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    MIRM
    Earnings call· Jun 2026(Q2 FY26)

    Mirum Pharmaceuticals Q2 FY26 earnings call MIRM

    Aug 5, 2026 Source

    Executive summary

    Mirum Pharmaceuticals Q2 FY26 — Strong Commercial Performance and Pipeline Progress Amidst Regulatory Delay for Volixibat in PSC

    Mirum Pharmaceuticals delivered a strong Q2 FY26, driven by robust commercial execution and increased full-year sales guidance. The company advanced its pipeline with key milestones, including Breakthrough Therapy Designation for volixibat in PSC and completed enrollment for the VANTAGE study in PBC. However, regulatory discussions for volixibat in PSC will delay its NDA submission, requiring further iterative engagement with the FDA.

    Highlights

    5

    • Net product sales reached $176 million in Q2 FY26, reflecting strong demand across the portfolio.

    • Full-year 2026 net product sales guidance increased to $680 million to $700 million, up from previous guidance.

    • Volixibat received Breakthrough Therapy Designation for cholestatic pruritus due to PSC, recognizing its potential.

    • VANTAGE study for volixibat in PBC completed enrollment with over 330 patients, with FDA feedback confirming its pivotal study potential.

    • Cash, cash equivalents, and investments increased to $561 million as of June 30, up from $391 million at the beginning of the year, strengthened by a $690 million convertible notes issuance.

    Concerns

    1

    • FDA recommended conducting a Phase 3 study for volixibat in PSC, delaying the NDA submission to H1 2027 from the previously planned timeline.

    Guidance & targets

    11
    CategoryTargetConfidence
    Full-year 2026 net product sales
    $680 million to $700 million
    high materiality
    High
    Volixibat NDA submission for cholestatic pruritus due to PSC
    First half of next year (2027)
    high materiality
    Medium
    Zilurgisertib PDUFA date for FOP
    Next month (September 2026)
    medium materiality
    High
    Zilurgisertib U.S. launch for FOP
    Fourth quarter (Q4 2026)
    medium materiality
    High
    Brelovitug AZURE-1 top line results
    Later this quarter (Q3 2026)
    medium materiality
    High
    Brelovitug AZURE-4 year four data
    Fourth quarter (Q4 2026)
    medium materiality
    High
    Brelovitug BLA submission
    First half of next year (2027)
    high materiality
    High
    LIVMARLI EXPAND study top line data
    Fourth quarter (Q4 2026)
    medium materiality
    High
    Volixibat VANTAGE study top line results (PBC)
    First quarter next year (Q1 2027)
    medium materiality
    High
    MRM-3379 proof-of-concept data (Fragile X syndrome)
    Next year (2027)
    low materiality
    High
    LIVMARLI sNDA submission (additional rare cholestatic diseases)
    Next year (2027)
    medium materiality
    High

    Segment performance

    4
    SegmentRevenueYoYQoQMargin
    LIVMARLI (Total)
    Strong performance driven by continued new patient starts, sustained persistence, and weight-based dose increases in Alagille, and growing contribution from new diagnoses in PFIC, particularly adult patients.
    Alagille growth: durablePFIC contribution: growingAdult PFIC population (US): at least 2,000 patientsAdult PFIC diagnosis: meaningful opportunity to expand through education
    		$129 million———
    LIVMARLI (U.S.)
    U.S. sales contributing significantly to overall LIVMARLI performance.
    		$92 million———
    LIVMARLI (International)
    Growth across direct and partner markets, with contributions from established markets and expanding reimbursement.
    		$37 million———
    Bile Acid Medicines
    Steady contribution to the rare genetic disease business.
    		$48 million———

    Operational metrics

    16
    Net product sales
    $176 millionvs $128 million in Q2 2025
    Q2 2026

    Total net product sales for the quarter.

    Cash, cash equivalents and investments balance
    $561 millionvs $391 million at beginning of year
    as of June 30, 2026

    Strengthened by convertible notes issuance.

    Cash contribution margin from commercial business
    high 50s percentapproximately a 5 percentage point improvement over prior year
    Q2 2026 and H1 2026

    Reflects scaling of commercial business.

    Total operating expense
    $219 million
    Q2 2026

    Excludes stock-based compensation and intangible amortization.

    In-process R&D expense
    $16 million
    Q2 2026

    Part of total operating expense.

    R&D expense
    $76 million
    Q2 2026

    Excludes stock-based compensation and intangible amortization.

    SG&A expense
    $66 million
    Q2 2026

    Excludes stock-based compensation and intangible amortization.

    Cost of sales
    $23 million
    Q2 2026

    Excludes stock-based compensation and intangible amortization.

    Stock-based compensation, intangible amortization and other noncash expenses
    $37 million
    Q2 2026

    Total non-cash expenses for the quarter.

    Addressable adult PFIC population
    at least 2,000 patients
    current

    Likely similar number in Europe. Majority don't yet have a PFIC diagnosis due to less established genetic testing in adult practices.

    Addressable adult PFIC population
    similar number
    current

    Likely similar number to the US estimate of at least 2,000 patients.

    VANTAGE study patients randomized
    over 330 patients
    completed enrollment

    Study for volixibat in PBC.

    VISTAS study patients randomized
    more than 180 PSC patients
    completed

    Largest randomized clinical study conducted in patients with pruritus due to PSC.

    VISTAS study safety exposure
    over 100 PSC patients
    one year

    One year safety exposure data.

    Total subjects across volixibat clinical program
    over 600 subjects
    to date

    Includes growing results from an independent committee evaluating liver safety.

    Diagnosed and managed FOP patients
    about 300 patients
    current

    Patients are highly specialized centers.

    Industry KPIs

    4
    MetricValueDetails
    Pipeline read out calendarMultiple pivotal readouts
    Regulatory approvals filingsBreakthrough Therapy Designation for volixibat in PSC
    Peak long term sales guidanceOver $1 billionUSD
    Clinical trial efficacy safety dataHighly significant improvements in pruritus

    Deals & partnerships

    1
    Not statedAcquisition of zilurgisertib program

    Program added to rare genetic business in Q2 2026, leading to potential launch later this year.

    Risks & headwinds

    2
    Regulatory delay for volixibat in PSCNear-term (next 6-12 months)

    NDA submission delayed from previously planned timing to H1 2027

    Mitigation: Iterative discussions with FDA to build familiarity with VISTAS data and history, leveraging Breakthrough Therapy Designation for more frequent interactions. Management believes VISTAS data is robust and sufficient.

    Lack of continuity in FDA review team for volixibat in PSCOngoing

    New FDA team encountered during pre-NDA meeting, potentially unfamiliar with prior alignment on VISTAS study design as pivotal.

    Mitigation: Engaging in iterative discussions to educate the new team on the VISTAS study's backdrop, design, and significance, especially given the Breakthrough Therapy Designation.

    What to watch in Q3 FY26

    5

    Volixibat PSC regulatory path

    Next quarter / H1 2027
    CurrentFDA recommended Phase 3, NDA submission delayed to H1 2027
    TargetClear path forward for NDA submission without additional Phase 3

    Why it matters

    Resolution of this regulatory hurdle is critical for the commercialization timeline and investment thesis for volixibat in PSC.

    This engagement will delay the planned timing of📎 our NDA submission, which we are now targeting for the first half of next year. We're positioned to move quickly once we have further clarity from the agency.

    Q&A highlights

    5

    What key topics will be addressed in future discussions with the FDA regarding the PSC NDA?

    Management explained that the FDA team encountered during the pre-NDA meeting was new, and the discussions will focus on familiarizing them with the VISTAS study's design, its successful results, and the history of prior FDA alignment on its pivotal intent. The Breakthrough Therapy Designation, granted after the meeting, provides a good opportunity for further engagement.

    “in the meeting, there was a new team from FDA. And so we think there's a lot of work to kind of get them up to speed on the backdrop here. So the history designing and conducting VISTAS and what this study means in a PSC setting.”

    asked by Ryan Deschner · answered by Christopher Peetz

    2 min read6 chapters

    Detailed Narrative

    01

    Commercial Performance and Outlook

    Mirum reported strong Q2 FY26 net product sales of $176 million, driven by LIVMARLI and bile acid medicines. LIVMARLI sales were $129 million, with $92 million from the U.S., showing durable growth in Alagille and increasing contribution from adult PFIC patients. The company raised its full-year 2026 net product sales guidance to $680 million to $700 million, excluding any potential contribution from zilurgisertib in Q4.

    02

    Volixibat Regulatory Update for PSC

    The FDA granted Breakthrough Therapy Designation for volixibat in cholestatic pruritus due to PSC based on strong VISTAS study results. However, during a pre-NDA meeting, the FDA recommended conducting a Phase 3 study, delaying the NDA submission to H1 2027. Management believes the VISTAS study is robust and plans iterative discussions with the agency to address concerns and supplement the planned submission.

    03

    Volixibat VANTAGE Study Progress for PBC

    The VANTAGE study for volixibat in pruritus due to PBC has completed enrollment with over 330 randomized patients. Following earlier Breakthrough Therapy Designation, the FDA has provided feedback confirming VANTAGE's potential to serve as a pivotal study if successful, with top-line results expected in Q1 2027. This provides a clearer path for PBC compared to PSC.

    04

    Zilurgisertib Launch Readiness

    Mirum is preparing for the potential U.S. launch of zilurgisertib for FOP in Q4 2026, following a September PDUFA date. The launch will target patients aged 12 and older, leveraging the existing rare genetics sales team that markets bile acid medicines, as FOP treaters are concentrated in similar specialized centers. A marketing application has also been submitted in Europe.

    05

    Pipeline Milestones

    Beyond volixibat, Mirum expects several key clinical readouts. Brelovitug AZURE-1 top-line results are anticipated later this quarter, with year four data from AZURE-4 expected in Q4 2026, supporting a BLA submission in H1 2027. The Phase 3 EXPAND study of LIVMARLI in additional rare cholestatic conditions is on track for top-line data in Q4 2026, potentially leading to an sNDA in 2027. MRM-3379 proof-of-concept data in Fragile X syndrome is expected in 2027.

    06

    Financial Strength

    The company reported a strong cash position of $561 million as of June 30, 2026, up from $391 million at the start of the year. This was significantly bolstered by the issuance of $690 million in 0% coupon convertible notes due June 2032, which also allowed for the settlement of 75% of outstanding 2029 notes and reduced interest expense. Operating cash flow was positive in Q2 despite increased R&D.

    AI-generated summary of the company’s earnings call. Not investment advice.