Talphera (TLPH) Maxim Group’s 2024 Healthcare Virtual Summit summary

Company overview and product background

• Focuses on acute care settings, developing Niyad, a unique anticoagulant for extracorporeal circuits, currently in phase III trials.

• Niyad's active ingredient, nafamostat, is a serine protease inhibitor with 30 years of use in Japan and South Korea for multiple indications.

• The product is designed to prevent clotting in continuous renal replacement therapy (CRRT) circuits, targeting critically ill ICU patients.

• Classified as a device by the FDA due to its action outside the body, resulting in a faster six-month review process and breakthrough designation.

• Breakthrough status was granted due to significant unmet needs and limitations of current U.S. anticoagulants, heparin and citrate.

Clinical trial design and progress

• The pivotal phase III NEPHRO CRRT study is a single, registrational trial with 166 patients, randomized to nafamostat or placebo.

• Primary endpoint is mean activated clotting time over 24 hours; study completion is defined at 72 hours.

• Trial is efficient and objective, with no placebo effect possible due to the nature of the endpoint.

• Site activation was delayed due to logistical challenges, but enrollment is now underway at multiple sites, with more high-potential sites coming online.

• Up to 14 sites will participate, and the study is expected to complete in 2025, with timing dependent on enrollment cadence.

Intellectual property and market potential

• Six years of data exclusivity expected upon PMA approval, with additional method-of-use patents filed to extend protection into the 2040s.

• U.S. market opportunity for CRRT anticoagulation estimated at $100 million with potential to double if used in chronic dialysis settings.

• Additional indications could include acute pancreatitis, disseminated intravascular coagulation, and possibly COVID-related uses.

• All resources are currently focused on completing the CRRT trial.