- The much-awaited one-year data for the entire low-dose cohort for UniQure NV's (NASDAQ: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.
