This really is a bi-polar market - you have so many stocks trading near 52-week lows, while the indices are near all-time highs. I suppose it makes sense that if most of the money going into the market is in the form of index funds/ETFs, the only action will be in the big-cap names that drive the indices?
You are reinforcing my whole point - you can't make precise assessments on what the change in 6MWT means in terms of overall efficacy. I agree that the "market" may react to further 6MWT declines as if they are meaningful, but do you really believe you can be that precise in assessing an average decrease? If one boy becomes non-ambulant, the average is sure to be decline by more than 30 meters, even if two or three remain stable on 6MWT - should the average 6MWT score be the "be-all, end-all" yardstick, even if they all remain stable on pulmonary and arm strength tests?
mauouo - you remind me of the analyst on the call that was pressing the idea that the company would need to "correlate" 6MWT scores to the dystrophin production numbers. As CG has pointed out many times, the twins who lost ambulation also have robust dystrophin measurements from the biopsy of their ARM muscles - that has little to do with how much functional LEG muscle remained at they time they started treatment and how quickly they will lose ambulation.
The enrollment criteria of baseline 6MWT and age is intended to isolate boys that have a certain level of functional muscle that can be salvaged, but that is an inexact methodology. IOW, the boys' post-treatment 6MWT performance is more a measure of how bad their legs really were at baseline than how well Etep is "working". If five years from now all twelve of the boys are in wheelchairs but their pulmonary function is stable and their arm muscles are still functional, did Etep "work"?
Your commentary is reinforcing a false premise/storyline - i.e. if the boys in this trial lose ambulation as they age, they have not been "cured". For DMD, a "cure" means salvaging the remaining viable muscle tissue, not restoring or regrowing muscle tissue that has been destroyed. The issue of how much functionality can be retained by the remaining viable muscle tissue has many factors at play - in the case of the boys in this trial, one of the most significant factors is how much height & weight they put on as they age. For some boys a cure may mean only retaining their pulmonary function, for others it may mean staying out of a wheelchair until their late teens, and hopefully for boys treated from a very early age it will mean a relatively normal life. The bottom line is that for a group of boys that have already lost a substantial portion of their leg muscles and are hitting a period of rapid growth in height and weight, the 6MWT data cannot expected to be stable. Anyone looking at the data and thinking "if the drug worked, their 6MWT scores should be stable or improving" doesn't understand the disease or how Etep works.
Wrong - the market is finally catching up to value of this company - Migalistat alone is worth the current market cap and it will be on the market in 2016, but the value of Migalistat pales in comparison to the value of their next generation ERT's. The share price will be $15+ upon approval of Migalistat and $50+ within 3-4 years.
So the basic message is you had no clue what was going on with the company, so you bash news because it goes against what you thought you knew, and now you are moving on to another "bet" where I'm guessing you are equally clueless? Thanks for sharing!
The "horse" in the case of Ebola has to be a govt contract - pursuing a pivotal trial/approval without a govt contract would be a waste of resources.
"Encouraging" because these patients were progressing on every other available treatment. I doubt partial responses and stable disease is going to create any buzz, but nothing else has accomplished this.
Nothing is going to change the filing time dramatically. Regardless of how positive the upcoming data may be , the FDA is not going to accelerate anything - their MO has been to stall until they figure out what they are doing. Do you think by some miracle they will see the light over the next two months and say don't wait until June, file it in April?
I knew that, but sometimes the market reaction to a giveback dismisses the nuances. I didn't think it was an antitrust issue facing NVS, but rather the simple conflict of having two potentially competing drugs being sold by one company?
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.