2024. 08
Date： 2024.08.26 -
Caliway Announces the Initiation of Subject Recruitment in CBL-514 Phase 2b Study for Dercum's Disease
New Taipei City, August 26, 2024 - Caliway Biopharmaceuticals (Caliway) announced that the subject recruitment of CBL-514 Phase 2b study for Dercum’s disease (CBL-0202 DD Phase 2b study, NCT06303570) has been initiated. The study results are anticipated in Q4 2025.
CBL-0202DD study is a randomized, placebo-controlled Phase 2b study evaluating CBL-514 injection’s efficacy and safety in treating Dercum’s disease. A total of approximately 30 Dercum’s disease subjects with four to ten painful lipomas will be enrolled in the study and randomized 1:1 into two groups to receive either CBL-514 or placebo once every 4 weeks for up to 5 treatments for each selected lipoma.
The CBL-514 Phase 2b study features notable changes compared with the earlier Phase 2 study (CBL-0201DD, NCT05387733). While the Phase 2 study evaluated a 4-week treatment period with the primary efficacy endpoint designed to evaluate the change of lipoma dimensions, the Phase 2b study design extends the treatment period to 16 weeks, with lipoma size evaluated by ultrasound at an independent third-party facility during each visit from 4 weeks to 24 weeks after the first treatment.
Additionally, the Phase 2b study design shifts the primary endpoint to the percentage of lipomas achieving a complete response compared to the placebo, providing a robust perspective on the drug’s efficacy. With a longer treatment period, a larger subject population, and a more challenging primary endpoint, the CBL0202DD Phase 2b study represents a significant advancement in the pursuit of innovative treatments for Dercum’s disease. Caliway will proceed with the IND of CBL-514 Phase 3 Pivotal study application for Dercum’s disease as soon as the Phase 2b study results meet the efficacy endpoints.
CBL-514 is the world's most advanced new drug in Dercum’s disease clinical development and is also the only clinical drug with statistically significant efficacy. The previous CBL-0201DD Phase 2 study results have demonstrated significant painful lipoma size reduction with a pain reduction of 4.7 points. With the potential to become the first approved drug for Dercum’s disease treatment, CBL-514 is currently the only new drug granted with both Orphan Drug Designation and Fast Track Designation for Dercum’s disease treatment by the U.S. FDA, which will help accelerate its clinical development and increase the product value once it launches to the market.
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 $19.96 billion.
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.
Caliway has completed CBL-0201DD, a Phase 2 study (NCT05387733) to evaluate the efficacy and safety of CBL-514 in treating Dercum's disease. The study results showed that after CBL-514 treatment, 64.5% of painful lipomas showed a dimensional reduction of more than 50%, and 38.7% of painful lipomas showed complete clearance, with a pain reduction of 4.7 points. In addition, CBL-0202DD, a Phase 2b IND (NCT06303570), has been approved by the U.S. FDA in January 2024, with subject recruitment initiating in August 2024 and the study results are anticipated in Q4 2025.
CBL-0202DD study is a randomized, placebo-controlled Phase 2b study evaluating CBL-514 injection’s efficacy and safety in treating Dercum’s disease. A total of approximately 30 Dercum’s disease subjects with four to ten painful lipomas will be enrolled in the study and randomized 1:1 into two groups to receive either CBL-514 or placebo once every 4 weeks for up to 5 treatments for each selected lipoma.
The CBL-514 Phase 2b study features notable changes compared with the earlier Phase 2 study (CBL-0201DD, NCT05387733). While the Phase 2 study evaluated a 4-week treatment period with the primary efficacy endpoint designed to evaluate the change of lipoma dimensions, the Phase 2b study design extends the treatment period to 16 weeks, with lipoma size evaluated by ultrasound at an independent third-party facility during each visit from 4 weeks to 24 weeks after the first treatment.
Additionally, the Phase 2b study design shifts the primary endpoint to the percentage of lipomas achieving a complete response compared to the placebo, providing a robust perspective on the drug’s efficacy. With a longer treatment period, a larger subject population, and a more challenging primary endpoint, the CBL0202DD Phase 2b study represents a significant advancement in the pursuit of innovative treatments for Dercum’s disease. Caliway will proceed with the IND of CBL-514 Phase 3 Pivotal study application for Dercum’s disease as soon as the Phase 2b study results meet the efficacy endpoints.
CBL-514 is the world's most advanced new drug in Dercum’s disease clinical development and is also the only clinical drug with statistically significant efficacy. The previous CBL-0201DD Phase 2 study results have demonstrated significant painful lipoma size reduction with a pain reduction of 4.7 points. With the potential to become the first approved drug for Dercum’s disease treatment, CBL-514 is currently the only new drug granted with both Orphan Drug Designation and Fast Track Designation for Dercum’s disease treatment by the U.S. FDA, which will help accelerate its clinical development and increase the product value once it launches to the market.
About Dercum’s Disease
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 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 $19.96 billion.
About CBL-0202DD Phase 2b Study
CBL-0202DD study (NCT06303570) is a randomized, placebo-controlled, Phase 2b study evaluating CBL-514 injection’s efficacy and safety in treating Dercum’s disease subjects. The study will enroll approximately 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 efficacy of CBL-514 on each lipoma will be evaluated during each visit from 4 weeks to 24 weeks after the first treatment at an independent third-party facility.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.
About CBL-514
CBL-514, a potentially first-in-class small-molecule drug, is a lipolysis injection that can induce adipocyte apoptosis to reduce subcutaneous adiposity in treatment areas without causing any systematic side effects on the central nervous system, cardiovascular system, and respiratory system. Caliway's preclinical studies have shown that CBL-514 upregulates the apoptosis mediators caspase 3 and Bax/Bcl-2 ratio and then induces adipocyte apoptosis in vivo and in vitro.Caliway has completed CBL-0201DD, a Phase 2 study (NCT05387733) to evaluate the efficacy and safety of CBL-514 in treating Dercum's disease. The study results showed that after CBL-514 treatment, 64.5% of painful lipomas showed a dimensional reduction of more than 50%, and 38.7% of painful lipomas showed complete clearance, with a pain reduction of 4.7 points. In addition, CBL-0202DD, a Phase 2b IND (NCT06303570), has been approved by the U.S. FDA in January 2024, with subject recruitment initiating in August 2024 and the study results are anticipated in Q4 2025.