Jaguar Health announces initial proof-of-concept results on crofelemer

Jaguar Health (JAGX) family companies Napo Pharmaceuticals and Napo Therapeutics announced that initial proof-of-concept results show that a novel liquid formulation of crofelemer reduced the required total parenteral nutrition and supplementary intravenous fluids in a third intestinal failure patient. This is the second pediatric patient with intestinal failure due to the orphan disease short bowel syndrome who was treated with crofelemer. To date, three patients with intestinal failure due to SBS-IF or microvillus inclusion disease have been treated with crofelemer in this exploratory, single-arm open label non-randomized IIT in Abu Dhabi. As announced, and as presented April 26, at the Annual ELITE PED-GI Congress, initial proof-of-concept results from this study show that crofelemer, Jaguar’s novel plant-based anti-secretory prescription drug, reduced the required TPN 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. Per the IIT protocol, as announced, the initial pediatric MVID patient participating in the study was taken off crofelemer after 12 weeks of treatment for an intended period of 30 days. However, after just 8 days, the patient’s parents requested reinitiation of crofelemer dosing, as the patient’s symptoms worsened – evidenced by increased stool output and decreased urine output – and the patient was restarted on daily treatment with crofelemer. The initial pediatric SBS-IF patient participating in the study also had crofelemer withdrawn per the protocol after 12 weeks. This patient then exhibited a relapse of symptoms and was put back on daily treatment with crofelemer, and has since been maintaining a significant TPN reduction. Jaguar plans to continue supplying crofelemer to Dr. Mohamad Miqdady, the principal investigator for this IIT, for the patients participating in this study for as long as Dr. Miqdady feels is medically necessary. Dr. Miqdady has submitted initial proof-of-concept data from the ongoing IIT in Abu Dhabi for all three above-referenced patients to a major international pediatric gastroenterological conference for consideration for presentation. Jaguar, through Jaguar family companies Napo and Napo Therapeutics, is currently supporting two independent proof-of-concept IITs, and conducting two placebo-controlled Phase 2 studies, of crofelemer in patients with intestinal failure due to MVID and SBS-IF in the United States, European Union, and/or Middle East/North Africa regions. 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.