Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $3.37B
• Dividend Yield: N/A
• Average Volume (3M): 950.09K
• Price to Earnings (P/E): ―
• Beta (1Y): 1.15
• Revenue Growth: 33.46%
• EPS Growth: 26.72%
• Country: US
• Employees: 1,294
• Sector: Healthcare
• Sector Strength: 42
• Industry: Biotechnology
• EPS (TTM): -1.57
• Shares Outstanding: 94,542,040
• 10 Day Avg. Volume: 740,419
• 30 Day Avg. Volume: 950,086
• PEG Ratio: -7.78
• Price to Book (P/B): 13.41
• Price to Sales (P/S): 0.00
• P/FCF Ratio: -8.06
• Enterprise Value/Market Cap: N/A
• Enterprise Value/Revenue: N/A
• Enterprise Value/Gross Profit: N/A
• Enterprise Value/Ebitda: N/A
• 1Y Price Target: $91.40
• Price Target Upside: 156.31% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 15

• Rating: 43 Neutral
• Price Target: $34.00 ▼( -4.66% Downside)

Ultragenyx Pharmaceutical shows strong revenue growth and promising clinical development progress, but these are offset by significant financial challenges, including ongoing losses and cash flow issues. The technical indicators suggest a bearish trend, and valuation metrics highlight profitability hurdles. The guidance from the earnings call provides some optimism for long-term profitability, but near-term risks remain high.

Positive Factors

Financial Performance

Ultragenyx reported total net product revenue of $139.3M, meeting expectations and reflecting steady business performance.

Product Pipeline

The company is considered a top pick primarily due to positive views on setrusumab and other promising pipeline opportunities.

Regulatory Approval

Management is encouraged by FDA Commissioner Marty Makary's supportive comments on rare disease approvals and ways to improve and accelerate the process.

Negative Factors

Industry Risks

RARE is susceptible to the standard risks that apply to the entire biotech industry, including development, regulatory, commercial, manufacturing, financing, and IP pitfalls.

Valuation Concerns

The stock is already at a well-described floor valuation.

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 internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.

How the Company Makes Money

Ultragenyx Pharmaceutical makes money primarily through the sale of its pharmaceutical products, which are developed to treat rare and ultra-rare genetic diseases. The company's revenue model is centered around the commercial launch of its approved therapies, which includes direct sales to healthcare providers and institutions. Additionally, Ultragenyx enters into strategic partnerships and licensing agreements to expand its research, development, and commercialization capabilities. These partnerships can provide milestone payments, royalties, and other financial incentives that contribute to its earnings. Key factors influencing the company's revenue streams include the successful clinical development of its pipeline products, regulatory approvals, and the ability to secure reimbursement from health insurance providers.

Ultragenyx Pharmaceutical Financial Statement Overview

Summary

Ultragenyx Pharmaceutical demonstrates strong revenue growth and a robust gross profit margin. However, the company faces substantial profitability and cash flow challenges, with ongoing net losses and negative free cash flow, despite a low debt-to-equity ratio.

Income Statement

Ultragenyx Pharmaceutical shows a consistent increase in revenue over the years, with a significant revenue growth rate of 29% from 2022 to 2023. However, the company continues to face substantial losses, as indicated by negative EBIT and net income margins. The gross profit margin remains strong, but high operating expenses contribute to persistent net losses.

Balance Sheet

The company maintains a relatively low debt-to-equity ratio, indicating prudent use of leverage. However, the declining stockholders' equity and a lower equity ratio suggest potential risks in financial stability. The return on equity remains negative due to ongoing net losses, which could affect long-term shareholder value.

Cash Flow

Ultragenyx has a negative free cash flow, although there is an improvement in free cash flow from 2023 to 2024. The operating cash flow to net income ratio indicates operational cash flow challenges. Despite positive financing cash flows, sustained negative operating cash flows highlight the need for enhanced operational efficiencies.

Breakdown
Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement		Total Revenue
560.23M	434.25M	363.33M	351.41M	271.03M
	Gross Profit
483.50M	389.04M	335.01M	335.40M	264.90M
	EBIT
-535.97M	-569.21M	-648.92M	-381.74M	-330.12M
	EBITDA
-469.00M	-516.45M	-640.51M	-410.32M	-139.81M
	Net Income Common Stockholders
-569.18M	-606.64M	-707.42M	-454.02M	-186.57M
Balance Sheet		Cash, Cash Equivalents and Short-Term Investments
610.02M	577.21M	896.73M	999.13M	1.21B
	Total Assets
1.50B	1.49B	1.55B	1.52B	1.76B
	Total Debt
10.30M	43.17M	31.59M	41.97M	48.23M
	Net Debt
-163.43M	-170.41M	-101.35M	-265.61M	-665.30M
	Total Liabilities
1.24B	1.22B	1.19B	599.84M	605.18M
	Stockholders Equity
255.30M	275.41M	352.49M	922.56M	1.15B
Cash Flow		Free Cash Flow
-421.68M	-521.57M	-526.59M	-411.79M	-176.13M
	Operating Cash Flow
-414.19M	-474.81M	-380.46M	-338.69M	-132.22M
	Investing Cash Flow
-17.77M	168.00M	-291.65M	-195.37M	-179.12M
	Financing Cash Flow
399.24M	388.14M	501.21M	118.55M	600.27M

Ultragenyx Pharmaceutical Technical Analysis

• Technical Analysis Sentiment: Negative
• Last Price: 35.66
• 50DMA: Negative
• 100DMA: Negative
• 200DMA: Negative
• MACD: Negative
• RSI: Neutral
• STOCH: 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 35.66 is below the 20-day moving average (MA) of 36.43, below the 50-day MA of 36.39, and below the 200-day MA of 45.68, indicating a bearish trend. The MACD of -0.28 indicates Negative momentum. The RSI at 47.71 is Neutral, neither overbought nor oversold. The STOCH value of 38.22 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for RARE.

Ultragenyx Pharmaceutical Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
PTCT	69 Neutral	$3.64B	7.05	33.08%	―	91.12%	―
ACLX	55 Neutral	$3.59B	―	-35.56%	―	-41.66%	-180.15%
SWTX	54 Neutral	$3.49B	―	-51.57%	―	730.42%	31.63%
Sector *	53 Neutral	$5.14B	3.03	-43.89%	2.83%	16.75%	-0.06%
ZLAB	52 Neutral	$3.54B	―	-32.05%	―	43.72%	28.63%
CRSP	50 Neutral	$3.18B	―	-19.71%	―	-86.13%	-67.29%
RARE	43 Neutral	$3.37B	―	-386.33%	―	33.46%	26.72%

Ultragenyx Pharmaceutical Corporate Events

Ultragenyx Pharmaceutical Approves Key Proposals at Annual Meeting

On May 15, 2025, Ultragenyx Pharmaceutical held its Annual Meeting of Stockholders where several key proposals were approved. The stockholders elected Class III directors, approved the Second Amended and Restated 2023 Incentive Plan, ratified Ernst & Young LLP as the independent accounting firm for 2025, and supported the executive compensation plan. These decisions reflect the company’s strategic direction and governance priorities, potentially impacting its operational focus and stakeholder confidence.

The most recent analyst rating on (RARE) stock is a Buy with a $83.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.