Ultragenyx Pharmaceutical (RARE)
NASDAQ:RARE
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Ultragenyx Pharmaceutical
(NASDAQ:RARE)
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Rating:45Neutral
Price Target:
$19.00
▼(-11.30% Downside)
Action:ReiteratedDate:03/30/26
The score is held down primarily by weak financial durability (large operating losses, substantial cash burn, and a sharply reduced equity base). Technicals also indicate a sustained downtrend. Offsetting these are earnings-call signals of a credible cost-reduction plan with profitability targeted for 2027 and supportive pipeline/regulatory milestones, but these benefits are still dependent on successful execution and regulatory outcomes.
Positive Factors
Revenue Growth & Gross Margin
Ultragenyx demonstrates multi-year top-line expansion to $673M (2025) alongside very high gross margins (~84%). That combination signals durable pricing power in orphan drugs and efficient product economics, supporting scalable product-level cash generation as sales grow.
Negative Factors
High Cash Burn
Persistent large negative operating and free cash flows indicate the company remains cash‑consumptive and reliant on external funding. High absolute burn limits financial flexibility, raises refinancing/dilution risk, and makes successful execution of approvals and cost cuts critical to runway beyond 2026.
Read all positive and negative factors
Positive Factors
Negative Factors
Revenue Growth & Gross Margin
Ultragenyx demonstrates multi-year top-line expansion to $673M (2025) alongside very high gross margins (~84%). That combination signals durable pricing power in orphan drugs and efficient product economics, supporting scalable product-level cash generation as sales grow.
Read all positive factors
Ultragenyx Pharmaceutical (RARE) vs. SPDR S&P 500 ETF (SPY)
Market Cap
$2.07B
Dividend YieldN/A
Average Volume (3M)2.13M
Price to Earnings (P/E)―
Beta (1Y)1.17
Revenue Growth20.63%
EPS Growth8.09%
CountryUS
Employees1,294
SectorHealthcare
Sector Strength45
IndustryBiotechnology
Share Statistics
EPS (TTM)-1.29
Shares Outstanding96,629,790
10 Day Avg. Volume2,626,891
30 Day Avg. Volume2,129,114
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 Cap1.41
Enterprise Value/Revenue4.33
Enterprise Value/Gross Profit5.16
Enterprise Value/Ebitda-6.07
Forecast
1Y Price Target
$51.82Price Target Upside141.92% Upside
Rating ConsensusStrong Buy
Number of Analyst Covering19
EPS Forecast (FY)-4.22
Revenue Forecast (FY)$769.09M
Ultragenyx Pharmaceutical Business Overview & Revenue Model
Company Description
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and i...
How the Company Makes Money
Ultragenyx primarily makes money by selling its approved rare-disease medicines in markets where it has commercialization rights, generating product revenue recognized on shipments to customers (such as specialty pharmacies, distributors, or other...
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 Earnings Call Summary
Earnings Call Date:Feb 12, 2026
(Q4-2025)
| % Change Since: |
Next Earnings Date:Apr 30, 2026
Earnings Call Sentiment Neutral
The call balanced clear commercial momentum and promising late-stage clinical progress with meaningful near-term challenges. Highlights include 20% revenue growth in 2025, strong product-level growth (Crysvita +17%, Evkeeza +84%), an expanded global commercial footprint, multiple imminent clinical/regulatory catalysts (DTX401 rolling BLA with expected Q3 2026 PDUFA, GTX-102 Phase III readout H2 2026), and a restructuring plan targeting profitability in 2027 (notably R&D and SG&A reductions). Offsetting these positives are a significant FY2025 net loss ($575M), a CRL for UX111 that requires a resubmission and delays potential approval, high combined R&D/SG&A spend (~$1.1B), a 10% workforce reduction, and regulatory uncertainty around biomarker acceptance and reliance on PRVs and approvals to reach profitability. Overall, the call conveys progress and a credible path forward but with substantial execution and regulatory risks that temper near-term optimism.Positive Updates
Strong Full-Year Revenue Growth
Total revenue for 2025 was $673 million, up 20% year-over-year and exceeding the upper end of guidance.
Negative Updates
Complete Response Letter for UX111 BLA
The FDA issued a Complete Response Letter (CRL) for the UX111 BLA requiring additional supportive CMC documentation (materials typically provided during inspection), necessitating a resubmission and causing regulatory delay for MPS IIIA.
Read all updates
Q4-2025 Updates
Positive
Negative
Strong Full-Year Revenue Growth
Total revenue for 2025 was $673 million, up 20% year-over-year and exceeding the upper end of guidance.
Read all positive updates
Company Guidance
Ultragenyx guided 2026 total revenue of $730–760 million (8–13% growth vs. 2025), excluding potential revenue from new product launches, with Crysvita forecast at $500–520 million and Dojolvi at $100–110 million; 2026 combined R&D and SG&A are expected to be flat to down low single digits versus 2025. Looking to 2027, management expects R&D to fall ~38% (~$280 million) versus 2025 and combined R&D+SG&A to be down at least 15% versus 2025 as SG&A increases to support launches, all as part of a plan to reach profitability in 2027; the company also announced a 10% workforce reduction (~130 FTEs). For context, full‑year 2025 results were: total revenue $673 million (20% YoY), Crysvita $481M (17%; NA $275M, LatAm & Turkey $177M, Europe $29M), Dojolvi $96M (9%), Evkeeza $59M (84%), MEPSEVII $37M; cost of sales $109M; combined R&D+SG&A ~$1.1B; net loss $575M ($5.83/sh); and cash, cash equivalents and marketable securities of $738M.Ultragenyx Pharmaceutical Financial Statement Overview
Summary
Income Statement
32
Negative
Balance Sheet
28
Negative
Cash Flow
22
Negative
| Breakdown | Dec 2025 | Dec 2024 | Dec 2023 | Dec 2022 | Dec 2021 |
|---|---|---|---|---|---|
Income Statement | |||||
| Total Revenue | 673.00M | 560.23M | 434.25M | 363.33M | 351.41M |
| Gross Profit | 564.00M | 483.50M | 389.04M | 335.01M | 335.40M |
| EBITDA | -500.00M | -469.00M | -516.45M | -640.49M | -410.32M |
| Net Income | -575.00M | -569.18M | -606.64M | -707.42M | -454.02M |
Balance Sheet | |||||
| Total Assets | 1.53B | 1.50B | 1.49B | 1.55B | 1.52B |
| Cash, Cash Equivalents and Short-Term Investments | 693.00M | 610.02M | 577.21M | 747.76M | 740.20M |
| Total Debt | 1.28B | 40.34M | 43.17M | 31.59M | 41.97M |
| Total Liabilities | 1.60B | 1.24B | 1.22B | 1.19B | 599.84M |
| Stockholders Equity | -80.00M | 255.30M | 275.41M | 352.49M | 922.56M |
Cash Flow | |||||
| Free Cash Flow | -472.00M | -421.68M | -521.57M | -526.59M | -411.79M |
| Operating Cash Flow | -466.00M | -414.19M | -474.81M | -380.46M | -338.69M |
| Investing Cash Flow | 236.00M | -17.77M | 168.00M | -291.65M | -195.37M |
| Financing Cash Flow | 478.00M | 399.24M | 388.14M | 501.21M | 118.55M |
Ultragenyx Pharmaceutical Technical Analysis
Neutral
21.42
Price Trends
22.29
Negative
26.49
Negative
28.94
Negative
Market Momentum
-0.67
Negative
51.36
Neutral
74.30
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 21.42 is above the 20-day moving average (MA) of 20.86, below the 50-day MA of 22.29, and below the 200-day MA of 28.94, indicating a neutral trend. The MACD of -0.67 indicates Negative momentum. The RSI at 51.36 is Neutral, neither overbought nor oversold. The STOCH value of 74.30 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 |
|---|---|---|---|---|---|---|---|
62 Neutral | $2.99B | -51.51 | -19.63% | ― | 182.44% | 47.21% | |
58 Neutral | $5.41B | -10.27 | -55.81% | ― | ― | -38.30% | |
57 Neutral | $2.18B | -22.67 | -32.94% | ― | ― | 9.60% | |
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
47 Neutral | $2.90B | -67.82 | -79.16% | ― | 114.22% | 77.02% | |
45 Neutral | $2.07B | -3.94 | -1024.42% | ― | 20.63% | 8.09% | |
43 Neutral | $2.47B | -13.21 | -32.80% | ― | ― | -34.96% |
* Healthcare Sector Average
RARE
Ultragenyx Pharmaceutical
21.42
-13.71
-39.03%
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Ultragenyx Pharmaceutical Corporate Events
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
Ultragenyx BLA for UX111 Accepted for FDA Review
Positive
Apr 2, 2026
On April 2, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Administration accepted for review its resubmitted Biologics License Application for UX111, an AAV9 gene therapy for patients with Sanfilippo syndrome Type A, also k...
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
Ultragenyx Wins FDA IND Clearance for UX016 Therapy
Positive
Mar 30, 2026
On March 30, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Administration cleared its Investigational New Drug application for UX016, an investigational small molecule prodrug of sialic acid being developed as a substrate r...
Business Operations and StrategyProduct-Related Announcements
Ultragenyx Announces Positive Phase 3 Results for DTX301
Positive
Mar 12, 2026
On March 12, 2026, Ultragenyx reported positive Phase 3 Enh3ance trial results for DTX301, its investigational AAV8 gene therapy for ornithine transcarbamylase deficiency, showing an 18% reduction in 24-hour plasma ammonia levels versus placebo at...
Product-Related AnnouncementsRegulatory Filings and Compliance
Ultragenyx Gains FDA Priority Review for DTX401 Therapy
Positive
Feb 23, 2026
On February 23, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Administration accepted for review its Biologics License Application for DTX401, an AAV gene therapy, for the treatment of Glycogen Storage Disease Type Ia. The ...
Business Operations and StrategyFinancial Disclosures
Ultragenyx Launches Restructuring Plan to Cut Costs
Negative
Feb 12, 2026
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company developing and commercializing therapies for serious rare and ultra-rare genetic diseases, anchored by products such as Crysvita, Dojolvi, Evkeeza and Mepsevii. The company has a meanin...
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
Ultragenyx Highlights New UX111 Data in FDA Resubmission
Positive
Feb 3, 2026
On February 3, 2026, Ultragenyx Pharmaceutical reported new long-term clinical data for UX111 (rebisufligene etisparvovec), its investigational AAV9 gene therapy for Sanfilippo syndrome type A, showing substantial and durable reductions in cerebro...
Business Operations and StrategyProduct-Related AnnouncementsRegulatory Filings and Compliance
Ultragenyx Resubmits UX111 Gene Therapy for FDA Review
Positive
Jan 30, 2026
On January 30, 2026, Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval for UX111, an AAV9 gene therapy for Sanfilippo syndrome type A, incorporating longer...
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.