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- 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

NEW TAIPEI CITY, Jan. 23, 2024 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway) announces that Australian Bellberry HREC has approved Caliway's application to initiate CBL-514's Pivotal Phase 3 study (CBL-0301 Phase 3) for subcutaneous fat reduction. "We are all delighted to see CBL-514 showing its potential to become the new, promising, non-invasive fat reduction treatment in the previous Phase 2 study," said Vivian Ling, CEO of Caliway. "The following CBL-514 Phase 3 study will further investigate its fat reduction efficacy compared with placebo with the U.S. FDA-suggested fat change assessment tool, Abdominal Fat Rating Scale (AFRS) and MRI." The CBL-0301 Pivotal Phase 3 study is a multicountry and multicenter study that will recruit 300 subjects to evaluate CBL-514's efficacy, safety, and tolerability for reducing abdominal subcutaneous fat. The primary endpoint will be the percentage of subjects with at least 1-grade improvement on AFRS compared with placebo. The AFRS, developed by Caliway under the U.S. FDA recommendation, is a scale to assess fat accumulation severity and categorize subjects into 5 grades (1-5). The number indicated the severity of abdominal fat accumulation, with 1 indicating none/minimal and 5 indicating very severe. Currently, there are two CBL-514 Phase 2b studies for subcutaneous fat reduction ongoing in the U.S., Australia, and Canada. To optimize the Phase 3 study protocol and to increase its successful rate of meeting endpoints, both Phase 2b studies were designed to simulate the Phase 3 study with the same efficacy assessment tools, AFRS and MRI. Caliway will submit the final Pivotal Phase 3 study protocol as soon as the preliminary result of the CBL-0204 Phase 2b study is announced. Following up, Caliway will submit the CBL-0301 Phase 3 IND application to the U.S. FDA, EMA, and other countries' regulatory authorities. The subject recruitment will be initiated after receiving IND approval from the U.S. FDA. Caliway will also prepare to submit the IND application of another CBL-514's Pivotal CBL-0302 Phase 3 study for subcutaneous fat reduction. About CBL-0301 Pivotal Phase 3 Study The CBL-0301 Pivotal Phase 3 study is a multicountry and multicenter, randomized, double-blind, placebo-controlled study evaluating CBL-514 injection for reducing abdominal subcutaneous fat. CBL-514 will be administered via injection into the subcutaneous adipose layer on the abdomen. The study will recruit a total of 300 subjects with moderate (Grade 3) to severe (Grade 4) abdominal subcutaneous fat reduction assessed by the Abdominal Fat Rating Scale (AFRS). The subjects will be randomized in a 2:1 ratio to receive up to 4 treatments of allocated CBL-514 or placebo administered on the abdomen once every 3 weeks, and complete 3 follow-up visits 4 weeks, 8 weeks, and 12 weeks after the last treatment. The treatment dose will depend on the level of subcutaneous fat accumulation on the participant's abdomen. The maximum dose is 600 mg per treatment. The primary efficacy endpoint is the percentage of subjects with at least 1-grade AFRS improvement compared with placebo at the second follow-up. The secondary efficacy endpoints include the percentage of subjects with at least 2-grade AFRS improvement compared with placebo at each follow-up visit, and the number of treatments required to the first occurrence of 1-grade AFRS improvement. About Subcutaneous Fat Reduction The current subcutaneous fat reduction procedures include invasive surgery, such as liposuction and abdominoplasty, and non-invasive products. Studies have shown that liposuction can remove an average of 183.3mL of abdomen fat, yet it involves a lengthy procedure that is generally performed in an operating room and may also carry the risk of unfavorable outcomes and moderate to severe side effects. The efficacy of non-invasive procedures is generally less optimal with limited treatment areas and potential side effects, including tissue necrosis, nerve damage, and paradoxical adipose hyperplasia (PAH). According to the American Society of Plastic Surgeons' report, more than 60% of people are reluctant to undergo subcutaneous fat reduction procedures due to the fear of the side effects. The medical needs for subcutaneous fat reduction remain unmet. 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