that we make for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered. Upfront payments under license agreements are expensed upon receipt of the license and annual maintenance fees under license agreements are expensed in the period in which they are incurred. Milestone payments under license agreements are accrued, with a corresponding expense being recognized, in the period in which the milestone is determined to be probable of achievement and the related amount is reasonably estimable.
Our research and development costs are primarily devoted to supporting our neuromuscular program development efforts. Our direct, external research and development expenses consist primarily of fees paid to outside consultants, CROs, CMOs and research laboratories in connection with our process development, manufacturing and clinical development activities. Our direct external research and development expenses also include fees incurred under license and intellectual property purchase agreements. Where specifically identifiable, we expect to track these external research and development costs on a program-by-program basis as we identify product candidates to advance into clinical development.
We do not allocate employee costs, costs associated with our development efforts and facilities, including depreciation or other indirect costs, to specific programs because these costs are deployed across multiple programs and, as such, are not separately classified. We use internal resources and third-party consultants primarily to conduct our research and development activities as well as for managing our process development, manufacturing and clinical development activities.
Therapeutic candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our research and development expenses will increase substantially in connection with our platform development efforts and planned preclinical and planned and current clinical development activities in the near term and in the future. We further expect that the research and development expenses of our programs will increase in the near term as we initiate or continue CTA/IND-enabling activities for our therapeutic candidates. Therefore, we cannot reasonably estimate or know the nature, timing, and costs of the efforts that will be necessary to complete the preclinical and clinical development of any of our therapeutic candidates. The successful development of our therapeutic candidates is highly uncertain. This is due to the numerous risks and uncertainties associated with product development, including the following:
•the scope, timing, rate of progress and expenses of our ongoing and potential future research activities, including preclinical and CTA/IND-enabling studies, clinical trials and other research and development activities we decide to pursue;
•the successful initiation, enrollment and completion of clinical trials under current good clinical practices ("GCPs");
•the timing of filing and acceptance of INDs or comparable foreign applications that allow commencement of future clinical trials for our therapeutic candidates;
•whether our therapeutic candidates show safety and efficacy in our clinical trials and an acceptable risk-benefit profile in the intended populations;
•our ability to hire and retain key research and development personnel to meet our strategic goals;
•our ability to successfully develop, obtain regulatory and marketing approvals of our therapeutic candidates for the expected indications and patient populations;
•our ability to establish and maintain agreements with third-party manufacturers for clinical supply for our clinical trials and commercial manufacturing, if our therapeutic candidates are approved;
•commercializing therapeutic candidates, if and when approved, whether alone or in collaboration with others;
•our ability to maintain a continued acceptable safety, tolerability and efficacy profile of our therapeutic candidates following approval;
•our ability to establish new licensing or collaboration arrangements to support our potential therapeutic candidates on favorable business terms;
•any decisions we make to discontinue, delay or modify our programs to focus on others;
•obtaining, maintaining, protecting and enforcing patent and trade secret protection and regulatory exclusivity for our therapeutic candidates; and
•obtaining and maintaining adequate coverage and reimbursement from third party payors.
A change in the outcome of any of these variables with respect to the development of any of our therapeutic candidates could significantly change the costs and timing associated with the development of that therapeutic candidate. We may never succeed in obtaining regulatory approval for any of our therapeutic candidates.
General and Administrative Expenses
General and administrative expenses consist primarily of salaries and personnel-related costs, including stock-based compensation, for our personnel in executive, legal, finance and accounting, corporate and business development, human resources and other administrative functions. General and administrative expenses also include: legal fees relating to intellectual property and corporate matters; professional fees paid for accounting, auditing, consulting and tax services; insurance costs; travel expenses; information technology expenses; and facility costs not otherwise included in research and development expenses.
We anticipate that our general and administrative expenses will increase in the future as we increase our headcount and expand our facilities to support our continued research activities and development of our programs and our EEV platform ("EEV Platform").
Interest and Other Income (Expense)
Interest and other income (expense) consists primarily of interest earned on our invested cash equivalents and marketable securities, gains and losses on disposal of fixed assets and gains and losses on foreign currency transactions.
Income Taxes
Provision for income tax expense (benefit) recorded in any interim period is based on the estimated effective tax rate for the fiscal year for those tax jurisdictions that can be reliably estimated. There were no significant income tax provisions or benefits for the three months ended March 31, 2026 and 2025.
Critical Accounting Policies and Significant Judgments and Estimates
Our management's discussion and analysis of financial condition and results of operations is based on our financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of our financial statements and related disclosures requires us to make judgments, estimates and assumptions that affect the reported amounts of assets and liabilities, costs and expenses and the disclosure of contingent
assets and liabilities in our financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions or conditions.
There have been no material changes to our critical accounting policies and estimates from those reported in the Annual Report, except as described further in Note 2 Summary of Significant Accounting Policies in the condensed consolidated financial statements elsewhere in this Quarterly Report.
Results of Operations
Comparison of the three months ended March 31, 2026 and 2025
Three Months Ended March 31,
(in thousands) 2026 2025 Change
Collaboration revenue $ 875 $ 20,558 $ (19,683)
Operating expenses:
Research and development 33,054 32,074 980
General and administrative 10,124 10,274 (150)
Total operating expenses 43,178 42,348 830
Loss from operations (42,303) (21,790) (20,513)
Other income:
Interest and other income 2,624 4,441 (1,817)
Total other income 2,624 4,441 (1,817)
Loss before provision for income taxes (39,679) (17,349) (22,330)
Provision for income taxes 38 - 38
Net loss $ (39,717) $ (17,349) $ (22,368)
Collaboration Revenue
Collaboration revenue was $0.9 million for the three months ended March 31, 2026 and $20.6 million for the three months ended March 31, 2025. The decrease of $19.7 million was primarily a result of Entrada substantially completing our research plan activities for VX-670 during the first quarter of 2025.
Research and Development Expenses
Three Months Ended March 31,
(in thousands) 2026 2025 Change
Direct research and development expenses
ENTR-601-44 $ 4,755 $ 4,734 $ 21
ENTR-601-45 3,602 2,859 743
ENTR-601-50 1,369 692 677
ENTR-601-51 2,337 826 1,511
DMD franchise-wide(1)
1,739 - 1,739
Collaboration services 234 876 (642)
Ocular programs
461 270 191
Other preclinical and discovery 599 987 (388)
Unallocated research and development expenses
Personnel related (including stock-based compensation) 12,041 12,756 (715)
Facility related and other 5,917 8,074 (2,157)
Total research and development expenses $ 33,054 $ 32,074 $ 980
(1)Represents manufacturing and clinical costs that support across the Company's product candidates targeting DMD.
Research and development expenses were $33.1 million for the three months ended March 31, 2026, compared to $32.1 million for the three months ended March 31, 2025. The increase of $1.0 million in research and development expenses was primarily attributable to:
•an increase of $3.9 million in direct research and development expenses, driven by additional costs incurred related to the progress of our Duchenne programs, partially offset by fewer costs incurred related to our collaboration with Vertex; and
•a decrease of $2.9 million in unallocated research and development expenses driven by a decrease in personnel costs of $0.7 million and a decrease in facilities related costs of $2.2 million. Personnel costs are inclusive of stock-based compensation expense of $2.2 million and $2.4 million for the three months ended March 31, 2026 and 2025, respectively.
We expect that our research and development expenses will increase as we continue to advance ENTR-601-44 and ENTR-601-45 through clinical trials, ENTR-601-50 into clinical trials, ENTR-601-51 and our ocular programs through preclinical development and into clinical trials, and continue to perform discovery work for future product candidates.
General and Administrative Expenses
General and administrative expenses for the three months ended March 31, 2026 were $10.1 million, compared to $10.3 million for the three months ended March 31, 2025. The decrease of $0.2 million was primarily attributable to a decrease in professional services.
Interest and Other Income, net
Total interest and other income, net was $2.6 million for the three months ended March 31, 2026, compared to $4.4 million of interest and other income for the three months ended March 31, 2025. The decrease was driven by changes in interest earned from debt securities and money market funds as well as a decrease in the amount of marketable securities held.
Liquidity and Capital Resources
Overview
Since our inception, we have devoted substantially all our resources to research and development efforts relating to our EEV Platform, advancing development of our portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases and administrative support for these operations, including raising capital.
Since inception, we have incurred significant net losses. As of March 31, 2026, we had an accumulated deficit of $312.8 million. Other than the recognition of revenue related to collaboration payments, we expect to continue to generate operating losses and negative operating cash flows for the foreseeable future as we advance our platform and therapeutic candidates. We will not generate any revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for one or more therapeutic candidates, if ever. If we obtain regulatory approval for any therapeutic candidates, we expect to incur significant expenses related to developing our internal commercialization capability to support product sales, marketing and distribution.
Furthermore, we expect to incur additional costs associated with operating as a public company, including significant legal, accounting, investor relations and other expenses. As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy, as we advance therapeutic candidates through preclinical and, if successful, into clinical development, seek regulatory approval, prepare for and, if any therapeutic candidates are approved, proceed to commercialization and operate as a public company. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions.
If we are unable to obtain funding, we will be forced to delay, reduce, or eliminate some or all of our research and development programs, product portfolio expansion and ultimate commercialization efforts, which would adversely affect our business prospects, or we may be unable to continue operations. Although we continue to pursue these plans, we may not be successful in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.
Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased expenses or when or if we will be able to achieve or maintain profitability. Even if we can generate product sales, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.
As of March 31, 2026, we had cash, cash equivalents and marketable securities of $254.9 million. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of March 31, 2026 will be sufficient to fund our operations into the third quarter of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. To finance our operations beyond that point we will need to raise additional capital, which cannot be assured.
Sources of Liquidity
To date, we have raised over $850.0 million of gross proceeds from sales of stock to leading biotechnology investors and from the Vertex Agreement. As of March 31, 2026, we had cash, cash equivalents and marketable securities of $254.9 million.
In November 2025, we entered into a sales agreement (the "Sales Agreement"), with TD Securities (USA) LLC (f/k/a Cowen and Company, LLC), acting as our agent and/or principal (the "Sales Agent"), with respect to an "at the market offering" program under which we may, from time to time, at our sole discretion, issue and sell shares of our common stock having an aggregate offering price of up to $150.0 million through the Sales Agent. During the three months ended March 31, 2026, there have been no sales of common stock pursuant to the Sales Agreement.
Cash Flows
The following table summarizes our cash flows for each of the periods presented:
Three Months Ended March 31,
(in thousands) 2026 2025
Net cash used in operating activities $ (41,695) $ (38,507)
Net cash provided by investing activities 26,987 4,783
Net cash provided by financing activities 100 350
Net decrease in cash, cash equivalents and restricted cash $ (14,608) $ (33,374)
Operating Activities
For the three months ended March 31, 2026, net cash used in operating activities was $41.7 million and was driven by our net loss of $39.7 million, net cash used in changes in our operating assets and liabilities of $7.0 million, adjustments for non-cash expenses relating to stock-based compensation expense of $4.7 million and depreciation expense of $0.8 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $0.5 million.
For the three months ended March 31, 2025, net cash used in operating activities was $38.5 million and was driven by our net loss of $17.3 million, net cash used in changes in our operating assets and liabilities of $25.9 million, adjustments for non-cash expenses relating to stock-based compensation expense of $5.1 million and depreciation expense of $0.9 million, and adjustments for non-cash income relating to accretion of premiums and discounts of $1.3 million.
Investing Activities
Net cash provided by investing activities was $27.0 million for the three months ended March 31, 2026, consisting primarily of $38.9 million from the maturities of marketable securities, partially offset by $11.8 million in purchases of marketable securities and $0.1 million of purchases of property and equipment.
Net cash provided by investing activities was $4.8 million for the three months ended March 31, 2025, consisting primarily of $85.9 million from the maturities of marketable securities, partially offset by $80.0 million in purchases of marketable securities and $1.1 million of purchases of property and equipment.
Financing Activities
Net cash provided by financing activities was $0.1 million for the three months ended March 31, 2026, consisting of $0.1 million in proceeds from stock option exercises.
Net cash provided by financing activities was $0.4 million for the three months ended March 31, 2025, consisting of $0.4 million in proceeds from stock option exercises.
Future Funding Requirements
We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and, if successful, the clinical development of our programs. Our operating expenses and future funding requirements are expected to increase substantially as we continue to advance our portfolio of programs. Based on our current operating plans, we believe that our cash, cash equivalents and marketable securities as of March 31, 2026 will be sufficient to fund our operations into the third quarter of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect.
Because of the numerous risks and uncertainties associated with research, development, and commercialization of our candidates, we are unable to estimate the exact amount of our working capital requirements. Our future capital requirements will depend on many factors, including costs associated with:
•the continuation of our current research programs and our clinical and preclinical development of therapeutic candidates from our current research programs;
•advancing our existing and future therapeutic candidates into clinical development;
•initiating preclinical studies and clinical trials for any therapeutic candidates we identify and develop or expand development of existing programs into additional indications;
•maintaining, expanding, enforcing, defending and protecting our intellectual property portfolio and providing reimbursement of third-party expenses related to our patent portfolio;
•timing of manufacturing for our therapeutic candidates and commercial manufacturing if any therapeutic candidate is approved;
•establishing and maintaining clinical and commercial supply for the development and manufacture of our therapeutic candidates;
•seeking regulatory and marketing approvals for any of our therapeutic candidates that we develop, if any;
•seeking to identify, establish and maintain additional collaborations and license agreements, and the success of those collaborations and license agreements;
•ultimately establishing a sales, marketing and distribution infrastructure to commercialize any platforms for which we may obtain marketing approval, either by ourselves or in collaboration with others;
•generating revenue from commercial sales of therapeutic candidates we may develop for which we receive marketing approval;
•hiring additional personnel, including research and development, clinical and commercial personnel, to meet our strategic goals;
•adding operational, financial and management information systems and personnel, including personnel to support our product development;
•achieving sufficient market acceptance, coverage and adequate reimbursement from third-party payors and adequate market share and revenue for any approved products;
•acquiring or in-licensing products, intellectual property, and technologies; and
•the ongoing costs of operating as a public company.
Until such time, if ever, as we can generate substantial product revenue to support our cost structure, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, and other similar arrangements. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. If we raise funds through collaborations or other similar arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or therapeutic candidates or grant licenses on terms that may not be favorable to us and/or may reduce the value of our common stock. If we are unable to raise additional funds when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market our therapeutic candidates even if we would otherwise prefer to develop and market such therapeutic candidates ourselves.
Contractual Obligations and Commitments
Lease Commitments
During the three months ended March 31, 2026, there were no material changes to our lease commitments from those described in Note 11, Leases, of our financial statements in the Annual Report.
Purchase and Other Obligations
We enter into contracts in the normal course of business with CROs, third-party manufacturers, and other third parties for preclinical research studies and testing and manufacturing services. These contracts do not contain minimum purchase commitments and are cancellable by us upon prior written notice. Payments due upon cancellation consist only of
payments for services provided or expenses incurred, including non-cancelable obligations of our service providers, up to the date of cancellation.
We have also entered into license agreements under which we are obligated to make certain payments. During the three months ended March 31, 2026, there were no material changes to our commitments and contingencies related to our license agreements from those described in "Business-Intellectual property- License agreement with The Ohio State University" and Note 11, Commitments and Contingencies, to our financial statements in the Annual Report. For additional information regarding our license agreements, refer to Note 11, Commitments and Contingencies, to our condensed consolidated financial statements in this Quarterly Report.
Emerging Growth Company and Smaller Reporting Company Status
We are an "emerging growth company" ("EGC") under the Jumpstart Our Business Startups Act of 2012 (the "JOBS Act"). Section 107 of the JOBS Act provides that an EGC can take advantage of the extended transition period provided in Section 7(a)(2)(B) of the Securities Act of 1933, as amended (the "Securities Act"), for complying with new or revised accounting standards. Thus, an EGC can delay the adoption of certain accounting standards until those standards would otherwise apply to private companies. We have elected to avail ourselves of delayed adoption of new or revised accounting standards and, therefore, we will be subject to the same requirements to adopt new or revised accounting standards as private entities.
As an EGC, we may, and intend to, take advantage of certain exemptions and reduced reporting requirements under the JOBS Act. Subject to certain conditions, as an EGC:
•we may avail ourselves of the exemption from providing an auditor's attestation report on our system of internal controls over financial reporting pursuant to Section 404(b) of the Sarbanes-Oxley Act of 2002, as amended (the "Sarbanes-Oxley Act");
•we may avail ourselves of the exemption from complying with any requirement that may be adopted by the Public Company Accounting Oversight Board ("PCAOB") regarding mandatory audit firm rotation or a supplement to the auditor's report providing additional information about the audit and the financial statements, known as the auditor discussion and analysis;
•we may provide reduced disclosure about our executive compensation arrangements; and
•we may not require nonbinding advisory votes on executive compensation or stockholder approval of any golden parachute payments.
We will remain an EGC until the earliest to occur of (i) the last day of the fiscal year following the fifth anniversary of the completion of our initial public offering ("IPO"), (ii) the last day of the fiscal year in which we have total annual gross revenues of $1.235 billion or more, (iii) the date on which we have issued more than $1.0 billion in non-convertible debt during the previous rolling three-year period or (iv) the date on which we are deemed to be a large accelerated filer under the Securities Exchange Act of 1934, as amended (the "Exchange Act").
We are also a "smaller reporting company" because the market value of our stock held by non-affiliates was less than $250 million as of June 30, 2025. We may continue to be a smaller reporting company if either (i) the market value of our stock held by non-affiliates is less than $250 million or (ii) our annual revenue was less than $100 million during the most recently completed fiscal year and the market value of our stock held by non-affiliates is less than $700 million. If we are a smaller reporting company at the time we cease to be an emerging growth company, we may continue to rely on exemptions from certain disclosure requirements that are available to smaller reporting companies. Specifically, as a smaller reporting company we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report and, similar to emerging growth companies, smaller reporting companies have reduced disclosure obligations regarding executive compensation.
Recently Issued Accounting Pronouncements
See Note 2, Summary of Significant Accounting Policies to our condensed consolidated financial statements included elsewhere in this Quarterly Report.
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