Uniqure Notches Bullish Initiation On Gene Therapy Prospects

Uniqure NV (NASDAQ: QURE) has managed to win the seal of approval from Wells Fargo Securities due to its gene therapy pipeline candidates that target multiple diseases. 

The Analyst

Analyst Jim Birchenough initiated coverage of Uniqure with an Outperform rating and $47 price target.

The Thesis

Uniqure's dominant gene therapy platform with an AAVS vector; its potential "best-in-class" lead gene therapy candidate AMT-061 for treating hemophilia B; and pipeline optionality for treating Huntington's and genetically driven heart disease create a positive outlook, Birchenough said in the initiation note. 

AMT-061 is in a Phase 3 trial for hemophilia B. The analyst expects a positive update on the candidate by the end of 2018 following dose confirmation and a Phase 3 enrollment update.

AMT-130, the company's second gene therapy candidate, is being tested for Huntington's disease, with preclinical data supporting extensive Huntington gene silencing in all regions of the brain, Birchenough said. Huntington's disease is a $20-billion-plus opportunity, and AMT-130 is complementary to other gene-silencing approaches, he said. 

Ionis Pharmaceuticals Inc (NASDAQ: IONS) and Roche Holdings AG Basel ADR Common Stock (OTC: RHHBY) combined investigational gene therapy candidate IONIS-HTTRx was recently found to be effective in an ongoing Phase 1/2 clinical trial.

Uniqure is collaborating with Bristol-Myers Squibb Co (NYSE: BMY) for cardiovascular disease indications through its AMT-126, which is in preclinical development. Wells Fargo expects data by the end of the year that could support subsequent clinical development.

The relatively low expectations for this indication provide a favorable risk-reward, Birchenough said. 

The Price Action

Uniqure shares have added about 61 percent year-to-date.

Related Links:

Benzinga's Biotech Pulse: Verastem Uprooted On Stock Offering, Titan Pharma Earnings

Zoetis To Buy Abaxis In $2 Billion Deal