Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

• Market Cap: $2.38B
• Dividend Yield: N/A
• Average Volume (3M): 3.32M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.07
• 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: 2,554,526
• 30 Day Avg. Volume: 3,315,893
• 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: 1.24
• Enterprise Value/Revenue: 4.69
• Enterprise Value/Gross Profit: 5.53
• Enterprise Value/Ebitda: -6.11
• 1Y Price Target: $60.31
• Price Target Upside: 144.27% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 18
• EPS Forecast (FY): N/A
• Revenue Forecast (FY): N/A

• Rating: 47 Neutral
• Price Target: $24.50 ▼(-0.77% Downside)

The score is held down primarily by weak financial performance—ongoing losses, negative operating/free cash flow, and elevated leverage—despite strong gross margins and revenue growth. Technically, the stock remains in a broader downtrend below key longer-term moving averages with a negative MACD. Offsetting positives include upbeat revenue guidance, nondilutive financing that improves near-term flexibility, and constructive regulatory milestones for key gene therapy programs.

Positive Factors

High gross margins and recurring revenue growth

Ultragenyx's very high gross margin (83% TTM) and consistent product revenue growth reflect durable pricing power and efficient product economics for its approved therapies. That margin profile supports reinvestment in R&D and commercialization as revenues scale, improving long-term commercialization leverage even before new approvals.

Nondilutive $400M royalty financing strengthens runway

The $400M royalty sale to OMERS provides nondilutive liquidity and defers payments to 2028, materially improving near-term balance sheet flexibility. This reduces the immediate need for equity dilution and preserves capital to fund late-stage programs and regulatory activities through pivotal readouts.

Progress on multiple gene therapy BLAs and priority review

Resubmission of UX111 with longer-term outcomes and completion of DTX401's BLA mark structural progress toward potentially first-in-class approvals. Successful approvals would expand Ultragenyx's rare-disease franchise, create durable revenue streams, and materially de-risk program timelines versus earlier-stage pipelines.

Negative Factors

Elevated leverage on the balance sheet

A debt-to-equity ratio of 1.33 signals meaningful leverage for a development-stage biopharma that still faces regulatory milestones. Elevated debt increases fixed obligations and reduces financial flexibility, heightening refinancing and interest-rate risk if approvals or revenue ramps are delayed.

Negative operating and free cash flow with ongoing cash burn

Persistent negative operating and free cash flow and large YTD cash burn indicate ongoing funding needs to support operations and late-stage trials. This structural cash deficit increases dependency on external financing, which can dilute equity or add debt if commercial uplifts from new approvals are slower than expected.

Mixed Phase 3 results create program risk

Setrusumab's failure to meet primary endpoints in two Phase 3 trials is a structural setback: it reduces near-term product growth potential, may trigger program write-downs, and forces cost reductions that could slow other development activities, increasing execution risk across the pipeline.

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

Revenue is growing and gross margin is strong (83.32% TTM), but profitability remains weak with negative net profit/EBIT margins. Balance sheet leverage is elevated (TTM debt-to-equity 1.33) with negative ROE, and cash generation is pressured by negative operating and free cash flow despite modest improvement.

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: Neutral
• Last Price: 24.69
• 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 24.69 is above the 20-day moving average (MA) of 23.87, below the 50-day MA of 28.91, and below the 200-day MA of 31.82, indicating a neutral trend. The MACD of -0.96 indicates Negative momentum. The RSI at 45.60 is Neutral, neither overbought nor oversold. The STOCH value of 71.72 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
TARS	60 Neutral	$2.70B	-31.54	-28.35%	―	182.44%	47.21%
XENE	59 Neutral	$3.18B	―	-45.14%	―	―	-38.30%
TVTX	56 Neutral	$2.83B	-29.21	-410.78%	―	114.22%	77.02%
IRON	52 Neutral	$3.08B	-15.24	-34.78%	―	―	-34.96%
MLYS	52 Neutral	$2.42B	-10.35	-42.14%	―	―	9.60%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
RARE	47 Neutral	$2.38B	-4.16	-325.76%	―	20.63%	8.09%

Ultragenyx Pharmaceutical Corporate Events

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.

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.