The future looks bright for this high-flying biotech stock.Mirum Pharmaceuticals' (MIRM +2.79%) share price has more than doubled over the last 12 months. Is this biotech stock about to soar again in 2026? I think so – for two key reasons.At an inflection pointIn several important ways, Mirum is at an inflection point. It achieved positive cash flow from operations in 2025. That's a significant milestone for any small biotech company.NASDAQ : MIRMMirum PharmaceuticalsToday's Change( 2.79 %) $ 2.63Current Pr ...

Mirum Pharmaceuticals Inc. (NASDAQ:MIRM) is one of the best mid-cap growth stocks to buy right now. On January 13, Baird analyst Brian Skorney raised the firm’s price target on Mirum Pharmaceuticals to $95 from $81, while maintaining an Outperform rating. This revision came as the firm updated its valuation model after preliminary results delivered a beat-and-raise on earnings and guidance. The stronger-than-expected data provides a solid foundation heading into a high-activity year in 2026. Furthermore, ...

Core Insights - CorMedix (CRMD) and Mirum Pharmaceuticals (MIRM) attract investors in the rare-disease biotech sector due to smaller patient populations that can yield significant revenue through premium pricing and regulatory incentives [1] - Both companies benefit from clearer clinical endpoints and faster regulatory pathways, allowing quicker market entry compared to traditional drugs [1] CorMedix (CRMD) Overview - CorMedix's lead product, DefenCath, received FDA approval in late 2023 as the first antimicrobial catheter lock solution in the U.S., aimed at reducing catheter-related bloodstream infections in kidney failure patients [2] - DefenCath generated $167.6 million in net sales in the first nine months of 2025, indicating strong market adoption [7] - The company plans to expand DefenCath's label into total parenteral nutrition and has diversified its revenue through a $300 million acquisition of Melinta Therapeutics, adding seven approved therapies to its portfolio [7] - Despite initial success, CorMedix's future outlook is cautious, with 2026 revenue guidance of $300-$320 million and projected 2027 revenues of $100-$140 million, reflecting pricing pressures and limited growth [8][11] Mirum Pharmaceuticals (MIRM) Overview - Mirum's lead product, Livmarli, is approved for treating cholestatic pruritus in patients with Alagille syndrome and has seen a 70% year-over-year increase in net sales, reaching $161.4 million in the first nine months of 2025 [3][12] - The company has expanded its portfolio with a new tablet formulation of Livmarli and additional bile acid products from the acquisition of Travere Therapeutics, generating $118.8 million in the first nine months of 2025 [16] - MIRM's revenue guidance for 2026 is approximately $630-$650 million, supported by a broader product base and ongoing clinical studies for new therapies [16][28] Comparative Analysis - Both companies exhibit a similar risk-reward profile, but MIRM presents a clearer growth narrative with multiple revenue drivers, while CRMD's outlook is clouded by conservative guidance and reliance on a single product [4][9] - MIRM's shares have increased by 75.3% over the past six months, contrasting with CRMD's 38% decline, indicating market preference for MIRM's growth potential [23] - Valuation metrics show MIRM trading at a price/book ratio of 16.02, significantly higher than CRMD's 1.43, reflecting investor confidence in MIRM's growth prospects [24] Investment Recommendation - Mirum Pharmaceuticals is viewed as the better investment opportunity due to its strong sales growth, diversified revenue streams, and multiple growth levers, while CorMedix faces more uncertainty and competitive pressures [25][29]

Core Insights - Mirum Pharmaceuticals (MIRM) shares increased by 7.1% following the announcement of strong preliminary results for Q4 and full-year 2025, which boosted investor confidence [1][6] Financial Performance - Preliminary net product sales for Mirum in 2025 were approximately $520 million, exceeding the management's guidance of $500-$510 million provided in November 2025 [2][6] - Estimated net product sales for Livmarli were around $359 million for 2025, while Cholbam and Ctexli contributed approximately $161 million [4] - For Q4 2025, estimated net product sales were about $149 million, including $106 million from Livmarli and around $43 million from Cholbam and Ctexli [5] 2026 Sales Outlook - Mirum forecasts net product sales of approximately $630-$650 million for 2026 [6][7] - The company is conducting a phase III study for Livmarli aimed at treating pruritus in rare cholestatic conditions, with enrollment expected to complete in the first half of 2026 [7] Pipeline Developments - Mirum's lead pipeline candidate, volixibat, is under evaluation in two phase IIb studies for primary biliary cholangitis and primary sclerosing cholangitis, with top-line data from the VISTAS study anticipated in Q2 2026 [8] - A definitive agreement to acquire Bluejay Therapeutics was announced in December 2025, which will enhance Mirum's portfolio in rare liver diseases [9] - A phase II study has been initiated for MRM-3379, a PDE4D inhibitor targeting Fragile X syndrome [10]

PresentationGreat. Good morning, everyone. My name is Jess Fye. I'm a biotech analyst at JPMorgan, and we're continuing our 44th Annual Healthcare Conference this morning with Mirum. First, you're going to hear a presentation from the company and then we're going to go into a Q&A session. So for all of you in the room, if you do have a question, just raise your hand so someone can bring you a microphone for the webcast. And if you're listening online, you can submit questions to the portal, and I can read t ...

Summary of Mirum Pharmaceuticals FY Conference Call Company Overview - **Company**: Mirum Pharmaceuticals (NasdaqGM:MIRM) - **Focus**: Rare disease company delivering life-changing medicines to patients with rare diseases - **Key Products**: Livmarli, Volixibat, Chenodal, Cholbam, Brilovatug Financial Performance - **2025 Product Revenue**: Estimated at $520 million - **2026 Revenue Guidance**: Expected between $630 million and $650 million - **Potential Revenue from Pipeline**: Over $4 billion across current commercial and pipeline programs [3][25] Key Products and Pipeline Livmarli - **Indications**: Approved for Alagille syndrome and PFIC, with a third indication expected to read out later this year - **2025 Revenue**: $359 million, with strong growth driven by pediatric patient starts and label expansion into PFIC [8][30] - **Future Potential**: Estimated billion-plus potential opportunity [6][30] - **EXPAND Study**: Aiming for label expansion into other cholestatic pruritic indications, with top-line data expected in Q4 [10][12] Volixibat - **Indications**: Studied for PSC and PBC, targeting a significant unmet need in cholestatic pruritus [12][13] - **Market Size**: Approximately 30,000 patients with PSC, with two-thirds experiencing pruritus [13] - **Upcoming Data**: Top-line data from the VISTAS study expected next quarter, with NDA filing anticipated in the second half of the year [16][25] Brilovatug (Acquisition of BlueJ Therapeutics) - **Indication**: Targeting hepatitis delta, a rare co-infection with HBV - **Market Size**: Approximately 15,000 diagnosed and insured patients in the U.S., with potential for higher prevalence in other regions [22][48] - **Phase 2 Results**: Achieved 100% virologic response, with a 65%-82% response rate on FDA approval endpoints [23][47] - **Phase 3 Program**: AZURE studies currently enrolling, with top-line data expected in the second half of the year [24][25] Market Dynamics - **Livmarli**: Strong growth driven by increased awareness and diagnosis in adult PFIC settings, with a focus on genetic testing [9][28] - **Volixibat**: Positioned as the only potential therapy for PSC, with a significant market opportunity due to the lack of approved therapies [41][42] - **PBC Market**: More competitive with recent approvals of PPARs, but still presents opportunities for IBAT inhibitors [43] Commercial Strategy - **Geographic Focus**: Direct commercialization in North America and Western Europe, with partnerships in smaller countries [5] - **Field Expansion**: Plans to increase the U.S. field force by 30-40 people to target community physicians and GI specialists [44] Risks and Considerations - **Regulatory Risks**: Ongoing monitoring of clinical trial outcomes and regulatory approvals for pipeline products [2][25] - **Market Competition**: Need to navigate competitive landscapes, especially in the PBC market [42][43] Conclusion - Mirum Pharmaceuticals is positioned for significant growth with a strong pipeline and commercial strategy, focusing on rare diseases and addressing unmet medical needs in cholestatic conditions and hepatitis delta. The company anticipates pivotal data readouts in the coming year that could further enhance its market position and revenue potential [25][26].

Core Viewpoint - Mirum Pharmaceuticals is experiencing significant commercial growth and positive market response, reflected in its stock performance and future prospects [2][5]. Group 1: Company Overview - Mirum Pharmaceuticals is a biopharmaceutical company based in Foster City, California, focusing on therapies for rare liver diseases [1]. - The company has a market capitalization of approximately $4.42 billion, indicating its substantial presence in the biopharmaceutical industry [4]. Group 2: Stock Performance - The stock price of Mirum is currently $89.50, reflecting an increase of approximately 3.38% with a change of $2.93 [2]. - The stock has fluctuated between a low of $82 and a high of $90.07 today, with the latter being its highest price over the past year [3]. - The lowest price for MIRM in the past year was $36.88, showcasing substantial growth [3]. Group 3: Market Sentiment - Robert W. Baird has set a price target of $95 for NASDAQ:MIRM, indicating a potential upside of approximately 6.15% [1][5]. - The trading volume for MIRM is 1,229,452 shares, suggesting active investor interest and engagement [4].

Core Insights - Saira Ramasastry, a director at Mirum Pharmaceuticals, executed the exercise of 5,000 stock options and sold the resulting shares for a total transaction value of $400,000 on December 22 [2][7]. Transaction Summary - The transaction involved the direct sale of 5,000 shares, with a total value of $400,000, based on a weighted average purchase price of $80.00 per share [3]. - Post-transaction, Ramasastry holds no direct common shares, as the entire transaction accounted for 100% of her direct holdings [8]. Company Overview - Mirum Pharmaceuticals reported a revenue of $471.79 million and a net income of -$41.42 million for the trailing twelve months (TTM) [5]. - The company's stock price increased by 90.71% over the past year, calculated using December 22 as the reference date [5]. Company Snapshot - Mirum specializes in developing therapies for rare and orphan liver diseases, with its lead product being LIVMARLI and additional candidates like Volixibat in its pipeline [6]. Operational Performance - Preliminary results for 2025 indicate net product sales of approximately $520 million, exceeding guidance, with forecasts of $630 million to $650 million in global net product sales for 2026 [11]. - The company ended 2025 with an estimated $392 million in cash and investments and achieved positive operating cash flow, allowing for pipeline expansion and the proposed acquisition of Bluejay Therapeutics [11].

Financial Projections - Expected net product sales for 2026 are projected to be between $630 million and $650 million, with a compound annual growth rate (CAGR) of 91% from 2022 to 2025[3]. - The company expects FY 2026 net product sales guidance to be between $630 million and $650 million, with cash flow positive anticipated in 2027[39]. - The company has a cash balance of $392 million as of December 31, 2025, which is preliminary and subject to financial closing procedures[39]. Product Development and Pipeline - The peak revenue potential of Mirum's portfolio, including the proposed acquisition of Bluejay Therapeutics, is estimated to exceed $4 billion within the next 18 months[3]. - The ongoing EXPAND Phase 3 study for LIVMARLI® is expected to provide topline data by Q4 2026, targeting cholestatic liver disease patients[16]. - Volixibat, another product candidate, is undergoing a Phase 2b study for Primary Sclerosing Cholangitis (PSC) patients, with confirmatory topline data expected in Q2 2026[19]. - Four potentially registrational topline readouts are expected in the next 18 months, including VANTAGE (PBC) and VISTAS (PSC) results[39]. - The VANTAGE study for PBC patients with moderate-to-severe pruritus has completed enrollment and topline results are expected in H1 2026[25][39]. - Ongoing Phase 3 trials for Brelovitug are supporting FDA and EMA filings, with topline data expected in H2 2026[37]. - The Phase 2 study of MRM-3379 for Fragile X Syndrome is expected to provide topline data in 2027[33]. Market Expansion and Strategic Focus - The company aims to expand its international market presence and maintain intellectual property protection until at least 2040[9]. - The proposed acquisition of Bluejay Therapeutics is anticipated to close in January 2026, subject to customary conditions, which may enhance Mirum's operational capabilities[3]. - Mirum's strategic focus on rare diseases positions it as a leader in the global market, with significant commercial synergies anticipated from its bile acid portfolio[12]. - The company is actively pursuing market expansion and new product development strategies to enhance its portfolio and address unmet medical needs[45]. Product Performance and Safety - LIVMARLI® has shown significant year-over-year growth, with projected annual net product sales of $359 million for 2025, reflecting a 69% increase from 2024[8]. - LIVMARLI total net product sales for FY 2024 reached $336.4 million, with Q4 2024 sales projected at $99.4 million[45]. - LIVMARLI's US sales for Q1 2025 are estimated to be $49.5 million, increasing to $74 million by Q4 2025[45]. - 84% of patients treated with LIVMARLI experienced a clinically meaningful improvement in cholestatic pruritus, with 83% achieving a ≥20% reduction in serum bile acid (sBA) levels[48]. - In a post-hoc analysis, 93% of patients with a >1-point reduction in ItchRO[Obs] remained transplant-free six years after starting LIVMARLI[49]. - 62% of PFIC patients treated with LIVMARLI reported minimal to no itch after 26 weeks of treatment, compared to 28% in the placebo group[50]. - Significant reductions in serum bile acids were observed, with a mean decrease of 160 µmol/L in LIVMARLI patients compared to placebo[52]. - LIVMARLI has a well-characterized safety profile, with the most common adverse events being diarrhea and abdominal pain, occurring at rates of 41.6 and 38.6 events per 100 person-years, respectively[54]. Intellectual Property and Regulatory Designations - The company has intellectual property coverage for LIVMARLI in the United States extending to 2040, including multiple patents related to dosing and formulation[57]. - The company has multiple patents listed in the Orange Book, with key patents expiring between 2027 and 2043, indicating a strong pipeline for future treatments[60]. - The company has received orphan designation for its PBC indication, granting 10 years of market exclusivity from approval[60]. - The method of treatment for dosing is pending approval for several indications, showcasing ongoing innovation in the company's product offerings[60]. - The company has granted FDA Breakthrough Therapy Designation for Volixibat, which is expected to launch in H1 2026[25][39].

Core Insights - Enthorin Therapeutics, LLC is focused on circuit-modulating treatments for neurological and neurodevelopmental disorders [1] - The company announced that its licensing partner, Mirum Pharmaceuticals, initiated the BLOOM Phase 2 clinical study for MRM-3379 in Fragile X syndrome [1] - MRM-3379 is characterized as an orally available, highly brain-penetrant, selective phosphodiesterase inhibitor [1] Company Overview - Enthorin Therapeutics specializes in biotechnology with a focus on neurological and neurodevelopmental disorders [1] - Mirum Pharmaceuticals, Inc. is the licensing partner for MRM-3379 and is publicly traded on NASDAQ under the ticker MIRM [1] Clinical Development - The BLOOM Phase 2 clinical study aims to evaluate the efficacy of MRM-3379 in treating Fragile X syndrome [1] - MRM-3379 is noted for its ability to penetrate the brain effectively, which is crucial for its therapeutic application [1]