It was just 5 days ago that Bristol-Myers Squibb (BMY) partnered with uniQure (QURE) on the discovery and development of novel gene therapies for cardiovascular indications. QURE ripped to $34, the price at which BMY invested in QURE, but tumbled towards the end of this week with the disclosure that regulators in Europe may be changing their tune on Glybera, uniQure’s Europe-approved gene therapy for the treatment of LPLD. UniQure made the disclosure in the middle of conducting a secondary financing late this week, which priced at $29.50 on Thursday.
Disclosure Increases Headline Risk in 2015
First, some background.
As part of the conditional approval of Glybera three years ago (October 2012), uniQure was required to complete a post-approval trial 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 the summary product of characteristics of Glybera to include six-year follow-up data from the handful of patients treated years ago. This is essentially a data package to further characterize the product’s long-term effect on pancreatitis in LPLD individuals.
As part of this procedure, uniQure disclosed (voluntarily) the accidental destruction of some historical source data at a trial site in Canada. The EMA subsequently requested a good clinical practices inspection of the entire Glybera trial program.
This inspection was conducted in early 2015 and focused on the quality control mechanisms that were in place at uniQure during data acquisition and processing in 2009 and 2010, as well as the integrity of the data as a whole. Since then, the company says, they have implemented corrective quality control actions.
UniQure believes that these events “do not materially affect the previously reported results of our historical trials. The inspection team also concluded “that the quality of the data and the level of GCP compliance both are acceptable” and that the trial data can be used for the submitted Type II variations.”
The EMA is currently reviewing the benefit-risk analysis of Glybera in light of the additional six-year follow-up data (this analysis was complete last year) and the findings of the GCP inspection.
On April 8 uniQure received a copy of 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 (the CAT was created just before Glybera was initially submitted for approval in the EU). Based on the final study report, including the six-year follow-up data, the assessment report states that 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. Importantly, the initial report represents only the views of the rapporteur and does not bind the CAT or the CHMP.
In essence, this week’s news introduces new risk that Glybera is pulled from the market due to a “negative risk-benefit balance”.
There are numerous moving parts to this, but here is how we view the news. 
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One or more of PropThink’s contributors are long QURE or BMY.
