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UniQure Reports Mixed Results of AMT-060 in Patients with severe Hemophilia B

On Saturday, UniQure provided an update for their ongoing low dose AMT-060 and 26 week results from their high dose AMT-060 Ph1/2 clinical trial. This was the Q4 catalyst event we have been anticipating.

The updated low dose cohort data included data through 12 months of treatment with AMT-060. These data demonstrated that AMT-060 provides stable FIX expression up to 12 months where patients experienced significantly fewer bleeding episodes, achieving 85% less total FIX usage. This low dose cohort maintains an average steady state FIX activity of 5.2% of normal.

The 26 week high dose cohort data did not hit the magic >12% FIX activity, which was the upside result we were hoping for. Instead, with a mean steady state FIX activity of 6.9%, the market still believes UniQure inferior to their major competitor, Spark Therapeutics (ONCE). However, since ONCE still has issues with immune reactions, and QURE will likely be first to market, QURE price has held steady, which is consistent with the low downside risk we anticipated. Furthermore, it is estimated that 20-40% of patients may not be eligible for Spark’s therapy, potentially providing a commercial niche for UniQure. Finally, at a market cap of about $150 million, less than 1/10th the market cap of Spark Therapeutics, QURE still has an attractive valuation.

Find the UniQure press release here:

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