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Diagonal Therapeutics to Present Data at ASH 2024 Demonstrating the Economic and Clinical Burden of Hereditary Hemorrhagic Telangiectasia (HHT)

- Study finds annual healthcare costs per patient are significantly higher than previously described

- HHT costs are higher than other expensive-to-treat rare diseases and exceedingly higher in people who are anemic and require hematological support, highlighting the need for new treatment options

WATERTOWN, Mass., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Diagonal Therapeutics, a biotechnology company pioneering a new approach to discovering and developing agonist antibodies to address the root cause of genetic diseases, today announced that a study in health economics and outcomes research focused on the cost and clinical burden in people suffering with hereditary hemorrhagic telangiectasia (HHT) will be presented at the American Society of Hematology, taking place in San Diego, December 7-10, 2024. The study, performed jointly with Dr. Hanny Al-Samkari, Peggy S. Blitz Endowed Chair in Hematology/Oncology at the Massachusetts General Hospital and Associate Professor of Medicine at Harvard Medical School, confirms that actual healthcare costs associated with HHT are significantly higher than historical data suggest and are similar to or exceed that of other severe rare diseases. The presentation will be given by Dr. Al-Samkari.

In the study, researchers analyzed data from a large, nationally representative U.S. claims database with more than 330 million covered lives with commercial insurance, Medicare and Medicaid, and included inpatient, outpatient and pharmacy claims. The study reports on the per-patient, per-year costs for 24,407 and 23,524 HHT patients identified for 2022 and 2023, respectively.

Data Highlights:

  • Mean annual costs per individual across all people with HHT were $19,386 and $19,398 in 2022 and 2023, respectively.
  • Annual costs were higher for those with HHT-related anemia, $27,147 (2022) and $26,694 (2023). While only 57 percent (2022) and 56 percent (2023) of people in the dataset experienced anemia, those accounted for 80 percent and 77 percent of the total costs, respectively.
  • Moreover, costs for those requiring intravenous (IV) iron or red blood cell transfusions were $40,298 in 2022 and $37,827 in 2023, accounting for 25 percent and 24 percent of people with HHT, but 52 percent and 47 percent of the total cost, respectively.

"Our analysis demonstrates that the healthcare costs associated with HHT have been significantly underestimated. HHT is a devastating bleeding disorder that historically has been understudied,” said Alex Lugovskoy, Ph.D., Chief Executive Officer of Diagonal. "These data highlight the tremendous need for new therapies targeting the underlying cause of disease, which may help avoid later-stage complications associated with morbidity and mortality in people suffering from HHT.”

"We have long suspected that the true impact and cost of HHT is significantly underestimated,” said Marianne Clancy, MPA, Executive Director of Cure HHT. "This new information is highly valuable and underscores the significance of our effort to push for better disease-specific therapeutic options. This collaboration among Diagonal Therapeutics, MGH, Harvard Medical School, and Cure HHT is a critical step toward getting the appropriate recognition and care for people living with HHT.”

Presentation details:

  • Title: Characterizing the Healthcare Resource Utilization and Costs of Hereditary Hemorrhagic Telangiectasia (abstract)
  • Presenter: Hanny Al-Samkari, M.D., Massachusetts General Hospital and Harvard Medical School
  • Presentation Date: Saturday, December 7, 2024
  • Presentation Time: 2:15 PM EST
  • Room: Marriott Grand Ballroom 2-4

About Hereditary Hemorrhagic Telangiectasia (HHT)

HHT is a rare disease that affects more than 150,000 people in the U.S. and EU, and for which there are currently no approved therapies. In HHT, loss of function point mutations in the TGF-β receptor superfamily complex create two types of abnormal blood vessels - telangiectasias and arteriovenous malformations (AVMs) - that are fragile and susceptible to rupture and bleeding. These bleeding events lead to chronic anemia, necessitating frequent iron infusions or red blood cell transfusions. AVMs, if left untreated, may result in lung and brain hemorrhage, stroke, heart failure, and death. Diagonal's agonist antibody candidates reactivate dormant signaling, addressing the root cause of the disease.

About Diagonal Therapeutics

Diagonal Therapeutics is a biotech company pioneering a new approach to discovering and developing agonist antibodies. The Company's DIAGONAL platform combines proprietary computational and experimental techniques to overcome historical challenges associated with agonist antibody drug discovery. Diagonal's emerging pipeline - discovered using the DIAGONAL platform - has the potential to deliver life-changing therapies to patients by tackling the underlying cause of disease. For more information, please visit www.diagonaltx.com.

Media Contact:

Cory Tromblee

media@diagonaltx.com