Phase 2 Study Initiated to Evaluate Jaguar Health's Crofelemer for Microvillus Inclusion Disease (MVID), an Ultrarare Congenital Diarrheal Disorder
Jaguar Health (NASDAQ:JAGX) has initiated a Phase 2 study to evaluate crofelemer, its plant-based prescription drug, for Microvillus Inclusion Disease (MVID) in pediatric patients. This study is part of five clinical efforts, including three proof-of-concept trials and two Phase 2 studies, across US, EU, and MENA regions. The first patient dosing is expected between December 2024 and January 2025, with initial results potentially available in Q1 2025.
Crofelemer has received Orphan-Drug Designation from both FDA and EMA for MVID and SBS-IF. The study will evaluate a novel oral powder solution formulation, different from the FDA-approved Mytesi® tablets. MVID is an ultrarare pediatric disease affecting approximately 200 patients globally, characterized by severe diarrhea and malabsorption, with no currently approved treatments.
Jaguar Health (NASDAQ:JAGX) ha avviato uno studio di Fase 2 per valutare il crofelemere, il suo farmaco prescritto di origine vegetale, per la Malattia da Inclusione dei Microvilli (MVID) nei pazienti pediatrici. Questo studio fa parte di cinque sforzi clinici, tra cui tre studi di prova di concetto e due studi di Fase 2, nelle regioni USA, UE e MENA. La prima somministrazione ai pazienti è prevista tra dicembre 2024 e gennaio 2025, con i primi risultati potenzialmente disponibili nel primo trimestre del 2025.
Il crofelemere ha ricevuto la Designazione di Farmaco Orfano sia dalla FDA che dall'EMA per MVID e SBS-IF. Lo studio valuterà una nuova formulazione di polvere orale, diversa dai tablet Mytesi® approvati dalla FDA. La MVID è una malattia pediatrica ultr raro che colpisce circa 200 pazienti a livello globale, caratterizzata da grave diarrea e malassorbimento, senza trattamenti attualmente approvati.
Jaguar Health (NASDAQ:JAGX) ha iniciado un estudio de Fase 2 para evaluar el crofelemere, su medicamento recetado a base de plantas, para la Enfermedad de Inclusión de Microvellosidades (MVID) en pacientes pediátricos. Este estudio forma parte de cinco esfuerzos clínicos, incluyendo tres ensayos de prueba de concepto y dos estudios de Fase 2, en las regiones de EE. UU., UE y MENA. Se espera que la primera dosificación en pacientes ocurra entre diciembre de 2024 y enero de 2025, con resultados iniciales que podrían estar disponibles en el primer trimestre de 2025.
El crofelemere ha recibido la Designación de Medicamento Huérfano tanto de la FDA como de la EMA para MVID y SBS-IF. El estudio evaluará una nueva formulación de solución en polvo oral, diferente de las tabletas Mytesi® aprobadas por la FDA. La MVID es una enfermedad pediátrica ultrarrara que afecta aproximadamente a 200 pacientes a nivel mundial, caracterizada por diarrea severa y mala absorción, sin tratamientos aprobados en la actualidad.
재규어 헬스 (NASDAQ:JAGX)는 소아 환자의 미세융모 증후군(MVID)을 평가하기 위해 식물 기반 처방약인 크로펠레머의 2상 연구를 시작했습니다. 이 연구는 미국, EU 및 MENA 지역에서 세 개의 개념 증명 시험과 두 개의 2상 연구를 포함하는 다섯 개의 임상 노력의 일환입니다. 첫 번째 환자의 약물 투여는 2024년 12월에서 2025년 1월 사이에 있을 것으로 예상되며, 초기 결과는 2025년 1분기에 발표될 가능성이 있습니다.
크로펠레머는 MVID 및 SBS-IF에 대해 FDA와 EMA 모두로부터 희귀 약물 지정을 받았습니다. 이 연구는 FDA에서 승인된 Mytesi® 정제와는 다른 새로운 경구용 분말 제형을 평가할 것입니다. MVID는 전 세계적으로 약 200명의 환자에게 영향을 주는 극히 희귀한 소아 질환으로, 심한 설사와 흡수 장애가 특징이며, 현재 승인된 치료법이 없습니다.
Jaguar Health (NASDAQ:JAGX) a lancé une étude de Phase 2 pour évaluer le crofelemere, son médicament sur ordonnance d'origine végétale, pour la Maladie d'Inclusion des Microvillosités (MVID) chez les patients pédiatriques. Cette étude fait partie de cinq efforts cliniques, y compris trois essais de preuve de concept et deux études de Phase 2, dans les régions des États-Unis, de l'UE et de la MENA. La première administration chez les patients est prévue entre décembre 2024 et janvier 2025, avec des résultats initiaux potentiellement disponibles au premier trimestre 2025.
Le crofelemere a reçu la désignation de médicament orphelin de la part de la FDA et de l'EMA pour MVID et SBS-IF. L'étude évaluera une nouvelle formulation de solution en poudre orale, différente des comprimés Mytesi® approuvés par la FDA. La MVID est une maladie pédiatrique ultrarare qui touche environ 200 patients dans le monde, caractérisée par une diarrhée sévère et une malabsorption, sans traitements actuellement approuvés.
Jaguar Health (NASDAQ:JAGX) hat eine Phase-2-Studie zur Bewertung von Crofelemer, seinem pflanzenbasierten verschreibungspflichtigen Medikament, zur Mikrovillushyperplasie (MVID) bei pädiatrischen Patienten initiiert. Diese Studie ist Teil von fünf klinischen Bemühungen, darunter drei Machbarkeitsstudien und zwei Phase-2-Studien, in den Regionen USA, EU und MENA. Die erste Patientendosierung wird voraussichtlich zwischen Dezember 2024 und Januar 2025 erfolgen, wobei erste Ergebnisse möglicherweise im ersten Quartal 2025 verfügbar sind.
Crofelemer hat sowohl von der FDA als auch von der EMA die Orphan-Drug-Designation für MVID und SBS-IF erhalten. Die Studie wird eine neuartige orale Pulverlösung formulieren, die sich von den von der FDA genehmigten Mytesi®-Tabletten unterscheidet. MVID ist eine ultrarare pädiatrische Erkrankung, die ungefähr 200 Patienten weltweit betrifft, und ist durch schwere Durchfälle und Malabsorption gekennzeichnet, ohne derzeit genehmigte Behandlungen.
- Received Orphan-Drug Designation from both FDA and EMA
- Phase 2 study received regulatory clearances from FDA and EMA
- Multiple clinical trials running simultaneously across global regions
- Potential for early patient access and reimbursement in EU countries
- Targeting an unmet medical need with no current approved treatments
- Extremely small patient population (couple hundred globally) limiting market size
- Results not expected until Q1 2025
- Multiple clinical trials running simultaneously may increase operational costs
Insights
The initiation of a Phase 2 study for crofelemer in MVID represents a significant milestone in addressing an ultrarare pediatric condition with high unmet medical need. The multi-regional trial design across US, EU and MENA regions enhances the study's robustness and potential for regulatory acceptance. The novel oral powder formulation being tested differs from the currently approved Mytesi tablets, potentially offering better administration options for pediatric patients.
The parallel execution of five clinical efforts, including three proof-of-concept studies and two Phase 2 trials, demonstrates a comprehensive development strategy. With first results expected in Q1 2025, the company's approach of pursuing multiple studies simultaneously could accelerate the development timeline. The Orphan Drug Designation from both FDA and EMA provides regulatory benefits and market exclusivity, while potential PRIME designation could further expedite development.
This development is strategically significant for Jaguar Health, particularly given its small market cap of
The company's multi-pronged approach with five concurrent clinical efforts, while resource-intensive, could provide multiple shots on goal and de-risk the development program. Success in either MVID or SBS-IF indications could significantly impact the company's valuation, as orphan drugs typically command premium pricing and enjoy market exclusivity.
The study is one of five clinical efforts - three proof-of-concept investigator-initiated trials (IIT) and two Phase 2 studies - of crofelemer for the rare disease indications of MVID and/or short bowel syndrome with intestinal failure (SBS-IF) in the US, EU, and/or Middle East/North Africa regions; availability of first IIT proof-of-concept results potentially in Q1 2025
Crofelemer, Jaguar's novel plant-based prescription drug, has been granted Orphan-Drug Designation by the FDA and the European Medicines Agency (EMA) for both MVID and SBS-IF
SAN FRANCISCO, CA / ACCESSWIRE / December 18, 2024 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics today announced that Napo's Phase 2 study to evaluate the efficacy of crofelemer, Jaguar's novel plant-based anti-secretory prescription drug, for the indication of MVID in pediatric patients, has been initiated.
"The initiation of this double blind, placebo-controlled study in pediatric MVID patients is a key milestone for Jaguar," said Lisa Conte, Jaguar's founder, president, and CEO. "This study is one of five clinical efforts in rare diseases - three IIT proof-of-concept (POC) 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 Middle East/North Africa (MENA) regions. These studies are evaluating a novel oral powder for solution formulation of crofelemer - which is different from the FDA-approved oral formulation of crofelemer delayed-release tablets, Mytesi ® , available for people living with HIV/AIDS. Dosing of the first patient in each of these five studies is expected to occur during December 2024 and January 2025, with the availability of the first IIT POC result potentially in Q1 2025, with additional IIT POC 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 in those countries."
The clinical protocol for the Phase 2 study in pediatric MVID patients has received regulatory clearances from the U.S. Food and Drug Administration (FDA) and the EMA, and the trial will be conducted at clinical sites in the US, Europe, and the MENA region.
"MVID and SBS-IF, rare and severe diseases requiring intensive parenteral nutrition (PN) and support, have severe morbidity and mortality implications as well as impacting the quality of life of both patients and their caregivers," Conte said. "In addition to the clinical endpoints, we plan to assess the quality-of-life impact on patients and caregivers as part of both the above-referenced Phase 2 studies."
MVID is an ultrarare 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.
"We expect that, if even just a very small number of patients show benefit with the extremely safe profile of crofelemer, this may potentially allow approval in the U.S. for crofelemer for MVID; support reimbursed early patient access to crofelemer for MVID in specific EU countries; and qualify crofelemer for participation in PRIME for MVID. PRIME is an EMA program providing enhanced interaction and early dialogue with developers of promising medicines that target an unmet medical need, with the goal of optimizing development plans and speeding up evaluation so the medicine can reach patients earlier," Conte said.
Crofelemer has been granted Orphan-Drug Designation by the FDA and the EMA for SBS-IF and MVID.
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 dosing of the first patient in each of the five rare disease studies will occur during December 2024 and January 2025, Jaguar's expectation that the availability of the first IIT POC result may potentially occur in Q1 2025, Jaguar's expectation that additional IIT POC results may be available throughout 2025, 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, Jaguar's expectation that it will assess the quality-of-life impact of SBS-IF and/or MVID on patients and caregivers as part of both Phase 2 studies of crofelemer, and Jaguar's expectation that, if even just a very small number of patients show benefit with crofelemer, this may allow approval in the U.S. for crofelemer for MVID, support reimbursed early patient access to crofelemer for MVID in specific EU countries, and qualify crofelemer for participation in PRIME for MVID. 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.
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