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Analysts Optimistic on Uniqure NV (QURE) Following Latest Clinical Trial Data

By George MacDonald

On September 19th, Uniqure NV (NASDAQ:QURE), a leading player in gene therapy, announced topline line results of one-year follow up data in four Sanfilippo B syndrome patients, who were being treated with the company’s novel gene therapy.

The therapy consisted of uniQure’s proprietary AAV5 viral vector and a gene cassette including the N-acetylglucosaminidase, alpha (NAGLU) gene that was made with uniQure’s insect cell based-technology. The results of the Phase I/II clinical trial were announced at the European Society of Gene and Cell Therapy (ESGCT) and Finnish Society of Gene Therapy (FSGT) Collaborative Congress, held in Finland. Based on the results of the trial, two top analysts have revised their ratings for the company.

On September 21st, Roth Capital’s Debjit Chattopadhyay reiterated a Buy rating on the stock, and maintained a price target of $38.

Chattopadhyay’s optimism stemmed from the clinical trials data. He said, “This was the first demonstration of persistent restoration of NaGlu protein expression over 12 months achieved with gene therapy delivered directly into the CNS, and potentially opens up at least 11 other neuropathic LSD’s for which there are no treatments.” He added, however, that the current price target does not factor in the Sanfilippo program.

On September 20th, analyst Christopher Marai from Oppenheimer maintained an Outperform rating on the stock, and increased his price target to $50 (from the earlier target of $47). Marai said, “All patients demonstrated restoration of active NaGlu protein and maintenance of cognitive abilities. QURE plans to advance the program into a pivotal study.”

The company had announced a conference call on Monday, September 21. Prior to the call, Marai had stated some of his expectations from the call, and said, “On the call, we will look for (1) incremental safety information for AAV5-vector, including time-to-therapy clearance, (2) pivotal trial design information, (3) and efficacy details including brain MRI, neurological/biological markers.”

Marai and Chattopadhyay are not alone in their bullishness on uniQure; out of the five analysts polled by TipRanks in the last three months, all five analysts are bullish on the company. The average 12-month price target is $40.75, marking a 48% average return per rating.


Debjit Chattopadhyay is a top-ranked analyst. As of this writing, Chattopadhyay is ranked 13th out of 3,789 analysts ranked by TipRanks. Out of 171 ratings, Chattopadhyay has 63% success rate recommending stocks with a +24.6% average return per rating when measured over a one-year horizon and no benchmark.

Christopher Marai is also a top-ranked analyst as well, having earned a 57% success rate recommending stocks with a +27.9% average return per rating when measured over a one-year time horizon and no benchmark.