Spark Therapeutics Trial Success Seen As Positive For Gene Therapy Sector

NEW YORK (TheStreet) -- The shares of Spark Therapeutics  (ONCE) are surging after the company announced that its lead gene therapy product candidate, SPK-RPE65, had met its primary endpoint in a Phase 3 trial. In a note to investors at the end of last month, Cowen predicted that the stock could reach $70 per share if the trial was successful and Piper Jaffray told investors this morning that the trial is not only positive for Spark but for the gene therapy space as a whole.

NEWS: Spark Therapeutics announced this morning that SPK-RPE65, which is supposed to treat gene-induced blindness, had met its primary endpoint in a Phase 3 trial. Specifically, patients taking the product experienced a significant improvement in their vision, compared with those who did not receive the treatment, the company stated. After Spark announced the topline results for the trial, Roth Capital analyst Elemer Piros noted that this is the first successful controlled gene therapy trial in the field. The firm increased its price target on Spark shares to $90 from $71 and reiterated its Buy rating on the stock.

GOOD FOR SECTOR: Piper Jaffray analyst Joshua Schimmer said he views the news that Spark Therapeutics' "highly anticipated" SPK-RPE65 Phase 3 gene therapy study for congenital blindness met its primary endpoint as a positive for the ophthalmology gene therapy field as well as the broader gene therapy sector. Schimmer added that Applied Genetic Technologies' (AGTC) early clinical data in the second half of this year will be a further test of the ophthalmology gene therapy platform. The analyst noted that other companies with gene therapy programs include Avalanche  (AAVL) , Biogen  (BIIB) , bluebird bio  (BLUE) , BioMarin  (BMRN) , Cellectis  (CLLS) , Medgenics  (MDGN) , uniQure  (QURE) , Pfizer  (PFE) , Regeneron  (REGN) and Sangamo  (SGMO) .

EARLIER CALL: In a note to investors on September 24, research firm Cowen said it was optimistic about the trial's outcome and predicted that the stock could reach $70 per share if the results of the trial were positive. In his prior note, Cowen analyst Phil Nadeau had estimated that the trial had roughly a 70% chance of being successful. The Fly published a summary of Cowen's note on the day it was circulated.

DATA COMING FROM PEER: Applied Genetic Technologies is expected to report early data on its own gene therapy for patients with X-linked retinoschisis by year end. Wedbush analyst David Nierengarten has said he also expects the company's gene therapy for achromatopsia patients to enter the clinic "shortly," with data expected in 2016.

BUY BLUEBIRD: In a separate ratings action in the same space this morning, Morgan Stanley analyst Matthew Harrison upgraded bluebird bio to Overweight with a $143 price target. Harrison said valuation now heavily reflects any potential competition in sickle cell disease and expects data at ASH in early December to be a key near-term catalyst. The analyst expects the data to show significant increases in hemoglobin similar to the first sickle cell disease patient.

PRICE ACTION: In morning trading, Spark jumped 26% to about $55 per share after rising to as high as $66 per share earlier in the session. Meanwhile, Applied Genetic Technologies gained 6% to $13.88 and bluebird bio advanced 3.4% to $97.30.

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