Additional Infant Patient to Receive Jaguar Health's Crofelemer for Compassionate Use for Treatment of Intestinal Failure (IF)

Jaguar advances its rare pediatric intestinal failure program, adding a new compassionate-use infant case and targeting a mid-2027 NDA for MVID.

Rhea-AI Summary

Jaguar Health (JAGX) announced that affiliate Napo Pharmaceuticals is providing its novel liquid oral crofelemer formulation for compassionate use to an infant with intestinal failure (IF) due to congenital sodium diarrhea caused by a GUCY2C mutation.

The company remains focused on developing crofelemer as adjunctive therapy to parenteral support (PS) for pediatric IF diseases, including microvillus inclusion disease (MVID) and short bowel syndrome with IF (SBS-IF). Ongoing pediatric studies of liquid oral crofelemer have shown reductions of up to 48% and 40% in weekly PS requirements normalized to body weight in MVID and SBS-IF patients, respectively, after more than 12 months of high-dose therapy, with no significant clinical or laboratory abnormalities reported.

Jaguar has completed the randomized double-blind treatment phase of its pivotal trial in pediatric MVID and plans to file an NDA for crofelemer for MVID in mid-2027, and may seek Breakthrough Therapy Designation based on the trial data.

Positive

• Pediatric IF PS reduction data: up to 48% PS decrease in MVID and 40% in SBS-IF patients after >12 months of liquid oral crofelemer, with no significant clinical or laboratory abnormalities reported.
• Pivotal MVID trial milestone: completion of the randomized double-blind treatment phase in pediatric MVID supports plans for potential Breakthrough Therapy Designation and a mid-2027 NDA filing.
• Expanded clinical experience: additional infant with IF due to congenital sodium diarrhea now receiving crofelemer under compassionate use, broadening real-world exposure in ultrarare pediatric IF.
• Non-dilutive funding potential: the IF program is part of business development discussions that the company says may bring in non-dilutive funds from potential licensee partners.

Negative

• None.

News Explained

Jaguar’s affiliate Napo is providing oral crofelemer to an infant with intestinal failure caused by congenital sodium diarrhea; this adds compassionate-use treatment access but does not change the drug’s stated development or approval status.

Market Context

The August 19 earnings report was followed by a 5.5% decline, a historical comparator placing this c...

Analysis

The August 19 earnings report was followed by a 5.5% decline, a historical comparator placing this compassionate-use update alongside mixed company-specific news. Subsequent clinical milestones merit attention while the active S-3 shelf remains a financing risk.

Key Figures

MVID PS reduction: up to 48% SBS-IF PS reduction: up to 40% Treatment duration: >12 months +4 more

7 metrics

MVID PS reduction up to 48% one MVID patient after >12 months of liquid oral high-dose crofelemer

SBS-IF PS reduction up to 40% two pediatric SBS-IF patients after >12 months of therapy

Treatment duration >12 months selected pediatric IF patients

Parenteral support frequency up to seven days per week IF patients requiring lifelong PS

Daily PS duration up to 20+ hours per day IF patients requiring lifelong PS

Worldwide prevalence 200 patients estimated worldwide MVID prevalence

Planned NDA filing mid-2027 crofelemer for MVID

Historical Context

5 past events · Latest: Aug 26 (Positive)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Aug 26	Commercial launch	Positive	-0.6%	Neonorm Dog commercial launch was followed by a 0.59% decline.
Aug 19	Quarterly earnings	Negative	-5.5%	Second-quarter results included revenue declines and a larger net loss.
Aug 18	Earnings webcast notice	Neutral	+11.1%	The company scheduled a webcast to discuss second-quarter financial results.
Aug 5	Holdback payment	Positive	+5.5%	Jaguar satisfied conditions for a non-dilutive $2 million holdback payment.
Jul 28	Market research	Positive	-3.5%	Exploratory research supported Neonorm Dog’s commercial opportunity.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Recent company announcements showed mixed reactions: positive updates produced both gains and declines, while the earnings report coincided with a decline.

Key Terms

microvillus inclusion disease, short bowel syndrome, congenital sodium diarrhea, parenteral support, +2 more

6 terms

microvillus inclusion disease medical

"pediatric rare IF diseases, including microvillus inclusion disease (MVID)"

A rare, inherited condition in which the tiny, brush-like hairs on the inner lining of the small intestine are absent or abnormal, causing severe, lifelong diarrhea and inability to absorb nutrients. Think of the intestine’s surface as a shaggy carpet that helps soak up food; when those fibers are missing or disorganized, patients often need long-term intravenous feeding and specialized care. For investors, the disease matters because it creates clear demand for diagnostics, nutritional support, transplant solutions, and targeted therapies in the orphan-disease market.

short bowel syndrome medical

"microvillus inclusion disease (MVID) and short bowel syndrome with IF (SBS-IF)"

A chronic digestive disorder caused when a large portion of the small intestine is missing or not working, so the body cannot absorb enough nutrients and fluids from food. Investors should care because people with this condition often need long-term medical treatments, specialized nutrition or surgeries, and ongoing devices or drugs, creating sustained demand, higher healthcare costs, and opportunities for companies that provide therapies, supplies, or related services.

congenital sodium diarrhea medical

"infant patient due to congenital sodium diarrhea (CSD)"

A rare inherited disorder present from birth in which the intestines cannot absorb sodium properly, causing chronic, watery, sodium-rich diarrhea and frequent dehydration. Think of the gut as a leaky pipe that loses salt and water instead of holding them in. It matters to investors because the condition defines a small but well-defined patient population, clinical trial design, regulatory pathways, and potential markets for drugs, diagnostics, or medical devices targeting its underlying transport defect.

parenteral support medical

"adjunctive therapy to parenteral support (PS) for pediatric rare IF diseases"

Parenteral support is medical care that delivers nutrition, fluids or medications directly into a patient’s bloodstream or body tissues, bypassing the digestive system—think of it as feeding or hydrating someone through an IV rather than by mouth. Investors care because this service involves specialized products, durable revenue from long-term patient use, strict safety and reimbursement rules, and potential regulatory or supply risks that affect company earnings and growth.

breakthrough therapy designation regulatory

"plans to apply for Breakthrough Therapy Designation for crofelemer"

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

new drug application regulatory

"planned filing of an NDA (New Drug Application) with the U.S. Food"

A new drug application is a formal request submitted to government regulators seeking approval to market a new medicine. It is like a detailed proposal that shows the drug has been tested for safety and effectiveness. For investors, receiving approval signals that the drug may soon become available for sale, potentially leading to revenue growth and impacting the company's value.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Jaguar remains sharply focused on its ongoing global development program for oral crofelemer as adjunctive therapy to parenteral support (PS) for pediatric and adult rare IF diseases, including microvillus inclusion disease (MVID)

Company plans to file NDA for crofelemer for MVID in mid-2027 to obtain FDA approval

SAN FRANCISCO, CA / ACCESS Newswire / September 4, 2026 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) today announced that Jaguar family company Napo Pharmaceuticals (Napo) is providing the company's novel crofelemer powder for oral solution for treatment of intestinal failure (IF) in an infant patient due to congenital sodium diarrhea (CSD), which is caused by a mutation in the GUCY2C gene.

Napo remains sharply focused on its ongoing global development program for oral crofelemer as adjunctive therapy to parenteral support (PS) for pediatric rare IF diseases, including microvillus inclusion disease (MVID) and short bowel syndrome with IF (SBS-IF). In June 2026, as announced, data was presented at the 58^th Annual European Society for Pediatric Gastroenterology, Hepatology & Nutrition (ESPGHAN) Meeting in Lille, France from ongoing investigational studies with oral liquid crofelemer in pediatric patients with IF due to MVID and SBS-IF. The results of ongoing investigations of liquid oral crofelemer formulations demonstrate substantial reductions in weekly PS needs as well as in PS normalized to body weight in pediatric IF patients dosed orally for more than one year, with no significant clinical or laboratory abnormalities. In one MVID patient, the weekly PS requirements normalized to body weight have been reduced by up to 48% following >12 months of liquid oral high-dose crofelemer therapy. In two pediatric SBS-IF patients, weekly PS requirements normalized to body weight were reduced up to 40% following >12 months of liquid oral high dose crofelemer therapy.

IF patients, including those with MVID or short bowel syndrome (SBS), cannot orally absorb adequate nutrients, electrolytes and fluids, and require lifelong total parenteral nutrition (TPN) with or without supplemental intravenous fluids, which together comprise parenteral support (PS), for up to seven days per week and up to 20+ hours per day. While PS is supportive, palliative and lifesaving, it is associated with serious, often fatal complications.

Patients with GUCY2C mutation and CSD have a significant challenge for managing nutritional and electrolyte deficiencies - especially pediatric patients, given the higher impact on growth and developmental status. This pediatric patient with IF due to CSD requires significant daily PS and is receiving crofelemer as adjunctive therapy to the PS to reduce their daily and weekly PS needs, as observed in pediatric IF patients with MVID and SBS.

"Napo remains committed to supporting physicians who determine that the unmet medical need for their specific pediatric IF patients could be mitigated through the use of our novel crofelemer formulation as an adjunctive oral therapy to PS," said Pravin Chaturvedi, PhD, Napo's and Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board. "We wish these physicians and their patients the best outcomes and are grateful for their collaboration. Liquid oral crofelemer as adjunctive therapy to PS has been well tolerated with no significant clinical or laboratory abnormalities throughout the ongoing treatment period of >12 months in selected pediatric IF patients with MVID and SBS-IF. In addition, the reductions in weekly PS requirements normalized to body weight with excellent tolerability support the continued development of liquid oral crofelemer as the first oral adjunctive therapy for pediatric IF patients receiving long-term and/or lifelong PS."

"We're very pleased to be able to make our novel liquid oral formulation of crofelemer available to this infant for compassionate use," said Lisa Conte, Jaguar's founder, president, and CEO. "We remain sharply focused and committed to our global IF development program for our novel liquid oral crofelemer formulation as adjunctive therapy for the management of rare IF disorders. Our IF program is expected to continue to provide clinical milestones and is the subject of business development discussions with the potential to bring in non-dilutive funds from potential licensee partners. If appropriate, Jaguar plans to apply for Breakthrough Therapy Designation for crofelemer for the indication of MVID, with a planned filing of an NDA (New Drug Application) with the U.S. Food and Drug Administration (FDA) for this indication in mid-2027. We have completed the randomized double-blind treatment phase in our pivotal clinical trial of the novel liquid oral formulation of crofelemer in pediatric MVID patients. Upon completion of the trial, data from this study may serve as the basis for a Breakthrough Therapy Designation application. Because MVID is a devastating ultrarare pediatric disorder with a lethal natural history - and an estimated worldwide prevalence of only 200 patients - a trial of crofelemer in just a small number of MVID patients is expected to be clinically meaningful and significant."

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 Pharmaceuticals 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. Jaguar family companies Napo Pharmaceuticals, Inc. (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 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 its intestinal failure program will continue to provide clinical milestones, Jaguar's expectation that the opportunity may exist to bring in non-dilutive funds from potential licensee partners to support the intestinal failure program, Jaguar's plans to pursue Breakthrough Therapy designation for crofelemer for the indication of MVID, Jaguar's expectation that the company will file an NDA with the FDA for the MVID indication in mid-2027, and Jaguar's expectation that a trial of crofelemer in a small number of MVID patients may be statistically meaningful. 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 did Jaguar Health (JAGX) announce about crofelemer and an infant intestinal failure patient?

Jaguar Health announced that its affiliate Napo Pharmaceuticals is providing a novel liquid oral formulation of crofelemer for compassionate use to an infant with intestinal failure due to congenital sodium diarrhea, to be used as adjunctive therapy to parenteral support.

What clinical results has Jaguar Health reported for crofelemer in pediatric MVID and SBS-IF?

In ongoing investigational studies, liquid oral crofelemer has reduced weekly parenteral support requirements normalized to body weight by up to 48% in one MVID patient and up to 40% in two SBS-IF patients after more than 12 months of high-dose therapy, with no significant clinical or laboratory abnormalities reported.

When does Jaguar Health plan to file an NDA for crofelemer for MVID?

Jaguar Health plans to file a New Drug Application (NDA) with the U.S. Food and Drug Administration for crofelemer for microvillus inclusion disease (MVID) in mid-2027, subject to completion and assessment of its pivotal clinical trial in pediatric MVID patients.

Is Jaguar Health seeking Breakthrough Therapy Designation for crofelemer in MVID?

The company states that, if appropriate, it plans to apply for Breakthrough Therapy Designation for crofelemer for MVID. Data from the completed randomized double-blind treatment phase of its pivotal pediatric MVID trial may serve as the basis for such an application.

What stage is Jaguar Health's pivotal trial of crofelemer in pediatric MVID?

Jaguar Health has completed the randomized double-blind treatment phase of its pivotal clinical trial evaluating the novel liquid oral formulation of crofelemer in pediatric patients with microvillus inclusion disease (MVID). The trial’s full completion is expected to support regulatory filings.

How does crofelemer aim to help pediatric intestinal failure patients receiving parenteral support?

Crofelemer is being developed as an oral adjunctive therapy to parenteral support for pediatric intestinal failure conditions such as MVID, SBS-IF, and congenital sodium diarrhea, with the goal of reducing daily and weekly parenteral support needs while maintaining tolerability.

What is crofelemer and how is Jaguar Health sourcing it?

Crofelemer is a novel oral, plant-based prescription medicine purified from the red bark sap of the Croton lechleri tree in the Amazon Rainforest. Napo Pharmaceuticals has a sustainable harvesting program under fair trade practices to support ecological integrity and indigenous communities.