UniQure pivots to pipeline while Glybera sputters

To hear sellside analysts tell it, Glybera has little to do with the future of UniQure since the company has shifted its gene therapy programmes into other rare diseases like haemophilia and Sanfilippo syndrome.

That is not what investors believe: the announcement that the Netherlands-based, US-listed group will no longer pursue US approval of Glybera resulted in a 17% loss in share price to $18.78 yesterday. That drop came despite the fact that it has seen a commercial patient in Europe for the €1.1m ($1.2m) gene therapy, the first such agent to be launched in western markets.

The decision was a nod to reality, chief executive Jörn Aldag said in a conference call, given the small patient numbers in the ultra-rare familial lipoprotein lipase deficiency and the FDA’s demands for additional clinical work. The group will continue a phase IV clinical trial to meet obligations to the European Medicines Agency, as well as continuing to follow patients long term through a registry.

There is no other treatment for Sanfilippo syndrome, a triglyceride-raising genetic disorder that affects 1 in 250,000, but Mr Aldag said UniQure would focus on other diseases with high unmet medical needs.

Commercial success of sorts

The first commercial Glybera patient received treatment, at the Charité University Clinic in Berlin, at the end of October.

This came three years after European authorities approved the therapy, which requires 48 injections at 48 different sites for the average weight European. Partner Chiesi had delayed the launch until 2015 to incorporate six-year follow-up data on pancreatitis, a major complication of the condition, in pricing and reimbursement applications.

UniQure’s shift in focus has become increasingly clear over the past year, as the EU launch and US pivotal programme faced delays. The Sanfilippo B project AMT-110 has passed a phase I/II trial, and a haemophilia B candidate, AMT-060, is in phase II, not to mention UniQure's partnership with Bristol-Myers Squibb on cardiovascular disease (UniQure deal confirms red-hot interest in advanced therapies, April 7, 2015).

As such, abandoning Glybera in the US should not necessarily have been quite so damaging. Yet the spectre of regulatory stumbles haunts the group – after all, it was an earlier rejection of Glybera in Europe that sunk UniQure's predecessor company Amsterdam Molecular Technologies.

Shares bounced back 5% to $19.80 in early trading today, however, a sign that some investors saw a buying opportunity and have faith that the pipeline will come good.

Glybera remains a scientific achievement but a commercial disappointment. The good news for UniQure is that its discovery platform has the capacity to shift to other genetic disorders. As long as it has more projects in the pipeline it might be able to sustain the interest of some investors for a while – however, its ability to progress to a commercial-stage company remains unproven.

To contact the writer of this story email Jonathan Gardner in London at jonathang@epvantage or follow @ByJonGardner on Twitter

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