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8 April, 2021 - 16:10 By Tony Quested

Healx approaches seminal US clinical trial milestone

Cambridge rare disease specialist Healx has struck its latest patient group partnership – with Ataxia UK and FARA, The Friedreich’s Ataxia Research Alliance.

The partnership will seek to identify novel therapies for Friedreich's ataxia, the most common type of hereditary ataxia –  a degenerative disease of the nervous system. 

Friedreich's ataxia is a progressive condition and is estimated to affect one in 30,000 people in Europe and one in 50,000 people in the United States. 
Many patients living with the condition are wheelchair-bound by their late 20s. 

In this pioneering partnership, Healx will combine its AI technology and deep pharmacological expertise with Ataxia UK and FARA’s unparalleled patient insight to drive forward the world’s first treatments for the condition. 

This is the 13th project Healx has added to its discovery and development pipeline and the team hope to launch their first clinical trial – for Fragile X syndrome – in the US later this year. Fragile X syndrome is a genetic disorder characterised by mild-to-moderate intellectual disability. 

Healx is an AI-powered, patient-inspired technology business accelerating the discovery and development of rare disease treatments at scale. Working in collaboration with Ataxia UK and FARA, Healx will leverage its state-of-the-art AI platform and drug discovery expertise to develop novel treatments for Friedreich’s ataxia – a rare neurodegenerative condition that causes issues with balance, speech and coordination. 

Friedreich’s ataxia is a progressive, inherited form of ataxia and is caused by a genetic fault in a gene that encodes a protein called frataxin. Whilst the condition is the most common type of inherited ataxia, it is still very rare, with estimates suggesting that roughly one in 30,000 people in Europe and one in 50,000 people in the United States have the condition. 

Whilst symptoms vary, the condition is typically characterised by poor balance and coordination, difficulty speaking, a curved spine and heart problems. Some patients may also experience diabetes and chronic fatigue. 

On average, symptoms first appear when an individual is around 5 – 18 years old and will worsen over time, meaning many patients have to use wheelchairs by their late 20s. 

There is currently no cure or approved pharmacological treatment specifically for Friedreich’s ataxia but there are some treatments available to manage the symptoms. 

This partnership will aim to find, and accelerate to the clinic, some of the world’s first disease-modifying treatments for the condition. 

To do this, Healx will leverage its cutting-edge technology and innovative drug repurposing approach, and combine this with Ataxia UK and FARA’s unrivalled patient and scientific insight. 

At the heart of Healx’s technology is Healnet – the company’s AI drug discovery platform. As one of the only AI companies in the world to focus specifically on rare diseases, Healx has developed the world’s most comprehensive biomedical knowledge graph for rare diseases which comprises millions of data points from clinical, pharmacological and scientific literature. This graph is analysed by more than 10 different AI models to rapidly uncover novel connections between drugs, diseases and other relevant medical information. 

These AI models have been specially trained to predict the effectiveness of repurposing and combining drugs for rare disease treatments, and in the case of this project, the team have developed a novel metabolic model that they can use to identify drug candidates.  

Bruce Bloom, chief collaboration officer at Healx, says: “We are very excited to be working with Ataxia UK and FARA, who are both leaders in the field of ataxia research and support, and who share our commitment of putting patients right at the heart of treatment discovery.

“Together, we hope this pioneering partnership will accelerate much-needed treatments for Friedreich’s ataxia from AI prediction to clinical trial and directly to patients.”

This partnership is the latest to join Healx’s growing portfolio of rare disease collaborations. Last month, Healx announced that it would be working with the UK-based PKD Charity to find novel therapies for Autosomal Dominant Polycystic Kidney Disease and Autosomal Recessive Polycystic Kidney Disease. 

And in January, the team shared the news that they would be collaborating with Mission: Cure to identify the world’s first treatments for chronic pancreatitis. 

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