Albireo Pharma, Inc. (ALBO) on Q2 2022 Results - Earnings Call Transcript
Operator: Good afternoon, and welcome to the Albireo Pharma Second Quarter 2022 Earnings Call. . Please note that this conference is being recorded. I will now turn the call over to your host, Hans Vitzthum, Managing Director of LifeSci Advisors. Thank you. You may begin. Hans Vitzthum: Thank you, operator, and good afternoon, everyone. Thank you for joining today's call. This afternoon, Albireo issued a press release highlighting its recent business accomplishments and financial results for the second quarter ended June 30, 2022. 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 Investors section of our website at www.albireopharma.com, or on the SEC website. Any forward-looking statements represent our views as of today, August 15, 2022, and should not be relied upon as representing our views as of any subsequent dates. We undertake no obligation to update these statements publicly. Now it is my pleasure to turn the call over to Ron Cooper, Albireo's President and Chief Executive Officer. Ron? Ron Cooper: Thanks, Hans, and thank you, everyone, for joining us this afternoon. With me today are Simon Harford, our Chief Financial Officer; Pamela Stephenson, our Chief Commercial Officer; and Dr. Jan Mattsson, Chief Scientific Officer and Head of R&D. We estimate that there are about 100,000 cholestatic liver disease patients around the world that could benefit from Bylvay therapy. And in Q2, we had another strong quarter, accessing these patients through excellent execution of both our commercial and clinical development plans. Old standard Phase III studies like ours provide the highest quality of compelling data for payers, regulators and prescribers. Our approach resulted in build a U.S. and EU approvals in PFIC, and we expect the same for Alagille syndrome and biliary atresia. PFIC country commercialization rollout continues as planned. We're looking forward to a major Phase III readout Alagille syndrome around the corner and expect the bold biliary atresia study to be fully enrolled this year. We're building short-term and long-term corporate value at a steady pace to drive our aspiration of making build a $1 billion drug in the second half of the decade. On top of Bylvay, our promising earlier-stage assets, A3907 and A2342 are progressing exactly according to plan and will become valuable growth drivers. The strong financial footing and a team years of expertise in bile acid modulators, we're poised to deliver our plan to create additional value. As we shared with you in the first half of 2021, we have a global launch strategy for Bylvay, achieving global net product revenues of $5.9 million in the quarter. While sales are important, the more important metric is patient capture, which relates to the long-term value of our drug. I'm pleased that we now have 245 total Bylvay patients either on drug or in process. On a quarter-to-quarter basis, we've had consistent new patient capture. We continue to make excellent progress with global Bylvay launch and gain important market insights along the way. This quarter, we had a larger change in the mix of babies versus adult on therapy. We're also finding that in the U.S., patients are a bit more dispersed than expected as longer than originally anticipated to get to them. Pamela will provide more detailed update on these topics and the global launch metrics. You'll see that we've adjusted the metrics in a new format based on investor feedback. The metrics are simplified and now add up to total Bylvay patients. As we all know, PFIC is our smallest indication, but we're making excellent progress in our objective of expanding beyond PFIC as Bylvay is a pipeline in a product. We're on track in Alagille syndrome and expect to report top line data for the Phase III ASSERT study in the fall, which is really exciting. SERD is a gold standard prospective intervention trial with 35 sites across North America, Europe, Middle East and Asia Pacific. With that in mind, we're also very excited about the progress to date with our Phase III BOLD study in biliary atresia with enrollment on track to be completed by the end of this year and a top line data readout in 2024. Phase III BOLD trial is the first and only Phase III evaluating IBAT inhibitor in biliary atresia with clinical trial sites spanning the U.S., Europe, Latin America and China. For exchange with the FDA have been very productive, and we're excited about Bylvay's potential in this indication. Biliary atresia is the most common pediatric cholestatic liver disease and the #1 reason for pediatric liver transplants across all diseases. And finally, we're pleased with the 2 recent appointments to our Board of Directors, strengthen our company with the addition of Susan Alesina, Vice President, National Business Development and Alliances at Boston Children's Hospital; and Habib Dable, former President and CEO of Acceleron Pharma. Susan is a strong leader who in a role at Boston Children's Hospital provides us excellent insights into the health care system challenges and opportunities. In addition to our experience with a major Children's Hospital, Susan brings a rare blend of both big pharma and biotech strategic planning, business development and communications experience. Habib is another successful leader who grew Acceleron, which is eventually sold to Merck for over $11 billion. Habib brings excellent biotech CEO experience as well as big pharma scale experience for his many years at Bayer. We're fortunate to have both these great leaders during our board. Now let me turn it over to Pamela, who will take you through the progress of our global launch of Bylvay and PFIC. Pembina? Pamela Stephenson: Thanks, Ron. We are very pleased with the progress the team has made with Bylvay. To take you through our Q2 performance, I encourage you to reference Slide 15 in the corporate deck posted on the Investors section of our website. So let me start with our updated metrics methodology. We have revised the Q1 figures to match the new methodology. Having listened to feedback from you as investors and analysts, we have adjusted 3 of our key metrics and added a new metric we're calling total Bylvay patients so that our metrics are more intuitive and easier to understand. The net sales in unique prescribers metrics remain the same. The 3 updated metrics are; number of reimbursed patients, patients pending reimbursement and rollover patients. Reimbursed patients, meaning patients initiated on reimbursed drug and generating revenue, net of patients who have discontinued. Pending reimbursement, meaning patients with a prescription who are currently in our managed access programs or are in the process of getting on reimbursed drug. Rollover patients, meaning patients in the PFIC expanded access program or in the PEDFIC 2 open-label extension trial. It's important to note that this metric no longer includes patients in our managed access programs as they are now included in our new pending reimbursement metric. Together, these three metrics add up to our total Bylvay patients metric, which account for all patients currently on or in the process of getting on Bylvay worldwide. Let me give some detail on each of these, starting with total Bylvay patients, which is showing strong quarter-over-quarter growth from 170 patients at year-end 2021 to 207 at the end of Q1 to 245 at the end of Q2. Total Bylvay patients increased 18% in the second quarter. That is 38 new patients added that we did not have at the end of March, consistent with the double-digit quarter-over-quarter increase we saw in Q1. We are very pleased with this increase because it means more physicians are gaining experience with Bylvay and seeing firsthand the clinical benefit. We are seeing that once a physician has a positive experience, they are more likely to prescribe Bylvay again. Most importantly, this means that more and more patients are getting relief from their debilitating pruritus, seeing improvements in growth and sleep and enjoying better health outcomes. This is the reason to believe in the long-term value of our drug. Doctors are telling us this drug works, which is exactly what you want to hear in a launch and why we have 245 patients and that number continues to grow. Now let's turn to reimbursed patients. We are pleased that reimbursed patients have essentially doubled in the first 6 months of 2022 from 50 at the end of 2021 to 99 as of the end of June. This is a good indicator of coverage in the U.S., Germany and the U.K., and we will continue to see this number grow as pricing and reimbursement is granted in additional countries. In the U.S., nearly all patients across public and private insurers have a path to coverage for Bylvay. Now let's look at pending reimbursement. The number of patients pending reimbursement in the quarter increased to 68. This group of patients will eventually and hopefully as soon as possible, transition to becoming revenue-generating going forward. We are progressing well with completion of pricing and reimbursement in Italy, Belgium and Scotland. This is a direct result of the experience of our market access team and the recognition among payers of the innovation that Bylvay represents with our pivotal Phase III clinical trial data. In Italy, Bylvay has been granted full therapeutic innovation status by AIFA, the Italian Medicines Agency, which will ensure dedicated national funding and inclusion into regional formularies within 60 days from the publication of the decree in the National Gazette Journal anticipated next month. Together with Scotland and Belgium, these results confirm our thesis that payers see the high unmet need, added clinical benefit, robustness of the Phase III scientific evidence and overall value of Bylvay. With 14 dossiers submitted to-date, we anticipate coverage in additional European markets this year, which will trigger conversions of patients who are pending reimbursement to becoming revenue-generating. Let's now look at rollover patients. As a reminder, these 78 patients are either in the PFIC expanded access program or the PEDFIC 2 open-label extension trial, and they will roll over on to reimbursed drug over time. This metric is anticipated to decrease as patients roll off the extension trial at the end of 72 weeks and on to revenue-generating product in reimbursed markets. Moving on to net sales, Bylvay achieved $5.9 million of net revenue in the quarter, resulting in $10.5 million year-to-date with revenue from the U.S., Germany and recently the addition of the U.K. U.S. revenue was $3.5 million in Q2. In the U.S., we are pleased with the significant increase in the number of unique prescribers as our field team connects with patients as is common and rare diseases. We are seeing, however, that is taking longer than originally anticipated due to the broader prescribing base. International performance is going well with $2.4 million in revenue due to strong performance in Germany. This is proof of our ability to quickly connect patients and get them reimbursed on a global scale. As Ron mentioned earlier, the reality is that we are seeing greater variability than anticipated in terms of weight and patients starting on Bylvay. A larger proportion of our discontinuations have come from older, heavier and - higher revenue patients as they transition to liver transplant due to the progressive nature of their disease. I want to stress that we are in the early stages where small variations can make a big difference. The range of patients is wide in terms of age and weight with teenagers and adults bringing 10x the initial revenue of small babies. Yet in the long term, our smaller patients will create a larger revenue annuity as their health improves and they get back onto the growth curve. Given that both the compliance rate and refill rates are tracking well above 80%, we are confident in this revenue opportunity. Finally, our last metric is unique prescribers. We have increased the number of Bylvay prescribers in the U.S. to 73% by the end of June, up from 57 at the end of March. As a result of the expertise we are building and the new targeting approaches we are using to connect prescribers and patients. This is an additional 16 prescribers who had not previously written a prescription for Bylvay. The acceleration in the U.S. prescriber base confirms our belief that the available patients are there, but are finding -- but we are finding that they are taking longer to access due to 30% of prescribers being outside our key centers. These prescribers include community and office-based physicians as well as adult prescribers. Now that these HCPs have a therapeutic option to prescribe to their patients, they do not need to refer them to large centers as they had to do in the past for transplant. In summary, we are making excellent progress in terms of total Bylvay patients. We continue to expand the prescriber base to encourage more experience with Bylvay and we are gaining reimbursement in important markets such as Italy and Belgium due to the strong pivotal Phase III data and our ability to execute on market access strategies. Now I'll turn it back to Ron. Ron Cooper: Thanks, Pamela I wanted to quickly touch on our one-of-a-kind early development assets, A3907 and A2342. A3907 is the world's first and oral -- high systemically biovailable ASPD inhibitor in clinical development. And in our preclinical models, A3907 clearly demonstrates that it's different from the commercially available IBAT inhibitors. In addition, in the Phase I study, A3907 demonstrated excellent systemic exposure and good tolerability. We anticipate starting a Phase II study in adult liver disease by the end of 2022. We will share more details about our development approach for A3907 later this year. Our second asset for adult liver disease, A2342 deserves a few words. A2342 is the world's first and only oral NTCP inhibitor. We're working on advancing A2342 into a Phase I study by the end of this year, the intent of proving it to be a unique component of a combination treatment for hepatitis B or D. We have the ability and resources to develop both of these assets on our own for a single rare disease, but both product candidates have garnered interest from strategic partners considering wider development plans. Now let me turn it over to Simon. Simon Harford: Thank you, Ron. Let me summarize our financial results for the second quarter of 2022. Bylvay global net product revenue was $5.9 million. U.S. revenue was $3.5 million and international revenue was $2.4 million. Royalty revenue was $2.3 million compared to $2.4 million last year, a decrease of $100,000. All royalty revenue is passed on to healthcare royalty partners. Cost of product revenue was $800,000, following approval of Bylvay certain manufacturing and quality headcount costs are now included in cost of product revenue. There were no material costs as materials related to current products sold were expensed prior to approval. Bylvay was approved during the third quarter of 2021. Therefore, there was no cost of product revenue last year. R&D expenses were $22.9 million compared to $20.9 million for the same period of 2021, an increase of $2 million. The increase in R&D expenses were principally due to expenses related to clinical program activities, personnel expenses, including stock-based compensation and other costs as we continue to increase our headcount and program activities. The increase in program activities related to ongoing Phase III clinical trials, for biliary atresia and Alagille syndrome as well as Bylvay PFIC expenses and were partially offset by a decrease in preclinical expenses and A3907 due to completion of the Phase I study. Selling, general and administrative expenses were $21.6 million compared to $16.9 million last year, an increase of $4.7 million. The increase is attributable to personnel and related expenses as we continue to increase our headcount and commercialization activities related to Bylvay, including our sales force and support for global expansion efforts. Net loss for the second quarter of 2022 was $39.9 million or a loss of $2.04 per share compared to a loss of $36.4 million or a loss of $1.90 per share for the second quarter of 2021. The company had cash and cash equivalents of $181 million as of the end of the second quarter versus $216.7 million as of March 31, 2022. The company expects to have sufficient cash into 2024 based on current revenue and expense projections, while total Bylvay patients continue to progress well. The variability on weight of starting patients and time to find patients in the U.S. means we are adjusting our 2022 revenue guidance to $24 million. With that, let me turn it back to Ron for closing remarks. Ron Cooper: Thank you, Simon. Every week, we're capturing more and more patients, creating a stronger and stronger annuity for growth. Clinical trials are on track or ahead of expectations. As we advance our plans to make -- Bylvay $1 billion product, the key for us to take Bylvay from a PFIC drug to a leading pediatric cholestatic liver disease drug. We'll accomplish this by building our patient base quarter-by-quarter and plan to expand to Alagille and biliary atresia patients on the back of our Phase III studies. And finally, we continue to focus - our early assets in adult liver disease with A3907 and A2342. With this strong portfolio and financial position, we have a tremendous opportunity, and we'll continue to stay focused on our growth drivers. It's all very exciting for Albireo as a company with a first-in-class and first-to-market product for near and midterm growth. We thank everybody for joining us and I am pleased to open the call now for Q&A. Operator? Operator: . The first question is from Ritu Baral with Cowen. Ritu Baral: Can you hear me okay or no. Can you hear me? Ron Cooper: A little bit fuzzy but you start again. I think we are hearing you now. Ritu Baral: Okay sorry about ? Ron Cooper: Ritu I apologize, I apologize we're really having a hard time hearing you now. Ritu Baral: I'll back in the queue. Ron Cooper: All right. Operator: The next question is from Eun Yang with Jefferies. Eun Yang: Can you hear me okay? Ron Cooper: We hear you perfectly well. Eun Yang: Oh great, great okay, so I have a question on Alagille syndrome data expected this fall. So in Alagille syndrome study, the dose is 120, whereas in PFIC, it started as 40. So given the 3x higher dose with the same similar study design and same endpoint how do you see the efficacy and safety compared to what we saw in PFIC? Ron Cooper: I think we're pretty excited about the ASSERT study, and those results being announced just around the corner of this fall. As you know, ASSERT is the very first and only gold-standard randomized placebo-controlled study. And with that, we hope to show really strong efficacy and great tolerability in the ASSERT study similar to what we saw in the PEDFIC studies. As it relates to your question regards to dose, I think that's another ââ¬â it's one step at a time, right? Let's get the results from the ASSERT study. And then following the results of ASSERT study, you will be submitting that to the regulatory authorities, and we'll get into dialogue with them. Eun Yang: This so one follow-up question today is at 120 microgram per kilogram dose, do you expect side effects could it be potentially greater than a 40 microgram that you used in PFIC as a starting dose? Ron Cooper: Well, I think we'll have to wait till we see the data. But if you go back to the PEDFIC study where we use both the 40 and the 120 doses, we saw very similar levels of tolerability. So I think our expectation would be that we have excellent tolerability in the ASSERT study, and just like we saw in the PEDFIC study. Eun Yang: Okay that's helpful. And the second question is sorry, sorry for interruption. Ron Cooper: Go ahead, go ahead. Eun Yang: Yes so you -- in the prepared remarks, you mentioned that in PFIC, patients are more dispersed than expected in the U.S. and prescribing -- prescribers base is like a 30% outside the key centers. So do you think that there would be similar with the Alagille syndrome patients and with that, do you think you will need to expand your sales reps? Pamela Stephenson: So I'll just start by saying we're really pleased with how we're doing in terms of getting out to more unique prescribers with that 28% increase to 73 prescribers. For Alagille, we anticipate a similar type of prescriber base with those key centers that will be -- that we'll continue to call on. And there may be patients out in the community like we're finding with PFIC. And the good news is we're out there finding those unique prescribers and expanding our prescriber base. So we're really, really confident that we will not need to expand our field team and maybe 1 or 2 more reps, but nothing beyond that. Ron Cooper: That's the great thing about our model Eun. It's the exact same pediatric hepatologist that prescribed for PFIC, for Alagille and biliary atresia. So we think that we're sized appropriately to be able to take advantage of those opportunities. Eun Yang: Okay my last question. So Pam mentioned the compliance and refill rate is greater than 80%. Does that kind of imply the discontinuation rate, you mentioned that it's a local order and higher body weight patients. Do you think the discontinuation rate is somewhere around less than 20%? Pamela Stephenson: So let me just take a step back to clarify. So no, these are 2 different rates, right? So the compliance and persistency rate that I was speaking to really has to do in a given month, the patients who are on therapy at the end of the month, did they take all of their pills, so they get all their refills on time. And so that's -- we're really pleased with that being quite high. The second question -- a different question that you were asking was around the discontinuations. That's a different metric. That is a metric that we are seeing is right in line with expectations, what we would think for rare disease. But I do -- just want to clarify that those are 2 different metrics. Eun Yang: So you're not disclosing the discontinuation rate? Ron Cooper: Well, that's one of the reasons we changed our metrics slightly you see from previous quarters. You see that in -- we have a new metric called the reimbursed patients, right? And that's patients who are on drug at this time, and they connect directly to revenue. And that would be inclusive of individuals that have withdrawn from therapy. That's probably the more relevant metric for you. Operator: The next question is from Tim Lugo with William Blair. Lachlan Hanbury-Brown: This is Lachlan on for Tim. A couple here just -- so you mentioned the broader prescriber base. Can you maybe talk about how much that is than originally planned? I know you were just saying you don't think you expect to expand the sales force much if at all, but how many more physicians do you now targeting? And then second, on the international investment wins, do you have any color or how might be in each of those markets and then the pricing looks like? Ron Cooper: So Lachlan, you're a little choppy on us. I just want to make sure that we have the questions correct, right? I think that your first question was around the prescriber base in the U.S., how much broader that is? And your second question, I think, was around the international markets. And I believe what you were asking about what we -- in terms of the number of patients that are there -- we anticipate for price. I just want to make sure that that's what they were? Lachlan Hanbury-Brown: Correct. Ron Cooper: So Pamela, why don't you grab hold of that? Pamela Stephenson: Yes so first on the question on the broader prescriber base, I mean, what we're seeing is that we're still quite focused on the hundred physicians that we've always talked about in the 60 key centers. So that is there's depth there in terms of the physicians and centers and patients that are there. And then beyond that, as we look to the potentially around 1,000 other potential prescribers. What we're doing with that is we're able through advanced analytics to really have a better understanding of which of those physicians are most likely to prescribe. So, we've been really going sort of deep on the centers and then broad on the broader prescriber base. On your second question in terms of reimbursement, outside of the U.S as I mentioned, that is going well. The negotiations have gone well. We're really pleased with Italy and Belgium and Scotland, bringing those countries in even faster than we've seen for industry benchmarks. And the number of patients in each of the countries, I would just say, look to your country population estimates as a guide there because there's -- that's something that we're not able to disclose at this time. But it's something that I think you can see from your population models overall. And then the pricing, I think, again, as expected, the pricing is right in line with what we had expected in terms of rare disease pricing in European markets. Lachlan Hanbury-Brown: A quick follow-up here, you mentioned the depth in the key centers. Is it broader subscribers are you seeing as much debt are you seeing them or they like you are seeing -- 1 or 2..? Pamela Stephenson: No, you're absolutely right. 1 or 2 patients in the broader prescriber base. Ron Cooper: But again --- remember, we're still early in the launch process, right? So that's what we would expect. But that gets us pretty excited, right? Because once they start, we anticipate that as new patients as they identify new patients that they will be depth of prescribing. Operator: The next question is from Brian Skorney with Baird. Brian Skorney: I was hoping you can talk briefly about the uptake in the EU. Now of reimbursement in U.K., Italy and Belgium, but it sounds like Belgium doesn't start to contribute until October. Just wanted to get an idea of how much of a contributing factor U.K. and Italy were this quarter versus Germany and how much growth you may have seen in Germany? And then on the U.S., since last quarter, there was an inventory drawdown, where U.S. sales effectively flat over these last 3 quarters. I mean it just seems like if I kind of back out some reasonable inventory expectations for the fourth quarter stocking followed by destocking last quarter and today's front. It all seems to be sort of in that $3 million. Can you talk a little bit about the U.S. specific dynamics in terms of reimbursed patients and pending reimbursements? Simon Harford: Brian, this is Simon. In terms of the EU, really the way you need to sort of think about revenue going forward for the remainder of this year is that in sort of Q3, we would expect it to continue to be primarily patients from the countries that have already launched. So the U.S., Germany and the U.K as you know, the U.K. launched sort of in late May. As we fast forward then into Q4, that is where you should expect to see the Italian and Belgium patients start flowing through. As Pamela I think, was saying in Italy, for example. We've completed the negotiations, but we still have to wait for publication and then regional rollout region-by-region. So that will start happening strongly in the fourth quarter of this year. As you think about the topic of your question on inventory in the U.S., that really was a 2-quarter phenomenon. And as we said, we were back to sort of steady state inventory at the end of Q1. So specifically to your question on growth between Q1 and Q2 in the U.S., yes, it is fair to say the revenue numbers were flat due to that weight variability between patients. But the more meaningful metric here in our minds is really these reimbursed patients. And hence, why we've given it to you who are actually on drug and generating revenue at the end of the quarter, and that continues to grow. Operator: The next question is from Ed Arce with H.C. Wainwright. Ed Arce: Firstly for me, and I appreciate the extra detail on the patients. I wanted to first confirm that I think as you've mentioned, the revenue-generating patients right now stand at early as per - stand at 99%. And if that is in fact the case, could you help us understand the average time to conversion for both the pending and the rollover patients. And then why you think in the U.S., do you have a range that you could provide for us in terms of the gross to net pricing now -- and I have a follow-up? Ron Cooper: So Ed just -- so first off, you're asking about the revenue patients. So it is 99 patients that drive the revenue. Pamela will take the question about the average time and then Simon can talk about pricing in the U.S. so Pamela. Pamela Stephenson: So your question on the average time to conversion on the 2 categories, pending reimbursement and rollover patients, that was your question, correct? Ed Arce: Correct. Pamela Stephenson: Yes so -- that's going to differ based on the different countries. So depending -- those 2 categories are a number of patients, for example, in Italy in those countries in any of the markets that we have don't have reimbursement yet of the 14 dossiers in which we've submitted. So we'll have a good number of those patients from Italy, as Simon has just mentioned, rolling over at Q4. And then the other countries as they come online will have some -- patients from Belgium and then the other countries into 2023. So we're really excited that we're going to have patients rolling over Q4 as well into early next year. And then as you know, in the -- there is U.S. patients in that number to that are going through reimbursement and very pleased with our time to fill in the U.S., we're able to quickly get patients through that process of prior authorization and onto drug. Simon Harford: And yes, I think in terms of the question about gross to net, currently, close to 50% of patients in the U.S. are Medicaid patients. So they're at the standard Medicaid discount, the remainder of patients are obviously commercial payer and those discounts are very, very different, obviously, from Medicaid. So those 2 things sort of drive the mix as it relates to gross to net. Ed Arce: Okay all right. And then -- just one additional question, if I look at the total patients growing from 170 in the fourth quarter 207 in the first and 245 this past quarter, those are growth rates of 21.8% and then -- is that a reasonable way to think about the growth trajectory through the remainder of this year and into next year? Ron Cooper: Well I think, Ed I think, first of all, what we're really excited about is the new capture of patients, right, it's roughly the same quarter-on-quarter, right, is 37% and 38%. So that's very consistent -- that means patients are out there. I think that projecting had difficult to us to do so because, in fact, we have reimbursements that are coming in. There's a lot of dynamics. But all I can say is the fact that we've been consistent quarter-to-quarter is very pleasing to us. And these are new patients that will become revenue-generating over time. Operator: The next question is from Andreas Argyrides with Wedbush Securities. Andreas Argyrides: Just a couple here. Can you just provide clarity on the number of new rollover patients of 78 versus the 110 provided at the end of Q1. And then I don't know if I caught all the details here, the call has been a bit choppy, but you're seeing growth across 2 metrics. So what's driving the reduced sales guidance? And then I have one follow-up on ASSERT? Ron Cooper: So why don't we do this in reverse order. Why don't you talk about the sales guidance, Simon, and then maybe you could just help Andreas a little bit with the metrics, Pamela? Pamela Stephenson: Yes. Simon Harford: Yes Andreas, in terms of the sales guidance of $24 million for 2022 really what's driving that is the 2 items that have been referenced several times here. One is just the time to connect to patients in the U.S. given the broader prescriber base than we had originally anticipated. And then secondly, obviously, we've had quite some variability in terms of patient starts between sort of patient groups. And that's the other factor here in the equation, particularly as early on, we have probably more advanced sort of disease heavier patients who were quickly then sort of transitioning to liver transplant, whereas what we're seeing now is more the patient you would expect to get, which is the babies or infants. The good news about those is though they generate less revenue to begin with, we catch them and benefit them as patients as they continue to grow with this progressive disease PFIC. So that's the biggest driver. If you think about first half, second half, first half of the year, we had $10.5 million of revenue. This would imply to $24 million or $13.5 million in the second half. And again, I would expect to see continued growth in both quarters from the markets where we've already launched and then a little bit of a step-up in Q4 for maybe Italy and Belgium patients. Pamela Stephenson: And so Andreas, on your first question, the difference between what we reported in the Q1 category of potential rollover patients and the Q2 category of roll other patients. The first thing to keep in mind is that we changed and updated the definition of that category. So that's partially what's driving the difference. We took -- we removed the patients that are on our managed access program and put those into the pending reimbursement category. So that's partially why you're seeing the difference in the number there. And then in addition to that, we've been rolling over patients because within that category now, right, is the open-label PEDFIC 2 extension patients as well as the EAP patients. And so as we came online with the U.K., we rolled over patients. And as patients in Germany hit the 72-week mark, we rolled them over as well just a couple of different dynamics that would explain the difference in numbers there. Andreas Argyrides: Okay. Have you provided the target number of U.S. prescribers? And then if so, whatever that is, what's the average number of patients per prescriber that you estimate? Pamela Stephenson: What I can tell you is what we've provided is that 100 key top prescribers remains the same. They're the ones in the 60 centers that are very much focused on, have been focused on. And then beyond that, what we are looking at is because of the broader prescriber base and we're seeing patients -- some patients who are out more into the community within the community gastro urologists as well as with the adult hepatologist. We've shared, there's, a number of about 1,000 more potential prescribers out there that we are currently actively targeting. Ron Cooper: And in terms of depth, I think, Andrew, it's too early for us to be able to say. It's still early in the launch, right? So obviously, we'd like to see as much depth of prescribing with all of these, right? But to us, we're pretty excited that the number has gone up to ââ¬â sorry to 73, right? So there's more people prescribed and we'd anticipate that over time, there's going to be greater depth as well. Andreas Argyrides: Okay and the last one if I caught this correctly, the ASSERT timeline, the timeline for the readout on ASSERT has moved from year-end to the fall. I don't know if you caught this you discussed this, but what's what is the contributing factor for that timeline? Ron Cooper: We just wanted to refine our guidance to be a little bit more accurate, right? And so, we're pretty excited that the certain study is fully enrolled, the first gold standard Phase III study in Alagille syndrome, and we hope to see some good data around the corner. Operator: The next question is from Ritu Baral with Cowen. This is Yan on for Ritu. Unidentified Analyst: My question would be -- sorry, the line is still a little bit choppier, but I'm going to persevere. The question is around the discontinuations that you're seeing. Do you characterize it more as the patients sort of progressing towards leading surgery or is it more of a tolerability issue. And then sort of comparing that to what you saw in the Phase III study? Pamela Stephenson: Sure, let me take that question. First, I'll just start with saying that the discontinuations are right in line with what we would expect and what we've seen in other rare disease. And we're really pleased that we are not seeing discontinuations due to efficacy. So to your question, one of the reasons that we see in this quarter, at least, we've seen some patients discontinue is the fact that they were -- they had advanced liver disease. They were already on path to liver transplant. The physician was prescribing sort of as a last resort to help with the patient as they were getting ready for transplant. So those patients sort of just went on to transplant and had to stop Bylvay. And then the other reasons that we would see as sort of more family social dynamics that you often see in any disease category with some of the discontinuations. But again, what I would just say is that this is not concerning and the discontinuation - have been right in line with expectations. Unidentified Analyst: Okay. So I'm going to follow-up with a couple of questions. So that sort of ties into what you were describing last quarter about targeting the 2 patients. And my understanding of your category of patients would include perhaps more of those same patients that you characterize as probably having higher discontinuation rates? How do you expect that to sort of feed into your numbers? How do you expect your numbers to shake out? And then also, can you help quantify or give an idea of how the average size or age of the, patients have changed? Have you expect that to sort of start to steadying now? Pamela Stephenson: Sure. Let me talk a little bit about what we're seeing because what we are seeing is a lot of new younger babies starting therapy, and that is something that we're really excited about because they are -- they're going to be with us for the long-term. And so that is a trend that we're seeing in terms of patient starts. I think overall, the age, what I would say it's early in the launch. It's still early. We're seeing a lot of variability. We are seeing younger patients, older patients, heavier patients, lighter patients. And with our pricing dynamic, that does cause a lot of variability. And I think that's in the short-term. Over the long-term, we're really excited as the patient numbers grow we think things will smooth out. Ron Cooper: Yes and I would just add to that Yan, as you think about sort of revenue, when you start at the beginning with a relatively small number of patients, if you get a teenager, there were significantly more obviously with weight-based dosing than if you're starting with a sort of a baby or infant. Ironically though, over the long-term, those patients that you want to capture early in the progression of the disease, the younger and lighter ones will be the ones that create the continuous annuity over the long-term. So that's something to think about as you model. Operator: We have reached the end of the Q&A portion. I will now turn the call to Ron Cooper, for closing remarks. Ron Cooper: Right well, thank you, operator, and thank you all for attending today's conference call. I am very proud of our organization's ability to deliver and execute this plan and thank each every one of our employees for their commitment, drive and innovation. Keep you updated as we continue to advance Albireo's mission to provide hope to families with patients with liver disease in the entire liver community. Thanks very much. Operator: This concludes today's conference call. You may disconnect your lines. Thank you for participating, and have a pleasant day.ALBO Ratings Summary
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Albireo Pharma’s Bylvay EU Launch Begins in Germany
Albireo Pharma, Inc. (NASDAQ:ALBO), an emerging pharmaceutical company focusing on the development and commercialization of drugs for the treatment of liver and gastrointestinal diseases including Bylvay (odevixibat) for the treatment of progressive familial intrahepatic cholestasis (PFIC), announced that Bylvay is now available to patients in Germany, making it the first drug on the European market for PFIC.
With Germany having the largest EU market potential of the ~1,900 patients living with PFIC outside of the U.S., the launch of Bylvay in Germany is a major opportunity for the company to establish a following and build up revenue before expanding into additional European countries.
Analysts at Wedbush increased their price target on the company’s shares to $84 from $82, maintaining their outperform rating. While their current net revenue estimates of $5.8 million and $30.9 million for 2021 and 2022, respectively account for typical initial launch challenges, they anticipate net revenues reaching over $713 million in 2028 following a growth inflection point in 2023 as a result of likely broad payer coverage.
Albireo Pharma’s Bylvay EU Launch Begins in Germany
Albireo Pharma, Inc. (NASDAQ:ALBO), an emerging pharmaceutical company focusing on the development and commercialization of drugs for the treatment of liver and gastrointestinal diseases including Bylvay (odevixibat) for the treatment of progressive familial intrahepatic cholestasis (PFIC), announced that Bylvay is now available to patients in Germany, making it the first drug on the European market for PFIC.
With Germany having the largest EU market potential of the ~1,900 patients living with PFIC outside of the U.S., the launch of Bylvay in Germany is a major opportunity for the company to establish a following and build up revenue before expanding into additional European countries.
Analysts at Wedbush increased their price target on the company’s shares to $84 from $82, maintaining their outperform rating. While their current net revenue estimates of $5.8 million and $30.9 million for 2021 and 2022, respectively account for typical initial launch challenges, they anticipate net revenues reaching over $713 million in 2028 following a growth inflection point in 2023 as a result of likely broad payer coverage.
