Proof-of-Concept Results Show Crofelemer Reduced Total Parenteral Nutrition in Patients with Rare Diseases Microvillus Inclusion Disease (MVID) and Short Bowel Syndrome with Intestinal Failure (SBS-IF) by up to 27% and 12.5% - Potential to Modify Disease Progression in Intestinal Failure Patients
Jaguar Health announced promising proof-of-concept results for their drug crofelemer in treating rare intestinal diseases. The study showed crofelemer reduced total parenteral nutrition (TPN) requirements by up to 27% in patients with Microvillus Inclusion Disease (MVID) and by 12.5% in those with Short Bowel Syndrome (SBS-IF).
The trial, presented at the Annual ELITE PED-GI Congress, demonstrated reduced stool volume output and increased urine output, indicating improved nutrient absorption. Dr. Mohamad Miqdady, Chief of Pediatric Gastroenterology at Sheikh Khalifa Medical City, led this open-label study of crofelemer's novel liquid formulation.
Notably, when a pediatric MVID patient discontinued crofelemer after 12 weeks, symptoms worsened within 8 days, leading to treatment reinstatement. The company is pursuing expedited regulatory pathways through EMA's PRIME program and FDA's Breakthrough Therapies program, given the significant unmet need in treating these rare conditions.
Jaguar Health ha annunciato risultati promettenti di proof-of-concept per il loro farmaco crofelemer nel trattamento di malattie intestinali rare. Lo studio ha evidenziato che crofelemer ha ridotto le necessità di nutrizione parenterale totale (TPN) fino al 27% nei pazienti con Microvillus Inclusion Disease (MVID) e del 12,5% in quelli con Short Bowel Syndrome (SBS-IF).
La sperimentazione, presentata al Congresso Annuale ELITE PED-GI, ha mostrato una diminuzione del volume delle feci e un aumento della produzione di urina, segno di un miglior assorbimento dei nutrienti. Il dott. Mohamad Miqdady, capo della gastroenterologia pediatrica presso lo Sheikh Khalifa Medical City, ha guidato questo studio open-label sulla nuova formulazione liquida di crofelemer.
È importante notare che, quando un paziente pediatrico con MVID ha interrotto crofelemer dopo 12 settimane, i sintomi sono peggiorati entro 8 giorni, portando alla ripresa del trattamento. L'azienda sta perseguendo vie regolatorie accelerate tramite il programma PRIME dell'EMA e il programma Breakthrough Therapies della FDA, vista la significativa esigenza insoddisfatta nel trattamento di queste rare patologie.
Jaguar Health anunció resultados prometedores de prueba de concepto para su medicamento crofelemer en el tratamiento de enfermedades intestinales raras. El estudio mostró que crofelemer redujo los requerimientos de nutrición parenteral total (TPN) hasta en un 27% en pacientes con Microvillus Inclusion Disease (MVID) y en un 12,5% en aquellos con Short Bowel Syndrome (SBS-IF).
El ensayo, presentado en el Congreso Anual ELITE PED-GI, demostró una reducción en el volumen de heces y un aumento en la producción de orina, lo que indica una mejor absorción de nutrientes. El Dr. Mohamad Miqdady, jefe de gastroenterología pediátrica en Sheikh Khalifa Medical City, lideró este estudio abierto con la nueva formulación líquida de crofelemer.
Es destacable que, cuando un paciente pediátrico con MVID suspendió crofelemer después de 12 semanas, los síntomas empeoraron en 8 días, lo que llevó a la reanudación del tratamiento. La compañía está buscando vías regulatorias aceleradas a través del programa PRIME de la EMA y el programa Breakthrough Therapies de la FDA, dado el importante déficit en el tratamiento de estas enfermedades raras.
재규어 헬스(Jaguar Health)는 희귀 장 질환 치료를 위한 약물 크로펠머(crofelemer)의 개념 증명 결과가 유망하다고 발표했습니다. 연구 결과에 따르면 크로펠머는 미세융모 포함질환(MVID) 환자의 총 비경구 영양(TPN) 필요량을 최대 27%까지, 단장 증후군(SBS-IF) 환자에서는 12.5%까지 감소시켰습니다.
연례 ELITE PED-GI 학회에서 발표된 이번 임상시험은 대변량 감소와 소변량 증가를 보여 영양소 흡수가 개선되었음을 시사합니다. 셰이크 칼리파 메디컬 시티 소아 위장병학 책임자인 모하마드 미크다디 박사가 크로펠머의 새로운 액상 제형에 대한 공개 임상시험을 이끌었습니다.
특히, 한 소아 MVID 환자가 12주 후 크로펠머를 중단했을 때 8일 만에 증상이 악화되어 치료를 재개한 점이 주목됩니다. 회사는 EMA의 PRIME 프로그램과 FDA의 Breakthrough Therapies 프로그램을 통한 신속한 규제 절차를 추진 중이며, 이러한 희귀 질환 치료에 대한 큰 미충족 수요를 고려하고 있습니다.
Jaguar Health a annoncé des résultats prometteurs de preuve de concept pour leur médicament crofelemer dans le traitement de maladies intestinales rares. L'étude a montré que crofelemer réduisait les besoins en nutrition parentérale totale (NPT) jusqu'à 27 % chez les patients atteints de Microvillus Inclusion Disease (MVID) et de 12,5 % chez ceux souffrant du Syndrome de l'intestin court (SBS-IF).
L'essai, présenté lors du Congrès annuel ELITE PED-GI, a démontré une diminution du volume des selles et une augmentation de la production d'urine, indiquant une meilleure absorption des nutriments. Le Dr Mohamad Miqdady, chef du service de gastroentérologie pédiatrique au Sheikh Khalifa Medical City, a dirigé cette étude en ouvert sur la nouvelle formulation liquide de crofelemer.
Il est notable que lorsqu'un patient pédiatrique atteint de MVID a interrompu crofelemer après 12 semaines, les symptômes se sont aggravés en 8 jours, conduisant à la reprise du traitement. L'entreprise poursuit des voies réglementaires accélérées via le programme PRIME de l'EMA et le programme Breakthrough Therapies de la FDA, compte tenu du besoin important non satisfait dans le traitement de ces maladies rares.
Jaguar Health gab vielversprechende Proof-of-Concept-Ergebnisse für ihr Medikament Crofelemer bei der Behandlung seltener Darmerkrankungen bekannt. Die Studie zeigte, dass Crofelemer den Bedarf an totaler parenteraler Ernährung (TPN) bei Patienten mit Microvillus Inclusion Disease (MVID) um bis zu 27 % und bei Patienten mit Short Bowel Syndrome (SBS-IF) um 12,5 % senkte.
Die Studie, vorgestellt auf dem jährlichen ELITE PED-GI Kongress, zeigte eine Verringerung des Stuhlvolumens und eine erhöhte Urinausscheidung, was auf eine verbesserte Nährstoffaufnahme hinweist. Dr. Mohamad Miqdady, Leiter der pädiatrischen Gastroenterologie am Sheikh Khalifa Medical City, leitete diese offene Studie zur neuen flüssigen Formulierung von Crofelemer.
Bemerkenswert ist, dass bei einem pädiatrischen MVID-Patienten, der Crofelemer nach 12 Wochen absetzte, sich die Symptome innerhalb von 8 Tagen verschlechterten, was zur Wiederaufnahme der Behandlung führte. Das Unternehmen verfolgt beschleunigte Zulassungsverfahren über das PRIME-Programm der EMA und das Breakthrough Therapies-Programm der FDA, angesichts des erheblichen ungedeckten Bedarfs bei der Behandlung dieser seltenen Erkrankungen.
- Crofelemer reduced Total Parenteral Nutrition needs by up to 27% in MVID patients and 12.5% in SBS-IF patients
- Potential fast-track regulatory approval through EMA's PRIME program and FDA's Breakthrough Therapies program
- No other approved drugs exist for reducing TPN needs in MVID patients, giving the company first-mover advantage
- Multiple ongoing trials: 3 proof-of-concept studies and 2 Phase 2 studies across US, EU, and MENA regions
- Early patient access with reimbursement possible in EU countries based on clinical investigation data
- Small patient population due to MVID being an ultrarare disease, limiting market size
- Study was open-label and non-randomized, which may affect data credibility
- Only preliminary/initial results available, with full efficacy data still pending
- Multiple ongoing trials require significant R&D investment and resources
Insights
Jaguar's crofelemer shows promising TPN reduction in rare diseases, advancing potential for expedited regulatory pathways in markets with zero approved treatments.
Jaguar Health's proof-of-concept data for crofelemer represents a significant development in treating ultra-rare conditions with severe unmet needs. The data showing up to 27% reduction in TPN for MVID patients and 12.5% for SBS-IF patients addresses a critical clinical issue, as TPN dependence carries substantial risks including infections, metabolic complications, and organ dysfunction.
The exploratory trial's design as single-arm and open-label is appropriate given the ultra-rare nature of these diseases, particularly MVID. Notably, the observed withdrawal effect – where symptom worsening prompted treatment reinitiation after just 8 days – provides compelling supporting evidence for crofelemer's biological activity.
From a regulatory perspective, these results strategically position Jaguar to pursue accelerated approval pathways through the EMA's PRIME program and FDA's Breakthrough Therapy designation. For ultra-rare conditions like MVID where patient populations are extremely limited, regulatory authorities often consider robust proof-of-concept data more meaningfully.
The company's coordinated development approach, with multiple simultaneous proof-of-concept studies and two Phase 2 trials, demonstrates efficient resource allocation focused on generating sufficient data for regulatory engagement. The possibility of early patient access in EU countries based on published clinical investigation data represents a potential accelerated commercialization path.
Crofelemer's ability to reduce TPN requirements represents a potential disease-modifying advancement for intestinal failure patients currently without treatment options.
The reduction in TPN requirements demonstrated in this proof-of-concept study represents a clinically meaningful outcome for patients with MVID and SBS-IF. These conditions force lifelong dependence on parenteral support, with patients typically requiring TPN up to 7 days weekly for more than 50% of their nutritional needs.
The clinical significance extends beyond mere symptom management. By reducing stool volume/frequency and increasing urine output (indicating improved oral absorption), crofelemer appears to address the fundamental pathophysiology of these conditions. The rapid symptom deterioration observed during the planned withdrawal period provides compelling evidence of biological effect.
For context, any reduction in TPN dependency substantially impacts patient outcomes. TPN-associated complications include line infections, liver damage, metabolic derangements, and developmental delays in pediatric patients. Even modest reductions in TPN requirements can meaningfully reduce these risks.
Crofelemer's novel antisecretory mechanism appears well-suited for these secretory diarrheas. The drug's plant-based origin and established safety profile in other indications provides additional reassurance for vulnerable pediatric populations.
While further validation in placebo-controlled studies is necessary, these initial results suggest crofelemer could potentially modify disease progression rather than merely treating symptoms – a distinction particularly valuable in lifelong conditions with cumulative morbidity.
Jaguar to host investor webcast today at 8:30 AM Eastern to review the initial results from the proof-of-concept study of crofelemer for MVID and SBS-IF presented April 26 at the Annual ELITE PED-GI Congress; Click here to register
There are 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 including total parenteral nutrition (TPN) - which carries the risk of morbidity, infections, metabolic complications, liver and kidney problems, and neurodevelopmental delay
This proof-of-concept data in MVID supports crofelemer's potential inclusion in the European Medicines Agency's (EMA) PRIME program for expediated and assisted regulatory approval in the EU as well as FDA's Breakthrough Therapies program for expedited regulatory approval in the US
SAN FRANCISCO, CA / ACCESS Newswire / April 30, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics today announced initial proof-of-concept results showing that a novel liquid formulation of crofelemer reduced the required TPN and/or supplementary intravenous fluids, collectively referred to as parenteral support (PS) in patients with intestinal failure due to microvillus inclusion disease (MVID) and short bowel syndrome (SBS-IF) by up to
These initial results were recently presented at the Annual ELITE PED-GI Congress by Dr. Mohamad Miqdady, a recognized leader in pediatric gastroenterology who serves as the Chief of Pediatric Gastroenterology, Hepatology and Nutrition at Sheikh Khalifa Medical City (SKMC) in Abu Dhabi in the United Arab Emirates. He is the Principal Investigator for this ongoing exploratory, single-arm open label non-randomized investigator-initiated trial 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-IF.
"We are not aware of any other intervention that has shown the ability to reduce the required administration of lifelong parenteral support including TPN, thus potentially improving nutrient absorption in patients with MVID," said Lisa Conte, Jaguar's Founder and CEO.
"As I was pleased to announce at the Annual ELITE PED-GI Congress, the initial proof-of-concept results of this study indicate that the novel liquid formulation of crofelemer being evaluated in this investigational study reduced the required weekly amount of TPN and/or supplementary intravenous fluids in participating MVID and SBS-IF patients," said Dr. Miqdady, the principal investigator for the study. "Patients with MVID and SBS-IF suffer from devastating diarrhea and dehydration caused by these debilitating, lifelong conditions and often require lifelong TPN up to 7 days a week for greater than
Per the study protocol, the pediatric MVID patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days. "However, after just 8 days, the patient's parents requested reinitiation of crofelemer dosing, as the patient's symptoms were worsening - evidenced by increased stool output and decreased urine output. Hence we restarted the patient on daily treatment with crofelemer," continued Dr. Miqdady.
"I'm very excited by these preliminary results. There is no treatment for MVID, and these patients have to be on lifelong TPN. Every reduction in TPN counts. The more you manage to taper the time and amount of TPN in MVID patients, the better for these patients, and it has a significant impact on their quality of life as well as on the hepatic and renal complications these patient face," said Dr. Christos Tzivinikos, Founder of the Pediatric GI Department at Al Jalila Children's Specialty Hospital in Dubai, and a Clinical Assistant Professor at Mohammed Bin Rashid University of Medicine and Health Sciences in Dubai. Dr. Tzivinikos is a clinical investigator in Napo's recently initiated placebo-controlled double-blind Phase 2 study of crofelemer for MVID in pediatric patients.
The investigator-initiated trial in Abu Dhabi is evaluating the safety and effectiveness of crofelemer in pediatric patients with intestinal failure and includes an assessment of crofelemer's potential to be safely administered to patients with intestinal failure from MVID, SBS, and other congenital diarrheal disorders (CDD) in optimum dose levels for the patients.
"Based on preliminary communications with European Medicines Agency (EMA) regulatory members that focus on PRIME (Priority Medicines), a program that provides enhanced interactions and early dialogue for development of novel medicines targeting unmet medical needs, we are excited by these findings from Dr. Miqdady's study," said Pravin Chaturvedi, PhD, Chair of Napo's and Jaguar's Scientific Advisory Board (SAB) and Jaguar's Chief Scientific Officer. "With these initial results, given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in Europe. Crofelemer may qualify for participation in PRIME in the European Union and potentially in the U.S. Food and Drug Administration's (FDA) Breakthrough Therapies program."
This study is being conducted at SKMC, a flagship tertiary hospital in the UAE and the largest teaching medical center in Abu Dhabi, by Dr. Miqdady. He 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 SAB. Dr. Miqdady completed his Fellowship in Pediatric Gastroenterology at Baylor College of Medicine and Texas Children's Hospital in Houston.
Jaguar, through Napo Pharmaceuticals and Napo Therapeutics, is currently supporting three POC investigator-initiated trials (IITs), and conducting two placebo-controlled Phase 2 studies, for crofelemer for SBS-IF and/or MVID patients in the United States, European Union, and/or Middle East/North Africa regions. The company's Phase 2 study to evaluate the efficacy of crofelemer for MVID in pediatric patients has been initiated, as has the company's Phase 2 study to evaluate the efficacy of crofelemer for SBS-IF in adults. In addition to the initiation of the POC IIT of crofelemer in Abu Dhabi in pediatric patients for various CDDs, including SBS-IF or MVID, the dosing for the IIT in the U.S. to evaluate crofelemer for SBS-IF in adults has also begun. Initiation of the third IIT is planned for the second half of 2025.
Additional 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.
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 was 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 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 the company will host an investor webcast on April 30, 2025, Jaguar's expectation that crofelemer's novel antisecretory mechanism of action may have the potential to provide a novel therapeutic option to modify disease progression through reduction in TPN needs and associated comorbidities in patients with intestinal failure due to MVID, SBS and other dehydrating conditions and improve their quality of life, Jaguar's expectation that a third IIT of crofelemer will initiate in the second half of 2025, Jaguar's expectation that additional POC results from IITs will be available throughout 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs, 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 U.S. and other regions for crofelemer for MVID and qualify crofelemer for participation in PRIME and in the FDA's Breakthrough Therapies program for MVID, and Jaguar's expectation that, in accordance with the guidelines of specific European Union countries, published data from clinical investigations in MVID and SBS-IF could support reimbursed early patient access to crofelemer for these debilitating conditions in specific European Union 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.
Contact:
hello@jaguar.health
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SOURCE: Jaguar Health, Inc.
View the original press release on ACCESS Newswire
FAQ
What are the key results of JAGX's crofelemer proof-of-concept study for MVID and SBS-IF patients?
How could crofelemer's MVID treatment results affect JAGX's regulatory approval process?
What clinical trials is JAGX currently conducting for crofelemer in 2025?
Why is JAGX's crofelemer significant for MVID patients?
When will additional results from JAGX's crofelemer trials be available?
