Ultragenyx Pharmaceutical Inc. ( RARE ) NASDAQ Global Select

Cena: 29.27 ( -1.71% )

Aktualizacja 08-25 21:59
NASDAQ Global Select
Branża: Biotechnology

Notowania:

Opis firmy:

Ultragenyx Pharmaceutical Inc., firma biofarmaceutyczna, koncentruje się na identyfikacji, akwizycji, rozwoju i komercjalizacji nowych produktów do leczenia rzadkich i ultra-racjonalnych chorób genetycznych w Ameryce Północnej, Europie i na arenie międzynarodowej. Jego produkty biologiczne obejmują Crysvita (burozumab), przeciwciało ukierunkowane na czynnik wzrostu fibroblastów 23 do leczenia hipofosfatemii połączonej przez X, a także osteomalacji indukowanej przez nowotwory; MEPSEVII, enzym terapia zastępcza w leczeniu dzieci i dorosłych mukopolisacharydozą VII; DOJOLVI do leczenia zaburzeń utleniania kwasu tłuszczowego długotrwałego; i Evkeeza (Evinacumab) do leczenia homozygotycznej rodzinnej hipercholesterolemii. Kandydatury produktów firmy obejmują DTX401, kandydat kliniczny wirusa 8 (AAV8) do leczenia pacjentów z chorobą przechowywania glikogenu typu IA; DTX301, terapia genowa AAV8 w leczeniu pacjentów z ornityną przezkarbamylazy; UX143, ludzkie przeciwciało monoklonalne do leczenia osteogenezy Imperfecta; GTX-102, antysensowny oligonukleotyd do leczenia zespołu Angelmana; UX701, do leczenia choroby Wilsona; i UX053 do leczenia choroby przechowywania glikogenu typu III. Ultragenyx Pharmaceutical Inc. ma umowę o współpracy i licencji z Kyowa Kirin Co., Ltd.; Uniwersytet Saint Louis; Baylor Research Institute; Regenxbio Inc.; Bayer Healthcare LLC; Genetx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; oraz Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. został zarejestrowany w 2010 roku i ma siedzibę w Novato w Kalifornii.

Informacje o spółce:
Sektor: Zdrowie
Branża: Biotechnology
Zatrudnienie: 1 276
Giełda: NASDAQ Global Select
Ilość akcji w obrocie: 93.69
Ilość akcji: Brak danych
Debiut giełdowy: 2014-01-31
WWW: https://www.ultragenyx.com
CEO: Dr. Emil D. Kakkis M.D., Ph.D.
Adres: 60 Leveroni Court
Siedziba: 94949 Novato
ISIN: US90400D1081
Wskaźniki finansowe
Kapitalizacja (USD) 2 820 796 732
Aktywa: 1 538 363 000
Cena: 29.27
Wskaźnik Altman Z-Score: -1.5
Słaby (wysokie ryzyko bankructwa)
Dywidenda: 0
P/E: -5.3
Ilość akcji w obrocie: 94%
Średni wolumen: 1 962 937
Ilość akcji 96 371 600
Wskaźniki finansowe
Przychody TTM 522 745 000
Zobowiązania: 1 184 536 000
Przedział 52 tyg.: 25.81 - 60.37
Piotroski F-Score: 3
Słaby (niska jakość finansowa)
EPS: -5.5
P/E branży: 28.3
Beta: 0.581
Raport okresowy: 2025-11-04
WWW: https://www.ultragenyx.com
Zarząd
Imie i Nazwisko Stanowisko Wynagrodzenie Rok urodzenia
Mr. Erik Harris Executive Vice President & Chief Commercial Officer 878 767 1971
Mr. John Richard Pinion II Chief Quality Operations Officer & Executive Vice President of Translational Sciences 860 053 1966
Ms. Karah Herdman Parschauer J.D. Chief Legal Officer & Executive Vice President of Corporate Affairs 846 744 1978
Mr. Thomas R. Kassberg Chief Business Officer & Executive Vice President 714 838 1960
Mr. Dennis Karl Huang Chief Technical Operations Officer and Executive Vice President of Gene Therapy Research & Development 331 330 1965
Mr. Howard Horn Executive Vice President of Corporate Strategy & Chief Financial Officer 184 808 1978
Dr. Emil D. Kakkis M.D., Ph.D. Founder, President, Chief Executive Officer & Director 1 459 548 1960
Mr. Vimal Srivastava Senior Vice President of Business Development & Alliance Management 0 1965
Mr. Ernie W. Meyer Chief Human Resources Officer & Executive Vice President 0 1965
Mr. Theodore A. Huizenga Senior Vice President, Corporate Controller & Principal Accounting Officer 0 1971
Lista ETF z ekspozycją na akcje Ultragenyx Pharmaceutical Inc.
Symbol ETF Ilość akcji Wartość
VB 2 194 749 79 801 073
VBK 1 232 867 44 827 044
XBI 985 020 26 112 981
IWP 591 641 16 163 634
IBB 536 733 14 663 552
SCHA 447 853 11 942 328
IWF 335 562 9 167 566
IWR 312 045 8 525 081
VHT 262 701 9 551 808
ITOT 139 345 3 806 900
VONG 114 708 4 170 782
H1D5.DE 96 673 2 285 457
AUM5.DE 96 673 2 285 457
500U.L 96 673 2 609 192
500.PA 96 673 2 285 457
LABU 92 167 2 518 002
DBX1.DE 79 987 1 913 607
XMMD.L 79 987 2 185 231
XMEM.L 79 987 165 594 648
PFUT 79 529 2 172 728
PRFZ 66 056 1 792 759
IWB 62 413 1 705 124
BTEK.L 56 182 1 161 737
BTEC.L 56 182 1 534 902
2B70.DE 56 182 1 342 732
BTEE.L 56 182 1 534 902
SCHB 52 962 1 431 975
AGES.L 49 618 1 025 997
AGED.L 49 618 1 355 561
2B77.DE 49 618 1 185 845
IYH 49 498 1 352 277
GNOM 44 847 1 225 220
BBMC 41 812 1 142 303
FHLC 35 823 989 073
TMSL 32 033 875 141
ONEQ 31 069 857 815
ESML 26 234 711 990
IWV 24 694 674 646
ISCG 23 987 655 322
SMMD 20 966 569 017
ESGV 19 814 720 437
CUS1.L 16 989 351 304
CUSS.L 16 989 464 148
CSUSS.MI 16 989 406 036
SXRG.DE 16 989 406 036
FNY 15 934 421 294
KOMP 15 507 421 916
EQAL 15 006 407 262
SMMV 14 162 384 356
VONE 13 952 507 294
MMLG 12 563 332 165
ETHO 12 433 339 669
BBP 11 311 312 296
SCHK 7 410 200 397
IGSU.L 7 337 200 456
IGSG.L 7 337 151 721
IUSL.DE 7 337 175 358
VTHR 7 209 262 119
GINN 6 922 286 847
HELX 5 832 158 280
SMLV 5 367 147 271
RECS 4 811 131 436
TILT 4 620 126 218
ISCB 4 027 110 025
IYY 3 872 105 791
FAD 3 577 94 575
GDOC 3 490 144 625
BIB 3 482 95 128
IBBQ 3 387 91 923
STXK 2 829 77 288
XUU.TO 2 544 95 357
WDNA.L 1 576 58 564
WBIO.L 1 576 58 564
MMSC 1 489 39 369
R1GR.L 1 236 33 762
USSC.L 1 151 27 210
ZPRV.DE 1 151 23 828
GGUS 1 123 46 537
V3AL.L 1 066 38 759
V3AA.L 1 066 38 759
V3AB.L 1 066 29 371
JDOC 811 22 156
XBAL.TO 810 30 371
WDNA 700 19 124
USUE.DE 625 16 961
USFM.L 625 16 961
ANEW 483 13 195
CBUG.DE 394 9 425
EWSA.AS 394 10 774
XUH.TO 376 10 193
XTR.TO 15 412
SBIO.L 0 756 906
PZW.TO 0 8 407
SBIO.MI 0 663 201
CDX -10 781 -294 539
LSEQ -16 766 -455 029
Wiadomości dla Ultragenyx Pharmaceutical Inc.
Tytuł Treść Źródło Aktualizacja Link
Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report NOVATO, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2024 Impact Report today, which covers the company's approach to corporate responsibility. The report highlights efforts to transform the lives of individuals and families affected by rare diseases, empower its employees, and make a meaningful impact on the communities it operates within and serves. globenewswire.com 2025-05-14 20:30:00 Czytaj oryginał (ang.)
HENRY CATCHPOLE TAKES TO THE TRACK IN RARE HONDA NSX-R IN SWEDEN AHEAD OF BROAD ARROW'S FIRST EUROPEAN AUCTION HIGHLY COLLECTIBLE NSX-R TO FEATURE IN INAUGURAL BROAD ARROW CONCORSO D'ELEGANZA VILLA D'ESTE AUCTION ON 24-25 MAY HIGHLY COLLECTIBLE NSX-R TO FEATURE IN INAUGURAL BROAD ARROW CONCORSO D'ELEGANZA VILLA D'ESTE AUCTION ON 24-25 MAY globenewswire.com 2025-05-08 12:01:00 Czytaj oryginał (ang.)
Ultragenyx to Participate at Bank of America's 2025 Healthcare Conference NOVATO, Calif., May 07, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Howard Horn, the company's chief financial officer and executive vice president, will participate in a fireside at Bank of America's 2025 Healthcare Conference on Tuesday, May 13, 2025, at 8:00 a.m. PT. globenewswire.com 2025-05-07 21:00:00 Czytaj oryginał (ang.)
Ultragenyx's Q1 Loss Wider Than Expected, Revenues Increase Y/Y RARE reports weaker-than-expected first-quarter results, as earnings and revenues miss estimates despite a year-over-year increase in product sales. zacks.com 2025-05-07 15:45:36 Czytaj oryginał (ang.)
Ultragenyx Pharmaceutical Inc. (RARE) Q1 2025 Earnings Call Transcript Ultragenyx Pharmaceutical Inc. (RARE) Q1 2025 Earnings Call Transcript seekingalpha.com 2025-05-07 03:31:54 Czytaj oryginał (ang.)
Ultragenyx (RARE) Reports Q1 Earnings: What Key Metrics Have to Say The headline numbers for Ultragenyx (RARE) give insight into how the company performed in the quarter ended March 2025, but it may be worthwhile to compare some of its key metrics to Wall Street estimates and the year-ago actuals. zacks.com 2025-05-06 23:00:34 Czytaj oryginał (ang.)
Ultragenyx (RARE) Reports Q1 Loss, Misses Revenue Estimates Ultragenyx (RARE) came out with a quarterly loss of $1.57 per share versus the Zacks Consensus Estimate of a loss of $1.54. This compares to loss of $2.03 per share a year ago. zacks.com 2025-05-06 22:10:46 Czytaj oryginał (ang.)
Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update First quarter total revenue of $139 million,  Crysvita® revenue of $103 million and Dojolvi® revenue of $17 million globenewswire.com 2025-05-06 20:01:00 Czytaj oryginał (ang.)
5 Drug/Biotech Stocks Likely to Outperform Q1 Earnings Estimates Let us look at some drug/biotech stocks, NVO, PCRX, RARE, ACAD and DNLI, which are poised to beat on first-quarter 2025 earnings. zacks.com 2025-05-05 14:15:41 Czytaj oryginał (ang.)
Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update NOVATO, Calif., April 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, May 6, 2025, to discuss its financial results and corporate update for the quarter ending March 31, 2025. globenewswire.com 2025-04-30 21:00:00 Czytaj oryginał (ang.)
Ultragenyx (RARE) Expected to Beat Earnings Estimates: What to Know Ahead of Q1 Release Ultragenyx (RARE) possesses the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations. zacks.com 2025-04-24 15:08:33 Czytaj oryginał (ang.)
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., April 22, 2025 (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 36,831 restricted stock units of the company's common stock to 21 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 April 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4). globenewswire.com 2025-04-22 21:00:00 Czytaj oryginał (ang.)
Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline Ultragenyx shows strong revenue growth and disciplined expense management, making it a compelling long-term investment despite inherent biotechnology risks. The company's diverse revenue streams from Crysvita, Dojolvi, and Evkeeza fund innovation and buffer against financial volatility. Key catalysts include the FDA's Priority Review of UX111 for Sanfilippo syndrome type A, with a PDUFA date set for August 2025. seekingalpha.com 2025-04-16 13:49:56 Czytaj oryginał (ang.)
Ultragenyx (RARE) Soars 9.2%: Is Further Upside Left in the Stock? Ultragenyx (RARE) saw its shares surge in the last session with trading volume being higher than average. The latest trend in earnings estimate revisions may not translate into further price increase in the near term. zacks.com 2025-04-10 14:30:48 Czytaj oryginał (ang.)
BROAD ARROW ADDS RARE RUF CTR YELLOWBIRD TO PORSCHE AUCTION IN PARTNERSHIP WITH AIR|WATER Broad Arrow Auctions, a Hagerty (NYSE: HGTY) company, has announced an exceedingly rare 1989 RUF CTR Yellowbird, chassis no. W09BT0348KPR06023, to lead its second annual Porsche Auction in partnership with Air|Water. Set for April 26 at the Orange County Fairgrounds in Costa Mesa, California, the single-day auction held within the Air|Water event will once again feature more than 50 desirable Porsches along with a selection of coveted Porsche memorabilia pieces. globenewswire.com 2025-04-09 16:46:00 Czytaj oryginał (ang.)
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., March 19, 2025 (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 21,806 restricted stock units of the company's common stock to 16 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 March 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4). globenewswire.com 2025-03-19 19:00:00 Czytaj oryginał (ang.)
Ultragenyx to Participate at Investor Conferences in March NOVATO, Calif., Feb. 24, 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 participation in three upcoming investor conferences. globenewswire.com 2025-02-24 18:30:00 Czytaj oryginał (ang.)
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., Feb. 19, 2025 (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 23,760 restricted stock units of the company's common stock to 10 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 February 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4). globenewswire.com 2025-02-19 18:30:00 Czytaj oryginał (ang.)
AMERICAN SALARS ACQUIRES 18,083 HECTARE BRAZIL HARDROCK LCT PEGMATITE PROPERTY WITH RECENT SAMPLING UP TO 3.72% LI20 AND SIGNIFICANT RARE EARTH ELEMENT VALUES VANCOUVER, BC, Feb. 18, 2025 (GLOBE NEWSWIRE) -- AMERICAN SALARS LITHIUM INC. ("AMERICAN SALARS" OR THE "COMPANY") (CSE: USLI, OTC: USLIF, FWB: Z3P, WKN:  A3E2NY ) announces that it has entered into a share purchase agreement to acquire all of the outstanding shares of 1447377 BC Ltd. (the “Vendor”) that holds a 100% interest in a highly prospective 18,083 Hectares (180 sq km) Hardrock LCT (“Lithium-Cesium-Tantalum”) Pegmatite Project including Rare Earth Elements (“REE”) and a suite of Critical Minerals (the “Jaguaribe Project”). globenewswire.com 2025-02-18 05:01:00 Czytaj oryginał (ang.)
Ultragenyx Q4 Loss Wider Than Expected, Revenues Increase Y/Y RARE reports mixed fourth-quarter results, as earnings miss estimates while revenues beat the same. Product sales increase year over year. zacks.com 2025-02-14 12:41:35 Czytaj oryginał (ang.)
Ultragenyx Pharmaceutical Inc. (RARE) Q4 2024 Earnings Call Transcript Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Q4 2024 Earnings Conference Call February 13, 2025 5:00 PM ET Company Participants Joshua Higa - VP, IR Emil Kakkis - CEO and President Erik Harris - CCO Howard Horn - CFO Eric Crombez - CMO Conference Call Participants Tazeen Ahmad - Bank of America Salveen Richter - Goldman Sachs Gena Wang - Barclays Yaron Werber - TD Cowen Joon Lee - Truist Securities Jeff Hung - Morgan Stanley Rick Miller - Cantor Fitzgerald Luca Issi - RBC Capital Liisa Bayko - Evercore ISI Operator Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2024 Financial Results Conference Call. At this time, all participants are in a listen-only-mode. seekingalpha.com 2025-02-13 23:53:23 Czytaj oryginał (ang.)
Ultragenyx (RARE) Q4 Earnings: Taking a Look at Key Metrics Versus Estimates Although the revenue and EPS for Ultragenyx (RARE) give a sense of how its business performed in the quarter ended December 2024, it might be worth considering how some key metrics compare with Wall Street estimates and the year-ago numbers. zacks.com 2025-02-13 20:30:21 Czytaj oryginał (ang.)
Ultragenyx (RARE) Reports Q4 Loss, Tops Revenue Estimates Ultragenyx (RARE) came out with a quarterly loss of $1.39 per share versus the Zacks Consensus Estimate of a loss of $1.24. This compares to loss of $1.52 per share a year ago. zacks.com 2025-02-13 20:11:08 Czytaj oryginał (ang.)
Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update 2024 Total Revenue of $560 million, exceeding guidance Crysvita® revenue of $410 million and Dojolvi® revenue of $88 million globenewswire.com 2025-02-13 18:01:00 Czytaj oryginał (ang.)
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update NOVATO, Calif., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, February 13, 2025, to discuss its financial results and corporate update for the quarter and the year ending December 31, 2024. globenewswire.com 2025-02-06 18:30:00 Czytaj oryginał (ang.)
RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study Ultragenyx announces new safety and efficacy data from its pivotal study of its investigational candidate, UX111, for patients with Sanfilippo syndrome type A. zacks.com 2025-02-06 13:31:10 Czytaj oryginał (ang.)
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 Modified intent-to-treat group demonstrated +22.7 point (p globenewswire.com 2025-02-05 10:00:00 Czytaj oryginał (ang.)
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., Jan. 17, 2025 (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 7,750 restricted stock units of the company's common stock to eight 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 January 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4). globenewswire.com 2025-01-17 18:30:00 Czytaj oryginał (ang.)
Ultragenyx's Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside In conjunction with the 2025 J.P. Morgan Healthcare Conference, the analyst hosted an investor lunch with Ultragenyx Pharmaceutical Inc RARE. benzinga.com 2025-01-15 16:05:35 Czytaj oryginał (ang.)
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 globenewswire.com 2025-01-12 13:00:00 Czytaj oryginał (ang.)
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. zacks.com 2025-01-07 13:25:42 Czytaj oryginał (ang.)
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. globenewswire.com 2025-01-06 18:30:00 Czytaj oryginał (ang.)
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 globenewswire.com 2025-01-06 04:00:00 Czytaj oryginał (ang.)
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). globenewswire.com 2024-12-20 18:30:00 Czytaj oryginał (ang.)
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. zacks.com 2024-12-20 14:31:35 Czytaj oryginał (ang.)
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. globenewswire.com 2024-12-20 05:00:00 Czytaj oryginał (ang.)
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 globenewswire.com 2024-12-19 18:05:00 Czytaj oryginał (ang.)
HARMONY BIOSCIENCES TO PARTICIPATE IN OPPENHEIMER MOVERS IN RARE DISEASE SUMMIT PLYMOUTH MEETING, Pa. , Dec. 3, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced that Harmony's management team will participate in a fireside chat at the upcoming Oppenheimer Movers in Rare Disease Summit in New York, NY on Thursday, December 12, 2024, at 9:00 a.m. prnewswire.com 2024-12-03 10:05:00 Czytaj oryginał (ang.)
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. globenewswire.com 2024-11-26 18:30:00 Czytaj oryginał (ang.)
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). globenewswire.com 2024-11-20 18:30:00 Czytaj oryginał (ang.)
AMGEN PRESENTS NEW DATA ACROSS RARE INFLAMMATORY DISEASES AT ACR 2024 MITIGATE Phase 3 Study Results Reinforce Promise of UPLIZNA® as the First Potential Treatment tor IgG4-RD Phase 4 AGILE Data Support Shortening KRYSTEXXA® Infusion Time THOUSAND OAKS, Calif. , Nov. 14, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the presentation of new data across its rare disease portfolio and pipeline at the annual American College of Rheumatology (ACR) Convergence 2024 conference in Washington, D.C. prnewswire.com 2024-11-14 18:00:00 Czytaj oryginał (ang.)
Ultragenyx: Ready For More Growth After Q3 Earnings Beat Ultragenyx's strong Q3 earnings report, with a 42% YoY revenue increase, has reignited my interest in initiating a starter position in RARE. The company's promising pipeline, including late-stage programs like Setrusumab and DTX401, presents multiple catalysts for future growth and higher valuation. Ultragenyx's $825M cash position and path to profitability by 2026 suggest financial stability, potentially avoiding the need for extensive dilutive financing. seekingalpha.com 2024-11-12 13:12:09 Czytaj oryginał (ang.)
Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST's 17th Annual Global Science Summit Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish efficacy of GTX-102 globenewswire.com 2024-11-09 12:30:00 Czytaj oryginał (ang.)
Ultragenyx Q3 Loss Narrower Than Expected, Revenues Beat Estimates RARE reports encouraging third-quarter results, beating earnings and revenue estimates. Product sales increase year over year. zacks.com 2024-11-06 12:45:31 Czytaj oryginał (ang.)
Ultragenyx Pharmaceutical Inc. (RARE) Q3 2024 Earnings Call Transcript Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Q3 2024 Earnings Conference Call November 5, 2024 5:00 PM ET Company Participants Joshua Higa - VP, IR Emil Kakkis - CEO and President Erik Harris - CCO Howard Horn - CFO Eric Crombez - CMO Conference Call Participants Chris Raymond - Piper Sandler Tazeen Ahmad - Bank of America Malcolm Kuno - JPMorgan Gena Wang - Barclays Lydia Erdman - Goldman Sachs Maury Raycroft - Jefferies Mehdi Goudarzi - Truist Securities Yaron Werber - TD Cowen Yigal Nochomovitz - Citi Thomas Yip - H.C. Wainwright Jack Allen - Baird Joe Schwartz - Leerink Partners Michael Riad - Morgan Stanley Kristen Kluska - Cantor Fitzgerald Dae Gon Ha - Stifel Operator Good afternoon and welcome to the Ultragenyx Third Quarter 2024 Financial Results Conference Call. seekingalpha.com 2024-11-06 00:40:31 Czytaj oryginał (ang.)
Compared to Estimates, Ultragenyx (RARE) Q3 Earnings: A Look at Key Metrics While the top- and bottom-line numbers for Ultragenyx (RARE) give a sense of how the business performed in the quarter ended September 2024, it could be worth looking at how some of its key metrics compare to Wall Street estimates and year-ago values. zacks.com 2024-11-05 21:00:32 Czytaj oryginał (ang.)
Ultragenyx (RARE) Reports Q3 Loss, Tops Revenue Estimates Ultragenyx (RARE) came out with a quarterly loss of $1.40 per share versus the Zacks Consensus Estimate of a loss of $1.45. This compares to loss of $2.23 per share a year ago. zacks.com 2024-11-05 20:15:15 Czytaj oryginał (ang.)
Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update Third quarter 2024 total revenue grew 42% versus prior year to $139 million, including Crysvita® revenue of $98 million and Dojolvi® revenue of $21 million globenewswire.com 2024-11-05 18:01:00 Czytaj oryginał (ang.)
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., Oct. 18, 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 22,405 restricted stock units of the company's common stock to six 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 October 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4). globenewswire.com 2024-10-18 20:30:00 Czytaj oryginał (ang.)
RARE's Bone Disorder Drug Gets FDA's Breakthrough Therapy Tag Ultragenyx announces that the FDA grants Breakthrough Therapy designation to its investigational osteogenesis imperfecta treatment candidate, setrusumab. zacks.com 2024-10-08 16:10:20 Czytaj oryginał (ang.)
RARE's Wilson Disease Candidate Betters Standard Therapy in Study Ultragenyx reports superior efficacy of UX701 for Wilson disease compared with standard therapy from the Stage 1 cohorts of its pivotal phase I/II/III study. zacks.com 2024-10-04 16:05:20 Czytaj oryginał (ang.)
Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease NOVATO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus 2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off of standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. globenewswire.com 2024-10-03 20:30:00 Czytaj oryginał (ang.)
Ultragenyx Announces Upcoming Setrusumab (UX143) Presentations at the ASBMR 2024 Annual Meeting NOVATO, Calif., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it will present seven abstracts related to its ongoing late-stage program evaluating setrusumab (UX143) and osteogenesis imperfecta (OI), including a late-breaker oral presentation of the 14-month data from the Phase 2/3 Orbit study, at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. The meeting is being held September 27-30, 2024, in Toronto, Canada. globenewswire.com 2024-09-26 12:00:00 Czytaj oryginał (ang.)