uniQure N.V. (QURE) is having a rough stretch. The stock has plunged about 31% over the past month, and Tuesday's trading session offered a microcosm of that volatility. Shares climbed as high as $22 before pulling back during regular hours, though no specific news catalyst explained the intraday swing.
The real story here goes back to November, when the FDA delivered some unwelcome feedback about uniQure's investigational gene therapy for Huntington's disease. Here's the issue: the FDA doesn't agree that data from the Phase 1/2 studies of AMT-130, compared against an external control group, provides adequate primary evidence to support a biologics license application (BLA). This matters because uniQure had shared its prespecified protocols and statistical analysis plans with the agency ahead of time.
Last week, the FDA doubled down on that position in its final meeting minutes, consistent with what uniQure disclosed back in November. The agency conveyed that the Phase 1/2 data are "currently unlikely" to serve as the primary evidence needed for BLA submission. That's regulatory speak for "we need more convincing."
The frustrating part? Back in September, uniQure announced topline data from its pivotal Phase 1/2 study that looked genuinely promising. The trial met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 36 months. The comparison was against a propensity score-matched external control, which is where the FDA apparently sees problems.
At publication Tuesday, QURE stock was down 0.14% at $20.40, trading at what many would consider depressed levels given the uncertainty hanging over its regulatory pathway.
