Jaguar Health Begins Phase 2 Study of Crofelemer for Ultrarare MVID

Jaguar Health, Inc. has announced the commencement of a Phase 2 clinical trial to evaluate the effectiveness of crofelemer, a plant-based prescription drug, in treating microvillus inclusion disease (MVID) in children. This study marks a significant step in Jaguar's efforts to address rare diseases, as it is one of five clinical trials involving crofelemer for the rare conditions of MVID and short bowel syndrome with intestinal failure (SBS-IF) in various regions, including the United States, European Union, and Middle East/North Africa.

Founded by Lisa Conte, Jaguar Health is committed to addressing the needs of those with rare gastrointestinal diseases. The new study employs a double-blind, placebo-controlled approach to assess the efficacy of crofelemer in pediatric MVID patients. This new oral powder formulation of crofelemer differs from the FDA-approved delayed-release tablets previously used for alleviating diarrhea in adults with HIV/AIDS.

Regulatory bodies, including the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have approved the clinical protocol for the Phase 2 study, which will take place in the US, Europe, and the MENA region. The initial patient dosing is anticipated to begin in late 2024 and early 2025, with early proof-of-concept results expected by the first quarter of 2025.

MVID and SBS-IF are severe conditions that necessitate extensive parenteral nutrition and support, significantly affecting the quality of life for both patients and their families. Jaguar Health's research aims not only to determine clinical outcomes but also to evaluate the impact on the quality of life for these patients and their caregivers.

MVID is a particularly rare pediatric condition characterized by chronic diarrhea and nutrient absorption issues, affecting only a small number of patients globally. The management of this disease requires meticulous attention to nutritional and fluid needs, with no approved treatments currently available. Jaguar Health posits that if crofelemer demonstrates even modest benefits, it could pave the way for regulatory approval in the U.S., early patient access in certain European countries, and participation in the EMA's PRIME program. This initiative seeks to facilitate early patient access to medicines addressing unmet medical needs by optimizing development plans and expediting evaluations.

Crofelemer has been recognized as an Orphan-Drug by both the FDA and EMA for SBS-IF and MVID, reflecting its potential significance in treating these rare diseases. Derived from the sap of the Croton lechleri tree, commonly known as "dragon's blood," crofelemer is developed through sustainable and fair trade practices that support ecological balance and local communities.

Jaguar Health, alongside its subsidiary Napo Pharmaceuticals, is dedicated to creating innovative treatments from plant sources in rainforest areas for gastrointestinal conditions. Napo Pharmaceuticals focuses on human prescription medicines for managing neglected gastrointestinal symptoms, while Jaguar's Italian subsidiary, Napo Therapeutics, is expanding crofelemer's availability in Europe for rare diseases.

Overall, Jaguar Health's ongoing trials represent a hopeful advancement in the treatment of MVID and SBS-IF, offering potential relief for patients facing these challenging conditions.