Shuttle Pharmaceuticals Holdings, Inc. (NASDAQ: SHPH) has announced a significant milestone in its Phase 2 clinical trial of Ropidoxuridine (IPdR) for the treatment of glioblastoma, achieving 25% enrollment in the initial randomized portion. The trial is designed to evaluate the efficacy of Ropidoxuridine, a radiation sensitizer, in combination with radiation therapy for patients with IDH wild-type, methylation negative glioblastoma, an aggressive form of brain tumor with limited treatment options.
The Phase 2 trial (NCT06359379) is randomizing 40 patients into two dose levels: 1,200 mg/day and 960 mg/day, with 20 patients in each arm. The primary objective is to determine the optimal dose of Ropidoxuridine for glioblastoma patients. Following the dose optimization, an additional 14 patients will be enrolled at the selected dose to establish statistical significance in endpoint survival compared to historical controls. The study is being conducted at several leading cancer centers, including Georgetown University Medical Center, Allegheny Health Network Cancer Institute, UNC Medical Center, UVA Cancer Center, John Theurer Cancer Center at Hackensack University Medical Center, and Miami Cancer Institute under Baptist Health South Florida.
Ropidoxuridine: A Novel Radiation Sensitizer
Ropidoxuridine (IPdR) is Shuttle Pharma's lead candidate, designed to enhance the effectiveness of radiation therapy in treating glioblastoma. Glioblastoma remains a deadly malignancy with a poor prognosis, and Ropidoxuridine has received Orphan Drug Designation from the FDA, potentially granting marketing exclusivity upon approval for this indication.
The Need for Improved Glioblastoma Treatments
Patients with IDH wild-type, methylation negative glioblastoma face a particularly grim prognosis, with less than half surviving beyond 12 months post-diagnosis with the current standard of care, which is primarily radiation therapy. Anatoly Dritschilo, M.D., CEO and Chairman of Shuttle Pharmaceuticals, expressed optimism about the trial's progress, stating, "Enrollment into the trial is ahead of our expectations... as we look to develop radiation sensitizers to increase cancer cure rates, prolong patient survival and improve quality of life for patients suffering from glioblastoma."
Radiation Therapy and Market Opportunity
Radiation therapy is a common treatment modality for cancer, with an estimated 800,000 patients in the US receiving it annually. Approximately 50% of these patients are treated with curative intent. Shuttle Pharma aims to tap into the market opportunity for radiation sensitizers, targeting the 400,000 patients who undergo radiation therapy for curative purposes. This number is projected to grow by over 22% in the next five years, underscoring the need for effective therapies like Ropidoxuridine.
