Jaguar Health Announces First Patient Dosed in Short Bowel Syndrome with Intestinal Failure in Investigator-Initiated Trial of Crofelemer; Proof-of-Concept Data Potentially Available H1 2025

Rhea-AI Summary

Jaguar Health (NASDAQ:JAGX) has announced the dosing of the first short bowel syndrome with intestinal failure (SBS-IF) patient in an investigator-initiated pediatric proof-of-concept trial of crofelemer, their plant-based antidiarrheal prescription drug. The trial is being conducted at Sheikh Khalifa Medical City in Abu Dhabi.

This follows the dosing of the first microvillus inclusion disease (MVID) patient two weeks ago in the same trial. The study is part of five clinical efforts for crofelemer in rare diseases, including three proof-of-concept studies and two Phase 2 studies across the US, EU, and MENA regions.

Proof-of-concept data, potentially available in H1 2025, could lead to reimbursed early patient access in certain European countries. Both SBS-IF and MVID are rare diseases requiring intensive parenteral nutrition. SBS affects approximately 10,000-20,000 people in both the US and Europe, while MVID has an estimated global prevalence of a few hundred patients.

Positive

• FDA and European Medicines Agency granted Orphan Drug Designation for both SBS-IF and MVID indications
• Multiple clinical trials ongoing (5 total) across different regions
• Potential for reimbursed early patient access in European countries by H1 2025

Negative

• Small target market with MVID affecting only hundreds of patients globally
• Results from proof-of-concept trials not expected until H1 2025

Insights

Clinical Research Analyst

The initiation of patient dosing in this investigator-initiated trial (IIT) represents a strategic acceleration in Jaguar Health's rare disease program. This trial is particularly noteworthy for several reasons:

• The study's location in the MENA region at Sheikh Khalifa Medical City is strategically valuable due to the higher prevalence of autosomal recessive disorders in this population, potentially accelerating patient recruitment
• The potential for early reimbursed access in European markets based on proof-of-concept data could provide an expedited path to commercialization, significantly different from traditional approval pathways
• The company's multi-pronged approach with five concurrent clinical programs (3 POC IITs and 2 Phase 2 studies) demonstrates a comprehensive development strategy that could provide multiple shots on goal

The market opportunity is substantial given the severe unmet need in both indications. For SBS-IF, the U.S. and European prevalence of 10,000-20,000 patients each, combined with the pediatric SBS incidence of up to 43,000 cases, represents a significant commercial opportunity. The MVID population, though smaller at several hundred patients globally, typically requires intensive, costly care throughout life.

The dual orphan drug designations from both FDA and EMA provide regulatory benefits and market exclusivity, enhancing the commercial potential. The potential H1 2025 data readout could be a important catalyst, particularly if positive results lead to early access programs in Europe, which could generate revenue streams ahead of traditional regulatory approvals.

Crofelemer's plant-based nature and novel mechanism of action in treating diarrhea could provide advantages in these indications where chronic diarrhea management is important for patient outcomes. The absence of approved drug treatments for MVID particularly highlights the potential first-mover advantage in this ultra-rare indication.

Trial taking place at Sheikh Khalifa Medical City, a flagship United Arab Emirates hospital and largest teaching medical center in Abu Dhabi

Crofelemer, Jaguar's novel plant-based prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines Agency for both SBS-IF and MVID

Proof-of-concept (POC) data generated from this study could potentially lead to reimbursed early patient access in certain European countries for crofelemer for these rare/orphan diseases

SAN FRANCISCO, CA / ACCESS Newswire / February 3, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) 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.¹ Based on an estimate of 3.6 million live births in the U.S. in 2023,² 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.

About Crofelemer

Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as "dragon's blood," of the Croton lechleri tree in the Amazon Rainforest. Napo has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities.

About the Jaguar Health Family of Companies

Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo's crofelemer is FDA-approved under the brand name Mytesi^® for the symptomatic relief of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan and/or rare diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications.

For more information about:

Jaguar Health, visit https://jaguar.health

Napo Pharmaceuticals, visit www.napopharma.com

Napo Therapeutics, visit napotherapeutics.com

Magdalena Biosciences, visit magdalenabiosciences.com

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Forward-Looking Statements

Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that another IIT and another Phase 2 study of crofelemer will initiate in early Q1 2025, Jaguar's expectation that POC data from the IIT in Abu Dhabi may be available in H1 2025, Jaguar's expectation that additional POC IIT results for crofelemer may be available throughout 2025, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations could support early patient access to crofelemer for SBS-IF or MVID in these countries. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to several risks, uncertainties, and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

¹ Beth A. Carter, MD, et al. Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome. The Journal of Pediatrics, 2017; 181:102-11

² https://www.ncbi.nlm.nih.gov/books/NBK607756/

Contact:

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SOURCE: Jaguar Health, Inc.

View the original press release on ACCESS Newswire

FAQ

When will Jaguar Health (JAGX) release proof-of-concept data for crofelemer in SBS-IF and MVID trials?

The first proof-of-concept data is expected to be available in the first half of 2025, with additional results throughout 2025.

How many patients are affected by SBS that JAGX's crofelemer could potentially treat?

SBS affects approximately 10,000 to 20,000 people in the U.S., with a similar patient population in Europe. For pediatric SBS, the maximal incidence is estimated at approximately 43,000 based on 2023 U.S. birth rates.

What regulatory designations has JAGX's crofelemer received for SBS-IF and MVID?

Crofelemer has received Orphan Drug Designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency for SBS and MVID.

How many clinical trials is JAGX conducting for crofelemer in rare diseases?

Jaguar Health is conducting five clinical efforts: three proof-of-concept investigator-initiated trials and two Phase 2 studies across the United States, European Union, and Middle East and North Africa regions.