CymaBay Therapeutics Inc. (CBAY) Q2 2020 Earnings Call Transcript

CymaBay Therapeutics Inc. (CBAY 0.03%)
Q2 2020 Earnings Call
Aug 10, 2020, 4:30 p.m. ET

Contents:

• Prepared Remarks
• Questions and Answers
• Call Participants

Prepared Remarks:

Operator

Greetings, and welcome to CymaBay Therapeutics second-quarter 2020 earnings conference call. [Operator instructions] As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Dan Menold, vice president of finance. Please proceed, sir.

Dan Menold -- Vice President of Finance

Thank you, operator, and good afternoon, everyone. I hope that you've had a chance to review the press release we issued announcing our second-quarter 2020 financial results and business updates. You can access that release on our website under the Investors tab. Joining me on the call today are Sujal Shah, chief executive officer; Dr.

Chuck McWherter, chief scientific officer; and Klara Dickinson, chief regulatory and compliance officer. Sujal will provide an update on recent progress and plans on the development program for seladelpar, as well as a brief summary of our financials before we open the call up for Q&A. Before we begin, I'd like to remind everyone that statements made during this conference call, including the Q&A session, relating to CymaBay's expected future performance, business prospects, events or plans, including clinical plans, regulatory approvals and anticipated time lines and data release dates and cash runway are forward-looking statements as defined under the Private Securities Litigation Reform Act of 1995. Although the company believes that the expectations reflected in such forward-looking statements are based upon reasonable assumptions, actual outcomes and results are subject to risks and uncertainties and could differ materially from those forecast due to the impact of many factors.

The company assumes no obligation to update or supplement any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law. Participants are directed to the cautionary statements set forth in today's press release, as well as the risk factors set forth in CymaBay's quarterly and annual reports filed with the SEC for factors that could cause actual results to differ materially from those anticipated in the forward-looking statements. This conference call is the property of CymaBay, and any recording or rebroadcast is expressly prohibited without the written consent of CymaBay. At this time, I'd like to turn the call over to Sujal.

Sujal Shah -- Chief Executive Officer

Good afternoon, and thank you for joining us. Given our discussion of enhanced data and plans for continued development of seladelpar in PBC just last week, our prepared remarks today will be brief and will focus on four specific topics, key achievements in the first half of the year, planned development activities, upcoming milestones and summary financials. Over the past two months, we have experienced quite a reversal of fortunes, triggered by the conclusion of an in-depth, rigorous and independent analysis and review of safety data in the second quarter from a Phase 2 study of seladelpar in patients with nonalcoholic steatohepatitis, or NASH. First, the FDA lifted all clinical holds across all three liver diseases in which seladelpar had ongoing clinical studies that were halted late last year, namely NASH, primary biliary cholangitis, or PBC, and primary sclerosing cholangitis, or PSC.

FDA took this action in short order following receipt of CymaBay's submission of complete responses for each indication that included an independent report from a panel of some of the world's most renowned hepatopathologists and hepatologists expert in NASH and drug-induced liver injury and the supporting data package that led to their unanimous conclusion that there was no clinical, biochemical or histological evidence of seladelpar-related liver injury in the NASH Phase 2 study and that pending FDA review, clinical development of seladelpar should resume. Then barely over a week after the FDA lifted all clinical holds of seladelpar, we unblinded and reported positive top line results from ENHANCE, a global Phase 3 study of seladelpar in patients with PBC that was terminated early, in which the amended primary and two key secondary endpoints were met with statistical significance. For patients on seladelpar 10 milligrams, the results included a nearly 80% response on our primary composite endpoint and accepted regulatory approval endpoint versus 12.5% on placebo, almost 30% response on ALP normalization versus zero on placebo and a meaningful and significant placebo-controlled effect on reducing pruritus, a key clinical symptom of PBC, all in just three months. In addition, seladelpar appeared to be safe and well tolerated in this study.

We believe these data continue to support the potential for seladelpar to be a breakthrough therapy for patients with PBC. And I'll remind you that in the U.S., seladelpar has breakthrough therapy designation from the FDA. And in Europe, prime access designation from EMEA for PBC. The full details of this data were discussed last week.

And a recording of the call is available on our website. The full set of data from this study are being reviewed in detail and will serve to inform design considerations of a new Phase 3 study. We believe the results from ENHANCE, results from previous Phase 2 studies and our growing safety experience with seladelpar in patients with PBC have the potential to support a smaller overall study focused on comparing seladelpar 10 milligrams with placebo. We are confident that once finalized and approved by regulatory authorities, both in and outside of the United States, the global Phase 3 study will be one that we can utilize our experience to enroll and execute expeditiously.

We are moving quickly to finalize the design of this study, submit it to regulatory agencies and begin study start-up activities that will continue through the remainder of this year with the expectation that enrollment in a new Phase 3 study is likely to begin in the first quarter of 2021. We also hope to have the opportunity to present data from ENHANCE at an upcoming medical meeting, potentially at AASLD in November. In addition to the Phase 3 study, we plan as quickly as feasible to reinitiate the long-term study also terminated last year. This will provide the more than 100 patients previously treated with seladelpar in this study to once again receive treatment with seladelpar, while allowing us to continue building the safety database in support of an NDA.

We intend to allow all PBC patients previously enrolled in a seladelpar study the opportunity, should they qualify and have support of their treating physician, to participate in our long-term study, in a new Phase 3 study or in any of the other NDA enabling or supportive studies we initiate to support registration. For each of these studies, we also plan to incorporate necessary procedures to ensure patient safety in consideration of the current global pandemic involving COVID-19. Turning briefly to seladelpar programs beyond PBC, let me say a few words about PSC and NASH. When we halted development of seladelpar late last year, we had just begun a dose-ranging Phase 2 study of seladelpar in patients with PSC, a rare orphan cholestatic disease that shares some attributes with PBC, but for which key differences can make it a more heterogeneous and complex disease to treat.

Many patients with PSC also suffer from inflammatory bowel disease, have significant liver inflammation and fibrosis and have a significant risk for developing cholangiocarcinoma. We continue to believe the anti-cholestatic, anti-inflammatory, and antipruritic effects observed with seladelpar in PBC and the potential antifibrotic effects of seladelpar warrant exploring its safety and efficacy in patients with PSC. While we will remain highly focused this year on reinitiating the development program for seladelpar in PBC, we will continue to consider how and when we may look to reinitiate our development efforts in PSC and provide updates appropriately. There are currently no approved treatments for PSC.

And we continue to believe there may be a significant opportunity for us to explore seladelpar in this indication. With respect to our development of seladelpar in NASH, earlier this year, we shared top line results for seladelpar on endpoints of NASH resolution and fibrosis in our Phase 2 study. The effects on the two key endpoints accepted by regulatory authorities for registration in NASH was particularly encouraging for the seladelpar 50-milligram dose group versus placebo. For patients in the seladelpar 50-milligram dose group, 26.1% experienced NASH resolution with no worsening of fibrosis and 37% experienced at least one-point improvement in fibrosis with no worsening of NASH versus 8 and 20% for these two endpoints, respectively, for patients in the placebo group.

We believe these data along with the improvements in lipids and overall safety and tolerability profile observed for seladelpar in this study and the ease of its administration as an oral agent position seladelpar well to be combined with other complementary mechanisms being evaluated in NASH today, particularly those that have demonstrated effects on weight loss and reductions in total liver fat. Given the multifactorial nature of NASH and the significant resources required to establish safety and efficacy in clinical studies, we believe a thoughtful combination strategy and the potential opportunity to advance development through partnerships or collaborations is not only warranted but would be the most optimal development path forward. We will look to upcoming medical meetings once again, potentially AASLD in November, as an opportunity to share the full data from this study. And in parallel, we'll evaluate the best strategic path forward while our focus and capital resources remain on reinitiation and completion of the development program for seladelpar in PBC.

Finally, I'd like to highlight a brief summary of our key financial highlights from the second quarter. We have concentrated intensely on reducing expenses since the end of 2019, and the impact of this effort was particularly evident in the second quarter by the magnitude of our overall cost containment. While still in the midst of our investigation of NASH findings and expert panel review, we had guided to a June 30, 2020, expected cash balance of between approximately 156 to $161 million. This forecast was based on a six-month expected cash burn of between 30 and $35 million, incorporating costs associated with termination and closeout of contracts and clinical studies, severance benefit payments associated with previously announced headcount reductions and expenses for ongoing operations that were largely focused on the investigation and regulatory efforts that ultimately led to a lifting of the clinical holds for seladelpar, as well as advisory and consulting expenses associated with our ongoing evaluation of strategic options.

The financials that we report today reflect the focus and discipline of how we operate our business. We successfully dealt with the unexpected and rapid challenges of the coronavirus pandemic, delivered on resurrecting the promise of seladelpar for patients and our shareholders, while sharply decreasing the second-quarter cash expenditures to $7.3 million. This translates to a total cash burn during the first six months of the year of approximately $22 million, greater than 25% lower than the low end of our prior guidance. While some of this cash savings is due to a temporary delay in the timing of certain clinical trial shutdown costs, a portion also reflects certain cost savings due to the company's rapid resolution of the clinical hold on the seladelpar program and the completion of our review of strategic options much sooner than initially planned in our cash forecast.

Cash, cash equivalents and short-term investments totaled $168.9 million at June 30, 2020. We have not raised capital since the first quarter of 2019, and we believe our current cash is sufficient to fund our current operating plan, including the reinitiation of the full development program for seladelpar in PBC into 2022. Due to the ongoing impact of the global coronavirus pandemic, we continue to conduct operations remotely for all employees, which has allowed business activities to continue as seamlessly as possible. To date, these developments have not had a significant impact on our financial condition or our ability to execute our business plans.

We will continue to closely monitor pandemic development and their associated risk to the business including plans to restart clinical development of seladelpar, and will continue to take actions available to mitigate them where possible. Further, all of our actions will be guided by a commitment to taking all steps possible to ensure the health and safety of our employees, as well as patients enrolled in our clinical studies. We're now happy to take questions. Operator?

Questions & Answers:

Operator

[Operator instructions] Our first question comes from Yasmeen Rahimi with Piper Jaffray. Please proceed with your question.

Paul O'Brien -- Piper Jaffray -- Analyst

Hi. Paul on for Yasmeen. Congrats on all the progress this quarter. So we have two questions today.

First, what do we know about the market size for UDCA-treated patients, who are biochemically controlled but still experience significant level of pruritus? And second, connected to that question, how could an additional cohort be incorporated into the upcoming Phase 3 PBC trial to support an additional label indication of pruritus reduction in PBC-treated biochemically controlled patients?

Sujal Shah -- Chief Executive Officer

Thank you for the question. So, let me maybe start off and perhaps invite other team members to add any additional color. We've not really done an in-depth analysis fundamentally around what we would consider a commercial opportunity for pruritus, particularly patients that may be adequately treated, as you're asking, on UDCA itself. I think what I can point to, however, is the fact that despite a good portion of patients having some benefit and even those having benefit that deemed adequate to UDCA, UDCA is not, in fact, as you know, been associated with necessarily improving the symptom of pruritus in patients with PBC.

So, we do believe that overall, with the opportunity to potentially have an indication or a label claim or even data in a final Phase 3 study for registration that supports what we've seen thus far in at hand where seladelpar showed the significant benefit on reducing pruritus relative to placebo at the 10-milligram dose group, there may, in fact, be opportunities for us to continue to expand use in the overall PBC population. Now, I think part of your second question is whether or not there may be ways for us, of course, to incorporate this endpoint, if you will, or potentially another arm in the next Phase 3 clinical study. And so, here, perhaps what I'd like to be able to highlight is the most significant need today remains for these patients that remain at risk of disease progression. So, those patients that are either inadequate responders to UDCA or are intolerant, continue to have a significant need for a safe, efficacious, well-tolerated second-line treatment alternative.

Fundamentally, we want to do everything we can to accelerate the execution of a new Phase 3 clinical study to get seladelpar registered for this patient population. Now we will certainly be exploring the measure of pruritus in the next Phase 3 study as we have in ENHANCE. And that, along with other potential strategies, could, in fact, allow us to have multiple avenues to explore the effects of pruritus, not just in the second line treatment alternative setting, but also for patients at large. These will all be considerations that I think our team focuses in on with experts, with patient advocacy groups as we think about the overall development program for seladelpar moving forward.

Paul O'Brien -- Piper Jaffray -- Analyst

OK. Thanks so much for your answer.

Sujal Shah -- Chief Executive Officer

Thank you.

Operator

Our next question comes from Steve Seedhouse with Raymond James. Please procee with your question.

Steve Seedhouse -- Raymond James -- Analyst

Thanks so much. Two questions. First one is just on the PBC Phase 3 study with respect to running a study of 10 milligrams versus placebo, that enables a smaller Phase 3, could enroll quicker. Just curious if you can talk about whether or not the data you already have from titration or lower doses would still support those options in the eventual label, even if you don't have those arms in Phase 3?

Chuck McWherter -- Chief Scientific Officer -- Analyst

Steve, Chuck McWherter here. Yes, we do think we have a considerable data set with both safety and anti-cholestatic effects in our open label study. So, the 50-milligram group really carried -- sorry, the five-milligram group carried over more than 50 patients through a year with good durability and response. And so, I think that that really would -- if we need it, a lower dose, we would be able to have that available.

Our view though is the 10-milligram growth dose really shine. I mean, it shows really, as you've seen in the data set that we released, really great effects on alkaline phosphatase, some decreases in bilirubin and a host of other liver chemistries were improved. And the safety aspects of the drug really support it going forward as the preferred dose for patients.

Steve Seedhouse -- Raymond James -- Analyst

Makes sense. There's no reason to go lower, I guess, if the safety profile is just fine at 10 milligrams. And the second question is, you spoke, Sujal, confidently about PSC on this call, whereas some of your recent updates focused on -- more on PBC and obviously, that being the priority. But now that PSC is -- looks like a second priority for you, can you just talk about if you have any data from patients treated in the terminated Phase 2 study? If that's informing your confidence here or -- and/or if you've seen an evolution in, really, in understanding of what endpoints matter most and the type of trial design that the FDA would prefer, or does that last part remain a bit unclear still?

Sujal Shah -- Chief Executive Officer

Yes. Thank you for the question. It's a good question. I think first, I'd start off with the fact that I don't think our perspective, fundamentally, despite the challenging situation we've been through over the last nine months, really changes our outlook on PSC relative to where it was last year when we had embarked on the Phase 2 dose-ranging study in that setting.

I think we're largely encouraged by the fact that as a cholestatic liver disease, we are likely to see some benefit for patients, certainly on improving cholestasis given the mechanism of seladelpar and what we see in terms of reductions in transaminases in the PBC population. We're encouraged by the potential that seladelpar may in fact be also highly anti-inflammatory in what is cholestatic disease with a greater degree of inflammation and even fibrosis. I think one nuance point is the initiation and progression of fibrosis in NASH is quite unique to NASH and not necessarily the same as what occurs in PSC. Nevertheless, the mechanism and even what we've seen in NASH, I would say, it gives us some encouragement about the potential to see some antifibrotic benefit even in a PSC population.

Again, I caution that the etiology of fibrosis is unique in these two diseases. But I do -- I would say that we are, in fact, encouraged by what we've seen now across both now the PBC and NASH populations. So really, as we think about our focus in rare orphan cholestatic diseases, where there remains significant unmet needs for patients, that's really the key driver in our thoughts around continuing to expand seladelpar in -- from PBC into PSC. And I think as I mentioned in the prepared remarks, the key thoughts on our end are just about timing and how we would actually progress.

And I think it's a bit premature for us to be able to provide specific guidance there. But this is a patient population that, again, we do believe there's a potential for us to explore development.

Steve Seedhouse -- Raymond James -- Analyst

OK. And just a follow-up, did you have any data from the terminated study, or is there just not enough there?

Sujal Shah -- Chief Executive Officer

No. We had actually only randomized a single subject in that Phase 2 study when we halted the development at the end of last year, and it was a subject actually on placebo, so no data in that population as of yet.

Steve Seedhouse -- Raymond James -- Analyst

Thanks so much appreciate it.

Sujal Shah -- Chief Executive Officer

Thank you, Steve.

Operator

Our next question comes from Ellie Merle with Cantor Fitzgerald. Please proceed with your question.

Ellie Merle -- Cantor Fitzgerald -- Analyst

Thanks so much for taking my question, and congrats on all the progress. Just in terms of development plans in NASH, it seems like a collaboration approach is something that you're exploring. What could make that change where you would consider developing seladelpar in NASH by yourself? I mean, I know that it's a complex space with large studies, but you did see some encouraging histology data on fibrosis and NASH resolution. So, curious sort of what could change your outlook in terms of developing it yourself versus in collaboration? And then also, just in terms of the learnings of PPAR-delta biology.

From what we learned from your NASH pathology data versus what we've seen in NASH from other PPARs, such as alpha or gamma, what do you think this means for PBC long-term? I know that there's other PPARs in development for PBC as well. So, in terms of what we learned about PPAR biology from the NASH data, what do you think this means long-term for the competitiveness of your data in PBC long term?

Sujal Shah -- Chief Executive Officer

Yes. Thank you for the question, Ellie. Let me answer the first one, and then I'll ask Chuck to provide some color around seladelpar and PPAR delta specifically. Yes, I think when we look at the data set in NASH, as I mentioned, we are, in fact, highly encouraged by what was observed, particularly at the 50-milligram dose group on both NASH resolution and fibrosis, and this was a relatively small study, however.

And as we think about what this data informs us, I think it tells us quite a bit around the ability for seladelpar to provide some of the key benefits that NASH patients would require to treat the overall disease. At the same time, the totality of the data also gives us a pretty clear understanding. And I think this is the case, frankly, I'd have to say for other agents in development, as well around thoughts on what would be the best combinations in order to truly have a substantial benefit in a population of NASH patients, and I talk about this specifically as showing some significant in meaning benefit relative to placebo alone, which I'd argue, many agents have either shown no benefit or a marginal one. So much of our thought actually comes from the fact that if we were to design the most optimal development strategy, it would in fact be one in which we have some better understanding around complementary mechanisms to seladelpar to drive the kind of response that we believe you really want to be able to see to open up on this large patient population.

Now, of course, there's no question that this also requires significant resources. But it's a combination of both the most optimal resource, as well as the most optimal development plan that really informs us of wanting to make sure that we have this opportunity to explore some of these potential combinations.

Chuck McWherter -- Chief Scientific Officer -- Analyst

Ellie, this is Chuck, maybe I'll just kind of add in some additional information around different profiles for PPAR. The first thing I would want to caution everybody is about some of the challenges of making comparisons across studies where they're -- it's -- they're not head to head. Sometimes, there can be even minor differences in the population, so it always comes with some big qualifiers. I would say that if you looked at Resolve-It versus our Phase 2b -- as you know, in Resolve-It, there was no effect on fibrosis, where although we didn't hit statistical significance either, I think due to the small size of the study, only 46 in the 50 milligram group, it was very heartening to see 37% of patients with an improvement in one stage in fibrosis versus 20% in placebo.

And then, if you focus on some of the differences between an alpha delta and a strong delta, I point out the effects that we see on, for example, markers of liver injury. So, all reductions at a year, were about 38 units per liter with seladelpar and 50 milligrams placebo was very minor. And that is very different than what was seen in either Golden 505 and or in the GENFIT's Phase 2 study that they presented last year in Vienna. And of course, we haven't seen all the data on Resolve-It yet, so that's to be determined.

And then, if you turn to PBC, I think this is where the story really strengthens for seladelpar and the affected delta. It's not only on bile acid reduction and improving cholesterol disposition, which helps to reduce hepatocellular stress. But the reductions that we've seen in markers of inflammation and stress like ALT, both in ENHANCE and in our open-label Phase 2 study, really I think encouraged us to think that delta really provides everything that the patient will need. If you then think about the pan-PPAR, the lanifibranor and the NATIVE results that were provided just a month or so ago, you see that once again, I think if you look at the fibrosis effects at 37% versus roughly 40% and then the placebo rates were very similar again.

Again, they had a much larger study, and it was a long -- it was a shorter study, it's only a 24-week study. So there, again, I think that what lanifibranor and seladelpar have in common may be the delta component. In fact, I know that there's a publication that just appeared, it's in -- had print in Journal of Hepatology, where the authors basically come to the same conclusion that the anti-inflammatory effects of lanifibranor are driven by delta.

Ellie Merle -- Cantor Fitzgerald -- Analyst

Got it. Very helpful. Thank you.

Sujal Shah -- Chief Executive Officer

Thanks, Ellie.

Operator

Our next question comes from Ed Arce with H.C. Wainwright. Please proceed with your question.

Ed Arce -- H.C. Wainwright -- Analyst

Hi, everyone. Thanks for taking my question, and congrats on a remarkable turnaround in the last few months. I just wanted to perhaps drill a bit further in some of your remarks, Sujal, just now with regards to NASH and your decision strategically to focus on looking to partner that program going forward. And I wanted to see if you could just give us a bit more detail around your thinking in that strategic decision with regard to your overall focus as a company in rare cholestatic liver diseases and how that played a part of your overall decision.

And along with that, if you've had any sort of preliminary discussions with any potential partners.

Sujal Shah -- Chief Executive Officer

Yes. Thank you, Ed. Let me maybe start off by just saying that we're passionate and compelled by the data set we already have in hand for the benefit that we're seeing to date with seladelpar in PBC. We fundamentally believe there is a significant opportunity to advance care for these patients that's not fully reflected, for example, and where even the company is valued today.

We think about those patients and the significant need for those patients, we frankly believe that there's an opportunity to even expand the overall patient population based on the latest profile of seladelpar coming out of ENHANCE. And so, that keeps us incredibly laser-focused on getting seladelpar in the hands of these patients. So when we think about NASH then, obviously, an area of significant high unmet need as well, there still remain a significant number of open questions, whether they're clinical, regulatory or even commercial. I think it's fair to say that a lot of questions we all felt might be answered by now in the setting of NASH remain unanswered.

And so, we have an opportunity with the very clear data set we have in hand with seladelpar in PBC to drive forward and to do the things that we fundamentally need to do to create value and to do so in a very capital-efficient manner. As I mentioned, we're incredibly confident of the overall cash balance we entered the second half of the year with and our ability to really execute our operating plan into 2022. And so we'll remain very focused on this key opportunity while some of these questions in NASH are ultimately answered, particularly, again, as it pertains to regulatory, as well as eventually commercial opportunity. I think as we look at the data, we like to always say that the data really needs to drive our decision-making.

And I would tell you that I think it's the data for seladelpar that keeps us encouraged but also has us focused on thinking of complementary mechanisms to which seladelpar can be combined. And so when we talk about partnership, these can be even collaborations that are nonexclusive. They don't necessarily have to be arrangements with an exchange of economics. They're really driven toward thinking about, again, a thoughtful development approach to getting meaningful benefit in the population.

So, we'll continue to spend some time thinking about these opportunities but, again, won't take away our operating focus on the opportunity that's most near term to bring seladelpar to patients with PBC.

Ed Arce -- H.C. Wainwright -- Analyst

That's very helpful, Sujal. And then, just perhaps one other question on your planned pivotal study for PBC. Realize enrollment is targeted to begin first quarter of next year. When do you think you might be in a position to discuss the full protocol for the study and trial design?

Sujal Shah -- Chief Executive Officer

Yes. It's a good question. Thank you, Ed. I think as I mentioned, currently, what we're making sure that we do is to delve into the data set in front of us now to inform the final study design that we'll put in front of regulatory agencies.

So, we plan to continue to move that forward as expeditiously as possible with the idea that we do want to be thoughtful with the data set we have in hand, particularly the full data set from ENHANCE. I think when we think about time lines through the rest of this year, as I mentioned, the key will be for us in the relatively short-term to put a protocol in front of regulators. As soon as we have any of their input or require needs for any kinds of changes to that protocol or the green light to advance, we'll put it in front of IRBs and ethics committees very quickly. Again, as I'll remind folks, ENHANCE was a study that was conducted in over 100 centers across more than 20 countries globally.

And so our experience here, I'm confident will give us the opportunity to get back up and running as quickly as possible. But we do have to go through those necessary steps, of course, with regulators, ethics committees and investigational review boards. And so, that will be -- those will be activities that remain through the rest of this quarter and early into the fourth quarter as well. I think in the end, it's hard for us to say what the timing would be for those groups to get back to us, but we'll start the study as quickly as possible.

If we get through a process where those approvals come even before the end of this year, there won't be anything that holds us back from beginning enrollment even sooner. I just wanted to make sure that we set the proper expectations around overall timelines.

Ed Arce -- H.C. Wainwright -- Analyst

Fair enough. Thanks, Sujal.

Sujal Shah -- Chief Executive Officer

Thank you.

Operator

Our next question comes from Jay Olson with Oppenheimer. Please proceed with your question.

Jay Olson -- Oppenheimer and Company Inc. -- Analyst

Hey. Thanks for taking the question. I was wondering if you could maybe look into the future at the PBC market dynamics with potentially other new entrants in addition to seladelpar, including Intercept's own combination of OCA with Bezafibrate, and just speculate as to how you think those market dynamics may shake out.

Sujal Shah -- Chief Executive Officer

Thank you for the question, Jay. I think when we think about the unmet needs for patients today, we know that there are patients that remain inadequate responders even to second line treatment. We also know that patients continue to suffer from symptom burden. So, overall, those are two of the key things that we know from a rich set of data with seladelpar, particularly relative to some of these other agents, as you mentioned, that are looking to be explored in the population.

This is a rich set of data that continues to provide us confidence. In fact, I believe that the data set we have in hand relative, for example, to things like Bezafibrate or even elafibranor, where sponsors have discussed advancing these treatments into development, I would argue that our data set in terms of overall patients studied in Phase 2 and even in our ENHANCE Phase 3 study is a more rich data set overall in a very well controlled global study. So I can't speak necessarily to what those agents may show, I don't have that forward crystal ball, but I can tell you that the experience with seladelpar for patients is one that's been very positive. There's a tremendous amount of experience with physicians, as well as patients globally with seladelpar, and I think that gives us a tremendous advantage as we look to reinitiate development.

Maybe I'll ask Chuck to provide some of his thoughts on top of that as well.

Chuck McWherter -- Chief Scientific Officer -- Analyst

Yes. Thank you, Jay. So, just to add a little bit to what Sujal was describing, I think one comment to think about is some of the combination strategies, for example, Bezafibrate and OCA, are really trying to go after subjects who are not receiving a complete response on the single agent. So, it's designed to address maybe a shortfall or a perceived shortfall in terms of benefit to the patient.

And I think seladelpar's profile, as exhibited in ENHANCE, really speaks to its ability to help address that at least in some aspects. Another thing to consider, and we alluded this -- to this earlier, with the trend among medical experts to think about lower alkaline phosphatase is always a good goal. If you can normalize it, that's an advantage. So, there are currently patients who have alkaline phosphatase that are below, let's say, 1.67 upper limit of normal are candidates for entering clinical studies.

They still carry risk, they still itch, and so I think seladelpar's profile as we think about potential strategies with clinical data to expand the addressable population, I think, really bodes well. If we could, for example, in a life cycle management approach, think about exploring effectivity in patients who still have elevated alk phos with a drug that's well tolerated, that would be an advantage over some of the other agents that you alluded to that carry some tolerability issues. It's more challenging for them to go into lower-risk populations where maybe the tolerability doesn't offset the benefit as much.

Jay Olson -- Oppenheimer and Company Inc. -- Analyst

That's super helpful. Maybe as a follow-on, I was wondering if assuming that seladelpar's efficacy, safety and tolerability profile continue to look as favorable as they do in your registrational study, would you consider studying seladelpar in a first-line setting potentially in patients who can be identified in advance as potentially unresponsive or intolerant of UDCA?

Chuck McWherter -- Chief Scientific Officer -- Analyst

Yes. I absolutely think that our intention is really to understand the benefit and the safety of seladelpar in PBC patients, first, in the intended population for the program that we're pursuing right now but, ultimately, in the broadest population for which there might be benefit. And so, I'm not here to say that we're going to try to establish that it's frontline therapy at the moment, but I wouldn't rule it out. I think it would be quite interesting to take patients who are naive to UDCA and conduct a study to see what the benefit might be.

And I think that physicians and patients alike would be interested in the outcome of a study like that.

Jay Olson -- Oppenheimer and Company Inc. -- Analyst

Great. Super helpful. Thanks for taking the question.

Operator

Our next question comes from Thomas Smith with SVB Leerink. Please proceed with your question.

Thomas Smith -- SVB Leerink -- Analyst

Hey, guys. Thanks for taking the questions. Just a couple of questions on PBC. I guess as we look forward to a potential data presentation at AASLD, could you give us a sense of what additional data sets we should be looking for from the ENHANCE study? Could we see additional data looking at symptom burden or the ability to improve pruritus beyond just the NRS data that you presented in the top line release? And then, could you also just remind us, besides the new Phase 3 study, which trials or clinical activities remain as kind of the gating factors that you would need to complete prior to submitting an NDA for PBC?

Chuck McWherter -- Chief Scientific Officer -- Analyst

Yes. Thanks for that question. Yes. I think we're going to be receiving additional data sets now, so the data set that we received a little more than a week ago were the top line data.

So, we would have an expectation to be able to review a variety of information, including exploratory biomarkers, patient symptom responses, things like that, it -- that -- I think that we'll try to understand first. So, we'll let the data drive what's the most interesting that we would like to combine with what we've already released. But I think you can look forward with anticipation to really, I think, a very nice data set, and it remains to be seen whether it will be accepted at AASLD, but that's really our hope and our intention.

Sujal Shah -- Chief Executive Officer

Maybe I'll ask Klara, our chief regulatory officer and compliance officer, to talk a bit about the overall development program in some of these other studies.

Klara Dickinson -- Chief Regulatory and Compliance Officer

Thank you. Thank you for the question. Some of the additional studies are just the ancillary studies that most companies have to conduct. One is renal impairment studies, drug-drug interaction studies, looking at your -- for your to-be-marketed formulations, food effect studies.

And then, we do have, also, a specific study that's looking at hepatic impairment in our patient population. So, patients with PBC and there is a classification based on Child-Pugh's data.

Thomas Smith -- SVB Leerink -- Analyst

Got it. That's helpful. Thanks very much.

Operator

Thank you. At this time, I would like to turn the floor back to management for closing comments.

Sujal Shah -- Chief Executive Officer

Thank you. I'd like to make just a few comments as we close today. It's been a remarkable year to say the least for CymaBay, for our patients and for all of our stakeholders. In our most difficult days at the company, we found the will to investigate, to analyze, to learn, to forge a path forward and simply just keep fighting to make progress from the patients who we left behind late last year.

Today, I've spent several hours with members of our team speaking to leaders of several PBC and liver disease patient advocacy groups around the world. We have been giving these groups, as well as investigators an update on what we learned and what we plan to do next. I can't even begin to describe how important the human element is in everything we all do every day. Whether our motivation comes from a desire to support our colleagues, patients and their families, the medical communities or those groups who have invested in us at CymaBay to create value, we are grounded by the human element in everything we do.

I can assure you that we will continue to be focused on playing our part, focusing on improving the lives of patients with liver diseases. We'll continue to do so thoughtfully with patients at the forefront of our minds and efficiently to ensure we are meeting our obligations to all of our stakeholders. As I mentioned, we are confident we have the capital needed to reinitiate development of seladelpar. And I expect us to continue achieving key milestones and providing updates in the months ahead and look forward to our next call.

Thank you all again for joining us today.

Operator

[Operator signoff]

Duration: 50 minutes

Call participants:

Dan Menold -- Vice President of Finance

Sujal Shah -- Chief Executive Officer

Paul O'Brien -- Piper Jaffray -- Analyst

Paul OBrien -- Piper Jaffray -- Analyst

Steve Seedhouse -- Raymond James -- Analyst

Chuck McWherter -- Chief Scientific Officer -- Analyst

Ellie Merle -- Cantor Fitzgerald -- Analyst

Ed Arce -- H.C. Wainwright -- Analyst

Jay Olson -- Oppenheimer and Company Inc. -- Analyst

Thomas Smith -- SVB Leerink -- Analyst

Klara Dickinson -- Chief Regulatory and Compliance Officer

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