Lisata Therapeutics Announces US FDA Rare Pediatric Disease Designation Granted to LSTA1 for the Treatment of Osteosarcoma

Lisata Therapeutics, Inc. recently announced the US 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,” said Kristen K. Buck, MD, 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 US) 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 10 months to 6 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.

LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered (ie, 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 US and Europe as well as for glioblastoma multiforme (GBM) in the US. The product candidate has also received a Fast Track designation from the FDA for pancreatic cancer.

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.