enGene to Present Three Posters at the 2025 ASCO Genitourinary Cancers Symposium (ASCO GU) Highlighting the Ongoing Clinical Development of Detalimogene Voraplasmid for the Treatment of Non-Muscle Invasive Bladder Cancer (NMIBC)
enGene Holdings (Nasdaq: ENGN) announced three poster presentations at the 2025 ASCO Genitourinary Cancers Symposium, showcasing their lead investigational product detalimogene voraplasmid. The treatment targets high-risk, BCG-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS).
The company's pivotal LEGEND study is actively enrolling patients across the USA, Canada, Europe, and Asia-Pacific. The treatment utilizes enGene's Dually Derivatized Oligochitosan® (DDX) platform, designed to overcome viral gene therapy limitations. There are approximately 730,000 bladder cancer patients in the U.S., with 75-80% being NMIBC cases. Among high-risk NMIBC patients, 50-70% become BCG-unresponsive and experience recurrence or progression.
The presentations will cover preliminary LEGEND study results, mechanism of action details, and a phase 1/2 study of detalimogene voraplasmid intravesical monotherapy.
enGene Holdings (Nasdaq: ENGN) ha annunciato tre presentazioni di poster al simposio sui tumori genito-urinari ASCO 2025, mettendo in evidenza il loro principale prodotto sperimentale detalimogene voraplasmid. Questo trattamento è mirato a combattere il tumore della vescica non muscolare invasivo ad alto rischio, resistente al BCG, con carcinoma in situ (CIS).
Lo studio cruciale LEGEND dell'azienda sta attivamente reclutando pazienti negli USA, Canada, Europa e Asia-Pacifico. Il trattamento utilizza la piattaforma Dually Derivatized Oligochitosan® (DDX) di enGene, progettata per superare le limitazioni della terapia genica virale. Negli Stati Uniti, ci sono circa 730.000 pazienti con tumore della vescica, di cui il 75-80% presenta casi di NMIBC. Tra i pazienti ad alto rischio con NMIBC, il 50-70% non risponde al BCG e sperimenta recidive o progressioni.
Le presentazioni copriranno i risultati preliminari dello studio LEGEND, i dettagli del meccanismo d'azione e uno studio di fase 1/2 della monoterapia intravesicale con detalimogene voraplasmid.
enGene Holdings (Nasdaq: ENGN) anunció tres presentaciones de cartel en el Simposio sobre Cánceres Genitourinarios de ASCO 2025, presentando su principal producto de investigación detalimogene voraplasmid. Este tratamiento está dirigido al cáncer de vejiga no muscular invasivo (NMIBC) de alto riesgo que no responde a BCG y presenta carcinoma in situ (CIS).
El estudio crucial LEGEND de la empresa está llevando a cabo el reclutamiento de pacientes en EE.UU., Canadá, Europa y Asia-Pacífico. El tratamiento utiliza la plataforma Dually Derivatized Oligochitosan® (DDX) de enGene, diseñada para superar las limitaciones de la terapia génica viral. En EE.UU., hay aproximadamente 730,000 pacientes con cáncer de vejiga, de los cuales el 75-80% son casos de NMIBC. Entre los pacientes de alto riesgo con NMIBC, el 50-70% se vuelve resistente a BCG y experimenta recurrencia o progresión.
Las presentaciones cubrirán los resultados preliminares del estudio LEGEND, detalles del mecanismo de acción y un estudio de fase 1/2 de la monoterapia intravesical con detalimogene voraplasmid.
enGene Holdings (Nasdaq: ENGN)은 2025 ASCO 비뇨기암 심포지엄에서 detalimogene voraplasmid라는 주요 임상 시험 제품에 대한 세 가지 포스터 발표를 발표했습니다. 이 치료는 BCG에 반응하지 않는 고위험 비근육 침습성 방광암(NMIBC)과 제자리암(CIS)을 타겟합니다.
회사의 주요 LEGEND 연구는 미국, 캐나다, 유럽, 아시아-태평양 지역에서 환자를 적극적으로 모집하고 있습니다. 이 치료는 enGene의 이중 유도화 올리고키토산® (DDX) 플랫폼을 활용하여 바이러스 유전자 치료의 한계를 극복하도록 설계되었습니다. 미국에는 약 730,000명의 방광암 환자가 있으며, 이 중 75-80%는 NMIBC 사례입니다. 고위험 NMIBC 환자 중 50-70%가 BCG에 반응하지 않으며 재발 또는 진행을 경험합니다.
발표에서는 LEGEND 연구의 초기 결과, 작용 기전의 세부 사항, detaylimogene voraplasmid 단일 요법의 1/2상 연구에 대해 다룰 것입니다.
enGene Holdings (Nasdaq: ENGN) a annoncé trois présentations d'affiches au symposium ASCO sur les cancers génito-urinaires 2025, présentant leur principal produit d'investigation detalimogene voraplasmid. Ce traitement cible le cancer de la vessie non-muscle invasif (NMIBC) à haut risque résistant au BCG avec carcinome in situ (CIS).
L'étude clé LEGEND de l'entreprise recrute activement des patients aux États-Unis, au Canada, en Europe et dans la région Asie-Pacifique. Le traitement utilise la plateforme Dually Derivatized Oligochitosan® (DDX) d'enGene, conçue pour surmonter les limites de la thérapie génique virale. On estime qu'il y a environ 730 000 patients atteints de cancer de la vessie aux États-Unis, dont 75 à 80 % sont des cas de NMIBC. Parmi les patients à haut risque de NMIBC, 50 à 70 % deviennent réfractaires au BCG et connaissent des récidives ou une progression.
Les présentations couvriront les résultats préliminaires de l'étude LEGEND, des détails sur le mécanisme d'action et une étude de phase 1/2 de la monothérapie intravesicale avec detalimogene voraplasmid.
enGene Holdings (Nasdaq: ENGN) hat drei Posterpräsentationen beim ASCO-Symposium für genito-urinäre Krebserkrankungen 2025 angekündigt, die ihr führendes Prüfprodukt detalimogene voraplasmid vorstellen. Diese Behandlung zielt auf hochrisikobehafteten, BCG-resistenten, nicht-muskelinvasiven Blasenkrebs (NMIBC) mit Carcinoma in situ (CIS) ab.
Die wegweisende LEGEND-Studie des Unternehmens rekrutiert aktiv Patienten in den USA, Kanada, Europa und im asiatisch-pazifischen Raum. Die Behandlung nutzt die Dually Derivatized Oligochitosan® (DDX) Plattform von enGene, die entwickelt wurde, um die Einschränkungen der viralen Gentherapie zu überwinden. In den USA gibt es etwa 730.000 Blasenkrebs-Patienten, wobei 75-80% der Fälle NMIBC sind. Unter den hochrisikobehafteten NMIBC-Patienten werden 50-70% BCG-resistent und erleben Rückfälle oder Fortschritte.
Die Präsentationen werden die vorläufigen Ergebnisse der LEGEND-Studie, Details zum Wirkmechanismus und eine Phase 1/2-Studie zur intravesikalen Monotherapie mit detalimogene voraplasmid behandeln.
- Large addressable market with 730,000 bladder cancer patients in the U.S.
- Active patient enrollment across multiple global regions
- Novel non-viral treatment approach addressing limitations of current therapies
- None.
Detalimogene voraplasmid is a novel, investigational, pivotal-stage, non-viral genetic medicine designed to mitigate systemic risks and deliver durable efficacy for patients with high-risk NMIBC, including Bacillus Calmette-Guérin (BCG)-unresponsive disease
Ongoing, pivotal, registrational LEGEND study is actively enrolling patients with sites participating in the
enGene’s Dually Derivatized Oligochitosan® (DDX) platform is designed to potentially overcome the limitations of viral gene therapies and increase patient and clinician access to therapies
“Patients with NMIBC often face a debilitating, multi-year journey with limited treatment options and significant negative impacts to daily life. These patients are most commonly treated by community urologists who face unique challenges accessing and providing optimal care, including global treatment shortages and complex usage and handling,” said Ron Cooper, Chief Executive Officer of enGene. “We are determined to develop genetic therapies for underserved conditions like NMIBC and ultimately provide a long overdue treatment innovation designed to advance care and ease the treatment burden.”
There are an estimated 730,000 patients in the
“The current treatment options for patients with high-risk NMIBC are sadly sub-optimal, often resulting in disease progression, recurrence or removal of the bladder as a life-altering measure of last resort,” said Anthony Cheung, Chief Scientific Officer and Co-Founder of enGene. “If approved, we believe detalimogene voraplasmid will be uniquely compelling to both patients and physicians by combining a streamlined administration process designed to ease the treatment experience with durable efficacy and a favorable safety and tolerability profile.”
Presentation details are outlined below:
- Abstract #: 802
- Poster #: F29
- Clinical Trial Registration Number: NCT04752722
- Session Title: Poster Session B: Urothelial Carcinoma
- Presentation Date/Time: Friday, February 14 from 11:30am-12:45pm PT
- Location: Level 1, West Hall
- Presenting Author: John Taylor III, MD, MS, Professor of Urology & Cancer Biology, University of Kansas Cancer Center
- Abstract #: 826
- Poster #: G22
- Clinical Trial Registration Number: NCT04752722
- Session Title: Poster Session B: Urothelial Carcinoma
- Presentation Date/Time: Friday, February 14 from 11:30am-12:45pm PT
- Location: Level 1, West Hall
- Presenting Author: Vikram Narayan, MD, Assistant Professor in the Department of Urology at Emory University School of Medicine
- Abstract #: TPS886
- Poster #: J14
- Clinical Trial Registration Number: NCT04752722
- Session Title: Trials In Progress Poster Session B: Urothelial Carcinoma
- Presentation Date/Time: Friday, February 14 from 11:30am-12:45pm PT
- Location: Level 1, West Hall
- Presenter: Shreyas Joshi, MD, MPH, Assistant Professor in the Department of Urology at Emory University School of Medicine
About Non-Muscle Invasive Bladder Cancer (NMIBC)
Non-muscle invasive bladder cancer (NMIBC) is a disease with a significant patient burden, high clinical needs and massive economic impact on our 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 deeper into the muscle of the bladder wall. About 75
About Detalimogene Voraplasmid
Detalimogene voraplasmid (formerly known as EG-70) is a novel, investigational, non-viral genetic medicine 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 voraplasmid has received Fast Track designation from the
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, simplify safe handling and cold storage complexities and streamline both manufacturing processes and administration paradigms.
About the Pivotal LEGEND Trial
Detalimogene voraplasmid is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk, non-muscle invasive bladder cancer (NMIBC). LEGEND’s pivotal cohort (Cohort 1) consists of approximately 100 patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) (with or without papillary disease) and is designed to serve as the basis of the Company’s Biologics License Application (BLA) filing. In addition to this pivotal cohort, three additional cohorts are actively enrolling patients, 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).
The LEGEND trial is actively enrolling patients with sites participating in the
About enGene
enGene is a clinical-stage biotechnology company mainstreaming genetic medicines 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 mission is to deliver innovative non-viral medicines to improve the quality of life for people with urological cancers. enGene’s lead program is detalimogene voraplasmid for patients with non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) – a disease with a high clinical burden – who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG).
To learn more, please visit enGene.com and follow us on LinkedIn.
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 regarding enGene’s management teams’ expectations, hopes, beliefs, intentions, goals or strategies regarding the future. 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”, “intends”, “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: our beliefs as to the potential benefits of detalimogene, including its potential to provide a highly differentiated treatment option, if approved, and the potential benefits of medicines developed with the DDX platform.
Many factors, risks, uncertainties and assumptions 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 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 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
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.
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Media Contact:
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Investor Contact: investors@engene.com
Source: enGene Holdings Inc.
FAQ
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