Jaguar Health Inc. (NASDAQ: JAGX) reported progress in developing crofelemer, its FDA-approved botanical drug, for rare congenital gastrointestinal disorders including microvillus inclusion disease and short bowel syndrome with intestinal failure.

The company's family company Napo Pharmaceuticals is developing a novel crofelemer powder for oral solution formulation, distinct from the currently approved Mytesi delayed-release tablet form. Crofelemer is derived from the Croton lechleri tree and works through an anti-secretory mechanism targeting intestinal chloride ion channels.

Jaguar announced several development milestones, including continuation of a pivotal clinical trial extension phase in microvillus inclusion disease patients. The company targets submitting a New Drug Application for crofelemer powder for oral solution by mid-2027.

The European Society for Pediatric Gastroenterology, Hepatology & Nutrition accepted two late-breaking abstracts for presentation on liquid oral crofelemer treatment in pediatric intestinal failure patients. Previous proof-of-concept results showed the liquid formulation reduced required parenteral support by up to 37% in microvillus inclusion disease patients and up to 15% in short bowel syndrome patients.

Microvillus inclusion disease is a rare congenital condition causing life-threatening dehydration in infants, requiring lifelong parenteral support including total parenteral nutrition and intravenous fluids. Short bowel syndrome with intestinal failure prevents sufficient nutrient and fluid absorption through the intestine.

"Crofelemer represents a differentiated pharmaceutical development story: traditional medicinal knowledge helped identify a promising botanical source, and modern drug development transformed that knowledge into a highly characterized FDA-approved prescription drug," said Lisa Conte, Jaguar's president and CEO.

The company emphasized the complexity of crofelemer's chemistry, manufacturing, and controls as a barrier to generic replication. Jaguar has collaborated with 19 indigenous cultural groups across 30 locations in Peru for sustainable harvesting of the source material.