Albireo Pharma, Inc.
Q3 2018 Earnings Call Transcript

Published: 13 Nov 2018

[Operator:]

Greetings, and welcome to Albireo's Third Quarter 2018 Earnings Call. At this time, all participants will be in listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions]. As a reminder, this conference is being recorded. I’d now like to turn the conference over to Paul Arndt, Managing Director LifeSci Advisors. Please go ahead.

[Paul Arndt:]

Thank you, Rob, and good morning, everyone. Thank you for joining today's call during which management will provide an update on Albireo's performance in the third quarter of 2018. Earlier today, Albireo issued a press release highlighting recent business accomplishments and noting its financial results for the third quarter and nine-month period ended September 30, 2018. The press release is accessible via the company's Web site at www.albireopharma.com. Before proceeding, let me mention that comments made 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 prevalent 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 make to 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 any undue reliance on any forward-looking statement. Also, any forward-looking statement that is made speaks only as of today, Tuesday, November 13, 2018, 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:]

Great. Thank you, Paul, and thank you everybody for joining today's conference call. We’re speaking to you today from San Francisco where we’ve just attended a very successful AASLD The Liver Meeting. With me today is Dr. Patrick Horn, Albireo's Chief Medical Officer; and Simon Harford, our Chief Financial Officer. The third quarter was characterized by progress on a number of fronts, including advancing development of our lead asset A4250 for progressive familial intrahepatic cholestasis or PFIC, continuing to present data that improve our understanding of PFIC and cholestatic liver diseases, achieving key regulatory milestones for A4250 and building our company towards commercialization. So now I’ll go into greater detail in each of these areas. So first off, we’re continuing to advance A4250 in PFIC by activating sites and enrolling patients in PEDFIC1, our Phase 3 clinical trial. To bring you up to speed on the A4250 story, I’ll share a quick summary. The Phase 3 program includes a single randomized, double-blind, placebo-controlled multi-centered 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 FDA evaluation and a key secondary endpoint for the EMA is a change in pruritus. The primary endpoint for the EMA and key secondary endpoint for the FDA is a serum bile acid responder rate. Now at the end of October, 26 sites have been activated in 11 countries including the first site in the United States. We believe we will have our target number of 35 to 45 sites activated by year end. Our team continues to expect top line data to be available end of 2019 or early 2020, which puts us close to being within 12 months of this very important result. So that’s the pivotal trial. So as I said, we just came off a very successful AASLD, American Association for the Study of Liver Diseases, meeting as well as an excellent NASPGHAN, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition meeting. The data from these meetings are really contributing to our understanding of PFIC and other cholestatic diseases, the role bile acids play in these diseases and the role of A4250 as potential therapy. This week at the AASLD, there were highly informative data presented on the natural history of PFIC from the NAPPED or Natural course and Prognosis of PFIC and Effect of biliary Diversion consortium. There are also interesting data from the Childhood Liver Disease Research Network or as you may know the ChiLDReN network on a prognostic value of serum bile acids in biliary atresia. The NAPPED consortium is an independent international group of treatment centers in Europe, North America, Asia and Australia investigating a natural history of PFIC. They create the largest database of clinical information on industrial PFIC 1 and PFIC 2 patients. The database is approximately 500 patients in total and they’ve conducted a meta-analysis to characterize the natural course of the disease. Now there were a number of important learnings from the 204 genotype PFIC 2 patients such as one-third of the patients reached adulthood with their native liver. In patients who underwent biliary diversion surgery, native liver survival was significantly higher with a p-value of 0.007. Reduction of bile acids below 100 micromoles per liter was associated with increased native live survival. So very interesting outcomes. Now as for PFIC 1, the database is much smaller with 46 genotype patients and there’s greater diversity in this population as the FIC1 gene was also found in other tissues such as the pancreas and intestines. Intrahepatic cholestasis is the predominant manifestation but extrahepatic manifestation may also occur in these patients. Native liver survival was higher than in PFIC 2 overall. Improved native liver survival with diversion was not seen we believe due to the small sample size. The investigators hope to have more PFIC 1 patients enrolling in the coming months. So why are these NAPPED data important for A4250 and what are the implications for us. The NAPPED data served two important functions for A4250. They will augment Albireo’s planned regulatory filing following completion of our Phase 3 trial and further demonstrate the value of serum bile acid reduction that it can provide towards better outcomes for patients with cholestatic liver diseases. Now in our discussions with regulatory authorities, they’ve indicated to us that the totality of the data of our submission will be important for approval. That means that we’re going to utilize data from the PFIC 1 24-week double-blind study, also data available at the time of the filing from the PFIC 2 18-month open label study and the natural history data from the NAPPED study. Now for prescribers and payers, we hope to demonstrate both significant positive impacts on symptom and disease modification to establish value for A4250 for better patient outcome. PFIC 1 is designed to show improvement in pruritus, weak growth and quality of life. PFIC 2 will provide long-term outcomes data and NAPPED will provide historical data which the PFIC 1 and PFIC 2 data can be benchmarked. The NAPPED data demonstrated that surgical biliary diversion not only relieves the pruritus associated with PFIC, but gives a highly effective treatment option for children with PFIC 2 and that bile acid decreases are associated with increased native liver survival. The data further underscores the high unmet need for the development of oral drugs, like A4250, and that have been shown in studies to produce production in bile acids similar to biliary diversion surgery. To also add, AASLD data presented by the ChiLDReN network from their Steroids in Biliary Atresia Randomized Trial demonstrated the prognostic value of serum bile acids after Kasai procedure in biliary atresia. Now the investigators concluded that lower serum bile acids measure six months after successful Kasai correlated with less liver injury and portal hypertension at two years, as well as possibly less future need for liver transplant. The data further highlight the important role of elevated bile acids in pediatric liver disease and the potential for A4250 to be an effective therapy option in these patients. So in addition, the interesting data AASLD presented two different datasets at the NASPGHAN 2018 Annual Meeting. First, we show data from a qualitative study of symptoms of pediatric cholestatic liver diseases that may significantly reduced quality of life. Pruritus was reported to be the most frequent, most disturbing symptom in 77% of respondents reported pruritus-related sleep disturbance. In addition, we presented research from a systematic literature review of partial external biliary diversion surgery or PEBD in patients with PFIC. This found that changes in certain bile acids and bilirubin appear to be useful as biomarkers in predicting both early response such as relief of pruritus and long-term outcome such as the need for liver transplant following PEBD surgery. Pruritus and serum bile acids are both important endpoints in the PFIC 1 A4250 Phase 3 drug. So now onto the regulatory front, I’m pleased to share that A4250 has received additional special designation that the FDA has granted Fast Track designation for A4250 in the treatment of pruritus associated with PFIC. Fast Track designation allows for expedited review and approval of medicines for serious and life-threatening diseases. This designation really does reinforce the potential for A4250 to address a critical unmet medical need in the treatment of PFIC. You may recall that A4250 has also received several other special regulatory designations for the development of PFIC. The FDA has granted A4250 Orphan Drug designation and a Rare Pediatric Disease designation with eligibility to apply for Priority Review Voucher. The European Medicines Agency has granted A4250 Orphan Drug designation as well as access to the PRIority MEdicines, PRIME program, and its Paediatric Committee has agreed to Albireo's A4250 PIP, or Pediatric Investigation Plan. Now beyond PFIC, A4250 also received Orphan Drug designation from the FDA for the treatment of Alagille syndrome. It’s also a rare and life-threatening disease that affects the liver with no approved pharmacologic treatment. Orphan Drug designation provides seven years of market exclusivity for the product upon regulatory approval. This is another noteworthy achievement for us. While we’re highly focused on our Phase 3 program for PFIC, we’re also turning our attention this year towards advancing our development program in other rare cholestatic liver diseases. So lastly, we continue to make excellent progress preparing A4250 for regulatory submission and eventual commercialization. For regulatory submission, manufacturing readiness is progressing as planned and we’re pleased to announce that we’ve agreed with the FDA on our chemistry, manufacturing and controls or CMC plan for registration. In addition, the long-term preclinical tox studies are on track. We were also notified that odevixibat has been selected as the proposed International Nonproprietary Name for A4250 and that’s now in a four-month objection period. And A4250 has received conditional approval from the FDA for its proposed proprietary brand name which we will announce closer to launch. So now on the commercial front, we continue to deepen our understanding of the market opportunity, refine our population estimate and execute on our launch planning. We believe there are about 30,000 to 40,000 patients in the U.S. and Europe with rare cholestatic liver diseases that are of primary interest to us as additional targets. Within that population we estimate about 8 to 10 people would be PFIC and BRIC. I think these estimates are strong given the lack of published data. Currently, we’re studying several diseases in both the clinical and commercial standpoint to determine which represents the best near-term opportunity, and as a result we expect to advance development in a second rare cholestatic liver disease in 2019. From a company perspective, we continue to evolve and grow the company in key areas, we prepare for commercialization. As we announced last month, we strengthened our leadership team with the addition of Simon Harford as our new Chief Financial Officer. I really want to welcome Simon to the Albireo team. Simon has an absolutely outstanding background having held leadership roles in healthcare at PAREXEL, GlaxoSmithKline and Eli Lilly. And his experience is ideal for our needs at this stage of growth. At the same time, we’ve also strategically strengthened the organization in other areas, including preclinical, biostatistics and regulatory. So to summarize, we’ve advanced the development of our lead asset A4250 for PFIC, shared important scientific data that improves our understanding of PFIC in other cholestatic liver diseases, achieved key regulatory milestones for A4250 and begun to build our company towards commercialization. So, Simon, this is your first earnings call for Albireo and it’s with pleasure that I hand it over to you for a financial update. Simon.

[Simon Harford:]

Thank you, Ron. Let me quickly highlight our financial results for Q3 2018 and the nine months ended September 30, 2018, which we released earlier today. We closed the third quarter with a balance of 173.6 million in cash and cash equivalents compared to 53.2 million at December 31, 2017. Our cash balance reduced by 9.6 million during the quarter primarily as a result of our Phase 3 PFIC trial for A4250. Our revenue for the third quarter was $237,000 and $12.2 million for the nine months ended September 30, 2018. Revenue during Q3 was related to the estimated royalty revenue from EA Pharma for elobixibat. Year-to-date revenue was primarily driven by the milestone payment received earlier in the year from EA Pharma due to approval in Japan of elobixibat for chronic constipation in that market. Our research and development expenses for the third quarter were $9.7 million compared to $3.2 million in the third quarter of 2017. R&D expenses for the nine months ended September 30, 2018 were $22.2 million compared to $9 million in the same period of last year. R&D expenses in Q3 as well as year-to-date are primarily driven by costs associated with development of A4250 as we implement our strategy as well as preclinical work associated with NASH. Our G&A expenses for the third quarter of 2018 were $3.9 million compared to $3.7 million in the third quarter of last year. G&A expenses for the nine months ended September 30 were $12.2 million compared to $10.6 million in the same period 2017. While Q3 G&A expenses increased 4% year-on-year, the higher year-to-date G&A expenses were principally attributable to increases in professional fees and recruitment costs. Net loss for the third quarter ended September 30, 2018 was $14 million or $1.17 per share compared to a net loss of $6.5 million or $0.73 a share in the third quarter of 2017. Net loss for the nine months ended September 30 was $30.2 million or $2.60 per share compared to a net loss of $19.4 million also $2.60 a share in the same nine months period in 2017. Based on our current operating plans, we anticipate our total operating expenses including R&D and G&A expenses for 2018 to be at the high end of the range at $45 million to $50 million, driven primarily by expenses related to our Phase 3 PFIC clinical trial for A4250 as we implement our strategy. In terms of cash, we expect our current cash balance to be sufficient to meet our operating requirements into 2021 under our current plan. And with that, let me turn the call back over to Ron for some concluding remarks.

[Ron Cooper:]

All right. Good job, Simon, and thank you. So to summarize, we’re really pleased with our continued progress in the PFIC 1 Phase 3 trial in PFIC and the data emergence create a better understanding of this devastating disease. We look forward to top line data in late '19, early '20. We also continue to make good progress advancing A4250 in other cholestatic liver diseases, elobixibat and our preclinical assets in NASH and A3384 in bile acid malabsorption disease. These are all proceeding according to plan. We remain steadfast in our commitment to bring life-changing therapies to patients suffering with devastating liver and GI diseases and leveraging our bile acid modulation science. So with that, we’ll open the call to questions. Rob, could you open up the line please?

[Operator:]

Yes. Thank you. [Operator Instructions]. The first question today comes from the line of Eun Yang with Jefferies. Please proceed with your questions.

[Eun Yang:]

Thank you. So with the Phase 3 on the way, I’m sure there is increased awareness of the disease in the medical community. So by the time you have launched the drug, how many human eligible patients do you think you’d have identified by then?

[Ron Cooper:]

First of all, good morning. Thanks for joining us. I think what you’re asking us is by the time we launch the drug, how many patients we would have identified?

[Eun Yang:]

Yes.

[Ron Cooper:]

First of all, we’re here at the American Liver Meeting and to your point we spent a lot of time with key opinion leaders and prescribers and clearly since we have the PFIC 1 up and going, a lot of excitement in the community. The fact that there is actually an option other than surgery right now has really galvanized the growth. I think that when we think about the potential opportunity for A4250, we believe there’s somewhere between 30,000 to 40,000 individuals in the U.S. that would be eligible. As to when launch occurs, that’s a dynamic process, right, because at that time it depends when babies are being born and when they have this disease. But as we get closer to that time, we’ll start to be doing more rigorous market research so we’re ready to go.

[Eun Yang:]

And then since the trial is on track and data readout by end of next year or beginning of 2020, when do you expect to start your pre-commercialization activities? Thank you.

[Ron Cooper:]

Thank you, Eun. We’ve already started our activities now. So from a commercial perspective, we have a head of marketing and commercialization. We’re starting to build around that. We started to deploy field medical resources. We’ve begun planning, looking at overall brand plan, launch plan and as well market research. We’ve started to understand where the patients are, where the prescribers are in your deployment plan together. And we’re also working hard on the access front to make sure that we have a good access strategy for A4250 for both the U.S. and Europe. That’s been deployed already and that’s already part of our current guidance for expenses.

[Eun Yang:]

Thank you.

[Ron Cooper:]

Eun, I appreciate it.

[Operator:]

The next question is from the line of Katherine Xu with William Blair. Please proceed with your questions.

[Katherine Xu:]

Good morning. I just wonder, Ron, and perhaps you just provide some details on the responder criteria for the primary endpoint in Europe and then the secondary for the FDA? And then with regards to the current PFIC 2 data, how confident you are on that endpoint? And also secondarily, what is the safety database requirement? Can you remind us of that and how the Phase 3 study as well as NAPPED I guess sort of your thoughts with that?

[Ron Cooper:]

Yes. Thanks very much for the questions, Katherine. I’ll hand it over to Pat shortly but I’d remind everybody that our Phase 3 should be a study at least of 60 patients. The primary endpoint in the U.S. is pruritus; primary endpoint in Europe is serum bile acid responder definition. But it is the same study. We just looked at it kind of through two different doors. But, Pat, maybe you can talk a little more about the responder definition.

[Patrick Horn:]

Yes. Good morning. This is Pat Horn. So for the serum bile acid, the responder definition is a reduction of 70% from the baseline serum bile acids or reaching an absolute serum bile acid level of 70 or less. And based upon our Phase 2 study, that’s how we power this study. So we assumed – we were very conservative in our assumption of what we thought our active drug would do and very liberal in our assumption of what we thought the placebo patients would do and we’ve powered it that way. So we’re confident in that endpoint. With pruritus as well, it’s not a responder definition. It’s really a change in baseline compared to – of active compared to placebo. And again, we used the Phase 2 data when we did our power calculation. So again, we’re confident that we powered sufficiently. In terms of the safety database, this is an orphan product with an Orphan Disease designation and that really gives us a relief on the expected database from the FDA. So we have vetted our Phase 3 program both in study design and trial size with both the EMA and the FDA and both agencies have given us an indication that obviously if it’s successful, it will be sufficient to support a filing.

[Katherine Xu:]

And then I’m just wondering how that 70 micromoles, that number was decided? I guess like yesterday when we were looking at NAPPED, the line drawn was at 100 looking at the native liver survival. So just curious about how that 70 came about?

[Patrick Horn:]

So obviously the 70 was selective before the NAPPED data was available and we did – it was selected based on discussions with both the KOLs and the regulatory agencies.

[Ron Cooper:]

So, Katherine, it’s a bit of a balanced question. So as Pat had said, KOLs and regulatory agencies but also our Phase 2 data in some ways if you said the responder definition was a 95% reduction or something higher than that, that would really reduce the probability of a response in the placebo group. So we felt that in discussions with those key stakeholders, 70% really sets us up for success in both what’s going to occur in the placebo group and in the treatment group.

[Katherine Xu:]

Thank you.

[Ron Cooper:]

Thank you, Katherine.

[Operator:]

The next question comes from the line of Yasmeen Rahimi with ROTH Capital. Please proceed with your questions.

[Yasmeen Rahimi:]

Good morning team. Two quick questions. Question one is for Pat. Can you remind us again how bile acids are measured, not necessary how they actually work, but more around sort of their ability in regard to – and I know it is influenced by eating and fasting and how is that incorporated into the trial design? And then the second one is just maybe big picture as we all are here at The Liver Meeting, can you maybe give us two of the key takeaways that’s sort of changed your perspective on cholestatic diseases or in PFIC here at The Liver Meeting? Thank you.

[Patrick Horn:]

Okay. So serum bile acids do have some variability day-to-day and especially related to meals. But that variability is very small compared to the levels and the increases we’re looking at with these cholestatic liver diseases. So for a normal person, the serum bile acids are usually below 20. So they can vary anywhere from 15 to 18 or 19, but here we’re looking at children who start with levels of 200 or 300. So the variability introduced by mealtime is expected to be a very minor percentage. Plus in our study we are measuring fasted serum bile acids, so just to kind of get rid of that. I think for me kind of the most interesting thing that I’ve learned at The Liver Meeting, obviously there’s been a lot, is the whole kind of clinical thinking around the distinction between BRIC and PFIC and how people are now starting to think of that as the same disease with different presentations. When it was first described, BRIC and PFIC were kind of separated by the phenotype in the way they presented and they got to be distinct. And then with the genetic – the mutations are in the same gene. And then as they follow these people longitudinally, people who initially present with a BRIC phenotype almost always transition into a PFIC phenotype. So it really is now considered the same disease with different presentations.

[Ron Cooper:]

And I think for me, if you ask me, I would add that we’re committed with A4250 developed in multiple cholestatic liver diseases. Our plan is to launch in PFIC, BRIC area where we estimate somewhere between 8,000 to 10,000 patients. But actually seeing a lot of data which correlates to bile acids and pruritus across the different disease, and obviously the NAPPED data was excellent data but also data – biliary atresia, the discussion around primary sclerosing cholangitis where pruritus is really an issue further reinforces to me that bile acids do a lot in the body and do a lot in these diseases and that in fact a mechanism that controls bile acids and improves pruritus could have a lot of utility.

[Yasmeen Rahimi:]

Thank you so much and keep up the great work.

[Ron Cooper:]

Thank you, Yasmeen.

[Operator:]

The next question comes from the line of Liana Moussatos with Wedbush Securities. Please proceed with your questions.

[Liana Moussatos:]

Thank you for taking my question and congratulations on all your progress. How does Europe versus the U.S. differ in the percent of PFIC patients who get surgery and transplant?

[Patrick Horn:]

This is Pat again. So typically if you look at Western Europe and the United States, they’re very similar in terms of the people who get either biliary diversion or liver transplant. I think one of the interesting things too is now that NAPPED is also expanding, it will include multiple U.S. sites including most of the sites in the ChiLDReN network, so we’ll actually have that data. But the assumption is that if you look at Western Europe and the United States, they are similar. There are some places in the world, just some Middle Eastern countries where they don’t go into biliary diversion surgery. Instead they go directly into liver transplantation. So there are some pockets where it’s a little bit different. But in general in the geographies that Ron was talking about, the U.S. and Europe, it’s pretty similar.

[Liana Moussatos:]

Thank you.

[Ron Cooper:]

Thank you, Liana.

[Operator:]

The next question comes from the line of Ritu Baral with Cowen. Please proceed with your questions.

[Ritu Baral:]

Good morning, guys. Thanks for taking the questions. Now that we’re about a year away from Phase 3 data, can you frame how you are thinking about potential pricing? Yesterday obviously there were some very interesting data presented at your event on transplant burden as well as biliary diversion procedure rates. How do you look at transplant biliary diversion surgery and the value proposition for this trial?

[Ron Cooper:]

Great. Thanks very much for the question, Ritu, and thanks for joining us. I think for us in terms of pricing, pricing obviously will be driven by the quality of our data and what’s occurring in the market at that time. So we’ll make decisions closer to that timeframe. That being said, we’re very focused already in putting together a good access strategy overall and pricing is a component of that. And in that access strategy the things that you’re talking about modeling against burden of disease both from a quality of life perspective but also cost in the system of biliary diversion surgery, liver transplant, et cetera, will be part of that. So that’s work that we’re undertaking soon. We have done some pricing benchmark work already. And when we look at analogs of agents that have similar prevalence, diseases of similar severity, the range for those agents is around 300,000 to 500,000 a year. So that’s the book of work that we have, but we already focused on building a good access strategy so that we’re able to justify the value of A4250 in PFIC both for what it should be able to do for symptoms but as well for long-term disease modification.

[Ritu Baral:]

Got it. And a quick follow up, and I apologize if I missed this. It was part of the discussion yesterday. What actually drives the need for transplant in PFIC patients? Is it the HCC [ph] risk? Is it pruritus? Is it other liver events?

[Patrick Horn:]

So it’s interesting. PFIC is a little different than some of the others where in biliary atresia it’s really a form of liver failure and cirrhosis. But in PFIC, pruritus is the primary indication for the surgical intervention. So it is 100% the indication for biliary diversion. The reason they do biliary diversion is to relief the pruritus. And in – if you look at the same group of people that 50% of the patients gets their liver transplant when they still have a functioning liver and they get it to get rid of the pruritus. So I know sometimes when we talk about pruritus and itching as a symptom, people kind of say – they tend to discount that, the severity of it. But in this disease it leads to organ transplantation. It’s that severe. So that’s the numbers we have so far.

[Ritu Baral:]

Very helpful. Thanks for taking the questions.

[Ron Cooper:]

Thank you, Ritu.

[Operator:]

[Operator Instructions]. The next question is coming from the line of Alan Carr with Needham & Company. Please proceed with your questions.

[Alan Carr:]

Hi. Thanks for taking my questions. I wanted to come back to your plans for next years in terms of looking at other indications for 4250. Can you talk about I think the pluses and minuses of biliary atresia versus Alagille syndrome in terms of PFIC for 4250, and give meaning one way or the other at this point?

[Ron Cooper:]

Yes, Alan, thanks for joining us and thanks for the question. I think that we’ve guided that we will start a second indication next year. Part of being here at the American Liver Meeting is dialoging with key stakeholders and key opinion leaders to give us some feedback on these diseases. What we did with PFIC before we went ploughing ahead into PFIC 1 and PFIC 2, we spent a lot of time getting deep understanding of the disease to make sure we design a trial which we believe should produce positive results, but we’re trying to do the same with these other diseases. Pat, maybe you want to add a little bit to that.

[Patrick Horn:]

Yes. One of the convenient things for us in terms of these cholestatic liver diseases in children is that they’re cared for at the same centers and usually by the same investigators. So we’re well connected with our PFIC investigators now and we’re actively talking to them in terms of the different indications. I think from a mechanism of action point of view, I think that there’s – clearly we’re confident that we can have a positive impact in either of those diseases. One of the things for us to consider is what the study will look like, how easy it will be to design a study that will answer the questions we need to answer in a reasonable time for a reasonable cost in the different indications. So we’re looking at Alagille, we’re looking at biliary atresia, we’re looking at PSC, maybe PBC as potential expansion for the A4250.

[Alan Carr:]

So any of those – not just the pediatric, not just Alagille or biliary atresia, but the second indication that you might examine next year might be PSC or PBC looking into adults?

[Patrick Horn:]

We haven’t ruled that out. Again, as Ron said, we’re really focused on becoming a pediatric liver company. And so we’re focused primarily on biliary atresia or Alagille. There’s increasing evidence and from what we’ve learned this week out there in San Francisco is there certainly is a role for bile acid reduction and bile salt reduction in really any of the cholestatic liver diseases.

[Ron Cooper:]

Yes, and just clarify, Alan, in the PSC space we’d be thinking about the pediatric population there. So there’s a large number of individuals who have PSC that are children. That’s where our focus would be.

[Alan Carr:]

Okay. And then coming back to a comment – in the press release and your comment earlier, can you elaborate on where things are with CMC and the other preclinical tox work in terms of getting ready for NDA submission? Where do things stand in that process? Thanks.

[Patrick Horn:]

Yes, so this is Pat. So according to our plan and according to our anticipated data release and everything, the clinical data is on the critical path. CMC and toxicology should all be in place and completed by the time we get the clinical data. So it really is the clinical data that’s on critical path for that submission.

[Alan Carr:]

Okay, great. Thanks very much.

[Operator:]

Thank you. At this time, we’ve come to the end of our question-and-answer session. I’ll turn the call back to Ron Cooper for his closing remarks.

[Ron Cooper:]

Great. Thank you, Rob. So first of all, my thanks to those that have attended today’s call. I hope that you see that we’ve made tremendous progress in Albireo in this last quarter with A4250 advancing the PIFC 1 study in Phase 3 and looking carefully at other diseases. We continue to make good progress in the NASH space with elobixibat and our preclinical compounds and we continue to make progress with A3384. So we look forward to a strong close to the year. Thank you again.

[Operator:]

Thank you. This concludes today’s conference. You may disconnect your lines at this time. Thank you for your participation.