[verwijderd] 5 september 2018 12:06 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 quote:Frenky_Tornado schreef op 5 september 2018 11:30:De koers zou hierop moeten stijgen, maar ja het blijft de beurs he.nuchtere opmerking Frenky; je hebt logica en beurslogica...2 totaal verschillende uitgangspunten.Neemt niet weg dat ik bij een daling ga bijkopen...dus kopers zijn er sowieso."That's one small step for man, one giant leap for mankind"... haha Aanbevelingen 0 ” Quote Reageren Niet oké
Tom3 5 september 2018 12:44 auteur info Tom3 Lid sinds: 25 jan 2015 Laatste bezoek: 23 apr 2024 Aanbevelingen Ontvangen: 2788 Gegeven: 594 Aantal posts: 10.995 Op naar de $ 10 zou ik zeggen! Dit is het bewijs dat het werkt. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 14:56 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Net uitgekomen rapport JMR:ProQR Therapeutics N.V. Dramatic Gain of Vision for LCA10 Patients in First QR-110 Study PRQR: $7.95 | Market Cap: $254M Market Outperform | Price Target: $25.00Liisa A. Bayko | +1 312 768-1785 | lbayko@jmpsecurities.com ? Proof-of-concept study in LCA10 hits on multiple efficacy measures enabling Phase 3 to start in 1H19; we reiterate our Market Outperform rating and raise our risk-adjusted, DCF-derived price target on ProQR Therapeutics to $25 from $20. ? We are excited by the first look at data of QR-110 in patients with LCA10 – a rare retinal dystrophy leading to childhood blindness. In particular, we are impressed with the concordance of the data showing a rapid and sustained benefit on every metric of vision assessed, especially the change in visual acuity, which we viewed as a high hurdle (see our preview here). ? The logMAR scale is used to measure vision improvement (negative values) and vision loss (positive values) on the commonly used ETDRS letter chart where the clinically meaningful threshold is 0.3 logMAR (3 lines/15 letters). This is important as it is a commonly used regulatory metric for efficacy, and in this study, over 60% of patients responded with a mean response of 0.67 logMAR, well above the clinically meaningful threshold. Spark Therapeutics (ONCE, NC) used a novel endpoint for Luxturna approval utilizing a mobility test and the FDA determined two levels to be clinically relevant on this instrument. Here, QR-110 also showed positive data (Figure 1). ? Based on the data, the FDA has agreed to the Phase 2/3 ILLUMINATE study as a next step that should begin in 1H19, followed closely by a study in children under age six years, which could enable even greater therapeutic benefit. We think the quick expansion into the younger population underscores the clean safety profile of QR-110. ? We see LCA10 as a >$700M opportunity, which may or may not be shared by the only other competitor we are aware of: Editas Medicines’ (EDIT, CIT) gene editing approach. ? Following these data, we raise our probability of success (POS) for QR-110 to 60% from 20% and our POS for QR-421a in Usher’s syndrome slightly to 20% from 10%, as we see some read-through from the optic cup model from this study. Based on the accelerated Phase 3 start, we now model a U.S. launch of QR-110 in 2021 (2022 previously). We also remove PRQR’s cystic fibrosis program, eluforsen, from our model. QR-110 now accounts for ~$13/share of our $25 target ($5/share of our previous $20 target). ? Management is hosting a conference call this morning at 8:15 a.m. ET where we will look for additional color on the data. We are hosting a call with ophthalmic expert Dr. Mark Pennesi at 10:00 a.m. ET to discuss LCA10 and the QR-110 data. Please contact your JMP Securities sales representative for dial-in information. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 14:57 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Ik heb ook het volledige rapport voor je als pdf.Het ziet er dus echt goed uit (letterlijk en figuurlijk) Aanbevelingen 0 ” Quote Reageren Niet oké
Tom3 5 september 2018 15:11 auteur info Tom3 Lid sinds: 25 jan 2015 Laatste bezoek: 23 apr 2024 Aanbevelingen Ontvangen: 2788 Gegeven: 594 Aantal posts: 10.995 $13 voorbeurs. Gaat dus steeds beter. Was het koersdoel van Wainwright niet $ 20? ARWR , dat ik gisteren gekocht heb, noteert ook tegen de bovenkant van de taxaties. Aanbevelingen 0 ” Quote Reageren Niet oké
Ontop1 5 september 2018 15:15 auteur info Ontop1 Lid sinds: 11 nov 2014 Laatste bezoek: 23 apr 2024 Aanbevelingen Ontvangen: 117 Gegeven: 45 Aantal posts: 2.464 quote:Tom3 schreef op 5 september 2018 15:11:$13 voorbeurs. Gaat dus steeds beter. Was het koersdoel van Wainwright niet $ 20? ARWR , dat ik gisteren gekocht heb, noteert ook tegen de bovenkant van de taxaties.Mooie tijden op het moment Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 15:23 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 With promising data, ProQR Therapeutics' RNA-based eye drug heads to phase 3www.fiercebiotech.com/biotech/promisi... Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:45 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Nog eentje om het te bevestigen:FIRST TAKE ProQR Therapeutics N.V.PRQR: Price: $7.95; Market Cap (M): $254Rating: Buy; Price Target: $20.00Andrew S. FeinAlicia Yin, Ph.D.Li Wang WatsekWir Lieben QR-110 Data in Leber's; Reit Buy and $20 PTOur thoughts around the positive QR-110 Phase 1/2 interim data. We believe that the positive interim data from QR-110 Phase 1/2 trial in Leber congenital amaurosis 10 (LCA10) patients is highly informative from the following aspects: (1) the data demonstrated not only favorable safety but also rapid and sustained efficacy of QR-110 in treating LCA10 patients. Based on our conversations with KOLs, this data has exceeded the KOL expectations (i.e. safety profile without much efficacy signal), and should lower both the clinical and regulatory risk moving forward, in our view (discussed below); (2) provided first in human proof of concept data of antisense RNA intravitreal injection as a treatment modality for LCA10 patients, which may be superior to other approaches currently being tested, including CRISPR-mediated gene editing and AAV-based delivery methods (discussed below); and (3) given the much shorter than expected time to observe clinical benefits (patients have only been treated for 3~6 months), and the planned initiation of a Phase 2/3 study in 1H19 prior to the completion of the ongoing Phase 1/2 trial in 3Q19, we expect the timeline for the development of QR-110 may be significantly shortened.Early and sustained efficacy signals exceed our expectations. According to several KOLs, 6-month is a very short time frame for demonstrating functional changes in inherited retinal diseases (usually measured in terms of years). Thus, our initial expectation going into the data readout was focused on the safety profile with the assumption that any efficacy signal would be small and simply a bonus at this early time point. We believe that the efficacy signals across multiple measurements in the interim data, albeit in relatively few patients, suggest a real clinical benefit, especially given how severe these patients are, and could serve to lower the clinical risk in a pivotal trial: (1) the rapid onset of efficacy for QR-110, in our view, is consistent with QR-110’s mechanism of action (MOA) that allows for restoration of functional CEP290 protein levels in a short-term; (2) consistent improvement observed in four different outcome measures including functional, physiological and structural measurements, gives us confidence that the signal is real and likely to be reproducible; and (3) improvement in the median value, as well as the mean value of endpoints, suggest that the benefit observed is not driven by a single hyper-responder. Moreover, we believe these data compare favorably to Luxturna’s (Spark Therapeutics; ONCE; not rated) clinical outcomes, especially considering LCA 10 is often a more severe disease than LCA 2: (1) the mobility test developed by Spark is viewed favorably by the FDA, as it is a functional measurement and directly relates to patients’ quality of life (QoL). Although there is no single mobility test favored by the FDA, the feedback from the agency according to both KOLs and management is that a series of mobility tests with a broader dynamic range to better accommodate the range of disease severity in patients would be valuable. To that end, ProQR designed and developed multiple tests with a large dynamic range with scores from 0-20, that vary in difficulty of mobility, degree of obstacles, light sensitivity, and visual light contrast. Per management, >2 levels of improvement should be considered clinically meaningful, and QR-110 achieved a mean of 2.6 levels of improvement (57% patients improved >2 levels) which was maintained for more than 6 months; (2) most of the patients with LCA10 have very low vision at baseline and it is often difficult to show improvement on visual acuity. With LCA2, Luxturna failed to show significant improvement in best correlated visual acuity (BCVA) (-0.16 LogMAR, p=0.17). In comparison, QR-110 showed significant and clinically meaningful improvement (>=-0.3 LogMAR) in BCVA (-0.67 LogMAR, p=0.01; 62.5% patients improved >0.3 LogMAR), which lasted for at least 6 months. Recall that an improvement of -0.3 roughly translates to an improvement in 3 lines on an eye chart, thus the improvement of -0.67 is at least an improvement of 6 lines. We view this outcome as especially significant, since: (1) no change was expected this early in treatment; and (2) most of these patients barely have the ability to sense light penetrance yet alone to be able to read letters on a chart. If the results hold in a larger trial, we believe it would be highly compelling in the eyes of the FDA and could result in rapid uptake by the provider community.; (3) improvement in FST and OCI were comparable to those reported in Luxturna’s Phase 1 trial; (4) EZ line restoration detected by OCT is consistent with the proposed MOA that CEP290 restoration helps re-grow the outer segment of photoreceptors. We do note, however, that this improvement in OCT was only observed in 1 patient, although this is a significant outcome (regrowth of outer segments, does not occur spontaneously) it is important to state that the other patients tested did not have sufficient resolution to accurately distinguish the EZ line; and (5) no SAEs or patient discontinuations have been reported, and the independent Data and Safety Monitoring Committee (DSMC) agreed that there were no safety concerns. Moreover, we point out that the approval of Luxturna demonstrated that due to the grave unmet need, a modest improvement may be sufficient to score an FDA nod. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:46 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 What to look for in upcoming studies? In addition to completing the ongoing Phase1/2 trial with topline readout (12-month data) in 3Q19, ProQR expects to initiate a Phase 2/3 registration trial in 1H19. Moving forward, we will keep a keen eye on: (1) potential updated protocols on dose level and frequency pursuant to ProQR’s discussion with the FDA. Of note, in the current Phase 1/2 trial, only the lower two of the three proposed dose levels were tested, which produced similar outcomes. Meanwhile, we believe a lower dose frequency may further decrease the likelihood of complications related to intravitreal injections and facilitate patient adoption; (2) whether CEP290 homozygous patients (p.Cys998X mutation, also known as c.2991+1655A>G mutation, estimated at 10% ~ 20% of total) respond better than heterozygous patients. The current data from 10 patients are all compound heterozygous patients. QR-110 is mutation specific and can therefore only address the allele with p.Cys998X mutation, thus, it is hypothesized that patients with homozygous p.Cys998X mutation may benefit more from this therapy; and (3) whether younger patients respond better. LCA10 is a genetic disorder and disease onset is at birth. It is also a progressive disease since once the cells die, gene therapies may not be able to reverse it. As a result, it is generally believed that earlier treatment is better since younger patients may have more intact retinal cells, and earlier treatment may allow prevention of vision loss. In fact, Luxturna data showed that the best responses occurred in younger patients with LCA 2. We note that in Phase 1/2 interim data presented by ProQR, all pediatric patients are responders (youngest being 8 years old), which may suggest a better response in younger patients. That being said, according to management the highest responder on the visual acuity scale was an older adult patient that had very severe disease. Thus, suggesting that QR-110 treatment could be effective in a broad spectrum of patients irrespective of age or disease severity, providing that there are some functional cells remaining to restore. Of note, in parallel to the proposed Phase 2/3 pivotal study, ProQR plans to initiate a pediatric trial in patients <6 years old. We believe that QR-110 may be advantageous in younger pediatric patients based on: (1) according to our KOLs, physicians consider intravitreal injection as a relatively safe procedure and are comfortable with performing it in children as young as 12 months old; (2) the favorable safety profile demonstrated by QR-110 should remove physicians’ concerns around applying it to younger patients; and (3) we are further encouraged by the broad label (>12 months old, viable retinal cells and RPE65 mutations) obtained from the FDA by Luxturna, despite its invasive subretinal injection procedure and the fact that Phase 3 Luxturna trial enrolled LCA2 patients of 4~44 years old. Thus, we believe the FDA’s view on the risk/reward balance bodes well for a broad label for a potentially more efficacious and less invasive therapy (QR-110) in a more severe disease (LCA10).Antisense RNA intravitreal injection may be superior to CRISPR delivered by AAV in treating LCA10. QR-110 phase 1/2 data has provided important proof of concept validations for antisense RNA intravitreal injection as a noval modality for LCA10 patients, which we believe may be more advantageous compared to CRISPR-mediated gene editing and other AAV-based delivery methods (Editas Medicine; EDIT; not rated): (1) subretinal injection is considered a surgical approach, is invasive and has a greater risk profile since lifting the retina likely damages photoreceptor cells and may result in reduced vision if it has to be repeated. In contrast, the intravitreal injection as a treatment procedure is relatively simple and has become a commonly used procedure since the introduction of the combination treatment of verteporfin and intravitreal triamcinolone for neovascular age-related macular degeneration (AMD). The downside of the RNA approach vs. the gene therapy approach is that the injections have to occur multiple times a year as opposed to a one-time treatment. The likelihood of complications associated with repeated intravitreal injections may arise despite being a relatively safe procedure. The risk for endophthalmitis is up to 0.16% per injection typically due to bacterial infection, for traumatic cataract up to 0.07% per injection and for retinal detachment up to 0.17% per injection. According to KOLs, for a severe disease like LCA10, intravitreal injections every 3 months could be considered and every 6 months should be highly acceptable; (2) moreover, pan-retinal treatment by intravitreal injection may demonstrate more benefits than a limited treatment by AAV-mediated gene therapy (treating <20% of retinal surface) as the latter only affects a small region surrounding the injection site; (3) CRISPR may have off-target effects and may constantly cut the genome in an uncontrollable fashion; (4) Editas’s approach is to delete the mutated part of the intron by CRISPR, but may result in abnormal splicing that fails to produce sufficient levels of CEP290 mRNA, since the method depends on non-homologous end joining (NHEJ). The uncertain DNA sequence resulting from NHEJ may also raise safety concerns; (5) finally we believe that preclinical testing and manufacturing processes are both much more straightforward for RNAi therapies than AAV-based gene therapies. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:46 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Valuation and risks. Our price target of $20/share is based on an equally-weighted composite of: (a) $18.0/share, as a 25x multiple of taxed and diluted FY28 EPS of $9.92 discounted back to FY18 at 30% (in line with the expected PE multiple and discount rate of an early development-stage biotechnology company); and (b) an NPV of $21.9/ share (discounted cash flow analysis using a 18% discount rate and 2.0% growth rate, in line with the expected discount and growth parameters of an early development-stage biotechnology company). Risks to our investment thesis and target price include: (1) failure in clinical studies; (2) failure to secure regulatory approval; and (3) smaller than anticipated commercial opportunity due to market size, competition, and pricing.Andrew S. Fein 212-356-0546 afein@hcwresearch.comAlicia Yin, Ph.D. 646-975-6983 ayin@hcwresearch.comLi Wang Watsek 212-356-0513 lwatsek@hcwresearch.com Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:48 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Het complete rapport is te vinden onder deze link:hcwco.bluematrix.com/sellside/EmailDo... Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:52 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 En nog een bevestiging is te vinden op:chardan.bluematrix.com/sellside/Email... Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:53 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Uit bovenstaand rapport van Chardan:PRQR: Company Reports Positive Interim Data From LCA10 Clinical StudyProQR has reported positive interim data from its Phase 1/2 clinical trial of QR-110 in patients with Leber’s Congenital Amaurosis 10 (LCA 10): The data was presented today at the Retinal Degeneration 2018 meeting in Killarney, Ireland. In the study QR-110 demonstrated rapid and sustained improvement in vision in patients with LCA10, as measured by visual acuity and the mobility course performance, as well as being well-tolerated with no serious adverse events recorded. At three months of treatment, the mean improvement in best corrected visual acuity was -0.67 LogMAR (with a standard error of mean = 0.32) with 62.5% of subjects showing an improvement of greater than -0.3 LogMAR from baseline, which is considered clinically meaningful. The mean change in the contralateral eye was 0.02 LogMAR (SEM 0.05). At three months of treatment, the mean improvement in navigating the a mobility course was 2.6 levels (SEM 1.2) with 57.1% of subjects improving by more than 2.0 levels, which is regarded as clinically meaningful. The mean change in the contralateral eye was 1.36 (SEM 1.04). This open-label Phase 1/2 clinical trial enrolled 11 patients, 10 who received treatment, in their worst eye, at three sites in the U.S. and Belgium. Patients are scheduled to receive four doses of QR-110 via intravitreal injections, one loading dose, followed by three maintenance doses administered once every three months for 1 year. Three adults and two pediatric patients received a 160 µg loading dose and 80 µg maintenance doses, while three adults and two pediatric patients received a 320 µg loading dose and 160 µg maintenance doses. Based on early efficacy observed in this open-label study, the originally planned interim analysis at six months treatment was accelerated to the point when eight patients had reached three months of treatment, and a planned third cohort of patients who would have received a 500 µg loading dose and 270 µg maintenance doses was not enrolled. For the 10 subjects dosed in the study, one subject has received all four doses and three have received three doses, representing a combined total of more than 1,500 treatment days. So far QR-110 was well tolerated with no serious adverse events related to treatment or procedure. All data and safety monitoring committee (DSMC) reviews were completed with no restrictions on further dose escalation or pediatric dosing. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:54 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Key Takeaways From Topline Data: As shown in Exhibits 1, patients exhibited improvements in BCVA in the treated eye vs the contralateral eye at three months. Further, as shown in Exhibit 2, the BCVA effect was maintained at 6 months. As shown in Exhibits 3 and 4, there was concordant improvement in all of the outcome measure in patients who demonstrated a response to QR-110.Twelve-month data from this study in expected to be reported around mid-2019. Based on these interim results, the Company now plans to advance QR-110 into a Phase 2/3 clinical study (called “ILLUMINATE”). Based on discussions with the FDA, the preliminary design for this study will be a double-blind, sham controlled study that will target enrollment of 30-40 patients with LCA10, which could be adaptively repowered. The primary endpoints in this trial are expected to include the mobility course and visual acuity, among others, at twelve months followup. The trial is expected to be conducted at centers in North America and select European countries. The trial is expected to commence enrollment in the first half of 2019. The Company believes that it is possible that this could serve as a single registration study that could support FDA approval. In parallel to this Phase 2/3 trial, the Company plans to start a separate trial in patients <6 years old. LCA 10 is a rare ophthalmic disease caused by a genetic defect in 20 or more associated genes. The most common mutation leads to significant decrease of active CEP290 protein in the photoreceptor cells in the retina in the eye. The CEP290 protein is involved in the formation and stability of the connecting cilium, a hair-like structure involved in the transport of proteins from the inner segment to the outer segment of photoreceptor cells. The absence of this essential protein causes loss of vision. There is currently no disease modifying therapy available on the market or being tested in clinical development for this specific subtype of the disease. QR-110 is a single stranded RNA oligonucleotide, administered by intravitreal injections, which binds to the pre-mRNA at the site of the p.Cys998X mutation, such that it is silenced during the splicing process, restoring the mature mRNA to wild type, resulting in production of normal CEP290 protein. The Company's QR-421a and QR-411 therapies target another rare ophthalmic disease, Usher syndrome: QR-421a is on track to commence evaluation in a Phase 1/2 safety and efficacy clinical trial at the end of 2018. Usher syndrome 2A is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene. Patients with Usher syndrome 2A generally progress to a stage in which they have very limited central and peripheral vision and moderate to severe deafness. There are no therapies approved or product candidates in clinical development that treat the vision loss associated with Usher syndrome 2A. The Company is developing QR-421a for the ophthalmic manifestation of Usher syndrome 2A due to exon 13 mutations and QR-411 for the ophthalmic manifestations of Usher syndrome 2A due to the PE40 mutation. Both product candidates are single-stranded oligonucleotides intended to be administered by intravitreal injections, which act to splice the pre-mRNA to form a mature mRNA does does not include the mutated exon, resulting in a functional usherin protein to restore vision. The Phase 1/2 trial will consist of a single-dose arm and a six-month adaptive multiple dose arm. Top-line data from the single-dose arm are expected in the first half of 2019 and from the adaptive multipledose arm later in 2019. In February 2018, the Company announced a partnership agreement with Foundation Fighting Blindness (FFB), under which FFB has agreed to provide milestone driven funding of $7.5 million to advance QR-421a into the clinic in exchange for receiving future milestone payments. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:56 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 We maintain our BUY rating and are increasing our 12-month price target to $21.00: We continue to believe that ProQR has the potential to become a significant competitor in the oligonucleotide therapeutic market. Based on the data for QR-110 reported today, we are increasing the probability of success in our model for this indication from 20% to 50%. We believe that this result also provides some positive read across to the Company's other programs targeting other rare eye diseases. Hence we are increasing the probability of success in our model for its QR-421a for Usher syndrome mutation from 15% to 20%. Additionally, the recent increase in the stock price, has reduce the amount of dilution associated with projected future capital raises in our model, positively impacting our DCF valuation. We believe that future progress updates from pipeline programs could serve as value creation events for the stock over the next 6-18 months. The Company is now expected to advance QR-110, used as a therapy for LCA10, into a Phase 2/3 clinical study during the first half of 2019, which could serve as a single registration trial for possible FDA approval of this indication. The Company is expected to report 12-month follow-up data from the on-going Phase 1/2 study around mid-2019. The Company's QR-313, targeting Dystrophic epidermolysis bullosa (DEB) has commence evaluation in a phase 1b study, which should produce interim results by the end of 2018, with additional data in 2019. The Company's QR-421a therapy targeting Usher's syndrome, is expected to reach the clinic by the end of this year, resulting in interim data in mid-2019, and additional data in the second half of 2019. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 20:58 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 En wat doet de koers? Nauwelijks omhoog na zoveel bevestigingen. $20 is de laagste target. Dan is er nog veel te verdienen guys. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 21:33 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 er komt meer...markt loopt vaak vooruit...maar gezien deze koersstijging gisteren altijd achter de feiten aan bij ProQR. Als de markt meer feeling had, dan stegen we misschien 10%...zoiets van: natuurlijk hadden we dit verwacht.De instituten begrepen het wel...die sprokkelden bij afgelopen weken/maanden...en Piet Particulier doet vaak te weinig moeite om potentieeltjes te onderzoeken...tja dividend bij olies zijn blijkbaar meer sexy? Aanbevelingen 1 ” Quote Reageren Niet oké
Hans Igor 5 september 2018 22:08 auteur info Hans Igor Lid sinds: 01 jan 2000 Laatste bezoek: 01 apr 2024 Aanbevelingen Ontvangen: 548 Gegeven: 868 Aantal posts: 1.594 Gelukkig nog wat mensen in mijn omgeving kunnen interesseren, (ook op GLPG-draadje) maar velen ook niet. Nu de koers 120% is gestegen krabt menigeen zich toch achter de oren. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 5 september 2018 22:11 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 ProQR Announces Proposed Underwritten Public Offering of Ordinary Sharesir.proqr.com/news-releases/news-relea... Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 6 september 2018 08:29 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 finance.yahoo.com/news/apos-why-proqr...Here's Why ProQR Therapeutics N.V. Is Skyrocketing TodayMotley Fool Brian Feroldi, The Motley Fool,Motley Fool•September 5, 2018What happenedAfter reporting data from a phase 1/2 clinical trial, shares of ProQR Therapeutics N.V. (NASDAQ: PRQR), a clinical-stage biotech focused on RNA medicines that treat rare genetic diseases, rose 70% as of 12:13 p.m. EDT on Wednesday.So whatProQR reported results from its phase 1/2 trial that is testing its compound called QR-110 as a hopeful treatment for LCA10, which is a genetic mutation that usually leads to childhood blindness.Here are the key takeaways from the study:Use of QR-110 led to improvements in vision of the majority of patients when measured using visual acuity and the mobility course performance tests. Specifically, about 60% of patients showed a clinically meaningful response to the drug after three months of treatment. A secondary study also showed that the efficacy effects of the drug persisted beyond three months. The drug was well-tolerated by study participants, and no serious adverse events were reported.A pivotal phase 2/3 trial is set to start in the first half of 2019.Commenting on the study results, Dr. David Rodman, an executive vice president of R&D at ProQR, stated:We observed a clinically meaningful improvement in vision in the treated eye as measured by both mechanistic and potential registration endpoints. Consistent with predictions based on our patient derived optic-cup models, improvement in visual function was observed as early as two months after treatment and was maximal and stable by three months and thereafter. Given the news, it isn't hard to figure out why shareholders are having a great day.Now whatToday's update should provide investors with increased confidence that QR-110 might be the real deal. However, it's also important to remember that great phase 1/2 clinical news doesn't always repeat itself in larger late-stage trials. Given the risk, I'm content to root for ProQR's success from the safety of the sidelines. Aanbevelingen 0 ” Quote Reageren Niet oké
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Beurscodes, betekenis en hulp bij zoeken Europa AEX Euronext Amsterdam BRU Euronext Brussels PSE Euronext Paris LIS Euronext Lissabon CHX CBOE Europe, grote(re) EU aandelen NAV Investment Funds (NAV) Noord-Amerika NYS New York Stock Exchange OTC CBOE BZX Exchange (US) TSE Toronto Stock Exchange Kunt u een instrument niet vinden? Zoek dan via de zogenaamde ISIN code. Elk instrument, aandeel etc. heeft een unieke code. Kies vervolgens - wanneer er meerdere resultaten zijn - de notering op de beurs van uw keuze. Waar vind ik die ISIN code? Google de naam van het instrument, aandeel etc. met de toevoeging 'ISIN'. Als zoeken op ISIN code geen resultaten oplevert hebben wij het instrument of aandeel niet in onze koersendatabase.