Jaguar Health, Inc. is taking a bold step forward in its mission to make a difference in the rare and orphan disease space. Through its Italian subsidiary, Napo Therapeutics, the company is focusing on Short Bowel Syndrome (SBS), Microvillus Inclusion Disease (MVID), and Cholera, all of which are incredibly rare and devastating conditions. As the majority shareholder of Napo Therapeutics, Jaguar is committed to finding treatments that can improve the lives of those affected by these conditions.
Microvillus Inclusion Disease (MVID)
Crofelemer, an oral botanical drug, is set to be submitted in a Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the first half of 2023 in hopes of treating MVID, an ultra-rare congenital diarrheal disorder (CDD) condition. This life-threatening condition affects newborns and children, causing severe secretory diarrhea and potential secondary symptoms including dehydration, metabolic acidosis or alkalosis, and malnutrition, which may become life-threatening. MVID is a subset of the group of inherited chronic enteropathies known as CDDs, which are characterized by heterogeneous etiology and the common symptom of severe chronic diarrhea. With the potential approval of crofelemer, this debilitating condition may soon find relief.
The U.S. government is providing a helping hand to the development of drugs for rare diseases; this is evidenced by the Orphan Drug Act of 1983. In response, the Company has submitted an Orphan Drug Designation (ODD) application to the FDA for MVID, and is awaiting a response. If the application is accepted, then the Company will be eligible for various development incentives, such as tax credits for qualified clinical testing and filing fee relief. The European Medicines Agency (EMA) also granted ODD for crofelemer in October 2022 for MVID in the European Union.
At the 2022 Digestive Disease Week® (DDW) conference, a clinical collaborator of the Company presented exciting findings that suggest crofelemer may be a potential therapy to reduce chloride ion secretion in patients with MVID. The in vitro study highlighted the clinical potential of crofelemer as a potential symptomatic therapy, and/or treatment for MVID. This is a significant step forward in the effort to find beneficial treatments for this condition.
Short Bowel Syndrome
Crofelemer has been granted orphan drug designations (ODD) by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of Short Bowel Syndrome (SBS). SBS is a complex condition which is characterized by persistent diarrhoea and/or malabsorption of fluids and nutrients due to surgical resection of bowel segments, congenital anomalies, or diseases. The condition can be so severe that it requires parenteral support (PS) in the form of parenteral nutrition (PN) and/or intravenous (IV) fluids. To evaluate its safety and effectiveness, investigator-initiated clinical trials are planned to take place in 2023 to assess the efficacy of crofelemer in reducing PS in patients with SBS and intestinal failure (SBS-IF).
SBS-IF is a debilitating condition that affects around 10,000-20,000 Americans and an estimated equal number of Europeans. People suffering from this condition must endure the significant loss of their bowel, which necessitates the use of PN and/or IV fluids to maintain nutrient, electrolyte and fluid balance. This puts a huge limitation on their ability to carry out basic ADL such as attending school or work, and drastically affects their QOL. Moreover, the constant use of PS can lead to potentially life-threatening conditions such as sepsis and organ failure.
Cholera in the United States
The FDA recently activated the IND application of NP-300, a novel botanical drug product candidate, for the relief and treatment of moderate-to-severe diarrhea, with or without concomitant antimicrobial therapy, from bacterial, viral and parasitic infections including Vibrio cholerae. Cholera is an orphan indication in the United States, and due to its low incidence and prevalence, the Company is seeking orphan drug designation (ODD) for NP-300 in the U.S. to help bring symptomatic relief and a potential treatment to those affected.
Upon successful completion of the process to obtain approval of NP-300 by the FDA for the treatment of diarrhea caused by cholera, we intend to pursue a Tropical Disease Priority Review Voucher under the FDA’s incentive program. This voucher is transferable and has been known to sell for anywhere between $67 million and $350 million, offering a potentially lucrative return on investment for the approval of NP-300 for cholera-related diarrhea.
Jaguar is on the cusp of two game-changing clinical events that could revolutionize the company and open up a world of new value. Our highest priority is the Phase 3 OnTarget trial of crofelemer for the prophylaxis of cancer therapy-related diarrhea (CTD), with enrollment completion anticipated in the second quarter of 2023. Additionally, we are launching an investigator-initiated proof-of-concept trial of crofelemer for short bowel syndrome (SBS) and chronic diarrhea (CDD). These two events promise to be incredibly impactful and we are incredibly excited to see what the future holds.
Crofelemer, a plant-based drug derived from the red bark sap of the Croton lechleri tree in the Amazon Rainforest, is a true example of sustainability in action. Harvested through fair trade practices and approved by the FDA’s Botanical Guidance as one of only two botanical drug products, crofelemer is not only a safe and effective treatment but also a means of empowering indigenous communities.
NP-300 is an exciting new oral botanical drug product, sustainably harvested from the red bark sap of the Croton lechleri tree – the same source of crofelemer. This groundbreaking product is set to be developed in line with the FDA’s Botanical Guidance, offering a revolutionary new way of treating ailments.
About Jaguar Health, Napo Pharmaceuticals, Napo Therapeutics & Jaguar Animal Health
Jaguar Health, Inc. is a commercial stage pharmaceuticals company on a mission to develop innovative, plant-based, non-opioid, and sustainably sourced prescription medicines for both people and animals suffering from GI distress. Its wholly owned subsidiary, Napo Pharmaceuticals, Inc., is currently engaged in a pivotal Phase 3 clinical trial for the prevention of diarrhea in adult cancer patients receiving targeted therapy. Jaguar Health is also the majority shareholder of Napo Therapeutics S.p.A., an Italian corporation established in 2021 that works to expand crofelemer access throughout Europe. This is all part of Jaguar Health’s aim to provide a more natural, safe, and effective way to treat GI distress.