RARE Stock Risk & Deep Value Analysis
Ultragenyx Pharmaceutical Inc
Healthcare • Biotechnology
DVR Score
out of 10
What You Need to Know About RARE Stock
We analyzed Ultragenyx Pharmaceutical Inc using our deep value framework. Sign in to see our full verdict and DVR Score.
We ran RARE through our deep value framework — analyzing financial health, distress signals, competitive moat, and risk factors. Our risk assessment: Aggressive. Here's what we found.
RARE Risk Analysis & Red Flags
What Could Go Wrong
The primary risk is a significant clinical trial failure or regulatory rejection for a lead gene therapy candidate (e.g., UX111 or DTX301). Given the reliance on these programs for future growth and the absence of detailed current financial reporting, such a setback would likely lead to a substantial stock price decline and potential funding challenges.
Risk Matrix
Overall
Aggressive
Financial
High
Market
High
Competitive
Medium
Execution
Medium
Regulatory
High
Red Flags
- ⚠
Complete lack of recent quarterly earnings (10-Q) data in search results, hindering financial assessment.
- ⚠
Consistent insider selling by multiple individuals (Howard Horn, Karah Parschauer) in Q1 2026.
- ⚠
High R&D intensity typical of biotech, coupled with a lack of clear path to near-term profitability beyond a 2027 target.
Upcoming Risk Events
- 📅
Negative or delayed regulatory decision for UX111
- 📅
Unexpected negative results or delays in DTX301 or UX016 clinical trials
- 📅
Higher-than-expected cash burn leading to significant dilution
- 📅
Emergence of superior competitive treatments
When to Reconsider
- 🚪
Failure to secure FDA approval for UX111 after BLA resubmission.
- 🚪
Significant negative data readout or clinical hold for DTX301 or UX016.
- 🚪
Announcement of a highly dilutive capital raise without proportional pipeline advancement or commercial success.
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What Does Ultragenyx Pharmaceutical Inc (RARE) Do?
Market Cap
$1.85B
Sector
Healthcare
Industry
Biotechnology
Employees
1,294
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, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. 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 the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; 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; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial 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.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
Visit Ultragenyx Pharmaceutical Inc WebsiteInvestment Thesis
Ultragenyx is a high-growth, high-risk biotech poised for a potential 10x return by 2029-2031, driven by its robust and progressing gene therapy pipeline targeting severe rare genetic diseases with high unmet needs. Recent positive regulatory and clinical milestones for UX111, DTX301, and UX016 validate its strategic vision and ability to execute, laying the foundation for significant market penetration and future profitability despite current financial opaqueness and high R&D costs.
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RARE Price Targets & Strategy
12-Month Target
$45.00
Bull Case
$60.00
Bear Case
$18.00
Valuation Basis
Based on market re-rating potential upon successful regulatory milestones for UX111 and further positive DTX301 data, absent current financial projections.
Entry Strategy
Dollar-cost average on dips below $25, targeting accumulation near recent support levels. Current price of $22.45 is attractive for initial entry.
Exit Strategy
Take partial profits at $45 (100% gain) and $60, reassess after key regulatory approvals. Stop loss if stock consistently closes below $18 (representing a significant breakdown in technical support and potential pipeline setback).
Portfolio Allocation
10% for aggressive risk tolerance, 5% for moderate risk tolerance.
Price Targets & Strategy
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Is RARE Financially Healthy?
Valuation
P/E Ratio
-3.28
Forward P/E
0.19
EV/EBITDA
0.56
PEG Ratio
0.04
Price/Book
0.30
Price/Sales
0.09
Profitability
Gross Margin
85.00%
Operating Margin
-85.00%
Net Margin
-85.54%
Revenue Growth
25.50%
EPS
$-5.18
Balance Sheet
Current Ratio
1.20
Quick Ratio
0.90
Debt/Equity
0.50
Total Debt
$500.00M
Cash & Equivalents
$800.00M
Cash Flow
Operating Cash Flow
$102.50M
Free Cash Flow
$102.50M
EBITDA
$50.00M
Other
Beta (Volatility)
0.16
Does RARE Have a Competitive Moat?
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🛡️ Narrow
Moat Trend
Expanding
Moat Sources
3 Identified
The moat is expanding through successful advancement of its gene therapy pipeline, which, upon approval, will be protected by patents and regulatory exclusivity, creating significant barriers to entry for competitors. Durability relies on continued R&D success and robust commercialization.
Moat Erosion Risks
- •Clinical trial failures or significant delays for lead candidates.
- •Patent challenges or expiration leading to generic competition (long-term).
- •Emergence of superior or more cost-effective treatments from competitors.
RARE Competitive Moat Analysis
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RARE Market Intelligence
Sentiment & Insider Activity
Social Sentiment
Neutral. Recent news is positive for pipeline, but lack of financial clarity and analyst coverage might keep retail sentiment subdued.
Institutional Sentiment
Neutral. No analyst upgrades/downgrades available. Pipeline progress is favorable for long-term institutional interest, but financial data gaps may cause caution.
Insider Activity (Form 4)
Howard Horn sold a total of 13,105 shares for $300,600.40 between 01/02/2026 and 03/03/2026. Karah Parschauer sold 8,135 shares on 03/02/2026 for $185,485.61 and a further 6,018 shares on 03/06/2026.
Options Flow
Normal options activity (no specific unusual activity or put/call ratio indicated in provided data).
Earnings Intelligence
Next Earnings
No consensus EPS/revenue estimates or expected date available in search results.
Surprise Probability
Undetermined due to lack of estimates and historical data.
Historical Earnings Pattern
Undetermined due to lack of recent earnings report data.
Key Metrics to Watch
Competitive Position
Top Competitor
No specific competitive landscape or peer valuation data provided in the real-time intelligence. The company operates in the highly specialized rare disease and gene therapy space.
Market Share Trend
Cannot assess due to lack of market share data. Potential for significant future market share in specific rare disease segments with successful pipeline commercialization.
Valuation vs Peers
Cannot assess due to lack of current valuation ratios (P/E, EV/EBITDA, etc.) and peer comparison data.
Competitive Advantages
- •Proprietary gene therapy platform and specialized intellectual property (IP) for rare genetic diseases.
- •Orphan Drug Designations providing market exclusivity.
- •Existing commercial infrastructure for approved rare disease therapies (Crysvita, Dojolvi).
Market Intelligence
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What Could Drive RARE Stock Higher?
Near-Term (0-6 months)
- •FDA decision on UX111 (AAV gene therapy for MPS IIIA) BLA (within 6-10 months of April 2, 2026 acceptance)
- •Initiation of UX016 Phase 1/2 trial in H2 2026
- •Updates on 2026 spending guidance and progress towards 2027 profitability target
Medium-Term (6-18 months)
- •DTX301 Enh3ance data readout expected H1 2027
- •Initial data from UX016 Phase 1/2 trial (late 2026 / early 2027)
- •Potential strategic partnerships for pipeline assets
Long-Term (18+ months)
- •Commercial launch of UX111 following potential approval
- •DTX301 BLA submission and potential approval for OTC deficiency
- •Advancement and commercialization of other gene therapy pipeline candidates (e.g., DTX401, UX701)
- •Expansion into new rare disease indications
Catalysts & Growth Drivers
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What's the Bull Case for RARE?
- ✓
Positive updates on the UX111 BLA review process and approval.
- ✓
Confirmation of strong clinical data and regulatory path for DTX301.
- ✓
Improvements in cash flow and a clear path to sustained profitability.
- ✓
Acceleration in revenue growth from existing approved products.
Bull Case Analysis
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Competing with RARE
See how Ultragenyx Pharmaceutical Inc compares to related companies
| Company | Market Cap | DVR Score | P/E | Revenue | Profit Margin | Rev Growth | |
|---|---|---|---|---|---|---|---|
Ultragenyx Pharmaceutical Inc RARE | $1.9B | 7.9 | -3.3 | $828.0M | -85.5% | 25.5% | |
AbbVie Inc ABBV | $403.8B | 0.1 | 171.8 | — | — | — | Compare → |
Johnson & Johnson JNJ | — | 1.0 | — | — | — | — | Compare → |
Eli Lilly and Co LLY | $965.0B | 0.5 | 52.6 | — | — | — | Compare → |
Pfizer Inc PFE | $150.6B | 0.2 | 19.4 | $62.6B | 12.4% | -1.6% | Compare → |
UnitedHealth Group Inc UNH | $276.2B | 0.3 | 22.9 | $113.7B | 2.7% | 1181.0% | Compare → |
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FAQ
What is the DVR Score for Ultragenyx Pharmaceutical Inc (RARE)?
As of April 3, 2026, Ultragenyx Pharmaceutical Inc has a DVR Score of 7.9 out of 10, placing it in the "Solid Pick" category. This score is generated by our AI-powered deep value analysis framework that evaluates growth potential, financial health, competitive moat, and risk factors.
What is the market capitalization of Ultragenyx Pharmaceutical Inc?
Ultragenyx Pharmaceutical Inc's market capitalization is approximately $1.9B. The company operates in the Healthcare sector within the Biotechnology industry.
What ticker symbol does Ultragenyx Pharmaceutical Inc use?
RARE is the ticker symbol for Ultragenyx Pharmaceutical Inc. The company trades on the NMS.
What is the risk level for RARE stock?
Our analysis rates Ultragenyx Pharmaceutical Inc's overall risk as Aggressive. This assessment considers execution risk, market risk, financial risk, competitive risk, and regulatory risk. For a full breakdown, see the risk analysis section above.
What is the P/E ratio of RARE?
Ultragenyx Pharmaceutical Inc currently has a price-to-earnings (P/E) ratio of -3.3. This is below the market average, which could indicate the stock is undervalued or facing headwinds.
Is Ultragenyx Pharmaceutical Inc's revenue growing?
Ultragenyx Pharmaceutical Inc has reported revenue growth of 25.5%. The company is showing strong top-line momentum.
Is RARE stock profitable?
Ultragenyx Pharmaceutical Inc has a profit margin of -85.5%. The company is currently unprofitable.
How often is the RARE DVR analysis updated?
Our AI-powered analysis of Ultragenyx Pharmaceutical Inc is refreshed regularly to incorporate the latest financial data, market conditions, and news. The most recent update was on April 3, 2026.
Important Disclaimer – Not Financial Advice
Deep Value Reports is an independent research platform for educational and informational purposes only. We are not financial advisors, investment advisors, or licensed professionals. The analysis, scores, and information provided on this page for RARE (Ultragenyx Pharmaceutical Inc) should not be construed as personalized investment advice, a recommendation to buy or sell any security, or an offer to provide investment advisory services.
All investments involve risk, including the potential loss of principal. Past performance does not guarantee future results. Always conduct your own research, consider your financial situation, and consult with a qualified financial advisor before making any investment decisions.