Mirum Pharmaceuticals Q2 2023 Earnings Call Transcript

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August 3, 2023

There are 11 speakers on the call.

Operator

Good afternoon, and thank you all for joining. I would like to welcome you all to the Myriam Pharmaceuticals Report 2nd Quarter 2023 Financial Results and Provide Business Update. My name is Freeka, and I'll be your moderator for today's call. All lines are on mute for the presentation portion of the call today with an opportunity for questions and answers at the end. I would now like to turn the call over to Andrew McKibben, VP, Investor Relations and Finance.

Operator

So Andrew, please go ahead.

Speaker 1

Thanks, Brika, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceuticals' Q2 2023 conference call. I'm joined today by our President and CEO, Chris Peetz our Chief Operating Officer, Peter Radovich and our Head of Research and Development, Pam Vig. Earlier today, Myriam issued a news release announcing the company's results for the Q2 of 2023. Copies of this news release and SEC filings can be found in the Investors section of our website.

Speaker 1

Full details and updates from the quarter can be found in our news release. Before we begin, I'd like to remind you that during the course of this conference call, we will be making certain forward looking statements about Merriam. Our pending acquisition of Trevir's bile acid product portfolio and our program is based on management's current expectations, including statements regarding Merriam's current and future business and commercial plans, development programs, regulatory expectations, strategies, prospects, market opportunities and financial forecasts and guidance. Myriam is under no duty to update these statements and they are subject to numerous risks and uncertainties and actual results could differ materially from the results anticipated by these statements. Investors should read the risk factors set forth in Myriam's filings with the SEC.

Speaker 1

With that said, I'll turn the call over to Chris. Chris?

Speaker 2

Thank you, Andrew, and good afternoon to everyone joining us on the call today. We are thrilled to discuss yet another quarter of strong growth at Myriam. We are on track to become a leading multi product franchise in pediatric hepatology through the impact of LYT Marley for patients globally, Continued progress of valexovat in the clinic and the pending acquisition of Kenadol and Colbom. The last quarter saw substantial progress on our 5 part strategy, which is to grow Lidmarley in the U. S, launch Lidmarley in international markets, Establish new indications for Lip Marley, advance and commercialize velixibat in adult cholestatic indications and acquire additional high impact medicines for rare disease.

Speaker 2

We've made great strides on all of these areas of strategic focus so far this year. 1st, on growing LaMarley's commercial business, in the Q2, we performed well against our guidance of 50% year over year net product sales growth in the U. S. In 2023 as well as growth from international markets achieving $32,500,000 of Lip Marley revenue and $37,500,000 total revenue. We are also excited about the recent progress in international markets with our approval in Canada and a growing list of countries selling LIV Marley in distributor markets.

Speaker 2

LIV Marley indication expansion efforts are also making great progress Our PFIC application under review with the December 13, 2023 PDUFA date. We're looking forward to the potential label expansion based on the compelling March Phase 3 data and bringing LYFARLI to PFIC patients. In addition, the EMBARK biliary atresia study is fully enrolled heading towards top line data later this year. Elixibat is also progressing nicely towards interim analyses in PSC and PBC later this year, positioning those studies to expand into their potentially registrational portions. And finally, on the 5th strategic pillar of product acquisition.

Speaker 2

We are advancing towards closing the acquisition of Kenadol and Cold Balm, 2 commercial products with over an aggregate of $100,000,000 of annual net sales in 2022. As we've shared recently, we expect this acquisition to deepen our leadership in pediatric hepatology and accelerate our growth across our product line. We expect the transaction to close in the Q3 and are excited about of these bile acid replacement medicines with LYMARLE and the upcoming Phase 3 readout for Kenadol and CTX. Financially, we are well positioned to execute on our strategy. We ended the 2nd quarter with a $330,000,000 cash balance And Q2 was another quarter of financial growth with relatively steady operating expense levels and a growing top line compared to the Q1.

Speaker 2

The pending addition of Kienadol and Kholbaum offers an opportunity to further accelerate our revenue with a highly synergistic business. Concurrent to this pending acquisition, we entered a private placement agreement for $210,000,000 supported by several existing and new investors, which we will use to fund the upfront payment in connection with the acquisition. This financing allows us to maintain the strength of our balance sheet and positions us for continued growth. With that, I'll pass the call over to Peter to discuss our commercial business in more detail before Pam gives an R

Speaker 3

and D update. Peter? Thanks, Chris. We continue to see strong demand for Libmarley with $32,500,000 in Libmarley net product sales in the Q2 of 2023, which represents 86% growth over the Q2 of 2022. Our U.

Speaker 3

S. Business continues to perform well with net product sales of $26,200,000 This growth was driven by strong underlying and demand. Notably in Q2, we saw an increase in uptake by patients less than 12 months of age, consistent with the LibMarley label expansion to 3 months of age and older in March. Early treatment in Alagille syndrome is important to limit the impact of the disease And we're pleased to see Lebmarly addressing this need in the real world setting. The U.

Speaker 3

S. Business performance continues to track very well against our guidance of 50% growth in 2023 net product sales. Now turning to international markets, we continue to see strong de novo demand in Germany and France launches, in line with what we saw in the U. S. Launch of LibMarla.

Speaker 3

Pricing and reimbursement discussions are ongoing in major markets, which we expect will continue throughout 2023 with full launches in most European countries starting in 2024. We also anticipate use of named patient programs in European and distributor markets to continue to grow throughout 2023. Overall, we are making great progress and are looking forward to our potential label expansion for PFIC in December in the United States and the upcoming readout of the EMBARK study in biliary atresia. We see the total opportunity for Libmarly across Alagille syndrome, PFIC and BA as greater than $1,000,000,000 and that this opportunity is strengthened by our recently announced agreement to acquire Peanutol and Kolbom, which will deepen our presence in pediatric hepatology and establish a focused multiproduct franchise. There is a substantial amount of top and bottom line synergy to unlock by putting the bile acid portfolio and LivMarley together in the hands of 1 commercial team as the marketing efforts are similar in scope and the approach to market access, product distribution and patient services are quite complementary.

Speaker 3

We expect the bile acid products to continue to grow at low single digit rates annually with the potential indication expansion to CTX for penitol providing an upside opportunity to increase patient diagnosis and treatment upon approval. Financially, the strong second quarter net sales of $32,500,000 for Lidmarley And $27,500,000 for Kienodol and Kole Balm reported today by Travir totals approximately 60,000,000 for the future combined franchise with growth expected across all three products. In summary, we are building on the tremendous momentum and potential of our expanded portfolio to deliver these important medicines to patients with rare liver diseases. On that note, I'll turn the call over to Pam. Ma'am?

Speaker 4

Thanks, Peter. We are thrilled to be heading into a data rich second half of the year. First, as Chris mentioned, We are eagerly awaiting the December 13 PDUFA date for PFIC. Secondly, we announced in May the completed enrollment for the EMBARK Phase 2 study in biliary atresia. This study has a 6 month primary endpoint assessing bilirubin and we're on track for the top line data readout later this year, which will be the 1st placebo controlled data with an IBAT inhibitor in this setting.

Speaker 4

I'm also pleased to say that the Phase 2b VISTA study of elixibat in PSC Currently has the total number of patients randomized or in screening required for the interim analysis. This interim will be triggered when 45 randomized patients have been on study for a minimum of 3 months and is expected around year end. In addition, the interim analysis for the Phase 2b VANTAGE study in PBC is also expected around the same time. As a reminder, after these interim analyses, both studies will move into the potentially pivotal portion of the trial. And finally, turning towards the pending acquisition, we have high confidence in the success of the RESTORE Phase III trial evaluating Kenodal in patients with CTX and look forward to top line data in the Q4.

Speaker 4

If positive, this study could support a label expansion for CTX with the potential for orphan exclusivity. Now regarding our ongoing efforts to present the growing body of data on LYB Marley, this summer we presented 12 abstracts at both the EASL and European Pediatric Hepatology Congresses. These data demonstrated LIV Marley's significant ability to reduce bilirubin, Symptoms associated with Alagille Syndrome and PFIC as well as real world data in which patients with Alagille Syndrome came off of the liver transplant waiting list after Lib Marley treatment. In addition, we published a seminal analysis in hepatology demonstrating that pruritus, Serum bile acids and bilirubin are significantly predictive for transplant and event free survival in Alagille syndrome, showing that 93% of Libmarly treated patients with keratitis reductions remain transplant free after 6 years. So in summary, the upcoming data readouts across indications along with the ongoing robust clinical evidence of LIVMIRLI in both Allagel and PFIC cements our leadership position in pediatric hepatology, and we remain committed to growing our presence in rare disease.

Speaker 4

And with that, I'll turn the call back over to Chris. Chris?

Speaker 2

Thanks, Pam. Myriam is proud to be the leader in pediatric hepatology. The team continues to deliver high growth with Lidmarly and make important advances in treatment options for cholestatic disease across our clinical programs. At the close of our acquisition of Kenadol and Kollbaum expected in the Q3 this year, we will have 3 approved rare disease products, An upcoming PDUFA date for label expansion for LYF Marley and 4 additional indications in development in high need orphan settings with upcoming clinical readouts. We are exceptionally positioned to expand our leadership and impact in rare disease and look forward to bringing these important medicines to patients around the world.

Speaker 2

With that, operator, please open the call for questions.

Operator

Thank you.

Speaker 2

Operator, do we have any questions in the queue?

Operator

I apologize. We have our first question from Steve Seedhouse of Raymond James. Your line is open.

Speaker 5

Good afternoon, guys. I wanted to ask a couple of questions about, Embark. The first one is just, are you like our patients screened and consented And ultimately enrolled in the study before they've had their Kasai procedure or in the days or weeks After. And I'm curious if there's a lead in period sort of before treatment where you track bilirubin kinetics and see Sort of what the response is to the surgery before initiating treatment. I have a couple of follow ups as well.

Speaker 4

Great. Thanks for the question. Appreciate it. So for the EMBARK study, the patients immediately post the size of the discussion happens Sometimes prior to the Kasai procedure, when they learn of the diagnosis after the baby is born. And so the consent Process can happen either before the Kasai surgery or immediately after the Kasai surgery, but the goal is to get these patients onto the was to get we're now fully enrolled, was These patients into the study as quickly as possible.

Speaker 4

There are no criteria for bilirubin in the study. So We're not looking at any bilirubin effects post the site. We're enrolling all patients. And we're really excited to have the study readout towards the end of the year.

Speaker 5

Okay, great. Thanks for that. And then just on the 6 month analysis, I'm curious if there is a formal assessment of like transplant or other liver events already in that 6 month time point Or let's say just through last follow-up of all the patients that are enrolled by the time you announced the 6 month bilirubin analysis.

Speaker 4

Yes. Thanks. So for the 6 month primary endpoint, as you know, is bilirubin, But we are assessing in other key secondary outcomes transplant at that 6 month time point as well as other thresholds of bilirubin that are predictive of transplant. And then these patients at the end of the double blind treatment period roll over into open label extension. We'll continue to follow those patients long term.

Speaker 5

Okay. And then just the last question I have was just on that open label extension. So I think correct me if I'm wrong, I think it would go through 24 months total, so 18 months post the 6 month analysis. And the question is, Do you think you'll have the ability to leverage natural history control cohort data in similar ways that Just based on some quality databases in Alagille and PFIC, for instance, here for biliary atresia? Or If not, is that work that you would hope to do at Myriam in the coming year?

Speaker 5

Thanks so much.

Speaker 4

Yes. That's a great question. We're actually doing that right now. We're working with natural history registries looking at kind of aligning those patients with the patients that are enrolled in our study and understanding a little bit more about thresholds and And then we'll obviously continue to look at long term treatment versus natural history. So all of that will be kind of as much as we can in the timeframe.

Speaker 4

The package will contain our data set plus work that we're doing supported by the Natural History and we'll have discussion with the FDA at that time.

Speaker 5

Great. Thanks so much.

Speaker 3

Thank you.

Operator

Your next question comes from Manu Baruja of SEB Leerink.

Speaker 6

Just there now, I guess. Thanks for taking the question, guys. A quick commercial question. I know we had a little bit of discussion last year around 2Q, 3Q Seasonality, can you give us a sense of where those trends are now? How should we think about modeling?

Speaker 6

And sort of what are the sort of key metrics we should be following or thinking about as we think about going through each season this year?

Speaker 7

Thanks for the question, Manny. Yes, so we feel that what we've seen in Q2 is really strong Demand that feel really good about how the business is playing heading into Q3. But worth mentioning, we're just now at the beginning of August. So We'll have to see how things unfold over the next couple of months, but overall feel really good about how the business is pointed going forward.

Speaker 6

Great. That's helpful. And can you separately, Looking forward, post presumptive deal close of the acquired assets, how should we think about Margin evolving over time. Obviously, you're going to have a little bit of expense that you bring on, but that's going to be coming with a substantial amount of revenue. Is there room for further margin expansion going forward?

Speaker 6

On what time horizon might we see that as we transition from this Q4, Q1 with revenues from all the assets put together through next year.

Speaker 2

Thanks Mike for the question. The overall picture of bringing KienaDol and Kholbom into Myriam, We do see a tremendous amount of synergies. So they will contribute substantially to margin at Myriam because of the overlap in our business. A lot of that behind some of the rationale why we're so excited about bringing these products into our portfolio. We're not going to be guiding towards margin, but what I can say is that the this puts us on a very sound financial footing and We do expect all three products to continue growing for the foreseeable future.

Speaker 2

So that will continue to improve financial performance as we go.

Speaker 6

Great. That's really helpful. Thanks guys. Congrats on the quarter.

Speaker 2

Yes. Thanks, Marty.

Operator

We now have David Labouc of Citibank. Please go ahead when you're ready.

Speaker 8

Hi. Thanks for taking our question. This is Debanjana on for David. So we wanted to ask if you could update us on the timing of the closure of the TAVIA transaction. And also could we have some insight as to how the operating spend might shift post closure?

Speaker 8

Thank you.

Speaker 2

Yes, thanks for the question. We're tracking towards closing this quarter, so we do expect a Q3 close. And then as we move into a post close time period, we're excited about Jumping right in to bring these products over to Myriam. Overall, they'll have a high impact In terms of margin and cash contribution right at the start in Q4. So see that synergy will play through And having these products in our hands, so expect that impact to show up with improved performance In Q4 from having these products at Myriam.

Speaker 8

Okay. So maybe just a quick follow-up on that. So What's your strategy on incorporating this product into your current pipeline?

Speaker 2

Absolutely. Yes, we can I'll have Pierre speak through some of the planning, but these fit very well with Lip Marley and the current Lip Marley business. I'd love to hear again into some of the strategies we have planned.

Speaker 7

Thanks, Chris. Yes. We're really excited about bringing these products into Myriam and excited about bringing several really talented folks from Travir and to Myriam as well. We have a team right now that's been out promoting with Marley to pediatric hepatologists and anticipate that team And also we'll have a team that focuses on physicians, our specialties that are often involved in identifying Patients with bile acid synthesis disorders, Zillow and spectrum disorders, etcetera, that focus on neurology, Hemology,

Speaker 3

Metagenetics.

Speaker 7

So have a liver focused team and what we're calling a metabolic focused team and modest Expansion to our field footprint. So pretty excited about how that plays out going forward.

Speaker 8

Okay. That's very helpful. Thank you.

Speaker 3

Thanks for your question.

Operator

Thank you. We now have Ed Arce of H. C. Wainwright.

Speaker 9

Hi, good afternoon, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking my questions. Perhaps first question regarding the IMbark data regarding beta positive And given the LaMali's orphan drug designation for biliary atresia, do you anticipate in BARC top line data along with Natural history data that you mentioned earlier. Do you think collectively that's enough to support with Mali's approval?

Speaker 2

Thanks, Thomas, for the question. Overall, that's our strategy to take that package to FDA. It's too early to say what The determination will be at this point, that bilirubin has not yet been fully established as an endpoint for approval. And that's the work that we're doing with the Embark data and the natural history work. So looking forward to putting together what we think will be a really compelling data set.

Speaker 9

Okay. Got it. And then perhaps, can you go over some Major differences that you know of so far between the FDA and MAA and the EMA, What they are looking for, for a proof of endpoint for beta reutrisia?

Speaker 2

Thanks for the question there. I mean, we see a pretty common point of view in what we've had for our conversations. And I think it's worth noting that There really is not a lot of clinical data or research that's been done in this space. So the IMbark study Really is a landmark. It's going to be the largest and first study of IBAT in the setting.

Speaker 2

And one of the few studies that actually has an intervention that early after the Kasai, so really important data set to bring forward to advance care for these patients.

Speaker 9

Got it. And then One final question for valexibat. You mentioned the interim analysis for both These are the VANTAGE studies both expected later this year. Can you tell us what type of data we can expect to see?

Speaker 4

Yes. Thank you for the question. So for the PSC VISTA study, this is a blinded interim. So we won't be sharing any data, but it is for dose selection and then the study will upsize if need to Or not and then carry on a potentially registrational Part 2 of the study. So wouldn't expect any top line data out there, but would expect to be able to communicate that the study is carrying on and continuing on.

Speaker 4

For the PBC VANTAGE study, We will be sharing top line data. This is an open interim analysis. And so we'll be able to have a look at the data and we'll be sharing that with you later.

Speaker 9

Okay, understood. Thank you again for taking the questions and looking forward to data from the VANTAGE study.

Operator

Our next question comes from Brian Skorney, Baird.

Speaker 10

Hi. This is Charlie on for Brian. So I want to ask about the SG and A. We've seen it climbing somewhat steadily over the past several quarters. Just wanted to know, Particularly as you start to integrate the bile acid team from Trevir, if we should expect that to keep rising, what kind of trajectory that's going to take and along a similar line thinking about elixibat as that hopefully gets closer to Getting commercialized, what kind of regulatory strategy are you taking going forward?

Speaker 10

And considering that these are adult Liver diseases, will this require a different commercial sales force? Or do you think that you can also leverage the The yield force that you have currently. Thank you.

Speaker 2

Thanks for the questions. I'll comment a bit on SG and A and ready for strategy and pass over to Peter to talk about the field team. And for SG and A, what we have what we see is It's kind of just modest increases over prior quarters, really driven by initially the launch in the U. S. And now some of the international markets that we brought on.

Speaker 2

So just for the base business, some incremental SG and A increase over time is what we'd expect. Then with adding in KienaDol and kohlbaum, that will bring incremental SG and A expense, but really modest compared to the top line opportunity. As Peter was mentioning earlier, there's a lot of synergy in how we approach those markets and the fit with Marley. So we are bringing some people over who have Great experience with those products from Travir, excited about bringing those folks into the Myriam family. And but overall, the Total contribution will be quite attractive with these products in our hands.

Speaker 2

As we move forward to melixibat, which Will be an opportunity to expand into adult hepatology. First, the Endpoint and registration to comment on. These studies incorporate pruritus as the primary endpoint, Similar to Alagille syndrome and the ongoing review for PFIC, the pruritus is the outcome for approval And that's what we're using for the registration approach for the adult indications. I'll let Peter comment on the commercialization strategy.

Speaker 1

Yes. Interesting.

Speaker 7

So yes, I think we would be able to leverage our commercialization team and infrastructure to Launch will have added to adults. Our current team is primarily pediatric focused. It goes to the large higher decile, larger volume Adult liver doctors, but we would look to expand into a broader group of adult hepatologists and gastroenterologists. I think of it as kind of an incremental build for that launch.

Speaker 9

Great. Thanks a ton

Speaker 10

for the color and congrats on the quarter.

Speaker 3

Thank you.

Operator

I'd like to hand the call back over to Chris for any final remarks.

Speaker 2

Great. Thanks, operator. And thanks again to everyone for joining today's call. Have a great day. Goodbye.

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Key Takeaways

  • Myriam delivered 86% year-over-year net product sales growth in Q2 2023 with $32.5 million in Libmarley revenue and $37.5 million in total revenue, outperforming its 50% growth guidance.
  • International expansion is gathering momentum with approval in Canada, launches in Germany and France, and ongoing pricing and reimbursement discussions targeting full European rollouts in 2024.
  • The pipeline is advancing rapidly, highlighted by a December 13, 2023 PDUFA decision for PFIC label expansion, full enrollment in the EMBARK biliary atresia study (top-line data later this year), interim analyses for elixibat in PSC and PBC by year-end, and a Q4 readout of the RESTORE Phase III trial in CTX.
  • Myriam expects to close the Q3 acquisition of Travir’s Kenodal and Kolbam products—together generating over $100 million in 2022 net sales—financed by a $210 million private placement to deepen its pediatric hepatology franchise.
  • The company ended Q2 with $330 million cash, maintained steady operating expenses, and anticipates margin expansion from the pending product acquisition and ongoing top-line growth.
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Earnings Conference Call
Mirum Pharmaceuticals Q2 2023
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