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Following the recent U.S. Food and Drug Administration (FDA) orphan drug designation, an Investigational New Drug (IND) application has been submitted for a phase I/II study of PXS-5505, a pan-lysyl oxidase (LOX) inhibitor, which is currently under development for the treatment of myelofibrosis.
PXS-5505 has been previously tested in healthy volunteers in a phase I study. The IND submission protocol covers a dose escalation study for 1 month, and an open-label, 6-month treatment phase in patients who are not receiving a Janus kinase inhibitor. The study is planned to be started by the end of 2020 and be completed in 2022.
Inhibition of LOX family members is expected to show benefits of reduced fibrotic burden and to have favorable effects on aberrant blood cell production seen in myelofibrosis.
Myelofibrosis | FDA grants an orphan drug designation for PXS-5505
On July 14, 2020, the U.S. Food and Drug Administration (FDA) granted an orphan drug designation for PXS-5505.
Editorial theme | The management of relapsed/refractory myelofibrosis: Part 1 – JAK failure and challenges
In the management of myelofibrosis (MF), Janus kinase (JAK) inhibition as first-line treatment has been shown to play a crucial role in the...
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