Puretech Health’s Share Price Rose 1.98% on Orphan Drug Status

The Puretech Health PLC (LON: PRTC) share price rose 1.98% after announcing that the US Food and Drug Administration (FDA) has granted orphan drug designation to LYT-200, its drug candidate for treating acute myeloid leukaemia (AML). 

LYT-200, a fully humanised IgG4 monoclonal antibody, zeroes in on galectin-9. This target is known for its dual role as a significant oncogenic factor in leukaemia cells and as an immunosuppressive agent. 

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Through its innovative action mechanism, LYT-200 exhibits direct cytotoxic properties against leukaemia cells and holds promise as a pioneering therapeutic approach for various haematological malignancies.

The drug targets conditions such as relapsed or refractory AML and high-risk Myelodysplastic Syndromes (MDS), in addition to advanced or metastatic solid tumours with traditionally low survival rates, like specific head and neck cancers. 

LYT-200’s efficacy and safety are currently under examination in a Phase 1b clinical trial, focusing on patients with relapsed or refractory AML and MDS. This investigation explores LYT-200’s performance as an independent treatment and in conjunction with established therapies like venetoclax and hypomethylating agents (HMA).

Preliminary results of this study were shared in 2023, with further detailed findings anticipated to be disclosed at a scientific conference in 2024.

The FDA’s orphan drug designation is awarded to innovative therapies and biological products designed for treating, diagnosing, or preventing rare diseases affecting less than 200,000 people across the U.S.

The distinction brings several advantages under the Orphan Drug Act, such as tax benefits for specific clinical trials and the potential for seven years of exclusive marketing rights in the U.S. upon approval for the drug’s use in AML treatment.

Amir Fathi, MD, Director of the Leukemia Program at Massachusetts General Hospital and lead investigator of the trial, said: “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.”

Aleksandra Filipovic, MD, PhD, Head of Oncology at PureTech, noted: “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.”

Puretech Health share price. 

The PureTech Health share price rose 1.98% to trade at 206.0p from Tuesday’s closing price of 202.0p.