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...
those are planned sells. Very normal for BIO´s. Planned sells are away to subsidize income for executives as salaries are nominal. Stock is close to breaking out.... do your own Due diligence
Pfizer (NYSE: PFE ) is struggling to offset slowing sales tied to expiring patents, and that has some observers thinking that Pfizer remains on the hunt to buy another drugmaker that can spark its growth. Earlier this week, Pfizer announced that it would acquire Hospira, the top provider of injectable drugs. The deal will net Pfizer an intriguing biosimilar program, but some suspect that the company is still hungry for more. If Pfizer is indeed still on the lookout for another buyout, three of our top Motley Fool contributors think these three companies should be on Pfizer's wish list.
George Budwell: Over the past couple of years, Pfizer's R&D efforts have decidedly shifted away from small-molecule drugs and more toward next-generation types of therapies, such as biologics, evidenced most recently by the aforementioned acquisition of Hospira. So it's not entirely surprising that the pharma giant has taken a keen interest in cardiovascular gene therapy company Celladon Corporation (NASDAQ: CLDN ) . At last count, Pfizer owned nearly 1.8 million shares of Celladon, making it one of the company's largest shareholders.
My take is that Pfizer's initial interest could turn into a full-blown tender offer if Celladon's lead clinical candidate, Mydicar, reports positive mid-stage results this April as a treatment for systolic heart failure. Such a deal would give Pfizer access to a potential megablockbuster. And, as a biological-based treatment, Mydicar would benefit from the high standards for generic "biosimilars" set by the Affordable Care Act in 2010, meaning the drug could effectively be immune from generic rivals for several years past its patent expiration. Furthermore, as a gene therapy, it would hold a competitive moat simply by the lack of competitors that can easily manufacture the viral vector on a commercial scale.
Mydicar would also fit in nicely with the big pharma's strong cardiovascular product portfolio and clinical pipeline. All told, this pairing looks like a great match -- that is, if Celladon's gene therapy platform is validated by the upcoming clinical data readout.
Board Members, Upon reading about the newly registered trial on clinical. gov. I contacted Mr. Wiklund from CLDN IR DEPT. Please see his below response. I omitted a couple sentences that were not pertinent to the subject matter. He states as you can read below its normal protocol to test higher dosages to determine potential toxicity levels in a new therapy. As, the current dosage has not shown any safety issues. In my humble opinion this is still a risk on FDA event in April. As, his response to me does not speak to the efficacy in the current trial only the safety profile. Hopefully we will get more information on Tuesday. Please do your own due diligence. Blessings.
I was surprised to find that via Clinical.gov that a 36 person mydicar trial thats gated to start April with a dosage 2.5x that of CUPID.What is extremely concerning is if this is a signal that the efficacy in the current trial end points will be too low for the FDA.
With respect to the higher dose trial, we have not seen any dose limiting toxicities, safety concerns, or lab abnormalities with our current dose. Our DSMB has met four times during the CUPID trial without safety concerns. Proper Drug development then argues to initiate small scale studies to explore what a dose limiting tox may be. Particularly with a novel, first in man, gene therapy.
Fredrik Wiklund Less