It has been about a month since the last earnings report for Mirum Pharmaceuticals, Inc. (MIRM) . Shares have added about 4.5% in that time frame, outperforming the S&P 500. Will the recent positive trend continue leading up to its next earnings release, or is Mirum Pharmaceuticals due for a pullback? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at the most recent earnings report in order to get a better handle on the important catalysts. Mirum's Q2 Earning ...

Mirum Pharmaceuticals, Inc.'s (MIRM) lead product, Livmarli (maralixibat), an orally administered ileal bile acid transporter ("IBAT") inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) worldwide. Sales of Livmarli have been rising steadily since its approval and launch in 2021. The FDA approved a label expansion for Livmarli oral solution to include the treatment of cholestatic pruritus in patients aged five years and older with progressive familial i ...

Financial Data and Key Metrics - Net product revenue for Q2 2024 was $77.8 million, a 139% increase compared to $32.5 million in Q2 2023 [4][13] - Total operating expenses for Q2 2024 were $102 million, including R&D expenses of $32.7 million, SG&A expenses of $49.2 million, and cost of sales of $20.2 million [13] - Net loss for Q2 2024 was $24.6 million, or $0.52 per share [14] - Cash, cash equivalents, and investments totaled $295.4 million as of June 30, 2024, a decrease of $7.4 million from the prior quarter [14] Business Line Performance - LIVMARLI global net product sales grew to $47.2 million in Q2 2024, a 45% increase compared to the same quarter last year [8] - U.S. sales of LIVMARLI were $35.5 million, while international sales were $11.7 million [8] - CHOLBAM and CHENODAL combined net product sales were $30.5 million in Q2 2024 [9] Market Performance - LIVMARLI demand growth was strong internationally, with favorable price negotiations in Germany [8] - The European Commission endorsed LIVMARLI for PFIC patients aged 3 months and older, highlighting its significant benefit [9] Strategic Direction and Industry Competition - The company is focused on growing its commercial business, expanding indications for approved medicines, advancing volixibat in adult cholestasis, and seeking pipeline growth opportunities [14] - The company submitted an NDA for chenodiol in CTX, aiming to address an underdiagnosed population and secure orphan exclusivity [4] - The VISTAS PSC and VANTAGE PBC studies for volixibat showed positive interim results, positioning the company to bring the first medicine to the PSC patient community [7] Management Commentary on Operating Environment and Future Outlook - The company is on track to achieve its full-year revenue guidance of $310 million to $320 million [7][9] - Management expressed confidence in the continued growth of LIVMARLI, supported by new patient additions and label expansions [8] - The company is optimistic about the potential of volixibat to set a new standard in addressing cholestatic pruritus [11] Other Important Information - The company launched the EXPAND study to evaluate LIVMARLI in patients with cholestatic pruritus across various rare conditions, targeting enrollment of approximately 45 patients by 2026 [12] - The VISTAS PSC study is expected to complete enrollment in the second half of 2025, while the VANTAGE PBC study aims to complete enrollment in 2026 [10][11] Q&A Session Summary Question: How does the company plan to broaden LIVMARLI's reach within the PFIC segment? - The company has seen strong initial reception for LIVMARLI in both Alagille syndrome and PFIC indications, with equal footing in the U.S. and leadership in Europe [16] Question: What are the enrollment strategies for VISTAS and VANTAGE trials? - Enrollment is progressing well, with investigators excited about the potential impact of volixibat for PBC and PSC patients [18] Question: What is the duration and time horizon for the EXPAND study? - The EXPAND study is expected to complete enrollment in 2026, with results anticipated to support wider use of LIVMARLI in cholestatic pruritus [20] Question: How does the company view seasonality in its commercial business? - The company does not identify strong seasonality effects due to the ultra-rare nature of its products, though quarter-to-quarter variability can occur [21] Question: What is the timeline for OUS pricing reference dynamics? - The pricing reference impact is expected to play out in Q2 and Q3, with normalization anticipated by Q4 [22] Question: How was the sample size for the EXPAND study determined? - The sample size was based on powering for the primary endpoint, with confidence derived from prior experience with LIVMARLI [24] Question: What data supports enrolling biliary atresia patients in the EXPAND trial? - The EXPAND study targets a different patient population than EMBARK, focusing on chronic liver disease patients with cholestatic pruritus [26] Question: Where will the interim PBC data for volixibat be presented? - The company is preparing an abstract for submission to an upcoming congress, with additional details to be included [29] Question: What is the company's business development strategy? - The company is focused on rare disease opportunities, particularly in pediatric rare diseases, with a high bar for new opportunities [31] Question: Will the company pursue a label expansion for PFIC patients below 12 months? - The company is evaluating the potential for another sNDA based on infant data but feels the current label captures most targeted patients [33] Question: How does the FDA view the EXPAND trial for label expansion? - The EXPAND study was suggested by the FDA and is designed to address cholestatic pruritus across various conditions [36] Question: How will pruritus be measured in the EXPAND trial? - Pruritus will be measured using an observer-rated ItchRO for pediatric patients and self-reported endpoints for adults [37][39] Question: What is the formulation used in the EXPAND trial? - The EXPAND trial will use the same liquid formulation of LIVMARLI that is commercially available [40] Question: Are biliary atresia patients from EMBARK eligible for EXPAND? - The EXPAND study targets a different patient population, with no overlap expected from EMBARK [42] Question: What is driving demand growth for CHOLBAM and CHENODAL? - Demand growth is steady, with Q1 2024 impacted by a cyberattack, and no major plans for CHOLBAM label expansion [45] Question: How is the PFIC launch progressing in the U.S.? - The PFIC launch is progressing well, with modest revenue contribution expected in 2024 and stronger growth anticipated in 2025 [48] Question: What is the commercial opportunity for CHENODAL in CTX? - The CTX opportunity includes increasing diagnosis rates and promoting CHENODAL to a broader patient population [53] Question: Has the FDA or EMA reviewed interim data for VANTAGE and VISTAS? - Regulatory agencies have not reviewed interim data, with discussions planned after final results are available [55]

Mirum Pharmaceuticals, Inc. (MIRM) incurred second-quarter 2024 loss of 52 cents per share, wider than the Zacks Consensus Estimate of a loss of 48 cents. The company had reported a loss of 92 cents per share in the year-ago quarter. Revenues in the second quarter totaled $77.9 million, up almost 107.7% year over year. The figure beat the Zacks Consensus Estimate of $75 million. The top line comprised Livmarli (maralixibat) sales, newly acquired Cholbam and Chenodal sales and minimal license and other reven ...

Mirum Pharmaceuticals, Inc. (MIRM) came out with a quarterly loss of $0.52 per share versus the Zacks Consensus Estimate of a loss of $0.48. This compares to loss of $0.92 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -8.33%. A quarter ago, it was expected that this company would post a loss of $0.40 per share when it actually produced a loss of $0.54, delivering a surprise of -35%. Over the last four quarters, the company ...

Company Overview - Mirum Pharmaceuticals, Inc. (MIRM) shares increased by 7.3% to close at $43.33, with notable trading volume compared to typical sessions, and a total gain of 21.9% over the past four weeks [1][3] Earnings and Revenue Expectations - The company is projected to report a quarterly loss of $0.48 per share, reflecting a year-over-year change of +47.8%. Expected revenues are $74.57 million, which is an increase of 98.9% from the same quarter last year [2][6] Stock Performance and Trends - The consensus EPS estimate for Mirum Pharmaceuticals has remained unchanged over the last 30 days, indicating that stock price movements may not continue without earnings estimate revisions [3][6] - The stock currently holds a Zacks Rank of 3 (Hold), suggesting a neutral outlook [7] Industry Context - Mirum Pharmaceuticals operates within the Zacks Medical - Biomedical and Genetics industry. Another company in this sector, Oculis Holding AG (OCS), saw a 1.5% increase in its stock price, but has returned -2.1% over the past month [4][7] Recent Developments - The FDA approved a label expansion for Mirum's lead drug, Livmarli, for treating cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 12 months and older, which may have contributed to the recent share price rally [5]

Lastly, MIRM is also working on PBC and PSC treatments. For PBC, trials showed a 3.8-point reduction from baseline in the primary endpoint of pruritus, indicating a significant improvement in reducing this primary symptom of PBC. For PSC, outcomes were also encouraging, preliminary meeting efficacy thresholds that need to be confirmed in further studies with 20mg daily doses. Quality at a Premium: Valuation Analysis According to Seeking Alpha's dashboard on MIRM, the company is projected to generate roughly ...

Core Opinion - The European Commission has approved Livmarli for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and older, which is expected to drive sales growth for Mirum Pharmaceuticals [9][13]. Company Developments - Mirum Pharmaceuticals has submitted an additional supplemental new drug application to the FDA for a higher concentration formulation of Livmarli, aiming to include younger patients with PFIC in the treatment [2]. - Livmarli generated sales of $42.8 million in the first quarter of 2024, reflecting a year-over-year increase of nearly 47% [11]. - The FDA previously approved Livmarli for cholestatic pruritus in patients aged five years and older with PFIC in March [10]. Clinical Data - Data from the phase III MARCH study indicated that Livmarli significantly reduced pruritus and serum bile acids compared to placebo, and improved total bilirubin and growth [5][16]. Market Performance - Shares of Mirum Pharmaceuticals increased by 4.3% following the European Commission's approval announcement [13]. - Year-to-date, Mirum's shares have rallied 21.6%, contrasting with a 7.1% decline in the industry [15]. Competitor Analysis - Other healthcare stocks with favorable rankings include Acrivon Therapeutics, Anavex Life Sciences, and RAPT Therapeutics, with varying degrees of performance and loss per share estimates [3][4][8][12].

Last month, the company submitted a new drug application (NDA) seeking approval for chenodiol for treating cerebrotendinous xanthomatosis (CTX) patients in the United States. The NDA filing was based on positive data from the phase III RESTORE study. Chenodiol is not currently approved but has received a medical necessity medicine status from the FDA for the treatment of CTX, a rare autosomal genetic disorder. We remind investors that in August 2023, Mirum acquired all assets of Travere Therapeutics' bile a ...

Shares of Mirum Pharmaceuticals, Inc. (MIRM) were up 27.3% on Jun 17 after the company announced positive interim data from two phase IIb studies, which evaluated its oral ileal bile acid transporter inhibitor, volixibat, for treating patients with primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC). The phase IIb VISTAS study investigated volixibat for the treatment of PSC, while the phase IIb VANTAGE study evaluated volixibat for the treatment of PBC. Interim data from the VANTAGE s ...