Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $3.39B
• Dividend Yield: N/A
• Average Volume (3M): 1.57M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.19
• Revenue Growth: 26.77%
• EPS Growth: 24.18%
• Country: US
• Employees: 1,294
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -1.17
• Shares Outstanding: 96,371,640
• 10 Day Avg. Volume: 1,298,779
• 30 Day Avg. Volume: 1,565,194
• PEG Ratio: N/A
• Price to Book (P/B): N/A
• Price to Sales (P/S): N/A
• P/FCF Ratio: N/A
• Enterprise Value/Market Cap: 1.17
• Enterprise Value/Revenue: 6.51
• Enterprise Value/Gross Profit: 7.76
• Enterprise Value/Ebitda: -9.13
• 1Y Price Target: $91.50
• Price Target Upside: 160.16% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 13

• Rating: 55 Neutral
• Price Target: $36.00 ▲(2.36% Upside)

Ultragenyx Pharmaceutical's overall stock score is driven by financial performance challenges, including ongoing losses and negative cash flow. While technical indicators show positive momentum, the valuation is impacted by a negative P/E ratio. The earnings call provided optimistic guidance with strong revenue growth and clinical progress, but financial losses remain a concern.

Positive Factors

Revenue Growth

Strong revenue growth indicates robust demand for Ultragenyx's products, reflecting successful commercialization and market expansion, which is crucial for long-term sustainability.

Product Commercialization

Effective product commercialization enhances market presence and revenue streams, supporting the company's strategy to achieve profitability by 2027.

Breakthrough Therapy Designation

The Breakthrough Therapy designation accelerates development and review processes, potentially leading to faster market entry and competitive advantage.

Negative Factors

Profitability Challenges

Ongoing losses highlight significant profitability challenges, which may hinder financial flexibility and long-term shareholder value.

Cash Flow Concerns

High cash burn rates can strain financial resources, necessitating careful cash management to ensure operational sustainability and future growth.

Regulatory Delays

Regulatory delays can postpone product launches, affecting revenue projections and competitive positioning in the market.

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 modest revenue growth but faces significant profitability challenges with negative net profit and EBIT margins. The balance sheet is stable with moderate leverage, yet ongoing losses impact shareholder value. Cash flow management shows slight improvement, but operational cash flow remains negative.

Income Statement

Ultragenyx Pharmaceutical has shown modest revenue growth with a TTM increase of 3.3%. However, the company is facing significant profitability challenges, with negative net profit and EBIT margins indicating ongoing losses. The gross profit margin remains strong at over 83%, suggesting efficient cost management in production, but overall profitability is hindered by high operating expenses.

Balance Sheet

The company's balance sheet reflects a moderate debt-to-equity ratio of 0.27, indicating manageable leverage. However, the negative return on equity highlights ongoing losses impacting shareholder value. The equity ratio is relatively stable, suggesting a solid asset base, but the overall financial health is affected by persistent losses.

Cash Flow

Ultragenyx Pharmaceutical's cash flow statement shows a slight improvement in free cash flow growth, but operating cash flow remains negative, indicating cash burn. The free cash flow to net income ratio is above 1, suggesting some efficiency in cash management despite losses. However, the negative operating cash flow to net income ratio highlights ongoing operational challenges.

Breakdown	TTM	Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement
Total Revenue	610.16M	560.23M	434.25M	363.33M	351.41M	271.03M
Gross Profit	511.96M	483.50M	389.04M	335.01M	335.40M	264.90M
EBITDA	-435.33M	-469.00M	-516.45M	-640.51M	-410.32M	-139.81M
Net Income	-532.93M	-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	496.62M	610.02M	577.21M	896.73M	999.13M	1.21B
Total Debt	882.91M	910.01M	43.17M	31.59M	41.97M	48.23M
Total Liabilities	1.15B	1.24B	1.22B	1.19B	599.84M	605.18M
Stockholders Equity	151.29M	255.30M	275.41M	352.49M	922.56M	1.15B
Cash Flow
Free Cash Flow	-427.30M	-421.68M	-521.57M	-526.59M	-411.79M	-176.13M
Operating Cash Flow	-421.25M	-414.19M	-474.81M	-380.46M	-338.69M	-132.22M
Investing Cash Flow	18.30M	-17.77M	168.00M	-291.65M	-195.37M	-179.12M
Financing Cash Flow	98.17M	399.24M	388.14M	501.21M	118.55M	600.27M

Ultragenyx Pharmaceutical Technical Analysis

• Technical Analysis Sentiment: Positive
• Last Price: 35.17
• 50DMA: Positive
• 100DMA: Positive
• 200DMA: Positive
• 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 Positive. The current price of 35.17 is above the 20-day moving average (MA) of 32.88, above the 50-day MA of 31.16, and above the 200-day MA of 35.15, indicating a bullish trend. The MACD of 1.20 indicates Negative momentum. The RSI at 63.36 is Neutral, neither overbought nor oversold. The STOCH value of 67.34 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
SWTX	―	―	―	―	―	730.42%	31.63%
PTCT	―	$5.41B	9.77	―	―	96.00%	―
ACLX	―	$4.77B	―	-42.75%	―	-60.63%	-216.87%
RARE	―	$3.39B	―	-182.60%	―	26.77%	24.18%
CRSP	―	$5.56B	―	-25.34%	―	-81.18%	-69.19%
Sector *	―	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
ZLAB	―	$2.93B	―	-28.39%	―	32.38%	33.47%

Ultragenyx Pharmaceutical Corporate Events

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.

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.