L'intérêt à découvert pour uniQure donne aux investisseurs une idée du degré auquel les investisseurs parient sur la baisse de l'action de uniQure. Les données sur l'intérêt à découvert sont mises à jour toutes les deux semaines.

uniQure (NASDAQ:QURE) Stock, Rapport sur les intérêts à découvert

uniQure

uniQure NV is a gene therapy company. It develops treatments and platforms for patients suffering from genetic and other devastating diseases. Its products and services are focused on hemophilia, Huntington's disease, and cardiovascular diseases. The company is focused on the development of the pipeline of gene therapies with the collaboration of Bristol Myers Squibb for cardiovascular diseases.

uniQure NV

~ Alignment with FDA continues to support Accelerated Approval pathway ~
~ BLA submission planned for first quarter of 2026 ~
~ Conference call today at 8:30 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- <a href="https://www.globenewswire.com/Tracker?data=KJrUuvgpMd-Ddja3YfjTaW-sEFYSuEc8TNCL8DhDuaFHTkrvl5gSZYYVlr1J5ubOEYW0Znoht2JKxs4d8ldSNg==">uniQure</a> N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington's disease. Following recent Type B meetings and further guidance from the U.S. Food and Drug Administration (FDA), the Company has reached alignment with the FDA on several key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information that will support a Biologics License Application (BLA) submission expected in the first quarter of 2026.
Statistical Analysis Plan
In the second quarter of 2025, the Company held a Type B meeting with the FDA to discuss the proposed use of external control data and the prospectively defined statistical analysis plan (SAP) in support of the planned BLA submission for AMT-130. The FDA continued to support its prior agreement that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may serve as an acceptable registrational, intermediate clinical endpoint for accelerated approval. The FDA agreed that the primary efficacy analysis for the BLA will evaluate the 3-year change in cUHDRS in high-dose AMT-130 patients compared to a propensity score-adjusted external control arm. The Company plans to use propensity score-weighted external control derived from the ENROLL-HD dataset for the primary analysis and to submit certain sensitivity analyses, including one using a propensity score-matched external control, as additional support.
The FDA also agreed that ENROLL-HD – a large, prospective, longitudinal, natural history study of patients with Huntington's disease – may be acceptable as the external control dataset for the primary analysis of the trial data along with additional sensitivity analyses using the TRACK-HD/TRACK-ON and PREDICT-HD datasets. To date, approximately 33,000 patients have enrolled in ENROLL-HD. Compared to previously used natural history studies like TRACK-HD and PREDICT-HD, ENROLL-HD offers a substantially larger sample size, lower attrition rates, and longer average patient follow-up. The Company expects the larger sample size to reduce variability in the external control data and enhance the robustness of the SAP.
The Company plans to submit an updated SAP consistent with discussions from the recently held Type B meeting to the FDA in the second quarter of 2025.
Chemistry, Manufacturing and Controls (CMC)
In the first quarter of 2025, the Company held a Type B meeting with the FDA to discuss CMC requirements in support of the planned BLA submission for AMT-130. The FDA agreed that validation of the AMT-130 manufacturing process should be possible using experience and prior knowledge from the etranacogene dezaparvovec-drlb (HEMGENIX&#xAE;) process, complemented with additional full-scale AMT-130 GMP batches and a single Process Performance Qualification (PPQ) batch.
The FDA also agreed with the Company's proposed drug product release testing plan, including the proposed potency assay, pending completion of qualification and specification setting activities.
Next Steps for the Planned BLA Submission
Based on these recent Type B meetings with the FDA, the Company continues to prepare for a BLA submission for AMT-130 in the treatment of Huntington's disease. Certain key next steps and expected timing include:
<ul>
<li>Q2 2025: submit updated SAP to the FDA &nbsp;</li>
<li>Q3 2025: initiate PPQ run and present topline Phase I/II data per the SAP &nbsp;</li>
<li>Q4 2025: hold pre-BLA meeting with the FDA &nbsp;</li>
<li>Q1 2026: submit BLA with a request for priority review designation &nbsp;</li>
</ul>

News

QURE

uniQure Secures FDA Alignment On AMT-130, Targeting 2026 BLA Submission

Chardan Capital analyst Daniil Gataulin maintains uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Buy and maintains $38 price target.

Price Target

Analyst Ratings

Chardan Capital Maintains Buy on uniQure, Maintains $38 Price Target

HC Wainwright &amp; Co. analyst Patrick Trucchio reiterates uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Buy and maintains $70 price target.

Reiteration

HC Wainwright & Co. Reiterates Buy on uniQure, Maintains $70 Price Target

~ No serious adverse events and 92% reduction in seizure frequency observed in the&nbsp;first trial participant through first five months of follow up ~
~ Data to be presented today at Epilepsy Therapies &amp; Diagnostics Development Symposium (ETDD) ~
LEXINGTON, Mass. and AMSTERDAM, May 29, 2025 (GLOBE NEWSWIRE) -- <a href="https://www.globenewswire.com/Tracker?data=hSOiic6k5pW8K81kp2xoPfCxK7Ad4gSkFBM1R7qS6x-ja85V662-C8N0Ok8imcUf8N6O6XPJCDjh9RYh7brfCQ==">uniQure</a> N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of a clinical case study of the first participant dosed in the first cohort of its ongoing Phase I/IIa trial of an investigational gene therapy candidate, AMT-260, in patients with refractory MTLE. The data are being presented today, May 29, 2025 by Firas Taha, M.D., Medical Director, Clinical Development at uniQure, at the Epilepsy Therapies &amp; Diagnostics Development Symposium in Leesburg, Virginia.
The first participant in the GenTLE clinical trial has been followed for five months post-administration of AMT-260. Prior to treatment, the participant experienced an average of seven seizures a month, including five seizures in the 30-day period immediately prior to dosing. Since receiving AMT-260, the participant has reported two seizures during the five-month follow-up period, with no seizures reported during the last 60 days as of April 17th (cutoff date), the last date data were obtained on this participant for presentation in the ETDD symposium case study and no reported serious adverse events as of the cutoff date.

uniQure Presents Clinical Case Study Of First Participant Dosed In First Cohort Of Ongoing Phase I/Iia Trial Of Investigational Gene Therapy Candidate, AMT-260, In Patients With Refractory MTLE

Guggenheim analyst Debjit Chattopadhyay reiterates uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Buy and maintains $28 price target.

Guggenheim Reiterates Buy on uniQure, Maintains $28 Price Target

uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) reported quarterly losses of $(0.82) per share which beat the analyst consensus estimate of $(0.95) by 13.68 percent. This is a 39.71 percent increase over losses of $(1.36) per share from the same period last year. The company reported quarterly sales of $1.57 million which missed the analyst consensus estimate of $6.27 million by 75.02 percent. This is a 81.53 percent decrease over sales of $8.48 million the same period last year.

Earnings

Earnings Beats

Earnings Misses

uniQure Q1 EPS $(0.82) Beats $(0.95) Estimate, Sales $1.57M Miss $6.27M Estimate

<h3>Companies Reporting Before The Bell</h3>
• NeoGenomics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NEO#NASDAQ">NEO</a>) is projected to report quarterly earnings at $0.00 per share on revenue of $120.47 million. 
• Anika Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ANIK#NASDAQ">ANIK</a>) is estimated to report quarterly earnings at $0.09 per share on revenue of $28.07 million. 
• Gogo (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/GOGO#NASDAQ">GOGO</a>) is likely to report quarterly earnings at $0.08 per share on revenue of $214.44 million. 
• Protalix BioTherapeutics (AMEX:<a class="ticker" href="https://www.benzinga.com/stock/PLX#AMEX">PLX</a>) is estimated to report quarterly earnings at $0.08 per share on revenue of $21.60 million. 
• ANI Pharmaceuticals (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ANIP#NASDAQ">ANIP</a>) is projected to report quarterly loss at $0.06 per share on revenue of $180.69 million. 
• TELUS Intl (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/TIXT#NYSE">TIXT</a>) is projected to report quarterly earnings at $0.05 per share on revenue of $650.27 million. 
• EchoStar (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/SATS#NASDAQ">SATS</a>) is projected to report quarterly loss at $0.80 per share on revenue of $3.87 billion. 
• Outbrain (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/OB#NASDAQ">OB</a>) is projected to report quarterly loss at $0.05 per share on revenue of $285.18 million. 
• Essent Group (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ESNT#NYSE">ESNT</a>) is projected to report quarterly earnings at $1.65 per share on revenue of $310.79 million. 
• Veren (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/VRN#NYSE">VRN</a>) is likely to report quarterly earnings at $0.29 per share on revenue of $719.90 million. 
• TXNM Energy (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/TXNM#NYSE">TXNM</a>) is expected to report quarterly earnings at $0.20 per share on revenue of $432.72 million. 
• Algonquin Power (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/AQN#NYSE">AQN</a>) is projected to report quarterly earnings at $0.10 per share on revenue of $652.63 million. 
• Embecta (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EMBC#NASDAQ">EMBC</a>) is expected to report quarterly earnings at $0.59 per share on revenue of $261.77 million. 
• Docebo (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/DCBO#NASDAQ">DCBO</a>) is estimated to report quarterly earnings at $0.22 per share on revenue of $57.10 million. 
• American Strategic (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/NYC#NYSE">NYC</a>) is projected to report earnings for its first quarter. 
• Plains All American (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PAA#NASDAQ">PAA</a>) is projected to report quarterly earnings at $0.45 per share on revenue of $14.00 billion. 
• Enbridge (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ENB#NYSE">ENB</a>) is expected to report quarterly earnings at $0.95 per share on revenue of $10.31 billion. 
• Creative Media (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CMCT#NASDAQ">CMCT</a>) is estimated to report earnings for its first quarter. 
• uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) is estimated to report quarterly loss at $0.96 per share on revenue of $6.10 million. 
• Ocugen (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/OCGN#NASDAQ">OCGN</a>) is projected to report earnings for its first quarter. 
• Inuvo (AMEX:<a class="ticker" href="https://www.benzinga.com/stock/INUV#AMEX">INUV</a>) is likely to report quarterly loss at $0.01 per share on revenue of $23.68 million. 
• Ultralife (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ULBI#NASDAQ">ULBI</a>) is expected to report quarterly earnings at $0.21 per share on revenue of $54.50 million. 
• TeraWulf (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/WULF#NASDAQ">WULF</a>) is projected to report quarterly loss at $0.06 per share on revenue of $47.69 million. 
• NextNav (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NN#NASDAQ">NN</a>) is likely to report quarterly loss at $0.14 per share on revenue of $1.87 million. 
• PAR Technology (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/PAR#NYSE">PAR</a>) is expected to report quarterly loss at $0.04 per share on revenue of $105.16 million. 
• InspireMD (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NSPR#NASDAQ">NSPR</a>) is estimated to report quarterly loss at $0.20 per share on revenue of $1.45 million. 
• Telos (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/TLS#NASDAQ">TLS</a>) is projected to report quarterly loss at $0.05 per share on revenue of $29.22 million. 
• Calumet (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CLMT#NASDAQ">CLMT</a>) is projected to report quarterly loss at $0.52 per share on revenue of $918.50 million. 
• Koppers Holdings (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/KOP#NYSE">KOP</a>) is projected to report quarterly earnings at $0.56 per share on revenue of $488.00 million. 
• Rapid Micro Biosystems (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RPID#NASDAQ">RPID</a>) is likely to report quarterly loss at $0.27 per share on revenue of $6.63 million. 
• Associated Cap Gr (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/AC#NYSE">AC</a>) is estimated to report quarterly loss at $0.47 per share on revenue of $5.33 billion. 
• Federal Agricultural (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/AGM#NYSE">AGM</a>) is estimated to report earnings for its first quarter. 
• Kymera Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/KYMR#NASDAQ">KYMR</a>) is estimated to report quarterly loss at $0.90 per share on revenue of $10.73 million. 
• First Trust Switzerland AlphaDEX Fund (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/FSZ#NASDAQ">FSZ</a>) is projected to report quarterly earnings at $0.25 per share on revenue of $171.64 million. 
• Sylvamo (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/SLVM#NYSE">SLVM</a>) is likely to report quarterly earnings at $0.69 per share on revenue of $817.58 million. 
• Strawberry Fields REIT (AMEX:<a class="ticker" href="https://www.benzinga.com/stock/STRW#AMEX">STRW</a>) is projected to report earnings for its first quarter. 
• AMC Networks (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AMCX#NASDAQ">AMCX</a>) is estimated to report quarterly earnings at $0.81 per share on revenue of $567.23 million. 
• Alpha Metallurgical (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/AMR#NYSE">AMR</a>) is likely to report quarterly loss at $0.17 per share on revenue of $586.15 million. 
• Construction Partners (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ROAD#NASDAQ">ROAD</a>) is likely to report quarterly loss at $0.06 per share on revenue of $559.61 million. 
• Starwood Property Trust (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/STWD#NYSE">STWD</a>) is projected to report quarterly earnings at $0.49 per share on revenue of $468.00 million. 
• BKV (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/BKV#NYSE">BKV</a>) is likely to report quarterly earnings at $0.09 per share on revenue of $208.66 million. 
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<h3>Companies Reporting After The Bell</h3>
• Motorsport Games (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MSGM#NASDAQ">MSGM</a>) is estimated to report earnings for its first quarter. 
• ContextLogic (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/LOGC#NASDAQ">LOGC</a>) is projected to report earnings for its first quarter. 
• Hawaiian Electric Indus (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/HE#NYSE">HE</a>) is expected to report earnings for its first quarter. 
• Orla Mining (AMEX:<a class="ticker" href="https://www.benzinga.com/stock/ORLA#AMEX">ORLA</a>) is projected to report earnings for its first quarter. 
This article was generated by Benzinga's automated content engine and reviewed by an editor.

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Earnings Scheduled For May 9, 2025

uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) is set to give its latest quarterly earnings report on Friday, 2025-05-09. Here's what investors need to know before the announcement.
Analysts estimate that uniQure will report an earnings per share (EPS) of $-0.96. 
The announcement from uniQure is eagerly anticipated, with investors seeking news of surpassing estimates and favorable guidance for the next quarter.
It's worth noting for new investors that guidance can be a key determinant of stock price movements.
<h3>Past Earnings Performance</h3>
The company's EPS missed by $0.99 in the last quarter, leading to a 8.95% increase in the share price on the following day.
Here's a look at uniQure's past performance and the resulting price change:

<table>
<thead>
<tr>
<th>Quarter</th>
<th>Q4 2024</th>
<th>Q3 2024</th>
<th>Q2 2024</th>
<th>Q1 2024</th>
</tr>
</thead>
<tbody>
<tr>
<td>EPS Estimate</td>
<td>-0.51</td>
<td>-1</td>
<td>-1.25</td>
<td>-1.33</td>
</tr>
<tr>
<td>EPS Actual</td>
<td>-1.50</td>
<td>-0.91</td>
<td>-1.16</td>
<td>-1.36</td>
</tr>
<tr>
<td>Price Change %</td>
<td>9.0%</td>
<td>18.0%</td>
<td>-2.0%</td>
<td>1.0%</td>
</tr>
</tbody>
</table>

<img src="https://www.benzinga.com/files/images/story/2025/1746723712_0.png" alt="eps graph" />
<h3>Tracking uniQure's Stock Performance</h3>
Shares of uniQure were trading at $10.06 as of May 07. Over the last 52-week period, shares are up 147.36%. Given that these returns are generally positive, long-term shareholders are likely bullish going into this earnings release. 
<a href="https://www.benzinga.com/quote/QURE/earnings">To track all earnings releases for uniQure visit their earnings calendar on our site.</a>
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Earnings Outlook For uniQure

U.S. stocks were lower, with the Dow Jones index falling around 700 points on Monday.
Shares of Wolverine World Wide, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/WWW#NYSE">WWW</a>) rose sharply during <a href="https://www.benzinga.com/quote/WWW/analyst-ratings">Monday's session</a>.
Baird analyst Jonathan Komp upgraded the rating for Wolverine World Wide from Neutral to Outperform.
Wolverine World Wide shares jumped 7.2% to $11.56 on Monday.
Here are some other big stocks recording gains in today's session.
<ul>
<li>Verve Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/VERV#NASDAQ">VERV</a>) jumped 15% to $5.28. Cantor Fitzgerald analyst Rick Bienkowski, on April 15, upgraded Verve Therapeutics from Neutral to Overweight.</li>
<li>Waton Financial Limited (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/WTF#NASDAQ">WTF</a>) gained 14.8% to $7.43.</li>
<li>uniQure N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) shares rose 12.4% to $14.61. uniQure was recently granted FDA granted Breakthrough Therapy designation for AMT-130 for the treatment of Huntington&#8217;s disease.</li>
<li>Intellia Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NTLA#NASDAQ">NTLA</a>) gained 12% to $8.10.</li>
<li>Rocket Pharmaceuticals, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RCKT#NASDAQ">RCKT</a>) climbed 11.4% to $7.05.</li>
<li>Erasca, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ERAS#NASDAQ">ERAS</a>) climbed 11.2% to $1.2900.</li>
<li>Microvast Holdings, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MVST#NASDAQ">MVST</a>) rose 11% to $2.0324.</li>
<li>Beam Therapeutics Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) gained 10.6% to $18.81.</li>
<li>Replimune Group, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/REPL#NASDAQ">REPL</a>) shares gained 9.6% to $8.76.</li>
<li>Pacira BioSciences, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PCRX#NASDAQ">PCRX</a>) shares rose 6.3% to $26.21 as the company announced a $300 million share repurchase program.</li>
<li>CleanSpark, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CLSK#NASDAQ">CLSK</a>) gained 5.3% to $7.90.</li>
<li>B2Gold Corp. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/BTG#NYSE">BTG</a>) gained 4.8% to $3.4800.</li>
</ul>
Now Read This: 
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Photo via Shutterstock

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Wolverine World Wide, Pacira BioSciences And Other Big Stocks Moving Higher On Monday

U.S. stocks were lower, with the Dow Jones index falling around 550 points on Thursday.
Shares of uniQure N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) rose sharply during Thursday's session after the company was granted FDA granted Breakthrough Therapy designation for AMT-130 for the treatment of <a href="https://www.benzinga.com/quote/QURE">Huntington&#8217;s disease.</a>
uniQure shares jumped 45.3% to $13.65 on Thursday.
Here are some other big stocks recording gains in today's session.
<ul>
<li>Structure Therapeutics Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/GPCR#NASDAQ">GPCR</a>) gained 21.4% to $22.49.</li>
<li>Hertz Global Holdings, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/HTZ#NASDAQ">HTZ</a>) rose 20.3% to $6.99 after Pershing Square Capital Management disclosed a stake via a 13F. CNBC reported that Pershing Square&#8217;s stake is 19.8%, through shares &amp; swaps.</li>
<li>Eli Lilly and Company (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/LLY#NYSE">LLY</a>) gained 15.3% to $847.08 after the company announced topline Phase 3 results from its ACHIEVE-1 trial, which showed orforglipron demonstrated statistically significant efficacy results and a safety profile consistent with injectable GLP-1 medicines.</li>
<li>Capricor Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CAPR#NASDAQ">CAPR</a>) rose 13.8% to $10.97.</li>
<li>ClearPoint Neuro, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CLPT#NASDAQ">CLPT</a>) gained 13% to $13.02.</li>
<li>VersaBank (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/VBNK#NASDAQ">VBNK</a>) rose 12% to $10.91.</li>
<li>Evotec SE (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EVO#NASDAQ">EVO</a>) jumped 10.7% to $3.6200 following FY24 financial results.</li>
<li>Avis Budget Group, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CAR#NASDAQ">CAR</a>) gained 10.1% to $80.00. The move came as investors responded to news that billionaire investor Bill Ackman&#8217;s Pershing Square Capital Management significantly increased its stake in Hertz Global Holdings Inc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/HTZ#NASDAQ">HTZ</a>).</li>
<li>Insteel Industries, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/IIIN#NYSE">IIIN</a>) gained 9.1% to $29.15 following second-quarter results.</li>
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uniQure, Hertz Global, Eli Lilly And Other Big Stocks Moving Higher On Thursday

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to uniQure N.V.&#8217;s (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) AMT-130 <a href="https://www.benzinga.com/general/health-care" target="_blank" rel="noreferrer noopener">for Huntington&#8217;s disease</a> on Thursday
This designation is in addition to the Regenerative Medicine Advanced Therapy (RMAT) designation, the Orphan Drug designation and the Fast Track designation, all of which were previously granted <a href="https://www.benzinga.com/pressreleases/25/04/g44860315/uniqure-announces-fda-breakthrough-therapy-designation-granted-to-amt-130-for-the-treatment-of-hun" target="_blank" rel="noreferrer noopener">by the FDA to AMT-130</a>.
Huntington&#8217;s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms, including chorea, behavioral abnormalities and cognitive decline, resulting in progressive physical and mental deterioration.
Also Read: <a target="_blank" href="https://www.benzinga.com/general/biotech/25/02/43569013/uniqure-csl-behrings-hemophilia-gene-therapy-shows-sustained-efficacy-and-safety-at-four-years" rel="noreferrer noopener">uniQure/ CSL Behring&#8217;s Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years</a>
According to a 2021 study in Neuroepidemiology, approximately 70,000 people have been diagnosed with Huntington&#8217;s disease in the U.S. and Europe.
The Breakthrough Therapy designation is supported by clinical data from the ongoing Phase 1/2 trials of AMT-130 for Huntington&#8217;s disease.
In July 2024, uniQure presented interim data at 24 months, showing dose-dependent slowing of disease progression based on the cUHDRS in treated patients compared to a propensity-weighted natural history. To date, 45 patients have received AMT-130.
The Breakthrough Therapy designation is intended to expedite the development and review of investigational therapeutic candidates for treating a serious condition. Preliminary clinical evidence suggests that the drug may show substantial improvement over available therapy on one or more clinically significant endpoints.
In general, the preliminary clinical evidence should show a clear advantage over available therapy. A drug with Breakthrough Therapy designation is eligible for all Fast Track designation features and intensive guidance on an efficient drug development program.
<h2 class="wp-block-heading">uniQure Stock Predication For 2025</h2>
Equity research analysts, both on and off Wall Street, typically use earnings growth and fundamental research as a form of valuation and forecasting. But many in trading turn to technical analysis as a way to form predictive models for share price trajectory.
Some investors look to trends to help forecast where they believe a stock could trade at a certain point in the future. Looking at uniQure, an investor could make an assessment of a stock's long-term prospects using a moving average and trend line. If they believe a stock will remain above the moving average, which many consider a bullish signal, they can extrapolate that trend into the future using a trendline. For uniQure, the 200-day moving average sits at $9.61, according to <a href="https://pro.benzinga.com/">Benzinga Pro</a>, which is above the current price of $9.39. For more on charts and trend lines, <a href="https://pro.benzinga.com/">see a description here.</a>
Traders believe that when a stock is above its moving average, it is a generally bullish signal, and when it crosses below, it is a more negative signal. Investors can use trend lines to make an educated guess about where a stock might trade in the future if conditions remain stable.
Price Action: QURE stock is up 49.8% at $14.07 during the premarket session at the last check on Thursday.
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Photo: Shutterstock

Biotech

Health Care

General

Why Is Gene Therapy Developer uniQure Stock Trading Higher On Thursday?

WIIM

uniQure shares are trading higher after the company was granted FDA granted Breakthrough Therapy designation for AMT-130 for the treatment of Huntington's disease.

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~
~ Additional regulatory update and guidance on the Biologics License Application submission
expected in the second quarter of 2025 ~
LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- <a href="https://www.globenewswire.com/Tracker?data=JOV8TGQtSE6C9NWzaMvbCG-dm9N8uvfIH6VjMYQjF-8xR9NK2Ew8yFRfi--XIwq9-1J7opX1h6bLkIUMRUFRWQ==">uniQure</a> N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available. This designation is in addition to Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation and Fast Track designation, all previously granted by the FDA to AMT-130.
The Breakthrough Therapy designation is supported by clinical data from the ongoing Phase I/II trials of AMT-130 for the treatment of Huntington's disease. In July 2024, uniQure presented interim data at 24 months that showed dose-dependent slowing of disease progression based on the cUHDRS of treated patients compared to a propensity-weighted natural history. To date, a total of 45 patients have received AMT-130.
Breakthrough Therapy designation is intended to expedite the development and review of investigational therapeutic candidates that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). In general, the preliminary clinical evidence should show a clear advantage over available therapy. A drug that receives Breakthrough Therapy designation is eligible for all Fast Track designation features, intensive guidance on an efficient drug development program, and FDA commitment involving senior managers1.

FDA Grants Breakthrough Therapy Designation To uniQure's AMT-130 For Treatment Of Huntington's Disease

Chardan Capital analyst Daniil Gataulin maintains uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Buy and raises the price target from $27 to $38.

Chardan Capital Maintains Buy on uniQure, Raises Price Target to $38

uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) reported quarterly losses of $(4.92) per share which missed the analyst consensus estimate of $(3.99) by 23.31 percent. This is a 23.96 percent increase over losses of $(6.47) per share from the same period last year. The company reported quarterly sales of $27.12 million which missed the analyst consensus estimate of $39.44 million by 31.24 percent. This is a 71.17 percent increase over sales of $15.84 million the same period last year.

uniQure FY 2024 GAAP EPS $(4.92) Misses $(3.99) Estimate, Sales $27.12M Miss $39.44M Estimate

CSL Limited (OTC:<a class="ticker" href="https://www.benzinga.com/stock/CSLLY#OTC">CSLLY</a>) and uniQure Inc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>)&nbsp;announced the four-year results from the pivotal HOPE-B study confirming the long-term durability and safety of a one-time infusion of Hemgenix (etranacogene dezaparvovec-drlb) for <a target="_blank" href="https://www.benzinga.com/pressreleases/25/02/n43560239/csl-behrings-gene-therapy-hemgenix-etranacogene-dezaparvovec-drlb-four-years-post-infusion-data-co" rel="noreferrer noopener">adults hemophilia B patients</a>.
The data showed that through four years, Hemgenix continues to deliver elevated and sustained factor IX activity levels, can offer long-term and greater bleed protection compared to prophylactic treatment, can eliminate the need for routine factor IX prophylaxis, and maintains a favorable safety profile.
Also Read: <a target="_blank" href="https://www.benzinga.com/24/12/42414454/why-is-uniqure-stock-skyrocketing-on-tuesday" rel="noreferrer noopener">Why Is uniQure Stock Skyrocketing On Tuesday?</a>
Approved in 2022 by the FDA, Hemgenix is the first gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes.
It is also the only approved gene therapy for hemophilia B to treat adult patients with and without AAV5-neutralizing antibodies.
In the Phase 3, open-label, single-dose, single-arm HOPE-B trial, 54 adult male participants with severe or moderately severe hemophilia B, with or without preexisting AAV5 neutralizing antibodies, were infused with a single dose of Hemgenix.
Of the 54 participants who received Hemgenix, 51 completed four years of follow-up. Hemgenix produced mean factor IX levels of 41.5 IU/dL (n=50) at year one, 36.7 IU/dL (n=50) at year two, 38.6 IU/dL (n=48) at year three and 37.4 IU/dL (n=47) at year four post-infusion.
In addition, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by approximately 90% from lead-in as compared to year four.
Furthermore, joint bleeds were reduced from a mean ABR of 2.34 at lead-in to 0.09 during year four. In year four, 94% of patients remained free of continuous prophylaxis treatment.
No patients returned to continuous prophylaxis between year three and year four.
There were no serious adverse events related to treatment.
Price Action: QURE stock is down 5.95% at $15.25 at last check Friday.
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CSLLY

uniQure/ CSL Behring's Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years

Press Releases

uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease

Small Cap

CSL Behring's Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B

uniQure Announces 2024 Financial Results and  Highlights Recent Company Progress

uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington's Disease

uniQure to Announce First Quarter 2025 Financial Results

uniQure Announces First Quarter 2025 Financial Results and Highlights of Recent Company Progress

Management

Corbus Pharmaceuticals Names Industry Veteran Rachelle Jacques as Board Chair

uniQure Presents Clinical Case Study of First Patient Dosed with AMT-260 in Refractory Mesial Temporal Lobe Epilepsy (MTLE)

uniQure Provides Regulatory Update on AMT-130 for Huntington's Disease

uniQure Announces Appointment of Kylie O'Keefe  as Chief Customer and Strategy Officer

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