Ultragenyx Pharmaceutical Inc. research snapshot
RARE AI stock analysis as of the August 4, 2026 data cutoff reads Ultragenyx Pharmaceutical as a high-risk pre-profit biotech whose shares fell sharply in late July and now trade below every major moving average. The stock traded at $25.04 on August 3, 2026 at 3:45 PM EDT, up 0.13 or 0.52% from the $24.91 close on July 31, within a session range reported near $24.01 to $25.40 after opening at $24.92, with the daily history table showing an August 3 close of $25.02 up 0.44%, carrying a market capitalization near $2.46 billion, matching the $25.04 price times 98.19 million shares outstanding, with StockAnalysis.com reporting $2.46 billion and Macrotrends reporting $2.453 billion at the July 31 close. The shares peaked near $35.87 on July 7 and fell about 30% into the July 31 close before stabilizing around $25, now sitting below a 20-day moving average near $29.40, a 50-day moving average near $27.44, and a 200-day moving average near $26.98, with a 14-day RSI near 34.55. The Q1 2026 report, released May 5, 2026, showed total revenue of $136 million with Crysvita at $93 million, Dojolvi at $18 million, Evkeeza at $18 million, and Mepsevii at $7 million, a net loss of $185 million or $1.84 per share, cash, cash equivalents, and marketable securities of $534 million as of March 31, 2026, and reaffirmed 2026 revenue guidance of $730 million to $760 million and a path to profitability in 2027. Two gene therapy BLAs are under FDA review with PDUFA dates of August 23, 2026 for DTX401 in GSD Ia and September 19, 2026 for UX111 in MPS IIIA, and the Phase 3 Aspire study of GTX-102 for Angelman syndrome has data expected in the second half of 2026. A securities investigation into statements about the failed setrusumab Phase 3 ORBIT study in osteogenesis imperfecta is also active. The Q2 2026 results and corporate update are scheduled for August 4, 2026 after market close, after this page data cutoff. This is informational research and not investment advice.
Current price
$25.04 at the August 3, 2026, 3:45 PM EDT reading, up 0.13 or 0.52% from the $24.91 July 31 close, within a session range reported near $24.01 to $25.40 after opening at $24.92, with the daily history table showing an August 3 close of $25.02 up 0.44%
Market cap
$2.46 billion reported by StockAnalysis.com at the August 3 reading, matching the $25.04 price times 98.19 million shares outstanding, with Macrotrends reporting $2.453 billion at the July 31 close
AI score
48 / 100
Rating
High-risk pre-profit biotech with near-term catalysts
Trend status
Short-term downtrend below the 20-day, 50-day, and 200-day moving averages after giving back most of the June rally
Data cutoff (updated monthly)
August 4, 2026
Informational use only. This page is not investment advice.
| Dimension | Conclusion | Confidence |
|---|---|---|
| Business quality | Ultragenyx sells four approved rare-disease therapies (Crysvita, Dojolvi, Evkeeza, Mepsevii), with TTM revenue near $670 million and growing, but the business is not yet profitable and carries negative stockholders equity near $236 million. | Medium |
| Moat | The moat comes from orphan drug exclusivity, regulatory infrastructure for rare disease development, modality breadth (biologics, gene therapy, ASO), a commercial sales force, and an in-house gene therapy manufacturing plant, in a niche market with few alternatives. | Medium |
| Management | Founder and CEO Emil Kakkis has led the company since 2010 and the team has brought multiple products to market and guided to 2027 profitability. Key risks are key-person dependence on the founder, dilution, and the overhang from the failed setrusumab ORBIT study and related securities investigation. | Medium |
| Financial trend | FY2025 revenue was $673 million up 20.18%, Q1 2026 revenue was $136 million, and 2026 guidance of $730 million to $760 million was reaffirmed. The company still lost $185 million in Q1 2026, held cash, cash equivalents, and marketable securities of $534 million at March 31, 2026, and carries about $1.2 billion of royalty financing liabilities. | Medium-high |
| Valuation | At about 3.68x trailing revenue and an EV near $3.13 billion or 4.67x EV to sales, the stock prices meaningful pipeline upside. PE and P/FCF are not applicable given negative earnings, and book value is negative. | Low-medium |
| Technical trend | RARE gave back most of its June rally, falling from the July 7 close near $35.87 to the July 31 close of $24.91, and now trades below the 20-day, 50-day, and 200-day moving averages near $29.40, $27.44, and $26.98, with a 14-day RSI near 34.55. | Medium |
| Risk level | Key risks are binary pipeline outcomes, especially the GTX-102 Phase 3 Angelman readout in the second half of 2026, the DTX401 and UX111 PDUFA reviews, a securities investigation tied to the failed setrusumab ORBIT study, cash burn and dilution, negative equity, the $1.2 billion royalty financing liability, and a 13.68% short interest. | Medium-high |
| AI confidence | High for descriptive facts and cross-checked math that matched across StockAnalysis.com, Macrotrends, the Q1 2026 press release, and the Goldman Sachs healthcare conference transcript. Lower for pipeline probability-weighted outcomes because clinical trial results are inherently binary. | High data confidence |
| Investment certainty | Low to medium certainty. Pipeline catalysts could drive significant upside or disappointment, and the stock already trades below all major moving averages, so position sizing should account for binary risk. | Low-medium |
RARE AI stock forecast
The RARE AI stock forecast uses scenario ranges around the $25.04 August 3, 2026 reading. It does not claim that AI can predict a specific future price. The bullish case requires positive Phase 3 data for GTX-102 in Angelman syndrome, approval and successful launches of DTX401 and UX111, and progress toward the 2027 profitability target. The base case assumes mixed pipeline outcomes with some launches landing. The bearish case assumes a GTX-102 setback, a regulatory or launch disappointment, dilutive financing, or worsening litigation overhang. Q2 2026 results are due after the August 4, 2026 data cutoff and could change the ranges.
$50 to $70
More likely if GTX-102 shows strong efficacy in the Phase 3 Angelman readout, DTX401 and UX111 are approved and launch well, Evkeeza and Dojolvi keep growing, and management holds the 2027 profitability path without large dilution.
$30 to $45
More likely if pipeline readouts are mixed, gene therapy launches ramp gradually, the company raises capital at moderate dilution, and the stock trades in line with pre-profit biotech peers after a stabilization above the 200-day moving average.
$14 to $22
More likely if GTX-102 Phase 3 data disappoint, gene therapy approvals or launches slip, the securities investigation expands, cash burn forces dilutive financing, or the broad biotech sector faces risk-off sentiment.
RARE AI technical analysis
RARE AI technical analysis is bearish in the short term as of the August 4, 2026 data cutoff. The stock traded at $25.04 at 3:45 PM EDT on August 3, up 0.13 or 0.52% from the $24.91 July 31 close, after falling from a July 7 close near $35.87. It now sits below a 20-day moving average near $29.40, a 50-day moving average near $27.44, and a 200-day moving average near $26.98, with a 14-day RSI near 34.55. The 5-day and 20-day moving averages near $25.39 and $29.40 were computed from the StockAnalysis.com daily closing price history, and the 50-day and 200-day values come from the StockAnalysis.com statistics page. Because this page does not fetch request-time market data, traders should confirm levels on a live chart before acting.
| Level | Value | Why it matters |
|---|---|---|
| Current price | $25.04 | StockAnalysis.com real-time quote at the August 3, 2026, 3:45 PM EDT reading, up 0.13 or 0.52% from the $24.91 July 31 close within a session range reported near $24.01 to $25.40 after opening at $24.92; the daily history table shows an August 3 close of $25.02 up 0.44%, and the implied market cap near $2.46 billion matches the price times 98.19 million shares outstanding. |
| 5-day moving average | $25.39 | Computed as the simple average of the five closing prices through August 3, 2026 in the StockAnalysis.com daily price history, just above the current price, which signals very short-term momentum is still weak. |
| Near support | $24 to $24.9 | The July 31 close of $24.91, the August 3 intraday low of $24.01, and the June 16 low near $24.51 form a near-term support zone; a close below $24 would open the June consolidation area. |
| Secondary support | $21.4 to $22 | The June consolidation lows near $21.39 on June 8 and $21.78 on June 2 sit below, and a break there would challenge the broader June recovery structure. |
| Major support | $18.29 | The 52-week low set earlier in the cycle remains the last major downside reference below the stock. |
| Near resistance | $26.9 to $27.5 | The StockAnalysis.com 200-day moving average near $26.98 and the 50-day moving average near $27.44, together with the late-July swing highs, form the first resistance cluster above price. |
| Next resistance | $29.4 to $30 | The 20-day moving average near $29.40 and the July 17 to July 20 highs near $30 sit above and mark the next recovery test. |
| 50-day moving average | $27.44 | StockAnalysis.com statistics reported the 50-day moving average near $27.44 on August 3, 2026, above the current price and confirming the medium-term downtrend. |
| 200-day moving average | $26.98 | StockAnalysis.com statistics reported the 200-day trend line near $26.98 on August 3, 2026, above the current price, so the longer-term trend is under pressure until reclaimed. |
| 20-day moving average | $29.40 | Computed as the simple average of the twenty closing prices through August 3, 2026 in the StockAnalysis.com daily price history, well above price and a key recovery reference. |
| Momentum | RSI near 34.5 | The 14-day RSI was near 34.55 on StockAnalysis.com statistics on August 3, 2026, weak but not yet deeply oversold. |
| Volume | About 1.9 million average shares | StockAnalysis.com statistics listed 20-day average volume near 1.86 million shares on August 3, 2026, and the August 3 session showed about 1.03 million shares traded, so the selloff cooled on below-average participation. |
| Volatility | Elevated, low beta | The 14-day realized volatility computed from the StockAnalysis.com daily closing price history was near 65% annualized as of August 3, 2026, with beta near 0.31 on StockAnalysis.com statistics, so position sizing should account for large binary moves around data readouts and PDUFA dates. |
| Invalidation | Close below $24, reclaim above $27.5 | A decisive close below the near support near $24 would open the $21.4 to $22 area and then the $18.29 low. A reclaim and hold above the 50-day and 200-day zone near $27 to $27.5 would start to repair the downtrend. |
RARE AI trading strategy
The RARE AI trading strategy is a rules-based research framework for trading and watching a binary biotech, not personalized advice. It pairs catalyst awareness with technical confirmation, position sizing for clinical and regulatory outcomes, and defined invalidation levels.
Wait for RARE to reclaim and hold above the 50-day and 200-day zone near $27 to $27.5 with rising volume before treating the downtrend as reversing. Use the $29.40 area and then the July highs near $30 as the next confirmation levels.
A failed reclaim of the $27 to $27.5 cluster or a close back below $26 should reduce confidence. Reduce position size ahead of the GTX-102 Phase 3 readout, the August 23 and September 19 PDUFA dates, and the August 4 Q2 report.
If RARE stabilizes near the $24 to $24.9 support with an RSI bounce from oversold levels, evaluate cash position, pipeline timelines, PDUFA dates, and the pending Q2 report before considering a countertrend entry.
Do not average down solely because the stock is cheap. Binary clinical outcomes can drive 30% to 50% moves, negative equity limits downside protection, and the securities investigation can hit sentiment without warning.
Track the Q2 2026 results and call on August 4, 2026 after market close, the DTX401 PDUFA on August 23, 2026, the UX111 PDUFA on September 19, 2026, GTX-102 Phase 3 Aspire data in the second half of 2026, DTX301 second-endpoint data in the first half of 2027, quarterly revenue trends, cash balance, financing announcements, and litigation news.
Lower the rating if cash burn accelerates without pipeline progress, PDUFA timelines slip, the securities investigation expands, or key trial timelines are pushed out significantly.
Investment research summary
Ultragenyx discovers, develops, and commercializes therapies for rare and ultra-rare genetic diseases. Customers are patients, specialty pharmacies, and healthcare systems seeking treatments for conditions with few or no alternatives, with TTM revenue near $670 million across Crysvita, Dojolvi, Evkeeza, and Mepsevii.
The moat is built on orphan drug exclusivity, regulatory expertise in rare disease drug development, broad modality capabilities (biologics, gene therapy, ASO), a commercial sales force, and an in-house gene therapy manufacturing plant. Approved products generate recurring revenue but face long-term competition.
The thesis can fail if GTX-102 or other key pipeline candidates show weak efficacy, gene therapy approvals or launches disappoint, the securities investigation tied to the failed setrusumab ORBIT study expands, cash burn forces dilutive financing, negative equity worsens, or competition erodes approved product economics.
Founder and CEO Emil Kakkis has led the company since 2010 with a focus on rare disease drug development and a stated target of profitability in 2027. The main risks are key-person dependence on the founder, the overhang from the setrusumab ORBIT failure and the related investigation, and the challenge of funding a broad pipeline with limited capital.
Rare disease gene therapy and precision medicine represent a durable secular trend supported by regulatory incentives and improving delivery technologies. Ultragenyx is positioned across multiple rare disease modalities and has two gene therapy BLAs under FDA review, but the sector is vulnerable to binary readouts and financing cycles.
At about 3.68x trailing revenue and an EV near $3.13 billion, the stock prices meaningful pipeline upside. With negative earnings and negative book value, traditional valuation metrics have limited relevance. The margin of safety depends on successful pipeline execution, disciplined capital management, and resolution of the litigation overhang.
Source-backed data
Every metric below includes a source and last verification date.
| Metric | Value | Source | Last verified |
|---|---|---|---|
| RARE price | $25.04 at the August 3, 2026, 3:45 PM EDT reading, up 0.13 or 0.52% from the $24.91 July 31 close, within a session range reported near $24.01 to $25.40 after opening at $24.92, with the daily history table showing an August 3 close of $25.02 up 0.44% | StockAnalysis.com real-time quote and daily price history | August 4, 2026 |
| Market capitalization | Near $2.46 billion, reported by StockAnalysis.com at the August 3 reading and matching the $25.04 price times 98.19 million shares outstanding, with Macrotrends reporting $2.453 billion at the July 31 close | StockAnalysis.com quote and statistics, Macrotrends market cap | August 4, 2026 |
| Enterprise value | $3.13 billion reported by StockAnalysis.com statistics, which minus the $2.46 billion market cap implies net debt near $670 million, consistent with the statistics page net cash figure of negative $666 million | StockAnalysis.com statistics | August 4, 2026 |
| Q1 2026 revenue | Total revenue of $136 million with Crysvita at $93 million, Dojolvi at $18 million, Evkeeza at $18 million, and Mepsevii at $7 million | Ultragenyx Q1 2026 press release | August 4, 2026 |
| Q1 2026 net loss | Net loss of $185 million, or $1.84 per share basic and diluted, compared with a net loss of $151 million or $1.57 per share in Q1 2025 | Ultragenyx Q1 2026 press release | August 4, 2026 |
| Cash and marketable securities | Cash, cash equivalents, and marketable securities of $534 million as of March 31, 2026, down from $737 million at December 31, 2025; the StockAnalysis.com balance sheet breaks this into $175 million cash, $238 million short-term investments, and $121 million long-term investments | Ultragenyx Q1 2026 press release and StockAnalysis.com balance sheet | August 4, 2026 |
| 2026 revenue guidance | Full year 2026 total revenue guidance of $730 million to $760 million reaffirmed, with Crysvita at $500 million to $520 million and Dojolvi at $100 million to $110 million, excluding revenue from potential new product launches | Ultragenyx Q1 2026 press release | August 4, 2026 |
| Operating expense guidance | Combined R&D and SG&A expenses in 2026 expected flat to down low-single digits versus 2025, and 2027 combined R&D and SG&A expenses expected to decrease by at least 15%, supporting a path to profitability in 2027 | Ultragenyx Q1 2026 press release | August 4, 2026 |
| FY2025 revenue | $673 million in FY2025, up 20.18% from $560 million in FY2024, matching StockAnalysis.com financials and the 2025 Impact Report narrative | StockAnalysis.com financials | August 4, 2026 |
| TTM revenue | $670 million over the trailing twelve months ended March 31, 2026, up 13.48%, consistent with the reconciliation FY2025 revenue plus Q1 2026 minus Q1 2025 | StockAnalysis.com financials | August 4, 2026 |
| TTM net income and EPS | TTM net loss of about $609 million and loss per share of $6.11, matching the reconciliation FY2025 EPS of $5.83 plus Q1 2026 EPS of $1.84 minus Q1 2025 EPS of $1.57 | StockAnalysis.com statistics | August 4, 2026 |
| Gross margin | TTM gross margin of 83.52% per StockAnalysis.com financials and an implied product gross margin near 78% from the Q1 2026 press release (cost of sales of $30 million on $136 million of revenue); the statistics page reports a negative TTM gross margin of 28.06% under S&P Global classification, which this page treats as a classification difference | StockAnalysis.com financials, Q1 2026 press release, StockAnalysis.com statistics | August 4, 2026 |
| TTM cash flow | Operating cash flow of negative $497 million and free cash flow of negative $503 million over TTM, with capital expenditures of only $6 million because manufacturing costs are largely expensed in operating expenses | StockAnalysis.com cash flow statement | August 4, 2026 |
| Book value and net cash | Total stockholders equity deficit of $236 million per the balance sheet (statistics page reports $229 million including minority interest), book value per share of negative $2.41, cash and short-term investments of $413 million, total debt of $1.20 billion, and net cash of negative $666 million or negative $6.78 per share per the statistics page | StockAnalysis.com balance sheet and statistics | August 4, 2026 |
| Shares outstanding | 98.19 million shares outstanding as of August 3, 2026, up 6.60% year over year, with a float of 94.61 million and 3.61% owned by insiders | StockAnalysis.com statistics | August 4, 2026 |
| Valuation check | 3.68x trailing revenue, 4.67x EV to sales, and a forward PS ratio of 3.04, with PE and P/FCF not applicable given negative earnings | StockAnalysis.com statistics | August 4, 2026 |
| Analyst consensus | Strong Buy with an average target of $53.37 across 19 analysts, median $48, low $27, and high $96, which is about 113% above the $25.04 price | StockAnalysis.com forecast and analyst ratings | August 4, 2026 |
| Analyst rating mix, July 2026 | 12 Strong Buy, 5 Buy, 2 Hold, and 0 Sell out of 19 analysts | StockAnalysis.com forecast recommendation trends | August 4, 2026 |
| Recent analyst target updates | Goldman Sachs $25 to $27 on July 21, Bank of America Buy maintained on July 27, Wedbush $26 to $30 around July 14 with a Hold rating, RBC Capital $35 to $40 on July 7, J.P. Morgan Buy at $72 on July 7, and Cantor Fitzgerald $84 to $96 about two months ago | StockAnalysis.com forecast and news feed | August 4, 2026 |
| 2026 and 2027 consensus estimates | FY2026 revenue estimate of $748.3 million with EPS of negative $4.19, and FY2027 revenue estimate of $990.93 million with EPS of positive $3.13, implying analyst expectations for profitability in 2027 | StockAnalysis.com forecast financial forecast | August 4, 2026 |
| Short interest | 13.44 million shares short, 13.68% of shares outstanding and 14.20% of float, with 4.24 days to cover, down from 16.22 million the prior month | StockAnalysis.com statistics short selling | August 4, 2026 |
| 52-week range and performance | 52-week range of $18.29 to $39.89, down 12.03% over the trailing 52 weeks, with a beta of 0.31 | StockAnalysis.com quote and statistics | August 4, 2026 |
| Earnings schedule | Q2 2026 financial results and corporate update scheduled for August 4, 2026 at 5:00 PM ET, after this page data cutoff, so Q2 results are not included in this analysis | Ultragenyx press release | August 4, 2026 |
| Securities investigation | Johnson Fistel announced on July 15, 2026 it is investigating potential claims on behalf of long-term shareholders over alleged misstatements tied to setrusumab and the Phase 3 ORBIT study in osteogenesis imperfecta, which missed its primary endpoint earlier in the year | Johnson Fistel press release | August 4, 2026 |
| Technical snapshot | 5-day moving average near $25.39 and 20-day moving average near $29.40 computed from StockAnalysis.com daily price history, 50-day moving average near $27.44 and 200-day moving average near $26.98 on StockAnalysis.com statistics, 14-day RSI near 34.55, 14-day realized volatility near 65% annualized computed from StockAnalysis.com daily closes, and 20-day average volume near 1.86 million shares | StockAnalysis.com statistics and daily price history | August 4, 2026 |
This RARE AI stock analysis is an informational tool for research and education only. It is not investment advice, a recommendation, or a guarantee of future performance. Forecast ranges are scenarios based on available data as of August 4, 2026 and can be wrong. Biotech investments carry elevated risk due to binary clinical trial outcomes, regulatory decisions, and the ongoing securities investigation.
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