Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

Investigational Drug Shows Promising Results in Phase II Study of Aggressive, Fatal Blood Disorder

By MD Anderson Cancer Center | November 14, 2018

A Phase I/II study, led by investigators at The University of Texas MD Anderson Cancer Center, reports an investigational drug called tagraxofusp has demonstrated high response rates in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare but highly aggressive – and often fatal bone marrow and blood disorder – for which there are no existing approved therapies.

Findings from the study are being presented Dec. 3 at the 60th American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego by Naveen Pemmaraju, M.D., associate professor of Leukemia.

“This was the largest prospectively designed multi-center, multi-cycle clinical trial specifically dedicated to patients with BPDCN,” said Pemmaraju. “We observed high response rates including an overall response rate of 90 percent among frontline-treated patients. “These findings give us hope for patients who have had no treatments specific to this disorder.”

BPDCN patients historically have experienced poor outcomes and low response rates with a variety of cytotoxic chemotherapy approaches following diagnosis. While there are no approved therapies or standard of care, patients quite often receive chemotherapy approved for other blood cancers, and/or stem cell transplants, however the majority of patients are older or do not qualify for standard chemotherapy approaches.

Forty-five patients with a median age of 70 years were enrolled in the seven-site study which provided tagraxofusp as a first-line or relapsed/refractory treatment. Median follow up for the study was 13.8 months.

Patients are continuing to be enrolled for the next stage of the clinical trial which will ensure ongoing access to the drug. Tagraxofusp is a novel targeted therapy that targets CD-123, a cell surface receptor, expressed in BPDCN and other hematologic malignancies.

The drug is also being investigated in other clinical trials, including for patients with chronic myelomonocytic leukemia (CMML) and myelofibrosis. Tagraxofusp received a Breakthrough U.S. Food and Drug Administration therapy designation with a rolling biological license application submitted to the FDA.


Filed Under: Drug Discovery

 

Related Articles Read More >

Sai Life Sciences exec: GLP-1 boom has ‘exploded the peptide field’ as firm opens new center
Novartis in the Pharma 50
Swissmedic approves first malaria treatment for infants
Korean team reports all-in-one cancer nanomedicine in pre-clinical studies
Nektar’s Phase 2b atopic dermatitis win triggers 1,746% analyst target surge, but legal tussle with ex-partner Lilly could complicate path forward
“ddd
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in the drug discovery and development industry.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Drug Discovery and Development
  • MassDevice
  • DeviceTalks
  • Medtech100 Index
  • Medical Design Sourcing
  • Medical Design & Outsourcing
  • Medical Tubing + Extrusion
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE