Beste Mensen,
Weet niet of het hier de juiste plek is maar heeft een van jullie zich verdiept in Celladon?
Wat ik zie is een aandeel dat bijna 50% gezakt is en dat deze maand zijn data voor een pivotal fase 2B trial vrijgeeft. Drager van de therapie is een AAV1 virus en dus een concurrent van QURE/BMS. GSK, J&J en ik meen Pfizer hebben allemaal een aandeel in Celladon.
Ik zie het als een interessante mogelijkheid. Want deze maand komen de pivotal data en als die goed zijn haal je een goed rendement. Maar de fase 2a data moeten erg goed zijn geweest voor een breakthrough status. Inderdaad de significantie van het therapeutisch effect was meen ik p=0,0003, en dat betekent dat er of echt een stevig effect is OF dat er structureel op een verkeerde manier is gemeten, zodat de uitkomst in feite trivial is. Maar de kans dat zo'n significantie toeval is, is heel klein.
Ik wil wel instappen geloof ik. Iemand zich al in dit aandeel verdiept?
Hieronder wat quotes:
Celladon's most advanced drug in its pipeline is Mydicar, an enzyme-targeting therapy for systolic heart failure patients that is currently in phase 2b studies. Last year, the FDA awarded Mydicar breakthrough therapy designation, and Celladon expects that it will be able to release top-line data from this Mydicar trial by the end of April.
The potential for Celladon's positive upcoming data did little to support shares, however, as investors moved to the sidelines to avoid the risk of a Mydicar failure.
Regardless, despite Celladon's investments to ramp up facilities and staff that could support an eventual commercialization of Mydicar, the company appears to be in solid financial shape.
We are the first company to enter clinical development with a product candidate, MYDICAR, that selectively targets SERCA2a. We refer to our Phase 1 trial and Phase 2a trial of MYDICAR together as our CUPID 1 trial. In Phase 2a of our CUPID 1 trial, 39 patients with heart failure for reduced ejection fraction, or HFrEF, which is caused by the inability of the heart to pump blood efficiently due to weakening and enlargement of the ventricles, were enrolled in a randomized, double-blind, placebo-controlled trial. MYDICAR was safe and well-tolerated, reduced heart failure-related hospitalizations, improved patients’ symptoms, quality of life and serum biomarkers and improved key markers of cardiac function predictive of survival, such as end systolic volume. Based on these results, as well as our previous preclinical studies and clinical trials, we advanced MYDICAR to a 250-patient randomized, double-blind, placebo-controlled international Phase 2b trial in patients with HFrEF, which we refer to as CUPID 2. We completed enrollment of CUPID 2 in February 2014, reached the primary analysis data cutoff in February 2015 and expect to un-blind the data and announce results in late April 2015. If successful, these results, along with other studies, could form the basis for regulatory submissions for approval with the United States Food and Drug Administration, or FDA, and European Medicines Agency, or EMA.
In April 2014, the FDA’s Center for Biologics Evaluation and Research, or CBER, granted Breakthrough Therapy designation to MYDICAR for reducing hospitalizations for heart failure in patients who test negative for adeno-associated viral vector 1, or AAV1, neutralizing antibodies, are class III or IV heart failure patients under the New York Heart Association.