Rare Daily Staff

Healx has entered into an agreement with Boehringer Ingelheim to identify indications related to rare neurological diseases.

The project will leverage Healx’s AI-based drug discovery platform, Healnet, and expertise in rare diseases and pharmacology, to identify potential new indications for assets from Boehringer Ingelheim’s pipeline.

Healx will use its data analysis and technological capabilities to help Boehringer Ingelheim prioritize selected indications for further research by predicting and matching treatments to rare diseases.

Healx has significant experience in the area of rare neurological disorders, such as Fragile X syndrome and Pitt-Hopkins syndrome. The company’s AI-based drug discovery platform Healnet 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 neurodevelopmental disorder Pitt-Hopkins syndrome.

“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,” said Tim Guilliams, founder and CEO of Healx. “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, those affecting fewer than 1 in 2,000 people, have been identified to date, with 250 new rare diseases classified annually. Collectively, this impacts more than 400 million individuals worldwide with less than 5 percent of these rare diseases having approved treatments. There is therefore a substantial unmet medical need.

Founded in 2014, Healx’s mission is to advance 100 rare disease treatments into the clinic by 2025 using 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 organizations to explore novel pharmacological routes and indication expansion opportunities.

Photo: Tim Guilliams, founder and CEO of Healx

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