New Taipei City, Taiwan, September 22, 2026
Caliway Biopharmaceuticals has announced positive final results from its randomized, single-blind, placebo-controlled Phase 2 CBL-0202DD clinical trial evaluating CBL-514 in patients with Dercum’s disease, a rare and debilitating disorder associated with painful lipomas. The investigational therapy achieved its primary efficacy endpoint, demonstrating statistically significant reductions in pain compared with placebo in both the Full Analysis Set and Per-Protocol populations. Caliway reported that the treatment effect remained evident across multiple assessment points, with pain reduction increasing through Week 24. The findings provide further clinical evidence supporting continued development of CBL-514 for a disease with significant unmet medical need and no approved pharmacological therapy specifically established for Dercum’s disease.
CBL-514 Achieves Phase 2 Primary Endpoint
The CBL-0202DD Phase 2 trial was designed to assess the efficacy and safety of CBL-514 in people with Dercum’s disease. The condition is characterized by abnormal deposits of painful adipose tissue, commonly involving lipomas, and can cause persistent pain and substantial impairment in daily life. Caliway’s final analysis showed that CBL-514 produced a statistically significant reduction in pain compared with placebo at the study’s primary assessment at Week 20. In the Full Analysis Set, patients receiving CBL-514 experienced a mean pain-score reduction of 3.1 points at Week 20, compared with a 1.9-point reduction for placebo, producing a statistically significant treatment effect with p=0.005. The Per-Protocol analysis produced a 3.1-point reduction for CBL-514 versus 1.4 points for placebo, with p=0.0002. These findings supported the primary endpoint across both analysis populations.
The company also reported sustained improvements at earlier and later assessments. At Week 16, mean pain-score reductions were 3.5 points for CBL-514 versus 1.7 points for placebo in the Full Analysis Set. At Week 24, the reduction reached 4.1 points compared with 1.7 points for placebo. Both comparisons were statistically significant, with the treatment effect becoming larger over the course of follow-up.
Sustained Pain Reduction and Safety Profile
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An exploratory analysis provided additional evidence of the potential treatment response. Caliway reported that approximately 90% of patients treated with CBL-514 achieved a clinically meaningful pain reduction of at least three points in more than half of their treated painful lipomas. The company said the finding was substantially higher than the response observed with placebo. From a safety perspective, CBL-514 was generally well tolerated during the Phase 2 study. Caliway reported that no drug-related serious adverse events were observed. The most frequently reported treatment-emergent adverse events were mild-to-moderate local injection-site reactions, and the overall safety profile was described as consistent with the expectations of ongoing clinical development.
CBL-514 is a proprietary small-molecule injectable drug candidate designed to selectively induce adipocyte apoptosis, or programmed death of fat cells, at the injection site. Caliway is developing the candidate across several potential indications involving abnormal or unwanted subcutaneous adipose tissue, including Dercum’s disease and other conditions.
Caliway Explores Broader Therapeutic Potential
The positive Dercum’s disease findings could also support investigation of CBL-514 in lipedema, a chronic disorder involving abnormal fat accumulation that can be accompanied by pain, swelling and tenderness. Caliway said it plans to work with clinical experts to evaluate the feasibility and development strategy for CBL-514 in this additional indication. The company has previously reported that CBL-514 received FDA Fast Track and Orphan Drug Designations for Dercum’s disease, while the European Medicines Agency also granted Orphan Drug Designation. The investigational therapy remains in clinical development, and additional clinical studies will be required before its safety and efficacy can support any potential regulatory approval.
The latest Phase 2 results represent an important milestone for Caliway’s rare-disease drug development program. By demonstrating statistically significant and sustained pain reduction against placebo, CBL-514 has generated clinical data that can inform future development decisions. Further studies will be necessary to confirm the findings in larger populations and establish the candidate’s long-term safety and therapeutic benefit. For the biopharmaceutical industry, the program highlights continued efforts to develop targeted treatments for rare diseases with limited therapeutic options. As Caliway advances CBL-514, future clinical and regulatory milestones will determine whether the investigational therapy can ultimately provide a new treatment option for people living with Dercum’s disease.
Source: Caliway Biopharmaceuticals press release
