uniQure reports longer-term Huntington’s disease gene therapy data

• uniQure reported 36- and 48-month follow-up results for ifezuntirgene inilparvovec, its investigational gene therapy for Huntington’s disease.
• High-dose patients showed slower disease progression compared with matched external controls on several measures, although the 48-month cUHDRS result was not statistically significant.
• Five high-dose patients experienced treatment-related serious adverse events involving central nervous system inflammation, all of which resolved.

uniQure (NASDAQ:QURE) reported additional Phase I/II data for ifezuntirgene inilparvovec, its investigational gene therapy for Huntington’s disease, with follow-up results extending to 36 and 48 months.

The company reported that at 36 months, 15 high-dose patients showed an 80% slowing in progression on the composite Unified Huntington’s Disease Rating Scale (cUHDRS) compared with updated matched external controls. The result had a nominal p-value of 0.005.

At the same 36-month time point, high-dose patients showed a 67% slowing on Total Functional Capacity (TFC), with a nominal p-value of 0.011, compared with matched external controls.

At 48 months, 12 high-dose patients showed a 44% slowing on the prespecified primary cUHDRS endpoint compared with matched controls.

The result had a p-value of 0.144 and was not statistically significant.

The same group showed a 61% slowing on TFC, with a nominal p-value of 0.008.

uniQure noted that matched external controls had 53% missing data at 48 months and said the missing data may have reduced the estimated treatment effect.

The company said the newly reported results were not included in its submitted license application, which relied on 36-month data from 12 high-dose patients.

Safety findings showed that five high-dose participants experienced treatment-related serious adverse events involving central nervous system inflammation. uniQure said all cases resolved.