(RARE) Ultragenyx - Overview
Stock: Enzyme, Gene, Antibody, Metabolic, Rare
EPS (Earnings per Share)
Revenue
| Risk 5d forecast | |
|---|---|
| Volatility | 49.9% |
| Relative Tail Risk | -12.2% |
| Reward TTM | |
|---|---|
| Sharpe Ratio | -0.55 |
| Alpha | -60.69 |
| Character TTM | |
|---|---|
| Beta | 0.955 |
| Beta Downside | 0.574 |
| Drawdowns 3y | |
|---|---|
| Max DD | 66.78% |
| CAGR/Max DD | -0.26 |
Description: RARE Ultragenyx January 12, 2026
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is a U.S. biotech that discovers, acquires, develops, and commercializes therapies for rare and ultra-rare genetic diseases across North America, Europe, the Middle East, Africa, and the Asia-Pacific.
Its approved biologics include Crysvita (burosumab) for X-linked hypophosphatemia and tumor-induced osteomalacia, Mepsevii for mucopolysaccharidosis VII, Dojolvi for long-chain fatty-acid oxidation disorders, and Evkeeza (evinacumab) for homozygous familial hypercholesterolemia.
The pipeline features several Phase 3 candidates: UX143 (osteogenesis imperfecta), UX111 (AAV9 gene therapy for Sanfilippo A/MPS IIIA), DTX401 (AAV8 gene therapy for glycogen storage disease Ia), DTX301 (AAV8 therapy for ornithine transcarbamylase deficiency), and GTX-102 (antisense oligonucleotide for Angelman syndrome). Phase 2 assets include UX701 (Wilson disease) and UX053 (glycogen storage disease III).
Ultragenyx maintains a network of collaborations and license agreements with partners such as Kyowa Kirin, Regeneron, REGENXBIO, GeneTx, Mereo, and Arcturus, which provide viral-vector and antisense platforms and help share development risk.
Key recent KPIs (Q4 2023): revenue $274 million, net loss $156 million, cash and equivalents $1.1 billion giving roughly an 18-month cash runway; R&D expense rose 12% YoY, reflecting the heavy pipeline investment.
Sector drivers that materially affect Ultragenyx’s outlook include: (1) sustained payer willingness to reimburse high-price orphan drugs, (2) FDA’s accelerated approval pathways that shorten time-to-market for rare-disease therapies, and (3) a projected 13% CAGR for the global gene-therapy market through 2030, which expands the addressable market for its AAV candidates.
Investors should watch cash burn rate, partnership milestone payments, and regulatory decisions closely, as any delay or adverse data could materially impair the company’s ability to fund its pipeline.
For a data-driven deep-dive, you might explore ValueRay’s analyst toolkit for Ultragenyx’s valuation sensitivities.
Piotroski VR‑10 (Strict, 0-10) 2.0
| Net Income: -579.8m TTM > 0 and > 6% of Revenue |
| FCF/TA: -0.38 > 0.02 and ΔFCF/TA -9.35 > 1.0 |
| NWC/Revenue: 48.08% < 20% (prev 98.44%; Δ -50.36% < -1%) |
| CFO/TA -0.37 > 3% & CFO -445.7m > Net Income -579.8m |
| Net Debt/EBITDA: error (EBITDA <= 0) |
| Current Ratio: 1.89 > 1.5 & < 3 |
| Outstanding Shares: last quarter (99.8m) vs 12m ago 4.48% < -2% |
| Gross Margin: 84.69% > 18% (prev 0.86%; Δ 8383 % > 0.5%) |
| Asset Turnover: 46.22% > 50% (prev 33.98%; Δ 12.24% > 0%) |
| Interest Coverage Ratio: -8.93 > 6 (EBITDA TTM -483.4m / Interest Expense TTM 58.1m) |
Altman Z'' -15.00
| A: 0.25 (Total Current Assets 643.2m - Total Current Liabilities 340.0m) / Total Assets 1.19b |
| B: -3.70 (Retained Earnings -4.40b / Total Assets 1.19b) |
| C: -0.38 (EBIT TTM -518.7m / Avg Total Assets 1.36b) |
| D: -3.74 (Book Value of Equity -4.39b / Total Liabilities 1.17b) |
| Altman-Z'' Score: -16.87 = D |
Beneish M -3.08
| DSRI: 1.00 (Receivables 112.5m/93.3m, Revenue 630.6m/522.7m) |
| GMI: 1.02 (GM 84.69% / 86.25%) |
| AQI: 0.82 (AQ_t 0.25 / AQ_t-1 0.30) |
| SGI: 1.21 (Revenue 630.6m / 522.7m) |
| TATA: -0.11 (NI -579.8m - CFO -445.7m) / TA 1.19b) |
| Beneish M-Score: -3.08 (Cap -4..+1) = AA |
What is the price of RARE shares?
Over the past week, the price has changed by +2.62%, over one month by +9.58%, over three months by -20.19% and over the past year by -46.05%.
Is RARE a buy, sell or hold?
- StrongBuy: 13
- Buy: 6
- Hold: 1
- Sell: 0
- StrongSell: 0
What are the forecasts/targets for the RARE price?
| Issuer | Target | Up/Down from current |
|---|---|---|
| Wallstreet Target Price | 64 | 159.1% |
| Analysts Target Price | 64 | 159.1% |
| ValueRay Target Price | 20.4 | -17.6% |
RARE Fundamental Data Overview February 03, 2026
P/B = 255.6513
P/EG = -0.24
Revenue TTM = 630.6m USD
EBIT TTM = -518.7m USD
EBITDA TTM = -483.4m USD
Long Term Debt = 33.8m USD (from capitalLeaseObligations, last quarter)
Short Term Debt = 77.7m USD (from shortTermDebt, last quarter)
Debt = 863.1m USD (from shortLongTermDebtTotal, last quarter)
Net Debt = 660.6m USD (from netDebt column, last quarter)
Enterprise Value = 2.76b USD (2.32b + Debt 863.1m - CCE 425.2m)
Interest Coverage Ratio = -8.93 (Ebit TTM -518.7m / Interest Expense TTM 58.1m)
EV/FCF = -6.11x (Enterprise Value 2.76b / FCF TTM -451.4m)
FCF Yield = -16.35% (FCF TTM -451.4m / Enterprise Value 2.76b)
FCF Margin = -71.58% (FCF TTM -451.4m / Revenue TTM 630.6m)
Net Margin = -91.95% (Net Income TTM -579.8m / Revenue TTM 630.6m)
Gross Margin = 84.69% ((Revenue TTM 630.6m - Cost of Revenue TTM 96.5m) / Revenue TTM)
Gross Margin QoQ = 82.50% (prev 86.18%)
Tobins Q-Ratio = 2.32 (Enterprise Value 2.76b / Total Assets 1.19b)
Interest Expense / Debt = 1.64% (Interest Expense 14.1m / Debt 863.1m)
Taxrate = 21.0% (US default 21%)
NOPAT = -409.7m (EBIT -518.7m * (1 - 21.00%)) [loss with tax shield]
Current Ratio = 1.89 (Total Current Assets 643.2m / Total Current Liabilities 340.0m)
Debt / Equity = 94.24 (Debt 863.1m / totalStockholderEquity, last quarter 9.16m)
Debt / EBITDA = -1.37 (negative EBITDA) (Net Debt 660.6m / EBITDA -483.4m)
Debt / FCF = -1.46 (negative FCF - burning cash) (Net Debt 660.6m / FCF TTM -451.4m)
Total Stockholder Equity = 140.0m (last 4 quarters mean from totalStockholderEquity)
RoA = -42.50% (Net Income -579.8m / Total Assets 1.19b)
RoE = -414.2% (Net Income TTM -579.8m / Total Stockholder Equity 140.0m)
RoCE = -298.4% (out of range, set to none) (EBIT -518.7m / Capital Employed (Equity 140.0m + L.T.Debt 33.8m))
RoIC = -292.7% (out of range, set to none) (NOPAT -409.7m / Invested Capital 140.0m)
WACC = 7.23% (E(2.32b)/V(3.19b) * Re(9.44%) + D(863.1m)/V(3.19b) * Rd(1.64%) * (1-Tc(0.21)))
Discount Rate = 9.44% (= CAPM, Blume Beta Adj.)
Shares Correlation 3-Years: 100.0 | Cagr: 10.90%
Fair Price DCF = unknown (Cash Flow -451.4m)
EPS Correlation: 73.77 | EPS CAGR: 27.10% | SUE: 4.0 | # QB: 1
Revenue Correlation: 94.95 | Revenue CAGR: 18.97% | SUE: -0.62 | # QB: 0
EPS next Quarter (2026-03-31): EPS=-1.38 | Chg30d=+0.096 | Revisions Net=+2 | Analysts=8
EPS next Year (2026-12-31): EPS=-4.27 | Chg30d=+0.195 | Revisions Net=+2 | Growth EPS=+24.2% | Growth Revenue=+18.6%