On April 11, 2023 Pharmaxis Ltd (ASX: PXS) reported it will add a combination treatment arm to the current Phase 2 clinical trial of PXS‐5505 in myelofibrosis (MF). Following helpful feedback from the U.S. Food and Drug Administration (FDA), the trial will be widened to include myelofibrosis patients already receiving a JAK inhibitor as standard of care in combination with PXS‐5505 (Press release, Pharmaxis, APR 11, 2023, View Source;utm_campaign=Following%20FDA%20Review%20Pharmaxis%20to%20Accelerate%20Plans%20for%20PXS-5505%20Combination%20Study&utm_content=Following%20FDA%20Review%20Pharmaxis%20to%20Accelerate%20Plans%20for%20PXS-5505%20Combination%20Study+CID_03240385beb62faf2fb8d4378d022ac5&utm_source=Campaign%20Monitor&utm_term=View%20Full%20Media%20Release [SID1234629961]).
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Pharmaxis has previously reported interim data from MF‐101 in a monotherapy setting demonstrating a well‐tolerated drug that leads to stable or improved symptoms, haematological cell counts and fibrosis grades. In a Type C Meeting review, the FDA examined a package of safety and efficacy information from the monotherapy trial and provided guidance on the number of patients, treatment dosage, study duration and endpoints for a study in combination with a JAK inhibitor.
Recruitment for the current study has reached 21 out of a targeted 24 patients on monotherapy with 20 sites active worldwide. Pharmaxis now plans to submit a protocol amendment to global regulators, including the FDA, that will add an arm to the existing MF study MF‐101 and utilise its existing trial sites. Based on the FDA feedback, it is anticipated that the trial design can be streamlined to initiate the combination arm at the same dose currently used in the monotherapy arm and commence later this year.
Pharmaxis CEO, Gary Phillips said, "The agreement with the FDA to expand the patient population in the ongoing phase 2 study to include those patients currently on a JAK inhibitor is an important step forward in realising the benefits of lysyl oxidase inhibition for all myelofibrosis patients and in maximising the commercial opportunity for PXS‐5505. We are already in discussion with the existing trial site investigators who have welcomed the opportunity to extend the patient population for the study and anticipate significantly accelerated recruitment."
In recent months Pharmaxis has reported interim data from MF‐101, two poster presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) and the publication of ground breaking pre‐clinical data in myelodysplastic syndrome (MDS) in Nature Communications for the Company’s lead asset, PXS‐5505. Following a review of its development strategy Pharmaxis has decided to focus its resources on these haematological malignancies and will not at this point be progressing the previously planned study in hepatocellular carcinoma (HCC) patients in an investigator initiated clinical trial by the University of Rochester.
Pharmaxis CEO Gary Phillips said, "Our collaboration with the research team at University of Rochester remains highly valued and their work is continuing with further pre‐clinical evaluation of our pipeline assets but for now we have decided not to pursue HCC given the timelines for recruitment and the need to focus our resources."
Pharmaxis will provide further details of study design, timelines and costs for the PXS‐5505 / JAK Inhibitor combination arm of the MF‐101 study after it receives feedback from regulators on the amended protocol, expected in Q2 2023.