fox - no worries. I wonder if the parents of the Etep boys are going to be part of the advisory panel hearing or whether it will be limited to discussing the nuances of dystrophin, 6MWT, etc. If their stories are not part of the equation, that would be a crime against the rest of the DMD population.
Sawyer's mom, who reported last month that her son more than doubled his running endurance in a "Pacer" test at school, reported on Thursday that Sawyer's 6-Month check-up showed "Improved Strength, Improved Lung Function/Capacity and increased ROM (range of motion)". At some point, even the most clueless naysayers will have to acknowledge that the improvements these boys are experiencing on Etep trumps any issues about dystrophin measurement issues, Intent to Treat issues, and any other neurotic issues they may have about the Phase II trial. Reasonably likely? Not even close - it is "bloody obvious" that this drug works.
simp - The timeline for submission is end of the year - approval is not expected until mid-2015. This has been known since the last press release/call and was just reiterated at this morning's presentation - why would claim a year-end approval is possible?
Given the recent guidance by the FDA, the earliest Sarepta COULD have filed the NDA was sometime in the 3rd quarter. They are delaying that 3-4 months in order to bring certain information into the NDA and allow the FDA time to do a more thorough review of their dystrophin measurement methodology. Assuming the FDA has no issues with that methodology, that will open the door for the potential for a 4th biopsy on the Phase II boys to be done and included in the NDA. Personally, I think it is shameful that the FDA is asking for this - the clinical data is overwhelming enough to establish efficacy as being "reasonably likely" and confirms the validity of the dystrophin data. Delaying the NDA also allows 144 week data to be included in the NDA and 168 week data to be available during the review period. Finally, the FDA required that the confirmatory studies be well along as part of any approval, so the delay gives them time to get those studies enrolled and early safety data to be available by the time the FDA makes a decision by mid-2015. IMO the FDA's ignorance has delayed Etep getting to market by 6-9 months (the company had to get manufacturing up to speed, so it wasn't all on the FDA), so SRPT taking another 3-4 months to make sure they give the FDA no wiggle room on the approval is a smart thing to do.
GSA - Good point on the strong growth of PINPOINT and LUNA equipment sales. This was supposed to be a razor-razor-blade story, with all the profit driven by recurring kit sales, but it is clear they are getting good margins out of the equipment sales, so in the near-term, strong growth there can drive us to profitability. Still missed on the EPS number by $0.03, so not sure the market will reward us?
A ho-hum number of $10.3M - the good news is that there was a sequential increase in equipment sales, but recurring/kit sales were flat, partly due to pricing on kits for sold vs leased equipment. Overall, the gross margin rate (65%) increased from last year, which was good, but the net loss increased, as sales and marketing expenses grew faster than sales. Story still in tact - not sure how the market will respond?
endo - It was discussed earlier on the board that the Jan preannouncement was not because the strength of the number, but because they were presenting at the JP Morgan conference the next day and wanted to include the Q4 data in the presentation. I agree that the chances of a $12M+ number are slim, but anything less than the $10.7M posted in Q4 would be the first sequential quarterly decline in over a year. I'm hoping to see $11M and change?
EU is dead - that is already priced into the stock. EU was considering a "conditional approval" for ovarian cancer - i.e. allow marketing the drug while the Phase III trial is underway. Without a Phase III trial, the conditional approval is not possible - everyone knows that already.
It takes a special kind of person to totally misinterpret the facts of a situation, endure an onslaught of people telling him he is a complete moron for that misinterpretation and then carry on with the same gibberish. I'm not sure there is a proper name for vukken - #$%$ is politically incorrect and vukken would tarnish the reputation of the mentally challenged. Where do these people come from - 16.6% must be the portion of his brain cells that are still functional?
To put some context around this, the Phase III PROCEED trial enrolled patients whose lesions were 100% folate-receptor positive (FR100). In the Phase II trial for FR100 patients (n=38), the combination arm had a PFS of 5.5 months vs 1.5 months in the control. Even in the full population of 149 patients, the control arm PFS was 2.7 months. They knew that FR100 patients historically had worse outcomes, so the 1.5 made sense in that context, and there was never any hint that the 1.5-2.7 month PFS was anything out of the historical norm? So, what went wrong here to drive the control arm PFS to double over historical outcomes?
What rock did you crawl out from under and why did you decide to defecate here. Obviously, you are clueless as to this company and its prospects, so the only mystery is why you felt the need to prove it to this particular MB?
They said on the call today that they were looking to provide more detail on the lung cancer trial, including OS data on the combination arm, at ESMO, which is at the end of September. Whether the ovarian collapse will change that schedule, who knows, but within 6 months you will have a read on Merck's commitment to vina and some preliminary data on the Phase I folate-tubulysin drug, which has much stronger pre-clinical data that vina. The PSMA-tubulysin trial probably won't have data until very late this year. I always considered the tubulysin program to be the payoff here - if vina was successful, Merck would capture most of its value, but ECYT owns 100% of the tubulysin pipeline and has plenty of cash to move it forward.
Correct - the median survival numbers for the various arms would not have caused the 4+month delay, so is there a "long tail" of survival in the treatment arms, or maybe the placebo arm?
Exactly - even with the lower guidance, they will probably earn $2 this year. Unless you believe the search business is going to evaporate completely over time, there is value here.
I'm always fascinated when people take the time to post some detailed bashing of a stock that, just by making a couple of clicks, is easily discovered to be total BS. Chegg had $137M of cash and investments (inc $24M of long-term investments) at the end of 2013 and at the end of the most recent quarter that number was $131M (inc $37M of long-term investments), so they used up $6M during the quarter. At that pace, they have about 5 years worth of cash, but given the growth in the digital business, they will probably be cash flow positive by the end of this year. Your humble opinion is garbage - did you even look at the press release before you made up this story? LMFAO douchebag!
kart - yes, I am still in FOLD and will hopefully be taking on more before the 2nd Phase III trial results are announced - people have written off Migalistat, due to the messed up statistical analysis used in the 6-month results of the first Phase III trial, but that is a mistake. It will ultimately be approved and will generate substantial revenue, because (1) it is an oral drug, versus IV infusions required for enzyme replacement and (2) enzyme replacement poses a lot of issues with dosing and immune responses. The one overhang on the company is that it will take at least 3 years before any of the their "next generation" ERT#$%$ the market, so they will need a lot more cash to get there, and because of the skepticism over Migalistat, they may need to raise money at dilutive prices?
Exactly - in 1st line pancreatic cancer, median PFS with Gem is about 3-4 months and OS is about 7-8 months. Abraxane added less than 2 months to that equation and was approved. The 1.9 months improvement in OS for MM-398 is a more dramatic increase to the 2nd line equation - i.e. a 50% improvement vs a 25% improvement for Abraxane in 1st line. As some other posters pointed out, given that the 1.9 month improvement was statistically significant at P
Makes sense - given the funk the biotech market is in, most of my holdings are trading at about 40% of analyst estimates. It is hard to rationalize values at times - this stock was trading over $8 about 18 months ago - the pipeline has advanced dramatically since then and they are within a year of generating significant revenues with MM-398. So, was it a case of crazy buyers 18 months ago or is it crazy sellers today?
The interesting thing about your "disappointing" estimate of $10.1M is that it is right in line with analyst estimates of $10.3M, which is consistent with the company's 40% revenue growth target for 2014 - Q1 of 2013 revenue was $7.3M. No doubt there could be some seasonality in the placement/sales number for equipment, as Q4 is traditionally the strongest month for hospital equipment sales, but if they do less than Q4 2013's revenue of $10.7M, that will be the first time in over a couple of years without quarterly sequential growth. I think there is enough momentum in the recurring/kit sales to keep the sequential revenue growth train going, so I like your $11.5M revenue number. What the share price does with that number, who knows - we are at a very fickle time for the market, in general, and biotech even more - a lot of low volume, high volatility days?