Biotech founded on charity science raises £48 million to develop next-generation dementia treatments

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By Liz Burtally | Friday 13 October 2023

Earlier this week, we heard some great news: AstronauTx, a UK-based biotech company founded on science funded by Alzheimer’s Research UK, has shown such promise that it’s attracted £48 million from private investors. This funding will allow it to take its work to a whole new level.   

The biotech was launched in 2019, to capitalise on discoveries made at the Alzheimer’s Research UK UCL Drug Discovery Institute (DDI). The company initially focused on modifying the behaviour of brain cells called astrocytes, to try to prevent them from causing damage to nerve cells that causes Alzheimer’s disease. 

By raising more investment, AstronauTx can now expand the number of experimental drugs in its pipeline. The funds will also be used to launch a clinical trial for one of its most promising drugs in people with Alzheimer’s, which is an incredible feat. 

So how did science carried out in a lab lead to a multimillion-pound spin-out company that’s developing cutting-edge treatments for people with Alzheimer’s?  

It’s a success story that has involved Alzheimer’s Research UK every step of the way. 

 

The ground-breaking science 

Earlier this year, two new Alzheimer’s drugs – lecanemab and donenemab – made waves around the world after they showed success in clinical trials. Both of these drugs work in a similar way – they remove the hallmark amyloid protein that that builds up in the brain during Alzheimer’s. But amyloid build-up isn’t the only issue in Alzheimer’s – there are many other disease processes at play, presenting more opportunities to develop effective treatments.  

Astrocytes are cells that play a key role in helping nerve cells work properly, as well as keeping the blood-brain barrier healthy. Historically overlooked as bystanders, these star-shaped cells are now becoming an increasing focus for researchers and drug developers, as more information emerges about their role in neurological diseases like Alzheimer’s.  

In Alzheimer’s disease, astrocytes’ protective functions become disrupted, leaving nerve cells vulnerable to damage from the disease. A key insight into this process emerged in the mid-2010s, as researchers at UCL revealed new ways to restore astrocytes’ beneficial effects. They were in the perfect place to take their research further into clinical development.  

 

Taking the promising discoveries to the next stage 

The UCL Drug Discovery Institute, alongside the ALBORADA Drug Discovery Institute in Cambridge and Oxford Drug Discovery Institute, is part of our flagship Drug Discovery Alliance (DDA). Launched in 2015, the DDA acts as a bridge between academic research and the pharmaceutical industry, ensuring any promising discoveries in the lab are rapidly translated to life-changing treatments for people with dementia.  

At the heart of UCL’s world-class neurology research environment, the UCL DDI has cutting-edge drug discovery capabilities within its laboratories. Here the team were able to take their work on astrocytes to the next stage, screening chemicals that could restore these cells’ protective function, and which also had the potential to form the basis of experimental drugs. 

Although the team had unearthed a promising new drug target avenue that looked viable, more investment was needed to prove this could be successfully applied to treating Alzheimer’s disease.  

 

Growing and nurturing the company 

While the ground-breaking research was being carried out at the UCL DDI, Alzheimer’s Research UK was working with industry and government to create the Dementia Drug Fund (DDF) – a collection of venture capital funds set up in 2015 to exclusively support the development of companies developing new treatments for dementia.  

By supporting these companies to develop these promising ideas, the Fund, managed by SV Health Investors, fills a vital gap between lab-based drug discovery and larger-scale clinical trials. It is now the world’s largest venture capital fund focused on neurodegenerative conditions like dementia.  

In 2019, the UCL DDI secured a £6.5m ‘seed’ investment from the DDF, UCL Technology Fund and the UK Future Fund, and AstronauTx was officially born. The biotech assembled an expert team to take their astrocyte work forward, which included Dr Ruth McKernan CBE, former chair of Innovate UK and one of Alzheimer’s Research UK’s Trustees, who took the helm as Chair of the company.  

AstronauTx is just one of a growing number of enterprises whose work is being made possible thanks to investment from the DDF. 

 

The first milestone and next steps 

A few years later, AstronauTx has now been able to raise nearly £50 million in what’s known as ‘Series A’ funding. This type of finance is only available once a company has shown a viable business model, with strong potential for growth, and it marks the first significant financing of a start-up company.  

This is an incredibly exciting development, as many so biotech start-ups never get this far.  

The company will use the investment to develop treatments applicable across multiple neurological conditions, according to Dr McKernan. Progress towards a compendium of new drugs is thankfully well underway,” she said. Our treatments will be oral drugs, applicable across multiple neurodegenerative conditions,” which will, she hopes, work alongside others that are currently in late-stage development. 

 

A turning tide 

Until recently, dementia research has been seen globally as a costly, highly risk endeavour, beset by failure and disappointment. As a result, investors have tended to chased larger rewards in areas like cancer. But the tide is now turning – and this will accelerate thanks to successes like AstronauTx.  

As our chief executive, Hilary Evans, told the media, the news is a huge testament to the hard work of everyone involved. “It’s incredible to see a UK-based spinout able to raise £48m in a such a challenging environment,” she said, pointing to the “real sense of hope and optimism around dementia research today” thanks to the imminent arrival of first-generation Alzheimer’s treatments like donanemab and lecanemab.  

Dementia research is now starting to thrive and the momentum from lecanemab and donanemab – the first treatments that can slow the progression of Alzheimer’s – is building. 

As UCL Drug Discovery Institute’s Chief Scientific Officer, Professor Fiona Ducotterd, said, the investment sends an “important message” that the field is moving forwards, and that new medicines are on the way.  

“We are delighted for our collaborators at AstronauTx,” she said, pointing to the “strong support” from the investment community to progress the company’s work to patient trials. 

 

Wider benefits 

As the announcement shows, dementia research doesn’t just promise to deliver benefits to people living with dementia. It also represents a £48m investment in UK science. In fact, as our recent report shows, investment in dementia research boosts the wider UK economy, bringing benefit to the whole of society. For every £1 invested in dementia research since 2019, £2.59p has been generated for the UK economy – and our analysis suggests that this beneficial effect is predicted to rise even further to £3.15p by 2040.  

But to nurture and maintain these benefits, and keep up momentum towards a cure for dementia, we need to see sustained investment from government, industry and the charity sector. As Hilary said, “We’ll need to keep our feet on the accelerator to really deliver for people with dementia. The condition remains one of the world’s largest unmet medical needs, affecting over 55m people across the globe.  

“We will keep searching for newer and better treatments, that target a range of diverse biological processes, to tackle this growing crisis and continue our mission for a cure,” she added. 

AstronauTx’s success brings a multitude of gains – hope for the future where we may have multiple Alzheimer’s treatments, greater innovations in drug discovery, and a boost to the UK economy. It’s also a shining example of how innovation and investment will bring us closer to a world free of the fear, harm and heartbreak of dementia. 

Ducotterd agrees.It’s essential that we continue to break ground, discovering and developing the next generation of Alzheimer’s and other dementias therapeutics, to help all patients and their families living with these terrible conditions.” 

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About the author

Liz Burtally

Senior Science Communications Manager