Ultragenyx PharmaceuticalRARE
RARE
About: Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi, and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
Employees: 1,276
0
Funds holding %
of 6,815 funds
0
Analysts bullish %
of 9 analysts
Fund manager confidence
Based on 2024 Q3 regulatory filings by fund managers ($100M+ AUM)
97% more first-time investments, than exits
New positions opened: 59 | Existing positions closed: 30
31% more capital invested
Capital invested by funds: $3.65B [Q2] → $4.78B (+$1.13B) [Q3]
23% more repeat investments, than reductions
Existing positions increased: 111 | Existing positions reduced: 90
18% more call options, than puts
Call options by funds: $28.2M | Put options by funds: $23.9M
10% more funds holding
Funds holding: 272 [Q2] → 298 (+26) [Q3]
0% more funds holding in top 10
Funds holding in top 10: 6 [Q2] → 6 (+0) [Q3]
4.69% less ownership
Funds ownership: 98.16% [Q2] → 93.48% (-4.69%) [Q3]
Research analyst outlook
9 Wall Street Analysts provided 1 year price targets over the past 3 months
Low target
$48
20%
upside
Avg. target
$96
139%
upside
High target
$140
250%
upside
9 analyst ratings
8 positive
89%
1 neutral
11%
0 negative
0%
Piper Sandler Christopher Raymond 17% 1-year accuracy 5 / 30 met price target | 250%upside $140 | Overweight Maintained | 13 Jan 2025 |
Wedbush Laura Chico 26% 1-year accuracy 16 / 61 met price target | 20%upside $48 | Neutral Reiterated | 13 Jan 2025 |
HC Wainwright & Co. Ed Arce 51% 1-year accuracy 80 / 157 met price target | 137%upside $95 | Buy Reiterated | 24 Dec 2024 |
Wells Fargo Tiago Fauth 50% 1-year accuracy 11 / 22 met price target | 120%upside $88 | Overweight Maintained | 20 Dec 2024 |
JP Morgan Anupam Rama 25% 1-year accuracy 15 / 59 met price target | 155%upside $102 | Overweight Maintained | 21 Nov 2024 |
Financial journalist opinion
Based on 8 articles about RARE published over the past 30 days
Neutral
GlobeNewsWire
2 days ago
Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones
Preliminary 2024 total revenue of $555 million to $560 million, exceeding top end of guidance, including Crysvita® revenue of $405 million to $410 million, and Dojolvi® revenue of $87 million to $89 million
Positive
Zacks Investment Research
1 week ago
RARE's Cholesterol Drug Evkeeza Gains EC Approval for Expanded Use
EC expands the label of Ultragenyx's Evkeeza in the EU to treat younger patients aged 6 months to 5 years suffering from a rare cholesterol disorder.
Neutral
GlobeNewsWire
1 week ago
Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference
NOVATO, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 AM PT.
Neutral
GlobeNewsWire
1 week ago
European Commission (EC) Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)
First and only medicine approved in the EU for paediatric patients aged 6-months to 5 years old with HoFH, an ultrarare, inherited form of high cholesterol First and only medicine approved in the EU for paediatric patients aged 6-months to 5 years old with HoFH, an ultrarare, inherited form of high cholesterol
Neutral
GlobeNewsWire
3 weeks ago
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
NOVATO, Calif., Dec. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 15,175 restricted stock units of the company's common stock to 15 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of December 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).
Positive
Zacks Investment Research
3 weeks ago
RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
Ultragenyx submits a BLA to the FDA seeking the accelerated approval of UX111 for patients with Sanfilippo syndrome type A in the United States.
Neutral
GlobeNewsWire
3 weeks ago
NHS England Rolls Out Evkeeza® ▼(evinacumab) for Eligible Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolaemia (HoFH)
LONDON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that NHS England has implemented the commissioning of Evkeeza (evinacumab) following the National Institute for Health and Care Excellence (NICE) final guidance in September. The use of Evkeeza in eligible people aged 12 years and older will be routinely commissioned by NHS England in line with the NICE TA and will be available in seven hospital trusts in England. In addition, prior approval forms are in place to enable access for children aged 5 to 11 years, via the NHS England Commissioning Medicines for Children policy.
Neutral
GlobeNewsWire
3 weeks ago
Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025 Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025
Neutral
GlobeNewsWire
1 month ago
Ultragenyx to Participate in Investor Conferences in December
NOVATO, Calif., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences.
Neutral
GlobeNewsWire
1 month ago
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
NOVATO, Calif., Nov. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 20,820 restricted stock units of the company's common stock to 13 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of November 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).
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