Ingelheim, Germany and Sydney, Australia, 7 September 2020?? Boehringer Ingelheim and Pharmaxis Ltd today announced the discontinuation of the development of anti-inflammatory AOC3 inhibitor BI 1467335 for the treatment of patients with moderate-severe non-proliferative diabetic retinopathy (NPDR). BI 1467335 was acquired from Pharmaxis in 2015 and Boehringer Ingelheim will now terminate the agreement.

In a Phase IIa trial in patients with moderate-severe NPDR, BI 1467335 met its primary endpoint in ocular safety with the treatment being well tolerated. Boehringer Ingelheim decided not to further develop BI 1467335 in this indication based on the lack of a clear efficacy signal and risk of dose dependent drug interactions of the compound in NPDR patients identified in another Phase I study.

Boehringer Ingelheim takes a holistic approach to the development of novel retinal disease therapies, targeting key mechanisms in the pathogenesis of retinal diseases. By leveraging existing expertise in oncology, inflammation, neurodegeneration, fibrosis and cardiometabolic diseases, the company is building a growing portfolio of next generation retinal therapy approaches in various stages of development up to Phase 2 in macular degeneration and diabetic retinal diseases.

Pharmaxis CEO, Gary Phillips said, ?We understand Boehringer Ingelheim?s decision to stop development of BI 1467335 in NPDR based on the risk of dose dependent drug interactions at the dose level that was tested in the Phase IIa trial. Based on recent publications AOC3 remains an important clinical target. We will review the data collected in more detail to evaluate potential opportunities in other indications that already have supportive pre-clinical data and where the risk of drug interactions are of less concern. In the meantime, we look forward to the upcoming FDA decision on granting a marketing authorisation for Bronchitol in the US cystic fibrosis market and our focus remains on advancing our anti-cancer pan-LOX inhibitor program into a phase 2 myelofibrosis study following the recent IND approval.?

The Phase IIa trial (ClinicalTrials.gov Identifier: NCT03238963) was a Phase IIa multi-centre, double-masked design in 79 patients with moderately severe to severe non-proliferative diabetic retinopathy (NPDR) without center-involved diabetic macular edema (CIDME). The primary objective was to evaluate the safety and tolerability of BI 1467335. Secondary objective was the proportion of patients with a two step improvement in diabetic retinopathy severity score (DRSS). Patients were randomized to either BI 1467335 or to placebo for a 12-week treatment period with an additional 12-week follow-up period afterwards.

Diabetic retinopathy (DR) is the leading cause of vision-loss in adults aged 20-74. It progresses from mild nonproliferative diabetic retinopathy to moderate and severe nonproliferative diabetic retinopathy (NPDR), characterized by retinal hemorrhages and vascular changes in the retina, to proliferative diabetic retinopathy (PDR), characterized by the growth of new blood vessels on the retina. Diabetic Macular Edema (DME), characterized by retinal thickening from leaky blood vessels, can develop at all stages of retinopathy. Of an estimated 285 million people with diabetes mellitus worldwide, approximately one third have signs of DR and of these, a further one third of DR is vision-threatening DR (severe NPDR, PDR and DME).

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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 with drug candidates in clinical trials.

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. After an anti-inflammatory AOC3 inhibitor developed in 2015, 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/