Pharmaxis Ltd (ASX:PXS, OTC:PMXSF) plans to accelerate a combination study for its lead PXS-5505 drug candidate in myelofibrosis patients following a US Food and Drug Administration (FDA) review.
The regulatory body analysed interim safety and efficacy data from Pharmaxis’ ongoing monotherapy trial, providing guidance on the number of patients, treatment dosage, study duration and endpoints for an add-on trial in combination with a JAK inhibitor.
Following the FDA feedback, Pharmaxis plans to kick off the expanded combination therapy trial later this year, teaming the myelofibrosis standard of care with its PXS‐5505 candidate.
Maximising commercial opportunities
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.”
Pharmaxis unveiled interim data from its monotherapy trial earlier this year, presenting at American Society of Hematology and publishing groundbreaking pre‐clinical data in myelodysplastic syndrome (MDS) in Nature Communications for PXS‐5505.
Following a review of its development strategy, Pharmaxis has decided to focus its resources on these haematological malignancies, meaning it has put an investigator-initiated clinical trial into hepatocellular carcinoma with the University of Rochester on hold.
Phillips continued: “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 hepatocellular carcinoma given the timelines for recruitment and the need to focus our resources.”
More to come
Pharmaxis has been hard at work recruiting for the current study, which has reached 21 out of a targeted 24 patients on monotherapy with 20 sites active worldwide.
Moving forward, the clinical research company 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, Pharmaxis believes the trial design can be streamlined to initiate the combination arm at the same dose currently used in the monotherapy study.
The company will outline the study’s design, timelines and costs once it receives feedback from regulators later this quarter.