Q4 2024 Mirum Pharmaceuticals Inc Earnings Call and Business Update
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Hello, Thanks for watching.
Unknown Executive, Eric Bjerkholt
Elliot: Hello and welcome to Miriam Pharmaceuticals Report's fourth quarter and year-end 2024 financial results and provides business update. My name is Elliot and I'll be your coordinator today. If you would like to register your question during today's event, please press star one on your telephone keypad.
Speaker Change: These statements represent our judgment as of today, an 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-K, and subsequent SEC filings for more information, but that said I would like to turn the call over to Chris Chris.
Chris: Thanks, Andrew and good afternoon, everyone I'm excited to start today's call with a recap of <unk> achievements over the last year share an update on our strategic goals for 2025.
Speaker Change: In the short time since our founding through our focus on bringing important medicines to patients.
Speaker Change: Have created a strong business an ultra rare disease that is cash flow positive and attractive pipeline in broader patient populations.
Speaker Change: We are still early in our journey and we firmly believe the best guest counts are.
Speaker Change: Our strategic priorities over the next two years arching one further the global growth of our commercial medicines highlight.
Speaker Change: Highlighted by the borrowers continued hours Youll syndrome expansion early success and the recent approval of <unk> for cerebral tentative xanthomatosis or Cts in the U S.
Speaker Change: To advance our high impact pipeline driving Melissa that potentially pivotal data in adult cholestasis next year, initiating our fragile X program for MRI and the $3 79, and completing enrollment of the expand study from a borrowing a whole set of credits.
Speaker Change: Three selectively pursue product acquisition and license opportunities in rare disease with potential for significant patient.
Speaker Change: And patient impact and value creation, just as we have since the launch of <unk>.
Speaker Change: And for accomplished all of the above with continued scientific and financial discipline.
Speaker Change: 2024 was a big year for delivering on strategic goals total net product sales were $336 4 million exceeding the upper end of our revised guidance range.
Speaker Change: We finished the year with total net product sales of $213 million, including approximately $155 million from our U S business and $59 million internationally.
Speaker Change: In the second half of last year, <unk>, new patient starts and international uptake were drivers of the step up in growth.
Speaker Change: We believe the strong demand we are seeing with Marley Analogist sender and Patrick will continue.
Speaker Change: Beyond <unk>, we also saw healthy growth in our bile acid medicine.
Speaker Change: We think we are starting to bend the curve here with total 2024 net product sales of $123 million.
Speaker Change: I'm also happy to say that last week, the FDA approved <unk>, which is our new brand name for Ciena dial for CTX in adults.
Speaker Change: With this approval comes seven years of orphan exclusivity.
Speaker Change: And the opportunity again promotional efforts with our current field team to reach patients and this under diagnosed condition.
Speaker Change: Ultimately the robust growth dynamics across all of our medicines positions us well for 2025, where we expect to add close to $100 million to our top line.
Speaker Change: <unk> net product sales between 420 and $435 million for the year.
Speaker Change: 124 was also a significant year for our pipeline, we expanded the <unk> label with its approval for <unk> in both the U S and Europe, we initiated the phase III expand study of Marley in broader settings of course that it created a significant growth opportunity for the brand.
Speaker Change: And beyond the Marley, we achieved positive interim results for <unk> in both vis vis PSC advantage PBC studies, resulting in breakthrough designation in PBC.
Speaker Change: And finally, we have expanded our pipeline with the addition of mrna 379 with fragile X syndrome.
Speaker Change: This is another high unmet need orphan settings that aligns well with the capabilities. We have built in rare genetic neurology for the launch of <unk>.
Speaker Change: Pulling all this together we are set up for an exciting 2025 and beyond with continued strong execution, both our commercial business and pipeline of potentially high impact medicines.
Speaker Change: And with that I'll turn it over to Joanne and give a brief update on the pipeline Joanne. Thanks.
Joanne: Thanks, Chris plenty plenty for was a highly productive year for clinical.
I'm excited to continue this momentum.
Joanne: Starting with a potentially Registrational studies.
Joanne: All right.
Joanne: I think David the progress we made in 2024.
Bill Clinton: Bill Clinton.
Joanne: Second this mechanism.
Joanne: Right.
Joanne: Best of luck.
Joanne: LNG.
Joanne: Hello Pierre.
Joanne: Okay.
Joanne: We've done some clinical evidence.
Joanne: I think the division.
Joanne: Mark.
Joanne: Okay.
Joanne: Starting it looks at that at all.
Joanne: All of the business.
Joanne: Volume in our single active dose 20 milligram.
Joanne: Okay.
Joanne: Okay.
Joanne: Following the presentation.
Joanne: But we.
Joanne: Okay.
Joanne: Special.
Joanne: We've had great engagement.
Joanne: Thank you.
Joanne: We are pleased with the team.
Joanne: Thanks.
Joanne: We are on track to complete enrollment second half of this year.
Joanne: Given that this is a play.
Joanne: Okay.
Joanne: Yes.
Joanne: There are no approved treatments for <unk> and look forward to another update.
Joanne: To work with global Health authorities.
Joanne: Okay.
Joanne: Okay.
Joanne: Okay.
Joanne: Turning to PBC enrollment.
Joanne: Awesome.
Joanne: We remain on track to complete full enrollment.
Joanne: Given the results of our interim analysis, what we said.
Joanne: Typically.
Joanne: Rapid.
Joanne: Right.
Joanne: We've seen a positive.
Joanne: That's C a N patient.
Joanne: The majority of patients alright.
Joanne: Okay.
Joanne: First of all I see.
Joanne: But do not have adequate options.
Joanne: The strength of the vantage interim results.
Joanne: First line second line patients.
Joanne: So looking at that could be potentially.
Joanne: Our last question.
Joanne: Okay.
Joanne: Brinci therapy designation.
Joanne: The last year technology viewpoint.
Joanne: Potentially.
Joanne: We're also excited about the phase III expansion.
Joanne: This is an excellent opportunity externally.
Joanne: To include additional study close out.
Joanne: Right.
Joanne: There's a clear need.
Joanne: We are encouraged by the enthusiasm.
Joanne: Yes.
Joanne: The study is ramping up well and we are on track.
Joanne: Okay.
Joanne: Shifting to MRI.
Joanne: Okay.
Joanne: Got it.
Joanne: Right.
Joanne: We're preparing to initiate a phase II study this year.
Joanne: Yeah.
Speaker Change: Alan discussions.
Joanne: As a reminder, this mechanism has demonstrated proof of concept.
Speaker Change: And.
Speaker Change: Even the extension.
Speaker Change: And part of this pathway.
Speaker Change: Keep them around.
Speaker Change: Great.
Speaker Change: Potentially.
Speaker Change: Somebody great coffee with continued momentum all of them.
Speaker Change: The active engagement fostered investigators and patients.
Speaker Change: Look forward to providing further updates on our progress.
Speaker Change: Yeah.
Craig: I'll now turn it over to Craig.
Craig: Thank you Joanne I am pleased to say that we are in an.
Craig: Excellent financial position supported by our strong performance in 2024, which I will now briefly summarize.
Craig: 2024 was another year of continued strong growth in net product sales of about three commercial medicines.
Craig: Total net product sales in the fourth quarter 2024 was $99 4 million compared to $69 5 million the year before representing a 43% year over year increase.
Craig: For the full year 2024, total net product sales were $336 4 million compared to $178 9 million in 2023.
Craig: For the moly total net product sales in the fourth quarter 2024 was $64 1 million.
Craig: Totally modelled in net product sales for 2024 was $213 3 million, representing a 50% increase compared to 2023.
Craig: Turning to our bile acid medicines total net product sales in the fourth quarter 2024 was $35 million, representing an approximately 25% growth over the fourth quarter of 2023, the first full quarter the portfolio was undrawn.
Craig: Operator.
Craig: Total net product sales for the portfolio for all of 2024, it was $123 1 million.
Craig: Total operating expense for 2024 was $424 5 million, which includes R&D expense of $140 6 million.
Craig: G&A expense of $202 2 million.
Craig: Cost of sales of $81 6 million.
Craig: Expenses for the year included non cash stock based compensation expense of $48 4 million in intangible amortization and other noncash items of $31 million.
Craig: This intangible amortization and other noncash items expense is largely reflected in our cost of goods sold.
Craig: In the fourth quarter and full year R&D expense also reflects the $75 million upfront payment, we paid to them so and for the MRM 3379 license.
Craig: When adjusting for the noncash items I'm happy to say the business cash flow positive in 2024, and we expect that to continue this year.
Craig: We ended 2024 with cash cash equivalents and investments of $293 million, an increase of approximately $7 million from the start of the year.
Craig: Looking ahead, we expect net product sales of $420 million to $435 million in 2025, and we are guiding toward positive cash flow again this year.
Craig: We are well funded and we have the resources to execute on our plans and progress toward our multiple upcoming pipeline Cadillac.
Speaker Change: Now I'll turn the call back over to Cliff for final comments.
Cliff: Thanks, Eric.
Speaker Change: <unk> hundred $25 is set to be another big year for merit.
Speaker Change: With industry, leading commercial execution and 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.
Speaker Change: I am proud of what we've accomplished and excited for the opportunities ahead, as we continue to deliver life changing medicines for patients and their families.
Speaker Change: And with that operator, please open the call for questions.
Speaker Change: Thank you if you would like to ask a question. Please press star followed by one on your telephone keypad.
Speaker Change: If you would like to withdraw your question. Please press star followed by check.
Speaker Change: When preparing to ask a question. Please ensure you tomorrow on mute locally.
Kevin: First question comes from Kevin <unk> with Evercore ISI.
Speaker Change: Your line is open. Please go ahead.
Speaker Change: 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 at the back of really strong earnings performance pipeline Advancement does this change how you think.
Speaker Change: BD and capital allocation in general Thank you.
Speaker Change: Yeah.
Speaker Change: Okay. Thanks, Kevin for the question I'll make a first comment there and ask her to add anything else on the balance sheet.
Speaker Change: The thing that I pointed out kind of the dynamic behind what you're pointing out there is that we've been able to navigate merit to a really unique position in particular in this market backdrop for rare disease, where we can take advantage of our financial position and performance.
Speaker Change: To acquire and basically roll up some of these rare disease products that.
We see as great value creation opportunities for the company.
Eric: Eric speak more specifically to the financing plans.
Eric: Thank you.
Speaker Change: The convert matures in four years, so that it doesn't really factor into our plans meaningfully I think are.
Eric: Financial and financing flexibility.
Eric: Continue to progress the business.
Eric: Significant and gives us a lot of flexibility.
Eric: You consider.
Eric: Alternative, but we might find that attractive.
Eric: Thanks for the question Kevin.
Speaker Change: Let me now turn to Jessica Fye with Jpmorgan. Your line is open. Please go ahead.
Jessica Fye: Hey, guys. Good afternoon. Thanks for taking my question I was hoping you could talk about the underlying assumptions like a market penetration of share.
Speaker Change: In terms of competition et cetera.
Speaker Change: Behind the $1 billion revenue potential for <unk>.
Speaker Change: 3379 in fragile X Inc. Thank you.
Speaker Change: While we're talking about that asset are there any other indications with the mechanistic rationale that could make sense to pursue.
Thanks for the question Jessica I'll speak to the market sizing and then have Joanne kind of comments about applicability of.
Speaker Change: The mechanism in other settings and quite simply the way to think about the market potential when we look at just mail.
Patients with fragile X and its a 50000.
Speaker Change: Patient market. So a lot of different ways that you can look at the assumptions to see that the total addressable market is $1 billion or more quite easily.
Speaker Change: When we talked about that number we're looking at the U S opportunity do you see substantial upside beyond that number even.
Speaker Change: It's pretty simple kind of watermark too.
Speaker Change: Joanne on the comments about.
Speaker Change: Yes, so actually I think Jessica you sort of a kind of rightly kind of ask the question in terms of what are the opportunities.
Speaker Change: That <unk> is highly expressed in the brain and areas, where learning and memory are very important and so there are a wide number of.
Speaker Change: Conditions that this could be you could envision this could be used for the way. We're really approaching this is to kind of use fragile X is our kind of first entry point conducting a proof of concept there identifying a dose and then.
Speaker Change: Once we have that then I think it opens up the possibility for entering into some other indications associated intellectual disability.
Speaker Change: So certainly a lot of potential there that we can kind of attack.
Jeff: So the question Jeff.
Speaker Change: Leerink partners. Your line is open. Please go ahead.
Speaker Change: Hey, guys you have Ryan on for Mani Thanks for taking our question.
Speaker Change: So I'm curious when you look at the allergy 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 diagnoses or kind of greater penetration into those that are not on buyback yet.
Speaker Change: Thanks, Ryan for the question Eric in terms of overall penetration for the eligible treatment population in the U S. So <unk> syndrome patients.
Speaker Change: With greatest that are pre transplant.
Speaker Change: We're probably in the range of 40% or so penetrated.
Speaker Change: So just from that statistic alone, we see a lot of growth potential and the prevalent patients and thinking about what's driving that growth over time.
Speaker Change: But we're actually.
Speaker Change: Several tailwind on that one is just.
Speaker Change: Further penetration into the prevalent population.
Speaker Change: There is continued new diagnoses that come along the way so new patients entering.
Speaker Change: Usually intense so they're generally lower dose patients, but there are new patients but.
Speaker Change: Are diagnosed and start each year.
Speaker Change: And then behind that this is weight based dosing. So you do see some of those dose adjustments over time. So all of that combines for the U S to show that there's a long runway of continued growth for <unk> syndrome in the U S.
Speaker Change: We now turn to David Lebowitz with Citi. Your line is open. Please go ahead.
David Lebowitz: Thank you very much for taking my question and in terms of the recent approval for CTX congratulations on that.
Speaker Change: Could you.
Speaker Change: Yes elaborate on how you expect this could affect sales trajectory.
Speaker Change: From this point going forward given theres already use in that particular population.
Speaker Change: Thanks for the question David Yeah, So that you're kind of understanding your question does the dynamic here, where we already have.
Speaker Change: We think there is a substantial number of the diagnosed CTX patients.
Ben: Ben on keynote all.
Ben: The plan is to have those convert over to <unk> in the coming months.
Ben: And 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 and bringing new patients to diagnosis earlier and preventing accumulation and buildup of some of these irreversible.
Ben: Next up the disease.
Ben: Got it.
Ben: Thanks for the question.
Speaker Change: Our next question comes from Michael <unk> with Morgan Stanley. Your line is open. Please go ahead.
Speaker Change: Hi, This is Robert on for Mike. Thanks for taking our questions can you just talk about the patient mix between our child syndrome and P. Fix for all the Marley in fourth quarter of 2004, and how you expect this to play out in 2025.
Speaker Change: And then additionally, how many of the patients on the <unk> expanded access program or that one Pedro Thank you.
Speaker Change: Thanks for the question Rohit touch.
Speaker Change: Touch on the last one there first.
During the year last year, we were able to convert all of the.
Speaker Change: U S expanded access patients over to commercial product so that was complete.
Speaker Change: Really most of it into the third quarter last year.
Speaker Change: And.
Speaker Change: In terms of the patient mix, we don't we don't post specific numbers, but throughout kind of where we stand today most live marley patients by far our <unk> syndrome patients. So that's the vast majority of the contribution today in terms of new patient starts since really Q3 of next year we.
Speaker Change: Have seen.
Speaker Change: With the steady.
Speaker Change: I was youll patient starts we have seen a nice step up in <unk> patient starts. So they are contributing quite substantially to new patient starts going forward and you see that reflected in our topline performance.
Speaker Change: Thank you.
Speaker Change: Thanks for the question.
Speaker Change: We now turn to Brian <unk> with Raymond James Your line is open. Please go ahead.
Speaker Change: Okay.
Speaker Change: Hi, there congrats on the quarter.
Speaker Change: My question is how.
Speaker Change: How much commercial team expansion would you anticipate doing for expanded pruritus and ultra record static patients pending the results of the phase III expansion study and then I have a quick follow up.
Speaker Change: Alright, Thanks for the question Ryan.
Speaker Change: We're looking at planning for the lift Barclays potential label expansion with expand we anticipate that's largely the same prescribing universe.
Speaker Change: The <unk> and <unk> prescribers, so it wouldn't be an expansion of the.
Speaker Change: Field team that's already in place so a lot that we can leverage their commercially and you said you had a follow up question.
Speaker Change: Yes.
Speaker Change: Or do you feel at this point, but do you have a feel at this point for how many how many I guess.
Speaker Change: Typically how much of an impact the growing application weights are.
Speaker Change: Impacting sales groups.
In our Gilles.
Speaker Change: How big actually this growth component now compared to some of the other tailwind here, but how do you expect it to grow moving forward.
Speaker Change: I mean, the way that I would describe it as.
Speaker Change: We look at.
Speaker Change: And kind of thinking about this as the average.
Speaker Change: Expense 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.
Speaker Change: Patient starts along the way and that kind of tempers the overall effects, but we haven't seen it kind of gradually increase over the years.
Speaker Change: Got it thank you very much.
Speaker Change: Thanks for the questions.
Speaker Change: Let me now turn to Brian <unk> with Baird. Your line is open. Please go ahead.
Speaker Change: This is Luke on for Brian Thanks for taking the question.
Speaker Change: Hoping you could just talk a little bit about how the vantage study takes into account prior OCA Houston enrollment.
Speaker Change: And do you think patients recently discontinuing OCA could see an impact on <unk>.
Speaker Change: Alright measurements as compared to a patient or not.
Speaker Change: Not discontinuing.
Speaker Change: Thanks for the question I'll try and jump into that one.
Thanks for the question.
Speaker Change: So.
Speaker Change: When the study was started we actually thought there would be a bigger contribution in terms of patients who have an LTA. It turns out it's really not an issue at this point.
Speaker Change: And so.
Speaker Change: Our enrollment is actually picked up quite nicely since.
Speaker Change: The interim results came out.
Speaker Change: We're not really seeing that there is an issue in terms of patients and a lot of patients coming forward with with LTA.
Speaker Change: Who are candidates for the study otherwise.
Speaker Change: And I think that just reflects overall kind of usage patterns within CA in general.
Speaker Change: Not really that much of a contender.
Speaker Change: And this space kind of in addition to that you recall that for PBC. The vantage study about two thirds of those patients are first line. So that would be before OCA would be kind of would've been used or before the new P. Pars.
Speaker Change: <unk>.
Speaker Change: Thank you.
Speaker Change: Thanks for the question.
Speaker Change: Our next question comes from Jonathan <unk> with citizens JMP. Your line is open. Please go ahead.
Jonathan: Hey, Thanks for taking the question just wondering piggybacking on a prior expand question what do you envision the label looks like if you have success in the phase II trial, and then how do you wrap your hands around a thousand patient U S and EU prevalent given it's a collection of a bunch of small small indications.
Jonathan: Jonathan Thanks for the question.
Jonathan: The label indication statement.
Jonathan: Something to nail down frankly it is.
Jonathan: As you point out it's a definition somewhat of exclusion, it's a basket of.
Jonathan: Several different causes of cholestasis, excluding PSC PVC ICP <unk>. So it's all of the many.
Jonathan: Less common causes of cholestasis exactly.
Jonathan: Exact wording.
Jonathan: We'll need to be ironed out as we get to that label expansion.
Jonathan: Okay in terms of how we think about the patient number though.
Jonathan: We think it's.
Jonathan: Quite simply the way to think about it it's a <unk> sized opportunity or larger.
Jonathan: And as we looked at kind of real compassionate use requests and interest from physicians.
Jonathan: Rounding up some of those patient numbers and where that demand was coming from that is how we.
Jonathan: Landed at that 1000 patient estimate for the U S and Europe.
Jonathan: It's a very tangible.
Jonathan: Uh huh.
Speaker Change: Market estimate as opposed to maybe an epidemiology study for example.
Jonathan: So I feel confident that.
Jonathan: Quite sizable opportunity for Philip Marley.
Jonathan: Okay, Alright thats helpful. Thanks, Chris.
Speaker Change: Thanks for the question.
Ed Arce: And our final question comes from Ed Arce with H C. Wainwright. Your line is open. Please go ahead.
Ed Arce: Hello. Good afternoon, everyone. This is Thomas Yip asking a couple of questions. Thank you so much for the questions.
Ed Arce: First for you to look too bad.
Speaker Change: <unk> and PSC.
Ed Arce: Good to hear.
Speaker Change: We're on target to complete woman in second half this year.
Ed Arce: Just wonder.
Speaker Change: How many months.
Speaker Change: Right.
Speaker Change: When should we expect to see top line data after completing enrollment because.
Speaker Change: You can outline the details of the process.
Yes. Thanks for the question Thomas I mean, the simple way to think of it as it is.
Speaker Change: Approximately six month end point in time to close and cleaning the database so kind of project some amount of time like that after enrollment completion, we do plan to announce.
Speaker Change: Completion, when we get there.
Speaker Change: Got it.
Speaker Change: And then perhaps one question on the phase <unk>.
Speaker Change: Studying it.
Speaker Change: As well.
Speaker Change: Just wonder if you can share some patient experience that you have received to date and which type of.
Sure Chris that the conditions are most common.
Speaker Change: So far.
Speaker Change: Yes, thanks for the question.
Speaker Change: Maybe I'll ask Joanne to speak to some of the data at <unk> last year on that point, yes, we do actually have a number of patients that were treated.
Speaker Change: Compassionate use for biliary atresia.
Speaker Change: And those results are quite strikingly positive for too many other pruritus and of note. This is a different patient group <unk> was studied in the earlier embark study. So these are Billy Ashish of patients.
Speaker Change: That have likely had a kasai when they were quite young and then they continue on and we do know that over time patients will progress in terms of their disease and often require a liver transplant.
Speaker Change: And as part of that progression of disease that will develop pruritus and so we do have an abstract with handful of cases, where there has been quite good treatment outcome.
Speaker Change: Using I bet inhibition, so that's partly what gives us confidence.
Speaker Change: In conducting a study Olympics brand study that ultimately there isn't a need and also that we do have a product that can really address that.
Speaker Change: Yeah.
Speaker Change: Understood.
Speaker Change: One follow up on that.
Speaker Change: We expect to see some subsequent interim data from this study.
Speaker Change: Medical conferences.
Speaker Change: Actually we do not plan to do an interim analysis for the study our plan is to complete enrollment in 2026 next year. So.
Speaker Change: If we did an interim that would actually slow things down.
Speaker Change: So we do think we're.
Speaker Change: We're on track to complete enrollment in 2026 and look forward to sharing the results when they are available for the fourth quarter.
Speaker Change: Yes.
Speaker Change: Thank you again for taking my questions.
Speaker Change: Thank you.
Speaker Change: This concludes our Q&A.
Speaker Change: Back to Chris <unk> for any final remarks.
Speaker Change: Great. Thank you all for joining us today.
Speaker Change: A great afternoon.
Speaker Change: Ladies and gentlemen states call has now concluded. Thank you for your participation you may now disconnect your lines.
Speaker Change: [music].
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