CAGE Bio Announces Start of Phase 2 Clinical Trial of Novel Topical DNA Aptamer Therapy CGB-600 for Vitiligo Treatment

SAN CARLOS, Calif. and DALLAS, Oct. 23, 2025 /PRNewswire/ -- CAGE Bio Inc., a clinical-stage biotechnology company pioneering novel immunomodulatory therapies for dermatological diseases, today announced the initiation of a randomized, double-blind, placebo-controlled clinical trial evaluating its proprietary DNA Aptamer therapy, CGB-600, for the treatment of vitiligo.

The investigational active pharmaceutical ingredient, licensed from TAGCyx Biotechnologies Inc., Japan, is a first-in-class DNA Aptamer and new chemical entity specifically designed to selectively bind to interferon gamma and downregulate autoimmune activity implicated in the localized destruction of melanocytes. By targeting disease-specific immune pathways, CGB-600 aims to offer more durable re-pigmentation with fewer side effects compared to existing treatments.

"Vitiligo remains an area of high unmet medical need with limited durable treatment options" said Dr. Nitin Joshi, Chief Executive Officer of CAGE Bio. "Our DNA Aptamer program opens an entirely new therapeutic modality in immune-targeted skin therapeutics. Prior preclinical proof-of-concept studies suggest CGB-600 can achieve significant and sustained re-pigmentation. We believe this could be a transformative advancement for patients and providers."

"Nucleic acids are large, charged molecules and their localized, topical delivery is a huge unmet need. This human clinical trial, where a drug of this size is being delivered non-invasively into the skin, is a major advancement in the drug delivery field", said Dr. Samir Mitragotri, Professor of Bioengineering at Harvard University, who pioneered the ionic liquid drug delivery platform during his tenure at the University of California, Santa Barbara. He continues to advance the science and application of ionic liquids for therapeutic delivery in his current research at Harvard.

About the Phase 2 Clinical Trial
The randomized, double-blind, placebo-controlled study will enroll 36 adult patients with nonsegmental facial vitiligo. The primary endpoints are tolerability and improvement in F-VASI (Facial Vitiligo Area Scoring Index) score at Week 24. Top-line results are anticipated in 3Q26. 

The global vitiligo treatment market is projected to exceed $2 billion in the coming years, but options with sustained benefits remain scarce. Recent advances with JAK inhibitors have expanded possibilities for patients; however, CGB-600 offers a differentiated approach with the potential for enhanced selectivity, lower side effects, and sustained benefits.

About CAGE Bio
CAGE Bio Inc. is a clinical-stage biotechnology company advancing next-generation dermatology and immunology therapies using nucleic acid and targeted delivery platforms. The company's pipeline includes multiple candidates addressing inflammatory and autoimmune skin disorders.

About TAGCyx Biotechnologies Inc.
TAGCyx Biotechnologies Inc. https://tagcyx.com/en/ is a Japan-based preclinical company specializing in the discovery and optimization of therapeutic oligonucleotide medicines, applying its proprietary unnatural base technology.

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SOURCE CAGE Bio Inc.