Articles from Ultragenyx Pharmaceutical Inc.

NOVATO, Calif., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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.

Modified intent-to-treat group demonstrated +22.7 point (p<0.0001) treatment effect in the mean Bayley-III cognitive raw score compared to natural history data (ages 24-60 months)

NOVATO, Calif., Jan. 17, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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).

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

NOVATO, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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.

NOVATO, Calif., Dec. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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).

Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025

If approved, UX111 would be the first approved therapy in the U.S. for Sanfilippo Syndrome Type A

NOVATO, Calif., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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.

NOVATO, Calif., Nov. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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).

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

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

NOVATO, Calif., Oct. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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 Tuesday, November 5, 2024, to discuss its financial results and corporate update for the quarter ending September 30, 2024.

NOVATO, Calif., Oct. 18, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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).

NOVATO, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type I, III, or IV in patients 2 years of age and older.

NOVATO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE) today reported that the Phase 1/2/3 Cyprus2+ 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.

NOVATO, Calif., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE) 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.

NOVATO, Calif., Sept. 18, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 62,555 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 September 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Morgan Stanley 22nd Annual Global Healthcare Conference on September 4

NOVATO, Calif., Aug. 21, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 37,975 restricted stock units of the company’s common stock to 12 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 August 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Second quarter total revenue of $147 million, Crysvita® revenue of $114 million and Dojolvi® revenue of $19 million

NOVATO, Calif., July 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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, August 1, 2024, to discuss its financial results and corporate update for the quarter ending June 30, 2024.

Presentations will include an encore of the positive interim Phase 1/2 results presented in April at the 76th Annual American Academy of Neurology (AAN) Meeting

NOVATO, Calif., July 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 19,875 restricted stock units of the company’s common stock to 14 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 July 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Alignment with FDA on Phase 3 study primary endpoint of Bayley-4 cognition and key secondary endpoint of Multi-Domain Responder Index (MDRI)

NOVATO, Calif., June 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 45,525 restricted stock units of the company’s common stock to 26 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 June 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

NOVATO, Calif., June 13, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced the pricing of its underwritten public offering of 7,435,898 shares of its common stock at a price to the public of $39.00 per share. In addition, in lieu of issuing common stock to certain investors, the company is offering pre-funded warrants to purchase 1,538,501 shares of its common stock at a purchase price of $38.999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.001 exercise price per share of each pre-funded warrant. The aggregate gross proceeds to the company from this offering is expected to be $350 million, before deducting underwriting discounts and commissions and other offering expenses, and excluding the exercise of any pre-funded warrants. In addition, the company has granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 1,346,153 shares of the company's common stock at the public offering price, less the underwriting discount.

NOVATO, Calif., June 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that it has commenced an underwritten public offering of up to $350,000,000 of shares of its common stock and, in lieu of issuing common stock to certain investors, pre-funded warrants to purchase shares of its common stock. In addition, the company is expected to grant the underwriters of the offering an option for a period of 30 days to purchase up to an additional $52,500,000 of shares of common stock at the public offering price, less the underwriting discount.

Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval

14-month data show treatment with setrusumab resulted in a large, sustained 67% reduction in annualized fracture rate and persistent median annualized fracture rate of 0.00 (p=0.0014)

NOVATO, Calif., June 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 45th Annual Global Healthcare Conference on Tuesday, June 11, 2024, at 10:00 a.m. ET.

Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control

NOVATO, Calif., May 17, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 17,180 restricted stock units of the company’s common stock to ten 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 May 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

NOVATO, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Eric Crombez, M.D., the company's chief medical officer and executive vice president, will participate in a fireside at Bank of America’s 2024 Healthcare Conference on Tuesday, May 14, 2024, at 1:40 p.m. PT.

First quarter total revenue of $109 million, Crysvita® revenue of $83 million and Dojolvi® revenue of $16 million

NOVATO, Calif., April 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE) today released its 2023 Corporate Responsibility Report, which showcases the company’s efforts to transform the lives of individuals and families impacted by rare and ultrarare diseases, empower its employees, and make a meaningful impact on the communities it partners with.

NOVATO, Calif., April 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE) today announced that all patients have been enrolled across the Phase 3 Orbit and Cosmic studies evaluating setrusumab (UX143) in pediatric and young adult patients with osteogenesis imperfecta (OI). The pivotal Phase 3 portion of the Orbit study has randomized 158 patients ages 5 to 25 years, and the Cosmic study has completed enrollment of 66 patients ages 2 to <7 years.

NOVATO, Calif., April 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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, May 2, 2024, to discuss its financial results and corporate update for the quarter ending March 31, 2024.

NOVATO, Calif., April 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 44,965 restricted stock units of the company’s common stock to 14 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, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains; improvements consistent or exceeding Dose-escalation Cohorts data at Day 170

Conference call now scheduled for Monday, April 15 at 8:00 a.m. ET

Company will host investor call on April 17 at 8:00 a.m. ET to discuss new data from GTX-102 Phase 1/2 clinical study in patients with Angelman syndrome  

NOVATO, Calif., March 22, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 13,020 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 March 18, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

44th Annual Cowen Healthcare Conference on March 4

NOVATO, Calif., Feb. 23, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 10,995 restricted stock units of the company’s common stock to nine 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, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

2023 Total Revenue of $434 million, Crysvita® revenue of $328 million and Dojolvi® revenue of $71 million

NOVATO, Calif., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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 15, 2024, to discuss its financial results and corporate update for the fourth quarter and the year ending December 31, 2023.

The latest data from the pivotal Transpher A and long-term follow-up studies will be presented at WORLDSymposium™ 2024

GTX-102 is the first Angelman syndrome therapeutic candidate to receive PRIME designation

Safety and initial efficacy data from Stage 1 expected in the first half of 2024

NOVATO, Calif., Jan. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 12,130 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, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Preliminary 2023 Total Revenue of $430 million to $435 million, Crysvita® Revenue of $325 million to $330 million and Dojolvi® revenue of $70 million to $71 million

NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition.

Data from at least 20 patients enrolled in dose expansion cohorts anticipated in the first half of 2024

NOVATO, Calif., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 3:00 PM PT.

NOVATO, Calif., Dec. 21, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 30,325 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 December 16, 2023, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

For the first time, paediatric patients aged 5-11 years with HoFH in the EU have access to a first-in-class medicine to treat an ultrarare, inherited form of high cholesterol

6th Annual Evercore ISI HealthCONx on November 28 in Miami

NOVATO, Calif., Nov. 17, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 22,765 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 November 16, 2023, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

NOVATO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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 participate in a fireside chat at the upcoming Jefferies London Healthcare Conference on Tuesday, November 14, 2023, at 12:30 p.m. GMT.

Third quarter 2023 total revenue of $98.1 million, Crysvita® revenue of $74.9 million and Dojolvi® revenue of $16.6 million

NOVATO, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), 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, November 2, 2023, to discuss its financial results and corporate update for the quarter ending September 30, 2023.

NOVATO, Calif., Oct. 23, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced the closing of its previously announced underwritten public offering of 9,833,334 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,500,000 shares, at a price to the public of $30.00 per share, and, to certain investors in lieu of common stock, pre-funded warrants to purchase 1,666,722 shares of its common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.001 exercise price per share of each pre-funded warrant. The estimated net proceeds to the company, after deducting underwriting discounts and commissions and other offering expenses payable by the company, were approximately $326.1 million.

NOVATO, Calif., Oct. 20, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 18,865 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 October 16, 2023, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

NOVATO, Calif., Oct. 18, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced the pricing of its underwritten public offering of 8,333,334 shares of its common stock at a price to the public of $30.00 per share. In addition, in lieu of issuing common stock to certain investors, the company is offering pre-funded warrants to purchase 1,666,722 shares of its common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.001 exercise price per share of each pre-funded warrant. The aggregate gross proceeds to the company from this offering is expected to be $300 million, before deducting underwriting discounts and commissions and other offering expenses, and excluding the exercise of any pre-funded warrants. In addition, the company has granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 1,500,000 shares of the company's common stock at the public offering price, less the underwriting discount.

NOVATO, Calif., Oct. 17, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced that it has commenced an underwritten public offering of up to $300,000,000 of shares of its common stock. In addition, the company is expected to grant the underwriters of the offering an option for a period of 30 days to purchase up to an additional $45,000,000 of shares of common stock at the public offering price, less the underwriting discount.

Treatment with setrusumab (UX143) for at least 6 months resulted in 67% reduction in annualized fracture rate in patients with OI in Phase 2/3 Orbit study

NOVATO, Calif., Oct. 12, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 80,366 restricted stock units of the company’s common stock and the grant of non-qualified stock options to purchase an aggregate of 140,469 shares of common stock of the company to Howard Horn, its newly appointed Chief Financial Officer and Executive Vice President, Corporate Strategy. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan, as amended, with a grant date of October 9, 2023, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

NOVATO, Calif., Oct. 09, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE) today announced that data from its ongoing late-stage program evaluating setrusumab (UX143) in osteogenesis imperfecta (OI) will be presented at the American Society for Bone and Mineral Research 2023 Annual Meeting (ASBMR) October 13-16, 2023, in Vancouver, British Columbia.

Analyst Day will be webcast beginning at 8:30 a.m. ET on Monday, October 16

NOVATO, Calif., Sept. 20, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 33,475 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 September 16, 2023, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Citi BioPharma Conference on September 6 in Boston

NOVATO, Calif., Aug. 18, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQRARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 42,840 restricted stock units of the company’s common stock to 18 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 August 16, 2023, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Second quarter 2023 total revenue of $108.3 million, Crysvita® revenue of $83.0 million and Dojolvi® revenue of $16.5 million

Initial data expected in first half of 2024