Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $2.93B
• Dividend Yield: N/A
• Average Volume (3M): 1.55M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.27
• Revenue Growth: 33.46%
• EPS Growth: 26.72%
• Country: US
• Employees: 1,294
• Sector: Healthcare
• Sector Strength: 39
• Industry: Biotechnology
• EPS (TTM): -1.57
• Shares Outstanding: 94,542,040
• 10 Day Avg. Volume: 2,480,686
• 30 Day Avg. Volume: 1,545,492
• 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: 1.07
• Enterprise Value/Revenue: 1.46
• Enterprise Value/Gross Profit: 6.35
• Enterprise Value/Ebitda: -1.68
• 1Y Price Target: $80.64
• Price Target Upside: 173.26% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 16

• Rating: 50 Neutral
• Price Target: $29.00 ▼(-1.73%Downside)

Ultragenyx Pharmaceutical faces significant financial and technical challenges, including negative profitability and bearish technical indicators. Despite these hurdles, the company shows promising revenue growth and progress in its clinical pipeline, with expectations of future profitability. Recent corporate events present both challenges and opportunities, contributing to a balanced but cautious outlook.

Positive Factors

Earnings

Management has guided towards full year GAAP profitability driven largely by the growth of the current commercial portfolio.

Future Prospects

The rest of the pipeline is effectively free at this point, indicating significant potential upside not yet reflected in the current stock price.

Valuation

The current stock price offers a discounted entry point into the base business, with the entire pipeline effectively being a free addition.

Negative Factors

Clinical Trial Expectations

The setrusumab annualized fracture rate is expected to be worse than in the previous phase due to expected degradation of effect.

Clinical Trial Results

The Ph3 Orbit study for setrusumab did not clear the predetermined p-value threshold, which is seen as an incremental negative.

Market Reaction

Shares of Ultragenyx fell approximately 25% following the announcement of the Ph3 Orbit study results, reflecting market concerns.

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 generates revenue through the sale of its approved therapies for rare diseases. The company's revenue model is primarily based on the commercialization of its products in different markets, with income derived from direct sales as well as collaborations and partnerships with other pharmaceutical firms. Key revenue streams include sales of Crysvita, a therapy for X-linked hypophosphatemia, and Mepsevii, for mucopolysaccharidosis VII. Additionally, Ultragenyx enters into strategic alliances and licensing agreements, which may provide upfront payments, milestone payments, and royalties. These partnerships are critical to expanding the reach of their therapies and enhancing their financial performance.

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
EBITDA	-449.90M	-469.00M	-516.45M	-640.51M	-410.32M	-139.81M
Net Income	-549.58M	-569.18M	-606.64M	-707.42M	-454.02M	-186.57M
Balance Sheet
Total Assets	1.31B	1.50B	1.49B	1.55B	1.52B	1.76B
Cash, Cash Equivalents and Short-Term Investments	494.44M	610.02M	577.21M	896.73M	999.13M	1.21B
Total Debt	38.27M	40.34M	43.17M	31.59M	41.97M	48.23M
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: Negative
• Last Price: 29.51
• 50DMA: Negative
• 100DMA: Negative
• 200DMA: Negative
• MACD: Positive
• 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 29.51 is below the 20-day moving average (MA) of 37.16, below the 50-day MA of 36.59, and below the 200-day MA of 42.70, indicating a bearish trend. The MACD of -0.44 indicates Positive momentum. The RSI at 30.73 is Neutral, neither overbought nor oversold. The STOCH value of 34.97 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	$3.92B	7.46	33.08%	―	91.12%	―
Sector *	64 Neutral	¥340.97B	10.86	-2.99%	2.57%	11.77%	-9.96%
ACLX	58 Neutral	$3.79B	―	-35.56%	―	-41.66%	-180.15%
CRSP	54 Neutral	$5.10B	―	-19.71%	―	-86.13%	-67.29%
ZLAB	53 Neutral	$3.84B	―	-32.05%	―	43.72%	28.63%
RARE	50 Neutral	$2.93B	―	-386.33%	―	33.46%	26.72%

Ultragenyx Pharmaceutical Corporate Events

Ultragenyx Receives FDA Response on UX111 Application

On July 11, 2025, Ultragenyx Pharmaceutical announced that the FDA issued a Complete Response Letter for its Biologics License Application for UX111 gene therapy, intended for treating Sanfilippo syndrome type A. The FDA requested additional information and improvements related to chemistry, manufacturing, and controls, as well as observations from facility inspections. Ultragenyx plans to address these issues and resubmit the application, expecting a six-month review period post-resubmission. The clinical data was acknowledged as robust, with no issues noted, and updated clinical data will be included in the resubmission.

The most recent analyst rating on (RARE) stock is a Buy with a $81.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx Advances Phase 3 Study for UX143 Drug

On July 9, 2025, Ultragenyx Pharmaceutical announced that the Phase 3 portion of the Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta is progressing as planned, with final analysis expected by the end of the year. The Data Monitoring Committee confirmed the drug’s acceptable safety profile, allowing the study to continue, with final analyses to be conducted after 18 months of therapy, indicating potential positive implications for the company’s operations and stakeholders.

The most recent analyst rating on (RARE) stock is a Buy with a $81.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx Receives FDA Breakthrough Therapy Designation

On June 27, 2025, Ultragenyx Pharmaceutical announced that it received Breakthrough Therapy Designation from the FDA for GTX-102 as a treatment for Angelman syndrome. This designation is based on positive data from a Phase 1/2 study showing significant developmental improvements in patients, potentially expediting the drug’s development and review process.

The most recent analyst rating on (RARE) stock is a Buy with a $81.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

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.