Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $2.84B
• Dividend Yield: N/A
• Average Volume (3M): 2.35M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.17
• Revenue Growth: 26.77%
• EPS Growth: 24.18%
• Country: US
• Employees: 1,294
• Sector: Healthcare
• Sector Strength: 47
• Industry: Biotechnology
• EPS (TTM): -1.17
• Shares Outstanding: 96,371,640
• 10 Day Avg. Volume: 1,405,768
• 30 Day Avg. Volume: 2,345,089
• 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: 1.40
• Enterprise Value/Revenue: 6.51
• Enterprise Value/Gross Profit: 7.76
• Enterprise Value/Ebitda: -9.13
• 1Y Price Target: $84.38
• Price Target Upside: 183.34% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 16

• Rating: 49 Neutral
• Price Target: $30.00 ▲(0.74% Upside)

Ultragenyx Pharmaceutical's overall stock score is primarily impacted by its financial performance, which shows ongoing profitability challenges despite revenue growth. Technical indicators suggest a bearish trend, and valuation metrics are weak due to negative earnings. While the earnings call and corporate events provide some positive outlooks, regulatory setbacks and financial losses weigh heavily on the score.

Positive Factors

Pipeline and Commercial Momentum

The Overweight rating on RARE shares is based on a positive outlook on setrusumab, an underappreciated pipeline, and ongoing commercial momentum.

Potential Market Opportunity

Considering the significant burden of disease, including the requirement to ingest large quantities of cornstarch multiple times each day, a treatment that can reduce this burden would be well received by the community.

Regulatory Progress

Ultragenyx has initiated the rolling submission of the BLA for DTX401 in Glycogen Storage Disease Type Ia, which may begin to get more credit as it progresses towards commercialization.

Negative Factors

Investor Concerns

Ultragenyx shares have been under pressure due to investor disappointment in the interim readout from the pivotal Orbit trial of UX143 (setrusumab) in osteogenesis imperfecta (OI).

Regulatory Delays

The BLA submission for DTX401 for GSD1a has been delayed to the fourth quarter of 2025.

Stock Valuation Concerns

Despite the continued growth in the base business, the stock is trading at <$30/share, which is below what the commercial business is worth, with seemingly zero value ascribed to the pipeline.

Ultragenyx Pharmaceutical Business Overview & Revenue Model

Company Description

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing innovative therapies for rare and ultra-rare genetic diseases. Established in 2010, the company operates primarily in the biotechnology sector and specializes in the research, development, and commercialization of treatments that address unmet medical needs for patients suffering from these conditions. Ultragenyx's core products include enzyme replacement therapies and other novel therapeutics targeting specific genetic disorders, with a portfolio that includes therapies like Crysvita and Mepsevii.

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 reflects manageable leverage, yet ongoing losses impact shareholder value. Cash flow management shows slight improvement, but operational cash flow remains negative, indicating cash burn.

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: Neutral
• Last Price: 29.78
• 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 Neutral. The current price of 29.78 is above the 20-day moving average (MA) of 28.48, below the 50-day MA of 31.71, and below the 200-day MA of 38.71, indicating a neutral trend. The MACD of -0.31 indicates Negative momentum. The RSI at 52.44 is Neutral, neither overbought nor oversold. The STOCH value of 78.85 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Neutral sentiment for RARE.

Ultragenyx Pharmaceutical Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
ACLX	64 Neutral	$3.95B	―	-42.75%	―	-60.63%	-216.87%
PTCT	58 Neutral	$3.89B	7.25	33.08%	―	96.00%	―
Sector *	51 Neutral	$7.88B	-0.12	-39.86%	2.18%	21.38%	-1.56%
ZLAB	51 Neutral	$3.79B	―	-28.39%	―	32.56%	33.33%
CRSP	50 Neutral	$4.80B	―	-25.34%	―	-81.18%	-69.19%
RARE	49 Neutral	$2.84B	―	-182.60%	―	26.77%	24.18%

Ultragenyx Pharmaceutical Corporate Events

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.

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.

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.

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.

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.

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.