Talphera (TLPH) investor relations material

Key catalysts and development timeline

• Three major catalysts expected in the next year, including pivotal study readout for nafamostat mesylate (Niyad) in anticoagulation for extracorporeal circuits, with data targeted by year-end and PMA filing within three months after data readout.

• Product has breakthrough designation and a six-month FDA review clock, aiming for approval and market launch in the second half of next year.

• Study enrollment is over 75% complete, with improved site selection and protocol changes accelerating progress; full enrollment expected before year-end.

• Data cleaning is ongoing in real time, enabling top-line data release within four to five weeks after last patient out.

• Pre-PMA meeting with FDA planned to maintain alignment, with submission expected within three months of final data.

Study design, endpoints, and risk assessment

• Primary endpoint is activated clotting time (ACT), an objective measure, with the study powered at over 90% to meet this endpoint using conservative assumptions.

• Protocol modifications, including reduced sample size and alignment with FDA, have streamlined enrollment and execution.

• Dropouts are rare and mainly due to withdrawal of care in critically ill patients; no deaths observed within 24 hours.

• Secondary endpoints include filter life and clotting events, which are highly relevant for hospital operations and cost management.

• Recent KDIGO guidelines now recommend nafamostat for CRRT, citing studies showing fewer clotting events and filter changes.

Market opportunity and competitive landscape

• Updated market research, using scrubbed CPT code data and post-COVID growth trends, estimates 100,000–165,000 CRRT procedures annually in the U.S., with a 5–6% growth rate.

• Current practice often involves no anticoagulant or use of heparin (systemic, with bleeding risk) or citrate (complex, not FDA-approved), both of which have significant limitations.

• Nafamostat is expected to replace heparin and citrate as first-line or rescue therapy due to its efficacy and ease of use.

• KDIGO guidelines' recent update to recommend nafamostat is expected to drive adoption in the U.S. once approved, especially as current alternatives are suboptimal.

• U.S. market adoption is anticipated to be strong, with nafamostat positioned as a primary agent for CRRT anticoagulation.