They already ran their pivotal/Phase III trials and they failed. Per their last earnings call, they have had no discussions with the FDA about the design or requirements for of any confirmatory/Phase IV trials. As jrrt posted about earlier this week, once a drug is approved, recruiting patients for a clinical trial in that drug becomes very difficult - if Etep is approved, BMRN would have no chance of recruiting any patients into a confirmatory trial.
What AF fails to point out is that theoretically, SRPT has a much lower bar to clear, given that they are filing for conditional/accelerated approval. BMRN has made it clear that they aren't running any confirmatory studies, so they have to rest entirely on the data in hand, which is weak, at best. If the FDA accepts the biopsy data as confirming the presence of newly produced dystrophin, then between the pulmonary data and the patient reported outcomes, how could they not conclude that there is a "reasonable likelihood" of clinical benefit.
jrrt - The recruiting issue is an interesting one - the sad fact is that some families may not have insurance and the only access to the drug may be through participation in a clinical trial. I can't imagine the FDA (in the face of a positive AdCom decision) would delay approval in order to force parents into participating in the confirmatory trial?
bf - I thinking you are giving the FDA too much credit. They don't have the balls to to take a stand - if they did, they would have kicked BMRN's NDA to the curb, where it belongs - no dystrophin data, failed primary endpoints and clear safety issues. As much as I would like to believe their filing of SRPT's NDA is a sign that they are coming on board, I think they (again) don't have the balls at this stage to take a stand on Etep - they will push that responsibility to the AdCom. Ultimately, between the new dystrophin data and the AdCom results, I hope the FDA is left with no choice, as we have seen that when it is not spelled out for them, they tend to mess it up.
Simple - 192wk and dystrophin data at WMS meeting in early Oct and then the AdCom meeting in Nov. Not saying it might not be volatile, but it will be above $48 by Thanksgiving.
Yeah, money and a fancy marketing brochure is going to change doctors' and parents' minds about what is the safest and most effective drug for their terminally ill child. Even if marketing muscle was important in this situation, which it is not, it's not as if BMRN didn't have the financial resources prior to the MDVN deal to market Drisa aggressively. They can throw as much money as they want at Drisa - it will always be an inferior choice to Etep.
Thanks, jrrt. I hope the FDA assesses the 6MWT data in the same manner that you have, but I think the dysrophin data is even more important - not just in providing further evidence to support approval, but in providing a clear distinction between Etep and Drisa. The idea that you can have an effective drug without providing evidence of dystrophin production is just senseless - that is the drug's intended "mechanism of action", so for Biomarin to file a NDA without any solid evidence of consistent dystrophin production is laughable, IMO.
They don't rescind BTD/Priority Review/Fast Track based on trial results, just like they don't grant them based on probability of approval - it is based on the severity of the disease and the lack of effective treatments. You're as clueless as they come.
simp - you moron - how long have you been on this board - the Etep dosing was at 30mg and 50mg!
In what sense were my messages anti ARWR - I was as pleased as anyone to see that a reputable? analyst had put a huge target on the potential value of ARC-520, but when someone makes an illogical statement claiming that the value estimate by Goldman could be doubled simply by partnering it, I questioned it - how is that anti-ARWR?
post - I am long ARWR - I just questioned his senseless post and he got bent out of shape. Apparently, there are a few people on this board that consider him knowledgeable?
holden - With every new "lesson" you provide further evidence of how clueless you are. Let's assume Goldman is not as clueless as you and as part of their future projection, they took into account the dilution involved in getting ARC-520 to the finish line, including building the marketing infrastructure, etc.. Let's assume the purpose of their valuation was to show what the full potential value (not just US markets and not at some discounted value adjusted for execution risk) of a FC HBV drug would mean to ARWR's share price - the $150 number (even at a pre-dilution levels) represents approximately $10B in market cap. But without even knowing the assumptions that went into Goldman's valuation (it is likely they assumed a marketing deal outside the US), or specifying the scope of your fantasy "strong partnership deal, your "expertise" allows you to speculate that a partnership" would add another $10B of value? What a tool!
kart - if holden really meant that, that is what he would have stated. If the Goldman value theoretically did not include global markets or an inefficient marketing infrastructure, he could have specified those things, but he didn't - he made his comment based purely on a partnership doubling the ESTIMATED FUTURE VALUE of ARC-520 to the ARWR shareholders and I called him on his stupidity. Now he is trying to play professor and it is making him look even more clueless.
holden - What a moronic retort - I specifically challenge you on the premise of your post (that the Goldman value is a future value, not a DCF value taking into account the risk of clinical and commercial success) and you give me CAPITALIZED definition of a DCF valuation. Answer the question - is the $150 value you cited a CURRENT DCF value (i.e. based on todays facts and circumstances)? Secondly, I never stated ARWR shouldn't give up revenue/profit share in a partnership, I simply stated that giving a partner a share of an asset's future cash flows BY DEFINITION reduces the potential value of that asset to the company. If you are refuting that, you truly are a moron.
You're playing mathematical masturbation and the result is an illogical mess. First off, Goldman's value is not a current "present value of future cash flows discounted for risk and time", it is by your own admission a future value - i.e. it assumes the risk of clinical and commercial success (including putting together a sales force) are taken out of the equation. Your declaration of a partnership resulting in the use of a "dramatically lower discount rate" is stating the same thing I did - i.e. you would see an earlier spike in the share price. This is meaningless in the context of Goldman's future $150 value, which assuredly does not include the sharing of future profits or payment of royalties. Face it - your statement that a partnership would double Goldman's future value estimate is senseless - accept it and move on!!!
What you talkin' bout Willis? Let me re-state the issue as clearly as possible - Goldman supposedly placed a value on ARC-520 (assuming it is a FC) that resulted in a per share value of $150. The value of the drug is presumably based on the profit it would generate from future sales. In what universe would giving a partner a cut of the future profits (or royalty payments that would reduce future profits) result in a greater value of that drug to the company, unless the upfront payment and milestones are greater than offsetting profit-share/royalties? It really is a simple question of math - unless you believe a prospective partner is stupid enough to strike a deal where they pay the company more than they get back?
How would a partnership double the value of the drug - most partnerships involve giving up a chunk of the profits for some upfront money and clinical development support. Assuming ARC-520 is a success, a partnership deal would provide an earlier spike in the share price, but it would reduce the potential long-term value, unless there is some new finance/math paradigm I am not familiar with?
Has there been a "debate"? I have read numerous stories or twitter posts from parents of Etep boys convinced that Etep is providing true clinical benefit and now a story of parents flying coast-to-coast to get their boy in a trial. Has anyone seen any similar stories or postings from parents of Drisa boys - there certainly are many more of them out there than boys on Etep, but not for long.
usingaliase - You sound as clueless as Tran - read the press releases and investor presentations, listen to the investor calls/presentations and decide for yourself if Tran's "issues" are valid. His two most prominent statements about the trials are clearly false - if you can't grasp that, then you should be on the sidelines permanently.