‘Pivotal time’ as Healx raises $47m in Series C round

Healx’s $47m Series C raise propels AI-driven biology from the theoretical realm straight into a Phase 2 clinical trial as the Cambridge company pushes towards FDA approval for HLX-1502, an oral treatment for NF1 (neurofibromatosis Type 1).

Healx is exploring how models trained on raw biological data that speak the language of the cell can identify and exploit targets and result in greater understanding of rare disease biology.

The company was founded in Cambridge by Tim Guilliams, a biochemical engineer and tech entrepreneur, and David Brown, the co-inventor of Viagra and former global head of drug discovery at Roche. That was back in 2014, and the company has since raised more than $110m for its mission to apply emerging technologies to speeding the discovery of rare disease treatments.

“It is truly a pivotal time at Healx,” says Tim. “By December, we will advance HLX-1502 into a Phase 2 clinical trial in patients with NF1 both for plexiform neurofibroma, a genetic condition with limited treatment options, and for cutaneous neurofibroma, for which there are no FDA-approved options.

“We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine. Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumour disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman Syndrome.”

NF1 is a rare genetic disorder associated with predisposition to develop multiple benign and malignant tumours. It affects approximately one in 2,500 individuals. Two notable types of tumours associated with NF1 are plexiform neurofibromas and cutaneous neurofibromas.

Plexiform neurofibromas are complex tumours growing aggressively along nerves which often leads to significant morbidity and risk of malignant transformation. These tumours can affect various parts of the body, causing functional impairments, disfigurement and pain and requiring multidisciplinary management.

Currently, only one treatment option is available for some children with plexiform neurofibromas which is, however, associated with tolerability and safety concerns including gastrointestinal, heart, eye and skin toxicity.

Cutaneous neurofibromas are benign tumours that often cause significant symptoms, leading to impairment in quality of life, and also result in considerable cosmetic concerns. There are no approved treatments for NF1-associated cutaneous neurofibromas, leaving a major unmet need for the estimated 3 million people with NF1 worldwide.

HLX-1502 has received Orphan Drug and Rare Pediatric Disease designations from the FDA for treating NF1. These FDA designations provide several benefits to encourage the development of treatments for rare diseases and further highlight HLX-1502’s potential to significantly improve the lives of NF1 patients.

Simone Manso, Healx head of neurofibromatosis therapy development, said: “I could not be more excited to start this trial soon.

“Our treatment – with its novel mechanism of action and potential for a compelling and differentiated safety profile – represents a significant advancement in the NF1 field and a much-needed hope for the NF1 community. It has the potential to greatly improve NF1 patients’ lives, which is by far what matters the most and what drives us.”

Tim, speaking to the Cambridge Independent on a video call, outlined the company’s development since raising $56m in a Series B round in 2019.

“Healx is now 55 people, 35 are in the Cambridge area, then there’s some people in Europe and the US – both in Chicago and New York.

“It’s fantastic news that HLX is about to start Phase 2 trials and the proceeds of fundraising is firstly to fund the trials, second to progress the wider therapeutic pipeline towards clinic, and thirdly to enhance our AI platform HEALNET, which has some very exciting things happening.”

Healnet is Healx’s unique AI-driven drug discovery engine that’s tailored to discover treatments for rare diseases. The platform enables the design of second generation compounds using virtual screening, predictive modelling and phenomics (see panel).

So we heard a lot about repurposing existing drugs in Healx’s earlier days, is that still the process or are entirely new drug treatments in the pipeline?

“Yes,” says Tim. “Part of the improvement and development since the last round has been to build a platform that is agnostic, whether it’s a new molecule or an existing molecule. The goal is to match molecules to a new approach for a disease and best candidates for novel rare disease discovery and translate them to clinic in a cost-efficient way.

“We work with them to really understand biology in a different way and we have a lab as well – a research lab where data is being generated on particular diseases. It could be that other companies do the clinical trials but we’re focused on using the platform ourselves to find opportunities so we’re developing our own pipeline, and we do some partnerships. After that, we have the option to continue trials or partner depending on what’s best for the programme. But we don’t hire out Healnet.”

Healx’s platform has taken cancer treatments beyond kinase inhibitors. The kinase inhibitors are a large group of unique and potent antineoplastic agents which specifically target protein kinases that are altered in cancer cells and that account for some of their abnormal growth.

AstraZeneca has spent two decades on a significant number of kinase drug discovery programmes. Since the first approval of imatinib in 2001, there have been more than 30 FDA-approved kinase inhibitors brought to market. But the side effects – depending which kinase inhibitors (TKIs) and type of cancer are involved – can include tiredness, gastrointestinal disorders, muscle cramps and bone pain, hypertension and skin rashes.

“We are the only company discovering drugs with so few side effects,” says Tim.

There is also a cost implication. Discovering drugs is a complex process – from every 5,000 compounds made in the discovery phase, one drug is approved. Most don’t survive the first rounds of profiling. That’s changed with Healnet.

“There are 10,000 rare diseases, and 95 per cent don’t have any treatments, and so there’s an opportunity here to use AI at a fraction of the cost and time,” concludes Tim.

“We started in 2019 so we’re exceptionally proud of what we’ve achieved here and we work very closely with groups and foundations, to really understand patient needs and how we can find a breakthrough.”

The Series C round was co-led by Silicon Valley-based R42 Group and Atomico, one of Europe’s largest venture capital firms, with participation from new and existing investors including Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h and VU Venture Partners.

Proceeds of the financing will be used to advance Healx’s pipeline of medicines in rare oncology, renal and neurodevelopmental disorders, including advancing its lead programme HLX-1502 through a Phase 2 clinical trial for the treatment of NF1.

In conjunction with the financing, Stanford Medicine adjunct professor Ronjon Nag, founder of R42 Group and 2024 Silicon Valley Hall of Fame AI inductee, joins the board of Healx.