JAGX News & Events

Jaguar Health family company Napo Pharmaceuticals announced the acceptance of two late breaking abstracts for the treatment of pediatric IF from MVID and SBS at the 58th Annual Meeting of ESPGHAN. These pediatric IF patients are receiving liquid oral crofelemer as an investigational drug under two separate investigator-initiated trials. One infant MVID IF patient is being treated with crofelemer as adjunctive therapy to parenteral support for more than 6 months and other pediatric patients with IF due to MVID and/or SBS received crofelemer adjunctive therapy for over 1 year together with reduced amounts of parenteral support. "We are pleased with these results for the safety and effectiveness of liquid oral crofelemer as adjunctive therapy to PS pediatric IF patients on life-sustaining PS. MVID is a congenital enteropathy and has a lethal natural history if not treated with life-sustaining and lifelong total parenteral nutrition with or without supplemental intravenous fluids. MVID is estimated to have a prevalence of approximately 200 patients, and to the best of our knowledge, crofelemer is being used to treat approximately 4% of the prevalent MVID patients, with the inclusion of additional MVID patients in an ongoing blinded study. Crofelemer has demonstrated reductions in parenteral support needs for IF patients with both MVID and SBS. Crofelemer has been well tolerated, and the pediatric IF patients are gaining weight and height, demonstrating reductions in their needs for PS, especially when adjusted for body weight. This is important because TPN is associated with significant comorbidities and reductions in PS needs are expected to improve clinical outcomes as well as quality of life measures for these pediatric IF patients," said Dr. Pravin Chaturvedi, Napo's CSO. Napo is continuing to enroll adult patients with IF due to SBS using the same formulation of crofelemer. This randomized double-blind placebo-controlled trial is being conducted at multiple sites in Germany and Italy. An investigational study in adult SBS-IF patients is also being partially supported by the company in the US.

Jaguar Health plans to use AI platforms to enhance its development, commercialization, and healthcare delivery objectives for crofelemer programs for the treatment of intestinal failure in both adult and pediatric patients. "Our IF program in MVID could leverage AI platforms that could help guide personalized regimens for each patient for this ultrarare disease and include real-world evidence to support the effectiveness and benefit of new therapies like crofelemer in mitigating disease progression by potentially providing credible links between genetics, histopathology, and clinical manifestation. AI platforms could lead to more efficient drug development and quicker access to drugs for patients with no alternative treatments for diseases with lethal natural history, such as MVID." said Pravin Chaturvedi, CSO. "We are strategically focused on demonstrating crofelemer's effectiveness and achieving faster commercialization to meet our healthcare delivery objectives for IF indications," said Lisa Conte, CEO. "Our IF development programs continue to make progress in meeting key clinical milestones, including the ground-breaking and consistent reduction of parenteral support of up to 37% in an investigator-sponsored MVID treatment program. We are currently in business development discussions with the goal of bringing in non-dilutive funds from potential partners. We are encouraged to report that our development plan for MVID could include a plan for obtaining Breakthrough Therapy designation from the U.S. Food and Drug Administration in the coming months, which would augment expedited regulatory submission pathways for this pediatric disorder. The very low prevalence of MVID and its lethal natural history without any available therapies underscores the need for rapid and efficient development and commercialization of crofelemer. In addressing this very serious unmet medical need, and we are exploring AI platforms to assist in achieving our objectives."