Leerink notes uniQure (QURE) reported topline results from the pivotal Phase 1/2 study of AMT-130 in Huntington’s disease, meeting their primary endpoint with statistical significance slowing of disease progression as measured by the composite United Huntington’s Disease Rating Scale at 3 years that did not meaningfully erode vs. 2 years plus several key secondaries, which the firm thinks limits the regulatory risk for this program going forward. Leerink thinks the data can support an approval with minimal need for regulatory flexibility from the FDA, which has been a concern for investors, and the firm would not be surprised to see the stock trade up 100% or more on today’s news. Leerink has an Outperform rating on the shares.
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