Ultime notizie per uniQure (NASDAQ:QURE) Azioni

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

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>
<li>Fidelity National Information Services, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/FIS#NYSE">FIS</a>) shares jumped 7% to $73.41. Fidelity National Information Services unveiled two strategic moves—acquiring Global Payments' Issuer Solutions unit and selling its remaining stake in Worldpay.</li>
<li>Dollar Tree, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/DLTR#NASDAQ">DLTR</a>) gained 4.4% to $76.40.</li>
</ul>
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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

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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.

Price Target

Analyst Ratings

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

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

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.

Earnings

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

<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.

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.

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

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.

Press Releases

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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