Caliway Biopharmaceuticals has announced final statistical results from its Phase 2 CBL-0202DD clinical trial evaluating CBL-514 for the treatment of Dercum’s disease (DD), a rare disorder characterised by painful lipomas. The randomised, single-blind, placebo-controlled study met its primary efficacy endpoint, with CBL-514 demonstrating statistically significant and clinically meaningful reductions in lipoma-associated pain.

In the Full Analysis Set (FAS), patients treated with CBL-514 recorded a mean pain-score reduction of 3.1 points at Week 20, compared with 1.9 points for placebo, with a statistically significant treatment effect (p=0.005). At Weeks 16 and 24, mean pain-score reductions with CBL-514 were 3.5 and 4.1 points, respectively, compared with 1.7 points at both time points for placebo (p=0.0017 and p<0.0001).

The findings were also confirmed in the Per-Protocol (PP) population. At Week 20, the mean pain-score reduction was 3.1 points with CBL-514 versus 1.4 points with placebo (p=0.0002). At Weeks 16 and 24, reductions of 3.5 and 4.1 points, respectively, were reported for CBL-514, compared with 1.2 points at both time points for placebo (p=0.0001 and p<0.0001).

An exploratory FAS analysis showed that approximately 90 percent of patients receiving CBL-514 achieved a clinically meaningful pain reduction of at least three points in more than half of their treated painful lipomas.

CBL-514 was generally well tolerated during the study, with no drug-related serious adverse events reported. The most frequently reported treatment-emergent adverse events were mild-to-moderate local injection-site reactions.

Caliway stated that the treatment effect increased over the course of treatment, with statistically significant pain reductions observed at Weeks 16, 20 and 24. The company plans to further evaluate CBL-514 in other adipose tissue disorders, including lipedema, in collaboration with clinical experts.

CBL-514 is a patented small-molecule injection designed to selectively induce adipocyte apoptosis and reduce subcutaneous fat at the injection site. The company is also developing CBL-514 for non-surgical subcutaneous fat reduction and moderate-to-severe cellulite, while CBL-514D is being developed for Dercum’s disease.

More than 544 subjects have participated in clinical trials related to CBL-514, according to Caliway. The company said its completed clinical trials with final statistical reports have met their primary and key secondary efficacy endpoints, with safety and tolerability supporting continued development.