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Ultragenyx Pharmaceutical
(NASDAQ:RARE)
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Rating:49Neutral
Price Target:
$14.50
▼(-45.20% Downside)
Action:Reiterated
Date:10/07/26
The score is held down primarily by weak financial quality (large ongoing losses, heavy cash burn, and balance sheet deterioration) and bearish technical trend signals. Offsetting these are constructive earnings-call messaging on revenue execution and a 2027 profitability plan, plus meaningful corporate catalysts (gene-therapy approvals and non-dilutive PRV monetization), though pipeline setback risk remains material.
Positive Factors
Commercial revenue growth and product breadth
Revenue has more than doubled since 2021 while sales are spread across several approved rare-disease therapies. This commercial base provides recurring operating scale, supports patient access investments, and creates a platform for further launches rather than relying solely on one clinical program.
Negative Factors
Deep operating losses and sustained cash burn
Ultragenyx continues to consume significant cash while operating losses remain very large, showing that commercial revenue has not yet covered research, development, and selling costs. Persistent negative cash generation may require funding actions and limits flexibility to invest across the pipeline.
Read all positive and negative factors
Positive Factors
Negative Factors
Commercial revenue growth and product breadth
Revenue has more than doubled since 2021 while sales are spread across several approved rare-disease therapies. This commercial base provides recurring operating scale, supports patient access investments, and creates a platform for further launches rather than relying solely on one clinical program.
Read all positive factors
Ultragenyx Pharmaceutical Key Performance Indicators (KPIs)
Any
Revenue by Geography
Breaks 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.
Breaks 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.
Data provided by:
The Fly
The FlyUltragenyx Pharmaceutical (RARE) vs. SPDR S&P 500 ETF (SPY)
Market Cap
$1.53B
Dividend YieldN/A
Average Volume (3M)5.97M
Price to Earnings (P/E)―
Beta (1Y)1.43
Revenue Growth17.50%
EPS Growth-5.66%
CountryUS
Employees1,371
SectorHealthcare
Sector Strength45
IndustryBiotechnology
Share Statistics
EPS (TTM)-5.84
Shares Outstanding98,588,875
10 Day Avg. Volume4,565,523
30 Day Avg. Volume5,965,443
Financial Highlights & Ratios
PEG Ratio0.54
Price to Book (P/B)-28.35
Price to Sales (P/S)3.37
P/FCF Ratio-4.80
Enterprise Value/Market Cap2.82
Enterprise Value/Revenue6.01
Enterprise Value/Gross Profit7.23
Enterprise Value/Ebitda-9.11
Forecast
1Y Price Target
$28.47Price Target Upside7.61% Upside
Rating ConsensusModerate Buy
Number of Analyst Covering20
EPS Forecast (FY)-3.96
Revenue Forecast (FY)$759.02M
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 internation...
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 throug...
Ultragenyx Pharmaceutical Earnings Call Summary
Earnings Call Date:Aug 04, 2026
(Q2-2026)
| % Change Since: |
Next Earnings Date:Nov 04, 2026
Earnings Call Sentiment Positive
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.Positive Updates
Record Quarterly Revenue
Total revenue of $214 million in Q2 2026, the highest quarterly revenue in company history, reinforcing reaffirmed full-year revenue guidance.
Negative Updates
Operating Loss and Expense Run-Rate
Total operating expenses of $289 million in Q2 2026 (cost of sales $34M; combined R&D and SG&A $255M), resulting in a net loss of $92 million ($0.90 per share) for the quarter; operating expenses exceed quarterly revenue ($289M vs $214M).
Read all updates
Q2-2026 Updates
Positive
Negative
Record Quarterly Revenue
Total revenue of $214 million in Q2 2026, the highest quarterly revenue in company history, reinforcing reaffirmed full-year revenue guidance.
Read all positive 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
Income Statement
32
Negative
Balance Sheet
28
Negative
Cash Flow
30
Negative
| 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
Neutral
26.46
Price Trends
20.07
Negative
23.71
Negative
23.61
Negative
Market Momentum
-1.28
Negative
43.12
Neutral
74.72
Neutral
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For RARE, the sentiment is Neutral. The current price of 26.46 is above the 20-day moving average (MA) of 14.62, above the 50-day MA of 20.07, and above the 200-day MA of 23.61, indicating a neutral trend. The MACD of -1.28 indicates Negative momentum. The RSI at 43.12 is Neutral, neither overbought nor oversold. The STOCH value of 74.72 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Neutral 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.
Finance & Corporate - Financial and accounting risks. Risks related to the execution of corporate activity and strategy
Latest Risks Added 0 New Risks
Ultragenyx Pharmaceutical Peers Comparison
UnderperformOutperform
Sector (51)
Name | Overall Rating | Market Cap | P/E Ratio | ROE | Dividend Yield | Revenue Growth | EPS Growth |
|---|---|---|---|---|---|---|---|
65 Neutral | $3.42B | -67.18 | -13.53% | ― | 105.17% | 53.58% | |
62 Neutral | $2.07B | -5.51 | -57.97% | ― | ― | -19.00% | |
58 Neutral | $4.91B | -115.83 | -55.79% | ― | 77.12% | 64.40% | |
54 Neutral | $2.38B | -9.42 | -37.17% | ― | ― | -47.00% | |
52 Neutral | $3.53B | -7.69 | -43.98% | ― | -100.00% | -33.88% | |
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
49 Neutral | $1.53B | -2.64 | 392.35% | ― | 17.50% | -5.66% |
* Healthcare Sector Average
RARE
Ultragenyx Pharmaceutical
15.48
-16.54
-51.66%
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Ultragenyx Pharmaceutical Corporate Events
Business Operations and StrategyPrivate Placements and Financing
Ultragenyx Monetizes FDA Priority Review Voucher Asset
Positive
Oct 7, 2026
On 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...
Business Operations and StrategyProduct-Related Announcements
Ultragenyx wins FDA approval for FAYUVI gene therapy
Positive
Sep 17, 2026
On 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 sy...
Business Operations and Strategy
Ultragenyx pivots strategy after Aspire Phase 3 setback
Negative
Sep 2, 2026
On 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 n...
Business Operations and StrategyProduct-Related Announcements
Ultragenyx Wins FDA Accelerated Approval for GENGLYCOS
Positive
Aug 19, 2026
On 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 gly...
Business Operations and StrategyLegal Proceedings
Ultragenyx Settles DOJOLVI Patent Dispute with Esjay
Positive
Aug 11, 2026
On 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. Und...
Glossary
BuyA stock rated as a "Buy" is expected to perform better than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock is likely to deliver higher returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
HoldA stock rated as a "Hold" is expected to perform in line with the overall market or a specific benchmark. This rating indicates that the stock is neither particularly compelling nor unfavorable for investment. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
SellA stock rated as a "Sell" is expected to perform worse than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock may deliver lower returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
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.