CytRx begins aldoxorubicin Phase 2b clinical trial in patients with extensive-stage SCLC

CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced the initiation of a global Phase 2b clinical trial evaluating aldoxorubicin compared to topotecan in subjects with extensive-stage small cell lung cancer (SCLC) who have relapsed or were refractory to prior chemotherapy. Aldoxorubicin is CytRx's modified version of the widely-used chemotherapeutic agent, doxorubicin. CytRx has received Orphan Drug Designation for the treatment of SCLC from the U.S. Food and Drug Administration (FDA).

"The significant toxicities associated with topotecan, the only approved chemotherapy for the treatment of second-line SCLC, prevent many patients from even finishing one course of treatment, translating to poor response and overall survival rates," said Dr. Daniel Levitt, Executive Vice President and Chief Medical Officer of CytRx. "Based on its mechanism of action, data observed in a broad range of tumor types, and compelling Phase 1 pharmacokinetic data in metastatic, treatment relapsed or refractory solid tumors, aldoxorubicin may offer patients an improved tolerability profile together with significant improvements in clinically meaningful efficacy measures in this indication. Our previous data in relapsed or refractory solid tumor patients indicates that the dose of aldoxorubicin being administered to these patients is well tolerated and without any treatment-limiting side effects. Up to 21 cycles (4.8 grams/m²) have been given to one patient with small cell lung cancer with minimal side effects and good anti-tumor activity."

The open-label Phase 2b clinical trial is expected to enroll approximately 132 patients (1:1 randomization) with extensive-stage SCLC who have relapsed or were refractory to prior chemotherapy. Patients will receive either aldoxorubicin or topotecan. The primary endpoint is progression-free survival (PFS) and the secondary endpoints are overall survival (OS), overall response rates (partial and complete) and the safety of aldoxorubicin compared to topotecan in this population. Enrollment is expected to be completed in 2015 and PFS data are anticipated by mid-2016. The study is expected to involve approximately 40 clinical trial sites in the U.S., Spain, Italy and Hungary.

"Initiation of this global clinical trial builds on our substantial experience evaluating aldoxorubicin in soft tissue sarcomas (STS), glioblastoma multiforme and Karposi's sarcoma, and reflects our strategy to leverage the broad potential of aldoxorubicin into a variety of solid tumor and hematological indications, " said CytRx CEO Steven A. Kriegsman. "We are particularly interested in the potential of aldoxorubin in small cell lung cancer, as this tumor type typically is more sensitive to chemotherapy than STS."

In 2013, CytRx presented results from its Phase 1b clinical trial evaluating the pharmacokinetics and safety of aldoxorubicin in patients with metastatic solid tumors who have either relapsed or not responded to treatment with standard therapies, demonstrating a prolonged duration of treatment with aldoxorubicin. In that trial, treatment with aldoxorubicin extended to 21 cycles (a cycle is 21 days) in a patient with small cell lung cancer as well as to over 10 cycles in several other patients.

Aldoxorubicin is also currently being studied in a pivotal global Phase 3 clinical trial evaluating the efficacy and safety of aldoxorubicin as a second-line treatment for patients with STS under a Special Protocol Assessment with the FDA. CytRx is also evaluating aldoxorubicin in two Phase 2 clinical trials, one in patients with late-stage glioblastoma (GBM) and the other in HIV-related Kaposi's sarcoma, and a Phase 1b trial in combination with ifosfamide in patients with STS.