{
  "@context": "https://orbyd.app/schemas/dossier.v1.json",
  "ticker": "MIRM",
  "name": "Mirum Pharmaceuticals, Inc.",
  "url": "https://orbyd.app/dossiers/MIRM/",
  "json_url": "https://orbyd.app/dossiers/MIRM.json",
  "status": "DORMANT",
  "current_conviction": "MEDIUM",
  "graded_conviction": "MEDIUM",
  "archetype": {
    "code": "a5",
    "n": 5
  },
  "current_thesis": "Rare-disease franchise ran to a $125 ATH on FOP data, then cooled ~9% into a confirmed 2026-07-30 Q2 print. Base compounds (Q1 sales +43% YoY, FY guide $660–680M) with FOP PDUFA 2026-09-26 and HDV Phase 3 topline 2H26 stacked ahead — but sell-side has fully caught up, so the print is the near-term binary and the theme is now MATURING.",
  "invalidation_trigger": "A weekly close below $100 breaks the post-June breakout structure and voids the momentum leg. Secondary: a 2026-07-30 Q2 print that cuts the $660–680M FY guide, a zilurgisertib PDUFA setback near 2026-09-26, or an AZURE-1 Phase 3 topline miss in 2H26.",
  "catalyst_date": null,
  "outcome": "INVALIDATED",
  "outcome_date": "2026-08-06",
  "invalidation_fired": true,
  "themes": [
    "rare-disease-gene-therapy",
    "precision-biotech-therapeutics"
  ],
  "tags": [],
  "sources": [],
  "notes": [
    "Insider Form 4s ~2026-07-08: CEO sold 20k post-option-exercise, CFO 5k under 10b5-1 — routine, not a conviction-buy signal.",
    "Q2 2026 earnings confirmed for 2026-07-30 (~3 trading days out) — hard binary blocker for fresh entries; avoid chasing into the print, prefer post-print confirmation or a hold of the $100–105 base.",
    "Zilurgisertib is licensed from Incyte (INCY); FOP economics are shared — watch INCY around the 2026-09-26 PDUFA as a same-direction tell.",
    "Not gene therapy: MIRM is small-molecule IBAT/ALK2 (zilurgisertib) plus a monoclonal (brelovitug). Correct any auto-theme tagging it as rare-disease-gene-therapy.",
    "Stacked 2H26 binaries: FOP PDUFA 2026-09-26, brelovitug AZURE-1/AZURE-4 HDV topline 2H26, volixibat NDA H2 26 — size for event risk, not trend alone.",
    "Sell-side has fully caught up (six PT raises 2026-06-11 to 2026-07-07); theme is MATURING, not accelerating — favor pullback-to-base entries over breakout chases.",
    "Early-July insider Form 4s (CEO ~20k, CFO ~5k under 10b5-1) and 2026-07-13 inducement grants are routine, not conviction buys.",
    "Price refresh: ATH $125.41 on 2026-07-02, pulled back to $114.48 on 2026-07-22; 52-week range $50.25–$130.00; market cap ~$5.7B."
  ],
  "body_markdown": "## Current Thesis\nA commercial-stage rare-disease franchise that ran to an all-time high on best-in-class FOP data, then cooled roughly 9% into a confirmed Q2 print. The base business is compounding — Q1 2026 net product sales of $159.9M (+43% YoY), FY guide lifted to $660–680M — while three discrete regulatory binaries stack into 2H26: a zilurgisertib FOP PDUFA on 2026-09-26, brelovitug HDV Phase 3 topline, and a volixibat NDA. The wrinkle since the stock's early-July peak: sell-side has now caught up (six PT raises already banked), so the frictionless re-rating leg is largely behind it. The immediate gate is the 2026-07-30 earnings print, ahead of the September FOP decision. Theme state is MATURING, not accelerating.\n\n## Bull Case\n- **Guidance raised, not just met.** On the 2026-05-06 Q1 print, net product sales were $159.9M (+43% YoY vs $111.6M), Livmarli $113.8M and bile-acid medicines $46.1M; 2026 net-sales guide moved up to $660–680M against FY2025 revenue of ~$521M.\n- **Zilurgisertib FOP data is class-leading.** PROGRESS Phase 2 results (ENDO 2026, 2026-06-14) showed a 99.9% reduction in new heterotopic-ossification lesion volume, durable through Week 48. FDA granted Priority Review with a PDUFA of 2026-09-26; the NDA covers FOP patients aged 12 and older. FOP is ultra-rare (~300 U.S. patients) — orphan pricing, minimal competition.\n- **Brelovitug HDV is a de-risking second pillar.** AZURE-1 Phase 3 enrollment and AZURE-4 screening are complete (announced July 2026), confirming 24-week topline data timing in 2H26. Phase 2b AZURE-1 hit its primary endpoint on 2026-04-27 (combined virologic response + ALT normalization at Week 24). Breakthrough Therapy designation supports the U.S. BLA these two studies underpin.\n- **Volixibat optionality.** U.S. NDA targeted for H2 2026 in primary sclerosing cholangitis, extending the IBAT franchise beyond Livmarli.\n- **Sell-side confirmed the move.** Six PT raises 2026-06-11 → 2026-07-07 (MS $150, RBC $148, Citizens $146, JPM $125, HC Wainwright $185) landed as price printed new highs.\n\n## Bear Case\n- **The whole 2H26 is binary-stacked.** PDUFA 2026-09-26, AZURE-1/AZURE-4 topline, and the volixibat NDA cluster into one window; any single disappointment re-rates a name up ~130% off its 52-week low of $50.25.\n- **Momentum is cooling into the print.** Price fell from the 2026-07-02 all-time high of $125.41 to $114.48 on 2026-07-22 (–2.2% that session); the early-July breakout has stalled rather than extended.\n- **The easy re-rating is spent.** Six PT raises already banked means the narrative is public — further upside now needs fresh data, not analysts catching up.\n- **Acquisition accounting noise.** The Bluejay deal drove a $726.3M in-process R&D charge and a Q1 net loss of $790.2M, or $(13.43)/share.\n- **Insider stance is neutral-to-soft.** Early-July Form 4s show routine 10b5-1 sells (CEO ~20k, CFO ~5k) and 2026-07-13 inducement grants at a $122.13 strike — no insider buying to flag fresh conviction.\n- **FOP economics are shared with Incyte,** so the headline approval's P&L contribution is split.\n\n## Setup & Price Structure\nPrice sits near $114–115 (2026-07-22), roughly 9% below the $125.41 all-time high (2026-07-02), inside a 52-week range of $50.25–$130.00; market cap is ~$5.7B. The breakout to new highs has stopped extending and the tape is digesting into earnings. The June breakout shelf near $100–105 is the structure that matters — holding it on a weekly basis keeps the uptrend alive; losing it voids the momentum leg. With the print 2026-07-30 and roughly three trading days out, buying ahead of it is a guidance coin-flip, not a setup. Cleaner entries are post-print confirmation of the $660–680M FY guide or a controlled pullback that holds the breakout base. Because the sell-side upgrade cycle that powers a first leg has largely fired, the correct stance into the print is to stand aside and let the binary resolve rather than chase a stalling tape.\n\n## Catalyst Calendar (next 30 days)\n\n- *(Beyond 30 days, flagged for sizing):* 2026-09-26 zilurgisertib FOP PDUFA; 2H26 brelovitug AZURE-1/AZURE-4 24-week topline; H2 2026 volixibat NDA submission.\n\n## Elapsed catalysts\n\n- **2026-07-30 — Q2 2026 earnings (confirmed).** The read on Livmarli/PFIC demand and whether the $660–680M FY guide holds or is raised. Binary; ~3 trading days out — the dominant near-term event. *(passed 10d ago)*\n\n## What Would Change Our Mind\n- A weekly close below $100 breaks the post-June breakout structure and voids the momentum leg. Secondary invalidations: a 2026-07-30 Q2 print that cuts the $660–680M FY guide, a zilurgisertib PDUFA setback near 2026-09-26, or an AZURE-1 Phase 3 topline miss in 2H26.\n- On the upside, a post-print guide raise plus a reclaim of the $125 all-time high would re-accelerate the story and flip the theme back toward ACCELERATING.\n\n## Correlation Notes\n- **Incyte (INCY):** zilurgisertib is licensed from Incyte and FOP economics are shared — INCY headlines around the 2026-09-26 PDUFA read as a same-direction tell.\n- **Small/mid-cap biotech beta (XBI):** binary-event names amplify sector risk-on/off swings; a hostile biotech tape can drag MIRM independent of company news.\n- **HDV competitive read-through:** Gilead's bulevirtide (ex-U.S.) and other HBV/HDV programs frame brelovitug's commercial opportunity and any read-across from competitor data.",
  "first_seen": "2026-07-10",
  "last_analyzed": "2026-07-26T12:29:35+00:00",
  "last_synthesized": "2026-07-26",
  "last_update_source": "watchlist_research",
  "license": "Content © orbyd. Cite the canonical URL."
}