Shuttle Pharmaceuticals Holdings, Inc. has announced the successful dosing of the first three patients in its Phase 2 clinical trial evaluating Ropidoxuridine for the treatment of glioblastoma. This trial marks a significant step forward in the development of new therapies for this aggressive brain tumor, which currently has no known cure.
The Phase 2 trial (NCT06359379) is designed to assess the efficacy and safety of Ropidoxuridine in patients with IDH wild-type, methylation negative glioblastoma, a particularly aggressive subtype. These patients currently have limited treatment options, with radiation therapy being the standard of care. More than half of these patients survive less than 12 months after diagnosis.
The trial will initially randomize 40 patients into two dose cohorts: 20 patients receiving 1,200 mg/day and 20 patients receiving 960 mg/day of Ropidoxuridine. The goal of this initial phase is to determine the optimal dose. Following dose optimization, an additional 14 patients will be enrolled at the selected dose to achieve statistical significance in survival compared to historical controls. The company anticipates the trial will take 18 to 24 months to complete.
Rationale for Ropidoxuridine
Ropidoxuridine (IPdR) is Shuttle Pharma’s lead candidate, acting as a radiation sensitizer. It is designed to enhance the effectiveness of radiation therapy (RT) in treating brain tumors. The mechanism of action involves increasing the sensitivity of cancer cells to radiation, potentially leading to improved tumor control and patient outcomes.
Management Commentary
"The initiation of patient dosing in our Phase 2 trial of Ropidoxuridine for the treatment of patients with glioblastoma is a significant milestone for both Shuttle Pharma and the thousands of patients with brain tumors who currently lack effective therapies," said Anatoly Dritschilo, M.D., Chairman and CEO of Shuttle Pharma. "The results of this trial will be important as Shuttle Pharma looks to leverage radiation sensitizers to increase cancer cure rates, prolong patient survival and improve quality of life for patients suffering from glioblastoma."
Market Opportunity
Approximately 800,000 patients in the US receive radiation therapy for cancer treatment annually. Of these, about 50% are treated for curative purposes. Shuttle Pharma is targeting the 400,000 patients treated for curative purposes, with the expectation that this number will grow by over 22% in the next five years. The company believes that radiation sensitizers like Ropidoxuridine have the potential to significantly improve outcomes for these patients.
Shuttle Pharma received Orphan Drug Designation from the FDA for Ropidoxuridine, which could provide marketing exclusivity upon FDA approval.
