Mirum(MIRM) - 2025 Q4 - Annual Results

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].