Refsum Disease, Infantile

New patents issued for core rare disease target indications for crofelemer Napo has approximately 200 patents issued and pending Jaguar is supporting investigator-initiated proof-of-concept studies of crofelemer for the rare disease indications of SBS and microvillus inclusion disease (MVID), an ultrarare CDD, in the US, EU, and Middle East/North Africa regions, with results expected in 2024 and 2025 SAN FRANCISCO, CA / ACCESSWIRE / July 8, 2024 / Napo Pharmaceuticals (Napo), a Jaguar Health (NASDAQ:JAGX) (Jaguar) family company, today announced that the European Patent Office and IP Australia, the Australian patent office, have each issued a new patent to Napo. The European patent covers methods for treating SBS, bile acid diarrhea, and diarrhea associated with small intestine resection or gallbladder removal, in patients with an inhibitor of chloride-ion transport such as crofelemer, Napo's novel plant-based prescription drug. The Australian patent covers methods of preventing, ameliorating and/or treating diarrhea associated with CDDs using a proanthocyanidin polymer composition such as crofelemer. "We are very happy that these patents have been issued and believe that they further enhance our intellectual property protection for crofelemer for our core rare disease target indications of SBS and MVID," said Lisa Conte, Jaguar's founder, president, and CEO. "As previously announced, Jaguar, with strong leadership and participation from Jaguar family companies Napo and Napo Therapeutics, is supporting investigator-initiated proof-of-concept studies of crofelemer for SBS with intestinal failure and MVID in the US, EU and Middle East/North Africa (MENA) regions, with results expected in 2024 and 2025," Conte said. "In accordance with the guidelines of specific EU countries, published data from clinical investigations in SBS and MVID could support early patient access to crofelemer for these debilitating conditions in those countries." Some SBS patients are subject to intestinal failure, often requiring parenteral nutrition (PN) from a few days 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. MVID, an ultrarare CDD, is a severe infantile disease characterized by diarrhea, malabsorption, and acid/base instability, requiring intensive parenteral support for nutritional and fluid management, and there are currently no approved drug treatments. As with all potential follow-on indications, Jaguar and Napo prioritize IP protection. Napo currently holds approximately 152 patents and approximately 43 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 (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. Crofelemer also is the subject of Napo's phase 3 OnTarget clinical trial for preventive treatment of cancer therapy-related diarrhea (CTD). 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 the Make Cancer Less Shitty patient advocacy program at makecancerlessshitty.com and on X, Facebook & 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 proof-of-concept studies of crofelemer for SBS with intestinal failure and MVID will be available in 2024 and 2025, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from such clinical investigations could support 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

Jaguar's novel plant-based prescription drug crofelemer has been granted Orphan Drug Designation by the FDA and the European Medicines Agency for both MVID and SBS SAN FRANCISCO, CA / ACCESSWIRE / May 9, 2024 / Jaguar Health, Inc. (NASDAQ:JAGX) ("Jaguar") today announced that Jaguar family companies Napo Pharmaceuticals ("Napo") and Napo Therapeutics have submitted a Clinical Trial Application (CTA) to the Italian and German health authorities to initiate a phase 2 clinical trial of Jaguar's novel plant-based prescription drug crofelemer for treatment of short bowel syndrome (SBS) with intestinal failure in adults. Napo Therapeutics has also submitted a CTA to AIFA, the Italian health authority, for the conduct of a clinical trial in pediatric patients with microvillus inclusion disease (MVID), and the approval request process is also underway for this trial in the United Arab Emirates with Dubai Health. The MVID clinical trial is being conducted under an active US Investigational New Drug (IND) in the US. "We're very pleased that these important CTAs have been submitted," said Lisa Conte, Jaguar's president and CEO. "This is a key regulatory milestone in our ongoing development efforts for a novel powder formulation of crofelemer for oral solution for patients with MVID and SBS with intestinal failure - two devastating and often catastrophic rare diseases for patients, who are frequently on parenteral nutrition for as long as 20 hours a day, seven days a week." "As previously announced, Jaguar, with strong leadership and participation from Napo Pharmaceuticals and Napo Therapeutics, is also supporting investigator-initiated proof-of-concept studies of crofelemer for MVID and SBS with intestinal failure in the US, EU and Middle East/North Africa (MENA) regions, with proof-of-concept results expected in 2024. Additionally, in August 2023 the FDA activated Napo Pharmaceuticals' Investigational New Drug (IND) application for a novel formulation of crofelemer for the treatment of MVID, which allows us to initiate our planned US-based phase 2 trial of crofelemer for treatment of this indication in pediatric MVID patients," Conte said. "In accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID and SBS could support early patient access to crofelemer for these debilitating conditions in those countries." MVID, an ultra-rare congenital diarrheal disorder, is a severe infantile disease characterized by diarrhea, malabsorption, and acid/base instability, requiring intensive parenteral support for nutritional and fluid management, and there are currently no approved drug treatments. Some SBS patients are subject to intestinal failure, often requiring parenteral nutrition (PN) from a few days 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. 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, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID and SBS could support early patient access to crofelemer for these conditions in those 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 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. Contact: hello@jaguar.health Jaguar-JAGX SOURCE: Jaguar Health, Inc. View the original press release on accesswire.com

Jaguar's novel plant-based prescription drug crofelemer has been granted Orphan Drug Designation by the FDA and the European Medicines Agency for both MVID and short bowel syndrome (SBS) with intestinal failure Proof-of-concept studies of crofelemer for MVID and SBS planned for 2024 SAN FRANCISCO, CA / ACCESSWIRE / April 29, 2024 / Jaguar Health, Inc. (Nasdaq:JAGX) today announced that Jaguar family company Napo Pharmaceuticals (Napo) is a Bronze Sponsor of the 10th Annual Elite Ped-GI Congress, which takes place May 2-4, 2024 in Abu Dhabi in the United Arab Emirates (UAE). Additionally, at the conference Napo is sponsoring a panel discussion titled "Masterclass in Congenital Diarrhea" which will focus on microvillus inclusion disease (MVID), an ultra-rare congenital diarrheal disorder. Jaguar's novel plant-based prescription drug crofelemer has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for both MVID and short bowel syndrome (SBS) with intestinal failure. "As previously announced, Jaguar, with strong leadership and participation from Jaguar family companies Napo Pharmaceuticals and Napo Therapeutics, is supporting investigator-initiated proof-of-concept studies of crofelemer for the rare disease indications of MVID and SBS with intestinal failure in the US, EU and Middle East/North Africa (MENA) regions, with results expected in 2024. Additionally, in August 2023 the FDA activated Napo Pharmaceuticals' Investigational New Drug (IND) application for a novel formulation of crofelemer for the treatment of MVID, which allows us to initiate our planned phase 2 trial of crofelemer for treatment of this indication in pediatric MVID patients," said Lisa Conte, Jaguar's president and CEO. "In accordance with the guidelines of specific EU countries, published data from clinical investigations in such rare diseases could support early patient access to crofelemer for these debilitating conditions in those countries." MVID is a severe infantile disease characterized by diarrhea, malabsorption, and acid/base instability, requiring intensive parenteral support for nutritional and fluid management, and there are currently no approved drug treatments. Dr. Mohamad Miqdady, a member of Napo's Scientific Advisory Board (SAB), will serve as the moderator of the Napo-sponsored panel discussion. The session panelists include Pravin Chaturvedi, PhD, who is Jaguar's Chief Scientific Officer and Chair of the Napo SAB, as well as Dr. Christos Tzivinikos and Dr. Antonella Diamanti, who are leading experts in the treatment of MVID patients in the UAE and Italy. Dr. Miqdady is the Division Chief of the Pediatric Gastroenterology, Hepatology & Nutrition Division at Sheikh Khalifa Medical City, a flagship tertiary hospital in the UAE and the largest teaching medical center in Abu Dhabi. He is also an Adjunct Professor at Khalifa University's medical school in Abu Dhabi, and completed his Fellowship in Pediatric Gastroenterology at Baylor College of Medicine and Texas Children's Hospital in Houston. Dr. Christos Tzivinikos is a Consultant Pediatric Gastroenterologist and Founder of the Pediatric GI Department at Al Jalila Children's Specialty Hospital in Dubai, and an Adjunct Clinical Assistant Professor at Mohammed Bin Rashid University of Medicine and Health Sciences in Dubai. Dr. Diamanti is Head of the Artificial Nutrition Unit at Bambino Gesù Children's Hospital in Rome, Italy. She has served as Medical Director of the hospital's Gastroenterology Department since 2001. Dr. Tzivinikos and Dr. Diamanti will be clinical investigators in Napo's planned phase 2 MVID study, titled Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants with Microvillus Inclusion Disease (MVID). About the Elite Ped-GI Congress This 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. The annual event provides a venue for healthcare professionals to share their best practices, to network within groups of interest, and to create and strengthen clinical collaborations. Additional information about the Elite Ped-GI Congress can be viewed here on the event website. 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 innovative, patient-centric therapeutic solutions for essential supportive care and the management of neglected side effects across complicated disease states. Napo's goal is to redefine what is possible in supportive care, providing hope and improving outcomes for patients worldwide. Napo's 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 investigator-initiated and IND proof-of-concept studies of crofelemer for MVID and SBS with intestinal failure will be available in 2024, the expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations of crofelemer for MVID and SBS could support early patient access to crofelemer for these rare diseases in those countries, and the expectation that Jaguar will conduct a phase 2 clinical study of crofelemer for the treatment of 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 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 Jaguar-JAGX SOURCE: Jaguar Health, Inc. View the original press release on accesswire.com