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Home  /  Forum  /  uniQure  /  uniQure Announces Financial Results for the First Quarter 2015

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uniQure Announces Financial Results for the First Quarter 2015

24 Posts
Pagina: 1 2 »» | Laatste | Omlaag ↓
  1. flosz 11 juni 2015 12:34
    Amsterdam, the Netherlands, June 11, 2015 — uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced financial results for the first quarter ending March 31, 2015, and provided an update on multiple gene therapy programs.

    Pipeline Updates

    Hemophilia B: The Company has commenced screening patients in its first clinical trial site in Germany for its Phase I/II study of AMT-060 in hemophilia B patients and anticipates providing a preliminary readout of safety and efficacy data in the second half of 2015.
    Sanfilippo B: All patients have been dosed in the collaborator-sponsored Sanfilippo B program with Institut Pasteur. One year follow-up results will be available in the second half of 2015 and presented at a relevant scientific meeting.
    Glybera®: uniQure remains on target to initiate in early 2016 a pivotal clinical study for Glybera® (alipogen tiparvovec) to support a future regulatory submission in the U.S.
    Parkinson’s Disease: uniQure’s investigator-sponsored Phase I clinical study of glial cell line-derived neurotrophic factor (GDNF) in Parkinson’s disease, led by Krystof Bankiewicz, MD, PhD, at the University of California, San Francisco, has completed enrolment of its first dosing cohort and will be commencing dosing of its second cohort in the third quarter of 2015.

    Corporate Highlights

    Strategic Collaboration with Bristol-Myers Squibb

    On May 26, 2015, uniQure announced the closing of its collaboration with Bristol-Myers Squibb Company, triggering an initial $50 million upfront payment. The partnership, announced April 6, 2015, provides BMS with exclusive access to uniQure’s gene therapy technology platform for multiple targets in the cardiovascular space. The collaboration includes uniQure’s proprietary congestive heart failure program targeting S100A1, a calcium sensor and master regulator of heart function, and the companies will collaborate on up to nine other gene therapy targets addressing a broad range of cardiovascular disease and other target-specific disease areas.

    On June 10, 2015, the shareholders of uniQure approved the equity component of the BMS collaboration, and the Company will issue to BMS an initial tranche of approximately 1.1 million ordinary shares, at a purchase price of $33.84 per share, for aggregate consideration of approximately $38 million. The transaction is expected to close on June 12, 2015.

    Business Development

    Collaboration with Treeway: In January 2015, uniQure entered into a license and collaboration agreement with Treeway B.V., a private company founded by entrepreneurs Bernard Muller and Robbert Jan Stuit, both diagnosed with amyotrophic lateral sclerosis, or ALS, to develop a gene therapy treatment for ALS.

    Collaboration with Synpromics: In January 2015, uniQure entered into a collaborative license agreement with Synpromics Limited to strengthen its technology platform with respect to therapeutic indications that require high-level therapeutic gene expression or comprise large therapeutic genes. uniQure will exclusively own the results of this collaborative effort.

    Other Corporate Highlights

    Follow-on Public Offering: On April 15, 2015, uniQure completed a follow-on public offering of 3,000,000 ordinary shares at $29.50 per ordinary share. After deducting the underwriting discounts and other estimated offering expenses payable by uniQure, the net proceeds to the Company were approximately $82.5 million (€77.9 million).
    Infrastructure: In the first quarter of 2015, the Company completed the build-out of its 53,000 sq. ft. manufacturing facility in Lexington, Massachusetts and remains on target to achieve GMP readiness by the end of 2015.
    Human Resources: In January 2015, uniQure announced the appointment of Matt Kapusta to Chief Financial Officer. On June 10, the shareholders of uniQure approved the appointments of Mr. Kapusta to the Management Board and Philip Astley-Sparke to the Supervisory Board. In addition, uniQure leadership accepted the request by Chief Commercial Officer Hans Christian Rohde to proceed with early retirement effective August 1, 2015.
    “The completion of our landmark collaboration with Bristol-Myers Squibb begins an exciting new chapter for uniQure by advancing our goal to bring the promise of gene therapy to the millions of patients with cardiovascular disease,” Jörn Aldag, uniQure Chief Executive Officer, commented. “In conjunction with our successful follow-on offering, we also now have the necessary financial resources to achieve proof-of-concept in hemophilia B, Sanfilippo B and Parkinson’s disease and further the clinical development of these important programs. We also plan to expand our proprietary pipeline in rare liver/ metabolic and CNS diseases by advancing several preclinical product candidates and pursuing acquisitions and in-licensing opportunities.”

    Mr. Aldag added: “All of us at uniQure would like to thank Hans Christian for his leadership and collaboration over the last three years and we wish him the best for his well-deserved retirement.”

    Financial Highlights

    As of March 31, 2015, the Company held cash and cash equivalents of €43.2 million, compared with €53.2 million as of December 31, 2014. Licensing and collaboration revenues for the three months ended March 31, 2015 were €1.1 million, compared with €1.2 million for the comparable period in 2014. The majority of 2015 revenues are related to development activities that were reimbursable by Chiesi under the Company’s co-development agreement for hemophilia B.

    Research and development expenses were €10.1 million for the three months ended March 31, 2015, compared with €6.2 million for the comparable period in 2014. The increase is related to the initiation of our Phase I/II clinical study of AMT-060 in hemophilia, additional development and clinical activities required to support the planned commercial launch of Glybera, the continued progression of uniQure’s other product candidates and increased activity in our U.S. facility.

    Selling, general and administrative expenses were €4.2 million for the three months ended March 31, 2015, compared with €2.3 million or the comparable period in 2014. The increase is primarily due to expenses related to consultants and professional fees associated with business development and other general and administrative activities. The net loss for the first quarter 2015 was €12.6 million, or €0.69 per share, compared with €7.8 million, or €0.52 per share, for the first three months of 2014.



    www.uniqure.com/news/254/182/uniQure-... $QURE
  2. flosz 12 juni 2015 18:00
    Dd 11-05-2015:

    AMT-021 for Acute Intermittent Porphyria

    We are developing AMT-021 as a gene therapy for acute intermittent porphyria, or AIP, a severe liver disorder. AMT-021 consists of an AAV5 vector carrying a therapeutic porphobilinogen deaminase, or PBGD, gene that we exclusively license from Applied Medical Research Center of the University of Navarra in Spain. Our former collaborator Digna Biotech has completed a Phase I

    clinical trial of AMT-021 in eight patients in Spain. The collaboration achieved its primary goal in completing a successful Phase I study, thereby establishing the preliminary safety profile of liver-directed gene therapy with AAV5 that we expect will support future clinical studies.

    Phase I Clinical Trial Sponsored by Digna Biotech

    Digna Biotech conducted a multicenter, open label, prospective, interventional, single dose, dose-escalation Phase I clinical trial to investigate the safety and tolerability of AMT-021 in eight patients with severe AIP. Digna Biotech conducted this clinical trial at two sites in Spain. There were four dosing cohorts in the trial, with two patients per cohort. Digna Biotech monitored all patients for one year following treatment. The primary objective of this Phase I clinical trial was to assess the safety of systemic administration and to determine the maximum tolerated dose of AMT-021. Secondary objectives included measuring urinary levels of toxic metabolites to determine whether these metabolites can be used as a biomarker of potential treatment effect. Preliminary analysis of the one-year follow-up data indicates the safety and successful transduction of liver cells with the PBGD gene. Key findings were as follows:

    · There were no liver enzyme perturbations.

    · There was no evidence of cellular immune response against the AAV vector or the PBGD gene.

    · Vector genomes were detected in patients’ liver biopsies obtained under one year after vector injection, indicating that AMT-021 sustainably transduced human liver cells.

    Digna has advised us that there were four serious adverse events in this trial; however the events were determined by the investigator not to be treatment related. Digna further reported that there were no treatment-related adverse events or liver events related to AMT-021. Digna did not observe a reduction in the urinary levels of toxic metabolites in trial participants that might have served as a surrogate marker for efficacy. We believe that this result may suggest that a relatively high level of transgene expression will be required for a gene therapy to provide a clinical benefit in this indication. This contrasts with an indication such as hemophilia, in which the near or total absence of a protein in the patient means that a relatively low level of gene expression may result in a clinical benefit. In light of the absence of dose-limiting toxicities in the Phase I clinical trial, we are currently assessing whether higher dose levels or a more potent vector may be the relevant next step in the project. Under our consortium agreement with Digna Biotech and the other consortium members, we have an exclusive right to use all data related to the program.

    tinyurl.com/okdtbkp

    Dd 11-06-2015:

    Acute intermittent porphyria (AIP). We have incurred costs related to the development and manufacture of clinical supplies of AMT-021 for the treatment of AIP provided to our collaborator, Digna Biotech, for its ongoing Phase I clinical trial in this indication.

    tinyurl.com/prh5v5g
  4. [verwijderd] 12 juni 2015 19:34
    Voor de veligheid van de Hemofilie B trial is het super. Maar er werd bij geen van de doses een aanwijzing voor effectiviteit gevonden. Dus moeten ze of hogere doses geven of een meer 'potente' vector. In beide gevalen moet zo'n phase 1 trial overgedaan worden. Dus voor de onwikkleing van deze indicatie is dit een vertragende uitkomst. In apen wekte deze dosering namelijk wel:
    cohPBGD ) administered intra- venously as part of a safety program to start a clinical study in patients with AIP. Macaques injected with either 1x1013 or 5x 1013 vector genomes/kg of clinical-grade rAAV5-
    cohPBGD were monitored by standardized clinical parameters, and vector shedding was analyzed. Liver transduction efficacy, biodistribution, vector integration, and histopathology at day 30 postvector administration were determined. There was no evidence of
    acute toxicity, and no adverse effects were observed. The vector achieved efficient and homogenous hepato-cellular transduction, reaching transgenic PBGD expression levels equivalent to 50% of the naturally expressed PBGD mRNA. No cellular immune response was detected against the human PBGD or AAV capsid proteins. Integration site analysis in transduced liver cells revealed an almost random integration pattern supporting the good safety profile of rAAV5-cohPBGD. Together, data obtained in nonhuman primates indicate that rAAV5-cohPBGD
    represents a safe therapy to correct the metabolic defect present in AIP patients.
    HUMAN GENE THERAPY 24:1007–1017 (December 2013)

  5. flosz 23 juni 2015 11:19
    quote:

    flosz schreef op 11 juni 2015 12:34:

    Sanfilippo B: All patients have been dosed in the collaborator-sponsored Sanfilippo B program with Institut Pasteur. One year follow-up results will be available in the second half of 2015 and presented at a relevant scientific meeting.

    Mbt Sanfilippo B:
    In art.spreekt men over gebruik AAV10 vector, QURE/Pasteur program. maakt gebruik van AAV5. Foutje? Tevens vermelding aantal patiënten:4

    Institut Pasteur (France) / Sanfilippo Type B
    The Pasteur Institute in France has recently completed treatment of 4 patients with Sanfilippo Type B as part of a phase I/II clinical trial for a gene therapy product. This trial involved an AAV10 vector delivered to the brain intracerebrally by a neuro-surgical procedure and is very similar in its approach to the Lysogene treatment, but focused on type B instead of A.

    Results are expected to be published later in 2015.
    tinyurl.com/o7t4xeg

    AAV5 vector for liver-directed gene therapy. AAV5 is an important part of our gene therapy platform and the vector used in our hemophilia B and Sanfilippo B gene therapies, www.uniqure.com/news/198/182/uniQure-...
  7. [verwijderd] 23 juni 2015 12:18
    Ja denk echt dat dat een foutje is met die AAV10.
    Wel goed om te weten dat er een concurrerend consortium is:
    Abeona Therapeutics & Nationwide Children's Hospital (USA) / Sanfilippo Types A and B

    Abeona Therapeutics is a biotech formed specifically to develop two products; ABX-A and ABX-B, for the treatment of Sanfilippo subtypes A and B. The program is building on successful pre-clinical studies conducted by Drs Fu (Type B) and McCarty (Type A) at the Nationwide Children's Hospital in Columbus, Ohio.

    Therapie kan intravenous en hoeft niet in de hersenen of ruggemerg toegediend te worden zoals de QURE therapie. Maar goed dit is een minor detail. Effectiviteit is het belangrijkste!

    Patients on the clinical trial will be administered the gene therapy product intravenously using an AAV9 (Adeno-Associated Viral serotype 9) vector, which has the ability to cross the Blood-Brain-Barrier.

    Hun trial start pas ergens in 2015! Dus $QURE heft hier wel een voorsprong.

  9. flosz 23 juni 2015 15:43
    Goed overzicht(en meer):
    twitter.com/bioterp/status/6133359513...

    June 23, 2015
    Zack Fink

    This week I have been giving some thought to the Sanfilippo B gene therapy program (AMT-110) that uniQure has rights to in-license from Institut Pasteur. With AMT-110 data expected within the next 6 months, I decided it would be prudent to give a brief overview of gene therapy for lysosomal storage diseases, especially with Plasmatech Pharmaceuticals (PTBI) completing their reverse merger with the private gene therapy company Abeona Therapeutics (ABEO) last week.

    Lysosomal Storage Disease Overview

    Lysosomal storage diseases (LSDs) are inherited metabolic storage diseases characterized by progressive accumulation of undigested materials in the lysosomes of affected cells – a consequence of a genetic defect resulting in missing/improperly functioning digestive enzyme(s). LSDs are an ideal class of disorders to target with gene therapy due to their monogenic nature and clearly defined biology. Further, modest enzymatic activity may be sufficient for clinical efficacy in storage disease with CNS involvement – see Bluebird’s Adrenoleukodystrophy data [1,2]. LSDs with central nervous system (CNS) involvement are the first group of LSDs to be treated with gene therapy most likely because of the poor existing standard of care – limited protein replacement therapy (ERT/SRT) and hematopoietic stem cell transplantation (HSCT). A comparison of the advantages and disadvantages of each therapeutic option for LSDs can be seen below, along with a table of LSDs treated with ERT such as Shire’s (SHPG) Elaprase (idursulfase) and Sanofi/Genzyme’s (SNY) Aldurazyme (lardonidase) [3]:

    Protein replacement therapies tend to be ineffective for the CNS disease in LSDs because of their inability to cross the blood-brain barrier. In order to promote transgene expression in the CNS, in-vivo gene therapy for LSDs with CNS involvement primarily rely on three routes of administration: intravenous delivery (IV), CSF injection, or direct injection into the brain. In addition, ex-vivo gene therapy in these indications rests on traditional HSCT with modified stem cells, resulting in modified progenitor cells migrating to the CNS. There is, however, clinical proof-of-concept data in LSD for CNS involvement in both in-vivo and ex-vivo gene therapy.

    Clinical Proof of Concept for LSD Gene therapy

    In 2013, Biffi et al. were the first to publish clear clinical proof-of-concept data for a gene therapy in an LSD with CNS involvement [4]. Investigators used a lentiviral vector for ex-vivo transduction of stem cells from three patients diagnosed with late infantile metachromatic leukodystrophy (LI-MLD).

    MLD is a caused by mutations in the ARSA gene resulting in buildup of sulfatide in CNS cells, leading to demyelination and neurodegeneration. Late infantile MLD patients present symptoms by their third birthday and usually die within a few years of symptom onset. There are no treatments that substantially delay the progression of MLD.

    In this trial, investigators used a “control group” that consisted of disease progression data from older siblings of the enrollees, who also were diagnosed with LI-MLD. Patients demonstrated above-normal expression of ARSA throughout the hematopoietic cell lineages at one year post-treatment. Importantly, at 18-24 month follow-up all three treated patients had their disease progression halted and/or slowed compared to “controls” and a natural history cohort of untreated LI-MLD patients.

    Despite the immense read-through for the potential of ex-vivo lentiviral gene therapies in other LSDs with CNS involvement, the use of this treatment modality may be limited – the transgene is only expressed in the CNS by cells with hematopoietic lineage, such as microglia. Gene therapy for LSDs rely on individual cells expressing adequate amounts of missing protein to prevent storage buildup. In order to maximize efficacy, transgene expression should be widespread to all cells in the CNS – not just those with hematopoietic lineage. This is why much of the excitement for LSD gene therapy (and CNS gene therapy as a whole) has risen from the use of AAV vectors which have the ability to lead to widespread transduction in the CNS.

    In 2014, Tardieu et al. were the first group to publish clear clinical proof-of-concept data for in-vivo gene therapy of a LSD with CNS involvement [5]. The investigators enrolled four patients diagnosed with Mucopolysaccharidosis Type IIIA (MPSIIIA, Sanfilippo A) and treated these patients with an AAVrh.10 vector carrying the SGSH and SUMF genes. As a reminder, Sanfilippo A is caused by a mutation in the SGSH gene resulting in the typical pathophysiology seen in LSDs with CNS involvement. The investigators delivered a total dose of 7.2E11 vector genomes (vg) per patient at 12 deposits in the brain (via 6 image-guided tracks). Based on neurological and clinical evaluations, three treated patients were considered clinically stable, with the fourth showing improvement, at 52 weeks post-treatment. This contrasts the disease’s natural history.

    Lysogene obtained an exclusive license to develop AAVrh.10-SGSH-SUMF in 2013, and renamed the asset SAF-301. Lysogene is currently conducting a 5-year follow-up study of the original four patients treated with SAF-301 [6].

    Looking into the Future

    With the recent improvement in AAV vector manufacturing and delivery, I’m optimistic that within the next several years future gene therapy trials designed to treat the CNS disease involvement in LSDs could show modest clinical efficacy. These future positive clinical trial data could position gene therapy as a treatment option (not a cure) to add to the treatment arsenal for LSDs.

    Further down the road, novel AAV vectors (via rational design and directed evolution) with improved transgene cassettes are going to be moving into the clinic. These novel AAV vector/transgene cassettes are the future of in-vivo CNS gene therapy – as well as in-vivo AAV gene therapy as a whole.
    Bijlage:
  11. flosz 8 juli 2015 19:17
    Int.Conference Sanfilippo Syndrome and Related Lysosomal Storage Diseases, Geneva, Switserland cisml.org/cisml/en/

    Conference Day 2 Afternoon - Friday 27th November
    13h30-15h00 Session 3 : Specific Clinical Aspects of MPS III and New Treatment Approaches

    1) Keynote Presentation - Intra-Cerebral Administration of AAV2/5 Vector containing Human NAGLU cDNA in Children with

    MPS IIIB : Results at 12 months of a Phase I/II trial

    Prof. Marc Tardieu, Paediatric Neurology Service, INSERM, Unit 1012, University Paris-South, France
    Bijlage:
  14. flosz 14 september 2015 16:41
    EXPECTATIONS FOR UNIQURE’S NEXT DATA EVENT: SANFILIPPO GENE THERAPY RESULTS
    UniQure’s (QURE) presentation of 1-year follow-up data from patients with Sanfilippo B (MPS IIIB) and treated with the gene therapy AMT-110 is only a week away, on September 19 (here). Expectations are modest in front of what could be equivocal results for this early product candidate.

    By way of background, AMT-110 consists of an AAV5 vector carrying a therapeutic NaGlu (a-N-acetylglucosaminidase) gene. Institut Pasteur began a phase I/II study in four patients in October of 2013, and this will be the first look at 1-year results. UniQure has an agreement to acquire the clinical results and commercial rights to the program following completion of this small study.

    For a deeper look at QURE, read our previous coverage.

    First, I want to re-emphasize what I stated in a blog post on gene therapies for lysosomal storage diseases last month: that clinical proof-of-concept data does exist for gene therapies in lysosomal storage diseases for both lentiviral vectors (late infantile metachromatic leukodystrophy) and adeno-associated virus (AAV) vectors (Sanfilippo A, MPSIIIA). But, the existing results are not highly indicative.

    Privately held Lysogene tested SAF-301 (AAVrh.10-SGSH-SUMF) in 4 patients diagnosed with Sanfilippo A (MPSIIIA) in an open-label phase I/II trial that began in 2011. With these types of heterogeneous progressive neurodegenerative conditions, long-term follow-up is required to truly understand the treatment effect, if any, thus existing results from the Lysogene program should be viewed as preliminary. At one year post-treatment, investigators determined that patients 1-3 were considered clinically stable, and patient 4 was considered to have improved. In addition, neuropsychological evaluations suggested a possible improvement in behavior, attention, and sleep. Exhaustive neuropsychological evaluations performed at inclusion and 52-weeks post gene transfer are shown below [1]:

    The neuropsychological evaluations used are objective and do not provide a robust efficacy signal with 52-week follow-up. As mentioned, I suspect longer-term follow-up is required to tease out any meaningful cognitive effect. Lysogene continues towards 5-year follow-up of the original four patients treated with SAF-301, but we do have parent testimony on one subject. Karen Aiach, co-founder and CEO of Lysogene, has a diagnosed daughter who received SAF-301 in the phase I/II clinical trial. Mrs. Aiach was interviewed in Forbes in July: “[Ornella] was dosed with treatment at the age of 6, which is pretty advanced in the disease. Since then she’s been much better for the last three years from a behavioral standpoint – not hyperactive, sleeps well. That’s changed her life, and the lives of her family around her” [2]. Nevertheless, an objective signal is somewhat weak, and 5-year follow-up will be critical to determine the robustness of this efficacy.

    Enzyme replacement therapy (ERT) such as Biomarin’s (BMRN) laronidase (MPS I) and Shire’s (SHPG) idursulfase (MPS II) have been the standard-of-care for somatic MPS. Unfortunately, due to CNS involvement in MPS IIIA/B, ERT has not been shown to be effective in these indications, and hematopoietic stem cell transplant (HSCT) has not been adequately assessed for MPSIII.

    In order to determine where investors should place the efficacy hurdle for CNS symptoms and disease course for MPSIIIA/B, we turned to cognitive outcomes for MPS I patients – a type of MPS with CNS-involvement – treated with ERT and HSCT. To date, intravenous ERT for MPS I has not been sufficient to prevent decline or improve cognition in patients with MPS I [4]. HSCT, meanwhile, has been shown to improve disease severity and cognition in patients with MPS I, but It was only after multi-year follow-up that efficacy could be adequately assessed [3,5]. The importance of multi-year follow-up in determining treatment efficacy is further supported by the significant natural history variability in patients diagnosed with MPSIIIA/B [3, 6-8].

    So what does this mean for the impending AMT-110 52-week follow-up data? It’s likely much too early for a robust efficacy signal on the cognitive assessments. Biomarkers are a much more appropriate outcome to asses, such as glycosaminoglycan (GAG) accumulation and NaGlu expression. These may offer validation that the AAV5 vector has successfully transfected cells in the CNS and are functionally active.

    We sat down with uniQure last week to discuss the endpoints being used in this trial. Recall that the Pasteur group that conducted the Lysogene SAF-301 trial are the same group conducting the AMT-110 Sanfilippo trial, and they will be using the same cognitive and neuropsychological endpoints that were assessed in the Lysogene trial: Psychoeducational Profile (PEP-3), Vineland adaptive behavior scale (Vineland-II), and Toddler Behavior Assessment Questionnaire (TBAQ). GAG accumulation will not be assessed in this trial because the Pasteur group was unable to develop an adequate assay to measure GAG. UniQure stated if they do in-license the program they will work on developing a validated assay for GAG measurement.

    Upside in Uniqure Stock?
    Put directly, I don’t expect a robust cognition efficacy signal at the 52-week follow-up. I would, however, be satisfied with these results suggesting CNS disease stabilization, as seen in the SAF-301 trial. Despite AMT-110 being dosed five times higher than SAF-301, the AAV5 vector appears to be inferior to the AAVrh.10- vector for CNS applications [9-12]. Despite this, there is reason to believe that AMT-110 could show a cognitive/neuropsychological efficacy signal considering that the AMT-110 trial enrolled much younger patients (2-4 years old) than those dosed in the SAF-301 trial (5-6 years old). In addition, AMT-110 was delivered via 8 burr holes, which includes delivery to the cerebellum. SAF-301 was delivered via 6 burr holes and excluded the cerebellum.

    In terms of AMT-100’s potential value, upside (or downside) is difficult to quantify. First, multi-year follow-up are absolutely necessary to determine who should be administered these gene therapies and when in their disease progression. This was highlighted with laronidase, where a robust efficacy signal was only apparent after six-years of follow-up [13]. In attempting to quantify potential sales, we turned to revenue numbers from Shire’s Elaprase (idurasulfase). We calculated a current worldwide market penetration of idurasulfase in MPS II of ~20%. For simplicity sake, we assume a similar, aggressivemarket penetration for AMT-110; but, we also assume only 40% of patients will be eligible for AAV5 gene therapy due to pre-existing neutralizing antibodies. With a one-time WAC of $800k, U.S./EU sales peak in the $200 million-range. Using these assumptions, we feel comfortable with current available data assigning a maximum fundamental upside of ~$4.00/share for Uniqure, with a 33% probability of success and a 20% discount rate. Of note, the 33% probability of success is our pre-data estimate and assumes that AMT-110 will show a similar modest cognitive/neuropsychological signal as SAF-301.

    Perhaps more importantly, these AMT-110 results may give investors a better idea of gene therapy’s applicability in lysosomal storage diseases, a fairly broad, unmet orphan need. Success now, or further down the road, opens the opportunity in a number of CNS-driven LSDs.

    twitter.com/bioterp/status/6433988824...
  20. [verwijderd] 18 september 2015 16:23
    toch denk ik dat de massa relatief weinig ervan snapt of volgt. Die uitzending met QURE-tip zal helpen en zo ook de winst ah eind vd dag.

    Als de data mbt San F.B maandag goed in de picture is, dan kan het echt stormen.

    Er komt niet zomaar 7-8$ bij in 5 dagen!!! (ook nog voor Flosz bericht was de stijging al ingezet)
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