Beursonline.nl

flosz 21 september 2015 09:58

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@DrMedicalValue: @El_Sequenco thx, $QURE All: must read! Good overview. Nugget: data open possibility to develop an aditional FIFTEEN (yes 15) LS diseases.

twitter.com/drmedicalvalue/status/645...

flosz 21 september 2015 13:09

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oppenheimer $qure Raising PT to $50, Quick Take: AMT-110 a Meaningful Treatment for Sanfilippo B SUMMARY (cont) t.co/EgVqM7KiOt
twitter.com/el_sequenco/status/645914...

Bijlage:

flosz 24 september 2015 15:32

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@TipRanks: Analysts Optimistic on $QURE Following Latest Clinical Trial Data #biotech #stocks #NASDAQ blog.tipranks.com/2015/09/analysts-op...
twitter.com/tipranks/status/646948716...

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[verwijderd] 10 november 2015 22:05

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weet niet of dit al ergens geplaatst was, maar beter dubbel dan niet;

www.traders-choice.com/pdf/?s=QURE

finance.yahoo.com/news/technical-cove...

Highlights from today's reports include:

On Tuesday, November 03, 2015, the NASDAQ Composite ended at 5,145.13, up 0.35%, the Dow Jones Industrial Average edged 0.50% higher, to finish the day at 17,918.15, and the S&P 500 closed at 2,109.79, up 0.27%.

- Uniqure NV's stock advanced 1.33% to close Tuesday's session at USD 19.03. The share price vacillated between USD 18.29 and USD 19.23. The stock recorded a trading volume of 0.23 million shares, which was below its 50-day daily average volume of 0.35 million shares and below its 52-week average volume of 0.26 million shares. Over the last three days Uniqure NV's shares have advanced 6.55% and in the past one week it has moved up 3.14%. However, in the last six months, the stock has lost 22.96% but year to date, the shares have picked up 28.49%. Further, the stock is trading below its 50-day and 200-day moving average of USD 22.88 and USD 25.29, respectively. Additionally, the company has market capitalization of USD 432.2 million. Uniqure NV is a pharmaceutical company based in the Netherlands. It is primarily engaged in the field of gene therapy for orphan diseases.

---dat laatste schetst misschien de onderwaardering; 'orphan diseases'

Noem Congestive Heart Failure maar 'orphan'... misschien zit daar de clou voor een fors hogere waardering: een lucratieve samenwerking op gentherapy-gebied met blockbuster-potentie dat ook duidelijk in de media wordt vermeld!

flosz 1 december 2015 09:58

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COWEN $QURE Strong Reception of Ph1/2 MPS3b at?ICSS; Competitive Landscape Mature As?Well?QURE Ph1/2 12 mo. MPS3b AMT-110 data was presented again at ICSS Fri. and very?well received by MPS3 KOLs. The trial PI noted 18 more mos. of follow-up is needed?to establish strong evidence of benefit, due to the poor natural history data, but noted?the 12-mo. data to date is highly encouraging. 18-mo followup data is due mid-2016.?QURE’s next catalyst is first Ph1/2 hemophilia B data due in Dec.?AMT-110 MPS3b Very Well Received at ICSS, But More Followup Needed?QURE Ph1/2 12-mo. AMT-110 MPS3b data was presented again at the 2nd?International Conference on Sanfilippo Syndrome and Related Lysosomal Storage?Diseases (Geneva) meeting Friday. Data was very well received by MPS3 KOLs and?disease stakeholders. The trial PI noted 18 more mos. of follow-up is needed to?establish strong evidence of benefit, due to the poor natural history data, but noted?the 12-mo. data to date is highly encouraging. 18-mo follow-up data is due mid-2016.?QURE’s next catalyst is first Ph1/2 hemophila B data due in Dec.?QURE Ph1/2 MPS3b (San Fillipo B) AMT-110 12-mo data very well received at?IASFC, but more follow-up needed.?¦ QURE Ph1/2 MPS3b (San Fillipo B) AMT-110 12 mo data was presented again at?the ICSS meeting this past Friday (Geneva). MPS3b is a genetic defect of GAG?(glycosaminoglycans) breakdown caused by a mutation in the NaGlu enzyme. It?is characterized by only mild physiological abnormalities but profound cognitive?impairment and severe behavioral disturbances before progressing to catatonia and?early death by 20 years of age. QURE’s AMT-110 is an AAV5-NAGLU gene therapy?that is delivered through intracerebral injection. Data was very well received by?MPS3 KOLs and there was significant enthusiasm for the program. The trial PI did?note, however, 18 more mos of treatment experience is needed to establish strong?evidence of benefit. The PI and other MPS3 KOLs at the conference noted the 12-?mo. data to date (see our note here) is highly encouraging due to concordance of?biomarker and clinical neurocognitive development endpoints. CSF GAG levels in?patients did not decline, although the trial PI noted in dog experiments, treatment?resulted in higher CSF NAGLU levels, lower tissue GAG levels, but also no decline?in CSF GAG. This is hypothesized to be a function of continued GAG production in?the meninges. QURE and KOLs have noted the upcoming 18-mo followup data (due?around mid-2016) will be even more meaningful than the data in hand. Additional?CSF Naglu, CSF GAG, MRI and neurocognitive measure data will be collected then.?Clinical Endpoints: The current rate-limiting step in MPS3 pivotal trials.?¦ One clear takeaway from the conference is that there is no clear universally?accepted clinical endpoint or set of endpoints for measuring either cognitive or?behavioral defects in MPS3. The most impressive data generated in the QURE trial?psychometric social interactions. There appeared to be generally little familiarity?with the PEP3 scale, a score used more frequently to measure communication?ability in autism, and geography-specific familiarity with the Brunet-Lezine score?(developed for French-speaking clinical evaluators). None of these 3 scales (the 3?used by QURE trial) track the behavioral disturbances that are a major pathology of?MPS3. The Sanfilippo Behavior Rating Scale (SBRS), a 68-item questionnaire which?uses the Vineland as its motor subscale, was only recently specifically developed?to assess the behavioral phenotype of MPS3 children and its disease progression?over time. It has not yet been used in clinical trials. Other trials also use the Muller?behavioral score, and the TBAQ (toddler behavioral assessment questionaire)?has also been proposed as an appropriate scale. However, none of these scales?have been universally accepted by MPS KOLs, discussed to date with regulatory?authorities, or validated in MPS3.?Better MPS3b natural history data needed for optimal development.?¦ The trial PI suggested the main reason a total of 30 mo of Ph1/2 followup would?be needed to best judge the benefit of QURE’s therapy is due to the distinct lack of?natural history data in MPS3b, particularly in regards to clinical neurocognitive data?as well in children under 5 years of age. While MPS3 KOLs believe neurocognitive?development slows and then plateaus between 2-5 years of age, and then declines?after 5 years of age, there is no quantification of this widely accepted decline (either?by clinical scale or magnitude of change). Since the Ph1/2 AMT-110 trial has no?control arm and 3 of 4 patients were under the age of 2.5, the trial PI believes that a?total of 2.5 years of followup data (when all patients will be over 5 years of age and?therefore ought to be declining) will be needed to strongly argue clinical benefit to?accepted natural history. Based on our discussions with KOLs at the conference,?we believe there will be key publications on the natural history of MPS3b and?MPS3a (which is thought to have more rapid decline than MPS3b). We believe an?understanding of natural history will be key for the understanding of future MPS3b?datasets both from QURE, as well as other developers.?Other MPS3b candidates in the pipeline, already in or almost ready for Ph1/2?trials.

?¦ We note MPS3b development programs from a number of other companies that?were either presented or widely discussed at the conference. Of note, ALXN’s?trial of SBC-103, and ERT that is hypothesized to cross the blood brain barrier,?is in a high-profile (at least in the MPS3 community) Ph1/2 trial. Also presented?was Abeona's Ph1/2 ready AAV9-NaGlu gene therapy (the company is currently?completing a MPS3b natural history study to support clinical endpoints). Finally,?BioMarin’s preclinical BMN-250 (a GILT/IGF2 tagged rhERT) while not presented,?was a significant topic of hallway discussion at the conference. KOLs cited the?highly positive interim data from the company’s CLN-2 program (an intrathecal GILT?ERT), noting that ’250 was based on similar technology and may have a similar,?incredibly rapid development pathway.?Prevalence of MPS3 could be 5x current estimates with growing recognition?of late-onset variants.?¦ One point of significant discussion at the conference was the underdiagnosis or?misdiagnosis of late-onset MPS3. Presentations on prevalence and screening noted?a number of incidental diagnosis of MPS3 in patients presenting with dementia in?their 40's. The overall incidence of all MPS3 syndrome in the US is thought to be??about 16,000 children, with MPS3a being the most common. Prevalence of MPS3b?is thought to be only ~330 in the US. However, based on new MPS3 screening?studies, KOLs indicated that actual prevalence could 5x these numbers, given?underdiagnosis of these late-onset variants
twitter.com/liquid_biopsy/status/6713...

flosz 1 december 2015 09:59

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Jefferies cuts $QURE PT to $36.00Wider 3Q Loss; Disappointing, but Not
Surprising Glybera Update QURE no longer pursuing U.S. approval of Glybera does not come as a total surprise to us given frustrating progress to date & disappointing EU launch (& lower treatable pts #s vs. prior expectation per QURE). This underscores yet-tobeproven commercial potential of gene therapies (vs. demonstrating clinical
PoC). While current EV of ~$240M is not expensive, meaningful upside, barring
potential buyout, likely depends on clear clinical benefits of its products.
Wider 3Q15 net loss of €25.8M (vs. our loss of €15.1M) due to one-time noncash
impairment charge of ~€11.6M associated with Glybera. Revenues in 3Q15
were ~€3.2M (vs. ours of €2.1M). QURE stated that the impairment charge was primarily related to a reduction in the number of pts that can be treated with Glybera for lipoprotein lipase deficiency (LPLD). Cash of ~€215M at end-3Q15 should be sufficient into ~2018 by our estimates.
Stops pursuing FDA approval of Glybera, while noting reduced # of treatable
pts in EU (unquantified) - not surprising given the frustrating clinical progress
with Glybera in the U.S. and protracted/disappointing EU launch since its
conditional approval in EU on 10/31/12. Lowering our estimated cumulative # of pts
treated (2015-2032) to 170 (vs. 570 previously); and no longer including Glybera in our PT.
Lowering PT to $36/sh (vs. $40/sh previously) on removal of Glybera and
increased discount rate to 13% (from 12%) to reflect yet-to-be proven commercial
potential/success of gene therapy products (even after approval).
Upcoming clinical events include: (1) Ph1/2 preliminary data from 2 pts for AMT-060
in hemophilia B in Jan. 2016 & preliminary data from 5 pts in the low-dose cohort in 1H16;
(2) regulatory pathway for AMT-060 & AMT-110 to be established in 2016/early-2017; (3)30-mo follow-up Ph1/2 data for AMT-110 in Sanfilippo B in 2016; (4) IND-enabling studies for Huntington's Disease & hemophilia A to begin in 2016; (5) at least 1 new program each in liver & CNS to be nominated in 2016; and (6) validation testing of 4D Synthetic AAV candidates to begin in 2016.
Valuation/Risks
Our $36 PT is based on an NPV analysis of ~$13/sh for S100A1 in CHF/~$9/sh for additional CV targets with BMY, ~$3/sh for AMT-110 in Sanfilippo B, and ~$11/sh for technology value, discounted at 13% annually. Risks include (1) yet-to-be proven clinical utility/commercial potential of gene therapy & (2) pipeline delays/failure.
twitter.com/liquid_biopsy/status/6715...

flosz 1 december 2015 10:00

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ROTH $QURE : Persistence Pays
Note that we are re-publishing this note due to the 3Q earnings announcement
this morning, along with an updated timeline for hemophilia data release. We
are re-establishing active coverage of uniQure with a Buy rating and 12-month
target price of $37/share. Following a long and arduous road, uniQure secured
approval for the first gene therapy in the Western world in 2012.
Preparing for Pivotal: uniQure is exploring a pivotal trial regulatory strategy,
following the announcement of encouraging data in four Sanfilippo syndrome
patients. The gene therapy, manufactured by uniQure, was delivered directly
to the brain of young children suffering from the fatal condition. Twelve months
later, corrected enzyme levels were measured (14-17% of normal) in the
cerebrospinal fluid. These enzyme levels are higher than in patients with the
mild form of the disease.
Beat the Remarkable: In a clinical program for hemophilia B, uniQure
will attempt to further improve on the remarkable results of an academic
consortium (Nathwani et al.,). Factor IX gene therapy successfully corrected
the defect in the majority of patients treated with the highest dose. uniQure
employs a liver-targeted vector (AAV5) at higher doses to deliver a potential
cure for all patients. The company plans to release data from the first dose
cohort in January 2016.
Bristol Sees it: uniQure licensed a cardiovascular gene therapy asset,
coupled with some other preclinical programs to Bristol-Myers Squibb and
received $140MM to date. The cardiovascular asset was originally purchased
for €3MM.
Valuation: We have determined the fair value of uniQure shares by summing
the risk-adjusted cash flows of its lead gene therapy program in hemophilia B.
The sum of the risk-adjusted NPV for the program yields an after-tax (35%),
discounted (15%) value of over $470MM ($20/share), in our estimation. By
adding $300MM ($12/share) for the platform and the estimated $123MM ($5/
share) YE16E cash, the total value is ~$900MM, in our calculation. Using
24MM shares outstanding, the fair value per share equates to $37/share - our
12-month target price on uniQure.
twitter.com/liquid_biopsy/status/6713...

PIPER $QURE 3Q Earnings: Lowering Glybera Expectations but Improving SFB Outlook?This morning QURE released 3Q earnings disclosing that the first patient was treated?with commercial Glybera in Germany during Q4 but also that the company will no?longer pursue Glybera registration in the US. We note that investors have broadly?looked past Glybera to the emerging pipeline, though appreciation of the relevance of?the Sanfilippo B data and awareness of the company's recent progress to become an?integrated gene therapy platform name are very low. We encourage investors to return to this story to see the progress the company is making targeting neuro, liver and heart diseases as well as evolving vectors, developing synthetic promoters, and manufacturing.?Reiterate OW, though our PT is lowered from $40 to $35 as we add in Sanfilippo B but?remove US Glybera estimates.?• Hemophilia B data set for early January: QURE intends to report preliminary gene?therapy hemophilia B results from the first 2 patients in the first dose cohort treated?for 12 weeks. Safety and levels of FIX expression will be disclosed. Data from the?rest of the cohort (5 total pts) is set to be presented at a scientific meeting in 1H?2016. We note that patients are not receiving prophylaxis immunosuppression, and?this should help determine whether QURE's baculovirus manufacturing process does?indeed confer some advantages. At the current valuation, we're rather agnostic as?to whether hemophilia B itself turns into a relevant commercial opportunity for?QURE, since the modularity of its GT platform means if the company sees meaningful?expression in this indication, there should be plenty more Orphan diseases to target.?• Sanfilllipo B advancing: QURE noted that CSF glycosaminoglycans (GAG) levels were?unchanged following gene therapy despite durable expression of NaGlu seen in the?CSF. QURE seems unconcerned by this apparent contradiction, arguing the effect as?an artifact of the deep brain delivery modality and that the supportive dog models?do not show a reduction in CSF GAGs also show a substantial benefit in brain GAGs?and clinical outcome. Recall that expression levels and natural history data suggest?the experience with 4 patients does reflect a meaningful benefit. QURE plans to move?this program in a registration study, though how this profile differentiates with other?approaches in development remains to be seen.?• Model updates: We are removing US Glybera estimates and adding in Sanfilippo B?to our model, and the net result is a reduction in the PT. We are introducing 2016?quarterly estimates.
twitter.com/liquid_biopsy/status/6713...

flosz 1 december 2015 10:19

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twitter.com/tomsilver39/status/671540...

Equities researchers at Janney Montgomery Scott assumed coverage on shares of Uniqure NV (NASDAQ:QURE) in a research note issued to investors on Monday, Analyst Ratings Network.com reports. The firm set a “buy” rating and a $40.00 price target on the stock. Janney Montgomery Scott’s price objective indicates a potential upside of 112.99% from the stock’s previous close. www.marketbeat.com/stocks/NASDAQ/QURE/

Bijlage:

T. Montana 1 december 2015 11:29

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Dank floss voor deze analisten updates! Ik ben niet helemaal gerust over de vaagheid omtrent de endpoints van Sanfilippo, doet me denken aan Glybera

flosz 1 december 2015 21:25

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chardan: $QURE Buy on the market's -17% reaction to old or expected?news?We reiterate our Buy rating on uniQure and advocate using?yesterday's share price weakness to buy into recent positives on?AMT-110 (Sanfilippo B) that were inadequately priced during the?recent biotech market turmoil. Per our 27 August research?("UniQure 2Q results provide visibility on key September to?December catalysts"), we removed US Glybera (lipoprotein lipase?deficiency) from our model after uniQure announced the FDA would?require more than one additional study to support a BLA filing for?Glybera in the US. Our decision was based on Glybera's overall?commercial profile, which we did not believe justified US investment.?The impact on our price target at the time was 7.5%, and with far?more important assets like AMT-060 (hemophilia B) and AMT-110?(Sanfilippo B) making progress, and having upcoming catalysts, we?affirmed our Buy rating on uniQure. Yesterday, uniQure reported?3Q15 results and provided an update on key programs, including the?disclosure that, as we expected, uniQure would not pursue regulatory?approval for Glybera in the US, confirming a positive for us, that?uniQure is pursuing the value-enhancing decision (i.e. not to pursue?US Glybera). We further note that a 1- to 4-week delay on the release?of preliminary AMT-060 hemophilia B data is immaterial to models.?The following are what we consider key incremental items from the?3Q2015 results release and conference call:?• AMT-060 (hemophilia B): In early January 2016 (previously due?by end-2015), uniQure will release preliminary, top-line data for 2?patients who have completed at least 12 weeks of treatment, with?full results from the first cohort of 5 patients at a scientific?conference in 1H2016.?•?- Our take: A delay of a few weeks is immaterial to our?thesis, or any reasonably-modeled hemophilia B view, and?we continue to expect a signal of factor IX expression at 12?weeks in each of the 2 patients shown in early 2016.?- Another take: We believe Dimension Therapeutics (Neutral,?PT$13) traded up 9% yesterday, as some in the market?wondered why uniQure is presenting data for only 2 patients?of 5 in the interim data. Our catalyst sheets for months have?consistently showed uniQure's expectations of "2-3 patients"?of data out of the 10 in the trial, and we note this approach?enhances the ability of QURE to present the data at a major?scientific conference. As such, we continue to prefer uniQure?over Dimension Therapeutics.?• AMT-110 (Sanfilippo B): uniQure is still working to complete?in-licensing of the recent positive phase I/II data from Institut?Pasteur and will follow the patients through the end of the?30-month study, which is expected around year-end 2016.??Our take: We continue to believe long-term durability of AMT-110's benefits in a deadly disease like Sanfilippo B would represent a landmark result in gene therapy and in lysosomal storage diseases, as patients who should have been heading towards a vegetative state by the age of 7 or so (and death in their teens) showed age-dependent and almost normal brain development without atrophy at sequential MRIs.?• Glybera US: As above, the company has decided not to pursue U.S. regulatory approval of Glybera in lipoprotein?lipase deficiency and as a consequence reported a one-time, non-cash impairment of €11.6 million, which drove an EPS of -€1.08, versus our expectation of -€0.60.?•?- Our take: Prior to this news, we modeled €0 million in 2025E revenues for Glybera US, meaning no impact to our sales model and no impact to our valuation.
twitter.com/liquid_biopsy/status/6717...

[verwijderd] 1 december 2015 21:57

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www.capitalcube.com/blog/index.php/un...

uniQure NV – Value Analysis (NASDAQ:QURE) : December 1, 2015
Posted by CapitalCube on December 1, 2015 in Fundamental Analysis, Yahoo Finance | 86 Views | Leave a response
Click here to see latest analysis

Capitalcube gives uniQure NV a score of 25.

Our analysis is based on comparing uniQure NV with the following peers – Isis Pharmaceuticals, Inc., Amgen Inc., Sangamo BioSciences, Inc., bluebird bio, Inc., BioMarin Pharmaceutical Inc. and Sanofi Sponsored ADR (ISIS-US, AMGN-US, SGMO-US, BLUE-US, BMRN-US and SNY-US).
Investment Outlook

uniQure NV has a fundamental score of 25 and has a relative valuation of OVERVALUED.
Fundamental Score

Access our research and ratings on uniQure NV
Click here to view
Company Overview

Compared to peers, relative outperformance over the last year has faded more recently.

uniQure N.V.’s current Price/Book of 3.08 is about median in its peer group.

QURE-US‘s earnings and EBITDA are both negative which suggest that P/E or Price/EBITDA are not meaningful to make this analysis between operating advantage (ROE) and growth expectations (as suggested by P/E or P/EBITDA).

QURE-US‘s relatively low net margins and poor asset turns suggest a problematic operating strategy.

Changes in annual revenues (relative to peers) are better than the change in its earnings (relative to peers), implying the company is focused more on revenues.

QURE-US‘s return on assets currently and over the past five years has trailed the peer median and suggests the company might be operationally challenged relative to its peers.

The company’s capital investment program and to-date returns suggest that the company is likely making big bets on the future.

QURE-US‘s operating performance may not allow it to raise additional debt.

Access our research and ratings on uniQure NV
Click here to view
Leverage & Liquidity

QURE-US would seem to have a hard time raising additional debt.

With debt at a relatively low 9.38% of its enterprise value compared to an overall benchmark of 25% (Note: The peer median is currently 9.38%), and relatively tight interest coverage level of -12.77x, QURE-US would have a hard time raising much additional debt. Thus, the company is classified as having Limited Flexibility when it comes to raising more debt.

Of the 6 chosen peers for the company, only 5 of the stocks have an outstanding debt balance. Companies with no debt include SGMO-US.

QURE-US has maintained its Constrained profile from the recent year-end.

QURE-US‘s interest coverage has declined 1.09 points from last year’s high but remains above its five-year average interest coverage of -64.88.

While its interest coverage decreased to -12.77x from -11.68x (in 2014), its peer median increased during this period to 7.37x from 6.28x.

Interest coverage fell 2.17 points relative to peers. It is also below the 2.50x coverage benchmark unlike the peer median.

QURE-US‘s debt-EV is similar to last year’s high of 9.38%, which compares to a low of 9.38% in 2014.

Though its debt-EV has remained relatively stable at 9.38% compared to 2014, its peer median has increased to 9.38% from 6.58% during this period.

Relative to peers, debt-EV fell 2.80 percentage points.

Access the detailed analysis for uniQure NV
Click here to view
Key Liquidity Items
Company Debt/Enterprise Value (%) Current Ratio Interest Coverage (x) Cash Flow To Total Debt (%)
Isis Pharmaceuticals, Inc. 10.51 7.11 -0.08 13.89
Amgen Inc. 30.23 5.42 7.37 25.55
Sangamo BioSciences, Inc. 0 10.29 No interest exp 999
bluebird bio, Inc. 1.77 21.24 No interest exp -434.97
BioMarin Pharmaceutical Inc. 3.93 3.01 -7.04 -13.71
Sanofi Sponsored ADR 13.96 1.35 12.97 47.79
uniQure N.V. 9.38 8.39 -12.77 -198.51
Peer Median 9.38 7.11 7.37 13.89
Best In Class 1.77 21.24 No interest exp 999

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Company Profile

uniQure NV engages in the discovery, development, and commercialization of gene therapies. Its core gene therapies include AMT-060 for the treatment of hemophilia B; preclinical S100A1 therapeutic for the treatment of congestive heart failure; and Glybera 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. uniQure was founded on January 9, 2012 and is headquartered in Amsterdam, Netherlands.
Disclaimer

The information presented in this report has been obtained from sources deemed to be reliable, but AnalytixInsight does not make any representation about the accuracy, completeness, or timeliness of this information. This report was produced by AnalytixInsight for informational purposes only and nothing contained herein should be construed as an offer to buy or sell or as a solicitation of an offer to buy or sell any security or derivative instrument. This report is current only as of the date that it was published and the opinions, estimates, ratings and other information may change without notice or publication. Past performance is no guarantee of future results. Prior to making an investment or other financial decision, please consult with your financial, legal and tax advisors. AnalytixInsight shall not be liable for any party’s use of this report. AnalytixInsight is not a broker-dealer and does not buy, sell, maintain a position, or make a market in any security referred to herein. One of the principal tenets for us at AnalytixInsight is that the best person to handle your finances is you. By your use of our services or by reading any our reports, you’re agreeing that you bear responsibility for your own investment research and investment decisions. You also agree that AnalytixInsight, its directors, its employees, and its agents will not be liable for any investment decision made or action taken by you and others based on news, information, opinion, or any other material generated by us and/or published through our services. For a complete copy of our disclaimer, please visit our website www.analytixinsight.com.

T. Montana 1 december 2015 22:46

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@flosz: dank wederom; heel lucide rapport van chardan, toch niet een van de top huizen, goed onderbouwd en roerend mee eens.
@ ivet: dit is me nogal een appels en peren vergelijking; ik hoop dat niemand dit als basis voor investeren gebruikt of liever, desinvesteren, trading bots misschien?

Fartknock 2 december 2015 05:54

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Idd zeer goede analyse van Chardan. Dank Flosz.

[verwijderd] 2 december 2015 10:15

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quote:
T. Montana schreef op 1 december 2015 22:46:
@flosz: dank wederom; heel lucide rapport van chardan, toch niet een van de top huizen, goed onderbouwd en roerend mee eens.
@ ivet: dit is me nogal een appels en peren vergelijking; ik hoop dat niemand dit als basis voor investeren gebruikt of liever, desinvesteren, trading bots misschien?

klopt, heb er niets mee en toont flauwekul/willekeur van analisten(rapporten) aan.

Genoeg mensen die minder krijgen voor beter en degelijker werk...

Toch vermeld ik dit soort berichten omdat er helaas genoeg mensen zijn die niet een eigen koers varen, maar soms blind varen op mensen die zeggen er verstand van te hebben. Commentaar op rapporten als deze geeft die mensen soms weer nieuwe inzichten.

Ik ben zelf niet academisch opgeleid, maar heb dat ook niet als gemis ervaren in het beleggen. Belangrijk is om je eigen vaardigheden en talenten te ontdekken/ontwikkelen en aan te wenden en respect te hebben voor de kunde/kennis en inzet/moeite van medemensen.
Soms vertellen mensen vanuit hun vakgebied of laten communicatieve vaardigheden zien waarbij ik met mijn oren sta te klapperen en voel me dan soms net een holbewoner zo achterlijk...maar als ik dan op mijn beurt weer verhaal van mijn kennis, staan die mensen op hun beurt weer met hun oren te klapperen. Het is mooi als mensen samenwerken om al hun kunde en kennis te bundelen! Ik zei wel eens eerder: als ik alleen al de lijm kon zijn tussen alle afzonderlijke deskundigen, dan is dat al heel wat!

Fartknock 2 december 2015 16:54

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UniQure (QURE) is Reiterated by WallachBeth to Buy, Raises Price Target to $60
December 2, 2015 ·

uniQure (QURE) is Reiterated by WallachBeth to Buy according to the research report released to the investors. The brokerage firm has raised the Price Target to $60 from a previous price target of $33 . The shares recommendation by the Brokerage Firm was released on Dec-2-2015.

www.moneyflowindex.org/uniqure-qure-i...

flosz 7 december 2015 12:10

1

Leerink $QURE Top-Line Hemophilia B Data in Jan 2016 Next Key
Catalyst
• Bottom Line: QURE reported 3Q results and provided a pipeline
update. QURE expects to report preliminary top-line data from its Phase
1/2 hemophilia B study in early January 2016 which we think could
incrementally de-risk QURE's AAV5-based gene delivery platform. QURE
also announced that it will not be pursuing US regulatory approval of
Glybera, which we view favorably given the small commercial opportunity
relative to other strategic priorities. QURE also expects to advance an
addl. preclinical CNS program focused on Huntington's Disease in 2016,
while preparing for the launch of a company-sponsored Phase II/III trial
in MPS-IIIB (mucopolysaccharidosis type IIIB) in late 2016/early '17.
Updating estimates and reiterating OP rating.
• Top-line hemophilia B data expected in January 2016. QURE guides
to announcing preliminary top-line data from the Phase 1/2 hemophilia
B study by the 2nd week of January 2016. These data will include topline
data including safety results and Factor IX (FIX) expression levels
for two patients with at least 12 weeks of follow-up. In our conversation
mgmt. emphasized their desire to have 12 weeks of follow-up data before
presenting given that elevations of liver enzymes are often seen at 6-12
weeks and also reliable FIX expression from the gene therapy may not be
obtained prior to 12 weeks given that the patients are still being weaned
off recombinant F9. Full data from the first cohort of 5 patients will be
presented at a scientific conference in 1H16. QURE is still on track to
initiate dosing in the 2nd cohort in January 2016 and completing this
Phase I/II trial in 2016.
• We would be buyers ahead of the hemophilia B data. We view
positive hemophilia B data as potentially validating for AAV5-based gene
delivery to the liver in humans. Based on our prior MEDACorp KOL
discussions, consistent and durable Factor IX expression of greater than
5% of normal represents the well-established bar to success. Whereas
higher expression is desirable, striking clinical outcomes have been
achieved with mean steady-state expression levels of only 5%. We view
several factors as working in QURE's favor relative to published positive
clinical data, including: (1) existing clinical validation of QURE's gene
cassette incorporating the native F-IX gene, (2) existing validation of
AAV5 vector safety at 10x higher doses vs. those tested previously, (3)
lower prevalence of preexisting neutralizing antibodies to AAV5 vs. AAV8,
and (4) higher product purity/potency (less empty capsids) potentially
leading to higher durability and less immunogenicity.

• Next Sanfilippo B update (30-month follow-up) expected by
YE16. QURE continues to work on in-licensing of the Sanfilippo B
data from their academic collaborator and complete 30 month followup
of the Phase 1/2 patients around YE16. We continue to view the
presented Phase 1 data as promising with durable and persistent
NAGLU activity (from 0% at baseline to 14-17% of normal). Based on
our prior MEDACorp KOL discussions, as little as 5% of normal NAGLU
expression across the brain could be clinically meaningful.
• US regulatory approval of Glybera will not be pursued; not
surprising to us. We view this as positive and mostly expected given the
small commercial opportunity associated with Glybera

twitter.com/liquid_biopsy/status/6738...

flosz 5 januari 2016 14:21

1

Piper: twitter.com/liquid_biopsy/status/6843...

Bijlage:

T. Montana 5 januari 2016 19:18

0

Dank flosz, zoals altijd to the point en informatief.

flosz 7 januari 2016 14:25

0

COWEN $QURE 1st AMT-060 Hemophilia B Gene Tx?Data: #Solid POC from Early Low Dose?QURE released first 2 patient preliminary data from the low dose cohort of its?AMT-060 hemophilia B AAV5 gene therapy drug. In our view, the overall efficacy at 12?and 20wks gives solid POC and is encouraging when combined with precedent from?the related St. Jude's product, which has best in class long-term data thus far. Early?safety is also solid; we look forward to LT data in mid '16.?AMT-060 Hemophilia B Data Shows Solid POC with Encouraging FIX?Production Rates and Manageable Safety?¦ Overall FIX 12 week production rates of 5.5% and 4.5% of normal in first 2?pts highly encouraging for low dose - Company conference call at 8:30am?ET. AMT-060 is QURE's AAV5 based wild-type Factor IX gene therapy for hemophilia?B that entered clinical trials in late 2015. We believe this first data from the low?dose (5x1012) cohort gives encouraging proof of concept given the strength of the?efficacy signal seen in these first patients at 12 weeks. QURE also noted that 2 of?the 3 remaining patients in the low-dose cohort have discontinued prophylaxis?(factor reduction rate was at clinician's discretion), which is also encouraging, and?we look forward to full 12-week data later this year.?¦ Liver enzyme elevations appears to be highly manageable. QURE reported?that patient #1 experienced a "mild, transient and asymptomatic elevation" of liver?transaminases at week 10 that resolved with an 8 week steroid taper. Encouragingly,?this patient still expressed solid factor levels at week 20, after discontinuation of?steroid (steroid tapers frequently permanently blunt transgene expression as seen in?the Ph1/2 Baxalta Hemophilia B gene therapy data presented at ISTH 2015).?¦ 0% screen failure rate for AAV5(+) boosts commercial opportunity. Of?note, QURE indicated the trial had no screen failures due to baseline AAV5?seropositivity. This supports our conclusions from the literature which indicate?an overall population AAV5 positivity rate of <25%, which is much lower than?other AAV subtypes (usually 50%). This is thought to be in part because AAV5 is?thought to be the most genetically divergent and therefore susceptible to less cross?positivity. Bottom line, this means that likely larger percentage of Hemophilia B?patients would be clinically eligible for AMT-060 compared to other treatments,?which has important commercial ramifications.?¦ Fair balance: Longer term expression levels are key to elucidate therapeutic?profile, but St. Jude's product bodes well for '060. We do note the true clinical?profile of AMT-060 will require years of followup data to fully elucidate. Transgene?expression levels can fall over time, especially as non-replicating transgene?containing plasmids could be cleared from rapidly regenerating tissues (e.g. liver).?However, we do note that encouraging precedent data exists from the St. Jude's?AAV8 hemophilia B product which uses the same transgene payload. The St. Jude's?publication suggests transgene expression is relatively steady in most patients by 12??weeks of followup (with minor fall-off) through ~40 weeks. Longer term followup?suggests profound reductions in factor requirements and improvements in disease?burden. Overall we think this, as well strong 2H15 QURE San Filipo B gene therapy?data, bodes very well for the '060 program, including full low dose and first high?dose (2x1013) data around mid 2016.

twitter.com/liquid_biopsy/status/6850...

[verwijderd] 8 januari 2016 10:54

0

www.medicaldaily.com/uniqure-shares-j...

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