ALBO (2020 - Q1)

Greetings. Welcome to Albireo Pharma’s First Quarter 2020 Earnings Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions] Please note, this conference is being recorded. I will now turn the conference over to Paul Arndt with LifeSci Advisors. Please proceed. 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 first quarter ended March 31, 2020. 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 Investors section of our website, albireopharma.com, or on the SEC’s website. Any forward-looking statements represent our views as of today, Thursday, May 7, 2020, and should not be relied upon as representing our views as of any subsequent dates. We undertake no obligation to publicly update these statements. And now, I will turn the call over to Ron Cooper, Albireo’s President and CEO. Ron?

Thank you, Paul, and thanks to all of you who have joined us this morning. Virtually with me is Albireo’s Chief Medical Officer, Dr. Pat Horn; our Chief Commercial Officer, Pamela Stephenson; and our Chief Financial Officer, Simon Harford. We would like to do the call slightly differently today. I will provide you an update on our programs and then I’d like to go into a little deeper into the few key questions we have received. So first, the COVID-19 virus has obviously been on everyone’s mind. We have been monitoring the situation and following the recommendations of authorities. Our top priority is the health and safety of our employees and our commitment to the patients we are working to serve. Our organization is used to working virtually, and I would say, that the level of engagement and productivity has been remarkable. We continue to move the business forward problem solving as needed. We are fortunate that our two clinical trials that we were recruiting in PFIC and NASH are fully enrolled. Furthermore, there is sufficient inventory of clinical trial material to conduct these ongoing programs. Having raised $43 million early in the quarter, our cash position is strong and we have sufficient cash to get us into the second half of 2021. Though we continue to monitor the evolution of the COVID-19 crisis and the potential impact to our business, our fundamentals remain unchanged, and our prior guidance regarding clinical trials and cash runway remain unchanged. Let’s move into a brief update on our programs. Starting with odevixibat in PFIC, we are just months away from the topline readout of our pivotal trial data. The PEDFIC 1 trial is fully enrolled with 62 out of a planned 60 patients and we expect topline results in the middle of the year. With our current clinical trials already having achieved full enrollment and with adequate clinical drug supply, the COVID-19 risk is less significant than it would have been -- it would have been the case a couple of months ago. And more than three-quarters of the patients have completed the PEDFIC 1 trial, which is excellent progress versus our most recent update when we indicated that we are at more than two-thirds. While taking the utmost care to protect the safety of patients in the trial, we are confident in our ability to collect the remaining data. Why do we believe this? For the U.S. pruritus primary endpoint, we collect the data remotely with a smartphone-like tool and this is unchanged since the COVID-19 crisis began. For the EU serum bile acid primary endpoint, we expect to collect the remaining blood samples as many of the trial sites are children’s hospitals that are often away from the emergency rooms and given that odevixibat maybe a life-saving therapy this trial is often prioritized in these settings. For the few other sites where there may not -- where this may not be the case, we will implement the workarounds. Thus far, our team has been doing a really great job of addressing each situation on a case-by-case basis and we are now in the home stretch for data collection. We look forward to potential approval launch in the second half of 2021. In the meantime, we are laying the groundwork for our regulatory submissions and accelerating commercial launch preparations. We continue to make excellent progress in manufacturing and supply chain planning, select packaging partners for the U.S. and the EU, selected a partner to help us execute our patient support program, deepening our collaboration with physician and patient communities, executing on the CMC plan agreed with the FDA in the fall of 2018. We are using the planned commercial formulation in our Phase III studies and registration batches are on stability. So moving biliary atresia. The BOLD pivotal trial, biliary atresia and the use of Odevixibat in treating liver disease, is now underway. We are very pleased to announce that we have initiated the first clinical trial sites for this precedent setting pivotal trial. Regarding COVID-19, we are in the site activation stage and we plan to conduct most of the work remotely during this period. In the latter part of the year, we will be in hospitals for some site initiation visits, but would hope that access would have resumed by that time. BOLD is a double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of odevixibat compared with placebo in children with biliary atresia who have undergone a Kasai procedure. We plan to initiate 70 to 75 sites globally with an enrollment target of approximately 200 patients. Biliary atresia is the most common rare pediatric cholestatic liver disease and given the size of the patient population, it represents a significant commercial opportunity. This study will provide valuable data on disease modification that will be helpful to payers and other stakeholders, both the FDA and the EMA have granted odevixibat orphan designation in biliary atresia. Now quickly looking at Alagille syndrome. We received FDA feedback on our trial designand we will be sharing more details when the design is finalized. But this trial will be more like the PFIC pivotal trial than the biliary atresia pivotal trial. We continue to anticipate beginning the Alagille pivotal program by the end of the year and expect Alagille topline data to be available between the announcement of the PFIC and biliary atresia topline results. There are approximately 100 key pediatric hepatologists in both the U.S. and Europe. Many of these physicians will be participating in all three pivotal programs and these investigators will be very familiar with odevixibat and Albireo. So shifting to our efforts in NASH and adult liver disease. In Q1, we announced full enrollment of 47 out of a planned 46 patients in our first Phase II trial with elobixibat in NAFLD and NASH. We continue to expect topline data by the middle of this year. Most of the trial sites in the study are community-based clinics rather than hospitals, which should help with continuity during COVID-19. This proof-of-concept study is the first-ever study ever conducted in both NAFLD and NASH patients with an IBAT inhibitor and we are looking for the combination of positive trends in liver markers, cardiovascular risk factors and favorable GI tolerability. In addition, our Japanese partner, EA Pharma is sponsoring a study in Japan with 100 patients using a higher dose of elobixibat 10 milligrams. We expect the data by the end of this year or early next year. Now given the unmet need in NASH, we believe our approach could provide the potential for the optimal balance of liver and cardiovascular risk efficacy with excellent convenience and tolerability. These two studies will provide valuable insights to inform the next step to development for a potential partner. We are working at the complete IND enabling studies with our lead preclinical candidate that offers a novel mechanism of action. We believe that the combination of the two elobixibat Phase II studies and the emerging data on the lead preclinical candidate could create a compelling case for a potential NASH partner and create additional value for Albireo. Finally, given that we are rapidly approaching what is arguably the most critical data event in Albireo’s development as a public company. I’d like to take a moment to talk about why we are confident that PEDFIC 1 will yield results that will position odevixibat for regulatory and commercial success in the U.S. and EU. On the topic of the pruritus endpoint in PEDFIC 1, the confidence in our ability to demonstrate odevixibat’s impact on pruritus begins with our proprietary pruritus measurement tool named Precision. We developed this tool for our Phase III patient population and testing it rigorously with both patients and caregivers and in close consultation with the FDA. It uses a simple intuitive four-point rating scale like the Whitington scale used in our Phase II study that distinguishes each rating with pictures, words, numbers and colors. Now we recognize there may be some variability in measuring pruritus and have taken steps to mitigate this risk through powering assumptions trial sizing and trial length. We have powered the study conservatively, assuming a moderate treatment effect and a significant placebo response. Additionally, we have made the study larger and these elements lead to a powering assumption of well over 80%. As in many other studies, we expect that any placebo effect would tend to diminish over time and once again, in consultation with regulatory authorities, we lengthened the study to 24 weeks. While there have been other trials in other therapeutic areas that have failed to reach our primary endpoint using rating scales, we believe that the PEDFIC 1 trial is different due to the extreme severity of the disease. Patients tell us that pruritus is either better or not. We believe that the ease of use and expected high compliance with our measurement tool, as well as the trial design will underpin the results. As a result, we have a high degree of confidence in our potential to achieve success for the pruritus endpoint. Why do we believe that odevixibat fits our aspiration to develop best-in-class products? It is born, first, from the Albireo team’s multiple decades of experience in developing IBAT inhibitors. Among other things, this domain expertise is evidenced by our ability to lay claim to having developed the first-ever commercially available IBAT inhibitor with elobixibat. When we think of a potential best-in-class product, we must consider the product itself and the data supporting its safety and efficacy. Odevixibat is the most potent IBAT inhibitor ever synthesized and it is highly selective with minimal systemic exposure. Odevixibat can give -- be given once daily with choice of a convenient sprinkle formulation for infants that can be mixed with a puree or small capsules for older children. These capsules do not need to be refrigerated. The clinical data supports odevixibat are strong and consistent. In Phase I, odevixibat demonstrated consistent bile acid lowering and a low rate of diarrhea in the two lowest doses. In Phase II, those PFIC patients eligible for the Phase III study achieved a mean reduction in bile acids of over 70% in four weeks, which is similar to what’s observed in bile acid diversion surgeries. In addition to powerful bile acid reduction, odevixibat reduced pruritus and the improved sleep with no diarrhea observed during the four-week treatment period. The Phase III PEDFIC 1 trial is the first and the largest pivotal prospective, randomized, placebo-controlled trial in PFIC ever initiated. We will analyze approximately 40 patients on active drug versus 20 on placebo. Now looking at odevixibat as a commercial proposition, I want to revisit how we think about the PFIC, biliary atresia and Alagille patient populations. There’s no prevalence data or registries that exist to provide a clear view of population size. Therefore, we use secondary resources that are available and primary market research to estimate populations. We applied the published incidence rate and estimating median survival rates to regional populations. We look at emerging natural history sources like the NAPPED study as points of comparison along the way. We are focused mainly on the U.S. and EU, but are now broadening our analysis to other regions as part of our regional partnering discussions. There is a significant opportunity in the rest of the world and we believe that the total number of pediatric cholestatic patients in the world is in excess of our current estimate of 30,000 to 40,000. We have ongoing efforts to refine these numbers and we will share further details as the work is completed. So I hope you can see that we made significant progress with our development programs and remain on track to deliver both compelling new data and forward progress throughout the year. With that, it’s my pleasure to turn the call over to Simon for a financial update. Simon?

Thanks, Ron. Let me quickly summarize our financial results for Q1 2020. Revenues were $1.5 million for the first quarter of the year, compared to $0.6 million in the same period last year. The increase was primarily due to royalty revenue received from EA Pharma, which is passed on to HealthCare Royalty Partners as part of an agreement in December 2017 to monetize the royalty stream. Research and development expenses were $16.1 million for the first quarter of 2020, up from $8.3 million in the same period in 2019. The increase for the first quarter was primarily the result of program expenses for odevixibat and elobixibat, as well as personnel costs as we continue to increase our program activities and headcount. Our Q1 general and administrative expenses were $8.2 million, compared to $5.3 million for the same quarter in 2019. The increase was attributable to headcount and commercial readiness expenses in preparation for the -- into approval and launch of odevixibat in PFIC next year. Other operating expense was $6.8 million in the quarter versus $2.3 million last year, an increase of $4.5 million due to the impact of foreign exchange rates on intercompany loans as the Swedish kroner declined in value. There is no cash impact of these changes. Net loss for the first quarter was $31.5 million or a loss of $2.23 per share, compared to a net loss of $16.7 million or $1.39 loss per share in the first quarter of 2019. As of March 31, 2020, we had a balance of $150.5 million in cash and cash equivalents, compared to $131.8 million on December 31, 2019. The higher cash balance is a result of $43 million of net proceeds from our equity financing in early February. Excluding the impact of foreign exchange on intercompany loans, we continue to anticipate operating expenses for 2020 of approximately $100 million. We 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. Ron?

In summary, we all know that COVID-19 is having a sweeping impact across the industry and our society as a whole. Thus far, Albireo has persevered extremely well and we are in a fortunate position due to the status of our programs. As we eagerly await topline results from the odevixibat pivotal trial in PFIC, we are making excellent progress across a wide range of launch preparations, including manufacturing and supply chain, our patient support program, invaluable groundwork with physician, families and patient organizations, market access work and field force planning. At the same time, our biliary atresia pivotal trial is moving ahead and we are near finalization of our pivotal trial design in Alagille syndrome. Behind all of this, we continue to make progress in NASH with the elobixibat Phase II trial fully enrolled and IND enabling studies underway with our lead preclinical product candidate. We remain solidly optimistic the second half of 2020 is shaping up to be the culmination of many years of great science and hard work at Albireo. 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. Thank you for taking our questions and thank you for the update. Two questions, one is, as we are headed into the NASH data, we are expecting also to see serum bile assets. Can you may be shed some light into how valuable the transferability will be from bile acid reductions in that patient population to the upcoming PFIC study? And then the second question is, can you give us a little bit more color in regards to what elements of the Alagille pivotal study remains to be finalized and what are aspects of it that are known? And thank you again for taking our questions.

Hey. Good morning, Yasmeen and thanks very much for joining the call. I will make a few comments and then maybe, Pat, you can just add on if I have missed anything. I think that, we think about the NASH data and the impact of elobixibat in NASH, these are different compounds, elobixibat and odevixibat. And so in general, we are going to be expecting reductions in bile acids but I do not think that these are transferable because odevixibat is the most potent IBAT inhibitor ever synthesized and it’s very much specifically being used in NASH, I am sorry, in the pediatric cholestatic [Technical Difficulty] That’s why we have designed it for that. And then as it relates to the Alagille studies, it’s pretty much we are just putting some bows on things. We have got some nice feedback from the regulatory agencies, we have a direction of what we need to do and we will provide you some further details. Did I miss anything there, Pat?

Yeah. So I think the thing that ties these together is the reduction in bile acid and the fact that elevated bile acids cause ongoing hepatic damage and that’s kind of regardless of the indication. It’s more pronounced with odevixibat and the pediatric cholestatic diseases where the serum bile acids are much higher and we expect to see disease modification not only in PFIC but in biliary atresia and the other cholestatic diseases. In NASH, though, there’s also data that show that as you move from a normal liver to a fatty liver to NASH, you get this elevation in serum bile acids and preclinical work shows that reducing bile acids with IBAT inhibition actually decreases the fibrotic component of NASH. In addition, in the NASH, the other attributes of the IBAT inhibition, the increased glucose sensitivity, the reduction in cholesterol and the other things that will add to the impact in the NASH space. And with terms to the Alagille study, I think, Ron is exactly right. So we agreed on the major things, the endpoints, the timing, we are working on other things like exactly how we are going to look at the daily algorithm for drug abused liver injury, because this is a group of patients that start out with high levels. And so there are just a few little things we need to sort out. But we are very close to our final design.

Thank you, Pat.

Yeah.

Thank you, Ron.

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

Thank you for taking my questions. Simon, you mentioned $100 million in guidance for OpEx this year. What does Q2 look like versus Q1, are we going to see a steady increase or will Q2 be lower because I think they are being done virtually?

So we haven’t guided explicitly to what expenses will be quarter-by-quarter. But, obviously, $100 million roughly this year is roughly $25 million a quarter on an underlying basis when you exclude the any foreign exchange movements, which, as I mentioned, isn’t on cash-related. And I think it’s fair to say that as things like the PFIC trial start to wind down, we actually do have some typically slightly higher expenses at the end of the trial rather than in the middle. But that’s sort of offset by the fact to some extent that there’s less travel now to sites, et cetera, because stuff is being done remotely. And from a hiring perspective, I think, really what you should think about from a people point of view is we will be adding through the year and in a gated manner, so the way we are treating this is we are hiring those people who are absolute must have for the launch ahead of topline data readouts. So things like commercial and medical affairs leadership, but we are not adding until after topline data headcount for things like field forces and those who are not mission critical until we have done the topline data readout. But, overall, I would say, we are building over time, but I am not going to be exactly specific quarter-to-quarter.

Okay. Thank you.

Thanks, Liana.

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

Thank you. So for biliary atresia, Phase III initiations are right on track. So congrats. So looking at the clinicaltrials.gov, data is expected in May 2024 primary completion date. So question to you is, is there a kind of an interim look that you could look at to provide some data read between now and then?

Yeah. Thank you for the kind words Eun and I will let Pat answer the question. But I think we are pretty excited about getting this precedent setting study up and going, real credit to Pat and to his organization. Pat, do you want to add a little bit to that?

Yeah. So I think there were multiple conversations with the regulatory agencies about looking at kind of interim analysis and based on biomarkers. But the regulatory agencies are really set on the hard clinical endpoint of survival with native liver at two years and that really -- there wasn’t much of an appetite from a regulatory point of view to look at the biomarkers early. So there is not a formal interim analysis, there will be kind of ongoing review by the data monitoring committee but not as a formal interim analysis.

Thank you. And on PFIC, Ron, you mentioned that you guys are working on more of the commercial opportunity refining, given the lack of prevalence and registry data for this patient population. That said, based on your Phase III enrollment and screen processes. Do you have any kind of number of patients in mind that you could have identified by the time you launch the product?

Yeah. Thank you, Eun. That’s the type of work that we are -- that’s ongoing right now. I think we are pretty pleased with to being able to slightly over enroll the study. We actually had a lot of interest in the study. We consented over 100 children as well. We believe the demand is there. Pamela and her team right now are looking at that on a site-by-site basis, and as time goes on, we hope to provide you more details in that regard.

Thank you for taking the question.

Thanks, Eun.

Our next question is from Ed Arce with H.C. Wainwright and Company. Please proceed.

Hi. Good morning. Thanks for taking my question and congrats on the continued progress, especially the initiation of the BOLD study. So two questions for me. Starting with that study in BA, I know you mentioned there will be sort of transition throughout the year from mostly sort of remote or virtual activities over the next few months and then more on-site activities later in the year if access is allowed given the pandemic. I was just wondering if you could give us a bit more details as you are going through the year on what your plans are, not only what you have in plan, but contingencies if the access is less than you would hope for?

Yeah. Ed, first of all, good morning, and thanks very much for the question. I think from a COVID perspective, from a biliary atresia trial perspective as well, we are doing exactly what we would be doing, right? So a lot of the work right now is with the CROs, it’s IRBs, it’s contract, and Pat and his team have done a great job in doing that work, getting some sites up and going. We just anticipate though for some sites we need to get into the hospitals and it will -- we cannot do that remote. But as the year progresses, we anticipate getting into those hospitals and so that should be able to get us up and going with sites. Now obviously, if there are some reoccurrence of COVID and it makes it more difficult that we are not able to get into the hospitals that will make it challenging. But I think given what’s in the public domain now, every indication suggests that as we get into the late summer and the fall, we should be able to be into these institutes and that should be fine from a time line perspective.

Okay. Great. And the other question I had was, as you mentioned, there’s a lot of interest in better understanding the U.S. primary endpoint here, the pruritus endpoint for the PEDFIC study. And you shared a little bit of the specifics around your proprietary Precision tool, as you call it, and so I was just wondering if you could also share a bit more of the details of that tool with regard to, in particular the improvements that you made on the Whitington tool, also a four-point rating scale, as an analog as you were developing that? Thank you.

Yeah. Great. Thanks. Pat, why don’t you take that?

Sure. So as you point out, Ed, the Whitington scale is a zero to four-point scale and that’s what we use in, with zero being no itching and four being the most intense itching. And that was developed for patients with cholestatic liver disease early on in the early transplant by Peter Whitington at the University of Chicago at the time. So what we have done is we have taken that scale and put it into an electronic format, and so it fits into something that’s about the size of the phone, and the patient is asked to rank their pruritus either overnight or during the day. In addition to the numeric scale, as Ron points out, it’s also matched with kind of the smiley or frowny cases and these frowny faces are actually colored green being good to red being the fourth. And the development work, kind of the content validity and the questionnaires that went into both the patients and the caregivers, show that this really kind of improves the ability to differentiate on both the parent’s point of view in the -- or the observer reported outcome and in the patient reported outcome. So it really just takes the same concept that’s been used since the ‘80s really in terms of rating pruritus in pediatric cholestatic liver disease and puts it in a format that is much more patient and caregiver-friendly and enhances it with both the colors and the text as well.

And Ed, and just to add, we are blinded to the data in the PEDFIC 1 study. But we do know the rate of usage of the tool and we are actually very pleased at the high rate of usage and the amount of data that we have. So that would suggest that the tool is relatively easy to use for the parents.

Great. That’s excellent detail. That was it for me and congrats again on the continued progress especially in the current environment.

Yeah. Thanks very much, Ed.

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

Hi. Thanks for taking the questions. A couple of them, one around PFIC in terms of regulatory turning, are you planning in submitting an MAA and NDA at the same time or are these going to be staggered? And I am also curious about Alagille syndrome, we have had some conversations with regulators, is there some alignment between what the EMA and FDA want? Thanks.

So a couple of regulatory questions, Alan. Thanks very much. Thanks for joining us. Yeah. I think our plans are on the back of PEDFIC 1 data to hit the both authorities around the same time. They obviously can’t do them all on the exact same day. So around the same time, so that’s the overall intent and we would be looking to have a single protocol for Alagille pivotal program for both the U.S. and Europe.

And one other one that I have for you, you mentioned earlier that you are looking at rest of the world, outside Europe and U.S. in terms of market research. What are your -- what’s your strategy there in terms of outlicensing, are you having discussions already or is that something that comes when or after Phase III data available?

Yeah. So I think we are excited about the potential of odevixibat outside the U.S. and Europe. That’s where we focused. As we have done more research in that, we find there are a lot of countries, particularly from a genetic perspective where there are clusters for PFIC, which is very interesting for us and for biliary atresia as well. So we continue to have good dialogue with potential partners. Our intent to have -- is to have a small focused commercial organization in the U.S. and in Europe. In those countries where we don’t have as much expertise is to engage partners and those discussions are ongoing.

Thank you for the updates.

Thank you, Alan.

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

Okay. Hi. Good morning, guys. Thanks for taking the question. Just want to focus in on your commercial preparation for PEDFIC 1 after the readout. What are your thoughts on reimbursement, and specifically, what will the commercial team look like at launch?

Great. Thanks for the question, Matt. Pamela that’s right down in the middle for you. So why don’t you answer that question, please?

Sure. Hi, Matt. Thanks for the question. As we are thinking about utilization, our preparation is really in three areas. We are looking across market access reimbursement, as you suggested, also physician, KOL engagement and patient advocacy efforts. And towards reimbursement, we have done a lot of work. We are really excited about where we are. We have spoken with payers across Europe and in the U.S., and have begun to educate them about the disease. And when we do educate them about the disease, about PFIC, in particular, right away they see the severity, the high unmet need, the lack of available options and they acknowledge the relatively small budget impact. So we have gone forward in creating our cost effectiveness and other economic models, our value story and generating evidence that we will need that we know that payers want to see as we prepare the economic dossiers that they will need. So, overall, that’s where we are with access, as well as physicians and with patients.

Great. And then in terms of what the commercial team will look like at launch, what’s your vision there?

Well, we are really excited about this opportunity because, as has been mentioned, the small focused prescriber base is going to allow us to have a very concentrated effort. We envision 15 to 25 field deployed individuals in the U.S. and about the same amount in Europe, so a very focused and nimble team.

The nice thing, Matt, is we estimate that there are about 100 key pediatric hepatologists in the U.S. and Europe, right? So we don’t have to go to many, we actually -- many of those individuals will be involved in all three of our pivotal trials. We are developing strong relationships with them and to Pamela’s comment about access, remember that Pamela has a lot of experience that goes in access have the global pricing and access in her previous role and has launched for us [inaudible] products pretty successfully. So I think I feel pretty comfortable and are ready from an access perspective.

Great. Thanks, And then one question on the Alagille pivotal study. Can you give us a sense in terms of now with your thinking and the feedback and the FDA and the design? What your proposed time line is for that study, you mentioned that it would fall between the readout from the PEDFIC 1 study and the BOLD study in 2024. What’s -- what can you kind of fine-tune that a little bit for us?

Yeah. Give us a little time to put the bow on the study, and we will give you more details in that regard. But as we are thinking right now, we plan to be ready by the end of the year, and as I stated, this study will look more like the [Technical Difficulty] study in terms of the size and length. As a result, we would expect the results to come in between the topline results for the PEDFIC study and the BOLD study. We are all I think just reiterates our commitment to building odevixibat into a major drug for pediatric cholestatic liver disease and speaks to the large commercial opportunity.

Okay. Fair enough and look forward to added detail. Thanks for taking the question.

Yeah, Matt.

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

Hi, guys. Thanks for taking the question. Have you publicly stated what the last patient last visit in the PEDFIC 1 trial will be and can you walk us through any required safety follow-up? And how are you looking at cleaning up the data in the age of COVID, we have heard from other companies that you can’t visit the sites to sort of go through their records and hunt people down over weird chart comments or whatever. So how are you thinking about all of that and then I have a follow-up.

Yeah. So let me start with that, and then I will hand it over to Pat to perhaps give a little bit of color as well. From a guidance perspective, we announced in our Q4 earnings that the trial was fully enrolled. We previously announced that we were at 59 patients around the end of January and a few more to go. That kind of gives you a sense of what the last patient looks like. Pat, do you want to talk a little bit about safety follow-up and how we are thinking about collecting the data?

Yeah. So, yeah, this is Pat. So as you recall, the patients in the PEDFIC 1 study are eligible to enroll into the PEDFIC 2 study. So once they are done with the PEDFIC 1 study, there really is no further follow-up. They go directly into the study. So last patient, last visit is just that there’s not an extended safety follow-up period in PEDFIC 1. I think in terms of getting on site and monitoring remotely, I think, that’s something we are very actively pursuing and I think the regulatory agencies have come out with a number of guidance, actually surprisingly quickly to help with that. So there are ways to do remote monitoring. Some sites have the ability to redact the patient information and send that out or actually one of the things that people are exploring now is actually having, for example, a webcam at the site where someone from the site actually shows the monitors, the source data and the monitor can actually source data verify, looking at the real source data but looking at it remotely. So, obviously, all of these things are under consideration, all of these are being actively pursued and put in place. Our hope is that we will be able to get people on site because that obviously is what everyone’s used to and that will be the quickest. But there are workarounds and we are actually exploring all of those. I think in terms of getting hold of people. I think with -- kind of with teams, with all the different remote, it’s very easy to access the people in the -- at the investigative sites and interact with them. And there’s the talk about how busy the hospitals are and that’s exactly true. But that’s only true in certain parts of the hospital. So, for example, the research staff at a number of hospitals actually have relatively more time now to work on active studies, because there are -- some of the observational studies have been discontinued, whereas the interventional studies especially the Phase III studies, are allowed to go on. So we have found that helpful actually, not only in PEDFIC and interaction there but even in some of the start-up with biliary atresia that the sites have more time to dedicate that. So, again, I think, like everybody else, we are learning as we go but we see potential workarounds for all of these.

Yeah. I think just to add, Ritu, and thanks, Pat. We are in pretty good shape. So remember that we have more than 3/4 of the patient through the PEDFIC 1 study and so we have collected a fair chunk of data and have done some good chunk of data cleaning already. As Pat says, there’s work to be done, but I think, we are planning on some reasonable workarounds and we are hopeful of the ability to get to the sites as well in a reasonable timeframe.

That’s great to hear. I mean I can barely get grocery delivery here in New York. So good to see you guys are on top of it. What about PEDFIC 2 data release at the same time as PEDFIC 1 topline, will we get anything?

Pat, why don’t you address that?

So the answer to your question, we haven’t had that internal discussion yet. The plan is we will do an interim cut of the PEDFIC 2 data and that will be included as part of the regulatory submission when we do it, so we will analyze the interim cut and include that. We haven’t -- to answer your question directly, we haven’t had any kind of internal discussion about when and if we would do a press release on that data or whether we would save it for a scientific meeting or what we would do with that. It clearly doesn’t have the -- I mean, it’s supportive data. It is important but it doesn’t have the same importance level as the PEDFIC 1 study.

Got it. And last question, why is EA Pharma doing a higher NASH show? Why are they more interested and why didn’t you elect to do it as part of the Phase II?

Well, thank you for that question, Ritu. I think it’s just about our overall strategy learn as much as possible and to share costs, right? So I think what we are looking for in this NAFLD, NASH space is a signal of some sort and what we are looking to do is as how and improvement to liver diseases, cardiovascular risk and the GI tolerability part of it. And you do know that with these IBAT inhibitors that you go to higher doses, by definition, you are going to induce diarrhea. So we believe by pulling up resource with EA Pharma and us being focused on a 5-milligram arm versus placebo, whereas their folks in the 10-milligram, 10 milligram with cholestyramine, cholestyramine on its own and placebo, the totality of that data will give us a signal and that will give us sufficient information to inform the next stage development.

Is there a study being run in Japan?

It is being run in Japan.

Got it. Okay. Great. Thanks for taking the questions.

Thank you, Ritu.

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

Thank you for taking the question and congratulations to your team as everyone continues to manage through a difficult environment. For Alagille syndrome, you mentioned the trial that you are discussing with the agency would be more similar to PEDFIC 1 and BOLD? I assume that’s a comment based more on endpoint and design and can you just maybe discuss why you would not use a randomized withdrawal type of trial design similar to what some others have been successful with?

Yeah. That -- thank you, Tim. Thanks for joining us. That comment is more around to give you a sense more about size and length more than anything else. As you know, the biliary atresia study is larger, it’s 200 patients, right, and it will be longer, it’s a two-year period. So take our comments to help you sort of gate sort of the size and length of the study, more like the PEDFIC 1 study, and as I said, we will provide you more details once we put a bow on this.

Understood. Thank you for that. And following up on PEDFIC 1 and the Precision to your commentary, we all kind of understand pruritus in this indication is very severe. But -- and now that you fully randomized the trial and if the force [ph] are completed, can you just give us a sense of the baseline pruritus scores with Precision in patients that entered the trial, were they all three and four, were there any two?

Yeah. So thanks for the question. I think we are not prepared to give the details on the baseline is yet to suffice to say the intra criteria says you have the baseline of two or greater.

Okay. Understood. And can you just maybe update us as well on the rollover from PEDFIC 1 into PEDFIC 2, I think, last quarter, you mentioned those almost all the patients. Does that continue to be the case?

We have not provided any guidance in regards to the number of patients that have rolled over. As we had -- all we have said is that PEDFIC 2 is an attractive study and that we now have patients that have been on drug for well over the year.

Okay. Thank you very much and congratulations again.

Thanks for joining us, Tim.

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

All right. Thank you, Operator, and thank you everybody for tuning in to today’s conference. I think you find our conference an exciting and pivotal time. I think we all know that COVID-19 is on everyone’s mind and it does make things more difficult. But regardless of the challenges of COVID-19 we as a company are on track. We expect to deliver data for PFIC in the PEDFIC 1 study and data for elobixibat in the NASH study mid this year. I am also very proud of our organization in that we are building odevixibat into a pediatric cholestatic liver disease drug by getting the BOLD study up and going. We also have an organization that is growing and we have the financial resources to accomplish all of our objectives. So look forward to a strong back half of 2020. Thanks again for joining us.

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