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uniQure NV (QURE) Sees Significant Decrease in Short Interest
Posted by James Conley on Nov 17th, 2016 // No Comments

uniQure NV logouniQure NV (NASDAQ:QURE) saw a significant drop in short interest in October. As of October 31st, there was short interest totalling 874,899 shares, a drop of 27.0% from the October 14th total of 1,198,353 shares. Based on an average trading volume of 131,108 shares, the short-interest ratio is currently 6.7 days. Currently, 6.4% of the shares of the stock are short sold.
rationeel
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rationeel
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DAG grafiek: Met laag volume een groen candeltje. Steun 7,01 Weerstand 7,19 Sk 7,10

WEEK grafiek: Low 6,94 High 8,32
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T. Montana
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Jefferies, November 21, 2016/part 1
uniQure N.V. (QURE)
November 21, 2016
Execution Risks & Lack of Major Near-term Catalysts; Downgrading to Hold

Key Takeaway
Once viewed as the most advanced gene therapy platform company (& landmark gene therapy deal w/ BMY in 2015), today QURE's position is overshadowed by lack of progress, competition & poor execution. Its strategic review & prioritization announced last wk comes up short in providing confidence in strategy/execution. In addition, with no apparent NT key catalysts, we move to Hold until we see a turnaround in execution.

Reduction/delays in its gene therapy programs no longer support our prior valuation; downgrading QURE to Hold & reducing PT to $8 from $28. Following its strategic review, QURE pared down clinical programs & outlined general, but delayed timelines for key programs last wk. Our downgrade (D/G) is based on (1) disappointing progress to date; (2) overshadowing competition; (3) execution risk; & (4) no apparent key catalyst to offer meaningful upside in next ~12mo. Risks to our D/G include buyout by partner BMY/other or significant partnership. Cash at end-2Q16 of €166M sufficient into 2019, per QURE.

Significant reduction in our PT stems from (1) lowered technology value (62% of our PT reduction); (2) delayed timeline for S100A1 (23%) & (3) AMT-110 discontinuation (15%). For biotech companies, the longer it takes to make a successful/ timely transition from platform to products, the further diminishing platform value there is, in our view. While quite subjective, our prior QURE tech value was largely on probability- adjusted potential milestones from partner BMY on up to 10 targets ($217M per target #2-10). In the absence of data, lack of apparent progress/extended timelines has lowered our confidence in BMY collaboration. Thus, we're basing tech value on BMY initial fee alone, reducing by 62% to $3.50/sh (vs. $16/sh prior).

We view BMY collaboration in S100A1 for CHF is key to share appreciation; human proof-of-concept data unlikely until ~2018+. When QURE formed the landmark gene therapy partnership with BMY on 4/6/15, first pt dosing for lead S100A1 was expected potentially in 2016; but now, it would be unlikely until ~2018+. Thus, we're pushing out previously anticipated BMY milestone payments in our projections (~$245M in total), resulting in NPV of $4.50/sh for S100A1 (vs. $9/sh prior).

Valuation/Risks
Our PT of $8 is based on a NPV analysis of ~$4.50/sh for S100A1 & ~$3.50/sh for technology, discounted at 14% annually. Risks include: (1) yet-to-be proven clinical utility/commercial potential of gene therapy & (2) pipeline delays/failure.
T. Montana
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Jefferies, November 21, 2016/part 2

QURE’s review and prioritization of its gene therapy programs comes up short in providing confidence in its execution. Timelines for its new key programs are delayed but yet-to-be laid out clearly, including AMT-060 for hemophilia B (Phase 1/2), AMT-130 for Huntington’s disease in (preclinic) & Bristol-Myers Squibb (BMY, Hold) partnered CHF program (preclinic). In addition, QURE is discontinuing two programs (AMT-110 for Sanfilippo B in Phase 1/2, AMT-140 in Parkinson's disease in Phase 1/2). Particularly, discontinuation of AMT-110 for Sanfilippo B is surprising because QURE had been excited about early clinical data and as recently as late-August, QURE was expecting additional data from 30-month follow-up in 4 treated patients in 1Q17. For AMT-140 in Parkinson's disease (PD), it noted it took ~3 years to enroll the first 2 of 4 dose cohorts (total n=12) and concluded that AMT-140 does not align with its core strategy; its collaborators, UCSF and NIH, could complete the ongoing Phase 1/2 trial.

With its planned 20-25% reduction in personnel (50-60 positions) and lower OpEx (R&D
refocus), QURE expects €5-€6M in savings by end-2017 & €11-€15M cost savings over next two years. Its cash of €166M at end of 2Q16 should be sufficient into ~2019 per QURE. In addition, QURE will move all GMP manufacturing to its Lexington, MA facility (vs. another in Amsterdam).

For AMT-060 in hemophilia B, QURE now expects discussions with regulators on a pivotal trial design to begin in 1Q17 (vs. previously planned to establish a regulatory pathway for approval in 2016). See Chart 1 for competitive products in development. QURE expects to disclose data for the high-dose cohort (2x1013 gc/kg, n=5) at ASH on 12/3/16 (Oral presentation titled “Interim results from a dose escalating study of AMT-060 (AAV5-hFIX) gene transfer in adult patients with severe hemophilia B”). At our Gene Editing/Therapy Summit last month, QURE stated that ~5- 10% FIX activity is a reasonable expectation for the high-dose cohort (vs. 5.4% activity at 4x lower dose cohort, n=5). Based on these data, QURE expects to begin discussions with regulators about the design of a pivotal study and to begin manufacturing product for a pivotal study in 1Q17. We continue to view hemophilia B is not a medically unmet need (relatively well served market with current factor replacement therapies), aside from a crowded hemophilia B gene therapy field, including competitors such as Spark Therapeutics' (ONCE, Hold)/Pfizer's (PFE, Hold) SPK-9001 that has shown higher FIX activity of mean 32.2% +/- 6.5% (but with one participant exhibiting an immune response for the AAV capsid, characterized by a rise in LFTs above the subject's own baseline, but not above the upper range of normal, accompanied by decline in factor IX activity level). We continue to see little commercial opportunity for AMT-060. Currently we assign 75% probability for success and assume a commercial launch in the U.S. in 2020.

For hemophilia A, QURE plans to provide an update in 1H17 (vs. previously identification of a candidate by YE16/early-2017). Though not specifically mentioned as a priority, QURE will assess a go/no-go decision for this program in 1H17.

For AMT-130 in Huntington’s disease (HD), it is prioritizing the program with additional preclinical data expected in 2017 & potential IND filing in ~2018 (vs. previously entering clinic in 2H17). See Chart 2 for competitive products in development. Animal studies in rats and mouse models demonstrated that AMT-130, a gene therapy product candidate for HD, produces ~80% knockdown of the Huntingtin gene (HTT) in the cortex and putamen brain regions. IND-enabling studies were commenced earlier this year. Given its early stage, we assign ~25% probability for success for now and assume a potential commercial launch in ~2022
DeZwarteRidder
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quote:

T. Montana schreef op 22 november 2016 00:13:

Jefferies, November 21, 2016/part 1
uniQure N.V. (QURE)
November 21, 2016
Execution Risks & Lack of Major Near-term Catalysts; Downgrading to Hold

Key Takeaway
Once viewed as the most advanced gene therapy platform company (& landmark gene therapy deal w/ BMY in 2015), today QURE's position is overshadowed by lack of progress, competition & poor execution. Its strategic review & prioritization announced last wk comes up short in providing confidence in strategy/execution. In addition, with no apparent NT key catalysts, we move to Hold until we see a turnaround in execution.

Reduction/delays in its gene therapy programs no longer support our prior valuation; downgrading QURE to Hold & reducing PT to $8 from $28. Following its strategic review, QURE pared down clinical programs & outlined general, but delayed timelines for key programs last wk. Our downgrade (D/G) is based on (1) disappointing progress to date; (2) overshadowing competition; (3) execution risk; & (4) no apparent key catalyst to offer meaningful upside in next ~12mo. Risks to our D/G include buyout by partner BMY/other or significant partnership. Cash at end-2Q16 of €166M sufficient into 2019, per QURE.

Significant reduction in our PT stems from (1) lowered technology value (62% of our PT reduction); (2) delayed timeline for S100A1 (23%) & (3) AMT-110 discontinuation (15%). For biotech companies, the longer it takes to make a successful/ timely transition from platform to products, the further diminishing platform value there is, in our view. While quite subjective, our prior QURE tech value was largely on probability- adjusted potential milestones from partner BMY on up to 10 targets ($217M per target #2-10). In the absence of data, lack of apparent progress/extended timelines has lowered our confidence in BMY collaboration. Thus, we're basing tech value on BMY initial fee alone, reducing by 62% to $3.50/sh (vs. $16/sh prior).

We view BMY collaboration in S100A1 for CHF is key to share appreciation; human proof-of-concept data unlikely until ~2018+. When QURE formed the landmark gene therapy partnership with BMY on 4/6/15, first pt dosing for lead S100A1 was expected potentially in 2016; but now, it would be unlikely until ~2018+. Thus, we're pushing out previously anticipated BMY milestone payments in our projections (~$245M in total), resulting in NPV of $4.50/sh for S100A1 (vs. $9/sh prior).

Valuation/Risks
Our PT of $8 is based on a NPV analysis of ~$4.50/sh for S100A1 & ~$3.50/sh for technology, discounted at 14% annually. Risks include: (1) yet-to-be proven clinical utility/commercial potential of gene therapy & (2) pipeline delays/failure.
Tjonge, jonge, beter laat dan nooit...!!

Wat een halve garen met hun veel te hoge koersdoel van 28 en de veeel te late bijstelling naar 8.

T. Montana
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Jefferies, November 21, 2016/part 3 & laatst

BMY collaboration, QURE expects IND filing for S100A1 in CHF in 2018 (vs. dosing of first patient in Phase 1b in 2016 when the BMY deal was signed on 4/6/15). We view timely progress/success of S100A1 for chronic heart failure (CHF) in collaboration with BMY is a key driver for QURE. Unfortunately we would not be able to see human proof-of-concept data until ~2018-2019. Currently we assign 40% probability for success and assume a commercial launch by BMY in ~2023.

Resetting PT to $8 (from ~$28) on delayed/discontinued programs, lowered technology value and increased discount rate. Our $8 PT is based on an NPV analysis of ~$4.50/sh for S100A1 (vs. ~$9/sh prior) assumed delays in achieving potential milestone payments from partner BMY; and ~$3.50/sh for technology value (vs. ~$16/sh prior) on disappointing progress to date (delays, discontinuation), overshadowing competition, & unclear development timelines, discounted at 14% annually to reflect execution risks (vs. 13% prior). We have removed AMT-110 in Sanfilippo B (~$3/sh prior) in our valuation due to QURE’s discontinuation.

Given the low commercial potential for hemophilia B gene therapy in general (a well controlled condition with currently available therapies & ~5x smaller market size vs. hemophilia A), AMT-060 does not contribute to our QURE valuation. For AMT-130 for HD, given the very early-stage of development (potential IND filing in ~2018), even with our projected cumulative sales of >$1B by 2026, on a ~25% probability-adjusted, AM-130 contribution to our valuation is minimal at present.
Given the commercial failure of QURE’s Glybera to date, success of a gene therapy product is yet to be materialized. We acknowledge that perceived technology value is highly subjective. For gene therapy companies, enterprise value (EV) ranges widely from bluebird bio (BLUE, BUY) at ~$1.9B to Adverum Biotechnologies (ADVM, Buy) at (~$83M). Thus, technology value of a gene therapy company can be less than $0 on lead product failure and lack of confidence in programs & management, particularly for money-losing companies (as cash is a moving target). In our view, a technology value for biotech companies diminishes if the company fails to make a successful, timely transition from a platform-based to a product-based company. For QURE, we view its current valuation is largely supported by the collaboration with BMY in the CV area.

Upcoming events include: (1) AMT-060 data at ASH on 12/3/16; (2) update on next generation AAV technology in liver diseases in early-2017; (3) preclinical data for AMT-130 in HD in 2017 & IND filing in ~2018; and (4) IND filing of S100A1 in 2018.
Prof. Dollar
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quote:

T. Montana schreef op 22 november 2016 00:13:

Jefferies, November 21, 2016/part 1
uniQure N.V. (QURE)
November 21, 2016
Execution Risks & Lack of Major Near-term Catalysts; Downgrading to Hold
Dank voor het plaatsen. Ik ben ook benieuwd naar je eigen opinie. Wil je die delen?
Prof. Dollar
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uniQure Announces Financial Results For Third Quarter 2016 and Provides Update on Company Progress

"Our near-term priorities are to aggressively prepare for a pivotal study in hemophilia B and [..]"

Eindelijk, de woorden zijn er. Nu de daden!

uniqure.com/investors-newsroom/press-...
DeZwarteRidder
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quote:

Fartknock schreef op 22 november 2016 15:29:

Er waait een nieuwe wind.
Ja, een fart.....
DeZwarteRidder
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uniQure N.V. (QURE)

NasdaqGS - NasdaqGS Real Time Price. Currency in USD
6.66-0.47 (-6.59%)

As of 3:57 PM EST. Market open.
rationeel
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DeZwarteRidder
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quote:

rationeel schreef op 22 november 2016 22:57:

Met een GROTER dan gemiddeld volume een rode candle. Steun 6,45 weerstand 7.06 Sk 6,66
Bijlage:
rationeel
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Met een kleiner dan gemiddeld volume een groene candle. Steun 6.49 Weerstand 6,80 Sk 6,68
De laagste koers was vandaag 6,41. Dit lijkt de voorlopige? low.
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DAG grafiek: Met klein volume een groene candle. Steun 6,70 Weerstand 6,95 Sk 6,84

WEEK grafiek: LOW 6,41 ATL High 7,27
Er is sprake van positieve divergentie.
T. Montana
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quote:

Prof. Dollar schreef op 22 november 2016 14:30:

[...]
Dank voor het plaatsen. Ik ben ook benieuwd naar je eigen opinie. Wil je die delen?
Opinie is niet zo veel waard als de perceptie zo negatief is. De banken die QURE volgen zijn 2de rangs en Jefferies zoekt m.i. al lang naar een reden om de TP naar beneden te schroeven en dit is de perfecte aanleiding, of die nu terecht is of niet. Katherine High heeft in de VS een veel hoger profiel dan wie dan ook bij QURE en maakt daar handig gebruik van. Hans Bishop van Juno is ook zo iemand. Eerst gecashed bij Dendreon voordat het tegen een ijsberg te gronde ging en nu net in juli 20 miljoen in opties gecashed bij Juno die net weer slachtoffers meldden in hun trial en een hold. V.w.b. QURE, veel hangt af van de data bij ASH. Als die goed zijn zal het lek wat boven water komen. Hoe veel, geen idee. Dat hangt ook af van de Spark data en de agressieve spin die ze er aan geven. Een charismatische CEO is voor QURE heel belangrijk denk ik, iemand die de geloofwaardigheid kan herstellen en de belofte van het platform zo kan communiceren dat de waardering stijgt. Een mooie deal zou ook helpen, maar met wie is de vraag. Het veld is behoorlijk dicht bevolkt inmiddels, veel kapers op de kust, dus prijzen zullen niet van het niveau Inocard zijn. Ik ben teleurgesteld, maar heb mijn horizon twee jaar opgeschoven naar 2019. En zoals ik eerder schreef, ik kan me niet voorstellen dat Forbion de boel de boel zal laten. Daarvoor zit er teveel bloed, zweet en tranen in om nog maar te zwijgen van reputatie. Ik heb ooit ergens gelezen dat van Deventer betrokken was bij de ontwikkeling van de eerste monoclonal antibody, Centoxin van Centocor. Dat is in 1993 gigantisch door het ijs gezakt met dramatische gevolgen voor Centocor. Maar uiteindelijk is dat Remicade geworden en Centocor is 6 jaar later voor $5 miljard door J&J gekocht. Kortom het kan verkeren en het team van Forbion weet dat beter als geen ander.
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