Dosing of the first patient in Boehringer Ingelheim’s Phase 2a clinical trial in patients with diabetic retinopathy triggered the payment.
It is the second disease targeted by the drug developed by Pharmaxis, which is being used in a Phase 2a clinical trial for the chronic liver disease NASH (non‐alcoholic steatohepatitis).
The studies into NASH and diabetic retinopathy are due to report in the second half of 2018.
Today’s payment further strengthens the Pharmaxis balance sheet, which held $39 million in cash at the end of September 2017.
Gary Phillips, chief executive officer, said: “This milestone payment marks the expansion of the Boehringer Ingelheim development plan for BI 1467335 into two diseases.
“All the potential development milestones in the $625 million deal would be payable to Pharmaxis should both indications be approved.
“Both development plans attract the same total development payments through to approval, with the second indication milestone payments weighted more towards approval.
“The next milestones from BI are payable if they choose to commence phase 3 trials, at which time the two indications would attract €62 million [circa $94 million] in milestones.”
A leading cause of vision loss
Diabetic retinopathy is the leading cause of vision loss in adults aged 20‐74.
Of an estimated 285 million people with diabetes mellitus worldwide, 33% have signs of the disease.
The Phase 2a clinical trial of BI 1467335 in diabetic retinopathy will randomise 100 patients to drug or placebo for a 12‐week treatment period with an additional 12‐week follow‐up period.
After the study reports later this year, a subsequent Phase 2b study will seek to confirm and extend the findings.
Two-pronged clinical program
Boehringer Ingelheim’s two‐pronged clinical program with BI 1467335 is focused on tackling the common diseases related to the complications of diabetes which affect millions worldwide.
The drug was acquired by Boehringer Ingelheim in 2015 with an upfront payment of $41 million to initially study NASH.
Pharmaxis has a portfolio of products at various stages of development and approval.
Its product Bronchitol for cystic fibrosis is marketed in Europe, Russia and Australia.
Another product, Aridol, for the assessment of asthma, is sold in Europe, Australia and Asia.
The company’s development pipeline is centred on its expertise in amine oxidase chemistry.
It includes a series of Lysyl Oxidase Inhibitors that have entered clinical development targeting fibrotic diseases of the heart, kidney, liver and lung.