SL-701 is designed to activate the immune system to kill malignant gliomas, which are aggressive malignancies of the brain that occur in both adults and children.
Antitumor activity of SL-701 is currently being evaluated in multicenter Phase II clinical trial in adult patients with second-line glioblastoma multiforme (GBM), a particularly aggressive type of glioma.
The company said that an earlier version of the therapy showed clinical activity, including durable complete responses (CRs) and partial responses (PRs) as well as prolonged disease stabilizations and an overall survival signal, in both adults and children with malignant glioma.
Stemline chief medical officer and head of Research and Development Dr Eric Rowinsky said: "We are pleased with the FDA’s decision to grant Orphan Drug designation to SL-701 as it provides Stemline with a number of benefits through development and commercialization of this novel therapy.
"GBM is a highly aggressive disease with few effective treatment options and remains a major unmet need. Instead of targeting a single component of the cancer, SL-701 is designed to activate and direct the immune system against multiple targets overexpressed on glioma cells."
Currently, the company is developing two clinical stage product candidates, SL-401 and SL-701, and a pipeline of preclinical candidates including SL-801 and SL-501.