Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $3.55B
• Dividend Yield: N/A
• Average Volume (3M): 1.02M
• 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: 58
• Industry: Biotechnology
• EPS (TTM): -1.57
• Shares Outstanding: 94,542,040
• 10 Day Avg. Volume: 1,183,032
• 30 Day Avg. Volume: 1,020,377
• PEG Ratio: -0.09
• Price to Book (P/B): 9.19
• Price to Sales (P/S): 0.00
• P/FCF Ratio: -6.15
• Enterprise Value/Market Cap: 0.88
• Enterprise Value/Revenue: 1.46
• Enterprise Value/Gross Profit: 6.35
• Enterprise Value/Ebitda: -1.68
• 1Y Price Target: $87.87
• Price Target Upside: 134.38% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 15

• Rating: 46 Neutral
• Price Target: $35.00 ▼(-6.64%Downside)

The overall stock score of 46 reflects persistent financial challenges, notably poor profitability and cash flow issues, despite promising revenue growth and clinical advancements. Technical indicators suggest a bearish momentum, and valuation metrics are unattractive. However, positive aspects from the earnings call, including revenue growth and strategic milestones, provide some balance to the otherwise cautious outlook.

Positive Factors

Clinical Trials

The phase 3 Orbit study evaluating setrusumab in OI patients is designed with interim looks that could allow for early approval, increasing the potential for success.

Product Pipeline

The current valuation of RARE assigns minimal value to the pipeline, creating an attractive risk/reward profile.

Revenue Growth

The company has commercialized four products, which in aggregate generated $560 million in revenue in 2024, up 29% year-over-year.

Negative Factors

Biotech 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.

Macroeconomic Factors

RARE shares have faced significant downward pressure largely due to macro headwinds.

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 shows substantial revenue growth, but this is heavily outweighed by significant operational challenges, including high negative net profit and EBIT margins. The consistent operating losses have eroded shareholder equity and placed financial stability at risk, despite low debt levels.

Income Statement

Ultragenyx Pharmaceutical shows a significant revenue growth rate of 8.24% in TTM, indicating a positive trajectory, but is hindered by negative net profit margins of -93.03% and EBIT margins of -86.92%, reflecting ongoing operational challenges. The company has been consistently operating at a loss, which impacts its financial health negatively.

Balance Sheet

The debt-to-equity ratio stands at 0.27 in TTM, indicating low financial leverage, but the equity ratio has decreased to 10.99%, indicating reduced shareholder equity in the asset base. Consistent net losses have eroded equity, pushing the return on equity (ROE) further into negative territory at -381.06%.

Cash Flow

The company has a troubling free cash flow growth rate of 2.04% in TTM, reflecting minor improvement, yet free cash flow remains negative at -$413 million. High negative operating cash flow to net income ratio of 0.71 indicates cash flow challenges, yet the financing activities provide a necessary cash influx.

Breakdown
TTM	Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement		Total Revenue
590.69M	560.23M	434.25M	363.33M	351.41M	271.03M
	Gross Profit
493.97M	483.50M	389.04M	335.01M	335.40M	264.90M
	EBIT
-513.57M	-535.97M	-569.21M	-648.92M	-381.74M	-330.12M
	EBITDA
-449.90M	-469.00M	-516.45M	-640.51M	-410.32M	-139.81M
	Net Income Common Stockholders
-549.58M	-569.18M	-606.64M	-707.42M	-454.02M	-186.57M
Balance Sheet		Cash, Cash Equivalents and Short-Term Investments
494.44M	610.02M	577.21M	896.73M	999.13M	1.21B
	Total Assets
1.31B	1.50B	1.49B	1.55B	1.52B	1.76B
	Total Debt
38.27M	40.34M	43.17M	31.59M	41.97M	48.23M
	Net Debt
-88.78M	-133.39M	-170.41M	-101.35M	-265.61M	-665.30M
	Total Liabilities
1.16B	1.24B	1.22B	1.19B	599.84M	605.18M
	Stockholders Equity
144.25M	255.30M	275.41M	352.49M	922.56M	1.15B
Cash Flow		Free Cash Flow
-413.04M	-421.68M	-521.57M	-526.59M	-411.79M	-176.13M
	Operating Cash Flow
-389.94M	-414.19M	-474.81M	-380.46M	-338.69M	-132.22M
	Investing Cash Flow
7.69M	-17.77M	168.00M	-291.65M	-195.37M	-179.12M
	Financing Cash Flow
399.46M	399.24M	388.14M	501.21M	118.55M	600.27M

Ultragenyx Pharmaceutical Technical Analysis

• Technical Analysis Sentiment: Neutral
• Last Price: 37.49
• 50DMA: Positive
• 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 Neutral. The current price of 37.49 is above the 20-day moving average (MA) of 36.31, above the 50-day MA of 35.93, and below the 200-day MA of 44.52, indicating a neutral trend. The MACD of 0.48 indicates Negative momentum. The RSI at 54.29 is Neutral, neither overbought nor oversold. The STOCH value of 68.59 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Neutral sentiment for RARE.

Ultragenyx Pharmaceutical Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
PTCT	70 Outperform	$4.03B	7.83	33.08%	―	91.12%	―
CRSP	60 Neutral	$3.59B	―	-19.71%	―	-86.13%	-67.29%
ZLAB	59 Neutral	$4.85B	―	-32.05%	―	43.72%	28.63%
ACLX	58 Neutral	$3.67B	―	-35.56%	―	-41.66%	-180.15%
SWTX	54 Neutral	$3.52B	―	-51.57%	―	730.42%	31.63%
Sector *	54 Neutral	$5.41B	3.27	-45.10%	3.29%	16.81%	0.02%
RARE	46 Neutral	$3.55B	―	-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.