Investigational New Drug Submitted to the FDA for the treatment of MF
Great news in upcoming therapies for myeloproliferative neoplasms! Australia based pharmaceutical company, Pharmaxis Limited has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for a planned phase 1/2 study of PXS-5505 for the treatment of myelofibrosis.
"This IND submission incorporates the pre-IND feedback Pharmaxis received from the FDA earlier this year and is the next major step forward in the clinical development program of PXS-5505 for the treatment of myelofibrosis," said Mr. Gary Phillips, Chief Executive Officer of Pharmaxis. "We are leveraging our leadership in lysyl oxidase science to bring new treatment options for these severely underserved patients and strongly believe that our novel approach of inhibiting all of the lysyl oxidase family members could reduce bone marrow fibrosis and have beneficial effects on blood cell production and consequently other aspects of the disease."
Pharmaxis Limited is an Australian pharmaceutical research company and a global leader in drug development for inflammation and fibrotic diseases. The company has a highly productive drug discovery engine, drug candidates in clinical trials and significant future cash flows from partnering deals. Leveraging its small-molecule expertise and proprietary amine oxidase chemistry platform, Pharmaxis has taken four in-house compounds to Phase 1 trials in just five years. Boehringer Ingelheim acquired the Pharmaxis anti-inflammatory AOC3 inhibitor in 2015 to develop it (BI 1467335) for two diseases: the liver condition Non-alcoholic Steatohepatitis (NASH) and diabetic retinopathy (DR). The company’s successor amine oxidase program has developed an oral anti-fibrotic LOXL2 inhibitor, aimed at NASH, pulmonary fibrosis (IPF) and other high-value fibrotic heart and kidney diseases, with a commercial partnering process underway, a systemic pan-LOX inhibitor for acute fibrosis and cancer that will enter a phase 2 study in 2020 and a topical pan-LOX inhibitor for scarring that is expected to commence phase 1 studies in 2H 2020. Pharmaxis' Mannitol platform has yielded the products Bronchitol for cystic fibrosis, which is marketed in Europe, Russia, and Australia, with United States FDA approval pending; and Aridol for the assessment of asthma, which is sold in the United States, Europe, Australia, and Asia. Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au/