Earnings and revenue growth expectations certainly give a good sense of the potential strength in a stock, but empirical research shows that trends in earnings estimate revisions are strongly correlated with near-term stock price movements. For Mirum Pharmaceuticals, the consensus EPS estimate for the quarter has remained unchanged over the last 30 days. And a stock's price usually doesn't keep moving higher in the absence of any trend in earnings estimate revisions. So, make sure to keep an eye on MIRM goi ...

Loading... Interim results from the VANTAGE study evaluating volixibat in patients with PBC demonstrated a statistically significant (-3.82, p<0.0001) improvement in pruritus (itch) for volixibat and a placebo-adjusted difference of -2.32 points in the primary endpoint, p=0.0026, as measured by the Adult ItchRO scale. 75% of patients on volixibat achieved a greater than 50% reduction in serum bile acids. No new safety signals were observed, and adverse events were similar between the 20 and 80 mg treatment ...

It has been about a month since the last earnings report for Mirum Pharmaceuticals, Inc. (MIRM) . Shares have lost about 3.4% in that time frame, underperforming the S&P 500. Will the recent negative trend continue leading up to its next earnings release, or is Mirum Pharmaceuticals due for a breakout? 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 drivers. Mirum's Q1 Earnings ...

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. In March 2024, 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 progres ...

Financial Data and Key Metrics - Total revenue for Q1 2024 was $69.2 million, a significant increase from $31.6 million in Q1 2023 [43] - Net product sales for Q1 2024 were $68.9 million, representing a 137% increase from Q1 2023 [31] - LIVMARLI global net product sales grew to $42.8 million in Q1 2024, up from $41.4 million in Q4 2023 and $29.1 million in Q1 2023 [34] - U.S. LIVMARLI sales were $30.8 million, while international sales were $12.1 million [34] - Net loss for Q1 2024 was $25.3 million or $0.53 per share [45] - Cash, cash equivalents, and investments increased to $302.8 million as of March 31, 2024, up from $286.3 million at the end of 2023 [45] Business Line Data and Key Metrics - LIVMARLI continues to show strong performance with demand growth across all geographies [27][34] - CHOLBAM and CHENODAL net product sales were $26.1 million in Q1 2024 [29] - The company is advancing volixibat in PSC and PBC, with interim analyses scheduled for June 2024 [32][37] Market Data and Key Metrics - The U.S. business was impacted by a healthcare cybersecurity incident, resulting in a temporary $3 million disruption to Q1 sales [28] - The company is preparing for the European market launch of LIVMARLI for PFIC, pending EMA approval [17][105] Company Strategy and Industry Competition - The company is focused on expanding the indications of its commercial medicines, including LIVMARLI for PFIC and CHENODAL for CTX [24][25] - Volixibat is being developed for PSC and PBC, with no approved therapies currently available for these conditions [32] - The company remains disciplined in its business development approach, focusing on rare disease settings [81] Management Commentary on Operating Environment and Future Outlook - Management is confident in achieving the full-year revenue guidance of $310 million to $320 million, driven by demand growth and new approvals [27][31] - The company expects reimbursement and payer discussions for LIVMARLI in PFIC to materialize over the next few quarters [35] - The interim analysis for volixibat in PSC and PBC is expected to provide meaningful treatment effect data and dose selection [39][40] Other Important Information - The company is preparing for the NDA submission of CHENODAL for CTX treatment [25] - The interim analysis for volixibat in PSC and PBC will focus on pruritus improvement, safety, and serum bile acids [40] Q&A Session Summary Question: Impact of the Change Healthcare cybersecurity incident on revenue - The $3 million impact was a one-time effect, with no lingering effects expected in future quarters [58][59] - The impact was balanced across products, proportional to their size [60] Question: Expectations for volixibat studies - The studies are designed to be broadly applicable, allowing patients on or off ursodiol and with varying levels of alphos [52] - The interim analysis will focus on pruritus improvement, safety, and serum bile acids, with no statistical significance expected [40][83] Question: European market preparation for LIVMARLI in PFIC - The company is ready to launch LIVMARLI in Europe upon EMA approval, with prescribers identical to those for Alagille syndrome [105] Question: Revenue contribution from PFIC in Q1 2024 - There was no significant PFIC contribution in Q1 2024, with revenue expected to grow in the back half of the year [103] Question: Drivers for bile acid product sales - The Change Healthcare cyberattack impacted the entire portfolio, with quarter-to-quarter volatility due to the ultra-rare nature of the diseases [106]

Mirum Pharmaceuticals, Inc. (MIRM) incurred first-quarter 2024 loss of 54 cents per share, wider than the Zacks Consensus Estimate of a loss of 40 cents. The company had reported a loss of 80 cents per share in the year-ago quarter.Revenues in the first quarter totaled $69.2 million, up almost 118.9% year over year. The figure, however, missed the Zacks Consensus Estimate of $70 million. The top line comprised Livmarli (maralixibat) sales, newly acquired Cholbam and Chenodal sales and minimal license and ot ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _____________________________________________________________________________ FORM 10-Q _____________________________________________________________________________ (Mark One) x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transit ...

FOSTER CITY, Calif. – May 8, 2024 - Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for first quarter 2024 and provided a business update. "In the first quarter, we made great progress across a number of our strategic objectives that support both the near- and long-term growth of Mirum while maintaining a strong financial position," said Chris Peetz, chief executive officer at Mirum. "Our commercial business is performing well, and we saw continued growth in demand for LIVMARLI g ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number 001-38981 Mirum Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) | Delaware | 83-1281555 | | --- | --- | | (S ...

Financial Data and Key Metrics Changes - Total revenue for Q4 2023 was $69.6 million, with total revenue for the year ending December 31, 2023, at $186.4 million, compared to $27.9 million and $77.1 million for Q4 and the full year 2022 respectively, indicating significant growth [60] - Net product sales for the year were $178.9 million, reflecting a year-over-year growth of 142% [45][60] - The net loss for the year was $160.9 million or $3.94 per share, which included non-cash stock-based compensation of $35 million and intangible amortization of $10.4 million [61] Business Line Data and Key Metrics Changes - LIVMARLI's total global net product sales grew to $41.4 million in Q4, with U.S. sales at $31.4 million and $111 million for the year, representing approximately 63% year-over-year growth [54][60] - CHENODAL and CHOLBAM generated net product sales of $28.1 million in Q4, marking the highest quarter-to-date for bile acid products [70] Market Data and Key Metrics Changes - The company expects net product sales to reach $310 million to $320 million in 2024, representing over 70% growth from 2023 [55][60] - The international market for LIVMARLI is showing strong uptake, although variability in quarterly revenue is anticipated [54] Company Strategy and Development Direction - The company aims to leverage its expertise in cholestasis and expand its commercial portfolio, focusing on upcoming label expansion opportunities and pipeline growth [46][50] - The strategy includes preparing for multiple regulatory and clinical catalysts, with a PDUFA date for LIVMARLI and PFIC set for March 13, 2024 [48][57] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of LIVMARLI and the overall business, highlighting the strong demand and progress in international markets [19][54] - The company is well-funded with cash and investments totaling $286.3 million as of December 31, 2023, indicating a strong financial position to support future growth [62] Other Important Information - The company welcomed Dr. Joanne Quan as the new Chief Medical Officer, emphasizing her leadership and expertise in driving future value creation [51] - The company is focused on addressing unmet medical needs in rare diseases, particularly through the development of therapies for conditions like PSC and PBC [59][117] Q&A Session Summary Question: Can you discuss the 2024 net product sales guidance and contributions from LIVMARLI versus CHENODAL and CHOLBAM? - Management refrained from providing specific product-level guidance but noted strong dynamics across products, with expectations of mid-single-digit growth for bile acid products and continued strong growth for LIVMARLI [66] Question: What are the expectations for the VANTAGE study and its impact on outcomes? - Management indicated that significant impact on pruritus would be clinically meaningful and that the study is designed to address an important unmet medical need [88][90] Question: Can you provide details on the sales numbers for LIVMARLI and bile acid products for Q4? - LIVMARLI totaled $41.4 million worldwide, with $31.4 million from the U.S. and $10 million internationally, while bile acid products generated $28.1 million for Q4 [105]