RARE

Ultragenyx Pharmaceutical Inc

Oct 21 • 11:26AM ET

34.68

+0.28 0.81 %

Healthcare • Biotechnology • USA • NASD

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

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

Index	-	P/E	-	EPS (ttm)	-5.53	Insider Own	7.07%	Shs Outstand	96.18M	Perf Week	9.44%
Market Cap	3.34B	Forward P/E	-	EPS next Y	-3.64	Insider Trans	-0.20%	Shs Float	89.55M	Perf Month	17.68%
Enterprise Value	3.74B	PEG	-	EPS next Q	-1.23	Inst Own	96.78%	Short Float	8.55%	Perf Quarter	23.81%
Income	-532.93M	P/S	5.48	EPS this Y	20.67%	Inst Trans	2.70%	Short Ratio	4.47	Perf Half Y	-3.43%
Sales	610.16M	P/B	22.05	EPS next Y	26.98%	ROA	-36.44%	Short Interest	7.65M	Perf YTD	-17.57%
Book/sh	1.57	P/C	6.73	EPS next 5Y	45.45%	ROE	-182.60%	52W High	56.38 -38.49%	Perf Year	-38.91%
Cash/sh	5.15	P/FCF	-	EPS past 3/5Y	2.09% 2.45%	ROIC	-55.60%	52W Low	25.81 34.37%	Perf 3Y	-6.42%
Dividend Est.	-	EV/EBITDA	-	Sales past 3/5Y	16.82% 40.12%	Gross Margin	79.51%	Volatility	4.57% 4.37%	Perf 5Y	-62.58%
Dividend TTM	-	EV/Sales	6.12	EPS Y/Y TTM	24.18%	Oper. Margin	-82.78%	ATR (14)	1.28	Perf 10Y	-57.96%
Dividend Ex-Date	-	Quick Ratio	2.30	Sales Y/Y TTM	26.77%	Profit Margin	-87.34%	RSI (14)	65.74	Recom	1.19
Dividend Gr. 3/5Y	- -	Current Ratio	2.45	EPS Q/Q	23.19%	SMA20	10.99%	Beta	0.28	Target Price	86.10
Payout	-	Debt/Eq	5.84	Sales Q/Q	13.24%	SMA50	13.92%	Rel Volume	0.49	Prev Close	34.40
Employees	1294	LT Debt/Eq	5.34	Earnings	Aug 05 AMC	SMA200	-2.15%	Avg Volume	1.71M	Price	34.68
IPO	Jan 31, 2014	Option/Short	Yes / Yes	EPS/Sales Surpr.	9.81% 2.77%	Trades		Volume	281,715	Change	0.81%

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

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)
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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)
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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)
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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)
Jun-03-25 05:00PM	Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference (GlobeNewswire)
May-20-25 05:00PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire) -5.19%
May-14-25 04:30PM	Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report (GlobeNewswire)
May-08-25 10:25AM	Ultragenyx price target lowered to $100 from $140 at Truist (TipRanks) +7.66%
May-07-25 05:00PM	Ultragenyx to Participate at Bank of America's 2025 Healthcare Conference (GlobeNewswire)
12:15PM	Q1 2025 Ultragenyx Pharmaceutical Inc Earnings Call (Thomson Reuters StreetEvents)
10:45AM	Ultragenyx's Q1 Loss Wider Than Expected, Revenues Increase Y/Y (Zacks)
03:23AM	Ultragenyx Pharmaceutical Inc (RARE) Q1 2025 Earnings Call Highlights: Strong Revenue Growth ... (GuruFocus.com)
May-06-25 11:32PM	Ultragenyx projects $640M-$670M revenue for 2025 with strong product growth (Seeking Alpha) -9.80%
11:31PM	Ultragenyx Pharmaceutical Inc. (RARE) Q1 2025 Earnings Call Transcript (Seeking Alpha)
06:00PM	Ultragenyx (RARE) Reports Q1 Earnings: What Key Metrics Have to Say (Zacks)
05:10PM	Ultragenyx (RARE) Reports Q1 Loss, Misses Revenue Estimates (Zacks)
04:01PM	Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update (GlobeNewswire)
May-05-25 09:13AM	5 Drug/Biotech Stocks Likely to Outperform Q1 Earnings Estimates (Zacks)
Apr-30-25 05:00PM	Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update (GlobeNewswire)
Apr-29-25 10:01AM	Jazz Pharmaceuticals (JAZZ) Earnings Expected to Grow: Should You Buy? (Zacks)
Apr-24-25 10:01AM	Ultragenyx (RARE) Expected to Beat Earnings Estimates: What to Know Ahead of Q1 Release (Zacks)
Apr-22-25 05:00PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Apr-21-25 10:00AM	Pz-Cel Has Massive Potential For RDEB Patients (Seeking Alpha)
Apr-16-25 09:49AM	Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline (Seeking Alpha)
Apr-13-25 08:50PM	Ultragenyx Pharmaceutical (RARE): Among Billionaire Stanley Druckenmiller's Top Stock Picks with Huge Upside Potential (Insider Monkey)
Apr-10-25 09:30AM	Ultragenyx (RARE) Soars 9.2%: Is Further Upside Left in the Stock? (Zacks)
Mar-29-25 04:20PM	Why Ultragenyx Pharmaceutical (RARE) Is Among the Best Mid Cap Biotech Stocks to Buy (Insider Monkey)
Mar-19-25 05:00PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Mar-18-25 09:16AM	Ultragenyx price target lowered to $115 from $140 at Piper Sandler (TipRanks)
Feb-24-25 04:30PM	Ultragenyx to Participate at Investor Conferences in March (GlobeNewswire)
Feb-19-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Feb-18-25 08:00AM	Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) (GlobeNewswire)
Feb-14-25 09:48AM	Q4 2024 Ultragenyx Pharmaceutical Inc Earnings Call (Thomson Reuters StreetEvents)
02:23AM	Ultragenyx Pharmaceutical Inc (RARE) Q4 2024 Earnings Call Highlights: Strong Revenue Growth ... (GuruFocus.com)
Feb-13-25 04:19PM	Ultragenyx: Q4 Earnings Snapshot (Associated Press Finance)
04:01PM	Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update (GlobeNewswire)
Feb-06-25 04:30PM	Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update (GlobeNewswire)
08:21AM	Ultragenyx announces new data on UX111 AV gene therapy (TipRanks)
Feb-05-25 08:00AM	Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS (GlobeNewswire)
Jan-17-25 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
11:14AM	Why Ultragenyx Pharmaceutical (RARE) Is the Biotech Stock with the Biggest Upside Potential (Insider Monkey)
Jan-15-25 02:05PM	Ultragenyx's Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside (Benzinga)
Jan-12-25 11:00AM	Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones (GlobeNewswire)
Jan-06-25 04:30PM	Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference (GlobeNewswire)
08:07AM	Ultragenyxs Evkeeza secures EU approval for young children with rare cholesterol disorder (Pharmaceutical Technology)
02:00AM	European Commission (EC) Extends the Approval of Evkeeza (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH) (GlobeNewswire)
Dec-20-24 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
03:00AM	NHS England Rolls Out Evkeeza (evinacumab) for Eligible Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolaemia (HoFH) (GlobeNewswire)
Dec-19-24 04:05PM	Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome (GlobeNewswire)
08:00AM	Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA) (GlobeNewswire)
Nov-26-24 04:30PM	Ultragenyx to Participate in Investor Conferences in December (GlobeNewswire)
Nov-20-24 04:30PM	Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) (GlobeNewswire)
Nov-09-24 10:30AM	Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST's 17th Annual Global Science Summit (GlobeNewswire)

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	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
KAKKIS EMIL D	President & CEO	Dec 30 '24	Sale	42.23	11,727	495,231	2,183,985	Dec 31 02:05 PM
KAKKIS EMIL D	Officer	Dec 30 '24	Proposed Sale	42.23	11,727	495,264		Dec 30 04:32 PM
KAKKIS EMIL D	President & CEO	Dec 10 '24	Sale	50.00	8,273	413,650	2,195,712	Dec 11 06:44 PM
KAKKIS EMIL D	Officer	Dec 10 '24	Proposed Sale	50.00	8,273	413,650		Dec 10 04:12 PM

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RARE

Ultragenyx Pharmaceutical Inc

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