Pharma Focus Europe

PureTech Secures Orphan Drug Designation for LYT-200 in Acute Myeloid Leukemia

Thursday, March 14, 2024

PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a clinical-stage biotherapeutics company, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to LYT-200 for the treatment of AML (Acute Myeloid Leukemia).

Dr. Amir Fathi, Director of the Leukemia Program at Massachusetts General Hospital and lead investigator of the trial, highlighted the urgent need for more effective treatments for patients with relapsed or refractory AML, given the poor long-term survival rates.

LYT-200, a fully human IgG4 monoclonal antibody, targets galectin-9, an oncogenic driver in leukemia cells and an immune-suppressive protein. It has shown promising anti-leukemic effects via multiple mechanisms and is being explored as a potential therapy for hematological malignancies like relapsed/refractory AML and high-risk MDS, as well as challenging solid tumors with poor survival rates, such as head and neck cancers.

Dr. Aleksandra Filipovic, Head of Oncology at PureTech, expressed confidence in LYT-200's innovative approach and its potential to offer patients a more effective and better-tolerated treatment option.

The FDA's orphan drug designation for LYT-200 acknowledges its potential to address unmet medical needs. This designation provides PureTech with various incentives under the Orphan Drug Act, including tax credits for certain clinical trials and seven years of market exclusivity in the U.S. upon approval for AML treatment.

LYT-200's mechanism of action involves targeting galectin-9, which is implicated in various blood cancers and solid tumor types, correlating with poor survival rates. Preclinical data have demonstrated its cytotoxic effects and synergy with standard chemotherapy and venetoclax.

Early findings from the Phase 1b clinical trial of LYT-200 in AML and MDS have shown promising safety, tolerability, and potential clinical activity. PureTech continues to advance LYT-200 through its Founded Entity Gallop Oncology, utilizing its hub-and-spoke model to explore its therapeutic potential further.



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