US-FDA accepts Caliway’s CBL-514 IND application for phase 2 study to treat Dercum's disease

Caliway Biopharmaceuticals, a Taiwan-headquartered, clinical-stage biopharmaceutical company driven to breakthrough drug discovery of novel small-molecule therapeutics, announced that the US FDA has accepted its CBL-514 Investigational New Drug (IND) application of CBL-0202 DD for phase 2 study to treat Dercum's disease.

Dercum's disease is a rare disease characterized by the growth of painful lipomas. The pain can be severe, chronic (> 3 months) and often disabling. So far, no approved treatment for Dercum's disease has led to the 2032 global treatment market expansion to 22.75 billion. Previous CBL-514 Phase 2 study (CBL-0201DD, NCT05387733) has demonstrated its efficacy in lipoma dimension reduction by more than 50% and significant pain improvement by 4.7 points, showing its potential in becoming the first approved treatment for Dercum's disease.

The CBL-0202DD phase 2 study will further evaluate the efficacy, safety, and tolerance of CBL-514 in treating Dercum's disease compared with placebo. The subject recruitment will be initiated in Q2 2024.

Dercum's disease is a rare disorder that is characterized by the development of painful lipomas primarily located on the trunk region, and the extremities close to the trunk. The pain is chronic (>3 months), symmetrical, often disabling, and resistant to traditional analgesics. The main symptoms of Dercum's disease, according to the early reports, are obesity, pain from the lipomas, and numerous psychiatric manifestations, including sleep disturbances, depression, and anxiety.

The etiology of Dercum's disease remains unknown. There is no approved drug nor recommended standard treatment for Dercum's disease yet. The current treatment options, including surgical removal, liposuction, electrotherapy, and inflammation inhibitors, are symptomatic, and the efficacies are limited with numerous adverse events. The clinical need for Dercum's disease remains unmet.

According to the Global Dercum's Disease Market Research Report, the global Dercum's disease treatment market size in 2021 was $11.3 billion. With a compound annual growth rate (CAGR) of 6.76%, the global market of Dercum's disease treatment in 2030 is estimated to expand to $19.95 billion.

CBL-0202DD study is a randomized, placebo-controlled, single-blind Phase 2b study evaluating CBL-514 injection's efficacy and safety in Dercum's disease subjects.

The study will recruit 30 Dercum's disease subjects with four to ten painful lipomas and randomize them 1:1 into two groups to receive CBL-514 injection or placebo. Each subject will receive up to 5 courses of CBL-514 injection or placebo administered into selected lipomas once every 4 weeks for 16 weeks in total, and the injection volume per lipoma will be based on the lipoma size, determined by ultrasound. The recurred lipomas will also be treated.

The efficacy of CBL-514 on each lipoma will be evaluated at 4 and 8 weeks after the last treatment. The primary endpoint is the percentage of lipomas with complete response compared to placebo. The secondary endpoints include the percentage of lipomas with partial response compared to placebo, the duration of complete response/partial response lasting on the lipomas compared to placebo, the percentage change in lipoma volume compared to placebo, and the change of pain scores on the lipomas compared to placebo.

CBL-514, a potentially first-in-class small-molecule drug, is an injection lipolysis drug that can induce adipocyte apoptosis and lipolysis to reduce subcutaneous adiposity in treatment areas in animal studies without causing any systematic side effects on the central nervous system, cardiovascular system, and respiratory system. Caliway's nonclinical studies showed that CBL-514 upregulates the apoptosis mediator caspase 3 and Bax/Bcl-2 ratio, and then induces dose-dependent adipocyte apoptosis in vivo and in vitro.

Caliway is investigating multiple indications for CBL-514, including non-invasive subcutaneous fat reduction, Dercum's disease, cellulite, and lipoma treatment.

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