Jan 16, 2024
Pleco Therapeutics receives FDA Orphan Drug Designation

Nijmegen, The Netherlands – 16th January 2024 – The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Pleco Therapeutics BV’s lead compound, PTX-252 for the treatment of Acute Myeloid Leukaemia (AML). PTX-252 is a novel molecular entity developed to increase the sensitivity of cancer cells to chemotherapy.

The FDA’s designation acknowledges the potential of PTX-252 in addressing AML, a rare blood cancer with a poor prognosis. Orphan Drug Designation is granted to medicines that treat or prevent life-threatening or chronically debilitating rare disease, and with either no currently approved method of prevention or treatment or with significant clinical benefit when compared with existing treatments. 

Orphan Drug Designation qualifies companies for incentives, including tax credits for clinical trials, exemption of user fees, and seven years of market exclusivity after approval.

"We are very excited to receive this designation for PTX-252 from the FDA," states Ivo Timmermans, Chief Executive Officer of Pleco Therapeutics. "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."

In the development of the lead compound PTX-252, Pleco has collaborated with Belgian company Hyloris Pharmaceuticals SA (Euronext Brussels: HYL). Stijn Van Rompay, Chief Executive Officer of Hyloris, comments: "Securing orphan drug designation for PTX-252 stands as a testament to our unwavering commitment to advancing the frontiers of scientific discovery within the repurposing space."

For more information, please contact:

Julie Powell, Commercial: M: +44 (0) 7774 827205 E: julie.powell@plecotherapeutics.com

Helen Pope, Media: M: +44 (0) 7879 818247 E: helen@helenpopepr.co.uk 


About Acute Myeloid Leukaemia (AML)

AML is a heterogenous haematological malignancy that originates from immature white blood cells (blasts) in the bone marrow. AML generally spreads quickly to the bloodstream and can then spread to other parts of the body including lymph nodes, spleen, central nervous system, and testicles. AML is an orphan disease, and it is the most common type of acute leukaemia in adults. The median age of newly diagnosed AML patients is around 67 years. Worldwide the incidence of AML is estimated to be 350,000 cases per year (4.7 cases per 100,000 population, Globocan). In the U.S., there were an estimated 20,050 new cases of AML in 2022 and 11,540 deaths. It can arise de novo or secondary to the progression of other diseases or following treatment with cytotoxic agents.

About Pleco Therapeutics 

Pleco Therapeutics is a clinical stage specialty biopharmaceutical company that aims to improve the survival rate of cancer patients through its novel Plecoid™ therapies that increase the effectiveness of current cancer treatments. A private company, Pleco is headquartered in Nijmegen, the Netherlands, with a U.S. subsidiary, Pleco Therapeutics USA Inc, based in Newark, New York.

For more information about Pleco Therapeutics and its work in rare diseases, including AML, visit www.plecotherapeutics.com

Follow us on LinkedIn: https://www.linkedin.com/company/pleco-therapeutics-bv/ 

About Hyloris Pharmaceuticals SA

Hyloris is a specialty biopharma company focused on innovating, reinventing, and optimizing existing medications to address important healthcare needs and deliver relevant improvements for patients, healthcare professionals and payors. Hyloris has built a broad, patented portfolio of 17 reformulated and repurposed value-added medicines that have the potential to offer significant advantages over available alternatives. Hyloris is based in Liège, Belgium. 

For more information, visit www.hyloris.com 

Follow us on LinkedIn: https://www.linkedin.com/company/hyloris-pharmaceuticals/