FDA grants Fast Track Designation to RAG-01 in NMIBC
The FDA has granted a Fast Track Designation to RAG-01, a first-in-class small activating RNA (saRNA) therapeutic under review for the treatment of patients with BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC).
The FDA’s Fast Track designation is designed to expedite the review and development of novel treatments that will fill an unmet medical need. According to Ractigen Therapeutics, the developer of the therapy, RAG-01 is the first saRNA worldwide to receive a Fast Track Designation.
RAG-01 is an saRNA therapy that is delivered via intravesical instillation and is designed to target and activate p21, a tumor suppressor gene.
An Investigational New Drug (IND) application for RAG-01 in BCG-unresponsive NMIBC was accepted by the FDA earlier this year, in April 2024. The approval of the IND application initiated the launch of clinical trials of the therapy in the United States.
Ractigen Therapeutics also revealed the launch of a phase 1 trial of RAG-01 in Australia earlier this year by announcing the dosing of the first patient. The trial was initiated in December 2023, and has successfully enrolled and dosed 3 patients in the study to date.
Overall, the multi-center, open-label, first-in-human, phase 1 trial (NCT06351904) of RAG-01 in Australia is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of the treatment in patients with NMIBC who have failed prior BCG therapy. All patients will receive a starting dose of RAG-01 at 30 mg, but they will be distributed into 4 dose cohorts: 30 mg, 100 mg, 300 mg, and 600 mg.
There are 2 primary outcome measures for the study. The first is safety and tolerability of RAG-01 in patients with NMIBC during the period from screening to 6 months following the initial instillation of RAG-01. Safety and tolerability will be measured through adverse events (AEs), serious AEs, and treatment emergent AEs. The other primary outcome measure is the maximum tolerated dose and/or recommended phase 2 dose of RAG-01. These will be assessed through the evaluation of any dose-limiting toxicities with 21 days of the first instillation of RAG-01.
The estimated enrollment for the trial is 15 patients, and the estimated primary completion date is June 30, 2025. The study is being conducted through a collaboration between Ractigen Therapeutics and GenesisCare, a leading provider of cancer care services in Australia.
May 24, 2024
