The US Food and Drug Administration (FDA) has granted Lisata Therapeutics a rare pediatric disease designation (RPDD) for its osteosarcoma treatment, LSTA1 (certepetide).
Lisata’s lead candidate is a cyclic peptide that is designed to activate the CendR uptake pathway, leading to an improved uptake of co-administered drugs. The intravenous therapy also works as a neuropilin-1 (NRP1) antagonist that prevents tumour cell migration and adhesion.
Currently, the US-based biotech is investigating LSTA1 across six Phase I and Phase II studies. One of these is the Phase II BOLSTER study (NCT05712356), in which Lisata is investigating the therapy for the treatment of solid tumours. The multi-centre trial is enrolling participants and is expected to complete accrual by the end of 2024, as per a 29 February company update. In addition to studying LSTA1 in a basket solid tumour trial, Lisata is also exploring the therapy’s potential more specifically for colon and appendiceal cancers.
In June 2022, the FDA also rewarded the drug a fast-track designation for pancreatic cancer, which was later followed by a European Medicines Agency (EMA) orphan drug designation in the same indication in September 2023. The FDA also granted LSTA1 orphan status a month later. Due to the most recent designation for osteosarcoma, if the FDA later approves a new drug application for LSTA1 in the indication, the company will receive a priority review voucher.
In a 21 March press release announcing the rare pediatric drug designation, Dr Kristen Buck, Lisata’s Chief Medical Officer, said “LSTA1 has already demonstrated promise in preclinical and clinical settings for the treatment of several advanced solid tumours, 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.”
The American Cancer Society reports that each year 100 new cases of osteosarcoma are diagnosed in the US, with approximately 50% of cases accounted for by children and teenagers. The organisation also found that osteosarcoma cases make up approximately 2% of childhood cancer cases.
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