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CBL-514, Caliway's novel small molecule drug, was selected for data presentation at the World Orphan Drug Congress USA 2025, highlighting its promising Phase 2 study results (CBL-0201DD) for Dercum's Disease Treatment. The Phase 2 topline results met all primary and secondary endpoints, with 64.5% of painful lipomas showing ≥ 50% dimension reduction or complete clearance (per-protocol population), and pain being reduced by 4.7 points (on a 10-point scale). The program has advanced to a U.S. FDA-approved Phase 2b study (CBL-0202DD), with subject recruitment expected to be completed by Q3 2025, representing a potential breakthrough for patients with no approved treatment options. TAIPEI, April 17, 2025 /PRNewswire/ -- Caliway Biopharmaceuticals (TWSE: 6919) announced today that it has been invited to present the clinical results of its CBL-514 Phase 2 study (CBL-0201DD) in Dercum's Disease at the upcoming World Orphan Drug Congress USA 2025, to be held in Boston on April 24, 2025. CBL-514 is a novel 505(b)(1) small-molecule drug developed by Caliway. It has previously demonstrated promising results in the field of medical aesthetics. CBL-514 is also being investigated for Dercum's Disease— a rare disease characterized by the growth of painful lipomas, with no approved treatment to date. According to the CBL-0201DD Phase 2 study results, 64.5% of painful lipomas showed dimensions reduction of more than 50% or complete clearance after CBL-514 treatments, with more than half of painful lipomas achieved this outcome after only one CBL-514 treatment. The pain score was also reduced by 4.7 points (out of 10). These results support CBL-514's potential as a first-in-class treatment for Dercum's disease, offering new hope to patients by significantly reducing lipoma size and pain. The World Orphan Drug Congress USA is a leading global conference focused on rare disease and orphan drug development. Bringing together over 2,000 participants from academia, regulatory agencies, pharmaceutical and biotech companies, patient advocacy groups, and investors. The event serves as a key platform for advancing scientific innovation, regulatory policies, and access strategies in rare disease therapies. Being selected to present at the most important event for all rare disease stakeholders, not only highlights the clinical potential of CBL-514 in rare disease treatment, but also underscores Caliway's ongoing commitment to addressing unmet medical needs. The drug has entered a U.S. FDA-approved Phase 2b clinical study (CBL-0202DD), which is currently ongoing and expected to complete patient recruitment by Q3 2025. If successful, CBL-514 could become the first approved treatment for Dercum's Disease, offering a long-awaited therapeutic option for patients worldwide. CBL-514 Study Presentation Info at World Orphan Drug Congress USA: Conference: World Orphan Drug Congress USA 2025 Date & Time: April 24, 2025 (Thursday), 10:40 AM (Eastern Time) Presentation Title: Efficacy and Safety of CBL-514, a Novel Lipolytic Injectable, in the Treatment of Dercum's Disease: Phase 2 Study Results Location: Boston Convention & Exhibition Center | Theater 2 – Hall C For more details on the World Orphan Drug Congress USA 2025, please visit: the official website. About Dercum's Disease Dercum's disease is a rare disorder 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 2022 was $11.8 billion. With a compound annual growth rate (CAGR) of 6.76%, the global market of Dercum's disease treatment in 2032 is estimated to expand to $22.7 billion. About CBL-514 CBL-514, a 505(b)(1) and first-in-class small-molecule drug developed by Caliway, is the world's first injectable lipolysis drug that induces adipocyte apoptosis to reduce subcutaneous fat in targeted areas without causing any systematic side effects on the central nervous system, cardiovascular system, and respiratory system. CBL-514 has demonstrated fewer side effects, superior efficacy, and fewer required treatments compared with existing surgical procedures and other non-invasive localized fat reduction solutions. Caliway is currently investigating multiple indications for CBL-514, including non-surgical fat reduction, moderate-to-severe cellulite and weight rebound management. A derivative compound, CBL-514D, is being developed for indications including rare diseases such as Dercum's disease and Obstructive Sleep Apnea (OSA). As of the end of November 2024, Caliway has enrolled a total of 520 subjects in clinical studies related to CBL-514. To date, 10 clinical studies have been completed with results available, and all these studies have met their primary efficacy endpoints and safety parameters. CBL-514 has demonstrated precise efficacy with a favorable safety profile across multiple indications, showing outstanding statistical significance ( p<0.00001 or p<0.0001) in both ITT and FAS populations. Caliway plans to initiate subject recruitment for two pivotal global Phase 3 studies for localized fat reduction in the second half of 2025. About Caliway Biopharmaceuticals Caliway Biopharmaceuticals (Caliway) is a clinical-stage biopharmaceutical company driven to breakthrough drug discovery of novel small-molecule therapeutics. Listed on the Taiwan Exchange (TWSE-6919), 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 WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- CBL-514 is the first-in-class new drug candidate for large-area fat reduction. CBL-0204 Phase 2b study, approved by the U.S. FDA and AU TGA, met all the primary and secondary efficacy endpoints in FAS (full analysis set) population and PP (per-protocol) population, paving its way to a global pivotal Phase 3 study in 2025. - Over 80% of participants in the PP population achieved at least 1-grade improvement using CR-AFRS (Clinician Reported-Abdominal Fat Rating Scale) and PR-AFRS (Patient Reported-Abdominal Fat Rating Scale). - Almost 60% of participants in the PP population achieved at least 1-grade improvement using CR-AFRS after one CBL-514 treatment. - Compared to the placebo group, CBL-514 can reduce over 30% of abdominal subcutaneous fat thickness in the PP population. NEW TAIPEI CITY, Dec. 11, 2024 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway) announced the study results of the CBL-514 Phase 2b study for subcutaneous fat reduction (CBL-0204 Phase 2b study, NCT05736107), which met all the primary and secondary efficacy endpoints in the FAS (Full analysis set) and PP (Per-protocol) population analysis. Notably, the primary endpoint in the FAS population analysis showed that, at 12 weeks after the final treatment, 76.7% of patients treated with CBL-514 compared with placebo (18.9%) achieved at least 1-grade improvement reported by the Investigator using the Clinician Reported-Abdominal Fat Rating Scale (CR-AFRS) (p=0.00004). Additionally, the study results demonstrated a favorable safety and tolerability profile, paving its way to a global muti-center Pivotal Phase 3 study in 2025, shading light of becoming the first approved injectable for large area fat reduction. CBL-0204 is a multi-center, randomized, placebo-controlled Phase 2b study evaluating CBL-514's efficacy and safety in reducing subcutaneous fat in the abdominal area. The study enrolled 107 participants with moderate to severe abdominal subcutaneous fat assessed by the Abdominal Fat Rating Scale (AFRS). The participants were randomized 1:1 to receive up to 4 courses, once every 3 weeks of CBL-514 or placebo treatments, followed by three follow-up visits at 4, 8, and 12 weeks after the last treatment. The maximum dose was 600 mg per treatment. To enhance the success rate of meeting efficacy endpoints in the future CBL-514 Pivotal Phase 3 study, the CBL-0204 study adopted the U.S. FDA-recommended efficacy assessment tool, AFRS and MRI (Magnetic Resonance Imaging) to assess the subcutaneous fat volume change in the treated area. Primary Endpoint In FAS population, at 12 weeks after the last treatment, 76.7% of participants treated with CBL-514 compared with placebo (18.9%) achieved at least 1-grade improvement reported by the Investigator using the CR-AFRS (p=0.00004). In PP population, at 12 weeks after the last treatment, 81.8% of participants treated with CBL-514 compared with placebo achieved at least 1-grade improvement reported by the Investigator using the CR-AFRS (p<0.0005). Secondary Endpoints At 12 weeks after the last treatment, 32% of participants treated with CBL-514, compared with placebo (0%), achieved at least 2-grade improvement reported by the Investigator using the CR-AFRS (p<0.002). In FAS population, at 12 weeks after the last treatment, 76.7% of participants treated with CBL-514, compared with placebo (19.4%), achieved at least 1-grade improvement reported by participants using the PR-AFRS (p=0.00004). In PP population, at 12 weeks after the last treatment, 81.8% of participants treated with CBL-514, compared with placebo, achieved at least 1-grade improvement reported by participants using the PR-AFRS (p<0.0002). At 4 weeks after the last treatment, 50% of participants treated with CBL-514 lost at least 150mL (p< 0.00001) of subcutaneous fat volume, compared with placebo (0%), as assessed by MRI. Compared to the placebo group, the CBL-514 group showed a change of -27.1% in abdominal subcutaneous fat thickness (95% CI: -32.3%, -21.8%, p< 0.00001) at 4 weeks after the final treatment. 58.8% of the participants only required one CBL-514 treatment to achieve at least 1-grade CR-AFRS improvement. Overall, the safety and tolerability profiles of CBL-514 demonstrated in the study were favorable and consistent with previous clinical studies of CBL-514. The major TEAEs were ISRs (injection site reactions) that were mild and moderate. For more information regarding CBL-0204 Phase 2b study, please visit: About AFRS AFRS is a scale developed by Caliway and validated following U.S. FDA recommendations and relevant regulations. In accordance with the FDA's guideline of Patient-Focused Drug Development, the primary endpoint of this study was to assess the efficacy of CBL-514 in improving the abdominal fat level using the Abdominal Fat Rating Scale (AFRS). The AFRS includes the Clinician Reported-AFRS (CR-AFRS) designed for physicians and the Patient Reported-AFRS (PR-AFRS) designed for participants. They were used to assess the response rate based on the physician's evaluation and the participant's self-assessment, respectively. The scale categorizes abdominal fat into five grades from Grade 1 (None/Minimal) to Grade 5 (Very Severe), with higher scores indicating higher severity of abdominal fat, and different grades indicating clinically significant differences. About the Full Analysis Set (FAS) population The FAS population includes all eligible participants who were randomized to receive at least one course of study drug and contributed qualified baseline efficacy data, and at least one post-dose efficacy data point. According to ICH E9 Statistical Principles for Clinical Trials, the analysis results of FAS and the Intention-to-Treat (ITT) are the same. About CBL-514 CBL-514, a first-in-class small-molecule drug, is a lipolysis injectable that can induce adipocyte apoptosis and lipolysis 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 is investigating multiple indications for CBL-514, including non-invasive subcutaneous fat reduction, Dercum's disease, and cellulite. To date, a total of 520 subjects have enrolled in CBL-514 clinical studies. According to 10 completed clinical studies of three indications, CBL-514 has demonstrated precise efficacy with a favorable safety profile. The global Pivotal Phase 3 study for non-surgical fat reduction will be conducted in 2025. 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 Taipei Exchange (TWSE-6919), Caliway aims to become an innovative pharmaceutical leader in aesthetic medicine and other diseases. For more information, please visit: https:/ 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 WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- 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