I have been reading some of your posts here and on seeking alpha. You are in the biotech industry and I accept your description of patient demographics and I will not ask you to reveal your source. I am trying to model their results and perform some analytics on the 2b trial results. The only info on the net about this trial ( that I could find ) is "Randomization and Blinding
Subjects will be assigned in a 1:1 ratio to MYDICAR® or placebo. Randomization will be stratified by country and the ability to walk between 150 to 425 meters in the 6MWT." In my model if I stratify Class Two as 20% and Three and Four as $40% each and assuming no effect on stage two and four patients and a 50% reduction in hospitalization in 2/3 of the Class Three patients; I get a figure similar to the hazard ratio they got. This is obviously a very simple model and has to be wrong as the information is incomplete. This shows a trend for Class Three but is not statistically significant. Obviously if there is no stratification this model does not apply. Can you give me your thoughts? Thanks.
Thanks again. The only stratification I can find in Cupid 2b is by country and a modified 6 minute walk test where the subject has to walk 150-450 meters. I have been looking at the walk test and its not very sensitive in the short term and does a poor job of stratifying NYHA Class. In the figures you have given above both for Cupid 2 a av. distance was 345 meters and in 2b 326 meters implying that all CHF patients except the very severe Stage four who are dyspnoeic at rest would be able to walk 150 meters. This means stratification was by country and in terms of CHF there were two groups severe stage four and the other group was stage two, three and milder stage four patients. This would definitely lead to uneven distribution of stage two, three and four patients in the two arms. Would you agree with this?
I hope sooner! Its only 250 patients. Its really hard to explain that a 88% risk reduction at one year with a p value of 0.003 would be reduced to a 8% improvement with a p value of 0.8 unless there are serious issues with the trial.
The factors you site were really peripheral to my decision to invest.
I made my decision on two facts which stood out to me. First the One Year Relative Risk reduction for the highest dose which was employed in the 2b tail was 88% at a p value of 0.003 and at three years there was a 90% reduction in mortality at 3 years compared to placebo. These are incontrovertible findings and the fact that on average the mortality and morbidity rate in placebo follows general accepted cardiovascular trial morbidity and mortality curves proved to me the noise about placebo patient patients being less sick was just that noise. I also realized since Mydicar was not improving Ejection fraction it was merely stabilizing the heart but as long as it was reducing hospitalization and mortality it did not really matter to me.
Where I erred was in not realizing the impact that adding two previously untested groups ( Class two and Class Four) and lack of proper stratification would play havoc. This is the opposite of what ACAD did. They had a failed phase two but certain subgroups showed clinical significance and they concentrated their phase three trial on those subgroups and worked hard to reduce the subjectivity by rigorously training the observers and succeeded in their phase three. CLDN in contrast had one
dose work in one group of patients and they messed it up by including other groups of patients and not stratifying properly. Corporate Greed at its finest!
Management is inexperienced and panicked and anounced the topline results which include three groups of which only one group was evaluated in the previous 2a trial. That group showed a 90% efficacy witha p value of 0.003. It is very possible that particular subgroup would show efficacy or a favorable trend on further analysis.
Its not him; the shorts are taking position. Look at the volume.
They can not do that legally. My understanding is that any material information has to be made public. Also the fact they are looking at another acquisition seems to suggest they are valuing the whole mydicar platform at zero.
Actually the ex CEO in the conference call stated the data would be made public and published in a journal.
Typically if they find something positive in the post-hoc analysis they announce it to the public in a PR and state they will discuss with the FDA next steps. As in this specific instance ( as you and I were hoping) if the data would show a favorable trend in Class Three you don't think they would announce it to the public and talk about a confirmatory trial. On such a PR the SP would be at least 50.
I hope you are right. I did not like them talking about an acquisition so I am out, I will be back if they announce a confirmatory trial. GL to you.
I thought I would reply to this posting as seems one post relevant to NYMX among the nonsense posted on this board. Wonder if any of the old posters are still in this stock. Maybe we need a show of hands. :)
I am cautiously optimistic but not quite sure of what they are doing with these new pivotal results. I know initially when they declared the initial phase three results Dr. A stated the drug performed as expected but the placebo response was higher than in previous trials and that is why the topline results were a failure. As per my conversations with other people on this board who had been in the trial a questionnaire was sent to all participants subsequently (to phase three results) asking them if they had participated in other therapies apart from NX1207 when in the trial. Since the trial lasted several years its logical to assume that the placebo patients tried other things while in the trial and those that did should ideally be no longer included in the analysis and I presume this is what they are doing now after excluding the noncompliant patients.
I do wonder though why would any of those placebo patients admit to being noncompliant as they were being paid to participate in the trial.
You have the right to be skeptical but you wont get in on the other side of 2. It will either be other side of ten cents or ten dollars. I personally think they would not have reteirated the results news again last week unless they were fairly confident of having something.