Napo Therapeutics, a Jaguar Health family company, is making strides in the development of crofelemer, a novel oral plant-based prescription drug, for rare diseases in Europe. The company was recently named 'Best Pharmaceuticals Innovator of the Year - Europe' by The European business publication, underscoring its commitment to addressing unmet needs in rare gastrointestinal disorders.
Clinical Development of Crofelemer
Crofelemer is currently the subject of five clinical efforts, including three proof-of-concept investigator-initiated trials (IIT) and two Phase 2 studies, for the rare disease indications of microvillus inclusion disease (MVID) and/or short bowel syndrome with intestinal failure (SBS-IF) in the US, EU, and/or Middle East/North Africa regions. Initial proof-of-concept results are expected in Q2 2025.
Focus on Microvillus Inclusion Disease (MVID) and Short Bowel Syndrome (SBS)
Napo Therapeutics is supporting proof-of-concept studies for both MVID and SBS, with results expected by the end of 2024 and throughout 2025. Phase 2 trials for both conditions are planned to start before the end of 2024. These studies could support early patient access to crofelemer in specific EU countries.
MVID, an ultrarare congenital diarrheal disorder, is a severe infantile disease characterized by diarrhea, malabsorption, and acid/base instability, requiring intensive parenteral support for nutritional and fluid management. Currently, there are no approved drug treatments for MVID.
Some SBS patients are subject to intestinal failure, often requiring parenteral nutrition (PN) up to 7 days a week. Intestinal failure is associated with significant morbidity and mortality, as well as high medical expenses associated with PN. SBS patients with intestinal failure also experience severe chronic diarrhea and associated sequelae, including significant dehydration, metabolic acidosis or alkalosis, and malnutrition, which can become life-threatening.
Scientific Showcase and Recognition
Ladenburg Thalmann hosted a Scientific Showcase webinar on December 18, 2024, focusing on Jaguar Health's clinical development of crofelemer. The event highlighted the five ongoing clinical efforts for rare disease indications. Furthermore, Massimo Radaelli, PhD, CEO of Napo Therapeutics and President of Jaguar International, was named 'Best Pharmaceuticals Innovator of the Year - Europe' by The European.
Lisa Conte, Jaguar's president and CEO, stated, "This award is well deserved, reflecting Massimo's lifetime of dedication and pioneering vision in the industry... additional welcome recognition of ongoing efforts by Massimo and Napo Therapeutics to expand access to crofelemer in Europe for orphan and/or rare diseases, initially for MVID and SBS-IF."
