Caliway’s CBL-514 Shows Significant, Sustained Pain Reduction in Phase II Dercum’s Disease Trial
Taiwan’s Caliway Biopharmaceuticals (TW: 6919) has reported positive final results from a Phase II trial of CBL-514D, demonstrating statistically significant and sustained reductions in pain associated with Dercum’s Disease, a rare disorder characterized by painful lipomas.
The randomized, single-blind, placebo-controlled CBL-0202DD trial met its primary efficacy endpoint, with CBL-514 producing a significantly greater reduction in pain scores than placebo at Week 20 in both the Full Analysis Set (FAS) and Per-Protocol (PP) populations.
In the FAS population, patients receiving CBL-514 recorded a mean 3.1-point reduction in pain at Week 20, compared with 1.9 points for placebo, producing a statistically significant treatment effect (p=0.005). Pain reductions were also significant at Weeks 16 and 24, with mean reductions of 3.5 and 4.1 points, respectively, compared with 1.7 points at both time points for placebo.
The results were consistent in the PP population. CBL-514 produced a 3.1-point mean reduction at Week 20, compared with 1.4 points for placebo (p=0.0002). At Weeks 16 and 24, mean reductions were 3.5 and 4.1 points, respectively, versus 1.2 points for placebo at both time points. The differences were statistically significant, with p-values of 0.0001 and less than 0.0001, respectively.
According to Caliway, the magnitude of pain reduction increased over successive treatment assessments, indicating a sustained treatment effect through Week 24. An exploratory analysis also found that approximately 90% of patients in the FAS population achieved at least a three-point clinically meaningful pain reduction in more than half of their treated painful lipomas, substantially higher than the placebo group.
CBL-514 was generally well tolerated during the study, with no drug-related serious adverse events reported. The most common treatment-emergent adverse events were mild-to-moderate local injection-site reactions.
Dercum’s Disease is a chronic condition involving painful deposits of subcutaneous adipose tissue, typically affecting the trunk and extremities. Patients can experience persistent pain, tenderness and burning sensations that can significantly affect daily activities. There are currently no therapies specifically approved for Dercum’s Disease, while existing approaches such as surgical removal of lipomas may provide temporary relief.
Caliway cited market research estimating the global Dercum’s Disease market at approximately US$11.3 billion in 2021, with the market projected to reach nearly US$20 billion by 2030.
The company also plans to explore CBL-514D for other adipose tissue disorders, particularly Lipedema. The condition, which predominantly affects women, is associated with abnormal fat accumulation in the lower body and symptoms including pain, swelling and tenderness. Caliway said market and epidemiological research indicates that Lipedema may affect around 10% of women worldwide and represents a potential market exceeding US$30 billion.
Caliway plans to work with clinical experts to assess the feasibility and development strategy for CBL-514D in Lipedema.
CBL-514 is a small-molecule injectable designed to selectively induce apoptosis, or programmed cell death, in adipocytes while reducing subcutaneous fat without causing necrosis or damage to surrounding tissue. The company said more than 544 subjects have participated in clinical studies involving CBL-514, with 10 completed trials reporting positive primary and key secondary efficacy endpoints.
Founded in 2012, Caliway was listed on the Taiwan Stock Exchange in October 2024 and develops therapeutics targeting aesthetic medicine and biomedical applications.