On March 13, 2024 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to LYT-200 for the treatment of AML (Press release, PureTech Health, MAR 13, 2024, View Source [SID1234641116]).

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"The current long-term survival rates of patients with relapsed or refractory AML are very poor, and there remains a tremendous unmet need for more effective therapies," said Amir Fathi, MD, Director of the Leukemia Program at Massachusetts General Hospital and lead investigator of the trial.

LYT-200 is a fully human IgG4 monoclonal antibody (mAb) targeting galectin-9, a potent oncogenic driver in leukemia cells and an immunosuppressive protein. LYT-200 has demonstrated direct cytotoxic, anti-leukemic effects through multiple mechanisms and is being developed as a potential novel treatment for hematological malignancies, such as relapsed/refractory AML and high-risk MDS, as well as locally advanced/metastatic solid tumors that have poor survival rates, including head and neck cancers. LYT-200 is currently being evaluated in an ongoing Phase 1b clinical trial in relapsed/refractory AML and MDS, both as a single agent and in combination with standard-of-care venetoclax and hypomethylating agents (HMA). Initial findings from this trial were announced in 2023, and additional data are expected to be presented in a scientific forum in 2024.

"Orphan drug designation from the FDA validates our belief that targeting galectin-9 with LYT-200 is a novel, promising approach that may offer patients a better tolerated, more effective treatment," notes Aleksandra Filipovic, MD, PhD, Head of Oncology at PureTech.

The FDA grants orphan drug designation to novel drug and biologic products for the treatment, diagnosis, or prevention of conditions affecting fewer than 200,000 persons in the U.S. Orphan drug designation qualifies PureTech for incentives under the Orphan Drug Act, including tax credits for some clinical trials and eligibility for seven years of market exclusivity in the U.S., if the drug is approved for AML.

About LYT-200

LYT-200 is a fully human IgG4 monoclonal antibody targeting galectin-9 for the potential treatment of hematological malignancies, such as acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS), as well as locally advanced/metastatic solid tumors that have poor survival rates, including head and neck cancers. Galectin-9 is a potent oncogenic driver and immunosuppressor, and in AML it has been described to work via cytotoxic CD8 T cells and natural killer cells. A wide variety of preclinical data underscores the importance of galectin-9 as a target and suggests a potential opportunity for biomarker development. These data demonstrate high expression of galectin-9 across various blood cancers and solid tumor types and show that galectin-9 levels correlate with poor survival in several cancers.

LYT-200 has demonstrated direct cytotoxic, anti-leukemic effects through multiple mechanisms, as well as synergy with standard of care chemotherapy and venetoclax in preclinical models. In the ongoing, Phase 1b clinical trial evaluating LYT-200 as a single agent in AML and MDS, LYT-200 has demonstrated a favorable safety and tolerability profile as well as early signals of potential clinical activity. This trial is ongoing, in addition to a Phase 1/2 adaptive design trial for the potential treatment of advanced/metastatic solid tumors. Consistent with its hub-and-spoke model, PureTech intends to advance LYT-200 via its Founded Entity Gallop Oncology.