With a share price that has effectively doubled over the past few months and a drug pipeline beginning to snowball its successes, investors that identified Pharmaxis (ASX: PXS) early on are justifiably ecstatic.

The clinical stage drug developers have had a strong start to FY22 with their lead asset, oncology drug PXS-5505 and anti-skin scarring topical drug, PXS-6302 both performing well in clinical trials.

Designed for the treatment of rare blood cancer myelofibrosis, PXS-5505 could potentially corner a market worth (a conservatively estimated) $1 billion per year.

The drug works to inhibit the lysyl oxidase enzymes responsible for damage and scarring to the bone marrow, a disease mechanism that ultimately leads to reduced immunity, bleeding issues and poor patient outcomes. Unlike current therapies, Pharmaxis’ drug has unique disease modifying potential as demonstrated in preclinical studies.

Last week Pharmaxis achieved a milestone, announcing the progression of PXS-5505 to Phase 2 trials after positive results throughout Phase 1.

Keen to maintain momentum, the Company has today announced that dosing for Phase 2 has commenced in myelofibrosis patients across sites in Australia and South Korea. Additional sites in both Taiwan and the US are scheduled for commencement of dosing shortly.

Already cleared by the FDA under the Investigational New Drug Scheme, the trial aims to demonstrate the efficacy and safety of PXS-5505 as a monotherapy for patients not eligible or tolerant to JAK inhibitors, the current standard of care therapy. Additionally, the trial will seek to evaluate the impact of the drug on several disease metrics such as bone marrow fibrosis, cytopenia and spleen volume.

After garnering international attention from renowned researchers independent study results have begun to surface demonstrating the drug’s potential for the treatment of other diseases such as liver cancer.

CEO of Pharmaxis, Gary Philips commented on the news of first dosing, saying: “the first patients entering the Phase 2 study were participants in the just completed Phase 1c dose escalation study. We are pleased that these patients want to continue on PXS-5505 allowing us a rapid start to the next stage of the clinical trial. Having shown that the drug has a good tolerability profile at a dose which can fully inhibit all the family of lysyl oxidase enzymes that are implicated in myelofibrosis, we are confident of being able to assess the impact of this mechanism of action in the ongoing study. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022.”

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