AI Startup Healx Looks to Find Repurposable Drugs for Rare Diseases

Off the heels of a $56 million Series B funding round, Cambridge, UK-headquartered Healx is looking to partner with rare disease patient groups to advance treatments toward the clinic.

The company’s artificial intelligence-powered drug discovery platform, Healnet, uses both public and proprietary biomedical data to identify and develop repurposed treatments, which are existing drugs that have potential applications treating additional diseases. With a total budget of $20 million, Healx expects to allocate $1 million in AI and drug discovery resources per project over the next two years.

The goal of the so-called Rare Treatment Accelerator is to advance 100 rare disease treatments toward the clinic by 2025, Healx’s new chief collaboration officer, Bruce Bloom, tells Xconomy. Patient groups have no financial commitment as Healx partners, but bring the research and data that Healx can crunch with its AI software.

“We want the real-life understanding of the day to day that patients and caregivers live with. Sometimes the science points you in one direction, but the patients and caregivers point us in completely different directions,” says Bloom, who joins Healx from Cures Within Reach (CWR), where he was founder, president, and chief scientific officer from 2005, and CEO since 2017.

Bloom tells us the platform was designed to use AI to augment human intelligence by better predicting what kind of repurposed drugs would work for a disease. To do this, Healnet uses several AI modalities, including natural language processing, and has demonstrated a 70 percent success rate in predicting preclinical outcomes, Bloom says.

“Science has clearly demonstrated how many diseases a particular drug can impact,” he adds. “We interrogate all the information that we have from a null hypothesis. That way we don’t bias the answers we get by starting at a particular place. The advantage is that we find some really nonobvious connections.”

The Series B funds also will be used to further develop the company’s AI platform and onboard new staff. Healx employs approximately 40 as of now but expects this number to be between 55 and 75 mid next year.

Among the company’s current partners is the FRAXA Research Foundation, with which Healx is developing new treatments for fragile X syndrome, a genetic condition that causes intellectual disability. A clinical study is expected in early 2020.