The AMT-130 Study
An Exciting development in the Huntington’s research space.
uniQure has released encouraging interim results from the AMT-130 therapy trials for Huntington’s Disease (HD). Since the trials began, AMT-130 has faced some challenges. Initial treatments seemed promising, but in August 2022, serious side effects were reported in some participants receiving the high dose of AMT-130. This led to a three-month pause in trial enrollment. Fortunately, after implementing new safety measures, the trials resumed, and uniQure announced that the trials would continue as planned. The positive interim results bring hope and optimism to the Huntington’s Disease community.
About AMT-130
Developed by uniQure, AMT-130 is the first gene therapy for Huntington’s Disease (HD). Unlike other therapies in clinical trials, AMT-130 is a one-and-done treatment designed to reduce the levels of the huntingtin protein in the brain. This therapy uses a harmless virus to deliver genetic material that lowers huntingtin in brain cells, administered through a specialised brain surgery.
Study Details
The AMT-130 gene therapy is being tested in two clinical trials: HD-GeneTRX-1 in the US and HD-GeneTRX-2 in Europe. These trials involve 39 participants who received either a high dose, a low dose of AMT-130, or underwent sham surgery (an equivalent of someone receiving a placebo). Participants are tracked for four years post-surgery with various clinical, biomarker, and brain imaging assessments to evaluate the safety and effectiveness of AMT-130.
Interim Outcomes:
The primary aim of these trials is to determine whether AMT-130 is safe for people with HD. Additionally, the trials gather data that might suggest how well AMT-130 works and its impact on HD symptoms.
Key points from the latest interim update include:
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- Safety: Both low and high doses of AMT-130 appear relatively safe, with manageable side effects like headaches and procedural pain. Importantly, no new serious side effects have been reported since the trial pause.
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- NfL Biomarker: Neurofilament light (NfL) levels, which indicate brain health, initially spiked post-surgery but then declined rapidly. This suggests that AMT-130 may be slowing HD progression. Recent data shows that NfL levels in treated participants are significantly below expected levels, indicating a potential slowing of brain health decline.
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- Clinical Measures: Using the Composite Unified Huntington’s Disease Rating Scale (cUHDRS), uniQure found that disease progression was slowed by about 80% in high-dose participants compared to natural history data. However, this finding must be interpreted cautiously due to the small sample size.
Next Steps
Although the results are promising, the trials are ongoing, and more data from a larger group of participants is needed. Excitingly, the FDA recently granted AMT-130 Regenerative Medicine Advanced Therapy (RMAT) designation, facilitating more frequent interactions and priority review. uniQure plans to meet with the FDA in the second half of 2024 to discuss the development and potential approval pathway for AMT-130.
If you are a professional who is looking get more information on the interim update on the AMT-130 trial, please visit the press release statement for a detailed update:
If you are wanting to read more about the trial and the drug for yourself or for your loved ones, we encourage that you click the click below to HDBuzz, where the press release statement has been simplified for an improved understanding for everyone.
Conclusion:
While it’s early days, the interim results from the AMT-130 trials are promising, offering cautious optimism for the HD community. Continued research and data collection will be crucial in determining the long-term effectiveness and safety of this innovative gene therapy.