DYRK1B x BAX

- CBL-514 is the first and only drug to receive both Orphan Drug Designation and Fast Track designation for Dercum's disease treatment. NEW TAIPEI CITY, March 3, 2024 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway) announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to CBL-514 for Dercum's disease treatment. CBL-514 is a lipolysis injection that can reduce local subcutaneous fat by inducing adipocyte apoptosis, and is the first drug to receive both ODD and Fast Track designation for Dercum's disease treatment. "Receiving Orphan Drug Designation and Fast Track designation from the U.S. FDA is a huge milestone in CBL-514 development. Moreover, of all Dercum's disease treatment studies, CBL-514 is by far the most advanced program with the clinical efficacy being proven to reduce the size of the painful lipoma by more than 50% significantly," said Vivian Ling, CEO of Caliway. "With these two designations, we look forward to accelerating CBL-514 clinical development, and becoming the first approved drug for Dercum's disease treatment with a 7-year marketing exclusivity." Caliway has completed a Phase 2 study (CBL-0201DD, NCT05387733) to evaluate CBL-514's efficacy and safety in treating Dercum's disease last year. The study results demonstrated that after treating with CBL-514, 64.5% of painful lipomas showed dimension reduction of more than 50%, 38.7% of painful lipomas showed complete clearance, with pain being reduced by 4.7 point. In January 2024, a placebo-controlled Phase 2b study (CBL-0202DD) to evaluate efficacy of CBL-514 for treating Dercum's disease has been approved by the U.S. FDA. The subject recruitment will be initiated in Q2 2024. About Orphan Drug Designation To support the development and evaluation of new treatments for rare diseases, the U.S. FDA grants orphan drug designation to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer than 200,000 people in the United States. Orphan drug designation qualifies sponsors for incentives including 7-year marketing exclusivity to sponsors of approved orphan products, federal tax credit for expenses, waiver of Prescription Drug User Fee Act (PDUFA) fees, and eligibility for regulatory assistance and guidance from the FDA in the design of a drug development plan. For more information regarding Orphan Drug Designation, please visit the U.S. FDA official website: 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 $19.95 billion. About CBL-514 CBL-514, a potentially 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 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 subcutaneous fat reduction, Dercum's disease, cellulite, and lipoma treatment. About Caliway Biopharmaceuticals Caliway Biopharmaceuticals (Caliway) is a Taiwan-headquartered, clinical-stage biopharmaceutical company driven to breakthrough drug discovery of novel small-molecule therapeutics. Listed on the emerging stock market in Taiwan (TPEX6919), Caliway aims to become an innovative pharmaceutical leader in aesthetic medicine and other diseases. 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

- Fast Track designation indicates CBL-514's potential in filling the unmet medical need of Dercum's disease. - CBL-514 is the first drug to receive Fast Track designation for Dercum's disease treatment. - CBL-514 Phase 2 study in Dercum's disease demonstrated its efficacy in significant painful lipomas' size reduction of more than 50%, complete clearance and pain improvement by 4.7 points. NEW TAIPEI CITY, Feb. 14, 2024 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the investigation of CBL-514 in Dercum's disease treatment. CBL-514 is a lipolysis injection that can reduce local subcutaneous fat by selectively inducing adipocyte apoptosis, and is the first drug to receive Fast Track designation for Dercum's disease treatment. "We are very pleased to see the FDA's decision on granting Fast Track designation to CBL-514," said Vivian Ling, CEO of Caliway. "Dercum's disease is a rare disease characterized by the growth of painful lipomas, the pain can often be chronic and disturbing. With the Fast Track designation, Caliway can benefit from more frequent engagement with the FDA, which will support the collection of appropriate clinical data to accelerate CBL-514 development, and provide the treatment to patients who have been suffering with this disease." Fast Track Designation Fast track designation is a process that the FDA designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need, providing a therapy where none exists, or providing a therapy which may be potentially better than available therapy. For more information on the Fast Track process, please visit the U.S. FDA official website: . The FDA's decision was based on Caliway's preclinical and CBL-0201DD Phase 2 (NCT05387733) clinical study results, showing that CBL-514 is the first and only product to demonstrate clinically meaningful and statistically significant in painful lipomas complete clearance or dimensions reduction of more than 50%, as well as significant pain improvement by 4.7 points. Therefore, the CBL-0201DD Phase 2 study demonstrated the substantial improvement over available therapies to address the unmet medical need for Dercum's disease. Expanded Access Program The Expanded Access Program is a program recognized by the FDA as a follow-on to their Fast Track Designation. An EAP provides a potential pathway for patients with a serious or life-threatening condition to gain access to an investigational drug for treatment outside of a clinical trial, particularly when no comparable or satisfactory alternative therapy options are available. For physicians that have identified a patient, or have spoken to a patient who contacted you, please contact Caliway at [email protected]. Caliway's goal is to acknowledge receipt to the requesting physician within (10-12) business days. 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 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. About CBL-514 CBL-514, a potentially 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 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 subcutaneous fat reduction, Dercum's disease, cellulite, and lipoma treatment. About Caliway Biopharmaceuticals Caliway Biopharmaceuticals (Caliway) is a Taiwan-headquartered, clinical-stage biopharmaceutical company driven to breakthrough drug discovery of novel small-molecule therapeutics. Listed on the emerging stock market in Taiwan (TPEX6919), Caliway aims to become an innovative pharmaceutical leader in aesthetic medicine and other diseases. 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

- 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