Pacylex Granted FDA Fast Track Designation for PCLX-001 for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

On November 22, 2022 Pacylex, a clinical-stage N-myristoyltransferase (NMT) inhibitor company developing first-in-class therapies for leukemia, lymphoma, and solid tumor cancers, reported that the U.S. Food and Drug Administration (FDA) has granted PCLX-001 Fast Track Designation for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Greenfire, NOV 22, 2022, View Source [SID1234624533]). Fast Track is a process to facilitate drug development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address unmet medical needs.

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The FDA’s decision was informed by the results of Pacylex’s nonclinical studies and an ongoing Phase 1/2 clinical study of PCLX-001 in non-Hodgkin Lymphoma (NHL) and solid tumor patients, which showed that treatment with PCLX-001 had a favorable safety and tolerability profile. "Receiving Fast Track designation from the FDA reinforces Pacylex’s belief in PCLX-001 as a potential new treatment for AML," said Michael Weickert, PhD, CEO of Pacylex.

"Because Acute myeloid leukemia is a rapidly progressing, difficult-to-treat blood cancer with a poor prognosis, and Pacylex’s nonclinical data with PCLX-001 suggests that AML may be the cancer type most sensitive to NMT inhibitors, we have been eager to advance PCLX-001 into clinical studies in AML patients in the near future," said John Mackey, MD, CMO of Pacylex.

Earlier this month the FDA cleared the IND for PCLX-001 for Phase 1/2 clinical study to start in AML patients in the coming months. The FDA also recently granted PCLX-001 Orphan Drug Designation for AML. PCLX-001 is currently being studied in NHL and solid tumor cancer patients at 4 sites in Canada. Pacylex has the first and only NMT inhibitor in clinical studies.

In the ongoing clinical studies of PCLX-001, seventeen patients have been accrued through 5 dose levels of oral, once-per-day PCLX-001 with no dose limiting toxicities observed. The ongoing clinical PCLX-001 trial in NHL and solid tumor patients is registered at Identifier: NCT04836195. Data on the scientific rationale for PCLX-001 in AML patients will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition from 10-13 December 2022.

About Acute Myeloid Leukemia
In 2020, more than 474,500 new cases of leukemia were reported globally with more than 311,500 deaths (Source: 1). In 2017, AML accounted for 23.1% of total leukemia cases worldwide, and it is one of the most common types of leukemia in adults (Source: 2,3). In the U.S., an estimated 20,050 new cases of AML will be diagnosed in 2022 with the five-year survival rate reported at 30.5% (Source: 2,4).

The conventional treatment for newly diagnosed AML is intensive induction and consolidation chemotherapy with HSCT for eligible patients (Source: 5). The introduction of new targeted therapies in recent years has added to the standard of care and improved outcomes for some patients with molecularly defined AML subtypes (Source: 6). However, there remains a need to improve survival for the majority of patients with AML (Source: 5).

PCLX-001 (aka DDD86481) is a first-in-class, small molecule NMT inhibitor originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness, funded by Welcome Trust. Pacylex is developing PCLX-001 in the form of a once-a-day pill initially to treat leukemia and lymphoma. PCLX-001 has also been shown to inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the levels of NMT2 are correlated with survival, suggesting an important biological role in these cancers.