Latest news with #MVID
USA Today
30-06-2025
- Business
- USA Today
Proof-of-Concept Results Show Jaguar Health's Crofelemer Reduced Total Parenteral Nutrition in Third Intestinal Failure Orphan Disease Patient
As recently announced, initial proof-of-concept results from this ongoing investigator-initiated trial (IIT) show crofelemer reduced the required total parenteral nutrition in the first participating microvillus inclusion disease (MVID) patient by up to 27% and in the first participating short bowel syndrome patient by up to 12.5% The first two patients in this IIT were taken off crofelemer after 12 weeks of treatment for a period intended to last 30 days, per the study protocol, but were promptly placed back on daily crofelemer treatment because their symptoms worsened Company strategy: Seek business development partnerships for license to develop and commercialize Jaguar's intestinal failure products, resulting in non-dilutive funding for Jaguar Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics today announced that initial proof-of-concept results show that a novel liquid formulation of crofelemer reduced the required total parenteral nutrition (TPN) and supplementary intravenous fluids in a third intestinal failure patient. This is the second pediatric patient with intestinal failure due to the orphan disease short bowel syndrome (SBS-IF) who was treated with crofelemer. To date, three patients with intestinal failure due to SBS-IF or microvillus inclusion disease (MVID) have been treated with crofelemer in this exploratory, single-arm open label non-randomized IIT in Abu Dhabi. As announced, and as presented April 26, 2025 at the Annual ELITE PED-GI Congress, initial proof-of-concept results from this study show that crofelemer, Jaguar's novel plant-based anti-secretory prescription drug, reduced the required TPN and supplementary intravenous fluids in the first participating MVID patient by up to 27% and in the first participating SBS-IF patient by up to 12.5%. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output – an indicator of improved nutrient oral absorption. 'We're very pleased to report that the second pediatric SBS-IF patient participating in this IIT exhibited a similar response for TPN reduction, further exemplifying that crofelemer's paradigm-shifting antisecretory mechanism of action may have the potential to provide a novel therapeutic option to modify disease progression due to the reductions in TPN and associated comorbidities in MVID and SBS-IF patients and improve their quality of life,' said Lisa Conte, Jaguar's Founder and CEO. 'This SBS-IF patient was administered the highest dose of crofelemer provided for in the approved protocol for the IIT, with no resulting safety issues.' Per the IIT protocol, as announced, the initial pediatric MVID patient participating in the study was taken off crofelemer after 12 weeks of treatment for an intended period of 30 days. However, after just 8 days, the patient's parents requested reinitiation of crofelemer dosing, as the patient's symptoms worsened – evidenced by increased stool output and decreased urine output – and the patient was restarted on daily treatment with crofelemer. The initial pediatric SBS-IF patient participating in the study also had crofelemer withdrawn per the protocol after 12 weeks. This patient then exhibited a relapse of symptoms and was put back on daily treatment with crofelemer, and has since been maintaining a significant TPN reduction. Jaguar plans to continue supplying crofelemer to Dr. Mohamad Miqdady, the principal investigator for this IIT, for the patients participating in this study for as long as Dr. Miqdady feels is medically necessary. Dr. Miqdady has submitted initial proof-of-concept data from the ongoing IIT in Abu Dhabi for all three above-referenced patients to a major international pediatric gastroenterological conference for consideration for presentation. Jaguar, through Jaguar family companies Napo and Napo Therapeutics, is currently supporting two independent proof-of-concept IITs, and conducting two placebo-controlled Phase 2 studies, of crofelemer in patients with intestinal failure due to MVID and SBS-IF in the United States, European Union, and/or Middle East/North Africa regions. Completion of Napo's randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients is expected in mid-2026 as planned. 'Our strategy is to seek business development partnerships for license rights for development and commercialization of Jaguar's intestinal failure products – with the goal of generating non-dilutive funding for Jaguar,' said Conte. 'Given the ultrarare nature of MVID, and the groundbreaking initial proof-of-concept results from the ongoing IIT in Abu Dhabi, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval.' Based on the initial findings of the ongoing IIT in Abu Dhabi, crofelemer's paradigm-shifting antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to reduce TPN and associated complications, including liver, renal, and cognitive deficits, as well as infections from IV infusion, in patients with intestinal failure due to MVID and short bowel syndrome. The observed groundbreaking TPN reduction is particularly compelling for MVID, an ultrarare pediatric disease characterized by severe diarrhea and malabsorption that requires intensive parenteral support for nutritional and fluid management and for which no approved drug treatments exist, or any potential approach to reduce TPN. The initial proof-of-concept data in MVID supports crofelemer's potential inclusion in the European Medicines Agency's (EMA) PRIME program that may accelerate regulatory approval pathways in the EU for this indication. This data may also support qualification of crofelemer for the FDA's Breakthrough Therapy program for expedited regulatory approval in the US. Additional proof-of-concept results from IITs are expected throughout 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs. 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. The IIT in Abu Dhabi is being conducted by Dr. Miqdady at Sheikh Khalifa Medical City (SKMC), a flagship tertiary hospital in the United Arab Emirates and the largest teaching medical center in Abu Dhabi. Dr. Miqdady, a recognized leader in pediatric gastroenterology, serves as the Chief of Pediatric Gastroenterology, Hepatology and Nutrition at SKMC. He is an American board-certified pediatric GI, hepatology and nutrition professor at Khalifa University in Abu Dhabi, and also serves as a member of Napo's Scientific Advisory Board. Dr. Miqdady 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 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. 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 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 Magdalena Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute 'forward-looking statements.' These include statements regarding Jaguar's expectation that completion of Napo's Phase 2 study of crofelemer in pediatric MVID patients will occur mid-2026, Jaguar's expectation that its strategy of seeking business development partnerships for license rights for development and commercialization of Jaguar's intestinal failure products may support generation of non-dilutive funding for Jaguar, Jaguar's expectation that even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval of crofelemer for MVID, Jaguar's expectation that crofelemer's mechanism of action may have the potential to provide a novel therapeutic option to reduce TPN and associated complications, including liver, renal, and cognitive deficits, as well as infections from IV infusion, in pediatric MVID and SBS-IF patients, Jaguar's expectation that proof-of-concept data in MVID may support crofelemer's potential inclusion in the EMA's PRIME program for expediated and assisted regulatory approval and in the FDA's Breakthrough Therapy program for expedited regulatory approval in the US, Jaguar's expectation that additional proof-of-concept results from IITs will be available throughout 2025, and Jaguar's expectation that published data from clinical investigations in MVID and SBS-IF could support reimbursed early patient access to crofelemer for MVID and SBS-IF. 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-JAGX SOURCE: Jaguar Health, Inc. View the original press release on ACCESS Newswire
Indianapolis Star
23-06-2025
- Business
- Indianapolis Star
Jaguar Health Provides Updates on Orphan Disease Intestinal Failure Development Program for Crofelemer
Enrollment in company's first-of-its-kind placebo-controlled Phase 2 study to evaluate the efficacy of crofelemer for microvillus inclusion disease (MVID) in pediatric patients has reached approximately 25% As recently announced, initial proof-of-concept results of the ongoing investigator-initiated trial (IIT) show crofelemer reduced the required total parenteral nutrition in patients with intestinal failure due to MVID and short bowel syndrome by up to 27% and 12.5% respectively; data from the third patient enrolled is expected Company strategy: Seek business development partnerships for license to develop and commercialize Jaguar's intestinal failure products, resulting in non-dilutive funding for Jaguar SAN FRANCISCO, CA / ACCESS Newswire / June 23, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) today provided updates on the company's orphan disease intestinal failure program. Jaguar, through Jaguar family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics, is currently supporting two independent proof-of-concept investigator-initiated trials (IITs), and conducting two placebo-controlled Phase 2 studies, of crofelemer, Jaguar's novel plant-based anti-secretory prescription drug, in patients with intestinal failure due to microvillus inclusion disease (MVID) and short bowel syndrome (SBS-IF) in the United States, European Union, and/or Middle East/North Africa regions. As announced, and as presented April 26, 2025 at the Annual ELITE PED-GI Congress, initial proof-of-concept results from the ongoing exploratory, single-arm open label non-randomized IIT of crofelemer in Abu Dhabi in pediatric intestinal failure patients show that crofelemer reduced the required total parenteral nutrition (TPN) and supplementary intravenous fluids in the first participating MVID patient by up to 27% and in the first participating SBS-IF patient by up to 12.5%. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output – an indicator of improved nutrient oral absorption. Data from the third patient enrolled in the IIT is expected. Completion of Napo's randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients is expected in mid-2026 as planned. 'Our strategy is to seek business development partnerships for license rights for development and commercialization of Jaguar's intestinal failure products – with the goal of generating non-dilutive funding for Jaguar,' said Lisa Conte, Jaguar's Founder and CEO. 'I attended the BIO International Convention in Boston last week and took part in productive meetings at the event.' 'Given the ultrarare nature of MVID, and the groundbreaking initial proof-of-concept results from the ongoing IIT in Abu Dhabi, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval,' said Conte. 'We're excited to report that enrollment in the company's first-of-its-kind placebo-controlled Phase 2 study to evaluate the efficacy of crofelemer for MVID in pediatric patients is at approximately 25% and patient screening is continuing. For the company's placebo-controlled Phase 2 study to evaluate the efficacy of crofelemer for SBS-IF in adults, enrollment is above 10%, and patient screening is continuing,' said Conte. 'Additionally, enrollment is continuing in the two ongoing proof-of-concept IITs. These are important milestones in development efforts for crofelemer for the treatment and management of intestinal failure related to these devastating orphan diseases and will continue to generate IIT data.' Based on the initial findings of the ongoing IIT in Abu Dhabi, crofelemer's paradigm-shifting antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to reduce TPN and associated complications, including liver, renal, and cognitive deficits, as well as infections from IV infusion, in patients with intestinal failure due to MVID and short bowel syndrome. The observed groundbreaking TPN reduction is particularly compelling for MVID, an ultrarare pediatric disease characterized by severe diarrhea and malabsorption that requires intensive parenteral support for nutritional and fluid management and for which no approved drug treatments exist, or any potential approach to reduce TPN. The initial proof-of-concept data in MVID supports crofelemer's potential inclusion in the European Medicines Agency's (EMA) PRIME program that may accelerate regulatory approval pathways in the EU. This data may also support qualification of crofelemer for the FDA's Breakthrough Therapy program for expedited regulatory approval in the US. Additional proof-of-concept results from IITs are expected throughout 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs. 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. 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. 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 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 Magdalena Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute 'forward-looking statements.' These include statements regarding Jaguar's expectation that completion of Napo's Phase 2 study of crofelemer in pediatric MVID patients will occur mid-2026, Jaguar's expectation that the two ongoing proof-of-concept IITs will continue to generate data, Jaguar's expectation that data from the third patient enrolled in the IIT in Abu Dhabi is expected, Jaguar's expectation that its strategy of seeking business development partnerships for license rights for development and commercialization of Jaguar's intestinal failure products may support generation of non-dilutive funding for Jaguar, Jaguar's expectation that even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval of crofelemer for MVID, Jaguar's expectation that crofelemer's mechanism of action may have the potential to provide a novel therapeutic option to reduce TPN and associated complications, including liver, renal, and cognitive deficits, as well as infections from IV infusion, in pediatric MVID and SBS-IF patients, Jaguar's expectation that proof-of-concept data in MVID may support crofelemer's potential inclusion in the EMA's PRIME program for expediated and assisted regulatory approval and in the FDA's Breakthrough Therapy program for expedited regulatory approval in the US, Jaguar's expectation that additional proof-of-concept results from IITs will be available throughout 2025, and Jaguar's expectation that published data from clinical investigations in MVID and SBS-IF could support reimbursed early patient access to crofelemer for MVID and SBS-IF. 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-JAGX SOURCE: Jaguar Health, Inc.
Miami Herald
23-06-2025
- Business
- Miami Herald
Jaguar Health Provides Updates on Orphan Disease Intestinal Failure Development Program for Crofelemer
Enrollment in company's first-of-its-kind placebo-controlled Phase 2 study to evaluate the efficacy of crofelemer for microvillus inclusion disease (MVID) in pediatric patients has reached approximately 25% As recently announced, initial proof-of-concept results of the ongoing investigator-initiated trial (IIT) show crofelemer reduced the required total parenteral nutrition in patients with intestinal failure due to MVID and short bowel syndrome by up to 27% and 12.5% respectively; data from the third patient enrolled is expected Company strategy: Seek business development partnerships for license to develop and commercialize Jaguar's intestinal failure products, resulting in non-dilutive funding for Jaguar SAN FRANCISCO, CA / ACCESS Newswire / June 23, 2025 / Jaguar Health, Inc.(NASDAQ:JAGX) (Jaguar) today provided updates on the company's orphan disease intestinal failure program. Jaguar, through Jaguar family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics, is currently supporting two independent proof-of-concept investigator-initiated trials (IITs), and conducting two placebo-controlled Phase 2 studies, of crofelemer, Jaguar's novel plant-based anti-secretory prescription drug, in patients with intestinal failure due to microvillus inclusion disease (MVID) and short bowel syndrome (SBS-IF) in the United States, European Union, and/or Middle East/North Africa regions. As announced, and as presented April 26, 2025 at the Annual ELITE PED-GI Congress, initial proof-of-concept results from the ongoing exploratory, single-arm open label non-randomized IIT of crofelemer in Abu Dhabi in pediatric intestinal failure patients show that crofelemer reduced the required total parenteral nutrition (TPN) and supplementary intravenous fluids in the first participating MVID patient by up to 27% and in the first participating SBS-IF patient by up to 12.5%. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output - an indicator of improved nutrient oral absorption. Data from the third patient enrolled in the IIT is expected. Completion of Napo's randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients is expected in mid-2026 as planned. "Our strategy is to seek business development partnerships for license rights for development and commercialization of Jaguar's intestinal failure products - with the goal of generating non-dilutive funding for Jaguar," said Lisa Conte, Jaguar's Founder and CEO. "I attended the BIO International Convention in Boston last week and took part in productive meetings at the event." "Given the ultrarare nature of MVID, and the groundbreaking initial proof-of-concept results from the ongoing IIT in Abu Dhabi, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval," said Conte. "We're excited to report that enrollment in the company's first-of-its-kind placebo-controlled Phase 2 study to evaluate the efficacy of crofelemer for MVID in pediatric patients is at approximately 25% and patient screening is continuing. For the company's placebo-controlled Phase 2 study to evaluate the efficacy of crofelemer for SBS-IF in adults, enrollment is above 10%, and patient screening is continuing," said Conte. "Additionally, enrollment is continuing in the two ongoing proof-of-concept IITs. These are important milestones in development efforts for crofelemer for the treatment and management of intestinal failure related to these devastating orphan diseases and will continue to generate IIT data." Based on the initial findings of the ongoing IIT in Abu Dhabi, crofelemer's paradigm-shifting antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to reduce TPN and associated complications, including liver, renal, and cognitive deficits, as well as infections from IV infusion, in patients with intestinal failure due to MVID and short bowel syndrome. The observed groundbreaking TPN reduction is particularly compelling for MVID, an ultrarare pediatric disease characterized by severe diarrhea and malabsorption that requires intensive parenteral support for nutritional and fluid management and for which no approved drug treatments exist, or any potential approach to reduce TPN. The initial proof-of-concept data in MVID supports crofelemer's potential inclusion in the European Medicines Agency's (EMA) PRIMEprogram that may accelerate regulatory approval pathways in the EU. This data may also support qualification of crofelemer for the FDA's Breakthrough Therapyprogram for expedited regulatory approval in the US. Additional proof-of-concept results from IITs are expected throughout 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs. 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. About CrofelemerCrofelemer 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 CompaniesJaguar 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. 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 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 Pharmaceuticals, visit Therapeutics, visit Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking StatementsCertain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that completion of Napo's Phase 2 study of crofelemer in pediatric MVID patients will occur mid-2026, Jaguar's expectation that the two ongoing proof-of-concept IITs will continue to generate data, Jaguar's expectation that data from the third patient enrolled in the IIT in Abu Dhabi is expected, Jaguar's expectation that its strategy of seeking business development partnerships for license rights for development and commercialization of Jaguar's intestinal failure products may support generation of non-dilutive funding for Jaguar, Jaguar's expectation that even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval of crofelemer for MVID, Jaguar's expectation that crofelemer's mechanism of action may have the potential to provide a novel therapeutic option to reduce TPN and associated complications, including liver, renal, and cognitive deficits, as well as infections from IV infusion, in pediatric MVID and SBS-IF patients, Jaguar's expectation that proof-of-concept data in MVID may support crofelemer's potential inclusion in the EMA's PRIME program for expediated and assisted regulatory approval and in the FDA's Breakthrough Therapy program for expedited regulatory approval in the US, Jaguar's expectation that additional proof-of-concept results from IITs will be available throughout 2025, and Jaguar's expectation that published data from clinical investigations in MVID and SBS-IF could support reimbursed early patient access to crofelemer for MVID and SBS-IF. 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@ SOURCE: Jaguar Health, Inc.
Miami Herald
20-05-2025
- Business
- Miami Herald
First Patient Randomized in Jaguar Health's Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial of Crofelemer in Pediatric Patients with Microvillus Inclusion Disease (MVID)
As announced, initial proof-of-concept (POC) results from distinct ongoing investigator-initiated trial (IIT) in Abu Dhabi show crofelemer reduced total parenteral support in a pediatric MVID patient with intestinal failure by up to 27%; per the IIT protocol, patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days, but was restarted on daily crofelemer treatment after just 8 days, as patient's symptoms were worsening Additional POC IIT results expected throughout 2025 for MVID; results from company's MVID Phase 2 study expected with completion of treatment in H2 2026 Ground-breaking results in single-digit number of MVID patients could potentially lead to expedited approval in the EU through PRIME and to Breakthrough Therapy designation in the US SAN FRANCISCO, CA / ACCESS Newswire / May 20, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals, Inc. (Napo) and Napo Therapeutics S.p.A. today announced that the first patient has been randomized in Napo's randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety and efficacy of the novel crofelemer oral powder for solution formulation in pediatric patients with intestinal failure due to the ultrarare disease MVID. Patients with MVID suffer from devastating diarrhea and dehydration caused by this debilitating, lifelong condition. These pediatric patients often require lifelong and life-sustaining total parenteral nutrition (TPN) with additional supplemental intravenous (IV) fluids, termed total parenteral support, which is the standard of care for MVID, for up to 7 days a week greater than 50% of the time each day to provide adequate nutrient and electrolyte support. While crucial for MVID patients, TPN carries a significant risk of morbidity and mortality due to infections, metabolic complications, liver and/or kidney disease, and a risk of neurodevelopmental delay. There are currently no approved drug treatments for MVID. As previously announced, and as presented last month at the 11th Annual ELITE PED-GI Congress in Abu Dhabi in the United Arab Emirates, the initial proof-of-concept results from the ongoing and independent proof-of-concept investigator-initiated trial (IIT) at Sheikh Khalifa Medical City (SKMC) showed that crofelemer reduced the required TPN and/or supplementary intravenous fluids - collectively referred to as parenteral support - in the first participating MVID patient by up to 27% at the end of 12-weeks of crofelemer treatment. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output - an indicator of improved nutrient oral absorption. Per the protocol for the IIT at SKMC, the pediatric MVID patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days. After just 8 days, the patient's parents requested reinitiation of crofelemer dosing, as the patient's symptoms were worsening - evidenced by increased stool output and decreased urine output. Hence, the patient was restarted on daily treatment with crofelemer. "We're very happy that the first patient has been randomized in Napo's randomized, double-blind placebo-controlled Phase 2 clinical trial of crofelemer in pediatric MVID patients," said Lisa Conte, Jaguar's founder, president, and CEO. "This is another important milestone in development efforts for crofelemer for the treatment and management of intestinal failure related to this devastating ultrarare pediatric disease. For this study, randomization represents the key milestone of assigning a participating patient to either the crofelemer or placebo treatment group in a double-blind, placebo-controlled within-patient crossover study." "Additional proof-of-concept results for MVID from the ongoing IIT of crofelemer at SKMC in pediatric patients with intestinal failure due to MVID and other disorders are expected throughout 2025," Conte said. "The results of Napo's randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients are expected to be available in the second half of 2026. Because the IIT of crofelemer for MVID is an unblinded, open-label study, results from this IIT are expected to be available sooner." Based on the initial proof-of-concept findings presented last month at the 11th Annual ELITE PED-GI Congress, crofelemer's novel antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients, as evidenced by the reductions in total parenteral support and potential to improve the quality of life of MVID patients. "Based on preliminary communications with European Medicines Agency (EMA) regulatory members that focus on PRIME (Priority Medicines), a program that provides enhanced interactions and early dialogue for development of novel medicines targeting unmet medical needs, we are excited by the initial findings from the IIT being conducted at SKMC," said Conte. "We are not aware of any other intervention that has shown the ability to reduce the required administration of life-sustaining parenteral support including TPN, thus potentially improving nutrient absorption in patients with MVID. Given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the European Union. Crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the U.S. Food and Drug Administration's (FDA) Breakthrough Therapy program for this ultrarare pediatric indication. Additionally, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition." 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 Pharmaceuticals, visit Therapeutics, visit Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that additional POC results for MVID from the IIT in pediatric patients with intestinal failure due to MVID and other disorders may be available throughout 2025, Jaguar's expectation that results from the company's MVID Phase 2 study will be available with completion of treatment in H2 2026, Jaguar's expectation that crofelemer's novel antisecretory mechanism of action may have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients and the potential to improve the quality of life of MVID patients, Jaguar's expectation that, given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the Europe Union, Jaguar's expectation that crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the FDA's Breakthrough Therapyprogram, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition. 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@ SOURCE: Jaguar Health, Inc.
Associated Press
20-05-2025
- Business
- Associated Press
First Patient Randomized in Jaguar Health's Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial of Crofelemer in Pediatric Patients with Microvillus Inclusion Disease (MVID)
As announced, initial proof-of-concept (POC) results from distinct ongoing investigator-initiated trial (IIT) in Abu Dhabi show crofelemer reduced total parenteral support in a pediatric MVID patient with intestinal failure by up to 27%; per the IIT protocol, patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days, but was restarted on daily crofelemer treatment after just 8 days, as patient's symptoms were worsening Additional POC IIT results expected throughout 2025 for MVID; results from company's MVID Phase 2 study expected with completion of treatment in H2 2026 Ground-breaking results in single-digit number of MVID patients could potentially lead to expedited approval in the EU through PRIME and to Breakthrough Therapy designation in the US SAN FRANCISCO, CA / ACCESS Newswire / May 20, 2025 / Jaguar Health, Inc. ( NASDAQ:JAGX ) (Jaguar) family companies Napo Pharmaceuticals, Inc. (Napo) and Napo Therapeutics S.p.A. today announced that the first patient has been randomized in Napo's randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety and efficacy of the novel crofelemer oral powder for solution formulation in pediatric patients with intestinal failure due to the ultrarare disease MVID. Patients with MVID suffer from devastating diarrhea and dehydration caused by this debilitating, lifelong condition. These pediatric patients often require lifelong and life-sustaining total parenteral nutrition (TPN) with additional supplemental intravenous (IV) fluids, termed total parenteral support, which is the standard of care for MVID, for up to 7 days a week greater than 50% of the time each day to provide adequate nutrient and electrolyte support. While crucial for MVID patients, TPN carries a significant risk of morbidity and mortality due to infections, metabolic complications, liver and/or kidney disease, and a risk of neurodevelopmental delay. There are currently no approved drug treatments for MVID. As previously announced, and as presented last month at the 11th Annual ELITE PED-GI Congress in Abu Dhabi in the United Arab Emirates, the initial proof-of-concept results from the ongoing and independent proof-of-concept investigator-initiated trial (IIT) at Sheikh Khalifa Medical City (SKMC) showed that crofelemer reduced the required TPN and/or supplementary intravenous fluids - collectively referred to as parenteral support - in the first participating MVID patient by up to 27% at the end of 12-weeks of crofelemer treatment. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output - an indicator of improved nutrient oral absorption. Per the protocol for the IIT at SKMC, the pediatric MVID patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days. After just 8 days, the patient's parents requested reinitiation of crofelemer dosing, as the patient's symptoms were worsening - evidenced by increased stool output and decreased urine output. Hence, the patient was restarted on daily treatment with crofelemer. 'We're very happy that the first patient has been randomized in Napo's randomized, double-blind placebo-controlled Phase 2 clinical trial of crofelemer in pediatric MVID patients,' said Lisa Conte, Jaguar's founder, president, and CEO. 'This is another important milestone in development efforts for crofelemer for the treatment and management of intestinal failure related to this devastating ultrarare pediatric disease. For this study, randomization represents the key milestone of assigning a participating patient to either the crofelemer or placebo treatment group in a double-blind, placebo-controlled within-patient crossover study.' 'Additional proof-of-concept results for MVID from the ongoing IIT of crofelemer at SKMC in pediatric patients with intestinal failure due to MVID and other disorders are expected throughout 2025,' Conte said. 'The results of Napo's randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients are expected to be available in the second half of 2026. Because the IIT of crofelemer for MVID is an unblinded, open-label study, results from this IIT are expected to be available sooner.' Based on the initial proof-of-concept findings presented last month at the 11th Annual ELITE PED-GI Congress, crofelemer's novel antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients, as evidenced by the reductions in total parenteral support and potential to improve the quality of life of MVID patients. 'Based on preliminary communications with European Medicines Agency (EMA) regulatory members that focus on PRIME (Priority Medicines), a program that provides enhanced interactions and early dialogue for development of novel medicines targeting unmet medical needs, we are excited by the initial findings from the IIT being conducted at SKMC,' said Conte. 'We are not aware of any other intervention that has shown the ability to reduce the required administration of life-sustaining parenteral support including TPN, thus potentially improving nutrient absorption in patients with MVID. Given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the European Union. Crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the U.S. Food and Drug Administration's (FDA) Breakthrough Therapy program for this ultrarare pediatric indication. Additionally, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition.' 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 Napo Pharmaceuticals, visit Napo Therapeutics, visit Magdalena Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute 'forward-looking statements.' These include statements regarding Jaguar's expectation that additional POC results for MVID from the IIT in pediatric patients with intestinal failure due to MVID and other disorders may be available throughout 2025, Jaguar's expectation that results from the company's MVID Phase 2 study will be available with completion of treatment in H2 2026, Jaguar's expectation that crofelemer's novel antisecretory mechanism of action may have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients and the potential to improve the quality of life of MVID patients, Jaguar's expectation that, given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the Europe Union, Jaguar's expectation that crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the FDA's Breakthrough Therapyprogram, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition. 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: [email protected] Jaguar-JAGX SOURCE: Jaguar Health, Inc. press release
