BTB Drug Development Research Center

BTB Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel treatments for pain, rare genetic diseases, and cancer. Established in June 2020 in Kyoto, Japan, the company's foundation is built upon the transcriptomics drug discovery programs of Professor Masatoshi Hagiwara of Kyoto University. Professor Hagiwara, a serial entrepreneur and a distinguished researcher in RNA biology, serves as the Chief Scientific Officer, bringing extensive experience in developing new therapeutic methods by manipulating the transcriptome with small molecules. The company is led by CEO Takashi Kiyoizumi, an experienced executive in the life sciences industry with a track record of successful drug development and fundraising, including co-founding and leading MediciNova (NASDAQ: MNOV) through its IPO.

The company's core technology revolves around transcriptomics drug discovery, an approach that identifies the mechanism of action of small molecules through the analysis of the transcriptome—all RNAs expressed in a cell. This allows for the development of small molecule-based drugs that can regulate RNA splicing, offering advantages like oral administration and more favorable manufacturing costs compared to nucleic acid-based medicines. In January 2025, the company completed a U.S. corporate inversion, relocating its headquarters to Escondido, California, while maintaining its research and development subsidiary in Kyoto. This strategic move aims to accelerate development, particularly in the United States, to address significant unmet medical needs.

BTB Therapeutics' pipeline is led by its non-opioid analgesic program, featuring BTB-101 (also known as ENDOPIN). This compound is a first-in-class small molecule that inhibits the α2B adrenergic receptor, demonstrating a strong analgesic effect comparable to opioids in preclinical studies but without the associated side effects or dependency. This positions ENDOPIN as a potential solution to the opioid crisis. The pipeline also includes an RNA regulatory drug development program. Key candidates include BTB-201 for Cardiac Fabry disease and BTB-202 for Long QT syndrome, both of which target abnormal RNA splicing. Furthermore, the company is advancing its cancer immunotherapy program with BTB-301, a small molecule designed to enhance the expression of cancer-specific spliced neoantigens, thereby boosting the effectiveness of immune checkpoint inhibitors.

The company's business model is centered on the research, clinical development, and eventual commercialization of its proprietary drug candidates. BTB Therapeutics has successfully secured funding through venture capital investments from firms like SMBC Venture Capital and Kyoto University Innovation Capital. It has also received significant non-dilutive financial support through grants from the Japan Agency for Medical Research and Development (AMED) and Kyoto Prefecture, totaling approximately $35 million.

Keywords: non-opioid analgesic, RNA modifying compounds, transcriptomics drug discovery, ENDOPIN, Masatoshi Hagiwara, Takashi Kiyoizumi, cardiac Fabry disease, long QT syndrome, cancer immunotherapy, α2B adrenergic receptor inhibitor, small molecule drugs, RNA splicing regulators, clinical-stage biopharmaceutical, rare genetic diseases, spliced neoantigens, drug development, Kyoto University, life sciences, pain management, genetic disorder treatment