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 ...

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]