Q4 2019 Earnings Call
Ladies and gentlemen, please standby your conference call will begin momentarily once again, ladies and gentlemen lease them along.
[music].
Ladies and gentlemen, thank you for standing by welcome to the intercept fourth quarter and for your earnings Conference call. At this time all participants are in a.
Listen only mode, where do we will conduct a question answer session and instructions will follow with the time if your car operator systems drone program. Please first berbizier Warner touched on telephone I would not much of yourself. This conference call must be Francisco head of Investor Relations you may begin.
Thank you good morning, everyone and thank you for joining us on today's call. This morning, we issued a press release announcing our fourth quarter and full year 2019 financial position in results and also posted accompanying slides are available on our website www dot intercept pharma dotcom.
Before we begin our discussion I'd like to note that during our call we will be making forward looking statements, including statements regarding our proof product in clinical development program.
It was pretty matters, including potential approval associated for liver fibrosis good enough.
And our strategy prospects financial guidance and future commercial and financial performance.
Listeners are cautioned not to place undue reliance on forward looking statements, which be big only as of the date of the call and we undertake no obligation to update such statements except as required by law. These forward looking statements are based on estimates and assumptions that although believed to be reasonable are inherently uncertain and subject to a number of risks and uncertainties, some but not necessary.
All the risk factors that could cause our actual results to differ materially from our historical results or those anticipated are predicted by are forward looking statements are discussed in this mornings press release and in our periodic public filings of yes.
Today's call will begin with prepared remarks from our CEO Dr. Mark Pruzanski, followed by those from our Chief operating officer, Jerry to ourselves and our Chief Financial Officer Sandeep, Yeah, well then open the call to take your question season limit yourself to one initial question in order to allow time for all questions to be addressed I mean, I'll turn the call Overdorf CEO [laughter].
Thanks, Lisa and good morning, everyone. Thank you for joining us on our fourth quarter and full year 2019 earnings conference call.
I'm very proud of our achievements over the past you're putting us on a great footing as we consider 2020 and the next exciting chapter for intercept as the pioneer leading the development of a first and foundational treatment for patients with advanced liver fibrosis skewed enough.
Recapping the year I have to begin with the first and so far only successful pivotal phase three Nash readout.
Regenerate 18 month interim results confirmed the robust and taught by brought a efficacy as opposed to yet still the only investigational Nash drug to have demonstrated the ability to reverse liver fibrosis into large well controlled trials.
And given the predominant use of noninvasive tests or Eni teas in clinical practice to assess fibrosis staging our recent scientific presentation, demonstrating that a range of commonly used some and imaging and ickes correlate well with fibrosis change was highly impactful.
It was capped by our reporting additional meaningful improvements in fibrosis beyond months 18 based on fiber scan imaging in our earliest recruited owes you treated regenerate patients who had been on study for up to three years at the time at the interim analysis.
Of course, our phase three results at this stage for the submission of our India now Wonder priority review by the FDA with anticipated approval and launch within the first half of this year.
This was followed by our M&A no under review by the M&A, putting us in position for a potential first up 2021 launch in Europe.
We also continued to deliver exceptional results in our PPC business with a strong finish in 2019 recording a quarter of a billion and net sales representing 40% growth over the prior year and at the top end of our guidance.
We don't caliber now approved in 36 countries, we continue to expand access globally for PBC patients in need.
And it was gratifying to report five plus your treatment data demonstrating durable safety and efficacy in our completed phase three open label extension study, while exceeding 10000 patient years of experience in the PBC population.
Of course, we continue to drive innovation and with a view of enhancing our long term product portfolio. We initiated a phase two combination study of those <unk> as a fibrate in PBC, while making good progress on the formulation on a fixed dose combination.
Finally, we shored up our balance sheet with a 470 million dollar financing to solidify the companys financial position heading into a launch here.
As we embark on 2020, we've maintained our operational momentum into the first quarter as we continue to build on our strong clinical and commercial foundation for the future.
Early in the new year, we completed enrollment of 919 patients in our groundbreaking <unk> phase three reverse study in Nash patients with compensated cirrhosis.
With an 18 month pointed treatment phase, we expect reversed to readout by the end of next year in this important more advanced sub segment of the Nash patient population.
In the meantime, all the pieces are in place as we focused simultaneously on our regulatory review and commercial preparatory efforts and the lead up to you anticipated FTC approval and launch of those Yang for the treatment of patients with advanced fibrosis Judah now.
Well much remains to be done it's gratifying to consider after almost a two decade journey of discovery and development and we are finally on the cost for bringing the first effective pharmacologic option to these patients were truly at risk of advancing to cirrhosis liver failure and premature death.
Before I hand, the call over to Jerry I'd like to review in a little more detail, where we are in the regulatory review process.
First as you know F.D.A. is planning to hold an advisory Committee Radcom meeting with a tentative April 22nd date, and we previously announced an updated PDUFA target action date of June 26.
Planning for an AD com is a major undertaking and we're confident we will be well prepared.
We have the prior experience of our successful AD com proceeding RPC approval and our team has been doing an excellent job to prepare for the one upcoming.
Engaging with a wide range of external experts, we continued to be confident that we'll be able to effectively demonstrate obviously, a strong value proposition for patients and positive benefit risk.
As I, often say, we did not take any short cuts in our extensive development program and have assembled a great amount of data supporting our breakthrough designated drugs safety and efficacy profile in Nash fibrosis.
With that Jerry will now provide an update on our global commercial PBC business and Nash pre launch activities Jerry.
Thanks, Mark and good morning, everyone.
2019 was a solid year in terms of our PBC commercial performance.
Full year, we reported $249.6 million level caliber net sales globally, which was 40% growth over the prior year as we continued to help bring to benefit civil caliber to more PBC patients and prescribers in the U.S. and abroad.
Quarter, four we reported $70.3 million and worldwide Calvin net sales our highest quarterly sales to date.
In the U.S., we achieved net sales of $53.5 million in the fourth quarter, reflecting consistent end market demand growth is more community based G.I. specialists gained experience with Ocala.
Now turning to the international region, we achieved ex U.S. O'callaghan net sales of $16.8 million in the fourth quarter. This growth reflected the continued strong performance in our key international markets. This was a trend we saw throughout the entire year at this point our caliber net sales outside the U.S. make up about one quarter of our global net.
Sales.
Of course in addition to PBC our teams continue to make progress on our preparations for the national launch.
Our investor event in December we spent a great deal of time talking about advanced fibrosis due to Nash as a disease.
Commercial focus and our launch plans for <unk>.
But I want to update you on how we're progressing on some of our critical initiatives.
We've identified our initial target population for launch has those patients with advanced fibrosis due to Nash without cirrhosis, who are under the care of a specialist.
This population represents a significant unmet need today and we estimated that it consists of approximately 500000 patients in the U.S. <unk>.
Again, we believe these patients already under the care Hepatologists and Gastroenterologists, which is where we'll focus our launch resources.
We continue to flex up our commercial organization in preparation for approval and for launch.
We've completed the hiring of our Salesforce leadership and to date, we've employed the majority of the expanded field based team were currently focused on disease education and customer profiling work and we're on track with the launch plans that we previously laid out.
The specialists will be targeting out launch often identify and manage Nash patients in practice noninvasively.
Based on our market research a strong majority of Hepatologists and Gastroenterologists surveyed said that they're able to reliably assess advanced fibrosis, using noninvasive measures without conducting a liver biopsy.
We're making good progress through our disease education efforts as we continue to encourage physicians to use the noninvasive tools that they have access to.
In addition to our work. It's also good to see momentum in the area from the efforts of other stakeholders.
One recent example of this momentum came earlier this quarter when labcorps one of the country's leading diagnostic companies.
Now this new CPT codes that triggered the automatic calculation of a patients did for score along with their normal liver panel and CBC.
We know that disease education is critical and a new category like Nash and we continue to focus our efforts in this area.
We've had productive face to face engagement with H.C. piece at recent Congresses and disease education meetings.
Our field medical team was expanded last year and has been in the field now for about six months focused solely on physician education, particularly with Gastroenterologist well I've been very interested in learning more about Nash.
When taking into account the educational efforts of our field force teams, we estimate that we've already reached over 8500 specialists.
During these interactions or education focuses on the increase risk, where cirrhosis that advanced fibrosis patients face and the importance of appropriately identifying and managing these patients.
We've been in discussions with payers as we prepare for the launch in Nash and as you would expect as we move closer to launch our interactions with payers are increasing.
Our market access team is making strong progress and speaking with keep pairs across the country and I can say that the level of engagement on both sides as high our goal remains to bring to payers on the journey and to ensure that they have a clear understanding of our target patient population of advanced fibrosis, do Dinesh and that there are no surprises.
This work will continue to be a key focus in the months ahead, and we remain very encouraged.
As a reminder, our launch in Nash will be a specialty launch where in the first six to nine months, we expect to see the typical building of reimbursement access on the commercial side. This is that expected to be followed by the government reimbursement, which usually takes a bit longer.
So across the board I'm very pleased with our efforts are in depth knowledge. In this market tells us that upon launch associate should be well positioned to be the first and only treatment of choice for patients with advanced fibrosis due to Nash.
Preparation is on track to execute the first launch in this category and provide access to associate for patients suffering from advanced fibrosis due to Nash.
And now I'll turn the call over to our Chief Financial Officer, Sandeep could pod yet for financial update.
Sandeep.
Thank you Jerry and good morning, everyone. Please refer to our press release issued earlier. This morning for both summary of our financial results for the quarter and full year ended December 31st 2019.
Our business performed very well in 2019, we closed the year with Calvin net sales at the high end of our guidance range.
We invested incremental resources in our national launch preparation activities, while also strengthening the balance sheet with 470 million in gross proceeds from a successful financing.
In the fourth quarter, we recognize 71.5 million in total revenue our highest quarter to date.
Up from 53.3 million in the fourth quarter of 2018.
For the full year 2019, total revenues were 252 million, representing a growth of 40% over the prior year.
Fourth quarter or Calvin net sales comprised the U.S. net sales of 53.5 million and ex us net sales of 16.8 million.
This represents a growth of approximately 30% at 43% respectively versus the prior year quarter.
We continue to see solid Ocala of a prescription demand growth, which we expect to continue as we move into 2020.
Total gross to net for the fourth quarter were towards the lower end of our previously communicated 10% to 15% range.
Our GAAP operating expenses for the fourth quarter 460.8 million in our non-GAAP adjusted operating expenses were 145.4 million.
For the full year 2019, GAAP operating expenses were 564.4 million and our non-GAAP adjusted operating expenses for 499.4 million.
As a reminder, our non-GAAP adjusted operating expenses exclude stock based compensation depreciation and non cash operating lease costs.
Our cost sales for the quarter were 2.5 million compared to 1 million in the prior year quarter.
Our selling general administrative expenses for the fourth quarter were 93.7 million. This was an increase of 22.7 million over the prior year quarter and it was driven primarily by increases related to our national launch preparation activity.
During Q4, we completed the majority of the buildup to support the Nash launch with the exception to the field force, which we anticipate to complete in Q1.
Our research and development expenses for the fourth quarter for 64.6 million and generally in line with prior year quarter.
As of December 30, Onest 2019, we had cash cash equivalents restricted cash and investable debt securities available for sale.
Our next probably 657.4 million.
Turning to our financial guidance for the year.
As you saw in our earnings release. This morning, as a result of uncertainties relating to our launch associate and liver fibrosis students.
And their potential impact on our 2020 financial performance, we're not providing 2020 net sales guidance.
We expect caliber sale in the first half of 2020 continued to perform well and reflect solid demand growth and continued year over year growth.
In terms of seasonality I'd like to remind you we do expect softness in the first quarter 2020 in the U.S. as insurance plans reset.
We are again expecting the typical increase in Q1 gross to net.
Which is impacted by many factors responsibility for the coverage gap.
With respect to expenses, we expect non-GAAP adjusted operating expenses for 2023 in the range or 560 million to 600 million.
Affecting our investments to support the launch of associate for liver fibrosis due to Nash, our commercial efforts in PBC and continuation of our clinical development and pipeline program and other operating expenses.
This expense guidance assumes both the approval on or about the PDUFA action date of June 26 2020.
In summary, we ended 2019, well positioned to achieve our strategic objectives in 2020.
We plan to capitalize on the momentum in our PBC business that we saw in 2019.
Focusing on regulatory and commercial milestones to bring the first fruit therapy to patients with liver fibrosis due to Nash.
Finally, as a reminder, non-GAAP adjusted operating expense is a non-GAAP financial measure under SEC regulation.
Please refer to our press release issued earlier this morning for full explanations and reconciliations of this measure.
I'd like to not turn call over to the operator operator.
Ladies and gentlemen, if you have a question or comment at this time. Please press the star than the one key on your touched on telephone. If your question. It's been answered you wish to move yourself from the Q. Please press the pound.
We also asked would you please limit yourself to one question to allow the questions of uterus.
Our first question comes from bright Abrams with RBC capital markets.
Hey, guys. Thanks, very much for taking my question and congratulations on all the progress.
It sounds like you are having increasing interactions with commercial payers was wondering if you could maybe drill down on that a little bit more I'm curious to learn.
Your latest use and how you expect reimbursement to be structured any feedback you're receiving on overall access any pressure points on on pricing.
The potential to priced differently in different indications.
And the perceptions right now amongst payers for non invasive diagnostics. Thanks.
Thanks, Brian last year to take that.
Thanks for the question Brian.
Obviously as I said in the <unk> prepared remarks, and as you can imagine though were heavily focused on our interactions with the payers as we progress.
Closer to launch the conversations with the payers get Baltimore frequent and frankly, we're speaking to a broad group about influencers across the payers in different departments. When you think about the construction of the national payers I think.
Our progress continues to go into right direction I think the discussion continues to be focused on a population of advanced patients that payers acknowledged.
Are the ones, who have the highest likelihood to progressed to cirrhosis, which is the largest concerned that the payers have because that's where both the complications and the cost. So we've seen a good progressive discussion I think there was a better understanding on the payers as Weve continued with these discussions on the target patient population.
And then depending on the payer you're at different points in the discussion as we now progress there are some elements.
Including a final label, which are of course part of the Finalization of that discussion and ultimately a price point, we would anticipate a you know, we'll we'll be able to progress on these discussions and come back at the time of approval with that I'll list price on Nash that we would it be prepared.
And look forward to communicating to you along with our overall approach in how we would expect to move forward with with the rest of our portfolio in PBC.
Thanks Jerry.
Our next question comes from lease you young with Cantor Fitzgerald.
Hey, guys. Thanks for taking my question Congrats on all the progress maybe one and a half.
What are the biggest questions that you anticipate heading into this panel and how are you prepared to answer them and can you just thought maybe how they compare and contrast, you know kind of.
The expectations for this panel on what you versus what you happened at the PBC Pell, obviously, two different diseases, but same drought. Thanks.
Yes, thanks, so we've yet to.
So again as I mentioned my prepared remarks.
We're well underway in terms of preparation for for this panel.
I think the difference from PVC and we do have that experience as I mentioned with a very successful outcome.
You know is at least twofold, one is first drug in a new category.
Second on Nash is obviously, a much bigger disease.
And the patients frankly that we're targeting with advanced fibrosis.
Our generally sick or right they have.
Typically multiple co morbidities so.
You know, we're preparing I would say more broadly.
We we are actively consulting with experts across a range of disciplines.
Matching the typical the typical Nash.
Patient.
I think you know look no surprises here, what do you typically puts to handle like this.
Spans the range of what is required to inform an assessment of benefit risk of any drop.
And Thats what were preparing for.
Great. Thanks.
Thanks.
Our next question comes from Michael Yee with Jefferies.
Hey, guys. Thanks for the question in some of your comments again going back to all your discussion with parents just wanted to think about or ask you about.
How those conversations have been around biopsy again, and you are conviction level that.
Our overall, you're not hearing any concerns about that and how to think about how we would learn about these requirements to launch gets underway from payers and then a follow up to that schedule.
I know that you're in discussions for two different pricing strategies.
Yes, you said you would communicate that lunch Nash is approved is Johnny Nevsky eight.
That needs to be two different NDC towns is not a payer thing some payers would have to get from any secrets, maybe just explain how that works just a little bit show Ob Gyn.
Yes, thank you very much.
Thanks, Mike I'll hand that to Jerry yes, thanks for the questions, Mike I guess I'll take the second one.
First so our goal has been to maintain optionality on our price points for potentially two different brands out there if we're able to be.
Successful, we have filed a separate end da which would.
If approved give us a separate brand a separate label a separate NDC code, which then we would have the option of how to price vis-a-vis.
The PBC brand. So there would be a regulatory a factor of that decision and then ultimately pricing decision based on our discussion with payers and what we felt was that the best up pricing strategy to take at the time and as we said, we'll we'll come back with that in the future. Once we get a label on Nash and give you an.
Indicator to our thinking on on PBC.
The discussion with the payers, a biopsy and how best to identify the advanced fibrosis patient that we're going to be targeting has been as you could imagine and I think as you.
Probably a are aware has been a part of the discussion with payers.
Again, it's been a good collaboration one of our objectives have has been to engage proactively with the payer. So that we're not surprising them that we're talking openly and of course different payers look at this situation.
Based on on their experience their experience in other liver conditions, where.
We did see.
Non invasive become adopted both from the physician and from the payer side. So while it's still too early to be completely definitive. We do remain confident we are having that discussion openly I think payers are aware of all of the emerging data and practice around noninvasive is along with.
The concerns in the limitations of a biopsy and suffice to say we remain encouraged but we also remain focused on the work that we're doing in the field with payers and with physicians to move the field along.
Got it thank you.
Our next question comes from.
Your next question comes from Salveen Richter with Goldman Sachs.
For taking the question. This is Ross on for Salveen with the launch coming up in the second half here, how should we be thinking about any inventory impact during the initial launch period and then secondly, if we think about the phase two reversal read out what confidence do you have you guys will people to read this trial out successfully given the.
The current failures, we've seen across the landscape by competitors.
Thanks, So I'll take the second question and then hand over to Sundeep for the inventory question. So on reverse.
Very proud of the fact that we essentially doubled the size. The study last year and then roll that same period of time, we've been guiding to.
With over 900.
Patients, who compensated cirrhosis enrolled in the study.
And as I mentioned my prepared remarks that.
Lets readout by the end of next year.
You know we've talked to this before we obviously have the robust anti fibrotic benefit that we've demonstrated in patients who are not yet cirrhotic, but with bridging fibrosis. The three.
Patients these patients with compensated cirrhosis or or frankly, not that much more advanced the early cirrhotics we insured.
That they don't have any development of soft Joel very seasoned therefore don't yet have clinically significant portal hypertension.
And we actually.
Believed that the results that we reported out last year.
And the competitor failings actually give us more confidence.
The ability of associate demonstrate fibrosis reversal.
In this patient with early compensated cirrhosis.
So we're very much looking forward to to reading out.
Towards the end of next year.
And then.
Yes.
The inventory Ross I assume you meant.
The selling.
Inventory are you thinking like how much product, but would have on hand or was the question more specific I assume it's how do you recognize to sell it.
Yes, exactly you guys going to be have any any inventory stocking impact yeah look I mean, we're planning.
This is specialty launch that will be through specialty pharmacies, yes. So there will obviously be some but but it's generally much less than than typically a broader launch. So yeah. I think it will obviously depend on the timing of the launch but there'll be some inventory that will be sold into initially, but I would say that's probably.
Rather minimal given the fact that it's there were a specialty pharmacy and we're not doing large wholesalers.
Got it thanks guys.
Our next question comes from Yasmeen, Rahimi with Roth Capital partners.
Hi team on congrats on the continued progress two questions for you my apologies for asking too.
The first why you.
How do you plan on addressing potential request by pairing the decline OTI responders versus non responders in the real world for reimbursement and then the second one it's very simplistic what do we think how what might end up on the label ITC versus protocol I'm, a little bit color on them.
Research that you guys have died in terms of how comfortable docs are with these two definitions and thank you for taking my question.
Sure, Yes, though in the first question too much Gary inject the second one in chart.
Yeah and.
Thanks for thanks for the question you asked me the payer discussions around monitoring for patients data that are on therapy. I mean, I think there is a and understanding.
That.
The physicians, who are seeing these patients will be doing typical.
Liver monitoring on an ongoing basis that tends to be the first thing that they're looking at in terms of the.
Follow up that tends to happen as these patients are seen a couple of times a year on average so looking at a combination of.
Of the normal lab values along with.
Noninvasive tends to be the way that these patients are managed in the real world and that's been part of the discussion with a with payers to date.
Yes, and then you guys as you know and stay tuned for more we presented some exciting noninvasive testing data correlating.
All the commonly used in itcs with.
The fibrosis.
Prudent.
We saw and most recently in December.
And with the 30 days with patients who on average has been on study on treatment for another year.
Beyond the 18 month readout was even further improvements in fiber scan.
Based elastography, suggesting.
Ongoing improvement in fibrosis, so in the real World. That's that's what we expect people to use monitor for response.
With respect to your first question on on labeling.
This study the primary efficacy analysis was.
In the ITC population. It was most rigorous gold standard approach and as much as we've got a categorical binary endpoints, so any patient who dropped out or otherwise didn't get a repeat.
IOP see who is considered a non responder so for sure.
We expect RTT data to to end up in the clinical studies section.
And would of course hope given the totality of data supporting primary efficacy analysis that we see more.
And just that.
In support of the efficacy profile of the drop.
Thank you.
Our next question comes from route to borrow column.
Hey, guys. This is to Sean offshore Ritu Baral, just two questions from our end versus just in terms of the non invasive and discussing acquisitions. How comfortable are they would not only diagnosis for then you're going to fall off with just fearing based scores versus meeting some sort of imaging modalities, such as like a fiber scanner and MRT to really track response.
And then secondly, I know Jared said the Nash launch was about 8500 specialists does that include the 5000 already here that PBC launch and if so.
Any further color just on timeline for how long you think it will check to expand that reach to 15000. Thank you.
Okay.
Yes, so maybe I'll take the second question first so.
So just a little a little lot context. A reminder, so we're currently calling on for PBC roughly 5000 target.
Hepatologists and Gastroenterologists, our target group for launch in Nashville be about 15000. This goes to the broad group of of Gastroenterologist community base that we've outlined where the 500000 target patients that we've.
Designated as the launch target reside the 8500 number that I issued earlier.
Was roughly the number of physicians that weve reached through all of our education in person education to date that number as you can imagine increases every week as we go to more so we would anticipate will continue to increase that we.
We are in the process of Ah of deploying the additional field forces that we talked about and so the ultimate target group that we will be hitting by the time, we launch will be that 15000, so think about it progressively we're educating a in a variety of different means and the 8500 is the the point in time.
Hi, Matt.
That weve reached to date.
Yes and ended the second question I guess the other question your base about certain based.
Yes. So there is look theres. Some variability you have a and I think this has been part of our strategy, we're not picking a winner.
In terms of the noninvasive is but we want physicians to.
And we're trying to help physicians use the tools that they have access to and they do feel comfortable with the tools they have access to.
There are some who are using primarily the CRM based somewhere or using multiple tools, including a fiber scan fiber shore elfas in the process of Oh being introduced.
So there is some variability and again I think the good message for US is that physicians are comfortable with what they have access to they're continuing to learn more NB educated and the availability of these tools continues to grow. So that's the momentum that we see in the market that we think we're going to be able to.
Yes.
And the only the only thing I'd add to that as Jerry mentioned in his prepared remarks.
You know that there's a lot of innovation happening and for example, labcorps adopting CPT codes with automatic generation of fit for as many fishnet was available liver function tests and CVC.
Major step forward. There are also doing a relate to fiber sure for any patients specs and based on the primary screening of potentially having.
Advanced fibrosis so.
We're also making great strides.
With big lap companies.
And are confident that with time there'll be more and more adoption.
Of these entities.
Great. Thank you.
Our next question comes from Steve So you'd hope of Raymond James.
Hi, Thank you disrespect to with respect to the India for Nash. So many clarify if you included any data from any of your clinical studies, including even reverse related to titration of O'shea from 10 to 25 or from 25 to 10 Megs I'm just curious if any titration data could possibly find its way into a label.
Thank you.
I mean, the short answer is no I mean reverse obviously remains blinded and ongoing.
We do have titration arm, there 10 to 20, but as you know and regenerate we didnt we had to 10 to 25, we file based on the on the efficacy demonstrated the 25.
And.
Yeah, ultimately it will be at Ftn discretion on reviewing the totality of data whether whether they believe theres a role for the for the 10, but we don't have in on the Nash side, we don't have Tetration data.
Tune tenant white box.
Great. Thank you.
Our next question comes from Evan pickup with CBS.
Hi, everyone. Thanks for taking the questions is shrinking another farm on for Spain.
A question given that yesterday, a competitor reported positive data on Russia, and Nash resolution what are your updated thoughts on the competitive landscape.
And how do you view this data center in context.
Well see a recorded Nash thanks.
Yes, as you mentioned, you're talking about the MGM announcement yesterday in their ongoing phase two study.
It's in the context of the broader competitive landscape is the first glimmer of hope.
In a long time over the last time was a year ago with with our positive readout.
You know we weren't surprised the result, I've often said that.
This compound yeah, Jeff 19 compound is squarely in the FX our pathway.
It's the only target that is currently validated we validated that and.
The bar remains very very hard to show.
The all important fibrosis benefit.
The results just as the quick comment on the results again, not surprising very much in line with with our Flint phase two results I think gives them forward.
In phase three and whether you are much larger unlikely longer term.
Study.
And.
We'll see its of course of daily injectable. So I think that the the path forward will be a little bit more challenging there as compared to a daily oral therapy lycos yet.
I think that.
At this point.
I can't comment on how Yesterdays news.
In past the rest of the field I think over the course of this year, we expected number more.
Redoubts.
A couple of phase three and mostly phase two.
But you know as I can say the only thing I can say is that the field is littered with.
Unfortunately failures in the borrowings.
Very high to succeed.
Great. Thanks very much.
Our next question comes from Brian Skorney with Baird.
Hey, good morning, guys. Thanks for taking my question I'm, just wondering if you could talk high level about the restructuring at the off new drugs that FDIC and how we should think about the impact.
This has on the renewable energy I think the review divisions getting within the next month.
So do we know will o'shea be evaluated officially under the new division of Gastroenterology or the division of Hepatology nutrition and and are there division directors named for either of those divisions, yet for for the office of the knowledge and information, which oversees those divisions.
Yes. Thanks.
Obviously, something we're following pretty closely.
No there's been a broader reward, though andy over the last year and the sort of the creation of new Standalone liver Hepatology Division.
As part of that plan I'm, not aware that thats been yet formally announced but but I know that it's underway.
As you mentioned.
What we've heard is what you've heard on like I think there was a recent call with the rents and you all were mentioned that.
Division director for the Hepatology Division has been.
Names.
But we haven't heard confirmation of that.
We do expect at the ongoing review of our end da and all Nash.
Hi, Andy is going to go to the technology.
Division and we don't expect any.
Disruption.
As we as that goes forward.
Great and then.
Second one in there in terms of the final analysis regenerate I know you guys have not wanting to guide for for readout for.
On timing of the readout will there be a point when you guys are willing to provide guidance should we think about this was maybe a time handler, we would get like a years notice I had a readout.
Well you know it at the present moment as you just mentioned, we're not in a position to provide such guidance.
That is we see on a blended basis events, comprising the composition outcomes endpoint accumulate and they are tracking well against our original assumptions.
That we might be in better position on a post marketing basis to provide.
Some clarity there.
Needless to say.
I just answered a question about competitive landscape. So it was important to remember because people tend to look.
Back.
At at those falling, but we continue to move forward and if it's on any any competing compound as chances.
Getting or in terms of the confidence in earlier phase development of a chance of getting to market will read out.
Verse study in compensated cirrhosis critically important segment of the population and of course, the regenerate outcomes to support full approval. So.
We will continue we look forward to continuing to improve our competitive positioning in this market overtime.
Thanks Mark.
Our next question comes from Jay Olson with Oppenheimer.
Oh, Hi, maybe just to follow on on that last comment.
I have a longer term strategic question since intercept is uniquely positioned to create and define the market for Nash therapeutics and your lead time over competitors seem to continue to grow.
But as you look out three or more years, how do you plan to maintain your leadership position Nash and defend against leader insurance, you may try to redefine efficacy or other attributes of Nash therapeutics in a way there that could threaten no CA. Thank you for taking the question.
Sure well I'll just reprise.
The comment I, just made which is you know we will continue just with our lead compound O'shea, which we expect to.
Approval to position as is the foundational therapy since this disease.
We'll continue to generate data reading out.
And further confirming.
We see his ability to ultimately to improve outcomes in these patients and with that that will be a game changer.
Very very difficult for follow on compounds to to do the same.
Well, it's fairly soon we'll take a very long.
Period of time.
I think we've also talked.
Over the years about.
Ultimately combination regimens that might emerge for the treatment of of this of patients with his disease.
And as everyone is seen as follow the field.
You know, it's easy to talk about combo much much more challenging to realize we've always said that the bar is exceeding the high just to get single assets.
Forward moving forward, let alone combos and most recent combo readout was frankly.
I think most people would agree with.
Yes.
Hard hard to interpret let's just say and hard to see how.
How how to go ahead there.
But certainly we're constantly on the lookout for other ammo ways that we can combine with us and we've talked.
In the past about potential to the extent that people are.
Looks looks promising that.
Combining and people are like Pesified, which of course, we have the rights to in the us with associated could could be promising.
And we'll continue to monitor for other opportunities.
Okay.
Great. Thank you.
Our next question comes from recent Breakover JMP Securities.
Hi, Good morning. This is John for Lisa Hi, just a question regarding regenerate.
Can you comment on any potential unblinding.
In the U.S increase their savings protocol to try to maintain the blind and then what are your current thoughts on.
Beneficial effects for stands in Nash patients.
Yes, so we have actually presented data.
And since nor our primary managed gifted the lancet which came out.
Before the end of last year, we actually show a breakdown.
That month, 18 patients who remains that naive patients who had a stat and added.
Patients, who are honest hadn't at baseline and how.
How they looked from from a little bit management standpoint. We've also said that stat news was not shown to make a difference in terms of.
Patients realizing the primary endpoint.
In the study.
That said you know.
As we've been saying since the days that Flint trial read out.
Status our recommended the current is still the unusual guidelines for the management of Dyslipidemia and this patient population, obviously an important.
And cost effective means of managing LDL.
In this patient so it's something that we certainly.
Advocate and we've shown that the comp combination of the two can be used safely and effectively in these patients.
Thank you. Your next question comes from Liana Moussatos with Wedbush Securities.
Thank you for taking my question, how much of that Opex guidance. This year is for the Nash launch.
Affects the question on the Opex guidance I mean, we havent really provided a breakdown in terms of national as to what I can tell you. It's a good good portion of certainly the agenda is allocated to support the national launch we would not last year, we incrementally increased about $100 million to support the launch efforts last year and this.
Sure we continued to obviously.
Leverage our PBT infrastructure that we had and reallocate resources from PBC to support the Nash launch, but suffice to say I mean, the guidance overall anticipates an approval.
On or about our PDUFA action date.
Thanks.
Our next question comes from Alan Carr with Needham and company.
Hi, Thanks for taking my questions, Jason can you comment more on.
PB sales in PBC sales in Europe.
Some of the details around the how its going and whether or not it's meeting your expectations.
Then mark.
Give us your thoughts on we think Nash trial endpoints might be headed.
[noise] noninvasive versus biopsy. Thanks.
Thanks, Alan This is Jerry Yeah, I would say a consistent with the overall picture were very positive on.
The progress we've made a across our international markets I think one of the really good elements that you've seen as things have progressed as the consistent performance across the main countries. It's not as if we see one or two markets that are doing.
Substantially better than the others I think it's been really good performance I think though the markets have.
Learned from the earlier market, so you've seen a sharing of the learnings in terms of the importance of the educational efforts in PBC I think the increase in the overall level of second line therapy prescribing has been one of the.
The consistent themes and so overall I think we feel good about the progress in Europe the trends throughout 2019, and the momentum that we have coming into into 2020 and of course that remains an important part of our of our PBC business as we look forward.
No one with respect to your question on endpoints. It's it's obviously the burning question.
In in the field.
Were you know on one hand, all stakeholders, including other regulatory authorities recognize the real limitations of of biopsy.
And these complex histologic.
Points.
And on the other hand at least from a regulatory standpoint.
It's unlikely that we're going to see.
Categorical shift away from the requirement to demonstrate histologic improvement.
In the near future.
There is an upcoming I'm sure you're where is all the easel close sponsoring.
Yes, Nash and points meeting.
Next month.
So so theres ongoing interest in the field deliver form is active in looking at this so I do think it's probably just a matter of time.
But right now we've got draft guidance is out there focusing on on histology.
In this population so.
We along with everyone else, we're going to continue driving.
Validating evidence for non invasive.
As endpoints.
But it's going to take time.
Great. Thanks for taking my questions.
Our next question comes from Joel Beatty with Citi.
Hi, Thanks for taking the question for the upcoming at a time do you anticipate that they'll be considering the role of noninvasive diagnostics and prescription and use of well see a or would that be a topic outside of the outcome.
Hi.
Could be something that.
Yes, the panel to consider.
And certainly we presented a lot of data.
In support of NFC based identification of patients.
But others, but its pure speculation on my part.
Got it thanks.
Thanks.
Our next question comes from Thomas Smith with SPP Leerink.
Hi, guys. Thanks for taking the questions.
Can you just talked about the rationale for the choice of doses in the reverse study and Cirrhotics, where you mentioned mark.
The 10 milligram dose along with the titration arm looking at the 10 to 25 Megs and then if this trial were to be positive do you imagine filing a separate on da for this indication or would this be a potential supplement to the Nash fibrosis idea.
Yes, so with respect to dose selection remember we designed the study prior to the read out of regenerate, where we test into 10 and 25.
And hence the selection of the same.
Doses on for reverse.
And and remember again, you know in particularly in cirrhotic patients.
Expect dose for those to see increased exposure.
Some of our drug in the liver right. So more critical it more more bang for your Buck.
At a given dose so we're happy with.
With the testing of of those two doses, we'll see what the result is on towards the end of next year.
The second part of your question.
Remind me was.
Sure just totally separate NDS thats right, yes. So.
As you know also in Nash and this is true for.
All the players in the field.
FDA has sort of considers the Nash population with cirrhosis a separate population.
Reverses.
Ongoing under a separate indeed.
And what will have to see what based on successful readout. What the path forward then is to get the expanded indication.
Okay, great. Thanks.
And I'm not showing any further questions at this time I turn the call back over to our hosts.
Yes, thanks, everyone for joining our yearend call. This morning.
Just to close.
Following anticipated FDA approval.
We believe oceana is very well positioned to become the foundational therapy in patients with advanced fibrosis due to Nash, which we consider to be truly a blockbuster opportunity for us.
The next few weeks and months, we'll continue to be solely about execution for everyone here at the company.
And with each day that passes were moving swiftly toward achieving our critically important objective.
Which is the ability to offer the first approved drug to patients suffering from advanced liver fibrosis due to Nash thanks very much.
Ladies and gentlemen. This concludes todays presentation you may now disconnect have a wonderful there.