Burosumab

Ultragenyx Reports First Quarter 2024 Financial Results and Corporate Update

Retrieved on: 
Thursday, May 2, 2024
Osteogenesis imperfecta, Burosumab, Patient, OI, Ageing, Ultragenyx

NOVATO, Calif., May 02, 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 reported its financial results for the quarter ended March 31, 2024 and reaffirmed its financial guidance for 2024.

Key Points: 
  • Total operating expenses for the first quarter of 2024 were $274 million, including non-cash stock-based compensation of $37 million.
  • Net cash used in operations for the quarter ended March 31, 2024 was $191 million.
  • For the full year 2024, the company reaffirms:
    Crysvita revenue in the range of $375 million to $400 million.
  • ET to discuss the first quarter 2024 financial results and provide a corporate update.

Athira Pharma Appoints Javier San Martin, M.D., as Chief Medical Officer

Retrieved on: 
Monday, April 15, 2024
Doctor of Philosophy, Denosumab, University, Arrowhead Pharmaceuticals, Eli Lilly, Compensated emancipation, Internal medicine, International development, Employment, HGF, Lundbeck Seattle Biopharmaceuticals, Athira Pharma, Therapy, Senior, Hypophosphatemia, Patient, ALS, DSM-IV codes, Ultragenyx, Drug development, Burosumab, Growth, Pharmaceutical industry

BOTHELL, Wash., April 15, 2024 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced the appointment of Javier San Martin, M.D., as Chief Medical Officer.

Key Points: 
  • BOTHELL, Wash., April 15, 2024 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced the appointment of Javier San Martin, M.D., as Chief Medical Officer.
  • “We are pleased to welcome Javier to the team and are confident his insights and guidance will be instrumental as we advance the Company’s next phase of growth,” said Mark Litton, Ph.D., President and Chief Executive Officer of Athira.
  • Before that, Dr. San Martin served as Senior Vice President of Clinical Development at Alder Biopharmaceuticals, where he managed medical, regulatory, and clinical operations.
  • In addition, Athira today announced, as required by The Nasdaq Stock Market Rules, an equity inducement award to Dr. San Martin, the Company’s new Chief Medical Officer.

Ultragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, February 15, 2024
Patient, Burosumab, Ultragenyx, OI

NOVATO, Calif., Feb. 15, 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 reported its financial results for the quarter and full year ended December 31, 2023 and reaffirmed its financial guidance for 2024.

Key Points: 
  • Total operating expenses for the fourth quarter of 2023 were $249 million, including non-cash stock-based compensation of $34 million.
  • Total operating expenses for the year ended December 31, 2023 were $1,003 million, including $135 million of non-cash stock-based compensation.
  • For the fourth quarter of 2023, Ultragenyx reported net loss of $123 million, or $1.52 per share basic and diluted, compared with a net loss for the fourth quarter of 2022 of $152 million, or $2.16 per share, basic and diluted.
  • ET to discuss the fourth quarter and full year 2023 financial results and provide a corporate update.

Kyowa Kirin Responds to NICE’s Publication of Appraisal Consultation Document for CRYSVITA® (Burosumab), for Treatment of Rare Disease X-Linked Hypophosphataemia (XLH) in Adults1

Retrieved on: 
Wednesday, November 29, 2023
Health, Pharmaceutical, Medicine, STA, Addenbrooke's Hospital, HST, Disease, Patient, XLH, NHS, Friends, ACD, Diagnosis, High, Pain, Family, Kyowa Kirin, NHS England, Pharmaceutical industry, Nursing, Burosumab

The decision would create a disparity in access between England and Wales on the one hand, and Scotland on the other.

Key Points: 
  • The decision would create a disparity in access between England and Wales on the one hand, and Scotland on the other.
  • To date, ten European countries, including France, Germany and Italy, also reimburse the medicine for the treatment of adults with XLH.
  • However, Kyowa Kirin remains committed to continued engagement with NICE to ensure patients living with XLH are able to access the treatment.
  • We fully support the UK Rare Disease Framework and specifically priority 4 - improving access to specialist care, treatments and drugs.

Ultragenyx Reports Third Quarter 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, November 2, 2023
Patient, Șaeș, Ageing, Incidence, Ultragenyx, BMD, Annual general meeting, Fracture, Society, Burosumab, OI, American Society for Bone and Mineral Research, Mineral, Beauty salon, Pharmaceutical industry, Orbit, Cosmic

NOVATO, Calif., Nov. 02, 2023 (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 reported its financial results for the quarter ended September 30, 2023 and provided its financial guidance for the year.

Key Points: 
  • For the full year 2023, the company expects:
    Crysvita revenue in the range of $325 million to $340 million.
  • This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
  • In May 2023, Ultragenyx announced the last patient had been dosed in the Phase 3 study.
  • ET to discuss the third quarter 2023 financial results and provide a corporate update.

Kyowa Kirin Presents Real-World Findings at ASBMR Annual Meeting Highlighting Progressive Disease Burden of X-linked Hypophosphatemia (XLH)

Retrieved on: 
Monday, October 16, 2023
Komodo, Diagnosis, Enthesopathy, Osteoarthritis, MBA, Health care, Physician, Hypertension, Obesity, Research, Calcitriol, Myalgia, Burosumab, Prevalence, Tendon, X-linked hypophosphatemia, XLH, Society, Kyowa Kirin, TSE, Spinal stenosis, Health, American Society for Bone and Mineral Research, Arthralgia, Fracture, Disease, Medicare, Kidney, Depression, Patient, DSM-IV codes, Kidney disease, Commercial, FH, Muscle weakness, Pharmaceutical industry

Most patients had received some form of conventional therapy for XLH prior to burosumab treatment, with 61% receiving calcitriol and 43% receiving phosphate supplements.

Key Points: 
  • Most patients had received some form of conventional therapy for XLH prior to burosumab treatment, with 61% receiving calcitriol and 43% receiving phosphate supplements.
  • Older patients were under-represented in the study population, limiting the generalizability of the findings to this group.
  • In this retrospective observational cohort study, researchers examined healthcare claims data from the Merative™ MarketScan® Commercial and Medicare Databases.
  • The differences between the study groups were apparent even among young children and persisted throughout a person's life.

Ultragenyx Reports Second Quarter 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, August 3, 2023
Burosumab, Serum, BMD, Sinusitis, Ultragenyx, Patient, Ageing, Headache, OI, Osteogenesis imperfecta, Phosphorus, Pharmaceutical industry, Cosmic, Orbit

NOVATO, Calif., Aug. 03, 2023 (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 reported its financial results for the quarter ended June 30, 2023 and provided its financial guidance for the year.

Key Points: 
  • For the second quarter of 2023, Ultragenyx reported net loss of $159.8 million, or $2.25 per share basic and diluted, compared with a net loss for the second quarter of 2022 of $158.2 million, or $2.26 per share, basic and diluted.
  • Howard Horn was appointed as Ultragenyx’s Chief Financial Officer (CFO) and Executive Vice President, Corporate Strategy, effective October 16, 2023.
  • Ultragenyx will host a conference call today, Thursday, August 3, 2023, at 2 p.m. PT/5 p.m.
  • ET to discuss the second quarter 2023 financial results and provide a corporate update.

Ultragenyx Reports First Quarter 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, May 4, 2023
Ageing, Burosumab, Ultragenyx, Patient, OI, Osteogenesis imperfecta, Beauty salon

NOVATO, Calif., May 04, 2023 (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 reported its financial results for the quarter ended March 31, 2023 and reaffirmed its financial guidance for 2023.

Key Points: 
  • This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
  • In addition, in the second quarter of 2023, Ultragenyx intends to initiate Cosmic, a randomized study in children under age five with serious bone disease, which compares bisphosphonates to UX143.
  • No additional treatment-related serious adverse events or lower extremity weakness adverse events have occurred since our prior update in January 2023.
  • ET to discuss the first quarter 2023 financial results and provide a corporate update.

Kyowa Kirin North America Assumes Commercial Leadership Role for CRYSVITA® (burosumab-twza) Injection in North America

Retrieved on: 
Thursday, April 27, 2023
Burosumab, Hypophosphatemia, Education, Pledge, Ultragenyx, Research, Kyowa Kirin, Patient, TIO, FGF23, Neoplasm, Rare, X-linked hypophosphatemia, XLH, TSE, Pharmaceutical industry

BEDMINSTER, N.J., April 27, 2023 /PRNewswire/ -- Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a top 50 global specialty pharmaceutical company, today assumes primary commercial leadership for CRYSVITA® (burosumab-twza) injection in the United States and Canada from Ultragenyx Pharmaceutical, Inc., a transition planned for from the start of their decade-long collaboration.

Key Points: 
  • BEDMINSTER, N.J., April 27, 2023 /PRNewswire/ -- Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a top 50 global specialty pharmaceutical company, today assumes primary commercial leadership for CRYSVITA® (burosumab-twza) injection in the United States and Canada from Ultragenyx Pharmaceutical, Inc., a transition planned for from the start of their decade-long collaboration.
  • The transition builds on the company's existing commercial portfolio and commitment to address the needs of people living with rare diseases.
  • Their contributions were instrumental in expediting late-stage trials, securing regulatory approval, and delivering treatment to thousands of patients," said Steve Schaefer, President, Kyowa Kirin North America.
  • To prepare for its new commercial leadership role in North America, Kyowa Kirin has engaged with community leaders and people living with XLH and TIO to gain perspective on their experiences and how best to support them.

Ultragenyx Reports Fourth Quarter and Full Year 2022 Financial Results and Corporate Update

Retrieved on: 
Thursday, February 16, 2023
Ultragenyx, Research, Burosumab, Ageing, Acquisition, Osteogenesis imperfecta, OI, Patient, Cryptocurrency

NOVATO, Calif., Feb. 16, 2023 (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 reported its financial results for the quarter and full year ended December 31, 2022 and reaffirmed its financial guidance for 2023.

Key Points: 
  • Total operating expenses for the fourth quarter of 2022 were $249.0 million, including non-cash stock-based compensation of $29.4 million.
  • For the fourth quarter of 2022, Ultragenyx reported net loss of $151.8 million, or $2.16 per share basic and diluted, compared with a net loss for the fourth quarter of 2021 of $122.5 million, or $1.79 per share, basic and diluted.
  • Cash, cash equivalents, and marketable debt securities were approximately $896.7 million as of December 31, 2022.
  • ET to discuss the fourth quarter and full year 2022 financial results and provide a corporate update.