Financial Data and Key Metrics Changes - Total net product sales for 2024 were $336.4 million, a significant increase from $178.9 million in 2023, representing an 88% year-over-year growth [18][20] - In Q4 2024, total net product sales reached $99.4 million, compared to $69.5 million in Q4 2023, marking a 43% increase [18][19] - The company ended 2024 with cash, cash equivalents, and investments of $293 million, an increase of approximately $7 million from the start of the year [20] Business Line Data and Key Metrics Changes - Lebmarly's total net product sales for 2024 were $213.3 million, reflecting a 50% increase compared to 2023 [19] - Bile acid medicines generated total net product sales of $123.1 million for 2024, with Q4 sales of $35 million, representing a 25% growth over Q4 2023 [19][20] Market Data and Key Metrics Changes - The company anticipates net product sales between $420 million and $435 million for 2025, indicating expected growth driven by strong demand for its medicines [9][20] - The market for Fragile X syndrome is estimated to have a total addressable market of over $1 billion, with a patient population of approximately 50,000 in the U.S. [30][32] Company Strategy and Development Direction - The strategic priorities for 2025 include expanding the global growth of commercial medicines, advancing the pipeline, selectively pursuing product acquisitions, and maintaining scientific and financial discipline [6][7] - The company aims to leverage its financial position to acquire rare disease products that present significant value creation opportunities [25][26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for Lebmarly and bile acid medicines, expecting continued growth in 2025 [9][10] - The company is well-funded and expects to maintain positive cash flow while progressing toward multiple upcoming pipeline catalysts [20] Other Important Information - The FDA approved Citexley for cerebrotendinous xanthomatosis (CTX) in adults, which is expected to enhance promotional efforts and patient reach [9][10] - The company has expanded its pipeline with the addition of MRM-3379 for Fragile X syndrome, indicating a focus on high unmet needs in rare genetic neurology [11][17] Q&A Session Summary Question: Strategic question regarding capital allocation and business development - Management highlighted the unique position of the company in the rare disease market, allowing for potential acquisitions of valuable products [25][26] Question: Market penetration and competition for MRM-3379 - Management discussed the substantial upside in the market potential for Fragile X syndrome and the applicability of the mechanism in other conditions [30][32] Question: Alagille syndrome market penetration - Management estimated that approximately 40% of the eligible treatment population in the U.S. has been penetrated, indicating significant growth potential [36][38] Question: Impact of recent CTX approval on sales trajectory - Management expects a gradual increase in patient diagnoses and treatment conversions to Citexley, positively impacting sales [40][41] Question: Patient mix between Alagille syndrome and PFIC - Management confirmed that most Lebmarly patients are Alagille syndrome patients, with a notable increase in PFIC patient starts contributing to growth [46][47] Question: Commercial team expansion for expanded pruritus - Management indicated that no significant expansion of the commercial team is anticipated, as the same prescribing universe will be leveraged [49][50] Question: VISTA study enrollment and timeline for data - Management projected a six-month timeline for top-line data after enrollment completion for the VISTA study [68][69] Question: Interim data from the EXPAND study - Management confirmed that no interim analysis is planned for the EXPAND study, with a focus on completing enrollment by 2026 [73][74]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 __________________________________________________________________________________________ FORM 10-K __________________________________________________________________________________________ ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR T ...

Mirum Pharmaceuticals, Inc. (MIRM) closed the last trading session at $49.93, gaining 2.9% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $63 indicates a 26.2% upside potential.The mean estimate comprises 10 short-term price targets with a standard deviation of $8.30. While the lowest estimate of $50 indicates a 0.1% increase from the current price level, the most optimistic analy ...

Most of us have heard the dictum "the trend is your friend." And this is undeniably the key to success when it comes to short-term investing or trading. But it isn't easy to ensure the sustainability of a trend and profit from it.Often, the direction of a stock's price movement reverses quickly after taking a position in it, making investors incur a short-term capital loss. So, it's important to ensure that there are enough factors -- such as sound fundamentals, positive earnings estimate revisions, etc. -- ...

When it comes to short-term investing or trading, they say "the trend is your friend." And there's no denying that this is the most profitable strategy. But making sure of the sustainability of a trend to profit from it is easier said than done.The trend often reverses before exiting the trade, leading to a short-term capital loss for investors. So, for a profitable trade, one should confirm factors such as sound fundamentals, positive earnings estimate revisions, etc. that could keep the momentum in the st ...

Mirum Pharmaceuticals, Inc. (MIRM) could be a solid addition to your portfolio given its recent upgrade to a Zacks Rank #2 (Buy). An upward trend in earnings estimates -- one of the most powerful forces impacting stock prices -- has triggered this rating change.A company's changing earnings picture is at the core of the Zacks rating. The system tracks the Zacks Consensus Estimate -- the consensus measure of EPS estimates from the sell-side analysts covering the stock -- for the current and following years.I ...

Shares of Mirum Pharmaceuticals (MIRM) were up 12.1% this week, driven by investor enthusiasm after the company announced encouraging preliminary sales numbers for the fourth quarter and full year 2024, along with an encouraging outlook and growth strategy for 2025.Mirum’s commercial portfolio comprises of lead product, Livmarli (maralixibat), which is approved for treating cholestatic pruritus in patients with Alagille syndrome worldwide. The drug is also approved for treating certain patients with progres ...

While "the trend is your friend" when it comes to short-term investing or trading, timing entries into the trend is a key determinant of success. And increasing the odds of success by making sure the sustainability of a trend isn't easy.Often, the direction of a stock's price movement reverses quickly after taking a position in it, making investors incur a short-term capital loss. So, it's important to ensure that there are enough factors -- such as sound fundamentals, positive earnings estimate revisions, ...

Mirum Pharmaceuticals: Transforming Lives in Rare Disease J a n u a r y 2 0 2 5 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to managem include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and oppor opportunities for LIVMARLI, CHOLBAM, chenodiol, and Mirum's clinica ...

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