Pharmaxis Ltd (ASX: PXS) today published a quarterly update in which the company reported three significant events. Consequently, the Pharmaxis share price has shot up 47.73% to 13 cents this morning. The pharmaceutical research company is working on inflammation and fibrosis. In addition it already has a portfolio of products at various stages of development and approval.
What's moving the Pharmaxis share price?
The company's announcement contained four very important issues for a drug research enterprise. One of which is linked to short term funding but all bode well for the Pharmaxis share price.
First, the Food and Drug Administration (FDA) granted permission to progress its myelofibrosis drug, PXS‐5505, into a phase 1c/2 study. Myelofibrosis is a rare bone cancer. The company has moved quickly to contract Parexel to conduct the study which will start recruiting in Q1 2021.
Second, the FDA granted Pharmaxis orphan drug designation for PXS‐5505 in myelofibrosis. This means it is a treatment for a rare disease or condition. Or more succinctly, one that affects less than 200,000 persons in the US. This designation means the drug marketing application is not subject to a prescription drug user fee.
Third, the Australian Government awarded Pharmaxis $1 million in funding for pre‐clinical drug. This was from the Biomedical Translation Bridge (BTB) program. It will significantly advance work on the company's drug discovery for the treatment of the devastating genetic disorder Duchenne Muscular Dystrophy (DMD).
In fact, the funding grant will take the company all the way to the start of phase 1 trials for this new drug candidate. Pharmaxis chief executive officer Garry Phillips said this was an example of the strategy to accelerate drug discovery until it could further define the commercial opportunity.
Bronchitol US FDA review
For the coming quarter, the company will be preparing to start the myelofibrosis study. However, there will also be considerable focus on the outcome of the Bronchitol NDA filed by US licensee Chiesi. The FDA have advised it has a 'goal action date' of 1 November 2020 which was yesterday. If successful, the company has a US$10m milestone payments attached to the approval and supply of launch stock to Chiesi.
