RAG-01 Gains FDA Fast Track Designation in NMIBC


RAG-01 has received an FDA fast track designation and is being investigated for the treatment of non-muscle invasive bladder cancer.

3D illustration of bladder cancer: © matthieu - stock.adobe.com

3D illustration of bladder cancer: © matthieu - stock.adobe.com

  • A fast track designation has been granted to RAG-01 by the FDA, making it the first small activating RNA drug worldwide to receive this designation.
  • This designation is designed to expedite the development and review process for novel therapies that address unmet medical needs.
  • A phase 1 clinical trial (NCT06351904) is ongoing in Australia and has successfully enrolled and dosed 3 patients.

The FDA has granted a fast track designation to RAG-01 for non-muscle invasive bladder cancer (NMIBC), marking it the first small activating RNA (saRNA) drug worldwide to receive this designation.1

"We are thrilled to receive fast track designation for RAG-01, marking a significant milestone not only for our program but also for the saRNA field as a whole. This designation underscores the urgency and importance of advancing innovative therapies like RAG-01 to address critical medical needs,” said Long-Cheng Li, MD, founder and chief executive officer of Ractigen Therapeutics, in a press release.

RAG-01 is a pioneering saRNA candidate delivered through intravesical instillation. The agent works to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. Using Ractigen's proprietary LiCO™ delivery technology, RAG-01 has demonstrated significant tumor suppression in preclinical bladder cancer models.

A phase 1 trialis currently evaluating RAG-01 for the treatment of patients with NMIBC. The trial is ongoing in Australia and started in December 2023. To date, 3 patients have been successfully enrolled and dosed in the study.

“We remain dedicated to accelerating the development of innovative saRNA therapies to address a wide range of diseases, including cancer, genetic disorders, and chronic conditions. Through strategic collaborations and pioneering research efforts, the company aims to deliver transformative treatments that improve patient outcomes and quality of life," added Li in the press release.

In the phase 1 study, patients are being treated with RAG-01 at a starting dose of 30 mg. There are 4 dose cohorts included in the study giving patients different doses of RAG-01, including 30 mg, 100 mg, 300 mg, and 600 mg.2

Enrollment is open to patients between 18 and 75 years old who have pathologically confirmed high-grade NMIBC defined as grade 2 or grade 3 disease. Patients must have a life expectancy of ≥ 6 months, an ECOG performance status of ≤2, and sufficient organ function. Further, patients must be able to tolerate catheterization, and females of childbearing potential, as well as their partners, must agree to adopt effective contraception from the time informed consent is signed up until within 6 months after the last instillation.

The primary end points are safety and tolerability of RAG-01 in patients with NMIBC and to determine the maximum tolerated dose and/or recommended phase 2 dose of the agent.

The FDA also recently approved the investigational new drug application for RAG-01.1 With the fast track designation, this further validates the therapeutic potential of this innovative saRNA therapy.

Following these regulatory decisions, it is possible for clinical trials evaluating RAG-01 in NMIBC treatment to expand to the United States and highlights the potential RAG-01 has in addressing unmet medical needs in this patient population.

1. Ractigen Therapeutics secures FDA fast track designation for RAG-01, a first-in-class saRNA therapy. News release. Ractigen Therapeutics. May 21, 2024. Accessed May 21, 2024. https://tinyurl.com/2xxbs29r
2. A study of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC) who have failed bacillus calmette guérin (BCG) therapy. ClinicalTrials.gov. Updated April 16, 2024. Accessed May 21, 2024. https://classic.clinicaltrials.gov/ct2/show/NCT06351904
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