Ultragenyx Pharmaceutical Inc.
$22.41
-1.9%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.
At a Glance
Live SnapshotIntrinsic Alpha Methodology
Thesis Lenses
Signal
Medium confidenceValuation multiple is compressed (P/E -3.8).
Valuation inputs: P/E -3.8, earnings yield N/A, momentum -1.9%.
If multiples stay elevated while momentum fades, downside repricing risk rises.
Driver
High confidenceReturn profile is soft (ROE +7.2%, ROIC -43.5%).
Profitability stack: net margin -85.4%, ROE +7.2%, ROIC -43.5%.
Quality deterioration often appears in margins before it shows up in headline EPS.
Risk
High confidenceDownside profile is elevated and scenario stress-testing is essential.
Risk factors: liabilities/assets +104.8%, momentum -1.9%, net margin -85.4%.
Higher leverage with weak momentum and thin margins can amplify drawdown severity.
Ultragenyx Pharmaceutical Inc. Market vs Earnings Trajectory
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Ultragenyx Pharmaceutical Inc. Fair Value Envelope
Ultragenyx Pharmaceutical Inc. Profit Bridge
Profitability Analysis
Gross Margin
Exceptional pricing power and cost management. The company retains over half of revenue after direct costs.
Operating Margin
Operating losses indicate the core business is unprofitable. Requires restructuring or revenue growth.
Net Profit Margin
Net losses indicate the company is burning cash. Sustainability depends on path to profitability.
Profitability Insight
Margin squeeze alert: Healthy gross margins but weak operating margins suggest excessive operating expenses. Cost optimization could unlock significant value.
Ultragenyx Pharmaceutical Inc. Cash Conversion Engine
Cash Flow Quality Analysis
FCF Conversion Rate
Strong cash conversion. Most reported earnings translate into actual cash flow, suggesting solid fundamentals.
CapEx Intensity
Asset-light business model. Low capital requirements leave more cash available for shareholders and growth investments.
FCF Growth Trend
Declining FCF warrants attention. Investigate whether due to temporary factors or fundamental deterioration.
Working Capital Impact
Minimal working capital impact on FCF. Changes in operating assets and liabilities well-managed.
Cash Flow Quality Insight
Working capital strain: Significant cash tied up in operations. Monitor receivables collection, inventory turns, and payment terms to optimize cash conversion cycle.
Ultragenyx Pharmaceutical Inc. Funding & Solvency Profile
Financial Health Analysis
Current Ratio
Excellent liquidity position. The company can easily cover its short-term obligations with substantial cushion.
Working Capital
Strong working capital position provides significant operational flexibility and financial cushion.
Asset Composition
Balanced asset mix between current and non-current assets, typical of many stable businesses.
Ultragenyx Pharmaceutical Inc. Street Expectations Map
Wall Street analysts project that RARE stock may rise significantly over the coming 12 months. The consensus 1-year price target stands at 48.36, with estimates ranging from a low of 26.00 to a high of 67.00.
The consensus 1-year price target stands at 48.36, with estimates ranging from a low of 26.00 to a high of 67.00.
Analyst Consensus Analysis
Upside Potential
Substantial upside potential. Analysts see significant value gap, suggesting the stock may be materially undervalued at current levels.
Analyst Agreement
High uncertainty. Very wide target spread indicates significant disagreement among analysts—major valuation uncertainty or transformation underway.
Analyst Conviction
High uncertainty: Wide dispersion indicates analysts struggle to agree on valuation—proceed with caution and independent analysis.
Market Sentiment Insight
High risk, high reward: Substantial upside potential exists, but wide analyst disagreement signals execution risk or unclear catalysts. Thorough due diligence essential before committing capital.
Ultragenyx Pharmaceutical Inc. Insider Positioning
During the last 12 months, insiders have sold $3.42M worth of RARE shares, with no buying activity reported.
3 Months
0
0.00
29.6K
686.49K
-686K
-$686K
3-6 Months
0
0.00
99.4K
2.31M
-2.3M
-$2.31M
6-9 Months
0
0.00
10.5K
328.48K
-328K
-$328K
9-12 Months
0
0.00
2.5K
92.56K
-93K
-$93K
Top Buyers
No buying activity
Top Sellers
Kakkis Emil D
Director, Officer: President & Ceo
$1.24M
Horn Howard
Officer: Chief Financial Officer
$952K
Parschauer Karah Herdman
Officer: Evp And Chief Legal Officer
$470K
Harris Erik
Officer: Evp & Chief Commercial Officer
$240K
Pinion John Richard
Officer: See Remarks
$229K
Insider Activity Analysis
Net Insider Sentiment
Strong bearish signal with $3.42M net selling. Heavy insider selling may indicate concerns about valuation or near-term prospects.
Buy/Sell Ratio
Very poor ratio. Heavy selling with minimal buying suggests insiders may be concerned about valuation or outlook.
Recent Trend (3 Months)
Heavy recent selling activity. Recent insider sales notably exceed purchases—may warrant investigation into company developments.
Insider Participation
5 insider sellers vs. 0 buyers. Widespread selling across multiple insiders may signal concerns.
Insider Activity Insight
Multiple red flags: Heavy selling, numerous sellers, and continued recent selling create a concerning pattern. Investigate fundamental drivers and consider whether company guidance or industry conditions have deteriorated.
No Dividend Yield Data
RARE has not reported any dividend yield values in the available annual periods.
rare Dividend History
Learning Layer
Related Guides
Build conviction on RARE by pairing this stock hub with focused valuation guides.
Research Paths
Snapshot
Start with context, operating signals, and key market metrics.
Value Model
Stress test fair value across bear, base, and bull assumptions.
Statements
Validate revenue quality, margins, and balance sheet durability.
Earnings Call
Read management commentary and compare it with reported outcomes.
Dividends
Check payout sustainability and long-term distribution behavior.
Analyst Expectations
Review consensus spread and where estimate risk is concentrated.