Positive End-of-Phase 2 Meeting with FDA
Management reported a collaborative End-of-Phase 2 meeting with the FDA for the IPF-related chronic cough program with alignment on pathway to NDA, primary endpoint (objective cough monitor), and agreement to proceed with 2 pivotal Phase III trials.
Planned Phase III Program and Timelines
Company plans two parallel global Phase III trials for IPF-related chronic cough: a 52-week trial (primary efficacy at 24 weeks, ~300 patients) and a confirmatory 12-week trial (~130 patients) with initiation of the first trial expected in Q2 and the second in H2 of the year.
Strong 2025 Clinical Data Momentum
Positive 2025 readouts from CORAL (IPF chronic cough) and RIVER (refractory chronic cough) trials supported capital raises and enabled progression to registrational programs.
Cash Runway to Fund Key Milestones
Trevi ended 2025 with approximately $188 million in cash, cash equivalents and marketable securities, providing an expected runway into 2028 to reach top-line data for: Phase IIb RCC, Phase IIb non-IPF chronic cough and the 12-week pivotal Phase III IPF readout.
Market Opportunity and Addressable Patient Populations
Estimated U.S. IPF population of ~150,000 with ~2/3 (≈66.7%) having uncontrolled chronic cough; non-IPF ILD estimated at ~228,000 with 50%–60% (≈114k–136.8k) having uncontrolled cough — more than doubling the market opportunity beyond IPF alone.
RCC and Dose Optimization Strategy
Planned Phase IIb parallel-arm dose-ranging RCC trial (3 doses + placebo) to determine dosing; crossover data suggest activity at low dose and interest in exploring lower or once-daily dosing and related IP/formulation strategies.
Operational Readiness and Enrollment Expectations
Management expects enrollment for the larger Phase III to take ~1 year with ~80–100 sites (majority U.S.); strong physician and site interest reported and plans for patient advocacy engagement to support recruitment.
Secondary Endpoints and Patient-Reported Outcomes
Primary objective cough monitor retained; key secondary PROs (cough frequency/severity) and breathlessness moved into key secondary category after encouraging CORAL findings, strengthening potential label-relevant data.
Regulatory and Safety Preparations
Agreement with FDA on remaining standard label-enabling Phase I studies (renal/hepatic impairment, food effect) and DDI assessments (including interaction with new antifibrotic nerandomilast and CYP inducer/inhibitor studies) to support NDA.
Controlled-Substance/Scheduling Outlook
Management reported constructive discussions with controlled-substances staff, submitted human abuse potential and respiratory safety data, and expressed growing conviction the product will remain unscheduled.