Microvillus Inclusion Disease

Cholera is an acute diarrheal illness caused by infection of the intestine with the bacterium Vibrio cholerae Crofelemer previously granted Orphan Drug Designation by the FDA and the EMA for both short bowel syndrome and microvillus inclusion disease SAN FRANCISCO, CA / ACCESSWIRE / February 12, 2024 / Jaguar Health (NASDAQ:JAGX) today announced that Jaguar family company Napo Pharmaceuticals (Napo) has submitted an Orphan Drug Designation (ODD) application to the U.S. Food and Drug Administration (FDA) for crofelemer, the company's novel plant-based prescription drug, for the symptomatic relief and treatment of moderate-to-severe diarrhea, with or without concomitant antimicrobial therapy, from bacterial, viral, and parasitic infections including Vibrio cholerae, the bacterium that causes cholera. Due to the very low incidence and prevalence of cholera in the United States, cholera is an orphan indication in this country. Rare diseases, also referred to as orphan indications, are a core Jaguar development focus. In 2024 and 2025, Jaguar expects results from 5 proof-of-concept studies of crofelemer, the company's novel plant-based prescription drug, from at least 8 clinical sites, including Cleveland Clinic, on three continents for the rare disease indications of microvillus inclusion disease (MVID) - an ultrarare congenital diarrheal disorder (CDD) - and short bowel syndrome (SBS). In accordance with the guidelines of specific European Union countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for these debilitating conditions in 2025 while the company pursues approval of crofelemer for SBS and MVID from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). In August 2023 the FDA activated Napo's Investigational New Drug (IND) application for a new crofelemer powder for oral solution formulation for the treatment of MVID. "We are very pleased that the ODD application for crofelemer for cholera-related diarrhea has been submitted," said Steven King, PhD, Jaguar's Chief Sustainable Supply, Ethnobotanical Research & IP Officer. "The company previously presented Phase 2 data on crofelemer for the treatment of devastating dehydration in cholera patients from the renowned International Centre for Diarrhoeal Disease Research (icddr,b) in Bangladesh. Receipt of orphan drug designation would qualify the company for various development incentives, including tax credits for qualified clinical testing and relief of filing fees, and provide the company with a seven-year period of marketing exclusivity if marketing approval is approved for crofelemer for this indication in the U.S. We expect to pursue ODD designation for crofelemer for this indication from the European Medicines Agency (EMA) as well in the future." Crofelemer has been granted ODD by the FDA and the EMA for both MVID and SBS. The Orphan Drug Act (ODA) in the U.S. provides for granting special status to a small molecule drug or biological product to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan drug designation (or sometimes "orphan status"). In the EU, receipt of ODD supports some specific regulatory pathways, and sponsors who obtain ODD for their drug can benefit from Scientific Advice from the EMA for clinical trials for the orphan indication and receive market exclusivity for a period of ten years once the medicine is approved for commercialization. Although cholera is an orphan indication in the U.S., it is estimated that, worldwide, 1.3 to 4 million cholera cases and 21,000 to 143,000 cholera-related deaths occur each year, according to the Centers for Disease Control and Prevention of the U.S. Department of Health & Human Services. Approximately one in 10 infected persons will have severe disease characterized by profuse watery diarrhea, vomiting, and leg cramps. In these people, rapid loss of body fluids leads to dehydration and shock. Without treatment, death can occur within hours. Cholera is now endemic in many countries outside the U.S. Preliminary data issued January 11, 2024 by the World Health Organization (WHO) indicates that the number of cholera cases reported in 2023 in WHO Member States as of December 15, 2023 surpassed that of 2022. Nearly a year has passed since WHO classified the global resurgence of cholera as a grade 3 emergency, the highest internal level for a health emergency requiring a comprehensive response at the three levels of the organization. About Crofelemer Crofelemer is a botanical (plant-based) drug extracted and purified from the red bark sap, also referred to as "dragon's blood," of the medicinal Croton lechleri tree in the Amazon Rainforest. Jaguar Health family company 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, 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. Napo Pharmaceuticals' crofelemer drug product candidate is the subject of the OnTarget study, a pivotal Phase 3 clinical trial for preventive treatment of chemotherapy-induced overactive bowel (CIOB) in adults with cancer on targeted 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 Napo Pharmaceuticals, visit Napo Therapeutics, visit napotherapeutics.com Magdalena Biosciences, visit magdalenabiosciences.com Visit Jaguar on LinkedIn: Visit Jaguar on X: Visit Jaguar on Instagram: Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that results from 5 proof-of-concept studies of crofelemer from at least 8 clinical sites, including Cleveland Clinic, on three continents will be available in 2024 and 2025 for the indications of MVID and SBS, Jaguar's expectation that, in accordance with the guidelines of specific European Union countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for MVID and SBS in 2025 while the company pursues approval of crofelemer for SBS and MVID from the EMA and the FDA, and Jaguar's expectation that it will pursue ODD designation for crofelemer for cholera-related diarrhea from the EMA in the future. 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 a number of 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. Source: Jaguar Health, Inc. Contact: hello@jaguar.health SOURCE: Jaguar Health, Inc. View the original press release on accesswire.com

New patent issued for core rare disease target indication for crofelemer Jaguar is supporting investigator-initiated proof-of-concept studies of crofelemer for the rare disease indications of microvillus inclusion disease - an ultrarare CDD - and short bowel syndrome (SBS) in the US, EU, and Middle East/North Africa regions, with results expected in 2024 SAN FRANCISCO, CA / ACCESSWIRE / January 4, 2024 / Napo Pharmaceuticals (Napo), a Jaguar Health (NASDAQ:JAGX) (Jaguar) family company, today announced that the United States Patent and Trademark Office on January 2, 2024 issued a new U.S. patent to Napo for methods for treating congenital diarrheal disorders (CDDs) in patients with an inhibitor of chloride-ion transport such as crofelemer, Napo's novel plant-based prescription drug. "We are very happy that this patent has been issued and believe that it further enhances our intellectual property protection for crofelemer for our core focus on CDD indications," said Lisa Conte, Jaguar's founder, president, and CEO. Jaguar is supporting investigator-initiated and investigator IND proof-of-concept studies of crofelemer for the rare disease indications of microvillus inclusion disease (MVID) - an ultrarare CDD - and short bowel syndrome (SBS) in the US, European Union, and Middle East/North Africa (MENA) regions, with results expected in 2024. In accordance with the guidelines of specific EU countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for MVID or SBS for these debilitating conditions. MVID is a catastrophic medical situation for pediatric patients, and there are currently no approved drug treatments. Some SBS patients are subject to intestinal failure, often requiring parenteral nutrition (PN) from a few to up to 7 days a week. Intestinal failure is associated with significant morbidity and mortality; and high medical expenses associated with PN. SBS patients with intestinal failure also have severe chronic diarrhea, and the associated sequelae from diarrhea, including significant dehydration, metabolic acidosis or alkalosis and malnutrition, and other secondary symptoms, and these symptoms emerge either early or late, and many times become life-threatening. Crofelemer has been granted Orphan Drug Designation (ODD) by the FDA and the European Medicines Agency for both MVID and SBS with intestinal failure. The ODD programs in the US and EU qualify sponsors to receive potential incentives to develop therapies for the diagnosis, prevention, or treatment of rare diseases or conditions. As with all potential follow-on indications, Jaguar and Napo prioritize IP protection. Napo currently holds approximately 148 patents and approximately 50 patents pending. To date, crofelemer is the only oral plant-based prescription medicine approved under the FDA's Botanical Guidance, which provides an important additional exclusivity advantage due to the inherent practicalities limiting the pathway by which a generic version of the drug could be produced. 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, 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. Napo Pharmaceuticals' crofelemer drug product candidate is the subject of the OnTarget study, an ongoing pivotal Phase 3 clinical trial for preventive treatment of chemotherapy-induced overactive bowel (CIOB) in adults with cancer on targeted 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 Napo Pharmaceuticals, visit Napo Therapeutics, visit napotherapeutics.com Magdalena Biosciences, visit magdalenabiosciences.com Visit Jaguar on LinkedIn: Visit Jaguar on X: Visit Jaguar on Instagram: Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that results of investigator-initiated and investigator IND proof-of-concept studies of crofelemer for MVID and SBS will be available in 2024, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for SBS or 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: hello@jaguar.health Jaguar-JAGX SOURCE: Jaguar Health, Inc. View the original press release on accesswire.com

Top line data for Jaguar Health's pivotal phase 3 OnTarget trial of crofelemer for prophylaxis of diarrhea in adult cancer patients receiving targeted therapy forthcoming SAN FRANCISCO, CA / ACCESSWIRE / November 30, 2023 / Jaguar Health, Inc. (NASDAQ:JAGX) today announced that Lisa Conte, the company's founder, president and CEO, will present virtually on December 5, 2023 at the MedInvest Oncology Investor Conference. Participation Instructions for Jaguar Health's Virtual Presentation at the MedInvest Oncology Investor Conference When: Tuesday, December 5, 2023 from 9:55 AM - 10:10 AM Pacific Standard Time Registration link for presentation: Click Here About the Phase 3 OnTarget Clinical Trial The OnTarget trial is evaluating the company's plant-based prescription drug crofelemer for prophylaxis (prevention) of diarrhea in adult cancer patients with solid tumors receiving targeted therapy, with or without standard chemotherapy - an indication Napo also refers to as preventive treatment of chemotherapy-induced overactive bowel (CIOB) - which includes symptoms such as chronic debilitating diarrhea (loose and/or watery stools), urgency, and bowel incontinence. The study is a first-of-its-kind prophylactic clinical trial with a primary endpoint based on patient reported outcomes that address the highly neglected and unmet burden of CIOB, including specifically diarrhea/loose watery stools. This study is evaluating the benefit of crofelemer to prevent or substantially reduce the number of weekly loose and/or watery stools as a continuous measurement over the 12-week treatment period compared to placebo. By completing the targeted enrollment in stage 1, the OnTarget study has approximately 90% power to detect the difference in the incidence and severity of diarrhea between crofelemer and placebo. The company's expectation is that the double-blind placebo-controlled OnTarget trial will provide evidence that CIOB associated with targeted cancer therapies is chronic, not acute, and impacts the patient's quality of life as well as their ability to remain on their cancer therapy regimens at proven doses for better outcomes. The OnTarget trial is evaluating the effectiveness of crofelemer's novel mechanism of action, intestinal chloride ion channel modulation, to mitigate or substantially reduce chronic cancer therapy-related diarrhea and other symptoms of CIOB. About Chemotherapy-Induced Overactive Bowel (CIOB) A significant proportion of patients undergoing cancer therapy experience chemotherapy-induced overactive bowel (CIOB) - which includes symptoms such as chronic debilitating diarrhea (loose and/or watery stools), urgency, and bowel incontinence. Diarrhea has the potential to cause dehydration, and worsen fatigue and non-adherence to treatment in this population. Novel "targeted cancer therapy" agents, such as epidermal growth factor receptor (EGFR) antibodies and tyrosine kinase inhibitors (TKIs), with or without cycle chemotherapy agents, may cause increased electrolyte and fluid content in the gut lumen, which results in passage of loose and/or watery stools (i.e., diarrhea). Diarrhea has been reported as one of the most common side effects of TKIs and may result in cancer therapy drug holidays or reductions from therapeutic dose, potentially impacting patient outcome. Diarrhea is also a common side effect of some approved CDK 4/6 inhibitors. Patients with cancer-related diarrhea (CTD) are 40% more likely to discontinue their chemotherapy or targeted therapy than patients without CTD,1 and the cost of care of CTD patients is estimated to be 2.9 times higher than for patients who are not experiencing CTD.2 Rare Disease Business Opportunity Jaguar is supporting investigator-initiated and investigator IND proof-of-concept studies of crofelemer for microvillus inclusion disease (MVID) and short bowel syndrome (SBS) with intestinal failure in the US, EU and Middle East/North Africa (MENA) regions, with results expected before the end of 2023 and in 2024. In accordance with the guidelines of specific European Union countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for SBS or MVID, potentially in 2024, for these debilitating conditions. About Crofelemer Crofelemer is the only oral FDA-approved prescription drug under botanical guidance. It is plant-based, extracted and purified from the red bark sap of the Croton lechleri tree in the Amazon Rainforest. Napo Pharmaceuticals, a Jaguar family company, 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, and bowel incontinence. 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. Napo Pharmaceuticals' crofelemer drug product candidate is the subject of the OnTarget study, an ongoing pivotal Phase 3 clinical trial for preventive treatment of chemotherapy-induced overactive bowel (CIOB) in adults with cancer on targeted 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 Napo Pharmaceuticals, visit Napo Therapeutics, visit napotherapeutics.com Magdalena Biosciences, visit magdalenabiosciences.com Visit Jaguar on LinkedIn: Visit Jaguar on X: Visit Jaguar on Instagram: Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that Jaguar Health management will present December 5, 2023 at the MedInvest Oncology Investor Conference, Jaguar Health's expectation that top line results from the OnTarget study are forthcoming, the expectation that the OnTarget trial will provide evidence that diarrhea associated with targeted cancer therapies is chronic, not acute, and impacts the patient's quality of life as well as their ability to remain on their cancer therapy regimens at proven doses for better outcomes, and the expectation that results of proof-of-concept studies of crofelemer for MVID and SBS with intestinal failure will be available before the end of 2023 and in 2024. 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 Pablo C. Okhuysen, M.D., The impact of cancer-related diarrhea on changes in cancer therapy patterns: Real world evidence 2 Eric Roeland, M.D., FAAHPM, Healthcare utilization and costs associated with cancer-related diarrhea CONTACT: hello@jaguar.health Jaguar-JAGX SOURCE: Jaguar Health, Inc. View source version on accesswire.com: