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
BOSTON, MA, USA & MONTREAL. Canada I September 26, 2024 I enGene Holdings Inc. (Nasdaq: ENGN or “enGene” or the “Company”), is a clinical-stage genetic medicines company whose non-viral lead investigational product detalimogene voraplasmid (also known as detalimogene, and previously EG-70) is in an ongoing pivotal study in patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis). Today, the Company announced preliminary data from 21 patients assessed at three months, including 17 patients who were also assessed at six months, in the ongoing pivotal cohort of the LEGEND study. The Complete Response (CR) rate at any time was 71%, the CR rate at three months was 67% and the CR rate at six months was 47%. Detalimogene was generally well-tolerated by patients and no patients discontinued due to treatment-related adverse events.
“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 Colorado.
Bladder cancer is a top 10 cancer by incidence in the US with high annual treatment cost. NMIBC represents more than 75% of bladder cancer diagnosis and over 70% of urologists practice in the community setting where the majority of these patients are treated.
“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 (48%) experienced at least one treatment-related adverse event (TRAE), which were mainly Grade 1/2 in severity, except for two Grade 3 TRAEs (peripheral edema and urosepsis). The most common TRAEs (≥10%) were dysuria, bladder spasm, pollakiuria and fatigue. There were no Grade 4 or Grade 5 TRAEs reported.
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.
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.
SOURCE: enGene
Post Views: 193
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
BOSTON, MA, USA & MONTREAL. Canada I September 26, 2024 I enGene Holdings Inc. (Nasdaq: ENGN or “enGene” or the “Company”), is a clinical-stage genetic medicines company whose non-viral lead investigational product detalimogene voraplasmid (also known as detalimogene, and previously EG-70) is in an ongoing pivotal study in patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis). Today, the Company announced preliminary data from 21 patients assessed at three months, including 17 patients who were also assessed at six months, in the ongoing pivotal cohort of the LEGEND study. The Complete Response (CR) rate at any time was 71%, the CR rate at three months was 67% and the CR rate at six months was 47%. Detalimogene was generally well-tolerated by patients and no patients discontinued due to treatment-related adverse events.
“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 Colorado.
Bladder cancer is a top 10 cancer by incidence in the US with high annual treatment cost. NMIBC represents more than 75% of bladder cancer diagnosis and over 70% of urologists practice in the community setting where the majority of these patients are treated.
“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 (48%) experienced at least one treatment-related adverse event (TRAE), which were mainly Grade 1/2 in severity, except for two Grade 3 TRAEs (peripheral edema and urosepsis). The most common TRAEs (≥10%) were dysuria, bladder spasm, pollakiuria and fatigue. There were no Grade 4 or Grade 5 TRAEs reported.
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.
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.
SOURCE: enGene
Post Views: 193
