Pacylex Pharmaceuticals CEO Presents Zelenirstat for Acute Myeloid Leukemia at the World Orphan Drug Congress

We are excited to announce that CEO Michael Weickert will present Phase 1 safety and efficacy results for zelenirstat and evidence supporting advancing it into clinical development in the Orphan Drug indication of Acute Myeloid Leukemia (AML) at the World Orphan Drug Congress USA 2024, Apr 23-25, 2024, at the Boston Convention and Exhibition Center, Boston, MA, United States.

Details for the presentation are below. The Company’s CEO, Dr. Michael Weickert, will also be available during the conference for one-on-one meetings.

The Phase 1 dose escalation safety and tolerability study was conducted in 29 heavily pre-treated (median of 4 prior lines of drug therapy) solid tumor and lymphoma patients. Treatment related adverse events were observed in a minority of patients, and were self-limited mild to moderate gastrointestinal side effects. A recommended Phase 2 dose (RP2D) for expansion studies was established. Zelenirstat prolonged progression free and overall survival in Phase 1 solid tumor patients receiving the RP2D, compared to those receiving lower doses. Prolonged Stable Disease of 6 months or longer was observed in 57% (4/7) of the solid tumor patients receiving RP2D, including a patient with metastatic colorectal cancer who continues on treatment for more than 14 months with ongoing reductions of approximately 50% in CEA (carcinoembryonic antigen) and tumor volumes.

Preclinical studies including in vitro, ex vivo, and in vivo animal studies indicate NMT inhibition disrupts prosurvival signal initiation and oxidative phosphorylation in AML cells and completely regresses AML xenografts. Additional in vivo evidence indicates AML stem cells are even more sensitive to zelenirstat than blasts. This strongly suggests that AML patients are excellent candidates for zelenirstat, a once per day oral investigational therapy. Orphan and Fast Track designations have been granted for AML by the US FDA, and a US IND has cleared the FDA for an AML Phase 1/2 clinical study.

"Advancing zelenirstat into a second hematologic indication, AML, is a top priority for the company”, said Dr. Michael Weickert, CEO of Pacylex. “AML is among the cancers most likely to respond to NMT inhibition. The high mortality of patients who have failed other available therapies makes it urgent for us to bring zelenirstat to people with AML."

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