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Biotech Beat: Avalanche Biotechnologies Inc (AAVL), bluebird bio Inc (BLUE), Synergy Pharmaceuticals Inc (SGYP) News

Disparate results from two gene therapy developers are worth a mention in this week’s biotech recap.

Avalanche Biotechnologies Inc (NASDAQ:AAVL) blew up spectacularly this Monday with the release of top-line results for AV-101, a gene therapy directed at wet AMD. The study met its primary endpoint – safety – but failed to impress investors on just about every other outcome. Ideally, the gene therapy would have meaningfully reduced the number of Lucentis injections (standard of care in the setting) required to maintain vision over the course of a year, when compared to a placebo, and/or improve vision relative to the comparator.

Not so. Patients receiving AV-101 on top of standard of care needed, at the median, just 2 fewer injections than patients receiving normal care. In a setting where some patients receive Lucentis every month, it’s not enough to justify adding an expensive gene therapy. And confounding the findings further, patients receiving AV-101 had their retinas thicken during the course of the study, generally a sign of worsening condition.

The results were a mess, and even management couldn’t seem to come up with anything novel to say about the data. In a conference call after the close Monday, executives essentially stuck to the company line, answering analyst questions with boilerplate “the study was positive” commentary. That didn’t help the dribbling stock, and AAVL closed the week down 62%, at $15.56.

In contrast, bluebird bio Inc (NASDAQ:BLUE) presented impressive results  – yet again – from the handful of patients receiving its LentiGlobin gene therapy. Mr. Fink wrote all about the results on Monday, outlining key facets related to the first patient with Sickle Cell Disease treated: engraftment kinetics and the slow wash out of transfused Hemoglobin. Read it here.

Synergy Pharmaceuticals Inc (NASDAQ:SGYP) phase 3 results for plecanatide, its constipation drug, impressed even skeptics this week, and the stock ripped higher by nearly 100% into Friday.

Mr. King has been writing about Synergy since the stock was in the $5.00 range over a year ago, reiterating the buy thesis for PropThink’s Premium members at $3.50 this spring. With the binary event having finally played out (for a double, nonetheless), the focus now shifts to a second phase 3 trial, due as soon as next month, and commercial plans for the CIC drug. Ironwood Pharma’s (IRWD) Linzess is already on the market and has been an expensive product to launch (>$200 million annually). That’s a tall order for a smaller company like Synergy, so the question remains whether Synergy can find an acquirer or a quality partner for the commercial launch in 2016/17.

The biotech sector finally took out some overhead resistance on Thursday, opening up what most traders expect is the next leg higher in the sector’s bull run. You can read about the technicals at

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