Healx raises $56 million to combat rare diseases with AI

, a company that said it’s using intelligence (AI) to discover new drug treatments for rare diseases, has raised $56 million in a series B round of funding led by Atomico, with participation from Intel Capital, Balderton Capital, Global Brain, Btov Partners, Amadeus Capital Partners, and Cambridge Innovation Capital’s Jonathan Milner.

Founded out of Cambridge in 2014, Healx’s core AI Platform Healnet applies a range of machine learning techniques to public and proprietary data sources covering literature, clinical trials, patents, drug targets, chemical structures, symptoms, and more. Part of this involves using natural language processing (NLP) to extract insights and knowledge from all the published sources around specific diseases.

The culmination of all this data, Guilliams said, results in a knowledge graph of rare diseases which could help pharmacologists or biologists unearth effective new treatments that would otherwise be much more difficult to spot.

“We use a variety of machine learning algorithms to solve the many tasks necessary to predict drug treatments and translate them in the clinic effectively,” Healx CEO and cofounder Dr. Tim Guilliams told VentureBeat. “We use deep learning AI algorithms that predict novel connections between, for instance, drugs and disease. This is known as knowledge base completion, and relies upon our comprehensive knowledge graph of rare disease data.”

Rare diseases

While rare diseases are generally defined as diseases that impact a small percentage of people, there are up to 8,000 such diseases globally, according to the World Health Organization (WHO), affecting 400 million people globally. And the vast majority 95% still don’t have a FDA-approved treatment.

It’s worth stressing here that Healx isn’t setting out to develop completely new drugs, rather, it’s about getting the most value out of drugs that have already been approved, through combining them in new ways.

“We don’t develop new chemistry and molecules, we maximize the value for the existing drugs combining them in a clever way to improve their therapeutic effect when needed,” Guilliams continued.

Prior to now, Healx had raised around $12 million, the bulk of which came via a $10 million series A round 15 months ago, and with another $56 million in the bank, it said that it plans to build a clinical-stage portfolio for rare diseases such as Fragile X Syndrome (FXS), a known genetic cause of autism. To help, Healx is launching a global accelerator program that will strive to find clinic-ready treatments of rare diseases within two years, a significant reduction on the decade or more it can typically take using traditional drug discovery processes.

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“The current, expensive, trial-and-error-based model of drug discovery hasn’t changed in a century,” added Atomico principal and former surgeon Irina Haivas. “And it especially fails rare disease patients. 50 percent of these patients are children, many living with highly debilitating symptoms. Healx has shown that doesn’t have to be the case, by combining AI with world-class pharmacological expertise and putting patients first.”

The Rare Treatment Accelerator, as Realx’s new program is called, is intended to serve as an easy way for Healx to collaborate with clinicians and patient groups that represent people with rare diseases, and will involve reviewing shared data and resources.

“To date, it’s been families and patient groups who have had to become experts in the diseases affecting their loved ones and have often been the ones driving forward the efforts into finding new treatments,” Guilliams said. “With our unique combination of in-house R&D, industry collaborations and now the Rare Treatment Accelerator, we look forward to supporting these groups in their mission.

The AI treatment

AI and machine learning are infiltrating pretty every industry, and the drug discovery realm is no different. In the past few months alone Utah-based Recursion Pharmaceuticals took its total funding to $230 million with a fresh $121 million raise; London’s BenevolentAI secured $90 million, taking its total inbound investment to nearly $300 million; and Maryland-based Insilico Medicine locked in a further $37 million. As it happens, earlier this year Insilico Medicine partnered with A2A Pharmaceuticals to launch Consortium.AI, a joint venture designed to treat rare diseases with AI.

While all these companies share a common goal, vis-à-vis they’re using machines to speed up drug discovery and development, Healx is carving its niche by “maximizing the value of already-approved drugs,” rather than developing new molecules, while simultaneously focusing on rare genetic diseases.

Looking further down the line, Healx said that it’s targeting 100 rare diseases treatments by 2025.

“Because we start from safe, existing drugs, we can quickly and cheaply translate them in the clinic, which is key to Healx’s mission,” Guilliams said. “The trials for our Fragile X treatments are just the start of the impact we believe our technology is capable of having on drug discovery.”

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