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Mirum Pharmaceuticals, Inc. (MIRM)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 total revenue was $99.4M, up 43% year over year; net product sales were $99.4M with LIVMARLI at $64.1M and bile acid medicines at $35.0M, reflecting strong commercial momentum .
  • Management issued 2025 guidance for global net product sales of $420–$435M and expects positive cash flow, citing robust demand and multiple upcoming clinical/regulatory milestones .
  • CTEXLI (chenodiol) received FDA approval for CTX, creating a new on-label growth avenue and planned conversion of existing patients with an emphasis on faster diagnosis to expand the treated population .
  • Pipeline execution remains on track: volixibat PSC (VISTAS) enrollment completion expected H2 2025 with topline in 2026; volixibat PBC (VANTAGE) enrollment completion expected in 2026; LIVMARLI EXPAND enrollment completion expected in 2026, sustaining medium-term catalysts .

What Went Well and What Went Wrong

What Went Well

  • Strong commercial performance: Q4 net product sales $99.4M (+43% YoY); full-year net product sales $336.4M; LIVMARLI 2024 net product sales $213.3M and bile acid medicines $123.1M .
  • Management affirmed 2025 guidance of $420–$435M and indicated positive cash flow, highlighting a disciplined financial posture and durable growth drivers; CEO: “we expect this momentum to continue” .
  • Pipeline progress and designation: volixibat achieved breakthrough therapy designation in PBC; interim data showed statistically significant itch improvement and broader symptom benefit (fatigue), maintaining timelines for PSC/PBC programs .

What Went Wrong

  • Operating expenses elevated: Q4 opex was $123.6M; full-year opex $424.5M with non-cash items of $79.4M, pressuring GAAP profitability despite commercial strength .
  • Continued GAAP losses: Q4 net loss $(23.8)M; prior quarters also negative (Q3 $(14.2)M), reflecting investment in R&D, SG&A, and intangible amortization .
  • International pricing headwinds: management cited European price reference impacts in Q2/Q3, which muted international revenue translation before normalizing later in the year .

Financial Results

MetricQ2 2024Q3 2024Q4 2024
Total Revenue ($USD Millions)$77.875 $90.377 $99.414
Net Income (Loss) ($USD Millions)$(24.638) $(14.235) $(23.790)
Loss from Operations ($USD Millions)$(24.232) $(12.684) $(24.222)
EBIT Margin % (Operating Income/Revenue)−31.1% −14.0% −24.4%
Net Loss Margin % (Net Loss/Revenue)−31.7% −15.8% −23.9%
Diluted EPS ($)$(0.52) $(0.30) n/a (not disclosed in Q4 table)

Segment breakdown (net product sales):

SegmentQ2 2024Q3 2024Q4 2024
LIVMARLI ($USD Millions)$47.2 $59.1 $64.1
Bile Acid Medicines ($USD Millions)$30.5 $31.2 $35.0
Total Net Product Sales ($USD Millions)$77.8 $90.3 $99.4

Key KPIs:

KPIQ2 2024Q3 2024Q4 2024
Cash, Cash Equivalents & Investments ($USD Millions)$295.4 $293.8 $292.8
Total Operating Expenses ($USD Millions)$102.107 $103.061 $123.636
Stock-based Compensation ($USD Millions)$11.835 $11.908 $13.251
Intangible Amortization ($USD Millions)$5.593 $5.894 $5.894

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Global Net Product Sales ($USD Millions)FY 2024$310–$320 $330–$335 Raised
Global Net Product Sales ($USD Millions)FY 2025n/a$420–$435 New
Cash FlowFY 2025n/aPositive cash flow expected New
Volixibat PSC (VISTAS) Enrollment CompletionH2 2025H2 2025 H2 2025 Maintained
Volixibat PBC (VANTAGE) Enrollment Completion20262026 2026 Maintained
LIVMARLI EXPAND Enrollment Completion20262026 2026 Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q-2 and Q-1)Current Period (Q4 2024)Trend
LIVMARLI PFIC adoption & reimbursementPFIC U.S. label expansion to ≥12 months; early PFIC scripts; faster-than-expected EU pricing underway; price reference impacts in Q2/Q3 Expanded access conversions completed; PFIC now a substantial contributor to new patient starts alongside steady ALGS Accelerating
International pricing dynamicsGermany negotiation outcome and price referencing headwinds expected to abate by Q4 No new headwind commentary; commercial momentum intact Normalizing
Volixibat PSC/PBC progressPositive interim results; breakthrough designation in PBC; interim itch improvements ~2.3 points vs placebo; enrollment ramping PSC enrollment completion H2 2025; topline 2026; PBC enrollment continuing to 2026 Steady execution
CTEXLI (chenodiol) CTXPDUFA set for Dec 28, 2024 FDA approval achieved; plan conversion of existing patients; focus on increasing diagnosis to expand market Launch tailwind
Fragile X (MRM-3379)In-licensed; Phase 2 initiation planned 2025 Market sizing: ~50k male patients; $1B TAM; Phase 2 in 2025; flexible capital allocation Advancing program
ALGS penetrationGeneral growth runway (implicit) U.S. ALGS pre-transplant treatment penetration ~40%, leaving significant runway Underpenetrated opportunity

Management Commentary

  • CEO: “Total net product sales were $336.4 million… we expect to add close to $100 million to our top line with anticipated net product sales between $420 million and $435 million for the year [2025]” .
  • CFO: “When adjusting for the non-cash items, the business was cash flow positive in 2024, and we expect this to continue this year” .
  • CMO: “VISTAS [PSC]… on track to complete enrollment in the second half of this year [2025]… expect top line results in 2026” .
  • CEO on ALGS market: “Eligible treatment population… probably in the range of 40% penetrated… long runway of continued growth” .

Q&A Highlights

  • Capital allocation and convertibles: flexibility to pursue attractive BD alternatives; convert maturity in ~4 years not a near-term constraint .
  • Fragile X (MRM-3379): U.S. male patient TAM ~50k; management frames ~$1B revenue potential; PDE4D mechanism with high brain penetration profile; Phase 2 planned 2025 .
  • ALGS penetration: ~40% U.S. penetration of the eligible pre-transplant pruritus population; growth drivers include further penetration, new diagnoses, and weight-based dose adjustments over time .
  • CTEXLI rollout: plan to convert existing chenodiol patients to CTEXLI and invest in earlier diagnosis to bend the curve of new patient adds .
  • PSC/PBC timelines: PSC topline roughly six months post-enrollment completion plus database close/clean; PBC interim no major OCA confounders, majority first-line patients in VANTAGE .

Estimates Context

  • S&P Global consensus EPS and revenue data for Q4 2024 and the next quarter were unavailable at the time of analysis due to a temporary data access limit; therefore, comparisons to Wall Street estimates are not provided. Values would ordinarily be sourced from S&P Global consensus; in this case, consensus data were unavailable.

Key Takeaways for Investors

  • Commercial traction remains robust: Q4 revenue $99.4M and net product sales $99.4M; LIVMARLI mix strengthening with PFIC contributing to new starts while ALGS remains the majority, supporting 2025 growth .
  • 2025 outlook is a catalyst: guidance raised to $420–$435M with expected positive cash flow; multiple pipeline milestones in 2025–2026 provide visibility to continued narrative strength .
  • New on-label opportunity: CTEXLI approval for CTX broadens portfolio; management plans patient conversion and diagnosis expansion to drive incremental sales over time .
  • Pipeline de-risks medium-term: volixibat PSC/PBC programs remain on schedule with prior interim signals and FDA breakthrough designation in PBC, underpinning optionality in adult cholestatic indications .
  • Operating leverage likely to improve: despite elevated opex and non-cash charges, adjusted cash flow was positive in 2024 and guided positive in 2025, suggesting improving cash generation as revenue scales .
  • ALGS still underpenetrated: ~40% penetration implies a multi-year runway in the core U.S. market, supplemented by international expansion and PFIC ramps .
  • Near-term trading implications: the combination of 2025 guidance, CTEXLI approval, and reaffirmed pipeline timelines should support sentiment; watch for incremental disclosure on international pricing normalization and PFIC uptake cadence in subsequent quarters .