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  1. T. Montana 13 januari 2016 13:57

    • T. Montana

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    JEFFERIES
    uniQure N.V. (QURE): Update from Meeting with QURE Management
    Rating BUY
    Price Target $36.00
    Price $16.35
    Bloomberg NASDAQ: QURE

    *Shares Out. (MM): Reflects share balance as of 9/30/15
    Key Takeaway
    For 2016, we view clinical data catalysts to be on AMT-060 for hemophilia B. QURE expects additional 3 pts Ph1/2 data for AMT-060 low-dose cohort to be presented at a medical conference in 2Q16 and data from high-dose cohort (4x low-dose) potentially by YE16. On non-human primate data, QURE expects higher F9 expression (vs. 4.5-5.5% observed at low-dose in n=2), although not commenting on an anticipated magnitude of F9 expression.

    For AMT-060 for hemophilia B (human Factor IX [FIX] gene in an AAV5 vector), enrollment in high-dose cohort (2x10^13 gc/kg, over 4x low-dose; n=~5) in Ph1/2 to begin in ~1Q16 following a DMC meeting in ~early-Feb. with a potential update by YE16. Based on non-human primate data, QURE expects high-dose AMT-060 to produce higher F9 expression vs. the low-dose (~4.5-5.5% in n=2), although not commenting on the magnitude of anticipated increases. Given that potential liver toxicity is correlated to the high vector copy numbers, QURE recently amended the protocol for high-dose cohort to require 2x weekly liver enzyme screening in the first 12 wks tx and recommend prednisolone administration for ALT levels >1.5-2.0x baseline. QURE will also allow enrollment of >5 pts, noting high interests from patients seen to date. Final design of pivotal trial, expected to begin in 2017, is yet to be discussed; QURE has noted an open-label trial of ~40-50 pts with primary endpoints such as annual bleed rate (ABR) and PK as possible.

    Additional 3 pts in AMT-600 low-dose cohort (5x10^12 gc/kg) in Ph1/2 to be presented at a medical conference in 2Q16, potentially at World Federation of Hemophilia in Orlando, FL on 7/24-7/28/16 or European Hematology Association in Copenhagen, Denmark on 6/9-6/12/16. On 1/7/16, preliminary topline results from 2 pts in the low-dose cohort showed F9 expression of 5.5%/4.5% at ~20/12 wks of follow-up post treatment (vs. <1-2% prior to tx), validating ability of QURE's AAV5 vector to penetrate the liver. As of 1/6/16, all 5 pts had completed at least 10 wks of therapy and n=4/5 pts had discontinued prophylactic recombinant F9 therapy (a secondary endpoint for the trial). No patients developed antibodies against AAV5; the first pt treated experienced a mild, transient and asymptomatic ALT elevation at ~10 wks post tx, which resolved following 8 wks of tapering prednisolone doses. In a St. Jude's study utilizing the same F9 gene cassette (scAAV2/8-LP1-hFIXco; at the same dose as AMT low-dose, 2x10^12 vector genome/kg) in an AAV8 vector, n=4/7 pts were able to discontinue prophylactic rF9 therapy with F9 levels of up to ~7% of normal. Sustained F9 levels of the pts who could not discontinue prophylactic therapy were ~1-3%.

    Upcoming events include: (1) full low-dose Ph1/2 data (n=5) for AMT-060 in hemophilia B at a medical meeting in 2Q16 (potentially World Federation of Hemophilia on 7/24-7/28/16 or European Hematology Association on 6/9-6/12/16); ?(2) regulatory pathway for AMT-060 & AMT-110 to be established in 2016; (3) potential Ph1/2 high-dose data for AMT-060 in hemophilia B by YE16; (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) 30-month follow-up data for AMT-110 in Sanfilippo B in early-2017.
  2. [verwijderd] 13 januari 2016 14:03

    • [verwijderd]

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    There is excitement in the hemophilia community, both from the side of patients and from physicians and researchers, about the potential benefits of gene therapy for the health and quality of life of hemophiliacs. The potential that a successful gene therapy would allow hemophiliacs to forego prophylactic treatment in the future would be life changing.
  3. Prof. Dollar 13 januari 2016 15:27

    • Prof. Dollar

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    quote:

    T. Montana schreef op 13 januari 2016 13:57:

    JEFFERIES
    uniQure N.V. (QURE): Update from Meeting with QURE Management
    Rating BUY
    Price Target $36.00
    Price $16.35
    Bloomberg NASDAQ: QURE
    [..]

    Prrrr, het wordt steeds erger...

    "30-month follow-up data for AMT-110 in Sanfilippo B in early-2017."
    Tijdens de Investor Day is eind 2016 aangegeven! Worden de resultaten dan gepresenteerd tijdens JPM17? Een goedmakertje? En wat is de implicatie voor de beoogde pivotal trial? Ook uitstel?

    Over tweede cohort Hemofilie B:
    "data from high-dose cohort (4x low-dose) potentially by YE16"
    "with a potential update by YE16"
    Het rekensommetje leert dat ze in november/december van hooguit 3 patiënten de zogeheten topline resultaten kunnen presenteren. En dat er op de ASH door QURE niks gepresenteerd zal worden (of het moeten deze resultaten zijn).

    "QURE will also allow enrollment of >5 pts, noting high interests from patients seen to date."
    Hoe moeten we de bijzin "noting high interests" lezen? Ik neem toch aan dat er een bedrijfsstrategie achter zit. Verkorten DMC reviewtijd in trial?
  4. T. Montana 13 januari 2016 15:55

    • T. Montana

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    Voor een nieuwe CEO is nu wsch het beste moment om de timelines wat voorzichtiger in de markt te zetten. Eerlijk gezegd word ik er niet koud of warm van. In deze biz zijn ze altijd te laat. Het speciaal vernoemen van de "high interest" is misschien een signaal naar investeerders dat QURE geen recruitment problemen zal hebben; met alle concurrentie is dat een gewettigde angst.
  6. T. Montana 29 februari 2016 23:47

    • T. Montana

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    Dank flosz. Ik wacht eigenlijk vooral op "full ownership" van het Sanfilippo program. Dat was aangekondigd in september. Schrijft geen van de analisten daar iets over?
  7. Prof. Dollar 1 maart 2016 12:18

    • Prof. Dollar

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    quote:

    T. Montana schreef op 29 februari 2016 23:47:

    Dank flosz. Ik wacht eigenlijk vooral op "full ownership" van het Sanfilippo program. Dat was aangekondigd in september. Schrijft geen van de analisten daar iets over?
    In de presentatie van vorige week heeft Soland aangegeven dat QURE nu verantwoordelijk is voor de vier behandelde patiënten in het Sanfilippo B programma.
  10. T. Montana 2 maart 2016 00:46

    • T. Montana

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    is het een snelweg of een frans landweggetje en waar zijn de kilometerpaaltjes met een ongeveer datum erop; dat helemaal oranje is een positief signaal, maar als ze het echt helemaal hadden binnengehaald was er echt wel een persbericht aan besteed ...
  11. Prof. Dollar 2 maart 2016 10:02

    • Prof. Dollar

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    quote:

    T. Montana schreef op 2 maart 2016 00:46:

    is het een snelweg of een frans landweggetje en waar zijn de kilometerpaaltjes met een ongeveer datum erop; dat helemaal oranje is een positief signaal, maar als ze het echt helemaal hadden binnengehaald was er echt wel een persbericht aan besteed ...
    Haha. Sorry. Deden ze dat maar!

    Het kan haast niet anders of we krijgen straks een hele uitvoerige 4Q (en final year) + 1Q2016 rapportage.
  13. flosz 5 april 2016 11:20

    • flosz

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    Piper Jaffray analyst Joshua Schimmer reiterated an Overweight rating on uniQure NV (NASDAQ: QURE) yesterday and set a price target of $26. The company’s shares closed yesterday at $13.30, close to its 52-week low of $10.61.
    www.analystratings.com/2016/04/05/uni...

    Jefferies
    uniQure N.V. (QURE): Narrower 4Q15 Loss; AMT-060 High-Dose Data in Hemophilia B by ~YE16
    Rating BUY
    Price Target $36.00
    Price $12.75
    www.iex.nl/Forum/Topic/1286603/219/Wa...
  15. flosz 5 april 2016 16:56

    • flosz

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    Oppenheimer $QURE: Juni 2015:$47, Sept. 2015:$50 (San.B) -> April 2016:$23
  18. T. Montana 20 april 2016 22:20

    • T. Montana

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    JEFFERIES
    Bluebird (BLUE): Interim Lenti-D Data in CALD Somewhat Mixed, But Expecting Further Improvement
    Rating BUY
    Price Target $84.00
    Price $47.72
    Bloomberg NASDAQ: BLUE

    Key Takeaway
    Interim data of Lenti-D Ph2/3 Starbeam study in CALD were presented at AAN today. We see 16/17 stabilization in the neurological function scores as impressive. While Loes score progression and re-emergence of gadolinium enhancement in some pts at 12mon raise some questions on potential disease stabilization, we expect to see continued improvement at 24mon based on sustained VCN, protein expression and underlying science.

    None of 17 cerebral adrenoleukodystrophy (CALD) pts treated with Lenti-D experienced major functional disabilities (MFDs) with 6-24mon follow-up. Recall that Lenti-D (gene therapy for CALD) Ph2/3 Starbeam study enrolled 17 pts with % pts without MFDs at 24mon as the primary endpoint. MFDs include 6 serious symptoms (loss of communication, no voluntary movement, cortical blindness, tube feeding, wheelchair required and total incontinence) as measured in the neurological function scores (NFS, 0 means normal and the higher score, the more severe the symptom). With variable follow-up periods (=6mon for all 17 pts, 12-24mon for 14 pts including 3 pts with full 24mon follow-up), none of the 17 pts experienced MFDs as of Mar 31’16, suggesting the 3 pts with sufficient follow-up have already met the primary endpoint. With further follow-up, we expect to see ~80% pts to meet the primary endpoint at 24mon (link), comparable to current standard of care (allo-HSCT, allogeneic hematopoietic stem cell transplantation).

    Stabilization in NFS score appears better vs. natural history study of allo-HSCT treated pts and our KOL expectation. Sixteen of 17 pts (94%) achieved stabilization in NFS score (change of <3 points and an absolute NFS = 4). Two pts had NFS increase of 1 (worsening of disease) and one pt had NFS increase of 5 in 6mon after treatment. The results appear better vs. our KOL expectations (50% disease stabilization in 9-12mon) and the natural history study conducted by the company (ALD-101, 70% NSF stable at 24mon).

    While Loes score progression (measuring myelin degeneration using MRI) and re-emergence of gadolinium enhancement (GdE+, indicator of neuroinflammation) in some pts at 12mon raised some questions on potential disease stabilization, we expect to see continued improvement at 24mon. Fourteen of 17 pts achieved stabilization in Loes score (defined as change of <5 points and an absolute Loes score = 9). Note that several pts had Loes score increase of ~3-4 within 12mon follow-up. Although resolution of GdE+ was achieved in 16/17 pts at 6mon, re-emergence of GdE+ was observed in 5/14 pts at 12mon. GdE enhancement was resolved in 2 of 5 pts at 18mon follow up. Although it is unclear if GdE+ would be resolved for the remaining 3 pts, we expect to see continued improvement for both Loes score and GdE at 24mon because: 1) sustained vector copy numbers were observed for nearly every single pt throughout treatment period, 2) ALD protein expression showed steady increase for all pts, 3) natural history of the disease suggests rapid progression upon diagnosis (usually death within 5 yr) while it would require meaningful time (>6-9mon according to our KOL) for stem cells to differentiate to microglia cells in order to prevent the ongoing demyelination. Our KOL feedback also suggests improved disease stabilization over time based on stem cell transplant (70-80% at 24mon vs. 50% at 12mon), 4) one case presented at the meeting suggested Loes score decreased to baseline with longer followup (2 at the baseline, 3 at 12mon and 2 at 24mon).

    Safety profile of Lenti-D continues to be consistent with myeloablation treatment with 6mon+ follow-up vs. data in the abstract, highlighting its advantage as autologous transplantation. One possibly treatment-related SAE (grade 3 BK virus cystitis, common in bone marrow transplantation, link) and treatment related AE (grade 1 tachycardia) were reported and resolved with standard measures. Integration site analysis showed no dominant clones. Consistent with autologous transplantation, Lenti-D treatment led to no graft versus host disease (GvHD) vs. 45-75% in allo-HSCT-treated pts.
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