On October 19, 2022 Clinical stage drug development company Pharmaxis (ASX: PXS) reported that it has concluded an interim analysis of data from 6 patients who have completed 6 months’ treatment with PXS-5505 in its open label phase 2 clinical trial in patients with the bone marrow cancer myelofibrosis (Press release, Pharmaxis, OCT 19, 2022, View Source;utm_campaign=Pharmaxis%20Releases%20Promising%20Interim%20Data%20from%20Skin%20Scarring%20Study&utm_content=Pharmaxis%20Releases%20Promising%20Interim%20Data%20from%20Skin%20Scarring%20Study+CID_e06885616dc6e57f4d0378eea23abf49&utm_source=Campaign%20Monitor&utm_term=View%20Full%20Media%20Release [SID1234622192]).

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The phase 2 trial known as MF-101 was cleared by the FDA under the Investigational New Drug (IND) scheme and aims to demonstrate that PXS-5505, the lead asset in Pharmaxis’ drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. These patients have very limited treatment options and a life expectancy of approximately 1 year.

A total of 15 patients have been enrolled in the cohort expansion phase of the study with 6 patients having completed 24 weeks of treatment. Four patients have dropped out of the study due to a lack of clinical response.

PXS-5505 has been well tolerated with no serious treatment related adverse events reported.
2/6 patients had clinically important improvement in symptoms.
5/6 patients had either stable or improved bone marrow fibrosis scores of ≥1 grade.
5/6 have stable or improved platelet and/or haemoglobin scores
No reductions were seen in spleen volume
Dr Gabriela Hobbs MD, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukemia Service, Massachusetts General Hospital said, "PXS-5505 continues to be very well tolerated in the clinic with no severe treatment related adverse events reported. Though still early in the dose expansion phase of the study, PXS-5505 appears to be stabilising and in some cases, improving the hemoglobin and platelet counts, which has also been associated with symptom improvements in those patients that were treated to 24 weeks.

"This is encouraging given the poor prognosis seen after ruxolitinib discontinuation with a median overall survival of only 11-14 months, typical of this study population. These results support further clinical investigation of PXS-5505 in myelofibrosis."