enGene (Nasdaq: ENGN) updates pivotal LEGEND bladder cancer trial with mixed durability
Rhea-AI Filing Summary
enGene Therapeutics Inc. reported updated interim results from the pivotal Cohort 1 of its Phase 2 LEGEND trial of detalimogene voraplasmid in high-risk, BCG-unresponsive NMIBC, and amended the employment agreement of Dr. Hussein Sweiti. Among 125 patients, detalimogene achieved a 54% complete response rate at any time and 43% at six months, with a low 3.2% progression rate to muscle-invasive or more advanced disease. Treatment-related adverse events occurred in 55% of patients, were mainly Grade 1–2, and led to dose interruptions or discontinuations in 2.4% of patients each. A Kaplan-Meier estimate showed a 12‑month complete response rate of 25%, and later-enrolling patients had lower response rates than earlier subgroups, which the company is further analyzing.
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Insights
Interim data show solid initial responses and safety, but durability and later-cohort performance are weaker and under review.
enGene released interim data for 125 patients in the pivotal LEGEND Phase 2 cohort. Detalimogene delivered a 54% complete response rate at any time and 43% at six months, with a low 3.2% progression rate to muscle-invasive or more advanced disease. Safety appears manageable, with 55% of patients experiencing treatment-related adverse events, mostly mild.
Durability is more modest: landmark complete response rates were 32.7% at nine months and 13.3% at 12 months, with a Kaplan–Meier 12‑month estimate of 25%. Newly assessed patients since the prior October 2025 analysis showed lower responses (any-time complete response 39%, six‑month 32%), and the company notes that subgroup analyses so far have not found clear baseline differences.
Regulatory positioning remains important. Detalimogene has RMAT and Fast Track designations and is in the FDA’s CDRP program, with required manufacturing validation batches completed and a Statistical Analysis Plan submitted. The company plans further FDA engagement toward a potential BLA and expects to provide an update in the second half of 2026, while also presenting data at the American Urological Association meeting on May 15, 2026.
8-K Event Classification
Key Figures
Key Terms
complete response (CR) medical
Regenerative Medicine Advanced Therapy (RMAT) designation regulatory
Biologics License Application (BLA) regulatory
non-muscle invasive bladder cancer (NMIBC) medical
Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) regulatory
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Item 5.02 Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers.
On May 5, 2026, enGene USA, Inc. (“enGene USA”), an indirect, wholly-owned subsidiary of enGene Therapeutics Inc. (the “Company”) and Dr. Hussein Sweiti entered into an Amended and Restated Employment Agreement, effective as of May 6, 2026 (the “Sweiti Employment Agreement”), which amended and restated in its entirety the Employment Agreement, dated September 29, 2025, between enGene USA and Dr. Sweiti (the “Prior Employment Agreement”).
As amended and restated, the Sweiti Employment Agreement (i) reflects Dr. Sweiti’s current compensation arrangements with enGene USA, which includes an annual base salary of $561,750, an annual 40% bonus opportunity, and the ability to participate in employee benefit plans, (ii) revises Section 6(a) of the Prior Employment Agreement to include an additional proviso that, on or after June 1, 2026, Dr. Sweiti may resign for any reason or for no reason upon five business days’ prior written notice and shall remain entitled to receive his post-termination severance benefits from enGene USA, (iii) waives any obligation of Dr. Sweiti to repay the value of his sign-on bonus received under the Prior Employment Agreement, and (iv) provides for certain other administrative changes. Other substantive terms of the Sweiti Employment Agreement remain unchanged from the Prior Employment Agreement.
The foregoing description of the Sweiti Employment Agreement is qualified in its entirety by the complete text of the Sweiti Employment Agreement, a copy of which will be filed as an exhibit to the Company’s Quarterly Report on Form 10-Q for the period ended April 30, 2026.
Item 7.01 Regulation FD Disclosure.
On May 7, 2026, the Company issued a press release announcing updates regarding the pivotal cohort of its ongoing Phase 2 LEGEND trial of detalimogene voraplasmid (also known as “detalimogene” and previously EG-70) in high-risk, Bacillus Calmette-Guérin (“BCG”)-unresponsive patients with carcinoma in situ (“CIS”) with or without concomitant papillary disease, including new interim clinical data. In addition, as announced in the press release, the Company will host a conference call on May 7, 2026 to discuss the interim clinical data and has made available a slide presentation for the conference call. The full text of the press release is set forth in Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference. A copy of the slide presentation for the conference call is being furnished as Exhibit 99.2 to this Current Report on Form 8-K.
The information in this Item 7.01, including the slide presentation furnished under Exhibit 99.2, 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, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 8.01 Other Information.
As discussed above, on May 7, 2026, the Company issued a press release announcing updates regarding the pivotal cohort of its ongoing Phase 2 LEGEND trial of detalimogene, including new interim clinical data. A copy of the press release is filed herewith as Exhibit 99.1 to this Current Report on Form 8-K and the full text of the press release is incorporated into this Item 8.01 by reference.
The table below presents a summary of the newly announced interim clinical data supporting the press release; all terms used are as defined in the press release:
Any Time (N=124) |
6 Month (N=121) |
9 Month (N=113) |
12 Month (N=98) | |||||||
Total Population N=125 |
CR Rate* | 54.0% (CI: 44.9%, 63.0%) |
43.0% (CI: 34.0%, 52.3%) |
32.7% (CI: 24.2%, 42.2%) |
13.3% (CI: 7.3%, 21.6%) | |||||
| KM Estimate | 39.4% | 31.6% | 24.5% | |||||||
| * | Patients who discontinued without any disease evaluation or who discontinued after a CR are excluded from subsequent landmark response analysis. |
CR=complete response; defined as negative cystoscopy and negative urine cytology, or positive cystoscopy/cytology with negative bladder biopsy. |
CI: 95% Confidence Interval. |
Data as of 21 April 2026; Data collection and cleaning is ongoing. |
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
| Exhibit Number | Description | |
| 99.1 | Press Release of the Company, dated May 7, 2026 | |
| 99.2 | Slide Presentation, dated May 7, 2026 | |
| 104 | Cover Page Interactive Data File (Formatted as Inline XBRL) | |
SIGNATURES
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.
| ENGENE THERAPEUTICS INC. | ||||||
| Date: May 7, 2026 | By: | /s/ Ronald H.W. Cooper | ||||
| Name: Ronald H.W. Cooper Title: Chief Executive Officer and President | ||||||
Exhibit 99.1
enGene Announces Updated Interim Results From LEGEND Pivotal Cohort
54% complete response (CR) rate at any time; 43% six-month CR rate
Low rate of progression to muscle-invasive or higher disease (3.2%)
Low percentage of patients experienced treatment-related adverse events (TRAEs) (55%), nearly all of which were mild
Low percentage of patients experienced TRAEs leading to treatment interruption (2.4%) or discontinuation (2.4%)
Kaplan-Meier estimate of 12-month CR rate is 25%
enGene to host webcast to discuss results today, May 7, 2026, at 8:00 a.m. ET
BOSTON & MONTREAL, May 7, 2026 – enGene Therapeutics Inc. (Nasdaq: ENGN or “enGene” or the “Company”), a clinical-stage, non-viral genetic medicines company, today reported additional interim results from the pivotal cohort of its ongoing, Phase 2 LEGEND trial of detalimogene voraplasmid (also known as detalimogene) in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ (CIS) with or without concomitant papillary disease, which enrolled 125 patients. These data, as of April 21, 2026, demonstrated that patients treated with detalimogene achieved a 54% complete response (CR) at any time and a 43% CR rate at six months. The progression rate to muscle-invasive or advanced disease was low, at 3.2%. Detalimogene was generally well tolerated with 55% of patients having experienced a treatment-related adverse event, mostly mild (Grade 1 and 2).
“These updated data continue to reinforce the favorable safety and tolerability profile of detalimogene and its clinical activity in a heavily pretreated, high-risk NMIBC patient population with limited therapeutic options. Importantly, the low rate of progression to muscle-invasive disease leaves patients eligible for other bladder-sparing therapies,” said Ron Cooper, President and Chief Executive Officer, enGene. “While durability outcomes to date are not what we hoped, these data are preliminary. We are focused on evaluating the totality of the data as it evolves and plan to continue to engage with the FDA and the medical community.”
LEGEND Pivotal Cohort Data Update
The interim analysis is based on the 125 patients enrolled in Cohort 1. Patients who discontinued without any disease evaluation or who discontinued after a CR were considered not evaluable for subsequent landmark CR analysis.
Efficacy overview:
| • | 54% (95% CI: 45%, 63%) CR at any time (67/124) |
| • | 91% of responses occurred at first disease assessment |
| • | 43% (95% CI: 34%, 52%) CR rate at 6 months (52/121) with 14% (6/43) of patients having successfully converted from non-CR to CR post re-induction |
| • | Two re-induced patients have not yet had a 6-month assessment |
| • | Of the 52 responders at 6 months: |
| • | 37/44 patients who had a 9-month assessment were in CR; an additional 8 patients are pending evaluation |
| • | 13/22 patients who had a 12-month assessment were in CR; an additional 11 patients are pending evaluation |
| • | A maximum of 21 patients, including the 2 currently undergoing re-induction, still have the potential to achieve a CR at 12 months |
| • | The Kaplan-Meier (KM) estimate of the 12-month duration of response (DoR) is 25% (95% CI: 11%, 41%) |
| • | The KM estimate for median DoR is 37.3 weeks (range: 31.6-43.9 weeks) |
| • | Low rate of progression to muscle invasive or more advanced disease (3.2%) |
Safety overview:
| • | 55% of patients experienced a treatment-related adverse event (TRAE) |
| • | Among patients with any TRAE, 91% experienced Grade 1-2 TRAEs |
| • | Grade ≥3 TRAEs were reported in 6 patients (4.8%) |
| • | Low percentage of patients experienced TRAEs leading to treatment interruption (2.4%) and treatment discontinuation (2.4%) |
Among the 32 patients who had their first disease assessment after the last data analysis (October 24, 2025, reported November 11, 2025), CR rates were lower than previously reported results. The CR rate at any time was 39% and at 6 months was 32% for these patients. A preliminary subgroup analysis has not revealed any material differences in demographics or key disease characteristics. A more comprehensive analysis of these patients, including potential contributing factors, is ongoing.
As a beneficiary of FDA’s RMAT designation and a participant in the FDA’s CDRP program, enGene has completed the required FDA manufacturing validation batches and submitted a Statistical Analysis Plan (SAP) to the agency. The Company plans further engagement with the FDA as it approaches a potential Biologics License Application (BLA) filing and plans to provide an update in the second half of 2026.
The Company plans to share these data with the broader medical community at a Plenary Presentation at the upcoming American Urological Association meeting on May 15, 2026.
Safety Information
The overall tolerability profile associated with detalimogene is favorable. Of the 125 patients assessed for safety in Cohort 1, 69 patients (55%) experienced at least one TRAE, which were mainly Grade 1/2 in severity. The most common TRAEs were fatigue (22%), dysuria (14%), micturition urgency (12%), pollakiuria (12%), and bladder spasm (11%). Six patients experienced Grade ≥3 TRAEs, including one Grade 4 TRAE, which has resolved. There were no Grade 5 TRAEs reported. TRAEs leading to dose discontinuations (2.4%) and dose interruptions (2.4%) were rare.
Conference Call
enGene will host a conference call and live webcast at 8:00 a.m. ET today, May 7, 2026. Individuals interested in listening to the conference call may do so by clicking the following link, which is also available at the “Investors” section of the Company’s website at www.engene.com/investors. Following the live webcast, an archived version of the call will also be available on the Company’s website for 90 days.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on the healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can present as papillary outgrowths from the bladder wall, which are typically resected, or as carcinoma in situ (CIS), which consists of flat, multifocal lesions that cannot be resected. The two forms can also co-occur. About 75%-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50%-70%) and are potentially subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.
About Detalimogene Voraplasmid
Detalimogene is a novel, investigational, non-viral gene therapy for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (BCG)-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response.
Detalimogene was developed using the Company’s Dually Derivatized Oligochitosan® (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, reduce complexities related to safe handling and cold storage, and streamline both manufacturing processes and administration paradigms.
Detalimogene has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive carcinoma in situ (CIS) NMIBC with or without resected papillary tumors who are unable to undergo cystectomy. These designations are intended to expedite the development and review of drugs to serious or life-threatening conditions and fill an unmet medical need. Detalimogene has also been selected for the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program, designed to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy.
About the LEGEND Trial
Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk NMIBC. LEGEND’s pivotal cohort (Cohort 1) consists of 125 patients with high-risk, BCG-unresponsive NMIBC with CIS (with or without papillary disease) and is designed to serve as the basis of the Company’s planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, LEGEND includes three additional cohorts, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3).
About enGene
enGene is a clinical-stage biotechnology company mainstreaming non-viral genetic medicine through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene’s lead program is detalimogene voraplasmid (also known as detalimogene) for patients with non-muscle invasive bladder cancer (NMIBC), a disease with a high clinical burden. Detalimogene is being evaluated in the ongoing multi-cohort LEGEND Phase 2 trial, which includes a pivotal cohort studying detalimogene in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive patients with carcinoma in situ (CIS) with or without concomitant papillary disease. Detalimogene was developed using enGene’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA.
To learn more, please visit enGene.com and follow us on LinkedIn, X and BlueSky.
Forward-Looking Statements
Certain statements contained in this press release may constitute “forward-looking statements” within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”). enGene’s forward-looking statements include, but are not limited to, statements relating to the Company’s future plans, expectations, intentions, strategies and objectives. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “goal”, “intend”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar expressions (or the negative version of such words or expressions) may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: detalimogene’s potential efficacy, durability, safety, tolerability and ease of use profile, the development of detalimogene, the potential benefits of detalimogene, plans regarding regulatory interactions and a potential BLA submission for detalimogene, plans regarding updates on the LEGEND study, including clinical data and engagement with the FDA, and the potential benefits of medicines developed with the DDX platform. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond enGene’s control, that may cause actual events or results to differ materially from enGene’s current expectations. For example, there can be no guarantee that detalimogene will successfully complete necessary clinical development phases, including achieving positive results in the pivotal cohort of the LEGEND study, or that those results or any feedback from regulatory authorities will ultimately lead to BLA submission for, and the approval of, detalimogene.
Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks, uncertainties and assumptions relating to a number of other factors, which could cause the Company’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the inability of preliminary clinical data to predict the final results of the trial, changes in the results from enGene’s clinical trials, including due to new data collected from the ongoing LEGEND study or future studies, subsequent analysis of existing data, and audit and verification procedures; the content and timing of decisions made by the FDA and other regulatory authorities; the Company’s ability to recruit and retain qualified scientific and management personnel, establish clinical trial sites and enroll patients in its clinical trials, execute on the Company’s clinical development plans; and its ability to secure regulatory approval on anticipated timelines, and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission (“SEC”) on EDGAR, including those described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2025 (copies of which may be obtained at www.sedarplus.ca or www.sec.gov).
You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved.
###
Media contact:
JC Molina
Jmolina@engene.com
Investor contact:
Lauren Stival Hopfer
Lhopfer@engene.com
LEGEND Interim Data Update May 7, 2026 Exhibit 99.2
Disclaimers Cautionary Statement Regarding Forward-Looking Statements This Presentation contains certain forward-looking statements within the meaning of the federal securities laws and "forward-looking information" within the meaning of Canadian securities laws (collectively, "forward-looking statements"). Forward-looking statements may be identified by the use of the words such as “forecast”, “intend”, “develop”, “expect”, “anticipate”, “become”, “believe”, “continue”, “could”, “estimate”, “goal”, “intends”, “may”, “might”, “plan”, “possible”, “project”, “should”, “strategy”, “future”, “potential”, “opportunity”, “target”, “term”, “will”, “would”, “will be” or similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding detalimogene’s potential efficacy, durability, safety, tolerability and ease of use profile, the development of detalimogene, the potential benefits of detalimogene, plans regarding regulatory interactions and potential biologics license application (“BLA”) submission for detalimogene, plans regarding updates on the LEGEND study, including clinical data and engagement with the FDA, the development of detalimogene, estimates and forecasts of financial and performance metrics, projections of market opportunity and market share, the anticipated market acceptance of detalimogene, expectations and timing related to regulatory submissions and commercial product launches and the prospects for regulatory approval of detalimogene. These forward-looking statements are based on various estimates and assumptions, whether or not identified in this presentation, and on the current expectations of the management of enGene Therapeutics Inc. ("enGene"), are not predictions of actual performance, and are subject to risks and uncertainties. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond enGene's control, that may cause actual events or results to differ materially from enGene's current expectations. For example, there can be no guarantee that detalimogene will successfully complete necessary clinical development phases, including achieving positive results in the pivotal cohort of the LEGEND study, or that those results or any feedback from regulatory authorities will ultimately lead to a BLA submission for, and the approval of, detalimogene. Management's expectations and, therefore, any forward-looking statements in this presentation could also be affected by risks, uncertainties and assumptions relating to a number of other factors, which could cause the Company’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the inability of preliminary clinical data to predict the final results of the trial; changes in the results from enGene’s clinical trials, including due to new data collected from the ongoing LEGEND study or future studies, subsequent analysis of existing data, and audit and verification procedures; the content and timing of decisions made by the FDA and other regulatory authorities; the Company’s ability to recruit and retain qualified scientific and management personnel, establish clinical trial sites and enroll patients in its clinical trials, execute on the Company’s clinical development plans, and secure regulatory approval on anticipated timelines; and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission (“SEC”) on EDGAR, including those described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2025 (copies of which may be obtained at www.sedarplus.ca or www.sec.gov). You should carefully consider the risks and uncertainties described in the “Risk Factors” section of such Annual Report, as well as other documents if and when filed by enGene from time to time with the SEC and Canadian securities regulators. If any of these risks materialize or our assumptions prove incorrect, actual events and results could differ materially from those contained in the forward-looking statements. There may be additional risks that enGene does not presently know or that enGene currently believes are immaterial that could also cause actual events and results to differ. In addition, forward-looking statements reflect enGene’s expectations, plans, or forecasts of future events and views as of the date of this presentation. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. These forward-looking statements should not be relied upon as representing enGene’s assessments as of any date subsequent to the date of this presentation. Accordingly, undue reliance should not be placed upon the forward-looking statements contained herein. Intellectual Property This Presentation contains trademarks, service marks, trade names, copyrights, and products of enGene and other companies, which are the property of their respective owners. The use or display of third parties’ trademarks, service marks, trade names, copyrights, or products in this Presentation is not intended to, and does not, imply a relationship with enGene, or an endorsement of or sponsorship by enGene. Solely for convenience, the trademarks, service marks, and trade names referred to in this Presentation may appear without the ®, TM or SM symbols, but such references are not intended to indicate, in any way, that enGene will not assert, to the fullest extent permitted under applicable law, its rights or the rights of the applicable licensor in such trademarks, service marks and trade names. Industry and Market Data This Presentation relies on and refers to certain information and statistics based on estimates by enGene’s management and/or obtained from third party sources which enGene believes to be reliable. enGene has not independently verified the accuracy or completeness of any such third party information, which involves elements of subjective judgment and analysis that may or may not prove to be accurate. None enGene, or its affiliates or any third parties that provide information to enGene or its affiliates, such as market research firms, guarantees the accuracy, completeness, timeliness, or availability of any information. Neither enGene nor its affiliates nor any third parties that provide information to enGene and its affiliates, such as market research firms, is responsible for any errors or omissions (negligent or otherwise), regardless of the cause, or the results obtained from the use of such content. enGene may have supplemented such information where necessary, taking into account publicly available information about other industry participants and enGene management’s best view as to information that is not publicly available. Neither enGene nor its affiliates give any express or implied warranties with respect to the information included herein, including, but not limited to, any warranties regarding its accuracy or of merchantability or fitness for a particular purpose or use, and they expressly disclaim any responsibility or liability for direct, indirect, incidental, exemplary, compensatory, punitive, special, or consequential damages, costs, expenses, legal fees, or losses (including lost income or profits and opportunity costs) in connection with the use of the information herein. Lead Program (detalimogene voraplasmid) The lead program described herein is an investigational drug therapy that has not been subject to testing designed to demonstrate that the therapy is effective in humans or to provide a basis to predict in advance whether an adequate level of efficacy in humans will be demonstrated in further testing. Although the FDA has indicated that the Phase 2 portion of the current LEGEND study may potentially support BLA approval, that outcome will depend entirely on the final results of Phase 2 clinical testing.
Introductory Remarks
LEGEND Phase 2 Cohort 1: CONSORT Diagram Enrolled 3-month 6-month 9-month 12-month Maintenance 125 Patients 1 Discontinued 1 no disease evaluation (censored*) 124 Completed Evaluation 103 Completed Evaluation 2 pending evaluation at 6-mos 19 Discontinued 18 non-CR 1 CR (censored*) 44 Completed Evaluation 10 pending evaluation at 6- or 9-mos 22 Completed Evaluation 21 pending evaluation at 6- 9- or 12-mos 12 Ongoing 1 non-CR discontinued in follow-up 51 Discontinued 51 non-CR 11 Discontinued 7 non-CR** 4 CR (censored*) 9 Discontinued 9 non-CR at evaluation *Patients who discontinued without any disease evaluation or who discontinued after a CR are excluded from subsequent landmark response analysis. **One patient is displayed as non-CR at 9 months based on April 21 data analysis, and initiated Cycle 4. This patient has since been updated to CR post data analysis.
LEGEND Phase 2 Cohort 1: Baseline Demographics and Disease Characteristics Characteristics Pivotal Cohort 1 (N=125) Age, years Median (Range) 71.0 (35.0-90.0) Sex, n (%) Female 24 (19.2%) Male 101 (80.8%) Race n (%) White 114 (91.2%) Asian 4 (3.2%) Black or African American 2 (1.6%) Not Reported 5 (4.0%) ECOG PS, n (%) 0 119 (95.2%) 1 6 (4.8%) Characteristics Pivotal Cohort 1 (N=125) Tumor stage, n, % CIS with Ta/T1 disease 49 (39.2%) CIS only 76 (60.8%) Reason for no cystectomy, % Ineligible 9 (7.2%) Declined 116 (92.8%) Prior BCG doses, n Median (Range) 12 (6-50) Prior therapy other than BCG: Total 31 (24.8%) Intravesical chemotherapy 21 (16.8%) Systemic immunotherapy 8 (6.4%) Viral gene therapy 5 (4.0%) Other 4 (3.2%) Prior NMIBC recurrence(s) Median (Range) 2 (0-11) Note: BCG, bacillus Calmette-Guérin; CIS, carcinoma in situ; ECOG PS, Eastern Cooperative Oncology Group Performance Score; NMIBC, non-muscle invasive bladder cancer. Data as of 21 April 2026; Data collection and cleaning is ongoing.
Detalimogene Demonstrated Favorable Safety and Tolerability Profile TRAEs were Generally Mild and Reversible, Consistent with Catheterization TRAE= treatment-related adverse event. *One patient had a treatment-related pyelonephritis/sepsis that led to a dose interruption. No grade ≥ 3 TRAEs led to discontinuation. Six patients had grade 3 TRAEs, including one patient that had a grade 4 TRAE (elevated ALT) that has since resolved. Data as of 21 April 2026; Data collection and cleaning is ongoing Most TRAEs were localized, brief, low-grade events 69 patients (55.2%) had any grade TRAEs Of those with TRAEs, 91.3% had Grades 1-2 TRAEs resolved after a median of 8 days Only 6 patients (4.8%) had grade ≥3 TRAEs* TRAEs leading to dose interruption or discontinuation were rare Dose interruption in 3 patients (2.4%) Discontinuation in 3 patients (2.4%) Participants with events, n (%) Pivotal Cohort 1 (N=125) ≥ 1 Treatment-related adverse event 55.2% Most frequent TRAEs (≥10%) Fatigue Dysuria Micturition urgency Pollakiuria Bladder spasm 21.6% 13.6% 12.0% 12.0% 11.2%
LEGEND Cohort 1 Interim Efficacy Data * Patients who discontinued without any disease evaluation or who discontinued after a CR are excluded from subsequent landmark response analysis. **Results do not include one patient with potential CR at 9 months that is currently considered a non-CR in the database. CR=complete response; defined as negative cystoscopy and negative urine cytology, or positive cystoscopy/cytology with negative bladder biopsy. CI: 95% Confidence Interval. Data as of 21 April 2026; Data collection and cleaning is ongoing. Re-induction success rate of 14.0% (6/43 non-CRs at 3 months responded following re-induction) Low rate of progression to muscle-invasive or more advanced disease (3.2%) Preliminary Durability for the 52 patients who were 6-month responders: At the 9-month assessment, 37** CRs of 44 evaluable patients (8 patients pending 9-month assessment) At the 12-month assessment, 13 CRs of 22 evaluable patients (11 patients pending 12-month assessment) In total, 21 patients have the potential to reach the 12-month assessment, including 2 undergoing re-induction Any Time (N=124) 6 Month (N=121) 9 Month (N=113) 12 Month (N=98) Total Population N=125 CR Rate* 54.0% (CI: 44.9%, 63.0%) 43.0% (CI: 34.0%, 52.3%) 32.7% (CI: 24.2%, 42.2%) 13.3% (CI: 7.3%, 21.6%) KM Estimate 39.4% 31.6% 24.5%
Patients Post October 2025 Analysis Underperformed Earlier Subgroups *Data disclosed in press release dated November 11, 2025, and reflect a data analysis of October 24, 2025. ** Includes i) patients whose first post-baseline assessment took place following October 24, 2025, and ii) one patient that dropped out without any post-baseline assessment. Data as of 21 April 2026; Data collection and cleaning is ongoing. Preliminary subgroup analysis of baseline demographics and key disease characteristics demonstrated no material differences Marginal differences in efficacy observed across pre-protocol (n=31) and post-protocol (n=94) amendment patients Analysis remains ongoing Pre-Protocol Amendment October 2025 Analysis* N=31 Post-Protocol Amendment October 2025 Analysis* N=62 Newly Assessed Patients Since October 2025** N=32 Any time CR Rate 55% 63% 39% 6 Month CR Rate 41% 62% 32%
Bladder Rinse Opportunity
Surfactant Bladder Rinse Could Increase Transgene Expression Bladder lumen contains protective mucosal boundary layer DDX designed to spontaneously penetrate mucosal layer and transfect urothelium Surfactant bladder rinse designed to disrupt bladder mucosal barrier and further increase DDX permeability Potentially improves DDX penetration and urothelial transfection
Detalimogene and Surfactant Bladder Rinse (Polidocanol) Designed to Improve Gene Expression, Shorten Dwell Time, and Improve Efficacy Increases Magnitude & Duration of Gene Expression Shortens Bladder Dwell Time Improves Efficacy % Survived at Study End pg IL-12/mg tissue protein pg IL-12/mg protein A 5-min bladder rinse à >10× increase in IL-12 expression for longer duration A 5-min bladder rinse reduced the deta dwell time required to achieve maximal IL-12 expression from 60 to 15 min. A 5-min bladder rinse (BR) rescued the efficacy of a sub-optimal dose of deta in an orthotopic bladder cancer model p< 0.03 All data from mouse models. Polidocanol is an approved sclerosing agent for treatment of varicose veins and has an extensive clinical track record
Detalimogene and Surfactant Bladder Rinse LEGEND Cohort Underway 20 trial sites activated in US and Canada and actively recruiting patients Simple 5-min pre-treatment of the bladder with 50 mL of 0.25% polidocanol solution prior to detalimogene administration Total detalimogene dwell time shortened from 60 to 30 minutes to reduce patient and office burden Trial Study Design Target Population: Patients with BCG-unresponsive HR NMIBC with CIS +/- Papillary Enrollment: Potential to enroll up to 80 patients Target Number of Sites: 35-40 US, Canada and EU Status: Actively enrolling 1w 2w 3w 4w 5w 6w 7w 8w 9w 10w 11w 12w Cycle 2-4 x4 50 mL bladder rinse (5 min) 50 mL detalimogene (30 min) Urine collection for IL-12
Market Research Insights
Community Urologists: Higher Volume with Fewer Resources and Staff Source: enGene sourced market research (survey n=100 urologists and uro-oncologists). Small Private Practice Large Urology Group Health System / Hospital Academic Medical Center Patients per Procedure Room Patients per Urologist
Less Than Half of Urologists Prescribe Branded Therapies to BCG-UR Patients Small Private Practice Large Urology Group Practice Hospital / Health System Academic Medical Center “I prescribe [Product X] and [Product Y]. I just cannot administer them in my office. I have to go to a hospital I have a contract with.” – Small Private Practice Urologist Note: Branded therapies included Inlexzo, Adstiladrin, Anktiva , and Keytruda. Other included referral, chemotherapy, and radical cystectomy. Source: Market Research (survey n=100 urologists and uro-oncologists)
Low Adoption of Branded Therapies Driven by Logistics and Cost to Acquire Reasons for Limited Use of Branded Therapies Rank Top 3 Source: Market Research (survey n=100 urologists and uro-oncologists).
Market Research Findings: Opportunities for Detalimogene Challenge Detalimogene Opportunity Limited Resources Accessible in lower-resource settings: Nurse-administered without specialized procedure-room or BSL-2 hood requirements Burdensome Logistics Minimal logistical burden: No vial thaw, bench-top preparation, and refrigerated stability Cost to Acquire Commercial flexibility through potential low COGS: Supports access strategies such as consignment, reimbursement guarantees, and flexible billing
Preliminary Efficacy and Durability Estimates Data as of 21 April 2026; CI: 95% Confidence Interval. Data collection and cleaning is ongoing. These data are presented for informational purposes and are not based on any head-to-head or comparator clinical studies. Cross-trial comparisons are inherently limited and may suggest misleading similarities or differences. Accordingly, no direct comparisons should be made. Adstiladrin Detalimogene 51.0% (41.0, 61.0) 54.0% (44.9, 63.0) Product DOR % ≥ 12 Months Adstiladrin (n=98) 46% Detalimogene (n=125) 25% est** (CI: 11%, 41%) ** KM estimate Product 12 Months Landmark Adstiladrin (n=98) 24% Detalimogene (n=125) 25% est** ** KM estimate Months Percent Detalimogene → median 8.7 months Adstiladrin → median 9.7 months
Closing Remarks and Q&A
