The US Food and Drug Administration (FDA) has accepted the Stemline Therapeutics's Investigational New Drug (IND) application for SL-701, which enables the company to advance SL-701 into a phase 2 trial of adults with glioblastoma multiforme (GBM) in first recurrence.

SL-701 is a subcutaneously-administered cancer vaccine comprised of multiple synthetic peptides engineered for increased immunoreactivity against targets overexpressed on the cancer stem cells (CSCs) and tumour bulk of gliomas, the most common type of brain cancer.

It is based on a vaccine developed at the University of Pittsburgh that demonstrated single agent clinical efficacy, including complete responses (CRs) and partial responses (PRs), in Phase 1/2 trials conducted by the university in both adults and children with high-grade gliomas, including GBM.

The Stemline-sponsored trial is an 80-100 patient multi-centre study, designed to evaluate SL-701 in adults with GBM that has recurred following initial treatment with surgery, radiation, and chemotherapy. Overall response rate and survival are co-primary endpoints.

Eric Rowinsky, managing director, Stemline's chief medical officer, commented, "We are very excited to be opening our first of two expected INDs this year. If the data generated by our SL-701 study are in-line with the results of earlier studies, we intend to work with the regulatory authorities towards registration and commercialisation." Dr. Rowinsky continued, "In addition to SL-701, our other clinical programme, SL-401, a targeted therapy directed to the interleukin-3 receptor which is overexpressed on CSCs and tumour bulk of a wide range of haematologic malignancies, is also on track for IND filing and initiation of studies this year."

David Reardon, managing director, clinical director, centre of Neuro-oncology at the Dana-Farber Cancer Institute, and primary investigator of the upcoming study, commented, "GBM and other high-grade gliomas are particularly aggressive cancers that arise from glial tissue and account for approximately 14,000 new cases of brain cancer diagnosed in the United States each year. Treatment options are limited and expected survival is a little over one year. We are very excited about SL-701's unique mechanism of action and results to date, and look forward to advancing this novel agent."