Commercial base ($700M+ 2026E rev, 20% growth) provides a soft SotP floor at ~$10–11/sh vs current $13.61. Two consecutive Phase-3 failures (setrusumab Dec-2025, GTX-102 Aug-2026) drove −65% drawdown from 52W high. UX111 PDUFA on Sep-19-2026 (T-3 days) is a hard binary. Analyst PT range $23–$33 (+70%/+140%) implies material re-rating if pipeline delivers; class action pending on setrusumab is the tail risk.
SotP with peer-median P/S applied to commercial products (Crysvita, Dojolvi, Mepsevii, Evkeeza) risk-adjusted for concentration and KKC economic split; pipeline rNPV for UX111 and other Phase-3 assets; PV-discounted royalty liabilities as negative financial component; litigation reserve for setrusumab class action. Implicit fw P/S 2.3x, within peer range 1.5x–4.5x. Cross-check with median sell-side PT $28 shows sell-side gives fuller pipeline credit than we do — our base is more conservative. Sensitivity: ±0.5x P/S = ±$3.4/sh; ±20pp UX111 probability = ±$0.8/sh. ⚠️ Not investment advice.
| Component | Assumption | USD/share |
|---|---|---|
| Crysvita commercial franchise | 2026E rev $510M × 2.5x fw P/S (peer median 3.0x, −20% for KKC ex-US economic split & Latam ordering volatility) = $1,275M equity value | +12.93 |
| Dojolvi + Mepsevii + Evkeeza + other | 2026E rev $220M × 2.0x fw P/S (mature slow-growth orphan biologics, no growth optionality) = $440M | +4.46 |
| UX111 pipeline option (Sanfilippo A) | 60% approval probability × $400M NPV (peak US sales $200–300M, 10-yr NPV @ 10% WACC) = $240M | +2.43 |
| Other Phase-3 pipeline options | rNPV blend: setrusumab BMD-path 15% × $250M + UX701/DTX401 20% × $200M = $77M | +0.78 |
| Cash − PV royalty liabilities | $436M cash − PV of $1.2B non-recourse royalty debt @ 7% blended = ~$850M PV → net financial −$414M | −4.20 |
| Litigation reserve (setrusumab class action) | Expected settlement mid-case $40M (net of D&O insurance $20M), P(mat. exposure) 70% = $20M | −0.20 |
| FV base case | Exact sum: 12.93 + 4.46 + 2.43 + 0.78 − 4.20 − 0.20 = $16.20 → rounded to $17 | ≈ $17.00 |
Short interest moderate: bears are positioned but not extreme. UX111 approval could trigger short-covering rally worth +8–12% on top of fundamental re-rating. Note: no insider buying reported in 12M window; several 10b5-1 sales by executives (aggregate <$3M — routine, not a red flag on its own).
| Item ($M) | FY2023 | FY2024 | FY2025 | Q2-26 (LTM) | Guidance 2026 |
|---|---|---|---|---|---|
| Total revenue | 434 | 560 | 673 | ~760 | 730–780 |
| Crysvita | 328 | 410 | 481 | ~510 | 500–520 |
| Dojolvi | 71 | 88 | 96 | ~100 | 100–110 |
| Net loss | −410 | −440 | −395 | ~−380 | −350 to −370 |
| Cash & equivalents (EoP) | 620 | 710 | 540 | 436 | ~300–350 |
| Royalty liabilities | 800 | 1,050 | 1,180 | 1,200 | 1,200 |
| Metric | Q2 2025 | Q3 2025 | Q4 2025 | Q1 2026 | Q2 2026 |
|---|---|---|---|---|---|
| Revenue ($M) | 159 | 180 | 175 | 190 | 214 |
| Rev growth YoY % | +18% | +22% | +18% | +28% | +35% |
| Net loss ($M) | −115 | −105 | −95 | −100 | −92 |
| End-of-period cash ($M) | 620 | 590 | 540 | 500 | 436 |
Business model — Ultragenyx Pharmaceutical Inc.
Crysvita (burosumab) ~$500–520M FY26E (72% rev) 🟢 ramping X-linked hypophosphatemia + tumor-induced osteomalacia. Partnered with Kyowa Kirin ex-US. Growth +17% in 2025. Concentration risk = single-product dependency. Latin America order patterns volatile. Dojolvi (triheptanoin) ~$100–110M FY26E (15% rev) 🟢 stable growth Long-chain fatty acid oxidation disorders. +9% growth 2025. Slower but sticky orphan market. Small patient population capped upside; steady margin contributor. Mepsevii + Evkeeza ~$70M FY26E (10% rev) 🟡 mature Mepsevii (MPS VII) mature; Evkeeza (evinacumab in HoFH) small ramp. Combined not driving valuation; adds product diversification but limited growth optionality. UX111 (Sanfilippo A GT) Pipeline · PDUFA 2026-09-19 🟡 binary event T-3d AAV gene therapy for MPS IIIA. Second review cycle after facility CRL. Potential $200–400M peak. Approval = orphan launch + re-rating catalyst. Rejection = pipeline damage compounds. Setrusumab (UX143) Under reassessment 🔴 primary miss + class action Osteogenesis Imperfecta. Phase-3 Orbit/Cosmic missed primary (fracture reduction) Dec-2025 but BMD improved. Class action filed. Path forward unclear; company evaluating composite endpoint filings. Other Phase-3 assets Pipeline · 2027–2028 🟡 development DTX401 (GSDIa gene therapy), UX701 (Wilson disease gene therapy). Data readouts late 2027–2028. Multiple shots on goal but funding at risk if UX111 rejected.
Legal, regulatory and risk analysis
SWOT analysis
- +4 commercial products generating $730M+ annualized revenue growing 15–20%
- +Q2-26 record quarterly revenue $214M; commercial base intact despite pipeline setbacks
- +Multiple late-stage pipeline assets in gene therapy (UX111, DTX401, UX701)
- +Kyowa Kirin partnership on Crysvita ex-US caps commercial risk
- +Orphan-drug pricing power in ultra-rare indications
- −Two consecutive Phase-3 primary-endpoint failures in 9 months (setrusumab, GTX-102)
- −$1.2B non-recourse royalty liabilities reduce economic equity value
- −Cash burn ~$97M/qtr; runway ~5 quarters requires disciplined execution
- −72% revenue concentration on Crysvita
- −Zero buyback; no history of shareholder return; management credibility damaged
- →UX111 approval Sep-19 unlocks +50/+80% re-rating and Sanfilippo A orphan franchise
- →Peer P/S at 3–10x vs RARE 1.8x — re-rating room even without new catalysts
- →Setrusumab reprocessing on BMD/composite endpoint could salvage the program
- →M&A optionality: BMRN, ALNY historically active acquirers in the space
- →Cost-cut plan announced post-Angelman may extend runway meaningfully
- !UX111 second CRL would compound sell-off and shift narrative to going concern
- !Class action discovery may surface further disclosure issues on setrusumab
- !Dilutive equity raise probable within 6–9 months if UX111 rejected
- !Kyowa Kirin partnership renegotiation risk if commercial underperforms
- !Sell-side downgrades continued: Morgan Stanley cut to Equal Weight Sep-3; further cuts possible
Summary by assessment area
- Cash $436M vs quarterly burn ~$97M = ~5 quarters runway
- Royalty liabilities $1.2B reduce economic equity value
- Commercial base $730M growing 15–20% provides revenue floor
- Dilutive raise probable if UX111 rejected
- UX111 PDUFA Sep-19 (binary)
- Active securities class action on setrusumab
- Two Phase-3 primary-endpoint failures in 9 months
- Management credibility questioned by sell-side
- Downside to SotP floor ~$10–11 (−20/−25%)
- Base FV $17 (+25%); consensus PT $28 (+106%)
- Ratio upside/downside ~3–4x — passes ASYMMETRY gate
- Position sizing must reflect binary nature (T-3 days)
Sources: Ultragenyx Q2-2026 earnings release & press releases (ir.ultragenyx.com), 10-Q filings via SEC EDGAR, Yahoo Finance, Robinhood, TMX Money, StockAnalysis, Kessler Topaz Meltzer & Check LLP class action notice, Morgan Stanley/Cantor Fitzgerald/Wedbush/Barclays/Citi analyst reports (Sep-2026), fffinstill peer database. Market data — last verified close 2026-09-15 (T-1): RARE $13.61, market cap ~$1.34B, 52W range $13.81–$39.89, shares outstanding 98,588,873. Short interest ~9% (approx). Peer P/S multiples per Simply Wall St and StockAnalysis. Report as of 2026-09-16. ⚠️ This document is for informational purposes only and does not constitute financial or investment advice.