Jaguar Health provides update on orphan disease intestinal failure program

Jaguar Health (JAGX) provided updates on the company’s orphan disease intestinal failure program. Jaguar, through Jaguar family companies Napo Pharmaceuticals and Napo Therapeutics, is currently supporting two independent proof-of-concept investigator-initiated trials, and conducting two placebo-controlled Phase 2 studies, of crofelemer, Jaguar’s novel plant-based anti-secretory prescription drug, in patients with intestinal failure due to microvillus inclusion disease and short bowel syndrome in the United States, European Union, and/or Middle East/North Africa regions. As announced, and as presented April 26, 2025 at the Annual ELITE PED-GI Congress, initial proof-of-concept results from the ongoing exploratory, single-arm open label non-randomized IIT of crofelemer in Abu Dhabi in pediatric intestinal failure patients show that crofelemer reduced the required total parenteral nutrition and supplementary intravenous fluids in the first participating MVID patient by up to 27% and in the first participating SBS-IF patient by up to 12.5%. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output – an indicator of improved nutrient oral absorption. Data from the third patient enrolled in the IIT is expected. Completion of Napo’s randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients is expected in mid-2026 as planned. Based on the initial findings of the ongoing IIT in Abu Dhabi, crofelemer’s paradigm-shifting 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 patients with intestinal failure due to MVID and short bowel syndrome. The observed groundbreaking 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. The initial proof-of-concept data in MVID supports crofelemer’s potential inclusion in the European Medicines Agency’s PRIMEprogram that may accelerate regulatory approval pathways in the EU. This data may also support qualification of crofelemer for the FDA’s Breakthrough Therapyprogram for expedited regulatory approval in the US. Additional proof-of-concept results from IITs are expected throughout 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs. In accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID and SBS-IF could support reimbursed early patient access to crofelemer for these debilitating conditions.