Clinical stage drug development company Pharmaxis (ASX: PXS) is one step closer to seeing their revolutionary cancer drug reach commercialisation.

PXS-5505, the Company’s lead asset has received the go-ahead from the safety committee to begin Phase 2 trials following positive results from the Phase 1c study which saw patients with myelofibrosis treated for 28 days at 3 dosage levels.

Designed for the treatment of myelofibrosis, a rare blood cancer, PXS-5505 works by inhibiting lysyl oxidase enzymes, the enzymes responsible for damage and scarring in the bone marrow. Damage to the bone marrow disrupts the body’s normal production of blood cells leading to severe anaemia, reduced immunity and bleeding issues. Once diagnosed, the average lifespan is just five years. About 10% of those diagnosed will go on to develop leukaemia.

Independent peer reviewed research has shown that there is significant upregulation of lysyl oxidase enzymes in myelofibrosis. Inhibiting the action of these has been demonstrated in preclinical models. PXS-5505 effectively reduced bone marrow fibrosis, reduced spleen size and improved blood cell count – results that have secured major investment from a very successful pharmaceutical-focused hedge fund.

Current treatment options for myelofibrosis are symptom treating only. PXS-5505’s point of difference is its disease modifying action that is designed to slow the disease. As an effective, disease modifying drug for myelofibrosis, commercialisation of PXS-5505

would open Pharmaxis to a market that is “conservatively estimated” at US$1 billion per annum.

Phase 2 of the trial, known as the dose expansion phase, will involve treatment of 24 patients at the highest dose tested in the Phase 1c trial for six months. Trial sites across Australia, South Korea, Taiwan and the USA will recruit patients.

CEO of Pharmaxis, Gary Phillips welcomed the progression of the trials, commenting: “We are very pleased to have completed the dose escalation phase of this study with such clear and positive findings. We will now immediately progress to the phase 2 dose expansion study where we aim to show PXS-5505 is safe to be taken longer term with the disease modifying effects that we have seen in the pre-clinical models. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022.”

The Phase 1c/2a trials have already been cleared by the US Food and Drug Administration (FDA) under the Investigational New Drug scheme.

Due to its unique mechanism of action, renowned global researchers have conducted their own studies on the drug, revealing that PXS-5505 holds potential for the treatment of brain cancer, liver cancer and pancreatic cancer. The ability of the drug to break down fibrotic tissue at the cancer site has been shown to enhance the effect of chemotherapy in pre-clinical trials, and could potentially be used as an adjunct to chemo drugs, a $50 billion market.

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