Ultragenyx Pharmaceutical (RARE) AI Stock Analysis

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Ultragenyx Pharmaceutical

(BMV:RARE)

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Positive Factors

Revenue Growth and Gross MarginStrong revenue momentum combined with very high gross margins indicates improving commercial traction and attractive product economics. If sustained, this profile can provide increasing resources to fund launches, support selective pipeline investment, and strengthen the company’s ability to approach profitability.

Negative Factors

Persistent Losses and Cash BurnLarge negative operating and free cash flow show that the business has not yet converted commercial growth into internal funding. Continued cash consumption can constrain investment choices and increase dependence on successful launches, cost reductions, or additional capital before profitability is achieved.

Ultragenyx Pharmaceutical (RARE) vs. SPDR S&P 500 ETF (SPY)

Market Cap: MXN 1.70B
Dividend Yield: ―
Average Volume (3M): 54
Price to Earnings (P/E): -3.2
Beta (1Y): 0.13
Revenue Growth: 4.03%
EPS Growth: 21.40%
Country: Mexico
Employees: 1,371
Sector: Healthcare
Sector Strength: 51
Industry: Biotechnology

Ultragenyx Pharmaceutical Business Overview & Revenue Model

Company Description

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical firm dedicated to discovering, developing, and commercializing innovative treatments for rare and ultra-rare genetic conditions. Its operations span North America, Europe, and other international...

Ultragenyx Pharmaceutical Earnings Call Summary

Earnings Call Date:Nov 03, 2026(Q3-2026)|% Change Since:0.00%|Next Earnings Date:Feb 18, 2027

Earnings Call SentimentNeutral

The call had substantial positive developments, including two first-ever gene therapy approvals, strong demand and launch preparation, growth across established products, approximately $350 million in nondilutive proceeds, and reaffirmed 2027 profitability expectations. These were balanced by the GTX-102 Phase 3 failure, termination of UX701, a roughly 20% workforce reduction, restructuring charges, and uncertainty around the setrusumab regulatory path. Overall sentiment was mixed and balanced.

Positive Updates

Two First-Ever Gene Therapy Approvals

The FDA approved GENGLYCOS in August for Glycogen Storage Disease Type Ia and FAYUVI in September for Sanfilippo syndrome Type A. These approvals brought Ultragenyx to six approved medicines and its first two gene therapies.

Negative Updates

GTX-102 Phase 3 Efficacy Failure

The GTX-102 Phase 3 study in Angelman syndrome failed to prove efficacy, contrary to the company's expectations after compelling Phase 2 data. Management said variability in the control group across multiple endpoints prevented a distinct separation in a one-year randomized trial, and the clinical program is being discontinued.

Company Guidance

Ultragenyx reaffirmed 2026 total revenue guidance of $730 million to $760 million, excluding revenue from FAYUVI and GENGLYCOS, including CRYSVITA revenue of $500 million to $520 million and DOJOLVI revenue of $100 million to $110 million, and expects combined R&D and SG&A expenses to be flat to down low single digits versus 2025, including $30 million in severance and other nonrecurring restructuring charges from the first half of the year and approximately $25 million expected in the fourth quarter; for 2027, it expects to provide full year revenue guidance early in the new year, R&D expenses of less than $350 million, a reduction of more than 50% from 2025 levels, SG&A expenses of less than $375 million, an increase of up to 7% from 2025 levels, headcount reduced by approximately 20% to a revised workforce of approximately 1,000 employees, full year GAAP net income profitability and positive cash flow from operating activities, with net proceeds from the sale of two PRVs totaling approximately $350 million.

Ultragenyx Pharmaceutical Risk Analysis

Ultragenyx Pharmaceutical disclosed 61 risk factors in its most recent earnings report. Ultragenyx Pharmaceutical reported the most risks in the "Tech & Innovation" category.

Tech & Innovation - Risks related to the company’s reliance on technology and ability to make innovative products

Latest Risks Added

0 New Risks

Ultragenyx Pharmaceutical Peers Comparison

Overall Rating

Financial Indicators

MX:RARE against its peers
Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
Sector *	51Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%

* Healthcare Sector Average

Performance Comparison

MX:RARE and its peers over the selected period
Show on chart	Ticker	Company Name	Price	Change	% Change
	MX:RARE	Ultragenyx Pharmaceutical, Inc.	256.00	-555.00	-68.43%

Glossary

BuyA stock rated as a "Buy" is expected to perform better than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock is likely to deliver higher returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.

HoldA stock rated as a "Hold" is expected to perform in line with the overall market or a specific benchmark. This rating indicates that the stock is neither particularly compelling nor unfavorable for investment. Note: This is not investment advice; please consult a financial advisor before making investment decisions.

SellA stock rated as a "Sell" is expected to perform worse than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock may deliver lower returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.

Disclaimer

This AI Analyst Stock Report is automatically generated by our AI systems using advanced algorithms and publicly available financial, technical, and market data. While the information provided aims to be accurate and insightful, it is intended for informational purposes only and should not be considered financial advice. Any content created by an AI (Artificial Intelligence) system may contain inaccuracies and/or contain errors. Investing in stocks carries inherent risks, and past performance is not indicative of future results. This report does not account for your personal financial circumstances, objectives, or risk tolerance. Always conduct your own research or consult with a qualified financial advisor before making investment decisions. The analysis and recommendations provided are based on historical and current data and may not fully reflect future market conditions or unexpected developments. Neither the creators of this report nor its affiliated entities guarantee the accuracy, completeness, or reliability of the information presented. Use this report at your own discretion and risk.

Ultragenyx Pharmaceutical AI Stock Analysis