Healx and Boehringer battle rare neurological diseases
Cambridge-based Healx and German powerhouse Boehringer Ingelheim have joined forces to identify indications related to rare neurological diseases.
Healx will use its AI-driven data analysis and technological capabilities to boost Boehringer Ingelheim’s efforts in prioritising selected indications for further research. The Healx Healnet platform draws on the most comprehensive knowledge graph of rare disease data to intelligently predict and match treatments to rare diseases and will play a key part in the collaborative effort.
Healx also has significant experience in the area of rare neurological disorders such as Fragile X syndrome and Pitt-Hopkins syndrome. Healnet has proven success having identified eight repurposing drug candidates with potential benefits for Fragile X syndrome, which were not considered for the condition prior to Healx’s involvement.
As a result, several candidates have demonstrated efficacy in vivo and one candidate is now progressing toward a Phase 2a trial.
In another project, carried out in collaboration with the Pitt Hopkins Research Foundation, Healnet was successfully used to investigate, predict and progress novel treatment options for the ultra-rare neuro-developmental disorder Pitt-Hopkins syndrome.
Dr Tim Guilliams, founder and CEO of Healx, said: “Healx is dedicated to transforming the lives of rare disease patients by intelligently matching and developing potential drug treatments to their conditions.
“We believe that sophisticated technologies, such as AI and machine learning methods, will be fundamental to any drug discovery and development project in the future.
“The project with Boehringer Ingelheim presents an opportunity for further innovation in identifying rare disease candidates for patients whose medical needs are not currently met. Harnessing BI’s expertise for drug development, especially in rare neurological conditions, in combination with our AI-powered drug discovery platform, will enable faster identification of potential new treatments.”
More than 7,000 rare diseases have been identified to date (diseases affecting fewer than one in 2,000 people), with 250 new rare diseases classified annually.
Collectively, this impacts over 400 million individuals worldwide with less than five per cent of these rare diseases having approved treatments – evidencing a substantial unmet medical need.
Healx’s mission is to use its AI platform, deep pharmacological expertise and extensive patient engagement to improve the quality of life and care for patients with rare diseases. Its business model is to develop its own pipeline of proprietary drug candidates, as well as to work in partnership with a range of organisations to explore novel pharmacological routes and indication expansion opportunities.