Update on uniQure AMT-130 – Where Do We Stand?

The progress published by uniQure regarding the treatment of Huntington’s disease (HD) is a major and motivating milestone! This news brings great hope to our HD families.

In 2015, we organized the first clinical advisory meeting for AMT-130 at the GHI, and since then I have been the global lead study physician for the two AMT-130 studies in the USA and Europe. You can trust me when I say I am very excited about the data available!

The two studies have definitely achieved their main purpose: to investigate the safety and tolerability of the AMT-130 gene therapy.

And yes, the efficacy signals are also encouraging. However, we must remember that only 12 patients are included in this study, and there is no well-controlled comparison group (sham operation/placebo). This is normal for an early Phase I/IIa safety trial. Proof of efficacy typically requires a larger, confirmatory Phase IIb/III study.

The comparison made here with historical data from observational studies such as ENROLL-HD carries some caveats. In such studies, investigators expect HD progression and tend to rate patients as worsening over time more than in a clinical trial where treatment effects are anticipated. Such issues can only be controlled by a properly blinded placebo group in the study, or by using objective measures that do not show placebo effects, such as imaging or Q-Motor assessments.

Therefore, although uniQure reports a very encouraging step in the right direction, we should keep in mind that the efficacy of AMT-130 has not yet been conclusively proven. Additionally, not all data have been evaluated yet. Whether approval can be granted with the data currently available remains to be seen. It is reasonable to have hope, but we should be realistic and not overestimate the results.

Dr. Ralf Reilmann, Director of the GHI, Global Principal Investigator of the uniQure AMT-130 studies