Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $3.19B
• Dividend Yield: N/A
• Average Volume (3M): 1.38M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.10
• 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: 1,487,416
• 30 Day Avg. Volume: 1,380,094
• 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: 1.08
• Enterprise Value/Revenue: 5.45
• Enterprise Value/Gross Profit: 6.43
• Enterprise Value/Ebitda: -7.11
• 1Y Price Target: $80.91
• Price Target Upside: 144.59% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 13
• EPS Forecast (FY): -5.54
• Revenue Forecast (FY): $655.98M

• Rating: 50 Neutral
• Price Target: $32.00 ▼(-3.26% Downside)

Ultragenyx's overall stock score reflects a challenging financial position with significant profitability issues. The positive earnings call guidance and strategic financial maneuvers provide some optimism, but technical indicators and valuation metrics suggest caution.

Positive Factors

Revenue Growth

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

Financial Position

Securing nondilutive capital strengthens the balance sheet, providing financial flexibility to invest in growth opportunities.

Pipeline Developments

A robust pipeline with late-stage programs enhances future revenue potential and competitive positioning in the biotech sector.

Negative Factors

Profitability Challenges

Ongoing net losses highlight profitability challenges, which may impact long-term sustainability and require strategic cost management.

Regulatory Challenges

Regulatory setbacks can delay product launches, affecting revenue timelines and increasing development costs.

Cash Flow Challenges

Negative cash flows indicate operational inefficiencies, potentially limiting the ability to fund future growth and innovation.

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 primarily through the sale of its approved therapies and products. Key revenue streams include product sales from its marketed drugs, particularly those addressing rare diseases, which are often priced at a premium due to their specialized nature and the significant investment in research and development required to bring them to market. The company also engages in partnerships and collaborations with other pharmaceutical companies and research institutions, which can include co-development agreements and licensing arrangements that provide additional revenue through upfront payments, milestone payments, and royalties on future sales. Furthermore, Ultragenyx may benefit from government incentives for orphan drugs, including tax credits and extended market exclusivity, which can contribute 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: 33.08
• 50DMA: Positive
• 100DMA: Positive
• 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 Positive. The current price of 33.08 is below the 20-day moving average (MA) of 33.37, above the 50-day MA of 31.63, and below the 200-day MA of 34.61, indicating a neutral trend. The MACD of 0.21 indicates Positive momentum. The RSI at 52.44 is Neutral, neither overbought nor oversold. The STOCH value of 74.54 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
PTCT	74 Outperform	$6.05B	8.80	―	―	97.54%	―
SWTX	61 Neutral	―	―	―	―	730.42%	31.63%
ACLX	52 Neutral	$5.21B	―	-47.18%	―	-76.96%	-425.41%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
RARE	50 Neutral	$3.19B	―	-325.76%	―	20.63%	8.09%
ZLAB	45 Neutral	$2.45B	―	-28.96%	―	23.73%	30.16%
CRSP	40 Underperform	$4.97B	―	-25.33%	―	-81.10%	-99.21%

Ultragenyx Pharmaceutical Corporate Events

Ultragenyx (RARE) Earnings Call Highlights Growth and Challenges

Ultragenyx Pharmaceutical, Inc. recently held its earnings call, where the sentiment was notably optimistic. The company highlighted its strong financial positioning, consistent revenue growth, and promising pipeline advancements, despite facing challenges such as net losses and regulatory hurdles. This positive outlook is underpinned by strategic financial maneuvers and a robust growth trajectory.

Ultragenyx Reports Q3 2025 Financial Results

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, specializes in developing and commercializing therapies for rare and ultra-rare genetic diseases. The company has a diverse portfolio of approved medicines and treatment candidates addressing diseases with high unmet medical needs.

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.

The most recent analyst rating on (RARE) stock is a Buy with a $80.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

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.

The most recent analyst rating on (RARE) stock is a Hold with a $36.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx’s MPS VII Study: A Closer Look at Market Implications

Ultragenyx Pharmaceutical, Inc. is conducting a study titled ‘Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)’ to monitor the presentation and progression of MPS VII, also known as Sly Syndrome. The study aims to assess the long-term effectiveness and safety of vestronidase alfa, focusing on hypersensitivity reactions and immunogenicity.

Ultragenyx’s Ongoing Study on UX111: A Potential Game-Changer for MPS IIIA Treatment

Ultragenyx Pharmaceutical Inc. is conducting a follow-up study titled ‘A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH).’ The study aims to evaluate the safety, tolerability, and efficacy of UX111 (formerly ABO-102) in patients with Mucopolysaccharidosis IIIA (MPS IIIA), a rare genetic disorder. This research is significant as it could lead to improved treatments for this debilitating condition.

Ultragenyx’s LC-FAOD Study: A Strategic Move in Rare Disease Management

Study Overview: Ultragenyx Pharmaceutical Inc. is conducting a study titled ‘Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)’. The study aims to assess the long-term safety of patients with LC-FAOD, focusing on outcomes related to pregnancy, infants, and lactation. This research is significant as it seeks to provide critical insights into the management and treatment of LC-FAOD, a rare metabolic disorder.

Ultragenyx’s GTX-102 Study: A Potential Game-Changer for Angelman Syndrome

Study Overview: The clinical study titled ‘A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome’ aims to evaluate the long-term safety of GTX-102 in individuals with Angelman Syndrome. This study is significant as it addresses the need for sustained treatment options for this rare genetic disorder, potentially improving patient outcomes.

Ultragenyx’s DTX401 Study: A Decade-Long Commitment to Patient Safety

Ultragenyx Pharmaceutical, Inc. is currently conducting a study titled ‘GSDIa Disease Monitoring Program’ to evaluate the long-term safety and effectiveness of DTX401 in patients with Glycogen Storage Disease Type Ia (GSDIa). This observational study aims to monitor patients for at least ten years following the administration of DTX401, highlighting its significance in understanding the long-term impact of the treatment.

Ultragenyx’s GTX-102 Study: A Potential Game-Changer for Angelman Syndrome

Ultragenyx Pharmaceutical, Inc. is conducting a Phase 2 clinical study titled A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS). The primary objective of this study is to assess the safety and efficacy of GTX-102, a treatment aimed at individuals with Angelman Syndrome, a rare genetic disorder. This study is significant as it explores potential therapeutic options for a condition with limited treatment avenues.

Ultragenyx Reports Positive Phase 3 Gene Therapy Results

Ultragenyx Pharmaceutical announced positive long-term results from its Phase 3 study of DTX401 AAV gene therapy for glycogen storage disease type Ia, presented at the International Congress of Inborn Errors of Metabolism 2025 in Kyoto, Japan. The study showed significant reductions in cornstarch intake and improved glycemic control, with participants reporting enhanced quality of life and the therapy demonstrating an acceptable safety profile. These findings underscore the potential of DTX401 to transform treatment for GSDIa, reinforcing Ultragenyx’s position in the rare disease market and offering promising implications for patients and stakeholders.

The most recent analyst rating on (RARE) stock is a Buy with a $105.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx Begins FDA Application for DTX401 Gene Therapy

On August 18, 2025, Ultragenyx Pharmaceutical announced the start of a rolling submission of a Biologics License Application to the FDA for DTX401, a gene therapy for Glycogen Storage Disease Type Ia. The submission includes promising 96-week data from a Phase 3 study showing significant reductions in daily cornstarch intake among patients. The company plans to complete the submission by the fourth quarter of 2025, addressing FDA observations related to manufacturing and controls.

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 Earnings Call: Clinical Wins Amid Financial Challenges

Ultragenyx Pharmaceutical, Inc. recently held its earnings call, revealing a balanced sentiment characterized by significant clinical advancements and robust revenue growth, juxtaposed with financial losses and regulatory challenges. The completion of the GTX-102 Phase III enrollment ahead of schedule and the attainment of Breakthrough Therapy designation were notable highlights, although these were offset by a complete response letter (CRL) for UX111, indicating a cautious optimism moving forward.

Ultragenyx Reports Growth Amidst Strategic Advancements

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, is dedicated to developing and commercializing novel therapies for rare and ultra-rare genetic diseases. The company operates in the biotechnology sector and is known for its focus on addressing high unmet medical needs with innovative treatments.