Chardan Capital Markets Starts uniQure BV (QURE) at Buy; Sees 2015 as 'Breakthrough' Year

Chardan Capital starts coverage on Uniqure NV (Nasdaq: QURE) at Buy with a price target of $40.

Analyst Gbola Amusa, MD, CFA, commented, We continue rolling out coverage on leading names in segments of therapeutics (e.g. gene therapy) that are likely to generate excess returns in the coming decade. uniQure is a gene therapy company that has already achieved, via Glybera (for lipoprotein lipase deficiency), the first gene therapy approval in Europe. The value of the company we think is much more dependent on AMT-060, which is in phase I/II trials for the treatment of hemophilia B and has important catalysts upcoming. We expect outperformance for QURE due to likely overall success on AMT-060 and other nearing catalysts, namely: 1) the possible Glybera European roll-out in 2H15, 2) phase I/II AMT-060 hemophilia B interim data in 3Q15, and 3) phase I/II Sanfilippo B data in 3Q15.

Defining phase I/II hemophilia B data for AMT-060 are due in 3Q15 and could build upon landmark St. Jude results. uniQure is testing AMT-060 (AAV5/factor IX) in a 10-patient trial, with interim data from the first 2-5 patients due in 3Q15, and final data due in 1Q16. Because hemophilia B is due to a single genetic defect and because even 5 percent expression of factor IX leads to a mild disease outcome, it is thought to be an ideal disease area for gene therapy. We put a 70 percent probability that the data in 3Q15/1Q16 will be positive, given: 1) St. Jude has already produced a landmark proof of concept study using a gene cassette uniQure has licensed, 2) uniQure is dosing at 3x and 10x the highest dose used by St. Jude to possibly address any issues on tropism, 3) the AAV5 vector and doses uniQure is using have been validated for safety in acute intermittent porphyria (AIP) trials, and 4) uniQure's AAV5 vector might have lower levels of neutralizing antibodies than the AAV8 vector tested in the St. Jude study. Our $292 million consolidated sales forecast for AMT-060 in 2025 is based on gene therapies converting 38 percent of the factor IX concentrate market, with uniQure taking 44 percent of the converted value. AMT-060 contributes $26.83, or 66 percent, of our $40 uniQure price target.

Amusa noted, We value uniQure using a discounted cash flow analysis, utilizing a 11.5 percent WACC and a 2.5 percent perpetuity growth rate.

For an analyst ratings summary and ratings history on uniQure BV click here. For more ratings news on uniQure BV click here.

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