RZ-001 Gains FDA Fast Track Designation in Glioblastoma


A phase 1/2 trial is evaluating the safety, efficacy, and tolerability of RZ-001 in patients with glioblastoma.

  • RZ-001 is the RNA replacement enzyme-based cancer gene therapy being evaluated for the treatment of glioblastoma (GBM).

  • With enhanced anti-cancer efficacy and higher survival rates, RZ-001 has already demonstrated promising responses in preclinical trials.

  • With fast track designation (FTD), RZ-001 may be eligible for accelerated approval and priority review, if relevant criteria are met.

The FDA has granted a FTD to RZ-001, for the treatment of patients with GBM, according to Rznomics Inc.1

RZ-001 is an RNA replacement enzyme-based cancer gene therapy which targets and cleaves hTERT mRNA. The agent also replaces the mRNA with the therapeutic gene RNA and induces effective immune cell infiltration into treated tumors. By reducing VEGF expression in the tumors, RZ-001 produces anti-cancer efficacy and transformation of the tumor microenvironment to be more responsive to immunotherapy.2

"We are proud to receive fast track designation from the FDA," said Seong-Wook Lee, PhD, chief executive officer of Rznomics, in a press release.1 "This designation signifies an important milestone in developing a safe and effective treatment for patients with GBM who are in need of new therapeutic options."

Human brain © Sergey Nivens - stock.adobe.com.jpg

Human brain © Sergey Nivens - stock.adobe.com.jpg

Previously, the FDA granted approval to a phase 1/2 investigational new drug application for RZ-001 in GBM. The trial (NCT06102525), which has an estimated study completion date of May 2029, plans to assess the safety, tolerability, and efficacy of RZ-001 in this patient population and will consist of 2 parts: a dose-escalation part (part 1) and a dose-expansion part (part 2).3

Patients aged 18 years and older with histologically-confirmed grade 4 astrocytoma, GBM who have human telomerase reverse transcriptase-positive expression confirmed during the screening period, an ECOG score of ≤ 2, a Karnofsky performance score of ≥ 60, and a life expectancy ≥ 3 months are eligible for enrollment in the study. If patients have a diagnosis of other malignant tumors within 5 years prior to RZ-001 administration, have extracranial metastases of the tumor cells, and are currently HIV positive or have a history of being HIV positive will be excluded from the trial.

The primary end points being evaluated are dose-limiting toxicities, maximum tolerated dose, treatment-related adverse events, number of participants with significant laboratory abnormalities, and overall survival (OS). Secondary end points include change in concentration of serum VEGF and anti-adenovirus antibody, overall response rate, duration of response, progression-free survival, OS, and neurologic function assessment using the Neurologic Assessment in Neuro-Oncology scale ranging from 0 to 3 in each assessment domain.

Additional end points being evaluated include concentration of adenovirus DNA in plasma at specified time points, change in concentration of serum anti-adenovirus antibody, change in concentration of serum cytokines, concentration of biomarker in peripheral blood, and concentration of biomarker in fresh tumor biopsy tissue.

Preclinically, RZ-001 has demonstrated therapeutic activity and improved anti-cancer efficacy in a dose-dependent manner in combination with atezolizumab (Tecentriq).2 The clinical study will evaluate the safety and efficacy in patients with hepatocellular carcinoma (HCC), and it is anticipated that the combination of RZ-001 with atezolizumab may enhance response rate and anti-cancer efficacy that could benefit patients with HCC.

  1. Rznomics Inc. secures fast track designation from the U.S. FDA for RZ-001. Rznomics Inc. News release. November 10, 2023. Accessed November 10, 2023. https://tinyurl.com/3vh5v99x
  2. RZNOMICS Inc. announces clinical trial collaboration and supply agreement to evaluate RZ-001 in combination with anti-PDL1 in liver cancer patients. News release. Rznomics Inc. November 10, 2023. Accessed November 10, 2023. https://tinyurl.com/mrybcehy
  3. A study to evaluate the safety, tolerability and efficacy of RZ-001 with valganciclovir (VGCV) in subjects with glioblastoma. ClinicalTrials.gov. Updated October 26, 2023. Accessed November 10, 2023. https://classic.clinicaltrials.gov/ct2/show/NCT06102525
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