SAN FRANCISCO, CA – Jaguar Health, Inc. (NASDAQ:JAGX) family company Napo Pharmaceuticals (Napo) today announced that, based on the U.S. Food and Drug Administration (FDA) support for Napo’s protocol amendment for its clinical trial to evaluate the safety and efficacy of its novel crofelemer powder-for-oral-solution formulation in pediatric patients with intestinal failure due to MVID, an ultrarare pediatric disorder, Napo plans to initiate a single-blind extension phase for the trial. The extended treatment period has the potential to improve clinical benefit of crofelemer through reduction of parenteral support (PS) requirements, which may result in disease progression-modification of MVID.
“We are pleased to have the opportunity to continue treatment with crofelemer powder for oral solution in our ongoing clinical trial of crofelemer in pediatric MVID patients,” said Pravin Chaturvedi, PhD, Jaguar’s Chief Scientific Officer and Chair of the Jaguar and Napo Scientific Advisory Board. “This would allow patients who have completed their double-blind treatment period to be eligible for the single-blind phase of continued treatment with crofelemer for further assessment of clinical benefit through reduction of PS for a longer period. PS reduction could potentially reduce co-morbidities associated with the disease and TPN; thus improving clinical benefit and modifying the disease progression to help address the lethal natural history of MVID.”
The dose selection for the single-blind extension phase will be determined by the study’s independent Data Monitoring Committee (DMC), which will ensure that the study clinicians and Napo personnel will remain blinded. Currently, approximately 30% of the participating MVID patients have completed the study’s double-blind treatment phase and the clinical investigators are supporting the patients’ participation in the single-blind extended phase.
This trial of crofelemer in MVID patients has clinical sites in the US, Italy and the UAE, and the consent from the FDA, the European Medicines Agency (EMA), The Ministry of Health and Prevention (MOHAP), and the Institutional Review Boards (IRBs) for each site.
“Our development plan for this ultrarare disease, with an expected improved clinical benefit from the longer treatment in the extension phase, may also support the possibility for a Breakthrough Therapy designation from the FDA in the coming months, potentially supporting opportunities for expedited regulatory submission pathways to the FDA for new drug approval for this ultrarare pediatric disorder,” said Lisa Conte, Jaguar’s founder, president, and CEO. “The extremely low incidence and prevalence of MVID, its lethal natural history and the fact that no therapies are available for MVID other than lifelong PS, underscores the need for new therapies. Together with the support of MVID clinical key opinion leaders (KOLs), we are exploring expedited regulatory pathways for crofelemer.”
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. Jaguar family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics S.p.A. focus on the development and commercialization of novel crofelemer powder for oral solution for the treatment of rare and orphan gastrointestinal disorders with intestinal failure, including MVID and short bowel syndrome.
For more information please visit:
Jaguar Health, visit https://jaguar.health
Napo Pharmaceuticals, visit www.napopharma.com
Napo Therapeutics, visit napotherapeutics.com
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