Mizuho Comments On UniQure's Upcoming Low-Dose Huntington's Gene Therapy Trial Data

The much-awaited one-year data for the entire low-dose cohort for UniQure NV's QURE AMT-130 gene therapy in Huntington's disease is expected at any time.
The initial results from the first four patients released in December 2021 disappointed expectations as the company unexpectedly did not have MRI or mutant HTT protein data.
Related: UniQure Shares Fall After Clinical Update From Huntington's Disease Gene Therapy Trial.
Mizuho says the risk/reward looks favorable, though it remains cautious.
The analysts expect acceptable safety data. The biomarker data will probably be noisy and variable, citing early pre-symptomatic stage patients, a small sample size, and a low dose cohort.
In Cohort 1, uniQure targets a ~50% knockdown in the striatum using a lower dose of 6e12 gc/subject (vs. 75% striatum KD in Cohort 2 with a higher dose of 6e13 gc/subject).
According to Mizuho, the investors expect modest to no change in mHTT based on the mini-pig model, which at low dose did not show any knockdown of mutant HTT in the CSF.
At the high dose, lowering of mHTT was observed in the CSF to be 30% at 12 months post-dosing.
The company does not expect to present volumetric MRI or functional data until all patients (low and high cohorts) have 12 months of follow-up, expected in 1H 2023.
Mizuho keeps a Buy rating with a price target of $52.
Price Action: QURE shares are down 8.43% at $13.18 during the market session on the last check Monday.

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