Lipoma

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

NEW TAIPEI CITY, Sept. 3, 2024 /PRNewswire/ -- 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 CBL-514 Phase 3 Pivotal study IND 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. 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 (TPEX-6919), Caliway aims to become an innovative pharmaceutical leader in aesthetic medicine and other diseases. 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. For additional contact: [email protected] SOURCE Caliway Biopharmaceuticals

CBL-514 is the first product to treat cellulite at the raised areas. Currently, there is no effective and safe cellulite treatment on the market. The global market for cellulite treatment is estimated to expand to $7.37 billion in 2034. CBL-0201EFP Phase 2 study demonstrated CBL-514 statistically significant efficacy in reducing cellulite severity scores at 4 and 12 weeks after treatment. Over 50% of participants experienced at least one level of improvement in cellulite severity 12 weeks after the final CBL-514 treatment evaluating with the modified Hexsel Cellulite Severity Scale among 4 levels of severity. CBL-514 is under development for multiple indications, including Cellulite, Local Fat Reduction (currently in Phase 2b), and Durum's disease (currently in Phase 2), which brought Caliway the Fast Track Designation and the Orphan Drug Designation by the FDA in Feb. 2024. NEW TAIPEI CITY, April 25, 2024 /PRNewswire/ -- Caliway Biopharmaceuticals (Caliway) today announced the topline results of the CBL-0201EFP Phase 2 study, which met all the primary and secondary efficacy endpoints in the ITT (Intent-to-treat) and PP (per-protocol) analysis population, demonstrating CBL-514's statistically and clinically significant efficacy in improving cellulite severity with favorable safety and tolerance profiles. CBL-514, Caliway's lead pipeline, is a multi-indication lipolysis injection that can reduce subcutaneous adiposity by inducing adipocyte apoptosis. Among all the current cellulite treatment products, CBL-514 is the first and only product to treat cellulite in the raised areas. Apart from cellulite treatment, CBL-514 is also under Phase 2 study for subcutaneous fat reduction and Dercum's disease. Moreover, the U.S. FDA granted CBL-514 Fast Track Designation and Orphan Drug Designation based on the CBL-0201DD Phase 2 study results for Dercum's disease in February 2024. CBL-0201EFP Phase 2-stage 1 Topline Results: Significant Reductions in Cellulite Severity Across Three Dosage Groups CBL-0201EFP Phase 2 study is an open-labeled study to evaluate the safety, tolerability, and efficacy of CBL-514 injection for the treatment of Edematous Fibrosclerotic Panniculopathy (EFP, cellulite). The efficacy was assessed using the modified Hexsel Cellulite Severity Scale (Modified HCSS), with the highest score (9) indicating the most severe cellulite severity and the lowest score (0) indicating no cellulite. The severity level of cellulite will be scored as mild (1-3), moderate (4-6), or severe (7-9), which will be determined by the total scores. In the CBL-0201EFP Phase 2-stage 1 dose-finding study, a total of 12 participants were enrolled in 3 sequential escalating CBL-514 dose groups, 40mg, 60mg, and 80mg, to receive 1 CBL-514 treatment administered on both sides of the posterolateral thighs. The statistical analysis revealed significant reductions in cellulite severity across three dosage groups. Follow-up assessments at 2 and 4 weeks post single treatment on both thighs showed statistically significant improvements in cellulite severity for all dosage groups, with the high-dose group of 80 mg showing the most promising efficacy, reducing cellulite severity score (mean±SD) of -2.0±0.9 points (p= 0.00049，95%CI: -2.77, -1.23) and -2.6±1.5 (p= 0.00169，95%CI: -3.88, -1.37) at 2 and 4 weeks after treatment. CBL-0201EFP Phase 2-stage 2 Topline Results: Over 50% of Participants Demonstrated at least One Level of Improvement Among 4 Cellulite Severity Levels In the CBL-0201EFP Phase 2-stage 2 study is a single-arm study, a total of 23 participants were enrolled to receive up to 2 CBL-514 treatments based on cellulite severity on the thighs, with a maximum single dose not exceeding 320 mg administered at 4-week intervals. The baseline of participants' average cellulite severity score (mean±SD) is 6.5 ± 1.0 points. Four weeks after the final CBL-514 treatment, using Modified HCSS score of 1 ~9 in 4 severity levels, there was a reduction in cellulite severity with a mean change of -1.3 ± 1.3 points (p < 0.00001, 95% CI: -1.70, -0.90), demonstrating CBL-514's statically significant efficacy in cellulite severity improvement at both 4 weeks (p < 0.00001) and 12 weeks (p < 0.00001) after CBL-514 treatment. Moreover, over 50% of participants experienced at least one level of improvement in cellulite severity at the 12 weeks after the final CBL-514 treatment. In addition to the primary efficacy endpoint, a secondary efficacy endpoint assessed by the Global Aesthetic Improvement Scale (GAIS) demonstrated notable improvements in cellulite. Following the final treatment, both the Principal Investigator (PI) and participants assessed over 95% of individuals as having improved cellulite appearance at the four-week mark. The safety and tolerability profiles of CBL-514 demonstrated in both stages of the study were favorable and consistent with previous clinical studies of CBL-514. Most adverse events were mild to moderate injection site reactions, including injection site bruising, pain, swelling, and warmth, and were resolved within 28 days post-treatment. These findings underscore the potential of CBL-514 as a highly effective treatment option for reducing cellulite severity, offering renewed confidence and satisfaction to those affected by this common cosmetic concern. Following up, Caliway will further develop the cellulite severity scale for future CBL-514 clinical studies to evaluate cellulite improvement efficacy based on the U.S. FDA's instruction. About Cellulite (Edematous Fibrosclerotic Panniculopathy, EFP) Cellulite is characterized by the nonpathological appearance of the dimpled skin surface (likened to orange peel, cottage cheese, or mattress appearance), which occurs on the thighs and buttocks. The relief alterations of cellulite include depressions and raised areas. The depressions are caused by skin retraction by subcutaneous fibrous septa, while raised areas are projections of fat and subcutaneous structures to the skin surface. According to the American Society of Plastic Surgeons (ASPS) report, around 80 to 90% of women experience cellulite dimpling. The current treatment for cellulite includes non-invasive (medical devices and collagenase drug) and invasive options. However, their efficacy remains limited and temporary. Additionally, most products would cause significant side effects after administration, including severe bruising, pain, and hyperpigmentation, making most patients reluctant to receive them. The clinical need for cellulite treatment remains unmet. The global market for cellulite treatment is estimated to expand to $7.37 billion in 2034. About the CBL-0201EFP Phase 2 Study CBL-0201EFP Phase 2 study is an open-labeled study assessing the efficacy and safety of CBL-514 in participants with Edematous Fibrosclerotic Panniculopathy (EFP) cellulite. The study has an integrated design consisting of a single ascending dose (SAD) part in Stage 1 followed by a single-arm design in Stage 2. The Stage 1 study (clinicaltrial.gov ID: NCT05632926) included a total of 12 participants enrolled in 3 sequential escalating CBL-514 dose groups, 40mg, 60mg, and 80mg. Eligible participants will be sequentially assigned to receive 1 course of allocated CBL-514 dose administered by subcutaneous injection on both sides of the posterolateral thighs. The Stage 2 study (clinicaltrial.gov ID: NCT05836779) included a total of 23 participants enrolled in 1 selected CBL-514 dose. Eligible participants will be sequentially assigned to receive up to 2 courses of allocated CBL-514 dose administered by subcutaneous injection on both sides of the posterolateral thighs. About CBL-514 CBL-514, a potentially first-in-class small-molecule drug, is a lipolysis injection that can induce adipocyte apoptosis and lipolysis to reduce subcutaneous adiposity in treatment areas in animal studies without causing any systematic side effects on the central nervous system and cardiovascular system, and respiratory system. Caliway's preclinical 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 by the discovery of breakthrough small-molecule therapeutics with a focus on medical aesthetics and inflammatory disease. 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. For additional contact: [email protected] SOURCE Caliway Biopharmaceuticals