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Novel sustained-release NDV-01 achieved a 92% CR rate in high-risk patients with NMIBC with strong safety, supporting advancement to phase 3 studies.
Treatment with sustained release of gemcitabine and docetaxel (NDV-01) led to complete response (CR) at any time in 92% (n = 23 of 25) of patients with high-risk non–muscle-invasive bladder cancer (NMIBC), according to data from the TRCG-011 trial (NCT06663137) presented during the 26th Annual Meeting of the Society of Urologic Oncology.1
The rates of CR at 3, 6, and 9 months were 84% (n = 21 of 25), 87% (n = 20 of 23), and 85% (n = 17 of 20), respectively.
“NDV-01 is a novel sustained formulation of gemcitabine/docetaxel for intravesical use. NDV-01 provides excellent 9-month safety and efficacy in patients with high-risk NMIBC. Data support effectiveness in patients who are BCG-naive, -exposed, and -unresponsive. The study is ongoing, including second-year follow-up,” Yair Lotan, MD, lead study author and professor of urology, chief of Urologic Oncology, and holder of the Jane and John Justin Distinguished Chair in Urology, at UT Southwestern Medical Center in Dallas, Texas, and coauthors wrote in the poster.
Sequential intravesically administered gemcitabine and docetaxel have been identified as a potential treatment approach for patients with high-risk NMIBC. NDV-01 is an investigational intravesical agent intended to deliver sustained release of gemcitabine and docetaxel over the course of 10 days while minimizing the time required to administer traditional gemcitabine and docetaxel and the need for a specialized pharmacy or hood.
The single-arm, open-label TRCG-011 trial was intended to evaluate the safety and efficacy of the novel formulation in patients with high-grade NMIBC. Inclusion criteria mandated that patients have high-risk disease with carcinoma in situ (CIS)/Tis, Ta, or T1 tumors and be naive, unresponsive, intolerant, or exposed to BCG.
Eligible patients received 6 bi-weekly instillations followed by monthly maintenance instillations through month 12. Subsequent follow-up consisted of urinary cytology, cystoscopy, upper tract imaging, and transurethral resection of bladder tumor or bladder biopsy, if necessary.
The primary end points were safety and 12-month CR rate. Secondary end points included duration of response, event-free survival, and pharmacokinetics.
CR was defined as a negative cystoscopy, cytology, and biopsy, if indicated. The first evaluation for CR was performed at month 3. Patients who did not have a CR at that point were allowed to be reinduced with another 6 bi-weekly courses of treatment. CR assessments also occurred at months 6, 9, and 12.
Baseline characteristics revealed that patients (n = 29) were mostly male (n = 30; 89%) vs female (n = 6; 17%) and had a median age of 73 years (range, 54-93). Most patients were either naive (n = 15; 42%) or unresponsive (n = 17; 47%) to BCG; 11% (n = 4) of patients had been exposed to BCG and had received a median of 6 doses (range, 0-21) of BCG. Disease stage was classified as pure CIS (n = 3; 8%), Ta/T1 plus CIS (n = 7; 19%), Ta (n = 21; 58%), and T1 (n = 6; 17%).
All patients had an ECOG performance status of 0 or 1.
Twenty-three patients reached the first disease assessment at 3 months and were included in the per-protocol analysis. Eleven patients are awaiting their first response assessment. All patients who received at least 1 dose of treatment were included in the safety analysis.
A total of 36 patients received at least 1 dose of NDV-01; 22 (61%) had a treatment-emergent adverse effect that manifested as dysuria (62%), asymptomatic positive urine culture (9%), and hematuria (7%). Grade 3 or greater TRAEs did not occur, and no patients discontinued therapy because of an AE.
Notably, there were no cases of progression to muscle-invasive disease, and no patients underwent radical cystectomy.
The developer of the agent, Relmada Therapeutics, announced that it has received feedback from the FDA following its Type B pre-IND meeting regarding the phase 3 program for NDV-01 in NMIBC.2 The company is expected to move forward with two studies with the intention of pursuing approval in high-grade, second-line BCG-unresponsive NMIBC and intermediate-risk NMIBC in the adjuvant setting.
“Relmada is engaged with regulators for a registrational program.1 Pivotal studies in both intermediate-risk and high-risk BCG-unresponsive NMIBC populations are planned for the first half of 2026,” the authors wrote in conclusion.
Disclosures: Lotan disclosed being a paid consultant of Relmada Therapeutics.
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