Ultragenyx Pharmaceutical, Inc. (RARE)
NASDAQ:RARE
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Ultragenyx Pharmaceutical
(NASDAQ:RARE)
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Rating:50Neutral
Price Target:
$25.00
▲(0.77% Upside)
Action:Reiterated
Date:05/19/26
The score is held back primarily by weak financials—deep ongoing losses, significant cash burn, negative equity, and substantial debt. This is partially offset by a constructive earnings-call outlook with reaffirmed growth guidance, cost-reduction plans, and multiple near-term regulatory catalysts, while technicals are neutral-to-modestly improving and valuation remains constrained by negative earnings and no dividend.
Positive Factors
Strong product economics (high gross margin)
Sustained ~84% gross margins reflect durable pricing power in orphan drugs and efficient cost of goods. This high margin base provides operating leverage as revenue scales, meaning incremental sales can meaningfully improve profitability if SG&A/R&D discipline continues.
Negative Factors
Weakened balance sheet and elevated leverage
Negative equity and roughly $1.2B of debt materially limit financial flexibility. Elevated leverage raises refinancing and covenant risk, constrains capital allocation choices, and makes the company more dependent on successful operational execution or external financing to support growth and launches.
Read all positive and negative factors
Positive Factors
Negative Factors
Strong product economics (high gross margin)
Sustained ~84% gross margins reflect durable pricing power in orphan drugs and efficient cost of goods. This high margin base provides operating leverage as revenue scales, meaning incremental sales can meaningfully improve profitability if SG&A/R&D discipline continues.
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.
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The Fly
The FlyUltragenyx Pharmaceutical (RARE) vs. SPDR S&P 500 ETF (SPY)
Market Cap
$2.16B
Dividend YieldN/A
Average Volume (3M)1.67M
Price to Earnings (P/E)―
Beta (1Y)1.29
Revenue Growth13.33%
EPS Growth-3.82%
CountryUS
Employees1,294
SectorHealthcare
Sector Strength45
IndustryBiotechnology
Share Statistics
EPS (TTM)-6.11
Shares Outstanding98,489,230
10 Day Avg. Volume1,582,213
30 Day Avg. Volume1,671,200
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 Cap1.57
Enterprise Value/Revenue5.05
Enterprise Value/Gross Profit6.05
Enterprise Value/Ebitda-6.58
Forecast
1Y Price Target
$51.87Price Target Upside109.06% Upside
Rating ConsensusStrong Buy
Number of Analyst Covering16
EPS Forecast (FY)-4.51
Revenue Forecast (FY)$741.44M
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 Earnings Call Summary
Earnings Call Date:May 05, 2026
(Q1-2026)
| % Change Since: |
Next Earnings Date:Jul 30, 2026
Earnings Call Sentiment Positive
The call balances strong commercial execution, clear revenue guidance (reaffirmed $730M–$760M for 2026), rapid pipeline progress (two near-term PDUFAs and promising long-term GTX-102 data with Bayley-4 and MDRI signals), and manufacturing/launch readiness against meaningful near-term financial pressure (Q1 net loss of $185M, $197M cash used in operations, high operating expenses and restructuring charges). Management presented a credible path to profitability in 2027 with planned R&D/SG&A reductions and assumed PRV monetization, but timing uncertainty for Phase 3 readouts, quarter-to-quarter ordering variability and execution risks around launches and PRV realization remain. Overall the positives around product performance, global commercialization scale, and upcoming catalysts outweigh the near-term financial and execution risks.Positive Updates
Total Revenue and Product Contributions (Q1 2026)
Total revenue of $136 million in Q1 2026. Product contributions: Crysvita $93M (North America $39M, Latin America & Turkey $46M, Europe $8M), Dojolvi $18M, Evkeeza $18M, Mepsevii $7M.
Negative Updates
Significant Q1 Operating Loss and Cash Burn
Net loss of $185M (or $1.84 per share) in Q1 2026. Net cash used in operations of $197M for the quarter, resulting in a cash balance of $534M — highlighting cash burn and the importance of future launches/PRV monetization for runway.
Read all updates
Q1-2026 Updates
Positive
Negative
Total Revenue and Product Contributions (Q1 2026)
Total revenue of $136 million in Q1 2026. Product contributions: Crysvita $93M (North America $39M, Latin America & Turkey $46M, Europe $8M), Dojolvi $18M, Evkeeza $18M, Mepsevii $7M.
Read all positive updates
Company Guidance
On the call management reaffirmed 2026 revenue guidance of $730–760 million (8%–13% growth vs. 2025), excluding potential launch revenue, and reiterated a path to profitability in 2027; Crysvita revenue guidance is $500–520 million and Dojolvi $100–110 million for 2026, while combined R&D and SG&A are expected to be flat to down low single digits vs. 2025 (with a targeted ≥15% reduction vs. 2025 in 2027). Q1 results included total revenue of $136 million (Crysvita $93M — NA $39M, Latin America & Turkey $46M, Europe $8M; Dojolvi $18M; Evkeeza $18M, +64% YoY; Mepsevii $7M), total operating expenses of $305M (cost of sales $30M; R&D+SG&A $275M) which included $30M stock‑based comp and $30M restructuring charges, net loss of $185M or $1.84 per share, cash and equivalents of $534M as of March 31, and net cash used in operations of $197M (including $38M for UX143 manufacturing and $5M severance) with operating cash use expected to decline in later quarters. They plan to monetize two PRVs modeled at a little over $100M each (with upside potential), target PDUFA dates for DTX401 on Aug 23, 2026 and UX111 on Sept 19, 2026, and highlighted commercial scale (sales in >35 countries) and patient counts (Crysvita >950 patients in LATAM with ~30 starts in Q1; Dojolvi >675 reimbursed in NA and ~300 in Europe; Evkeeza ~370 patients across 18 countries).Ultragenyx Pharmaceutical Financial Statement Overview
Summary
Income Statement
24
Negative
Balance Sheet
18
Very Negative
Cash Flow
22
Negative
| Breakdown | TTM | Dec 2025 | Dec 2024 | Dec 2023 | Dec 2022 | Dec 2021 |
|---|---|---|---|---|---|---|
Income Statement | ||||||
| Total Revenue | 669.43M | 673.00M | 560.23M | 434.25M | 363.33M | 351.41M |
| Gross Profit | 559.44M | 564.00M | 483.50M | 389.04M | 335.01M | 335.40M |
| EBITDA | -514.35M | -500.00M | -469.00M | -516.45M | -640.49M | -410.32M |
| Net Income | -609.36M | -575.00M | -569.18M | -606.64M | -707.42M | -454.02M |
Balance Sheet | ||||||
| Total Assets | 1.30B | 1.53B | 1.50B | 1.49B | 1.55B | 1.52B |
| Cash, Cash Equivalents and Short-Term Investments | 413.00M | 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 | 1.52B | 1.60B | 1.24B | 1.22B | 1.19B | 599.84M |
| Stockholders Equity | -236.00M | -80.00M | 255.30M | 275.41M | 352.49M | 922.56M |
Cash Flow | ||||||
| Free Cash Flow | -487.20M | -472.00M | -421.68M | -521.57M | -526.59M | -411.79M |
| Operating Cash Flow | -496.52M | -466.00M | -414.19M | -474.81M | -380.46M | -338.69M |
| Investing Cash Flow | 67.11M | 236.00M | -17.77M | 168.00M | -291.65M | -195.37M |
| Financing Cash Flow | 477.84M | 478.00M | 399.24M | 388.14M | 501.21M | 118.55M |
Ultragenyx Pharmaceutical Technical Analysis
Negative
24.81
Price Trends
23.77
Negative
23.19
Negative
27.28
Negative
Market Momentum
-0.52
Positive
40.10
Neutral
43.60
Neutral
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 24.81 is above the 20-day moving average (MA) of 23.98, above the 50-day MA of 23.77, and below the 200-day MA of 27.28, indicating a bearish trend. The MACD of -0.52 indicates Positive momentum. The RSI at 40.10 is Neutral, neither overbought nor oversold. The STOCH value of 43.60 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.
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 | $4.26B | -196.46 | -26.79% | ― | 96.03% | 82.27% | |
61 Neutral | $5.00B | -11.79 | -49.23% | ― | -100.00% | -46.29% | |
58 Neutral | $2.55B | -52.76 | -14.19% | ― | 128.99% | 58.46% | |
55 Neutral | $1.95B | -12.93 | -27.92% | ― | ― | 44.87% | |
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
50 Neutral | $2.16B | -3.62 | 1566.94% | ― | 13.33% | -3.82% | |
47 Neutral | $2.56B | -10.67 | -36.95% | ― | ― | -68.57% |
* Healthcare Sector Average
RARE
Ultragenyx Pharmaceutical
21.91
-15.13
-40.85%
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Ultragenyx Pharmaceutical Corporate Events
Business Operations and StrategyExecutive/Board ChangesShareholder Meetings
Ultragenyx Shareholders Approve Incentive Plan, Elect Directors
Positive
May 18, 2026
At its Annual Meeting of Stockholders held on May 14, 2026, Ultragenyx Pharmaceutical shareholders approved the Third Amended and Restated 2023 Incentive Plan, reinforcing the company’s long-term equity-based compensation framework for emplo...
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 ...
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.