Jaguar Health (JAGX) Study Result summary

OnTarget trial overview

• The Phase 3 OnTarget trial evaluated crofelemer in 287 adult cancer patients with 10 solid tumor types and 24 targeted agents, with over 75% having breast or respiratory cancers.

• The study was multicenter, patient-centric, randomized, double-blind, placebo-controlled, and included a 12-week treatment phase plus a 12-week extension.

• Patients were enrolled from 49 sites in the US, Argentina, Georgia, Serbia, and Taiwan.

• Patient-reported outcomes on bowel habits were used as the primary endpoint, specifically reduction in average weekly loose/watery stools.

• The trial did not meet its primary endpoint across all tumor types.

Subgroup findings and clinical relevance

• Clinically relevant and meaningful benefits were observed in pre-specified subgroups of breast and respiratory (lung) cancer patients.

• Pre-specified subgroups were chosen due to high incidence of diarrhea from targeted therapies, such as pertuzumab, abemaciclib, and osimertinib, with rates up to 85%.

• Clinically relevant outcomes were defined by patient input, aiming for a reduction to 14 loose stools per week.

• Improvement in diarrhea incidence was observed in these subgroups over the first 12 weeks; extension phase data are pending.

• Crofelemer was well tolerated, with adverse events similar to placebo and mostly related to underlying cancer or therapy.

Next steps and future plans

• Further analysis of both Stage 1 and Stage 2 data will focus on pre-specified and exploratory endpoints, including non-prespecified subgroups.

• Plans to engage with the FDA to explore regulatory options for crofelemer in responsive subgroups.

• Full data from both 12-week periods will be presented and published in peer-reviewed settings.

• Jaguar expects to launch Gelclair for oral mucositis in the US in Q4 2024.

• Additional clinical investigations of crofelemer for orphan indications are ongoing, with proof-of-concept data expected by end of 2024 and into 2025.