Rafael craters as devimistat fails in Phase III trials

Shares in Rafael Holdings (NYSE: RFL) closed down 73% at $8.09 yesterday, after its majority-owned affiliate Rafael Pharmaceuticals revealed that a Phase III study of its leading candidate devimistat in first-line patients with pancreatic cancer failed to meet its primary endpoint of overall survival (OS).

Moreover, a late-stage trial of the experimental anti-mitochondrial drug in patients with relapsed or refractory acute myeloid leukemia (AML) has also been halted due to a lack of efficacy.

In this multi-national Phase III randomized clinical trial, 528 patients with metastatic adenocarcinoma of the pancreas who had no prior therapy were randomized to receive either devimistat in combination with modified FOLFIRINOX (mFFX) or FOLFIRINOX, a current standard of care chemotherapy regimen. Devimistat given with mFFX did not significantly improve overall survival (HR=0.95, p=0.66). The median overall survival in the devimistat and mFFX arm was 11.1 months, compared to 11.7 months in the FOLFIRINOX arm.