QURE Stock Is On Track To Shed About Half Its Market Cap Despite Encouraging Huntington’s Disease Data – Here’s What Investors Need To Know

• Slowing of disease progression fell to 44% over a 48-month period.
• Five patients experienced serious treatment-related inflammation in the central nervous system, though all of them recovered fully
• uniQure submitted the BLA to the FDA based on the 36-month data.

Shares of uniQure (QURE) crashed more than 50% in pre-market trading on Tuesday after data for its experimental Huntington’s disease gene therapy raised questions about how well its treatment effect holds up over time.

The therapy is particularly important because there are currently no approved treatments that can slow the underlying progression of Huntington’s disease, a genetic brain disorder that progressively affects movement, thinking and behavior.

QURE shares are on track to clock their sharpest single-day losses and could also fall below their 200-day moving average (200-DMA) for the first time in over three months.

What Did The Data Show?

The results were largely positive at 36 months. Among 15 high-dose patients, uniQure’s Ifezuntirgene slowed disease progression by 80% on cUHDRS, a measure combining movement, thinking and daily function, compared with an external control group. The result was statistically significant.

The treatment also slowed decline by 67% on Total Functional Capacity (TFC), which measures patients’ ability to work and perform everyday activities.

Importantly, the 36-month data was the basis of uniQure’s Biologics License Application (BLA). The company filed for BLA earlier this month. The FDA previously agreed that 36-month data could form the primary basis for the application under the accelerated approval pathway.

So Why Did QURE Plunge?

The concern centers on the 48-month data. Among 12 high-dose patients, disease progression slowed on cUHDRS in 44% of cases, compared with 80% at 36 months, and the result was not statistically significant. TFC held up better, showing a statistically significant 61% slowing in decline.

The biotech firm said most side effects linked to the treatment procedure have been resolved. Five high-dose patients experienced serious treatment-related inflammation in the central nervous system, but all recovered fully.

Retail Calls Selloff An Overreaction

Despite the pre-market crash, retail sentiment surrounding QURE on Stocktwits turned ‘extremely bullish’ from ‘bullish’ a day earlier, amid ‘extremely high’ message volumes.

One user called the selloff an overreaction.

Another user said they would be “shocked” if the treatment doesn’t get approved.

The stock is down about 16% so far this year.

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