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Albireo Pharma, Inc. (ALBO)
Q2Â 2020 Earnings Call
Aug 7, 2020, 10:00 p.m. ET
Contents:
- Prepared Remarks
- Questions and Answers
- Call Participants
Prepared Remarks:
Operator
Greetings, ladies and gentlemen, and welcome to the Albireo Pharma's Second Quarter 2020 Earnings Conference Call. [Operator Instructions] It is now my pleasure to introduce your host, Paul Arndt of LifeSci Advisors. Thank you. You may begin.
Paul Arndt -- Investor Relations, LifeSci Advisors
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 second quarter ended June 30, 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 at www.albireopharma.com or on the SEC's website.
Any forward-looking statements represent our views as of today, Thursday, August 6, 2020, and should not be relied upon as representing our views as of any subsequent date. We undertake no obligation to publicly update these statements.
Now, it is my pleasure to turn the call over to Ron Cooper, Albireo's President and Chief Executive Officer. Ron?
Ron Cooper -- President and Chief Executive Officer
Great. Thank you, Paul, and thank you everybody for joining us this morning. With me today are Dr. Pat Horn, our Chief Medical Officer; Pamela Stephenson, our Chief Commercial Officer; and Simon Harford, our Chief Financial Officer.
So before I dive into an update on each of our programs, I wanted to give my heartfelt thanks to our hardworking and tremendous team here at Albireo, who have shown extraordinary commitment in moving each of our programs forward in the face of the COVID-19 global pandemic. Executing upon our strategy and keeping our programs on track is no small feat, even in the best of times.
Despite these challenges, we're pleased to report that last patient, last visit has been achieved for the odevixibat PFIC Phase 3 study and the elobixibat NASH Phase 2 study. The first patients have been enrolled in the odevixibat biliary atresia Phase 3 study, and we've come to agreement with the U.S. and European regulatory agencies on the design of a third odevixibat pivotal program in Alagille syndrome. We continue to execute and build upon our plans for commercialization as we prepare for top-line data. Following our top-line data announcements, we have a rich second half with key milestones to deliver on, which is possible because of our team's focus and quality of execution. With our strong balance sheet supporting our activities since the beginning of 2022, we feel optimistic about our position as a company.
Now for a closer look at each of our programs. In odevixibat for Progressive Familial Intrahepatic Cholestasis or PFIC, we were fortunate to have our Phase 3 trial fully enrolled when many of the COVID-19 restrictions were first put in place. We have now achieved last patient visit and expect top-line results in the coming weeks, aligned to our mid-2020 guidance. I'm really pleased that the data will include 62 out of a planned 60 enrolled patients, with no patients lost to follow-up due to COVID-19. We anticipate approval, issuance of a priority review voucher and commercial launch in the second half of 2021.
At Albireo, we continue to be fiercely dedicated to supporting patients, and we've committed to two important programs. First, we announced the launch of an Expanded Access Program, or EAP, for patients with PFIC in the U.S., Canada, Australia and Europe. Through our open-label PEDFIC 2 trial and now our EAP, we hope to increase access to odevixibat for all eligible patients to the greatest extent possible until odevixibat is commercially available. We're pleased to launch the program in these first countries, and we're working closely with local regulators in other countries to offer access and continue to provide hope for PFIC families worldwide.
Second, genetics testing. We are happy to be supporters of a free genetic testing program for PFIC and Alagille syndrome in patients in the U.S. with other industry partners. Both programs demonstrate a strong movement toward planned commercialization, but more importantly, our commitment to helping to provide support for patients who are in need.
We are confident that our PEDFIC 1 study has been carefully designed to incorporate FDA's input into the pruritus primary endpoint. We hosted a Key Opinion Leader event earlier this quarter to provide background on the design and implementation of clinical outcome assessments, including patient-reported and observer-reported outcomes in PEDFIC 1 and 2. Our precision measurement tools were designed using best practices for clinical outcome assessments in consultation with patients and caregivers, expert clinicians and the FDA, who provided considerable feedback through the development process to the design of the tools, including the interview study, draft items and response scales.
Based on the content validity work that we have done, we believe it will provide the quality measurements the FDA requires for an NDA submission of odevixibat for PFIC.
Now, plans for commercializations are in high gear. We've been very active on that front. Our supply chain and manufacturing plants are taking shape with packaging partners selected for the U.S. and the EU, and our patient support program partner is in place as well. Stability testing for our registration batches is well under way, and we are executing on the CMC plan with input received from the FDA in the fall of 2018.
As our commercialization plans begin to take shape, we're convinced today, more than ever, of the opportunity in PFIC. With a clear need for better treatment options for patients and an orphan drug designation offering market exclusivity, we believe that we are well positioned with our once-a-day oral dosing with minimal systemic exposure and a favorable safety profile. We'll have much more to say on this as we get closer to launch.
Moving on to our Phase 3 program with odevixibat in biliary atresia. We fulfilled our 1H '20 guidance and initiated our BOLD pivotal study last quarter. And we're delighted to have randomized the first patients in the trial despite the challenges of COVID-19. BOLD is the largest prospective intervention trial ever conducted in biliary atresia, expanding the development of odevixibat to a second rare cholestatic liver disease indication. This study is a double-blind, randomized, placebo-controlled study in approximately 200 infants designed to evaluate the efficacy and safety of odevixibat compared with placebo in children with biliary atresia, who have undergone a Kasai procedure.
The primary endpoint is the proportion of patients who are alive and have not undergone a liver transplant after two years of treatment. There are no approved pharmacological treatments for biliary atresia, which is the most common pediatric cholestatic liver disease and the leading indication for pediatric liver transplant. Clinical sites in the United States are currently active for patient enrollment. We have received orphan drug designation for biliary atresia in the U.S. and EU.
Rounding out our odevixibat program is Alagille syndrome, where we plan to initiate our third pivotal Phase 3 trial by the end of the year. We've come to agreement with the regulatory authorities in the U.S. and EU on the design of a protocol for a single study necessary for approval and have received orphan drug designations in both regions.
Let me now move to our pipeline products. It is estimated there are approximately 16 million people in the U.S. with NASH with a tenfold greater risk of liver-related mortality compared to the general population. This presents a massive market opportunity with no approved treatments. While many of the products in current development are struggling with the balance of efficacy and safety, it is our thesis that a non-systemic drug could have the right balance of efficacy and safety.
In our elobixibat program, we have two ongoing Phase 2 trials in NASH and NAFLD. For the U.S. elobixibat 5-milligram study, we completed last patient, last visit and are expecting top-line data in the coming weeks, fulfilling our mid-2020 guidance. This study will read out ahead of the odevixibat PEDFIC 1 Phase 3 study. We will have data on 43 of the 47 patients randomized as four were lost to follow-up, primarily due to COVID 19.
For the second elobixibat 10-milligram trial being conducted through our partner, EA Pharma in Japan, we reiterate our guidance and anticipate top-line data at the end of this year or early next year.
We're eagerly anticipating these important data as both studies are designed to provide a proof-of-concept of IBAT inhibitors in NASH/NAFLD. There are no biopsy endpoints in this study. For both studies, the primary endpoint is change in LDLC and important secondary endpoints such as reduction of liver fat measured by MRI-PDFF and reduction of liver enzymes. With this combination of parameters, we are looking for positive trends in lowering LDL, liver health, potentially with at least 10% to 20% improvement in liver fat and stabilization of liver enzymes and positive trends in cardiovascular disease factors with good GI tolerability, meaning a manageable rate of diarrhea. Our intent is to accumulate this data from both studies and seek a partnership for further development for elobixibat and potentially one of our emerging preclinical assets.
So in summary, we are very excited by the opportunities before us in each of our programs. We truly believe we are building something very special with our focus on progressing our late-stage programs and validating our pipeline with our best-in-class product assets and look forward to increased momentum in the second half of the year, starting with; odevixibat PFIC top-line data anticipated in the coming weeks; elobixibat data for one of our two Phase 2 trials in NASH anticipated ahead of the odevixibat data and data from our Japanese partners by the end of the year or early next year; BOLD Phase 3 trial, which will continue to enroll patients with biliary atresia; as well as a third Phase 3 trial in Alagille syndrome to be initiated by year-end; additional milestones for odevixibat into 2021, including completion of a study initiation for the biliary atresia trial and the anticipated approval and issuance of a priority review voucher and commercial launch in PFIC next year.
We are truly on the cusp of a very and exciting meaningful period for Albireo. We're certain of the potential and opportunity that lies ahead and really appreciate your support.
So with that, it's my pleasure now to turn the call over to Simon for a financial update. Simon?
Simon Harford -- Chief Financial Officer
Thank you, Ron. Revenue was $1.9 million for the quarter, year ended June 30, 2020, compared to $1.3 million in the same period last year. The increase was due primarily to royalty revenue received from EA Pharma in Japan, which is passed on to the HealthCare Royalty Partners as part of an agreement to monetize the royalty stream.
Research and development expenses were $18.4 million for the second quarter of 2020, up from $11 million for the same period last year. The increase for the quarter was primarily the result of program expenses for odevixibat, as well as people costs as we continue to build out indications for our lead asset and expand headcount.
General and administrative expenses were $8.5 million for the second quarter, compared to $5.5 million in the same period last year. The increase was primarily due to additional headcount as we continue to prepare for commercialization.
Net loss for the quarter ended June 30, 2020, was $20.6 million or a loss of $1.38 per share compared to a net loss of $16.6 million or a loss of $1.35 per share for the second quarter of 2019. As of June 30, 2020, we had a cash balance of $152 million. During the quarter, we restructured our royalty agreement with HealthCare Royalty Partners, netting an additional $15 million in non-dilutive capital.
Additionally, we established an $80 million debt facility with Hercules Capital, drawing down an initial amount of $10 million. As a result of these two financings, our cash runway now extends into the beginning of 2022.
With that, let me turn the call back over to Ron for closing remarks.
Ron Cooper -- President and Chief Executive Officer
Great. Thanks very much, Simon. We're moving forward toward our vision of becoming a fully integrated biotechnology company with an active commercial organization and a pipeline of therapies under development. We believe odevixibat has the potential to be a meaningful therapy in PFIC. We anticipate much more to come.
Thanks, everybody, for joining us. We're now pleased to open the call for questions. Operator?
Questions and Answers:
Operator
Thank you. [Operator Instructions] Our first question comes from the line of Yasmeen Rahimi with Piper Sandler.. Please proceed with your question.
Yasmeen Rahimi -- Piper Sandler -- Analyst
Hi, team. Thank you so much for the great update so we're very excited for all the results that we will be seeing soon. Two questions for you. The first question is in regards to NASH. Can you kindly share with us what data you hope to see to give you confidence or make the decision to go further in development in NASH? And then the second question is, have you had a chance to speak with the FDA on the potential use of bile acids as the regulatory endpoint? And thank you again for taking our questions.
Ron Cooper -- President and Chief Executive Officer
Good morning, Yasmeen. Thanks for joining us. I think we're very excited about the upcoming NASH data. Our program with elobixibat has always been designed as a proof-of-concept program, the study we're doing here in the U.S. and the study that our partners, EA Pharma, are doing. Our thesis for elobixibat in NASH is a thesis of where we believe we can do multiple things that can help with the disease. So what we're looking for is the combination, right, in improvement of liver health parameters, in reduction of cardiovascular risk parameters and doing that with good tolerability, namely, making sure there's a reasonable level of diarrhea. So that's what we're looking for between those two studies. And as we've talked to potential partners, they said they're looking for that type of information. So we're pleased to provide that data.
And then to your second question, as it relates to the regulatory question about using serum bile acids as an end point, we had very good discussions with both regulators in the U.S. and in Europe about the design of the PEDFIC study. Originally, we had proposed serum bile acids in both regions. And within Europe, they agreed that, that would be a good primary endpoint. In the U.S., their focus is much more on a clinical endpoint, and they wanted a pruritus endpoint. That being said, both regulatory agencies have communicated to us that the totality of data is very important for approval and that they are looking for all the parameters to be swimming in the right direction, and we hope to provide that information to them.
Yasmeen Rahimi -- Piper Sandler -- Analyst
Thank you so much and best of luck.
Ron Cooper -- President and Chief Executive Officer
Thank you, Yasmeen. Appreciate it.
Operator
Thank you. Our next question comes from the line of Ritu Baral with Cowen. Please proceed with your question.
Ritu Baral -- Cowen and Company, LLC -- Analyst
Good morning, guys. Thanks for taking the question. Can you comment on any potential missing data that you guys have on the serum bile acid endpoint, given the conduct of the trial over COVID? And at least at this point, high level, how are you thinking about the commercial for PFIC? About how many reps do you think you would need? Are you going to be focusing on centers of excellence? How broad does that marketing campaign need to be?
Patrick Horn -- Chief Medical Officer
So -- hi, Ritu. This is Pat. So in regards to the first question, missing data, so we have been very fortunate that we have a clinical operations team and a CRO that we're able to put into -- deep into place enough of the different measures and really handle these patients that were remaining in the study on a patient-by-patient basis. So looking at the data, we will have no missing bile acid data for the primary endpoint. There may be missing data at one or two of the intermediate endpoints or primary endpoint, which is the change from baseline to the end of the study, there will be no missing data. And for the commercial question, I'll turn it to Pamela.
Pamela Stephenson -- Chief Commercial Officer
Hi, Ritu. Thanks for the question. So we are planning, in terms of your first part of the question, for about 15 to 25 field-facing individuals. As you well know, this is a concentrated prescriber base, and we will be deploying sort of a center of excellence model focused on the 60 relative centers across the U.S. And we have spent a fair amount of time, first half of this year, profiling those accounts. So we have a depth of understanding of those accounts, and we feel confident in being able to cover them with the 15 to 25 field-facing personnel.
And to your final part of the question, certainly, there are important influencers and potential prescribers beyond those centers of excellence. We are doing a lot of research now to better understand them. But we don't believe we'll need more in terms of number of reps to cover any additional prescribers.
Ritu Baral -- Cowen and Company, LLC -- Analyst
Great. And let me squeak in one last...
Ron Cooper -- President and Chief Executive Officer
Thanks...
Ritu Baral -- Cowen and Company, LLC -- Analyst
Yeah, I'm going to squeak in one last question. Please forgive me. Key secondary analysis that we should be looking out for with top-line release. And thanks for taking the questions.
Patrick Horn -- Chief Medical Officer
So for a top-line release, again, we haven't had the final internal discussion, but my expectation is we'll only be releasing the primary analysis in top-line. The secondary analysis will be presented at the scientific meetings and publications.
Ron Cooper -- President and Chief Executive Officer
You have to remember, Ritu, that the PEDFIC study will probably be the largest prospective study ever conducted in PFIC. And when we speak with the scientific community, they're very excited about that data because that's going to provide a huge bank of information on not only odevixibat, but the treatment of the disease. So we'll be trying to preserve as much of that as possible for upcoming scientific meetings and publications.
Ritu Baral -- Cowen and Company, LLC -- Analyst
Fair enough. Thanks for taking all the questions.
Ron Cooper -- President and Chief Executive Officer
Thank you, Ritu.
Operator
Thank you. Our next question comes from the line of Eun Yang with Jefferies.. Please proceed with your question.
Eun Yang -- Jefferies -- Analyst
Thank you. So I look forward to the data in coming weeks. So when you present PFIC Phase 3 data at the Phase I, are you also presenting some interim data from PEDFIC 2?
Ron Cooper -- President and Chief Executive Officer
So for the top-line, Ritu [Eun], we'll be focused on just PEDFIC 1. We will be doing some analysis of PEDFIC 2, but that will be part of our regulatory submission.
Eun Yang -- Jefferies -- Analyst
I see. And then on the NASH data there we are expecting, I think that you mentioned briefly, but can you talk about what we should be looking for with the IBAT inhibitor? My question is, what do you think would be the key benefits compared to other products that we've seen in NASH? Thanks.
Ron Cooper -- President and Chief Executive Officer
Yeah, Thanks, Eun. I think our thesis in NASH is a little bit different than the other agents. So we don't -- when you think about the transition from NAFLD to NASH, what are the things that changed? Cholesterol changes, glucose changes, liver information and liver fibrosis, and increasing those information that there's an increase in bile acids. So our thesis is if we can affect multiple parameters by removing toxic bile acids from the body and toxic cholesterol, taking it out of the body, we believe that we could have some really interesting efficacy. That efficacy, though, is combined without a but, right? If you look at many of the current agents that are being developed, there is -- we're getting efficacy, but there are some side effects or convenience issues that make it difficult to give this medicine to a largely asymptomatic population.
So with elobixibat, in particular, think about the convenience of this drug. It's an oral drug. So it's not injectable. It's once-a-day, it's not multiple times a day. And it's a drug that is not systemic. So we do not anticipate much in the way of off-target effects like we've seen in some of our other studies. So therefore, it can be combined quite nicely with other cardiovascular drugs or combined with other NASH drugs. So for that -- from that perspective, we believe that the safety, convenience, tolerability is attractive for IBAT inhibition. Now, what we'd like to share is efficacy against multiple parameters of liver health and cardiovascular health to help with NASH patients. So we're pretty excited about the upcoming data to be able to demonstrate that.
Eun Yang -- Jefferies -- Analyst
Thank you.
Ron Cooper -- President and Chief Executive Officer
Thanks, Eun.
Operator
Thank you. Our next question comes from the line of Brian Skorney with Baird. Please proceed with your question.
Brian Skorney -- Baird -- Analyst
Hey, good morning everyone and thanks for taking my questions. I know you've talked a bit about the powering around the pruritus endpoint in the PFIC study and the assumption that there's a sizable placebo effect there. I was wondering how should we think about this for the serum bile acid endpoint, though. The cutoff of 70% reduction or less than 70 micromoles [Phonetic] per liter, pretty high threshold. Is there an assumption that any placebo patients, which you [Technical Issues] study under your powering? And maybe you could just kind of characterize what would be a reasonable variability around FDA be it for placebo and if you're looking at this over the leading period to ensure stability, before putting patients on treatment or placebo? And have you seen any blinded serum bile acid data from the study so far and is it consistent with expectations?
Patrick Horn -- Chief Medical Officer
So let me see if I can address all those points, if I can remember. So we did -- there were multiple bile acids obtained during the screening period. And that was to confirm that the bile acids were above 100, because that was an inclusion criteria, they had to be above 100. And so there was a little variability in some patients, but relative -- it was relatively constant.
After screening -- after the first dose, bile acids have been drawn throughout the study, but those remain blinded. So we have not seen any of the bile acid data after the first dose. 70% is a high bar. I would agree, it doesn't seem likely that placebo patients would, in fact, reach that. There is one other kind of component to the responder definition, though, is getting below 70. So it might not have to be a 70% reduction to be a responder. But even so, the thinking is it's unlikely that any placebo would reach it. But during our power assumption, we included a fairly significant response in the placebo arm to allow for any variations that we might be seeing. So that was accounted for in our powering assumptions.
Ron Cooper -- President and Chief Executive Officer
Brian, just said differently, our powering assumptions are really driven around the pruritus endpoint because we recognize there's more variability in that. So in the pruritus endpoint, we're powered at well over 80% to show a difference. In certain bile acids, there's just less variability over time. And as Pat has indicated, what we've done is we picked the number where we believe we will knock out placebo effect. So think about this, we need to show, in the treated group, that there is a reduction of 70% or reaching 70% versus the placebo group. Right? And so that's the big difference, the versus part. So you may recall that from our Phase 2 study within the PFIC group, within a four-week timeframe, we had a mean reduction of over 70%. So we feel pretty confident in our ability to reach the serum bile acid endpoint.
Brian Skorney -- Baird -- Analyst
Great, thanks, that's helpful.
Ron Cooper -- President and Chief Executive Officer
Thanks, Brian.
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, good morning. Thanks for taking my questions. Can you talk a bit more about this -- about the genetic testing program, which you highlighted earlier today and the scale of the Expanded Access Program? Trying to get a sense of where you stand in terms of identifying patients or number of identified patients ahead of launch next year. Thanks.
Ron Cooper -- President and Chief Executive Officer
So, Alan, we're not hearing you really well, but just to make sure, I think you asked some questions around the genetic testing program and a little more information around the Expanded Access Program and kind of where we are with that. Pamela, maybe you could address those.
Pamela Stephenson -- Chief Commercial Officer
Sure. Hi, Alan.
Alan Carr -- Needham & Company -- Analyst
Yes.
Pamela Stephenson -- Chief Commercial Officer
Yeah.
Alan Carr -- Needham & Company -- Analyst
The other element to that was just understanding -- or where you all stand in terms of understanding the number of identified patients as you get closer to launch.
Pamela Stephenson -- Chief Commercial Officer
Great. Thanks, Alan. So on your first question on the genetic testing program. This is a program that's sponsored by multiple industry partners to provide financial support for a free genetic testing program in the U.S. So as we understand it from what we hear from physicians that this is a really valuable program for physicians and patients and families. And so we are providing financial support to continue this program, and we're really happy that we're going to be able to continue to support the PFIC community in this way.
On your second question on the EAP program that we announced and launched on July 20, we are very pleased with the response thus far from physicians and parents. We're getting requests. We are processing those requests. And certainly, that will be one mechanism to sort of identify patients. But to the latter part of your question, we have other efforts ongoing to better understand the number of patients out there, so through centered at health mapping, and other research that we're doing, we continue to kind of hone our understanding -- we will continue to hone our understanding of those numbers prior to launch.
Alan Carr -- Needham & Company -- Analyst
Okay. Thanks for taking my questions.
Ron Cooper -- President and Chief Executive Officer
Thanks, Alan.
Operator
Thank you. Our next question comes from the line of Ed Arce with H.C. Wainwright. Please proceed with your question.
Thomas Yip -- H.C. Wainwright -- Analyst
Hi, good morning, everyone. This is Thomas Yip asking a couple of questions for Ed. Congratulations on a very busy second quarter. So first question, regarding your upcoming Alagille syndrome pivotal study that's expected to kick off before year-end. So while you're still figuring out the detailed design studies -- study designs, can you give us a rough idea of the enrollment size, treatment duration and also the primary endpoint? Would it be the same for both the FDA and the EMA?
Ron Cooper -- President and Chief Executive Officer
Thomas, good morning and thanks very much for the question. We're really pleased to have come to agreement with the regulatory authorities in the U.S. and Europe on a single pivotal program for Alagille syndrome. And so we're really pleased with that. And the team is up and going and trying to execute on that. When you think about the Alagille study, think about it kind of like our PEDFIC 1 study of scale like that. And we'll provide more details on that as we work our way through sort of the IRBs in the various sites. But as I said, we're excited to have a single pivotal that should be sufficient for approval for Alagille syndrome in both the U.S. and Europe, and we anticipate being up and going by the end of this year.
Thomas Yip -- H.C. Wainwright -- Analyst
Okay, thanks. Thanks for the clarification. And then for the NASH proof-of-concept study, assuming that you'll see positive liver and cardio perimeters, as you mentioned, what are the next steps for you guys? Would it be before the data readout from EA Pharma or are you guys waiting for that data set to make a bigger decision?
Ron Cooper -- President and Chief Executive Officer
Yeah, thanks, Thomas. Our business development discussions for our NASH assets are ongoing activities. So we continue to have dialogue with potential partners. I think we need to see the data from the Phase 2 study in the U.S., first of all, and we'll be sharing that with our potential partners. And then we'll kind of take it from there. Then we'll look forward to our Japanese data, which will be a larger study. It's for 100 patients. It's at a higher dose. It's also in combination with cholestyramine. So that will be part of the ongoing dialogue that we will have with potential partners. But we're pretty excited to be delivering data to them, because when we spoke to potential partners before, they said, look, really like this concept of IBAT inhibition in NASH, liked the little bits of data that you have, but we'd like to see data in NASH and NAFLD patients. And so we're going to be delivering that data very soon.
Thomas Yip -- H.C. Wainwright -- Analyst
Okay, that makes sense. One final question from us. For your new pre-clinical candidate, what therapeutic area do you have in mind for now? Would it be more along the line of a pediatric liver disease or would it be a broader potential market like NASH?
Ron Cooper -- President and Chief Executive Officer
We have a lot of expertise in our organization in bile acid modulation. The history of our company goes back into AstraZeneca in multiple decades. And so our understanding of these mechanisms is really strong. We love IBAT inhibitors because, as I've said before, the safety profile, the convenience, and the efficacy that we've been able to demonstrate in both -- in diseases like chronic constipation and children's pediatric cholestatic liver disease that it's just been great. But we are limited in the therapeutic window with these agents. Right? And so what we're trying to do is to widen that therapeutic window, the balance between being able to drive down bile acids and have good tolerability. And we're pretty excited that our team in Sweden has come up with some really interesting approaches.
Now at this time, we've not disclosed the mechanism of action, and we've not disclosed where we're going other than to say we're looking at adult diseases. But we have filed for patents, we have IND-enabling studies ongoing, and we're looking forward to sharing more data with you as the year progresses and as we get into next year.
Thomas Yip -- H.C. Wainwright -- Analyst
Okay. It sounds good. We'll look forward to that. Thank you for taking our questions again, and we look forward to [Indecipherable] the readouts in the next few weeks.
Ron Cooper -- President and Chief Executive Officer
Thank you, Thomas.
Operator
Thank you. Our next question comes from the line of Tim Lugo with William Blair. Please proceed with your question.
John Boyle -- William Blair & Company -- Analyst
Hi, team. This is John on for Tim. Thanks for the questions. Just two from us. So just with regards to biliary atresia, I'm just wondering if you guys are seeing any issues with getting the trial fully up and running, given the ongoing pandemic and if you're seeing any issues with enrollment cadence or any issue specific to specific regions. And then just a follow-up -- quick follow-up on a prior question on the Alagille syndrome trial. Just wondering if you have any -- a time line from when you'll share some more details on that trial design. Thanks.
Ron Cooper -- President and Chief Executive Officer
Yeah, Thanks very much, John. I have to say I'm really proud of Pat's clinical team and his clin ops team. You think about trying to get a trial up and going during this COVID crisis and really it's something else. So we've been able to get trial sites up and going in the U.S. They're actively recruiting. We actually have patients enrolled. Pretty excited about that. We've given guidance that we should complete, having all the sites up and going by -- in the first half of next year. And I know that Pat and his team are working really hard on that.
A key factor in that will be our ability to actually get on-site in the hospital. Right? And so in some circumstances from the -- with COVID, it is difficult for us to get into those hospitals. But we're hopeful in our ability to get into those hospitals and get trial sites up and going. Because, as I said earlier, biliary atresia is the number one cause of pediatric liver transplant. The unmet medical need is super high. And there are a number of children that could potentially benefit from our study. So we're working very hard to do that.
In regards to timelines for Alagille syndrome, we'll give more guidance on that as time goes on. But I think the way to think about it is we're going to be delivering PFIC data in the coming weeks. Alagille syndrome data should be next and then biliary atresia data over time.
John Boyle -- William Blair & Company -- Analyst
All right, thanks. Congrats again on the productive quarter.
Ron Cooper -- President and Chief Executive Officer
Thanks very much, John.
Operator
Thank you. ladies and gentlemen, our next question comes from the line of Liana Moussatos with Wedbush. Please proceed with your question.
Liana Moussatos . -- Wedbush Securities -- Analyst
Thank you for taking my questions. So I see three ways to win on PEDFIC. You can either win with both endpoints or either endpoint. If serum bile acid hits but pruritus doesn't, would you pursue European commercialization -- European approval and commercialization without a partner or with a partner? And how long -- if both endpoints hit, how long will it take for you to submit the regulatory documents?
Ron Cooper -- President and Chief Executive Officer
Thanks for the question, Liana. I think we remain really confident in the outcome of the PEDFIC study and in both endpoints. So I think the real answer to your question is we've got to see what the data says. Right? And once we see what the data -- what we do have, then we will proceed from there. Our current plans are to commercialize in the U.S. and in Europe. As Pamela has said, this is a key account approach. There's about 100 key pediatric hepatologists in the U.S., the same in Europe. So we think that is quite manageable. So that -- the answer to your question really is dependent upon the data.
And then your second question...
Patrick Horn -- Chief Medical Officer
Timing of filing.
Ron Cooper -- President and Chief Executive Officer
The timing of filing. Right -- yeah, right now, we're not giving guidance on that. Our guidance has been that we expect -- in the second half of 2021, we expect approval, issuance of a priority review voucher and launch during that time frame.
Liana Moussatos . -- Wedbush Securities -- Analyst
Thank you.
Ron Cooper -- President and Chief Executive Officer
Thank you, Liana.
Operator
Thank you. Ladies and gentlemen, at this time, there are no further questions. I would like to turn it back to management for closing comments.
Ron Cooper -- President and Chief Executive Officer
Great, thank you operator. First of all, I'd like to express my thanks to everybody that has joined us for today's call. Hopefully, what you've seen is that we have made tremendous progress during this quarter, despite the challenges of COVID-19 and eagerly anticipate seeing top-line data for odevixibat and PFIC, as well as elobixibat in NASH. Thanks again for tuning in.
Operator
[Operator Closing Remarks]
Duration: 42 minutes
Call participants:
Paul Arndt -- Investor Relations, LifeSci Advisors
Ron Cooper -- President and Chief Executive Officer
Simon Harford -- Chief Financial Officer
Patrick Horn -- Chief Medical Officer
Pamela Stephenson -- Chief Commercial Officer
Yasmeen Rahimi -- Piper Sandler -- Analyst
Ritu Baral -- Cowen and Company, LLC -- Analyst
Eun Yang -- Jefferies -- Analyst
Brian Skorney -- Baird -- Analyst
Alan Carr -- Needham & Company -- Analyst
Thomas Yip -- H.C. Wainwright -- Analyst
John Boyle -- William Blair & Company -- Analyst
Liana Moussatos . -- Wedbush Securities -- Analyst
