uniQure: Some hope yet for bulls?
NIHR and DHSC in the UK are backing AMT-130
I don’t have a position in QURE, though I have been following the story closely since I hold a position in CLPT and QURE is a biopharma partner of theirs. Today, the UK Government Office for Science published a very interesting article; it appears the NIHR and DHSC are officially backing AMT-130.
Link to the article: AMT-130 Gov UK
The 2 most notable quotes in my view are:
“The National Institute for Health and Care Research (NIHR), funded by the Department of Health and Social Care, supported a landmark clinical study into a new gene therapy, AMT-130 – the first gene therapy to be tested in people with Huntington’s disease. The study, funded by the life sciences company uniQure, showed that participants receiving a higher dose of the treatment experienced 75% less disease progression after three years, alongside slower decline in movement and thinking. For patients and families, this represents long-awaited hope”
“The NIHR Research Delivery Network provided further research staff and supported a long-term observational research study, which provided a suitable comparison group of those with Huntington’s who were not receiving the treatment, ensuring the results were robust”. As I see it, this directly questions the FDA’s suggestion for a sham procedure RCT
If not familiar with CLPT, UK backing of AMT-130 is a potential catalyst for $CLPT for three reasons:
Platform Validation: Confirms that AMT-130’s delivery requires ClearPoint’s specific MRI-guided navigation and SmartFlow cannula.
Proof of Concept: Successful trials serve as “live demos,” proving the hardware’s precision in complex clinical settings.
Faster Revenue: Regulatory flexibility in the UK/EU could lead to earlier commercial sales of ClearPoint’s disposables compared to the US.
The full article is below:
Slowing Huntington’s disease: how science advice enabled a breakthrough treatment
From: The Government of Science
”Around 8,000 people in the UK are living with Huntington’s disease, a rare and fatal condition caused by a single genetic mutation. Until now, there has been no treatment proven to slow its progression. Through science advice and publicly funded research infrastructure, that is beginning to change.
The National Institute for Health and Care Research (NIHR), funded by the Department of Health and Social Care, supported a landmark clinical study into a new gene therapy, AMT-130 – the first gene therapy to be tested in people with Huntington’s disease. The study, funded by the life sciences company uniQure, showed that participants receiving a higher dose of the treatment experienced 75% less disease progression after three years, alongside slower decline in movement and thinking. For patients and families, this represents long-awaited hope.
An NIHR Biomedical Research Centre and NIHR Clinical Research Facility provided expert scientific advice, specialist NHS facilities, and skilled research staff. The NIHR Research Delivery Network provided further research staff and supported a long-term observational research study, which provided a suitable comparison group of those with Huntington’s who were not receiving the treatment, ensuring the results were robust.
This study is one of nearly 18,000 projects enabled by NIHR research infrastructure in a single year. By bringing together trusted science advice, the NHS, and industry, NIHR helps turn cutting-edge ideas into real-world treatments that improves the health and wealth of the nation. It also strengthens the UK’s position as a global leader in life sciences and innovation.
This is science advice in action – supporting better decisions, safer research, and breakthroughs that can save lives. It Celebrates Science Advice, highlighting how evidence and expertise underpin progress across public services and deliver real benefits for people and communities”
Check out my post below explaining what I am doing with my CLPT position also:
