Jaguar Health Says First Patient Randomized In Napo's Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Trial To Evaluate Safety And Efficacy Of Crofelemer In Pediatric Patients With MVID

As announced, initial proof-of-concept (POC) results from distinct ongoing investigator-initiated trial (IIT) in Abu Dhabi show crofelemer reduced total parenteral support in a pediatric MVID patient with intestinal failure by up to 27%; per the IIT protocol, patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days, but was restarted on daily crofelemer treatment after just 8 days, as patient's symptoms were worsening

Additional POC IIT results expected throughout 2025 for MVID; results from company's MVID Phase 2 study expected with completion of treatment in H2 2026

Ground-breaking results in single-digit number of MVID patients could potentially lead to expedited approval in the EU through PRIME and to Breakthrough Therapy designation in the US

SAN FRANCISCO, CA / ACCESS Newswire / May 20, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals, Inc. (Napo) and Napo Therapeutics S.p.A. today announced that the first patient has been randomized in Napo's randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety and efficacy of the novel crofelemer oral powder for solution formulation in pediatric patients with intestinal failure due to the ultrarare disease MVID.

Patients with MVID suffer from devastating diarrhea and dehydration caused by this debilitating, lifelong condition. These pediatric patients often require lifelong and life-sustaining total parenteral nutrition (TPN) with additional supplemental intravenous (IV) fluids, termed total parenteral support, which is the standard of care for MVID, for up to 7 days a week greater than 50% of the time each day to provide adequate nutrient and electrolyte support. While crucial for MVID patients, TPN carries a significant risk of morbidity and mortality due to infections, metabolic complications, liver and/or kidney disease, and a risk of neurodevelopmental delay. There are currently no approved drug treatments for MVID.

Based on the initial proof-of-concept findings presented last month at the 11th Annual ELITE PED-GI Congress, crofelemer's novel antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients, as evidenced by the reductions in total parenteral support and potential to improve the quality of life of MVID patients.