FDA Grants Orphan Drug Designation to PTX-252 in AML


PTX-252, a novel molecular drug, has received an FDA orphan drug designation for the treatment of patients with acute myeloid leukemia.

  • The FDA has granted an orphan drug designation to PTX-252 in acute myeloid leukemia (AML).

  • Orphan drug designation is granted to drugs that treat or prevent life-threatening rare diseases.

  • A phase 2 study evaluating PTX-252 plans to begin in 2024.

An orphan drug designation has been granted by the FDA to PTX-252 for the treatment of patients with AML, according to Hyloris Pharmaceuticals.1

“Securing orphan drug designation for a product candidate incorporating a novel molecular entity not yet approved by any regulatory agency underscores our unwavering commitment to advancing the frontiers of scientific discovery within the repurposing space,” said Stijn Van Rompay, chief executive officer of Hyloris, in a press release.

Drugs are granted orphan drug designations from the FDA if they treat or prevent life-threatening rare diseases, including conditions that currently have no approved treatments. Drugs given orphan drug designation can provide a significant clinical benefit when compared with existing treatments.1

PTX-252 is a novel molecular drug which aims to improve the responsiveness and increase the sensitivity of cancer cells to chemotherapy with a molecular entity derived from an established molecule.

Photomicrograph of bone marrow aspirate showing myeloblasts of AML: © David A Litman - stock.adobe.com

Photomicrograph of bone marrow aspirate showing myeloblasts of AML: © David A Litman - stock.adobe.com

Pleco Therapeutics worked with Hyloris Pharmaceuticals SA for the development of PTX-252. Following his appointment in September 2023, Henno Welgemoed, MD, chief medical officer of Pleco Therapeutics, will lead the development of PTX-252 in AML. The agent will undergo evaluation in a phase 2 study which plans to launch in 2024.2

Welgemoed also is overseeing the development of PTX-062 for the treatment of patients with lung cancer, PTX-142 for treating pancreatic cancer, and PTX-081 in other cancers. Currently, all these agents are undergoing candidate selection or being evaluated in preclinical studies.3

“This milestone underscores our commitment to innovative therapies for rare diseases and it brings hope to AML patients who have limited treatment options. Our team is dedicated to advancing this therapy through clinical development as swiftly as possible,” added Ivo Timmermans, MD, MBA, chief executive officer of Pleco Therapeutics, in a press release.1

  1. Orphan drug designation granted to PTX-252 by US FDA for the treatment of acute myeloid leukemia (AML). News release. Hyloris Pharmaceuticals. January 16, 2024. Accessed January 22, 2024. http://tinyurl.com/5xems5f2
  2. Pleco Therapeutics announces the appointment of Dr Henno Welgemoed as chief medical officer. News release. Pleco Therapeutics. September 5, 2023. Accessed January 22, 2024. http://tinyurl.com/45trwkyx
  3. Pipeline. Pleco Therapeutics. 2021. Accessed January 22, 2024. https://www.plecotherapeutics.com/pipeline.html
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