Trial Outcomes & Findings for Study to Evaluate Safety, Pharmacokinetic and Pharmacodynamic Dose Escalation and Expansion Study of PXS-5505 in Patients With Primary, Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (NCT NCT04676529)

Population: Patients participated in multiple dose levels. * One patient participated in Cohorts A, B, and C. * Two patients participated in Cohorts B and C. * Two patients from the dose escalation phase subsequently enrolled into the cohort expansion phase. Overall, across all the study arms a total of 43 unique patients were treated. Therefore the automatically calculated totals are incorrect.

Population: Patients participated in multiple dose levels. * One patient participated in Cohorts A, B, and C. * Two patients participated in Cohorts B and C. * Two patients from the dose escalation phase subsequently enrolled into the cohort expansion phase. Overall, across all the study arms a total of 43 unique patients were treated. Summaries at each timepoint include patients reaching that timepoint, with the assessment performed.

Population: Patients participated in multiple dose levels. * One patient participated in Cohorts A, B, and C. * Two patients participated in Cohorts B and C. * Two patients from the dose escalation phase subsequently enrolled into the cohort expansion phase. Overall, across all the study arms a total of 43 unique patients were treated. Summaries at each timepoint include patients reaching that timepoint, with the assessment performed.

Population: All 24 expansion patients are included in at least one row. 1 patient had no pre-dose Day 0 LOX activity result and is not included in LOX inhibition results. A different patient had no pre-dose Day 0 LOXL2 activity result and is not included in LOXL2 inhibition results. 14 patients are included from the add-on. 2 patients had no pre-dose Day 0 LOX or LOXL2 results. An additional patient had no pre-dose Day 0 LOXL2 activity result and is not included in LOXL2 inhibition results.

Population: 15 of the 24 patients enrolled in the expansion phase were analysed. 8 patients withdrew without having any post-baseline biopsy samples taken and 1 patient's sample could not be graded at baseline. 13 of the 16 patients enrolled in the add-on phase were analysed. 3 patients withdrew without having any post-baseline biopsy samples taken. Summaries at each timepoint include patients reaching that timepoint, with the assessment performed.

Population: 15 of the 24 patients enrolled in the expansion phase were analysed. 8 patients withdrew without having any post-baseline biopsy samples taken and 1 patient's sample could not be graded at baseline. 13 of the 16 patients enrolled in the add-on phase were analysed. 3 patients withdrew without having any post-baseline biopsy samples taken. Summaries at each timepoint include patients reaching that timepoint, with the assessment performed.

Population: 16 of the 24 patients enrolled in the expansion phase were analyzed. 8 patients withdrew without having any post-baseline response assessment. 14 in the add-on phase were analyzed, including 1 patient who discontinued just before 12 weeks but had a response assessment at withdrawal. 2 patients withdrew without having any post-baseline response assessment. Summaries include those still in the trial at each timepoint, and with assessments performed.

Population: 16 of the 24 patients enrolled in the expansion phase were analysed. 8 patients withdrew without having any post-baseline spleen volumes measurements taken. 1 patient had a missing assessment at week 12 but is included in week 24 results. 13 of the 16 patients enrolled in the add-on phase were analysed. 3 patients withdrew without having any post-baseline spleen volume measurements. Summaries at each timepoint include patients reaching that timepoint, with the assessment performed.

Population: 22 patients were enrolled in the expansion phase with an enlarged spleen and 13 in the add-on phase. Summaries at each week are for the patients still on trial at that timepoint who had the assessment performed. Summary of overall response at any visit are for all enrolled patients with an enlarged spleen at baseline.

Population: 22 patients were enrolled in the expansion phase with an enlarged spleen and 13 in the add-on phase. Summaries at each week are for the patients still on trial at that timepoint who had the assessment performed. Summary of overall response at any visit are for all enrolled patients with an enlarged spleen at baseline.

Population: 24 patients were enrolled in the expansion phase, 13 patients completed the 24 week treatment period, 11 patients withdrew early from the study. 16 patients were enrolled in the add-on phase, 7 patients completed the 52-week treatment period, 9 patients withdrew early from the study. Summaries at each week are for the patients still on trial at that timepoint. Summary of overall response at any visit are for all enrolled patients.

Population: 16 of the 24 patients enrolled in the expansion phase were analyzed. 8 patients withdrew without having any post-baseline TSS assessment. 3 patients were missing the assessment at week 12 but are included at week 24. 13 in the add-on phase were analyzed. 3 patients withdrew without having any post-baseline TSS assessment. Summaries include those still in the trial at each timepoint, and with assessments performed.

Population: Anemia and minor anemia response summaries include only patients with hemoglobin \<100g/L at baseline and who remain in the study until at least week 12. Reduction in transfusion burden summary includes only those receiving transfusions in the 12 weeks prior to treatment and who remain in the study until at least week 12