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
thanks Banman.... I'm adding here as well. I hate to ask a tea leaf question as I don't want to annoy anyone. But, I was thinking based on similar story stocks that CLDN could run to 30- 40 pps prior to announcement then if approved could perhaps get to 75... call me crazy. any thoughts on this weegie board question... lol !
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
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...
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.
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.
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.
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.
Float is sub 10mm.Shares will be chased up. supply and demand. this is when things get PARABOLIC. wait to we see real Volume
GS will broker a buyout sooner then later.
PPS TG $50
buy and hold .....
Thanks for framing the bull /bear case so clearly. On the bull side, I was also factoring in the FDA had granted them a Breakthrough Therapy designation based on risk reduction on phase 1. Furthermore, Ogut comments regarding the dosses being to inadequate for humans versus rate confuses me as the finding of phase 1 data was based on humans ? or was the phase 1 on rats and the phase 2 data to be released is on humans ? sorry to sound so ignorant. please advise ? thanks for the assistance.
CLDN could potentially see 30 prior to April FDA news, the float Is 12mm , if the FDA momentum traders show up it could go parabolic. Its got a huge chance of getting approved if that happens it could see 60 plus overnight. as for now, definitely under the radar screen. the market makers are having a field day taking out stops. I'm long of course and feel good about this one.