regarding LOI and contracts all based on approval.. nothing more. stay objective brother... I´ve been long since the teens... took some off but still holding some through binary event. remove fear...
I said well written . Dr. Ogut is leading GT specialists on the plannet. read his blog. and please remember character counts when communicating with people. blessings to you whether long or short. god speed.
In experiments with larger animals, however, you don't see the same effect. You don't see the overexpression of the (SERCA2a) protein in the heart tissue," Ogut said. Ogut has no affiliation with Celladon and has no investment position in the company.
Measuring SERCA2a levels in heart-failure patients would be the most definitive way to resolve the question about whether the Mydicar gene therapy does what it's designed to do. Unfortunately, this analysis requires removing a small sample of heart tissue from patients, which can be done only if the patient dies or undergoes invasive cardiac surgery.
In the 2014 published study of CUPID-1 follow-up, researchers were able to obtain biopsies of cardiac tissue from 11 patients. In three of four patients infused with the high dose of Mydicar, persistent presence of the transferred gene was found. Five patients treated with the low and middle dose of Mydicar showed no presence of the transplanted gene, neither did two placebo patients.
Celladon skeptics, including investors shorting the stock, believe otherwise. Although it hit the primary efficacy goal, the CUPID-I was not entirely clean. Nine heart-failure patients were enrolled in the high-dose Mydicar arm versus 14 patients on a placebo. The small number of patients raises the risk that the benefit observed is an outlier that won't be replicated in the much larger CUPID-2 study.
This risk is amplified by the relatively poor performance of heart-failure patients treated with low and middle doses of Mydicar in CUPID-1. On some efficacy measures, patients treated with the middle dose of Mydicar performed worse than a placebo.
The absence of a consistent dose response in CUPID-1 is worrisome because higher numbers of re-engineered viral particles infused into patients should lead to more healthy, SERCA2a-producing genes taken up inside the cells of the heart muscle. Data on whether Mydicar can actually lead to successful replacement of diseased genes and produce higher levels of SERCA2a protein are unclear.
"When experiments are run in small animals like mice and rats, the gene therapy works well. The re-engineered virus makes its way into the heart tissue and the [SERCA2a] protein is over-expressed," says Ozgur Ogut, who until late last year was the co-director of the Cardiac Muscle Contractility Laboratory at the Mayo Clinic in Rochester, Minn.
The unbinding of data and analysis is done by the FDA not CLDN MGMT. The results are provided in a letter called a CRO. The announcement today is based on approval from the FDA.
Is doing you a service by highlighting the inherent risk in any major trial. Instead of blasting him you should thank him, Its called objectivity. Its should be a step in everyone investors due diligence otherwise your just drinking the cool aide and following the tea leaves. Its very possible that CLDN hits a homerun and gets permission to get to market yet why not understand the other side of the opportunity. Blessings everyone and good luck on whatever side of the trade you sit...