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Drug intelligence / Profile preview
JNJ-0237 (also known as AT-20494) is an orally bioavailable, selective small molecule inhibitor of the mechanistic target of rapamycin complex 1 (mTORC1). Originally discovered by Navitor Pharmaceuticals and developed by its spinout Anakuria Therapeutics, the asset was acquired by Janssen (a subsidiary of Johnson & Johnson) in 2022. JNJ-0237 was designed to selectively target mTORC1 while sparing mTORC2, thereby avoiding the mTORC2-associated toxicities (such as hyperglycemia and hyperlipidemia) that have historically limited the clinical utility of first-generation rapalogs. It was evaluated in Phase 1 clinical trials for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and chronic kidney disease (CKD) to slow cyst growth and disease progression, but development was discontinued by Johnson & Johnson in late 2025.
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