Caliway Announced Orphan Drug Designation Granted to CBL-514 for the Treatment of Dercum's Disease

-       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: https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

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: www.Caliway.com.tw/en

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SOURCE Caliway Biopharmaceuticals