Relmada gains FDA support for dual Phase 3 study paths of NDV-01 in non-muscle invasive bladder cancer

Relmada Therapeutics Inc. (NASDAQ: RLMD) has received formal written feedback from the U.S. Food and Drug Administration confirming that two separate registrational study designs for its lead intravesical therapy NDV-01 are acceptable to support potential approval in non-muscle invasive bladder cancer (NMIBC). The decision gives Relmada an unprecedented degree of flexibility in advancing its Phase 3 development strategy and represents a turning point in how the company positions itself in the urologic oncology landscape.

The FDA’s correspondence outlines a dual-path strategy: a single-arm registrational trial targeting the high-grade, BCG-unresponsive NMIBC population, and a randomized, controlled adjuvant trial in intermediate-risk NMIBC following transurethral resection of bladder tumor (TURBT). This regulatory nod allows Relmada to tailor NDV-01’s trajectory toward both an expedited niche indication and a larger-scale commercial indication in the adjuvant setting.

How the FDA’s two-path feedback accelerates NDV-01’s route toward potential approval and market positioning

The FDA’s acknowledgment of two distinct registrational paths is a major regulatory milestone for Relmada, particularly because NDV-01’s development pathway now mirrors the flexibility historically afforded to other breakthrough intravesical agents. The agency’s feedback suggests that a single-arm study design may be sufficient for BCG-unresponsive patients—a population with limited treatment options and high recurrence risk—provided that NDV-01 continues to demonstrate strong complete response (CR) durability and safety outcomes.

In contrast, the randomized adjuvant trial in intermediate-risk NMIBC will compare NDV-01 against observation following TURBT, using a time-to-event primary endpoint. This design opens the possibility of a broad label that could capture patients earlier in the disease continuum. Importantly, the FDA confirmed that no additional non-clinical studies are required to support a 505(b)(2) New Drug Application, enabling Relmada to leverage previously established safety profiles for gemcitabine and docetaxel—the two active components in NDV-01’s proprietary gel formulation.

For Relmada’s clinical and regulatory teams, this alignment effectively derisks the program. It not only eliminates redundant preclinical work but also provides clarity for protocol development and potential fast-track or breakthrough designations. The company stated that Phase 3 initiation remains on track for the first half of 2026, contingent on completion of long-term follow-up from its Phase 2 study.

Why NDV-01’s Phase 2 data have drawn scientific and regulatory interest despite limited patient numbers

Relmada’s updated Phase 2 data, presented alongside the FDA feedback, highlight NDV-01’s clinical promise. In the ongoing open-label study, NDV-01 achieved a 92 % overall response rate at any time point among evaluable NMIBC patients (23 of 25). The 9-month complete response rate stood at 85 % (17 of 20), with 91 % (10 of 11) achieving CR in the BCG-unresponsive subgroup and 88 % (7 of 8) maintaining CR at 9 months. Notably, no patients progressed to muscle-invasive disease, and no radical cystectomies were performed during the evaluation period.

Equally significant is the safety profile: no Grade ≥ 3 treatment-related adverse events were observed, suggesting that the intravesical combination’s local delivery mechanism effectively limits systemic toxicity. The hydrogel vehicle used in NDV-01 provides prolonged bladder wall exposure while minimizing systemic absorption—a pharmacologic feature that may help differentiate it from older agents with higher systemic risk.

Nevertheless, scientific caution persists. The sample size remains small, and durability beyond 12 months remains unproven. In bladder cancer, recurrence rates often rise sharply after the first year, meaning NDV-01’s long-term benefit must be validated before clinical enthusiasm translates into widespread adoption. Moreover, while FDA feedback provides regulatory latitude, approval will depend on achieving prespecified endpoints in statistically robust populations across both study arms.

How NDV-01’s design leverages known chemotherapeutic agents in a novel delivery system to target high-risk NMIBC

Mechanistically, NDV-01 represents a formulation-driven innovation rather than a novel chemical entity. It combines gemcitabine and docetaxel—two long-established cytotoxic drugs—within a biodegradable hydrogel designed for sustained intravesical release. This allows for higher local drug concentrations and prolonged dwell time without increasing systemic exposure.

This design addresses a long-standing challenge in bladder cancer therapy: conventional intravesical chemotherapy often fails to maintain sufficient mucosal contact time for durable tumor cell eradication. NDV-01’s formulation ensures that therapeutic concentrations are maintained throughout the bladder surface, reducing washout effects that typically compromise efficacy.

The strategy aligns with a growing emphasis on repurposing known agents through novel delivery systems to accelerate clinical development under the 505(b)(2) framework. The approach significantly reduces development cost and time by relying on pre-existing toxicology and pharmacology data. This, in turn, enhances investor confidence that NDV-01 can progress through pivotal trials without unforeseen regulatory or safety setbacks.

How Relmada’s regulatory milestone is influencing broader market confidence in oncology innovation

Following the announcement, Relmada Therapeutics’ share price (NASDAQ: RLMD) experienced mild volatility but trended upward in pre-market and intraday trading, reflecting cautious optimism among biotech investors. Sentiment indicators across institutional forums showed that traders interpreted the FDA’s two-path alignment as a de-risking event rather than a binary outcome catalyst. The regulatory clarity provides a definable roadmap for NDV-01’s transition into Phase 3, improving the stock’s narrative coherence within the mid-cap biotech segment.

From a financial standpoint, Relmada still faces execution risk. The Phase 3 program, spanning two major indications, will demand substantial capital. Analysts expect the company to pursue non-dilutive financing or a potential co-development partnership to preserve balance-sheet stability. The 505(b)(2) route should lower trial costs relative to first-in-class NMIBC therapies, but manufacturing scale-up of the hydrogel delivery system may offset part of that savings.

In broader context, investor sentiment toward oncology-focused mid-caps has improved in Q4 2025 amid increased FDA approval activity and renewed M&A interest from large pharmaceutical companies. If NDV-01’s 12-month data confirm durability and safety, Relmada could emerge as an attractive acquisition target or licensing partner for firms seeking bladder-sparing therapeutic portfolios.

What comes next for NDV-01 as Relmada advances toward Phase 3 and long-term durability readouts

The company plans to finalize protocol submissions for both registrational studies in early 2026, with trial initiation expected in the first half of the year. Concurrently, 12-month follow-up data from the ongoing Phase 2 study are expected to be released in 2026 Q1, providing the first look at NDV-01’s durability curve. These data will be crucial in determining whether the single-arm design can convincingly support an NDA.

Relmada’s management has also emphasized that manufacturing readiness and clinical site selection are underway, particularly in U.S. academic urology centers familiar with NMIBC trial infrastructure. The company aims to leverage its existing relationships with key opinion leaders in urologic oncology to accelerate enrollment—a critical differentiator in a field where patient identification remains challenging.

If the Phase 3 studies replicate the observed CR rates with durable, bladder-sparing outcomes, NDV-01 could redefine intravesical therapy standards. However, achieving this will require meticulous adherence to trial design, adequate statistical powering, and transparent data dissemination to maintain regulatory and investor confidence.

What Relmada’s FDA feedback reveals about biotech market confidence

Relmada’s announcement illustrates how regulatory alignment can become a sentiment catalyst in a biotech environment dominated by binary risk perception. By securing FDA endorsement of both a single-arm and randomized registrational path, Relmada effectively reframed NDV-01 from a high-risk exploratory project into a late-stage development asset with multiple exit routes.

Investor discussions following the news showed moderate bullish sentiment, with traders emphasizing the 505(b)(2) designation’s cost-saving potential. Still, analysts highlighted that Relmada’s broader pipeline diversification remains limited, meaning NDV-01 must deliver to sustain valuation.

From a macro-industry viewpoint, this FDA communication reinforces the agency’s continued openness to adaptive design in oncology trials—particularly those addressing high-unmet-need indications like BCG-unresponsive NMIBC. It signals that data quality and clinical durability, rather than sheer novelty, remain the decisive factors guiding regulatory acceleration.