Benzinga | Sep 1, 2020 07:08AM EDT

Plus Therapeutics, Inc. Receives FDA Orphan Drug Designation For Novel Glioblastoma Treatment

AUSTIN, Texas, Sept. 01, 2020 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), today announced that the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for its lead investigational drug, Rhenium NanoLiposomes (RNL(tm)) for the treatment of patients with recurrent glioblastoma.

RNL is being evaluated in the NIH/NCI-supported, multi-center ReSPECT(tm) Phase 1 dose-finding clinical trial (NCT01906385). Last week, the trials' Data Safety and Monitoring Board approved the Company to proceed to Cohort 6 which includes increasing both the drug volume and radiation dose to 8.8 milliliters (mL) and 22.3 millicuries (mCi), respectively.

"Receiving FDA orphan drug designation and approval to advance the trial into Cohort 6 are key milestones in our development of this treatment for recurrent glioblastoma," said Dr. Marc Hedrick, President and Chief Executive Officer of Plus Therapeutics. "We believe RNL has the potential to prolong survival for patients with malignant brain tumors and that of other difficult to treat radiosensitive tumors."

RNL is designed to safely, effectively, and conveniently deliver a very high dose of radiation - up to 25 times greater concentration than currently used external beam radiation therapy - directly into the brain tumor for maximum effect.

The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation provides to the Company certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.

Glioblastoma (Grade IV astrocytoma) is the most common and most aggressive of the primary malignant brain tumors in adults. According to the most recent CBTRUS Statistical Report, annually there are approximately 11,833 cases of glioblastoma diagnosed within the U.S., with historical 1-year and 5-year median survival rates of 40.8% and 6.8%, respectively.