Mirum Pharmaceuticals, Inc. Price & Performance (MIRM)

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 Merrim's programs and market opportunities for its approved medicines and product 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 ks and subsequent SEC filings for more information. With that said, I'd like to turn the call over to Chris. Chris? Thanks, Andrew, and good afternoon, everyone. I'm excited to start today's call with a recap of Merrim's achievements over the last year share an update on our strategic goals for 2025. In the short time since our founding, through our focus on bringing important medicines to patients, we have created a strong business in ultra rare disease that's cash flow positive with an attractive pipeline and broader patient populations. We are still early in our journey and we firmly believe that the best is yet to come. Our strategic priorities over the next two years are to: one, further the global growth of our commercial medicines, highlighted by Libmarly's continued Allogeal Syndrome expansion early PFIC success the recent approval of Cytexly for cerebral tendinitis, dysenthematosis or CTX in The U. S. Two, advance our high impact pipeline driving valexivat potentially pivotal data in adult colostasis next year. Initiating our Fragile X program for MRN-three thousand three hundred and seventy nine, and completing enrollment of the EXPAND study for lipmarly and cholestatic arthritis.

Three, selectively pursue product acquisition and license potential for significant patient and patient impact and value creation. Just as we have since the launch of Mirum, and four accomplished all of the above, with continued scientific and financial discipline. 2024 was a big year for delivering on strategic goals. Total net product sales were $336,400,000 exceeding the upper end of our revised guidance range Liv Marley finished the year with total net product sales of $213,000,000 including approximately $155,000,000 from our U. S. Business and $59,000,000 internationally. In the second half of last year, PFIC new patient starts and international uptake were drivers of the step up in growth. We believe the strong demand we are seeing for Leb Marley and Allogene Syndrome and PJ will continue. Beyond McMarley, we also saw healthy growth in our bile acid medicine, We think we are starting to bend the curve here with total twenty twenty four net product sales $123,000,000 I'm also happy to say that last week the FDA approved CITEXLY which is our new brand name for Kenadiol, for CTX in adults.

With this approval comes seven years of work in exclusivity, and the opportunity to begin promotional efforts with our current field team to reach patients this under diagnosed condition.

Ultimately, the robust growth dynamics across all of our medicines where we expect to add close to $100,000,000 to our top line. With anticipated net product sales between $420,000,000 and $435,000,000 for the year. 2024 was also a significant year for our pipeline. We expanded Libmarly's label with its approval for PFIC in both The U. S. And Europe we initiated the Phase three EXPAND study of Lufmarly and broader settings [indiscernible] a significant growth opportunity for the brand And beyond with Marley, we achieved positive results for Velixadat in both VISTUS PSC and VANTAGE PBC resulting in breakthrough designation in PBC.

And finally, we have expanded our pipeline with the addition of MRM-three thousand three hundred and seventy nine for Fragile X syndrome. This is another high unmet need orphan setting that aligns well with the capabilities we have built in rare genetic neurology for the launch of CITEXXA. Pulling all this together, we are set up for an exciting 2025 and beyond continued strong execution in both our commercial business and pipeline of potentially high impact medicines And with that, I'll turn it over to Joanne to give a brief update on the pipeline. Thanks, Chris. 2024 was a highly year for our clinical program, I'm excited to continue this momentum Starting with our three potentially registration studies of our Avid monolytosapat. I'm pleased to say that the progress we made in twenty twenty four builds further confidence in the [indiscernible] Across multiple segments, including allergy, We have done seen clinical evidence [indiscernible] [indiscernible] [indiscernible] [indiscernible] Based on the one interim analysis we conducted in June, following the positive interim analysis, We've had great engagement with investigators and patient We are on track to complete enrollment in the second half of this year. [indiscernible] There are no approved treatments for PSC and look forward to another opportunity to work with global health to bring a high impact medicine Turning to PVC.

Enrollment in the VANTAGE study results and remain on track to Polycomic Given the results of our interim analysis, what we showed a statistically significant rapid and sustained improvement We've seen a positive response from the investigator and patient community. Majority of PBC patients [indiscernible] in the first line and second line patients [indiscernible] For a large proportion of patients with PD-two Bracy therapy designation received in the FDA last year acknowledges the importance of [indiscernible] We're also excited about Phase III expansion This is an excellent opportunity to expand our label to include additional settings where there's a clear need for improved effective treatment and we are encouraged by the enthusiasm for CECL The study is ramping up well. We are on track to Shifting to 379. We're preparing to initiate a Phase two study [indiscernible] as a reminder, mechanism has demonstrated proof of concept [indiscernible] given the high expression of TD-forty eight and the importance of this pathway and learning curve [indiscernible] [indiscernible] In summary, we're very happy with and the active engagement investigators and patient We look forward to providing further updates on our progress Thank you, Joanne. I'm pleased to say that we are in an excellent financial position supported by our strong performance in 2024, which I will now briefly summarize. 2024 was another year of continued strong growth in net product sales of our three commercial medicines.

Total net product sales in the fourth quarter twenty twenty four was 99,400,000.0 compared to $69,500,000 the year before representing a 43% year over year increase. For the full year 2024, total net product sales $336,400,000 dollars compared to $178,900,000 in 2023. For Lidmali, total net product sales in the fourth quarter twenty twenty four was $64,100,000 Total modeling net product sales for 2024 was $213,300,000 representing a 50% increase compared to 2023. Turning to our bile acid medicines.

Total net product sales in the fourth quarter twenty twenty four was $35,000,000 representing an approximately 25% growth over the fourth quarter of twenty twenty three. The first full quarter the portfolio was under net product sales for the portfolio for all of 2024 was $123,100,000 Total operating expense for 2024 was $424,500,000 which includes R and D expense of $140,600,000 SG and A expense of 202,200,000.0 and cost of sales of 81,600,000 Expenses for the year included non cash stock based compensation expense of $48,400,000 dollars and intangible amortization and other non cash items of $31,000,000 This intangible amortization and other non cash items [indiscernible] is largely reflected in our cost of goods sold. In the fourth quarter and full year, R and D expense also reflected the 7,500,000.0 upfront payments we paid to and through in for the MRN 3,379 license. When adjusting for the non cash items on happy to say the business was cash flow positive in 2024 and we expect this to continue this year.

We ended 2024 with cash, cash equivalents and investments of $293,000,000 an increase of approximately $7,000,000 from the start of the year. Booking ahead, we expect net product sales of $420,000,000 to $435,000,000 in 2025 and we are guiding toward positive cash flow again this year. We are well funded and we have the resources to execute on our plans and progress toward a multiple upcoming pipeline catalysts. Now, I'll turn the call back over to Chris for final comments. Thanks, Eric. 2025 is set to be another big year for Miram. With industry leading commercial execution, advancing pipeline and a disciplined operating model we have built a leading rare disease company one that is positioned for sustained growth in the years to come. I'm proud of what we have accomplished and excited for the opportunities ahead as we continue to deliver life changing medicines for patients And with that, operator, First question comes from Gavin Clark Gratner. With Evercore ISI. Your line is open. Please go ahead. Hey, guys. Congrats on the great progress.

So I just had one more strategic question.

I know that you aren't able to call for redemption of convertible notes until next year, but just bigger picture as your stock has traded well above the conversion price, to back a really strong earnings performance, pipeline advancement, Does this all change how you think about BD and capital allocation in general? Thanks, Gavin, for the question. I'll make a first comment there and ask Eric to add anything else on the balance sheet and the thing that I point out kind of the dynamic behind what you're pointing out there Is that we've been able to navigate Merrim to a really unique position particular in this market backdrop for rare disease. Where we can take advantage of our financial position and to acquire and basically roll up some of these rare disease products that we see as great value creation opportunities for the company. I'll ask Eric to speak more specifically to the financing plans. Yes.

Thank you.

The convert matures in four years, so that doesn't really factor into our plans meaningfully. I think our financial and financing, like flexibility as we continue to progress the business is is significant and gives us a lot of flexibility to consider alternative that we might find attractive. Thanks for the question, Gavin.

We now turn to Jessica Fye with JPMorgan. Your line is open. Please go ahead.

Hey, guys. Good afternoon. Thanks for taking the question. Hoping you could talk about the underlying assumptions like market penetration or [indiscernible] Potential competition, etcetera, that are behind zone $1,000,000,000 revenue potential for Mirum 3,379 in Fragile X. And I guess while we're talking about that asset, are there any other indications with the mechanism to crash now that could make sense to pursue? Thank you. Thanks for the question, Jessica.

I'll speak to the market sizing and then have Joanne kind of comment about applicability of the mechanism and other settings. And market potential when we look at just mail patients with Fragile X, it's a 50,000 patient market. So a lot of different ways that you can look at the assumptions to see that the total addressable market is $1,000,000,000 or more quite easily. So really when we talk about that number, we're looking at The U. S. Opportunity do see substantial upside beyond that number even It's pretty simple kind of watermark It's a [indiscernible] [indiscernible] Yes. So, actually, I think Jessica, you sort of kind of rightly kind of asked the question in terms of what are the opportunities We know that PD-four d is highly expressed in the brain.

In areas where learning and memory are very important.

And so there are a wide number of conditions that this could be could envision this could be used for The way we're really approaching this is to kind of use Fragile X as our kind of first entry point Conducting a proof of concept there identifying a dose, once we have that then I think it opens up the possibility for entering into some other [indiscernible] Discipline. So certainly a lot of potential there that we're excited to access We now turn to the line for Ruja with Liwing partners.

Hey, guys. You have Ryan on for Marni. Thanks for taking our question. I'm curious when you look at the Alagile market, where do you think we are in terms of penetration into the prevalent population? And kind of going forward, is U. S. Growth really going to be driven more by new diagnosis or kind of greater penetration into those that are not on IVAT yet? Thanks, Ryan, for the question. In terms of overall penetration For the eligible treatment population in The U. S. So [indiscernible] with pruritus that are pre transplant We're probably in the range of forty percent or so penetrated. So just from that statistic alone, we see a lot of growth potential in the prevalent patients. Thinking about what's driving that growth over time, we're actually Several tailwinds on that. One is just to further penetration into the prevalent population There's continued new diagnoses that come along the way, so new patients entering Usually infants, so they're generally lower dose patients, but there are new that are diagnosed and start each year. And then behind that, this is weight based dosing, so you do see some of those dose adjustments over time. So all of that kind of combines for The U. S. To show that there's a long runway of continued growth for allergy syndrome in The U. S. Now turn to David Lebowitz with Citi.

Your line is open. Please go ahead. Thank you very much for taking my question. In In terms of the recent approval for CTX, congratulations on that. Could you I guess, elaborate on how you expect this could affect the sales trajectory. From this point going forward given there's already use in that particular population?

Thanks for the question, David. Yes, so that you're kind of understanding in your question there's the dynamic here where we already have we think that there's a substantial number of the diagnosed CTX patients had been on ketone all The plan is to have those convert over to SITEX Lee in the coming months.

And the the goal here is to impact diagnosis and getting more patients diagnosed and then to treatment. So we do expect it to be a gradual kind of bending of the curve [indiscernible] new patients to diagnosis earlier and preventing the accumulation and buildup of some of these irreversible effects of the disease.

Got it.

Thanks for the question.

Our next question comes from Mike Oles with Morgan Stanley. Your line is open. Please go ahead. Hi. This is Rohit on for Mike.

Thanks for taking our questions. Can you just talk about the patient mix [indiscernible] fourth quarter of twenty twenty four? And how you expect this to play out in 2025? And then additionally, how many of the patients on the PFIC extended access program are now on paid drug? Thanks for the question, Rohit. Touch on the last one there first. During the year last year, we were able to convert all of the U. S. Expanded access patients over to commercial products. So that was complete really most of it into the third quarter last year. And in terms of the patient mix, we've we don't post specific numbers. Throughout kind of where we stand today, most LIV Marley patients by far are Allogene's syndrome patients. So that's the vast majority of the contribution today. In terms of new patient starts, since really Q3 of next year, have seen with the steady allogeneal patient starts, we have seen a nice step up in PFIC patient starts. So they are contributing quite substantially to new patient starts going forward. And you see that reflected in the top line performance.

Thank you.

Thanks for the question.

We now turn to Ryan Deschner with Raymond James. Your line is open. Please go ahead.

Hi there. Congrats on the quarter. My question is how much commercial team expansion would you anticipate doing for expanded pruritus in ultraricholostatic patient pending result of the Phase three EXPAND study and then have a quick follow-up.

Thanks for the question, Ryan. Yes, for looking at planning for Lymphmarly's potential label expansion with Xpand, we anticipate that's largely the same prescribing universe as the AlloSure syndrome and PFIC.

Prescribers. So it wouldn't be an expansion of the field team that's already in place. A lot that we can leverage there commercially. And you said you had a follow-up question Yes. Thanks.

You feel at this point do you have a feel at this point for how many specifically how much of an impact the growing average patient weights are impacting sales growth in Allogeal how big actually is this growth component now compared to some of the other tailwinds here?

And how do you expect it to grow moving forward?

Mean, the way that I would describe it is, we look at kind of think about this as the average dispense and it changes quite modestly over time. In part because you have we continue to have new diagnoses come in. So there are new infant patient starts gradually increase over the years.

Got it. Thank you very much.

Thanks for the question.

We now turn to Brian Skorney with Baird. Your line is open. Please go ahead.

This is Luke on for Brian. Thanks for taking the question. We were hoping you could just talk a little bit about how the VANTAGE study takes into account prior OCA use and enrollment And do you think patients recently discontinuing OCA could see an impact on pruritus measurements as compared to a patient, not not discontinuing Thanks for the question. I'll let Joanne jump into that. [indiscernible] For the question. So So when the study was started, we actually thought there would be a bigger contribution in terms of patients who are in OTA. Turns out it's really not an issue At this point, Our enrollment is actually picked up quite nicely since the interim results came out We're not really seeing that there's an issue in terms of patients, a lot of patients coming forward with who are candidates for the study otherwise. And I think that just reflects overall kind of usage patterns with OTA In general. Not really that much of a contender in this space.

Yes. Kind of in addition to that, recall that for PBC, the VANTAGE study, about two thirds of those patients are first line. So that would be before OCA kind of would have been used or before the new PPARs are used.

Thank you. Our next question comes from Jonathan Wallivan with Citizens JMP.

Your line is open. Please go ahead. Hey, thanks for taking the question. [indiscernible] what do you envision the label looks like if you have success in that Phase III trial? And then how do you wrap your heads around that 1,000 patient U. S. And EU prevalence given it's a collection of a bunch of small Jonathan, thanks for the question. The label, the indication statement is something nail down frankly. It is as you point out, it's a definition somewhat of exclusion. It's a basket of several different causes of colostasis excluding PSE, PVC, ICP, Atlas Shiller and PFIC, it's all of the many less common causes of cholestasis So exact wording will need to be ironed out as we get to that label expansion. In terms of how we think about the patient number though, We think it's Quite simply, the way to think about it, it's a PFIC sized opportunity or larger And as we looked at kind of real compassionate use requests and interest from physicians kind of counting up some of those patient numbers and where that demand was coming Mirum. That's how we landed at that 1,000 patient estimate for The U. S. And Europe.

So I feel it's a very tangible market estimate as opposed to maybe an epidemiology study, for example. So I feel confident that's a quite Sizable.

Opportunity for [indiscernible] Okay. All right. That's helpful. Thanks, Chris. And our final question comes from Ed Arce with H. C. Wainwright.

Your line is open. Please go ahead. Hello. Good afternoon, everyone. This is Thomas Sief asking a few questions for Ed. Thank you so much So first for the relative that this is study in PSC, Good to hear that as [indiscernible] Just wonder how many months or when should we expect expect to see a top line data after complete enrollment is announced? You can outline some details on the process Yes. Thanks for the question, Thomas. I mean, the simple way to think of it is it's a approximately six month endpoint and time to close and clean the database. So kind of project some amount of time like that after enrollment completion. We do plan to announce enrollment completion when we get there.

Got it.

And then perhaps one question on the expense study with MALI as well. Just wonder if you can share some patient experience that you have received to date and which type of rare glaucoma cystic conditions are most common. So far.

Yes. Thanks for the question.

Maybe ask Joanne to speak to some of the data at ASLD last year. On that point, Yes. We do actually have a number of patients that were treated compassionate use for biliary atresia. And those results are quite strikingly positive for treatment of their pruritus. And of note, this is a different patient group than was studied in the earlier ENBARK study. So these are biliary atresia patients that have likely had a Kasai when they were quite young.

And then they continue on. We do know that over time patients will progress in terms of their disease and often a prior liver transplant. And as part of that progression of disease, they will develop pruritus. And so we do have an abstract with handful of cases where there's been quite good treatment out [indiscernible] So that's partly what gives us confidence in conducting a study like the XpAN study that ultimately there is an unmet need and also that we do have a product that can really address that Understood. Perhaps one follow-up on that. Should we expect to see some kind of interim data from this study perhaps in the medical conferences, posters, Actually, we do not plan to do an interim analysis for the study. Our plan is to complete enrollment in 2026 next year. So if any if we did an interim, it would actually slow things down. So we do think we're we're on track to complete enrollment in 2026 and look forward to sharing the results Thank you again for taking my questions. Thank you, Sones.

This concludes our Q and A. I'll now hand back to Chris Pietz, CEO for any final remarks.

Great. Thank you all for joining us today. Have a great afternoon.

Thanks.

Patient