Q4 2019 Earnings Call
Reading.
Welcome to Abu <unk> pharma fourth quarter full year 2019 earnings call.
Yes, I'm all participants are in listen only mode. A question and answer session will follow the formal presentation.
Then in what's your operators. This is starting with <unk>. Please press star zero on your telephone keypad. Please note. This conference is being recorded I will now turn the conference over to your host well Arts managing director of Lifesize advisors. They can you may begin.
Thank you operator, and good morning, everyone. Thank you for joining today's call.
This morning, I'll be <unk> issued a press release, highlighting its recent business accomplishments and reporting its financial results for the fourth quarter and year ended December 31 2019.
This press release is accessible via the company's website at www dot out real pharma Dot com.
Before proceeding we would like to note that management's comments today may include forward looking statements regarding the company's plans and expectations. These statements are being made under the private Securities Litigation Reform Act of 1995.
Subject to various risks and uncertainties.
Actual results may differ materially due to various important factors, including those described in the risk factor section of our most recent form 10-K under subsequent FCC filings.
These filings can be accessed from the media and Investor section of our website at Www Dot ALD rail <unk> dot com or on the Fccs website <unk>.
Any forward looking statements represent our views as of today March 2nd 2020, and should not be relied upon as representing our views as of any subsequent dates.
We undertake no obligation to publicly update these statements.
Now I'll turn the call over to run Cooper, <unk>, President and Chief Executive Officer Ron.
Great. Thank you Paul and thanks to all of you who joined US. This morning with me today is up rails, Chief Medical Officer, Dr. Patrick Horn.
Our chief commercial officer.
Pamela Stevenson and our Chief financial Officer assignment Harper.
We expect that Twentytwenty, what are your momentous year for Albert rail and the communities that were striving to help.
We have major milestones on the horizon and the team is ramping up activity to capitalize fully on the opportunities ahead.
In today's call I'd like to share our plans for the year and one update you on the advances in our development plans for all the VIX about.
To outline our go to market strategy and our road map for success.
And three provide an update on our emerging pipeline.
Now we have a number of exciting developments with the older affects about clinical development program, but let's first look at auto VIX about <unk>.
We enrolled more than 100 patients in the Patrick one pivotal trial and are very pleased to randomize 62 patients versus a plan 60 patients.
We expect topline data mid this year.
We are immensely grateful to the patients families and physicians, who have participated in the pet Big program and I've worked with us toward bringing what could be the first pharmacological treatment option to the piece that community.
Meanwhile, patients have been rolling over from Patrick one.
Two cohort one of the Patrick to long term open label extension study and we now have patients who've been on older VIX about therapy for well over a year.
At the same time continue to enroll the expanded second cohort of that Patrick to which includes people patients who did not meet the eligibility criteria for Patrick one.
With Patrick one fully randomize will capitalize on this opportunity to generate longer term data and brought into body of Olympics about evidenced in different patient types.
We are preparing for regulatory submissions following the topline announcement and expect order books about approval at walk for people in the second half up 2021.
Turning to our next indication Galleria Teresa, we're very pleased to recede investigational new drug I Indy clearance from the FDA following productive discussions on trial design.
The whole trial the older Patricia and the use of voted VIX about in treating liver disease is a precedent setting first ever pivotal trial conducted in Galleria trio.
We're extremely excited to initiate this program as are many members of the physician community who treat this devastating condition.
We believe the ability to treat the is the most common rare pediatric liver disease with roughly 15 to 20000 patients in the U.S. and you combine and for which there is no approved pharmacological therapy virtually all of these children are easily diagnosed in the first month of life, that's their stools earthquake color or not covered at all.
After a biochemical work up ultra sound and confirmation of door entry into the child Mili undergoes a five procedure.
A surgery in which a child damage bile ducts are removed and the intended attached to the lever to restore entrant paddick circulation.
This is a very serious disease as despite undergoing a surgery about 50% of the pieces required liver transplant within two years and about 80% of the piece is required transplant by age 20.
There are accumulating natural history data that indicate that those children, well lower serum bile acid have an improvement in key liver biomarkers and need of liver survival.
Oh, the VIX about lower CERN bile acid levels and potentially could be the first pharmacological treatment that slows ongoing damage to the lever.
Both will be a double blind randomized placebo controlled study to evaluate the efficacy and safety about a VIX about compared with placebo in children with builder, Trisha, who undergone a guess I procedure.
Children in the treatment arm will receive Olympics about 120 micrograms per kilogram per day orally administered for 24 months.
The primary efficacy endpoint is a proportion of patients who are alive and who have not undergone liver transplant. After two years of treatment.
The enrollment target is larger than the pet pick one trial with approximately 200 infants given that bill or atresia is a more common disease.
We initially proposed study designs using biomarkers and a shorter duration, but regulator desired clinical outcome, given the complexity and severity of the disease.
This study will provide valuable data on disease modification that will be helpful to payers and other stakeholders, both the FDA and the I may have granted OTA VIX about orphan designation in bill or intra Asia.
We're confident in our ability to initiate and conduct this trial and book and plans to begin the study in the first half of Twentytwenty, we will provide more detail on the trial at the program progresses.
[noise] not guided by recent regulatory actions and the duration of the Billerica treatment trial, we've decided to accelerate development in Alphaville syndrome and plan to initiate a third OTA VIX about pivotal program by the end of the year, we have a schedule interaction with the FDA this quarter to gain feedback on our pivotal program.
Both the F. D N. The may have granted OTA VIX about orphan designation in eligible syndrome.
We would expect the allergy all top line data to be available between the announcement of the P. FICC and Billerica trio topline results.
[noise] with phase three data in P., FICC and pivotal trials underway in belier trees, and knowledgeable syndrome, we will be generating significant data that ultimately could or support OTA VIX about to use in a larger patient population estimated to be roughly 30000 in the U.S.
And you combine.
Manny pediatric Hepatologist, we participating in all three pivotal programs and these physicians will have considerable experience with OTA VIX about and Albert rail.
We're planning for success at El Dorado, and we made excellent progress in our go to market work in 2019 and are accelerating our readiness activities in twentytwenty.
So our key areas of focus our manufacturing.
Key opinion leader engagement pricing and reimbursement and patient advocacy, while we build DAP and expertise in the organization to execute on our plants. Let me go for these a little bit more deeply.
So first of all we made excellent progress in manufacturing and supply chain planning, we're using the planned commercial formulation in our phase three studies and registration batches are on stability.
We're executing on to plan agreed with the FDA in the fall of 2018, and we do not expect manufacturing to be a gating factor for regulatory filings.
There are approximately 100 key pediatric hepatologist in the U.S. and the same in Europe.
We're planning activities to continue our strong and positive interactions with this give me the much of which is participating in our three important programs.
We've begun to treatment site mapping exercise to help us getting better insights on patient management and flow.
Of course pricing and reimbursement continues to be an emerging challenge for the industry broadly, but we plan to be ready with strong arguments and data.
The drive value stories ready at work his comments on the cost effectiveness model budget impact model burden as these study and other key elements of our access evidence.
We're very grateful for the patient advocacy associations further insight and absolutely wonderful collaboration.
We're continuing to build in our relationships and have created keep it voices dot com, which is an opportunity for p. families to tell their story the patient advocacy community continues to provide feedback, which we used to develop study designs recruitment inpatient services initiatives.
Let me close todays call by providing an update on our rapidly emerging pipeline.
In Q2 last year, we initiated our first phase two trial with Elagolix about monopoly and Nash. We're pleased with enrollment we continue to expect topline data by middle of this year.
As a reminder, this is a phase two proof of concept study investigating the effective elagolix about five milligram and NAFLD and Nash patients over 16 weeks.
I would like to set appropriate expectations for this study.
As this the first study ever conducted in both NAFLD and Nash patients with an eye bad inhibitor. We're looking for the combination of positive trends in liver markers CV risk factors and favorable Gi tolerability.
Now in addition to this study our Japanese partner EA farmers sponsoring an investigator sponsored study in Japan with 100 patients randomized to a higher dose of Elagolix about 10 milligrams bile acid sequestrant coal starving the combination of cost RMB and Alabama about 10 milligrams.
Or placebo.
Patients will be study for 16 weeks and we expect data by the end of this year or early next year.
These two studies will provide valuable information to inform the next stage of development.
Alber rail is funded the U.S. study and EA pharma has funded the larger Japanese study.
Given the unmet medical need in Nash, we believe our approach could provide the potential for the optimal balance of liver and cardiovascular risk efficacy with excellent convenience and tolerability, we have promising clinical and preclinical data showing that I bet inhibitors have a pause.
Effect I lived bids glucose liver inflammation liver fibrosis and elevated bile acids, all the hallmarks of Nash.
I look back so that is also oral once daily compound that a minimal systemic absorption. So we think it can be combined easily with other cardiovascular risk and Nash products.
With the data from these two NAFLD Nash studies, we believe our Nash program could create strong value for potential partnering opportunities.
[noise]. We also continue to drive innovation and are excited to be making progress with our pre clinical compounds.
Our focus has been to build on our expertise in bile acid modulation and develop new compounds that can offer a wider therapeutic window than I bet inhibitors.
We're pleased to select a lead candidate for our very productive compound library file for new patents and plant to complete hi, Andy enabling studies this year.
We're looking forward to bringing up the curve on our new approach and expect to speak more about this as the year progresses.
The last four Athree threes for which we are developing for bile acid malabsorption. We're pleased to have new patents issued but with competing priorities were evaluating next steps for this program.
I began to these call by referring to 2020 as a momentous year for Alberico pharma.
After reviewing in greater detail, the OTA VIX about and pipeline progress I hope that you can see why we're so excited about the potential for a tremendous twentytwenty.
So with that so my question turn the call over to Simon for a financial update Simon please.
Thank you Rob let me quickly summarize our financial results for the full yet in Q4 2019.
Revenues were 9.6 million to the a ended December 31st 2019, compared to 12.7 million for the year ended December 31st 2018.
The 3.1 million decline was primarily due to a milestone payment received in 2018 from EA pharma due to approval by the Japanese NHL W analytics back to the treatment of chronic constipation.
Offset by royalty revenue from EA pharma.
Revenues for 6.4 million for the fourth quarter of 2019 compared to point Sixmillion in the same period last year. The increase of 5.8 million was primarily due to royalty revenue received from EA pharma.
Research and development expense was 45 point Sixmillion for 2019 up from 31.7 million at the same period the before.
R&D expense was 14.2 million for the quarter compared to 9.5 million for the fourth quarter of 2018.
The increases for both the full Leah and fourth quarter were primarily the result of program expenses OTA VIX about Ela Bics bat and preclinical work as well as personnel costs as we continue to increase our program activities and the head count.
General and administrative expense was 23 million for 2019 compared to 18.1 million for the previous yet for the fourth quarter 2019, DNA expense was 6.2 million compared to 5.8 million for the same quarter in 2018, so base.
The full yet in fourth quarter. The increases were primarily attributable to headcount as we put in place support for the transition from an R&D to commercial organization.
As well as some additional stock based compensation.
Net loss for the a ended December 31st 2019.
It was 62.7 million or a loss of $5 from four cents per share compared to 46.1 million or a loss of 3.9 $4 per share for the year ended December 31st 2018.
For the fourth quarter 2019, the net loss was seven and a half million dollars or a loss of 57 cents per share compared to a net loss of 15.8 million or a $1.34 a share in the fourth quarter of 2018.
As of December 31st 2019, we had a balance of cash of 131.8 million compared to 142.7 million on September 32019.
In early February 2020, we closed a common stock offering which provided an additional 43.2 million of cash to fund the acceleration of pivotal trials the OTA VIX that.
In Billerica, Tricia and the eligible syndrome that Ron talked about earlier on the coal.
We are providing guidance from total operating expenses for 2020 of approximately $100 million given our recent equity financing. We now anticipate that our cash balance will be sufficient to meet our operating needs into the second top of Twentytwenty Guatemala.
With that let me turn the call back over to run for closing remarks.
Good job Simon Thanks, very much.
In summary, when we come to the end of Twentytwenty, we should have read out the data for order VIX bat NP FICC.
Started two additional pivotal OTA VIX about programs read out the fees to Ela because of that NAFLD and Nash study and given more insight on our pipeline programs at the same time, we would have strengthen the capabilities of the organization and built depth and expertise for successful commercial execution.
[noise], reflecting on all of the last several years something else has evolved and our understanding of the rare disease communities that were striving to help.
We know these children and families more intimately now and it's impossible to think about a year ahead without thinking about what our success could mean to them.
We sometimes see today somewhere a mother or father is making a terrible choice to relieve a child suffering or save a child's life.
This year, we're coming up upon major milestones for the company, but we recognize the true magnitude of what's at stake and the truth scope of the opportunity goes far beyond Albert rail.
Thats firmly in focus for us as we start this exciting year.
With that we'll open up the call the questions operator.
Thank you.
So I will be conducting a question and answer session as he would like to ask a question. Please press star one and your telephone keypad.
Information gel indicate your line is in the question Q you May Prescott.
Nice to remove your question friendly Q.
For participants using sneaker equipment and may be necessary to pick up your handset before passing the sorry Keith.
Our first question is from Yasmeen Rahimi with Roth Capital Partners. Please proceed.
Hi team Congrats will not continued great data.
Question as we're headed into the Pete Keepsake one study.
The first question is just a reminder.
He powered to show stats sake and both doses.
Or air Werent in or into tally of both our versus individual one and then the second one is it will be lovely to get a reminder, online what's your expectation. It's in regards to the placebo response and the primary endpoint and maybe also what our trial elements there weren't included or incorporated in order to.
Minimize that placebo response at the primary endpoint and thank you very much for taking my questions.
Yes, I mean this is Pat thank you very much further question.
Yes, the study its power and the statistical analysis into higher our kickoff statistical analysis, where it's clear Tivo horses pooled OTA VIX to that and then it steps down to placebo versus low dose and placebo versus high dose and it is powered above 80% for either of the.
Okay.
That single comparisons placebo versus low dose have a placebo versus high dose.
And with the second response it is always hard to know about that placebo response, and we looked at as we've looked at all of that data you know they haven't really been a lot of study then pick but there have been other studies in the call aesthetic liver disease and children and a couple of things on that so we are.
Looking across the entire 24 week period.
And the placebo response as we've seen in Twoq diminished during the first art tend to diminish after the first several weeks so you'll get a placebo response, and then with continued therapy that placebo response becomes less and less.
Regardless when when we hit our power calculation for pride. It we assumed relatively modest response in the active arm and a large response in the placebo arm than we did our typified calculations based on that.
Yes, yes, I mean, it's Ron year, just add thank you for the for that pad, yes, just to summarize this study is power to well over 80% against the parade this endpoint.
Therefore, given less variability with the serum bile acid in play, which the primary endpoint in Europe that powering assumption is even greater and I think we feel pretty comfortable that we understand that theres more variability with pruritis, but we've tried to mitigate that as Pat said by taking.
In an aggressive placebo response by making this study probably larger than than we had originally planned and by going to 24 weeks. These things we believe should mitigate.
Thanks very much your question does mean appreciate it.
Thank you.
Our next question is Fabulous Yang with Jefferies. Please proceed.
Thank you. So my understanding is dead Sage is pretty steady PC Watson enrolling patients we believe our PBD surgery. So question is.
Once approved the glop, Christina patients, who have undergone PVD surgery or would you be eligible score.
Yes, Tim.
Hi, you and good morning, Thanks, very much for your question.
Like usual the challenge in this space the rare orphan diseases. Yes. There is no published data on on your question, Yes, no or are there any any registries, you're absolutely correct, though that we.
We anticipate it our in our phase three study, we had patients who were before PBD surgery, and others, who had had PBD surgery, but still fulfilled our entry criteria. So I think it would be can be difficult for us to actually estimate the number of patients.
It would be eligible but we have from our estimates we believe there is somewhere between eight to 10000 individuals in the U.S. in Europe, who who have p. effectively are in various stages of treatment so big opportunity.
Thank you thanks for your question.
Question for sign those are good.
Hello.
Did the DC.
She's seizure of up 100 million.
Does that include.
Ready near running similar.
Sure Bob in 2019.
So if the revenue really ultimately at the end of the day is is sort of somewhat irrelevant because it flows through to the fact that we monetize the royalty and thats.
Thats revenue from the royalty in terms of the actual overall expenses.
Obviously 100 million.
Is a pickup from what we saw in 2019, well we had roughly.
70 million of total expenses combined.
And that pickup is really being driven by obviously the additional been Maria Trisha.
And I'll, let you will sit and going to studies as well as the Nash study.
And commercial sort of expectations.
I think its.
Sad to say that with the common stock offering that we completed in.
February we also feel pretty good as we've said that we have sufficient cash now to take us through into the second cost.
Next year.
Which is important given.
The sort of the topline data readout, we still have flexibility beyond that going forward.
Thank you very much.
Great. Thank you. Our next question is from Liana Moussatos with Wedbush Securities. Please proceed.
Congratulations congratulations on your progress.
In the press release, you mentioned as part of commercial preparation.
And that you're getting a detailed understanding of the treatment centers can you share some of that understanding that you've gained.
Yes, Hi, Liana Pamela.
Thank you for the question, Yes, we've recently started our account mapping initiative in which we're going out to pediatric libert treatment centers.
To really understand a couple of 91 is to profile.
I have a better understanding of the patient slow referral patterns and the unique needs from each of each of these centers in terms of education and support and were able to do this has you know because we'd have the concentrated small prescriber base in the U.S. and in Europe. So we are just starting with out we've gone through the pilot.
Yes, we are collecting a lot of.
Very useful information for commercialization efforts.
Thank you very much.
Thank you Ana.
Our next question is from Ed Arce with H.C. Wainwright. Please proceed.
Hi, everyone. Good morning, Thanks for taking my questions and congrats on all the progress last year.
Couple of questions for me.
On your.
Failure Atresia our program congrats on getting the I Andy.
For that.
Two questions one is.
What is the if you could go over the rationale on dose selection for the 120 make per keurig.
On that study.
And secondly.
Given that you've agreed with the agency on a proportion of patients alive.
It's explicitly mortality endpoint so.
I would assume that.
This would allow for full approval.
And then thirdly, maybe it's a bit too early but when could you expect data in this program.
And I have a couple of follow ups. Thanks.
Okay and so this is Pat so on.
Dose selection. So this really kind of mirrors, what we dive in a phase two and other studies.
The phase two study we looked at doses ranging from 10 to 200 and micrograms per kilogram per day in this ongoing phase three study we're looking at.
40 micrograms per kilogram per day, and 120 microgram per kilogram.
Per day.
Based on the preclinical model based on the PK, we did in the phase two study based on the exposure.
Okay, and kind of that the PK PD motto.
In the animals and then also it matches with the phase three to 120 microgram per kilogram per day, those should be the effective dose 95, so 95% of the patients exposed to this should actually.
The desired therapeutic effect, obviously, we're trying to prove that in in the ongoing pick program.
But we've also seen in that phase two study and even in the ongoing phase three study that this dose seems to be well tolerated and as a dose we want to carry forward.
I think for the end the commercialization and marketing it's ideal to have the same dose in the same indication. So so that's why we're going with 120 microgram per kilogram per day dose.
So we're not really going for mortality endpoint.
It's patients allied with their native liver. So the majority of these patients while still be alive, but we'll have undergone liver transplant. If you look at at if you look at the.
Overall statistics relatively few of these patients die from their billion area Trisha at least initially, but they get the liver transplant, but your second point years right yet as a study that will be if positive will lead to an indication for disease modification. Hence we are looking at disease modification.
We haven't given guidance on the timing of the data yet.
And we just need to get the sites up and going and once we start to see how how enrollment is going then we'll provide timelines.
And then as you're thinking of your supplementary questions you as it relates to dosing remember for the pet pick one study in pediatric we had selected two doses onetwenty.
And the 40.
And the 14 was really there as a backup to see if we would run into any additional diarrhea or anything like that and what we found is the 120 dose obviously were blinded to the pet FIC results, but the onetwenty dose there's been nothing thats popped up with us like gives us a lot of confidence going forward with 100.
20 in the middle retrieve study into pets Boyd connect with the 50 and it also connex, we'd like to have one commercial dose right across the diseases.
Have you had some other supplementary questions.
Just a couple of quick ones thanks for that.
On your Petrik, one phase three study.
If you could tell us when you reached that full enrollment target of 60.
And then.
Just a.
A quick finance question for Simon.
What is your following this recent raise last month what is your current shares out thanks.
Yeah, just quickly on on the on the on the first question at we reached full enrollment in this quarter earlier. This year I think we made an announcement in January that we were at 59, so at a little bit of time, a more often going from there Simon yes, the shares outstanding at the moment.
14.9 million following the raised that we dated to sort of the beginning of February.
Thanks for the questions that I appreciate it thank you.
Our next question is from Ritu Baral with Cowen and company. Please proceed.
Hey, guys do you sell more imports equals taken in question just two for me one is it because it's really the chooses study the phase three wondering what the powering assumptions are based off of do move going three patients in the phase two.
And then secondly for the NDA filing are there any other gating factors other than just the manufacturing studies, which is like preclinical tox studies or anything like that prior to filing thanks a lot.
Yes. So this is this is pat so in the phase three study.
What we did was we looked at the natural history data and we looked at the natural history data collected.
By the children's network through their group called contract analytics that looks at the natural history of the billing area trees, yet patient population over the last 10 to 15 year and then we also looked at the led the published literature on this and we did that and that is what we consider to be the placebo response.
And then we assumed a very modest increase based on.
Active treatment.
And you're right in the phase two study you only had the three patients denim is only a four week treatment, but we base, we based our assumption on a very modest.
Benefit to the active treatment and Thats why the biliary Atresia study is enrolling 200 patients rather than the fixing these on detail. So we took that into account, but this is really important question right because I think it's our belief.
That delivery at trees, the disease itself bile acid levels have a big impact on that so if you look on the natural history data those individuals that have bile acid levels lowered tend to be alive and have need of liver two years out there.
Those who have higher bile acid levels.
Tend to die or have a liver liver transplant. So thats the base that weve used to to power. The study not just a few patients that we had in the face phase two study and as it relates to the Anda filing.
There is nothing gaining to the filing other than the data itself.
And that's the way we believe it should be so we're in we're in very good shape from a manufacturing perspective in that we're using the plan commercial formulation.
The registration batches are our on stability and were following that lead that the FDA has given us some 2018 for CMC planning in terms of a long term talks as needed can stops reprotoxic cetera. Those are all done. So I think we're in very good shape.
For a filing following receiving topline data.
Great. Thank you.
Our next question is from the Tim Lugo with William Blair. Please proceed.
Hi, Thanks for taking my question and following up on believe Atresia can you just maybe share with us some initial pharmacoeconomic data or maybe just the.
Any of the feedback you're hearing around maintaining their native liver survival.
Any of the benefits of avoiding liver transplant disease modification.
Proof I mean that seems like a very powerful including versus just serum bile acid and you're right. It's endpoints I'm just wondering if that changes your thoughts around pricing eventually.
Yes, Tim is front here. Thanks very much for the question. We started to do a lot of market research with the payer community and I think.
First thing that that we know it is across these cola static liver diseases theres not much knowledge about them and you would expect that could these are ultra rare diseases. So that's sort of job one to educate them in regards to all of these diseases.
The second thing is that when we talk to them about P. FICC and these diseases. They say well. These are very serious diseases, and and I think that theyve indicates to us a willingness to entered the dialogue.
And talk to us about about access and what they said to US though is what do we need to achieve the access that what they want and doesn't surprise you. Yes. It will be about it will be about data and so.
The data from the Patrick One study will provide important data on on the symptoms of Pruritis and bile acids. The long term extensions Patrick to study will provide data on from the durability effect the expanded cohort will provide data in other.
Perfect patients and then the Napa data the world's largest natural history data will help us with long term outcomes as well and then if you throw on top of that are billerica trees study as we said earlier. This this is a study around disease modification. This just further strengthens our whole data package.
For access and so we feel very good about this study in helping us.
With answering some of the questions on we're going to receive from payers.
Thanks, Thank you slipped in.
And maybe a follow up question.
I noticed a simple company that does conclude day by day, obviously the street.
Hello down well have concern last week, but can you just talked about maybe any logistical issues.
May be coming up as you navigate pick one during those others public public health concerns around October 19.
Yeah, Tim of course, this is a fluid situation and obviously we're monitoring.
Really carefully we're following the CDC recommendations, thus far we're not experiencing any issues. We don't anticipate any disruptions that being said they'll will continue to monitor very carefully for all of our programs and all of our studies to make sure that we do the right things.
Thank you Sir.
Our next question is from Alan Carr with Needham and company. Please proceed.
[noise] questions.
Couple of them.
Give us an update on the number and nature patients and.
Let's take two cohort two and.
Implications for the label and then also Simon.
Analytics, a bad revenues in Japan.
I believe you're entitled to a $15 million payment sales milestone payment simply with or can you tell us about went when that might come. Thanks.
So in regards to the pet FICC a perfect to study I think at the same all we can really say is we're really pleased that number of patients are rolling in it to the study.
And that you already we have.
Patients that have been in the study we are for well over a year. We're pleased with the high rate of rollovers and Matt that data will actually be helpful.
In our regulatory filing as well.
I'm thinking of cohort.
Well remember for Petrik too there are two cohorts right. There is the the rollover cohort from Patrick one and then there's cohort to the expanded cohort.
In the rollover cohort, we are pretty pleased with very high rated rollover synthetic won and there are patients now that are over a year in the expanded cohort which are individuals that did not qualify for Patrick one. This will provide his data when a wider range of a pediatric patients and the.
A combination we believe will help us with our regulatory.
Our regulatory filings.
Yes.
Oh, Okay, all right I'll leave you alone on that one as Simon [laughter] and in terms of your question about potential.
$15 million.
Sales milestone from Japan.
Really we yes, the sales all the elevates about in Japan in chronic constipation.
Doing well, but as you know that sets sort of monetized in terms of $15 million that we could possibly get as a result of certain sales being achieved in the Japanese market. We just treat that as upside if it comes it comes if it doesnt.
It doesn't so it's not in any of our plans at this stage.
Okay. Thanks for taking my questions.
Thank you.
Our next question is from Matt Kaplan with Ladenburg Thalmann. Please proceed.
Hi, guys. Thanks for taking the questions.
Yes, just a follow up for Pat in terms of the hierarchical analysis that you plan to do surpassed the one.
You mentioned that first it was pooled analysis than low dose than high dose just a question in terms of why do the low dose first and hierarchical analysis versus the yes. So I actually think I misspoke, it's actually high dose first and then low dose okay. Great. Thank you okay.
And then in terms of the D.A. assays three can you remind us how long it. It took you to enroll the pet pick one study and are there any learnings I guess from that study in terms of facilitating role.
Mint and.
You know patients that.
I didn't qualify for whatever reasons during the screening period that you can apply to the.
The D.A. study were enrolling you know many more patients 200 patients and give us a sense in terms of.
How how long you think it could take into enrolling that study.
Yes so.
Looking it took us I guess about 15 to 18 months to enroll the pet pick one study.
But the studies are very different so had pick one had some pretty tight exclusion criteria. There was genetic analysis there was different bile acid levels. There was defined liver enzymes that they had any.
Yeah. So there were some restrictions on enrollment the Billy area treatment study is pretty much at all commerce study so that any baby that had their pets I procedure is eligible to enroll with very very few exceptions.
So so there's a different there I think the other thing is that bill area trees, the incidence and prevalence of biliary atresia is larger.
We are going for more sites.
Not quite double the number of sites, but we plan on at least 70 75 sites for the billing area treatment study. So we did learn a lot I think and as Ron said the majority of the investigators that were in that Patrick one and two study will be in the failure Treehouse study. So they know us we know.
Hopefully that will speed up some of that very practical operation point of view like contracting and all of that so again, we are optimistic we believe we have the right number of sites, we have that the people onboard they're very excited about this study.
And we look forward to really get started.
Matt I guess, the only other difference from the peak studies Bill earlier trees. Your went to 45 sites. They were predominantly Europe us aggressive world in this case as Pat said the 70 to 75 sites. We are we going to countries, where the disease occurs more frequently as well.
But I think we've learned an awful lot from the from the Patrick once I think as US This was actually the very first.
Randomized placebo controlled pivotal trial with this community and as Pat says, we built some really tremendous relationships with the sites and the investigators and we will build off of that as we go to biller atresia.
That's helpful. And then just just circling back to pets, Nick one does your second half 21 approval launch assume.
And approval and launch time.
Does it assume a priority review.
Yes, it does and we have fast track designation and an orphan designation.
Priority review is what you asked for when you do the filing but were fully expecting priority review.
Okay.
Great.
Thanks for taking the question.
And we now have a follow up question from and ours with H.C. Wainwright. Please proceed.
Hi, Thanks, Robert Thanks for taking the follow up just wanted to get a sense.
On.
The Algeo syndrome program given that you had.
Or is scheduled.
And the FDA meeting this quarter.
Is there any thoughts that you can share preliminarily on the design of that study. Thank you.
No I think we want to wait until we have clearance with the FDA has those regulatory discussions before we really start to release much about that.
Fair enough. Thank you.
Okay.
We have reached another question answer session I would like to turn the call back over to management for closing remarks.
Great. Thank you operator, thank you everybody off for for tuning in hopefully you can see is this as the year of data for Alvarado we expect.
In mid Twentytwenty topline results from the pet pick one pivotal program with order because of bad NP FICC, we expect.
These two data from the U.S. study for Elagolix about an Apple the Nash and we hope that later this year early next year, we expect data from the Japanese study available, it's about an Apple de Nash as well.
With that with the data coming I think hopefully you get a sense that we're Oregon, where we are organized as an organization, we're ready to execute on commercializing Ela VIX about and really building. The company. So again I want to thank you very much for your interest in Alberta and wish you all have very good day.
Thank you. This concludes today's conference you may disconnect your lines at this time and thank you for your participation.
[noise].