Lisata Therapeutics LSTA1 Receives Orphan Drug Designation from USFDA to Treat Malignant Glioma

Lisata Therapeutics, Inc., a clinical-stage pharmaceutical company, announced that the FDA designated LSTA1, the company's lead product candidate, as an orphan drug for the treatment of malignant glioma. "Malignant glioma is one of the most aggressive and deadly malignancies," said Kristen K. Buck, M.D., Lisata's executive vice president of R&D and chief medical officer.

"This Orphan Drug Designation acknowledges the high unmet medical need of this patient population, as well as the potential for LSTA1 to benefit patients in this setting." Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Orphan Drug Designation provides certain financial incentives to support clinical development as well as a seven-year period of US market exclusivity if the product receives FDA approval for such indication. LSTA1 is currently being studied in a number of ongoing or planned phase 1b/2a and 2b clinical trials in a variety of solid tumour types in combination with a variety of anti-cancer regimens all over the world.

In the coming months, the company intends to begin a clinical trial evaluating LSTA1 in previously untreated glioblastoma multiforme (GBM) in collaboration with researchers from Estonia's University of Tartu. The study is designed as a phase 2a double-blind, placebo-controlled, randomised proof-of-concept study evaluating LSTA1 when added to SoC temozolomide versus temozolomide alone and matching LSTA1 placebo in subjects with newly diagnosed GBM. It will be carried out at several locations in Estonia and Latvia, with a randomization of 2:1 LSTA1 + SoC versus placebo + SoC. The first patient is expected to be treated in the fourth quarter of 2023, according to the company. Lisata Therapeutics is a clinical-stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapies for advanced solid tumours and other major diseases.