Proof-of-Concept Data for Rare Disease Indications MVID and SBS-IF for Jaguar Health's Crofelemer to be Presented at April 2025 ELITE PED-GI Congress
Rhea-AI Summary
Jaguar Health (NASDAQ:JAGX) announced that preliminary results from an ongoing pediatric trial of crofelemer, their plant-based anti-secretory drug, will be presented at the April 2025 ELITE PED-GI Congress in Abu Dhabi. The study focuses on treating microvillus inclusion disease (MVID) and short bowel syndrome with intestinal failure (SBS-IF).
Crofelemer has received Orphan Drug Designation from both FDA and EMA for these conditions. The proof-of-concept study evaluates crofelemer's potential to improve stool formation, enhance nutrient absorption, and reduce total parenteral nutrition (TPN) needs in patients. Currently, there are no approved drug treatments for MVID, an ultrarare pediatric disease characterized by severe diarrhea.
Positive results could potentially qualify crofelemer for the EMA's PRIME program and FDA's Breakthrough Therapies program, expediting development and review processes. The study is being conducted at Sheikh Khalifa Medical City under Dr. Mohamad Miqdady's leadership.
Positive
- FDA and EMA Orphan Drug Designation secured for both MVID and SBS-IF
- Potential for expedited regulatory pathways through PRIME and Breakthrough Therapy programs
- First-mover advantage in MVID treatment where no approved drugs exist
- Multiple ongoing clinical investigations across US, EU, and MENA regions
Negative
- Early-stage proof-of-concept data - no confirmed efficacy yet
- Success depends on showing benefits in very small patient population
- Requires extensive clinical validation across multiple regions
Insights
Jaguar Health's crofelemer data presentation for rare diseases represents a potential regulatory milestone, though real impact depends on forthcoming clinical results.
Jaguar Health's announcement regarding the upcoming presentation of preliminary proof-of-concept data for crofelemer in ultra-rare pediatric conditions represents a modest but meaningful step in their clinical development program. The drug, which has secured Orphan Drug Designation from both the FDA and EMA for microvillus inclusion disease (MVID) and short bowel syndrome (SBS), is being evaluated in an investigator-initiated trial using a novel liquid formulation.
The significance lies primarily in addressing conditions with no FDA-approved treatments, particularly MVID. Patients with these disorders currently rely on total parenteral nutrition (TPN) for up to 20 hours daily, facing serious complications including infections and liver problems. Any therapy reducing TPN dependence would address a critical unmet need.
What makes this development potentially valuable is the regulatory pathway. Even modest efficacy in a small number of patients could potentially qualify crofelemer for the EMA's PRIME program and FDA's Breakthrough Therapy designation, expediting development and review. This accelerated pathway could substantially reduce time-to-market for these indications.
However, investors should recognize this represents early-stage data from a non-pivotal, investigator-initiated study. The presentation at the upcoming ELITE PED-GI Congress will provide preliminary insights, but comprehensive validation would require company-sponsored trials with larger patient populations.
For Jaguar's broader pipeline strategy, these indications align with their focus on gastrointestinal disorders and could provide multiple shots on goal with the same compound. The company is supporting additional clinical investigations with results expected throughout 2025, potentially creating a series of data-driven catalysts.
Crofelemer's development for MVID addresses a devastating condition with no approved treatments, though clinical proof-of-concept data remains preliminary.
The investigation of crofelemer for congenital diarrheal disorders addresses an area of profound unmet medical need. MVID in particular represents one of the most challenging pediatric gastrointestinal conditions, characterized by severe secretory diarrhea from birth, resulting in life-threatening dehydration and malnutrition.
The current standard of care—prolonged daily TPN—comes with serious complications including catheter-related bloodstream infections, liver disease, and metabolic disorders. These complications significantly impact quality of life and long-term survival, with many patients ultimately requiring intestinal transplantation.
Crofelemer's mechanism as an anti-secretory agent that inhibits both the CFTR chloride channel and calcium-activated chloride channel makes it theoretically well-suited for conditions characterized by excessive chloride secretion driving water loss. The novel liquid formulation being tested is particularly appropriate for the pediatric population.
A key endpoint in this study examining whether benefits persist after treatment discontinuation will help determine if crofelemer provides symptomatic relief or potentially modifies disease pathophysiology. For orphan diseases, demonstrating even modest improvements in clinical parameters can be sufficient for regulatory consideration.
The investigator-initiated nature of this trial under Dr. Miqdady, a respected pediatric gastroenterologist, provides external validation of the scientific rationale. However, these early results should be viewed cautiously, as proof-of-concept data in rare diseases often requires confirmation in larger trials.
The potential for reimbursed early access in certain EU countries based on published clinical investigation data could accelerate availability to patients while full-scale trials proceed—a critical consideration for life-threatening conditions with no approved therapies.
Crofelemer, a novel plant-based anti-secretory prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines Agency (EMA) for both short bowel syndrome (SBS) and microvillus inclusion disease (MVID) and is being evaluated to serve as a potential therapeutic option to manage intestinal failure in these patients
There are currently no approved drug treatments for MVID, an ultrarare pediatric disease characterized by severe diarrhea and malabsorption that requires intensive parenteral support for nutritional and fluid management
Proof-of-concept data in MVID from this study would provide support for crofelemer's potential inclusion in the EMA's PRIME program for novel medicines targeting unmet medical needs and the FDA's Breakthrough Therapies program
SAN FRANCISCO, CA / ACCESS Newswire / April 17, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics today announced that preliminary results from the ongoing pediatric investigator-initiated trial (IIT) of a novel liquid formulation of crofelemer, Jaguar's novel plant-based anti-secretory prescription drug, for various congenital diarrheal disorders (CDD), including MVID and SBS with intestinal failure (SBS-IF), will be presented by Dr. Mohamad Miqdady at the April 24-26, 2025 Annual ELITE PED-GI Congress in Abu Dhabi in the United Arab Emirates.
MVID and SBS-IF, rare orphan 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. There are currently no approved drug treatments for MVID. A key value driver would be a reduction in total parenteral nutrition (TPN).
"Patients with MVID and SBS-IF suffer from devastating diarrhea and dehydration caused by these debilitating, lifelong conditions. These patients are frequently on TPN for as long as 20 hours a day, seven days a week - and TPN carries a significant risk of morbidity, including infections, metabolic complications, and liver problems," said Dr. Miqdady, the principal investigator for this study. "I look forward to presenting preliminary, proof-of-concept results from this study at the ELITE PED-GI Congress."
This ongoing proof-of-concept study is evaluating crofelemer's potential to be safely administered to patients with MVID, SBS-IF, and other CDDs in escalating doses, improve stool formation, improve the absorption of nutrients, and lower TPN needs - which have never been achieved in an MVID patient. The study also provides the opportunity to evaluate whether any potential benefit provided by crofelemer in participating patients ceases after treatment with crofelemer is discontinued.
Napo expects that if even just a very small number of MVID patients show benefit with crofelemer, this may potentially allow pathways for regulatory approval in the US and other regions and qualify crofelemer for participation in PRIME, a European Medicines Agency (EMA) program providing enhanced interaction and early dialogue with drug developers of novel medicines targeting unmet medical needs; early patient access in certain EU countries; and in the U.S. Food and Drug Administration's (FDA) Breakthrough Therapies program. If a drug is designated as breakthrough therapy, the FDA will expedite the development and review of the drug.
This study is being conducted at Sheikh Khalifa Medical City (SKMC), a flagship tertiary hospital in the United Arab Emirates and the largest teaching medical center in Abu Dhabi, by Dr. Mohamad Miqdady, a recognized leader in pediatric gastroenterology who is SKMC's Division Chief of the Pediatric Gastroenterology, Hepatology & Nutrition Division.
Jaguar is supporting multiple clinical investigations, including additional IITs, for crofelemer together with additional clinical studies in adult SBS-IF and pediatric MVID patients in the US, European Union, and/or Middle East/North Africa regions. POC results from IITs are expected throughout 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs. 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.
Dr. Miqdady is an American board-certified pediatric GI, hepatology and nutrition professor at Khalifa University 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.
The Annual ELITE PED-GI Congress is designed to provide information about high level, clinically significant updates and comprehensive trends relevant to the practice of pediatric gastroenterological, nutrition and liver disorders. Napo is a Bronze-level sponsor of the 2025 Annual ELITE PED-GI Congress.
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. 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 POC data for MVID and SBS-IF for crofelemer will be presented at the April 2025 Annual ELITE PED-GI Congress, the expectation that a key value driver would be a reduction in TPN in MVID and SBS-IF patients, the expectation that crofelemer's mechanism of action may have the potential to serve as a therapeutic option to manage intestinal failure in patients with MVID and SBS-IF and improve the quality of life of these patients receiving chronic TPN, Jaguar's expectation that, if even just a very small number of MVID patients show benefit with crofelemer, this may potentially allow pathways for regulatory approval in the US and other regions and qualify crofelemer for participation in PRIME, early patient access in certain EU countries, and in the FDA's Breakthrough Therapies program for MVID, Jaguar's expectation that POC results from IITs will be available throughout 2025, and Jaguar's expectation that, 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. 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.
Contact:
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SOURCE: Jaguar Health, Inc.
View the original press release on ACCESS Newswire
FAQ
What are the potential benefits of Jaguar Health's crofelemer for MVID and SBS-IF patients?
When will JAGX present the proof-of-concept data for crofelemer?
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