GSA - Given the revenue implications of your point #5, don't you think the 40% overall revenue growth is weak? I haven't gone through the numbers in detail, but I would think converting the LifeCell business (where they were splitting revenues) to direct sales would have added 15%-20% to the 2014 numbers all by itself? Beyond that, I can't imagine the 40% includes much of anything from the "18 countries". Last year we were thinking the 40% revenue growth was a slam dunk and didn't make it, which is why I pray he is being conservative in targeting 40% as his "baseline". With all the irons in the fire, the conversion of the LifeCell business and the fact that they missed 2014 (so are growing from a smaller base), I would hope that 60% is the "median" target for 2015?
What has become abundantly clear is that the 6MWT is a very flawed clinical endpoint in DMD - there are too many variables that come into play in how a boy will perform on the test, so the efficacy of the drug gets lost in the sea of noise - how much muscle function was lost before treatment started, how much did the boy grow during the treatment period, etc., etc.. It would be different if the treatment reversed the damage and allowed muscle function to be restored or if the patients were all fully grown at the time of enrollment, but that is not the case here. Ultimately, maintaining ambulation may not be achievable, depending on when treatment begins, so why not focus on pulmonary and other clinical endpoints that may not have as much variability in the natural history. The trials may need to last longer than 48 weeks, but at least you might have some clear cut evidence at the end?
They said the biopsies were being scheduled along with the 6MWT measurements to minimize travel for the families. Assuming the biopsies were taken recently,the results would still take a couple months to process - presumably they will go through the same independent analysis by 3 separate readers?
Correct - the theory is start them early enough before they have significant deterioration/loss of muscle and their remaining muscle will grow and keep pace with their body into adulthood. At age 9, they have too much wasted muscle mass in their legs to allow them to remain ambulant into adulthood. The unknown is whether Etep is simply slowing or stopping muscle loss - the pulmonary data would suggest it is stopping it, but only time will tell.
You don't get it - theoretically if the drug is working, the "deterioration" of the muscles has slowed or stopped. What hasn't stopped is the boys growing - the remaining healthy muscle tissue in their legs can't support the increase in height and weight as they grow. The pulmonary measures show the exact same thing - actual MIP and MEP are stable or increasing, but there are marginal declines in "predicted" MIP and MEP, which takes into account their weight - i.e. as they grow they should be able to inhale and exhale at greater strengths, but the existing damage to their muscles does not allow them to keep pace with their body's growth.
"you need convincing data for early approval". Actually, no - theoretically under the AA regs all they need is clean surrogate (i.e. dystrophin production) data and a "reasonable likelihood" that a clinical benefit will be realized. You can argue with the N, but the 6MWT data is better than natural history over 3 years and the pulmonary data is clearly better than natural history would predict - the other clinical measures also show improvement over natural history. So, if the dystrophin data comes in clean, the clinical data on top of the parents' and doctors' testimony as to how Etep has improved the lives of their boys will be enough to convince an AdCom panel that there is a reasonable likelihood that Etep is providing a clinical benefit.
Are you really that clueless? Read the press release - they effectively doubled the share count (iow gave half the company away) to merge the two companies. The $750M value is simply stating that as long as the share price doesn't decline, the combined company will be worth $750M after the merger. I can't imagine people are buying the shares in the pre-market thinking the $750M is some kind of purchase price, but there is no other explanation. I tried to short, but there were no shares available. Unless there is something magical about OnCore's assets, I would think this will drift back down below $20 fairly quickly?
By comparison, RNA's Phase III trial has data on 47 boys that were older than 7 at baseline - the treated group (33 boys) lost 42 meters in 48 weeks and the placebo group (14 boys) lost 67 meters in 48 weeks - and BMRN thinks they can get approval with that data,despite all the safety issues. By all accounts the 6MWT decline accelerates once the boys start declining, but even if you assume a linear decline with RNA's data out to 168 weeks, the treated group would lose over 140 meters and the placebo group would lose over 230 meters over the same time period.
simp - you can't reread the FDA guidance for AA in Jul 2013 and again in Apr 2014, without also reading the reversals of those decisions and the clear confusion they placed in the path of the company with their guidance in Nov 2013 and Oct 2014. Is the company to blame for the FDA reversals and requests for further and further data?
nerd - you're off the rails here. CG always presented the case knowing that a confirmatory trial would be needed - the issue was what the design of that trial would be - placebo controlled, dystrophin measures required at what time points, how many patients, etc., etc.. How could CG "immediately plan and put into action" a confirmatory trial when the FDA couldn't make up their minds on what they wanted to see? If you recall, one of the earlier FDA communications spoke to including all kinds of new clinical measurements into the equation. And when it comes to dystrophin measurement, "everybody in the industry" knowing what the best method was didn't prevent to FDA from questioning it and dismissing the measurements from the Phase II trial. You've bought into the gibberish on this board that somehow CG messed up the process, when it is a clear case of mismanagement by the FDA.
The problem here is that the company has lost control of the narrative and allowed what should be accepted as powerful results to be discounted and dismissed. Granted, the company was treading on new ground, but CG got caught up in the initial stability of the 6MWT and focused the narrative too much on the Beckers analogy, which is a flawed comparison - Beckers patients have truncated but still functional dystrophin, so that when they are 10-12 years old they still have the majority of their muscle mass intact and functioning. The boys in this trial do not - by the time they entered the trial, a substantial portion of their muscle mass had already been irreversibly destroyed.
Hopefully, the Beckers analogy will hold up for boys that are treated from a very early age, but it has distorted the narrative in interpreting the results for this trial. Imagine how the 144wk and 168wk trial results would be interpreted if two years ago CG had said this - "The boys are experiencing remarkable stability in their performance on the 6MWT thru 48 weeks and we hope that will continue. However, you have to remember that these boys have experienced significant loss of functional muscle mass in their legs that will not be recovered through this treatment. Therefore, as they hit adolescence and add height and weight, we are not sure that their remaining healthy muscle tissue will allow them to stay ambulant, but it should allow them to stay on their feet longer than natural history would predict." Unfortunately, the "market" will likely hammer the shares for any continued decline in the 6MWT scores, regardless of how stable the remainder of the clinical measurements are.
His title is not CFO - it is "VP of Finance and Controller", which is typically one level below the CFO position. I never noticed that this company does not have a designated CFO, which is unusual. Although working in finance and accounting, I could never imagine what a CFO of a development stage biotech does all day - with little to no revenues to account for, the accounting, tax and filing issues are fairly simple - a nice gig that typically pays $500K+.
In any event, the point is that Fernandez has been with the company since 2006 and appointed to his current position in 2011, and he likely does not have an employment agreement. So, why after 3+ years in this position do they feel the need to give him (and the VP of Manufacturing) protection from a change of control - these things don't happen in a vacuum? I worked for a company that scrambled to put in similar agreements for certain managers when the company was suddenly put in play by the BOD.
Good question. If Merck is seeing broad applicability of the drug (beyond Pancreatic and STS indications) and wants to capture all the spoils, they may be pushing to buy while the share price is low. Whether THLD management would consider a sale at this stage is complicated - they could play hardball and put Merck off until the Phase III trials come in, betting on a much higher share price to start the negotiations, but they have a duty to the shareholders to consider current offers. What would you consider a good price today, taking into account the risk of the trials, etc.?
put in place for a couple mid-level managers. This is somewhat unusual - possibly triggered by preliminary talks to sell the company??? Most hi-level execs have employment agreements that already have these provisions and they usually don't push change of control benefits down to mid-level guys unless there is a real possibility of a transaction.
A 3-minute video of Billy dancing - didn't look like he was looking to sit down at the end, either. I suppose the shorts will write it off as New Year's Eve placebo effect?!
starfe - do you really count yourself and the rest of the moronic bashers on this board as part of "Wall Street"?!
Another lie - CG never said the trial was going to enroll in July - the guidance they gave in April was the trial would begin in the "3rd quarter" and when pressed on exact timing, he never said Jul, he said Sep. You can quibble over whether a trial begins when patients are enrolled or when the first patient is dosed, but at worst it was a two month delay. The fact that people want to stretch this out to a "6-month" delay in order to pile on CG is beyond me.