NDV-01’s 12-month Phase 2 follow-up in high-risk NMIBC patients produced a 95% complete response rate at any time and a durable 76% rate at 12 months. BCG-unresponsive patients achieved an 80% CR rate at 12 months, with no progression to muscle-invasive disease, no radical cystectomies, no ≥ Grade 3 treatment-related adverse events and zero treatment-related discontinuations.

In March 2026, the company closed an oversubscribed $160 million PIPE financing led by leading healthcare investors. These proceeds, combined with a $93 million cash balance at December 31, 2025, are expected to fund operations through 2029 and support initiation of the NDV-01 Phase 3 RESCUE program.

Regulatory alignment is in place for two registrational pathways: an open-label, randomized controlled trial in intermediate-risk NMIBC with disease-free survival as the primary endpoint, and a single-arm trial in second-line BCG-unresponsive NMIBC with complete response rate as the primary endpoint. Both studies are on track to initiate in mid-2026 under the RESCUE program.

The company reported a Q4 net loss of $19.9 million and R&D expenses of $8.1 million for Q4 2025, down from $11.0 million a year earlier. Upcoming corporate milestones include U.S. IND clearance for NDV-01, Phase 3 RESCUE initiation, initial three-month NDV-01 data by year-end 2026, and the start of a sepranolone Phase 2 study in Prader-Willi syndrome.