RARE Stock Risk & Deep Value Analysis

Ultragenyx Pharmaceutical Inc

Healthcare • Biotechnology

DVR Score

7.9

out of 10

Solid Pick

The Bottom Line on RARE

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. Here's what we found.

Updated Apr 3, 2026•Run Fresh Analysis →•

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RARE Stock Risk Analysis

Overall Risk

Aggressive

Financial Risk

High

Market Risk

High

About Ultragenyx Pharmaceutical Inc (RARE)

Sector

Healthcare

Industry

Biotechnology

Market Cap Category

mid

Market Cap

$1.85B

RARE Deep Value Analysis

Ultragenyx (RARE) maintains its compelling high-risk, high-reward profile, scoring 79/100 for 10x potential within 3-5 years. The company has made significant strides in advancing its gene therapy pipeline, with the FDA accepting the BLA resubmission for UX111, positive Phase 3 results for DTX301, and IND clearance for UX016. These milestones directly validate the strategic vision for addressing severe rare genetic diseases and are critical drivers for future market leadership and competitive advantage. While detailed current financial data is unavailable, the pipeline's progress reinforces the long-term growth thesis. Insider selling is noted but not substantial enough to outweigh the positive clinical developments. The 10x potential is critically tied to continued successful clinical and regulatory advancement, making it a speculative but potentially transformative investment.

RARE Research Sources

Research sources

No external source links for this analysis yet. Run a fresh analysis to capture SEC filings and financial news articles we used.

For educational context only. Not financial advice.

RARE Red Flags & Warning Signs

⚠
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

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RARE Financial Health Metrics

Market Cap

$1.85B

P/E Ratio

-3.28

Profit Margin

-85.54%

Debt-to-Equity

0.50

Dividend Yield

0.00%

Beta (Volatility)

0.16

Earnings Per Share

$-5.18

RARE Competitive Moat Analysis

Moat Rating

Narrow

Moat Trend

Expanding

Moat Sources

3 Identified

Intangible Assets/IP (patents, proprietary gene therapy platforms, orphan drug designations)Switching Costs (for patients on existing rare disease treatments, though less applicable for gene therapies aiming for curative effects)Regulatory Exclusivity (granted for orphan drugs)

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.

RARE Competitive Moat Analysis

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RARE Catalysts & Growth Drivers

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

RARE Bull Case: What Could Go Right

✓
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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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 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 and consult with a qualified financial advisor.