Jaguar Health Announces First Patient Dosed in Investigator-Initiated Trial (IIT) Evaluating Crofelemer for the Rare Disease Indication of Microvillus Inclusion Disease (MVID), with Proof-of-Concept (POC) Data Potentially Available H1 2025
Jaguar Health (NASDAQ:JAGX) announced the first patient dosing in an investigator-initiated proof-of-concept (POC) trial evaluating crofelemer for Microvillus Inclusion Disease (MVID) at Sheikh Khalifa Medical City in UAE. The trial, led by Dr. Mohamad Miqdady, will also include pediatric patients with short bowel syndrome with intestinal failure (SBS-IF).
This study is one of five clinical efforts for crofelemer in rare diseases, with POC data potentially available in H1 2025. Positive results could lead to reimbursed early patient access in certain European countries. Crofelemer has received Orphan Drug Designation from both FDA and European Medicines Agency for MVID and SBS-IF.
MVID affects a few hundred patients globally, while SBS affects 10,000-20,000 people in both the U.S. and Europe. Pediatric SBS incidence varies between 0.02% to 1.2% of live births, with a maximal U.S. incidence estimated at approximately 43,000 based on 2023 birth rates.
Jaguar Health (NASDAQ:JAGX) ha annunciato la somministrazione del primo paziente in uno studio clinico proof-of-concept (POC) avviato da ricercatori che valuta il crofelemer per la Malattia da Inclusione di Microvilli (MVID) presso il Sheikh Khalifa Medical City negli UAE. Lo studio, guidato dal Dr. Mohamad Miqdady, includerà anche pazienti pediatrici affetti da sindrome dell'intestino corto con insufficienza intestinale (SBS-IF).
Questa ricerca è uno dei cinque sforzi clinici per il crofelemer in malattie rare, con dati POC potenzialmente disponibili nella prima metà del 2025. Risultati positivi potrebbero portare a un accesso anticipato rimborsato per i pazienti in alcuni paesi europei. Il crofelemer ha ricevuto la designazione di Farmaco Orfano sia dalla FDA che dall'Agenzia Europea dei Medicinali per MVID e SBS-IF.
La MVID colpisce alcune centinaia di pazienti a livello globale, mentre la SBS interessa da 10.000 a 20.000 persone sia negli Stati Uniti che in Europa. L'incidenza della SBS pediatrica varia dallo 0,02% all'1,2% delle nascite vive, con un'incidenza massima stimata negli Stati Uniti di circa 43.000 in base ai tassi di natalità del 2023.
Jaguar Health (NASDAQ:JAGX) anunció la primera dosificación de un paciente en un ensayo proof-of-concept (POC) iniciado por investigadores que evalúa el crofelemer para la Enfermedad por Inclusión de Microvellosidades (MVID) en el Sheikh Khalifa Medical City de los EAU. El ensayo, dirigido por el Dr. Mohamad Miqdady, también incluirá pacientes pediátricos con síndrome de intestino corto con falla intestinal (SBS-IF).
Este estudio es uno de cinco esfuerzos clínicos para el crofelemer en enfermedades raras, con datos de POC potencialmente disponibles en la primera mitad de 2025. Resultados positivos podrían llevar a un acceso anticipado reembolsado para pacientes en ciertos países europeos. El crofelemer ha recibido la designación de Medicamento Huérfano tanto de la FDA como de la Agencia Europea de Medicamentos para MVID y SBS-IF.
La MVID afecta a unos pocos cientos de pacientes a nivel mundial, mientras que SBS afecta a 10,000-20,000 personas tanto en EE. UU. como en Europa. La incidencia de SBS pediátrica varía entre el 0.02% y el 1.2% de los nacidos vivos, con una incidencia máxima estimada en EE. UU. de aproximadamente 43,000 en base a las tasas de natalidad de 2023.
재규어 헬스 (NASDAQ:JAGX)는 아랍 에미리트의 셰이크 칼리파 메디컬 시티에서 크로펠레머를 미세융털 포함 질환(MVID) 평가를 위한 임상 시험에서 첫 번째 환자 투여를 발표했습니다. 미크다디 박사에 의해 이끌어진 이 연구는 장의 기능이 저하된 단장 증후군(SBS-IF) 아동 환자도 포함할 것입니다.
이 연구는 희귀 질환에서 크로펠레머에 대한 다섯 가지 임상 노력 중 하나이며, POC 데이터는 2025년 상반기에 제공될 수 있습니다. 긍정적인 결과는 특정 유럽 국가에서 환자에 대한 조기 접근에 대한 보상으로 이어질 수 있습니다. 크로펠레머는 MVID 및 SBS-IF에 대해 FDA와 유럽 의약청으로부터 희귀의약품 지정을 받았습니다.
MVID는 전 세계적으로 몇 백 명의 환자에게 영향을 미치며, SBS는 미국과 유럽에서 10,000-20,000명에게 영향을 미칩니다. 소아 SBS의 발생률은 출생 생존아의 0.02%에서 1.2%까지 다양하며, 2023년 출생률을 기준으로 미국의 최대 발생률은 약 43,000명으로 추정됩니다.
Jaguar Health (NASDAQ:JAGX) a annoncé la première dose administrée à un patient dans un essai clinique initié par un investigateur, visant à évaluer le crofelemer pour la Maladie à Inclusion de Microvillosités (MVID) au Sheikh Khalifa Medical City aux Émirats Arabes Unis. L'essai, dirigé par le Dr Mohamad Miqdady, inclura également des patients pédiatriques atteints de syndrome de l'intestin court avec défaillance intestinale (SBS-IF).
Cette étude fait partie des cinq efforts cliniques concernant le crofelemer dans les maladies rares, avec des données POC potentiellement disponibles au premier semestre 2025. Des résultats positifs pourraient conduire à un accès précoce remboursé pour certains patients dans des pays européens. Le crofelemer a reçu la désignation de Médicament Orphelin de la part de la FDA et de l'Agence Européenne des Médicaments pour la MVID et le SBS-IF.
La MVID affecte quelques centaines de patients dans le monde, tandis que le SBS touche entre 10 000 et 20 000 personnes tant aux États-Unis qu'en Europe. L'incidence du SBS pédiatrique varie entre 0,02 % et 1,2 % des naissances vivantes, avec une incidence maximale estimée aux États-Unis à environ 43 000 d'après les taux de natalité de 2023.
Jaguar Health (NASDAQ:JAGX) hat die erste Dosis eines Patienten in einer von einem Prüfer initiierten Proof-of-Concept-Studie (POC) bekannt gegeben, die Crofelemer zur Behandlung der Mikrovilluseinschlusskrankheit (MVID) am Sheikh Khalifa Medical City in den VAE bewertet. Die Studie, geleitet von Dr. Mohamad Miqdady, wird auch pädiatrische Patienten mit Kurzschlussdarm-Syndrom und Darmversagen (SBS-IF) einbeziehen.
Diese Studie ist eine von fünf klinischen Initiativen für Crofelemer bei seltenen Krankheiten, wobei POC-Daten voraussichtlich in der ersten Hälfte von 2025 verfügbar sein werden. Positive Ergebnisse könnten in bestimmten europäischen Ländern zu einem erstattungsfähigen frühen Zugang für Patienten führen. Crofelemer hat von der FDA und der Europäischen Arzneimittel-Agentur die Orphan-Drug-Designation für MVID und SBS-IF erhalten.
MVID betrifft weltweit einige Hundert Patienten, während SBS 10.000 bis 20.000 Menschen in den USA und Europa betrifft. Die Inzidenz von SBS bei Kindern variiert zwischen 0,02 % und 1,2 % der Lebendgeburten, wobei die maximale Inzidenz in den USA auf etwa 43.000 geschätzt wird, basierend auf den Geburtenraten von 2023.
- Potential early patient access and reimbursement in European markets if POC data is positive
- Orphan Drug Designation secured from both FDA and EMA for MVID and SBS-IF indications
- Multiple clinical trials ongoing simultaneously, indicating strong development pipeline
- Small patient population for MVID (only hundreds globally) may limit market potential
- Results from POC trials not expected until H1 2025
Insights
Jaguar Health has initiated a strategic clinical trial for crofelemer, marking a pivotal development in their rare disease program. The trial's location in the UAE's Sheikh Khalifa Medical City is particularly significant, as the MENA region has a higher prevalence of autosomal recessive disorders like MVID. This geographical strategy could accelerate patient recruitment and data collection.
The company is executing a comprehensive clinical development strategy with five concurrent trials - three proof-of-concept investigator-initiated trials and two Phase 2 studies - across multiple regions. This multi-pronged approach could expedite the development timeline and potentially lead to early market access in certain European countries, particularly significant given the lack of approved treatments for MVID.
From a market perspective, while MVID affects only a few hundred patients globally, orphan drug pricing could make this a viable commercial opportunity. The addition of SBS-IF to the trial, which affects approximately 10,000-20,000 patients in both the US and Europe, substantially expands the potential market. The dual Orphan Drug Designations from both FDA and EMA provide regulatory benefits and market exclusivity, strengthening the company's competitive position.
The timing of potential proof-of-concept data in H1 2025 is crucial, as positive results could trigger early patient access programs in Europe, potentially generating revenue before full approval. This could provide much-needed validation for crofelemer's broader development program and support Jaguar Health's commercialization strategy in rare diseases.
Trial taking place at Sheikh Khalifa Medical City, a flagship tertiary hospital in the United Arab Emirates
POC data generated from this study could potentially lead to reimbursed early patient access in certain European countries for crofelemer for MVID
Crofelemer, Jaguar's novel plant-based prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines Agency for both MVID and short bowel syndrome with intestinal failure (SBS-IF)
SAN FRANCISCO, CA / ACCESS Newswire / January 22, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics today announced that the first patient has been dosed in the independent investigator-initiated POC trial of crofelemer, Jaguar's novel plant-based anti-diarrheal prescription drug, for the rare disease indication of MVID in pediatric patients. POC data generated from this study potentially in the first half of 2025 could lead to reimbursed early patient access to crofelemer for this indication in certain European countries.
The trial will also include pediatric patients with SBS-IF. It 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 first patient in this unblinded study is an important milestone in clinical development efforts for crofelemer for MVID and SBS-IF - both of which are rare and severe diseases requiring intensive parenteral nutrition and support," said Lisa Conte, Jaguar's founder, president, and CEO.
The trial is taking place at SKMC. Autosomal recessive disorders and congenital anomalies such as MVID are more prevalent in the Middle East and North Africa (MENA) region.
"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 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."
MVID and SBS-IF, 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.
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.
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
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.
Crofelemer has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency for MVID and SBS.
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 MVID 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:
hello@jaguar.health
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SOURCE: Jaguar Health, Inc.
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FAQ
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