uniQure N.V. QURE surged 109.7% after it announced reaching alignment with the FDA on key elements of an accelerated approval pathway for AMT-130, a one-time administered investigational gene therapy, which is being developed to treat Huntington’s disease.

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 (RMAT) designation for AMT-130 to treat Huntington’s disease.

The alignment with the FDA was reached at an RMAT meeting with the regulatory body in November 2024. The FDA agreed that the reported data from the ongoing early to mid-stage studies of AMT-130 for Huntington’s disease compared with a natural history external control are sufficient to serve as the primary basis for a regulatory filing under the expedited pathway, avoiding the need for an additional pre-submission study.

Furthermore, the regulatory body has also agreed that cUHDRS may be used as an intermediate clinical endpoint and that reductions in the neurofilament light (NfL) chain measured in cerebrospinal fluid (CSF) may serve as supportive evidence of therapeutic benefit in the application for accelerated approval. Please note that the cUHDRS has been demonstrated to be the most sensitive measurement of clinical progression in Huntington’s disease patients. On the other hand, CSF NfL is an important biomarker of neurodegeneration and is strongly associated with the clinical severity of Huntington’s disease.

This is a significant milestone achievement for uniQure as it positions the company on the fastest and most efficient pathway to bring a potentially revolutionary therapy to people with Huntington’s disease. QURE is currently gearing up to submit a biologics license application to the FDA for AMT-130 to treat Huntington’s disease and plans to engage in further dialogues with the regulatory body in the first half of 2025 to discuss the statistical analysis plan and the technical requirements. In the past three months, shares of uniQure have skyrocketed 176.2% against the industry’s 6.2% decline.

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QURE’s AMT-130 Study Interim Data Shows Superior Efficacy

In July 2024, uniQure reported updated interim data, including up to 24 months of follow-up data, from 29 treated patients enrolled in the phase I/II studies of AMT-130 for Huntington’s disease in the United States and EU. Results demonstrated a potential long-term, durable clinical benefit and reduction of a key marker of neurodegeneration upon treatment with the candidate.

uniQure is simultaneously conducting two phase I/II studies of AMT-130, with 26 participants in the United States and 13 in the EU/UK to treat Huntington's disease. Among these, a total of 29 patients received either a low dose (12 patients) or a high dose (17 patients) of AMT-130, while 10 control patients underwent imitation surgery. As of March 31, 2024, follow-up data at 24 months were available for 21 patients, including 12 from the low-dose group and nine from the high-dose group. uniQure, for the first time, performed a statistical analysis of clinical outcomes at 24 months for the 21 treated patients.

Per the updated data, AMT-130 continues to be generally well-tolerated with a safety profile that is manageable at both doses. No new drug-related serious adverse events were reported.

Developmental Programs in QURE’s Clinical Pipeline

uniQure expects to complete enrolling patients in the third cohort of the U.S. phase I/II study exploring AMT-130 in combination with immunosuppression soon. Safety data from this cohort is anticipated in the first half of 2025.

Additionally, uniQure also shared its plan to present another interim analysis from the ongoing phase I/II studies of AMT-130 to treat Huntington’s disease in mid-2025. The data from this interim analysis will include a 36-month comparison of treated patients to the propensity score-weighted external control.

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

Please note that the company also markets an internally developed gene therapy for the treatment of hemophilia B in the United States and EU under the brand name Hemgenix. The approvals in the U.S. and EU markets in 2022 and 2023, respectively, marked a significant milestone in the field of genomic medicine as it brought 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 Other Stocks to Consider

uniQure currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks from the drug/biotech industryare Alnylam Pharmaceuticals ALNY, Pfizer PFE and Gilead Sciences GILD, each carrying a Zacks Rank #2 at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Over the past 60 days, Alnylam Pharmaceuticals’ loss estimates have narrowed from 42 cents to 39 cents per share for 2024, while that for 2025 has narrowed from 47 cents to 13 cents. ALNY shares have lost 3% in the past three months.

Alnylam Pharmaceuticals’ earnings beat estimates in three of the trailing four quarters and matched once, delivering an average surprise of 65.67%.

In the past 60 days, Pfizer’s earnings estimates have risen from $2.66 to $2.92 per share for 2024, while that for 2025 has increased from $2.86 to $2.94. PFE shares have lost 12.4% in the past three months.

Pfizer’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 74.50%.

In the past 60 days, Gilead Sciences’ earnings estimates have risen from $3.78 to $4.29 per share for 2024, while that for 2025 has increased from $7.24 to $7.38. GILD shares have risen 10.9% in the past three months.

Gilead Sciences’ earnings beat estimates in three of the trailing four quarters and missed the same in one, delivering an average surprise of 15.46%.

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