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Ultragenyx Pharmaceutical Inc

Feb 03 • 04:00PM ET

24.80

+0.04 0.16 %

Healthcare • Biotechnology • USA • NASD

Feb 03, 8:07 AMUltragenyx reports new longer-term UX111 gene therapy data showing sustained significant CSF-HS reductions and continued clinically meaningful functional improvements in children with Sanfilippo syndrome type A.

Peers: BIIB VRTX ALNY BMRN AMGN REGN IONS CRSP NBIX PBYI

| Held by: XBI VTI VB VBK VXF IBB IWP SCHA IWF IWR

Scroll to Statements

Index	-	P/E	-	EPS (ttm)	-5.94	Insider Own	6.94%	Shs Outstand	96.27M	Perf Week	0.73%
Market Cap	2.39B	Forward P/E	-	EPS next Y	-3.78	Insider Trans	-0.30%	Shs Float	89.78M	Perf Month	5.08%
Enterprise Value	2.83B	PEG	-	EPS next Q	-1.10	Inst Own	99.49%	Short Float	9.87%	Perf Quarter	-28.32%
Income	-579.83M	P/S	3.79	EPS this Y	10.86%	Inst Trans	3.31%	Short Ratio	3.61	Perf Half Y	-12.86%
Sales	630.60M	P/B	260.67	EPS next Y	32.61%	ROA	-42.50%	Short Interest	8.86M	Perf YTD	7.83%
Book/sh	0.10	P/C	5.50	EPS next 5Y	40.10%	ROE	-325.76%	52W High	46.50 -46.67%	Perf Year	-42.37%
Cash/sh	4.51	P/FCF	-	EPS past 3/5Y	2.09% 2.45%	ROIC	-72.97%	52W Low	18.41 34.71%	Perf 3Y	-43.28%
Dividend Est.	-	EV/EBITDA	-	Sales past 3/5Y	16.82% 40.12%	Gross Margin	79.09%	Volatility	4.60% 5.17%	Perf 5Y	-81.79%
Dividend TTM	-	EV/Sales	4.48	EPS Y/Y TTM	8.09%	Oper. Margin	-86.27%	ATR (14)	1.39	Perf 10Y	-66.34%
Dividend Ex-Date	-	Quick Ratio	1.74	Sales Y/Y TTM	20.63%	Profit Margin	-91.95%	RSI (14)	46.16	Recom	1.18
Dividend Gr. 3/5Y	- -	Current Ratio	1.89	EPS Q/Q	-29.33%	SMA20	4.36%	Beta	0.15	Target Price	64.00
Payout	-	Debt/Eq	94.23	Sales Q/Q	14.65%	SMA50	-14.61%	Rel Volume	0.84	Prev Close	24.76
Employees	1294	LT Debt/Eq	85.75	Earnings	Nov 04 AMC	SMA200	-22.19%	Avg Volume	2.45M	Price	24.80
IPO	Jan 31, 2014	Option/Short	Yes / Yes	EPS/Sales Surpr.	-46.69% -4.13%	Trades		Volume	2,059,803	Change	0.16%

Date	Action	Analyst	Rating Change	Price Target Change
Oct-20-25	Initiated	Wells Fargo	Overweight	$65
Jul-28-25	Resumed	H.C. Wainwright	Buy	$80
May-28-25	Initiated	William Blair	Outperform
Jun-06-24	Upgrade	Goldman	Neutral → Buy	$56 → $67
Apr-22-24	Initiated	RBC Capital Mkts	Outperform	$77
Dec-08-23	Initiated	Wells Fargo	Overweight	$72
Jun-14-23	Resumed	Credit Suisse	Outperform	$96
Jun-06-23	Upgrade	Evercore ISI	In-line → Outperform	$60 → $80
Apr-26-23	Initiated	Cantor Fitzgerald	Overweight	$114
Jan-18-23	Resumed	Canaccord Genuity	Buy	$90
Dec-30-22	Resumed	H.C. Wainwright	Buy	$82
Nov-03-22	Upgrade	Robert W. Baird	Neutral → Outperform	$50
Oct-13-22	Upgrade	Guggenheim	Neutral → Buy	$50
Aug-01-22	Downgrade	Evercore ISI	Outperform → In-line	$95 → $60
Mar-16-22	Upgrade	Credit Suisse	Neutral → Outperform	$99 → $105
Feb-11-22	Upgrade	JP Morgan	Neutral → Overweight	$132
Sep-30-21	Initiated	H.C. Wainwright	Buy	$106
Aug-19-21	Initiated	UBS	Sell	$73
Jul-15-21	Initiated	Guggenheim	Neutral
Jun-29-21	Upgrade	BofA Securities	Neutral → Buy	$162

Show Previous Ratings

Feb-03-26 12:58PM	Ultragenyx Says Gene Therapy Shows Lasting Benefits, Slows Brain Decline In Fatal Childhood Disorder (Benzinga)
08:00AM	Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA) (GlobeNewswire)
Feb-02-26 11:03AM	Ultragenyx Pharmaceutical Resubmits UX111 BLA for Sanfilippo Syndrome (Zacks)
Jan-30-26 08:30AM	Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA (GlobeNewswire)
Jan-23-26 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
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02:05PM	Ultragenyx Stock Declines After Missing Primary Endpoints on its Phase III Orbit and Cosmic Studies (PR Newswire)
Jan-21-26 04:58PM	Cathie Wood Has 13 Big Ideas For 2026: Here's The Ark Invest List And Stocks To Watch (Benzinga)
Jan-12-26 10:11AM	Morgan Stanley Trims Ultragenyx (RARE) PT to $50 While Betting on 2026 Biotech Recovery (Insider Monkey) +8.26%
07:30AM	Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference (GlobeNewswire)
Jan-09-26 03:16AM	Analysts Lower Ultragenyx (RARE) Target but Stay Bullish (Insider Monkey)
Jan-07-26 04:05PM	Ultragenyx to Present at the 44th Annual J.P. Morgan Healthcare Conference (GlobeNewswire) +7.36%
Jan-05-26 03:56AM	Novo launches Wegovy pill; Argenx to swap CEOs (BioPharma Dive)
Dec-31-25 09:50AM	RARE Rises on Completion of Rolling Submission for AAV Gene Therapy (Zacks)
09:40AM	DigitalBridge downgraded, Commvault initiated: Wall Streets top analyst calls (TipRanks)
09:01AM	Ultragenyx (RARE) Moves 15.5% Higher: Will This Strength Last? (Zacks)
07:37AM	Loading…
07:37AM	Ultragenyx (RARE) Rockets 15.5% on Bargain-Hunting After Steep Fall (Insider Monkey)
Dec-30-25 04:31PM	Stocks Fall Slightly in Thin Holiday Trade (Barchart) +15.52%
04:05PM	Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa) (GlobeNewswire)
03:58PM	Why Ultragenyx Pharmaceutical Stock Is Bouncing Back Today (Motley Fool)
12:39PM	Why Ultragenyx Stock Dropped 42% and What Comes Next (Barrons.com)
11:06AM	Stocks Slip as Bond Yields Rise (Barchart)
11:00AM	RARE, MREO Tank as Osteogenesis Study Fails to Meet Primary Endpoint (Zacks)
09:36AM	DigitalBridge downgraded, Commvault initiated: Wall Street's top analyst calls (The Fly)
08:11AM	Company News for Dec 30, 2025 (Zacks)
06:17AM	S&P Futures Tread Water Ahead of FOMC Meeting Minutes (Barchart)
Dec-29-25 04:21PM	These Stocks Moved the Most Today: Nvidia, Tesla, Newmont, DigitalBridge, Praxis Precision, Ultragenyx, and More (Barrons.com) -42.32%
03:34PM	Why Ultragenyx Pharmaceuticals Stock Is Plummeting Today (Motley Fool)
01:18PM	These Stocks Are Moving the Most Today: Nvidia, Tesla, Newmont, DigitalBridge, Praxis Precision, Ultragenyx, and More (Barrons.com)
01:05PM	These biotech stocks are getting hammered by shocking brittle-bone treatment study results (MarketWatch)
08:30AM	Ultragenyx Announces Phase 3 Orbit and Cosmic Results for Setrusumab (UX143) in Osteogenesis Imperfecta (GlobeNewswire)
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Dec-19-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Dec-11-25 07:44AM	Is Ultragenyx Pharmaceutical Inc. (RARE) One of the Best Biotech Stocks to Buy According to Wall Street Analysts? (Insider Monkey)
Dec-04-25 11:30AM	Ultragenyx (RARE) Up 14.4% Since Last Earnings Report: Can It Continue? (Zacks)
Nov-28-25 11:57AM	Barclays Remains Cautious on Ultragenyx (RARE), Cites Setrusumab Study Analysis Despite 70% Positive Result Outlook (Insider Monkey)
Nov-24-25 04:30PM	Ultragenyx to Participate in Investor Conferences in December (GlobeNewswire)
Nov-21-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire) +7.73%
Nov-10-25 08:19PM	Morgan Stanley Reaffirms "Buy" Rating on Ultragenyx Pharmaceutical (RARE) with $55 PT (Insider Monkey)
Nov-05-25 10:48AM	Ultragenyx Q3 Earnings and Sales Miss Estimates, Increase Y/Y (Zacks)
12:10AM	Ultragenyx Pharmaceutical Inc (RARE) Q3 2025 Earnings Call Highlights: Strong Revenue Growth ... (GuruFocus.com)
Nov-04-25 05:30PM	Here's What Key Metrics Tell Us About Ultragenyx (RARE) Q3 Earnings (Zacks)
05:15PM	Ultragenyx (RARE) Reports Q3 Loss, Misses Revenue Estimates (Zacks)
04:13PM	Ultragenyx: Q3 Earnings Snapshot (Associated Press Finance)
04:05PM	Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update (GlobeNewswire)
04:01PM	Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita (burosumab) for $400 Million to OMERS Life Sciences (GlobeNewswire)
Oct-30-25 08:00AM	Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups (GlobeNewswire)
Oct-29-25 10:00AM	Geron (GERN) May Report Negative Earnings: Know the Trend Ahead of Next Week's Release (Zacks)
Oct-28-25 04:30PM	Ultragenyx to Host Conference Call for Third Quarter 2025 Financial Results and Corporate Update (GlobeNewswire)
10:00AM	Will Ultragenyx (RARE) Report Negative Q3 Earnings? What You Should Know (Zacks)
Oct-24-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire) +5.31%
Oct-17-25 09:16AM	Strength Seen in DBV Technologies (DBVT): Can Its 6.8% Jump Turn into More Strength? (Zacks)
Oct-08-25 12:57AM	Bank of America Securities Remains Bullish on Ultragenyx Pharmaceutical (RARE) (Insider Monkey)
Oct-03-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Oct-02-25 09:45AM	Assenagon Boosts Ultragenyx Pharmaceutical Inc. (RARE) Stake by 439% Amid Breakthrough Pipeline (Insider Monkey)
Sep-30-25 04:05PM	Ultragenyx Appoints Eric Olson as Chief Business Officer and Executive Vice President (GlobeNewswire)
04:00AM	Cantor Fitzgerald Reiterates a Buy Rating on Ultragenyx Pharmaceutical (RARE) (Insider Monkey)
Sep-29-25 08:28AM	FDA Approves Label Extension of REGN's Cholesterol Drug Evkeeza (Zacks)
Sep-19-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Sep-16-25 09:17AM	Is Ultragenyx Pharmaceutical (RARE) a Compelling Long-Term Opportunity? (Insider Monkey)
Sep-10-25 11:11AM	IONS Up on FDA's Breakthrough Tag for ION582 in Angelman Syndrome (Zacks)
Sep-09-25 11:12AM	RARE Reports Positive Long-Term Data From Metabolic Disorder Study (Zacks)
Sep-08-25 04:05PM	Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa) (GlobeNewswire)
Aug-29-25 04:30PM	Ultragenyx to Participate at Investor Conferences in September (GlobeNewswire) +6.91%
Aug-21-25 05:00PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Aug-18-25 08:00AM	Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa) (GlobeNewswire)
Aug-06-25 11:43AM	Ultragenyx's Q2 Loss Narrower Than Expected, Revenues Rise Y/Y (Zacks)
03:29AM	Ultragenyx Pharmaceutical Inc (RARE) Q2 2025 Earnings Call Highlights: Strong Revenue Growth ... (GuruFocus.com)
Aug-05-25 05:30PM	Ultragenyx (RARE) Q2 Earnings: Taking a Look at Key Metrics Versus Estimates (Zacks)
04:06PM	Ultragenyx: Q2 Earnings Snapshot (Associated Press Finance)
04:01PM	Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update (GlobeNewswire)
Aug-04-25 03:32PM	Ultragenyx Pharmaceutical Inc. (RARE): A Bull Case Theory (Insider Monkey)
Jul-31-25 04:05PM	Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome (GlobeNewswire)
11:35AM	Ultragenyx Pharmaceutical Inc. (RARE) Gets FDA CRL for UX111, Vows Quick Resubmission (Insider Monkey)
10:00AM	Earnings Preview: Verastem (VSTM) Q2 Earnings Expected to Decline (Zacks)
Jul-30-25 08:30AM	Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update (GlobeNewswire)
Jul-24-25 11:09AM	BioHarvest Sciences Inc. (BHST) Surges 5.6%: Is This an Indication of Further Gains? (Zacks)
Jul-22-25 10:05AM	FDA Rejects Replimunes Skin Cancer Treatment, Shares Sink (Bloomberg)
Jul-18-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Jul-17-25 10:38AM	Bank of America Securities Reiterated a Buy Rating on Ultragenyx Pharmaceutical (RARE) (Insider Monkey)
Jul-16-25 09:38AM	William Blair Maintains a Buy on Ultragenyx Pharmaceutical (RARE) (Insider Monkey)
07:00AM	New Pedestrian Access from Inner Belt to East Somerville MBTA Station Now Open to the Public (PR Newswire)
Jul-15-25 08:28AM	Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA (Zacks) -6.94%
05:46AM	Health Canada extends Evkeeza approval for children with HoFH (Pharmaceutical Technology)
Jul-14-25 04:24PM	Ultragenyx Pharma Takes Another Hit As FDA Slaps Its Gene Therapy With A Rejection (Investor's Business Daily)
08:00AM	Health Canada Extends the Approval of Evkeeza (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH) (GlobeNewswire)
07:53AM	FDA rejects Ultragenyx gene therapy over manufacturing concerns (BioPharma Dive)
07:34AM	Ultragenyx receives complete response letter for UX111 BLA (Pharmaceutical Technology)
Jul-13-25 03:12PM	Ultragenyx (RARE) Falls 26% as New Treatment Fails to Get FDA Green Light (Insider Monkey)
Jul-11-25 04:30PM	Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) (GlobeNewswire)
07:51AM	Jazz names new CEO; Ultragenyx, Mereo shares slide on trial update (BioPharma Dive)
Jul-10-25 04:17PM	Ultragenyx, Mereo Hammered For Their Surprise Miss In Brittle Bone Disease (Investor's Business Daily) -25.11%
10:37AM	Biotech Stock Ultragenyx Is Falling on Unsatisfying Trial Update (Barrons.com)
10:17AM	RARE, MREO Fall as Osteogenesis Study Likely to Continue Till Year End (Zacks)
09:19AM	Ultragenyx (RARE) Surges 5.1%: Is This an Indication of Further Gains? (Zacks)
07:52AM	Kellogg shares soar on buyout talk, and other early market movers (MarketWatch)
Jul-09-25 04:05PM	Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis (GlobeNewswire) +5.12%
12:55AM	Ultragenyx's GTX-102 Receives FDA Breakthrough Therapy Designation for Angelman Syndrome (Insider Monkey)
Jun-30-25 11:40AM	RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome (Zacks)
Jun-27-25 08:00AM	Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome (GlobeNewswire)
Jun-20-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Jun-05-25 11:31AM	Ultragenyx (RARE) Up 9.6% Since Last Earnings Report: Can It Continue? (Zacks)

Ultragenyx Pharmaceutical, Inc. is a biopharmaceutical company, which engages in the identification, acquisition, development, and commercialization of novel products for the treatment of genetic diseases. Its products include Crysvita, Mepsevii, Dojolvi, and Evkeeza. The company was founded by Emil D. Kakkis in April 2010 and is headquartered in Novato, CA.

Insider Trading	Relationship	Date	Transaction	Cost	#Shares	Value ($)	#Shares Total	SEC Form 4
Horn Howard	Chief Financial Officer	Feb 02 '26	Sale	23.56	3,061	72,117	88,935	Feb 03 12:00 PM
HOWARD HORN	Officer	Feb 02 '26	Proposed Sale	23.56	3,061	72,117		Feb 02 04:09 PM
Horn Howard	Chief Financial Officer	Jan 02 '26	Sale	22.84	3,150	71,946	91,996	Jan 05 04:07 PM
HOWARD HORN	Officer	Jan 02 '26	Proposed Sale	22.84	3,150	71,946		Jan 02 04:10 PM
Huizenga Theodore Alan	SVP, Chief Accounting Officer	Dec 29 '25	Sale	34.38	85	2,922	50,450	Dec 30 01:49 PM
Horn Howard	Chief Financial Officer	Dec 10 '25	Sale	36.38	3,081	112,087	95,146	Dec 11 12:10 PM
HOWARD HORN	Officer	Dec 10 '25	Proposed Sale	36.38	3,081	112,087		Dec 10 04:05 PM
Horn Howard	Chief Financial Officer	Oct 13 '25	Sale	31.51	7,942	250,252	98,227	Oct 14 12:15 PM
Huizenga Theodore Alan	SVP, Chief Accounting Officer	Sep 18 '25	Sale	29.17	64	1,867	50,242	Sep 19 06:23 PM
KARAH PARSCHAUER	Officer	Sep 12 '25	Proposed Sale	31.17	2,450	76,366		Sep 12 04:17 PM
Parschauer Karah Herdman	EVP and Chief Legal Officer	Sep 12 '25	Sale	31.17	2,450	76,366	73,271	Sep 12 04:11 PM
Huizenga Theodore Alan	SVP, Chief Accounting Officer	Sep 05 '25	Sale	31.39	84	2,637	50,306	Sep 08 02:01 PM
Sanders Corazon (Corsee) D.	Director	Jun 20 '25	Sale	37.39	2,405	89,923	15,344	Jun 23 01:50 PM
CORAZON D. SANDERS	Director	Jun 20 '25	Proposed Sale	37.39	2,405	89,923		Jun 20 04:02 PM
Crombez Eric	EVP and Chief Medical Officer	May 05 '25	Sale	39.24	520	20,405	71,530	May 05 07:18 PM
Crombez Eric	EVP and Chief Medical Officer	Apr 21 '25	Sale	35.11	242	8,497	71,680	May 05 07:18 PM
Horn Howard	Chief Financial Officer	Mar 06 '25	Sale	40.40	1,785	72,114	106,169	Mar 10 12:53 PM
Kassberg Thomas Richard	CBO & EVP	Mar 03 '25	Sale	42.10	6,028	253,779	265,238	Mar 04 08:19 PM
Parschauer Karah Herdman	EVP and Chief Legal Officer	Mar 03 '25	Sale	42.10	12,846	540,817	75,287	Mar 04 08:18 PM
Crombez Eric	EVP and Chief Medical Officer	Mar 03 '25	Sale	42.10	8,945	376,584	71,922	Mar 04 08:16 PM
Pinion John Richard	See Remarks	Mar 03 '25	Sale	42.10	14,439	607,882	107,766	Mar 04 08:12 PM
KAKKIS EMIL D	President & CEO	Mar 03 '25	Sale	42.10	73,434	3,091,571	641,731	Mar 04 08:10 PM
KAKKIS EMIL D	President & CEO	Feb 28 '25	Sale	42.48	25,000	1,062,000	2,158,985	Mar 04 08:10 PM
Harris Erik	EVP & Chief Commercial Officer	Mar 03 '25	Sale	42.10	15,103	635,836	87,855	Mar 04 08:06 PM
Huizenga Theodore Alan	SVP, Chief Accounting Officer	Mar 03 '25	Sale	42.10	967	40,711	50,265	Mar 04 08:00 PM
Parschauer Karah Herdman	EVP and Chief Legal Officer	Feb 27 '25	Sale	42.88	2,990	128,224	54,991	Mar 03 01:58 PM
KAKKIS EMIL D	Officer	Feb 28 '25	Proposed Sale	42.48	25,000	1,062,100		Feb 28 04:11 PM
Parschauer Karah Herdman	Officer	Feb 27 '25	Proposed Sale	42.88	2,990	128,215		Feb 27 05:08 PM

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Ultragenyx Pharmaceutical Inc

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