Shares of uniQure QURE soared 78.4% on June 17, after the company announced a major regulatory milestone for AMT-130, its investigational gene therapy for Huntington's disease. The stock, however, lost 3.9% on June 18.
During a recent type B meeting, the FDA informed uniQure that the three-year data from the phase I/II study on AMT-130 study could serve as the primary basis for a biologics license application (BLA) seeking accelerated approval for the therapy for the treatment of Huntington's disease.
QURE expects to receive final minutes within 30 days of the recent type B meeting.
The FDA seeks alignment on the confirmatory study design before BLA submission, including consideration of a concurrent standard-of-care control group in place of a sham-controlled design. The agency stated that it would work expeditiously with uniQure to finalize these plans.
The company expects to complete alignment with the FDA ahead of its anticipated BLA filing in the third quarter of 2026 and is committed to initiating the confirmatory study as quickly as possible.
The latest update relieves anxious investors as QURE had earlier suffered a regulatory setback. Shares of the company crashed in March 2026 when the company announced that it had received final meeting minutes from the FDA regarding a type A meeting held in January 2026 to discuss AMT-130.
This meeting was held following an October 2025 pre-BLA meeting.
After receiving the final meeting minutes, the company disclosed that the FDA did not agree that data from the phase I/II studies, when compared with an external control group, were sufficient to serve as the primary evidence of effectiveness needed to support a marketing application for AMT-130.
The FDA has recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study.
More on QURE’s AMT-130
AMT-130 is a novel investigational gene therapy for Huntington's disease that leverages uniQure's proprietary miQURE platform. The therapy employs a microRNA (miRNA) designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.
AMT-130 is being evaluated in two ongoing multi-center, dose-escalating phase I/II studies designed to assess its safety, tolerability, and exploratory efficacy signals for the treatment of Huntington's disease.
Following discussions with the FDA, it was agreed that data from cohorts 1 and 2 in the phase I/II studies could be compared to a propensity score-matched external control derived from the Enroll-HD natural history data set, under a prespecified statistical analysis plan. These data can also form the primary basis of the company's planned BLA submission.
The phase I/II study in the United States enrolled 26 patients with early manifest Huntington's disease, who were randomized to receive either AMT-130 at a low dose (n=6), a high dose (n=10), or a sham procedure (n=10). Patients in the treatment arms received a one-time administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study includes a 12-month blinded phase followed by unblinded long-term follow-up of treated patients for five years. An additional four control patients crossed over to treatment.
In Europe, an open-label phase Ib/II study enrolled 13 patients with early manifest Huntington's disease, including six receiving the low dose and seven receiving the high dose of AMT-130.
Beyond these, uniQure enrolled 12 additional patients across U.S. and European sites in a third cohort evaluating both dose levels of AMT-130 in combination with immunosuppression using the established stereotactic administration procedure.
A fourth U.S.-based cohort enrolled six patients and is assessing the high-dose regimen in patients with lower striatal volumes than those included in earlier cohorts.
AMT-130 has received multiple designations from the FDA, including the Regenerative Medicine Advanced Therapy (RMAT) designation — the first ever granted to a Huntington's disease therapy —as well as Breakthrough Therapy and Fast Track designations.
A potential approval of this gene therapy will be a significant boost for QURE.
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.
QURE shares have surged 93.4% year to date against the industry’s 1.8% decline.
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QURE’s Other Pipeline Candidates
Apart from AMT-130, uniQure’s pipeline comprises a few candidates that are currently undergoing in development.
The company is evaluating AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE) in the phase I/IIa study. The other candidate in the pipeline is AMT-191 for the treatment of 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.
QURE’s Zacks Rank and Stocks to Consider
uniQure currently carries a Zacks Rank #3 (Hold). A couple of better-ranked biotech stocks are Immunocore IMCR and Liquidia Corporation LQDA, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, earnings per share (EPS) estimates for Immunocore have remained unchanged at 6 cents for 2026 and at 87 cents for 2027.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 46.66%.
Over the past 60 days, estimates for Liquidia’s 2026 EPS have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have surged 106.1% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
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This article originally published on Zacks Investment Research (zacks.com).