The RNA press release spoke to a treatment benefit in the younger boys (less than 7 years of age) - that goes against any logic - those boys are expected to show stability, so how do you show a treatment effect, unless the placebo boys under 7 years of age also declined over the 48 weeks, which would further taint the FDA's stance on the natural history of Exon 51 boys.
What's the driver going to be - the earnings announcement? I'm hoping for a pop in revenues from all the recently announced deals, but there is probably a lag to deal with between announcement and any substantive revenue?
You just described the inherent issue of trying to design a clinical trial - the efficacy of the drug (as evidenced by clinical measurements) is tied to how far the boy has progressed in the disease, something that is impossible to measure accurately/consistently. That is why the enrollment criteria are so important - you have to get boys that are on the verge of losing ambulation (as measured by the 6MWT) and the regulators have to believe in the natural history of this patient group, because allowing a placebo group that would be off drug for 6 months is highly unethical at this stage of the game.
Your message should have been titled - "GSK data confirms that the doubts raised by the FDA about the natural history of the disease are without merit". The placebo boys aged 7+ in the GSK Phase III trial declined by 83 meters in their 6MWT over 48 weeks. This is consistent with the natural history studies that break down boys by age and don't include Becker's patients. The sad thing is that the company cannot force the FDA to respond directly to the issue - i.e. force them to show whatever data they have that led to their erroneous conclusion. Ultimately, if there is enough pressure from the DMD parent groups, the FDA may be forced to answer this issue (and others) in front of a congressional hearing?
The standard protocol is to provide minutes within 30 days, so why nothing from the FDA? Based on the parent and researcher responses at the PPMD meeting, the response they likely got from SRPT management in their meetings and the general buzz in the community, they knew they missed the boat completely with the last minutes they provided the company, so they wanted to wait to see the 120 week data before potentially digging their grave further. At this stage of the game, if they came out and said the 6MWT natural history data is consistent with SRPT's 120 week data would be laughable. As CG pointed out in the conference, even GSK's data for the 7+ aged boys showed an 80M decline in 6MWT over 48 weeks. So, where does that leave them - do they continue to denigrate the dystrophin data, without providing any alternative measures that can be utilized in the confirmatory trial? Do they acknowledge the 6MWT is compelling but try to argue an insufficient trial size, something they've known from the beginning? There is no reasonable response they can come up with against AA that doesn't make them look foolish - they are between a rock (robust, consistent clinical data) and a hard place (the wrath of the DMD community), which is why they can't figure out what minutes to provide SRPT management.
Settle down - every company that is participating in the JPM conference is releasing a PR or 8K this morning providing updates - this is nothing more than reporting to the public any "material" details that they will be discussing in private over the next few days.
Listen to your own argument " you see SOME stabilization in CERTAIN patients" - in other words, not all the patients with baselines above 350 meters stabilize and those that do "show some" stabilization, meaning what, they didn't lose the full 78 meters? Compare that with the Etep data, where other than the boy with the broken ankle, EVERY boy stabilized with less than a 10% decline at 96 weeks. The argument for variability has to cut both ways - i.e. if you believe that boys above 350 baseline can show variable rates of decline, then the argument has to be that some of the 10 boys would show some rate of decline. The variability argument does hold water when you look at the full patient population of twelve boys - two of them declined rapidly before meaningful dystrophin levels were known to be produced - that proves variability existed in the recruited patient population and the fact that 100% of the remaining boys were stable after 36 weeks proves that Etep has eliminated any decline/variability.
"Hedge funds probably know the company better than anyone else" - how do you come to that assessment? The majority of hedge funds have consistently underperformed (vs market averages) for the past five years - any hedge fund that has tried to make a living shorting biotech stocks over the past few years has certainly gotten crushed. The idea that a particular fund or analyst has any special insight into what is going on at the company or the FDA is ridiculous.
micro - what are you talking about - the last several company presentations have show individual patient 6MWT data - see slide 11 of the Oct 17th update on their website. I'm not sure what all this hand-wringing is over the boy that broke his ankle is either - he was unable to perform the 6MWT at 84 weeks, but was included in the 96 week data, reflecting a 20% decline from week 36 (he was in the placebo-delayed arm). so he is bound to do as well as he did at week 84. Either way, if he is the only one not maintaining stabilization, it is easily explained, like it was at week 96.
A few months ago, I would have agreed with your "wait it out" strategy, but this biotech market is too hot to sit on the sidelines and wait for your perfect entry point. I was debating whether to wait for the dilution or if the sale by Pfizer would provide a meaningful bump, and last week decided to double down. More than likely, the terms of Pfizer's sale will not be revealed, but if the buyer is a high profile player in the field, it is going to bring renewed attention to the potential of iSONEP - with Phase II results only ~6 months away, the "market" will do the math (potential multi-billion $ drug and a market cap of less than $100M) and push the share price higher prior to the results - we may see $10 prior to the iSONEP results.
Just because Max tested in Dec does not mean all the data is available - if that was the case, then all the 96 week data was available in June, so why would they have waited until Sep to release it? There are obviously timing differences on when the boys enrolled - don't expect the 120 week data until March.
sayno - You claim there are other "robust dataset" that suggest stability in Etep's patient population - where are they - give the details? When you say we will "soon know" the Drisa Phase III placebo arm data - we already know - overall, both the treated and placebo arms declined by greater than 60 meters on average and a number of those in the placebo arm lost ambulation over 48 weeks - that is your definition of "stability"? Face it - you don't know the facts.
Then again, looking at the chart, there was some heavy volume in mid-Dec that took the price from $2.85 down to $2.25 , so maybe the bulk of the overhang has already been dealt with?
The 8K filed on 12/17 indicated that ~3,740,0000 shares were issued on exercise and the current share count is ~8,885,000. I assume all of those new 3.7M shares are registered and available to be sold - I'm not sure why you would think that would be bullish - I assume those that choose to exercise are looking to cash out quickly - why exchange a preferred share earning a stated dividend for a common share, unless you are looking to sell it?
Yeah - "medical tourism" will support orphan drug pricing of $300K+ per year - think again. The only reason the drugs for these rare diseases have a chance at development is because of the insurance systems we have in this country for supporting the ridiculous prices it takes to justify the development cost - that price support system requires FDA approval. Most DMD families, already burdened by unimaginable medical costs of having a DMD boy, don't have the money to cover an overseas trip, let alone the targeted price of one dose of Etep. Beyond that, this drug requires weekly infusions - what are they supposed to do - move overseas?
I don't get it - what is the point - who cares about your definition of a startup? The funniest thing is you advise people who doubt you to investigate on-line and they'll "probably" find a similar definition - does this mean you didn't bother to investigate yourself, instead you took the time to post your definition? Who would care enough to doubt you?
On what basis do you have the ability to assess the "professionals" at the FDA? While it is for the most part a black box when it comes to how they have come to decisions, we have a number of facts that have come to light showing them to be less than professional in their decision to curtail the AA path for Etep. Look at the excerpts from the meeting minutes that were quoted in SRPT's 11/12 press release - they cite the Drisa and PTC studies as evidence that increased dystrophin does not lead to clinical benefit. They later admit that they did not have detailed data on dystrophin production or 6MWT results from that trial when they sent that letter and the the PTC study does not relate to Exon 51 patients or an exon-skipping therapy, so how is that relevant? They cite "recent natural history data" that shows patients included in SRPT's trial are expected to show stability in 6MWT over 96 weeks, while a recognized expert in the field (McDonald) is saying the exact opposite and while the placebo patients in the Drisa trial (who were enrolled with criteria similar to the Etep trial) showed significant deterioration in 6MWT over 48 weeks. These are facts and your assessment is these are "professionals" to be respected?
another "commentator" that doesn't understand the situation. The anger coming from parents, investors and I assume SRPT management is due to the fact that there are clear rules in place for the "disciplined gatekeeper" to allow conditional approval of drugs in cases of rare, deadly diseases. By any rational account, SRPT's Phase II Etep data has provided the necessary proof to allow that conditional approval. Up until two months ago, the FDA agreed with that preliminary assessment, but then changed their stance based on irrelevant or misconstrued data coming from trials/research that had no bearing on the effectiveness of Etep. Patience, as an investor, is one thing, but asking parents of all DMD boys to be patient while the FDA comes to its senses is another.