Caliway to Present CBL-514 Phase 2 Results for Dercum's Disease at 2025 World Orphan Drug Congress USA

Caliway Biopharmaceuticals has announced a significant milestone for its novel small-molecule drug, CBL-514, which has shown promising results in the treatment of Dercum's Disease. CBL-514, a first-in-class injectable therapy, was selected for presentation at the World Orphan Drug Congress USA 2025, an event focusing on rare disease and orphan drug development. This opportunity underscores the drug's potential impact on rare disease treatment.

The Phase 2 clinical trial results for CBL-514, known as CBL-0201DD, demonstrated impressive efficacy in reducing painful lipomas associated with Dercum's Disease. The study met both primary and secondary endpoints, with 64.5% of the treated lipomas achieving a reduction in size by at least 50% or complete clearance. Additionally, patients experienced a notable reduction in pain, with an average decrease of 4.7 points on a 10-point scale. These outcomes suggest that CBL-514 could offer a new therapeutic option for a condition currently lacking approved treatments.

Dercum's Disease is a rare disorder characterized by the development of painful fatty tumors, primarily on the trunk and extremities. The condition is chronic and can be debilitating, with existing treatments offering limited relief and often accompanied by side effects. The promising results from the Phase 2 trial of CBL-514 present a potential breakthrough for patients suffering from this challenging condition.

The drug has now progressed to a U.S. FDA-approved Phase 2b study, CBL-0202DD, which is underway with patient recruitment expected to be completed by the third quarter of 2025. If the results from this study are favorable, CBL-514 could become the first approved treatment specifically targeting Dercum's Disease, providing hope for patients worldwide.

Caliway Biopharmaceuticals, the developer of CBL-514, is a clinical-stage company committed to the discovery and development of innovative small-molecule therapeutics. The company is listed on the Taiwan Stock Exchange and aims to lead in the field of aesthetic medicine and other disease areas. CBL-514 has shown effectiveness in multiple clinical studies, demonstrating precise efficacy and a favorable safety profile across various indications. The drug induces apoptosis in adipocytes, reducing subcutaneous fat in targeted areas without systemic side effects, offering a safer alternative to existing surgical and non-invasive fat reduction methods.

In addition to its application in Dercum's Disease, Caliway is exploring other indications for CBL-514, including non-surgical fat reduction and management of moderate-to-severe cellulite. A derivative compound, CBL-514D, is also in development for rare diseases such as Dercum's Disease and Obstructive Sleep Apnea.

The global market for Dercum's Disease treatment was valued at $11.8 billion in 2022 and is projected to grow to $22.7 billion by 2032, with a compound annual growth rate of 6.76%. The introduction of an effective treatment like CBL-514 could significantly impact this market by providing a long-awaited therapeutic option for patients.

Overall, the selection of CBL-514 for presentation at the World Orphan Drug Congress USA highlights its clinical potential and Caliway's dedication to addressing unmet medical needs in rare diseases. The ongoing Phase 2b study will be crucial in determining the drug's future role in the treatment of Dercum's Disease.