Pacylex Pharma's phase one trial: Zelenirstat safety in heavily pre-treated patients

By Liza Laws

- Last updated on GMT

© Getty Images
© Getty Images

Related tags Gastrointestinal lymphoma Clinical trials Leukemia Research

Pacylex Pharmaceuticals Inc. has announced the presentation of phase 1 safety and efficacy findings for small molecule drug zelenirstat for cancer treatment.

The presentation will be delivered by CEO Michael Weickert at the World Orphan Drug Congress USA 2024 which starts tomorrow (April 23).

“The phase 1 results mark a significant milestone in our journey towards addressing critical unmet needs in cancer treatment,” says Dr. Michael Weickert, CEO of Pacylex Pharmaceuticals.

“The data underscores the potential of zelenirstat as a promising therapeutic option for patients battling hematologic cancers and solid tumors.”

Solid tumors and lymphomas

The phase 1 study, focused on dose escalation safety and tolerability, involved 29 heavily pre-treated patients with solid tumors and lymphomas, each having a median history of four prior lines of drug therapy.

According to the findings, treatment-related adverse events were observed in a minority of patients, primarily consisting of mild to moderate gastrointestinal side effects. The establishment of a recommended phase 2 dose (RP2D) for expansion studies was a significant outcome of the trial.

“Zelenirstat demonstrated promising efficacy outcomes, particularly in solid tumor patients receiving the RP2D,” says Weickert. “This includes prolonged progression-free and overall survival rates, along with instances of prolonged Stable Disease, further highlighting its potential as a therapeutic breakthrough.”

Acute Myeloid Leukemia (AML) cells

Furthermore, preclinical studies, encompassing in vitro, ex vivo, and in vivo animal trials, have shown that NMT inhibition disrupts prosurvival signal initiation and oxidative phosphorylation in Acute Myeloid Leukemia (AML) cells, leading to complete regression of AML xenografts. These findings suggest a potential application of zelenirstat in AML treatment.

“AML patients, especially those who have exhausted other treatment options, represent a population with significant unmet needs,” Weickert adds. “The promising preclinical evidence, coupled with the Orphan and Fast Track designations granted by the US FDA, positions zelenirstat as a compelling candidate for further clinical investigation in AML.”

Investigational New Drug clearance

In light of these findings, Pacylex Pharmaceuticals is advancing zelenirstat into a Phase 1/2 clinical study for AML patients, following the clearance of a US Investigational New Drug (IND) application by the US Food and Drink Administration (FDA).

“Bringing zelenirstat to AML patients is a top priority for Pacylex Pharmaceuticals,” concludes Weickert. “The urgency stems from the high mortality rates associated with AML, particularly among patients who have exhausted other available therapies. We aim to provide a much-needed therapeutic option to this patient population.”

The company says the presentation of phase 1 results for zelenirstat at the World Orphan Drug Congress USA 2024 represents a significant step forward in Pacylex Pharmaceuticals' mission to innovate cancer treatment and improve patient outcomes.

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