Jaguar Health (JAGX) family companies Napo Pharmaceuticals and Napo Therapeutics 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 with additional supplemental intravenous 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. As previously announced, and as presented last month at the 11th Annual ELITE PED-GI Congress in Abu Dhabi in the United Arab Emirates, the initial proof-of-concept results from the ongoing and independent proof-of-concept investigator-initiated trial at Sheikh Khalifa Medical City showed that crofelemer reduced the required TPN and/or supplementary intravenous fluids – collectively referred to as parenteral support – in the first participating MVID patient by up to 27% at the end of 12-weeks of crofelemer treatment. 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 protocol for the IIT at SKMC, the pediatric MVID patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days. After just 8 days, the patient’s parents requested reinitiation of crofelemer dosing, as the patient’s symptoms were worsening – evidenced by increased stool output and decreased urine output. Hence, the patient was restarted on daily treatment with crofelemer.
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