PTX-252 Earns FDA Orphan Drug Designation in Acute Myeloid Leukemia

The novel agent was designed to include a novel molecular entity that is an alternative formation of a known established molecule, which is believed to enhance how cancer cells respond to chemotherapy.

The FDA has granted orphan drug designation to PTX-252 as a treatment for those with acute myeloid leukemia (AML), according to a press release from Hyloris Pharmaceuticals SA.¹

“This milestone underscores our commitment to innovative therapies for rare diseases and it brings hope to [patients with] AML who have limited treatment option,” Ivo Timmermans, chief executive officer at Pleco Therapeutics, said in the press release.¹ “Our team is dedicated to advancing this therapy through clinical development as swiftly as possible.”

The novel agent was designed to include a novel molecular entity that is an alternative formation of a known established molecule, which is believed to enhance how cancer cells respond to chemotherapy. With the orphan drug designation for PTX-252, developers may be eligible to receive financial incentives, market exclusivity, and support for managing regulatory processes.

According to the press release, the median age of patients with newly diagnosed AML is approximately 67 years. Additionally, there were 148,351 diagnosed cases of AML based on estimates from GlobalData; it is predicted that the number of diagnoses will increase to 185,323 by 2032.

“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 at Hyloris, said.

Pleco Therapeutics appointed Henno Welgemoed, MBChB, as its chief medical officer in September 2023.² As part of his new role, Welgemoed will lead the efforts in clinically developing and studying PTX-252 in the management of AML. It is anticipated that developers will initiate a phase 2 study evaluating PTX-252 in the aforementioned population in 2024. Additionally, Welgemoed will organize initiatives for studying other drug candidates in the preclinical setting across various disease types including small cell lung cancer (SCLC) and pancreatic cancer.

“With his deep expertise in [hematology-oncology] and a vast network among the world's leading experts, Henno will be a great asset to our team now that we advance our novel cancer therapies through clinical trials,” Ivo Timmermans, chief executive officer at Pleco Therapeutics, said in a press release at the time of the appointment.² “We have patient care at heart, and I am confident that under Henno's leadership and vision, our commitment to develop innovative solutions for the most difficult-to-treat types of cancer will be strengthened.”

According to developers at Pleco Therapeutics, a model system has been created for investigating how metals accumulate and interact with chemotherapy and cancer.3 It is believed that this model may allow for investigators to assess the association between chelation therapy and metal-related diseases including AML and SCLC.

In addition to PTX-252 for the treatment of those with AML, developers are researching several other candidates that may effectively remove toxic metals while saving essential ones. Other products include PTX-062, which is in the preclinical development stage for the management of lung cancer.³ Additionally, developers are investigating PTX-142 as a treatment for patients with pancreatic cancer.

References

1. Orphan drug designation granted to PTX-252 by U.S. FDA for the treatment of acute myeloid leukaemia (AML). News release. Hyloris Pharmaceuticals SA. January 16, 2024. Accessed January 23, 2024. http://tinyurl.com/ye22c537
2. Pleco Therapeutics announces the appointment of Dr Henno Welgemoed as chief medical officer. News release. Pleco Therapeutics BV. September 5, 2023. Accessed January 23, 2024. http://tinyurl.com/4tc48cvj
3. Pipeline. Pleco Therapeutics BV. Accessed January 23, 2024. http://tinyurl.com/mutu6sz9