Mirum Pharmaceuticals Q3 2024 Earnings Call Transcript

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November 12, 2024

There are 16 speakers on the call.

Operator

Hello, everyone, and welcome to the Mirum Pharmaceuticals Third Quarter 20 24 Financial Results and Business Update. My name is Seb, and I'll be the operator for your call today. I will now hand you over to Andrew McKibbin, Vice President of Investor Relations and Finance. Please go ahead.

Speaker 1

Thanks, Deb, and good morning, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q3 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, Merrim issued a news release announcing the company's results for the Q3 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.

Speaker 2

With that said, I'd like to turn

Speaker 1

the call over to Chris. Chris?

Speaker 2

Thanks, Andrew, and good morning, everyone. It has been another outstanding quarter for Miriam. 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. As a reminder, our goals this year have been to drive continued growth across our 3 commercial medicines, expand the labels for both with MARLYN PFIC and QINODial and CTX, leverage the strength of IVAT inhibition in the adult settings of PSC and PBC and continue expanding our portfolio of medicines for rare diseases. We have delivered on all of these this year.

Speaker 2

Specifically in the Q3, all three commercial medicines saw continued growth with net product sales of $90,300,000 an 89% increase from the Q3 of 2023. This very strong quarter was driven by the continued underlying trends across the brands with increased PFIC and international uptake for Lip Marley. With this strong execution from our commercial team, we are increasing our 2024 guidance to $330,000,000 to $335,000,000 in full year net product revenue. We have also made excellent progress with our pipeline. Valexovat has been granted breakthrough designation for cholestatic arthritis and PVC based on the VANTAGE study interim results.

Speaker 2

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. 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. 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. For kinodial, I'm pleased to announce we received priority review for our NDA and CTX with the PDUFA date of December 28. And finally, I'm also happy to share that we've expanded our early development pipeline with the addition of MRM-three thousand three hundred and seventy nine, a candidate for the treatment of patients with fragile X syndrome.

Speaker 2

Fragile X Syndrome is a rare genetic disorder that is a leading cause of intellectual disability that affects approximately 50,000 male patients across the U. S. And Europe with no approved therapies. Strategically, this program aligns well with the capabilities we are building in rare genetic neurology to support kinodial on CTX. We will continue to look for opportunities to leverage our industry leading rare disease expertise in patient settings where there's a clear need for high impact medicines.

Speaker 2

I'm proud of all of our progress to date in 2024 and the potential ahead for Mira. And I'll now turn the call over to Peter to go over the commercial business. Peter?

Speaker 3

Thanks, Chris. I'm pleased to say that we had another strong quarter with total net product sales of $90,300,000 For Lid Marley, total global net product sales grew to $59,100,000 in the 3rd quarter. U. S. Sales were $43,500,000 while international sales were $15,600,000 In the U.

Speaker 3

S, we continue to see solid growth in Alagille syndrome and are seeing added contribution from PFIC as reimbursement has come through earlier than we expected. Internationally, we continue to see strong underlying demand growth in Alagille syndrome from both our core markets and our distributor partners despite pricing headwinds. With LYMARLE now approved in PFIT in Europe, pricing and reimbursement negotiations are underway. We also saw continued steady growth from Hovol and Kinadol, which had $31,200,000 of net product sales in the Q3. And the commercial team is looking forward to our PDUFA date for CTX in December.

Speaker 3

Overall, it's been a tremendous year for the commercial business. We continue to grow our impact for Algill syndrome patients. We expanded the label for Loop Marley allowing us to extend our reach to the PFIC community. We effectively navigated price negotiations in Europe and we successfully integrated the bile acid portfolio leading to record sales. With the increased full year guidance of $330,000,000 to 335,000,000 we look forward to ending the year on a high note and continuing our strong execution into 2025.

Speaker 3

With that, I'll turn it over to Joanne. Joanne?

Speaker 4

Thanks, Peter. It was a productive quarter for our pipeline and I'm excited to share our progress. First, I want to highlight the progress we're making with valexovat. We're thrilled with the breakthrough therapy designation for valexovat as a potential treatment for cholestatic pruritus in patients with PBC. This designation highlights the significant burden patients face and the need for an effective treatment.

Speaker 4

The decision was based on the positive interim analysis of the Phase 2b VANTAGE study, which showed statistically significant improvement versus placebo in pruritus for PBC patients treated with olexivat. I'm happy to show that we will be presenting this data as a late breaker at AASLD's delivery meeting next week. For both the PSC and PBC programs, we've had great engagement with investigators and advocacy groups over the last few months. We're happy with how both studies are progressing and remain on track to have full enrollment by the second half of twenty twenty five for PSC and in 2026 for PBC. Moving on to Xpand.

Speaker 4

As a reminder, the study represents an exciting potential label expansion opportunity for Lymphomarley in additional settings of cholestatic arthritis. We have started opening sites and are excited to start screening patients. Looking toward the end of the year, we're on track for our December 28 PDUFA date for kenodiol CTX. This is an exciting step forward in extending our presence in rare genetic neurology. Next, I'm thrilled by the opportunity in fragile X syndrome with MRM-three thousand three hundred and seventy nine.

Speaker 4

Fragile X syndrome or FXS is the most common monogenic cause of intellectual disability and is also characterized by anxiety and language delays. It is an excellent disorder and males tend to be more severely affected. MRM-three thousand three hundred and seventy nine is a selective phosphodiesterase or PDE 4D inhibitor and this mechanism has shown proof of concept in Fragile X in the clinic. We believe MRM-three thousand three hundred and seventy nine's brain penetrant profile is differentiating and we plan to evaluate its potential in males with FXS in a Phase 2 study. We plan to start this study next year.

Speaker 4

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?

Speaker 5

Thanks, Joanne. Earlier today, we issued a press release that included financial results for the Q3, which I'll briefly summarize. 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. Net product revenue in the Q3 2024 was 90 $300,000 compared to net product revenues of $47,700,000 in the Q3 last year.

Speaker 5

Total operating expenses for the quarter ended September 30 were $103,900,000 which includes R and D expense of $31,700,000 SG and A expense of $50,500,000 and cost of sales of $20,800,000 The total operating expense for the quarter included approximately $18,000,000 of non cash stock based compensation and depreciation and amortization expense of which approximately $6,000,000 was included in cost of sales. For the quarter ended September 30, net loss was $15,000,000 or $0.32 a share. Our cash, cash equivalents and investments was $293,800,000 as of September 30, a reduction of $1,600,000 from the previous quarter. The 3rd quarter cash use included the payment of a $10,000,000 milestone to Takeda upon European approval of the Livmali PFIC indication. The upfront fee for MRM-three thousand three hundred and seventy nine was $7,500,000 which we will pay in the Q4 with cash on hand.

Speaker 5

Under the deal terms, we are subject to a $7,500,000 Phase III start milestone and certain regulatory and sales based milestones. 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. Now I'll turn the call back over to Chris for final comments.

Speaker 2

Thanks, Eric. It has been a great quarter for Miram. Our commercial programs are performing well with strong growth resulting from an increased full year guidance. Our pipeline is delivering with FOLXMAP's recent breakthrough designation and the initiation of the EXPAND study for LIVMARY and added MRM-three thousand three hundred and seventy nine with a planned Phase 2 start next year. And the Miram team has driven all of this with financial discipline, setting us up for a strong finish to the year.

Speaker 2

And with that, operator, please open the call for questions.

Operator

Thank The first question is from Gavin Clark Gartner from Evercore ISI. Please go ahead.

Speaker 6

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?

Speaker 6

And is there any opportunity to pursue an accelerated path?

Speaker 2

Gavin, thanks for the question. I'll actually turn it over to Joanne to talk about our strategy and thinking on endpoints.

Speaker 4

Yes. Hi, Gavin. Thanks for the question. 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 FDA.

Speaker 4

But we do think this is a very viable path forward. We've recognized it in the past, different endpoints have been used. And I think that's also been problematic for the field. And if you look at the history, there really have been no positive real positive studies other than for zetolomaz, which is also a PD-four inhibitor. However, with our molecule, we think that there's the selectivity and really the high brain penetration actually provides some advantages in developing forward.

Speaker 4

So we're really excited in terms of moving this forward and do think that there's a valid path forward with the FDA.

Speaker 7

And just to add

Speaker 2

one comment

Speaker 7

on the NIH toolbox endpoint. This is

Speaker 2

the patient conducted a test and some of all of the programs that we have here at Merrim, leaning on the patient reported outcomes and our experience with that, on what part of what gets us excited about this program and the clinical plan.

Speaker 7

Got it. That's helpful.

Speaker 6

And are you planning to have any engagement with regulators prior to initiating the Phase 2 or will next engagement likely be end of Phase 2 meetings?

Speaker 4

Yes. Thanks, Kevin, for the follow-up. We will be engaging with the FDA early next year on this program.

Speaker 8

Got it. Thanks. Thanks for the questions.

Operator

Our next question is from Jessica Fye at JPMorgan. Please go ahead.

Speaker 9

Hey, guys. Good morning. Thanks for taking my questions. I had a couple. First on valexibat, can you share a little more detail on the data we should expect from the late breaking presentation at AASLD from the Vantage interim?

Speaker 9

How much of an update will this be? What would you focus The Street on? And what subgroups, if any, will you present? That's one. 2nd, LiveMarley, it looks like growth was really solid quarter over quarter, year over year.

Speaker 9

I think last year, 3Q, you talked about seasonal headwinds. Did you see any of that this year? And then lastly on business development in the context of this latest in licensing, how do you see the company evolving with this asset kind of going beyond the kind of rare liver disease kind of legacy focus? And do you expect to kind of remain active on the biz def front on the back of this in licensing? Thank you.

Speaker 2

Thanks, Jess, for the question. I'll touch on the quick comments on valexovat and BD strategy and let Peter speak to the commercial trends. For the valexovat late breaker, we're just a handful of days away from these being released. So just kind of point you towards that and the updates coming soon with data there. Real focus, the top line is already out there, right, that we've seen just really strong response on pruritus.

Speaker 2

That is the registrational endpoint we're pursuing. So still very excited about this data and excited to share the update. On BD strategy, and I think just to take a step back and put the 3,379 addition to the as an early stage program, We do see this as one of several steps that we've been pursuing now for years here at Merrim, 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. So the 3,379 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, a thoughtful approach to endpoints and patient reported outcomes. And really the last comment we'll make is, I see this as kind of a bookend of example of things that we look at between the transaction last year bringing on commercial products.

Speaker 2

You'll see this as kind of the earlier end of the spectrum in terms of where we focus our time for BD strategy. And with that, maybe I'll pass it over to Peter on the commercial side.

Speaker 3

Yes. With regards to the Lid Marley trends, Jeff, as you mentioned in 2023, we did comment on a bit of a slowdown in some of the summer months with the Marley. Really has not been something we observed at all in 2024. This quarter was really solid continued growth in Algill syndrome in line with kind of what we've been seeing with Algill syndrome and a strong kind of launch performance from PFIC in the U. S.

Speaker 3

As well as strong growth in international.

Speaker 5

Thank you.

Speaker 8

Thanks for the question.

Operator

Our next question is from Mani Forooja from Leerink Partners. Please go ahead.

Speaker 10

Hey guys, good morning. You have Brian on for Mani. Thanks for taking our questions and congrats on the quarter. I guess a few on 3,379. Can you just provide any specifics around the deal terms such as royalties or future milestones that we should expect for this program?

Speaker 10

And then more specifically on the OpEx line, do you expect this program to impact OpEx moving forward or is it pretty negligible? And then just one on the commercial front, can you share any color on where you're seeing this acceleration in PFIC is coming from? So whether that be new patient starts or switches from a competitor and what that means for growth moving forward? Thanks.

Speaker 2

Great. Brad, thanks for the question. I'll pass it off to Eric and Peter to touch on both questions.

Speaker 5

Yes, thanks. So I did provide some comments on the financial terms in my commentary that we just went through. So the upfront was $7,500,000 and really the only sort of pre regulatory approval milestone is the $7,500,000 Phase 3 start milestone. Royalties, we're not going to comment on, but that is very typical for a deal of this stage, so pretty modest. In terms of the increase in spend, it's too early to be too specific because we're early in terms of developing our plans for this asset, both on the clinical side as well as the manufacturing side.

Speaker 5

But to give you just a rough sense, in terms of percentage of our R and D spend, you could think about it being sort of in the mid teens percentage wise.

Speaker 2

Yes. And just to kind of put that in perspective of how we looked at bringing this program into Mirum, see it as very much an efficient early stage program that is quite targeted investment to get to proof of concept. So the Phase 2 design and investment into that from the deal terms to the clinical spend, really not a big step in terms of the amount of dollars spent.

Speaker 3

And yes, Brian, to your question on what's driving the PFIC performance, 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 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. 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.

Speaker 3

That were ready to be rolled over after launch. And then we are seeing de novo demand. Probably the majority of those are IBAT naive and we are seeing some switches as well.

Speaker 7

Awesome. Thank you. Thanks

Speaker 8

for the questions.

Operator

Our next question is from Dae Gon from Stifel. Please go ahead.

Speaker 11

Hey, great. 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 11

Just curious if it is primarily driven by Alagille 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, but in terms of outlook on cadence of patient enrollment and any kind of time line that you envision in terms of enrollment completion, that would be great. And then lastly on the MRM-three thousand three hundred and seventy nine, 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?

Speaker 11

Is there anything that's lower hanging fruit after Fragilex? Thanks so much.

Speaker 2

Great, Dave. Thanks for the question. Maybe, John, you want to start with the Xpandz question and move from there?

Speaker 4

Yes. Thanks for the question. In terms of Xpandz, 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. Our guidance in terms of enrollment timeline has not changed from before.

Speaker 4

So we expect to hopefully 18 months or so and then proceed from there. So that's really right on track.

Speaker 2

And then on the sales trends. Yes, yes, Deane.

Speaker 3

So yes, we obviously raised our guidance to $3.30 to $3.35 in terms of what drives that. We continue to see strong alagiol growth in the U. S. Kind of in line with historical trends. And yes, 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.

Speaker 3

S.

Speaker 2

And just to kind of touch on the broader opportunity for PD-four d, 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 2 program before we consider kind of putting broader dollars against it. So, 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.

Speaker 11

Makes sense. Thanks for taking our questions, guys.

Speaker 7

Thanks, Neil.

Operator

The next question is from Brian Skorney at Baird. Please go ahead.

Speaker 12

Hey, good morning, guys. Thank you for taking my question.

Speaker 5

On the PD-four d, I

Speaker 12

know Shinobi is running the Phase 3 with ziplam molast in fragile X. And so I'm just kind of wondering, is the Phase 2 data with that sort of the basis of the licensing deal here? And then can you maybe compare and contrast your molecule with the shinobi 1? And would you anticipate initiating Phase 2 before the readout of that study next year? Or would you maybe wait on those results to decide on potential endpoints and power?

Speaker 12

Thanks.

Speaker 2

Yes. Thanks for the question, Brian. Yes, that did weigh into how we looked at the program. The Phase 2 proof of concept data from that program, I think, are quite interesting. I'll let Joanne speak to a little bit of the differentiation we see.

Speaker 2

But just in terms of study start timing, CDs that's somewhat independent though we are obviously monitoring that program for the newborn. But given the differentiated profile, 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.

Speaker 4

Yes. Hi, Brian. Thanks for the question. 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, simply because what we're trying to do is alter the cycloKNP levels within the brain.

Speaker 4

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 tomosynthesis program. We're excited by the potential for MRM-three thousand three hundred and seventy nine and other potential indications.

Speaker 5

Great. Thank you.

Speaker 8

Thanks for the question.

Operator

The next question is from David Labowicz from Citi. Please go ahead.

Speaker 13

Thank you very much for taking my question. Can you compare the decisions to in license the Travir assets with 3,379 and what you were thinking in general about your strategy relative to business development going forward?

Speaker 2

Thanks, David. I can give a little bit of color and I'll turn it over to Peter to talk about some of the things that we're active looking the spaces we're active looking at and kind of how the search came to both of these products. But the way I've put some perspective on these two transactions and frankly actually the founding transaction of Mirim and acquiring the Marley and Velixibat is really looking for overlooked value that the Mirim team can grow and expand on. And the spectrum of types of transactions we've looked at from commercial to Phase 1Phase 2 ready 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, gives us the ability to drive financial performance and on the clinical side, it gives us the ability to build future growth into the company.

Speaker 2

So see those, quite different transactions working together really well to build out the profile of Merum. I'll turn it over to Peter to talk about kind of what we look at.

Speaker 3

Yes. I mean, I think you can it's a great question. You can look at these two transactions. The transaction with Travere for the bile acid products came by way of us being in the pediatric liver space knowing what maybe underappreciated and create significant value for healthcare professionals, patients, caregivers and that led to our insight there and we're happy with the performance that we've had in over a year of having these products in our hands continuing to grow these to record highs. I think one point about Kenadol interestingly is most of the prescribers are neurologists.

Speaker 3

And so really for over the last year, we've kind of been in this neuro field even though it's a bile acid replacement product, the manifestations are neurologic and that's kind of gotten us into this field and the medical conferences, etcetera, where we kind of sort of gained insight and what may be another underappreciated program in 3,379. So I think one theme is high impact for patients and underappreciated rare disease programs. Thank you.

Speaker 14

Thanks for the question.

Operator

The next question comes from Steve Seedhouse at Raymond James Financial. Please go ahead.

Speaker 15

Hi, this is Timur Vanikup on for Steve Seedhouse. Congrats on the quarter. We have a couple of questions about Fragile X. So first one is, do you have plans to enroll patients younger than 18 years old? And at what point could you enroll those patients?

Speaker 15

And then in terms of the molecule specifics, do you know how much more Centimeters activity you can achieve in the brain? That is how much is the tomlast leaving on the table versus normal? Thank you.

Speaker 2

Yes. 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 2 elements. But do have some initial thinking on what we're going to propose.

Speaker 2

I'll caveat that with we'll update it with regulatory input.

Speaker 4

Yes. Thanks for the question. In terms of the age range, it's typical in these kinds of programs to start with adult patients and then try to step down in age. And that's the basic approach that we will have as well. Currently, we have information from the prior Phase 1 program, which included a fairly large number of adults, healthy volunteers and elderly patients.

Speaker 4

And so we'll use that strategy and then step down on age. 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 3. So having something for children will be particularly impactful for this disease. In terms of how much more central nervous kind of system activity, that's a pretty difficult thing to gauge. We do know that our brain to plasma ratio is actually quite a bit higher than the tomo last.

Speaker 4

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 differentiating feature for 3,379 compared to any of the other compounds here in the clinic.

Speaker 8

Thanks for the question.

Operator

The next question is from Mike Ulz at Morgan Stanley. Please go ahead.

Speaker 2

Good morning. Thanks for taking the question. Maybe just one on Kienodol 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.

Speaker 3

Yes. Thanks for the question, Mike. Yes, so we're really excited about the potential approval and the opportunity to be out there with active promotion. Just a reminder, while KEYTROL is approved under a different indication and available under emergency use here for CTX, this will be a chance to have it on label and with active promotion. We think the best literature estimates suggest there's about 1,000 to 2,000 prevalent patients in the U.

Speaker 3

S. With maybe only about 10% of those diagnosed. So a lot of our effort on disease state awareness and then ultimately upon approval promotion, we'll be trying to increase that diagnosis rate. And 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 the current numbers today.

Speaker 2

Great. Thank you.

Speaker 4

Thanks for the question.

Operator

The next question comes from John Wallerburn from JMP. Please go ahead.

Speaker 4

Hi. This is Catherine on for John. I just have a quick question about kind of what you're seeing ex U. S. And just global expansion for Lebomarley, if you could comment on that?

Speaker 4

And then any updates to your views on the PFIC launch, given how strong the quarter has been?

Speaker 2

Thanks for the questions, Catherine. Yes, the one comment I'd make on PFIC overall, this is early days and seeing good initial demand. But some of this was really just pull forward of reimbursement. So that's we do see this step up as really being a stronger quarter than others because of that earlier reimbursement. But I'll let Peter speak to some of the international aspects.

Speaker 3

Yes, absolutely. I mean, we've now have pricing and reimbursement approval in Alagille in the 4 major EU markets plus several midsized markets. So really proud of the execution our team has delivered there and distributor partners around the world continue to find more Alagille syndrome patients that we could potentially benefit with Marley. So So, the Alagille launch in Europe and international markets continues to go well. And yes, the PFIC approval came recently.

Speaker 3

So that is 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 online.

Speaker 4

Thanks so much.

Speaker 8

Thanks for the question.

Operator

The next question comes from Ed Arce from H. C. Wainwright. Please go ahead.

Speaker 7

Hi, good morning, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking our questions. So first question perhaps following on the biggest question, can you give us more details on payer discussions for PSEG in the U. S.

Speaker 7

And EU? I suppose specifically what percentage of target prescribers have you reached so far?

Speaker 3

Yes. I mean, the beauty of PFIC, really in every market where I could have been is the prescribers are the same as Alagille syndrome and maybe even a subset of those quite frankly. So I think we've at this point in the U. S. Launch of PFIC reached substantially all of them and have seen a nice interest in the profile and great demand in sales as you're seeing in the Q3 numbers.

Speaker 3

Early days in Europe, I think as we there's certainly a fair bit of outreach in Germany and places where we can launch more quickly, but I think we'll be reaching the vast majority of our PBIT providers as we move into next year and as the individual countries pricing reimbursement comes closer to where PBIT conversations are.

Speaker 7

Got it. And then perhaps another question for Lip Marley. Just wondering if you can discuss the growing progress for the EXPENSE study or perhaps any initial investigator feedback that you have so far?

Speaker 2

Thanks, Thomas, for the question. I mean, I can just touch on that briefly in that sites are just now being open. So we have sites open and starting to screen patients. 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.

Speaker 2

So this is an indication that's driven by physician and patient demand to add it to the Lib Marley label. So still see a lot of that same dynamic that led us to put the study together in the 1st place.

Speaker 7

Understood. Perhaps one more question from us. So with in licensing of the PDU-seven does that mean liver catheter disorder is now a busy target for Mirim or just rare genetic diseases in general?

Speaker 2

Yes. 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 with these genetic conditions, the thoughtful approach that you need on endpoints, that all plays across all of these different settings. And on the commercial team side, have commercialized and are active with a number of different therapeutic areas and prescribers.

Speaker 2

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.

Speaker 7

Understood. Thank you again for further questions. Looking forward to hearing more about 3 days seminar in coming months.

Speaker 8

Thanks for the questions.

Operator

This concludes the Q and A session. I'll now hand the call back to Chris Peet, CEO, to conclude the call.

Speaker 2

Great. Thanks everyone for joining us today and have a great day. Goodbye. Thank

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Key Takeaways

  • Net product sales of $90.3 million in Q3 2024, up 89% year-over-year, driven by robust uptake across all three commercial medicines and leading to an increased full-year guidance of $330 million–$335 million.
  • Breakthrough Therapy designation granted for valexivat in cholestatic pruritus associated with primary biliary cholangitis, based on positive interim Phase 2b VANTAGE data, with a late-breaking abstract accepted at AASLD.
  • Priority review received for the NDA of kinodial (for CTX) with a PDUFA date of December 28, 2024, marking a key step in expanding Mirum’s rare genetic neurology portfolio.
  • In-licensing of MRM-3379, a selective PDE4D inhibitor for fragile X syndrome, with plans to start a Phase 2 study next year and leverage Mirum’s expertise in rare genetic neurology.
  • Positive operating cash flow achieved for the first time in Q3, with cash, cash equivalents, and investments of $293.8 million as of September 30, reflecting strong financial discipline.
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Earnings Conference Call
Mirum Pharmaceuticals Q3 2024
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