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ProQR Therapeutics 2019
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Moeten we nog minimaal 4 maanden geduld hebben. Op facebook trouwens de sheets van Dr Girch bekeken. Het viel me op dat Usher van te voren wel gevalideerd is dmv een diermodel maar LCA10 niet. Omdat er verder veel overeenkomsten zijn , ga ik er van uit dat het met Usher ook wel goed komt
Wanneer verwachten we resultaten ?
4 maanden is kort voor een lt-er, belangrijker is dat het goede resultaten zijn en de recente toekenning van speciale status door de ema stemt me zeer positief.
Novartis wil uitbreiden in de RNA en oogheelkunde, ik zou zeggen bel even met Daniel de Boer:www.spglobal.com/marketintelligence/e...
Tom3 schreef op 10 augustus 2019 10:30 :
Novartis wil uitbreiden in de RNA en oogheelkunde, ik zou zeggen bel even met Daniel de Boer:
www.spglobal.com/marketintelligence/e... Misschien heeft de Boer al een stiekeme achterkamer deal met Galapagos ;-)
4finance schreef op 10 augustus 2019 12:08 :
[...]
Misschien heeft de Boer al een stiekeme achterkamer deal met Galapagos ;-)
Bart Filius is die achterkamer aan het klaarzetten :-)
www.americanbankingnews.com/2019/08/1... Er zit nog aardig wat opwaarts potentieel in.
moi!! schreef op 11 augustus 2019 19:43 :
www.americanbankingnews.com/2019/08/1... Er zit nog aardig wat opwaarts potentieel in.
Voor wat het waard is natuurlijk (ik hecht weinig aan analisten), maar wel erg positief, zes buy adviezen, gemiddeld koersdoel $ 29,83. Dat de koers omlaag is doorgeslagen past wel in het huidige beleggingsrealiteit, weinig hebben geduld en een lange termijn visie - laat staan in speculatieve en volatiele branche als biotech.
ir.proqr.com/news-releases/news-relea... ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP) 12 August 2019 at 7:00 AM EDT LEIDEN, Netherlands and CAMBRIDGE, Mass., Aug. 12, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-1123. ProQR plans to start enrolling patients in a Phase 1/2 trial for QR-1123 in 2019. QR-1123 is a first-in-class investigational oligonucleotide designed to address the underlying cause of the vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene. P23H is the most prevalent mutation associated with adRP in the U.S. This disease causes progressive vision loss in approximately 2,500 patients in the United States, leading to blindness in mid-adulthood. There are no approved therapies for adRP and QR-1123 is the first investigational medicine to be developed for patients that suffer from this disease. “We are pleased to have an open IND for QR-1123, based on which we will be advancing our next inherited retinal disease program into the clinic this year,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “This represents our fifth IND in less than five years and our third clinical program for severe genetic eye diseases. With a strong in vitro and in vivo proof-of-concept, we are excited about the potential of this medicine to make a positive impact on the lives of patients with adRP.” About QR-1123 QR-1123 is a first-in-class investigational antisense oligonucleotide that was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutant mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018. About the Phase 1/2 trial for adRP PQ-1123-001, is a first-in-human study that will initially include up to 12 adults with adRP due to the P23H mutation in in the rhodopsin (RHO) gene. The trial will include a single-dose escalation (open label) arm and a multiple-dose (double-masked) arm in which a single intravitreal injection of QR-1123 or sham procedure will be given in one eye. The objectives of the trial will include evaluation of safety, tolerability, pharmacokinetics and efficacy, as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity (BCVA), visual field and optical coherence tomography (OCT). Changes in quality of life as reported by trial subjects in patient reported outcomes, or PROs, will also be evaluated. Patients completing this trial will be able to participate in an extension study if eligible. The trial is designed to be conducted at expert sites in North America and is expected to start in 2019. About adRP Autosomal dominant retinitis pigmentosa, or adRP, is a severe and rare genetic disease that causes progressive reduction in night and peripheral vision during childhood and frequently leads to blindness in mid adulthood. In the United States, the most prevalent mutation associated with adRP is the P23H point mutation (also known as the c.68C>A mutation) in the rhodopsin (RHO) gene and affects approximately 2,500 people. This gain of function mutation causes misfolding of the rhodopsin protein that becomes toxic to the photoreceptor cells in the retina. Over time the cells die and vision is progressively lost. There are currently no therapies approved or in clinical development for P23H adRP. A natural history study in patients with P23H adRP has been conducted.
Mizuho Lowers Nektar Therapeutics (NASDAQ:NKTR) Price Target to $79.00 Posted by Rob Hiaasen on Aug 12th, 2019 Nektar Therapeutics logoNektar Therapeutics (NASDAQ:NKTR) had its price objective lowered by research analysts at Mizuho from $81.00 to $79.00 in a research note issued to investors on Friday, July 26th, BenzingaRatingsTable reports. The firm currently has a “buy” rating on the biopharmaceutical company’s stock. Mizuho’s price objective suggests a potential upside of 277.63% from the stock’s current price. Several other equities research analysts have also recently weighed in on the stock. BidaskClub cut shares of Nektar Therapeutics from a “sell” rating to a “strong sell” rating in a research report on Tuesday, July 16th. William Blair restated a “buy” rating on shares of Nektar Therapeutics in a research report on Friday, June 14th. Zacks Investment Research upgraded shares of Nektar Therapeutics from a “sell” rating to a “hold” rating in a research report on Tuesday, May 14th. Cowen set a $82.00 price objective on shares of Nektar Therapeutics and gave the company a “buy” rating in a research report on Thursday, June 13th. Finally, ValuEngine upgraded shares of Nektar Therapeutics from a “hold” rating to a “buy” rating in a research report on Monday, June 3rd. One equities research analyst has rated the stock with a sell rating, seven have issued a hold rating, six have given a buy rating and one has issued a strong buy rating to the company. Nektar Therapeutics currently has a consensus rating of “Hold” and a consensus price target of $50.33. Get Nektar Therapeutics alerts: Shares of Nektar Therapeutics stock opened at $20.92 on Friday. The firm has a market cap of $5.15 billion, a PE ratio of 5.53 and a beta of 2.67. The company has a quick ratio of 14.83, a current ratio of 14.94 and a debt-to-equity ratio of 0.21. The stock’s 50-day moving average price is $32.57. Nektar Therapeutics has a 12-month low of $17.50 and a 12-month high of $69.76. Nektar Therapeutics (NASDAQ:NKTR) last announced its earnings results on Thursday, August 8th. The biopharmaceutical company reported ($0.63) earnings per share (EPS) for the quarter, topping analysts’ consensus estimates of ($0.80) by $0.17. The business had revenue of $23.32 million during the quarter, compared to analysts’ expectations of $26.65 million. Nektar Therapeutics had a negative net margin of 354.90% and a negative return on equity of 24.67%. The firm’s revenue for the quarter was down 97.9% on a year-over-year basis. During the same quarter last year, the company posted $5.33 EPS. On average, equities analysts predict that Nektar Therapeutics will post -3.13 EPS for the current year.
In other news, SVP Stephen K. Doberstein sold 3,461 shares of Nektar Therapeutics stock in a transaction that occurred on Thursday, May 16th. The shares were sold at an average price of $31.37, for a total transaction of $108,571.57. Following the completion of the sale, the senior vice president now owns 95,913 shares in the company, valued at approximately $3,008,790.81. The sale was disclosed in a filing with the SEC, which can be accessed through this link. Also, CFO Gil M. Labrucherie sold 3,767 shares of Nektar Therapeutics stock in a transaction that occurred on Thursday, May 16th. The stock was sold at an average price of $31.37, for a total transaction of $118,170.79. The disclosure for this sale can be found here. In the last quarter, insiders have sold 253,283 shares of company stock valued at $8,356,254. 4.02% of the stock is owned by corporate insiders. Large investors have recently modified their holdings of the company. Raymond James Financial Services Advisors Inc. grew its stake in Nektar Therapeutics by 54.9% in the 4th quarter. Raymond James Financial Services Advisors Inc. now owns 6,761 shares of the biopharmaceutical company’s stock worth $222,000 after acquiring an additional 2,395 shares during the period. Advisor Group Inc. grew its stake in Nektar Therapeutics by 21.4% in the 4th quarter. Advisor Group Inc. now owns 6,638 shares of the biopharmaceutical company’s stock worth $218,000 after acquiring an additional 1,168 shares during the period. Dimensional Fund Advisors LP grew its stake in Nektar Therapeutics by 82.0% in the 4th quarter. Dimensional Fund Advisors LP now owns 408,526 shares of the biopharmaceutical company’s stock worth $13,433,000 after acquiring an additional 184,055 shares during the period. Principal Financial Group Inc. grew its stake in shares of Nektar Therapeutics by 0.9% during the 4th quarter. Principal Financial Group Inc. now owns 288,227 shares of the biopharmaceutical company’s stock valued at $9,474,000 after buying an additional 2,637 shares during the period. Finally, Metropolitan Life Insurance Co. NY grew its stake in shares of Nektar Therapeutics by 1.2% during the 4th quarter. Metropolitan Life Insurance Co. NY now owns 32,910 shares of the biopharmaceutical company’s stock valued at $1,082,000 after buying an additional 401 shares during the period. Institutional investors own 92.09% of the company’s stock. About Nektar Therapeutics Nektar Therapeutics, a biopharmaceutical company, develops drug candidates for cancer, auto-immune disease, and chronic pain in the United States. The company develops NKTR-181, an orally-available mu-opioid analgesic molecule, which is in Phase III clinical trial for moderate to severe chronic pain; ONZEALD, a topoisomerase I inhibitor that is in Phase III clinical trial for advanced metastatic breast cancer in patients with brain metastases; and NKTR-214, a CD122-preferential interleukin-2 (IL-2) pathway agonist, which is in Phase I to treat immuno-oncology.
Wat denk je zelf, dzr? Wordt het wat?
nb schreef op 12 augustus 2019 17:29 :
Wat denk je zelf, dzr?
Wordt het wat?
Nektar is in ieder geval goedkoop.
DeZwarteRidder schreef op 12 augustus 2019 18:05 :
[...]
Nektar is in ieder geval goedkoop.
Bij Nektar is alle vertrouwen weg nadat gepresenteerde data veel slechter was dan verwacht. De CEO wist dat kennelijk al. Die heeft recentelijk een enorm pak aandelen verkocht. ... Nektar zit in een niet te stoppen glijvlucht....
@Tom3; nog bijgestelde meningen mbt Affimed? aandeel lijkt vnl wat doelloos op en neer te bewegen...
Nektar Therapeutics Drops Sharply for Second Day After Admitting Batch Issues Nektar dropped 40% on Friday after announcing it had manufacturing issues in two batches of experimental cancer drugs. Tony Owusu Aug 12, 2019 1:30 PM EDT Nektar Therapeutics Pharmaceutical company Shares of Nektar Therapeutics (NKTR - Get Report) were down nearly 10% Monday, following on from a 40% decline on Friday as investors digested the company's announcement that its experimental cancer drug study ran into some issues. Last week, the company announced that it had manufacturing issues in two batches of experimental cancer drugs that the company gave to patients in a trial. Those two batches resulted in treatment responses that weren't as optimistic as expected, leading analysts at J.P. Morgan, Jefferies and Mizuho Securities to downgrade the stock to hold. "Nektar is making good progress advancing our multiple programs in immuno-oncology, immunology and pain," said Howard W. Robin, president and CEO of Nektar. "With our partner Bristol-Myers Squibb, although we've experienced some delays, we are working to finalize the development program for bempegaldesleukin in combination with nivolumab in a number of tumor types and which are designed to support registration for this unique I-O doublet." Separately, the company recently reported a quarterly loss of 63 cents a share on revenue of $23.3 million. Analysts were expecting a loss of 80 cents a share on revenue of $26.6 million. Nektar shares fell 9.7% to $18.90 on Monday.
Zullen we dit draadje voor PRQR houden? Misschien een apart draadje voor liefhebbers van andere bedrijven? Ondertussen gaan de fundamentele ontwikkelingen bij PRQR door. Het portfolio groeit en daarmee wordt het (toekomstige) risico gespreid. Goed teken dat er weer een Clinal Trial bij komt, betekent dat er geen extra focus nodig is op de anderen, die lopen blijkbaar naar wens. Sterker, kan me voorstellen dat ervaringen bij de ene oogaandoening helpen bij de oplossing voor andere oogaandoeningen.
ivet schreef op 13 augustus 2019 00:36 :
@Tom3; nog bijgestelde meningen mbt Affimed? aandeel lijkt vnl wat doelloos op en neer te bewegen...
Nee hoor, ik wacht op aankondiging mbt een deal met een nieuwe partner ( anti PD1 leverancier). Tot die tijd is het een speelbal van kortebaan speculanten.
Proqr heeft er precies ook last van vandaag.
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