Jaguar Health provides overview of 2025 crofelemer-related catalysts

Jaguar Health (JAGX) provided an overview of expected Q2 – Q4 2025 catalysts related to potential follow-on indications for crofelemer, the company’s novel plant-based prescription drug approved by the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research. Rare Disease Intestinal Failure Program: Initial proof-of-concept results were issued last week for the ongoing investigator-initiated trial of a novel liquid formulation of crofelemer in Abu Dhabi in the United Arab Emirates in pediatric patients with intestinal failure due to the rare orphan diseases microvillus inclusion disease and short bowel syndrome. The initial POC results, presented April 26, 2025 at the Annual ELITE PED-GI Congress, show crofelemer reduced the required total parenteral nutrition and/or supplementary intravenous fluids, collectively referred to as parenteral support, in patients with intestinal failure due to MVID and SBS-IF by up to 27% and 12.5% respectively. Crofelemer also reduced stool volume output, frequency of watery stools, and increased urine output – indicating improved nutrient oral absorption. Based on these initial findings, crofelemer’s novel antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to reduce TPN and associated complications, including liver, renal, and cognitive deficits, as well as infections from IV infusion, in this patient population. The observed TPN reduction is particularly compelling for MVID, an ultrarare pediatric disease characterized by severe diarrhea and malabsorption that requires intensive parenteral support for nutritional and fluid management and for which no approved drug treatments exist, or any potential approach to reduce TPN. This initial proof-of-concept data in MVID supports crofelemer’s potential inclusion in the European Medicines Agency’s (EMA) PRIME program for expediated and assisted regulatory approval in the EU as well as in the FDA’s Breakthrough Therapy program for expedited regulatory approval in the US. Jaguar, through Jaguar family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics, is currently supporting three POC IITs, and conducting two placebo-controlled Phase 2 studies, for crofelemer for MVID and/or SBS-IF patients in the US, EU, and/or Middle East/North Africa regions. Additional POC results from IITs in MVID and/or SBS-IF are expected throughout 2025. Cancer Therapy-Related Diarrhea Program: As announced, the FDA granted Napo a Type C Meeting in Q2 2025 to discuss the responder analysis results in the prespecified subgroup of patients with breast cancer in Napo’s recently conducted Phase 3 OnTarget trial. Patients with breast cancer accounted for 183 of the 287 participants in this unprecedented prophylactic clinical trial of crofelemer for diarrhea in adults with solid tumors receiving targeted therapy with or without standard chemotherapy. While the initial top line results from the OnTarget study showed that this multicenter, double-blind, placebo-controlled pivotal trial did not meet its primary endpoint for the prespecified analysis of all tumor types, the subgroup analysis in adult breast cancer patients indicates that crofelemer achieved significant results in this subgroup. The company’s goal for the meeting is to discuss the most efficient potential pathway to make crofelemer available to adult breast cancer patients for CTD.