CBL-514D significantly reduced Dercum’s Disease pain in a Phase 2 trial, with sustained benefits through Week 24 and potential for lipedema development.
Written By: Umesh Hanumante,
M.Pharm (Reg. Affairs)
Reviewed By: Pharmacally Editorial Team
Caliway Biopharmaceuticals reported statistically significant pain reductions with CBL-514D in the Phase 2 CBL-0202DD trial in patients with Dercum’s Disease. The treatment effect remained significant through Week 24, while the findings support further evaluation of the program in lipedema.
CBL-514D Meets the Phase 2 Primary Endpoint
The randomized, single-blind, placebo-controlled CBL-0202DD trial evaluated CBL-514D in patients with Dercum’s Disease, a rare and debilitating disorder associated with painful lipomas and abnormal subcutaneous adipose tissue. The study met its primary efficacy endpoint at Week 20, with statistically significant pain reductions in both the Full Analysis Set (FAS) and Per-Protocol (PP) populations.
In the FAS population, CBL-514D reduced mean pain scores by 3.1 points at Week 20, compared with 1.9 points with placebo (p=0.005). Mean pain reduction reached 3.5 points at Week 16 and 4.1 points at Week 24, compared with 1.7 points at both time points with placebo. The differences were statistically significant at both assessments (p=0.0017 and p<0.0001, respectively).
The PP analysis showed a similar treatment effect. Mean pain reduction at Week 20 was 3.1 points with CBL-514D versus 1.4 points with placebo (p=0.0002). At Weeks 16 and 24, mean reductions were 3.5 and 4.1 points, respectively, compared with 1.2 points at both time points in the placebo group (p=0.0001 and p<0.0001).
An exploratory FAS analysis found that approximately 90% of patients treated with CBL-514D achieved a pain reduction of at least 3 points in more than half of their treated painful lipomas, further supporting the consistency of the response.
Localized Adipocyte Apoptosis Underpins the Approach
CBL-514 is a small-molecule injectable that induces adipocyte apoptosis, or programmed cell death, at the treatment site to reduce subcutaneous fat. The approach is intended to remove adipocytes without causing necrosis or damage to surrounding tissues.
Caliway is developing CBL-514D as a formulation for Dercum’s Disease. The condition currently has no specifically approved treatment, leaving management focused largely on symptom control and procedures such as surgical removal of painful lipomas.
Consistent Safety and Tolerability
CBL-514D was generally well tolerated throughout the Phase 2 study. No drug-related serious adverse events were reported.
The most frequently reported treatment-emergent adverse events were mild-to-moderate local injection-site reactions. The reported safety findings support continued clinical development of the treatment.
The progressive reduction in pain from Week 16 through Week 24 also indicates that the observed treatment effect was sustained during follow-up, although longer-term studies will be needed to establish durability beyond the study period.
Lipedema Provides a Potential Expansion Opportunity
The Dercum’s Disease findings are also informing plans to evaluate CBL-514 in lipedema, a chronic adipose tissue disorder that predominantly affects women and is associated with abnormal fat accumulation, particularly in the lower extremities and buttocks. Patients may also experience pain, tenderness and swelling.
Caliway plans to work with clinical experts to assess the feasibility and clinical development strategy for CBL-514 in lipedema. The company cites estimates that lipedema affects approximately 10% of women worldwide and represents a potential global market opportunity exceeding $30 billion. Prevalence estimates, however, vary depending on diagnostic criteria and study methodology.
According to the company, more than 544 participants have received CBL-514 across clinical trials, with 10 completed studies having finalized statistical reports.
The Phase 2 CBL-0202DD results provide clinical support for continued development of CBL-514D in Dercum’s Disease, while the company evaluates whether the underlying adipocyte-targeting approach can be extended to other disorders involving abnormal and painful adipose tissue.
Reference
Caliway Announces Positive Phase 2 Results of CBL-514 in Dercum’s Disease, Achieving Primary Endpoint with Statistically Significant and Sustained Pain Reduction, Caliway Biopharmaceuticals, 21 September 2026
A Study to Evaluate the Efficacy and Safety of CBL-514 Compared to Placebo in Participants With Dercum’s Disease Lipomas, ClinicalTrials.gov ID NCT06303570
About the Writer
Umesh Hanumante (M.Pharm) (LinkedIn) is a pharmacy professional and healthcare writer with a background in Regulatory Affairs, pharmaceutical innovation, and clinical research. He has around two years of industry experience as an Executive PMT at Troikaa Pharmaceuticals Ltd and qualified GPAT 2024. His areas of interest include regulatory compliance, dossier preparation, clinical trials, emerging therapies, and advancements in the global pharmaceutical and healthcare sector.