FDA Grants Orphan Drug Status to RAD 301 for Imaging of Pancreatic Ductal Adenocarcinoma

The FDA has granted an orphan drug designation to RAD 301 (Ga68-Trivehexin) for the imaging of patients with pancreatic ductal adenocarcinoma (PDAC).¹

RAD 301 is a proprietary peptide-based molecule targeting αvβ6-integrin, which is a cellular marker for tumor invasion and metastatic growth. Expression of αvβ6 is associated with worse survival in several carcinomas, and the αvβ6-integrin receptor is found in high density on most pancreatic carcinoma cells.

“Orphan drug designation for RAD 301 comes on top of FDA investigational new drug approval for a phase 1 clinical trial [NCT05799274] in pancreatic cancer, which is planned to start in the next few weeks in the United States,” Riccardo Canevari, chief executive officer and managing director of Radiopharm Theranostics, stated in a news release. “This important designation further reinforces the excitement of investigators conducting the study. The FDA’s decision highlights the significant demand for effective imaging agents for improved and earlier diagnosis of pancreatic cancer, which has one of the highest levels of unmet needs among all cancer types.”

The phase 1 trial will evaluate the safety of RAD 301 in both health volunteers and patients with pancreatic cancer. Investigators will enroll volunteers who are subjectively healthy and likely to tolerate the imaging procedures.² Patients with pancreatic cancer will need to have a history of histologically or cytologically confirmed PDAC, and they will be allowed to participate if they are deemed likely to tolerate the procedures and survive throughout the follow-up period. Patients should have a life expectancy of at least 12 weeks per the judgement of the treating physician.

Within 30 days of administration of RAD 301, individuals will need to have a white blood cell count of at least 3000/µL, a neutrophil count of at least 1500/µL, a platelet count of at least 75,000/µL, hemoglobin of at least 9.0 g/dL, and creatinine of no more than 1.5 mg/dL. Aspartate transaminase/alanine transaminase (AST/ALT) must be no more than 2.5 x the upper limit of normal (ULN) for patients with no liver metastases, and AST/ALT must be no more than 5 x ULN for patients with liver metastases. Bilirubin must be no more than 1.5 mg/dL, except for individuals with Gilbert’s disease.

The study will exclude patients who are members of a vulnerable population or have a history suggestive of atopy. Patients will not be permitted to take any experimental study drugs 4 weeks prior to positron emission tomography (PET) scan.

Enrolled individuals will receive RAD 301 with a whole-body PET scan. The primary objectives of the study are to measure radiation absorbed doses in internal organs, observe changes in stable vital signs and electrocardiogram, and the determine the number of individuals with abnormal laboratory test results.

References

1. Radiopharm Theranostics receives FDA orphan drug designation for trivehexin (RAD 301) in pancreatic cancer. News release. Radiopharm Theranostics. May 9, 2023. Accessed May 10, 2023. https://assets.website-files.com/
2. Safety of RAD301 in healthy human volunteers and patients with pancreatic cancer. ClinicalTrials.gov. Updated April 5, 2023. Accessed May 10, 2023. https://clinicaltrials.gov/ct2/show/NCT05799274