Caliway Received U.S. FDA Fast Track Designation for CBL-514 for the Treatment of Dercum's Disease

- 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: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track.

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: www.Caliway.com.tw/en

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SOURCE Caliway Biopharmaceuticals