FDA Grants Orphan Drug Designation to PTX-252 for AML

The FDA has granted an orphan drug designation to PTX-252 for the treatment of patients with acute myeloid leukemia (AML), according to an announcement from Hyloris Pharmaceuticals.¹

PTX-252 is designed to improve the responsiveness of cancer cells to chemotherapy with a novel molecular entity derived from an established molecule.

“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,” Stijn Van Rompay, chief executive officer of Hyloris, stated in a news release.

In September 2023, joint collaborator Pleco Therapeutics announced the appointment of Dr Henno Welgemoed as chief medical officer. In this role, Henno will lead the development of PTX-252 in AML in 2024. Per the press release, the company is expected to launch the phase 2 study for the agent in 2024.²

In addition to PTX-252, Henno will oversee the development of several other products, including PTX-062 in lung cancer, PTX-142 in pancreatic cancer, and PTX-081 in other cancers. All of these agents are currently undergoing candidate selection or preclinical study.³

AML is a heterogeneous and highly proliferative cancer that originates from immature white blood cells in the bone marrow. It can present as de novo disease or occur secondarily due to the progression of other diseases or treatment with cytotoxic therapy. The median age of diagnosis is approximately 67 years, and the disease is more common in males. Global data estimates project that the number of newly diagnosed cases will increase from 148,352 in 2022 to 185,232 by 2032.¹

Each Plecoid™ therapy, coined after the Plecostomus fish that clean aquariums from the inside, is designed to target one of the underlying causes of cancer as opposed to the tumor itself. According to Pleco, each treatment is intended to change the balance of protein expression within the cancer microenvironment, removing toxic metals from the cell. This could result in improved chemotherapeutic anticancer activity.

“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,” Ivo Timmermans, MD, MBA, chief executive officer of Pleco Therapeutics,” added.¹

References

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 19, 2024. https://www.globenewswire.com/news-release/2024/01/16/2810252/0/en/Orphan-Drug-Designation-Granted-to-PTX-252-by-U-S-FDA-for-the-Treatment-of-Acute-Myeloid-Leukaemia-AML.html
2. Pleco Therapeutics announces the appointment of Dr Henno Welgemoed as chief medical officer. News release. Pleco Therapeutics. September 5, 2023. Accessed January 19, 2024. https://www.plecotherapeutics.com/news/38/86/Pleco-Therapeutics-Announces-the-Appointment-of-Dr-Henno-Welgemoed-as-Chief-Medical-Officer.html
3. Pipeline. Pleco Therapeutics. 2021. Accessed January 19, 2024. https://www.plecotherapeutics.com/pipeline.html