On December 11, 2023 Shuttle Pharmaceuticals Holdings, Inc. (Nasdaq: SHPH) ("Shuttle Pharma") reported submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to support the next phase of development of Ropidoxuridine (Press release, Shuttle Pharmaceuticals, DEC 11, 2023, View Source [SID1234638462]). Ropidoxuridine is Shuttle Pharma’s lead radiation sensitizer candidate for use in combination with radiation therapy (RT) to treat brain tumors (glioblastoma), a deadly malignancy of the brain with no known cure.

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RT is a proven modality for treating cancers. However, there is a significant need in the market to make radiation more effective. By developing radiation sensitizers, Shuttle Pharma aims to increase cancer cure rates, prolong patient survival and improve quality of life when used as a primary treatment, or in combination with surgery, chemotherapy and immunotherapy.

"Today’s announcement is an important milestone for Shuttle Pharma and the thousands of patients with brain tumors who currently lack effective therapies," stated Shuttle Pharma’s Chairman and CEO, Anatoly Dritschilo, M.D. "The IND submission is the culmination of many years of clinical development by the Shuttle Pharma team, as well as support from the broader cancer community, including the National Institutes of Health’s National Cancer Institute and Small Business Innovation Research program, who have provided guidance and grant funding to bring this potentially important new radiation sensitizing therapy to market."

An IND submission is a request submitted to the regulatory authorities seeking permission to test a new drug or therapeutic substance in humans. The submission includes detailed information about the drug, its composition, pharmacology, toxicology data from preclinical studies, proposed clinical trial protocols, and information on manufacturing and quality control. With the IND application submission now complete, the FDA is expected to provide Shuttle Pharma with its decision to proceed with the Phase II trial within approximately 30 days.

The submission of the IND follows recent receipt of written responses to questions submitted for a Type B pre-Investigational New Drug Application (PIND) meeting with the FDA in September 2023. During the PIND meeting, the FDA provided positive feedback and guidance on the Company’s Chemistry, Manufacturing, and Controls (CMC) and clinical protocol design for Ropidoxuridine, thus providing the pathway to this IND submission.

The planned Phase II trial will investigate whether a new treatment, Ropidoxuridine, taken during radiation treatment, will be a safe and possibly effective for treatment of patients with newly diagnosed IDH-wildtype glioblastoma with unmethylated MGMT promoter.

Dr. Dritschilo added, "Our mission is to improve the lives of cancer patients by developing therapies that are designed to maximize the effectiveness of RT while limiting the side effects of radiation in cancer treatment. This IND submission is an important next step in making this mission a reality."

An estimated 800,000 patients in the US are treated with radiation therapy for their cancers yearly. According to the American Cancer Society and the American Society of Radiation Oncologists, about 50% are treated for curative purposes and the balance for therapeutic care. The market opportunity for radiation sensitizers lies with the 400,000 patients treated for curative purposes, with this number expected to grow by more than 22% over the next five years.

Shuttle Pharma has received Orphan Drug Designation from the FDA, providing potential marketing exclusivity upon first FDA approval for the disease.