Story perspectives

UniQure Shares Plunge 40% After Trial Data Misses Significance

By Drooid · Sep 30, 2026, 2:28 PM UTC · How we work

Summary

uniQure Shares Plummet

uniQure’s Nasdaq shares fell about 40% after four-year data missed statistical significance.
At 48 months, high-dose patients slowed cUHDRS decline by 44%, a non-significant result.
Total Functional Capacity decline slowed 61% at 48 months, reaching significance (p=0.008).
Shares dropped to $20.50 in pre-market trading, the steepest single-session fall since November.
The company blamed the 48-month efficacy drop on survivor bias and missing external-control data.

Summary

AMT-130 Pricing Outlook

Analysts estimate AMT-130 could price at $1.7 million U.S. patient, aiming for $2.5 billion sales by 2030.
UniQure posted $9.4 million revenue and a $135 million net loss in the first half of 2026.
The therapy requires a roughly 10-hour neurosurgical procedure, raising operational costs and patient burden.
Five high-dose patients (17%) had serious CNS inflammation; all cases resolved, showing manageable safety.
One trial participant suicide was reported but deemed unrelated to AMT-130 treatment.

History

Historical Context

In December 2017 the FDA approved voretigene neparvovec (Luxturna), the first in-vivo gene therapy, after Spark Therapeutics and Children’s Hospital of Philadelphia presented Phase III data. The treatment, priced at $425,000 per eye, began U.S. sales in March 2018, establishing a precedent that now shapes investor and regulator expectations for later neuro-genetic therapies such as uniQure’s AMT-130.

1 of 3

uniQure Shares Plummet

uniQure’s Nasdaq shares fell about 40% after four-year data missed statistical significance.
At 48 months, high-dose patients slowed cUHDRS decline by 44%, a non-significant result.
Total Functional Capacity decline slowed 61% at 48 months, reaching significance (p=0.008).
Shares dropped to $20.50 in pre-market trading, the steepest single-session fall since November.
The company blamed the 48-month efficacy drop on survivor bias and missing external-control data.

1 / 3