ALBO (2019 - Q4)

Greetings. Welcome to Albireo Pharma's Fourth Quarter and Full-Year 2019 Earnings Call. [Operator Instructions] Please note, this conference is being recorded. I will now turn the conference over to your host Paul Arndt, Managing Director of LifeScience Advisors. Thank you. You may begin. Paul Arn

Thank you, operator and good morning everyone. Thank you for joining today's call. This morning Albireo 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.albireopharma.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 and they are subject to various risks and uncertainties. Actual results may differ materially due to various important factors including those described in the Risk Factors section of our most recent Form 10-K and our subsequent SEC filings. These filings can be accessed from the Media and Investor section of our website at www.albireopharma.com or on the SEC's website. Any forward-looking statements represent our views as of today March 2, 2020 and should not be relied upon as representing our views as of any subsequent date. We undertake no obligation to publicly update these statements. And now I'll turn the call over to Ron Cooper, Albireo's President and Chief Executive Officer. Ron?

Great. Thank you, Paul and thanks to all of you who have joined us this morning. With me today is Albireo's Chief Medical Officer, Dr. Patrick Horn; our Chief Commercial Officer, Pamela Stephenson; and our Chief Financial Officer, Simon Harford. We expect that 2020 will be a momentous year for Albireo and the communities that we're 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 odevixibat; two, outline our go-to-market strategy and our roadmap for success; and three, provide an update on our emerging pipeline. Now we have a number of exciting developments with the odevixibat clinical development program. But let's first look at odevixibat in PFIC. We enrolled more than 100 patients in the PEDFIC 1 pivotal trial and are very pleased to randomize 62 patients versus a planned 60 patients. We expect top line data mid this year. We are immensely grateful to the patients, the families and physicians who have participated in the PEDFIC program and have worked with us towards bringing what could be the first pharmacological treatment option to the PFIC community. Meanwhile, patients have been enrolling over on PEDFIC 1 to Cohort 1 of the PEDFIC 2 long-term open-label extension study. And we now have patients who've been on odevixibat therapy for well over a year. At the same time, we continue to enroll the expanded second cohort of PEDFIC 2, which includes PFIC patients who did not meet the eligibility criteria for PEDFIC 1. With PEDFIC 1 fully randomized, we will capitalize on this opportunity to generate longer term data and broaden the body of odevixibat evidence in different patient types. We're preparing for regulatory submission following the top line announcement and expect odevixibat approval a launch for PFIC in the second half of 2021. Turning to our next indication, biliary atresia, we are very pleased to receive investigational new drug IND clearance from the FDA, following productive discussions on trial design. Pivotal trial, biliary atresia and the use of odevixibat in treating liver disease is a precedent setting, first ever pivotal trial conducted in biliary atresia. We're extremely excited to initiate this program as our many members of the physician community who treat this devastating condition. We believe that biliary atresia is the most common rare pediatric liver disease with roughly 15,000 to 20,000 patients in the U.S. and EU combined, and for which there is no approved pharmacological therapy. Virtually all of these children are usually diagnosed in the first month of life as their stools are clay colored or not covered at all. After a biochemical workup, ultrasound and confirmation of biliary atresia, the child immediately undergoes a Kasai procedure, a surgery in which child's damage biles are to remove and the intestine is attached to the liver to restore enterohepatic circulation. This is a very serious disease that despite undergoing a surgery, about 50% of the patients require a liver transplant within 2 years, and about 80% of the patients require transplant by age 20. There are cumulating natural history data that indicate that those children who have lower serum bile acids have an improvement in key liver biomarkers and need of liver survival. Odevixibat lower sperm bile acid levels and potentially could be the first pharmacological treatment that slows ongoing damage to the liver. Both will be a double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of odevixibat compared with placebo in children with biliary atresia, who've undergone a Kasai procedure. Children in the treatment arm will receive odevixibat of 120 micrograms per kilogram per day, orally administered for 24 months. The primary efficacy endpoints with the 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 PEDFIC 1 trial with approximately 200 infants, given that biliary atresia is a more common disease. We initially proposed study designs using biomarkers in a shorter duration, but regulators desired at clinical outcomes, 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 EMA have granted odevixibat orphan designation in biliary atresia. We are confident in our ability to initiate and conduct this trial with both -- and plan to begin the study in the first half of 2020. We will provide more detail on the trial as the program progresses. Now guided by recent regulatory actions and the duration of the biliary atresia trials, we’ve decided to accelerate development in Alagille syndrome and plan to initiate a third odevixibat pivotal program by the end of the year. We’ve a scheduled interaction with the FDA this quarter to gain feedback on our pivotal program. Both the FDA and the EMA have granted odevixibat orphan designation in Alagille syndrome. We would expect the Alagille top line data to be available between the announcement of the PFIC and biliary atresia top line results. With Phase 3 data in PFIC and pivotal trials underway in biliary atresia and Alagille syndrome, we will be generating significant data that ultimately could support odevixibat use in a larger patient population estimated to be roughly 30,000 in the U.S. and EU combined. Many pediatric hepatologists will be participating in all three pivotal programs and these physicians will have considerable experience with odevixibat and Albireo. We are planning for success with Albireo and we made excellent progress in our go-to-market work in 2019 and are accelerating our readiness activities in 2020. So our key areas of focus are manufacturing, key opinion leader engagement, pricing and reimbursement and patient advocacy, while we build depth and expertise in the organization to execute on our plan. Let me go through these a little bit more deeply. So, first of all, we've made excellent progress in manufacturing and supply chain planning. We're using the planned commercial formulation in our Phase 3 study and registration batches are on stability. We are executing on the 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 pediatric hepatologists in the U.S and the same in Europe. We're planning activities to continue our strong and positive interactions with this community, much of which is participating in our three pivotal programs. We've begun a treatment site mapping exercise to help us get better insight in patient management and flow. Of course, pricing and reimbursement continues to be an emerging challenge for the industry broadly. We plan to be ready with strong arguments and data. The draft value story is ready and work is commenced on the cost effectiveness model, budget impact model, burden of disease study and other key elements of our access evidence. We're very grateful to Patient Advocacy Association for their insights and absolutely wonderful collaboration. We're continuing to build on our relationship and have created pficvoices.com, which is an opportunity for PFIC families to tell their story. The patient advocacy community provide feedback which we use to develop study designs, for improvement in patients services initiatives. Let me close this call by providing an update on our rapidly emerging pipeline. In Q2 of last year, we initiated our first Phase 2 trial with elobixibat, NAFLD and NASH. We're pleased with enrollment. We continue to expect top line data by middle of this year. As a reminder, this is a Phase 2 proof-of-concept study investigating the effect of elobixibat 5 milligram and NAFLD, NASH patients over 16 weeks. I would like to set appropriate expectations for this study. As this is the first study ever conducted in both NAFLD and NASH patients with an IBAT inhibitor, we're looking for the combination of positive trends in liver markers, TB risk factors and favorable GI tolerability. Now, in addition to this study, our Japanese partner, EA Pharma, sponsoring an investigator sponsored study in Japan with 100 patients randomized to a higher dose of elobixibat, 10 milligram, the bile acid sequestrant [indiscernible], the combination of [indiscernible] and elobixibat 10 milligram or placebo. Patients will be studied 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. Albireo has 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 means and tolerability. We have promising clinical and preclinical data showing that IBAT inhibitors have a positive effect on lipid, glucose, liver inflammation, liver fibrosis and elevated bile acid, all the hallmarks of NASH. Elobixibat is also oral once daily compound that has mineral systemic absorption. o we think it can be combined easily with other cardiovascular risks and NASH products. With the data from the two NAFLD/NASH studies, we believe our NASH program could create strong value for potential partnering opportunities. We also continue drive innovation and are excited to be making progress with our preclinical 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 IBAT inhibitors. We are pleased to select a lead candidate from our very productive compound library, file for new patents and plan to complete IND enabling study 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. So last, for A3384,which we're developing for bile acid malabsorption, we are pleased to have new patent issue, but with competing priorities, we're evaluating next steps for this program. I began today's call by referring to 2020 as a momentous year for Albireo Pharma. After reviewing in greater detail, odevixibat and pipeline progress, I hope that you can see why we are so excited about the potential for a tremendous 2020. So with that, it's my pleasure to turn the call over to Simon for a financial update. Simon, please.

Thank you, Ron. Let me quickly summarize our financial results for the full-year and Q4 2019. Revenues were $9.6 million for the year ended December 31, 2019 compared to $12.7 million for the year ended December 31, 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 MHLW of the elobixibat for the treatment of chronic constipation, offset by royalty revenue from EA Pharma. Revenues were $6.4 million for the fourth quarter of 2019, compared to $0.6 million in the same period last year. The increase of $5.8 million was primarily due to royalty revenue received from a EA Pharma. Research and development expense was $45.6 million for 2019, up from $31.7 million for the same period the year before. R&D expense was $14.2 million for the quarter, compared to $9.5 million for the fourth quarter of 2018. The increases to both the full-year and fourth quarter were primarily the result of program expenses for odevixibat, elobixibat and preclinical work as well as personnel costs as we continue to increase our program activities and headcount. General and administrative expense was $23 million for 2019 compared to $18.1 million for the previous year. For the fourth quarter 2019, G&A expense was $6.2 million compared to $5.8 million to the same quarter in 2018. For both the full-year and 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 year ended December, 31, 2019 was $62.7 million or a loss of $5.04 per share compared to $46.1 million or a loss of $3.94 per share for the year ended December 31, 2018. For the fourth quarter 2019, the net loss was $7.5 million, or a loss of $0.57 per share compared to a net loss of $15.8 million or a $1.34 per share in the fourth quarter of 2018. As of December 31, 2019, we had a balance of cash of $131.8 million compared to $142.7 million on September 30, 2019. In early February 2020, we close the common stock offering, which provided an additional $43.2 million of cash to fund the acceleration of pivotal trials for odevixibat in biliary atresia and the Alagille syndrome that Ron talked about earlier on the call. We are providing guidance on 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 half of 2021. With that, let me turn the call back over to Ron for closing remarks.

Good job, Simon. Thanks very much. In summary, when we come to the end of 2020, we should have read out the data for odevixibat, and PFIC, started two additional pivotal odevixibat programs, read out the Phase 2 elobixibat NAFLD/NASH study and giving more insights on our pipeline programs. At the same time, we will strengthen the capabilities of the organization and build depth and expertise for successful commercial execution. Reflecting on at the last several years, something else has evolved and our understanding of the rare disease communities that we're striving to help. We know these children and families more intimately now, and it's impossible to think about the year ahead without thinking about what our success could mean to them. We sometimes say today somewhere a mother or a father is making a terrible choice to relieve a child's 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 true scope of the opportunity goes far beyond Albireo. That's firmly in focus for us as we start this exciting year. With that, we will open-up the call to questions. Operator?

Thank you. [Operator Instructions] Our first question is from Yasmeen Rahimi with ROTH Capital Partners. Please proceed.

Hi, team. Congrats on the continued great update. I have two questions. As we are headed into the PEDFIC 1 study, the first question is just a reminder. Is the study powered to show that PFIC in both doses were in or in totality of both or versus individual one? And then the second one, it would be lovely to get a reminder on what your expectation is in regards to the placebo response on the primary endpoint? And maybe also what are trial elements that were included or incorporated in order to minimize that placebo response in the primary endpoint? And thank you very much for taking my questions.

Yes. Yes, Yasmeen, this is Pat. So thank you very much for the question. So, yes, the study is powered and the statistical analysis is the hierarchical statistical analysis, whereas placebo versus pool odevixibat, 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 single comparison, placebo versus low dose placebo versus high dose. And with the second response, it is always hard to know about the placebo response. And we looked at this, we've looked at all of the data, there haven't really been a lot of studies in PFIC, but there have been other studies in the cholestatic liver disease in children and a couple of things on it. So we are looking across the entire 24-week period and the placebo responses we've seen tend to diminish during the first part, 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. But regardless, when we did our power calculation for pruritus, we assumed relatively modest response in the active arm and a large response in the placebo arm. And we did our sample-sized calculations based on that.

It is -- Yasmeen, it's Ron here. Just to add, thank you for that, Pat. And just to summarize, the study is powered of well over 80% against the pruritus endpoint. Therefore, given less variability with the serum bile acid endpoint, which is the primary endpoint in Europe, that powering assumption, it is even greater. And I think we feel pretty comfortable that we understand that there is more variability with pruritus, but we've tried to mitigate that, as Pat has said, by taking an aggressive placebo response, by making this study probably larger than we originally planned. And by going to 24 weeks, these things we believe should mitigate that. Thanks very much for questions, Yasmeen. Appreciate it.

Thank you.

Our next question is from Eun Yang with Jefferies. Please proceed.

Thank you. So my understanding is that current Phase 3 study for PFIC, you are actually enrolling patients with or without PEBD surgery. So question is once approved, what percent of patients who have undergone PEBD surgery would be eligible for your treatment?

Hi, Eun. Good morning. Thanks very much for your question. Like usual, the challenge in this space, the rare orphan diseases, there is no published data on your question, nor are there any registries. You're absolutely correct. That we anticipate -- in our Phase 3 study, we had patients who were before PEBD surgery and others who would had PEBD surgery, but still fulfilled our entry criteria. So I think it'd be -- can be difficult for us to actually estimate the number of patients that would be eligible, but we -- from our estimates, we believe there's somewhere between 8,000 to 10,000 individuals in the U.S and Europe who have PFIC and they are in various stages of treatment. So big opportunity.

Thank you.

Thanks for your question, Eun.

And then a question for Simon. Current -- that is this year's cash…. of about $100 million. Does that include revenue running similar to that in 2019?

So the revenue really ultimately at the end of the day is sort of somewhat irrelevant because it flows through to the fact that we monetize the royalty and that's what -- that's revenue from the royalty. In terms of the actual overall expenses, obviously, $100 million is a pick up from what we saw in 2019 where we had roughly $70 million of total expenses combined. And that pick up is really being driven by obviously the additional biliary atresia and Alagille syndrome studies as well as the NASH study and commercial sort of expectations. I think it's fair 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 half of next year, which is important given the sort of the top line data readout, we still have flexibility beyond that going forward.

Thank you very much.

Thank you, Eun.

Our next question is from Liana Moussatos with Wedbush Securities. Please proceed.

Congratulations on all your progress. In the press release, you mentioned sort of commercial separation that you're getting a detailed understanding of the treatment center. Can you share some of that understanding that you've gained?

Yes. Hi, Liana. It's Pamela. Thank you for the question. Yes, we've recently started our account mapping initiatives, in which we're going out to pediatric liver treatment centers to really understand a couple of things. One is to profile and they have a better understanding of the patient flow, referral pattern and the unique needs of each these centers in terms of education and support. And we're able to do this, as you know, because we have a concentrated small prescriber base in the U.S. and in Europe. So we are just starting this out. We've gone through the pilot phase. We are collecting a lot of very useful information for commercialization efforts.

Thank you very much.

Thank you, Liana.

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 biliary atresia program, congrats on getting the INDI for that. Two questions. One is, what is the -- if you could go over the rationale on dose selection for the 120 mg/kg on that study? And secondly, given that you've agreed with the agency on a proportion of patients alive, it is explicitly a 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, Ed. Yes, so this is Pat. So on dose selection, so it's really kind of mirrors what we've done in the Phase 2 and other study. So, on the Phase 2 study, we looked at doses ranging from 10 to 200 micrograms per kilogram per day. In this ongoing Phase 3 study, we're looking at 40 micrograms per kilogram per day and 120 micrograms per kilogram per day. Based on the preclinical model, based on the PK we did in the Phase 2 study, based on the exposures and kind of the PKPD model in the animals, and then also it matches with the Phase 3. The 120 micrograms per kilogram per day dose should be the effective dose 95. So 95% of the patients exposed to this should actually have the desired therapeutic effect. Obviously, we're trying to prove that in the ongoing PFIC program. But we've also seen in the Phase 2 study and even in the ongoing Phase 3 study that this dose seems to be well tolerated. And that’s a dose we want to carry forward. I think for -- the commercialization and marketing is on deal to have the same dose in the same indication. So that's why we're going with 120 micrograms per kilogram per day dose. So we're not really going for a mortality endpoint if patients alive with their native [ph] liver. So the majority of these patients will still be alive, but will have undergone liver transplant. If you look at the overall statistics, relatively few of these patients die from their biliary atresia, at least initially, but they get their liver transplant. But your second point is right. This is a study that will be, if positive, will be good indication for disease modification since 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 enrollment is going, then we'll provide a timeline.

And, Ed, as you're thinking of your supplementary questions, as it relates to dosing, remember for the PEDFIC 1 study in PFIC, we had selected two doses, 120 and the 40. And the 40 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, we're blind to the PEDFIC results, but the 120 dose, there's been nothing that's popped up with us that gives us a lot of confidence going forward with 120 in the biliary atresia study and to Pat's point that connects with the ED 50 and it also connects we'd like to have one commercial dose right across the diseases. Ed, you had some other supplementary questions?

Yes, just a couple of quick ones. Thanks for that. On your PEDFIC 1 Phase 3 study, if you could tell us when you reached that full enrollment target of 60? And then, just a quick finance question for Simon. What is your -- following this recent raise last month, what is your current shares out? Thanks.

Yes, just quickly on the first question, Ed, we reach for 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 and we're often going from there. Simon?

Yes. The shares outstanding at the moment are now 14.9 million following the raise that we did at the sort of the beginning of February.

Thanks for the questions, Ed. Appreciate it.

Thank you.

Our next question is from Ritu Baral with Cowen & Company. Please proceed.

Hey, guys. [Indiscernible]. Thanks for taking the question. Just two for me. One is with regards to biliary atresia study for Phase 3, wondering what the powering assumptions are based off of [indiscernible] three patients in the Phase 2?And then secondly, for the NDA filing, are there any other gating factors other than just the manufacturing studies such as like preclinical fact studies or anything like that prior to filing? Thanks a lot.

Yes. So this is Pat. So in the Phase 3 study, what we did was we looked at the natural history data and we've looked at the natural history data collected by the children's network through their group called Contract Analytics that looked at the natural history of the biliary atresia, patient population over the last 10 to 15 years. And then we also looked at the list, 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 2 study we only had the three patients and it was only a 4-week treatment. But we based our assumption on a very modest benefit to the active treatment. And that's why the biliary atresia study is enrolling 200 patients rather than the 60 we saw in PFIC. So we took that into account.

But this is a really important question, right? Because I think it's our belief, that biliary atresia, the disease itself, bile acid levels, have a big impact on that. So if you look at the natural history data, those individuals that have bile acid levels can lower tend to be alive and have need of liver two years out. Those who have higher bile acid levels tend to die or have a liver transplant. That's the base that we've used to power the study, not just the few patients that we had in the Phase 2 study. And as it relates to the NDA filing, there is nothing gating to the filing other than the data itself. And that's the way we believe it should be. So we're in very good shape from a manufacturing perspective and that we're using the planned commercial formulation, the registration batches are on stability and we're following the lead that the FDA has given us in 2018 for our CMC planning. In terms of the long-term tox is needed, cancer tox [indiscernible] etcetera, those are all done. So I think we're in very good shape for a filing following receiving top line data.

Great. Thank you.

Thank you.

Our next question is from Tim Lugo with William Blair. Please proceed.

Thanks for taking my question. Following up on biliary atresia, can you just maybe share with us some of the initial pharmacoeconomic data, or maybe just the -- any of the feedback you're hearing around maintaining the native live survival? Any of the benefits of avoiding liver transplant, the disease modifications kind of curve? I mean, that seems like a very powerful endpoint versus just given bile acid and pruritus endpoint. I'm just wondering if that changes your thoughts around pricing eventually?

Yes, Tim. It's Ron here. Thanks very much for the question. We started to do a lot of market research with the payer community. And I think, the first thing that we note is the [indiscernible] cholestatic liver diseases, there is not much knowledge about them. And you would expect that, because these are all for rare diseases. That's for job one to educate them in regards to all of these diseases. The second thing is that when we talk to them about PFIC and these diseases, they say, well, these are very serious diseases. And I think that they've indicated to us, a willingness to enter the dialogue and talk to us about access. And what they said to us, though, is what do we need to achieve the access that they want, and it doesn't surprise you. It will be about data. And so, the data from the PEDFIC 1 study will provide important data on the symptoms of pruritus and bile acid. The long-term extension, PEDFIC 2 study will provide data on -- from the durability effect, the expanded cohort to provide data in other PFIC patients, and then the NAFLD 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 our biliary atresia study, as we said earlier, this is a study around disease modification. This just further strengthens our whole data package for access. So we feel very good about this study in helping us, with answering some of the questions that we're going to receive from payers.

Okay. Thank you.

Thank you.

And maybe a follow-up question. I know this is something companies are adjusting to day by day. Obviously, the [indiscernible] had all the -- although have concerns last week. But can just talk about maybe any [indiscernible] issues that maybe coming up as you navigate PEDFIC 1 during this -- of this public health concerns around COVID-19?

Yes, Tim, of course. This is a fluid situation and obviously we're monitoring really carefully. We're following the CDC recommendations. Yes, thus far we're not experiencing any issues and we don't anticipate any disruptions. That being said, we'll 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, Ron.

Our next question is from Alan Carr with Needham and Company. Please proceed.

Questions. A couple of them. Give us an update on the number and nature of patients in PEDFIC 2 Cohort 2 and implications for the label? And then also, Simon, elobixibat revenues in Japan, I believe you're entitled to a $15 million payment, a sales milestone payment at some point. What can you tell us about when that might come? Thanks.

So in regards to the PEDFIC 2 study, I think at this time all we can really say is that we're really pleased that a number of patients are rolling in to the study and that already we have patients that have been in the study for well over a year. We're pleased with the high rate of rollovers. And that data will actually be helpful, in our regulatory filing as well.

And thinking of cohort [multiple speakers]?

Yes. Well, remember for PEDFIC 2 there are two cohorts, right. There is the rollover cohort from PEDFIC 1. And then there is Cohort 2, the expanded cohort. In the rollover cohort, we are pretty pleased, with the very high rate rollover from PEDFIC 1. And there are patients now that are over a year. In the expanded cohort, which are individuals that did not qualify for PEDFIC 1, this will provide us data in a wider range of PEDFIC 1 patients. And the combination, we believe, will help us with our regulatory -- our regulatory filing.

Are you able to [multiple speakers].

[Indiscernible].

Okay. All right. I will leave you alone on that one, Simon.

In terms of your question about potential $15 million sales milestone from Japan. Really we -- the sales of elobixibat in Japan in chronic constipation of doing well. But as you know that that’s sort of monetized. In terms of the $50 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 doesn't, it doesn't. So it's not in any of our plans at this stage.

Okay. Thanks for taking my question.

Thank you, Alan.

Our next question is from Matt Kaplan with Ladenburg Thalmann. Please proceed.

Hi, guys. Thanks for taking the question. I guess just a follow-up for Pat in terms of the hierarchical analysis that you've planned to do, the PEDFIC 1. You mentioned that first it was pooled analysis and low dose and high dose. Just a question in terms of why do that low dose first then the 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. [Indiscernible]. And then in terms of the VA Phase 3, Can you remind us, how long it took you to enroll the PEDFIC 1 study and are there any learnings, I guess, from that study in terms of facilitating enrollment? You know, patients that didn't qualify for whatever reason during the screening period that you can apply to the VA study where you’re enrolling many more patients, 200 patients? Give us a sense in terms of how long you think it could take to enroll that study.

Yes. So looking, it took us, I guess, about 15 to 18 months to enroll the PEDFIC 1 study. But the studies are very different. So PEDFIC 1 had some pretty tight exclusion criteria. There was genetic analysis. There was different bile acid levels. There was different liver enzymes that they had in meet. And so there were some restrictions on enrollment. The biliary atresia study is pretty much all comers study so that any baby that has their precise procedure is eligible to enroll with very, very few exceptions. So there is a difference there. I think the other thing is that, biliary atresia, the incidence and prevalence of biliary atresia is the 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 biliary atresia study. So we did learn a lot, I think and as Ron said, the majority of the investigators that were in that PEDFIC 1 and 2 study will be in the biliary atresia studies. So they know us, we know them. Hopefully that will speed up some of this very practical operation point of view like contracting and all of that. So, again, and we are optimistic. We believe we have the right number of sites we have that the people on board, they're very excited about the study and we look forward to really get started.

Hey, Matt, I guess the only other difference from the PFIC study, biliary atresia went to 45 states. They were predominately Europe, U.S., the rest of world. In this case, as Pat said, the 70 to 75 sites, will we be going to countries where the disease occurs more frequently as well? But I think we've learned an awful lot from the bad pick ones, so they could test. This was actually the very first randomized placebo control 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 biliary atresia.

And it's helpful. And then just going back to the PEDFIC 1, Does your second half '21 approval launch to an approval of 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, would you do the filing? But we're fully expecting priority review.

Okay, great. Thanks for taking the questions.

And we now have a follow-up question from Ed Arce with H.C. Wainwright. Please proceed.

Thanks for taking the follow-up. I just wanted to get a sense on the ALGEO Syndrome program, given you had or scheduled an FDA meeting this quarter. Is there any thought that you could share preliminary on the design of that study? Thank you.

No, I think we -- what a great until we have clearance with the FDA and have those regulatory discussions before we really start to release much about that.

Fair enough. Thank you.

Okay.

We have reached the end of our question-and-answer session, I would like to turn the call back over to management for closing remarks.

Great. Thank you, operator, and thank you, everybody, for tuning in. Hopefully you could see is, this is the year of data for Albireo. We expect in mid 2020, top line results from the PEDFIC 1 pivotal 4 gram of odevixibat [indiscernible] we expect phase 2 data from the U.S. study for elobixibat and NAFLD/NASH and we hope that later this early, early next year and we expect data from the Japanese study of hope that later this year, early next year, we expect data from the Japanese study of elobixibat and that will be NASH as well. With the data coming, I think hopefully we had a sense that we are organized as an organization. We are ready to execute on commercializing, elobixibat and really building the company. Okay. Now we want to thank you very much for your interest in Albireo. Wish you all a very good day.

Thank you. This concludes today's conference. You may disconnect your lines at this time, and thank you for your participation.