Caliway Biopharmaceuticals announced final statistical results on September 21 from a randomized, single-blind, placebo-controlled Phase 2 trial of CBL-514 in Dercum's disease. The trial, designated CBL-0202DD, met its primary efficacy endpoint, the company said, with statistically significant pain reduction in both the full analysis set and the per-protocol population.
In the full analysis set, patients treated with CBL-514 recorded a mean pain-score reduction of 3.1 points at Week 20, against 1.9 points for placebo (p=0.005). At Weeks 16 and 24 the reductions were 3.5 and 4.1 points, against 1.7 points for placebo at both time points (p=0.0017 and p<0.0001). In the per-protocol population, the Week 20 reduction was 3.1 points against 1.4 points for placebo (p=0.0002), and the Week 16 and Week 24 reductions were 3.5 and 4.1 points against 1.2 points for placebo at both points (p=0.0001 and p<0.0001). Caliway said the size of the response continued to increase over the treatment courses.
No drug-related serious adverse events were reported, and the most commonly reported treatment-emergent adverse events were mild-to-moderate local injection-site reactions, with the overall safety profile consistent with expectations for the ongoing clinical program. An exploratory analysis in the full analysis set found that about 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, which Caliway said outperformed placebo.
Caliway said Dercum's disease is a chronic, highly recurrent rare disease marked by abnormal accumulation of subcutaneous adipose tissue in the trunk and extremities, producing painful lipomas. The company said no therapies are approved specifically for the condition, and that surgical removal provides only temporary symptomatic relief. It cited market research reports estimating the global Dercum's disease market at about US$11.3 billion in 2021 and projecting about US$19.96 billion by 2030, a compound annual growth rate of about 6.76%.
Caliway said key opinion leaders and potential pharmaceutical partners have recognized CBL-514's potential in other adipose tissue disorders that share clinical characteristics with Dercum's disease, including lipedema, which affects about 10% of women worldwide and represents a potential market opportunity exceeding US$30 billion. The company said there are no approved curative therapies for lipedema and that weight loss alone does not address the underlying fat accumulation. Caliway said it plans to work with clinical experts to assess the feasibility and clinical development strategy for CBL-514 in lipedema.
