Not sure the data is that "remarkable" - if you look at the detail in the slides, the one complete response they achieved was at the highest dose cohort and that patient had what looks like a severe reaction to the drug (slide 4). Of the 4 patients treated in the cohort just below the highest, there was only 1 PR with the remaining 3 achieving stable disease. It appears they were doing 3 patients in each cohort and the last patient in the final cohort was given the dose from the previous cohort - not sure if that means they determined the final dose was deemed too high from a safety perspective?
md - exaclty - for the believers that held while it went from below $10 to up over $50, back below $15, then over $35. then down close to $10, then back to where it is today, do you really think we are invested in smoothness? The science and the evidence never changed over the past few years - the only thing that was volatile was the prevailing sentiment on whether and when Etep would be approved, or if there would be any competition. I find it fascinating that the "market" could vacillate so violently on seemingly solid evidence of efficacy and safety. For the few morons that still believe that Drisa will have any impact on Etep's prospects, please read Christine McSherry's twitter recap of her son Jett's experience on Drisa. Will it be a "smooth" ride to $100? Probably not, but compared to the last few years, it will feel like a moonwalk to the bank!
To finish the point - I think the reality is actually to the contrary - amateur investors, including all the obnoxious momentum players that typically troll any biotech with an upcoming PDUFA date - are staying away from THLD, because they can't buy it on margin or the margin requirements are too high. The "professional" investors, as you call them, are more likely to play the options.
So, you think all the option activity is from "amateur" investors. There were 1,381 call contracts purchased for the Mar $10 strike - does that sound like an amateur taking a position?
The only "horror" between now and approval will be your idiotic, paranoid ramblings. I'm sure once Etep is approved, you will move on to the horror of patent issues or the horror of insurance reimbursement issues. You are like the anti-FDR - there is everything to fear including fear - give it a rest, already!
gig - "the law and the punishment in this country are too soft"? The US by far has the highest incarceration rate than any western nation - who exactly are we being soft on? And when you say "religions do not do the killing, evil people do", there is only one religion in the modern world that preaches to the destruction of "infidels". Of course it takes a "sick mind" to do these terrorist acts, but when a sick mind is brought up under a violent ideology, do the math!
Your "fifth detailed letter" to the FDA - I'm surprised you haven't been visited by some friendly men in white coats. Perhaps they will take you seriously, as your analysis reflects a real expertise in the field of DMD, although your apparent lack of functioning brain cells is a little "concerting"!
nerd - I would agree the matched cohort provides a significant level of validation to the clinical benefit picture, but for the ignorant masses that don't understand how impossible it would be to set up a placebo controlled trial in DMD where the two arms were tightly matched in baseline characteristics, a historical-controlled trial opens the door for criticism and dismissal of the results.
I fail to understand all the handwringing over the trial size, as if 3+ years into the process of pursuing accelerated approval the FDA is going to suddenly say "oh, you only have data on 12 patients"? A small N is a problem if there are safety signals that need to be weeded out in a larger population - not the case here. A small N is a problem if there is inconsistent pk/pd and other biomarker activity - not the case here. A small N is a problem if there is inconsistent clinical benefit - assuming you accept the rationale for the twins, not the case here. Most importantly, a small N is a problem if you are seeking full approval - not the case here.
You say this as if the company isn't already doing it? The FDA requested safety data from a certain number of patients as part of the final package, so there is no doubt the company is providing them any safety data they have from the confirmatory trial. When it comes to efficacy, I'm sure there will be more than just parents from the Phase II trial testifying to the benefits that Etep has provided their boys.
They still see the approval as a "toss up" and their spin on the AdCom was that "most panelists and patient representatives did not feel the safety concerns outweighed the overall benefit". How far up JJ's #$%$ must Jefferies be? The "panelists" did no weigh in on the benefits, except to agree that none of trials strengthened the case that the drug was effective. While I have sympathy for the patients who believe the drug has helped them, there were many more patients that had to discontinue the drug because of safety issues that weren't paid to come to the AdCom meeting. Having listened to most of the AdCom meeting, to label the potential approval as a "toss-up" is even more delusional than JJ's positive spin.
I appreciate this company's technology, but it seemed they were trying awfully hard to avoid admitting that they have to do another clinical trial, which seems to be the case, and the timeframe of "at least a year" is as wishy washy as you can get. Even if they had a solid clinical trial plan in place with a more definitive timeline, they don't have the capital to run one. Time to sell the company - selling enough shares for pennies to get FDA approval will dilute the existing shareholders into oblivion.
AF's rule technically does not apply to THLD, since they don't own the drug outright - with 50% of the future profits of Evo going to Merck, THLD's market cap does not reflect the full value of the drug. Secondly, AF dismisses Evo as being "similar" to the drug Palifosfamide, which failed in a Phase III trial for STS. While the drugs share the same warhead, the mechanism of action is completely different - Evo is targeting and active only against the hypoxic tumor regions - Pali targets the same tumor regions that most "conventional" chemo agents do, so the comparison is weak, at best. AF can provide some rational analysis at times (his assessment of NWBO is detailed and comprehensive), but he is dismissive of Evo without even understanding the drug.
bet - you bring up a great point and hammer it home with one of my lesson-learning trades. I had some shares in UTHR prior to approval and the price didn't move much when they got FDA approval - maybe 15-20% if I remember correctly - so I sold it. I didn't understand at the time what these orphan drugs were potentially worth, so I missed out on a 20X+bagger. As Ed Kaye has stated, SRPT is going to own the DMD space. These days, investors are more in tune with the orphan drug space and potential values, so a 20X return is probably not in the cards from here, but anyone who sells for less than $100 will surely miss out on some easy money.
I read the article - the guy is seriously delusional - he basically says if you exclude all the patients that did poorly on the drug the data looks great and when it comes to safety, the serious adverse events were in a "very small number of patients" and are "manageable" in the context of a lethal disorder - how would the very small number of patients changed if not for all the drop-outs and discontinuations. I suppose until they get rejected in Europe, he has to continue to posture for Drisa - after they get rejected there, I would hope for his shareholders' sake he would pull the plug on entire platform - there is no way Drisa can get to market without additional trials and who is going to enroll in them if Etep is approved?.
The quote in the Business Journal was him commending the FDA on how they handled the Drisa review. I've listened to every presentation and conference call over the last 4 years and I don't recall CG ever disparaging the FDA - he may have expressed frustration with the process and their start and stop decisions, but he was mostly disciplined in his comments about the FDA and how the company needed to work with them. CG was a scapegoat for people who though the FDA was making decisions based on personality conflicts with CG - if you look at the evidence, the FDA's decisions were based on ignorance and bad analysis.
Please provide examples of CG's "loose talk" - did he say something offensive about the FDA or their process - what exactly are you referring to when you conclude his loose talk got him fired?
"hopefully this is nearing completion" - it is highly unlikely the company has sold any shares through Cowen, yet. First off, they haven't even publicly announced the ATM agreement with Cowen - there has been no press release and they didn't discuss it at the recent Stifel conference - selling shares without a public disclosure is highly unethical and not consistent with the company's actions in the past. They also announced at Stifel that they are in the process of validating the "event" data from the two Phase III trials, so selling shares during this time frame would be an even more egregious act. Finally, there has only been five trading days since the Cowen agreement was disclosed in the SEC filing and the volume during that time has not been out of the norm (~850,000 shares per day).
The more likely agenda is that knowing they would be releasing top-line data around year-end, they wanted the ability to efficiently raise some funds to support the commercialization of Evo. Every action the company and Merck have taken over the past year is consistent with the expectation of a drug launch. While the data may still be officially blinded, they have some knowledge from the clinical sites how many patients are still being treated with Evo and for how long, so they have an idea which way the data is going to fall. For example, in the STS trial, the top-line results are based on 434 deaths out of total enrollment of 640 - they know how much drug they are shipping to the clinical sites and how that relates to the 200+ remaining patients in the study.
md - I don't mind someone "looking at both sides", but when the writer either ignores the relevant facts that would render one of those sides senseless or is ignorant of the facts, while portraying himself as an expert, then I think the criticism is justified. I am actually on the fence about AF - I have seen him make some very reasonable analyses of the validity of a certain company's drug or trials, but when it comes to SRPT and Etep, he has been very flaky with his analysis and has ignored some fairly evident facts, so you have to question his motives?
jrrt - given the harshness of the FDA assessment on safety and lack of mechanism of action, it would almost be negligent for BRMN to continue with their DMD platform. It is difficult to imagine the FDA actually asking them to do another trial to prove effectiveness, given their safety assessment. The FDA forced the AdCom's hand into focusing on the data flaws and safety issues, rather than the unmet need and patient testimony - will they force BMRN's hand and say don't come back until you can prove you can dose this safely in a preclinical trial?
When it comes to the issue of IV administration, I thought that the reason they didn't use that route originally was it was too harsh for IV - the risk of vasculitis was too high? No doubt SC provides some convenience, but when you are trying to get the drug dispersed to every corner of the body, I can't imagine SC is effective at that?