- The clinical-stage drug development company Pharmaxis has released the final interim analysis data from its phase 2 myelofibrosis (a type of bone marrow cancer) trial. The data shows that 60% of patients who completed 6 months' treatment with PXS-5505, a lysyl oxidase (LOX) enzyme inhibitor, demonstrated improved fibrosis grade and showed promising signs of clinical efficacy. These patients were ineligible, intolerant, or unresponsive to treatment with approved JAK inhibitor drugs.
- Pharmaxis has submitted the protocol for the next cohort study in combination with a JAK inhibitor to the FDA, following a positive type C meeting, with the trial start date set for later this year. This supports the company's promising skin scarring study outcome, offering clear proof of the concept in fibrosis.
- Out of 21 patients enrolled in the cohort expansion phase of the trial, 10 completed 24 weeks of treatment with no serious treatment-related adverse events reported. However, 10 other patients dropped out of the trial due to a lack of clinical response or adverse events unrelated to the drug.