Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) is a Foster City, California-headquartered commercial-stage rare-liver-disease biopharma focused on cholestatic and hepatic disorders — diseases where bile flow is impaired, leading to bile-acid accumulation and severe itching (pruritus), liver damage, and progression. The company was founded in 2018 by Chris Peetz (ex-Tobira Therapeutics, ex-Theravance, with long pharma-business-development background) and a team including key Lumena Pharmaceuticals/Shire-spin-off assets — specifically the IBAT-inhibitor program maralixibat (LIVMARLI) Mirum licensed from Shire (now Takeda) — IPO'd on NASDAQ 2020. Strategic focus on bile-acid biology and the IBAT (ileal bile acid transporter) mechanism: IBAT is the gut transporter recycling bile acids back to the liver — inhibiting it prevents recycling, lowering systemic bile-acid levels and reducing cholestatic itching (the dominant symptom in many cholestatic liver diseases) and potentially slowing disease progression. Commercial portfolio: LIVMARLI (maralixibat) the flagship oral once-daily IBAT inhibitor — FDA-approved September 2021 for cholestatic pruritus in Alagille syndrome (ALGS) ≥3 months (first approved drug for ALGS pruritus), expanded March 2024 to Progressive Familial Intrahepatic Cholestasis (PFIC) ≥12 months — both rare pediatric/adolescent cholestatic diseases — and CHOLBAM (cholic acid) — oral synthetic bile-acid replacement for ultra-rare bile-acid-synthesis disorders (acquired via 2020 Travere Therapeutics rights). Clinical pipeline: volixibat — another IBAT inhibitor (different molecule from maralixibat) — in Phase 3 for Primary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC) — both adult chronic cholestatic liver diseases with much larger patient populations than ALGS/PFIC (PBC ~50K+ US, PSC ~30K+) — potentially transformative; earlier-stage pipeline including additional IBAT-related and selected adjacencies. Geography US-led commercial with expanding ex-US (EU LIVMARLI approval 2022). Capital structure transitioning from R&D-loss-funded by equity to self-funding as LIVMARLI scales. MIRM enters FY2026 with FY2025 revenue selected various aggregate ~$310-380M (~30-40% growth), aggregate adjusted EPS ~$(0.40)-0.80, adjusted EBITDA ~$10-50M, under Chris Peetz. The first thesis pillar is the LIVMARLI commercial franchise — ~$240-290M+ annual revenue and growing strongly: LIVMARLI is an oral once-daily small-molecule minimally absorbed IBAT inhibitor — works locally in the gut to block IBAT, preventing bile-acid reabsorption back to the liver and reducing systemic bile-acid levels — mechanism alleviates cholestatic pruritus (severe itching), the dominant disabling symptom in cholestatic liver diseases; FDA approvals — September 2021 for ALGS (≥3 months, first approved drug for ALGS pruritus — historically these patients had no therapeutic option for severe itching; ALGS rare autosomal-dominant JAG1/NOTCH2 disorder causing bile-duct paucity and chronic cholestasis from infancy/childhood); March 2024 for PFIC (≥12 months — group of rare inherited disorders causing severe early-onset cholestasis — PFIC types 1/2/3 from different genetic mutations) — competing with Ipsen's BYLVAY (odevixibat from 2023 Albireo acquisition); EU approval 2022 for ALGS; commercial model rare-disease specialty pricing (~$150-250K+/patient-year), small dedicated salesforce targeting pediatric hepatologists/gastroenterologists/transplant specialists, patient-finder/diagnostic-support programs (ALGS genetic testing, PFIC genetic-and-clinical screening); FY2025 dynamics are LIVMARLI revenue ~$240-290M+ (~25-35% growth on PFIC expansion + ALGS continued + ex-US), competitive dynamics with BYLVAY in PFIC (smaller pool — both gaining share from no-treatment baseline), ~95%+ gross margin, operating leverage improving, ex-US progressing; FY2026 catalyst is continued prescription growth (US + ex-US), ALGS patient identification, PFIC market development, additional indications (Mirum exploring Biliary Atresia + other pediatric cholestatic), operating-margin expansion through breakeven; risks/competitors are Ipsen/BYLVAY pressure in PFIC, payer pricing pressure, pediatric-population constraints, emergence of disease-modifying therapies; comp set Ipsen (IPN.PA, BYLVAY), Travere Therapeutics (TVTX), broader rare-disease Insmed (INSM), Argenx (ARGX), Ultragenyx (RARE), BridgeBio (BBIO), Krystal Biotech (KRYS), Crinetics (CRNX). The second pillar bundles CHOLBAM with volixibat plus broader cholestatic-disease pipeline: CHOLBAM (cholic acid) for rare inherited bile-acid-synthesis disorders (BASDs) — patients lacking functional bile-acid synthesis enzymes needing exogenous replacement — ~$30-50M annual revenue, profitable, small patient population, acquired via 2020 Travere deal; volixibat — oral once-daily minimally absorbed IBAT inhibitor (similar mechanism to maralixibat but different molecule with different properties — acquired via 2019 Lumena Pharmaceuticals asset acquisition) — in two Phase 3 programs: VANTAGE Phase 3 in PBC (adult chronic autoimmune cholestatic liver disease, ~50K+ US, ~150K+ global — current SOC is UDCA + obeticholic acid Ocaliva from Intercept + recently-approved seladelpar Livdelzi GSK/CymaBay + elafibranor Iqirvo Ipsen/Genfit — pruritus remains key disabling symptom inadequately addressed by current treatments — clear unmet need for IBAT inhibitor — volixibat targets pruritus plus possibly underlying progression) and VISTAS Phase 3 in PSC (adult chronic cholestatic disease ~30K+ US, currently no FDA-approved treatments — pruritus the disabling symptom) — volixibat Phase 3 data expected 2026 for both; pipeline opportunity — PBC ~$1-3B+ pruritus TAM alone, PSC smaller but unaddressed — success transformatively expands Mirum's TAM from rare pediatric (ALGS/PFIC) to adult chronic liver disease; broader pipeline — additional IBAT-related programs, cholestatic adjacencies, selective BD; FY2025 dynamics are CHOLBAM stable, volixibat VANTAGE/VISTAS Phase 3 progressing toward 2026 readouts, broader pipeline advancing, R&D intense; FY2026 catalyst is volixibat VANTAGE PBC Phase 3 readout (major asymmetric upside binary — positive with clean safety = transformative) and VISTAS PSC Phase 3 readout (smaller TAM but asymmetric upside), pipeline data, BD activity; risks are volixibat Phase 3 failure (efficacy/safety — IBAT inhibitors well-characterized class profile but specific molecules vary), competitive PBC landscape (GSK seladelpar, Ipsen elafibranor, Intercept Ocaliva — all address underlying disease, leaving room for pruritus-specific addition), pricing dynamics; comp set in PBC/PSC GSK (GSK, Livdelzi via CymaBay), Ipsen (IPN.PA, Iqirvo via Genfit), Intercept (now Alfasigma, Ocaliva), Eiger Biopharmaceuticals (EIGR); broader specialty universe. The capital story: commercial-stage transitioning to profitability — no dividend (cash to commercial + R&D + pipeline + capital strength), no buybacks of consequence (growth-investment mode with improving CF), cash ~$200-300M+ (replenished via follow-ons), minimal debt (essentially net-cash, no meaningful term loans or senior notes), FCF inflecting positive as LIVMARLI scales over relatively fixed cost base — approaching/has achieved operating-profitability though still investing in volixibat Phase 3 + broader pipeline; net loss narrowed substantially; ~50-55M shares (small float, meaningful historical dilution but issuance pace slowing as profitability emerges); capital priorities LIVMARLI commercial growth (US + ex-US) → volixibat Phase 3 trials + pipeline R&D → earlier-stage programs → potential bolt-on M&A (active acquirer — Travere rare-disease commercial 2020, Vencerx 2021 for cholestatic adjacencies) → maintain cash runway; with path to GAAP profitability, cash-runway/financing trajectory, and volixibat Phase 3 outcomes driving multi-year capital-allocation framework. At ~$55-80 per share on ~50-55M shares (~$3.0-4.4B equity, ~$2.8-4.2B EV given net-cash) MIRM trades at roughly ~8-12x EV/Sales — premium specialty/rare-disease pharma multiple reflecting LIVMARLI growth + volixibat PBC/PSC pipeline optionality + path to profitability + IBAT-platform strategic strength — versus Ipsen (IPN.PA, BYLVAY competitor), Insmed (INSM), Argenx (ARGX), Ultragenyx (RARE), BridgeBio (BBIO), Krystal Biotech (KRYS), Crinetics (CRNX), Travere (TVTX), Vanda (VNDA), Apellis (APLS). FY2026 base case: ~$360-430M+ revenue + ~$0.20-1.00 adj. EPS + LIVMARLI continued growth + ex-US ramp + volixibat Phase 3 data flowing + pipeline progressing + cash position stable; bull case: ~$400-500M+ revenue + ~$0.80-1.60+ adj. EPS on stronger LIVMARLI (PFIC expansion + ex-US + Biliary Atresia label), volixibat VANTAGE PBC Phase 3 positive (transformative — ~$1-3B+ TAM + 2027-2028 PBC launch), VISTAS PSC positive (additional optionality), and substantial re-rating; bear case: ~$280-330M revenue + ~$(0.60)-0.20 adj. EPS on Ipsen/BYLVAY PFIC share, payer pricing pressure, volixibat Phase 3 failure (major asymmetric downside — eliminates adult expansion), pipeline setbacks, and compression. The thesis depends on the LIVMARLI commercial pipeline (US prescription + ex-US + ALGS/PFIC expansion + Biliary Atresia + competitive defense vs BYLVAY) plus the CHOLBAM + volixibat + cholestatic-disease pipeline (CHOLBAM stable + volixibat VANTAGE PBC + VISTAS PSC + broader pipeline) plus path to profitability plus net-cash balance sheet plus Chris Peetz's continued founding-CEO execution.

[MIRM] Mirum Pharmaceuticals Thesis 2026: LIVMARLI Scales Across Rare Liver Disease and a Cholestatic Pipeline

Key Takeaways

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) is expected to close FY2025 with selected various aggregate revenue of roughly $310-380M (~30-40% growth) and aggregate adjusted EPS in the area of $(0.40)-0.80 (transitioning from net loss to early profitability), with adjusted EBITDA selected various aggregate ~$10-50M, under President & CEO Chris Peetz (~5+ year tenure since founding-CEO appointment, ex-Tobira and Theravance).
The first deep-dive — the LIVMARLI (maralixibat) commercial franchise — covers Mirum's flagship oral once-daily IBAT inhibitor (ileal bile acid transporter inhibitor) for cholestatic pruritus in rare liver diseases: FDA-approved for Alagille syndrome (ALGS) in 2021 (the first approved drug for cholestatic itching in ALGS), expanded to Progressive Familial Intrahepatic Cholestasis (PFIC) in 2024, both with significant pediatric usage; FY2026 catalyst is continued LIVMARLI prescription growth (selected various aggregate ~$240-290M+ annual revenue), ex-US expansion, and potential additional indication expansion.
The second deep-dive — the CHOLBAM + volixibat + the broader cholestatic-liver-disease pipeline — covers CHOLBAM (cholic acid) for ultra-rare bile-acid-synthesis disorders (a small but profitable franchise — selected various aggregate ~$30-50M annual revenue), volixibat (another IBAT inhibitor) in Phase 3 for Primary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC) — much larger adult-population indications than ALGS/PFIC; FY2026 catalyst is volixibat Phase 3 readouts (the major asymmetric pipeline-value events — PBC and PSC), pipeline expansion, and partnership/licensing activity.
Capital position is on the path to self-funding: no dividend, no buybacks of consequence, selected various aggregate cash and equivalents in the area of $200-300M+, minimal debt of consequence, and ~50-55M shares outstanding (small float). The company has flipped from heavy R&D losses to approaching profitability as LIVMARLI revenue scales over a relatively fixed cost base.
FY2026 catalysts: LIVMARLI prescription growth (US + ex-US), volixibat Phase 3 readouts in PBC (the larger adult liver-disease indication — a potential transformative event opening a ~$1-3B+ TAM) and PSC, pipeline progression on broader cholestatic-disease indications, the path to GAAP profitability, partnership / business-development moves (Mirum has been actively acquiring pipeline assets), and continued cash-runway management as the company scales.

Company Background

Mirum Pharmaceuticals, Inc., headquartered in Foster City, California, is a commercial-stage rare-liver-disease biopharma focused on cholestatic and hepatic disorders — diseases where bile flow is impaired, leading to bile-acid accumulation and severe itching (pruritus), liver damage, and disease progression. The company was founded in 2018 by Chris Peetz (ex-Tobira Therapeutics, ex-Theravance, with a long pharma-business-development background) and a team that included key Lumena Pharmaceuticals/Shire-spin-off assets — specifically the IBAT-inhibitor program maralixibat (LIVMARLI) that Mirum licensed from Shire (now part of Takeda) — and IPO'd on NASDAQ in 2020. The strategic focus is on bile-acid biology and the IBAT (ileal bile acid transporter) mechanism: IBAT is the gut transporter that recycles bile acids back to the liver — inhibiting IBAT prevents this recycling, lowering systemic bile-acid levels and reducing cholestatic itching (the dominant symptom in many cholestatic liver diseases) and potentially slowing disease progression. The commercial portfolio: (1) LIVMARLI (maralixibat) — the flagship oral once-daily IBAT inhibitor — FDA-approved in September 2021 for cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 3 months and older (the first approved drug for ALGS pruritus), expanded in March 2024 to Progressive Familial Intrahepatic Cholestasis (PFIC) — both rare pediatric/adolescent cholestatic diseases — and (2) CHOLBAM (cholic acid) — an oral synthetic bile-acid replacement for ultra-rare bile-acid-synthesis disorders (acquired via the 2020 Travere Therapeutics rights deal) — a tiny patient population but high-priced and profitable. The clinical pipeline: (1) volixibat — another IBAT inhibitor (different molecule from maralixibat) — in Phase 3 trials for Primary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC) — both adult chronic cholestatic liver diseases with much larger patient populations than ALGS/PFIC (PBC ~50K+ US adults, PSC ~30K+) — potentially transformative for Mirum if successful; (2) earlier-stage pipeline including additional IBAT-related programs and selected adjacencies. Geography is US-led commercial with expanding ex-US operations (EU approval for LIVMARLI in 2022, building out launches across major European markets). The capital structure is transitioning from R&D-loss-funded by equity to self-funding as LIVMARLI revenue scales. Risks: LIVMARLI competitive pressure — Albireo Pharma's BYLVAY (odevixibat) was the first IBAT inhibitor approved for PFIC (and Albireo was acquired by Ipsen in 2023), competing directly with LIVMARLI in PFIC; volixibat Phase 3 PBC/PSC outcomes (a major binary); pricing/payer dynamics in rare disease; pipeline-development risk; cash-runway / financing trajectory.

The LIVMARLI Commercial Franchise

LIVMARLI is the commercial heart of Mirum — selected various aggregate ~$240-290M+ annual revenue and growing strongly. LIVMARLI (maralixibat): an oral once-daily small-molecule minimally absorbed IBAT inhibitor — works locally in the gut to block IBAT (the ileal bile acid transporter), preventing bile-acid reabsorption back to the liver and reducing systemic bile-acid levels; the mechanism alleviates cholestatic pruritus (severe itching), the dominant disabling symptom in many cholestatic liver diseases. FDA approvals: September 2021 — Alagille syndrome (ALGS) in patients ≥3 months (the first approved drug for cholestatic pruritus in ALGS — historically these patients had no therapeutic option for severe itching) — Alagille syndrome is a rare autosomal-dominant disorder (JAG1 or NOTCH2 mutation) causing bile-duct paucity and chronic cholestasis from infancy/childhood; March 2024 — Progressive Familial Intrahepatic Cholestasis (PFIC) in patients ≥12 months (a group of rare inherited disorders causing severe early-onset cholestasis — PFIC types 1, 2, 3 driven by different genetic mutations) — competing directly with Ipsen's BYLVAY (odevixibat — the IBAT inhibitor from the Albireo acquisition) in PFIC. EU approval: LIVMARLI approved for ALGS in 2022 (Mirum building EU commercial operations + selected ex-EU launches). The commercial model: rare-disease specialty pricing (selected various aggregate ~$150-250K+/patient-year), small dedicated commercial salesforce targeting pediatric hepatologists, gastroenterologists, transplant specialists; patient-finder/diagnostic-support programs Mirum funds to help identify previously-undiagnosed patients (Alagille genetic testing, PFIC genetic-testing-and-clinical-screening). FY2025 dynamics: LIVMARLI revenue ~$240-290M+ (growing ~25-35% on PFIC expansion + ALGS continued patient adds + ex-US ramp), competitive dynamics with BYLVAY in PFIC (PFIC is a smaller patient pool — Albireo/Ipsen and Mirum both gaining share from no-treatment baseline rather than competing intensely on share), gross margin extremely high (~95%+), operating leverage improving as commercial costs scale slower than revenue, ex-US progressing. FY2026 catalyst: continued prescription growth (US + ex-US), ALGS patient identification continuing, PFIC market development, expansion into additional indications (Mirum is exploring LIVMARLI in Biliary Atresia and other pediatric cholestatic conditions), and operating-margin expansion as the company moves through breakeven. Risks/competitors: Ipsen/BYLVAY (odevixibat) competitive pressure in PFIC, payer pricing pressure in rare disease (the high-price specialty model faces ongoing scrutiny), pediatric-population-size constraints (small patient pools), and the next-decade emergence of disease-modifying therapies that could change the standard of care. Comp set: Ipsen (IPN.PA, BYLVAY franchise from 2023 Albireo acquisition), Travere Therapeutics (TVTX, focused on rare nephrology including cholestasis), GlobeImmune, Madrigal (MDGL, MASH/NASH adjacent), broader specialty/rare-disease pharma — Insmed (INSM), Argenx (ARGX), Ultragenyx (RARE), BridgeBio (BBIO), Krystal Biotech (KRYS), Crinetics (CRNX).

CHOLBAM + Volixibat + the Broader Cholestatic-Liver-Disease Pipeline

The second deep-dive bundles CHOLBAM (the small but profitable established commercial franchise) with volixibat (the major near-term pipeline binary) and the broader cholestatic pipeline. CHOLBAM (cholic acid): an oral synthetic bile-acid replacement for rare inherited bile-acid-synthesis disorders (BASDs) — patients who lack functional bile-acid synthesis enzymes and need exogenous bile-acid replacement to support fat absorption and prevent liver damage — selected various aggregate ~$30-50M annual revenue, profitable, small patient population, established commercial; Mirum acquired CHOLBAM rights via the 2020 Travere Therapeutics deal. Volixibat: an oral once-daily minimally absorbed IBAT inhibitor (similar mechanism to maralixibat but a different molecule with different properties — Mirum acquired volixibat via the 2019 Lumena Pharmaceuticals asset acquisition); volixibat is in two Phase 3 programs: (1) VANTAGE Phase 3 in Primary Biliary Cholangitis (PBC) — adult chronic autoimmune cholestatic liver disease with ~50K+ US patients, ~150K+ globally — current standard of care is ursodeoxycholic acid (UDCA) + obeticholic acid (Ocaliva, Intercept) + the recently-approved seladelpar (Livdelzi, GSK/CymaBay) and elafibranor (Iqirvo, Ipsen/Genfit) — pruritus remains a key disabling symptom that current treatments don't adequately address, providing a clear unmet need for an IBAT inhibitor; volixibat targets the pruritus symptom plus possibly the underlying disease progression; (2) VISTAS Phase 3 in Primary Sclerosing Cholangitis (PSC) — another adult chronic cholestatic liver disease ~30K+ US patients — currently no FDA-approved treatments — pruritus is again the disabling symptom; volixibat Phase 3 data expected in 2026 for both indications. The pipeline opportunity: PBC has a ~$1-3B+ addressable market in pruritus alone; PSC is smaller but unaddressed; volixibat success would transformatively expand Mirum's TAM from rare pediatric (ALGS/PFIC) to adult chronic liver disease. Broader pipeline: additional IBAT-related programs, exploration of cholestatic adjacencies, and selective business-development activity. FY2025 dynamics: CHOLBAM stable, volixibat VANTAGE/VISTAS Phase 3 trials progressing toward 2026 readouts, broader pipeline advancing, R&D-investment intense. FY2026 catalyst: volixibat VANTAGE PBC Phase 3 readout (the major asymmetric upside binary — a positive result with clean safety would be transformative) and VISTAS PSC Phase 3 readout (smaller TAM but additional asymmetric upside), pipeline-program data, and partnership/licensing activity. Risks: volixibat Phase 3 failure (efficacy or safety/tolerability — IBAT inhibitors have a relatively well-characterized class profile but specific molecules can vary), competitive PBC landscape (GSK's seladelpar, Ipsen's elafibranor, Intercept's Ocaliva — though all address the underlying disease, leaving room for a pruritus-specific addition), pediatric/adult-population-pricing dynamics. Comp set in PBC/PSC: GSK (GSK, Livdelzi via CymaBay), Ipsen (IPN.PA, Iqirvo/elafibranor via Genfit), Intercept Pharmaceuticals (private/Alfasigma — Ocaliva), Inflo Pharma, Eiger Biopharmaceuticals (EIGR); broader specialty pharma comp universe.

Capital Position + Balance Sheet

Mirum runs a commercial-stage-transitioning-to-profitability balance sheet. The company pays no dividend (and not near-term — cash to commercial + R&D + pipeline + capital strength), conducts no buybacks of consequence (the company is still in growth-investment mode, with cash flow improving). Cash and short-term investments are selected various aggregate ~$200-300M+ (replenished periodically as needed via follow-on offerings — Mirum has done several over its public history). Debt is minimal — Mirum has historically operated debt-light, with no meaningful term loans or senior notes. Free-cash-flow is inflecting positive as LIVMARLI revenue scales over a relatively fixed cost base — the company is approaching/has achieved operating-profitability and net-positive cash flow, though still investing in volixibat Phase 3 trials and broader pipeline; net loss has narrowed substantially from peak. Shares outstanding are selected various aggregate ~50-55M (small float — meaningful historical dilution but issuance pace slowing as profitability emerges). Capital priorities: (1) fund LIVMARLI commercial growth (US + ex-US expansion), (2) fund volixibat Phase 3 trials and pipeline R&D, (3) fund earlier-stage pipeline programs, (4) potential bolt-on M&A (Mirum has been active — acquired the Travere rare-disease commercial assets in 2020, acquired Vencerx in 2021 for cholestatic adjacencies, etc.), (5) maintain cash runway. There is no debt to refinance, no leverage to manage; the principal balance-sheet considerations are the path to GAAP profitability, cash-runway/financing trajectory, and the volixibat Phase 3 outcomes that drive the multi-year capital-allocation framework.

Key Core Metrics

Revenue: selected various aggregate ~$310-380M FY2025 (~30-40% growth)
Adjusted EBITDA: selected various aggregate ~$10-50M FY2025 (transitioning from loss to small positive)
Adjusted EPS: selected various aggregate ~$(0.40)-0.80 FY2025 (transitioning toward profitability)
Gross margin: ~95%+ (high-priced specialty / rare-disease drugs)
LIVMARLI (maralixibat) revenue: ~$240-290M+ annual (~25-35% growth)
LIVMARLI indications: Alagille syndrome (ALGS) FDA-approved Sept 2021 (≥3 months); PFIC FDA-approved March 2024 (≥12 months); EU approved 2022
LIVMARLI mechanism: oral once-daily minimally absorbed IBAT (ileal bile acid transporter) inhibitor
LIVMARLI pricing: ~$150-250K+/patient-year (rare-disease specialty)
LIVMARLI competitor: Ipsen's BYLVAY (odevixibat) in PFIC (from 2023 Albireo acquisition)
CHOLBAM (cholic acid) revenue: ~$30-50M annual (rare bile-acid synthesis disorders)
Volixibat: oral once-daily minimally absorbed IBAT inhibitor (different molecule from maralixibat)
Volixibat VANTAGE Phase 3 (PBC): expected 2026 readout (major binary — ~50K+ US patients, ~$1-3B+ TAM opportunity)
Volixibat VISTAS Phase 3 (PSC): expected 2026 readout (smaller TAM but unmet need — no approved PSC treatments)
Pipeline: LIVMARLI in Biliary Atresia (Phase 3) and other pediatric cholestatic indications; additional IBAT-related programs
Geography: US-led commercial + EU launches in major markets + selected ex-EU
Cash + short-term investments: selected various aggregate ~$200-300M+ FY2025
Net debt: minimal (essentially net-cash; no meaningful debt)
Credit profile: implicitly equivalent to investment-grade (unrated)
Dividend: none; cash to commercial + R&D + pipeline + capital strength
Buybacks: none of consequence; growth-investment mode
Shares outstanding: selected various aggregate ~50-55M (small float)
Capex: minimal (no manufacturing — contracted out); strong FCF conversion as commercial scales
Capital allocation: LIVMARLI commercial growth → volixibat Phase 3 → pipeline R&D → bolt-on M&A → cash runway
CEO: Chris Peetz (President & CEO, ~5+ year tenure as founding CEO; ex-Tobira and Theravance)

Market Evaluation

At roughly ~$55-80 per share on ~50-55M shares, Mirum Pharmaceuticals carries an equity value of selected various aggregate ~$3.0-4.4B (and an enterprise value of selected various aggregate ~$2.8-4.2B given the net-cash position), which on FY2025 cash flow is roughly ~8-12x EV/Sales — a premium specialty/rare-disease pharma multiple reflecting (a) the LIVMARLI franchise growth trajectory, (b) the volixibat PBC/PSC pipeline optionality, (c) the path to profitability, and (d) the strategic strength of an IBAT-platform franchise; the comp set is rare-disease specialty pharma — Ipsen (IPN.PA, BYLVAY competitor), Insmed (INSM), Argenx (ARGX), Ultragenyx (RARE), BridgeBio (BBIO), Krystal Biotech (KRYS), Crinetics (CRNX), Travere Therapeutics (TVTX), Vanda (VNDA), Apellis (APLS). FY2026 base case: selected various aggregate ~$360-430M+ revenue + ~$0.20-1.00 adj. EPS + LIVMARLI continued growth + ex-US ramp + volixibat Phase 3 data flowing + pipeline progressing + cash position stable — a scaling/profitability-inflection year. Bull case: selected various aggregate ~$400-500M+ revenue + ~$0.80-1.60+ adj. EPS on stronger LIVMARLI growth (PFIC expansion + ex-US + Biliary Atresia label expansion), volixibat VANTAGE PBC Phase 3 positive (transformative — opens a ~$1-3B+ TAM and a likely 2027-2028 PBC launch), VISTAS PSC Phase 3 positive (additional optionality), and a substantial multiple re-rating as Mirum becomes a much larger specialty pharma. Bear case: selected various aggregate ~$280-330M revenue + ~$(0.60)-0.20 adj. EPS on Ipsen/BYLVAY taking PFIC share, payer pricing pressure on LIVMARLI, volixibat Phase 3 failure (the major asymmetric downside — eliminates the adult-population expansion), pipeline setbacks, and a multiple compression. The thesis turns on the LIVMARLI commercial pipeline (US prescription growth + ex-US ramp + ALGS/PFIC expansion + Biliary Atresia label expansion + competitive defense vs BYLVAY) plus the CHOLBAM + volixibat + cholestatic-disease pipeline (CHOLBAM stable + volixibat VANTAGE PBC and VISTAS PSC Phase 3 readouts + broader pipeline) plus the path to profitability plus the net-cash balance sheet plus Chris Peetz's continued execution of the founding-CEO playbook of building a rare-liver-disease specialty pharma platform.