Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $2.07B
• Dividend Yield: N/A
• Average Volume (3M): 2.18M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.12
• Revenue Growth: 20.63%
• EPS Growth: 8.09%
• Country: US
• Employees: 1,294
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -1.29
• Shares Outstanding: 96,629,790
• 10 Day Avg. Volume: 1,765,543
• 30 Day Avg. Volume: 2,181,012
• PEG Ratio: 0.54
• Price to Book (P/B): -28.35
• Price to Sales (P/S): 3.37
• P/FCF Ratio: -4.80
• Enterprise Value/Market Cap: 1.41
• Enterprise Value/Revenue: 4.33
• Enterprise Value/Gross Profit: 5.17
• Enterprise Value/Ebitda: -6.08
• 1Y Price Target: $53.35
• Price Target Upside: 148.60% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 21
• EPS Forecast (FY): -4.24
• Revenue Forecast (FY): $754.37M

• Rating: 46 Neutral
• Price Target: $22.50 ▲(4.85% Upside)
• Action: Reiterated Date: 02/24/26
• Date: Action: Reiterated 02/24/26

The score is primarily held back by weak financial performance: large ongoing losses, significant cash burn, and a sharply reduced equity base. Technicals also remain unfavorable with the stock trading below major moving averages and negative MACD. Offsetting these risks, the latest earnings call provided constructive forward guidance on revenue growth and planned cost reductions aimed at reaching profitability in 2027, but execution and regulatory risks remain significant.

Positive Factors

Sustained revenue growth & high gross margin

Steady top-line expansion paired with very high gross margins indicates durable product-level economics and pricing power typical of rare-disease specialty drugs. This supports reinvestment in launches and pipeline development and improves the long-term capacity to scale commercial operations if execution continues.

Commercial franchise scale & global footprint

An established, growing anchor franchise (Crysvita) plus rapid growth in newer products (Evkeeza) and expansion into 35+ countries lowers single-market dependence, strengthens payer relationships, and creates a repeatable commercial infrastructure to support future launches and geographic expansion over the next several quarters.

Near-term pipeline catalysts & regulatory progress

Multiple late-stage programs and recent regulatory filings represent structural upside: potential first-in-class approvals and pivotal readouts could materially expand the revenue base, leverage existing commercial channels, and generate non-recurring value (eg PRVs) that improve financial flexibility if approvals are achieved.

Negative Factors

Large, persistent cash burn

Consistent, sizeable negative operating and free cash flow indicates ongoing reliance on external funding or asset monetization to maintain operations. High absolute burn reduces runway, forces dilution or asset sales, and constrains strategic optionality absent material improvement or timely non-dilutive proceeds.

Severely weakened equity base / balance-sheet risk

A collapse in equity substantially reduces balance-sheet resilience: even with low nominal debt the elevated debt-to-equity amplifies financing risk and makes future capital raises more dilutive or costly. This limits flexibility to withstand prolonged losses or to invest aggressively without external support.

Dependence on approvals and PRVs for profitability

The company's profitability plan hinges on multiple regulatory outcomes and uncertain PRV proceeds. Approval delays, unfavorable regulatory decisions, or lower-than-expected PRV value would materially derail the plan, making near-term financial improvement contingent on execution and external regulatory timing.

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

Strong revenue growth ($271M in 2020 to $673M in 2025) and high gross margin (~84% in 2025) are outweighed by very large, persistent operating losses (2025 EBIT -$535M) and heavy cash burn (2025 FCF -$472M). The sharp drop in equity in 2025 (to ~$16M) reduces balance-sheet resilience despite low absolute debt ($12M).

Income Statement

Revenue has grown steadily over time (from $271M in 2020 to $673M in 2025), with strong gross profitability (gross margin ~84% in 2025). However, operating losses remain very large and persistent (2025 EBIT of -$535M), keeping net results deeply negative (2025 net margin about -85%). While losses have improved versus earlier years (e.g., 2022 net margin was roughly -195%), the company is still far from profitability and continues to scale expenses faster than profits.

Balance Sheet

Leverage is not the primary issue—total debt is low in 2025 ($12M). The bigger concern is the sharp deterioration in the equity base (stockholders’ equity fell from $255M in 2024 to ~$16M in 2025), which drives a much higher debt-to-equity ratio (0.74 in 2025 vs ~0.16 in 2024) and signals reduced balance-sheet resilience. Total assets have stayed relatively stable (~$1.5B), but the very weak equity position materially increases financial risk if losses continue.

Cash Flow

Cash generation remains a key weakness: operating cash flow and free cash flow are consistently negative, with 2025 operating cash flow at -$466M and free cash flow at -$472M. Negative cash flow has not shown a durable improving trend (burn increased versus 2024), implying continued reliance on external funding. A modest positive is that free cash flow roughly tracks net losses (2025 free cash flow to net income ~1.01), suggesting losses are not being driven by heavy non-cash earnings adjustments—but the absolute cash burn remains high.

Breakdown	Dec 2025	Dec 2024	Dec 2023	Dec 2022	Dec 2021
Income Statement
Total Revenue	673.00M	560.23M	434.25M	363.33M	351.41M
Gross Profit	564.00M	483.50M	389.04M	335.01M	335.40M
EBITDA	-500.00M	-469.00M	-516.45M	-640.49M	-410.32M
Net Income	-575.00M	-569.18M	-606.64M	-707.42M	-454.02M
Balance Sheet
Total Assets	1.53B	1.50B	1.49B	1.55B	1.52B
Cash, Cash Equivalents and Short-Term Investments	693.00M	610.02M	577.21M	747.76M	740.20M
Total Debt	1.28B	40.34M	43.17M	31.59M	41.97M
Total Liabilities	1.60B	1.24B	1.22B	1.19B	599.84M
Stockholders Equity	-80.00M	255.30M	275.41M	352.49M	922.56M
Cash Flow
Free Cash Flow	-472.00M	-421.68M	-521.57M	-526.59M	-411.79M
Operating Cash Flow	-466.00M	-414.19M	-474.81M	-380.46M	-338.69M
Investing Cash Flow	236.00M	-17.77M	168.00M	-291.65M	-195.37M
Financing Cash Flow	478.00M	399.24M	388.14M	501.21M	118.55M

Ultragenyx Pharmaceutical Technical Analysis

• Technical Analysis Sentiment: Negative
• Last Price: 21.46
• 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 Negative. The current price of 21.46 is below the 20-day moving average (MA) of 22.63, below the 50-day MA of 24.05, and below the 200-day MA of 30.42, indicating a bearish trend. The MACD of -0.67 indicates Negative momentum. The RSI at 38.73 is Neutral, neither overbought nor oversold. The STOCH value of 25.44 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
XENE	63 Neutral	$3.50B	-10.27	-51.76%	―	―	-38.30%
TARS	62 Neutral	$3.19B	-51.51	-23.39%	―	182.44%	47.21%
MLYS	52 Neutral	$2.11B	-18.12	-42.14%	―	―	9.60%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
TVTX	47 Neutral	$2.52B	-67.82	-57.80%	―	114.22%	77.02%
RARE	46 Neutral	$2.07B	-3.94	-656.54%	―	20.63%	8.09%
IRON	43 Neutral	$2.30B	-13.21	-35.86%	―	―	-34.96%

Ultragenyx Pharmaceutical Corporate Events

Ultragenyx Gains FDA Priority Review for DTX401 Therapy

On February 23, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Administration accepted for review its Biologics License Application for DTX401, an AAV gene therapy, for the treatment of Glycogen Storage Disease Type Ia. The FDA granted Priority Review status and set a Prescription Drug User Fee Act action date of August 23, 2026, potentially accelerating time to market and reinforcing Ultragenyx’s role as a leading player in rare disease gene therapies.

The most recent analyst rating on (RARE) stock is a Sell with a $19.50 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx Launches Restructuring Plan to Cut Costs

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company developing and commercializing therapies for serious rare and ultra-rare genetic diseases, anchored by products such as Crysvita, Dojolvi, Evkeeza and Mepsevii. The company has a meaningful international footprint, including Latin America and Türkiye, and pursues a strategy centered on high-value, first-in-class treatments for underserved patient populations.

For the year ended December 31, 2025, Ultragenyx reported revenue of $673 million, a 20% increase versus 2024, led by Crysvita’s 17% growth to $481 million and strong expansion in Evkeeza, but still posted a net loss of $575 million on $1.2 billion in operating expenses. On February 12, 2026, the company began implementing a strategic restructuring, including a 10% reduction in headcount, or about 130 employees, and the termination of UX143 manufacturing agreements, expecting about $50 million in related charges in the first half of 2026 as it seeks to reduce costs, focus resources on its largest value drivers, and support its stated path to achieving profitability in 2027.

The most recent analyst rating on (RARE) stock is a Buy with a $75.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx Highlights New UX111 Data in FDA Resubmission

On February 3, 2026, Ultragenyx Pharmaceutical reported new long-term clinical data for UX111 (rebisufligene etisparvovec), its investigational AAV9 gene therapy for Sanfilippo syndrome type A, showing substantial and durable reductions in cerebrospinal fluid heparan sulfate and clinically meaningful improvements in cognition, communication, and motor function versus natural history in younger children, as well as retention of key functional abilities in later-stage patients, with a favorable safety profile over a median 4.8-year follow-up as of a September 2025 data cutoff. These results, which will be presented at WORLDSymposium 2026, have been incorporated into a resubmitted Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval of UX111, positioning the program for a potential regulatory decision in the third quarter of 2026 and underscoring Ultragenyx’s bid to strengthen its presence in the rare-disease gene therapy market and deliver a first-of-its-kind treatment option for a fatal pediatric disorder.

The most recent analyst rating on (RARE) stock is a Buy with a $60.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Ultragenyx Resubmits UX111 Gene Therapy for FDA Review

On January 30, 2026, Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval for UX111, an AAV9 gene therapy for Sanfilippo syndrome type A, incorporating longer-term neurologic outcome data, biomarker evidence such as CSF heparan sulfate, and comprehensive responses to prior chemistry, manufacturing, and controls observations raised in a Complete Response Letter issued in July 2025. The new filing, which follows the FDA’s earlier acknowledgment of robust neurodevelopmental and biomarker data and builds on an additional year of follow-up showing durable treatment benefit and an acceptable safety profile, will proceed under Priority Review with a PDUFA date expected in the third quarter of 2026, positioning UX111 to become the first approved therapy for Sanfilippo syndrome type A and potentially strengthening Ultragenyx’s role in the rare disease gene therapy market.

The most recent analyst rating on (RARE) stock is a Buy with a $57.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

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.