The talk of a bubble in biotech seems to have subsided (temporarily, no doubt), and the sector-tracking iShares Nasdaq Biotech ETF (IBB) has continued higher after bouncing from its 50-day simple moving average in the last few weeks.
The week started off with a bang for PropThink Premium members, who knew our stance on uniQure (QURE) this year. The European gene therapy developer announced a collaboration with Bristol-Myers Squibb (BMY) on Monday, receiving $50 million up front, $15 million in near-term payments, and a >$30 million equity investment from BMS for the development of gene therapies directed at cardiovascular indications.This partnership was the missing piece for QURE, in our opinion, and will be viewed by the investment community as validation of the platform. The market had largely ignored QURE in favor of names like Spark (ONCE) and bluebird bio (BLUE) prior. Read UniQure Fills in the Missing Piece with Bristol-Myers Collaboration.
QURE ran to $34 on the news, the price at which BMS is buying stock in the smaller company, and uniQure pulled the trigger on a secondary financing late in the week. But in the middle of pricing the deal, uniQure revealed that European reviewers have recently taken a dim view of their approved gene therapy, Glybera’s, risk-benefit profile. As part of the EMA’s conditional approval of Glybera in October 2012, uniQure was required to complete a post-approval study and implement a disease registry for long-term surveillance of LPLD patients who received the product. Recently, the company submitted Type II variations to the EMA, which are intended to update with six-year follow-up data from the handful of patients treated years ago (complete last year). This package is designed to further characterize the product’s long-term effects.In light of the updated information, the EMA is currently re-analyzing the risk-benefit of Glybera, and on April 8 uniQure received an assessment report prepared by a rapporteur for the Committee for Advanced Therapy Medicinal Products (CAT), which is the committee that advises the EMA’s CHMP on gene therapies.
Based on the final study report, including the six-year follow-up data, the rapporteur considers that Glybera lacks efficacy, and that the benefit-risk balance of Glybera is negative. The rapporteur did, however, recommend that the application (Type II) be subject to a request for supplemental information before a final recommendation is made.Mr. King explained on Friday what to make of the update. You can read it here. Despite this latest, Mr. Fink’s call on QURE has worked nicely from $20 earlier this year.Dr. Paul Nunzio De Santis‘ suggestion in November (read it here, free) was to be long GlobeImmune (GBIM) from below $6.00 in anticipation of data this quarter for GS-4774, the company’s drug candidate for the treatment of Hepatitis B partnered with Gilead Sciences (GILD).GBIM ripped higher early this week in advance of new abstract titles from EASL at the end of this month being made available online on Wednesday, April 8. We wrote to PropThink Premium members in advance explaining why the stock was likely to fade on Wednesday, which was spot on. GBIM dropped 15% into the end of the week, and readers who followed our suggestion took profit at +75% on a 5-month trade. Kudos to Dr. De Santis for the idea last year.
Almost three years after an initial collaboration was announced, AstraZeneca (AZN) selected this week the first clinical candidate from a discovery program with Regulus Therapeutics (RGLS), which sent RGLS higher into the end of the week. Regulus announced on Wednesday evening that AstraZeneca has selected as a clinical candidate RG-125 (now called AZD4076), a GalNAc-conjugated anti-miR targeting microRNA-103/107 (miR-103/107) for the treatment of Non Alcoholic Steatohepatitis (NASH) in patients with type 2 diabetes/pre-diabetes. Under the terms of the deal, AstraZeneca will pay RGLS $2.5 million and will assume development of the program once an Investigational New Drug application has been accepted by the FDA.
One or more of PropThink’s contributors are long QURE, BMY, GBIM, AZN, or RGLS.
