QURE Stock Crashes 32% in a Week: Here's What You Should Know

Shares of uniQure N.V. QURE plunged 31.8% in a week, primarily after the company announced a disappointing regulatory update for its investigational gene therapy, AMT-130, for the treatment of Huntington’s disease, earlier in March.

uniQure recently received the final meeting minutes from the FDA following a Type A meeting held on Jan. 30, 2026, to discuss the development path for AMT-130 for treating Huntington’s disease. According to the agency, data generated from the ongoing phase I/II studies, when compared with an external control group, are not sufficient to serve as the primary evidence of effectiveness needed to support a potential marketing application for the candidate.

The FDA “strongly recommended” that QURE conduct a prospective, randomized, double-blind study with a sham surgery control to adequately evaluate the therapy’s efficacy. uniQure said that it plans to continue discussions with the regulator regarding phase III development and intends to request a Type B meeting in the second quarter of 2026 to explore potential study design options and determine a clearer regulatory pathway for AMT-130.

Management noted that although alignment on a regulatory submission strategy based on current early-stage data was not reached, the company believes the durability and overall body of evidence generated so far justify continued engagement with the FDA. uniQure plans to continue working with regulators to establish a scientifically grounded and efficient development path for AMT-130.

In the past six months, QURE shares have lost 38.9% against the industry’s 19.6% growth.

We remind the investors that in September 2025, uniQure reported meeting key goals in the phase I/II study of AMT-130 for the treatment of Huntington’s disease. Per the data readout, the high dose of AMT-130 achieved a statistically significant 75% slowing of the progression of Huntington's disease after three years. Patients also showed decreased levels of cerebrospinal neurofilament light protein after three years, an important biomarker linked to neurodegeneration and disease severity in Huntington’s disease.

Following the encouraging data readout, uniQure held a pre-Biologics License Application (BLA) meeting with the FDA regarding AMT-130 for Huntington’s disease in October 2025. Based on the discussions at the meeting, the regulator indicated that it no longer believes data from the phase I/II studies of AMT-130 would be sufficient to serve as the primary evidence supporting a potential BLA submission. Following this feedback, uniQure requested a Type A meeting with the FDA, which was held on Jan. 30, 2026.

Huntington's disease is a genetic disorder that causes the progressive breakdown of nerve cells in the brain, which leads to a decline in cognitive and physical abilities, often resulting in movement, thinking and psychiatric problems. uniQure enjoys the FDA’s Regenerative Medicine Advanced Therapy and Breakthrough Therapy designations for AMT-130 to treat Huntington’s disease.

Other Developmental Programs in QURE’s Clinical Pipeline

Apart from AMT-130, uniQure’s wholly-owned clinical pipeline comprises several other candidates that are currently undergoing early to mid-stage development for the treatment of patients with refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis and Fabry disease. 

Please note that the company also markets an internally developed gene therapy, in partnership with CSL Behring, for the treatment of hemophilia B in the United States and the EU under the brand name Hemgenix. The approvals in the United States and EU markets in 2022 and 2023, respectively, marked a significant milestone in the field of genomic medicine, bringing a new treatment approach for patients living with hemophilia.

uniQure N.V. Price and Consensus

uniQure N.V. price-consensus-chart | uniQure N.V. Quote

QURE’s Zacks Rank and Stocks to Consider

uniQure currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are USANA Health Sciences USNA, Catalyst Pharmaceuticals CPRX and ALX Oncology Holdings ALXO. While USNA and CPRX sport a Zacks Rank #1 (Strong Buy) each, ALXO carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for USANA Health Sciences’ 2026 earnings per share (EPS) have risen from $1.90 to $2.00. USNA shares have lost 41.2% over the past six months.

USANA Health Sciences’ earnings beat estimates in three of the trailing four quarters and matched once, with the average surprise being 21.92%.

Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 EPS have risen from $2.54 to $2.82. CPRX shares have soared 21.1% over the past six months.

Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.

Over the past 60 days, estimates for ALX Oncology Holdings’ 2026 loss per share have narrowed from $1.21 to 88 cents. ALXO shares have rallied 91.9% over the past six months.

ALX Oncology Holdings’ earnings missed estimates in each of the trailing four quarters, with the average negative surprise being 12.82%.

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This article originally published on Zacks Investment Research (zacks.com).

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