Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $2.69B
• Dividend Yield: N/A
• Average Volume (3M): 2.49M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.24
• Revenue Growth: 33.46%
• EPS Growth: 26.72%
• Country: US
• Employees: 1,294
• Sector: Healthcare
• Sector Strength: 38
• Industry: Biotechnology
• EPS (TTM): -1.57
• Shares Outstanding: 94,542,040
• 10 Day Avg. Volume: 2,593,394
• 30 Day Avg. Volume: 2,491,849
• PEG Ratio: -0.65
• Price to Book (P/B): 17.94
• Price to Sales (P/S): 0.00
• P/FCF Ratio: -6.15
• Enterprise Value/Market Cap: 0.92
• Enterprise Value/Revenue: 1.15
• Enterprise Value/Gross Profit: 5.03
• Enterprise Value/Ebitda: -1.33
• 1Y Price Target: $84.07
• Price Target Upside: 195.60% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 17

• Rating: 48 Neutral
• Price Target: $27.00 ▼(-5.06%Downside)

Ultragenyx Pharmaceutical faces significant financial challenges, including profitability and cash flow issues, which heavily impact the stock score. Technical indicators suggest bearish momentum, further weighing on the score. Although the earnings call provided optimistic guidance and the company has notable clinical advancements, these positives are currently overshadowed by financial and technical weaknesses.

Positive Factors

Earnings Potential

Ultragenyx's flagship CRYSVITA franchise is believed to be capable of achieving peak annual sales totaling over $3 billion.

Financial Valuation

The company's current commercial portfolio alone should support fair value in the roughly $35-40 per share range.

Pipeline Development

Analyst reiterates Buy rating with $79 price objective as there is strong opportunity for other pipeline programs.

Negative Factors

Clinical Uncertainty

Setrusumab's probability of success has been lowered to 55% due to increased clinical uncertainty.

Market Delay

The UX111 launch in the US and EU has been delayed to 2027 and 2028, respectively, due to regulatory issues.

Regulatory Issues

The FDA has issued a Complete Response Letter for UX111, which does not help the recent negative sentiment on the company.

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 consistent revenue growth but faces significant profitability challenges, with a negative net profit margin and high operating expenses. The balance sheet reflects moderate leverage but a declining equity base. Cash flow is under pressure with negative free cash flow, necessitating external financing.

Income Statement

Ultragenyx Pharmaceutical has shown consistent revenue growth, with a TTM revenue increase of 5.44% compared to the previous year. However, the company is facing significant profitability challenges, as indicated by negative EBIT and net profit margins. The TTM net profit margin is -93.05%, reflecting substantial losses. The gross profit margin remains strong at 83.64%, suggesting efficient cost management at the production level, but the overall financial performance is hindered by high operating expenses.

Balance Sheet

The company's balance sheet shows a high debt-to-equity ratio of 0.27, indicating moderate leverage. The equity ratio has decreased to 11.00%, suggesting a declining proportion of equity financing. Return on equity is negative at -381.06%, reflecting ongoing losses. The financial stability is concerning due to the shrinking equity base and persistent losses, although the company maintains a reasonable level of cash and short-term investments.

Cash Flow

Ultragenyx Pharmaceutical's cash flow statement reveals a challenging cash position, with negative free cash flow and operating cash flow. The TTM free cash flow to net income ratio is 0.75, indicating that cash outflows exceed net income losses. Despite negative cash flows, the company has managed to maintain liquidity through financing activities. The operating cash flow to net income ratio is 0.71, showing that cash losses are slightly less severe than accounting losses.

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
EBITDA	-469.00M	-516.45M	-640.51M	-410.32M	-139.81M
Net Income	-569.18M	-606.64M	-707.42M	-454.02M	-186.57M
Balance Sheet
Total Assets	1.50B	1.49B	1.55B	1.52B	1.76B
Cash, Cash Equivalents and Short-Term Investments	610.02M	577.21M	896.73M	999.13M	1.21B
Total Debt	40.34M	43.17M	31.59M	41.97M	48.23M
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: 28.44
• 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 28.44 is below the 20-day moving average (MA) of 29.61, below the 50-day MA of 33.89, and below the 200-day MA of 40.63, indicating a bearish trend. The MACD of -2.14 indicates Negative momentum. The RSI at 40.47 is Neutral, neither overbought nor oversold. The STOCH value of 67.21 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	74 Outperform	$4.13B	8.00	33.08%	―	91.12%	―
SWTX	61 Neutral	$3.54B	―	-51.57%	―	730.42%	31.63%
CRSP	58 Neutral	$4.84B	―	-19.71%	―	-86.13%	-67.29%
ACLX	58 Neutral	$3.92B	―	-35.56%	―	-41.66%	-180.15%
ZLAB	52 Neutral	$4.08B	―	-32.01%	―	43.59%	28.72%
Sector *	51 Neutral	$7.35B	0.50	-65.61%	2.47%	15.28%	1.44%
RARE	48 Neutral	$2.69B	―	-386.33%	―	33.46%	26.72%

Ultragenyx Pharmaceutical Corporate Events

Ultragenyx Completes Enrollment for GTX-102 Phase 3 Study

On July 31, 2025, Ultragenyx Pharmaceutical announced that the Phase 3 Aspire study for GTX-102, a treatment for Angelman Syndrome, is fully enrolled with 129 participants. The study, which began in December 2024, involves a 48-week trial comparing GTX-102 treatment to a sham comparator group, with primary and secondary endpoints focusing on cognitive improvements and multi-domain responses. Completion is expected in the second half of 2026, with plans to provide topline data and progress to regulatory submission.

The most recent analyst rating on (RARE) stock is a Buy with a $61.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

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.