Additional data from the ongoing Phase I/II clinical studies of AMT-130
What is the latest news about AMT-130?
uniQure has announced further results from its studies of AMT-130, an experimental gene therapy for Huntington’s disease.
The latest data continue to suggest that people receiving the higher dose may be experiencing slower progression of Huntington’s disease compared with an external comparison group of people who have not received the treatment. However, the studies involve relatively small numbers of people and the latest four-year results give a more mixed picture, so they need to be interpreted carefully.
At three years, updated data from all 15 people receiving the high dose showed an 80% slowing of disease progression using cUHDRS, a measure which combines movement, thinking and people's ability to carry out everyday activities. A separate measure called Total Functional Capacity (TFC), which looks at people's ability to function independently, showed a 67% slowing in decline.
What do the four-year results show?
At four years, data were available from 12 people receiving the high dose. Compared with an updated external control group, the results showed a 44% slowing of disease progression on cUHDRS and a 61% slowing in decline on TFC.
Importantly, the 44% result on cUHDRS did not reach statistical significance. This means we need to be cautious about how much can be concluded from this particular result. However, the result for TFC continued to show a meaningful difference compared with the external control group.
There are also limitations with the external comparison group at this later stage of the study. uniQure has said that missing data and what is known as “survivor bias” may have affected the four-year comparison. In simple terms, people whose disease was progressing more quickly were more likely to have dropped out of the external comparison group, potentially leaving a healthier group against which the people who received AMT-130 were being compared.
uniQure therefore also carried out an additional analysis using its previous external control group. This showed a 54% slowing in disease progression on cUHDRS and 68% on TFC at four years. However, because this was a post-hoc analysis, rather than the main planned comparison, these findings also need to be interpreted cautiously. The figures were included in the information shared by uniQure today.
What is happening in the UK?
There has also been an important development regarding possible approval of AMT-130 in the UK.
uniQure has held a pre-submission meeting with the Medicines and Healthcare products Regulatory Agency (MHRA), the organisation responsible for regulating medicines in the UK. The meeting included discussions about the clinical evidence and manufacturing information needed for a formal application. uniQure announced in April that it planned to submit a Marketing Authorisation Application (MAA) for AMT-130 in the UK during the third quarter of 2026.
This does not mean that AMT-130 has been approved in the UK. It means uniQure is taking the steps needed to ask the MHRA to consider the treatment for approval. The MHRA would then need to assess the evidence before deciding whether the treatment can be authorised for use in the UK.
What does this mean for people affected by Huntington’s disease?
These remain encouraging developments, particularly because there is currently no approved treatment that can slow the progression of Huntington’s disease. The results suggest that there may continue to be a treatment effect several years after the one-off gene therapy.
However, there are still important uncertainties. AMT-130 remains an investigational treatment, relatively small numbers of people have received it in these studies, and the difficulties with the external control group make some of the longer-term results harder to interpret.
The four-year results therefore shouldn't be viewed in isolation. While the main cUHDRS analysis at four years did not reach statistical significance, the TFC findings and additional analyses provide important context alongside the stronger three-year results.
So, while there continues to be reason for cautious optimism, we still don't know whether AMT-130 will ultimately be approved in the UK or, if it is approved, what future access to the treatment would look like.
uniQure has announced further results from its studies of AMT-130, an experimental gene therapy for Huntington’s disease.
The latest data continue to suggest that people receiving the higher dose may be experiencing slower progression of Huntington’s disease compared with an external comparison group of people who have not received the treatment. However, the studies involve relatively small numbers of people and the latest four-year results give a more mixed picture, so they need to be interpreted carefully.
At three years, updated data from all 15 people receiving the high dose showed an 80% slowing of disease progression using cUHDRS, a measure which combines movement, thinking and people's ability to carry out everyday activities. A separate measure called Total Functional Capacity (TFC), which looks at people's ability to function independently, showed a 67% slowing in decline.
What do the four-year results show?
At four years, data were available from 12 people receiving the high dose. Compared with an updated external control group, the results showed a 44% slowing of disease progression on cUHDRS and a 61% slowing in decline on TFC.
Importantly, the 44% result on cUHDRS did not reach statistical significance. This means we need to be cautious about how much can be concluded from this particular result. However, the result for TFC continued to show a meaningful difference compared with the external control group.
There are also limitations with the external comparison group at this later stage of the study. uniQure has said that missing data and what is known as “survivor bias” may have affected the four-year comparison. In simple terms, people whose disease was progressing more quickly were more likely to have dropped out of the external comparison group, potentially leaving a healthier group against which the people who received AMT-130 were being compared.
uniQure therefore also carried out an additional analysis using its previous external control group. This showed a 54% slowing in disease progression on cUHDRS and 68% on TFC at four years. However, because this was a post-hoc analysis, rather than the main planned comparison, these findings also need to be interpreted cautiously. The figures were included in the information shared by uniQure today.
What is happening in the UK?
There has also been an important development regarding possible approval of AMT-130 in the UK.
uniQure has held a pre-submission meeting with the Medicines and Healthcare products Regulatory Agency (MHRA), the organisation responsible for regulating medicines in the UK. The meeting included discussions about the clinical evidence and manufacturing information needed for a formal application. uniQure announced in April that it planned to submit a Marketing Authorisation Application (MAA) for AMT-130 in the UK during the third quarter of 2026.
This does not mean that AMT-130 has been approved in the UK. It means uniQure is taking the steps needed to ask the MHRA to consider the treatment for approval. The MHRA would then need to assess the evidence before deciding whether the treatment can be authorised for use in the UK.
What does this mean for people affected by Huntington’s disease?
These remain encouraging developments, particularly because there is currently no approved treatment that can slow the progression of Huntington’s disease. The results suggest that there may continue to be a treatment effect several years after the one-off gene therapy.
However, there are still important uncertainties. AMT-130 remains an investigational treatment, relatively small numbers of people have received it in these studies, and the difficulties with the external control group make some of the longer-term results harder to interpret.
The four-year results therefore shouldn't be viewed in isolation. While the main cUHDRS analysis at four years did not reach statistical significance, the TFC findings and additional analyses provide important context alongside the stronger three-year results.
So, while there continues to be reason for cautious optimism, we still don't know whether AMT-130 will ultimately be approved in the UK or, if it is approved, what future access to the treatment would look like.