Small biotech whiffs badly in PhIII pancreatic cancer study, prompting halt of another late-stage trial
A few months after backflipping onto Nasdaq in a strategic merger, Rafael Holdings is learning firsthand the drawbacks of becoming a public company.
The New Jersey-based biotech’s Phase III study for its lead program did not meet its primary endpoint in metastatic pancreatic cancer, Rafael announced Thursday, showing almost no difference in overall survival between the treatment and control arms. Efficacy results proved so troubling that the independent monitoring board recommended to halt a separate Phase III study in relapsed or refractory acute myeloid leukemia due to futility.
“We are disappointed by the results of these two Phase 3 clinical trials with devimistat,” CEO Ameet Mallik said in a brief statement that also thanked patients and noted the company plans to further evaluate the data with a subsidiary.
Investors ran for the hills at the news, with Rafael Holdings shares $RFL down nearly 80% in early morning trading. After trading as high as $66.44 in the wake of June’s merger, Rafael Holdings is now approaching penny stock territory at about $6 per share Thursday — more than a 90% decrease in less than four months.
Rafael Holdings had been researching whether its lead candidate devimistat could be used as a first-line treatment for Stage IV pancreatic cancer in combination with chemotherapy. The company randomized 528 patients evenly between the active and chemo control arms, and patients had not received any prior therapies for metastatic disease.
Going for the overall survival endpoint, Rafael Holdings saw a median OS of 11.1 months in the treatment arm compared to 11.7 months taking the control regimen. The results clocked in at a dismal p-value of p=0.66, prompting the suspension of the other AML study where the company wanted to measure devimistat against different regimens of chemotherapies.
The Phase III flop will likely knock this program back to the early stages, if Rafael Holdings chooses to continue development. As recently as August, the company had a number of Phase II studies prepped for different cancers, including locally advanced pancreatic cancer, r/r myelodysplastic syndrome and r/r Burkitt lymphoma.
But devimistat’s future now remains unclear, given the massive whiff in metastatic pancreatic cancer and the premature stop of the AML study.
Pancreatic cancer is notoriously difficult to treat and has one of the lowest survival rates among solid tumor types. There have been some potential advances recently, however. Eli Lilly-partnered Merus boasted of new early data at this year’s ASCO conference for NRG1 fusion-positive cancers, revealing five of 12 patients with such pancreatic tumors saw confirmed partial responses in a single-arm Phase I/II study.