Healx Raises $10M to Speed Therapy Discovery for Batten, Other Rare Diseases

Ana Pena, PhD avatar

by Ana Pena, PhD |

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Batten and rare disease therapies

Healx has raised $10 million to accelerate therapy discovery for rare diseases, including Batten disease.  The company uses artificial intelligence, pharmacology expertise, and patient engagement in an effort to give those with rare diseases a better quality of life.

Balderton Capital, Europe’s major early-stage venture investor, led the financing round, which also included Jonathan Milner and Amadeus Capital Partners.

“Healx has a simple but profound goal: to transform the lives of rare disease patients. Our technology helps us find treatments where none currently exist,” Tim Guilliams, co-founder and CEO of Healx, said in a news release.

Nearly all rare diseases remain without approved treatments. Worldwide, one in every 20 people is affected by some type of rare disease.

Last year, the United States and Europe approved the first medicine to treat Batten disease, BioMarin’s Brineura, an enzyme replacement therapy. Still, the medicine is indicated only for children with a specific type of Batten disease known as CLN2 disease. It slows disease progression but does not provide a cure.

Historically, pharmaceutical companies have invested in so-called “blockbuster” drugs, which are used to treat large numbers of people without regard for individual differences among patients. To serve those with rare diseases, Healx is focusing on artificial intelligence (AI) to speed therapy discovery.

The company owns the AI platform HealNet, which runs machine learning methods to screen large-scale databases (literature, patents, clinical trials, disease symptoms, drug targets, and biological and chemical lab data) in a search for optimal matches between patients and treatments.

The most promising candidates are reviewed by a panel of pharmacology experts to determine therapeutic strategies. Patient advocates, clinicians, and scientific curators also have input.

HealNet has also focused on finding new uses for existing medications, called drug repurposing, or their application in combination with other therapies. These strategies likely accelerate market entry, as existing medications require fewer trials. Laws such as the FDA’s Orphan Drug Act also help reduce the cost of rare disease therapy development.

“The traditional drug discovery process takes 10 to 15 years at a cost of $2 billion per new drug and with a failure rate of 95 percent — it’s broken, it’s slow, it’s high failure, and it’s not economic for rare diseases,” said David Brown, PhD, co-founder and chief scientific officer of Healx.

“However, today’s technology can change that and help 350 million underserved rare disease patients. Healx is showing that we can massively transform the rate of discovery of new medicines, reducing timelines and costs,” Brown said.

In a collaboration with the FRAXA Research Foundation, the company’s platform discovered a compound for fragile X syndrome that was ready for the clinic in less than 15 months and substantially reduced the cost of the drug’s development.

“Our proven approach is to start from existing drugs and apply artificial intelligence to niche disease populations, working with patient groups to accelerate treatment development,” Guilliams said. “Our success comes from our world-class multi-disciplinary team of experts, leveraging cutting-edge artificial intelligence techniques to cut the discovery-to-treatment time from years to months.”

Healx was founded in 2014 by Guilliams, a biochemical engineer and founder of the Cambridge Rare Disease Network; David Brown is the inventor of Viagra and former global head of drug discovery at Roche; and Andreas Bender is a lecturer and researcher at Cambridge University’s Centre for Molecular Informatics.