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Operator
Thank you for your patience, everyone. The Mirren Pharmaceuticals report second quarter 2024 financial results and provide business update will begin shortly. During the presentation, you will have the opportunity to ask questions by pressing star followed by one on your telephone keypad. We'll be right back.
Ed
Thank you.
Operator
Good afternoon everyone and welcome to the Miron Pharmaceutics report second quarter 2024 financial results and provides business update. My name is Carla and I will be coordinating your call today. During the presentation you will have the opportunity to ask questions by pressing star followed by one on your telephone keypad and if you change your mind please press star followed by two. I will now hand you over to Andrew McKeven, Vice President of Investor Relations and Finance, to begin. Andrew, please go ahead.
Andrew
Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceutical's second quarter 2024 conference call. I'm joined today by our CEO, Chris Peets, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Kwan, and Eric Bierkle, our Chief Financial Officer. Earlier today, Miriam issued a news release announcing the company's results for the second quarter of 2024. Copies of this news release and SEC filings can be found in the Investors section of our website. 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 Miriam'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 10Q and subsequent SEC filings for more information. With that said, I'd like to turn the call over to Chris.
Chris
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, important regulatory achievements, and positive Elixabet interim results. On the commercial side, adoption of our medicines continues to grow with total net product sales of $77.8 million across Ligmarly, Keenanol, and Colbon, representing a 139% increase from the second quarter of last year. Building on this strength, we've also achieved important regulatory milestones for our commercial medicines. We submitted our NDA for kinadiol and CTX, which, if approved, will allow us to take additional steps to reach this underdiagnosed population and provide an opportunity for orphan exclusivity. For Livmarli, I'm very happy to say that we are now approved for cholestatic pruritus and PFIC in the U.S. and for the treatment of PFIC in Europe. And we recently announced a U.S. label update to reduce that age to 12 months and older. We're looking forward to bringing Livmarly 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 Livmarly in cholestatic pruritus. I'll let Joanne speak to some of the details, but in short, cholestatic pruritus is not limited to algeal syndrome, PFIC, PSC, and PDC. 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 Live 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. alone that would be eligible for this indication. And finally, we took an important step towards advancing the looks of that towards potentially pivotal data with the positive interim readouts of the VISTA's PSC and Vantage PBC studies. Our VISTA's 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 in a patient population that spans first and second line PBC. 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?
Peter
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 revenue guidance of $310 to $320 million. Starting with Liv Marley, total global net product sales grew to $47.2 million this quarter, which represents a 45% increase compared to the same quarter last year. In the U.S., sales were $35.5 million, while international sales were $11.7 million. Growth continues to be driven by new algeol 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 PPIC patients, and our recent label expansion to include patients 12 months and older provides incremental opportunity, given that PPIC is generally diagnosed when children are young. Internationally, the Marley demand growth was strong, and I'm pleased to say that we achieved a favorable outcome in our price negotiations with Germany. Stemming 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 two. We were also very happy with the European Commission's recent endorsement of Livmarly for PFIC, three months and older, which highlights the significant benefit of Livmarly for these patients. Lastly, we saw nice demand growth from Colbaum and Keen et al. in the second quarter, where we recognized net product sales of 30.5 million. Overall, I'm thrilled with the continued strong commercial performance in the first half of the year and proud of the MARAM team's continued execution. We are on a solid path to achieve our full year guidance of 310 to 320 million and continued growth. And with that, I'll turn it over to Joanne. Joanne?
Joanne
Thanks, Peter. We had an exceptional quarter, highlighted by the impressive interim analyses for the VISTA study in PSE and the Vantage study in PVC. I'll give a quick recap of the results. Starting with the VISTA study in PSE, 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. The blinded 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 2025. Moving on to the Vantage study in PBC, We're thrilled with the interim results. Both doses of Elixabat showed a substantial and statistically significant reduction in age and approximately 2.3 point improvement over placebo. Based on this, the Vantage study will also continue with the 20 milligram VID dose consistent with the VISTA study. The results support Elixabat'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 study. Overall, these results are significant for PBC patients, suggesting that valixivat has the potential to set a new standard in addressing the burden of cholestasis. 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 III EXPAND study. Mirim has received a number of requests for compassionate use for Lipmarli in patients with cholestatic paritis 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 paritis. Based on the good responses we've seen in some individuals receiving compassionate use, we are optimistic that Lipmarli can play a significant role in the treatment of paritis for these patients. EXPAND is a randomized, double-blind, placebo-controlled study evaluating with Marley for treatment of pruritus over 20 weeks. EXPAND's 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 EXPAND in the coming quarters. I'll now turn it over to Eric to discuss our financial results. Eric?
Eric
Thanks, Joanne. Earlier today, we issued a press release that included financial results for the second quarter, which I'll briefly summarize. Net product revenue in the second quarter of 2024 was $77.8 million compared to net product revenues of $32.5 million in the second quarter last year. Total operating expense for the quarter end of June 30 were $102 million, which includes R&D expense of $32.7 million, SG&A expense of $49.2 million, and cost of sales of $20.2 million. The total operating expense for the quarter included approximately $17.7 million of non-cash charges, of which $5.7 million was included in cost of sales. For the quarter ended June 30, 24, net loss was 24.6 million or 52 cents per share. Our cash, cash equivalents and investments was 295.4 million as of June 30, 24, a reduction of 7.4 million from the end of the prior quarter. Cash used in the second quarter included the payment of a $10 million milestone to Takeda upon FDA approval of the Limali 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.
Chris
Thanks, Eric. It's been a great first half of the year. I'm proud of the Merrim team and our strong execution. We are well positioned to continue to advance our four strategic priorities, to grow our commercial business, expand the indications of our approved medicines, advance the Lixabet and 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 session. If you'd like to ask a question, please press star followed by one on your telephone keypad. If you change your mind, please press star followed by two. When preparing to ask your question, please ensure your device is unmuted locally. And our first question comes from Dagon Ha from Stifo.
Dagon Ha
Hey, good afternoon, guys. Thanks for taking our questions and congrats on the progress. Two from us, one commercial, one clinical, I guess, Starting with commercial, as we look at the approval label across AllerGeal and PFIC in the US, it's kind of a palindrome between Liv Marley and Bilve. So I was hoping if you can maybe comment a little bit on what you've learned from perhaps Bilve's experience in AllerGeal 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 VISTAs and Vantage trials? Thanks so much.
Chris
Thanks, Dagon, for the question. On the first kind of a comment on the labeling and label expansion we've seen for Live Marley, gone for the marley and it's it's played out really well i think we're in a position of a very strong leadership in europe where uh we're the only products uh approved for both indications uh in the us we're now uh we see it as very equal footing where we're now approved for both indications initial reception has been quite strong and you know the data for live marley and both indications we think tells a really compelling story for prescribers, and that's what we're seeing play out in the real world. So in terms of that kind of label sequencing, I think it's largely played out and to the favor of Marlene. And I'll let Joanne maybe comment on the enrollment strategies.
Joanne
Yeah, thanks for the question. You know, like I said, we're happy with how the enrollment is going. You know, as you've noticed, we've been very excited by the interim analysis results and 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. You know, we're continuing to work with our existing sites and continuing the expansion as we previously talked about. So, you know, we're happy with where our enrollment is going.
Dagon Ha
Great. Thank you very much. Thanks for the questions.
Operator
The next question comes from Ani Ferhoja from Luring Partners.
Chris
Hey, guys. Thanks for taking the question. A couple 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. Sure. Thanks for the questions. Maybe Joanne and Peter take the follow-up.
Joanne
Sure. So, you know, 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, you know, 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. So, you know, we're actually pretty confident in terms of our understanding the role of IVET inhibitors in treatment of cholestatic varitis and think that this extends its potential use to a wide variety of patients who, you know, for 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.
Embark
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.
Chris
And so the pendulum swings one way early in the year, tends to swing the other, certainly around 3Q as we saw a couple years ago. How should we think about the tempo of new patient ads and any sort of operational details we should think about in terms of seasonality across the next few quarters looking forward?
Peter
Yeah, thanks for the question. I mean, one thing to just kind of remind on, reflecting back to Q1, you know, we did see in the U.S. with Liv Marley and the bile acid products impact on our Q1 number from the changed healthcare cyber attacks. That's one thing to keep in mind as you think about quarter-to-quarter trends here. And, you know, as far as seasonality goes across three products, I can't say that we've identified, you know, any real seasonality in these products. I mean, they're know kind of ultra rare to rare products with you know relatively low underlying volume which can lead to kind of quarter to quarter variability uh quite frankly but whether that occurs in one quarter versus another i can't say that there's a really strong effect there okay and should we and on what time horizon should we expect sort of the sort of ous pricing reference
Chris
dynamics to play out? That's something we should think about sort of recurring and sort of eroding itself playing out over the course of a couple quarters. Is that primarily a 2Q, 3Q event? Like, how do we think about baking that into sort of how we model?
Peter
Yeah, exactly. We saw the effect in Q2, we expect it in Q3. And, you know, our expectation is by the time you're into Q4, that that effect is gone. and all of the kind of demand volume growth that we're seeing flows through to the top line.
Chris
Perfect. Thanks, guys. I know you've got a bunch of people in the queue. I'll hop off. Thanks for the questions.
Operator
Our next question comes from Gavin Clark-Gottener from Evercore ISA.
Gavin Clark - Gottener
Hi, guys. This is Yasheng for Gavin. Thanks for taking our question. We just have two. 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 and then one follow-up after that?
Chris
Yasheng, thanks for the question. I 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, in these cholestatic 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 charitas. So, the thinking behind this study design is based on having actually seen that same profile play out in a number of compassionate use patients. So, it really strong evidence from the individual case studies that, in aggregate, basically make this population. So I feel good about launching the study. That's why we designed it. That's what compelled us to put it together. Maybe Joanne can speak a little bit about sample size.
Joanne
Yeah, thanks for the question. So, you know, the sample size was, you know, based on powering based on our primary endpoint, which is an observer-rated visual. So this is a scale that we understand well, based on our previous experience, you know, prior studies with Marley. So we feel pretty confident that this is an appropriate sample size for us to see a solid treatment effect.
Gavin Clark - Gottener
Awesome. Thank you. And then one follow-up was, was there any data from MBART that makes you confident in enrolling VA patients in this trial?
Mike Ose
I'll pass that back to you. Sure.
Joanne
Yeah. Well, thanks for the question. You know, I think it's important to note that the patient population that we're enrolling in EXPAND is actually quite different than the one that we did enroll in EMBARQ. With EXPAND, we're really, you know, taking patients really at any point of their journey. And for bilirushesia, many of these patients will have had a CASI a number of years ago, but over the course of time, their condition deteriorates and they may develop the same. So this is quite a different population than EMBARQ, where we took really incident patients around the time that they had a cocci. So this is quite a different patient population, and on top of that, we're including other causes of cholestatic varitis with chronic disease.
Chris
Yes, I'd also add on that in those compassionate use case studies, there have been biliary atresia patients that are 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. I think we've now figured out how to dose these medicines and what settings to advance them in.
Gavin Clark - Gottener
Super helpful. Thank you so much.
Chris
Yep. Thanks for the questions.
Operator
The next question comes from Jessica from JP Morgan.
Jess
Hey, guys. Good afternoon. Thanks for taking my questions. I have a few. First, where should we look for presentation of the interim PPC data for Velixibat, and what kind of additional details 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 disease as defined by ALP levels or additional data on liver biomarkers or more details on the improvements observed in the fatigue dimension of the PPC-40? 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 Live Marley and Velixibat in terms of what IP you currently have and any pending applications? Thank you.
Chris
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 DD strategy. Your first comment on the further data from the PDC study, we're preparing an abstract. We'll work on getting it submitted for an upcoming Congress. You can't really predict when and where that lands and what's in it. So, you know, just say that we're looking at a number of the elements that you talked about there to consider for that abstract. 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 2040 family of granted and pending patents that really tie back to the quite unique dosing profile for IVAT in general and Livmarly and Felixivet 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 the backup of our public materials there that you can reference. Maybe Peter can talk about BD strategy. Sure.
Peter
Yeah, thanks for the question, Jess. We're focused in rare disease. We really like rare pediatric opportunities. You know, essentially asking ourselves the question, programs where we could add a lot of value, underappreciated programs, We have a really strong development, regulatory, commercial group in rare disease, so looking in 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, so we take a lot of scrutiny to these opportunities.
Jess
Thank you.
David Lebowitz
Questions?
Operator
The next question comes from Mike Ose from Morgan Stanley.
Mike Ose
Hi, this is Rohit Ose from Mike. Thanks for taking our questions. Just in PFIC, do you expect to pursue a label expansion to patients below 12 months? And can you just talk about how many patients are typically diagnosed that are below 12 months and what the opportunity there is? Thanks.
Chris
Thanks, Ryan, for the question. Overall, we feel that our label now with this PFIC expansion down to 12 months is in a really strong place. We are similar to what we do with Ellijill. We're evaluating the potential to submit yet another SMDA 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. I would feel that we're in a position now where we capture most of the Liv-Marley targeted patients for both Allergy Heal Syndrome and TIFIC. Thank you. Thanks for the question.
Operator
The next question comes from David Lebowitz from CT.
David Lebowitz
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?
Chris
Thanks for the question, David. Last, Joanne, maybe to talk a little bit about some of the thinking that went into that and discussion with FDA.
Joanne
Yeah. Thanks, David, for the question. You know, interesting that you asked the question that way because, in fact, the study came about because the FDA actually had suggested it. You know, we were receiving compassionate use request, and so the FDA at one point said, you know, wanted to put these into a study. So this, the EXPAND study has been designed, you know, keeping that input in mind. And so, you know, we do think the commonality is really cholestatic pruritus, you know, cholestasis, elevated serum bile acids, and we know the effect that it has, that I have on pruritus in that setting. So we're pretty confident in terms of, you know, our ability to execute the study and really in terms of the result that we'll see once it gets executed.
David Lebowitz
Got it. Thanks for taking my question. Thanks for the question.
Operator
The next question is from Steven Seedhouse from Raymond James Financial, Inc.
Embark
Yeah, good afternoon. Thanks for the questions. Just to expand, how are you going to 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.
Joanne
Yeah, thanks for the question. We have an observer-rated ITRO, which has been validated and which we do have experience with in prior studies. And that's really designed for pediatric population. So, you know, it's right of you to recognize that in a pediatric versus adult population, we can have different endpoints. But, you know, 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 and know-how.
Chris
Yeah, and just to kind of follow up on that, that pediatric data set, that's the primary cohort, 45 patients. You know, that's where the kind of primary analysis is. So the adults... scores will be part of a supplemental cohort and think of the analysis plan designed in that way. Primary is based on the pediatric score.
Embark
Ah, okay. And so, the adults would be self-reported itch, or would there be a second observer? How does the end point look?
Joanne
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 form, and we'll analyze those differently.
Embark
Okay, and then the formulation here, is this just going to be the same liquid formulation that's commercially available? That's right, yep. Okay. And then just last question, and thanks for the multi-part question. Are biliary atresia patients that enrolled in EXPAND that were maybe pruritic either on enrollment and that's, or excuse me, that enrolled in BARC 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 live myelitis?
Chris
Well, first, just to reiterate on the differences in the setting, the Embark 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 it is a very distinct setting to go after these, you know, what are going to be, think of them as... getting towards grade school age children for a lot of instances.
Embark
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?
Chris
I mean, the comment I'd make there, yeah, the Embark study results recall there that we really, in that setting, you're not seeing a response signal, right? So those patients either had successful CASAI procedures, you wouldn't expect them to progress again for several years, or they had a transplant already. So it's kind of, 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's just such a different patient population, you wouldn't see those necessarily connect.
Embark
Okay. Makes sense. Thanks so much.
Operator
The next question comes from Brian Scorni from Bayer.
Brian Scorni
Hi. This is Charlie on for Brian. Just a couple questions on the bile acid portfolio. Previously it was sitting around, you know, low to mid 20 per quarter. So just wondering, you know, 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 Coalbone. Thank you.
Chris
Yeah, thanks for the question. I'll ask Peter to give some color.
Peter
Yeah, you know, and I think I'd kind of go back to with the bile acid products that, you know, 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 Colbaum expansion, yeah, we think the Colbaum label is in a great spot. It facilitates the 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.
Chris
Thanks for the question, Trevor.
Operator
The next question comes from John from Citizens GMP.
John
Hey, thanks for taking the questions. A couple on PFIG for me. Wondering, you know, without talking about sales here, you know, any, you know, metrics on the PFIG launch in the U.S.? Things are going well. Things that you guys can 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? Any observation of that in the clinical trials, or is this just a risk that FDA wanted to include?
Chris
Yeah, I'll kind of break that up between Peter and Joanne to talk about the commercial and safety aspects. Yeah, thanks for the question.
Peter
Certainly real happy with how we've come out of the gate with PFIC, have seen We had a number of patients transition from our clinical trial expanded access programs to commercial drug. That's proceeded very well, as well as de novo prescriptions for PFIC patients come in. 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 And, you know, really expect 2025 to be where PFIC starts contributing more. So, you know, good start. And then with the presence of the propylene glycol in the clinical development program, I'll let Joanne speak to that.
Joanne
Yeah. 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 combed through all of the patient experience data. You know, this concern from the FDA really arose in light of the younger patients being considered for the expansion. And, you know, we have seen warns about PT toxicity with other labels, for instance, so this is not unique to us. You know, we do feel comfortable that the physicians who are prescribing this know their patients, they know what to look for, they're properly monitoring. So we think from a practical perspective that this is well handled in terms of the routine patient care.
John
Got it. Thanks.
Chris
Thanks for the question.
Operator
And the next question is from Ed Ars from HC Wainwright.
Ed
Hi, everyone. This is Thomas asking a couple questions for Ed. Thank you so much for taking our questions. Perhaps first, I'm just wondering for Kinadal, how will you characterize the additional commercial opportunity in CPX as a non-legal indication, I suppose in terms of quantity and also relative to our existing sales?
Chris
Thanks, Thomas, for the question. I'll ask Peter to comment on that, give some color on the undiagnosed population for CTX.
Peter
Yeah, you know, 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, and other physicians kind of take interest in that. And then, you know, if Kenadial is approved by FDA and we're able to promote, you know, certainly the product has never been promoted before. So I think there'll be an opportunity out there, and even for the diagnosis patients in clinic, you know, raise awareness of the benefits of Kenadial, support reimbursement, things like that.
Ed
in this case. Thanks. And maybe one more from us. This one for the lexibac. Regarding vintage in this case, has the FDA or the EMA reviewed the interim data that you have so far? Any feedback from any regulatory agency?
Chris
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.
Ed
OK. Thank you so much for the kind of questions.
Chris
Yeah. Thanks for the questions.
Operator
And that was our last question. So I will hand back over to Karin Peet. the CEO, for any final remarks.
Chris
Thank you, operator, and thank you all for joining us today. We appreciate the support for Miriam and our programs. Have a good evening. Goodbye.
Operator
And this concludes today's call. Thank you for joining. We will now disconnect from the call.
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