DYRK1B

- CBL-0202 Phase 2 study results met all the primary and secondary endpoints. - 69.9% and 60.9% of participants lost at least 150mL and 200mL of abdominal subcutaneous fat in the treated area after CBL-514 treatment. - Compared with placebo, an average of 312.1mL of subcutaneous fat was reduced in the treated area after CBL-514 treatment. - 42.9% of participants lost at least 150mL of subcutaneous fat in the treated area after only one CBL-514 treatment. TAIPEI, Oct. 2, 2023 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway) announced the topline results of the CBL-0202 stage 2 phase 2 study met all the primary and secondary efficacy endpoints compared with placebo in the ITT (Intent-to-treat) and PP (per-protocol) analysis population. "According to the study published in the Aesthetic Surgery Journal in 2012, liposuction could remove a mean of the subcutaneous fat volume of 183.3mL," said Vivian Ling, CEO of Caliway, "We are delighted to see that CBL-514 demonstrated the efficacy on subcutaneous fat reduction more effective than liposuction in the CBL-0202 stage 2 phase 2 study, reducing over 300mL of subcutaneous fat averagely." Topline Results from the CBL-0202 Phase 2-stage 2 Study Primary Endpoint 63.9% and 69.6% of participants in the CBL-514 group who lost at least 150 mL of subcutaneous fat in the treated area at 4 weeks and 8 weeks after treatment; while in the placebo group, 17.4% and 0.0% of participants who lost at least 150 mL of subcutaneous fat in the treated area at 4 weeks and 8 weeks after treatment. The percentage of participants who lost at least 150 mL of subcutaneous fat in the treated area at 4 weeks (p<0.001) and 8 weeks (p<0.00001) after CBL-514 treatment was significantly higher than the placebo group and achieved the primary endpoint. Secondary Endpoints 58.3% and 60.9% of participants in the CBL-514 group who lost at least 200 mL of subcutaneous fat in the treated area at 4 weeks and 8 weeks after treatment; while in the placebo group, 8.7% and 0.0% of participants who lost at least 200 mL of subcutaneous fat in the treated area at 4 weeks and 8 weeks after treatment. The percentage of participants who lost at least 200 mL of subcutaneous fat in the treated area at 4 weeks (p< 0.0002) and 8 weeks (p< 0.0001) after CBL-514 treatment was significantly higher than the placebo group. Compared with the placebo group, the LS mean (least square mean) difference of the subcutaneous fat volume change in the treated area at 4 weeks and 8 weeks after CBL-514 treatment were -312.08 mL (p<0.00001) and -276.83 mL (p<0.00005). 42.9% of participants lost at least 150 mL of subcutaneous fat in the treated area after one CBL-514 treatment. Overall, the safety and tolerability profiles of CBL-514 demonstrated in the study were favorable and consistent with previous clinical studies of CBL-514. Major TEAEs were ISRs (injection site reactions) that were mild and moderate, and most TEAEs were resolved before the end of the study visit. About the CBL-0202 Stage 2 Study The CBL-0202 stage 2 study (NCT04897412) is a randomized, single-blind, placebo-controlled phase 2 study to evaluate the efficacy, safety, and tolerability of CBL- 514 injection for abdominal subcutaneous fat reduction approved by the U.S. Food and Drug Administration (FDA) and Australian Therapeutic Goods Administration (TGA). CBL-514 will be administered via injection into the subcutaneous adipose layer on the abdomen. The trial randomized 76 participants across the U.S. and Australia in a 2:1 ratio to receive CBL-514 or a placebo. Each participant will receive up to 4 treatments of allocated CBL-514 or placebo administered on the abdomen once every 4 weeks and complete the two follow-up visits after the last treatment. The maximum dose is 600 mg per treatment, depending on the level of subcutaneous fat accumulation on the participant's abdomen. The primary efficacy endpoint of the CBL-0202 -stage 2 phase 2 study is the percentage of participants who lost at least 150 mL of abdominal subcutaneous fat as measured by ultrasound at 4 weeks and 8 weeks follow-up visits after treatment compared with placebo. The critical secondary efficacy endpoints include the percentage of participants who lost at least 200 mL of abdominal subcutaneous fat as measured by ultrasound at 4 weeks and 8 weeks follow-up visits after treatment compared with placebo, and reduction of subcutaneous fat volume over the treated area as measured by ultrasound from Baseline to follow-up visits compared with placebo. About Fat Reduction The current methods for fat reduction include non-invasive products and invasive surgery, such as liposuction and abdominal contour surgery. The efficacies of non-invasive products often take more than twelve weeks to show, and are generally poor with the limited treatment body area. The potential moderate to severe side effects of non-invasive products include tissue necrosis, nerve damage, scar tissue fibrosis, and paradoxical adipose hyperplasia (PAH). Though the efficacy of surgery is more significant compared to approved non-surgery local fat reduction products, the surgery still comes with severe side effects and a high risk of death. According to the American Society of Plastic Surgeons' report, more than 60% of people are reluctant to undergo local fat reduction due to the fear of the side effects. The medical needs for local fat reduction remain unmet. 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: SOURCE Caliway Biopharmaceuticals

- 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: Disclaimer This article and related information on this site contain forward-looking statements. The forward-looking information requires the Company to make numerous assumptions and is subject to inherent risks, uncertainties, and other factors that are beyond the control of the Company which may cause actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by such forward-looking statements. The Company undertakes no obligation to timely inform, update, or revise the information on this site if circumstances should change. SOURCE Caliway Biopharmaceuticals

- Caliway has initiated the subject recruitment in CBL-0204 Phase 2b study. - The study is expected to be completed in Q2 2024, and the topline results are anticipated in Q3 2024. TAIPEI, May 31, 2023 /PRNewswire/ -- Caliway Biopharmaceuticals, a biopharmaceutical company focusing on breakthrough medical aesthetics and inflammatory medicine discovery of small-molecule therapeutics, today announced the initiation of subject recruitment in CBL-0204 Phase 2b study. The study, approved by the U.S. FDA, will enroll 100 subjects in 14 clinical sites in the U.S. and Australia. CBL-0204 is a Phase 2b study evaluating the efficacy, safety and tolerability of CBL-514 injection, Caliway's leading candidate drug, in reducing abdominal subcutaneous fat. The study will enroll 100 adult subjects with mild, moderate, or severe abdominal subcutaneous fat as assessed by the abdominal fat rating scale (AFRS). The primary efficacy endpoint is the percentage of the subjects with at least one-grade improvement reported by the investigator assessing with the AFRS. Currently, CBL-514 is the only product worldwide with similar efficacy as liposuction and the only non-surgical fat reduction drug for a large treatment area. The previously completed clinical trial results have demonstrated significant differences in CBL-514's efficacy in reducing subcutaneous fat in the treated area with excellent safety and tolerability pro The CBL-0204 Phase 2b study is expected to be completed in Q2 2024. And the topline results are anticipated in Q3 2024. The Phase 3 pivotal study is expected to be applied in 2024. About Fat Reduction The current methods for fat reduction include non-invasive products and invasive surgery, such as liposuction and abdominal contour surgery. The efficacy of non-invasive products often takes more than twelve weeks to observe, and is generally poor with the limited treating body area. The potential moderate to severe side effects of non-invasive products include tissue necrosis, nerve damage, scar tissue fibrosis, and paradoxical adipose hyperplasia (PAH). Though the efficacy of surgery is more significant compared to approved non-surgery local fat reduction products, the surgery still comes with severe side effects and a high risk of death. According to the American Society of Plastic Surgeons' report, more than 60% of people are reluctant to undergo local fat reduction due to the fear of the side effects. The medical needs for local fat reduction remain unmet. About the CBL-0204 Phase 2b Study CBL-0204 is a multi-country, multicenter, randomized, placebo-controlled, Phase 2b study (ClinicalTrials.gov, NCT05736107) evaluating the efficacy, safety, and tolerability of CBL-514 injection in reducing subcutaneous fat. The study will enroll 100 adult subjects with mild, moderate, or severe abdominal subcutaneous fat as assessed by abdominal fat rating scale (AFRS). Each subject will receive up to four treatments of allocated CBL-514 or placebo administered subcutaneously to the abdomen, once every three weeks. The primary efficacy endpoint is the percentage of the subjects with at least one-grade improvement reported by the investigator using the AFRS. 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, based in Taiwan, is a 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: Company Codes: Taiwan:6919