Achieves critical first step toward priority review voucher

BASKING RIDGE, N.J., March  21, 2024  (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the "FDA") has granted Rare Pediatric Disease Designation ("RPDD") to LSTA1, the Company's lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults.

"LSTA1 has already demonstrated promise in preclinical and clinical settings for the treatment of several advanced solid tumors, and we believe that potential extends to a beneficial impact in osteosarcoma, a rare cancer that usually develops in the osteoblast cells that form bone," stated Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata. "Receiving RPDD from the FDA is an important milestone as we plan future clinical expansion for LSTA1, and, more importantly, it underscores the high unmet medical need of this patient population."

The FDA defines rare pediatric diseases as rare diseases (those with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals under 18 years of age. A substantial benefit of a RPDD is receipt of a priority review voucher, often referred to as a "golden ticket," once the FDA approves the new drug application ("NDA") for the product and indication having received the RPDD. Vouchers are especially valuable as they can be used to compel a priority review of an additional NDA or biologic license application for another product or indication, reducing the standard review time of approximately ten months to six months. The voucher may be used by the sponsor or sold to another sponsor for their use. Priority review vouchers have sold for as much as $350 million USD historically and, more recently, have sold for $75 to $100 million USD.

About LSTA1

LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered (i.e., covalently bound) 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, with the objective of 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 SoC 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. LSTA1 has been granted orphan drug designation for pancreatic cancer in the U.S. and Europe as well as for glioblastoma multiforme ("GBM") in the U.S. The product candidate has also received a Fast Track designation from the FDA for pancreatic cancer.