ENMD-2076 trial meets endpoint for fibrolamellar carcinoma

A phase 2 trial designed to evaluate ENMD-2076 for the treatment of fibrolamellar carcinoma met its stage 1 endpoint of objective response for nonfutility based on prespecified interim analysis criteria, according to the agent’s manufacturer.

As a result, the trial will advance to its second stage.

The trial is designed to evaluate the safety and efficacy of single-agent ENMD-2076 (CASI Pharmaceuticals) in 29 patients with locally advanced or metastatic fibrolamellar carcinoma, a rare primary hepatic malignancy.

The trial featured a Simon’s optimal two-stage design to allow for an early determination of potential futility in 16 patients in stage 1. The remaining 13 patients will be recruited for stage 2 for an overall final analysis.

Stage 1 remains ongoing, with approximately one-third of patients still on treatment, Rong Chen, MD, Chief Medical Officer of CASI, said in a press release. A full interim analysis will be performed after all 16 patients included in the first stage reach study endpoint responses, Chen said.

“We are pleased to see our fibrolamellar carcinoma trial advance into stage 2, which — upon completion — will increase the amount of data of ENMD-2076 in fibrolamellar carcinoma patients,” Ken Ren, PhD, CEO of CASI, said in the press release. “Fibrolamellar carcinoma is a life-threatening disease that typically affects young adults and is currently without any systemic treatment. Based on results to date, we are encouraged by the potential of ENMD-2076 to address the unmet medical needs of these patients. We are thankful to our investigators for their dedication to the program and to our fibrolamellar carcinoma patients who make the trial meaningful and drive our purpose.”

The FDA previously granted orphan drug designation to ENMD-2076 for the treatment of patients with hepatocellular carcinoma, ovarian cancer, acute myeloid leukemia and multiple myeloma.

A phase 2 trial designed to evaluate ENMD-2076 for the treatment of fibrolamellar carcinoma met its stage 1 endpoint of objective response for nonfutility based on prespecified interim analysis criteria, according to the agent’s manufacturer.

As a result, the trial will advance to its second stage.

The trial is designed to evaluate the safety and efficacy of single-agent ENMD-2076 (CASI Pharmaceuticals) in 29 patients with locally advanced or metastatic fibrolamellar carcinoma, a rare primary hepatic malignancy.

The trial featured a Simon’s optimal two-stage design to allow for an early determination of potential futility in 16 patients in stage 1. The remaining 13 patients will be recruited for stage 2 for an overall final analysis.

Stage 1 remains ongoing, with approximately one-third of patients still on treatment, Rong Chen, MD, Chief Medical Officer of CASI, said in a press release. A full interim analysis will be performed after all 16 patients included in the first stage reach study endpoint responses, Chen said.

“We are pleased to see our fibrolamellar carcinoma trial advance into stage 2, which — upon completion — will increase the amount of data of ENMD-2076 in fibrolamellar carcinoma patients,” Ken Ren, PhD, CEO of CASI, said in the press release. “Fibrolamellar carcinoma is a life-threatening disease that typically affects young adults and is currently without any systemic treatment. Based on results to date, we are encouraged by the potential of ENMD-2076 to address the unmet medical needs of these patients. We are thankful to our investigators for their dedication to the program and to our fibrolamellar carcinoma patients who make the trial meaningful and drive our purpose.”

The FDA previously granted orphan drug designation to ENMD-2076 for the treatment of patients with hepatocellular carcinoma, ovarian cancer, acute myeloid leukemia and multiple myeloma.