Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $3.29B
• Dividend Yield: N/A
• Average Volume (3M): 1.42M
• Price to Earnings (P/E): ―
• Beta (1Y): 0.98
• Revenue Growth: 20.63%
• EPS Growth: 8.09%
• Country: US
• Employees: 1,294
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -1.81
• Shares Outstanding: 96,477,570
• 10 Day Avg. Volume: 1,831,476
• 30 Day Avg. Volume: 1,419,895
• PEG Ratio: 0.28
• Price to Book (P/B): 14.92
• Price to Sales (P/S): 6.80
• P/FCF Ratio: -9.03
• Enterprise Value/Market Cap: 1.04
• Enterprise Value/Revenue: 5.45
• Enterprise Value/Gross Profit: 6.43
• Enterprise Value/Ebitda: -7.11
• 1Y Price Target: $76.31
• Price Target Upside: 123.59% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 14
• EPS Forecast (FY): -5.65
• Revenue Forecast (FY): $655.90M

• Rating: 57 Neutral
• Price Target: $38.00 ▲(11.34% Upside)

Ultragenyx's stock score is primarily influenced by its strong earnings call, which highlighted robust revenue growth and strategic financial moves. However, significant financial performance challenges and valuation issues weigh down the overall score. Positive technical indicators provide some support, reflecting market optimism.

Positive Factors

Revenue Growth

Consistent revenue growth indicates strong market demand and effective product adoption, supporting long-term business sustainability.

Strategic Financial Maneuvers

Securing nondilutive capital strengthens the balance sheet, providing financial flexibility for future growth and development initiatives.

Pipeline Developments

Advancing pipeline with late-stage programs enhances potential for future product launches, driving long-term revenue and market expansion.

Negative Factors

Profitability Challenges

Ongoing net losses highlight profitability challenges, which may strain financial resources and impact long-term sustainability.

Regulatory Challenges

Regulatory setbacks can delay product launches, affecting revenue timelines and increasing costs, impacting strategic goals.

High Leverage

High leverage increases financial risk, potentially limiting investment in growth opportunities and affecting long-term financial health.

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 generates revenue primarily through the sale of its approved therapies, including products like Crysvita (burosumab) and Mepsevii (vestronidase alfa). The company’s revenue model is based on direct product sales to healthcare providers and hospitals, as well as potential royalties from licensing agreements. Notably, Ultragenyx has entered into strategic partnerships with other pharmaceutical companies to enhance its research capabilities and expand its product pipeline, which can lead to milestone payments and shared revenues. Additionally, the company may receive grants and research funding related to its development programs, further contributing to its financial performance.

Ultragenyx Pharmaceutical Financial Statement Overview

Summary

Ultragenyx Pharmaceutical shows revenue growth but faces significant profitability and cash flow challenges. High leverage and negative returns on equity highlight financial risks, despite some improvements in cash flow metrics.

Income Statement

Ultragenyx Pharmaceutical shows consistent revenue growth, with a TTM growth rate of 3.35%. However, the company struggles with profitability, as indicated by negative net profit and EBIT margins. The gross profit margin remains strong at 83.32% TTM, but the high operating losses suggest significant cost challenges.

Balance Sheet

The company's balance sheet reflects high leverage with a TTM debt-to-equity ratio of 1.33, although this is an improvement from the previous year. The return on equity is negative, indicating that the company is not generating profits from its equity base. The equity ratio is not provided, but the overall financial health appears strained.

Cash Flow

Ultragenyx's cash flow statement reveals negative operating and free cash flows, although there is a slight improvement in free cash flow growth. The operating cash flow to net income ratio is negative, indicating cash flow challenges. The free cash flow to net income ratio is slightly above 1, suggesting some efficiency in converting income to cash.

Breakdown	TTM	Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement
Total Revenue	630.60M	560.23M	434.25M	363.33M	351.41M	271.03M
Gross Profit	534.05M	483.50M	389.04M	335.01M	335.40M	264.90M
EBITDA	-483.37M	-469.00M	-516.45M	-640.49M	-410.32M	-139.81M
Net Income	-579.83M	-569.18M	-606.64M	-707.42M	-454.02M	-186.57M
Balance Sheet
Total Assets	1.19B	1.50B	1.49B	1.55B	1.52B	1.76B
Cash, Cash Equivalents and Short-Term Investments	425.25M	610.02M	577.21M	747.76M	740.20M	1.20B
Total Debt	863.11M	40.34M	43.17M	31.59M	41.97M	48.23M
Total Liabilities	1.17B	1.24B	1.22B	1.19B	599.84M	605.18M
Stockholders Equity	9.16M	255.30M	275.41M	352.49M	922.56M	1.15B
Cash Flow
Free Cash Flow	-451.39M	-421.68M	-521.57M	-526.59M	-411.79M	-176.13M
Operating Cash Flow	-445.67M	-414.19M	-474.81M	-380.46M	-338.69M	-132.22M
Investing Cash Flow	410.98M	-17.77M	168.00M	-291.65M	-195.37M	-179.12M
Financing Cash Flow	86.99M	399.24M	388.14M	501.21M	118.55M	600.27M

Ultragenyx Pharmaceutical Technical Analysis

• Technical Analysis Sentiment: Positive
• Last Price: 34.13
• 50DMA: Positive
• 100DMA: Positive
• 200DMA: Positive
• 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 Positive. The current price of 34.13 is below the 20-day moving average (MA) of 34.97, above the 50-day MA of 33.90, and above the 200-day MA of 33.52, indicating a neutral trend. The MACD of 0.17 indicates Positive momentum. The RSI at 51.44 is Neutral, neither overbought nor oversold. The STOCH value of 44.27 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Positive sentiment for RARE.

Ultragenyx Pharmaceutical Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
TARS	60 Neutral	$3.49B	-40.79	-28.35%	―	182.44%	47.21%
RARE	57 Neutral	$3.29B	-5.75	-325.76%	―	20.63%	8.09%
TVTX	56 Neutral	$3.16B	-32.60	-410.78%	―	114.22%	77.02%
MLYS	53 Neutral	$2.96B	-12.63	-42.14%	―	―	9.60%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
XENE	48 Neutral	$3.44B	-11.47	-45.14%	―	―	-38.30%
IRON	43 Neutral	$3.01B	-14.90	-34.78%	―	―	-34.96%

Ultragenyx Pharmaceutical Corporate Events

Ultragenyx Signs $400M Royalty Purchase Agreement

On November 3, 2025, Ultragenyx Pharmaceutical entered into a Royalty Purchase Agreement with OCM LS23 Holdings LP, an investment vehicle of OMERS, for $400 million in cash. This agreement grants OMERS the right to receive a portion of future royalty payments from Crysvita® sales in the U.S. and Canada, with payments starting in January 2028. The deal aims to bolster Ultragenyx’s balance sheet ahead of pivotal milestones, including multiple late-stage data readouts and regulatory submissions. The company reported a 15% revenue growth for the third quarter of 2025 and reaffirmed its 2025 revenue guidance, expecting total revenues between $640 million to $670 million.

Ultragenyx Begins Dosing in Aurora Study for Angelman Syndrome

On October 30, 2025, Ultragenyx Pharmaceutical announced the dosing of the first patient in the Aurora study, which evaluates the efficacy and safety of GTX-102 for Angelman syndrome across a broader patient population. The Aurora study aims to include approximately 60 participants with various genotypes and age ranges, expanding beyond the Phase 3 Aspire study’s focus on patients with full maternal UBE3A gene deletion. This development signifies a strategic expansion in Ultragenyx’s clinical research efforts, potentially enhancing its market position and offering new treatment options for Angelman syndrome patients.