Caliway Biopharmaceuticals (Caliway) announced that all the primary and secondary efficacy endpoints were met in the analysis of the ITT (Intent-to-treat) population and PP (per-protocol) population from the CBL-0201DD Phase 2 study (NCT05387733), evaluating CBL-514's efficacy and safety on participants with Dercum's Disease lipomas. In addition, CBL-514 demonstrated good safety and tolerance profiles, with no systemic adverse events reported. Dercum's disease is a rare disease currently without approved or effective treatment, leading to the clinical need remaining unmet.

According to a published study by Emma Hansson, MD in 2011, liposuction may reduce pain in Dercum's disease by 3 points (evaluated by the Visual Analog Scale) at 3 months after liposuction compared to baseline. Topline Results from the CBL-0201DD Study: In the CBL-0201DD Phase 2 study, 12 participants with at least 4 painful and well-defined lipomas were recruited and randomized to 2 dose groups of CBL-514, with 6 participants per group. Each enrolled participant will receive up to 2 courses of their allocated dose of CBL-514 administered into selected lipomas, which the injection volume per lipoma will be based on the lipoma size, determined by ultrasound.

Primary Endpoint: Painful lipomas dimension in the high-dose group was reduced by 51.3% (p<0.0001) and 54.7% (p<0.0001) at 4 and 8 weeks after CBL-514 treatments.Painful lipomas dimension in the low-dose group was reduced by 40.0% (p=0.0002) and 34.7% (p<0.0001) at 4 and 8 weeks after CBL-514 treatments. Secondary Endpoints: 90.3% of painful lipomas in the high-dose group showed dimensions reduction after CBL-514 treatments compared to baseline. 64.5% of painful lipomas in the high-dose group showed complete clearance or dimensions reduction of more than 50% after CBL-514 treatments compared to baseline.

38.7% of painful lipomas in the high-dose group showed complete clearance after CBL-514 treatments compared to baseline. The mean absolute change in pain score of painful lipomas in the high-dose group, evaluated by the Comparative Pain Scale, was decreased by 4.7 points (p<0.0001) after CBL-514 treatments compared to baseline. The results in painful lipomas in the low-dose group showed statistical significance in primary and all secondary endpoints.

Detailed efficacy and safety topline results from the CBL-0201DD study will be published in the journal for rare diseases. Based on the promising results, Caliway is currently planning for CBL-0202DD Phase 2b study IND application to further investigate CBL-514's efficacy in treating Dercum's disease.