Caliway Begins Recruiting for CBL-514 Phase 2b Study on Dercum's Disease

Caliway Biopharmaceuticals has announced the commencement of subject recruitment for its CBL-514 Phase 2b study targeting Dercum's disease, known as CBL-0202 DD Phase 2b study (NCT06303570). The study aims to assess the efficacy and safety of CBL-514 injections in treating patients with Dercum's disease, and results are expected by the fourth quarter of 2025.

The CBL-0202DD study is a randomized, placebo-controlled Phase 2b trial designed to evaluate the impact of CBL-514 injections on Dercum's disease, specifically focusing on patients who have between four and ten painful lipomas. Approximately 30 subjects will be enrolled and divided into two groups in a 1:1 ratio. One group will receive CBL-514 injections, while the other will be given a placebo. Treatments will be administered once every four weeks, continuing for up to five sessions for each selected lipoma.

This Phase 2b study introduces significant advancements over the preceding Phase 2 study (CBL-0201DD, NCT05387733). The earlier study evaluated treatments over a four-week period, focusing on changes in lipoma dimensions. In contrast, the Phase 2b design extends the treatment period to 16 weeks. Lipoma size will be measured via ultrasound at an independent facility during visits from week 4 to week 24 post-initial treatment, providing a more extended observation period.

Another notable shift in the Phase 2b study is the primary endpoint, which now focuses on the percentage of lipomas achieving a complete response compared to the placebo. This change is intended to offer a more comprehensive view of the drug's effectiveness. With a longer treatment period, a larger participant pool, and a more stringent primary endpoint, the CBL-0202DD Phase 2b study represents a significant step forward in developing new treatments for Dercum's disease. Should the Phase 2b study meet its efficacy endpoints, Caliway plans to advance to a Phase 3 Pivotal study IND for Dercum's disease.

CBL-514 is currently the most advanced new medication in clinical trials for Dercum's disease and has shown statistically significant efficacy. Results from the earlier CBL-0201DD Phase 2 study demonstrated a substantial reduction in the size of painful lipomas, coupled with a pain reduction of 4.7 points. As the only treatment granted both Orphan Drug Designation and Fast Track Designation by the U.S. FDA for Dercum's disease, CBL-514 has the potential to become the first approved drug for this condition, expediting its clinical development and augmenting its market value upon release.

Dercum's disease is a rare and painful disorder characterized by the formation of painful lipomas, mainly on the trunk and nearby extremities. The pain is chronic, symmetrical, often debilitating, and resistant to conventional painkillers. The cause of Dercum's disease remains unknown, and no approved treatments currently exist. Existing treatment options, including surgical removal, liposuction, electrotherapy, and inflammation inhibitors, are primarily symptomatic and have limited efficacy and several adverse events. Therefore, there is a significant unmet clinical need for effective treatments for Dercum's disease.

According to the Global Dercum's Disease Market Research Report, the treatment market for Dercum's disease was valued at $11.3 billion in 2021 and is projected to grow to $19.96 billion by 2030, with a compound annual growth rate (CAGR) of 6.76%.

CBL-514 is a small-molecule drug designed as a lipolysis injection that induces adipocyte apoptosis to reduce subcutaneous fat without systemic side effects. Preclinical studies have indicated that CBL-514 upregulates apoptosis mediators, leading to effective fat reduction. Phase 2 studies have shown promising results, with significant reductions in lipoma size and pain. The ongoing Phase 2b study aims to further validate these findings and advance the drug toward potential approval.

Caliway Biopharmaceuticals, based in Taiwan, focuses on the discovery of novel small-molecule therapeutics. The company aims to become a leader in pharmaceutical innovation, particularly in aesthetic medicine and other diseases.

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