Good afternoon. Thank you for attending the Mirum Pharmaceuticals Fourth Quarter and Year-end 2022 Financial R esults and Business Update. My name is Matt, and I'll be your moderator for today's call. All lines will be muted during the presentation portion of the call with an opportunity for questions and answers at the end. If you would like to ask a question, please press star one on your telephone keypad. I would now like to turn the conference over to our host, Andrew McKibben, Vice President of Investor Relations and Finance. Andrew, please go ahead.
Thanks, Matt, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals fourth quarter and full year 2022 conference call. I'm joined today by our President and CEO, Chris Peetz, our Chief Operating Officer, Peter Radovich, our Head of R&D, Pam Vig, and our CFO, Ian Clements. Earlier today, Mirum issued a news release announcing the company's results for the fourth quarter and full year 2022. Copies of this news release and SEC filings can be found in the investors section of our website. Full details on updates from the quarter can be found in our news release and 10-K issued today.
Before we begin, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements about Mirum and our programs based on management's current expectations, including statements regarding Mirum's business plans, development programs, strategies, prospects, market opportunities, and financial forecasts and guidance. Mirum is under no duty to update these statements, and they are subject to numerous risks and uncertainties. Actual results could differ materially from the results anticipated by these statements. Investors should read the risk factors set forth in Mirum's 10-K for the year ended December 31st, 2022, and any subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris?
Thank you, Andrew, and good afternoon to everyone joining us on the call today. In 2022, Mirum made remarkable progress growing into our leadership position as a high-growth rare disease company focused on commercializing life-changing medicines. While we are all excited about our Q4 quarter-over-quarter sales growth of 48%, we are just getting started. Our clinical, regulatory, and business achievements during the year with net product sales for LIVMARLI of $75 million provide a strong base from which we will rapidly grow our business. We have put in place a five-part strategy to become a global leader in rare disease. First, build on the successful launch of LIVMARLI and Alagille syndrome in the U.S. LIVMARLI is the first and only treatment in Alagille syndrome to provide early improvements with long-term impact.
We are still early in making LIVMARLI available to the addressable Alagille syndrome patient population. In the second half of last year, we expanded our field presence and investment in the U.S. and now anticipate 50% growth in U.S. LIVMARLI sales in 2023. This is driven by the Mirum team and the tremendous potential of LIVMARLI for patients, which was highlighted this year by data showing that in Alagille syndrome patients who had an ICRO response with LIVMARLI, 88% of them remained event-free at six years. Second, grow LIVMARLI in markets outside the U.S. Following European approval in December, we have already launched in Germany, with France to follow shortly and further launches around the globe later this year and in 2024. Third part of our strategy, expand the label for LIVMARLI.
At the end of last year, we presented LIVMARLI PFIC phase III data. This is the largest study conducted in this devastating disease, covering the widest range of PFIC types. In addition to impressive reductions in serum bile acids and pruritus, we saw placebo-controlled improvements in various markers of liver health, namely bilirubin and growth, a first I've seen reported for IBAT clinical studies. We have submitted our sNDA and are excited about bringing this strong data profile to prescribers and patients. On our third potential indication for LIVMARLI, we have also made good progress in the EMBARK study in biliary atresia and expect to announce data in the second half of this year. Fourth, apply our expertise in IBAT inhibition to expand into adult cholestatic liver disease.
Building on earlier IBAT inhibition inhibitor data in primary sclerosing cholangitis and primary biliary cholangitis, we are conducting potentially pivotal studies for volixibat in these two underserved indications. Finally, the fifth part of our strategy, expand our development pipeline for business development activities. We are evaluating a number of interesting opportunities across rare and orphan diseases to leverage Mirum's industry-leading rare disease capabilities. We entered 2023 with great momentum, and I am excited by what the Mirum team can achieve in the near future, providing patients with life-changing medicines that they've been waiting for. With that, I'll pass the call over to Peter to discuss our commercial business in more detail before Pam gives an R&D update. Peter?
Thanks, Chris. We are pleased with the $27.9 million in LIVMARLI net product sales in the fourth quarter of 2022, which represents a 48% growth over the third quarter. Additionally, we reported $75.1 million in LIVMARLI net product sales for the full year 2022, which included $68.0 Million from the United States and $7.1 million from international markets. Needless to say, we are thrilled with LIVMARLI's performance in its first full year on the market, which represents one of the strongest rare pediatric launches in the industry. Now first, some comments on the U.S. business. The growth we saw in Q4 was driven by more new patient starts and a consistent refill cadence, which were both a direct result of our commercial team's strong execution.
We estimate that about 20% of currently diagnosed and addressable patients with Alagille syndrome in the United States have received LIVMARLI since launch. As the results reported today demonstrate, our commercial team has been highly effective at driving the LIVMARLI launch further into the addressable market. We remain confident in continued growth going forward, consistent with our guidance that 2023 LIVMARLI U.S. sales will grow by 50%. Turning to international, the $7.1 million in 2022 sales was predominantly in Q4 and came from partner markets in Eastern Europe and the Middle East, where Mirum's distribution partners were able to engage in pre-approval commercial access programs. The Q4 international sales number included an inventory build as a result of full-year product orders for identified patients in these partner markets.
Looking forward to 2023, we expect to see continued demand growth and sales contribution from partner markets in Eastern Europe and the Middle East, as well as new partner markets coming online in Latin America and Asia, including Korea, where LIVMARLI was recently approved. European commercialization has commenced following the December 2022 European Commission LIVMARLI approval. In early 2023, Germany became the first European country to launch and is off to a great start in the initial weeks out of the gate. In France, a pre-approval access program for LIVMARLI was recently approved, enabling access to commercial LIVMARLI before full reimbursement. Beyond Germany and France, we expect other Western European countries to launch upon completion of pricing and reimbursement discussions later this year and into 2024.
Our LIVMARLI marketing application is under review in Canada, which, if approved, would enable a launch later this year. LIVMARLI is the first and only treatment for Alagille syndrome that provides early improvements with long-term impact on event-free survival. With approximately 600 Alagille syndrome patients treated globally to date, clinicians have gained familiarity with and confidence in LIVMARLI's robust clinical profile, as well as its exceptional access and patient support. We believe this will propel a continued growth story for the Alagille syndrome launch going forward. On that note, I'll turn the call over to Pam. Pam?
Thank you, Peter. 2022 was an outstanding year for our clinical team as we presented meaningful data supporting both our commercial stage and pipeline programs. At the end of last year, we presented groundbreaking phase III data for LIVMARLI and PFIC, in which LIVMARLI drove a statistically significant reduction in pruritus and serum bile acids across the broadest genetic PFIC-type study to date, showing a magnitude of treatment effect which is approximately double from previous studies and exceeded our expectations. Notably, the majority of patients also achieved serum bile acid reductions below the threshold that is associated with transplant-free survival. Furthermore, LIVMARLI also drove early and significant placebo-controlled improvements in growth as well as in bilirubin, suggesting an improvement in the underlying liver health of these patients.
As a reminder, bilirubin is an important indicator of disease progression in cholestatic disease, and EMBARK is the first study to demonstrate statistically significant differences in bilirubin versus placebo in PFIC. It's remarkable that this was observed with only six months of treatment. These unprecedented clinical effects from the EMBARK PFIC study across broad genetic types situates LIVMARLI in a very strong position, and we are thrilled to deliver LIVMARLI to the broader PFIC population pending regulatory approval. As we look to biliary atresia, the bilirubin and serum bile acid reductions observed in PFIC are very encouraging. Both of these markers, particularly bilirubin, are strong prognostic indicators of disease progression, and our primary endpoint will measure the six-month change in bilirubin from baseline. We look forward to announcing this top-line data from the phase IIb EMBARK study in the second half of this year.
In the adult cholestatic indication, there's a similar significant unaddressed burden of cholestatic pruritus, which is associated with a buildup of toxic bile acids severely affecting quality of life in these patients. In the settings of both PSC and PBC, about over 60% of patients have active pruritus, of which about 80% or more are being treated with off-label anticholinergic medications that are largely ineffective or offer only partial response. The urgent need for new options is highlighted by a PSC survey we presented last year, where 75% of patients using two or more medications described only partial or no relief. Given the significant burden of these diseases, we're really excited to continue advancing volixibat in PSC and PBC with interim analyses expected later this year.
Lastly, I'm really proud of our team's academic and collaborative efforts, as well as our continued scientific leadership, which was showcased throughout 2022 through our congress presentations and a number of publications.
Characterizing the benefits of LIVMARLI in PFIC and beyond, mirroring what we're hearing from the real-world experience. 2023 is expected to be another outstanding year. With that, I'll turn the call over to Ian to review our financial results. Ian?
Thanks, Pam. The press release and 10-K filed earlier today provide a full financial update. I'll call out a couple of the highlights here. First, from a revenue perspective. Total revenue for the year ended December 31, 2022 was $77.1 million, including license revenue of $12 million or $2 million versus total revenue of $19.1 million for 2021. Of note, this is slightly higher than the $76 million preliminary estimate we announced back in January. Turning to operating expenses. Total operating expenses, including cost of sales for the year ended December 31, 2022, were $208.3 million versus $192.6 million for the prior year. Mirum remains well-funded with over three years of runway.
At the close of the year, we had $251.7 million on the balance sheet. Additional details on all of our operating expenses and our cash balance are provided in our 10-K filing. Overall, our financial position is strong and supports our continued growth and value creation across both our global commercial business and development pipeline. With that said, I'll turn the call back over to Chris. Chris?
Thanks, Ian. Mirum is poised for continued growth throughout the years ahead as we execute on commercial, regulatory and clinical value creation. We're in the midst of a strong launch in Alagille syndrome, growing the US business while launching in Europe and partner markets and unlocking expansion opportunities for LIVMARLI and volixibat ahead of us. We expect 2023 will be a catalyst-rich year. In addition to the continuing strong commercial growth for LIVMARLI adoption in Alagille syndrome, we have the potential PFIC label expansion and data readouts for our pipeline programs as we get further into the year. True to our name, Mirum, which comes from the Latin word for remarkable, our recent accomplishments and anticipated growth ahead in 2023 are full of unique potential as we work to bring important new medicines to patients around the world. With that, operator, please open the call for questions.
Absolutely. If you would like to ask a question, please press star followed by one on your telephone keypad. If for any reason you would like to remove that question, please press star followed by 2. Again, to ask a question, press star one. As a reminder, if you're using a speakerphone, please remember to pick up your handset before asking your question. We will pause here briefly as questions are registered. The first question is from the line of Jessica Fye with JPMorgan. Your line is now open.
Hey, guys. Good afternoon. Thanks so much for taking my questions. I was hoping you could provide us a little bit of a framework for how to think about Europe sales this year. Not sure if there's any, anything you can provide on that front. Second, I think in prepared remarks you mentioned that 20% of currently diagnosed and addressable patients have received LIVMARLI since launch. Can you talk through how we get to $500 million peak sales for LIVMARLI and Alagille in the U.S. based on what we saw for 4Q sales? In particular, I'm sort of curious what market share is assumed in that forecast, and also how much upward drift in price per patient is anticipated due to growth. Thank you.
Okay. Thanks, Jessica, for the question. Give a couple of intro comments and I'll let Peter speak to some of the details in particular on how Europe launch will roll out and some of the market dynamics in the U.S. I think, you know, just pointing to some, you know, pretty quick and easy math on Q4 U.S. revenue is a little bit of rounding to this, but you know, that's pretty close to a $100 million run rate at 20% penetrated. I think you can kinda get the picture of how that lines up with the $500 million or greater market opportunity that we talk about for the U.S. With that kind of just quick backdrop on U.S., I'll hand it over to Peter to maybe pick into the Europe question.
Sure, sure. Happy to, happy to comment on that. I think as you think about the international piece for LIVMARLI in 2023, you know, there's probably 3 components to think about. The key driver will be Germany. As I mentioned, we're in the market in Germany now, had a really strong start in the first handful of weeks. That's really the one country we'll be in where we have a kind of access to the full healthcare system, a full reimbursement. The second kind of bucket for 2023 is really named patient sales program or pre-reimbursement approval programs. I mentioned we have one in France that's opening up with commercial LIVMARLI. There's also a couple others potentially in Western Europe as well as partner markets.
That's those kind of programs are what drove our Q4 2022 number and, you know, opportunities to participate in those programs even in new geographies like Latin America. You know, those are a little bit less predictable. We have clear visibility to high unmet medical need. We hear from clinicians who have patients who are really, really interested in LIVMARLI. So the clinical side is high visibility, but it's really kind of country specific pricing reimbursement mechanisms that have to be navigated there.
That's the second bucket. I think the third bucket I'd kind of call out for 2023 is new country launches. In Western Europe, there's a, you know, we might have some new countries coming on at the end of the year or into 2024, depending on how pricing reimbursement timelines go. I mentioned Canada, which if approved by Health Canada, could support a launch with, you know, with contribution from the private market to Canada at the end of the year. There's a couple in that third bucket. There's a couple markets where you could start to see contribution in late 2023, but really more likely 2024.
Thank you.
Thanks for the question.
Thank you for your question. The next question is from the line of Mani Foroohar with SVB Securities. Your line is now open.
A quick question, more on the pipeline. As we think about the sort of portfolio of readouts that you're coming throughout this year on phase II, on what time horizons should we expect to get a little more clarity on what is and is not pivotal, just relative to when we're getting the data? I know there's a little bit of uncertainty around exactly what a pivotal endpoint might be, for example, in biliary atresia, et cetera. How should we think about the gap between data and when we'll get a little bit of clarity on what an approval endpoint is or isn't?
All right. Thanks for the question, Mani. I can touch on a little bit of context to put on these on these readouts. I'd point out that biliary atresia for LIVMARLI, you should think about it differently from the volixibat interims. First commenting on the biliary atresia readout, we won't know at the primary analysis readout later this year. That's gonna involve some conversations with FDA interpretation of the data, building our case to understand what the next step is for that program, whether it's putting together a submission or planning for another study. The volixibat program, though, these studies are very different in their positioning as potentially pivotal studies.
We have already aligned with FDA on using pruritus as the registrational endpoint and the analysis plans for both of these studies. Think of these upcoming interims as really the dose selection to move into the pivotal portion. When each volixibat study gets to its interim analysis, they essentially convert into pivotal studies for that adaptive part 2. We know once we've crossed that interim, as long as the study's continuing, it is a pivotal study in all kind of forms in the input from FDA that you want for a pivotal study.
Great. That's really helpful. Thanks, guys.
Great. Thanks for the question.
Thank you for your question. The next question is from the line of Josh Schimmer with Evercore. Your line is now open.
Great. Thanks so much for taking the questions. First, for the commercial spend, how should we think about the step-up as you add new indications to the franchise via LIVMARLI or volixibat? Then, for either product, but I guess LIVMARLI, any updates or progress identifying the path forward for the ICP indication? Thank you.
Great. Thanks for the question, Josh. I will. Actually, I'll pass it over to Peter to talk a little bit about commercial spend across the indications and products. But first I can comment on the ICP question in the interim. Really interesting data that came out of the OHANA study, albeit, you know, very small patient numbers we that we were looking at. But you can tell that volixibat's active for these patients. It addresses the pruritus, just a near impossible setting to conduct robust clinical studies in. We are seeing interest in potential investigator or sponsors approaches. We'll continue to explore that, but kind of until those come together, don't have a formal update on the ICP program. Peter, you wanna comment on commercial investment? Sure, happily.
You know, I think with LIVMARLI, the short answer is that it's largely stable. I think some of the puts and takes around that as we do geographic expansion with the first indication, ALGS, we've already built a lot of that team and invested in a lot of the commercial activities. There may be some kind of minor incremental things, but nothing big enough with the geographic expansion to change things. As we expand to PFIC, it's really that we're gonna take on the same job piece we're in now, and it's the same call universe. Again, maybe some very modest things that not enough to kinda show up on an overall SG&A number in a meaningful way that we would do to support that launch.
Biliary atresia also taken care of in the same physicians and children's hospital that beat PFIC and ALGS. Really, LIVMARLI is pretty stable. As we move to volixibat, that, you know, that gets us into the adult setting. Obviously, we'll have to have a team that can reach adult GIs and hepatologists. You know, that's we look to leverage the team we have now and build from that as we get to those milestones.
Got it. Thank you.
Thank you for your question. The next question is from the line of Steve Seedhouse with Raymond James. Your line is now open.
Hi, this is Ryan Deschner on for Steve Seedhouse. Curious in biliary atresia, what the timeline is you anticipate for completion of enrollment in EMBARK. Also, how will dosing in the EMBARK phase IIb be handled? Will there be any sort of titration or starting dose?
Ryan, thanks for the question. Actually, I'll pass that over to Pam to give a little color on the EMBARK study.
Yeah. Thanks for that question. We're just super excited about the EMBARK study, given that this will be the first study to read out in biliary atresia with an IBAT into the liver by the end of this year. That speaks to the timing of when we expect the study to read out and complete enrollment. With regard to your dosing question, the dose that we're using in this study is the same as our PFIC dosing, which is 600 micrograms per kilogram twice a day. You know, what we saw in the PFIC study was really remarkable with regard to the bilirubin reduction that were really unexpected. That gives us a lot of encouragement for what we are hoping to see in this biliary atresia study.
The dose is titrated as it is with our other studies, but the step-ups are few and early on in the study when patients are on stable dosing throughout the remainder of the, of the six months.
Got it. That's very helpful. Maybe if I could squeeze one more quick one in. Do you expect the biggest seasonality effect as it did in 2022 to come in 3Q, while the other quarters, I guess, going into 2023 remain fairly comparable?
Yeah, what we saw last year, and it's kind of hard to tell if that is a gonna be a recurring phenomenon. The summer travel season was a little more of a thing post-COVID last year. I think it's the simple way to put it. You know, a pretty good chance that we see some kind of effect like that, where there are just fewer visits to start new patients. Important to note that throughout third quarter last year and what we expect for this year is those refills and the persistence to continue to be a real highlight for families on drug, that they stay on drug. It's a real highlight of the performance.
Got it. Thank you very much.
Thank you.
Thank you for your question. The next question is from the line of David Lebowitz with Citi. Your line is now open.
Thank you very much for taking my question. Considering that LIVMARLI is going to be in a competitive dynamic by the end of this year in two different markets, both ALGS and PFIC, how do you see that affecting your commercial approach? Is there any risk of any competition on pricing in the market?
Thanks for the question, David. I'll make a couple comments. I mean, the one thing, though, on pricing is that's not something that we would expect to see, and particularly given some of the nuances of the position that we see in the market, in that LIVMARLI is really well-positioned with such a great lead in terms of prescriber experience. You know, nearly 600 ALGS syndrome kids treated with LIVMARLI over the history of the program. That's a tremendous amount of experience and most of that being overwhelmingly positive. Find ourselves in a position where I think we expect to see more of what we've seen in the launch to date, which is continued new patient starts and strong persistence compliance for patients that are on drug.
With the PFIC indication coming on, really a step up in terms of what you'd expect to see for efficacy from the clinical profile. That's gonna be really powerful. Excited for when our team can go out and talk about that data.
Thanks for taking my question.
Thank you.
Thank you for your question. The next question is from the line of Brian Skorney with Baird. Your line is now open.
Hey, good afternoon, everyone. Thanks for taking my question. On the EMBARK study, I was hoping you'd kind of talk through the relative importance as you see of mean reduction in serum bile acid versus the proportion of patients with total bilirubin below 2 mgs per deciliter. And maybe even in context there, what the average baseline bilirubin you were seeing in EMBARK, as well as what the lower threshold is that you're allowing and also there's another aside from that. Why isn't a 6 mg per deciliter cutoff also being included as a prospectively defined endpoint given the Schneider paper?
Thank you for the question. Basically, in biliary atresia, these kids, after Kasai, most patients continue to experience liver injury from accumulation of bile acids, right? By reducing bile acid retention, you should improve bile flow. Bilirubin is an indicator of how well that bile is flowing. You're looking at both of these as markers. Early on after Kasai, when you can reduce bilirubin, that has been shown, as you're mentioning in the Schneider paper, that if you are two or less, you have a really good chance of maintaining your native liver. If you're between 2 and 6, it's kind of mid-range, and if you're greater than six, you really are headed towards transplant.
That's because the liver is in fact not functioning. Bilirubin being the most predictive marker of transplants, is an indicator of bile flow in liver injury. I think that, as I mentioned, the data that we've seen from our PFIC study, as a result of reducing bile acid retention in that setting and thereby reducing bilirubin, is really encouraging for us on what we're hoping to see in the BA program when it reads out.
Just any comment on what the baseline in EMBARK would be from a bilirubin perspective?
There is no, no threshold. All of these kids are coming in. They're all pretty sick. They've had a Kasai procedure, they've got, you know, inflammation, they've got accumulation of bile acids, and they have elevated bilirubin.
Okay. Thank you.
Next question.
Thank you for your question. The next question is from the line of Ed Arce of H.C. Wainwright. Your line is now open.
Hi, everyone. Thanks for taking my questions. Appreciate it. A few from me. First, you mentioned earlier, there were or have been since launch, about 20% of patients diagnosed and addressable have received LIVMARLI. I just wonder if you could discuss, you know, the difference between that and actual market share now, given there's some that I would imagine some small numbers have cycled on or off. Is that, you know, pretty close to market share? And if not, what are the differences there? Secondly, wondering about PFIC, when you would expect to get the NDA acceptance letter and PDUFA date there.
Lastly, just thinking about the cadence of the three readouts later this year, two in the second half and one in the middle of the year. Wondering if you could perhaps delineate a little more precisely what kind of gaps in between those we could expect or if maybe a couple are expected pretty close? Thanks so much.
Thanks, Ed, for the question. The first one, kind of tick through these here. On the first one, looking at market share, we continue to, for the US business to really just highlight that these are real dispenses in that revenue number. You can look at those numbers as pretty direct, very direct, patient volume being dispensed, real demand. You can get a sense of that number just by looking at the US net revenue number. In terms of some of the kind of milestone timing items, PDUFA date assignment is typically about two months after submission. Expect that to be in the next month or so, based on when we submitted the PFIC sNDA. On the other kind of clinical endpoints or clinical readouts, don't really have any further color to get more precise than kind of mid-year and the two in the second half.
Thanks so much.
Okay, thanks for the question.
Thank you for your question. The final question is from the line of Yasmeen Rahimi with Piper Sandler. Your line is now open.
Good afternoon, team. Before I start my questions, just wanted to say thank you to Ian, and we will greatly miss you. Maybe this might be our last call you're gonna be hosting with us, so wish you the best of luck and thank you again for everything. definitely on behalf of all of us.
Thank you, Yas. Appreciate that.
I really wanted to say that. In regards to my questions, obviously a lot of investors are eagerly awaiting volixibat readouts. I think a fundamental question that comes up is what's gonna be the translation? Obviously, if we get the news that in PSC you can move forward, it's deemed as a pivotal study. Like, can you maybe help us understand sort of how much that de-risks the PBC opportunity and also what that means for you to put more dollars or more into continuing to really be behind volixibat. That's sort of my question for you guys. Thank you again.
Thanks, Yas, for the question. I'll have a couple comments then pass over to Pam to talk a little bit about the end runs and how it relates to the final data. I think the thing I'd say at the outset is there is really strong proof of concept for IBAT in both of these indications already. Some great clinical data from the maralixibat program in PSC showing that you can reduce bile acids and pruritus in patients that have elevations coming in, so the patient population that's in this study. I think we already have really strong clinical de-risking of knowing that an IBAT can be active in these settings on these endpoints. I mean, Pam can speak a little bit to the specifics of the study design and what the end runs mean.
Yeah. Thanks, Yas, for the question. Just to add on to what Chris is saying, in our own PSC experience, we've seen that patients who've had moderate to severe pruritus, and this was in an open label study with 70% itch reduction and almost 50% serum bile acid reduction in that population. Again, showing that we know what this mechanism can do in this disease, and frankly, also seeing analogy on PFIC across cholestatic diseases. Now if you look to our statistical assumptions for the studies in the interim, for PSC, the interim includes 45 patients, and that is triggered when all patients have reached week 12. That means some of the patients will be at week 24, some will be beyond, but all patients will have reached week 12.
This is a really great way for us to look at observational differences, and that will help us assess if we're on the right track for the final analysis. The interim is not powered for significance by design, but it allows us to determine if we're in the zone for a positive final analysis. A couple of notes on our kind of confidence in the way that we've conducted the design or that we've set up the design is that the 10x data in PBC showed statistical significance in their analysis when they looked at baseline over time.
They only had about 20 patients in each of those cohorts. That was seen in their BID dosing. I mean, importantly, that was seen in their high dose and their BID dosing regimen. We think given our dose, given our assumptions, given the number of patients we have, we're really confident about our assumptions and may even be overpowered.
Thank you so much, Pam.
Does that answer your question?
Yes, that was extremely helpful. Thank you, Pam.
Great.
Great. Thanks for the question.
Thank you for your question. There are no additional questions waiting at this time. I'll pass the conference back to Chris for any closing remarks.
Great. Thank you, operator. I just want to say thanks again to everyone for joining today's call. I hope you have a great day. Goodbye.
That concludes the conference call. Thank you for your participation. You may now disconnect your lines.