Osteogenesis imperfecta

Mereo BioPharma Reports Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Wednesday, March 27, 2024
Financial management, Patient, Ageing, ...Continued, OI, Epidemiology, EMA, Pediatrics, Osteogenesis imperfecta, FDA, Lithium

LONDON, March 27, 2024 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its financial results for the full year ended December 31, 2023, and provided an update on recent corporate highlights.

Key Points: 
  • “We reached a number of important milestones in 2023, which have set the stage for a potentially transformative 2024,” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo.
  • Cosmic was initiated in the second half of 2023 and is anticipated to be fully enrolled in the first half of 2024.
  • Accordingly, the full year 2023 financial results are presented in accordance with accounting principles generally accepted in the United States (U.S. GAAP) and in U.S. dollars.
  • Total ADS equivalents as of December 31, 2023 were 140,243,417, with each ADS representing five ordinary shares of the Company.

NextCure Provides Business Update and Reports Full Year 2023 Financial Results

Retrieved on: 
Thursday, March 21, 2024
Research, Toxicity, Bone disease, Agreement, Osteogenesis imperfecta, DAR, AML, Risk, Patient, MMAE, Mouse, OI, Public expenditure, Plaque, Apolipoprotein E, PRS, IND, ARIA, GMP, Osteoporosis, CRC, Workforce, Use, ADC, Osteoarthritis, FDA, GLP, Pharmaceutical industry

We plan to present the data of the CRC patients at a scientific conference within the second quarter of 2024.

Key Points: 
  • We plan to present the data of the CRC patients at a scientific conference within the second quarter of 2024.
  • Selected our first antibody drug conjugate (ADC) candidate of a potential of three from our collaboration with LegoChem Biosciences, Inc. (LegoChem).
  • Under the terms of the Agreement, both parties equally share the costs of developing the molecules and profits on commercialized products.
  • Implemented a restructuring plan to reduce operating costs and better align our workforce with the needs of our business.

Researchers at Phoenix Children's First in the World to Produce Mouse Lungs in Rats

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Monday, March 25, 2024
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PHOENIX, March 25, 2024 /PRNewswire/ -- Research conducted at the Phoenix Children's Research Institute at the University of Arizona College of Medicine — Phoenix reports the successful generation of a mouse lung in a rat, according to a paper published in the American Journal of Respiratory and Critical Care Medicine.

Key Points: 
  • The critical differentiating factor with this study is researchers incorporated CRISPR-Cas9, a unique genome editing technology that allows researchers to change genomes by altering sections of the DNA sequence.
  • Modifications of rat embryos by CRISPR-Cas9 allowed the formation of lung tissues consisting of almost all mouse lung cells.
  • This is one of many recent accolades for the Phoenix Children's Research Institute.
  • The Phoenix Children's Research Institute at the University of Arizona College of Medicine –Phoenix launched in May 2023, formalizing a longstanding research collaboration between the health system and the University of Arizona College of Medicine – Phoenix.

Mereo BioPharma Provides Update on Pipeline Progress and Corporate Developments

Retrieved on: 
Monday, January 8, 2024
U.S. FDA, FDA, EMA, Ageing, Osteogenesis imperfecta, Growth, Patient, OI

LONDON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today provided an update on its pipeline programs as well as an update on recent corporate developments.

Key Points: 
  • “2023 was a year of tremendous progress for Mereo.
  • “In addition, we significantly advanced the development of alvelestat, gaining valuable clarity on the regulatory path with both the FDA and EMA.
  • These developments are expected to further support our ongoing partnering activities for alvelestat.
  • We look forward to providing further updates on both setrusumab and alvelestat during the remainder of the year.

NextCure Provides Business Update and Reports Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023
Apolipoprotein E, Amyloid, Research, Acute myeloid leukemia, Plaque, Public expenditure, Conference, American Society for Bone and Mineral Research, Society, Osteoporosis, Mouse, ADC, Osteogenesis imperfecta, Annual general meeting, Cancer, Patient, AML, Mineral wool, Pharmaceutical industry, Cryptocurrency

Financial Results for Quarter Ended September 30, 2023

Key Points: 
  • Financial Results for Quarter Ended September 30, 2023
    Cash, cash equivalents, and marketable securities as of September 30, 2023, were $118.2 million as compared to $159.9 million as of December 31, 2022.
  • Research and development expenses were $11.0 million for the quarter ended September 30, 2023, as compared to $13.5 million for the quarter ended September 30, 2022.
  • General and administrative expenses were $4.6 million for the quarter ended September 30, 2023, as compared to $5.7 million for the quarter ended September 30, 2022.
  • Net loss was $14.3 million for the quarter ended September 30, 2023, as compared with a net loss of $18.9 million for the quarter ended September 30, 2022.

Mereo BioPharma Reports on Recent Program Developments and Provides Financial Update

Retrieved on: 
Monday, October 23, 2023
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LONDON, Oct. 23, 2023 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today provided an update on recent program developments and third quarter 2023 financial information.

Key Points: 
  • LONDON, Oct. 23, 2023 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today provided an update on recent program developments and third quarter 2023 financial information.
  • The Phase 3 portion of the study is currently enrolling approximately 195 patients at 50 sites across 12 countries.
  • There was no difference in the proportion of patients with treatment-related Adverse Events (AEs) between the alvelestat and placebo arms.
  • The data from the ATALANTa study are expected to further support the ongoing partnering process for the alvelestat Phase 3 program.

Preclinical Data Demonstrate Anti-Siglec-15 Treatment Reduced Bone Loss and Enhanced Bone Quality in Mice with Moderate-to-Severe Osteogenesis Imperfecta (OI)

Retrieved on: 
Tuesday, October 17, 2023
Hospital for Special Surgery, Male, Cortex, Spectroscopy, Female, BMD, Bone, Bone disease, Osteogenesis imperfecta, Society, Mab, Hospital, American Society for Bone and Mineral Research, Osteoporosis, Cancer, Fracture, Osteoclast, Calvarial doughnut lesions-bone fragility syndrome, Mouse, S15, Porosity, Life, OI, Patient, Medical imaging, Birth control

These results support development of NC605 as a potential highly effective treatment for osteogenesis imperfecta, a rare disease in which bones easily fracture.

Key Points: 
  • These results support development of NC605 as a potential highly effective treatment for osteogenesis imperfecta, a rare disease in which bones easily fracture.
  • Osteogenesis imperfecta is a rare disorder that results in high bone turnover, abnormal bone formation, bone fragility and recurrent fractures.
  • There is no cure for OI and current anti-resorptive treatments increase bone mineral density (BMD) primarily by inhibiting bone loss; however, these agents also inhibit bone formation.
  • Unlike anti-resorptive therapies, NC605 enhances osteoblast recruitment, resulting in overall enhanced bone quality.

Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease

Retrieved on: 
Monday, October 16, 2023
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NOVATO, Calif., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today will provide updates on its development pipeline, including setrusumab (UX143) for osteogenesis imperfecta (OI), GTX-102 for Angelman syndrome (AS), UX701 in Wilson disease and the rest of the company’s gene therapy portfolio at an Analyst Day held in New York City and by webcast.

Key Points: 
  • Long term data showed patients who stopped and restarted treatment reacquired previously gained developmental skills when they were re-dosed with the current regimen.
  • There have been no additional treatment-related SAEs, including lower extremity weakness, since November 2022.
  • ET on Monday, October 16, 2023 to discuss these data and to provide an update on the company’s development pipeline.
  • An archived version of the remarks will also be available through the Ultragenyx website.

Ultragenyx to Present Setrusumab (UX143) Update at ASBMR 2023 Including New Data from Phase 2/3 Orbit Study in Osteogenesis Imperfecta (OI)

Retrieved on: 
Monday, October 9, 2023
Mineral, Society, Ultragenyx, Ageing, Osteogenesis imperfecta, American Society for Bone and Mineral Research, OI, Patient, Rare-earth element, Pharmaceutical industry

The company will also present information on the Phase 3 Cosmic study design comparing setrusumab to intravenous bisphosphonate (IV-BP) in patients aged 2 to

Key Points: 
  • The company will also present information on the Phase 3 Cosmic study design comparing setrusumab to intravenous bisphosphonate (IV-BP) in patients aged 2 to
  • Details of the setrusumab presentations are as follows:
    Abstract Title: Evaluating Setrusumab for the Treatment of Osteogenesis Imperfecta: Phase 2 Data from the Phase 2/3 ORBIT Study (Lewiecki, E.M. et al.)
  • Abstract Title: Cosmic: An open-label, randomized, active-controlled, phase 3 study of setrusumab compared with bisphosphonates in pediatric subjects with osteogenesis imperfecta (Krolczyk, S. et al.)
  • The companies previously announced positive data from the dose-selection Phase 2 portion of the Orbit study showing that setrusumab rapidly induced bone production in OI-affected patients.

Ultragenyx to Present Pipeline Update at Analyst Day on Monday, October 16, 2023

Retrieved on: 
Thursday, October 5, 2023
Washington University School of Medicine, Angelman syndrome, Rehabilitation, Geriatric Orthopaedic Surgery & Rehabilitation, Medical director, Osteogenesis imperfecta, DSM-IV codes, Pediatric emergency medicine, Endocrinology, OI, Pharmaceutical industry, Pediatrics, Medicine, Ultragenyx

NOVATO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, will host an Analyst Day at 8:30 a.m.

Key Points: 
  • NOVATO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, will host an Analyst Day at 8:30 a.m.
  • ET on Monday, October 16, 2023.
  • Ultragenyx leaders and external experts will provide an update on the company’s development pipeline.
  • An archived version of the remarks will also be available through the Ultragenyx website.