Lisata Therapeutics Announces US FDA Orphan Drug Designation Granted for the Treatment of Malignant Glioma

Lisata Therapeutics, Inc. recently announced the US FDA has granted Orphan Drug Designation to LSTA1, the company’s lead product candidate, for the treatment of malignant glioma.

“Malignant glioma is one of the most aggressive and deadly malignancies,” said Kristen K. Buck, MD, Executive Vice President of R&D and Chief Medical Officer of Lisata. “This Orphan Drug Designation acknowledges the high unmet medical need of this patient population as well as the potential of 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 US. Orphan Drug Designation provides certain financial incentives to support clinical development as well as a 7-year period of US market exclusivity if the product receives FDA approval for such indication.

Currently, LSTA1 is the subject of multiple ongoing or planned Phase 1b/2a and 2b clinical studies being conducted globally in a variety of solid tumor types in combination with a variety of anti-cancer regimens. In the coming months, the company, in collaboration with investigators from the University of Tartu in Estonia, plans to initiate a clinical study evaluating LSTA1 in previously untreated glioblastoma multiforme (GBM). The study will be the first step in the clinical exploration of the impact of LSTA1 added to standard-of-care (SoC) for the treatment of GBM and is designed as a Phase 2a double-blind, placebo-controlled, randomized, 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 conducted at several sites in Estonia and Latvia and is targeted to enroll 30 patients with a randomization of 2:1 LSTA1 + SoC versus placebo + SoC. The company expects that the first patient will be treated in the fourth quarter of 2023.

LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. LSTA1 also has the potential to modify the tumor microenvironment, making tumors more susceptible to immunotherapies. Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies and RNA-based therapeutics. Additionally, LSTA1 has demonstrated favorable safety, tolerability and activity in clinical trials to enhance delivery of standard-of-care chemotherapy for pancreatic cancer. Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively.

Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s lead product candidate, LSTA1, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to target and penetrate solid tumors more effectively. Based on Lisata’s CendR Platform Technology, Lisata has already established noteworthy commercial and R&D partnerships. The Company expects to announce numerous clinical study and business milestones over the next 2 years and has projected that its current business and development plan is funded with available capital through these milestones and into early 2026. For more information, visit www.lisata.com.