HHT Cost & Impact: New Data from Diagonal Therapeutics
Diagonal Therapeutics Advances Novel Treatment for Rare Genetic Disorder, Hereditary Hemorrhagic Telangiectasia (HHT)
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BOSTON, MA – [Date] – Diagonal Therapeutics, a biotechnology company focused on disease-modifying therapies, is making significant strides in the development of a novel treatment for Hereditary Hemorrhagic Telangiectasia (HHT), a rare and debilitating genetic disorder. The company’s first-in-class bispecific ALK1 agonist antibody,DIAG723,has received orphan drug designation from the U.S. Food and Drug Governance (FDA) and a positive opinion from the European Medicines Agency (EMA), paving the way for potential disease-modifying treatment options for the over 150,000 individuals affected in the U.S. and EU.
Understanding Hereditary Hemorrhagic Telangiectasia (HHT)
HHT is a rare genetic disease characterized by the formation of abnormal, fragile blood vessels prone to rupture and bleeding. These malformations can occur in various organs, including the lungs, brain, and gastrointestinal tract, significantly impacting patients’ quality of life.
Currently, there are no approved therapies specifically designed to treat the underlying cause of HHT. patients often experience:
Frequent Bleeding: Nosebleeds, gastrointestinal bleeding, and skin lesions are common, leading to chronic anemia. Anemia & Transfusions: Many patients require regular iron infusions or red blood cell transfusions to manage anemia. Serious Complications: Untreated arteriovenous malformations (AVMs) can lead to life-threatening complications such as lung hemorrhage, brain hemorrhage, stroke, and heart failure.
reduced Quality of Life: The constant threat of bleeding and associated symptoms drastically reduce a patient’s ability to participate in daily activities.
recent data analysis utilizing Komodo Health’s Healthcare Map®, a database encompassing de-identified data from over 330 million patients across the United States, highlights the significant unmet need for effective HHT treatments and underscores the potential impact of DIAG723.
DIAG723: A First-in-Class Approach to HHT Treatment
Diagonal Therapeutics is tackling HHT at its root cause with DIAG723, a bispecific ALK1 agonist antibody. This innovative approach aims to repair the dysfunctional signaling pathways responsible for the abnormal blood vessel formation characteristic of HHT.
“We are committed to advancing a differentiated treatment that directly addresses the root cause of HHT and are encouraged by the compelling preclinical profile of our first-in-class,bispecific ALK1 agonist antibody,” said Eric Duhaime,Senior Vice President of Corporate Development at Diagonal and co-author of the study. “We look forward to advancing development of DIAG723 as a novel, potentially disease-modifying treatment option for people living with HHT.”
DIAG723’s unique mechanism of action offers the potential to not just manage symptoms, but to actually modify the disease course, offering a significant advancement over current standards of care.The orphan drug designation and positive EMA opinion are crucial milestones, accelerating the path towards potential patient access.
Diagonal Therapeutics: Pioneering Disease-Modifying Therapies
Diagonal Therapeutics is a biotechnology company dedicated to developing innovative, disease-modifying clustering antibodies. The company’s proprietary DIAGONAL Product Engine combines cutting-edge computational and experimental techniques to accelerate antibody drug discovery and optimize therapeutic assets.
Diagonal’s pipeline focuses on selectively addressing the underlying causes of disease across a range of therapeutic areas, including hematology, hepatology, and nephrology. The company’s commitment to innovation and patient-centric drug development positions it as a leader in the field of rare disease therapeutics.For more details, visit: