Jaguar Health Advances Crofelemer Trials for Rare Diarrheal Diseases
Key Insights
Jaguar Health has initiated multiple clinical trials to evaluate crofelemer for treating rare diarrheal diseases, including Short Bowel Syndrome with Intestinal Failure (search) (SBS-IF) and Microvillus Inclusion Disease (search) (MVID).
Crofelemer, a plant-based drug, has received Orphan Drug Designation from the FDA and EMA for both SBS-IF and MVID, potentially expediting its development and market access.
Clinical trials include investigator-initiated studies and Phase 2 trials across the US, EU, and MENA regions, with initial proof-of-concept results expected as early as H1 2025.
Jaguar Health, along with its family companies Napo Pharmaceuticals and Napo Therapeutics, is actively pursuing the development of crofelemer, a novel plant-based prescription drug, for the treatment of rare diarrheal diseases. The company has initiated several clinical trials, spanning multiple regions, to evaluate the efficacy and safety of crofelemer in patients with Short Bowel Syndrome with Intestinal Failure (search) (SBS-IF) and Microvillus Inclusion Disease (search) (MVID).
Clinical Trial Overview
Jaguar Health is undertaking five clinical efforts, including three proof-of-concept investigator-initiated trials (IITs) and two Phase 2 studies. These trials are being conducted across the United States, the European Union, and the Middle East/North Africa (MENA) regions. The studies are evaluating a novel powder formulation of crofelemer for oral solution, distinct from the FDA-approved oral formulation (Mytesi®) used for HIV/AIDS-related diarrhea.
Lisa Conte, Jaguar's founder, president, and CEO, stated, "This study is one of five clinical efforts - three IIT proof-of-concept studies and two Phase 2 studies - of crofelemer for the rare disease indications of SBS-IF and/or MVID in the US, European Union, and/or MENA regions."
Focus on Rare Diseases
SBS-IF and MVID are rare and severe diseases that require intensive parenteral nutrition and support. SBS affects approximately 10,000 to 20,000 people in the U.S., with a similar estimate for Europe, according to the Crohn's & Colitis Foundation. MVID is an ultrarare pediatric disease, affecting only a couple of hundred patients globally. Both conditions significantly impact the quality of life of patients and their caregivers, with high morbidity and mortality rates.
Regulatory and Strategic Advantages
Crofelemer has been granted Orphan Drug Designation by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for SBS-IF and MVID. This designation provides several benefits, including market exclusivity and potential tax credits. Furthermore, positive data from these trials could support reimbursed early patient access to crofelemer in specific EU countries, according to their guidelines.
Trial Locations and Key Investigators
One of the IITs is taking place at Sheikh Khalifa Medical City (SKMC) in Abu Dhabi, UAE, under the direction of Dr. Mohamad Miqdady, a leading pediatric gastroenterologist. Dr. Miqdady is also an Adjunct Professor at Khalifa University’s medical school and a member of Napo's Scientific Advisory Board.
Expected Milestones
Dosing of the first patient in each of these five studies is expected to occur throughout December 2024 and Q1 2025, with availability of IIT proof-of-concept results potentially in Q2 2025. The company anticipates that clinical investigation data could support early patient access to crofelemer in certain EU countries.
About Crofelemer
Crofelemer is derived from the red bark sap of the Croton lechleri tree in the Amazon Rainforest. Napo Pharmaceuticals has established a sustainable harvesting program to ensure quality, ecological integrity, and support for indigenous communities.