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Clinical Development
Programs

Our Dually Derivatized Oligochitosan® (DDX) platform, which enables a carrier designed to penetrate mucosal tissue and deliver a wide range of sizes and types of cargo, powers a deep pipeline of novel non-viral genetic developmental medicines across oncology and beyond.

Pipeline

Pipeline

Program

Cohort/Indication

Phase 1

Phase 2

Phase 3


EG-70
(detalimogene voraplasmid)

LEGEND Study
(intravesical)

Cohort 1: BCG-Unresponsive NMIBC with Cis

80%

Anticipated milestone: Interim Efficacy: Q3 2024*, BLA Filing: Q2/Q3 2026

Cohort 2a: BCG-Naïve NMIBC with Cis

60%

Anticipated milestone: Enrollment Commences: Q4 2024

Cohort 2b: BCG-Exposed NMIBC with Cis

60%

Anticipated milestone: Enrollment Commences: Q4 2024

Cohort 3: BCG-Unresponsive NMIBC, Papillary-Only

58%

Anticipated milestone: Enrollment Commences: Q4 2024

*The Company expects to release interim data from the LEGEND study’s BCG-unresponsive NMIBC with Cis cohort by the end of September 2024, comprising approximately 20 patients for which the company intends to report response status at both the 3m and 6m timepoints

Expected timelines and anticipated milestones reflect enGene management’s current estimate and are subject to change

EG-70 Clinical Program

Advancing a powerful non-viral gene therapy option for patients with NMIBC

More than half a million new cases of bladder cancer are diagnosed each year, and most new cases (about 70-80%) are non-muscle invasive bladder cancer (NMIBC). Patients suffering from Bacillus Calmette-Guerin (BCG)-unresponsive NMIBC with carcinoma in situ (Cis) are particularly underserved and have limited treatment options, and patients who fail BCG generally resort to having a radical cystectomy (bladder removal) – a life-altering, invasive and challenging procedure that significantly impairs patients’ quality of life. Furthermore, there is a chronic global shortage of BCG, making it difficult for these patients to get the therapy they need in the first place.

EG-70 has the potential to improve the standard of care for patients with NMIBC because of its optimized delivery to the bladder, safety in handling, and ease of administration.

EG-70 is a novel non-viral gene therapy designed to elicit a local immune response following the delivery to the bladder urothelium. This approach has the potential to induce a potent local immune response at the site of the tumor, resulting in greater therapeutic benefit while reducing undesirable systemic toxicity.  

The LEGEND Study

EG-70 in Patients with NMIBC BCG-Unresponsive and High-Risk NMIBC Incompletely Treated with BCG or BCG-Naïve

This study consists of two phases: a Phase 1 dose-escalation portion to establish safety and the recommended Phase 2 dose, followed by a Phase 2 portion to establish the effectiveness of EG-70. The study includes patients with NMIBC who have proven to be unresponsive to BCG therapy, as well as high-risk patients with NMIBC who are BCG-naïve or have received incomplete BCG treatment.

Additional information about the LEGEND clinical trial, including eligibility criteria, can be found on clinicaltrials.gov (identifier: NCT04752722). 

The LEGEND Study

EG-70 in Patients with NMIBC BCG-Unresponsive and High-Risk NMIBC Incompletely Treated with BCG or BCG-Naïve

This study consists of two phases: a Phase 1 dose-escalation portion to establish safety and the recommended Phase 2 dose, followed by a Phase 2 portion to establish the effectiveness of EG-70. The study includes patients with NMIBC who have proven to be unresponsive to BCG therapy, as well as high-risk patients with NMIBC who are BCG-naïve or have received incomplete BCG treatment.

Additional information about the LEGEND clinical trial, including eligibility criteria, can be found on clinicaltrials.gov (identifier: NCT04752722).