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Ultragenyx Pharmaceutical (RARE) AI Stock Analysis
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Ultragenyx Pharmaceutical
(NASDAQ:RARE)
The score is held down primarily by weak financial quality (deep losses, significant cash burn, and negative equity) and a bearish technical trend (price far below major moving averages with negative MACD). Offsetting factors include constructive business momentum and guidance from the latest earnings call (record revenue and planned cost reductions toward a 2027 profitability target) plus corporate catalysts/financing improvements from recent FDA approvals and PRV cash monetizations, though pipeline setbacks add risk.
Ultragenyx Pharmaceutical Key Performance Indicators (KPIs)
Revenue by GeographyBreaks down revenue across different regions, revealing where Ultragenyx Pharmaceutical is strongest and where it may face risk or growth potential due to local economic conditions or market share shifts.
North America remains the dominant revenue base, but the mix is materially diversifying: Latin America has become a high-growth — and lumpy — contributor driven by Crysvita bulk government orders and expanding patient starts, while Europe and Japan are steadily ramping from international launches (Evkeeza, Dojolvi). That geographic diversification supports management’s reaffirmed 2026 guidance, but quarter-to-quarter volatility from ministry orders and reliance on PRV monetization and successful launches mean investors should focus on sustained reimbursement and execution rather than any single quarter.
The FlyUltragenyx Pharmaceutical (RARE) vs. SPDR S&P 500 ETF (SPY)
- Market Cap
- $1.59B
- Dividend Yield
- ―
- Average Volume (3M)
- 4.21M
- Price to Earnings (P/E)
- -2.8
- Beta (1Y)
- 1.41
- Revenue Growth
- 17.50%
- EPS Growth
- -5.66%
- Country
- US
- Employees
- 1,371
- Sector
- Healthcare
- Sector Strength
- 51
- Industry
- Biotechnology
Ultragenyx Pharmaceutical Business Overview & Revenue Model
Company Description
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical firm dedicated to discovering, developing, and commercializing innovative treatments for rare and ultra-rare genetic conditions. Its operations span North America, Europe, and other international...
How the Company Makes Money
Ultragenyx makes money primarily by selling its commercial rare-disease medicines and recording product revenue from those sales. The company’s key revenue streams are: (1) Product sales of its approved therapies, where revenue is generated through d...
Ultragenyx Pharmaceutical Earnings Call Summary
Positive overall: the call highlighted record quarterly revenue, broad-based product growth (including Evkeeza +50% YoY), expanding patient counts across multiple geographies, improving cash consumption versus Q1, and clear near-term catalysts (two PDUFAs and a pivotal readout). Management articulated a credible path to profitability in 2027 with expense discipline and potential non-dilutive PRV monetization. Offsetting risks include a sizable operating loss and expense run-rate, reliance on binary regulatory outcomes, an unmet primary outcome for setrusumab requiring possible new trials, and readout/regulatory uncertainty for GTX-102, but these are framed as manageable and addressable within management's plan.
Q2-2026 Updates
Positive Updates
Negative Updates
Company Guidance
Ultragenyx reaffirmed full-year revenue guidance while reporting Q2 2026 results that included $214M total revenue (Crysvita $156M: $94M North America / $54M Latin America & Turkey / $8M Europe; DOJOLVI $27M; Evkeeza $21M, +50% vs Q2 2025; MEPSEVII $10M), total operating expenses of $289M (cost of sales $34M; combined R&D & SG&A $255M, including $34M non‑cash stock‑based comp), net loss of $92M ($0.90/share), $436M in cash & marketable securities as of June 30 and $97M net cash used in operations for the quarter; management reiterated combined R&D & SG&A guidance (2026 flat to down low‑single digits vs 2025) and a targeted ≥15% reduction in 2027 as part of a plan (plus double‑digit revenue growth, contributions from potential DTX‑401 and UX‑111 launches, and monetization of PRVs if approved) to reach profitability in 2027.
Ultragenyx Pharmaceutical Financial Statement Overview
Summary
Strong revenue scale-up ($351M in 2021 to $673M in 2025; $717M TTM) and very high gross margins (~84–95%) are key positives. However, profitability and cash generation remain weak with TTM net margin at -91% and large ongoing cash burn (TTM operating cash flow about -$485M; free cash flow about -$489M). Balance-sheet risk is elevated due to the shift to negative equity (TTM -$284M) and the large debt spike shown in 2025 (~$1.28B).
Income Statement
32NegativeBalance Sheet
28NegativeCash Flow
30Negative| Breakdown | TTM | Dec 2025 | Dec 2024 | Dec 2023 | Dec 2022 | Dec 2021 |
|---|---|---|---|---|---|---|
Income Statement | ||||||
| Total Revenue | 716.93M | 673.00M | 560.23M | 434.25M | 363.33M | 351.41M |
| Gross Profit | 595.94M | 564.00M | 483.50M | 389.04M | 335.01M | 335.40M |
| EBITDA | -473.00M | -474.00M | -469.00M | -516.45M | -640.49M | -410.32M |
| Net Income | -586.41M | -575.00M | -569.18M | -606.64M | -707.42M | -454.02M |
Balance Sheet | ||||||
| Total Assets | 1.26B | 1.53B | 1.50B | 1.49B | 1.55B | 1.52B |
| Cash, Cash Equivalents and Short-Term Investments | 292.00M | 693.00M | 610.02M | 577.21M | 747.76M | 740.20M |
| Total Debt | 1.20B | 1.28B | 40.34M | 43.17M | 31.59M | 41.97M |
| Total Liabilities | 1.55B | 1.60B | 1.24B | 1.22B | 1.19B | 599.84M |
| Stockholders Equity | -291.00M | -80.00M | 255.30M | 275.41M | 352.49M | 922.56M |
Cash Flow | ||||||
| Free Cash Flow | -489.47M | -472.00M | -421.68M | -521.57M | -526.59M | -411.79M |
| Operating Cash Flow | -485.25M | -466.00M | -414.19M | -474.81M | -380.46M | -338.69M |
| Investing Cash Flow | 39.94M | 236.00M | -17.77M | 168.00M | -291.65M | -195.37M |
| Financing Cash Flow | 397.19M | 478.00M | 399.24M | 388.14M | 501.21M | 118.55M |
Ultragenyx Pharmaceutical Technical Analysis
Price Trends
Market Momentum
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For RARE, the sentiment is Negative. The current price of 16.10 is above the 20-day moving average (MA) of 15.55, below the 50-day MA of 17.01, and below the 200-day MA of 22.90, indicating a neutral trend. The MACD of -0.01 indicates Negative momentum. The RSI at 49.82 is Neutral, neither overbought nor oversold. The STOCH value of 69.89 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for RARE.
Ultragenyx Pharmaceutical Risk Analysis
Ultragenyx Pharmaceutical disclosed 61 risk factors in its most recent earnings report. Ultragenyx Pharmaceutical reported the most risks in the "Tech & Innovation" category.
Latest Risks Added
0 New RisksUltragenyx Pharmaceutical Peers Comparison
Overall Rating
TARS78Outperform
TVTX68Neutral
IRON67Neutral
XENE60Neutral
MLYS49Neutral- RARE49Neutral
Financial Indicators
| Name | Overall Rating | Market Cap | P/E Ratio | ROE | Dividend Yield | Revenue Growth | EPS Growth |
|---|---|---|---|---|---|---|---|
| TARS | 78Outperform | $3.69B | -68.67 | -13.53% | ― | 105.17% | 53.58% |
| TVTX | 68Neutral | $4.75B | -111.98 | -55.79% | ― | 77.12% | 64.40% |
| IRON | 67Neutral | $3.59B | -12.25 | -37.17% | ― | ― | -47.00% |
| XENE | 60Neutral | $3.62B | -7.89 | -43.98% | ― | -100.00% | -33.88% |
| Sector * | 51Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% |
| MLYS | 49Neutral | $1.61B | -4.29 | -57.97% | ― | ― | -19.00% |
| RARE | 49Neutral | $1.59B | -2.76 | 392.35% | ― | 17.50% | -5.66% |
* Healthcare Sector Average
Performance Comparison
| Show on chart | Ticker | Company Name | Price | Change | % Change |
|---|---|---|---|---|---|
| RARE | Ultragenyx Pharmaceutical, Inc. | 16.10 | -17.37 | -51.90% | |
| TVTX | Travere Therapeutics, Inc. | 50.39 | 15.86 | 45.93% | |
| XENE | Xenon Pharmaceuticals | 37.41 | -3.71 | -9.02% | |
| IRON | Disc Medicine, Inc. | 80.84 | -3.85 | -4.55% | |
| TARS | Tarsus Pharmaceuticals, Inc. | 74.85 | 5.06 | 7.25% | |
| MLYS | Mineralys Therapeutics, Inc. | 18.18 | -20.99 | -53.59% |
Ultragenyx Pharmaceutical Corporate Events
Ultragenyx, Baylor Reach Settlement on DOJOLVI Patents
NeutralOn October 19, 2026, Ultragenyx Pharmaceutical Inc. and Baylor Research Institute reached a settlement with Navinta LLC, resolving patent litigation over patents covering Ultragenyx’s DOJOLVI triheptanoin oral liquid. The dispute stemmed from N...
Ultragenyx Sells FDA Priority Review Voucher for Cash
PositiveOn October 8, 2026, Ultragenyx Pharmaceutical Inc. agreed to sell a Rare Pediatric Disease Priority Review Voucher that it had received from the U.S. Food and Drug Administration on September 17, 2026, following approval of FAYUVI (UX111) for treatin...
Ultragenyx Monetizes FDA Priority Review Voucher Asset
PositiveOn October 6, 2026, Ultragenyx Pharmaceutical Inc. entered into an asset purchase agreement to sell a Rare Pediatric Disease Priority Review Voucher that it received from the U.S. Food and Drug Administration on August 19, 2026, following approval of...
Ultragenyx wins FDA approval for FAYUVI gene therapy
PositiveOn September 17, 2026, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration granted full approval to FAYUVI, also known as UX111, for treating pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndr...
Ultragenyx pivots strategy after Aspire Phase 3 setback
NegativeOn September 2, 2026, Ultragenyx reported Phase 3 results from the Aspire study of apazunersen (GTX-102) in Angelman syndrome, showing the trial failed to meet its primary cognitive endpoint and key secondary multidomain responder endpoint. With no e...
Ultragenyx Wins FDA Accelerated Approval for GENGLYCOS
PositiveOn August 19, 2026, Ultragenyx announced that the U.S. Food and Drug Administration granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, for adult and pediatric patients eight years and older with glycog...
Ultragenyx Settles DOJOLVI Patent Dispute with Esjay
PositiveOn August 7, 2026, Ultragenyx Pharmaceutical and Baylor Research Institute reached a settlement with Esjay Pharma resolving U.S. patent litigation over DOJOLVI, after Esjay filed an Abbreviated New Drug Application to market a generic version. Under...
Disclaimer
This AI Analyst Stock Report is automatically generated by our AI systems using advanced algorithms and publicly available financial, technical, and market data. While the information provided aims to be accurate and insightful, it is intended for informational purposes only and should not be considered financial advice. Any content created by an AI (Artificial Intelligence) system may contain inaccuracies and/or contain errors. Investing in stocks carries inherent risks, and past performance is not indicative of future results. This report does not account for your personal financial circumstances, objectives, or risk tolerance. Always conduct your own research or consult with a qualified financial advisor before making investment decisions. The analysis and recommendations provided are based on historical and current data and may not fully reflect future market conditions or unexpected developments. Neither the creators of this report nor its affiliated entities guarantee the accuracy, completeness, or reliability of the information presented. Use this report at your own discretion and risk.
Date of analysis: Oct 14, 2026