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Wat is de waarde van uniQure?

7.806 Posts
Pagina: «« 1 ... 331 332 333 334 335 ... 391 »» | Laatste | Omlaag ↓
  1. pim f 12 februari 2019 03:10
    quote:

    flosz schreef op 11 februari 2019 15:28:

    Hee pim, lang geleden!
    Leuk je weer “te zien”.
    Toen ik jouw naam zag staan bij uniQure, wist ik dat het goed zat...

    Na Crucell is dit weer een superbedrijf en ik denk dat de Pfizers van deze wereld al klaar staan met een enorme zak geld. Maar zo relatief kleinschalig bovenop de ontwikkelingen zitten zoals jij doet, is wel zo intellectueel bevredigend! Echter, als de grotere hemofilie-trial net zulke opwindende resultaten geeft als deze kleine, dan gaan we echt wat beleven. Leuk!
  5. Tex Mex 14 februari 2019 18:57
    quote:

    colt schreef op 14 februari 2019 17:38:

    ARK Invest-analist vertelde eerder aan Investors Business Daily dat de hemofilie-markt in 2025 $ 26 miljard waard zou kunnen zijn.
    Maar ik ben bang dat daarmee de hele hemofilie markt bedoeld wordt, of niet? Ik denk dat het goed is om het onderscheid tussen Hemofilie A, B en ziekte Van Willibrand in de gaten te blijven houden. Het aantal hemofilie B patiënten verhoudt zich ongeveer in een 1:5 verhouding tot het aantal hemofilie A patiënten. Dat betekent dat de markt voor uniQure (vooralsnog alleen een product voor hemofilie B in de kliniek) wel wat kleiner is dan het genoemde bedrag. Maar laten we hopen dat Hemofilie A dit jaar ook de klinische fase kan betreden en een succes gaat worden. Ik acht de kans op succes zeker niet denkbeeldig. Ook al is uniQure in de tijd geen voorloper op het gebied van gentherapie in Hemofilie A, ik zie wel duidelijk additionele waarde in de benadering die uniQure kiest in de behandeling van deze ziekte. Dus wellicht niet first to market, toch voordelen die het middel erg competitief kunnen laten zijn.
  9. Prof. Dollar 15 februari 2019 14:44
    Een week geleden stipte ik hier de nog niet officiële targets amyotrophic lateral sclerosis (ALS) en frontotemporal dementia (FTD) van QURE aan. Vandaag een persbericht over deze programma's.

    uniQure Announces Publications on AAV Gene Therapy Approach to Treating Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) in the Journal Molecular Therapy Nucleic Acids tools.eurolandir.com/tools/Pressrelea...
  11. forum rang 10 rationeel 20 februari 2019 14:48
    A Rare Disease Day Policy Event (the Second High-Level Event of the NGO Committee for Rare Diseases) will take place tomorrow at the United Nations in New York. Watch online to hear discussions on steps being taken to make rare diseases a global health priority, including a call for a UN resolution on rare diseases. The exceptional event, taking place to mark the occasion of Rare Disease Day 2019, is organised by EURORDIS, Ågrenska and Rare Diseases International.

    www.eurordis.org/news/watch-online-to...
  12. Prof. Dollar 22 februari 2019 10:35
    quote:

    flosz schreef op 21 februari 2019 13:58:

    Dr. Higgins is the Vice President of Clinical Development at uniQure. He is responsible for the early development of clinical gene therapy trials for neurological disorders www.blogtalkradio.com/help4hd/2019/02... #HuntingtonsDisease $QURE
    Wow! Goed luisteren vanaf minuut twintig: "[..] after treatment of AMT-130 (Huntington), we see a restoration of neuron function in the putamen". Het enthousiasme van Dr. Higgins is aanstekelijk. En niet onterecht mocht het straks klinische waarde hebben; dit kan wereldnieuws worden.

    Deze data worden waarschijnlijk komende week gepresenteerd tijdens de 14th Annual HD Therapeutics Conference in Palm Springs, California. www.regonline.com/builder/site/defaul...

    Aan het eind van die week volgt vrijdags een webcast van QURE tijdens de Leerink Global Healthcare Conference; March 1, 2019, 9:30 a.m. ET/ 15:30 CET. wsw.com/webcast/leerink32/register.as...

    En anderhalve week later een webcast tijdens de Cowen & Company 39th Annual Healthcare Conference; March 12, 2019, 10:40 a.m. ET/ 15:40 CET. wsw.com/webcast/cowen52/register.aspx...
  14. T. Montana 23 februari 2019 21:26
    Dit: Wall Street Journal, een uur geleden
    Roche Nears Deal to Buy Spark Therapeutics for Close to $5 Billion
    Biotech deal would represent a big premium, given Spark had a market value of just under $2 billion as of Friday’s close

    By Dana Cimilluca, Dana Mattioli and Jonathan D. Rockoff
    Feb. 23, 2019 1:25 p.m. ET
    Roche Holding AG RHHBY 0.51% is nearing a deal to buy Philadelphia biotechnology company Spark Therapeutics Inc., according to people familiar with the matter, as the Swiss drugmaker seeks to expand its presence treating hemophilia.

    A deal for Spark could be announced Monday—if not sooner—at a price tag of nearly $5 billion, some of the people said. That would represent a big premium, given Spark had a market value of just under $2 billion as of Friday’s close. As always, the deal could still fall apart before the companies manage to make it official.

    There was at least one other bidder for Spark as of Friday, some of the people said, though it isn’t clear who.

    Therapies that replace a defective gene with a healthy one are an emerging class of treatment pioneered by companies including Spark. It was founded in 2013 out of gene-therapy research at Children’s Hospital of Philadelphia.

    Doctors and patients have been looking forward for years to gene therapies treating intractable inherited diseases, but development of the therapies has proven more challenging than initially thought, including the death in 1999 of a young man who received an experimental gene therapy.

    Yet development of the therapies appeared to turn a corner in recent years, and big companies like Pfizer Inc. and Novartis AG have been making moves to offer such treatments. Pfizer has partnered with Spark on development of a hemophilia B treatment. Last year, Novartis paid $8.7 billion for gene-therapy developer AveXis.

    In 2017, Spark’s Luxturna, which treats a condition that can cause blindness, was the first gene therapy for an inherited disease to receive Food and Drug Administration approval. Spark is also developing gene therapies to treat the inherited blood disorder hemophilia.

    The company generated just $64.7 million in revenue last year and a net loss of $78.8 million. Even though that represents dramatic improvement from the prior year on both counts, it underscores how much Roche is having to pay up to secure the acquisition.

    Hemophilia is a new and emerging category for Roche. In 2017, the U.S. Food and Drug Administration approved the company’s hemophilia A treatment Hemlibra, which analysts expect will have billions of dollars in yearly sales.

    In January, the company described Hemlibra as one of its biggest growth drivers, with sales surpassing CHF100 million ($100 million) in the fourth quarter alone.

    If Spark’s hemophilia gene therapies pan out, Roche would be able to expand its offerings in the area, helping it compete with market rivals like Takeda Pharmaceutical Co. Ltd. and Sanofi SA .

    Among the challenges confronting companies like Roche seeking to sell the new gene therapies is gaining reimbursement. Spark has said it plans to sell Luxturna in the U.S. at a cost of $850,000 a patient, but it wants to offer partial refunds if patients don’t meet recovery targets.

    Roche is among a number of big drug companies hungry for biotechs that can help restock their pipelines and portfolios. There have been a raft of such deals already this year, including Bristol-Myers Squibb Co.’s roughly $74 billion planned purchase of Celgene Corp. and Eli Lilly & Co.’s agreement to pay $8 billion for cancer specialist Loxo Oncology Inc.

  15. T. Montana 23 februari 2019 21:48
    Jullie kunnen zelf allemaal het rekensommetje maken, maar omdat het er zo leuk uitziet: voor dezelfde premie gaat QURE voor meer dan $100 de deur uit - ergens tussen de 100-125. Niet gek. Als er al gegadigden zijn en je kan je niet voorstellen dat dat niet zo is dan is dit prijskaartje een aardig steuntje in de rug in de onderhandelingen. Zelfde premie vind ik uitstekend, maar ik denk dat ze het niet voor minder dan $5 miljard van de hand doen. Ook best!
  16. Prof. Dollar 23 februari 2019 22:02
    quote:

    T. Montana schreef op 23 februari 2019 21:48:

    Jullie kunnen zelf allemaal het rekensommetje maken, maar omdat het er zo leuk uitziet: voor dezelfde premie gaat QURE voor meer dan $100 de deur uit - ergens tussen de 100-125. Niet gek. Als er al gegadigden zijn en je kan je niet voorstellen dat dat niet zo is dan is dit prijskaartje een aardig steuntje in de rug in de onderhandelingen. Zelfde premie vind ik uitstekend, maar ik denk dat ze het niet voor minder dan $5 miljard van de hand doen. Ook best!
    Haha, ik was met hetzelfde rekensommetje bezig. En dan te bedenken dat QURE waardevolle troeven in handen heeft: schaalbare productiefaciliteit, vele patenten, ervaring/deskundigheid, beloftevolle pipeline, volledig eigenaar van programma's...
  17. Vitavita 25 februari 2019 08:44
    .. en nu is het definitief ;-))

    www.roche.com/de/investors/updates/in...

    Basel, 25. Februar 2019
    Roche enters into definitive merger agreement to acquire Spark Therapeutics

    - Roche to acquire Spark Therapeutics for US$ 114.50 per share
    - Spark Therapeutics is a leader in discovering, developing and delivering gene therapies with one commercial asset and four programmes currently in clinical trials
    - Transaction expected to close in Q2, 2019

    Roche (SIX: RO, ROG; OTCQX: RHHBY) and Spark Therapeutics, Inc. (NASDAQ: ONCE) today announced that they have entered into a definitive merger agreement for Roche to fully acquire Spark Therapeutics at a price of US$ 114.50 per share in an all-cash transaction. This corresponds to a total transaction value of approximately US$ 4.3 billion on a fully diluted basis. This price represents a premium of approximately 122% to Spark Therapeutics’ closing price on 22 February 2019 and a premium of approximately 19% to Spark Therapeutics’ 52 week high share price on 9 July 2018. The merger agreement has been unanimously approved by the boards of Spark Therapeutics and Roche.

    Under the terms of the merger agreement, Roche will promptly commence a tender offer to acquire all outstanding shares of Spark Therapeutics common stock, and Spark Therapeutics will file a recommendation statement containing the unanimous recommendation of the Spark Therapeutics board that Spark Therapeutics’ shareholders tender their shares to Roche.

    Spark Therapeutics, based in Philadelphia, Pennsylvania, is a fully integrated, commercial company committed to discovering, developing and delivering gene therapies for genetic diseases, including blindness, haemophilia, lysosomal storage disorders and neurodegenerative diseases.

    Spark Therapeutics’ lead clinical asset is SPK-8011, a novel gene therapy for the treatment of haemophilia A, which is expected to start Phase 3 in 2019. Spark Therapeutics also has SPK-8016 in a phase 1/2 trial aimed at addressing the haemophilia A inhibitor population. Additionally, Spark Therapeutics was the first company to receive FDA approval for a gene therapy for a genetic disease in 2017. LUXTURNA® (voretigene neparvovec-rzyl), a one-time gene therapy product indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy is currently marketed in the US by Spark Therapeutics. The European Commission granted marketing authorisation for LUXTURNA in 2018.

    Spark Therapeutics’ additional clinical assets include: SPK-9001, an investigational gene therapy for the potential treatment of haemophilia B in Phase 3 and SPK-7001 for choroideremia in Phase 1/2. The company is also developing SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease (a form of Batten disease) which are expected to be ready for clinical development in 2019, as well as additional preclinical programmes for Huntington's disease and Stargardt disease.

    Commenting on the transaction, Severin Schwan, CEO of Roche, said, “Spark Therapeutics’ proven expertise in the entire gene therapy value chain may offer important new opportunities for the treatment of serious diseases. In particular, Spark Therapeutics’ haemophilia A programme could become a new therapeutic option for people living with this disease. We are also excited to continue the investments in Spark Therapeutics’ broad product portfolio and commitment to Philadelphia as a center of excellence.” Spark Therapeutics’ will continue its operations in Philadelphia as an independent company within the Roche Group.

    “As the only biotechnology company that has successfully commercialised a gene therapy for a genetic disease in the US, we have built unmatched competencies in the discovery, development and delivery of genetic medicines. But the needs of patients and families living with genetic diseases are immediate and their needs vast,” said Jeffrey D. Marrazzo, CEO of Spark Therapeutics. “With its worldwide reach and extensive resources, Roche will help us accelerate the development of more gene therapies for more patients for more diseases and further expedite our vision of a world where no life is limited by genetic disease.”

    Terms of the Agreement

    Under the terms of the merger agreement, Roche will promptly commence a tender offer to acquire all of the outstanding shares of Spark Therapeutics’ common stock at a price of US$ 114.50 per share in cash. The closing of the tender offer will be subject to a majority of Spark Therapeutics’ outstanding shares being tendered in the tender offer. In addition, the transaction is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.

    Following completion of the tender offer, Roche will acquire all remaining shares at the same price of US$ 114.50 per share through a second step merger. The closing of the transaction is expected to take place in the second quarter of 2019.

    Citi is acting as financial advisor to Roche and Davis Polk & Wardwell LLP is acting as legal counsel to Roche. Centerview Partners is acting as financial advisor to Spark Therapeutics and Goodwin Procter LLP is acting as legal counsel to Spark Therapeutics. Cowen also acted as a financial advisor in this transaction to Spark Therapeutics.

  18. Vitavita 25 februari 2019 09:12
    ....
    Roche already sells the bispecific hemophilia A antibody Hemlibra, which currently has an edge over existing treatments, as it doesn't trigger factor VIII antibody formation, which makes existing blood clotting factor VIII replacement therapies inefficient in about 25% of hemophilia A patients. Hemlibra binds to factor IXa and factor X bridging factor VIII function required for effective homeostasis. Gene therapies, however, require just a single administration at best and thus could outcompete Roche's potential blockbuster drug in the longer term. As FDA Commissioner Scott Gottlieb announced in May 2018 he wanted to accelerate approval for factor VIII gene therapies, Roche had to react.

    The take over will take place according to Roche's proven recipe that grants as much scientific freedom to companies taken over as possible. Spark, however, is facing much competition, as other players Biomarin Pharma (Phase III), and uniQure NV (hemophilia B) have prospective treatments in the works.

    "Spark Therapeutics' haemophilia A programme could become a new therapeutic option for people living with this disease," said Severin Schwan, Chief Executive of Roche.

    Spark's stock tumbled last year after announcing two out of 12 patients showed an unfavourable immune response when treated with a higher dose of Spark's haemophilia therapy SPK-8011. The drug is expected to enter Phase III testing this year. It is also working on a therapy for haemophilia B, as well as treatments for Pompe disease, blindness-causing choroideremia and Huntington's disease.

    european-biotechnology.com/up-to-date...
  19. NielsjeB 25 februari 2019 13:25
    With permission to post.Nice layout by HCW $QURE -excellent analysis -indications that could be in the clinic over next 24 months, although suspect compelling POC data from Hemophilia A & HD might be sufficient for a competitive bid in excess of what Spark is currently commanding

    twitter.com/semodough/status/11000079...
    Bijlage:
7.806 Posts
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