immediate - you are dead wrong when you indicate the 4th biopsy results will be a "minor addition" to the NDA. The 4th biopsy results are significant because they will counteract any questions that may arise from the 6MWT data. As has been discussed previously, there is a contingent of ignorance out there that believes if Etep was working that there would be no declines in 6MWT performance for the 10 boys. The 192wk data will likely show further declines and raise ignorant questions about whether there is enough dystrophin being produced to be effective or perhaps the levels measured at wk 48 are not holding up in the long term. The 4th biopsy will help to counterbalance these questions and reinforce long-term effectiveness of the treatment.
mauouo - You've answered your own concern when you say it "seems management is very confident". Everything they are doing with respect to filing, hiring, financing, etc. indicates that they feel very good about the hand they are holding, which they have yet to reveal. When the dystrophin re-reads and 4th biopsy figures are revealed, it will all make sense. The FDA challenged them with their ridiculous accusation of "marked disparities" in the measurement of dystrophin last October and now the company has the data to put the FDA in its place - they also know Biomarin has no comparable data.
Accepting a filing is simply acknowledging that all the necessary components of the filing are complete - it means nothing regarding the substance of those components or is any indication the FDA is acknowledging the filing supports efficacy or safety. Most companies wouldn't file a NDA based on a failed Phase III trial not because they didn't think the filing would be accepted, but because it would have little chance of approval and a waste of company resources. Acceptance of the filing is a non-event.
I think the binary event will be the Advisory Committee, which hopefully be early Q4 at the latest? I doubt the FDA decision will come through before the end of the year, despite the fact that they have had access to much of the data for months/years and with the size of the trial, the volume of data is limited.
They haven't announced a specific date, but have spoke to it in their last several presentations and that it will be in the 3rd quarter, after the data is unblinded from their Phase 2a trial.
Do people think they "need" the analyst day to spin the data because it is still not definitive or will it be an "in your face" presentation that will overwhelm all the doubters with clearly positive data from the multiple studies? They have spoken about bringing "key opinion leaders" into the presentation - to me that sounds like they are looking to say "you don't have to believe us" about the data, because they are not sure how it will be received? At the core, they have acknowledged from the beginning that they aren't sure what it will take to create a "functional cure" in HBV - they are blazing a new treatment trail - so, regardless of where the numbers fall out, I guess it will still take a long-term, pivotal trial to really generate "proof of concept". Either way, with the other programs progressing nicely, ARWR seems way undervalued?
I love it - the guy is arguing that a kid on Etep would "look funny in school" as a competitive advantage to Drisa. Beyond on how stupid that statement is on so many levels, it won't be the "kid" who will be making the decision on what drug to use - it will be his doctor and parents. The safety profile of Etep alone is enough to push the decision in Etep's favor, but when it is combined with Etep's clinical proof of dystrophin production (when Drisa has no meaningful dystrophin data from its late stage trials), Drisa won't even be part of the conversation between parents and doctors.
He has been at the helm for 15 years now and has struck out on every major milestone. The only thing he has been successful at is dilutive financings and getting his comp raised. His cash comp last year (salary + bonus) was $821K - I'm guessing he has pulled out close to $10M out of this company in the last 15 years, while shareholders are left with nothing. Good work if you can get it!
Doesn't matter when Drisa hits the shelves - Etep will be the drug of choice by both parents and physicians. In the unlikely event Drisa gets approved before Etep, you will probably have parents wait until Etep is available, instead of starting their kids on the toxic Drisa and having to switch later.
The fact that they didn't do a conference call to discuss the agreement and the lack of any pre-market action pretty much confirms what I said - this is nothing that is going to move the needle on the company's share price. I'm not saying it is a bad agreement - the company made the right decision to focus its limited resources on the rest of the pipeline, and any incremental revenue will help the cause, but for a company with a market cap of ~$750M, this agreement is immaterial.
I don't think the "market" will respond much at all - given the clinical history of OMS103, the market had probably written OMS013 off as meaningless to the company's value. Given the structure of the agreement, with sales targeted to start this year, it is clear they are not going to do any additional clinical work that would support an FDA approval/label and CMS/insurance reimbursement, like Omidria has. Therefore, Fagron will be selling it as a "compounded" product at presumably a very moderate price point. They note 5 million arthroscopic procedures is the target market - I'd be surprised if they penetrated 10% of that market over the next 3-5 years, so the revenue potential is not anything that is going to move the needle on the share price.
The fact that they were so quick to lay off such a large part of the of the employee base means that they have no control over when or how the reimbursement issue will be resolved, or at least have no confidence that it will happen any time soon.
After listening to the recent presentation, I'm on the fence - the technology sure seems impressive, but there are so many hurdles to clear for widespread adoption to take place. For example, in the radiation treatment for breast cancer, their single treatment is supposed to as effective as weeks of traditional radiation treatments, but if radiologists adopt the iCAD treatment, they will effectively be slashing their revenues drastically, since they get currently get paid/reimbursed by the number of treatments. This is why they believe the breast cancer treatment market outside the US will be more significant (because countries w govt controlled health care will jump all over the cost savings), but that is a long, arduous marketing task.
This situation reminds me somewhat of GTHP - they have a technology that is clearly superior to traditional pap tests (quicker, less costly, just as accurate, etc.), but the billions being made by the institutions controlling the status quo will not allow it to be adopted in the US, except possibly as a secondary screening method.
simp - "consistent with prior disclosed readings" is not the issue with the dystrophin measurements. What makes them material information is the cloud the FDA placed over the original readings with their ridiculous assertion of "marked disparities" in the methodology used to generate the IHC measurements - the re-reads are material in clearing up that cloud and the 4th biopsies are material because they provide evidence of long-term efficacy, which is critical given the limitations of the 6MWT data. The company is currently withholding this data under the semi-legitimate excuse that the readings are immaterial until the FDA decides whether the data is sound and whether it will be deemed sufficient to treat dystrophin as a surrogate endpoint.
Unfortunately, that is not how it is typically handled in the biotech world. If funding is going to be needed shortly after a NDA decision, most biotech companies won't roll the dice on the FDA giving them a full and timely approval - they'll raise money before the decision. If that happens in the case of SRPT, I think it would be borderline illegal for them to do an offering without disclosing the results of the dystrophin re-reads and the 4th biopsy, so hopefully that will provide some strength going into an offering - ideally they would wait until after the AdCom?
What would really move the stock price is if they actually secure a partnership/distribution agreement for Europe. They are two months away from getting approval and they don't an agreement, yet - that is not how it is usually handled. Think of all the preliminary work that needs to be done to get this drug distributed through the various countries' health systems - the launch of this drug in Europe will be slower than it was in the US - remember, they got approval in the US in June 2014 and they just launched it in Apr 2015. It will probably be mid-2016 before Omidria gets launched anywhere in Europe.
I actually appreciate how they did a prelminary/limited launch in the US in order to ensure the reimbursement and other distribution mechanisms were in place before going to full launch, but it seems they didn't even start a lot of these activities until months after approval was received? If a partnership in Europe isn't announced until after full approval, we can expect the same drawn out process.
bionerd - do you think that dystrophin biopsy data from the new trials will be part of the rolling submission or just safety data? I believe the FDA only requested safety data from a certain number of patients as part of the submission and frankly the safety data, while important, is not a game changer. Assuming the biopsy re-reads and 4th biopsy data is clean and shows meaningful dystrophin levels in all of the boys, then you wouldn't think they would need dystrophin confirmation from the new trials and obviously there won't be any meaningful 6MWT data by mid-year from the new trials. Assuming the formal filing does not include any dystrophin or efficacy data from the new trial, but the AdCom is scheduled for Q4, does that open the door for additional data to be part of the AdCom discussion or is it limited to the data from the formal midyear filing?
Thanks, left_e - I went back and looked at the clinical trial description and the primary endpoint is the change in VA scores from baseline, so if Isonep does stabilize vs continued deterioration in the control arm, that will be a success. I seem to recall an endpoint that required an improvement in the # of letters read off some chart, but that is a secondary endpoint.
This is an interesting point - if Asonep is proven to reduce lesion size, but misses on improving VA, is the drug a bust? Like most degenerative diseases, I would think it would be meaningful to provide stability/lack of progression, while the hope for reversal or improvement might be a pipe dream. Did Lucentis and Avastin actually improve VA in their trials - if so, I assume that the improvement was only temporary, as most patients ultimately progress, right?