Detalimogene Demonstrates 71% Complete Response Rate at Any Time in Preliminary Analysis of LEGEND Pivotal Cohort
enGene Holdings Inc. (Nasdaq: ENGN) announced preliminary data from the pivotal cohort of the LEGEND study for detalimogene voraplasmid, their lead investigational product for high-risk, BCG-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis). The data showed:
- 71% Complete Response (CR) rate at any time
- 67% CR rate at three months
- 47% CR rate at six months
- Favorable tolerability with no drug-related discontinuations
The results are consistent with Phase 1 findings, supporting detalimogene's potential as a foundational therapy for NMIBC. The company plans protocol refinements to potentially enhance clinical benefits. Detalimogene's profile, including ease of handling and administration, minimal storage requirements, and lack of post-procedural patient restrictions, positions it as a promising treatment option, especially for community practice settings where over 70% of urologists practice.
enGene Holdings Inc. (Nasdaq: ENGN) ha annunciato dati preliminari dal cohort cruciale dello studio LEGEND per il detalimogene voraplasmid, il loro principale prodotto in fase di sperimentazione per il cancro della vescica non muscolare invasivo (NMIBC) di alto rischio, non responsivo al BCG, con carcinoma in situ (Cis). I dati hanno mostrato:
- 71% di tasso di Risposta Completa (CR) in qualsiasi momento
- 67% di tasso di CR a tre mesi
- 47% di tasso di CR a sei mesi
- Tollerabilità favorevole senza interruzioni correlate ai farmaci
I risultati sono coerenti con i riscontri della Fase 1, supportando il potenziale del detalimogene come terapia fondamentale per NMIBC. L'azienda prevede miglioramenti al protocollo per potenzialmente aumentare i benefici clinici. Il profilo del detalimogene, che include facilità di gestione e somministrazione, requisiti minimi di stoccaggio e assenza di restrizioni post-procedurali per i pazienti, lo posiziona come un'opzione terapeutica promettente, specialmente per contesti di pratica clinica dove oltre il 70% degli urologi esercitano.
enGene Holdings Inc. (Nasdaq: ENGN) anunció datos preliminares del cohorte clave del estudio LEGEND para el detalimogene voraplasmid, su principal producto en investigación para el cáncer de vejiga no muscular invasivo (NMIBC) de alto riesgo, no reactivo al BCG, con carcinoma in situ (Cis). Los datos mostraron:
- 71% de tasa de Respuesta Completa (CR) en cualquier momento
- 67% de tasa de CR a los tres meses
- 47% de tasa de CR a los seis meses
- Buena tolerabilidad sin interrupciones relacionadas con el fármaco
Los resultados son consistentes con los hallazgos de la Fase 1, apoyando el potencial del detalimogene como terapia fundamental para NMIBC. La compañía planea mejoras en el protocolo para posiblemente aumentar los beneficios clínicos. El perfil del detalimogene, que incluye facilidad de manejo y administración, requisitos mínimos de almacenamiento y la falta de restricciones post-procedimiento para los pacientes, lo posiciona como una opción de tratamiento prometedora, especialmente para entornos de práctica comunitaria donde más del 70% de los urólogos ejercen.
enGene Holdings Inc. (Nasdaq: ENGN)는 BCG 비반응성 고위험 비근육 침습성 방광암(NMIBC)과 제자리암종(Cis)에 대한 주요 연구 제품인 detalimogene voraplasmid의 LEGEND 연구의 중요한 코호트에서 초기 데이터를 발표했습니다. 데이터는 다음을 보여주었습니다:
- 어떠한 시점에서의 완전 반응(CR) 비율 71%
- 3개월 시점에서의 CR 비율 67%
- 6개월 시점에서의 CR 비율 47%
- 약물 관련 중단 없이 좋은 내약성
결과는 1상 조사 결과와 일관되며, NMIBC에 대한 기초 요법으로서 detalimogene의 잠재력을 뒷받침합니다. 회사는 임상적 이점을 향상시키기 위해 프로토콜 개선을 계획하고 있습니다. 사용 및 투여의 용이성, 최소한의 저장 요구 사항 및 시술 후 환자 제한이 없다는 등의 detalimogene의 프로필은 70% 이상의 비뇨기과 의사가 활동하는 지역사회 진료 환경에서 특히 유망한 치료 옵션으로 자리매김하게 합니다.
enGene Holdings Inc. (Nasdaq: ENGN) a annoncé des données préliminaires du cohort pivot de l'étude LEGEND pour le détailmogène voraplasmid, leur principal produit en phase d'investigation pour le cancer de la vessie non invasif à haut risque (NMIBC) résistant au BCG, avec carcinome in situ (Cis). Les données ont montré :
- taux de Réponse Complète (CR) de 71% à tout moment
- taux de CR de 67% à trois mois
- taux de CR de 47% à six mois
- bonne tolérance sans interruptions liées aux médicaments
Les résultats sont cohérents avec les constatations de la phase 1, soutenant le potentiel du détailmogène en tant que thérapie fondamentale pour le NMIBC. La société prévoit des améliorations du protocole afin de potentiellement augmenter les bénéfices cliniques. Le profil du détailmogène, y compris la facilité de manipulation et d'administration, les exigences minimales de stockage et l'absence de restrictions pour les patients après la procédure, le positionne comme une option thérapeutique prometteuse, en particulier dans les milieux de pratique communautaire où plus de 70% des urologues exercent.
enGene Holdings Inc. (Nasdaq: ENGN) gab vorläufige Daten aus der entscheidenden Kohorte der LEGEND-Studie für Detalimogen Voraplasmid bekannt, ihr führendes innovatives Produkt für hochriskantes, BCG-resistentes, nicht-muskelinvasives Blasenkarzinom (NMIBC) mit Carcinoma in situ (Cis). Die Daten zeigten:
- 71% vollständige Rücklaufquote (CR) zu jedem Zeitpunkt
- 67% CR-Quote nach drei Monaten
- 47% CR-Quote nach sechs Monaten
- Günstige Verträglichkeit ohne medikamentenbedingte Abbrüche
Die Ergebnisse sind konsistent mit den Erkenntnissen der Phase 1, die das Potenzial von Detalimogen als grundlegende Therapie für NMIBC unterstützen. Das Unternehmen plant Protokolländerungen zur potenziellen Verbesserung klinischer Vorteile. Das Profil von Detalimogen, einschließlich einfacher Handhabung und Verabreichung, minimaler Lageranforderungen und fehlender Einschränkungen für Patienten nach dem Eingriff, positioniert es als vielversprechende Behandlungsoption, insbesondere in Gemeinschaftspraxen, in denen über 70% der Urologen tätig sind.
- 71% Complete Response rate at any time in preliminary analysis
- 67% Complete Response rate at three months
- 47% Complete Response rate at six months
- Favorable tolerability profile with no drug-related discontinuations
- Consistency with Phase 1 results, increasing confidence in the emerging profile
- Potential for broad utilization in community practice settings due to ease of handling and administration
- Two Grade 3 treatment-related adverse events reported (peripheral edema and urosepsis)
Insights
The preliminary data from the LEGEND pivotal cohort for detalimogene voraplasmid in high-risk, BCG-unresponsive NMIBC with CIS is highly encouraging. A
The favorable tolerability profile, with no drug-related discontinuations, is particularly noteworthy. This could potentially allow for longer treatment durations and better outcomes. The predominantly Grade 1/2 adverse events align with what we'd expect for an intravesical therapy.
If these results hold up in the full cohort, detalimogene could become a valuable option for NMIBC patients who have alternatives after BCG failure. Its ease of administration and storage could make it particularly attractive for community practice settings, where most NMIBC patients are treated.
From a market perspective, detalimogene's potential is significant. Bladder cancer's position as a top 10 cancer by incidence in the US, coupled with high annual treatment costs, presents a substantial market opportunity. With over
The drug's design for practicality in urologist-managed NMIBC treatment aligns well with market needs. If approved, it could capture a significant share of the NMIBC market, especially in community practices. The potential for broad utilization due to its efficacy, tolerability and convenience could translate to strong revenue growth for enGene.
Investors should monitor the full pivotal cohort results and any regulatory developments, as positive outcomes could significantly impact enGene's market valuation, particularly given its current market cap of
Data from pivotal cohort demonstrate compelling clinical activity, consistent with Phase 1 results
Favorable tolerability profile with no drug-related discontinuations
Detalimogene’s profile to date supports its potential as a foundational therapy for NMIBC
enGene to host a conference call to discuss preliminary data today at 8:00 a.m. ET
“We are pleased with this preliminary dataset, which clearly demonstrates that detalimogene is highly active and generally well tolerated. The data are consistent with what was observed in Phase 1 and give us increased confidence in the emerging profile,” said Raj Pruthi, M.D., Chief Medical Officer of enGene. “We are also planning protocol refinements in LEGEND, which we believe will provide patients with additional clinical benefit.”
“The promising initial efficacy and safety data observed in LEGEND’s pivotal cohort, combined with detalimogene’s ease of handling, administration, minimal storage requirements, and lack of post-procedural patient restrictions underscore its potential to become a broadly utilized treatment option for NMIBC patients, especially in the community practice setting,” remarked Suzanne Merrill, M.D., a urologist specializing in bladder cancer with the United Urology Group in
Bladder cancer is a top 10 cancer by incidence in the US with high annual treatment cost. NMIBC represents more than
“Detalimogene was designed to be the most practical therapy for urologists to manage NMIBC,” said Ron Cooper, Chief Executive Officer of enGene. “The early results from our pivotal LEGEND study clearly demonstrate that detalimogene has the potential to offer a highly differentiated profile by striking an optimal balance between efficacy, tolerability, and ease of use.”
Safety Information
The overall tolerability profile associated with detalimogene was favorable, and there have been no drug-related discontinuations in the study. Of the 42 patients assessed for safety, inclusive of all Phase 2 cohorts, 20 patients (
About LEGEND
The LEGEND study is a multi-cohort study to establish efficacy of detalimogene in high-risk NMIBC patients with Cis, as well as those with papillary-only NMIBC. The pivotal cohort is an approximately 100 patient, open-label study evaluating detalimogene in BCG-unresponsive NMIBC patients with Cis. Patients receive 800µg/mL of detalimogene intravesically at weeks one, two, five, and six during a 12-week cycle, for up to four cycles, with assessments at three, six, nine, and 12 months. The study’s primary efficacy endpoint is the percentage of patients with a complete response at 12 months, based on cystoscopy, urine cytology and biopsy.
In addition to LEGEND’s pivotal cohort, the Company plans to initiate enrollment of three additional cohorts in the LEGEND study: (i) high-risk BCG-exposed patients with Cis and BCG-naïve patients with Cis in cohorts 2a and 2b, respectively, and (ii) high-risk BCG-unresponsive NMIBC patients with papillary-only disease in cohort 3. Enrollment for cohorts 2a, 2b, and 3 is expected to begin in the fourth quarter of 2024. The Company remains on track to file a Biologics License Application (BLA) for detalimogene in its pivotal cohort in mid-2026.
Investor Conference Call
enGene will host a conference call and live webcast at 8:00 a.m. ET today, September 26, 2024. Individuals interested in listening to the conference call may do so by using the webcast link 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 website.
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 lead program is detalimogene voraplasmid, (also known as detalimogene, and previously EG-70) 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 study, which includes a pivotal cohort studying detalimogene in Bacillus Calmette Guérin (BCG)-unresponsive patients with carcinoma in situ (Cis). 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. For more information, visit enGene.com.
Forward-Looking Statements
Some of the 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 expectations, hopes, beliefs, intentions, goals, strategies, forecasts and projections. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “intend”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar 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, our plans to amend the LEGEND protocol, our plans regarding expansion and modification of the LEGEND study for potential additional bladder cancer indications for detalimogene, our expectations regarding timing of the enrollment of patients in the additional cohorts of the LEGEND study, and our expectations regarding regulatory submissions.
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, that preliminary clinical data may not accurately reflect the complete results of a particular study and remain subject to audit and verification, and final data may differ materially from preliminary data; 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240926869206/en/
For media contact: media@engene.com
For investor contact: investors@engene.com
Source: enGene Holdings Inc.
FAQ
What is the Complete Response rate for detalimogene in the LEGEND pivotal cohort?
How well-tolerated is detalimogene in the LEGEND study?
What are the potential advantages of detalimogene for NMIBC treatment?
What percentage of patients experienced treatment-related adverse events in the LEGEND study?
