Q3 2024 Mirum Pharmaceuticals Inc Earnings Call
David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz
The End
Speaker Change: Hello everyone and welcome to the MIRM Pharmaceuticals third quarter 2024 financial results and business update. My name is Seb and I'll be the operator for your call today. If you would like to ask a question during the Q&A session please press star 1 on your telephone keypad. If you would like to withdraw your question please press star 2. I will now hand you over to Andrew McKibben, Vice President of Investor Relations and Finance. Please go ahead.
Unknown Speaker .
Thanks, Seb, and good morning, everyone.
Speaker Change: I'd like to welcome you to Merrim Pharmaceutical's third quarter 2024 conference call.
and Chief Operating Officer, Peter Radovich.
Speaker Change: Our Chief Medical Officer, Joanne Quan, and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Merrim issued a news release announcing the company's results for the third quarter 2024. Copies of this news release and SEC filings can be found in the investors section of our website.
Speaker Change: 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 Marist's programs and market opportunities for its approved medicines and product candidates.
Speaker Change: These statements represent our judgment as of today and inherently involve risk and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements.
Speaker Change: Please refer to the risk factors in our latest Form 10-Q and subsequent SEC filings for more information.
Chris: With that said, I'd like to turn the call over to Chris. Chris?
Chris: Thanks, Andrew. And good morning, everyone. It has been another outstanding quarter for Merrim. With strong commercial execution and advancing pipeline, we have made excellent progress across our 2024 strategic objectives, accelerating our growth as a leader in rare disease.
Chris: As a reminder, our goals this year have been to drive continued growth across our three commercial medicines.
Chris: Expand the labels for both Livmarly and PFIC and KenaDial and CTX. Leverage the strength of IVAT inhibition in the adult settings of PSC and PVC. And continue expanding our portfolio of medicines for rare diseases. We have delivered on all of these this year.
Chris: Specifically, in the third quarter, all three commercial medicines saw continued growth, with net product sales of $90.3 million, an 89% increase from the third quarter of 2023.
Chris: This very strong quarter was driven by the continued underlying trends across the brands with increased PFIC and international uptake for Lipmarly.
Chris: With this strong execution from our commercial team, we are increasing our 2024 guidance to $330 to $335 million in full year net product revenue. We've also made excellent progress with our pipeline.
Chris: The list of that has been granted breakthrough designation for cholestatic periodis in PBC based on the Vantage Study interim results.
Chris: As a reminder to qualify for this designation, the FDA requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapy on at least one clinically significant endpoint.
Chris: This designation emphasizes the continued unmet need in PBC and the strong early treatment effects seen in the interim results, which have now been accepted as a late-breaking abstract at the upcoming American Association for the Study of Liver Diseases meeting.
Chris: Our clinical studies in PBC and PSC remain on track with our enrollment guidance and the phase 3 EXPAND studies evaluating with Marley in additional settings of cholestatic arthritis is now underway.
Chris: For CUNADAO, I am pleased to announce we received priority review for our NDA and CTX with the PDUFA date of December 28.
Chris: And finally, I'm also happy to share that we've expanded our early development pipeline with the addition of MRM3379, a candidate for the treatment of patients with Fragile X syndrome.
Chris: Fragile X syndrome is a rare genetic disorder that is the leading cause of intellectual disability that affects approximately 50,000 male patients across the US and Europe with no approved therapies.
Chris: Strategically, this program aligns well with the capabilities we are building in rare genetic neurology to support cunodile on CTX.
Chris: We will continue to look for opportunities to leverage our industry-leading rare disease expertise in patient settings where there is a clear need for high-impact medicines.
Chris: I'm proud of all of our progress to date in 2024 and the potential ahead from here.
Chris: I'll now turn the call over to Peter to go over the commercial business. Peter?
Peter Radovich: Thanks, Chris. I'm pleased to say that we had another strong quarter with total net product sales of $90.3 million.
Peter Radovich: For Lidmarli, total global net product sales grew to $59.1 million in the third quarter.
Peter Radovich: U.S. sales were $43.5 million, while international sales were $15.6 million.
Peter Radovich: In the U.S., we continue to see solid growth in Alajil Syndrome and are seeing added contribution from PFIC as reimbursement has come through earlier than we expected.
Peter Radovich: Internationally, we continue to see strong underlying demand growth in Algael syndrome from both our core markets and our distributor partners despite pricing headwinds.
Peter Radovich: With Lamarly now approved in PFIT in Europe, pricing and reimbursement negotiations are underway.
Peter Radovich: We also saw continued steady growth from Holbaum and Kienedahl, which had $31.2 million of net product sales in the third quarter, and the commercial team is looking forward to our Paducah date for CTX in December.
Overall, it's been a tremendous year for the commercial business.
Peter Radovich: We continue to grow our impact for algae syndrome patients. We expanded the label for LUDE MARLE, allowing us to extend our reach to the PFIC community.
Peter Radovich: We effectively navigated price negotiations in Europe and we successfully integrated the bioasset portfolio leading to record sales.
Peter Radovich: With that, I'll turn it over to Joanne. Joanne. Thanks, Peter. It was a productive quarter for our pipeline, and I'm excited to share our progress.
Joanne Quan: First, I want to highlight the progress we're making with FELIXABAT.
Joanne Quan: We're thrilled with the breakthrough therapy designation for valixabat as a potential treatment for cholestatic arrhythmias in patients with PBC.
Joanne Quan: This designation highlights the significant burden patients face and the need for an effective treatment.
Joanne Quan: I'm happy to share that we will be presenting this data as a late breaker at AASLB's deliver meeting next week.
Joanne Quan: For both the PSC and PBC programs, we've had great engagement with investigators and advocacy groups over the last few months.
Joanne Quan: We're happy with how both studies are progressing and remain on track to full enrollment by the second half of 2025 for PSE and in 2026 for PBC.
Moving on to Expanse.
Joanne Quan: As a reminder, the study represents an exciting potential label expansion opportunity for lymphomorally in additional settings of cholestatic arthritis. We have started opening sites and are excited to start screening patients.
Joanne Quan: Looking toward the end of the year, we're on track for our December 28th PDUFA date for KinoDial and CTX.
Joanne Quan: This is an exciting step forward in extending our presence in rare genetic roles.
Joanne Quan: Next, I'm thrilled by the opportunity in Fragile X Syndrome with MRM-3379. Fragile X Syndrome, or FXS, is the most common monogenic cause of intellectual disability and is also characterized by anxiety and language delays.
Joanne Quan: It is an X-linked disorder, and males tend to be more severely affected.
Joanne Quan: MRM3379 is a selective phosphodiesterase, or PDE, 4D inhibitor, and this mechanism has shown proof of concept in Fragile X in the clinic.
Joanne Quan: We believe MRM3379's brain penetrant profile is differentiating, and we plan to evaluate its potential in males with FXS in a Phase II study. We plan to start this study next year.
Joanne Quan: I'm pleased with the progress we made this quarter across our different assets and look forward to providing updates in the future.
I'll now turn it over to Eric. Eric?
Eric Bjerkholt: Thanks, Joanne. Earlier today, we issued a press release that included financial results for the third quarter, which I'll briefly summarize.
Eric Bjerkholt: Overall, we continued our trend of strengthening quarter over quarter financial performance. I'm excited to announce that in Q3, we achieved positive operating cash flow for the first time.
Eric Bjerkholt: Net product revenue in the third quarter of 2024 was $90.3 million compared to net product revenues of $47.7 million in the third quarter last year.
Eric Bjerkholt: Total operating expenses for the quarter ended September 30 were $103.9 million, which includes R&D expense of $31.7 million, SG&A expense of $50.5 million, and cost of sales of $20.8 million.
Eric Bjerkholt: The total operating expense for the quarter included approximately $18 million of non-cash stock-based compensation and depreciation and amortization expense, of which approximately $6 million was included in cost of sales.
Eric Bjerkholt: For the quarter ended September 30, net loss was $15 million or $0.32 a share.
Eric Bjerkholt: Our cash, cash equivalents, and investments was $293.8 million as of September 30, a reduction of $1.6 million from the previous quarter.
Eric Bjerkholt: The third quarter cash use included the payment of a $10 million milestone to Takeda upon European approval of the Livmarli PFIC indication.
Eric Bjerkholt: The upfront fee for MRM 3379 was $7.5 million, which we will pay in the fourth quarter with cash on hand.
Eric Bjerkholt: Under the deal terms, we are subject to a $7.5 million phase 3 stock milestone and certain regulatory and sales-based milestones.
Eric Bjerkholt: In summary, we continue to grow the commercial business and advance and enhance our drug development pipeline, all while remaining financially independent and fiscally disciplined.
Chris: Now, I'll turn the call back over to Chris for final comments.
Thanks, Eric. It has been a great quarter for Miriam.
Chris: Our commercial programs are performing well with strong growth resulting from an increased full year guidance.
Chris: Our pipeline is delivering with Felix Batts' recent breakthrough designation and the initiation of the EXPAND study for Luv Marley, and added MRM 3379 with a plan phased to start next year. And the MIRM team has driven all of this with financial discipline.
Chris: Setting us up for a strong finish of the year. With that, Operator, please open the call for questions.
www.unc.org.au
Speaker Change: Thank you. As a reminder to ask a question, please press star one on your telephone keypad. If you would like to withdraw your question, please press star two. First question is from Gavin Clark Gartner from Evercore ISI. Please go ahead.
Speaker Change: Hey guys, congrats on all the progress and thanks for taking the questions. So I just wanted to focus in on Fragile X. So, first question here, maybe just for those who are less familiar, what's the current registrational endpoint in Fragile X? Is it behavioral or cognitive clinical endpoints? And is there any opportunity to pursue an accelerated path?
David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz
Speaker Change: Kevin, thanks for the question. I'll actually turn over to Joanne to talk about our strategy and thinking on endpoints.
Joanne Quan: Yeah, hi, hi, Gavin. Thanks for the question. You know, our thinking is that we can use the NIH toolbox as a registrational endpoint. Obviously, we've just acquired the program. So we haven't had further discussions with the FTA. But, you know, we do think this is a very viable path forward. We've recognized that in the past, different endpoints have been used. And I think that's also been problematic for for the field. And if you look at the history, there really have been no positive, real positive studies, other than for the Tomex, which is also a PD-14 inhibitor.
Joanne Quan: So we're really, you know, excited in terms of moving this forward and do think that there's a valid path forward with the FDA.
Speaker Change: And just to add one one comment on the NIH toolbox and point this is a you know the patient
Speaker Change: Conducted a test, and it was similar to all of the programs that we have here at Merrim, leaning on the patient reported outcomes and our experience with that on what is part of what gets us excited about this program and the clinical plan.
Unknown Speaker 0
Speaker Change: That's helpful. And are you planning to have any engagement with regulators prior to initiating the phase two or will next engagement likely be at the end of phase two meeting?
Speaker Change: Yeah, thanks, Kevin, for the follow up. We will be engaging with the FDA, you know, early next year.
on this program.
Got it. Thanks.
Thanks for the questions.
Speaker Change: Our next question is from Jessica Fye at J.P. Morgan. Please go ahead.
Unknown Speaker Okay.
Speaker Change: Hey guys, good morning. Thanks for taking my questions. I had a couple.
Speaker Change: First on the Lexibat, can you share a little more detail on the data we should expect from the late-breaking presentation at AASLD from the Vantage Interim?
How much of an update will this be?
Speaker Change: What would you focus the street on and what subgroups, if any, will you present?
That's one.
Speaker Change: Second, Liv Marley, it looks like growth is really solid, quarter over quarter, year over year. I think last year, 3Q, you talked about seasonal headwinds.
Speaker Change: kind of legacy focus and do you expect to kind of remain active on the BizDev front on the back of this in licensing? Thank you.
Speaker Change: Thanks, Jess, for the question. I'll touch on the quick comment on the Velixiv ad and BD strategy and let Peter speak to the commercial trend.
Speaker Change: Point you towards that and the updates coming soon with what data they're real focus The top line is already out there right that we've seen just really strong response on on caritas. That is the registration one point We're pursuing So still very excited about this data and excited to share the update
Speaker Change: On BD strategy, I think just to take a step back and put the 3379 addition to the early stage program,
Speaker Change: We do see this as one of several steps that we've been pursuing now for years here at MIRM with a team that's been focused on rare disease, in particular rare genetic disease and programs that are overlooked that can add a lot of value.
Speaker Change: So the 3379 program is a great example of that, where it fits well with our capabilities in terms of how genetic diseases get diagnosed and treated on the development side, you know, a thoughtful approach to endpoints.
and patient-reported outcomes.
Speaker Change: And really, the last comment I'll make is see this as kind of a bookend of an example of things that we look at between the transaction last year, bringing on commercial products. You'll see this as kind of the earlier end of the spectrum in terms of where we focus our time.
for BD Strategy.
Speaker Change: With that, maybe I'll pass it over to Peter on the commercial side.
Peter Radovich: Yeah, with regards to the Live Marley trends, Jeff, as you mentioned, in 2023, you know, we did, we did comment on a bit of a slowdown in some of the summer months with Live Marley, you know, really has not been something we observed at all in 2024, this, this quarter.
Peter Radovich: It's really solid continued growth in algae syndrome in line with kind of what we've been seeing with algae syndrome and a strong kind of long performance from PBIC in the U.S. as well as strong growth in the international.
Thank you.
Thanks for the question.
Speaker Change: Our next question is from Mani Baruha from Learink Partners. Please go ahead.
Unknown Speaker
Speaker Change: Hey guys, good morning. You have Brian on for morning. Thanks for taking our questions and congrats on the quarter. I guess a few on 3379. Can you just provide any specifics around the deal terms such as royalties or future milestones that we should expect for this program?
Speaker Change: And then more specifically on the OPEX line, you know, do you expect this program to impact OPEX moving forward or is it pretty negligible?
Speaker Change: and then just one on the commercial front, you know, can you share any color on where you're seeing this acceleration in PFIC is coming from? So, you know, whether that be new patient starts or switches from a competitor and what that means for growth moving forward. Thanks.
David Lebowitz, Jonathan Wolleben, Eric Bjerkholt
Speaker Change: Great, thanks for the question. I'll pass it off to Eric and Peter to touch on both questions. Yeah, thanks. So I did provide some comments on the financial terms in my commentary that we just went through. So the upfront was $7.5 million, and really the only sort of pre-regulatory approval milestone is the $7.5 million phase three stock milestone.
Speaker Change: Royalties, we're not going to comment on, but they're very typical for a deal of this stage. So pretty modest in terms of.
Speaker Change: The increase in span is too early to be too specific because we're early in terms of
Speaker Change: Developing our plans for this asset, both on the clinical side as well as the manufacturing side, but to give you just a rough sense in terms of percentage of our R&D spend, you could think about
it being sort of in the mid-teens.
Percentage-wise.
Speaker Change: Yeah, just to kind of put that in perspective of how we looked at bringing this program into MIRAM, see it as a very much an efficient early stage program that is quite targeted investment to get to proof of concept. So the phase two design and investment into that from the deal terms to the clinical spend.
Speaker Change: Really, not a big step in terms of the amount of dollars spent.
Speaker Change: And yeah, Brian, to your question on what's driving the PFIT performance.
Speaker Change: I think an overarching comment is really reimbursement, our market access and reimbursement team did a great job securing coverage for PFIC kind of sooner than we would anticipate.
Speaker Change: based on even our LGO approval as well as other rare disease approvals when you usually expect a few more quarters of new-to-market policies where reimbursement might be slower to come online. So that came online faster than we thought.
Speaker Change: In terms of sources of demand, we've talked in the past about our expanded access clinical trial rollover patients being in about the low 20s in the U.S. that were, you know, ready to be rolled over after launch. And then we are seeing de novo demand. Probably the majority of those are IVAT naive, and we are seeing some switches as well.
Thank you. Thank you.
Awesome, thank you.
Thanks for the questions.
The End
Speaker Change: Our next question is from Dagon Ha from Stiefel. Please go ahead.
Speaker Change: Hey, Greg, good morning. Thanks for taking our questions and congrats on the quarter, looking pretty bright ahead. I guess a couple of questions from me as well, just thinking about the OPEX growth, I guess not OPEX, but the revenue guidance increase.
Speaker Change: I'm just curious if it is primarily driven by allergical growth ahead in Q4, or is it really going to be about PFIC as we think about modeling purposes? When we think about EXPAND, Joanne, can you expand a little bit more? You said sites are opening.
Speaker Change: But in terms of outlook on cadence of patient enrollment and any kind of timeline that you envision in terms of enrollment completion.
Speaker Change: That would be great. And then lastly, on the MRM3379, just curious about your overall strategy on the neurology side. I mean, Fragile X is the starting point here, but are you thinking about expanding on the PDE4 inhibition for other indications? Is there anything that's lower hanging fruit after Fragile X? Thanks so much.
Unknown Speaker
Thank you. Thank you. Thank you.
Speaker Change: Great, thanks for the question. Joanne, do you want to start with the EXPAND question and move from there?
Joanne Quan: Yeah, thanks for the question. In terms of Expanse, we're right on track in terms of where we are in terms of the study starts. So we have sites up and running and we expect to start screening shortly. You know, our guidance in terms of enrollment timeline has not changed from before. So we expect to, you know, roughly 18 months or so.
Thank you. Thank you.
So that's really right on track.
David Lebowitz, Jonathan Wolleben,
Joanne Quan: And then on the sales trends. Yeah, yeah. So yeah, we obviously raised our guidance to 333, 335 in terms of what drives that. You know, we continue to see strong allergial growth in the U.S. kind of in line with historical trends. And yeah, the uptick that we saw in Q3 and that is kind of coming forward in our guidance in Q4 is really driven by the PFIC launch in the U.S.
Joanne Quan: And just to kind of touch on the, you know, the broader opportunity for PD4D,
Joanne Quan: There may be potential in other indications, but we're approaching this first in a very focused way to get to proof of concept in Fragile X in the Phase II program before we consider putting broader dollars against it.
Joanne Quan: Really looking forward to generating that first proof of concept data and what that can mean for Fragile X patients, and we'll go from there.
Thank you. Thank you.
Makes sense. Thanks for taking our questions, guys.
Thank you.
[inaudible]
Speaker Change: The next question is from Brian Scorney at Baird. Please go ahead.
Brian Scorney: Hey, good morning, guys. Thank you for taking my question. On the PDE 40, I know Shinobu is running a Phase 3 with Zipolma.
Brian Scorney: Malast in Fragile X. And so I'm just kind of wondering, is the phase two data with that sort of the basis of the licensing deal here? And then can you maybe compare and contrast your molecule with the Shinogi one? And would you anticipate initiating phase two before the readout of that study next year? Or would you maybe wait on those results to decide on potential endpoints and power? Thanks.
Speaker Change: Yeah, thanks for the question, Brian. Yeah, that did weigh into how we looked at the program and the phase two proof of concept data from that program.
Speaker Change: I think are quite interesting. I'll let Joanne speak to a little bit of the differentiation we see. But just first in terms of study start timing, CDs are somewhat independent, so we are obviously monitoring that.
Speaker Change: I think that we could see stronger results than what comes out of that program. I'll let Joanne speak to some of what we liked about this to bring it into the pipeline.
Joanne Quan: Yeah, hi Brian, thanks for the question. You know, the molecule we have is highly selective, and I think one of the key differentiation points from the other molecule is just the high brain to plasma ratio.
Joanne Quan: Simply because, you know, what we're trying to do is alter the cyclic AMP levels within the brain, and so we think that our molecule has some potential advantages here. So I think that's the key differentiation point, and obviously we'll keep that in mind as we look at results for the autonomous program.
Joanne Quan: We're excited by the potential for MRM3279 and other potential applications.
David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz
Great, thank you.
Thanks for the question. END
David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz
Speaker Change: The next question is from David Lebowitz from City. Please go ahead.
David Lebowitz, David Lebowitz, David Lebowitz
Speaker Change: Thank you very much for taking my question. Can you compare the decisions to license the Trevere assets with 3379 and what you were thinking in general about it, about your strategy relative to business development going forward?
Speaker Change: Thanks, David, I can, I can give a little bit of color and I'll turn over to Peter to talk about some of the things that we're active looking at, you know, the spaces we're active looking at and kind of how this search came to both of these products. But the way I put some perspective on these two transactions, and frankly, actually, the founding transaction of MIRM and acquiring with Marley and Belixibet, is really looking for overlooked value that the MIRM team can grow and expand on. And the spectrum of types of transactions we've looked at from commercial to, you know, phase one slash phase two ready.
Peter Radovich: It is where I think our team has really deep expertise in rare disease, in particular, in rare disease settings that are genetically driven, where you can use patient reported outcomes, have a lot of experience with that.
And so on the commercial side.
Speaker Change: gives us the ability to drive financial performance. And on the clinical side gives us the ability to build future growth into the company. So see those quite different transactions working together really well to build out the profile of MIRM. I'll turn it over to Peter to talk about
Peter Radovich: of what we look at. Yeah, yeah. I mean, I think you can, it's a great question. You can look at these two transactions.
Speaker Change: You know, the transaction with Trevere for the bioasset products came by way of us being
Peter Radovich: In the pediatric liver space, knowing what may be underappreciated and creates significant value for healthcare professionals, patients, caregivers, and that led to our insight there, and we're happy with the performance.
Peter Radovich: that we've had in over a year of having these products in our hands, continuing to grow these to record highs.
Peter Radovich: I think, you know, one point about Kenadol, interestingly, most of the prescribers are neurologists, and so really for over the last year, we've kind of been in this.
Peter Radovich: Neurofield, even though it's a bile acid replacement product, the manifestations are neurologic, and that's kind of gotten us into this field and, you know, the medical conferences, etc., where we kind of, you know, sort of gained insight and, you know, what may be another underappreciated program in 3379, so.
Peter Radovich: I think, you know, one theme is, you know, high impact for patients and underappreciated rare disease programs.
Thank you.
Thanks for the question.
Speaker Change: The next question comes from Steve Seedhouse at Raymond James Financial. Please go ahead.
Thank you. Thank you. Thank you.
Speaker Change: Hi, this is Timur Vonnekepon for Steve Seathouse. Congrats on the quarter. We have a couple of questions about Fragile X.
Speaker Change: So the first one is, do you have plans to enroll patients younger than 18 years old?
Speaker Change: And at what point could you could you enroll those patients? And then in terms of the molecule specifics, do you know how much more activity you can achieve in the brain? That is how much how much is the total last leading on the table versus normal? Thank you.
Speaker Change: I'll start and let Joanne finish on some of the details, but I appreciate the question here. We are planning to interact with FDA early next year to finalize some of the Phase II elements.
Speaker Change: But, you know, do have some initial thinking on what we're going to propose, caveat that with, you know, we'll update it with regulatory input.
Speaker Change: Yeah, thanks for the question. In terms of the age range, you know, it's typical in these kinds of programs to start with adult patients and then try to step down in age. And that's, that's the basic approach that we will have as well.
Speaker Change: Currently, you know, we have information from the prior phase one program, which included a fairly large number of adults, healthy volunteers and elderly patients, and so we'll use that strategy and then step down on age.
Speaker Change: And that we think going down in age will be particularly helpful for Fragile X because most patients with Fragile X are diagnosed around age three. So having something for children will be particularly impactful for this disease.
Speaker Change: In terms of how much more central nervous kind of system activity
Speaker Change: That's a pretty difficult thing to gauge. We do know that, you know, our brain-to-plasma ratio is actually quite a bit higher than the tomolab, so we think that that will give us some big advantages here. Obviously, the proof will be in the pudding in terms of looking at results, but we're pretty confident that this is an important
Speaker Change: Differentiating Feature for 3379 compared to any of the other compounds that are in the clinic.
www.unc.org.au
Thank you.
Thanks for the question.
Unknown Speaker
Speaker Change: The next question is from Mike Olds at Morgan Stanley. Please go ahead.
Speaker Change: Good morning. Thanks for taking the question. Maybe just one on Kenadol and CTX. Now that you sort of have a PDUFA date, late December, maybe just remind us what the potential market opportunity is there and what impact getting that in the label might have on sales. Thanks.
Yeah, thanks for the question, Mike.
Speaker Change: Yeah, so we're really, really excited about the potential approval and the opportunity to, you know, be out there with active promotion. Just a reminder, well,
Speaker Change: Keen and all is approved under a different indication and available under emergency use here for CTS.
Speaker Change: you know, this will be a chance to have it on label and with active promotion. We think that the best literature estimates suggest there's about 1,000 to 2,000 prevalent patients in the U.S., with maybe only about 10% of those diagnosed.
Speaker Change: So a lot of our effort, you know, on disease state awareness and then ultimately upon approval promotion
Speaker Change: will be trying to increase that diagnosis rate. And, you know, to the extent we can increase it even a little bit, I think it could have a meaningful impact on the current sales trajectory you see reflected in our numbers today.
Great, thank you.
Thanks for the question.
Thank you. Thank you. Thank you.
Speaker Change: The next question comes from John Wallaban from JMP. Please go ahead. Hi, this is Catherine on for John. I have a quick question about kind of what you're seeing at CUS and just global expansion for Marley, if you could comment on that. And then any updates to your views on the PFIC launch, given how strong the quarter's been?
David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz
That's, we do see this step up as
Peter Radovich: really being a stronger quarter than others because of that earlier reimbursement. I'll let Peter speak to some of the international aspects. Yeah, yeah, absolutely. I mean, we've now, you know, have pricing and reimbursement approval in Alajil and the four major EU markets plus several mid-sized markets. So really,
Peter Radovich: Really proud of the execution our team has delivered there and you know distributor partners around the world continue to
Speaker Change: Find more allergies in the patients that we potentially benefit with Marley. So, you know, the allergy launch in Europe and international markets continues to go well.
Speaker Change: And yeah, the PFIP approval came recently. So that is, you know, obviously not reflected in international sales now, maybe as you move into the back half of next year, mid to back half of next year with pricing and reimbursement coming.
Thanks so much.
Thanks for the question.
Speaker Change: The next question comes from Ed Arthur from HC Wainwright. Please go ahead.
Speaker Change: Hi, good morning, everyone. This is Thomas here asking a couple of questions for Ed. Thank you for putting in your questions.
Speaker Change: So, first question, perhaps following on to this question, can you give us more details on the discussions for P-TECH in the US and EU, I suppose specifically, what percentage of target subscribers have you reached so far?
Speaker Change: Yeah, I mean, the beauty of PFIC really in every market where I've been is the prescribers are the same as Alajil Syndrome, and maybe even a subset of those, quite frankly, so I think we've
Speaker Change: At this point, you know, in the U.S. launch of PBIT, we've substantially all of them and have seen, you know, a nice, nice interest in the profile and great, great demand and sales as you're seeing in the Q3 numbers.
Speaker Change: Early days in Europe, I think, as we prepare for outreach in Germany and places where we can launch more quickly, I think we'll be reaching the vast majority of our PBIC providers as we move into next year.
Speaker Change: at the Individual Countries Pricing and Reimbursement comes closer to where we've had conversations.
Thank you. Thank you.
Speaker Change: Got it. Another question for Lekmari, just wonder if you can discuss enrollment progress for the expense study or perhaps any initial investigative feedback that you have so far?
David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz
Speaker Change: Thanks, Thomas, for the question. I mean, I can just touch on that briefly in that, you know, sites are just now being open. So we have sites open and starting to screen patients.
Speaker Change: So too early to comment on the initial progress there, but I will remind you that the whole idea for the study and the study design really came from prescriber interest and investigator interest and looking to get compassionate use access for patients that are, that can fit within the protocol of the study. So this is, this is an indication that's driven by physician and patient demand to add it to the live Marley label.
Speaker Change: So still see a lot of that same dynamic that led us to put the study together in the first place.
Speaker Change: Understood. That's one more question from us. So, with the licensing of the COVID-7-I, does that mean leukemia disorder is now a PD target for mirum or just rare genetic diseases in general?
Thank you. Thank you. Thank you.
Speaker Change: Thanks for the question there. I mean, overall, it doesn't change what we're looking for. And we see rare disease itself as somewhat of a therapeutic area, because the commonality and how patients are diagnosed in these genetic conditions.
The thoughtful approach that you need on endpoints.
Speaker Change: That all plays across all of these different settings, and on the commercial team's side,
Speaker Change: have commercialized and are active with a number of different therapeutic areas and prescribers. So while we see there's opportunities to build in some of these subspecialties, we do see opportunity outside of them as well, because we've shown that we can add value across a number of different settings, both clinically and commercially.
David Lebowitz, Jonathan Wolleben, Eric Bjerkholt
Speaker Change: Understood. Thank you again for the questions. Looking forward to hearing more about 3379 in the coming months.
Thanks for the questions
Speaker Change: This concludes the Q&A session. I'll now hand the floor back to Chris Peetz, CEO, to conclude the call.
Chris Peetz: Great. Thanks, everyone, for joining us today and have a great day. Goodbye.
David Lebowitz, Peter Radovich,
Chris Peetz: Thank you all for joining today's Miriam Pharmaceuticals third quarter 2024 results call. You may now disconnect.
Transcription by ESO. Translation by —