International Society on Thrombosis and Haemostasis

World Thrombosis Day encourages public to "Move Against Thrombosis" to celebrate 10 years of raising awareness worldwide

Retrieved on: 
Friday, October 13, 2023

World Thrombosis Day engages 5,000 partner organizations from 120 countries to advance understanding of the treatment and prevention of blood clots.

Key Points: 
  • World Thrombosis Day engages 5,000 partner organizations from 120 countries to advance understanding of the treatment and prevention of blood clots.
  • For its 10th anniversary year, the campaign encourages people to Move Against Thrombosis with simple movements to increase blood flow.
  • Thrombosis can develop in, or travel to, blood vessels throughout the body, often causing symptoms that can be mistaken for other conditions.
  • Incorporating movement in blood clot prevention inspired World Thrombosis Day's Move Against Thrombosis theme.

World Thrombosis Day encourages public to "Move Against Thrombosis" to celebrate 10 years of raising awareness worldwide

Retrieved on: 
Friday, October 13, 2023

World Thrombosis Day engages 5,000 partner organizations from 120 countries to advance understanding of the treatment and prevention of blood clots.

Key Points: 
  • World Thrombosis Day engages 5,000 partner organizations from 120 countries to advance understanding of the treatment and prevention of blood clots.
  • For its 10th anniversary year, the campaign encourages people to Move Against Thrombosis with simple movements to increase blood flow.
  • Thrombosis can develop in, or travel to, blood vessels throughout the body, often causing symptoms that can be mistaken for other conditions.
  • Incorporating movement in blood clot prevention inspired World Thrombosis Day's Move Against Thrombosis theme.

FDA Grants Orphan Drug Designation to GC Biopharma's Drug Candidate for Thrombotic Thrombocytopenic Purpura

Retrieved on: 
Friday, October 6, 2023

YONGIN, South Korea, Oct. 5, 2023 /PRNewswire/ -- GC Biopharma Corp. announced today that GC1126A, its drug candidate for Thrombotic Thrombocytopenic Purpura (TTP), received an Orphan Drug Designation on September 27 from the U.S. Food and Drug Administration (FDA).

Key Points: 
  • YONGIN, South Korea, Oct. 5, 2023 /PRNewswire/ -- GC Biopharma Corp. announced today that GC1126A, its drug candidate for Thrombotic Thrombocytopenic Purpura (TTP), received an Orphan Drug Designation on September 27 from the U.S. Food and Drug Administration (FDA).
  • When designated as an orphan drug, there are incentives such as tax credits for clinical development costs and exemptions from PDUFA user fees Initial Pediatric Study Plan.
  • Speaking on this achievement, GC Biopharma stated, "We are dedicated to collecting data to develop a Best-in-Class treatment for such rare disorders.
  • We remain committed to our mission of providing patients with new treatment options by continuously developing innovative drugs."

Anthos Therapeutics Supported Global Survey of 3000+ Patients Prescribed Anticoagulants Detailing Impact of Patient-Relevant Bleeding (PRB) Presented at ISTH 2023

Retrieved on: 
Monday, June 26, 2023

CAMBRIDGE, Mass., June 26, 2023 (GLOBE NEWSWIRE) -- Anthos Therapeutics, a clinical-stage biotechnology company developing innovative therapies for cardiovascular and metabolic diseases, presented new data in collaboration with two internationally recognized patient advocacy groups, detailing the impacts of patient-relevant bleeding (PRB) on individuals prescribed anticoagulants. The oral presentation, Patient-Relevant Bleeding Events Among Patients Taking Anticoagulant Medication, was presented yesterday at ISTH – The International Society on Thrombosis and Haemostasis’ annual meeting, being held in Montreal, June 24-28, 2023.

Key Points: 
  • The oral presentation, Patient-Relevant Bleeding Events Among Patients Taking Anticoagulant Medication, was presented yesterday at ISTH – The International Society on Thrombosis and Haemostasis’ annual meeting, being held in Montreal, June 24-28, 2023.
  • More than 3,000 adult patients were recruited in partnership with StopAfib.org and the National Blood Clot Alliance (NBCA), to participate in an anonymous, online global survey with their members, designed to better understand so-called “minor” bleeding problems experienced by patients who take anticoagulants.
  • “We partnered with Anthos because they are putting patients first, such as through this patient survey.
  • This forward-thinking survey explores the impact of patient-relevant bleeding events on lifestyle, emotional and mental well-being, and adherence,” said Tarin Patrikis, Deputy Director of the National Blood Clot Alliance.

Pivotal Phase 3 Data Presented at ISTH 2023 Congress Spotlight TAK-755 Prophylaxis for Patients with Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

Retrieved on: 
Sunday, June 25, 2023

In the pivotal trial, no patient had an acute TTP event while receiving TAK-755 prophylactic treatment.

Key Points: 
  • In the pivotal trial, no patient had an acute TTP event while receiving TAK-755 prophylactic treatment.
  • In the pivotal trial, treatment-emergent adverse events (TEAEs) were reported in 10.3% of patients ages 12-68 receiving TAK-755 compared to 50% of patients receiving plasma-based therapy.
  • (Presentation Number: OC 14.4)
    Results were presented today in three oral presentations delivered at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress.
  • The FDA has accepted and granted Priority Review for Takeda’s Biologics License Application (BLA) for TAK-755 for the treatment of cTTP.

Hemab Therapeutics Presents New Preclinical Research Demonstrating Effects of Its Bispecific Antibody HMB-001 in Factor VII Deficiency

Retrieved on: 
Saturday, June 24, 2023

COPENHAGEN, Denmark and BOSTON, June 24, 2023 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced results today from preclinical research of HMB-001 in models of factor VII (FVII) deficiency at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress in Montreal.

Key Points: 
  • "The new preclinical data presented today show HMB-001 successfully targeted and accumulated endogenous FVIIa to levels that would be expected to provide clinical benefit in FVII deficiency, supporting the potential for HMB-001 in an additional underserved bleeding disorder."
  • FVII is a protein necessary in the formation of hemostatic plugs to control bleeding.
  • The total accumulation of FVIIa observed with HMB-001 was comparable to the normal range seen in healthy animals.
  • These initial results suggest HMB-001 may have potential application as a treatment for FVII deficiency.

VarmX Raises €30 Million Series B2 Financing for IND Approval and Preparations for Pivotal Trial

Retrieved on: 
Thursday, May 25, 2023

VarmX, a biotech company focusing on the development of innovative approaches for the reversal of anticoagulation, today announces it has successfully raised an additional €30 million in a Series B2 financing round.

Key Points: 
  • VarmX, a biotech company focusing on the development of innovative approaches for the reversal of anticoagulation, today announces it has successfully raised an additional €30 million in a Series B2 financing round.
  • Existing investors EQT Life Sciences (formerly LSP), Inkef, Lundbeckfonden BioCapital, Ysios Capital, BioGeneration Ventures (BGV), InnovationQuarter (IQ) and Libertatis Ergo Holding (LEH) all participated in the Series B2 round.
  • The proceeds from the financing will enable VarmX to obtain investigational new drug (IND) approval for its lead compound VMX-C001 and to complete preparations for the pivotal clinical trial, including large scale manufacturing.
  • VarmX has completed enrollment of VMX-C001’s first-in-human (FIH) study, to demonstrate safety and provide clinical proof of concept of the compound.

Rallybio Announces Clinical Proof-of-Concept Results for RLYB211, an Anti-HPA-1a Polyclonal Antibody for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT), Published in the Journal of Thrombosis and Haemostasis

Retrieved on: 
Thursday, April 6, 2023

FNAIT is a rare and life-threatening bleeding disorder in which maternal alloantibodies directed against fetal platelets can lead to devastating outcomes for the fetus/neonate.

Key Points: 
  • FNAIT is a rare and life-threatening bleeding disorder in which maternal alloantibodies directed against fetal platelets can lead to devastating outcomes for the fetus/neonate.
  • There are currently no approved therapies for the prevention or treatment of FNAIT.
  • The findings were published in the Journal of Thrombosis and Haemostasis, the official journal of the International Society on Thrombosis and Haemostasis.
  • “We are pleased that these clinical results were published in the Journal of Thrombosis and Haemostasis, indicating that RLYB211 markedly accelerated the elimination of HPA-1a-positive platelets.

Bayer Announces First Patients Enrolled in Global Phase III Studies for Investigational Oral FXIa Inhibitor Asundexian

Retrieved on: 
Wednesday, February 8, 2023

“Additional research into the combination of FXI inhibition and antiplatelet agents is needed to explore potential treatment options for the future.”

Key Points: 
  • “Additional research into the combination of FXI inhibition and antiplatelet agents is needed to explore potential treatment options for the future.”
    “Through our OCEANIC clinical trial program, Bayer leverages decades of cardiovascular expertise to investigate a potential new class of antithrombotics and explore the potential of asundexian as an additional antithrombotic treatment option across multiple patient populations,” 1-2 said Maria Borentain, M.D., Vice President and Head of Thrombosis and Vascular Disease Clinical Development, Bayer.
  • Effect of factor XIa inhibition with asundexian on recurrent ischemic stroke according to baseline patterns of infarction on brain MRI: PACIFIC-Stroke.6
    Khatri P, Shoamanesh A, Mundl H, et al.
  • Effects of the oral factor XIa inhibitor asundexian on intracranial bleeding among patients with acute non-cardioembolic ischemic stroke: PACIFIC-Stroke randomized trial.
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WFH Gene Therapy Registry: now live!

Retrieved on: 
Wednesday, January 18, 2023

MONTREAL, Jan. 18, 2023 /PRNewswire/ - To ensure patient safety remains at the forefront of the work of the World Federation of Hemophilia (WFH), we have developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR).

Key Points: 
  • MONTREAL, Jan. 18, 2023 /PRNewswire/ - To ensure patient safety remains at the forefront of the work of the World Federation of Hemophilia (WFH), we have developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR).
  • The launch of the WFH GTR coincides with the approval of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA)¹ and the European Medicines Agency (EMA)².
  • This worldwide endeavour aims to collect important data on all patients who receive gene therapy, whether through a clinical trial or through a post-marketed product.
  • The WFH GTR is now ready to enroll all PWH who have received gene therapy, through a clinical trial or through a marketed product.