Caliway Announces CBL-514 Phase 2 Study Topline Results for Dercum's Disease, Showing 64.5% of Lipomas with Complete Clearance or Dimensions Reduction of Over 50% and Reduced Pain by 4.7

-       CBL-0201DD Phase 2 study met the primary and all secondary endpoints.

-       64.5% of painful lipomas showed complete clearance or dimensions reduction of more than 50% after CBL-514 treatments.

-       54.8% of painful lipomas showed complete clearance or dimension reduction of more than 50% after one CBL-514 treatment.

-       The pain score of CBL-514 treated painful lipoma was decreased by 4.7 points.

-       CBL-514 is the first and only drug to demonstrate clinically and statistically significant efficacy in lipoma size reduction, complete clearance, and pain improvement of greater than 4.0 points.

TAIPEI, Sept. 13, 2023 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway), a Taiwan-based biopharmaceutical company focusing on breakthrough medical aesthetics and inflammatory medicine discovery of small-molecule therapeutics, today announced that all the primary and secondary efficacy endpoints were met in the analysis of the ITT (Intent-to-treat) population and PP (per-protocol) population from the CBL-0201DD Phase 2 study (NCT05387733), evaluating CBL-514's efficacy and safety on participants with Dercum's Disease lipomas. In addition, CBL-514 demonstrated good safety and tolerance profiles, with no systemic adverse events reported.

Dercum's disease is a rare disease currently without approved or effective treatment, leading to the clinical need remaining unmet. According to a published study by Emma Hansson, MD in 2011, liposuction may reduce pain in Dercum's disease by 3 points (evaluated by the Visual Analog Scale) at 3 months after liposuction compared to baseline.

"The topline results of CBL-0201DD study demonstrated that CBL-514 is the first and only product to show clinically meaningful and statistically significant in painful lipomas complete clearance or dimensions reduction of more than 50%, while also demonstrating significant pain improvement by 4.7 points," said Vivian Ling, CEO of Caliway.

Topline Results from the CBL-0201DD Study

In the CBL-0201DD Phase 2 study, 12 participants with at least 4 painful and well-defined lipomas were recruited and randomized to 2 dose groups of CBL-514, with 6 participants per group. Each enrolled participant will receive up to 2 courses of their allocated dose of CBL-514 administered into selected lipomas, which the injection volume per lipoma will be based on the lipoma size, determined by ultrasound.

Primary Endpoint

• Painful lipomas dimension in the high-dose group was reduced by 51.3% (p<0.0001) and 54.7% (p<0.0001) at 4 and 8 weeks after CBL-514 treatments.
• Painful lipomas dimension in the low-dose group was reduced by 40.0% (p=0.0002) and 34.7% (p<0.0001) at 4 and 8 weeks after CBL-514 treatments.

Secondary Endpoints

• 90.3% of painful lipomas in the high-dose group showed dimensions reduction after CBL-514 treatments compared to baseline.
• 64.5% of painful lipomas in the high-dose group showed complete clearance or dimensions reduction of more than 50% after CBL-514 treatments compared to baseline.
• 38.7% of painful lipomas in the high-dose group showed complete clearance after CBL-514 treatments compared to baseline.
• The mean absolute change in pain score of painful lipomas in the high-dose group, evaluated by the Comparative Pain Scale, was decreased by 4.7 points (p<0.0001) after CBL-514 treatments compared to baseline.

The results in painful lipomas in the low-dose group showed statistical significance in primary and all secondary endpoints. Detailed efficacy and safety topline results from the CBL-0201DD study will be published in the journal for rare diseases. Based on the promising results, Caliway is currently planning for CBL-0202DD Phase 2b study IND application to further investigate CBL-514's efficacy in treating Dercum's disease.

About Dercum's Disease

Dercum's disease is a rare disorder with a strong tendency to recurrence 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, painful adipose tissue, 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.

About the CBL-0201DD Phase 2 Study

The CBL-0201DD study is a randomized, open-label, Phase 2 trial (NCT05387733) evaluating the efficacy and safety of CBL-514 injections in subjects with Dercum's disease.

To determine the optimal CBL-514 dose for Dercum's disease treatment, the study enrolled twelve Dercum's disease patients with at least four painful lipomas and randomized 1:1 to two groups to receive one of two doses of CBL-514 injections. Each subject would receive up to two courses of their allocated dose of CBL-514 administered into selected lipomas, and the injection volume per lipoma will be based on the lipoma size, determined by ultrasound.

The efficacy of CBL-514 on each lipoma was evaluated at Week 4 and Week 8 after the last treatment. The primary endpoint is the change of lipoma dimensions measured by ultrasound following CBL-514 treatment. The secondary endpoints include the percentage of lipomas with dimensions change and/or complete clearance measured by ultrasound, and the improvement of pain of individual lipomas following CBL-514 treatment.

About CBL-514

CBL-514, a first-in-class small-molecule drug, is an injection lipolysis drug that can induce adipocytes 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 inhibits the cell survival kinase DYRK1b, upregulates the apoptosis mediators 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 fat reduction (reducing subcutaneous fat), Dercum's disease, cellulite, and lipoma treatment.

About Caliway Biopharmaceuticals

Caliway Biopharmaceuticals (Caliway), is a Taiwan-based, clinical-stage biopharmaceutical company driven to breakthrough drug discovery of novel small-molecule therapeutics to help patients suffering from illness or life-threatening diseases. In Caliway, we aim to become an innovative pharmaceutical leader in medical aesthetics and inflammatory disease. For more information, please visit: www.Caliway.com.tw/en

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SOURCE Caliway Biopharmaceuticals