Jaguar Health (JAGX) Study Result summary

Study background and objectives

• Proof-of-concept results for crofelemer in pediatric patients with microvillus inclusion disease (MVID) and short bowel syndrome (SBS/SBS-IF) were presented, targeting ultra-rare causes of intestinal failure requiring total parenteral nutrition (TPN).

• The main goal is to reduce TPN dependence, which is critical for survival but associated with severe complications and high costs.

• The study was highlighted at the Elite Pediatric Gastroenterology Congress in Abu Dhabi, attended by global experts.

• Crofelemer is a novel, oral, plant-based anti-secretory prescription drug derived from Croton lechleri.

• The study included assessment of safety, effectiveness, and optimal dosing for various congenital diarrheal disorders.

Study design

• Single-arm, open-label, non-randomized investigator-initiated trial conducted at Sheikh Khalifa Medical City in Abu Dhabi.

Key study results

• Crofelemer reduced TPN requirements by up to 27% in an MVID patient and 12.5% in an SBS/SBS-IF patient, considered groundbreaking by healthcare professionals.

• Decreased stool volume and frequency, and increased urine output, indicating improved nutrient absorption.

• Upon withdrawal of crofelemer, patients experienced worsening symptoms, which reversed upon reinitiation, supporting the drug's benefit.

• No other interventions have shown similar reductions in lifelong parenteral support for MVID.

• Indirect evidence suggests improved oral intake and potential for better cognitive development due to enhanced nutrition.