copp - your "humanitarian" argument is consistent with CG arguing in the past on why the company has to be focused on getting the right trial done as quickly as possible - that is the quickest way to get the drug approved and before it is approved, it can't help anyone. SRPT has already focused the company's resources on getting the confirmatory trial started in Q1, which means they probably already have the sites and clinicians set and will probably start screening patients in the 4th quarter, and your argument is to drop all that and step into the mess created by GSK and take over their trial sites and patients. I'll say it again - what an idiotic post!
What an idiotic post. As evidenced by by SRPT's trial, the most critical aspect of the trial is choosing the right patients - i.e. boys that are on the verge of starting to lose leg strength. The boys in the Prosensa trial are already on the decline and by time SRPT's confirmatory trial starts, many may have already lost ambulation. Not to mention the fact that the last thing you would want is a patient to be tainted by any effects of a prior exon-skipping therapy.
This "may" be wrong - are you kidding me? Anybody betting on the failure of Drisa as a sign that Etep doesn't work is simply clueless - Drisa failed to produce ANY dystrophin in a large chunk of patients, while Etep produced robust amounts in every patient.
life - do you know if any new data was released at SOHO - is it something that the company should be issuing a press release for? You seem to be all over this stock - what is your timeline for the next major clinical milestone? Thanks.
thig - exactly - the DMD community is very tight and vocal - where are the parents of the boys in the GSK trial posting pictures/videos/stories of the remarkable changes they are seeing in their sons? Instead, you hear rumors of drop-outs, injection site issues, other side effects and parents/boys being treated poorly. They may be able to spin the clinical data to generate some hint of a benefit or to minimize the safety/dosing issues, but that is not going to fool the parents or their doctors into believing that Drisa is in the same class as Etep.
t_c16 - you are correct - in fact, I was thinking about adding to my position, but was worried about the coming dilution. I originally bought a stake thinking they would partner OMS302 in order to minimize any dilution. Then the raise they did in May at $5 was only a about 3 months worth of operating burn. This analyst upgrade doesn't change that risk - not really sure why it carries more weight than the several other analysts that had targets in the $15 range?
It is amazing that there were 6M+ shares short. It's not as if this was a high flying biotech - it is trading about half of what they priced a secondary last July and in the interim had a succesful PIII trial,filed an NDA and generated some pretty impressive early clinical data for the rest of the pipeline. With a number of legit analysts having targets in the $15 range even before today, what was the short thesis?
solely on the market expectations for OMS302. I would have thought that it was based on the early clinical data on OMS824, their PDE10 inhibitor - this drug truly has blockbuster potential. OMS302 may be a $300M+ annual revenue market, but OMS824 targets a multi-billion $ market. Granted, it is a few years behind 302 in the clinic, but with Phase II trials starting by year-end, you may have "proof of concept" data on OMS824 before OMS302 hits the market.
The short thesis is (1) the FDA would never approve a drug based on a trial with only 12 patients (2) the fact that two of the boys lost ambulation during the study throws doubt into whether the drug works or at least taints the the remaining clinical stats on 6MWT and (3) the FDA's reluctance to verify dystrophin as a valid surrogate is a sign that they are doubting the validity of SRPT's dystrophin measurements or that the dystrophin being produced can be proven to be functional.
Anyone that has paid attention to the presentations over the past two years knows that these issues have been addressed and what little doubt may remain over the clinical evidence proving the effectiveness of Etep is overridden by the glaring evidence from the videos and stories of how Etep has changed the lives of these boys. That is where the shorts are really missing the boat - they believe that this anecdotal evidence has no place or influence in the FDA's decision. To the contrary, the FDA official that presented at PPMD about how the accelerated approval regs work and how they approach the reviews clearly stated that "patient reported outcomes" are a significant part of the review process when dealing with rare, deadly diseases and trials with a limited number of patients.
No doubt - they just raised $27M back in May - $22M of debt and $5M of equity - sold to a William Blair managed fund at $2.60 per share - they must be happy! How can they raise a that much money, spend the next few months talking about how they have the funds to get to cash flow positive and then do this - it is borderline fraud.
Not a scam - it is a venue for micro-caps to get their story out - the companies pay Red Chip for the promotion, since they are too small to get invited to investor conferences. Whether the company is legit and worthy of investment is up to you to decide. I didn't find GALT on Red Chip, but saw a couple of their presentations after I invested - it has been a nice ride from $2 to $10 over the last few months - I'll take that kind of scam any day!
kart - no worries - I know how flaky "response" stats can be - I just didn't understand the charge of misrepresentation? Whatever?
The PR clearly states that the secondary endpoints in the trial (PFS and OS) are not yet available and will reported later. Since it is a single-arm trial, those stats probably will be dismissed, regardless of how good they are, so there is nothing wrong with them announcing the final response data. I don't see anything out of the ordinary here that would warrant an accuastion of misrepresentation - I'm not pumping the data or the stock, just seeing the PR as a fairly standard take on an early clinical trial in cancer, with the exception of distinguishing the percent of patients acheiving tumor shrinkage, which is worthy of mention.
nerd - I understand where you are coming from, but the press release clearly discusses traditional response rates, as well. Correct me if I am wrong, but the standard categories for RECIST responses are Partial Response (20% or greater shrinkage of the tumor); Complete Response (tumor completely gone); Stable Disease (between 20% increase and 20% decrease in tumor size); and Progressive Disease (20% or greater increase in tumor size). The press release discloses the fact that they had a 40% Partial Response rate, but then hi-lites that 92% of the patients had tumor shrinkage - that is to distinguish it from the Stable Disease category, where patients might have had increases in tumor size. You can call that a misrepresentation, but that is a bit harsh.
nerd - When you say they are "misrepresenting" their lung cancer results, are you referring to AF's take on the recent press release, saying they were supposed to enroll 55 patients but only enrolled 25? The fact is that the study design had enrollment up to 55 patients, but they met the pre-specificed endpoint for success (number of responses) in the first 21 patients, so they discontinued enrollment. They disclosed this important fact in a press release announcing the preliminary results, but failed to disclose it in the current press release, leaving it open to false interpretation.
Beyond the lung cancer trial issues, I consider ONCY to be a speculative bet at this stage - but one worth putting a little money into, because if Reo is effective in metastatic disease (as the early results seem to indicate) , the breadth of the market potential is enormous. Having said that, one of the things that impressed me earlier in the year as I was investigating this company is how enthusiastic and surprised the execs seemed to be over the early clinical results they were seeing. However, the CEO seemed downright depressed on both of this weeks presentations and seemed to hint that the pending results in the current Phase III trial in head and neck cancers may not be definitive - he keeps speaking to "Stage I" of the results, which I am not sure I understand, since they are waiting OS data - what results beyond survival could they generate? I know from earlier analysis they have determined that Reo seems to be having a greater impact on metastatic lesions in the trial, but unlike many cancers, in head and neck it is the primary/local cancer that leads to death, so the metastatic impact may not generate an OS benefit. Bottom line - I am cautiously optimistic, but getting more concerned that the current Phase III trial may fail in OS.
They have less than 6 months worth of cash on hand at their current burn rate. Also, given their plans to market OMS302 themselves, they will need a major chunk of cash to build the marketing platform for that. Unless they do a JV for one of their programs with a large up-front payment, dilution is assured - that is why the stock is floundering. They have completely mis-managed their capital - they did an offering in July 2012 at $10.25 and took the gamble that their Phase III results in OMS302 (which are stellar) would propel the price to where they could do a less costly offering. Instead, the price is half of what it was last year and despite their efforts to pump it up with news on their early-stage products, the market is saying " sorry, maybe we'll buy after you figure out how you are going to fund the company's plans for the next two years".
simp - I actually have to agree with you on this one - i.e. no doubt that if they go ahead with a 4th biopsy the results will almost assuredly show that 100% of the boys continue to produce "robust' levels of dystrophin. The question is what that will actually prove that they shouldn't be able to reasonably assess from the current data and/or verify with data from the confirmatory trial. Putting a boy through painful surgery and recovery just to satisfy some curiosity from a FDA official(s) is wrong. The confirmatory trial offers them all the opportunity they need to verify different measurement methods, etc. and, as has been pointed out before on this board, the idea that there needs to be a tight mathematical "correlation" developed between a couple of dystrophin measures from the arm muscles against 6MWT performance over a 2-year period of time is ridiculous. These boys and their families have done enough to show the promise of Etep - leave them alone, give SRPT conditional approval and answer the remaining questions in the confirmatory trial.
I agree - with 100% of the boys showing solid dystrophin production after 24 weeks of treatment, what could another biopsy possibly prove? If they do 24-week biopsies in the confirmatory trial, that data will be available in Q3 of 2014, so they can use the confirmatory trial data to erase whatever doubt they may have before giving approval.
I don't think that is uncommon in this field - most trials for depression or other psychiatric/mood disorders are designed to assume a fairly significant drop-out rate - people get into the trial and hope to feel better and when they don't, assume they are on placebo and drop out. Not much you can do about it.