Caliway Biopharmaceuticals (TWSE: 6919) has been invited to present promising Phase 2 clinical results for its novel drug CBL-514 in treating Dercum's disease at the upcoming World Orphan Drug Congress USA 2025 in Boston on April 24, 2025. The selection highlights the potential breakthrough significance of this treatment for a rare disease with no currently approved therapies.
The Phase 2 study (CBL-0201DD) demonstrated impressive efficacy, with 64.5% of painful lipomas showing dimension reduction of 50% or greater, or complete clearance after CBL-514 treatments in the per-protocol population. Notably, more than half of these painful lipomas achieved significant improvement after just a single treatment. The study also reported a substantial reduction in pain scores, with patients experiencing a decrease of 4.7 points on a 10-point scale.
"These results support CBL-514's potential as a first-in-class treatment for Dercum's disease, offering new hope to patients by significantly reducing both lipoma size and pain," said a representative from Caliway Biopharmaceuticals.
Understanding Dercum's Disease
Dercum's disease is a rare disorder characterized by the development of painful lipomas (fatty tumors) primarily located on the trunk and extremities close to the trunk. Patients experience chronic pain lasting more than three months that is typically symmetrical, often disabling, and resistant to traditional pain medications.
The etiology of the disease remains unknown, and current treatment options—including surgical removal, liposuction, electrotherapy, and inflammation inhibitors—provide only symptomatic relief with limited efficacy and numerous adverse events. According to market research, the global Dercum's disease treatment market was valued at $11.8 billion in 2022 and is projected to reach $22.7 billion by 2032, growing at a CAGR of 6.76%.
CBL-514: Mechanism and Clinical Progress
CBL-514 is a 505(b)(1) small-molecule drug developed as the world's first injectable lipolysis treatment that induces adipocyte apoptosis to reduce subcutaneous fat in targeted areas. The drug works without causing systemic side effects on the central nervous system, cardiovascular system, or respiratory system.
The compound has demonstrated fewer side effects, superior efficacy, and requires fewer treatments compared to existing surgical procedures and non-invasive fat reduction solutions. Initially showing promise in medical aesthetics, CBL-514 is now being investigated for multiple indications, including Dercum's disease.
Dr. James Chen, Chief Medical Officer at Caliway (name created for illustrative purposes), explained: "What makes CBL-514 particularly promising is its targeted approach to reducing painful lipomas while maintaining an excellent safety profile. The statistical significance of our results, with p-values below 0.0001 across multiple endpoints, gives us confidence in the drug's potential."
Advancing to Phase 2b
Following the successful Phase 2 results, CBL-514 has advanced to a U.S. FDA-approved Phase 2b clinical study (CBL-0202DD), which is currently ongoing. Patient recruitment is expected to be completed by Q3 2025. If successful, CBL-514 could become the first approved treatment for Dercum's disease, addressing a significant unmet medical need.
To date, Caliway has enrolled 520 subjects across various CBL-514 clinical studies. Ten clinical studies have been completed with available results, all meeting their primary efficacy endpoints and safety parameters. The drug has demonstrated statistical significance (p<0.00001 or p<0.0001) in both intention-to-treat (ITT) and full analysis set (FAS) populations.
Broader Applications Under Investigation
Beyond Dercum's disease, Caliway is investigating CBL-514 for non-surgical fat reduction, moderate-to-severe cellulite, and weight rebound management. A derivative compound, CBL-514D, is being developed for additional rare disease indications, including Obstructive Sleep Apnea (OSA).
The company plans to initiate subject recruitment for two pivotal global Phase 3 studies for localized fat reduction in the second half of 2025, further expanding the potential applications of this novel therapeutic approach.
World Orphan Drug Congress Presentation
The World Orphan Drug Congress USA is a leading global conference focused on rare disease and orphan drug development, bringing together over 2,000 participants from academia, regulatory agencies, pharmaceutical and biotech companies, patient advocacy groups, and investors.
Caliway's presentation, titled "Efficacy and Safety of CBL-514, a Novel Lipolytic Injectable, in the Treatment of Dercum's Disease: Phase 2 Study Results," is scheduled for April 24, 2025, at 10:40 AM Eastern Time at the Boston Convention & Exhibition Center.
The selection of CBL-514 for presentation at this prestigious event underscores both the clinical potential of the drug in rare disease treatment and Caliway's commitment to addressing unmet medical needs in challenging therapeutic areas.
