Mirum Pharmaceuticals Q2 2024 Earnings Call Transcript

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Provided by Quartr
August 7, 2024

There are 16 speakers on the call.

Operator

Good afternoon, everyone, and welcome to the Miron Pharmaceuticals Reports Second Quarter 2024 Financial Results and Provides Business Update. My name is Carla and I will be coordinating your call today. I will now hand you over to Andrew McCabein, Vice President of Investor Relations and Finance to begin.

Operator

Andrew, please go ahead.

Speaker 1

Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Mirim Pharmaceuticals' Q2 2024 Conference Call. I'm joined today by our CEO, Chris Peetz our President and Chief Operating Officer, Peter Radovich our Chief Medical Officer, Joanne Kwan and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Miriam issued a news release announcing the company's results for the Q2 2024. Copies of this news release and SEC filings can be found in the Investors section of our website.

Speaker 1

Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward looking statements based on management's current expectations, including statements regarding Merum's programs and market opportunities for its approved medicines and product candidates. These statements represent our judgment as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10 Q and subsequent SEC filings for more information. With that said, I'd like to turn the call over to Chris.

Speaker 2

Chris? Thanks, Andrew, and good afternoon to everyone. I'm excited to share with you the outstanding progress we've made this quarter with our commercial medicines and pipeline. It's been a strong quarter across the board with continued growth and partner regulatory achievements and positive elixibat interim results. On the commercial side, adoption of our medicines continues to grow with total net product sales of $77,800,000 across the Marley, Kenadol and Colbom, representing a 139% increase from the Q2 of last year.

Speaker 2

Building on the strength, we've also achieved important regulatory milestones for our commercial medicines. We submitted our NDA for quinodiol and CTX, which, if approved, will allow us to take additional steps to reach this under diagnosed population and provide an opportunity for orphan exclusivity. For Lymphmarley, I'm very happy to say that we are now approved for cholestatic pruritus in PFIC in the U. S. And for the treatment of PFIC in Europe.

Speaker 2

And we recently announced a U. S. Label update to reduce that age to 12 months and older. We're looking forward to bringing the Marley to PFIC patients given the impressive clinical impact that we've seen in this population. I'm also excited to announce the initiation of another potentially label enabling study for LID MARLI in cholestatic pruritus.

Speaker 2

I'll let Joanne speak to some of the details, but in short, cholestatic pruritus is not limited to Alagille syndrome, PFIC, PSC and PBC. We see multiple additional rare disease settings where patients develop cholestasis and experience significant pruritus. Supported by high interest from physicians and compelling response case studies, we are launching the EXPAND study to bring LYF Marley to patient communities that would otherwise be challenging to study individually. Collectively, across these settings, we estimate there are at least 500 patients in the U. S.

Speaker 2

Alone that would be eligible for this indication. And finally, we look we took an important step towards advancing valexadat towards potentially pivotal data with the positive interim readouts of the VISTA's PSC and VANTAGE PBC studies. Our VISTAs PSC study exceeded the pre specified efficacy threshold and is continuing enrollment to the full study readout. With no approved therapies in PSC, we are positioned to bring the first medicine to this patient community. The interim results of the VANTAGE study in PBC were also very encouraging with a statistically significant improvement in pruritus and a patient population that spans first and second line PBC.

Speaker 2

I'm happy to say that enrollment is progressing well for both programs. It was a packed second quarter for Mira. So to dive into the details, I'll turn the call over to Peter to start with our commercial business. Peter?

Speaker 3

Thanks, Chris. I'm pleased to report another strong quarter across all three commercial products, and we continue to track well towards our full year year revenue guidance of $310,000,000 to $320,000,000 Starting with LibMarley, total global net product sales grew to $47,200,000 this quarter, which represents a 45% increase compared to the same quarter last year. In the U. S, sales were 35,500,000 dollars while international sales were $11,700,000 Growth continues to be driven by new Algeal syndrome patient additions comprised of both prevalent patients and newly diagnosed, a dynamic we expect to persist going forward. We're also beginning to see prescriptions for PFIC patients and our recent label expansion to include patients 12 months and older provides incremental opportunity given that PFIC is generally diagnosed when children are young.

Speaker 3

Internationally, LID MARLE demand growth was strong. I'm pleased to say that we achieved a favorable outcome in our price negotiations with Germany. Stepping from this, we saw some price reference impact on international sales in Q2. We expect this to run its course in the next quarter or 2. We were also very happy with the European Commission's recent endorsement of Libmarly for PFIC 3 months and older, which highlights a significant benefit of Libmarly for these patients.

Speaker 3

Lastly, we saw nice demand growth from Colbom and Kenadol in the Q2, where we recognized net product sales of 30,500,000 dollars Overall, I'm thrilled with the continued strong commercial performance in the first half of the year and proud of the Miriam team's continued execution. We are on a solid path to achieve our full year guidance of $310,000,000 to $320,000,000 and continued growth. And with that, I'll turn it over to Joanne. Joanne?

Speaker 4

Thanks, Peter. We had an exceptional quarter highlighted by the impressive interim analyses for the VISTA study in PSC and the VANTAGE study in PBC. I'll give a quick recap of the results. Starting with the VISTA study in PSC, we set a pre specified threshold for continuation based on both efficacy and safety. The blinded interim analysis met this threshold and our independent data review committee recommended proceeding with the 20 milligram BID dose and that the study continue without any changes.

Speaker 4

The BLIND analysis confirms a meaningful treatment effect and also allows us to include these patients in the total patient number for the final analysis. We're happy with how the study is enrolling and anticipate completing enrollment in the second half of twenty twenty five. Moving on to the VANTAGE study in TBC, we're thrilled with the interim results. Both doses of elixibat showed a substantial and statistically significant reduction in itch and approximately 2.3. Improvement over placebo.

Speaker 4

Based on this, the VANTAGE study will also continue with the 20 milligram VID dose consistent with the VISTA study. The results support elixibat's potential as an important advance for patients suffering from cholestatic pruritus. We also observed reductions in serum bile acids and improvements across multiple dimensions on the PBC40, most notably fatigue. We look to complete enrollment in 2026 for this larger step. Overall, these results are significant for PBC patients suggesting that meloxicivat has the potential to set a new standard in addressing the burden of cholestasis.

Speaker 4

We've already ramped up enrollment efforts and are targeting up to 100 total sites. Shifting gears a bit, I would like to talk about the new Phase 3 EXPAND study. Miriam has received a number of requests for compassionate use of Marley in patients with cholestatic pruritus across a variety of ultra rare indications. We believe these conditions share a common pathogenic mechanism, cholestasis leading to elevated serum bile acids, which results in persistent pruritus. Based on the good responses we've seen in some individuals receiving compassionate use, we are optimistic that LIVMARY can play a significant role in the treatment of pruritus for these patients.

Speaker 4

EXPAND is a randomized double blind placebo controlled study evaluating LIV Marley for treatment of pruritus over 20 weeks. SPAN study will enroll patients with cholestatic pruritus associated with a range of conditions such as biliary atresia, secondary sclerosing cholangitis and other less common conditions. Our target is to enroll approximately 45 patients and we expect to complete enrollment in 2026. I look forward to providing further updates on Vistas, Vantage and Xtand in the coming quarters. I'll now turn it over to Eric to discuss our financial results.

Speaker 4

Eric?

Speaker 5

Thanks, Joanne. Earlier today, we issued a press release that included financial results for the Q2, which I'll briefly summarize. Net product revenue in the Q2 2024 was $77,800,000 compared to net product revenues of $32,500,000 in the Q2 last year. Total operating expense for the quarter ended June 30 were $102,000,000 which includes R and D expense of $32,700,000 SG and A expense of $49,200,000 and cost of sales of $20,200,000 The total operating expense for the quarter included approximately $17,700,000 of non cash charges, of which $5,700,000 was included in cost of sales. For the quarter ended June 30, 20 4, net loss was $24,600,000 or $0.52 per share.

Speaker 5

Our cash, cash equivalents and investment was $295,400,000 as of June 30, 24, a reduction of $7,400,000 from the end of the prior quarter. Cash used in the Q2 included the payment of a $10,000,000 milestone to Takeda upon FDA approval of the RIMALI PFIC indication. With our robust commercial performance and continued financial discipline, we are in an excellent position to support the development of our pipeline and growth of our commercial business. Now I'll turn the call back over to Chris for final comments.

Speaker 2

Thanks, Eric. It's been a great first half of the year. I'm proud of the Miram team and our strong execution. We are well positioned to continue to advance our 4 strategic priorities: to grow our commercial business, expand the indications of our improved medicines, advance felixibat in adult cholestasis and continue to look for opportunities to grow the pipeline. With that, operator, please open the call for questions.

Operator

We will now begin the question and answer And our first question comes from Dae Gon from Stifel.

Speaker 6

Hey, good afternoon guys. Thanks for taking our questions and congrats on the progress. 2 from us, 1 commercial and 1 clinical, I guess. Starting with commercial, as we look at the approval label across Alagille and PFIC in the U. S, it's kind of a palindrome between LIVMARY and BILVAY.

Speaker 6

So I was hoping if you can maybe comment a little bit on what you've learned from perhaps Bilvay's experience in Alagille that you can implement to broaden LIV Marley's reach within the PFIC segment before further label expansion can come down the pike? And then in terms of clinical side, Joanne, enrollment progressing favorably sounds great. Is there any initiative for you to perhaps accelerate enrollment into VISTUS and VANTAGE trials? Thanks so much.

Speaker 2

Thanks, Taycan, for the questions. On the first kind of a comment on the labeling and label expansion we've seen for LYF Marley and can circle back for any follow-up question. But what I think you're asking is just how the sequencing has gone for the Marley. And it's played out really well. I think we're in a position of kind of very strong leadership in Europe where we're the only products approved for both indications.

Speaker 2

In the U. S, we're now we see it as very equal footing. We're now approved for both indications. Initial reception has been quite strong. And the data for Libmarly in both indications, we think tells a really compelling story for prescribers, and that's what we're seeing play out in the real world.

Speaker 2

So in terms of that kind of label sequencing, I think it's largely played out and to the favor of Marlin. And I'll let Joanne maybe comment on the enrollment strategies.

Speaker 4

Yes. Thanks for the question. Like I said, we're happy with how the enrollment is going. As you've noticed, we've been very excited by the interim analysis results on the PBC and the PSC and we share that with our investigators and they share the excitement on that and really are seeing the potential impact that this has as a medicine for both patient populations. We're continuing to work with our existing sites and continuing the expansion as we previously talked about.

Speaker 4

So we're happy with where our enrollment is

Speaker 2

going. Great. Thank you very much. Thanks for the questions.

Operator

The next question comes from Ani Faroohar from Leerink Partners.

Speaker 2

Hey guys, thanks for taking the question.

Speaker 7

A couple of quick ones. First, when we think about the novel expansion population in cholestatic pruritus, can you give us a sense of how you think about the duration of that study and the time horizon which we might see results, recognizing it's a little bit of a heterogeneous population? And then we have a commercial quick follow-up.

Speaker 2

Sure. Thanks for the questions. And maybe Joanne and Peter take a follow-up.

Speaker 4

Sure. So we are just launching the city now. And as we said, we plan to complete enrollment in 2026. It is a bit of a heterogeneous group of patients, but we expect that some patients will have biliary atresia as a cause, some secondary sclerosing cholangitis and then a variety of other causes. And this is really based on our experience in compassionate use.

Speaker 4

So we're actually pretty confident in terms of our understanding the role of IVAD inhibitors and treatment of polycystic pruritus and think that this extended extends its this potential use to a wide variety of patients who each of these, it would be difficult to study. So we're excited to study them all in this EXPAND study and get some results and hopefully support some wider use.

Speaker 7

That's helpful. And then when you think about sort of more on the commercial side, obviously, there's a little bit of a there's some element of seasonality in this market. And so the pendulum swings one way early in the year, tends to swing the other, certainly around 3Q as we saw a couple of years ago. How should we think about the tempo of new patient adds and any sort of operational details we should think about in terms of seasonality across the next few quarters, looking forward?

Speaker 3

Yes. Thanks for the question. I mean, one thing to just kind of remind on, reflecting back to Q1, we did see in the U. S. With Lidmarly and the bile acid products impact on our Q1 number from the changed healthcare cyber attacks.

Speaker 3

That's one thing to keep in mind as you think about quarter to quarter trends here. And as far as seasonality goes across 3 products, I can't say that we've identified any real seasonality in these products. I mean, they're kind of ultra rare to rare products with relatively low underlying volume, which can lead to kind of quarter to quarter variability, quite frankly. But whether that occurs in 1 quarter versus another, I can't say that there's a really strong effect there.

Speaker 7

Okay. And should we and on what time horizon should we expect sort of the sort of OUS pricing reference dynamics to play out? Is that something we should think about sort of recurring and sort of eroding itself playing out over the course of next couple of quarters? Is that primarily 2Q, 3Q events? Like how should we think about baking that into sort of how we model?

Speaker 3

Yes, exactly. We saw the effect in Q2. We expect it in Q3. And our expectation is by the time you're Q4 that that effect has gone and all of the kind of demand volume growth that we're seeing flows through to the top line.

Speaker 7

Perfect. Thanks guys. I know you got a bunch of people in the queue. I'll hop off.

Speaker 8

Thanks for the questions.

Operator

Our next question comes from Gavin Clarksonur from Evercore ISI.

Speaker 9

Hi, guys. This is Yash on for Gavin. Thanks for taking our question. And we just have 2. The first one, could you just touch on the level of confidence in that 45 patient sample size for EXPAND and maybe how you powered the study?

Speaker 9

And then one follow-up after that.

Speaker 2

Yes. Yes. Thanks for the question. I would think I'll let Joanne kind of comment a bit. But I think the one thing to kind of say to give some context here is that now at this point using IVAT in these full static settings, I think we've seen a pattern here that you can drive really dramatic response if you're at the right dose on bile acids and psoriasis.

Speaker 2

So the thinking behind the study design is based on having actually seen that same profile play out in the number of compassionate use patients. So really strong evidence from the individual case studies that in aggregate basically make this population. So feel good about launching the study. That's why we designed it and that's what compelled us to put it together. Maybe Joanne can speak a little bit about sample size.

Speaker 4

Yes. Thanks for the question. So the sample size was based on powering based on our primary endpoint, which is an observer rated vitro. So this is a scale that we understand well based on our previous experience in the prior studies with Marley. So we feel pretty confident that this is an appropriate sample size for us to see a solid treatment effect.

Speaker 9

Awesome. Thank you. And then one follow-up. Was there any data from IMbark that makes you confident in enrolling BA patients in this trial?

Speaker 10

I'll pass that

Speaker 2

to Doctor. Sharon. Sure.

Speaker 4

Yes. Well, thanks for the question. I think it's important to note that the patient population that we're rolling at EXPAND is actually quite different than the one that we did enroll in a mark. With EXPAND, we're really taking patients really at any point of their journey. And for biliary atresia, many of these patients will have had a number of years ago.

Speaker 4

But over the course of time, their condition deteriorates and they may develop. So this is quite a different population than embark where we took really incident patients around the time that they had a good time. So this is quite a different patient population. And on top of that, we're including other causes of cholestatic pruritus with a chronic disease.

Speaker 2

Yes. I'd also add on that in those compassionate use case studies, there have been biliary atresia patients and they're quite a bit older than the EMBARK age where they're enrolled as infants. And you do see that kind of hallmark response to IVAD treatment. So I think we've now figured out how to dose these medicines and what settings to advance them in.

Speaker 9

Super helpful. Thank you so much.

Speaker 8

Yes. Thanks for the questions.

Operator

The next question comes from Jessica Fye from JPMorgan.

Speaker 11

Hey, guys. Good afternoon. Thanks for taking my questions. I had a few. First, where should we look for presentation of the interim PBC data for volexivat?

Speaker 11

And what kind of additional detail should we expect when you present those results? For example, are there subgroups where we should expect to learn more? Could we see itch results broken down by severity of diseases defined by ALP levels or additional data on liver biomarkers or more details on the improvements observed in the fatigue dimension of the PBC40? And then separately, on the business development front, can you provide a bit of color about just what type of assets and which of these areas are most interesting to you? And lastly, can you just comment on your IP estate across Liv Marley and valexibat in terms of what IP you currently have and any pending applications?

Speaker 11

Thank you.

Speaker 2

Thanks, Jess, for the questions. I can kind of maybe I'll take a shot at the first and the last and then pass it over to Peter to talk about our PD strategy. Your first comment on the further data from the PBC study, We're preparing an abstract. We'll work on getting it submitted for an upcoming Congress and can't really predict when and where that lands and what's in it. So just say that we're looking at a number of the elements that you talked about there to consider for that abstract.

Speaker 2

And then on intellectual property, actually I'll direct you to our corporate deck and the backup slides. We have a summary of that that's been recently added and highlight the 2,040 family of granted and pending patents that really tie back to the quite unique dosing profile for IVAT in general and LOVARLI and valexovat in particular that's led to all of these great advances we've seen across the programs that we're talking about here. And there's more detail in this, in the backup of our public materials there that you can reference. Maybe Peter can talk about BD strategy.

Speaker 3

Sure. Yes, thanks for the question, Jess. We're focused in rare disease. We really like rare pediatric opportunities. Essentially asking ourselves the question, programs where we could add a lot of value, underappreciated programs.

Speaker 3

We have a really strong development, regulatory, commercial group in rare disease. So looking into that kind of a corridor. And we have a high bar. We're very disciplined. We have a really strong base business with a great runway of catalysts in front of it.

Speaker 3

So we take a lot of scrutiny at these opportunities.

Speaker 9

Thank you. The

Operator

next question comes from Mike Ulz from Morgan Stanley.

Speaker 12

Hi, this is Rohit on for Mike. Thanks for taking our questions. Just in PFIC, do you expect to pursue a label expansion to patients below 12 months? And then can you just talk about how many patients are typically diagnosed that are below 12 months and what the opportunity there is? Thanks.

Speaker 2

Thanks, Ryan, for the question. Overall, feel that our we feel that our label now with this PFIC expansion down to 12 months, it's in a really strong place. We are similar to what we do with Allogeneal, we're evaluating the potential to submit yet another SMVA based on the infant data that's now mature, but frankly haven't come to a decision on that yet. So it's something that we could pursue. So I feel like we're in a position now where we capture most of the LIDMARLE targeted patients for both Alagille syndrome and PFIC.

Speaker 8

Thank you. Thanks for the question.

Operator

The next question comes from David Lebowitz from Citi.

Speaker 8

Thank you very much for taking my question. With respect to the EXPAND trial, given the heterogeneous population, how do you think the FDA would view it from a label expansion perspective?

Speaker 2

Thanks for the question, David. I'll ask Joanne maybe to talk little bit about some of the thinking that went into that and discussion with FDA.

Speaker 4

Yes. Thanks, David, for the question. Interesting that you asked the question that way because in fact the study came about because the FDA actually had suggested it. We were receiving compassionate use request and so the FDA at one point said, why don't you put these into a study? So this the expense study has been designed keeping that input in mind.

Speaker 4

And so we do think the commonality is really cholestatic pruritus, cholestasis, elevated serum bile acids and we know the effect that it has that I have on setting. So we're pretty confident in terms of our ability to execute the study and really in terms of the results that we see see once we get executed.

Speaker 8

Got it. Thanks for taking my question. Thanks for the question.

Operator

The next question is from Steven Seedhouse from Raymond James Financial Inc.

Speaker 10

Yes, good afternoon. Thanks for the questions. Just on EXPAND,

Speaker 5

how are you going to

Speaker 10

be measuring pruritus? Because it seems like this trial would include pediatric and adult patients depending on the condition. So I'm just curious how you're going to standardize measuring the endpoint across disease types.

Speaker 4

Yes. Thanks for the question. We have an observer rated ITRO, which has been validated and which we do have experience within prior studies. And that's really designed for pediatric population. So it's right of you to recognize that in a pediatric versus adult population, we can have different endpoints.

Speaker 4

But we feel pretty confident in terms of the design of the study and the selection of the endpoint and the fact that we have experience with this endpoint in Novo.

Speaker 2

Yes. And just to kind of follow-up on that, that pediatric data set, that's the primary cohort, 45 patients. That's where the primary analysis is. So the adult scores will be part of a supplemental cohort and think of the analysis plan designed in that way. Primary is based on pediatric score.

Speaker 10

Okay. And so the adults would be self reported itch or would there be a second observer?

Speaker 4

Yes. So it does.

Speaker 10

How does the endpoint look at both?

Speaker 4

Right. So for the adults, it's a self reported endpoint. And that's one reason for us having them in a separate cohort. Based on our discussions with clinicians and just our experience with compassionate use, we do believe the majority of the patients will be pediatric. But we do think there will be some adult patients and so we're studying them in a supplemental and we'll analyze those 2.

Speaker 10

Okay. And then the formulation here, is it just going to be the same liquid formulation that's commercially available?

Speaker 2

That's right. Yes.

Speaker 7

Okay.

Speaker 10

And then just last question, and thanks for the multipart question. Our biliary atresia patients that enrolled and expand that were maybe pruritic either on enrollment and that's excuse me, that enrolled in Embark eligible to enroll in EXPAND? Or is anyone who's been treated compassionately eligible to enroll in this study? Or are you excluding anyone with prior exposure to Lidmarily?

Speaker 2

Well, first, just on the to reiterate on the differences in the setting, the IMbark population was so young. They were too young even really to recognize pruritus for the most part. You don't see that show up until, basically what would have been the very end or even after the time period that Embark was looking at. So there's a very distinct setting to go after these what are going to be thinking about as getting towards grade school age children for a lot of instances.

Speaker 10

So just to follow-up on that, Chris, if there are patients that responded by bile acids or bilirubin or some metric in EMBARK, are they eligible to enroll and expand if they're a biliary atresia patient?

Speaker 2

The comment I'd make there, yes, the EMBARK study results recall there that we really in that setting, you're not seeing a response signal, right? So those patients are either had successful Kasai procedures, you wouldn't expect them to progress again for several years or they had a transplant already. So it's kind of it's we didn't even see it as a really relevant question. We can circle back on I don't know the exclusion criteria off the top of my head right now, but it was it's just such a different patient population, you wouldn't see those necessarily connect.

Speaker 10

Okay. Makes sense. Thanks so much.

Operator

The next question comes from Brian Skorney from Baird.

Speaker 13

Hi. This is Charlie on for Brian. Just a couple of questions on the bile acid portfolio. Previously, it was sitting around lowtomid20 per quarter. So just wondering, you said it's demand growth, but what you're seeing, if you could give a little more detail there as well as if you're planning for any other opportunities to expand, the value you're getting out of coal bond?

Speaker 13

Thank you.

Speaker 2

Yes. Thanks for the question. I'll ask Peter to give some color. Yes.

Speaker 3

And I think I'd kind of go back to with the bile acid products that if you're looking at Q1 versus Q2, just a reminder that Q1 was artificially low because of the cyber attack that occurred that impacted pharmacy claim processing in the U. S. So that's one dynamic to keep in mind. We do expect kind of steady demand growth kind of in line with historical averages and what you've seen over time with reminding you that there is quarter to quarter variability with these products. And in terms of coal bomb expansion, yes, we think the coal bomb label is in a great spot.

Speaker 3

It facilitates the use and reimbursement of that product across the various settings where it's been established. So no major plans right now to focus on expansion there.

Speaker 8

Thanks for the question, Trent.

Operator

The next question comes from John Willoughlin from Citigroup.

Speaker 8

Hey, thanks for taking the questions.

Speaker 14

A couple on PFIC for me. Wondering without talking about sales here, any metrics on the PFIC launch in the U. S? Things are going well, things that you guys could improve upon throughout the rest of the year? And then with the label expansion recently, can you discuss the added risk of the propylene glycol toxicity?

Speaker 14

Any observation of that in the clinical trials? Or is this just a risk that FDA want to include?

Speaker 2

Yes. I'll kind of break that up between Peter and Joanne to talk about the commercial and safety aspects.

Speaker 3

Yes. Thanks for the question. Certainly real happy with how we've come out of the gate with PFIC. We've seen we had a number of patients transition from our clinical trial expanded access programs to commercial drug. That proceeded very well as well as de novo prescriptions for PVIP patients come in.

Speaker 3

We've talked about it before that we expect revenue contribution from PFIC in 2024 to be pretty modest given reimbursement. We expect a fair bit of free drug shipment this year for PFIC. It is contemplated a little bit in our guidance, but really expect 2025 to be where PFIC starts contributing more. So good start. And then with the rest of the propylene glycol and the clinical development program, I'll let Joanne speak to that.

Speaker 4

Yes. Thanks for the question. Just to be clear, we have not seen, PG toxicity in our clinical studies. And in the context of the PFIC label expansion, we and the FDA looked pretty carefully and kind of come through all of that patient experience data. This concern from the FDA really arose in light of the younger patients being considered for the expansion.

Speaker 4

And we have seen, warrants about PT toxicity with all label for instance. So this is not unique to us. We do feel comfortable that the physicians who are prescribing this know the patients, they know what to look for, they're appropriately monitoring. So we think from a practical perspective that this is well handled in terms of the routine patient care.

Speaker 2

Got it. Thanks.

Speaker 8

Thanks for the question.

Operator

And the next question is from Ed Arce from H. C. Wainwright.

Speaker 15

Hi, everyone. This is Thomas here asking a couple of questions for Ed. Thank you so much for taking our questions. Perhaps first, just wondering for QINODAR, how would you characterize the additional commercial opportunity in CTX as a non label indication, I suppose in terms of harmid and also

Speaker 7

relative to our cumulative existing sales?

Speaker 2

Thanks, Thomas, for the question. I'll ask Peter to comment on that, give some color on the undiagnosed population for CTX.

Speaker 3

Yes. We as we look at the CTX opportunity, the best estimates from literature, market research, KOLs, probably somewhere between 1,000 to 2,000 prevalent patients with CTX in the United States, but only 10% of those are diagnosed right now is the best estimate of what we see. So probably the biggest opportunity is if we can increase that 10% to something higher. So we are investing in disease state awareness initiatives, reaching out to physicians who may see these patients as they're presenting with various symptoms on their journey to a diagnosis and trying to both increase the rate of diagnosis as well as speed up the time to get to the diagnosis and have seen a lot of interest from prescribers, neurologists other physicians kind of take interest in that. And then if Kenadial is approved by FDA and we're able to promote, certainly the product has never been promoted before.

Speaker 3

So I think there'll be an opportunity out there and even for the diagnosis patients in clinic, raise awareness of the benefits of potential benefits of penodial, support reimbursement, things like that.

Speaker 15

Understood. Thanks. And maybe one more from us. This one for valixibat. Regarding vintage in this case, has the FDA or the EMA review interim data that you have so far, any feedback from any regulatory agencies?

Speaker 2

Thanks, Thomas, for the question. In the cadence here of regulatory review, we have had a pre IND discussion designing the studies and the next opportunity would be after we have final results is what our plans are. So no kind of interim discussions on plans.

Speaker 15

Okay. Thank you so much for taking my questions.

Speaker 8

Yes. Thanks for the questions.

Operator

And that was our last question. So I will hand back over to Chris Stade, the CEO for any final remarks.

Speaker 2

Thank you, operator, and thank you all for joining us today. We appreciate the support for Merum and our programs. Have a good evening. Goodbye.

Operator

And this concludes today's call. Thank you for joining. You may now disconnect from the call.

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