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Ultragenyx Pharmaceutical Inc.

35 indications, side-by-side phase, drug & LoA

$24.35

+0.12 (+0.5%)

C 51Pipeline Score Fair Value Pharma · Commercial
Market Cap
2.30 B
EPS
-6.11
P/E Ratio
-
Value Trade
40.12 M
SEC Financials
Q1 2026

  • Dilution Risk

    80%

  • Revenue

    136.00 M

  • R&D Expenses

    187.00 M

  • Operating CF

    -197.00 M

  • Total Assets

    1.30 B

  • Total Liabilities

    1.52 B

  • Equity

    -236.00 M

  • D/E Ratio

    12,345

-9.02 %
Week
-3.78 %
1 Month
8.49 %
3 Month
-25.79 %
6 Month
-79.38 %
5 Year
-48.93 %
All Time
Cash Data
Distressed

  • Cash Position

    413.00 M

  • Monthly Burn

    65.67 M

  • Runway

    5.2 mo

  • Burn Trend

    Accelerating

  • SEC Filing

    May 6, 2026
Overview
Volume
1.85 M
52 Week Range
18.29 - 42.37
% held by Insiders
8.96 %
% held by Institutions
97.7 %
Enterprise Value
3.09 B
Total Shares
96.41 M
Short %
16.4 %
Float Shares
91.50 M
Company Description
HQ: 60 LEVERONI COURT, NOVATO, CA,...
Employees:1,371
https://www.ultragenyx.com

locked

Upcoming Catalyst
Catalyst Drug/Treatment Stage Probability of Approval Description Drug Type Therapeutic Area Source
Regulatory Decision example
UX111 Sanfilippo syndrome type A
BLA
—
Gene Therapies
Rare Diseases
UX111 Sanfilippo syndrome type A
BLA
—
Gene Therapies
Rare Diseases
UX111 Sanfilippo syndrome type A
BLA
—
Gene Therapies
Rare Diseases
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Drug Pipeline Intelligence
C51
Pipeline Score
$1.3B
Pipeline Value
Fair Value
Valuation Signal
17
Drugs Scored
1.9x
rNPV / MCap
Top 73%
Small Cap
(rank 245 of 905)
Percentile Rank
Ultragenyx Pharmaceutical Inc. carries a moderate pipeline score (51/100), with $4.3B risk-adjusted pipeline value, led by Triheptanoin in Adult Polyglucosan Body Disease (Phase 2), but cash runway is a concern.
Showing 1 of 1 assets
DrugIndicationPhaseNCT IDPTRSrNPVStatusEnrollmentVelocityDesignCompletionML SignalLast Change
Triheptanoin
Small molecule		Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)Phase 3NCT0593320055% $1.5B ACTIVE NOT_RECRUITING 69 STALLED B (73) Aug 1, 2027MODERATE_RISKLOW
May 26, 2026
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Clinical Trial Results
Drug Name Indications Phase Date Trial Results Summary Title Source
GTX-102 (apazunersen)
Angelman syndrome
Phase 3
2025-06-27
GTX-102 (apazunersen)
Angelman syndrome
Phase 3
2025-06-27
GTX-102 (apazunersen)
Angelman syndrome
Phase 3
2025-06-27
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Inside Trades
TREND
CORPORATE INSIDERS BOUGHT SHARES WORTH 1.8M IN THE LAST 3 MONTHS
YEARLY INSIDER TRANSACTIONS
Sector Avg.
INSIDERS
SOLDINSIDERS
BOUGHT
POSITIVE SENTIMENT Based on 22 Insiders Transactions
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Show Data
Hedge Funds
TREND
HEDGE FUNDS INCREASED HOLDINGS BY 200.0K SHARES IN THE LAST QUARTER
Shares Held
2040.00B1530.00B1020.00B510.00B0
Q3
2024
Q4
2024
Q1
2025
Q2
2025
HEDGE FUNDS
SOLDHEDGE FUNDS
BOUGHT
POSITIVE SENTIMENT Based on 27 hedge funds in the last quarter
18 buying (3 new)·9 selling (1 exited)·2 unchanged
Fund Count
60%
Share Volume
25%
Conviction
15%
Hedge Funds invested in RARE
HedgeFund Name
( 3 )
% of Portfolio Current MV
-
Shares Owned
-
Activity
Avg Price $0

Example Capital Management

2.5 %
15.00 M
250.00 K

Example Capital Management

2.5 %
15.00 M
250.00 K

Example Capital Management

2.5 %
15.00 M
250.00 K
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Biotech Analyst Ratings
Symbol
Firm
Rating
Action
Price Target
Upside
date
RARE
Example Securities
Buy
Initiated
$150.00
+25%
2026-01-15
RARE
Example Securities
Buy
Initiated
$150.00
+25%
2026-01-15
RARE
Example Securities
Buy
Initiated
$150.00
+25%
2026-01-15
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RARE Stock Forecast & Analyst Consensus
BUY
Analyst Ratings
Buy65.0%
Hold25.0%
Sell10.0%
Price Target Trend
Average$24.00
Low$18.00
High$32.00
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RARE Institutional Ownership Trends
Current Insider %
5.20%
—+0.00%
Current Institutional %
62.40%
—+0.00%
Total Ownership
67.60%
Insider + Institutional
Data Points
1
1 Ticker(s)
Option Chain Statistics
ExpirationVolumeOpen InterestImplied Volatility CallsImplied Volatility Puts
CallsPutsPut-Call RatioCallsPutsPut-Call RatioIVOiWaIvVWaIvIVOiWaIvVWaIv
2026-09-18 0.00 0 - - - - - -
2026-09-18 0.00 0 - - - - - -
2026-09-18 0.00 0 - - - - - -
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Option Chain
CallsStrikePuts
Last PriceVolumeOpen InterestLast PriceVolumeOpen Interest
No data available
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Open interest
0 600K 1.2M 1.8M Avg OI 1.00 M Open Interest
0 1 2 3 1.1 Put-Call Ratio
Today's Open Interest
1.00 M
Put-Call Ratio
1.1
Put Open Interest
480.00 K
Call Open Interest
520.00 K
Open Interest Avg (30-day)
900,000
Today vs Open Interest Avg (30-day)
11.11%
Option Volume
0 450K 900K 1.4M Avg OV 750.00 K Option Volume
0 1 2 3 0.95 Put-Call Ratio
Today's Volume
750.00 K
Put-Call Ratio
0.95
Put Volume
360.00 K
Call Volume
390.00 K
Volume Avg (30-day)
800,000
Today vs Volume Avg (30-day)
-6.25%
Latest Ultragenyx Pharmaceutical Inc. (RARE) News & Alerts
-
Saturday, June 13, 2026
(%)
+ More

Demo Biotech Announces Positive Phase 3 Results

Competitive Position
How RARE ranks across every disease it competes in
Indication Rank Phase Best DrugTop 3 Competitors Market $B LoA Position
Rare Neurological Disorders #4 of 46 PHASE 3 GTX-102
IONS BIIB SRPT
$139.4B 0.88 CONTENDER
Rare Metabolic Disorders #4 of 30 PHASE 3 DTX301 + Corticosteroids
AZN SNY TVTX
$21.15B 0.85 CONTENDER
Rare Genetic Disorders #5 of 61 PHASE 3 setrusumab
BMRN ASND NVS
—0.84 CONTENDER