Quantumup
Sep 30, 12:22 PM
H.C. Wainwright reitd
$QURE Buy-
$80/said—AMT-130's four-year cUHDRS result complicates the durability case, although the sustained TFC benefit and the three-year data underlying the submitted BLA remain encouraging.
$TEVA $NBIX PTCT RHHBY WVE ANNX ALNY - REGN
H.C. Wainwright added—In 12 high-dose patients, the prespecified four-year CUHDRS analysis showed 44% slowing versus an updated external control but missed nominal statistical significance (p = 0.144).
However, Total Functional Capacity (TFC) showed 61% slowing (nominal p = 0.008 ) which management and a treating clinician emphasized reflects patients' ability to work, manage finances, and live independently, making its sustained four-year benefit an important part of their interpretation of the data alongside the cUHDRS result.
The four-year results were not part of the submitted BLA.
We reiterate our Buy rating, with the BLA already submitted on the earlier three-year data and FDA acceptance for review the next regulatory milestone.
