Caliway Announces Positive Phase 2 Results of CBL-514 in Dercum's Disease, Achieving Primary Endpoint with Statistically Significant and Sustained Pain Reduction

Caliway Biopharmaceuticals (TWSE: 6919) announced final statistical results from its randomized, single-blind, placebo-controlled Phase 2 clinical trial (CBL-0202DD) evaluating CBL-514 for the treatment of Dercum's Disease (DD), a rare and debilitating disorder characterized by painful lipomas. The study successfully achieved its primary efficacy endpoint, with CBL-514 demonstrating clinically meaningful and statistically significant pain reduction in both the Full Analysis Set (FAS) and Per-Protocol (PP) populations. The results showed robust and durable efficacy, with treatment benefits consistently observed across multiple assessment time points and increasing over all treatment courses. The Phase 2 CBL-0202DD trial was designed to evaluate the efficacy and safety of CBL-514 in patients with Dercum's Disease. Final statistical analyses showed clinically significant and sustained reductions in lipoma-associated pain following treatment with CBL-514 , while both primary and secondary efficacy endpoints achieved statistical significance. For the primary efficacy endpoint assessed at Week 20 in the FAS population, patients treated with CBL-514 achieved a mean pain-score reduction of 3.1 points, compared with 1.9 points in the placebo group, representing a statistically significant treatment effect (p=0.005). For the secondary efficacy endpoints assessed at Weeks 16 and 24, patients receiving CBL-514 achieved mean pain-score reductions of 3.5 points and 4.1 points, respectively, compared with a reduction of 1.7 points at both time points in the placebo group. Both comparisons reached statistical significance (p=0.0017 and p<0.0001, respectively). The efficacy findings were further confirmed in the PP population. For the primary efficacy endpoint assessed at Week 20, patients treated with CBL-514 achieved a mean pain-score reduction of 3.1 points, compared with 1.4 points in the placebo group, representing a statistically significant treatment effect (p=0.0002). For the secondary efficacy endpoints assessed at Weeks 16 and 24, patients receiving CBL-514 achieved mean pain-score reductions of 3.5 points and 4.1 points, respectively, compared with a reduction of 1.2 points at both time points in the placebo group. Both comparisons reached statistical significance (p=0.0001 and p<0.0001, respectively). CBL-514 delivered statistically significant and clinically meaningful pain reduction at Weeks 16, 20, and 24. Importantly, the magnitude of response continued to increase over time, supporting the durability of the treatment effect. Notably, an exploratory analysis in the FAS population showed that approximately 90% of patients treated with CBL-514 achieved a clinically meaningful pain reduction of at least 3 points in more than half of their treated painful lipomas, markedly outperforming placebo . From a safety perspective, CBL-514 was generally well tolerated throughout the study. No drug-related serious adverse events (SAEs) were reported. The most commonly reported treatment-emergent adverse events were mild-to-moderate local injection-site reactions, and the overall safety profile was consistent with expectations for ongoing clinical development.