Healx

Building the world’s first AI-native rare disease pharma. End-to-end.

Healx doses the first patient in a Phase 1/2 trial testing **HLX-4310**, an oral combination of a selective HDAC inhibitor and an mTOR inhibitor, for adolescents and adults with relapsed or refractory sarcoma. The study focuses on metastatic recurrence and progression, prioritizing **osteosarcoma** patients. The trial assesses both safety and efficacy in dose-escalation and dose-confirmation phases.

Jul 2026GlobeNewswireNews article

Healx applies its AI-driven drug discovery platform to Sanofi’s proprietary compound data to uncover new treatment opportunities for rare diseases. The partnership leverages Healx’s technology to analyze chemical structures and biological pathways, aiming to accelerate the identification of novel indications. Both companies focus on underserved rare disease areas where conventional methods have yielded limited progress.

Nov 2024GEN - Genetic Engineering and Biotechnology NewsNews article

Healx advances its AI-driven drug discovery pipeline with a **$47 million funding round**. The company secures regulatory approval to begin a **Phase II trial** for its lead candidate targeting **neurofibromatosis type 1 in adults**, with testing set to start in December 2024. This trial marks a key milestone in translating AI-generated insights into clinical validation for rare genetic disorders.

Aug 2024GEN - Genetic Engineering and Biotechnology NewsNews article

Healx, an AI-enabled drug discovery company focused on rare diseases, raises $47 million in a Series C round co-led by Atomico and R42. The company says it has regulatory clearance to begin U.S. Phase 2 trials of HLX-1502, its candidate for Neurofibromatosis Type 1 (NF1). The trial is expected to start by the end of 2024 and focus on young adults with NF1 and inoperable plexiform neurofibroma.

Aug 2024TechCrunchNews article