NDV-01, a sustained-release intravesical gemcitabine/docetaxel formulation, has reported a 92% complete response at any time point at nine months in an ongoing single-arm Phase 2 study in high-risk NMIBC, with the latest six-month follow-up highlighting favorable overall safety. The Phase 2 trial’s primary efficacy readouts are 12-month complete response rate and safety, with duration of response and event-free survival as key secondary measures.
The immediate news is a poster at SUO 2025 updating six-month follow-up from the single-center Phase 2 and a development timeline: Relmada plans to initiate Phase 3 in the first half of 2026 across two indications, high-risk and intermediate-risk NMIBC. The company cites recent FDA interactions that outline a potential registrational pathway. NDV-01 is engineered for 10-day bladder retention through a soft matrix designed to enhance local exposure and limit systemic toxicity; the formulation is positioned for in-office administration without anesthesia and is patent-protected through 2038.
Strategically, this is a bid to formalize and differentiate a widely used off-label regimen. Gemcitabine/docetaxel has become a pragmatic option amid chronic BCG shortages and constrained efficacy of existing salvage therapies. A sustained-release, ready-to-use product could offer operational and pharmacologic advantages over sequential instillations that rely on compounding, variable dwell times, and site-specific workflows. It also positions Relmada against an evolving standard in high-risk BCG-unresponsive disease, where single-arm CRR at defined timepoints remains acceptable to regulators, while the intermediate-risk segment will likely demand randomized, recurrence-focused evidence. For Relmada, which has been better known in CNS development, NDV-01 is a pivot into a large, procedure-driven oncology market with clearer adoption mechanics inside urology practices.
The operational impact for sites is straightforward: an in-office, short-prep instillation without anesthesia is aligned with existing cystoscopy schedules and could reduce dependence on external compounding pharmacies. The 10-day retention model, however, will require consistent catheter protocols, patient counseling on post-instillation management, and vigilant monitoring for chemical cystitis and device-related adverse events. CROs planning Phase 3 will need to translate a single-center signal into multicenter consistency, standardize cystoscopy/cytology adjudication, and ensure specimen handling minimizes bias in response ascertainment. For sponsors, the key regulatory tension is trial design bifurcation: a single-arm, CR-focused study may suffice in high-risk BCG-unresponsive cohorts, but intermediate-risk disease typically compels randomized comparisons versus established intravesical regimens, with recurrence-free survival and durability as critical endpoints. Payers will evaluate whether a proprietary SR formulation justifies premium pricing against low-cost compounded agents; demonstrable gains in durability, fewer instillations, or reduced procedural burden will be central to value arguments.
What to watch next is the 12-month CRR and durability curve from the ongoing Phase 2, including relapse kinetics post-CR and any cystectomy-free survival signal. Phase 3 design choices will be telling: enrichment for CIS-heavy populations in the high-risk study, the control arm and non-inferiority versus superiority framing in intermediate-risk, and the handling of BCG availability variability across regions. Safety granularity matters, particularly discontinuation rates due to local toxicity over repeated monthly maintenance. Manufacturing scale-up and CMC robustness for the gel matrix will be under scrutiny ahead of multicenter initiation. If Relmada can pair a reproducible efficacy signal with operational simplicity that reduces site friction and compounding dependencies, NDV-01 could find a practical niche; if not, entrenched off-label practice and payer caution will be the headwinds.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
