UniQure yo-yos as data raise questions about its place in hem B gene therapy race

Shares in uniQure ($QURE) briefly hit a new low following a negative knee-jerk reaction to fresh data on its hemophilia B gene therapy. The sharp slide, which was quickly reversed, was triggered by data showing that, for better or worse, the 9-month data on hemophilia B gene therapy AMT-060 were comparable to results generated after 6 months.

On one level, this represents a success for uniQure. With the data, the Dutch biotech has shown its gene therapy can deliver improvements in Factor IX (FIX) gene activity for at least 9 months after treatment. Four of the 5 patients in the study no longer need to receive prophylactic FIX infusions. FIX use is down 75%. And the latest FIX readings for the subjects are comparable to their steady-state levels. With the durability of gene therapies being a key factor, such sustained improvements are a boost for uniQure, particularly given that the 5 patients received a low dose of AMT-060.

The doubts about uniQure center on the response, or lack thereof, of one patient and comparisons to its rivals. After 9 months, patient 3 continues to need prophylactic FIX infusions and has FIX activity levels of below 2%. The FIX actiivity levels of the other participants range from 2.9% to 7.2%. While sufficient to end the need for prohylactic FIX infusions, data on the four stronger responders have still been used as a stick with which to beat uniQure. Rival gene therapy player Spark Therapeutics ($ONCE) has achieved 27% to 35% FIX activity in a four-person study.

Unfavorable comparisons to Spark’s data caused uniQure’s stock to slide 16% following the release of its 6-month results in June. A perception that uniQure faces stiff competition has contributed to the company’s market cap falling to $176.4 million (€159.1 million). As of the end of March, uniQure had €184.6 million in cash. While life on Nasdaq has been rough for uniQure over the past 9 months, it represents part of what the company signed up for when it chose to list in the U.S. The presence of other gene therapy biotechs--and the comparisons this enables--was a motivation for the U.S. IPO.

The future for uniQure rests, in part, on data from the high-dose cohort. While, barring a turnaround for patient 3, the 9-month low-dose data were always likely to receive a muted response at best, the high-dose readout gives uniQure a chance to show AMT-060 can increase FIX activity levels up toward 10% and beyond. While the full real-world implications of upping FIX activity to these levels are unclear, it has the potential to reduce the number of spontaneous bleeds and, in doing so, cut the need for on-demand FIX use.

When paired with the fact that uniQure has so far treated older patients than Spark, it uses a different vector--so may be able to carve out its own niche in the market--and it has manufacturing capacity in place, the release of upbeat data could quickly shift perceptions. The production capacity alone could prove to be critical, particularly now that other companies, notably BioMarin Pharmaceutical ($BMRN), are pursuing baculovirus-based gene therapy manufacturing.

For now, though, uniQure remains in the gene therapy doghouse.

- read the release
- view the presentation (PDF)

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UniQure slides toward historic lows after its hemB gene therapy goes up against Spark