Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q1 2023 Earnings Call Transcript

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q1 2023 Earnings Call Transcript May 7, 2023

Operator: Operator Ladies and gentlemen, welcome to the Mirum Pharmaceuticals Reports First Quarter 2023 Financial Results and Profile Business Update. My name is Glenn, and I will be the operator for today’s call. . I’ll now hand over to your host, Andrew McKibben to begin. Andrew, please go ahead.

Andrew McKibben: Thanks, Glenn, and good afternoon, everyone. I’d like to welcome you to Mirum Pharmaceuticals’ first quarter 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, Mirum issued a news release announcing the company’s results for the first quarter of 2023. Copies of this news release and SEC filings can be found in the Investors section of our website. Full details and updates from the quarter can be found in our news release and 10-Q issued today. 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 Mirum and our programs based on management’s current expectations, including statements regarding Mirum’s current and future business plans, development programs, and regulatory expectations, strategies, prospects, market opportunities and financial forecasts and guidance.

Mirum 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 Mirum’s 10-K for the year ended December 31, 2022, and any subsequent reports filed with the SEC. With that said, I’d like to turn the call over to Chris. Chris?

Chris Peetz: Thank you, Andrew, and good afternoon to everyone joining us on the call today. It’s been another exceptional quarter for Mirum, as we continue to advance our leadership position as a high growth rare disease company focused on life-changing medicines. Our clinical, regulatory and business achievements in the first quarter with total revenue of $31.6 million are driven by our team’s passion and expertise in addressing urgent unmet needs for patients with rare disease. With our five part strategy to become a global leader in rare disease in place, we’re pleased with the progress we’ve already made in 2023. First, we’ve continued to build on the successful launch of LIVMARLI in Alagille syndrome in the U.S., tracking well on our guidance of 50% year-over-year net product sales growth driven by new patient starts.

Second, our international business is also building well with the recent launch in Germany and early access programs in France and other international partner markets. We look forward to additional launches around the globe later this year and into 2024. Third, we’ve accomplished an important step in our strategy to expand the label for LIVMARLI. In the first quarter, we announced label expansion to include patients three months and older. In addition, we submitted a supplemental NDA and European equivalent with our positive LIVMARLI PFIC Phase 3 data. Our team is preparing for the December 13 PDUFA date and is excited about bringing LIVMARLI as a new treatment option advancing care for PFIC patients. We also anticipate providing top-line data from the EMBARK study in Biliary Atresia in the second half of this year, a third potential indication for LIVMARLI.

Photo by Raimond Klavins on Unsplash

And fourth, in advancing into adult indications, we are conducting potentially pivotal studies for Volixibat in Primary Sclerosing Cholangitis and Primary Biliary Cholangitis and look forward to providing interim updates from these studies in the second half of this year. And the fifth part of our strategy, we continue to evaluate opportunities across rare diseases for leverage Mirum’s industry-leading capabilities. We also recently took a significant step towards enabling our strategy with convertible note financing of approximately $316 million aggregate principal amount. This financing enabled the repurchase of the nearly 10% royalty committed to our prior royalty investor and added additional growth capital to the balance sheet. We were excited to see the high level of interest in supporting our strategy in this well oversubscribed transaction.

We continue to project that our balance sheet provides three plus years of runway beyond cash flow breakeven. We have started 2023 off with great momentum, and I’m excited about what the Mirum team is poised to achieve this year and beyond as we provide patients with life-changing medicines. And with that, I’ll pass the call over to Peter to discuss our commercial business in more detail before Pam gives an R&D update. Peter?

Peter Radovich: Thanks, Chris. We are pleased with the $29.1 million in LIVMARLI net product sales in the first quarter of 2023, which included $24.7 million from the U.S. and $4.4 million from international markets. The U.S. growth was driven by the addition of new patients as well as a strong retail cadence. And we’re pleased to report that substantially all of the key pediatric liver accounts in the U.S. have prescribed LIVMARLI. Based on their positive clinical experience and ease of access with Mirum Access Plus, health care professionals report that they intend to increase the use of LIVMARLI across their Alagille syndrome patients. Taken together, the U.S. business performance is tracking well against our guidance for 50% growth in 2023 net product sales.

Now turning to international markets. In Q1, we launched LIVMARLI in Germany, and it’s off to a great start. We’ve already converted early access program patients to commercial drug, and we have seen impressive de novo demand, in line with what we saw in the U.S. launch. Pricing and reimbursement discussions are ongoing in major European markets, which we expect to unfold throughout 2023, with full launches in European countries beyond Germany starting in 2024. Also, we expect named patient sales in European and distributor markets to continue throughout 2023, although we anticipate quarter-to-quarter variability in order patterns. In summary, LIVMARLI is in a strong position after year and a half in market as the first and only treatment for Alagille syndrome that is providing rapid symptomatic relief and long-term improvement in outcomes.

LIVMARLI is becoming standard of care in treating cholestatic pruritus with over 600 Alagille syndrome patients treated globally to date. Clinicians have gained familiarity with and confidence in LIVMARLI’s robust clinical profile as well as its exceptional access in patient support. And we believe this provides a strong competitive position, propelling a continued growth story in Alagille syndrome with exciting pediatric cholestasis indication expansion opportunities just on the horizon. And on that note, I’ll turn the call over to Pam. Pam?

Pamela Vig: Thanks, Peter. In the first quarter, we achieved important milestones in our pipeline that support the growth of our programs and ability to impact more patients. As a reminder, that such a problem in all of these diseases studied in our pipeline is accumulation of bile acids. In a classic cholestatic setting, IBAT inhibition has been shown to directly reduce toxic bile acid accumulation, the associated debilitating symptomatic burden and an Alagille LIVMARLI showed improved long-term outcomes versus standard of care. The benefits of profound IBAT inhibition was shown by the impressive data from our March PFIC Phase 3 study announced at the end of last year. And we’re happy to share that our supplemental NDA submission was accepted in the PDUFA date of December 13 of this year.

And in April, we also submitted for approval of LIVMARLI in PFIC to the EMA for patients two months of age and older. Now the data from the March PFIC study proved our hypothesis with greater IBAT inhibition using higher dosing ratio for LIVMARLI drove a highly statistically significant reduction in both pruritus and serum bile acids across the broadest range of genetic PFIC types studied to date. In addition, early and statistically significant improvements in growth as well as in bilirubin, we’ve seen in patients treated with LIVMARLI, where 40% of patients with abnormal bilirubin levels that they find normalized for bilirubin versus zero in the placebo group. Now these data suggest an overall clinical improvement beyond pruritus for these patients, and we have heard strong positive feedback from the physician community and if approved, look forward to the opportunity to expand LIVMARLI to a broad pivot population.

We’re also happy to announce that we remain on track for top-line data readout for our Biliary Atresia program for the second half of this year. This study will provide the first placebo-controlled data for an IBAT inhibitor in this study, and we’re really excited to share them all when the study is completed. As a reminder, the primary endpoint is the Biliary Atresia study assessment bilirubin at 26 weeks as bilirubin is the most predictive marker of disease progression and transplant in this setting. And the dosing regimen in the BA study is the same as the March PFIC study and the bilirubin improvements we observed in March are extremely encouraging as we look forward to our Biliary Atresia readout. Now turning to our pipeline programs in adult cholestatic indications.

We expect interim analysis from our studies of Volixibat in both PSC and PBC in the second half of this year. We’ve previously shown that marked reductions in pruritus and serum bile acids can be achieved with IBAT inhibition in PSC, underscoring the potential for Volixibat in this setting. And currently, there are no approved therapies for PSC and roughly 60% of patients are being actively managed for pruritus with largely ineffective off-label therapies, none of which lower bile acids. Building off of our learnings from our pediatric program, we are dosing at a higher level and BID dosing regimen, which adds to our confidence in these upcoming interim analysis. And finally, I’m proud of our team’s academic and collaborative efforts as we continue to advance the benefit for patients from clinical studies to the real world.

We’re committed to leveraging our deep experience across all of these cholestatic disease settings as we continue our scientific leadership. And with that, I’ll turn the call back over to Chris. Chris?

Chris Peetz: Thanks, Pam. Mirum is poised for continued growth throughout the years ahead. We anticipate that 2023 will be another transformative year with strong commercial growth, potential PFIC label expansion, top-line data of the first randomized dataset of an IBAT inhibitor in Biliary Atresia and interim analysis from our potentially pivotal Volixibat studies. We remain excited about the growth prospects and potential of Mirum as we work to provide new medicines to patients with rare disease. And with that, operator, please open the call for questions.

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Q&A Session

Operator: Thank you. . We have our first question comes from Jessica Fye from JPMorgan. Jessica your line is now open.

Unidentified Analyst: Hey, this is Nick on for Jess. Thanks for taking our questions. Two quick ones for me. One, could you just talk a little bit about the factors that resulted in that timing shift for the Phase 2b VISTAS trial from mid-2023 to the second half ’23? Is that more of a narrowing or a shift?

Pamela Vig: Yes, hi. I’m happy to answer that question. Thanks for the question. So the reason for the delay in the PSC study, it’s really a slight delay and mostly based on screen sales and there’s a number of reasons why patients are screening out. One is the mix of lab values some pruritus for compliance during screening where they have to put in their pruritus scoring. And if they’re not compliant, they also get kicked out, because we want to make sure that they can remain in the study. And then also some for itch score that is not meeting the minimum threshold to enter the study. What we’re doing with some of this is we’ve got plans on advancing tools and education on how we standardize training and interpretation of those itch scores and just continuing to drive forward, and we’re starting to see more movement and good activity.

Unidentified Analyst: Great. And as a quick follow-up. I know there have been some PBC trials in the past that have run into issues with high placebo responses on itch. Can you kind of just discuss how the VANTAGE trial is designed to help kind of mitigate those risks?

Pamela Vig: Yes. Thanks for the question. So in our PBC clarity study, as you know, we — there was a — we ourselves had a placebo response rate. So we took those learnings applied them into this study design. We baked some of that into the assumptions for our analysis, and we also adjusted our study designed to accommodate for placebo failures in account for that. I don’t think I can give specifics about the way that we’re doing that in the trial, but we have thought very deeply about that and taken that into account.

Unidentified Analyst: Great. Thanks so much.

Chris Peetz: Thanks for the questions.

Operator: Thank you. Our next question comes from Mani Foroohar from SVB Securities. Mani your line is now open.

Mani Foroohar: A quick question on Biliary Atresia. How should we think about the scale of the opportunity, both in terms of the number of patients, which I think is fairly well defined, but also at what point would patients be treated and how long would they be treated? Is the expectation here that you’d pursue a lifelong study? Or would you expect it from your clinical feedback that position in the case of a successful study commercialization would after one year, two years, some period of time, look to withdraw patients rather than leave them on drug for life?

Chris Peetz: Thanks, Mani, for the question. I’ll — I mean, I want to just kind of first comment, Biliary Atresia is the most common indication for transplant in liver transplant and pediatrics. So really in terms of scale of unmet need and impact you can have, this is a really important indication. I shall ask Peter to maybe speak to a little bit of how to think about how this evolves over time, the duration of treatment, because there are some nuances on how we think this indication can build over time.