Healx begins Phase II of AI-found rare disease treatment with new $47M

Healx is set to push its leading drug candidate for neurofibromatosis Type 1 (NF1) into a Phase II clinical trial by the end of this year. This advancement is facilitated by a substantial $47 million Series C funding round, co-led by R42 Group and Atomico. With this latest investment, the AI-driven rare disease company has now secured a total funding of $110 million.

The focal point of Healx's development is HLX-1502, its top candidate for NF1. This drug, along with other projects in oncology, renal, and neurodevelopmental disorders, has been discovered through Healx’s proprietary Healnet AI platform. This technology is instrumental in identifying therapeutic candidates that have reduced clinical risk by linking biological and chemical entities, making them apt for new treatments.

Healnet's capabilities extend beyond just identification; it can also reverse disease multiomic signatures, which allows it to pinpoint potential therapeutics. This is made possible by leveraging Healx’s extensive library of lab-generated phenotypic and transcriptomics data. By uncovering new disease mechanisms via AI, Healx aims to design second-generation compounds, employing predictive modelling and other advanced tools.

A further ambition of Healx is to develop "chemically-aware" large language models (LLMs). These models will be trained on biological data to "speak the language of the cell" and unveil new targets for rare diseases. This innovative approach is set to revolutionize the way new treatments are discovered and developed.

The recent funding will also be allocated towards generating crucial preclinical data for several additional compounds identified via Healx’s generative AI drug discovery engine. CEO Tim Guilliams emphasized that these compounds focus on rare diseases with significant unmet needs. This includes additional nerve-related tumor disorders, autosomal dominant polycystic kidney disease, and neurodevelopmental disorders such as Angelman syndrome.

The Series C round saw participation from both new and existing investors. Notable contributors include Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h, and VU Venture Partners. This mix of investors underscores the confidence and support in Healx’s innovative approach to tackling rare diseases through AI-driven solutions.

In summary, Healx’s recent $47 million funding round is a significant milestone that will propel the company’s NF1 drug candidate into Phase II trials and drive further development of its robust pipeline. By leveraging cutting-edge AI technology and a rich data library, Healx is poised to make groundbreaking advancements in the treatment of rare diseases, addressing crucial unmet medical needs.

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