Ultragenyx Pharmaceutical Inc. (RARE) AI Stock Analysis

NASDAQ: RAREAI research summary: business, growth, valuation, analysts, bull and bear case.

RARE AI stock analysis summary

AI Research

Ultragenyx Pharmaceutical Inc. (RARE) is a commercial-stage biopharmaceutical company focused on treatments for rare and ultra-rare genetic diseases. Revenue expanded at a 19.9% 5-year CAGR to $673.0M in FY2025, but the company remained unprofitable with a net loss of -$575.0M. With total debt of $1.20B and negative shareholders' equity of -$291.0M as of June 30, 2026, the company continues to rely on external capital to support its operations.

Market cap
$1.43B
Revenue (TTM)
$717.0M
P/E
—
1Y return
-57.2%
S&P 500 16.8%
Avg. target
$26.25
80.5% vs last close

Bull case

  • Revenue expanded at a 19.9% 5-year CAGR through FY2025 and increased 28.1% year-over-year in the quarter ended June 30, 2026.
  • Six analysts project the company will achieve positive EPS of $0.23 alongside $989.3M in revenue for the next fiscal year.
  • The consensus mean price target of $26.25 indicates 80.5% upside potential from the last close of $14.54.

Bear case

  • Ultragenyx posted a net loss of -$575.0M and free cash flow of -$472.0M in FY2025.
  • As of June 30, 2026, the company holds $1.20B in total debt against $292.0M in cash, leading to negative shareholders' equity of -$291.0M.
  • The stock declined 57.2% over the trailing one-year period while the S&P 500 gained 16.8%.

What Ultragenyx Pharmaceutical does

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on identifying, acquiring, developing, and commercializing therapies for rare and ultra-rare genetic diseases across North America, Europe, and other regions. Its commercialized biologic portfolio includes Crysvita for X-linked hypophosphatemia and tumor-induced osteomalacia, Mepsevii for Mucopolysaccharidosis VII, Dojolvi for long-chain fatty acid oxidation disorders, and Evkeeza for homozygous familial hypercholesterolemia. The company is also advancing clinical pipeline candidates including gene therapies DTX401 and DTX301, monoclonal antibody UX143, antisense oligonucleotide GTX-102, as well as UX701 and UX053.

Growth and profitability

Ultragenyx grew annual revenue from $351.4M in FY2021 to $673.0M in FY2025, reflecting a 5-year revenue CAGR of 19.9%. Over that timeframe, gross margin compressed from 95.4% in FY2021 to 83.8% in FY2025. Operating margins improved from -108.6% in FY2021 to -79.5% in FY2025 as research and development expenses reached $750.0M. For FY2025, net income was -$575.0M, or -$5.84 per share. Trailing twelve month revenue reached $717.0M through June 30, 2026, with quarterly revenue growing 28.1% year-over-year. TTM operating margin stood at -35.0% and profit margin was -81.7%, resulting in a TTM diluted EPS of -$5.82.

RARE income statement →

Balance sheet and cash flow

As of June 30, 2026, Ultragenyx held $292.0M in cash and short-term investments against $1.20B in total debt, leaving net debt at $910.0M. The company operates with negative shareholders' equity of -$291.0M, though its current ratio was 1.73 on total assets of $1.26B. Cash flow from operations was -$466.0M in FY2025, and free cash flow was -$472.0M after $6.0M of capital expenditures. Over the past five years, the company's share count increased 48.6%.

RARE balance sheet →

Valuation and performance

Ultragenyx trades at a price-to-sales ratio of 2.0x and an enterprise-value-to-revenue ratio of 1.1x, below the median of its US-listed Healthcare peers at 4.1x price-to-sales. Its price-to-book ratio is 19.7x. The company's market capitalization is $1.43B, while enterprise value stands at $824.0M. Over the past year, RARE shares fell 57.2%, compared to a 16.8% gain for the S&P 500 (SPY). Over five years, the stock dropped 81.8% while the S&P 500 advanced 83.0%.

RARE key metrics →

What analysts expect

Wall Street analysts maintain 8 Strong Buy, 2 Buy, and 11 Hold ratings on RARE, with no Sell or Strong Sell ratings. The mean price target is $26.25, representing an 80.5% upside from the last close of $14.54. For FY2026, consensus estimates project an EPS of -$4.08 on revenue of $759.6M. For the next fiscal year, six analysts project positive EPS of $0.23 on $989.3M in revenue. Ultragenyx beat EPS estimates in 1 of the last 4 quarters, with its next earnings report scheduled for November 3, 2026.

RARE forecast and price target →

Key risks and what to watch

  • Shareholder dilution has been significant, as the share count increased 48.6% over the past 5 years to fund ongoing operations.
  • Short interest is elevated, with short percent of float standing at 21.3%.
  • Ultragenyx has struggled to meet earnings targets consistently, topping EPS estimates in only 1 of the last 4 quarters.
  • Substantial research and development expenses of $750.0M in FY2025 continue to drive operating losses and cash burn.

AI-generated research summary. Written with AI from Quotelinks data as of October 16, 2026 and checked so every figure matches that data. For information only, not investment advice. Disclaimer

About this RARE AI stock analysis

This page is an AI-written research summary of Ultragenyx Pharmaceutical Inc. (RARE). The text was generated on October 30, 2026 from Quotelinks data as of October 16, 2026 (company profile, annual results, latest balance sheet, valuation, price performance and analyst estimates). An automatic check rejects any draft that contains a figure not found in that data. It is not investment advice; verify figures on the linked tabs before making decisions.

Related: RARE income statement, RARE balance sheet, RARE key metrics, RARE analyst forecast, RARE chart. See the RARE stock overview for the latest quote and news.

RARE AI stock analysis FAQ

What products does Ultragenyx Pharmaceutical commercialize?

Ultragenyx offers approved commercial biologic treatments including Crysvita, Mepsevii, Dojolvi, and Evkeeza for various rare genetic diseases.

When is Ultragenyx's next earnings report?

The company's next earnings report is scheduled for November 3, 2026.

Is Ultragenyx currently profitable?

No, Ultragenyx generated a net loss of -$575.0M in FY2025 and reported a trailing twelve month net profit margin of -81.7%.

How much debt does Ultragenyx carry?

As of June 30, 2026, Ultragenyx had $1.20B in total debt and $292.0M in cash and short-term investments, resulting in net debt of $910.0M.

Does Ultragenyx pay a dividend?

No, Ultragenyx does not pay a dividend.

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