Healx secures $47 million for AI-driven drug discovery in rare diseases

Healx, a UK-based startup that uses AI to discover new drugs for rare diseases, has secured $47 million in a Series C funding round. The company, which identifies connections between existing chemical compounds and rare diseases, has also received regulatory approval to begin Phase 2 clinical trials of a new drug in the US later this year.

Healx's unique approach to drug discovery involves analyzing millions of drug and disease data points to find previously undetected connections that could lead to new treatments with known compounds. This method promises to be less time-consuming and have lower failure rates than the traditional “one disease, one target, one drug” approach commonly used by pharmaceutical companies.

The company was founded in 2014 by Tim Guilliams, a PhD in biophysics and neuroscience from the University of Cambridge, and David Brown, former global head of drug discovery at Roche. With the new funding, Healx is preparing to launch a Phase 2 clinical trial for a new treatment it developed for neurofibromatosis type 1 (NF1), a genetic disorder that causes tumor growth in human nerves.

Healx's lead NF1 candidate, HLX-1502, is expected to begin trials in late 2024, focusing on young adults with NF1 and inoperable plexiform neurofibroma, a specific type of tumor associated with NF1. The company arrived at HLX-1502 using an existing drug developed for a different purpose that had never been launched in the US. The company is reformulating this drug, and since it already had existing safety data attached due to previous testing in healthy patients, it gives the company a greater degree of confidence that it will have fewer side effects.

Healx holds the patents for HLX-1502 specifically for use in the treatment of NF1 and other nerve-related diseases, and the FDA has also granted it new drug exclusivity in the US market. The company is also in the early preclinical or discovery stages of AI for a range of other conditions including oncology, renal and neurology.

Despite a challenging market for deep tech and biotech funding over the past two years, Healx's latest funding round was oversubscribed. The company is now looking to expand into the United States, where its clinical trials will begin in December.