Jaguar Health Provides Recap of November 8, 2025 Presentation on Groundbreaking Results of Proof-of-Concept Study of Crofelemer for Treatment of Pediatric Intestinal Failure at NASPGHAN Annual Meeting

Rhea-AI Summary

Jaguar Health (NASDAQ:JAGX) recap of a November 8, 2025 NASPGHAN presentation reported initial proof-of-concept results for crofelemer in pediatric intestinal failure (including MVID and SBS-IF).

The independent IIT in the UAE showed reductions in total parenteral support (PS) ranging from 12% to 37%: two SBS-IF patients had PS reductions of 12.5–15.6% over 12 weeks; one MVID patient showed up to 27% reduction in 12 weeks and up to 37% during an extension after reinitiation. Findings included reduced loose watery stools and support continued evaluation and placebo-controlled trials across U.S., EU and Middle East sites.

Positive

• PS reduced up to 37% in an MVID patient during extension
• Two SBS-IF patients had PS reductions of 12.5–15.6% over 12 weeks
• Reduced loose watery stools reported alongside PS reductions
• Placebo-controlled pediatric MVID trial ongoing in U.S., EU, Middle East

Negative

• Data from a small cohort: two SBS-IF and one MVID patient completed treatment
• Study design was single-arm, open-label, non-randomized, limiting control comparisons
• Results reported are initial and from an independent IIT, not yet from larger randomized trials

Insights

Clinical development expert

Initial IIT data show clinically meaningful PS reductions in pediatric intestinal failure, supporting further controlled study.

The data describe reductions in total parenteral support (PS) of 12.5% to 15.6% in two SBS-IF patients over the 12-week treatment period, and reductions up to 27% (initial 12 weeks) and up to 37% during an extension period in the initial MVID patient; reduced frequency of loose watery stools was also reported. These results, from an investigator-initiated, open-label, single-arm trial, indicate a biological signal where oral crofelemer corresponded with lower PS needs in a very small cohort.

The key dependencies and risks are clear: the dataset is extremely limited in size and open-label, so confounding and placebo effects cannot be excluded; safety, durability beyond the reported periods, and consistency across the broader MVID and SBS-IF populations remain unproven. Controlled, randomized data and prespecified endpoints are required to confirm disease‑modifying effect and to quantify variability in response.

Concrete items to watch are topline results from the ongoing placebo-controlled trial in pediatric MVID at sites in the U.S., E.U., and Middle East, safety and PS reduction durability during follow-up, and any regulatory interactions or guidance; expect meaningful readouts or milestones over the next 6–24 months depending on trial timelines.

Crofelemer can potentially extend and save lives of microvillus inclusion disease patients, reducing the volume of the total parenteral support (PS) necessary for them to survive

Groundbreaking PS reduction of up to 37% is unprecedented; No approved treatments exist for MVID

SAN FRANCISCO, CA / ACCESS Newswire / November 13, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family company Napo Pharmaceuticals (Napo) today provided a recap of the November 8, 2025 presentation at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) Annual Meeting describing the initial groundbreaking results of an independent investigator-initiated trial (IIT) of crofelemer in the UAE for treatment of pediatric intestinal failure, which includes patients with 'intestinal failure' due to microvillus inclusion disease (MVID) and short bowel syndrome (SBS-IF).

"The intestines of patients with the ultrarare genetic disorder MVID do not function properly, as is often the case with patients having intestinal failure due to short bowel syndrome. The intestines of these patients are unable to absorb the fluids, electrolytes and nutrients required to survive and thrive. Intestinal failure is a debilitating, lifelong condition that often requires patients to receive life-sustaining fluids, electrolytes and nutrients through intravenous administration, which consists of total parenteral nutrition (TPN) with supplemental intravenous fluids, which together constitute parenteral support (PS). Most intestinal failure patients require PS up to 7 days a week, and sometimes for 20 hours or more per day," said Lisa Conte, Jaguar's founder, president, and CEO.

While crucial for these patients, total parenteral support is associated with significant toxicities to patients, similar to some toxicities associated with chemotherapy, often causing serious health problems including infections, metabolic complications, liver and kidney function problems - as well as a risk of neurodevelopmental delay. These symptoms may emerge at any time in intestinal failure patients, and often become life-threatening.

As presented at NASPGHAN, the results of this ongoing and independent proof-of-concept trial of crofelemer demonstrate disease progression modification through reduction of total parenteral support (PS) in pediatric intestinal failure patients that ranged from 12 to 37%. Specifically, in the two SBS-IF patients who have completed treatment, the results show crofelemer reduced PS between 12.5 to 15.6% at the highest dose over the 12-week treatment period, together with reduced loose watery stools frequency. For the initial MVID patient who has completed treatment, PS needs were reduced by up to 27% at the highest dose over the initial 12-week treatment period and up to 37% during the extension period upon reinitiation of crofelemer treatment, and also showed reduced frequency of loose watery stools. These findings support continued evaluation of crofelemer to reduce PS needs for pediatric intestinal failure patients.

The presentation on November 8, 2025 at NASPGHAN, was titled Exploratory Single-Arm, Open-Label Non-Randomized Trial Evaluating the Safety and Effectiveness of Crofelemer in Pediatric Patients with Intestinal Failure. The study's primary investigator, Dr. Mohamad Miqdady, Division Chief of the Pediatric Gastroenterology, Hepatology & Nutrition Division at Sheikh Khalifa Medical City (SKMC), a tertiary care center in Abu Dhabi in the UAE, described these initial findings in pediatric intestinal failure patients with crofelemer treatment.

A recognized leader in pediatric gastroenterology, Dr. Miqdady is Professor of Pediatric Gastroenterology at Khalifa University's medical school, and completed his Fellowship in Pediatric Gastroenterology at Baylor College of Medicine and Texas Children's Hospital in Houston. He is a member of Napo's Scientific Advisory Board.

MVID is a devastating ultrarare pediatric disorder, with an estimated worldwide prevalence of 100-200 patients, characterized by severe malabsorption that requires life-sustaining parenteral support to meet the nutritional, fluid and electrolyte requirements of the child, and for which there are currently no approved treatments. MVID has a lethal natural history along with significant co-morbidities. Short bowel syndrome (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.

In addition to supporting the IIT in the UAE, Napo is also conducting the placebo-controlled clinical trial of crofelemer in pediatric MVID patients at sites in the U.S., E.U., and Middle East under appropriate regulatory approvals in each of these geographies. The company is also providing crofelemer powder for oral solution for use in two expanded access programs in intestinal failure patients the U.S. to treat adult and pediatric intestinal failure patients with short bowel syndrome and MVID, respectively.

The mission of the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) is to be a world leader in research, education, clinical practice and advocacy for pediatric gastroenterology, hepatology and nutrition in health and disease. NASPGHAN strives to improve the care of infants, children and adolescents with digestive disorders by promoting advances in clinical care, research and education.

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. Jaguar family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics S.p.A. focus on the development and commercialization of novel crofelemer powder for oral solution for the treatment of rare and orphan gastrointestinal disorders with intestinal failure, including microvillus inclusion disease and short bowel syndrome.

For more information about:

Jaguar Health, visit https://jaguar.health

Napo Pharmaceuticals, visit www.napopharma.com

Napo Therapeutics, visit napotherapeutics.com

Forward-Looking Statements

Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that crofelemer can potentially extend and save lives of MVID patients, reducing the volume of the PS necessary for them to survive. 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:

hello@jaguar.health
Jaguar-JAGX

SOURCE: Jaguar Health, Inc.

View the original press release on ACCESS Newswire

FAQ

What PS reductions did Jaguar Health report for crofelemer in pediatric intestinal failure (JAGX) at NASPGHAN on November 8, 2025?

The IIT reported PS reductions ranging from 12% to 37%, including 12.5–15.6% in two SBS-IF patients and up to 27–37% in one MVID patient.

How many patients completed crofelemer treatment in the NASPGHAN IIT recap from Jaguar Health (JAGX)?

The recap describes treatment completion for two SBS-IF patients and one MVID patient in the investigator-initiated trial.

Does crofelemer have approved treatments for MVID according to Jaguar Health (JAGX) November 13, 2025 update?

No; the release states there are currently no approved treatments for MVID.

What study design was used in the crofelemer proof-of-concept trial presented by Jaguar Health (JAGX)?

The study was an exploratory single-arm, open-label, non-randomized investigator-initiated trial.

Will Jaguar Health (JAGX) run further trials of crofelemer for pediatric MVID?

Yes; Jaguar/Napo are conducting a placebo-controlled pediatric MVID trial at sites in the U.S., EU and Middle East.

What clinical benefits besides PS reduction were reported for crofelemer in the NASPGHAN presentation?

The presentation reported a reduced frequency of loose watery stools alongside PS reductions.