Healx , a U.K. startup using AI to discover new drugs for rare diseases, has raised $47 million in a Series C round of funding co-led by Europe’s Atomico and Silicon Valley-based R42 .
The company also revealed it has received regulatory clearance to start phase two clinical trials for a new drug in the U.S. later this year.
Healx pitches itself as a “drug discovery pipeline,” one that identifies hidden connections between existing chemical compounds (e.g. drugs and pharmaceuticals at various stages of the development cycle) and rare diseases. It does this by collating myriad public and proprietary data sources, including biomedical literature, disease and biochemical datasets, clinical trials, patents, and more, creating a “biomedical knowledge graph” of rare diseases.
“Our AI platform and expert teams enable us to identify and discover novel disease biology and match it with suitable small molecules,” Healx co-founder and CEO Tim Guilliams told TechCrunch. “Our digital biology team focuses on understanding complex biological disease signatures, while our digital chemistry team uses AI to match these signatures with chemicals, small molecules and drugs, using computational techniques such as virtual screens and generative chemistry to do so.”
Typically, pharmaceutical companies adopt what is often referred to as a “one disease, one target, one drug” approach to drug discovery. This means they start by focusing on a single molecular target that’s responsible for a disease, and then set about designing a drug to target that specifically. The problem though is that this is hugely time consuming with a high fail-rate , which is where Healx is setting out to help, with the promise that it can analyze millions of drug and disease data points in tandem to find hitherto undetected connections that could present new treatments using known compounds.
Founded out of Cambridge in 2014, Healx is the brainchild of Guilliams, who has a PhD in biophysics and neuroscience from the University of Cambridge; and chairman David Brown , formerly global head of drug discovery at Roche , though he is perhaps better known as co-inventor of famed erectile dysfunction drug Viagra .
Drug design
With a fresh $47 million in the bank, Healx is gearing up to launch a Phase 2 clinical trial for a novel treatment it developed for Neurofibromatosis Type 1 ( NF1 ) — a genetic disorder that causes tumor growth on human nerves. Though a rare disease, NF1 is considered one of the more common genetic disorders , affecting up to 1 in 3,000 individuals. While the tumors are usually benign, they can cause other symptoms related to the skin, eyes and nervous system, and they can also eventually become cancerous.
Healx had previously secured investigational new drug ( IND ) approval from the U.S. Food and Drug Administration (FDA) in 2021 for Fragile X syndrome , while it’s also in the early AI-discovery or preclinical stages for a number of other conditions spanning oncology (cancer), renal (kidney), and neurology (nervous system).
However, the FDA has now cleared the way for Healx to proceed with a Phase 2 trial of its NF1 lead candidate, dubbed HLX-1502, which is a tablet taken orally. These trials are expected to start by the end of 2024, and will focus on young adults with NF1 and inoperable plexiform neurofibroma , a specific type of tumor associated with NF1.
The company arrived at HLX-1502 using an existing drug that had been developed for something else entirely, but which never launched in the U.S. The company says it is reformulating it, and because it already had existing safety data attached to it due to previous testing in healthy patients, this gives the company a greater degree of confidence that it will have fewer side-effects.
And this is important, given that Healx is focusing on a disease that often involves benign tumors in otherwise healthy patients.
“There’s a lot of cancer treatments out there where, basically, they kill everything — the healthy cells, the unhealthy cells, and there are a lot of side effects,” Guilliams said. “And so for the patient community and the families, for those who have the benign form it can live 40 to 50 years. So it doesn’t make sense to put them on chronic cancer drugs for decades with side effects that are very strong.”
Healx’s technology looks at all manner of existing drugs that are in development, those that have perhaps been shelved for various reasons but which have enough data attached to them to make them useful for others to build on.
“There’s hundreds of thousands of compounds and small molecules,” Guilliams said. “But once you start seeing patterns between different data types and predictions, you can try to select the drug that you believe is the most de-risked. AI helps us to find the best possible drug for a particular disease biology. And so here, we wanted something that went specifically to the nerve and where we believed there would be fewer side effects.”
This all leads to one obvious question: if Healx has developed its leading NF1 candidate by building on work previously carried out by others, does it actually own this candidate? According to Guilliams, Healx filed and owns the patents for HLX-1502 specifically for the use of treating NF1 and “other nerve related diseases,” while the FDA has also given it new drug product exclusivity in the U.S. market. This is usually given out for new chemical entities (NCEs), which is defined as drugs that “are innovative” or where there have been “significant changes in already approved drug products, such as a new use.”
“Comprehensive commercial and IP analysis is always conducted when selecting our lead drug candidate taken to the clinic,” Guilliams said.
However, Healx didn’t reveal the actual origins of the original compound or the safety data.
Drug money
Prior to now, Healx had raised around $68 million, the bulk of which came via a $56 million Series B round in 2019 . The company also recently announced an investment from one of its long-term partners called the Children’s Tumor Foundation (CTF). The CTF will provide “milestone-driven payments” to Healx as it works toward and through its clinical trials — it’s worth noting that although the CTF is a nonprofit, this financial arrangement isn’t a grant, though Healx hasn’t revealed the terms of the investment.
What’s particularly notable is that Healx’s Series C round is $9 million less than its Series B round five years ago which, while not entirely unheard of, is a little unusual in the startup funding realm. Indeed, when asked what the most capital-intensive part of Healx’s business was, Guilliams said that it’s carrying out clinical trials — a phase the company is only just now embarking on with its NF1 candidate.
TechCrunch has learned independently that Healx did initially try to raise significantly more than what it had hoped for its Series C round, and the company also went through a roughly 45% reduction in headcount last year via a series of voluntary and compulsory redundancies — the equivalent of around 70 staff.
Healx confirmed as much to TechCrunch, with Guilliams pointing to the economic downturn and macroeconomic factors being less than kind to the startup fraternity these past few years .
“It is important to note that when the markets crashed, we were indeed in the process of raising a larger round,” Guilliams said. “We pulled that offering off the table at that time, and we then adapted effectively to the new market conditions by streamlining operations, refocusing on our core strengths, and becoming more cost-effective and lean. This allowed us to maintain our momentum and continue making significant progress, which we are proud to announce now. Our advancements in generative AI have further enhanced our capital efficiency, enabling us to achieve more with fewer resources. We then opened up the new financing round with a better perspective on what was needed to accomplish our goals.”
Thus, Guilliams is presenting the latest incarnation of its Series C fundraise as being oversubscribed, noting that it has now raised more than it targeted.
“Given the challenging market conditions for deep tech and biotech funding over the past two years, our ability to secure this investment underscores the confidence our investors have in our approach and technology,” Guilliams added.
Healx’s raise also comes amid a swathe of investments into AI-driven drug discovery platforms, including Sam Altman-backed Formation Bio which recently raised $372 million , as well as a newly founded startup called Xaira which launched with $1 billion in funding — both significantly more than what Healx has raised in the entirety of its history.
Guilliams stressed that much of the foundation work required to get its first drug to market has been done already, meaning that it doesn’t necessarily require as much capital this time around as you might think. And he also noted that his company’s valuation has gone up since its last funding round, though declined to disclose specifics.
“What’s important is the quality of your programs — picking those that have the most potential,” Guilliams said. “The market size for NF1 is $16 billion [according to Healx’s own internal commercial analysis], and if it [its candidate] works on NF1, it’s likely to work on other nerve-related diseases — we’re testing those pre-clinically already. In rare diseases, the clinical trials are smaller, so we don’t need a ton of money, which, in the current markets, is a good place to be.”
Guilliams also confirmed the company did undergo redundancies, though declined to go into specifics around numbers or circumstances beyond the fact that it included “both voluntary and compulsory measures.”
“Despite the challenge of letting go of exceptional people, this process ultimately made us a stronger and more focused team,” he said. “This restructuring was part of our strategy to streamline operations and enhance our focus on key therapeutic areas and technological advancements.”
Market entry
Today, Healx counts around 55 employees, roughly half of whom “code,” be that in the realm of machine learning or computational biology. The other half are the clinical and pharmacology team, who “work hand-in-hand with the techies,” Guilliams said.
While the bulk of its team is based in Cambridge (U.K), the company is now looking to expand on the ground in the U.S. where its clinical trials are set to start by December. After that, we are likely looking at several more years before anything comes close to hitting the market — such is the stringent regulatory requirements around any new drug.
“Conservatively, I would say we would expect clinical results the first half of 2026, and then depending on those results and interactions with the regulators, we may need to do a second clinical trial — and then potentially register the drug,” Guilliams said.
Aside from lead backers Atomico and R42, Healx’s Series C round included participation from Ayana Capital, Balderton, Btov, Global Brain, Jonathan Milner, O2h and VU Venture Partners.