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Preliminary results from LEGEND: a Phase 2 study of detalimogene voraplasmid, a novel, investigational, non-viral genetic medicine for high-risk non-muscle invasive bladder cancer (NMIBC)
Mark Preston, MD
1, John A. Taylor, MD
2, Ashish M. Kamat, MD
3, Suzanne Merrill, MD
4, Sam S. Chang, MD
5, Vignesh T. Packiam, MD
6, Shreyas Joshi, MD
7, Jen-Jane Liu, MD
8, Ricardo Rendon, MD
9, Wassim Kassouf, MD
10, Jeff Holzbeierlein, MD
2, Mark Tyson, MD
11, Rian Dickstein, MD
12, Raj Satkunasivam, MD
13, Neal Shore, MD
14, Amy N. Luckenbaugh, MD
5, Alberto Martini, MD
15, Raj Pruthi, MD
16, Katherine Chan, MD
16, Anthony Cheung, MD
17, Christine Tosone, MD
16, Yair Lotan, MD
18.
1Brigham and Women's Hospital, Boston, MA, USA, 2University of Kansas Cancer Center, Kansas City, KS, USA, 3University of Texas MD Anderson Cancer Center, Houston, TX, USA, 4Colorado Urology, Brighton, CO, USA, 5Vanderbilt University, Nashville, TN, USA, 6Rutgers RWJ Barnabas Health, West Orange, NJ, USA, 7Emory University School of Medicine, Atlanta, GA, USA, 8Oregon Health Science University, Portland, OR, USA, 9Dalhousie University, Halifax, NS, Canada, 10McGill University, Montreal, QC, Canada, 11Mayo Clinic, Phoenix, AZ, USA, 12Chesapeake Urology, Hanover, MD, USA, 13Houston Methodist-Weill Cornell Medical College, Houston, TX, USA, 14Carolina Urology Research Center, Myrtle Beach, SC, USA, 15University of Cincinnati Cancer Center, Cincinnati, OH, USA, 16enGene Inc.,, Waltham, MA, USA, 17enGene Inc. St-Laurent, QC, Canada, 18University of Texas Southwestern Medical Center, Dallas, TX, USA.
Background: Detalimogene voraplasmid (EG-70) is a novel, investigational, non-viral genetic medicine for high-risk NMIBC, including BCG-unresponsive disease. The Phase 1 portion of LEGEND (NCT04752722) demonstrated a promising safety profile and an overall complete response (CR) of 73% at any time. Phase 2 is ongoing; preliminary efficacy results of the pivotal Cohort 1 (BCG-unresponsive NMIBC with CIS) and safety for all Cohorts are reported here.
Methods: Key eligibility criteria: ≥18 years; ECOG PS 0−2; high-risk NMIBC, ± resected coexisting papillary (Ta/T1) tumors, ineligible for/elected not to undergo cystectomy. Cohorts: BCG-unresponsive with CIS (1; pivotal Cohort); BCG-naïve with CIS (Cohort 2A); BCG-exposed with CIS (Cohort 2B); BCG-unresponsive with high-grade papillary disease without CIS (Cohort 3). Patients received four-doses, 50 mL intravesically at Weeks 1, 2, 5 & 6 of a 12-week cycle x 4 cycles. Primary endpoint: CR rate at Week 48; safety. Secondary endpoints: PFS; CR rate at Week 12, 24, 36 & 96, % of patients with a durable CR at 12 Months.
Results: As of September 13, 2024, 21 patients were evaluable for efficacy in Cohort 1. In the 42 safety-evaluable patients (all Cohorts), treatment-related adverse events were observed in 20 (47.6%; all G1/2), most commonly (≥10%): dysuria (21.4%); bladder spasm (19.0%); pollakiuria (11.9%); fatigue (11.9%). Overall CR rate 71%; CR rate 67% at 3 Months and 47% at 6 Months.
Conclusions: Preliminary data from the pivotal Phase 2 portion of LEGEND suggest a promising safety/tolerability profile. Overall, 71% of patients achieved a CR, with 67% achieving a CR at 3 Months and 47% achieving a CR at 6 Months.
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