Jaguar Health's crofelemer, initially developed as a diarrhea drug, has shown statistically significant benefits in a prespecified subgroup of breast cancer patients within the Phase III OnTarget trial (NCT04538625). The findings suggest that crofelemer could help these patients better adhere to their cancer therapies by managing diarrhea induced by chemotherapy, immunotherapy, or targeted drug therapy.
The OnTarget trial, conducted by Jaguar's family company Napo Pharmaceuticals, initially failed to meet its primary endpoint of reducing diarrhea frequency across all tumor types. However, the company noted clinically relevant signals in prespecified subgroups, including patients with breast and respiratory cancers.
Statistical Significance in Breast Cancer Subgroup
In a recent announcement, Jaguar Health revealed that a prespecified subgroup of adult patients with breast cancer who were treated with crofelemer achieved statistical significance in the primary endpoint. This suggests that crofelemer could play a crucial role in managing diarrhea, a common and debilitating side effect of cancer treatments.
Lisa Conte, Jaguar’s president and CEO, stated that the data from the breast cancer subgroup has been submitted to an oncology conference by the study’s primary investigators. A full study report is expected to be submitted to a peer-reviewed journal. The company is also conducting additional analyses of other prespecified subgroups from the OnTarget trial, with potential future submissions to peer-reviewed forums.
Context of Diarrhea in Cancer Treatment
Diarrhea is a frequent adverse event in patients undergoing chemotherapy, immunotherapy, or targeted drug therapy. Current treatments include drugs like loperamide (Imodium) and IV fluids for rehydration. Crofelemer offers a different mechanism of action and could provide an alternative or adjunctive therapy to manage this condition.
Trial Details and Patient Population
The OnTarget trial included nearly 180 breast cancer patients out of a total of 287 adult participants with solid tumors receiving targeted therapy with or without standard chemotherapy. This subgroup analysis provides valuable insights into the potential benefits of crofelemer in this specific patient population.
Crofelemer's Existing Approvals and Future Directions
Crofelemer is already FDA-approved for symptomatic relief of non-infectious diarrhea in adults with HIV/AIDS on antiretroviral therapy, marketed as Fulyzaq or Mytesi. The drug has also shown clinically meaningful benefits in patients with diarrhea-predominant irritable bowel syndrome (IBS-D), specifically functional and chronic idiopathic diarrhea. Data related to IBS-D will be presented at the American College of Gastroenterology Annual Scientific Meeting.
