Voxelotor

Centessa Pharmaceuticals Announces Addition of Patrick Yue, MD, as Senior Vice President of Clinical Development, Innovative Medicines

Retrieved on: 
Mercredi, mars 1, 2023
Beth Israel Deaconess Medical Center, Pfizer, Therapy, Sickle cell disease, MD, Senior, Voxelotor, CNTA, Washington University School of Medicine, PAH, BS, Patient, Haemophilia, Drug, Stanford University School of Medicine, California Institute for Regenerative Medicine, Narcolepsy, Computational Biology and Chemistry, Pharmaceutical industry, Biology, Chemistry, California Institute of Technology

BOSTON and LONDON, March 01, 2023 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company that aims to discover and develop medicines that are transformational for patients, today announced the appointment of Patrick Yue, MD, to Centessa’s management team as Senior Vice President of Clinical Development, Innovative Medicines.

Key Points: 
  • BOSTON and LONDON, March 01, 2023 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company that aims to discover and develop medicines that are transformational for patients, today announced the appointment of Patrick Yue, MD, to Centessa’s management team as Senior Vice President of Clinical Development, Innovative Medicines.
  • “Patrick is a physician-scientist who brings extensive clinical development experience to this newly created position at Centessa.
  • Prior to joining Centessa, Dr. Yue was Vice President of Clinical Science at Global Blood Therapeutics, where he oversaw four development programs, including voxelotor (Oxbryta) a treatment for sickle cell disease.
  • Dr. Yue has spent 12 years in the biotech/pharmaceutical industry, taking on roles of increasing responsibility at Gilead, Portola, Alexion and Pfizer.

Folia Health Technology Platform Used in First-of-its-Kind Real World Evidence Study for Sickle Cell Disease

Retrieved on: 
Jeudi, septembre 15, 2022
Infection, Hip, Center, Sickle cell disease, Acute chest syndrome, Voxelotor, Senior, High reliability organization, Stroke, End organ damage, Patient, GBT, ASCEND, CEO, SCD, Data collection, Degenerative disease, Blood, Anti-transglutaminase antibodies, Doctor of Pharmacy, Centers for Disease Control and Prevention, Founder, Caregiver, Statistics, Research, GLOBE, Health, Splenectomy, Disease, Individual, Pharmaceutical industry, Medical imaging

BOSTON, Sept. 15, 2022 (GLOBE NEWSWIRE) -- Today, Folia Health announced the initiation of the ASCEND Study, a first-of-its-kind, at-home observational real-world evidence study in which people living with sickle cell disease (SCD) will use Folias innovative technology platform to track their individual changes in symptoms and treatment use.

Key Points: 
  • BOSTON, Sept. 15, 2022 (GLOBE NEWSWIRE) -- Today, Folia Health announced the initiation of the ASCEND Study, a first-of-its-kind, at-home observational real-world evidence study in which people living with sickle cell disease (SCD) will use Folias innovative technology platform to track their individual changes in symptoms and treatment use.
  • The ASCEND Study, which is currently enrolling patients, is being conducted by Folia Health with funding from Global Blood Therapeutics, Inc. (GBT).
  • The ASCEND study will utilize the Folia mobile app or desktop platform to capture this evidence directly from patients in real time.
  • We hope that using the Folia Health platform in the ASCEND Study will deliver new insights by enabling sickle cell disease patients to track their experiences and participate in meaningful research from their home.

GBT Initiates Phase 2/3 Clinical Trial of GBT601 in Patients with Sickle Cell Disease

Retrieved on: 
Mercredi, juin 29, 2022
Hemolysis, Discovery (observation), Patient, Sickle cell disease, Research, GLOBE, COVID-19, Blood, United States $1 Coin Act of 1997, Oxygen, Polymerization, GBT, FDA, HBS, Securities Act of 1933, Securities Exchange Act of 1934, Tissue, National Heart, Lung, and Blood Institute, PD, Compliance, Pharmacokinetics, SAN, Anemia, Drug Quality and Security Act, SCD, Center, Sickle, Acute chest syndrome, Goal, PK, Private Securities Litigation Reform Act, Capillary, NASDAQ, Lung, Pharmakon—Danish College of Pharmacy Practice, Adult, Stroke, U.S. Securities and Exchange Commission, End organ damage, Disease, United, Registry, Volatile organic compound, Maintenance, Government, Safety, Centers for Disease Control and Prevention, Pharmaceutical industry, HB, Voxelotor

The study ( NCT05431088 ) is a randomized, multicenter Phase 2/3 clinical trial evaluating the safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of GBT601.

Key Points: 
  • The study ( NCT05431088 ) is a randomized, multicenter Phase 2/3 clinical trial evaluating the safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of GBT601.
  • Based on compelling preclinical and clinical data, we believe GBT601 has the potential to be a best-in-class therapy for patients with sickle cell disease, said Kim Smith-Whitley, M.D., executive vice president and head of research and development of GBT.
  • The initiation of our Phase 2/3 trial is an important milestone in our efforts to bring GBT601 to patients.
  • GBT601 is being studied in a Phase 1 clinical trial and Phase 2 portion of a Phase 2/3 clinical trial.

NFL Running Back Tevin Coleman and Family Team Up with GBT to Share Playbook for Families Affected by Sickle Cell Disease

Retrieved on: 
Jeudi, juin 16, 2022
Sickle cell disease, Capillary, United States $1 Coin Act of 1997, Disease, Drug Quality and Security Act, Health, Caregiver, Lung, National Heart, Lung, and Blood Institute, U.S. Securities and Exchange Commission, GLOBE, Hemolytic anemia, Tissue, Polymerization, Compliance, Private Securities Litigation Reform Act, NASDAQ, Social media, Twitter, Oxygen, Akilah, Center, United, FDA, End organ damage, COVID-19, Hope, Government, HBS, Acute chest syndrome, Parent, Anti-transglutaminase antibodies, Erik Coleman, Securities Act of 1933, SCD, Patient, Degenerative disease, Instagram, Blood, Securities Exchange Act of 1934, Safety, Stroke, Centers for Disease Control and Prevention, Volatile organic compound, Research, Family, GBT, Sickle, Partnership, Pharmakon—Danish College of Pharmacy Practice, Awareness, SAN, Goal, Pharmaceutical industry, Voxelotor, Facebook

This World Sickle Cell Day and Fathers Day, I am especially grateful for my daughter, Nazaneen, who lives and thrives with sickle cell disease, said Tevin Coleman, New York Jets running back and sickle cell dad.

Key Points: 
  • This World Sickle Cell Day and Fathers Day, I am especially grateful for my daughter, Nazaneen, who lives and thrives with sickle cell disease, said Tevin Coleman, New York Jets running back and sickle cell dad.
  • Too many children and adults with sickle cell go untreated, dont have regular checkups, and only get care when they suffer from symptoms.
  • Tevin and Akilah will share their playbook for families impacted by sickle cell via media opportunities, in-person appearances, social media, and sickle cell community engagement.
  • To learn more, follow Tevin Coleman on Twitter and Instagram , Akilah Coleman on Twitter and Instagram , and Sickle Cell Speaks on Instagram and Facebook .

GBT Presents Positive New Real-World Evidence Data at EHA2022 Congress Further Supporting Clinical Use of Oxbryta® (voxelotor) in Sickle Cell Disease

Retrieved on: 
Vendredi, juin 10, 2022
United Kingdom, Polymerization, EDT, Sickle cell disease, Roger Hawkins (drummer), Research, Hemolysis, Clinical Practice, Anemia, CEST, Clinical Practice Research Datalink, European Hematology Association, End organ damage, Â, SCD, GLOBE, Risk, NHS Digital, EHA, NASDAQ, Royal commission, European Commission, HBS, Poster, Cohort study, Safety, GBT, Pharmaceutical industry, Voxelotor, HB, Patient

SOUTH SAN FRANCISCO, Calif., June 10, 2022 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced new data from a large multicenter real-world evidence study supporting the clinical use of Oxbryta® (voxelotor) for the treatment of sickle cell disease (SCD) in patients 12 years of age and older. In addition, Phase 1 data of GBT021601 (GBT601), the company’s next-generation sickle hemoglobin (HbS) polymerization inhibitor, support its progression into the Phase 2 portion of a Phase 2/3 trial that is anticipated to commence by mid-year. These data, as well as new research from the United Kingdom demonstrating that an improvement of anemia as measured by hemoglobin (Hb) reduces the risk of end-organ damage in SCD patients, will be presented at the European Hematology Association (EHA) 2022 Hybrid Congress from June 9-12, 2022 in Vienna, Austria and online.

Key Points: 
  • A first-in-class, once-daily oral therapy, Oxbryta directly inhibits sickle hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD.
  • Oxbryta was well tolerated, with the majority of treatment-emergent adverse events (TEAEs) being mild to moderate.
  • Overall safety data was consistent with the Phase 3 HOPE Study of SCD patients ages 12 years and older.
  • The EHA2022 oral presentation (#S268) will be held on June 12 at 11:30 a.m. CEST (5:30 a.m. EDT).

GBT’s Inclacumab and GBT601 Receive U.S. FDA Orphan Drug and Rare Pediatric Disease Designations for the Treatment of Sickle Cell Disease

Retrieved on: 
Lundi, juin 6, 2022
Oxygen, Goal, Sickle cell disease, RPD, Acute chest syndrome, China Biologic Products, National Heart, Lung, and Blood Institute, Pain, Blood, COVID-19, Food and Drug Administration, European Medicines Agency, HBS, Tissue, Capillary, U.S. Securities and Exchange Commission, Volatile organic compound, Stroke, Food, Pharmacokinetics, Awareness, Hemolytic anemia, Degenerative disease, Securities Exchange Act of 1934, Lung, Securities Act of 1933, Pharmakon—Danish College of Pharmacy Practice, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, GBT, Polymerization, Registry, Roche, Government, Cell adhesion, Patient, Compliance, Private Securities Litigation Reform Act, Research, Drug Quality and Security Act, HCUP, United, Disease, Maintenance, Center, FDA, SCD, GLOBE, NASDAQ, SAN, P-selectin, Safety, Pharmaceutical industry, Voxelotor

SOUTH SAN FRANCISCO, Calif., June 06, 2022 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations for inclacumab and GBT021601 (GBT601) for the treatment of sickle cell disease (SCD). Inclacumab is a novel P-selectin inhibitor currently in Phase 3 clinical trials to evaluate its potential to reduce the occurrence of vaso-occlusive crises (VOCs) and readmissions due to VOCs in patients with SCD. GBT601, a next generation sickle hemoglobin (HbS) polymerization inhibitor, is being studied in a restarted Phase 1 clinical trial and is expected to advance into the Phase 2 portion of a Phase 2/3 trial by mid-year. “The FDA’s orphan drug and rare pediatric disease designations for both inclacumab and GBT601 are an acknowledgment of the critical and ongoing unmet need in sickle cell disease and the potential of GBT’s innovative pipeline of investigational medicines,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development at GBT. “We believe that both inclacumab and GBT601 have the potential to be best-in-class therapeutic options for the treatment of this devasting disease. We are excited to continue these clinical development programs to make progress on our goal of transforming sickle cell disease into a well-managed condition via multiple therapeutic approaches.”

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., June 06, 2022 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations for inclacumab and GBT021601 (GBT601) for the treatment of sickle cell disease (SCD).
  • We believe that both inclacumab and GBT601 have the potential to be best-in-class therapeutic options for the treatment of this devasting disease.
  • Inclacumab selectively targets P-selectin, a protein that mediates cell adhesion and is clinically validated to reduce pain crises.
  • The second registrational trial, GBT2104-132 ( NCT04927247 ), is evaluating the effect of a single dose of inclacumab on hospital readmission rates.

GBT Announces Pricing of Upsized Offering of $300.0 Million of Convertible Senior Notes

Retrieved on: 
Mercredi, décembre 15, 2021
Therapy, Tablet, U.S. Securities and Exchange Commission, FDA, Government, Pharmakon—Danish College of Pharmacy Practice, Securities Exchange Act of 1934, Securities Act of 1933, Exercise, Private Securities Litigation Reform Act, SEC Rule 144A, SAN, Sale, COVID-19, Time, NASDAQ, Sickle cell disease, SEC, Debt-to-GDP ratio, Nasdaq, GBT, Research, Disease, Observation, Investment, GLOBE, Person, SCD, Compliance, Polymerization, Security (finance), Cryptocurrency, Voxelotor

The offering size was increased from the previously announced offering size of $250.0 million aggregate principal amount of notes.

Key Points: 
  • The offering size was increased from the previously announced offering size of $250.0 million aggregate principal amount of notes.
  • GBT also granted the initial purchasers of the notes a 13-day option to purchase up to an additional $45.0 million aggregate principal amount of notes (the additional notes).
  • GBT may not redeem the notes prior to December 20, 2027.
  • GBT intends to use approximately $40.7 million of the net proceeds from the offering of notes to pay the cost of the capped call transactions.

GBT Announces Proposed Offering of $250.0 Million of Convertible Senior Notes

Retrieved on: 
Mardi, décembre 14, 2021
Therapy, Tablet, U.S. Securities and Exchange Commission, FDA, Government, Pharmakon—Danish College of Pharmacy Practice, Securities Exchange Act of 1934, Securities Act of 1933, Exercise, Private Securities Litigation Reform Act, SEC Rule 144A, SAN, Sale, COVID-19, Time, NASDAQ, Sickle cell disease, SEC, Debt-to-GDP ratio, GBT, Research, Disease, Observation, Investment, GLOBE, Person, SCD, Compliance, Polymerization, Security (finance), Voxelotor

GBT also intends to grant the initial purchasers of the notes a 13-day option to purchase up to an additional $37.5 million aggregate principal amount of notes (the additional notes).

Key Points: 
  • GBT also intends to grant the initial purchasers of the notes a 13-day option to purchase up to an additional $37.5 million aggregate principal amount of notes (the additional notes).
  • The notes will be senior, unsecured obligations of GBT, and interest on the notes will be payable semi-annually in arrears.
  • The notes will be convertible into cash, shares of GBTs common stock or a combination thereof, at GBTs election.
  • The interest rate, conversion rate and other terms of the notes are to be determined upon pricing of the offering.

GBT Reports Second Quarter 2021 Financial Results

Retrieved on: 
Mardi, août 3, 2021
Economy of New York City, NASDAQ, Voxelotor, Finance

SOUTH SAN FRANCISCO, Calif., Aug. 03, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported financial results for the second quarter ended June 30, 2021 and recent business progress.

Key Points: 
  • ET
    SOUTH SAN FRANCISCO, Calif., Aug. 03, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported financial results for the second quarter ended June 30, 2021 and recent business progress.
  • GBT believes that if the pandemic subsides in the second half of 2021, the number of new prescriptions will improve incrementally.
  • Total net product sales for the second quarter of 2021 were $47.6 million, resulting from sales of Oxbryta, compared to $31.5 million for the second quarter of 2020.
  • ET to discuss the financial results for the second quarter 2021 and provide a general business update.

GBT’s Voxelotor is First Sickle Cell Disease Treatment to Receive Promising Innovative Medicine (PIM) Designation in the UK

Retrieved on: 
Lundi, juin 14, 2021
Health sciences, Health, Breakthrough therapy, Clinical medicine, Department of Health and Social Care, Orphan drugs, MHRA, Medicines and Healthcare products Regulatory Agency, Sickle cell disease, Voxelotor, EAMS

The sickle cell disease community, which for decades has been dramatically underserved, deserves innovative treatments that address the underlying cause of this debilitating disease, said Nigel Nicholls, UK general manager of GBT.

Key Points: 
  • The sickle cell disease community, which for decades has been dramatically underserved, deserves innovative treatments that address the underlying cause of this debilitating disease, said Nigel Nicholls, UK general manager of GBT.
  • Voxelotor is the first SCD treatment to receive the PIM designation, and this is a significant milestone in our efforts to potentially make this therapy available in the UK.
  • If approved for EAMS, the voxelotor early access program would be further extended to eligible UK patients.
  • Oxbryta was granted Promising Innovative Medicine (PIM) designation in Great Britain from the Medicines and Healthcare Products Regulatory Agency (MHRA).