Jaguar Health family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics today announced that the first short bowel syndrome with intestinal failure (SBS-IF) patient has been dosed in the independent investigator-initiated pediatric POC trial (IIT) of crofelemer, Jaguar’s novel plant-based antidiarrheal prescription drug. The first infant with microvillus inclusion disease (MVID) was dosed as part of the same IIT two weeks ago. POC data generated from this study potentially in the first half of 2025 could lead to reimbursed early patient access to crofelemer for these indications in certain European countries.
The trial is being conducted by Dr. Mohamad Miqdady, a recognized leader in pediatric gastroenterology who is the Division Chief of the Pediatric Gastroenterology, Hepatology & Nutrition Division at Sheikh Khalifa Medical City (SKMC), a flagship tertiary hospital in the UAE and the largest teaching medical center in Abu Dhabi.
“Dosing of the SBS-IF first patient in this unblinded study is another important milestone in clinical development efforts for crofelemer for SBS-IF and MVID – both of which are rare and severe diseases requiring intensive parenteral nutrition and support,” said Lisa Conte, Jaguar’s founder, president, and CEO.
“This study is one of five clinical efforts in rare diseases – three POC IIT studies and two Phase 2 studies – for crofelemer for the orphan disease indications of MVID and/or SBS-IF in the United States, European Union, and/or Middle East and North Africa (MENA) regions,” Conte said. “The Company’s Phase 2 study to evaluate the efficacy of crofelemer for MVID in pediatric patients has been initiated, as has the independent IIT in the U.S. to evaluate crofelemer for SBS-IF in adults. The two additional studies are expected to initiate in early Q1 2025, with the availability of the first POC IIT result potentially in H1 2025, and with additional POC IIT results expected throughout 2025. In accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID and SBS-IF could support reimbursed early patient access to crofelemer for these debilitating conditions.”
SBS-IF and MVID, rare and severe diseases requiring intensive parenteral nutrition and support, have severe morbidity and mortality implications and impact the quality of life of both patients and their caregivers.
SBS affects approximately 10,000 to 20,000 people in the U.S., according to the Crohn’s & Colitis Foundation, and it is estimated that the population of SBS patients in Europe is approximately the same size. Pediatric SBS is a malabsorptive condition caused by surgical intestinal resection due to congenital abnormalities, vascular insufficiency or severe inflammatory intestinal disease. The incidence of pediatric SBS varies between 0.02% to 1.2% of live births. Based on an estimate of 3.6 million live births in the U.S. in 2023, the maximal incidence of pediatric SBS is estimated to be approximately 43,000.
Pediatric SBS-IF is associated with significant morbidity and mortality; and high medical expenses, and the patients also have severe chronic diarrhea. The associated sequelae from chronic diarrhea include significant dehydration, metabolic acidosis or alkalosis, and malnutrition.
MVID is a pediatric disease with an estimated prevalence of a couple of hundred patients globally. It is characterized by severe diarrhea and malabsorption, requiring intensive parenteral support for nutritional and fluid management. Each MVID patient is a unique patient; their journey requires very careful management of their nutritional needs, and there are currently no approved drug treatments for MVID.
The trial is taking place at SKMC. Autosomal recessive disorders and congenital anomalies such as MVID are more prevalent in the MENA region.
Crofelemer has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency for SBS and MVID.
Dr. Miqdady is an Adjunct Professor at Khalifa University’s medical school in Abu Dhabi, and serves as a member of Napo’s Scientific Advisory Board. He completed his Fellowship in Pediatric Gastroenterology at Baylor College of Medicine and Texas Children’s Hospital in Houston.
