Caliway Announces Positive Phase 2 Results of CBL-514 in Dercum's Disease, Achieving Primary Endpoint with Statistically Significant and Sustained Pain Reduction

• Primary Endpoint Achieved in CBL-0202DD Phase 2 Trial—CBL-514 delivered statistically significant pain reduction across both FAS and PP populations compared with placebo.
• Expansion Opportunity Beyond Dercum's Disease—Study findings support the potential development of CBL-514 for Lipedema, a condition affecting approximately 10% of women globally and representing a market opportunity of more than US$30 billion.

NEW TAIPEI CITY, Sept. 22, 2026 /PRNewswire/ -- Caliway Biopharmaceuticals (TWSE: 6919) on September 21 announced final statistical results from its randomized, single-blind, placebo-controlled Phase 2 clinical trial (CBL-0202DD) evaluating CBL-514 for the treatment of Dercum's Disease (DD), a rare and debilitating disorder characterized by painful lipomas. The study successfully achieved its primary efficacy endpoint, with CBL-514 demonstrating clinically meaningful and statistically significant pain reduction in both the Full Analysis Set (FAS) and Per-Protocol (PP) populations. The results showed robust and durable efficacy, with treatment benefits consistently observed across multiple assessment time points and increasing over all treatment courses.

Phase 2 CBL-0202DD Demonstrated Robust and Sustained Efficacy Across Primary and Secondary Endpoints

The Phase 2 CBL-0202DD trial was designed to evaluate the efficacy and safety of CBL-514 in patients with Dercum's Disease. Final statistical analyses showed clinically significant and sustained reductions in lipoma-associated pain following treatment with CBL-514, while both primary and secondary efficacy endpoints achieved statistical significance.

For the primary efficacy endpoint assessed at Week 20 in the FAS population, patients treated with CBL-514 achieved a mean pain-score reduction of 3.1 points, compared with 1.9 points in the placebo group, representing a statistically significant treatment effect (p=0.005). For the secondary efficacy endpoints assessed at Weeks 16 and 24, patients receiving CBL-514 achieved mean pain-score reductions of 3.5 points and 4.1 points, respectively, compared with a reduction of 1.7 points at both time points in the placebo group. Both comparisons reached statistical significance (p=0.0017 and p<0.0001, respectively).

The efficacy findings were further confirmed in the PP population. For the primary efficacy endpoint assessed at Week 20, patients treated with CBL-514 achieved a mean pain-score reduction of 3.1 points, compared with 1.4 points in the placebo group, representing a statistically significant treatment effect (p=0.0002). For the secondary efficacy endpoints assessed at Weeks 16 and 24, patients receiving CBL-514 achieved mean pain-score reductions of 3.5 points and 4.1 points, respectively, compared with a reduction of 1.2 points at both time points in the placebo group. Both comparisons reached statistical significance (p=0.0001 and p<0.0001, respectively).

CBL-514 delivered statistically significant and clinically meaningful pain reduction at Weeks 16, 20, and 24. Importantly, the magnitude of response continued to increase over time, supporting the durability of the treatment effect.

Notably, an exploratory analysis in the FAS population showed that approximately 90% of patients treated with CBL-514 achieved a clinically meaningful pain reduction of at least 3 points in more than half of their treated painful lipomas, markedly outperforming placebo. From a safety perspective, CBL-514 was generally well tolerated throughout the study. No drug-related serious adverse events (SAEs) were reported. The most commonly reported treatment-emergent adverse events were mild-to-moderate local injection-site reactions, and the overall safety profile was consistent with expectations for ongoing clinical development.

No Approved Therapies for Dercum's Disease and a Global Market Opportunity Approaching US$20 Billion

Dercum's Disease is a chronic, highly recurrent, and debilitating rare disease characterized by abnormal accumulation of subcutaneous adipose tissue in the trunk and extremities, resulting in painful lipomas or adipose masses. Patients frequently experience persistent pain lasting longer than three months, often accompanied by burning sensations and tenderness that can severely impact daily life. Although the exact cause and underlying pathophysiology of Dercum's Disease remain incompletely understood, growing evidence suggests that abnormalities in adipose tissue metabolism may play an important role in disease development. To date, there are no approved therapies specifically indicated for the treatment of Dercum's Disease, and currently available treatment approaches such as surgical removal provide only temporary symptomatic relief and are often associated with undesirable side effects.

Given the substantial unmet medical need, Dercum's Disease represents a meaningful therapeutic and commercial opportunity. According to market research reports, the global Dercum's Disease market was estimated at approximately US$11.3 billion in 2021 and is projected to reach approximately US$19.96 billion by 2030, representing a compound annual growth rate (CAGR) of approximately 6.76%.

In addition, based on the results of the CBL-0202DD study, several key opinion leaders (KOLs) and potential pharmaceutical partners have recognized the therapeutic potential of CBL-514 in other adipose tissue disorders that share clinical characteristics with Dercum's Disease, including Lipedema. Lipedema is a chronic disorder that predominantly affects women and is characterized by abnormal fat accumulation in the lower extremities and buttocks, frequently accompanied by pain, swelling, and tenderness. Epidemiological and market research data suggest that Lipedema affects approximately 10% of women worldwide, representing a substantial patient population and a potential global market opportunity exceeding US$30 billion. Currently, there are no approved curative therapies for Lipedema, and weight loss alone does not effectively address the underlying pathological fat accumulation.

Based on the promising findings from the Dercum's Disease study, the Company plans to collaborate with clinical experts to further evaluate the feasibility and clinical development strategy of CBL-514 for Lipedema. By leveraging CBL-514's unique mechanism of action as an inducer of adipocyte apoptosis, Caliway aims to expand into additional indications with high unmet medical needs and underserved commercial opportunities, ultimately bringing innovative treatment options to a broader patient population.

About CBL-514

CBL-514 is a patented, first-in-class small-molecule 505(b)(1) injection developed by Caliway. It is designed to selectively induce adipocyte apoptosis to precisely reduce subcutaneous fat at the injection site without causing necrosis or damage to surrounding tissues or cells.

CBL-514 has demonstrated broad therapeutic potential across multiple indications, including non-surgical subcutaneous fat reduction and moderate-to-severe cellulite. A separate formulation, designated as CBL-514D, is being developed for the treatment of Dercum's Disease, a rare disease. To date, more than 544 subjects have participated in clinical trials related to CBL-514. Across the 10 completed clinical trials with finalized statistical reports, all primary and key secondary efficacy endpoints have been met, demonstrating excellent safety and tolerability.

About Caliway Biopharmaceuticals

Caliway Biopharmaceuticals Co., Ltd. (TWSE: 6919), founded in 2012, is dedicated to developing breakthrough therapeutics that redefine standards of care and transform clinical practices. Caliway aims to leverage Taiwan's robust pharmaceutical research and development capabilities to serve the global aesthetic medicine and biomedical markets. The company was officially listed on the Taiwan Stock Exchange (TWSE: 6919) on October 2, 2024.

Disclaimer

This press release and related web pages contain forward-looking statements that are based on the Company's current expectations, forecasts, and assessments. These statements involve risks, uncertainties, and other factors beyond the Company's control. Actual events or results may differ materially from those projected in these forward-looking statements due to various external factors. Caliway undertakes no obligation to update or revise any information in this press release.

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