uniQure (NASDAQ:QURE) Stock

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.

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CSLLY

CSL and uniQure&#39;s Hemgenix shows sustained efficacy in hemophilia B, reducing bleeding rates by 90% and eliminating prophylaxis need for most patients at year four.

uniQure/ CSL Behring&#39;s Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years

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uniQure NV

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

<a href="https://www.globenewswire.com/Tracker?data=z0AWl3tJoSIUl76KC4vUVUAzdm1l2rsX7LsixFW855l2-sT40eG8RxNmqZMlS5i3Qp494Ylft9o_TKMdGS0ctQ==">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 completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease. Additionally, the Independent Data Monitoring Committee (IDMC) reviewed safety data from the initial two patients enrolled in the first cohort. The IDMC's review identified no significant safety concerns and recommended proceeding with enrollment in the second cohort.

uniQure Completes First Cohort Enrollment in Gene Therapy Trial for Fabry Disease, Safety Review Clears Path for Next Phase

uniQure Announced Independent Data Monitoring Committee For EPISOD1, uniQure's Phase I/II Clinical Trial Of AMT-162 Investigational Gene Therapy Has met And Reviewed 28-day Safety Data From First Study Cohort

HC Wainwright &amp; Co. analyst Patrick Trucchio 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 $25 to $70.

Price Target

Analyst Ratings

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

RBC Capital analyst Luca Issi reiterates uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Outperform and maintains $24 price target.

Reiteration

RBC Capital Reiterates Outperform on uniQure, Maintains $24 Price Target

WIIM

UniQure shares are trading lower after the company announced a public offering of 4.41 million shares.

The aggregate gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by uniQure, are expected to be approximately $75 million.&nbsp;

Offerings

uniQure Prices Public Offering Of 4.41M Of Its Ordinary Shares At $17.00 Per Share

uniQure 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 it has commenced an underwritten public offering of its ordinary shares and pre-funded warrants to purchase its ordinary shares. All securities to be sold in the offering will be offered by uniQure. In addition, uniQure intends to grant the underwriters a 30-day option to purchase up to 15% additional ordinary shares at the public offering price, less underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.
Leerink Partners is acting as the bookrunning manager for the proposed offering.

uniQure Announces Proposed Public Offering; No Size Or Amount Disclosed

Mizuho analyst Uy Ear maintains uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Neutral and raises the price target from $7 to $20.

Mizuho Maintains Neutral on uniQure, Raises Price Target to $20

UniQure shares are trading higher after Stifel maintained a Buy rating on the stock and raised its price target from $12 to $32.

Stifel analyst Paul Matteis 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 $12 to $32.

Stifel Maintains Buy on uniQure, Raises Price Target to $32

Goldman Sachs analyst Salveen Richter maintains uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Neutral and raises the price target from $9 to $20.

Goldman Sachs Maintains Neutral on uniQure, Raises Price Target to $20

UniQure shares are trading higher after RBC Capital and Leerink Partners maintained Outperform ratings on the stock and raised their respective price targets.

Leerink Partners maintains uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Outperform and raises the price target from $26 to $44.

Leerink Partners Maintains Outperform on uniQure, Raises Price Target to $44

Guggenheim analyst Debjit Chattopadhyay reiterates uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) from Buy to Buy.

Guggenheim Reiterates Buy on uniQureto Buy

RBC Capital analyst Luca Issi maintains uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) with a Outperform and raises the price target from $14 to $20.

RBC Capital Maintains Outperform on uniQure, Raises Price Target to $20

U.S. stocks traded mixed toward the end of trading, with the S&amp;P 500 falling by around 0.1% on Tuesday.
The Dow traded down 0.02% to 44,393.25 while the NASDAQ rose 0.03% to 19,743.54. The S&amp;P 500 also fell, dropping, 0.06% to 6,049.44.
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Leading and Lagging Sectors Communication services shares jumped by 2.3% on Tuesday.
In trading on Tuesday, real estate shares fell by 1%.
Top Headline
On Tuesday, AutoZone, Inc (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/AZO#NYSE">AZO</a>) reported weaker-than-expected earnings and sales results for its fiscal first quarter.
The company posted earnings per share of $32.52, missing the Street view of $33.76. Quarterly revenues of $4.28 billion (+2.1% year over year) missed the analyst consensus of $4.30 billion.
Equities Trading UP &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp; &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;
<ul>
<li>Chimerix, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CMRX#NASDAQ">CMRX</a>) shares shot up 213% to $2.71 after the company announced it will submit dordaviprone for accelerated approval to the FDA for patients with recurrent H3 K27M-mutant diffuse glioma.</li>
<li>Shares of Cumberland Pharmaceuticals Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CPIX#NASDAQ">CPIX</a>) got a boost, surging 94% to $2.4001 after the company announced the FDA has approved its supplemental new drug application for acetadote.</li>
<li>uniQure N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) shares were also up, gaining 129% to $16.71 after the company announced an agreement with the FDA on key elements of an accelerated pathway for AMT-130.</li>
</ul>
Equities Trading DOWN
<ul>
<li>CervoMed Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CRVO#NASDAQ">CRVO</a>) shares dropped 77% to $2.34 after the company announced its Phase 2b trial of Neflamapimod failed to meet its key endpoints for the treatment of dementia.</li>
<li>Shares of Yext, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/YEXT#NYSE">YEXT</a>) were down 17% to $7.07 following third-quarter results.</li>
<li>MEDIROM Healthcare Technologies Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MRM#NASDAQ">MRM</a>) was down, falling 46% to $1.52. MEDIROM Healthcare priced its $5 million public offering of 2.86 million American Depositary Shares at $1.75 per ADS.</li>
</ul>
Commodities In commodity news, oil traded up 0.7% to $68.84 while gold traded up 1.2% at $2,718.80.
Silver traded up 0.4% to $32.73 on Tuesday, while copper fell 0.1% to $4.2730.
Euro zone
European shares closed lower today. The eurozone's STOXX 600 fell 0.52%, Germany's DAX fell 0.08% and France's CAC 40 fell 1.14%. Spain's IBEX 35 Index fell 0.38%, while London's FTSE 100 fell 0.86%.
Asia Pacific Markets
Asian markets closed mixed on Monday, with Japan's Nikkei 225 gaining 0.18%, Hong Kong's Hang Seng Index gaining 2.76%, China's Shanghai Composite Index falling 0.05% and India's BSE Sensex falling 0.25%.
Economics
U.S. nonfarm business sector labor productivity increased by 2.2% in the third quarter, in-line with the preliminary reading.&nbsp;&nbsp;&nbsp;&nbsp;
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S&P 500 Edges Lower; AutoZone Posts Downbeat Earnings

U.S. stocks traded mostly higher midway through trading, with the Nasdaq Composite gaining around 50 points on Tuesday.
The Dow traded up 0.02% to 44,412.05 while the NASDAQ rose 0.25% to 19,786.14. The S&amp;P 500 also rose, gaining, 0.07% to 6,057.30.
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Leading and Lagging Sectors Communication services shares jumped by 2.5% on Tuesday.
In trading on Tuesday, real estate shares fell by 0.9%.
Top Headline
Ollie's Bargain Outlet Holdings, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/OLLI#NASDAQ">OLLI</a>) shares jumped over 14% on Tuesday after the company released third-quarter results.
The company reported third-quarter adjusted earnings per share of 58 cents, beating the street view of 57 cents. Quarterly sales of $517.43 million (+7.8%) beat the analyst consensus of $517.36 million. The increase in net sales was the result of new store unit growth.
Equities Trading UP &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp; &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;
<ul>
<li>Chimerix, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CMRX#NASDAQ">CMRX</a>) shares shot up 207% to $2.67 after the company announced it will submit dordaviprone for accelerated approval to the FDA for patients with recurrent H3 K27M-mutant diffuse glioma.</li>
<li>Shares of Cumberland Pharmaceuticals Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CPIX#NASDAQ">CPIX</a>) got a boost, surging 93% to $2.3888 after the company announced the FDA has approved its supplemental new drug application for acetadote.</li>
<li>uniQure N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) shares were also up, gaining 130% to $16.78 after the company announced an agreement with the FDA on key elements of an accelerated pathway for AMT-130.</li>
</ul>
Equities Trading DOWN
<ul>
<li>CervoMed Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CRVO#NASDAQ">CRVO</a>) shares dropped 78% to $2.2250 after the company announced its Phase 2b trial of Neflamapimod failed to meet its key endpoints for the treatment of dementia.</li>
<li>Shares of Yext, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/YEXT#NYSE">YEXT</a>) were down 18% to $7.01 following third-quarter results.</li>
<li>MEDIROM Healthcare Technologies Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MRM#NASDAQ">MRM</a>) was down, falling 47% to $1.50. MEDIROM Healthcare priced its $5 million public offering of 2.86 million American Depositary Shares at $1.75 per ADS.</li>
</ul>
Commodities In commodity news, oil traded up 0.8% to $68.94 while gold traded up 1.2% at $2,718.90.
Silver traded up 0.3% to $32.710 on Tuesday, while copper fell 0.3% to $4.2635.
Euro zone
European shares closed lower today. The eurozone's STOXX 600 fell 0.52%, Germany's DAX fell 0.08% and France's CAC 40 fell 1.14%. Spain's IBEX 35 Index fell 0.38%, while London's FTSE 100 fell 0.86%.
Asia Pacific Markets
Asian markets closed mixed on Monday, with Japan's Nikkei 225 gaining 0.18%, Hong Kong's Hang Seng Index gaining 2.76%, China's Shanghai Composite Index falling 0.05% and India's BSE Sensex falling 0.25%.
Economics
U.S. nonfarm business sector labor productivity increased by 2.2% in the third quarter, in-line with the preliminary reading.&nbsp;&nbsp;&nbsp;&nbsp;
Now Read This: 
<ul>
<li><a href="https://www.benzinga.com/24/12/42412458/amazon-to-rally-around-15-here-are-10-top-analyst-forecasts-for-tuesday">Amazon To Rally Around 15%? Here Are 10 Top Analyst Forecasts For Tuesday</a></li>
</ul>

Gold Gains Over 1%; Ollie's Bargain Outlet Shares Jump After Q3 Earnings

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 $25 price target.

HC Wainwright & Co. Reiterates Buy on uniQure, Maintains $25 Price Target

Raymond James analyst Danielle Brill upgrades uniQure (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) from Outperform to Strong Buy and raises the price target from $20 to $52.

Upgrades

Raymond James Upgrades uniQure to Strong Buy, Raises Price Target to $52

Press Releases

uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease

uniQure Announces Dosing of First Patient in Phase I/II Clinical Trial of AMT-162 for the Treatment SOD1-ALS

uniQure Announces Third Quarter 2024 Financial Results and Highlights Recent Company Progress

uniQure Announces Dosing of First Patient in GenTLE Phase I/IIa Clinical Trial of AMT-260 for the Treatment of Refractory Mesial Temporal Lobe Epilepsy

uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington's Disease

uniQure Announces Proposed Public Offering

uniQure Announces Pricing of its Public Offering

uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALS

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

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