$26.36 +3.36 (+14.61%)
As of Nov-23-20224:00:00 PM ET
FDA Approves UniQure's Hemgenix, First Hemophilia B Gene Therapy For Adults
BENZINGA 8:12 AM ET Nov-23-2022
UniQure N.V.'s (NASDAQ:QURE) partner CSL Limited (OTC:CSLLY) has received FDA approval for Hemgenix (etranacogene dezaparvovec-drlb), a one-time gene therapy for hemophilia B.
CSL licensed the exclusive global rights to Hemgenix from uniQure in May 2021.
UniQure has received about $500 million so far from CSL and will also be eligible to get up to an additional $1.5 billion in commercial milestone payments and royalties.
Cowen analyst Joseph Thome said that Hemgenix's price of $3.5 million is higher than the brokerage's estimated $1.9 million, but its consultants have been optimistic about the therapy's uptake, Reuters reported.
Results from the pivotal HOPE-B trial support the FDA approval, the largest gene therapy trial in hemophilia B.
Results from the study demonstrated that Hemgenix allowed patients to produce mean factor IX activity of 39% at six months and 36.7% at 24 months post-infusion.
Seven to 18 months post-infusion, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54% compared to the six-month lead-in period on factor IX prophylactic replacement therapy (4.1 to 1.9).
In addition, 94% of patients treated with Hemgenix discontinued prophylaxis and remained free of previous continuous routine prophylaxis therapy.
The FDA has already cleared two gene therapies, Zynteglo and Skysona, from Bluebird bio Inc (NASDAQ: BLUE) in August and September, respectively.
Erratic Trading Showing Unusual Volume in Shares of Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) and uniQure N.V. (NASDAQ:QURE)
Patient is a virtue.
If you know what you are doing it pays off.
That's why it's down another 3%
Trust me they are. They just don't want you to think that way.
Great newsGo QURE
uniQure N.V. (NASDAQ:QURE) announced the completion of a company-wide strateghttp://www.benchmarkmonitor.com/2016/11/17/uniqure-n-v-nasdaqqure-announced-the-completion-of-a-company-wide-strategic-review/ic review
uniQure N.V. (NASDAQ:QURE), announced the completion of a company-wide strategic review aimed at refocusing its pipeline, consolidating its manufacturing and enhancing overall execution to drive shareholder value. As a result of this initiative, the Company will prioritize programs in hemophilia B, Huntington’s disease and those associated with its landmark collaboration with Bristol-Myers Squib (BMS) in cardiovascular disease.
uniQure N.V. (NASDAQ:QURE), a Healthcare sector firm, traded 495604 shares in last trading session with closing price of $7.04 per share. Company gross margin stands at 94.90% whereas its return on investment (ROI) is -49.30%. Stock value has moved between $6.64 – 22.71 in last one year. Analyst’s mean target price for uniQure N.V. (NASDAQ:QURE) is $30.10 while analysts mean recommendation is 1.60. QURE EPS growth this year is -49.60%.
Adding all under 9s
QURE data will show in PPS hang on tight
Laaaaaid back QURE love it
QURE run to 12+ on it watch
Panzer nailing biotechs sniper
Looking fantastic QURE
This trade turning into pure genius muhahahhaha
End of summer it hits $12
In de past few weeks way oversold ..
So much for that -- now see 15K bid @ .41.
Ended up selling @ $7.37 this afternoon. Still greatly oversold but not sure can make it over .40 today and market too volatile.
no kiddin man... ez $$$ here...
Posted message 177 to myself. Was meant for you. Good luck.
Yep. You never know do you. Yesterday it went back close to that level early am but didn't buy. Who knew that bios would have the best day in about 4 years. If you have been holding you are doing just fine.
Overbought now so I'll wait and get back in. Great at this level and better when it dips:
too bad... $qure is at bottom...
Uniqure NV (QURE) Earns “Buy” Rating from Chardan Capital
March 28th, 2016 - By Renee Johnson - 0 comments
Uniqure NV logoChardan Capital reaffirmed their buy rating on shares of Uniqure NV (NASDAQ:QURE) in a research report sent to investors on Wednesday morning, MarketBeat.com reports. They currently have a $40.00 price objective on the stock.
A number of other analysts have also recently commented on the stock. Zacks Investment Research cut shares of Uniqure NV from a buy rating to a hold rating in a research report on Wednesday, November 25th. Cowen and Company restated a buy rating on shares of Uniqure NV in a research report on Monday, November 30th. Leerink Swann restated an outperform rating and issued a $48.00 price objective on shares of Uniqure NV in a research report on Friday, January 8th. Janney Montgomery Scott started coverage on shares of Uniqure NV in a research report on Monday, November 30th. They issued a buy rating and a $40.00 price objective for the company. Finally, Piper Jaffray restated an overweight rating and issued a $35.00 price objective on shares of Uniqure NV in a research report on Wednesday, January 6th. One analyst has rated the stock with a hold rating and eleven have given a buy rating to the company. The stock has a consensus rating of Buy and an average target price of $40.36.
Uniqure NV (NASDAQ:QURE) opened at 11.95 on Wednesday. The firm’s market capitalization is $289.41 million. Uniqure NV has a one year low of $10.61 and a one year high of $36.38. The company has a 50 day moving average price of $13.92 and a 200-day moving average price of $18.07.
Several institutional investors have bought and sold shares of the company. Perceptive Advisors LLC increased its position in Uniqure NV by 40.1% in the fourth quarter. Perceptive Advisors LLC now owns 967,093 shares of the company’s stock worth $15,996,000 after buying an additional 276,824 shares in the last quarter. Eventide Asset Management LLC increased its stake in shares of Uniqure NV by 12.9% in the fourth quarter. Eventide Asset Management LLC now owns 174,900 shares of the company’s stock valued at $2,893,000 after buying an additional 20,000 shares during the period. Columbus Circle Investors increased its stake in shares of Uniqure NV by 493.7% in the fourth quarter. Columbus Circle Investors now owns 113,548 shares of the company’s stock valued at $1,878,000 after buying an additional 94,422 shares during the period. Deutsche Bank AG increased its stake in shares of Uniqure NV by 53.2% in the fourth quarter. Deutsche Bank AG now owns 112,088 shares of the company’s stock valued at $1,853,000 after buying an additional 38,943 shares during the period. Finally, Tocqueville Asset Management L.P. increased its stake in shares of Uniqure NV by 5.6% in the fourth quarter. Tocqueville Asset Management L.P. now owns 79,750 shares of the company’s stock valued at $1,319,000 after buying an additional 4,200 shares during the period.
Uniqure NV is a pharmaceutical company based in the Netherlands. It is primarily engaged in the field of gene therapy for orphan diseases. The Company is focused on patients suffering from genetic or acquired diseases by directing the expression of a therapeutic protein or restoring the expression of a missing protein through a single administration.
3/24/2016 HC Wainwright Reiterated Rating Buy $30.00
uniQure N.V. (uniQure) is a gene therapy company. The Company is engaged in developing single treatments with curative results for patients suffering from genetic and other severe diseases. The Company is advancing a focused pipeline of gene therapies that have been developed both internally and through partnerships. The Company’s product candidates include AMT-060 for Hemophilia B, AMT-130 for Huntington's disease, S100A1 for congestive heart failure and Glybera for lipoprotein lipase deficiency (LPLD). uniQure has established clinical proof-of-concept in its lead indication, hemophilia B and has achieved pre-clinical, proof-of-concept in Huntington's disease. The Company, through its collaboration with Bristol Myers-Squibb (BMS), is focused on building a portfolio of gene therapies led by the S100A1 program for congestive heart failure.
AMT-060 for Hemophilia B
AMT-060 is a gene therapy for the treatment of Hemophilia B. Hemophilia B is an inherited disease in males characterized by insufficient blood clotting. AMT-060 consists of the adeno-associated virus (AAV) vector carrying a human Factor IX (FIX) gene cassette that the Company has exclusively licensed from St. Jude Children’s Research Hospital in Memphis, Tennessee. The Company produces this vector with its insect cell-based manufacturing process. It has an ongoing Phase I/II clinical trials.
AMT-130 for Huntington's Disease
AMT-130 is developed for the treatment of Huntington's disease (HD). HD is caused by an inherited defect in a single gene that codes for protein called Huntingtin. The Company’s product candidate AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA, which silences the Huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein.
S100A1 for Congestive Heart Faliure
The Company has entered into an agreement with BMS that provides exclusive access to the Company’s gene therapy technology platform for multiple targets in cardiovascular (and other) diseases (Collaboration and License Agreement). The collaboration includes its proprietary gene therapy program for congestive heart failure, which aims to restore the heart's ability to synthesize S100A1. . S100A1 is a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction. S100A1 deficient hearts show accelerated progression to severe heart failure and increase mortality after cardiac damage. The Company is leading the discovery, non-clinical, analytical and process development effort and is responsible for manufacturing of clinical and commercial supplies using our vector technologies and industrial, proprietary insect-cell based manufacturing platform, while BMS leads development and regulatory activities across all programs and is responsible for all research and development costs. BMS will be solely responsible for commercialization of all products from the collaboration.
Glybera for Lipoprotein Lipase Deficiency
Glybera is a gene therapy that is designed to restore the LPL enzyme activity required to enable the processing, or clearance, of fat-carrying chylomicron particles formed in the intestine after a fat-containing meal. The product consists of an engineered copy of the human LPL gene packaged with a tissue-specific promoter in a non-replicating AAV1 vector, which has a particular affinity for muscle cells. In order to improve activity, the Company uses a naturally occurring variant of the LPL gene that has higher enzyme activity than the normal version of the gene that encodes the protein. It produces Glybera using its insect cell-based manufacturing process. In the European Union, the Company has been granted orphan drug exclusivity for Glybera for treatment of LPLD until October 2022. The first commercial patient in Europe was treated with Glybera in September 2015.
Other Early-Stage Research
The Company is also engaged in the research of several other gene therapy candidates targeting rare and orphan diseases. It focuses on genetic diseases affecting the liver, including hemophilia A, as well as various central nervous system (CNS) disorders.
uniQure seeks to develop next-generation vectors with increased potency to target liver indications in which high relative percentage increases in the secretion of a protein above the disease state would be required for therapeutic benefit. These next-generation vectors may be used in the development of a gene therapy for hemophilia A as well as other therapeutic indications. In January 2014, the Company has entered into a collaboration and license agreement with 4D Molecular Therapeutics, LLC for the discovery and optimization of next-generation AAV vectors targeting the liver and the brain.
The Company competes with AGTC, Abeona Therapeutics, Adverum Biotechnologies, Asklepios BioPharmaceutical, Audentes Therapeutics, Avalanche Biotech, AveXis, Bayer, BioMarin, Bioveratiy, bluebird bio, Dimension Therapeutics, Errant Gene Therapeutics, Expression Therapeutics, Freeline Therapeutics, Genethon, Genzyme, GlaxoSmithKline, Homology Medicines, Lysogene, Megenics, Milo Therapeutics, Nightstarx, Pfizer, REGENXBIO, Renova Therapeutics, Retrosense Therapeutics, Sangamo BioSciences, Shire, Solid Biosciences, Spark Therapeutics, Takara, Voyager, Amgen, Bayer, Biogen, BioMarin, Genzyme, Novartis, Novo Nordisk, Pfizer, and Shire.