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Daar is-tie al: de 2Q19 update. uniQure Announces Second Quarter 2019 Results and Highlights Recent Company Progress tools.eurolandir.com/tools/Pressrelea... Geen update over de samenwerking met BMY. Mogelijk dat de SEC files later wat informatie bevat.
Prof. Dollar schreef op 29 juli 2019 13:36 :
Daar is-tie al: de 2Q19 update.
uniQure Announces Second Quarter 2019 Results and Highlights Recent Company Progress
tools.eurolandir.com/tools/Pressrelea... Geen update over de samenwerking met BMY. Mogelijk dat de SEC files later wat informatie bevat.
Wat verder opvalt is de paragraaf 'Upcoming Investor Events'. Doorgaans heeft men het over Industry Conferences (die gisteren voor de komende maand bekend zijn gemaakt) en in deze update staan de (investor) events tot half september. Kort samengevat: voortgang programma's hemofilie B en huntington voorlopen volgens planning. De eerste verwacht voor het einde van het jaar de inschrijving van patiënten af te ronden (fase III) en de tweede verwacht voor het einde van het jaar de eerste patiënt(en) te behandelen (fase I). Daarnaast worden er drie programma's klaargestoomd voor hun klinische doop: hemofilie A, Fabry en Spinocerebellar Ataxia Type 3 (SCA3). Eind dit jaar volgt waarschijnlijk meer informatie over de planning daarvan (R&D Day).
Significance of Neutralizing Antibodies in Gene Therapy in Hemophilia Sander Van Deventer Chief Scientific Officer , uniQure Conference Day 2 Thursday, August 22 2019 August 20-22, 2019 Boston, MA
Financial Highlights Cash Position: As of June 30, 2019, the Company held cash and cash equivalents of $184.1 million, compared to $208.8 million as of March 31, 2019. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2021. Revenues: Revenue for the three months ended June 30, 2019 was $2.5 million, compared to $3.1 million during the same period 2018. The decrease reflects the October 2018 termination of the development of S100A1 under our collaboration with Bristol-Myers Squibb. R&D Expenses: Research and development expenses were $24.2 million for the three months ended June 30, 2019, compared to $18.5 million during the same period 2018. The change was primarily related to increased activities associated with our ongoing clinical studies of AMT-061, the planned Phase I/II study of AMT-130, increased share-based compensation and the hiring of additional clinical and operations staff at our Lexington site. SG&A Expenses: Selling, general and administrative expenses were $7.9 million for three months ended June 30, 2019, compared to $5.9 million during the same period 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based compensation expenses. Net Loss: The net loss for the three months ended June 30, 2019 was $31.4 million, or $0.83 per share, compared to $20.6 million, or $0.57 per share during the same period 2018.
Prof. Dollar schreef op 29 juli 2019 13:57 :
[...]
Wat verder opvalt is de paragraaf 'Upcoming Investor Events'. Doorgaans heeft men het over Industry Conferences (die gisteren voor de komende maand bekend zijn gemaakt) en in deze update staan de (investor) events tot half september.
Hoi Dollar, het ontgaat mij even wat je hier precies opvallend aan vindt? Wat is de impliciete boodschap die je wil meegeven?
Niks. Enkel dat het afwijkt van de doorgaans gehanteerde communicatie. In de 2Q19 update laat men deze keer al weten welke investors events - pas over twee maand - worden aangedaan, terwijl men dat een dag eerder nalaat (enkel bericht over de komende maand). Op zich netjes om verder vooruit belangstellenden te informeren, maar deze vroege 'uitnodiging' viel mij wel op.
Why biotech companies are often at the whim of things they cannot control, in one chartwww.statnews.com/2019/07/31/why-biote... twitter.com/adamfeuerstein/status/115...
BioMarin drops lower dose of its hemophilia gene therapy as it eyes submissions by year-end - www.fiercebiotech.com/biotech/biomari... Wat denken wij er van? Gaa BMRN die goedkeuring verkrijgen?
ZÜRICH (AFN/BLOOMBERG) - Farmaceut Novartis heeft mogelijk gesjoemel met testgegevens voor een duur medicijn stilgehouden tot het middel was goedgekeurd. Dat heeft toezichthouder FDA bekendgemaakt. Het Zwitserse concern was sinds 14 maart intern op de hoogte van het probleem met de onderzoeksdata, maar meldde dat pas op 24 mei bij de FDA. Toen was het middel Zolgensma, met een prijskaartje van 2,1 miljoen dollar het duurste medicijn ter wereld, al voorzien van een stempel. De FDA onderzoekt wat er precies is gebeurd bij Novartis en bekijkt of er civiele stappen of een rechtszaak moet worden opgestart. De veiligheid en effectiviteit van het middel tegen een zeldzame spierziekte zou overigens niet in twijfel zijn. Een concurrent minder??
Het lijkt toch dat de verkoop op 17-7-2019 door CEO Kapusta de daling in gang heeft gezet, sinds transactie is de koers gestaag 25% naar beneden gedaan vanaf de top naar nu $ 55,65. Ook een andere investeerder Gilde Health Care heeft zijn positie eind van het 2e kwartaal 2019 verkocht. Is dit gewoon markt sentiment??? of speelt er wat anders.
QURE heeft afgelopen week een vacature uitgezet voor een Medical Lead Neurogenetics , Sr. Director Clinical Development. uniqure.csod.com/ats/careersite/JobDe... Past mooi bij de wetenschappelijke publicatie van eerder dit jaar: "Taken together, these findings support the feasibility of a gene therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) based on the reduction in toxicity caused by the repeat-containing C9orf72 transcripts." www.researchgate.net/publication/3310...
$QURE Announces Leadership Promotions and Executive Transition tools.eurolandir.com/tools/Pressrelea...
Goed dat ze scherp blijven op de eigen bedrijfsvoering. Lean and mean. Mooi dat Sander en Alex verbonden blijven!
Prof. Dollar schreef op 20 augustus 2019 13:45 :
Goed dat ze scherp blijven op de eigen bedrijfsvoering. Lean and mean. Mooi dat Sander en Alex verbonden blijven!
Wellicht dat ik geen verstand van Amerikaanse titulatuur heb, maar ik zou denken dat een EVP lager in de pikorde zit dan een C-suite executive zoals een CSO.
T. Montana schreef op 20 augustus 2019 15:12 :
[...]
Wellicht dat ik geen verstand van Amerikaanse titulatuur heb, maar ik zou denken dat een EVP lager in de pikorde zit dan een C-suite executive zoals een CSO.
Vanuit de Amerikaanse titulatuur bezien - en dan met name die in de financiële wereld - denk ik dat je helemaal gelijk hebt. Daarbuiten zijn er uitzonderingen. Gezien de uitleg in het persbericht zijn deze twee heren zeker niet in pikorde gezakt. Het zou mij niks verbazen als de volgende EVP verantwoordelijk wordt voor de commercie.
uniQure to Participate in Multiple Upcoming Industry Conferences in September tools.eurolandir.com/tools/Pressrelea... Met enkele webcasts om naar uit te kijken. En mogelijk nieuwe data 'recent progress' hemofilie B.
uniQure Announces Achievement of Planned Enrollment in HOPE-B Pivotal Trial of AMT-061 (Etranacogene Dezaparvovec) in Patients with Hemophilia B LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Sept. 03, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. Due to the high level of interest in the study from both patients and study investigators, uniQure expects to over-enroll up to six additional patients before the end of September. Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency. “We are extremely pleased to reach this important milestone in our ongoing development of etranacogene dezaparvovec, which we believe has the potential to be the first and best-in-class gene therapy for patients with hemophilia B,” stated Matt Kapusta, chief executive officer of uniQure. “We appreciate the tremendous support from the hemophilia patient community in achieving this important goal ahead of schedule and look forward to sharing top-line data from the Phase III trial, which we expect to do next year.” The pivotal Phase III HOPE-B study builds on the success of the interim 36-week results of the Company’s ongoing Phase IIb study of etranacogene dezaparvovec, which demonstrated that a single administration of the investigational gene therapy resulted in sustained increases in Factor IX (FIX) levels up to 54% of normal, and a mean FIX level of 45% of normal. During that time, no patient reported any bleeding events or required any infusion of FIX replacement therapy for bleeds or experienced any material loss of FIX activity. Additionally, an ongoing Phase I/II study of AMT-060, the Company’s first-generation gene therapy for the treatment of hemophilia B, demonstrated that all 10 patients continue to show sustained and stable increases in FIX activity and long-term clinical benefits at up to 3.5 years of observation. “We are very excited and proud to have achieved the targeted patient enrollment in the HOPE-B study in just a little more than one year from study initiation,” added Robert Gut, M.D., Ph.D., chief medical officer at uniQure. “This multi-center, multinational trial involves 39 clinical sites across 9 countries, and highlights the outstanding effort and passion of our clinical operations, clinical development, medical affairs and project management teams. We would like to thank all study participants, advisors, primary investigators and the whole study staff in the United States and Europe for their great contribution and support.” About the Pivotal Phase III HOPE-B Study The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Adult hemophilia B patients classified as severe or moderately severe are enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients will receive a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Dosing of patients in the HOPE-B pivotal trial was initiated in January 2019. The study’s primary endpoint is the assessment of Factor IX activity 26 weeks after dosing. Secondary endpoints include annualized bleeding rate (ABR) and usage of Factor IX replacement therapy over a 52-week time frame, as well as other efficacy and safety aspects. Post-treatment, patients will be followed for 5 years. Patients enrolled in the HOPE-B pivotal trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers. Previous studies performed by uniQure suggest that AAV5-based gene therapies may be viable treatments for at least 97% of patients. About Etranacogene Dezaparvovec (AMT-061) Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company’s AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com
Steve schreef op 3 september 2019 14:10 :
Mooi voor op schema!
Kan je wel zeggen. Op 29 juli zeiden ze nog: "In the second quarter of 2019, we continued to make substantial progress across our pipeline. Patient enrollment in our ongoing Phase III HOPE-B pivotal study of AMT-061 continues to advance, and we remain on track to complete patient enrollment by the end of this year ,” stated Matt Kapusta, chief executive of uniQure.
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