Excellent deal - will it be strong enough to offset the ordinarily negative taint surrounding a drug giveback by big Pharma?
All their studies have shown Iluvien to be superior in accuracy, let alone quicker and less costly, than pap tests - so far, that hasn't done them much good. I'm not a big conspiracy advocate, but you have to believe there is a huge vested interest among numerous, large international companies in keeping pap tests as the standard of care in detecting cervical cancer, so GTHP will probably be stuck trying to create value in the 3rd world markets for some time?
Ian Estepan, a biotech analyst/blogger, just tweeted that the offering was 4-times oversubscribed - there is a lot of institutional demand to get a piece of this company at $6.50 - will those locked out take it over $7 today?
Terri Ellsworth is always posting pictures and stories on Twitter about how well Billy is doing. Do you get the sense she thinks Billy is following the "natural history" of DMD?
You're asking the wrong question - presumably because you think the recent declines in 6MWT are a sign the disease is "progressing" (i.e. their remaining muscle tissue is continuing to be destroyed). If that was the case, then their pulmonary tests should be declining, as well. A 9-year old boy who has had half of his leg muscle tissue destroyed by this disease may not have enough healthy tissue to salvage and keep him ambulant as his body grows - if Etep keeps the remaining leg muscle tissue functional, but it is not enough to support his increasing body weight, is the disease "progressing"?
The reality is that whether Etep is halting the disease or delaying it is not going to be answered with the current trial - the trial in earlier boys will provide better proof, but that will take 5+ years to play out. That's the whole point behind AA - answers to the full clinical benefit that Etep can provide probably won't be known for a decade or more, but that is no reason to deny access to the drug to the boys that may benefit from it during that time.
Remember that before FOLD made a bigger commitment to develop their own ERT's, they were working toward doing combination studies with migalistat and the existing Fabry ERT's on the market. Migalistat as a single agent only works with 30%-50% of "amenable" Fabry patients (those whose bodies create some form of the enzyme, but it doesn't function well, vs non-amenable patients that produce no enzyme and have to be on ERT), but given that migalistat is showing that it may improve cardiac function vs ERT, this may open the door for doctors to prescribe migalistat off-label to the non-amenable patients on ERT in a combination therapy.
No doubt the MM results were sketchy at best - I was surprised at the SAE's that they attributed to TH-302 - to date, none of dozens of TH-302 trials have resulted in any significant toxicity with TH-302 - it is normally the combination drug that limits the dosing and creates any SAE's. The Glio data from the SNO poster, though, is solid. The historical response rate in that patient group is minimal, at best, so the 18% response rate and an additional 40%+ experiencing stable disease is meaningful and the safety profile is consistent with other TH-302 combination trials. Either way, TH-302's value will be driven in the near term by the pivotal STS and pancreatic trials - a failure in an early-stage MM trial is not meaningful.
zwerp - great post. And don't forget about all the prescription painkiller drugs that cause more overdose deaths than cocaine and heroin combined. Too much money being made by the right companies to make any waves in those markets.
If you're such a nimble trader, why would you have ever been "in" this stock - it has been known for at least a year that the pivotal trial results would not be known until at least late 2015?
grey - the answer is simple - ignorance. Just as the FDA pointed to the failed top-line Prosensa data as the basis for backtracking on Etep, they are taking the same position here. To put it into words, it is along the lines of "We don't have the knowledge or experience to make a rational analysis of the issue at hand, but we know that it concerns us, and when we are concerned we deny and delay". I said it before - what are the odds the FDA had an experienced expert on hand to inspect/review the dystrophin measurement methodology and results generated by Mendell's group. The fact that it took the FDA 5 months to express their concerns speaks volumes - they had some bureaucratic hack that had no experience in dystrophin measurement do the site visit, then spent the next five months trying to figure out what he saw, couldn't make sense of it, so then trashed the data and people that did the analysis and pushed off the responsibility onto an independent review of the same thing they couldn't figure out for themselves.
I wish this was just a simp-like paranoid tale, but I really believe that is the gist of what happened. Every issue the FDA has raised so far has turned out to be easily refuted by the company, so why would this be any different? Why do you think CG pulled out the slides for the last presentation - to show that anyone with half a brain would look at the dystrophin measurements and think, "even if they are 50% off in their post-treatment measurements, there is something real going on here" - instead they got "marked disparities".
flex - I am in a similar position, having bought all my shares in early 2012 and having no plans/desire to liquidate them until the full value of the DMD program is realized, at which point I will probably sell half and sit on the rest to see where the platform can take the company. But that doesn't minimize my frustration with how this company has been jerked around by the FDA. I can sympathize with the people who bought into the promise at much higher prices only to see capricious and senseless backtracking by the FDA cut their positions in half and/or force them to liquidate if they bought on margin. I can also relate to people who bought shares with shorter term investing goals in mind, having those plans to liquidate in order to cover the cost of a car, tuition, vacation, etc. trashed by what has happened over the past 18 months. Consider yourself lucky to have the luxury to sit back and be psychologically unscathed by the endless hoops the FDA places in front of this company, but please don't dismiss the frustrations of those that don't have that luxury. Finally, if you have paid enough attention to social media postings of the moms of DMD boys and the hopes that have been dashed and fears they live with every day for their sons, there is nothing here to laugh at.
I recall posting a couple years ago about how an FDA official (I think it was Temple?) made a presentation to PPMD about the accelerated approval process and specifically how they approach it in the case of rare and deadly diseases where there is limited clinical data and I was struck by how he talked about how important "patient reported outcomes" were in the process of determining whether AA was warranted. Obviously that was a load of BS, as you have had parents of these Etep boys screaming about how their boys are thriving on this drug for the past two years to no avail. So, if it isn't clinical data, surrogate data or patient reported outcomes (SRPT has all three), what exactly is it going to take for the FDA to do the right thing? I really believe our last hope is the expert panel review, which will now probably take place in Q3/4 of 2015 - if there is any justice the experts will lambaste the FDA reps about how Etep should have granted AA two years prior.
What a ridiculous commentary - "management doesn't know how to get a drug approved"? They only have one drug and it has followed the normal course of clinical progress with no significant issues at this point - what do you expect him to do, shortcut the Phase III trial process? The company's limited market cap make it difficult for many institutional investors to take a meaningful position in, so you are at the mercy of the fickle retail environment, and in the absence of buyers, even limited selling can push the price down to ridiculously low levels. The company may not be as aggressive in hyping TH-302 as some other companies, but they attend most conferences, have the backing of Merck and several legit analysts with double-digit price targets - what more do you want?
I think the question is what factual evidence do you have that trading was halted? If it was halted, there would have been a formal announcement. I agree that the call had a lot of positive aspects to it, but the idea that the company would have requested a halt in trading to announce earnings that were "spot on" with analyst estimates seems a bit far-fetched.
It is clear to anyone with half a brain that any "conflict" that the FDA has with SRPT or CG is simply because CG has been completely transparent about the company's communications with the FDA. He did this in order to provide the investment community clarity around the AA process in general and the specific requests the FDA was making with respect to the Etep filing. The FDA hates transparency - they hide behind the cloak of confidentiality and don't want their decisions scrutinized in the light of facts. Those of you who bash CG for the way he has handled this are in essence supporting how the FDA has handled it - in other words, you're morons!
THLD has fallen and it can't get up. It seems the small-cap biotech market is back to the days where you need positive pivotal data to move the stock. I've seen this type of rut before and you can never be sure when the wheels escape the rut and investors start focusing on the potential multiple at hand. There has been nothing but positive news from this company over the past two years, they are within a year or so of two pivotal trials being completed and the share price is less than half of what it was 3 years ago (after the Merck deal) and less than a quarter of its consensus analyst target. Go figure?
Unfortunately, I had a few small biotechs that were down over the past year and saw the same type of action on Friday. I've heard the term "window dressing" used to describe the mutual funds dropping losers before their annual report date - more like a dressing down!