From clinical and statistical significance points of view, CDx-301 could rive this company to multi-billion dollars co. The study produced very impressive statistical significance and you can shout enough hallelujah for the clinical significance. If you are lucky to have some CLDX stocks at this level, hold them. And when it is good for you, remember me. Good luck longs.
You definitely don't know what you are talking about as you ignorantly demonstrate your zero knowledge of Biotech science. Even if I direct you to look at their pipeline, you don't have the capacity to comprehend it!
CLDX is giant Biotech of the future. I told you so
You are extremely wrong! She is perfect for Oval office. Who wants a crazy bully to rule the greatest country in the universe? I'll throw up to see nude 1st lady. Think twice
It is not the only company without profit. For years AMZN made no cent and yet, it was senselessly pumped up! Most of the Biotech companies have no revenue talk of profit.
My guess is that the wall street criminals are artificially pushing it down so that they can buy at the end of the day. Trump or Bernie Sanders needs to go after wall street crooks! Be patient, I think tomorrow will be better.
Wall Street is crazy, I personally do not see any chance of positive votes. Prove me wrong!
al cannot be used to compensate for weak or inconsistent clinical findings (i.e., approval
FOOD AND DRUG ADMINISTRATION (FDA)
Center for Drug Evaluation and Research (CDER)
Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) Meeting
April 25, 2016
DRAFT QUESTIONS (cont.) ______________________________________________________________________________________________
Page 2 of 3
based on marginal data, to be buttressed with better data post-approval). When accelerated approval is used,
post-approval studies to verify the expected clinical benefit are generally required.
Discussion and Voting Questions
For DMD, there is obvious interest in dystrophin expression as a potential surrogate marker to support
accelerated approval. Whether an effect on a biomarker such as dystrophin is reasonably likely to predict
clinical benefit in DMD depends on a number of factors including, but not limited to, the reliability of the data,
the magnitude of the effect on the biomarker, and confidence that the dystrophin produced is functional.
Eteplirsen’s putative mechanism of action is to increase production of a truncated form of dystrophin. By
Western blot, the most accurate quantitative method used by the Applicant, mean dystrophin levels after 180
weeks of eteplirsen treatment are 0.93% ± 0.84% of normal (mean ± standard deviation). The Applicant
reported a control (untreated) value of 0.08% dystrophin based on retained samples from the pre‐treatment
biopsy in 3 patients from Study 201/201, combined with data from six patients with DMD who were not
enrolled in any study. FDA identified, however, some important limitations with respect to interpretation of
the results of the untreated controls (e.g., limits of assay detection, different muscles sampled).
1. DISCUSSION: Discuss the evidence presented about dystrophin production, including the following:
Misleading! Per Yahoo finance, cash on hand is $215.13M. Check it out. It seems the stock is pumped up for potential offering.
But is chapter 11 more likely to buy time for reorganization? If so, how will the market look at such a plan?
has the applicant provided substantial evidence of the effectiveness for pimavanserin for the treatment of psychosis associate with Parkinson's disease?
Discussion going on before vote
The lady sounded nervous as if she was reading under duress. She is a loaner! I do not see much sense in some of her negativity as she doe not have adequate training in clinical trial design, biostatistics, etc.. Her's will be in the garbage basket!
" I am here to plead that you recommend the approval..."
"As you know, there is nothing in the market... to treat..."
Personally, I feel for these patients.
Both ACADIA and FDA are also very passionate for these patients.
It will take a real COLD heart to reject the approval.
The testimonies are powerful.
Half-time impression / score on 3 questions
1. VOTE: Has the applicant provided substantial evidence of the effectiveness for pimavanserin for the treatment of psychosis associated with Parkinson’s disease?
YES, Clearly both Acadia and FDA agree there is efficacy established.
2. VOTE: Has the applicant adequately characterized the safety profile of pimavanserin?
Jury still out on this question. Yes, they described the risks accurately. Which leads to the next question. Is it an acceptable risk? It's no worse than what they have now. Lots of questions and concerns regarding the safety.
3. VOTE: Do the benefits of pimavanserin for the treatment of psychosis associated with Parkinson’s disease outweigh the risk of treatment?
Yes, and we get the black box warning for sure. I hope we get some good input from real patients that benefit from the treatment this afternoon to help persuade the committee to vote for approval.