UniQure shares plunged after new four-year results for AMT-130, its experimental one-time gene therapy for Huntington’s disease, showed a smaller estimated benefit than earlier analyses.
In 12 high-dose patients, disease progression on the cUHDRS measure was estimated to be 44% slower than in an external control group at 48 months. That difference was not statistically significant (p=0.144).
On a functional measure, Total Functional Capacity, the company reported 61% slower decline at 48 months (nominal p=0.008). Updated 36-month results in 15 high-dose patients showed 80% slower cUHDRS decline and 67% slower TFC decline.
UniQure says the four-year comparison may understate the treatment’s effect:
more than half of the external-control data were missing, and patients who dropped out appeared to decline faster than those who remained. A post-hoc analysis using an earlier control dataset produced a statistically significant 54% slowing on cUHDRS, but that was not the prespecified comparison.
The results raise questions about how durable and reliable the apparent benefit is. They add uncertainty as the FDA reviews UniQure’s US application; the application was based on earlier data, and a confirmatory study is expected.
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