Key Takeaway: Thank you. Mr. Tattory, you may begin your conference. John Tattory: Thank you (Melissa). Good morning and thank you for participating in Discovery Labs conference call. This morning's call will provide an update regarding the company's Surfaxin complete response initiatives.
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Full Press Release Details
Thank you. Mr. Tattory, you may begin your conference.
John Tattory:
Thank you (Melissa). Good morning and thank you for participating in Discovery Labs conference call. This morning's call will provide an update regarding the company's Surfaxin complete response initiatives.
Tom Amick:
Thank you, John. And thanks to everyone who is participating in this call. I know you all have busy schedules, but we really appreciate your interest - continued interest in Discovery Labs and our Surfaxin technology.
On January 10, of this year, we did issue a press release in which we detailed to you that we had received direction from the FDA. In that press release we also committed to get back to you to give you a further update. Today is the purpose of that update.
On the regulatory front I am very pleased with the recent progress relating to Surfaxin Complete Response initiative. And I'm joined today by our Vice President of research and development, Dr. Rusty Clayton, who will provide additional perspective in this regard. Rusty, please.
Rusty Clayton:
Thanks, Tom. As some of you will recall, since our last complete response letter from the FDA, our primary regulatory focus has been the execution of a nonclinical program that was intended to support the FDA approval of Surfaxin.
That concludes my comments and, with that, I'd like to turn the call back over to Mr. Amick.
Tom Amick:
Thank you Rusty. As you can see, we're at a very important point in the history of our company. I'm pleased with the progress that our technical team has made during the conduct of the comprehensive nonclinical program, which is intended to support the Surfaxin Complete Response filing.
John Tattory:
Good morning (Kim).
(Kim Lee):
Good morning. Thank you so much for the clarifications on the Surfaxin program today. Much appreciated. Just have a few follow up questions here. First is as far as optimization goes, what percentage reduction in assay variability relative to the prior BAT methodology do you think is acceptable to the FDA?
I know you've found the percentage to be 40 percent but do you think that's a reasonable reduction or do you think the FDA - has a different number in mind?
Rusty Clayton:
Yes, Kim. This is Rusty Clayton. Thanks, for the question. That's a really difficult question to answer. First of all a biological activity test of any sort, the baseline variability range is very wide.
(Kim Lee):
Understood. Great. And can you remind us what is the proposed shelf life of this optimized BAT?
Rusty Clayton:
Well, there's - the - I think what you mean to ask is what is the proposed shelf life of our product, Surfaxin
(Kim Lee):
Right. Right, using this - yes.
Rusty Clayton:
Yes. I'm sorry.
(Kim Lee):
OK, very helpful, thank you. And what further data is needed through - for the concordance testing?
Rusty Clayton:
We had proposed some data that we had generated in both the rabbit and the lamb. Some of those data have already been generated. We had asked that question to the FDA specifically and they had indicated that they would like some additional time points with some additional lots tested in both the rabbit and the lamb.
(Kim Lee):
Finally, look - I guess looking at the bigger picture, will this final stability validation be necessary to be used in the rest of your programs, like Surfaxin LS, Aerosurf and KL4?
Rusty Clayton:
The release and stability of the KL4 surfactant product, we believe, at least in the United States, will include a biological activity test. And it is our intention that this biological activity test optimized, as it is, and with as much effort as we've spent on this, will be used for the release and stability of all KL4 surfactant products.
(Kim Lee):
OK. OK. So, just to liquid form. So any aerosolized product will not need - would not - you would not make a use of that test. Is that correct?
Rusty Clayton:
Well, if you're going to aerosolize the surfactants before you even aerosolize the surfactant you have to assure that it's biologically active. And then that the rest of it becomes a developmental issue, what we have to demonstrate and what we have demonstrated so far to date is that when we aerosolize our product it is biologically active after aerosolization.
(Kim Lee):
OK. Thanks, again for the clarity.
John Tattory:
Thanks, (Kim).
Operator:
Your next question comes from (Larry Smith).
(Larry Smith):
Hi. The FDA, as we've all seen as being ultra, ultra conservative. You know must recently we've just seen today with the (inaudible) where they asked for a long (inaudible) cardiovascular study.
And in the case of Surfaxin the ultra conservative strategy would be why don't you just go out and do a human trial and demonstrate that the BAT is - works in a small human trial. But what kind of confidence or what kind of assurance can you give me - I mean give us that the FDA will accept this bridging study between - on the BAT between the lamb and the rabbit model as opposed to asking for a human study at the end of the day?
Rusty Clayton:
(Larry), this is Rusty Clayton. Thanks, for the question. First, let me point out that in our entire history with this particular new drug application the FDA has not once called into question our clinical program. And at our last face to face meeting with them, and a review meeting in 2009, they in fact indicated that our clinical program from and efficacy and safety point - standpoint was very robust.
So your question really regards to validating the biological assay by doing a clinical trial. And we in fact explored that with the FDA in 2009, early 2010 timeframe. Ultimately, after back and forth correspondence with the FDA, it was determined that we could not proceed with that route. It was not because of issues with the BAT, the biological activity test, or issues with the clinical trial.
So after we, I think, very exhaustively explored that option with them, the FDA advised us that this bridging study would be the preferred method to go.
(Larry Smith):
OK. Thank you.
Operator:
Again, if you would like to ask a question, press star one. Your next question comes from (Shiv Kapoor).
John Tattory:
Good morning.
(Shiv Kapoor):
Good morning. Thanks, for taking my questions. I am a little new to the story, so forgive me if I ask some questions that takes you back in history. But can you - can you tell me what the possible excipients are in your - in your testing and were there certain excipients that historically the FDA's been more concerned about? And what have we learned over the - over your testing about these excipients?
Rusty Clayton:
Well, I can't share some of the fine details with our excipients at this time because that remains proprietary. I will tell you that in our reviews of the FDA, dating back as long as I've been with the company in 2005, they have not expressed any concerns with regard to the excipients.
So we do not have any outstanding issues with regard to excipients, active pharmaceutical ingredients or impurities in our product.
(Shiv Kapoor):
OK. That's good to know. What about the optimization of the dosage of your surfactant? When was that done and were there any questions with relating - related to the activity of vis- -vis the optimal dosage of the surfactant?
Rusty Clayton:
The dosage optimization in general in pharmaceutical development occurs during the preclinical phase and that's when our optimization occurred. That optimized dose was then confirmed in clinical studies and we had a dose that we went forward in the Phase 3 clinical trial. That Phase 3 clinical trial was actually constructed with FDA input all along the way and ultimately accepted by the FDA.
Tom Miller:
The results of that successful phase three clinical initiative, again, has been published in "Pediatrics." The efficacy associated with our selected dose for that trial is quite comprehensively depicted and I would advise you to review those manuscripts.
(Shiv Kapoor):
Fair enough. One more question. Once you have the filing complete perhaps by third quarter, how long - how long of a response do you think the FDA - how long would you think the FDA will take? It seems like you've been in very active discussions with them.
Rusty Clayton:
Well, I mean to be fair to the FDA, with regard to our status as a new drug application, and given the data that they need to review, the guidance that we are providing is that the FDA will deem this a class two review and this typically, by guidance, will take six months.
So once from the date we - they receive the complete response, we expect that they will issue a PDUFA date within six months of that submission.
(Shiv Kapoor):
OK. Thanks, a lot.
Rusty Clayton:
No problem.
Operator:
There are no further questions at this time.
John Tattory:
OK. Then I would like to thank everyone for their participation, thank everyone for their questions and look forward our next conference call that will be later on this quarter. Thank you all very much.