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RARE · NASDAQ · Biotechnology

Ultragenyx Pharmaceutical

Develops and commercializes therapies for serious rare and ultra-rare genetic diseases.

$25.68

After-hours close+0.07 (+0.27%)

At close$25.61(−2.48%)

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Profile

Ultragenyx Pharmaceutical Inc.RAREHealth Care

Develops and commercializes approved therapies and product candidates for serious rare and ultra-rare genetic diseases. Its operating model combines patient and caregiver engagement with collaborations, acquisitions, and commercial execution.

Identifies, acquires, develops, and commercializes therapies for serious rare and ultra-rare genetic diseases with clear biology and high unmet need. The portfolio focuses on diseases where the biology is clear and unmet need is high. Its approved portfolio includes Mepsevii, Crysvita, and Dojolvi, alongside a broader product-candidate portfolio and patient and caregiver engagement that informs clinical protocol, endpoint, analysis, commercialization, and access approaches.

Founder Emil D. Kakkis has remained President and CEO since the 2010 formation, providing continuity through the public-market and commercial stages. Strategic collaborations and targeted acquisitions add capabilities, while product approvals and collaboration arrangements shape operations.

Company facts

CEOEmil D. Kakkis, M.D., Ph.D.

ListedJan 31, 2014

ExchangeNASDAQ

HeadquartersNovato, CA

CountryUnited States

Employees1,371

CIK0001515673

ISINUS90400D1081

Key statistics

Market cap$2.52B

EPS (ttm)−$5.84

P/E (ttm)−4.39

ROE392.4%

P/B−8.97

P/S3.52

Dividend yield—

Beta1.44vs XLV 0.62

Year to date +8.5% · Average volume (30d) 2.0M

Analysts

Price target, next 12 months$46.50+81.6%Median of 33 analysts · vs the Aug 28 close

ConsensusBuy26 of 33 analysts rate it buy or better

Ratings

Strong buyBuyHoldSellStrong sell

Target range

Last close $25.61

High$83.00+224.1%

Median$46.50+81.6%

Low$30.00+17.1%

12 months agoNowIn 12 months

Latest analyst notes

Date	Firm	Action	Rating	Prior target	Target
Aug 28, 2026	Wells Fargo	Target		$46	$50
Aug 21, 2026	Morgan Stanley	Reiterate	Overweight	$67	$74
Aug 20, 2026	Canaccord Genuity	Target		$111	$83
Aug 20, 2026	Wells Fargo	Reiterate	Overweight	$45	$46
Aug 4, 2026	Guggenheim	Reiterate	Buy	$43	$35

Earnings

EPS

Estimate−$0.63

ReportedDue Nov 3, 2026

Revenue

Estimate$184.1M

ReportedDue Nov 3, 2026

History

Report date	EPS est	EPS	Surprise	Revenue est	Revenue	Surprise	Next session
Aug 4, 2026Q2 FY26	−$1.22	−$0.90	+26.2%	$183.1M	$214.0M	+16.9%	−3.41%
May 5, 2026Q1 FY26	−$1.49	−$1.84	−23.7%	$158.2M	$136.0M	−14.0%	+3.68%
Feb 12, 2026Q4 FY25	−$1.20	−$1.29	−7.5%	$198.6M	$207.0M	+4.2%	−12.68%
Nov 4, 2025Q3 FY25	−$1.23	−$1.81	−47.2%	$199.8M	$159.9M	−19.9%	−1.82%

View details

Financial highlights

	Q1 FY25	Q2 FY25	Q3 FY25	Q4 FY25	Q1 FY26	Q2 FY26
Revenue	$139.3M	$166.5M	$159.9M	$207.0M	$136.0M	$214.0M
Gross profit	$110.6M	$143.5M	$131.9M	$178.0M	$106.0M	$180.0M
Operating income	−$142.9M	−$107.9M	−$170.9M	−$114.0M	−$169.0M	−$75.0M
Net income	−$151.1M	−$115.0M	−$180.4M	−$129.0M	−$185.0M	−$92.0M
Diluted EPS	−$1.57	−$1.17	−$1.81	−$1.29	−$1.84	−$0.90
Gross margin	79.4%	86.2%	82.5%	86.0%	77.9%	84.1%
Operating margin	-102.6%	-64.8%	-106.9%	-55.1%	-124.3%	-35.0%

Fiscal years (4-quarter sums)FY21 $351.4M · FY22 $363.3M · FY23 $434.2M · FY24 $560.2M · FY25 $672.7M

Revenue by product · Q2 FY26

Product$112.0M · 52%

Royalty$102.0M · 48%

Revenue by geography · Q2 FY26

North America$121.0M · 57%

Latin America$51.0M · 24%

EMEA$35.0M · 16%

Asia Pacific$7.0M · 3%

Balance sheet, Jun 30, 2026Cash & short-term investments $292.0M · Total debt $1.20B · Total assets $1.26B · Shareholders' equity −$291.0M

Dividends

No dividends on record.

Ownership

Institutions 112.44%Insiders 4.22%

A 13F quarter can total more than the shares outstanding — sub-advisers file the same block twice, and a filer's own share count can be a quarter stale. No split is drawn from it.

Shares outstanding 98.49M · Float 94.61M · 96.1% free float

Top shareholders13F · 13D/G · Form 4

Holder	Shares	% outstanding	Form	Filed
BlackRock, Inc.	10.65M	10.82%	13F	Aug 7, 2026
RTW Investments, LP	7.80M	7.90%	13G	Aug 14, 2026
Vanguard Portfolio Management LLC	6.79M	6.89%	13F	Aug 13, 2026
AQR Capital Management, LLC	6.56M	6.66%	13G	Aug 13, 2026
State Street Corporation	6.16M	6.30%	13G	Aug 7, 2026

Insider tradesForm 4

Date	Insider	Role	Shares	Avg price
Aug 3, 2026	Howard Horn	officer: Chief Financial Officer	−4,696	$24.92
Aug 3, 2026	Karah Herdman Parschauer	officer: EVP and Chief Legal Officer	−1,900	$24.92
Jul 1, 2026	Howard Horn	officer: Chief Financial Officer	−4,698	$33.13
Jun 15, 2026	Karah Herdman Parschauer	officer: EVP and Chief Legal Officer	−1,899	$24.62
Jun 15, 2026	Corazon (Corsee) D. Sanders	director	−2,000	$25.05