Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $2.32B
• Dividend Yield: N/A
• Average Volume (3M): 3.09M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.08
• 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: 3,165,492
• 30 Day Avg. Volume: 3,092,293
• 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: 1.48
• Enterprise Value/Revenue: 5.45
• Enterprise Value/Gross Profit: 6.43
• Enterprise Value/Ebitda: -7.11
• 1Y Price Target: $60.31
• Price Target Upside: 156.64% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 18
• EPS Forecast (FY): -3.83
• Revenue Forecast (FY): $789.07M

• Rating: 46 Neutral
• Price Target: $23.50 ▲(0.00% Upside)

The score is held down primarily by weak financial performance (ongoing losses, negative cash flow, and leverage) and a bearish technical setup (price well below key moving averages with negative MACD). These are partially offset by constructive earnings-call guidance and balance-sheet support from nondilutive financing, while recent corporate updates are mixed due to a major Phase 3 miss alongside a BLA filing milestone.

Positive Factors

High gross margin

An 83% gross margin indicates strong unit economics from approved therapies, providing durable pricing power and cash generation potential as volumes scale. High gross margins help fund R&D and commercialization, supporting a sustainable path to profitability even while operating losses persist.

Consistent revenue growth & guidance

Management’s multi-year revenue growth guidance reflects expanding commercial traction for Crysvita and other products. Persistent top-line growth supports reinvestment in late-stage programs and underpins the company’s stated path to GAAP profitability by 2027, reducing execution risk over the medium term.

Nondilutive capital via royalty sale

The $400M royalty purchase improves liquidity without equity dilution, lengthening runway for pivotal readouts and regulatory submissions. This structural funding reduces near-term financing pressure, enabling focus on clinical milestones that drive long-term value creation for rare-disease franchises.

Negative Factors

Negative cash flow and burn

Sustained operating cash outflows and large net losses deplete capital and force reliance on external financing. Persistent cash burn increases execution risk for late-stage programs and raises the importance of successful launches or further nondilutive financing to avoid dilution or curtailed R&D over the medium term.

High leverage

A debt-to-equity ratio above 1 indicates material leverage, which can constrain strategic flexibility and increase fixed obligations. High leverage reduces optionality for expensive trials or M&A and elevates refinancing and interest-rate risk, especially if cash generation remains negative over several quarters.

Phase 3 miss and program reassessment

A Phase 3 miss on a primary endpoint weakens expected commercial upside and forces program reassessment and expense cuts. This reduces pipeline optionality, delays potential revenue from osteogenesis imperfecta, and increases reliance on other programs and existing products to deliver longer-term growth.

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: Negative
• Last Price: 23.50
• 50DMA: Negative
• 100DMA: Negative
• 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 Negative. The current price of 23.5 is below the 20-day moving average (MA) of 26.90, below the 50-day MA of 31.07, and below the 200-day MA of 32.47, indicating a bearish trend. The MACD of -2.63 indicates Positive momentum. The RSI at 36.89 is Neutral, neither overbought nor oversold. The STOCH value of 60.87 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
TARS	60 Neutral	$3.19B	-36.58	-28.35%	―	182.44%	47.21%
XENE	59 Neutral	$3.21B	-10.49	-45.14%	―	―	-38.30%
TVTX	56 Neutral	$2.59B	-26.18	-410.78%	―	114.22%	77.02%
IRON	52 Neutral	$2.91B	―	-34.78%	―	―	-34.96%
MLYS	52 Neutral	$2.64B	-10.61	-42.14%	―	―	9.60%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
RARE	46 Neutral	$2.32B	―	-325.76%	―	20.63%	8.09%

Ultragenyx Pharmaceutical Corporate Events

Ultragenyx completes BLA filing for GSDIa gene therapy

On December 30, 2025, Ultragenyx Pharmaceutical announced it had completed the rolling submission of its Biologics License Application to the U.S. Food and Drug Administration for DTX401, an AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia. The application is supported by a clinical program involving 52 treated patients with up to six years of follow-up, including Phase 3 GlucoGene trial data showing significant and clinically meaningful reductions in patients’ daily cornstarch intake, improved glycemic control and fasting tolerance, and better patient-reported quality of life, alongside an acceptable safety profile. Completion of the BLA, including the final chemistry, manufacturing and controls module submitted in 2025, marks a key regulatory milestone that could advance Ultragenyx’s position in the rare disease gene therapy space and potentially bring a first-of-its-kind treatment option closer to patients with GSDIa, pending FDA review and decision.

The most recent analyst rating on (RARE) stock is a Buy with a $45.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx Reveals Mixed Phase 3 Results for Setrusumab

On December 29, 2025, Ultragenyx reported Phase 3 results from its Orbit and Cosmic trials of setrusumab (UX143) in Osteogenesis Imperfecta, showing that neither study met its primary endpoint of statistically significant reduction in annualized clinical fracture rates versus placebo or bisphosphonates, despite both trials demonstrating substantial and statistically significant improvements in bone mineral density and no change in the safety profile. The mixed efficacy outcome, with fracture-rate reductions in the pediatric Cosmic study not reaching statistical significance, has prompted the company to conduct further analyses of additional bone health and clinical endpoints and to plan significant expense reductions as it reassesses the future of the setrusumab program and its broader operational priorities.

The most recent analyst rating on (RARE) stock is a Buy with a $70.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

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.