RE:Clinical Trial Size in Orphan and Rare Diseases "We begin the Stage 2 expansion with substantial optimism for patients and the potential of pelareorep, especially considering the initial efficacy signal observed for pelareorep-based therapy in pancreatic cancer.
There is currently no established standard therapy for patients with anal carcinoma who have failed first-line treatment.
Continued positive results could potentially expand the opportunity for pelareorep beyond the lead indications of breast cancer and pancreatic cancer and open the door to a rapid regulatory pathway in this rare and significantly under-served patient population”
Thomas Heineman, M.D., Ph.D., Chief Medical Officer at Oncolytics guided that “Careful consideration of recently published clinical trial results indicates that a modest expansion of fewer than 20 patients will be sufficient to solidify the efficacy signal we have observed to date and lay the groundwork for a potential future registrational study in this population." Heineman explained that this small single arm would likely be sufficient for the FDA to grant an expedited drug approval in this rare disease.
ONCY's GOBLET-1 will be reporting on the anal cohort soon.