Jaguar Health provides update on Rare, Orphan disease indications

Jaguar Health provided updates regarding the Company’s focus on rare and orphan disease indications: short bowel syndrome, microvillus inclusion disease, and cholera. Rare disease indications are the sole focus of Napo Therapeutics, the corporation established by Jaguar in Italy in 2021, with an initial focus on SBS. Jaguar is the majority shareholder of Napo Therapeutics. The Company intends to submit an Investigational New Drug application to the U.S. Food and Drug Administration in the first half of 2023 for crofelemer, the Company’s oral botanical drug, for the indication of MVID, an ultra-rare congenital diarrheal disorder condition. MVID is a life-threatening autosomal recessive disease that affects newborns and children and leads to significant morbidity and even death from severe secretory diarrhea. MVID is a subset of various CDDs, which are a group of inherited chronic enteropathies characterized by heterogeneous etiology, and each type of CDD is thus a different disease with a different pathogenetic mechanism. CDDs share a primary common symptom of severe chronic diarrhea, which may produce secondary symptoms, including significant dehydration, metabolic acidosis or alkalosis and malnutrition, which may become life-threatening. As previously announced, the Company has submitted an Orphan Drug Designation application to the FDA for MVID and is awaiting a response on the application. The Orphan Drug Act of 1983, a law passed in the U.S. to facilitate development of drugs for rare diseases, provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. ODD qualifies the sponsor of the drug for various development incentives, including tax credits for qualified clinical testing and relief of filing fees. The European Medicines Agency granted ODD for crofelemer in October 2022 for MVID in the European Union. Crofelemer has received ODD from both the FDA and the EMA for SBS, which is a complex condition characterized by diarrhea and/or severe malabsorption of fluids and nutrients due to surgical resection of bowel segments, congenital anomalies, or disease-associated loss of absorption; any or all of which may require parenteral support in the form of parenteral nutrition and/or intravenous fluids. Investigator-initiated proof-of-concept clinical studies of the safety and effectiveness of crofelemer for reduction of PS in SBS patients with intestinal failure are expected to be conducted throughout 2023. Cholera, an acute diarrheal illness caused by infection of the intestine with the bacterium Vibrio cholerae, is an orphan indication in the United States. As announced in September 2022, the FDA has activated the Company’s Investigational New Drug application for NP-300, a novel botanical drug product candidate for the symptomatic relief and treatment of moderate-to-severe diarrhea, with or without concomitant antimicrobial therapy, from bacterial, viral and parasitic infections including Vibrio cholerae. Due to the very low incidence and prevalence of cholera in the United States, the Company plans to request orphan drug designation for NP-300 in the U.S. Upon completion of the requisite development activities to support the New Drug Application and subsequent approval of NP-300 by the FDA for the symptomatic relief and treatment of diarrhea from cholera, the Company intends to pursue a Tropical Disease Priority Review Voucher under the FDA’s financial incentive program to develop NP-300 for this indication. Priority review vouchers are transferable, and in past transactions by other companies have sold for values ranging from $67M to $350M, which provides for a potential immediate return on investment upon approval of NP-300 for the cholera-related diarrhea indication.