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Albireo Pharma, Inc.  (NASDAQ:ALBO)
Q4 2018 Earnings Conference Call
March 07, 2019, 8:30 a.m. ET

Contents:

Prepared Remarks:

Operator

Good morning, ladies and gentlemen. And thank you for joining us for Albireo's Conference Call to provide a Business Update for Year-End 2018. Following management's prepared remarks, we'll open the call for questions. I would now like to turn the call over to Paul Arndt from LifeSci Advisors, representing the Investor Relations company. Please go ahead, sir.

Paul Arndt -- Investor Relations

Thank you, operator, and good morning, everyone. Thank you for joining today's call during which management will provide an update on Albireo's performance in 2018. Earlier today, Albireo issued a press release highlighting recent business accomplishments and noting its financial results for the full year ended December 31, 2018. The press release is accessible via the Company's website at www.albireopharma.com.

Before proceeding, let me mention that comments today may include forward-looking statements made under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements other than statements of historical fact regarding the plans for or progress, scope, cost, duration or results of, development of A4250, elobixibat, A3384, or any other Albireo product candidate or program, including prevalence data, target indications for development, size, design, population, location, conduct, objective, duration, endpoints, timing for initiation or completion of, or for reporting of results from or regulatory feedback regarding any clinical trial, including Albireo's Phase 3 trial of A4250 in patients with PFIC, the timing for submission for approval of A4250, the commercial outlook for any Albireo product candidate, program or opportunity in any target indication, including potential coverage, pricing or reimbursement, any additional payment that HealthCare Royalty Partners or EA Pharma may take -- may make through Albireo, the period for which Albireo's cash resources will be sufficient to fund its operating requirements or Albireo's plans, expectations or future operations, financial position, revenues, costs or expenses.

Actual results may differ materially from those expressed or implied by any forward-looking statement as a result of many factors, including those described under the heading Forward-Looking Statements in the Albireo's press release from earlier today or under the heading Risk Factors in its most recent Form 10-K or in later fillings with the SEC.

Albireo cautions you not to place undue reliance on any forward-looking statement. Also, any forward-looking statement that is made speaks only as of today, Thursday, March 7, 2019 and should not be relied upon as representing Albireo's views as of any future date. Albireo disclaims any obligation to update any forward-looking statement, except as required by applicable law.

With that, I'll turn the call over to Ron Cooper, Albireo's President and CEO. Ron?

Ron Cooper -- President and Chief Executive Officer

Thank you, Paul, and thank you everybody for joining today's conference call. With me today is Dr. Patrick Horn, Albireo's Chief Medical Officer; and Simon Harford, our Chief Financial Officer. We are very pleased to be here today to review our delivery versus plan for 2018 and outline our priorities for 2019, 2020.

2018 was an important year in which we executed on our commitments. We expect 2019 and 2020 will be a transformative period for Albireo and I will provide a roadmap for the development of the Company during that period. So, let me begin with a quick review of 2018. We delivered on our commitment of starting the A4250 PEDFIC 1 trial in this spring. PEDFIC 1 is proceeding according to plan with 37 trial sites initiated versus our guidance of 35 to 45 as of February 28, 2019. And we continue to expect top line results end of this year or early 2020. We delivered on our commitment on the approval of elobixibat, our other IBAT inhibitor in the first half of 2018. Elobixibat was approved in Japan for chronic constipation, making it the first IBAT inhibitor approved in the world further validating our bile acid modulator scientific platform.

We delivered on our commitment to communicate important supportive data on A4250 pediatric liver diseases and PFIC natural history, which were presented at the major US and European liver scientific meetings. We delivered on our commitment to strengthen our regulatory position achieving our eighth A4250 orphan drug designation in the US and EU combined as well gaining priority review voucher eligibility and fast track designation in the US.

We delivered on our commitment to prepare for a high-quality A4250 launch agreeing with the FDA on a manufacturing plant, gaining acceptance of a proposed INN and gaining FDA conditional approval of the brand name. And we began our commercialization efforts with key leadership, commercial and medical hires, including Simon as our CFO & Treasurer, Pat as our CMO and Jason Duncan as our General Counsel. So as you can see, 2018 was a year in which we made excellent progress and it's provided a strong base to prepare us for success in 2019, 2020.

Now let's move to 2019, 2020 and the roadmap for Albireo. Our three development priorities are: number one, complete enrollment of the PEDFIC 1 A4250 Phase 3 study, submit to regulatory authorities for approval and ready for launch; number two, bring A4250 to a significantly larger patient population by initiating a trial in biliary atresia; number three, expanded to NASH with advancement of elobixibat and acceleration of our preclinical bile acid modulator compounds as candidates to partner in the future.

Let me go into a greater detail. First in PFIC, we began 2019 with much anticipation for the PEDFIC 1 study results, which are expected end of this year or early 2020. PEDFIC 1 remains our highest priority. So, as a reminder, this Phase 3 program is a single randomized, double-blind, placebo-controlled, multicenter clinical trial designed to enroll approximately 60 patients with PFIC, type 1 or type 2 and an open label extension study to assess long-term safety and durability of response.

The placebo-controlled trial is evaluating two doses of A4250 for 24 weeks. The primary endpoint for the FDA evaluation and a key secondary endpoint for the EMA is improvement in pruritus. The primary endpoint for the EMA and a key secondary endpoint for the FDA is serum bile acid responder rate. PEDFIC 1 is progressing well, and we remain optimistic about the outcome. We are preparing for potential approval and launch of A4250 in the first half of 2021, and we'll refine guidance as we get closer.

Second, we're extremely excited to begin study, A4250 in biliary atresia. Our intent has always been to expand the use of A4250 beyond PFIC into biliary atresia, Alagille syndrome, primary sclerosing cholangitis and other areas. We are pleased to announce that we will begin a pivotal study of A4250 in biliary atresia, a rare pediatric liver disease with major unmet medical need and relatively large population. And we'll do this in the second half of 2019.

Now, biliary atresia is a disease of the liver and bile ducts with symptoms developing about two to eight weeks after birth. Damaged or absent bile ducts result in bile and bile acids being trapped inside the liver and that results in liver cirrhosis and failure. About 80% of the patients require liver transplant within the first two decades of life making biliary atresia the most common indications for pediatric liver transplant.

Now, although there are no published prevalence data available, our estimates show biliary atresia is one of the most common rare pediatric liver diseases, with 15,000 to 20,000 patients estimated in the US and EU combined. We've received orphan drug designation for A4520 in biliary atresia in the US and the EU, and we've engaged regulators on trial design. We're in the process of finalizing a trial design with regulatory authorities. The study will build on the encouraging results we saw in our Phase 2 trial in pediatric cholestasis, and we anticipate starting the trial in the second half of 2019 and will provide more details in the coming months.

Biliary atresia and PFIC are the beginning for Albireo in rare pediatric liver disease. We continue to see potential for A4250 beyond these initial indications and plan to expand development into additional orphan pediatric liver diseases in 2020. Now, while Albireo is primary focused on pediatric orphan liver diseases, the potential of our NASH pipeline has continued to merit greater attention. And this year, we plan to take important steps forward in NASH. This is our third clinical development priority.

Bile acid modulation provides a strong rationale for development in NASH from both in efficacy and a convenience tolerability perspective. Elevated bile acids, along with elevated cholesterol, insulin sensitivity, liver inflammation and liver fibrosis are some of the key markers in NASH patients. We have clinical and preclinical data that show IBAT inhibitors may have a positive impact on these parameters. Additionally, elobixibat is a once-daily oral medicine that is showing minimal systemic exposure potentially allowing for dosing with cardiovascular disease risk-reducing medicines or other NASH products. Finally, elobixibat has a safety database with more than 1,500 exposures, which our understanding of its safety profile.

While NASH is projected to become the leading cause of liver transplants in the US, research has shown that most NAFLD patients die of cardiovascular complications. There is evidence of the elevated cholesterol and bile acids in the liver maybe major contributors to the disease and that reducing some of them to the elimination from the body could result in significant clinical effects. We're, therefore, excited to move elobixibat into a Phase 2 trial in NASH in Q2.

In addition, we are delighted with the progress of our preclinical compounds and have made a decision to allocate additional resources to accelerate their development. We hope to provide more detail on these compounds at a later date. The intention of the NASH program is to create additional value for the Company through potential partnering. As the new preclinical compounds warrant more resources and we are now accelerating multiple compounds in NASH, we will be reallocating resources from A3384 in bile acid malabsorption.

As we've previously guided, we believe A3384 has potential to become the first drug approved to treat bile acid malabsorption in the US, but further development has been engaged on the issuance of certain patents, which remain pending. We're confident these patents will issue, but have delayed trial initiation into 2020 and we will focus our resources on our activities with A4250 and in NASH.

So, in summary, 2018 was a historic year for Albireo in which we executed on our plan and built momentum coming into 2019. We expect 2019 to be a transformative year for Albireo. While we advance our Phase 3 trial in anticipation of top line results at the end of the year or early 2020, we also plan to take a major step forward with our broader pipeline, including A4250 in biliary atresia and elobixibat and our preclinical compounds in NASH.

Simon, it's my pleasure to hand it over to you for a financial update. Simon?

Simon Harford -- Chief Financial Officer

Thank you, Ron. Let me quickly highlight our financial results for the full year ended December 31, 2018. We closed the fourth quarter with a balance of $163.9 million in cash and cash equivalents, compared to $53.2 million at the end of December 2017. Our cash balance was reduced by $9.7 million during the fourth quarter, primarily as a result of our Phase 3 PFIC trial for A4250. Our revenue for the year ended 2018 was $12.7 million, compared to nominal revenue in the same period last year. Higher revenue was related to the $11.2 million milestone payment received earlier in the year from EA Pharma due to the approval of elobixibat for chronic constipation in Japan, as well as $1.5 million of royalty revenue received from EA Pharma for elobixibat since approval.

As you may recall, we monetized the royalty and sales milestone revenue in Japan with Healthcare Royalty Partners. Therefore, the royalty revenue that we are reporting is offset by non-cash interest expense in the income statement, which will continue going forward. Our research and development expenses for the full year ended December 31, 2018 were $31.7 million, compared to $13 million in the same period of 2017.

R&D expenses were primarily driven by costs associated with development of A4250, including costs incurred for manufacturing and clinical development activities for our Phase 3 trial in PFIC, higher headcount costs as we implement our strategy, and preclinical work primarily associated with NASH. Our G&A expenses for the full year were $18.1 million, compared to $15.2 million in the same period of 2017. G&A expenses increased 18.5%, principally attributable to increases in headcount and recruitment costs, as well as stock-based compensation costs.

Total expenses for the full year 2018 were $50.6 million, slightly above our guidance for total expenses of the high end of the range of $45 million to $50 million as we continue to make progress with the execution of our strategy. Net loss for the full year ended December 31, 2018 was $46.1 million. The loss was $3.94 per share, compared to a net loss of $24.4 million or $3.12 per share in the same period of 2017.

Based on our current operating plans, we anticipate our total operating expenses, including R&D and G&A expenses, for 2019 to be between $75 million and $80 million, driven primarily by expenses related to our Phase 3 PFIC clinical trial for A4250, as well as funding for our other clinical development priorities in biliary atresia and NASH. In terms of cash, we continue to expect our current cash balance to be sufficient to meet our operating requirements into 2021 under our current plan for clinical development.

And with that, let me turn the call back over to Ron for closing remarks.

Ron Cooper -- President and Chief Executive Officer

Great. Thank you, Simon. So to summarize, we expect this year will be truly transformational. We're fortunate compelling assets that continue to show promise as we leverage our novel bile acid modulation science and move forward in our commitment to bring potentially life-changing therapies to patients suffered from devastating liver and GI diseases. So with that, we'll open up the call to questions. Operator?

Questions and Answers:

Operator

Thank you. We will now be conducting a question-and-answer session. (Operator Instructions) Our first question comes from the line of Eun Yang with Jefferies. Please proceed with your question.

Eun Yang -- Jefferies -- Analyst

Thank you. I have a question on biliary atresia. So in your Phase 2, I think you had about 30 (ph) patients in the efficacy trial, so can you go over why you have seen there and also the patients -- are you going to be focusing on patients whoever undergone the biliary duct reconstruction surgery, and if so, what percent of biliary atresia patients actually undergone such a surgery? Thank you.

Ron Cooper -- President and Chief Executive Officer

Good morning, Eun. Thanks very much for your question. I think that first of all, we're pretty excited to announce that we will be stepping into a trial in biliary atresia. I think for A4250, we've always talked about A4250 becoming important pediatric cholestatic liver disease drug and our strategy has always been to start with PFIC and to expand, and our thoughts on expansion are based upon the Phase 2 results that you referred to. And within the Phase 2 study, we are able to show good improvements in serum bile acids and improvements in pruritus. We had a handful of patients in biliary atresia where we showed some good effects as well. Pat, you want to talk a little bit more about kind of the patient population you were thinking of?

Patrick Horn -- Chief Medical Officer

Yes. And so, in terms of the results we saw in our Phase 2 biliary atresia study, we're actually going to have a poster that specifically addresses that at the upcoming EASL meeting and so that data will be in there. But in general, we saw the same that we saw with the other population in the decrease in pruritus and a decrease in bile acids. In terms of the population, you're absolutely right. So patients -- when they are diagnosed with biliary atresia, immediately you go for the cup -- Kasai portoenterostomy, so they try to reestablish blood flow. So any study we do will be in patients post that Kasai procedure.

Ron Cooper -- President and Chief Executive Officer

Thanks for the question, Eun.

Operator

Thank you. Our next question comes from the line of Katherine Xu with William Blair. Please proceed with your question.

Katherine Xu -- William Blair & Company -- Analyst

Good morning. I'm just wondering whether you have applied for breakthrough designation for A4250, any indication and also (inaudible) atresia versus Alagille, just a little curious about that. It's just that data or the aetiology or just avoid direct competition with other company. And if you could just comment a little bit on the NASH Phase 2 design and thoughts and strategy going forward, what kind of data you're expecting and what class of agents you think is the most of my combinable is? Thank you.

Ron Cooper -- President and Chief Executive Officer

Okay. So Katherine, so, let's just make sure we -- first of all, good morning Katherine. Thanks for the questions. So question on breakthrough, question on our selection at the indication and a little bit about our NASH design, maybe, I'll start with the first one. We will hand the second one over to Pat. So breakthrough -- no, we've not applied for breakthrough designation as yet. I guess we probably have the opportunity to do so in the biliary atresia if we choose through. We already have for A4250 a number of important regulatory badges. So, we have eight orphan designations in the Europe, in the United States, and we have fast track in the US, which as you know, allows us for hopefully a speedier process as well. And quite frankly, the agency has been fantastic with us. We've enjoyed a really good dialog back and forth and they have been really helpful in the process. So we would anticipate that going forward. To talk about the -- our selection of biliary atresia, Pat?

Patrick Horn -- Chief Medical Officer

Yes. So you're absolutely right. When we looked at the biliary atresia, Alagille (inaudible), PSC and PBC are also possibilities. We look at it, there is truly a huge unmet medical need in the biliary atresia population. Right now, there really is no medical therapy for them and that disease progresses very rapidly, and it's among the fastest that lead to cirrhosis of any of the patient population. So, just in terms of that unmet medical need, there are some strong data presented at last year's Liver Meeting where -- even where post-biliary atresia elevated bile acids can lead to a decreased liver function. There is a real possibility to build to (ph) that population.

I think in terms of study feasibility, it's also one patient population size, it's also another factor, it certainly doesn't exclude us from going into Alagille (inaudible) and we are certainly considering it. One of the other real indications is in our discussions with the FDA, they expressed a real enthusiasm about having a trial in the biliary atresia population since this is an unmet need. So again, we need to just from resources -- predominantly people resources, we need to stage these. We can't start big programs at the same time, but clearly, we hope to expand into other indications as quickly as possible, but biliary atresia will be our first one.

Ron Cooper -- President and Chief Executive Officer

So just to summarize that one, yeah, Katherine, I think we're committed overall to developing A4250 across multiple pediatric cholestatic liver diseases. Our approach has been one of really trying to understand the disease really deeply, work with key opinion leaders, work with patient advocates and obviously, work with the regulators to get to with the trial design that we're comfortable with. And I think in this case with biliary atresia, we feel pretty confident from that perspective. We'll continue to evaluate some of the other pediatric cholestatic liver diseases. And as we've guided, our intent is to chip away at those over time as well. Pat, you want to talk a little bit about the NASH program.

Patrick Horn -- Chief Medical Officer

Yeah. So the NASH study is a Phase 2 study. The population is going to be patients with metabolic syndrome and either NAFLD or NASH and it is -- our primary endpoint is going to be change in LDL cholesterol. Obviously, we're going to look at liver, fat and other markers that Ron mentioned are applicable such as incidence sensitivity and cholesterol. It's a pretty standard Phase 2 study that really meets the new FDA guidance this for Phase 2 studies in NASH and NAFLD.

Ron Cooper -- President and Chief Executive Officer

So Katherine. Thank you, Pat. As we previously guided, we think about this Phase 2 study as a good proof-of-concept study to indicate some of the things, the effects that we've talked about previously in the study that would be consistent with the guidance that the FDA has put out on NASH. So hope we've got all your questions, Katherine. Thank you very much for them.

Operator

Thank you. Our next question comes from the line of Yasmeen Rahimi with ROTH Capital Partners. Please proceed with your question.

Katie An -- ROTH Capital Partners -- Analyst

Hi, everyone. Good morning. This is Katie An on for Yasmeen today. I have a few questions regarding biliary atresia. First, can you provide us with any additional color about the size, design and primary endpoint for the pivotal study? And can you describe how easy or difficult you think it will be to enroll? I guess, do you think it will be faster to enroll in PFIC?

Ron Cooper -- President and Chief Executive Officer

Hi, Katie. Good morning. Thanks very much for the questions. It's probably a little early for us to talk a little bit about trial design at this time because we're still in discussions with the regulatory authorities, but we're pleased about the regulatories that really expressed a lot of enthusiasm for study in biliary atresia. And we plan to be able to provide more information of our plan in the coming months. Pat, maybe you want to talk about how we think about recruitment.

Patrick Horn -- Chief Medical Officer

Yes, so the biliary atresia population is a larger population. And so, we would anticipate being able to enroll patients faster. I think ultimately, the length of this study will depend upon the number of sites we recruit and ultimately, how many patients we need in the final study design.

Ron Cooper -- President and Chief Executive Officer

Yeah, I think the other thing in that study as well, Katie will also dependent upon birth. Right. So, because in this case, these are patients that are newborns that undergo the Kasai procedure. So we feel pretty confident on our ability to (inaudible) start of the study. We look forward to providing you a little bit more detail as we finalize our discussion sooner with the regulators. Thanks, Katie.

Katie An -- ROTH Capital Partners -- Analyst

Okay. Yeah, just a couple of -- one more follow-up, how many Kasai procedures are performed annually and what percent of patients are not responders and what do you guys think will be the percentage of bile acid reduction needed to improve liver outcome? Will it be similar to what you found in the NAPPED Consortium with PFIC patients?

Patrick Horn -- Chief Medical Officer

Yeah. So, this is Pat again. So we're looking in feasible -- at the feasibility and we're looking at kind of the projected numbers. We would anticipate that in the United States, one number we have is, if you look at the Children's network, which is a consortium of 14 of the leading hepatology centers in the United States. They have on average 50 biliary atresia patients and Kasai patients per year. So, we're trying to expand that into the overall population, and what that number will be then? It also means that Europe has about the same prevalence there. So that was the first question. What was the other part of the question?

Katie An -- ROTH Capital Partners -- Analyst

The percentage of bile acid reduction needed to improve liver outcome?

Patrick Horn -- Chief Medical Officer

So again, that will be something we have to determine yet. We would anticipate, obviously, with our mechanism of action being able to show a significant reduction in serum bile acids. What degree will be required remains to be seen.

Katie An -- ROTH Capital Partners -- Analyst

Okay. Thank you guys so much.

Ron Cooper -- President and Chief Executive Officer

Thanks, Katie.

Operator

Thank you. Our next question comes from the line of Ed Arce with HC Wainwright. Please proceed with your question.

Ed Arce -- H.C. Wainwright & Co -- Analyst

Hi guys. Good morning and thanks for taking my questions and congrats on all the recent progress.

Ron Cooper -- President and Chief Executive Officer

Thanks, Ed.

Ed Arce -- H.C. Wainwright & Co -- Analyst

Some of my questions have already been asked, but I have a couple of others. First, now as you look toward a pivotal readout, perhaps as early as the end of this year. I know you -- Ron had touched upon some preparations early on that have begun in terms of commercial readiness for PFIC and so, perhaps you could expand a little bit about what you're doing, -- what you're planning throughout this year to prepare for that. And then secondarily, you had mentioned also pretty excited about the potential for some preclinical compounds in NASH. And so, I would imagine you're keeping some of that pretty close to the vest for now, but what could you share with us in terms of your potential differences with elobixibat and how you see it, particularly in terms of combinations? Of course, everyone is thinking about that in terms of combination strategies for NASH today. Thank you so much.

Ron Cooper -- President and Chief Executive Officer

Great. Thanks for the questions, Ed. I think that we're starting to pivot the organization now. We only went from an organization that was discovery organization to development organization. We're becoming a parent (ph) commercial organization. And I think one of the things that I made in my prepared comments was that, I think some of the people we brought into the organization are those that have significant experience in commercializing products around the world successfully. So our understanding of what's needed to successfully launch products, I think, is different from others significantly. You might recall that Simon spent many years with both Lilly and GSK. He was the CFO of the Global Vet Medicines (ph) business, so had a huge geographic approach in multiple therapeutic areas. Pat has spent years at Abbott, and I spent many years at Bristol-Myers Squibb in multiple commercial roles around the world.

So we're trying to do is, first of all, pivot the organization, hire appropriate individuals. And then right now, our budget calls for making sure that we invest in things that are pre-commercial activities that ensure that they are not rate-limiting things to a successful launch. So the types of things that we're involved in is making sure we're banging through things like brand strategy, commercialization plan. We're starting to be more active on the advocacy front, more active on the market access front, thinking through patient support, deepening our market research and understanding both the market and the need from our key stakeholders. So I think we're pretty excited about that and then underneath all of that work as well is the work to ensure that we file the product appropriate, so the regulatory work and the manufacturing work.

So as I said, I think it's going to be a big year from a pre-commercialization perspective. And then, I'm pretty excited about our preclinical compounds. The expertise in our organization extends multiple decades in bile acids. We've talked before that elobixibat is a validation of that bile acid platform with A4250 in a Phase 3 study and I think that this is really the strength of the platform and we're excited about the new agents. These are new chemical entities. These have a different mechanism of action. They will have new IP associated with them. And as we've indicated, they are products that build off of our understanding of bile acid modulation. And we're hopeful that they will be an important part of our overall product portfolio and as the year progresses, we'd hope to give you more color on that, Ed.

Unidentified Participant -- -- Analyst

All right. Thank you, guys.

Operator

Thank you. Our next question comes from the line of Ritu Baral with Cowen and Company. Please proceed with your question.

Ritu Baral -- Cowen and Company -- Analyst

Good morning, everyone. Thanks for taking my questions. Did I hear or interpret your -- one of your comments about the preclinical NASH drugs correctly that you're evaluating potential partnership strategies for one of them or for some of them. Could you go into that a little bit, how you think about the candidates for partnership versus moving it forward yourself and also your strategy on timing of any potential partnership and value creation as you think about this?

Ron Cooper -- President and Chief Executive Officer

Yes. And good morning, Ritu. Thanks very much for the question. I think, just to back a bit, it starts with our overall corporate strategy. So our intent is to build a pediatric liver company on the back of A4250. We plan to commercialize in the US and in Europe and use partners for the rest of the world. That's where we're going to build the Company around and that's where we have the expertise and I believe that we're credible in our ability to do that. We've always been pretty clear though that we'd like to provide to our investors a stream of non-dilutive capital, demonstrates our ability to do that, having signed a pretty good -- a really good agreement with our partners, EA Pharma, Eisai/Ajinomoto pharmas in Japan for elobixibat.

So our intent for those products that are outside of our footprint in pediatric cholestatic liver diseases, we will seek partnerships with them. And I think the timing part of that, I think, is pretty much dependent upon when we have data and who -- but I think what's exciting about our compounds goes back to what I said in my comments earlier that these agents in NASH, what's beautiful about them is, currently, elobixibat, it's a once-a-daily product, right, very low systemic exposure as a result it co-travels quite nicely with other potential NASH products and co-travels quite nicely with other cardiovascular risk products, right. And so, we'd anticipate something similar for our preclinical compounds as well.

So, just to summarize, strategy of the organization, build a pediatric cholestatic liver disease company on the back of A4250, generate non-dilutive capital for the organization through some of our other products through good partnerships.

Ritu Baral -- Cowen and Company -- Analyst

In NASH, Ron, do you necessarily think that optimal value creation can be reached before biopsy-generated data?

Ron Cooper -- President and Chief Executive Officer

I think, Ritu, it's really dependent upon the partner and dependent upon the assets. So we continue to work hard in generating data. We have had some nice dialog with some potential partners, so ongoing -- ongoing discussions, I think the number one thing is we're going to generate some data and then, we'll kind of see where that takes us.

Ritu Baral -- Cowen and Company -- Analyst

Got it. I've got a follow-up on the PEDFIC trial conduct. Do you feel that you guys have reached your final number of sites? How is enrollment in the -- in the sites that are up and going meeting your expectations?

Ron Cooper -- President and Chief Executive Officer

Yeah. So we've guided 35 to 45 sites. So we have reached our trial activation target. There still are a few sites that we are -- that we're still sort of finishing up. So there's a handful of those, right and that will help us. And in terms of enrollment, enrollment is progressing. We're pleased with what's occurred thus far. We just reiterate that, our plan for top line results by the end of this year, early next year.

Ritu Baral -- Cowen and Company -- Analyst

Got it. And then moving to biliary atresia and specifically that disease and it's -- I guess pharmacoeconomic burden and comparing that to PFIC, I'm just wondering, if you are commercializing one drug for two indications, how do you sort of view the burden of those two diseases and what it could mean for pricing down the line? And if you have any preliminary thoughts on how Alagille might impact that as well?

Ron Cooper -- President and Chief Executive Officer

Thanks for that question. I think the burden of this disease is significant right across the board. These children are suffering with pediatric cholestatic liver diseases. Most cases, they have elevated bile acids, they have terrible pleuritis, they don't sleep, they don't grow. Whether it's biliary atresia, PFIC or Alagille or PSC, there is no drug currently available. And I think that we've guided overall that patient population is between 30,000 to 40,000 in US -- in Europe. So while there's differences in these diseases, I think there's a lot of homogeneous situation in this case, right, seriousness, no other drug. And so, I think that, when we think about biliary atresia versus PFIC, both are very rare, both have a high burden on the family and the patient. So I think, we think about these with -- in terms -- as being comparable pricing as we go forward. Now, obviously, it's highly dependent upon the data that we generate and highly dependent upon the label that we have. I think our current thinking is that this is a more homogeneous group versus a heterogeneous group when it comes from a pricing perspective.

Ritu Baral -- Cowen and Company -- Analyst

Very helpful. And last question. Appreciate your patience. As you think about your NASH study and enrolling potentially both NAFLD and NASH patients, is there a specific mix of certain number of NASH patients that you're strategically thinking about making sure you have as you run that study?

Ron Cooper -- President and Chief Executive Officer

Yeah. So thanks, Ritu. I think that as we said before, we've given you a sense of what that study is going to look like. We think of it as a proof-of-concept study and think of it as something that's consistent with the FDA, has given from a guidance perspective. We haven't given all the details you have in that study. So as we finalize that and going in Q2, we will give you a little bit more color on that.

Ritu Baral -- Cowen and Company -- Analyst

Fair enough. Thanks for taking all the questions.

Ron Cooper -- President and Chief Executive Officer

Yeah. Thank you, Ritu.

Operator

Thank you. Our next question comes from the line of Liana Moussatos with Wedbush Securities. Please proceed with your question.

Liana Moussatos -- Wedbush Securities, Inc. -- Analyst

Thank you. You mentioned expanding A4250 in 2020 into other indications. What are your thoughts about indication selection for the next -- in 2020 with Alagille, PSC or others? And then following up on Katherine's question about breakthrough, why not apply for breakthrough since you could potentially get a voucher and monetize it?

Ron Cooper -- President and Chief Executive Officer

So I start with the second question. I think just to remind you, Liana, first of all, thank you for the questions, and good morning. Start off with -- our product is A4250 is already being given designation for priority review voucher from the FDA. So, we feel pretty good about that and we believe that's a value-creating opportunity over time. And frankly, the question about breakthrough, I don't think that's an unreasonable question. But right now, we're sort of focused in on finalizing the protocol with the agency and perhaps, we'll get it to that over time.

And then in terms of our selection of diseases, we continue to believe there is a really significant rationale for A4250 across multiple pediatric cholestatic liver diseases, but the approach that Pat and the rest of our team is taking is, these are ultra-orphan diseases. We want to make sure that we have trials that really capture the ability of A4250 in these patient populations and that takes a fair bit of work and trying to put together little bits of information, the competitive information where we are really comfortable that we can show the true effect of A4250. So we have ongoing discussions with key opinion leaders with patient advocates on trial designs in these other diseases. We went forward with biliary atresia first basically because it's a pretty significant opportunity. There are quite a few patients. As Pat had indicated earlier, the unmet need is super high.

And we've really enjoyed a wonderful dialog with the agencies about this because they actually recognize this is an unmet medical need as well. So I think that's where we are with the biliary atresia to comply the same criteria to the other potential diseases.

Liana Moussatos -- Wedbush Securities, Inc. -- Analyst

Thank you.

Operator

Thank you. Our next question comes from the line of Alan Carr with Needham & Company. Please proceed with your question.

Alan Carr -- Needham & Company -- Analyst

Hi, thanks for taking my questions, and couple of them. So I wonder you're talking a bit more about rationale for going straight to a Phase 3 as opposed to maybe running a larger Phase 2 in biliary atresia and then also it would point and do you start to think more aggressively about building a commercial organization, is that after Phase 3 or is that something you might do before? Thanks.

Ron Cooper -- President and Chief Executive Officer

Let me start with the second question on the commercial aspects of things. I think the way to think about our commercial planning is really around making sure nothing that is rate limiting to a successful launch is, it is not there, like it has to be there, right. I got, it's too many double negatives in that one. So it's certainly -- so our focus really is in preclinical activities. What we don't want to find ourselves is we're getting toward launch that we're missing a key component. So, that's what's being done now. Obviously, as we get closer to the data readout and when we put the card from the data readout perspective and finally, A4250 for regulatory approval, then we'll start accelerating our efforts. So I think just to summarize, the way to think about our commercial operations right now are very careful planning, deployment of careful resources making sure that rate-limiting issues are out of the picture such as things like as INN name and brand name not -- it should not be an issue for us. Those are things we're focused in on and we will accelerate as the data presents itself as we get into the regulatory pathway. Pat, maybe you take the first question.

Patrick Horn -- Chief Medical Officer

Yeah, so in terms of clinical development programs in rare diseases, it's always a difficult decision in how to proceed. So we're looking at biliary atresia. There are some strong scientific rationale why A4250 should be a benefit there. We have some data in our previous Phase 2 study. Like I said, that data is going to be presented and all of that support moving forward. So what we did was we designed a study to really demonstrate the efficacy of A4250 in biliary atresia.

And in the discussion with the regulatory agencies, we are designing a study that if successful, will not only proved the efficacy of A4250, but be considered a pivotal study that will support a registration application. So it's kind of the best of both worlds and partly, that's because in these rare diseases, the studies, even a relatively small study, are longer than in a different indication. So, I think we were very pleased that the regulatory agencies were willing to work with us and have this study, if it's positive, support a regulatory application.

Alan Carr -- Needham & Company -- Analyst

Great. Thanks for taking my questions.

Patrick Horn -- Chief Medical Officer

Thank you, Alan.

Operator

Thank you. Our next question comes from the line of Matt Kaplan with Ladenburg Thalmann. Please proceed with your question.

Matt Kaplan -- Ladenburg Thalmann & Co. -- Analyst

Hey guys, good morning. Couple of follow-up questions. I'm just digging a little bit more into BA pivotal program, can you give us more from kind of a 30,000-foot view, what you're thinking about in terms of the potential timeline for the program? Obviously, launching them the study in the second half of this year, but kind of help us understand kind of the timeline to data and potential NDA filing, if it's successful.

Ron Cooper -- President and Chief Executive Officer

Yeah. Good morning, Matt. Thanks very much for the question. I think this morning, we wanted to introduce to you that we're excited about expanding the potential from A4250 just from PFIC into other pediatric cholestatic liver diseases and particularly biliary atresia being that it is a major disease with huge unmet medical need. We're still in discussions with the regulatory authorities to finalize the protocol. So we've given guidance that we will start the study in the second half of this year. So I think it will be premature probably for me to make further comment. Let us finish up the discussion with the regulatory authorities and then we can probably give you more that over time.

Matt Kaplan -- Ladenburg Thalmann & Co. -- Analyst

Okay, fair enough. And then just also on the commercialization plans, could you give us a little bit of a sense in terms of the size of our commercial organization that you would need to go after the kind of your focus on the pediatric cholestatic liver disease area, what could it looked like?

Ron Cooper -- President and Chief Executive Officer

Sure. I think it starts with what is the customer base look like, right. So you think about in the US and Europe and (inaudible) let's say orders of magnitude, 100 key pediatric hepatologists and there is probably another 1,200 other individuals that might be prescribers as well. And think about it as most of these individuals are teaching centers, right. So there are tertiary centers. So we would not need a very significant field force to deploy against the large potential, right. So think of it, some are along the lines of both for Europe or the US somewhere between 15 to 20 to 25 individual field-deployed individuals and remember for -- remember that these are the same prescribers for all of these cholestatic liver diseases, right. So the pediatric hepatologist sees (ph) the PFIC patients, the biliary atresia patients, the eligible patients at (inaudible). So there's a lot of synergy over time.

Matt Kaplan -- Ladenburg Thalmann & Co. -- Analyst

Okay, very good. That's helpful. And last question just jumping back to NASH for a minute, just so kind of fully understand your strategy there with respect to elobixibat and your earlier-stage preclinical molecules, how do they fit into your NASH strategy, is it -- are you developing proof-of-concept data with elobixibat and putting that on the sidelines and then moving forward with your next-generation programs to or preclinical compounds to from a commercial point of view and development point of view or is it that you'll bring -- you potentially bring elobixibat to the next stage in Phase 3 with a partner?

Ron Cooper -- President and Chief Executive Officer

Yeah, that's great question, Matt. I think it starts with -- we are very confident about elobixibat in the NASH space given that it's a well-characterized compound, we have a significant level of exposures, over 1,500 exposures, we understand that compound. And so, as a result, the risk moving forward is somewhat less than the preclinical compounds, which we're still trying to understand. So there are some differences in that. I think really what's going to determine how we go forward with our partnership strategy is what the data we generate. And so, I think it's probably again premature to speculate as to exactly how we deal with both the compounds, but I think what our plan is let's generate the data, let's continue the dialog with potential partners and we'll see what works best from that perspective.

Matt Kaplan -- Ladenburg Thalmann & Co. -- Analyst

Okay, very good. And then just last question, elobixibat, how is it performing in EA's hands and now following approval in Japan?

Ron Cooper -- President and Chief Executive Officer

So, thanks for the question as well. Matt, I was in Japan with our partners late last year and I have to say the enthusiasm is fantastic. They've shared some market research on satisfaction levels. It was pretty impressive. The nice thing about elobixibat. It is the second of the new generation CIC, chronic idiopathic constipation products being launched in Japan and what's special about elobixibat is, it has a unique mechanism of action because it has both an ability to bring secretions into the colon, but a motility effect as well. And so, it seems that patients that had not responded to other medications have done pretty well. And as a result, I think our Japanese partners are pretty pleased with the uptake. That being said, it's still relatively early. They only have half year of sales, but they're around a half year of sales, they remain pretty optimistic, and we're pretty optimistic of the potential for elobixibat in Japan.

Matt Kaplan -- Ladenburg Thalmann & Co. -- Analyst

Great. Well, congrats on the progress. Thanks for taking the question.

Ron Cooper -- President and Chief Executive Officer

Yeah. Thanks very much, Matt.

Operator

Thank you. Our next question is a follow-up from Ed Arce with HC Wainwright. Please proceed with your question.

Ed Arce -- H.C. Wainwright & Co -- Analyst

Hi, Ron. Thanks for taking the follow-up. So I was just thinking through this group of rare pediatric cholestatic diseases. And as you've mentioned several times in the call, there's a lot of similarities given the high unmet medical need, the no-approved therapies for each of these and as you mentioned, the pricing at least at this point would seem to be pretty similar. And even from a commercial perspective, they are largely into these concentrated treatment centers that you just mentioned. So as I think about all of this and your announcement today where you're sort of unveiling your second indication for A4250, what exactly is the deciding factor for going for BA versus Alagille or PSC? Is it the faster enrollment that you mentioned? Is it the larger population? I noticed there's much -- significantly bigger than some of the other ones. What exactly led you with this one or more factors that led you to that decision? Thanks.

Ron Cooper -- President and Chief Executive Officer

Thanks, Ed, for the follow-up question. I think it's not one thing. It's multiple things that have led us to come to BA, some of them we've alluded to earlier, but there -- but the things that we think about is, first of all, unmet medical need, right. These are maybe that are born and very quickly afterlife have the Kasai procedure and have no treatment right now. So pretty significant unmet medical need. And as we talked to both key opinion leaders and we talked to the regulators, I think people want us to do something in this space. So I think that's the first thing.

The second thing is, as you pointed out, the size of the opportunity is pretty significant and if you look out what we're trying to do from a commercial perspective of the A4250, we're trying to build a pediatric cholestatic liver disease product. And so, I think it's not illogical to start with a very large GI indication for our next indication. And I think then the third part really is about the insight. Pat and his scientific team have spent a considerable amount of time with key opinion leaders and patient advocates to really understand this disease and put together small pieces of information to the point where I think we have pretty deep insight into what's happening with this disease and where A4250 could make an impact.

So I think those are the three reasons that why we're focused on biliary atresia with this -- at this point. That being said in the background, we continue to do work on other cholestatic liver diseases and our intent is to go forward with those over time, but we'll use the same criteria against those as well, unmet need, size of opportunity, and when we feel we have a good handle on what's going on from a size perspective, then we'll go forward. Thanks for the question, Ed.

Ed Arce -- H.C. Wainwright & Co -- Analyst

That's great.

Operator

Thank you. We have reached the end of our question-and-answer session. I would like to turn the call back over to Mr. Cooper for any closing remarks.

Ron Cooper -- President and Chief Executive Officer

Great. Thanks, Michelle. And thank you, everybody. So to summarize, I think what you -- I hope you saw that we had a strong 2018. We really delivered against our commitments. We expect the upcoming period to be truly transformational for Albireo with the continued progress with A4250 in PFIC, the beginning of a trial in biliary atresia, and we plan a major step forward with NASH. So again, thank you all for joining us today. Have a good day.

Operator

Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation and have a wonderful day.

Duration: 59 minutes

Call participants:

Paul Arndt -- Investor Relations

Ron Cooper -- President and Chief Executive Officer

Simon Harford -- Chief Financial Officer

Eun Yang -- Jefferies -- Analyst

Patrick Horn -- Chief Medical Officer

Katherine Xu -- William Blair & Company -- Analyst

Katie An -- ROTH Capital Partners -- Analyst

Ed Arce -- H.C. Wainwright & Co -- Analyst

Unidentified Participant -- -- Analyst

Ritu Baral -- Cowen and Company -- Analyst

Liana Moussatos -- Wedbush Securities, Inc. -- Analyst

Alan Carr -- Needham & Company -- Analyst

Matt Kaplan -- Ladenburg Thalmann & Co. -- Analyst

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