Jaguar Receives Regulatory Clearance in Germany and Italy for Crofelemer for Rare Disease Study - Short Bowel Syndrome with Intestinal Failure (SBS-IF)
Jaguar Health (NASDAQ:JAGX) has received regulatory clearance from Italian and German health authorities for a Phase 2 study of crofelemer in adults with Short Bowel Syndrome with Intestinal Failure (SBS-IF). The study, initiated on February 18, 2025, is part of five clinical efforts including three proof-of-concept trials and two Phase 2 studies for SBS-IF and microvillus inclusion disease (MVID).
The company expects the first proof-of-concept results potentially in H1 2025, with additional results throughout the year. Crofelemer, a plant-based anti-secretory prescription drug, has received Orphan-Drug Designation from both FDA and EMA for SBS-IF and MVID. These rare diseases affect approximately 10,000 to 20,000 people each in both the US and Europe, requiring intensive parenteral nutrition and significantly impacting patient quality of life.
Jaguar Health (NASDAQ:JAGX) ha ricevuto l'autorizzazione normativa dalle autorità sanitarie italiane e tedesche per uno studio di Fase 2 su crofelemer in adulti con Sindrome dell'Intestino Corto con Insufficienza Intestinale (SBS-IF). Lo studio, avviato il 18 febbraio 2025, fa parte di cinque sforzi clinici che includono tre studi di prova di concetto e due studi di Fase 2 per SBS-IF e malattia da inclusione microvillosa (MVID).
L'azienda prevede di ottenere i primi risultati della prova di concetto potenzialmente nella prima metà del 2025, con risultati aggiuntivi nel corso dell'anno. Crofelemer, un farmaco anti-secreto di origine vegetale, ha ricevuto la designazione di farmaco orfano sia dalla FDA che dall'EMA per SBS-IF e MVID. Queste malattie rare colpiscono circa 10.000-20.000 persone ciascuna sia negli Stati Uniti che in Europa, richiedendo un'alimentazione parenterale intensiva e influenzando significativamente la qualità della vita dei pazienti.
Jaguar Health (NASDAQ:JAGX) ha recibido la autorización regulatoria de las autoridades sanitarias italianas y alemanas para un estudio de Fase 2 de crofelemer en adultos con Síndrome de Intestino Corto con Insuficiencia Intestinal (SBS-IF). El estudio, iniciado el 18 de febrero de 2025, forma parte de cinco esfuerzos clínicos que incluyen tres ensayos de prueba de concepto y dos estudios de Fase 2 para SBS-IF y enfermedad por inclusión de microvellosidades (MVID).
La compañía espera obtener los primeros resultados de prueba de concepto potencialmente en la primera mitad de 2025, con resultados adicionales a lo largo del año. Crofelemer, un medicamento anti-secretor de origen vegetal, ha recibido la designación de medicamento huérfano tanto de la FDA como de la EMA para SBS-IF y MVID. Estas enfermedades raras afectan aproximadamente a 10,000 a 20,000 personas cada una en EE. UU. y Europa, requiriendo nutrición parenteral intensiva y afectando significativamente la calidad de vida de los pacientes.
Jaguar Health (NASDAQ:JAGX)는 이탈리아와 독일의 보건 당국으로부터 장의 기능 부전이 있는 단장 증후군(SBS-IF) 성인을 대상으로 한 크로페레머의 2상 연구에 대한 규제 승인을 받았습니다. 이 연구는 2025년 2월 18일에 시작되었으며, SBS-IF 및 미세융모 포함 질환(MVID)에 대한 3개의 개념 증명 시험과 2개의 2상 연구를 포함한 5개의 임상 연구의 일환입니다.
회사는 2025년 상반기 내에 첫 번째 개념 증명 결과를 기대하고 있으며, 연중 추가 결과를 발표할 예정입니다. 크로페레머는 식물 기반의 항분비 처방약으로, SBS-IF와 MVID에 대해 FDA와 EMA 모두에서 희귀약물 지정을 받았습니다. 이러한 희귀 질환은 미국과 유럽에서 각각 약 10,000명에서 20,000명에게 영향을 미치며, 집중적인 경구 영양이 필요하고 환자의 삶의 질에 상당한 영향을 미칩니다.
Jaguar Health (NASDAQ:JAGX) a reçu l'autorisation réglementaire des autorités sanitaires italiennes et allemandes pour une étude de phase 2 sur le crofelemer chez des adultes atteints de syndrome de l'intestin court avec insuffisance intestinale (SBS-IF). L'étude, lancée le 18 février 2025, fait partie de cinq efforts cliniques, y compris trois essais de preuve de concept et deux études de phase 2 pour le SBS-IF et la maladie à inclusion microvillaire (MVID).
L'entreprise s'attend à obtenir les premiers résultats de preuve de concept potentiellement au premier semestre 2025, avec des résultats supplémentaires tout au long de l'année. Le crofelemer, un médicament anti-sécrétoire d'origine végétale, a reçu la désignation de médicament orphelin à la fois de la FDA et de l'EMA pour le SBS-IF et la MVID. Ces maladies rares touchent environ 10 000 à 20 000 personnes chacune aux États-Unis et en Europe, nécessitant une nutrition parentérale intensive et ayant un impact significatif sur la qualité de vie des patients.
Jaguar Health (NASDAQ:JAGX) hat von den italienischen und deutschen Gesundheitsbehörden die regulatorische Genehmigung für eine Phase-2-Studie zu Crofelemer bei Erwachsenen mit Kurzschluss-Syndrom mit intestinaler Insuffizienz (SBS-IF) erhalten. Die Studie, die am 18. Februar 2025 begonnen wurde, ist Teil von fünf klinischen Bemühungen, darunter drei Proof-of-Concept-Studien und zwei Phase-2-Studien zu SBS-IF und Mikrovillushyperplasie (MVID).
Das Unternehmen erwartet die ersten Ergebnisse der Proof-of-Concept-Studie voraussichtlich im ersten Halbjahr 2025, mit weiteren Ergebnissen im Laufe des Jahres. Crofelemer, ein pflanzenbasiertes anti-sekretorisches Rezeptmedikament, hat sowohl von der FDA als auch von der EMA die Orphan-Drug-Designation für SBS-IF und MVID erhalten. Diese seltenen Krankheiten betreffen jeweils etwa 10.000 bis 20.000 Menschen in den USA und Europa, erfordern eine intensive parenterale Ernährung und haben erhebliche Auswirkungen auf die Lebensqualität der Patienten.
- Received regulatory clearance in Germany and Italy for Phase 2 clinical trial
- Multiple ongoing clinical trials (5 in total) across different regions
- Dual Orphan-Drug Designation from FDA and EMA for both target conditions
- Potential early patient access in EU countries based on clinical data
- First proof-of-concept results not expected until H1 2025
- market size (10,000-20,000 patients in US and similar in Europe)
Insights
The regulatory clearance in Germany and Italy for crofelemer's Phase 2 study represents a strategic expansion of Jaguar Health's rare disease program. The company's multi-pronged clinical approach, running five concurrent trials across different regions, demonstrates a sophisticated development strategy that could accelerate time-to-market while diversifying clinical risk.
The Orphan-Drug Designation from both FDA and EMA is particularly valuable, offering several benefits including:
- Market exclusivity (7 years in US, 10 years in EU)
- Tax credits for clinical research costs
- Waiver of regulatory fees
- Protocol assistance
The potential for early patient access in EU countries through published clinical data is a significant advantage, as it could generate revenue streams before full approval. With a combined US-EU patient population of 20,000-40,000 for SBS-IF alone, the market opportunity is substantial. Rare disease treatments typically command premium pricing, often exceeding
The company's strategy of conducting multiple trials simultaneously is noteworthy for three reasons:
- Accelerated data generation across different patient populations
- Risk mitigation through multiple proof-of-concept opportunities
- Potential for expanded indications (both SBS-IF and MVID)
The expected availability of initial proof-of-concept results in H1 2025 could serve as a significant catalyst for the company's valuation, particularly if positive results emerge from multiple trials.
This study, initiated yesterday, is one of five clinical efforts - three proof-of-concept (POC) investigator-initiated trials (IIT) and two Phase 2 studies - of crofelemer for the rare disease indications of SBS-IF and microvillus inclusion disease (MVID) in the US, EU, and/or Middle East/North Africa regions
Availability of first IIT proof-of-concept results potentially in H1 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 SBS-IF and MVID
SAN FRANCISCO, CA / ACCESS Newswire / February 19, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) ("Jaguar") family companies Napo Pharmaceuticals ("Napo") and Napo Therapeutics today announced that the clinical protocol for Napo's Phase 2 study to evaluate the efficacy of crofelemer, Jaguar's novel plant-based anti-secretory prescription drug, for the indication of SBS-IF in adults has received regulatory clearance from health authorities in Italy and Germany, where the trial will be conducted. The study initiated yesterday, February 18, 2025.
"The initiation of this double blind, placebo-controlled study in adult SBS-IF patients is another 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 POC IIT studies and two Phase 2 studies - for crofelemer for the orphan disease indications of SBS-IF and/or MVID in the United States, European Union, and/or Middle East and North Africa (MENA) regions."
"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 United Arab Emirates to evaluate crofelemer for SBS-IF and MVID in pediatric patients, and the independent IIT in the U.S. to evaluate crofelemer for SBS-IF in adults. The additional IIT is expected to initiate in 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," Conte said.
SBS-IF and MVID, 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. 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.
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 an additional IIT will initiate in Q1 2025, Jaguar's expectation that availability of the first IIT POC result may potentially occur in H1 2025, Jaguar's expectation that additional IIT POC results 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.
Contact:
Jaguar-JAGX
SOURCE: Jaguar Health, Inc.
View the original press release on ACCESS Newswire
FAQ
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