Caliway Selected to Present CBL-514 Phase 2 Study Results for Dercum's Disease at World Orphan Drug Congress USA 2025

• 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: https://www.caliway.com.tw/en

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SOURCE Caliway Biopharmaceuticals