Pharmaxis (ASX:PXS) has completed a busy June quarter, which involved three key milestones, recruitment for the phase 2 NASH trial, advancement of the LOXL2 development program and clinical trial results for Bronchitol®.
When the first patient in the phase 2 NASH trial is dosed it will trigger a milestone payment to Pharmaxis of circa A$26 million.
The advancement of the LOXL2 development program remained on track to progress into a phase 1 clinical study in the second half of 2017.
At the end of June, Pharmaxis had $21.5 million cash at bank.
Gary Phillips, CEO, commented: “Our Drug Discovery team have done outstandingly well to generate five lead candidate small molecule drugs to four different targets in the last two years since we sold the first phase 1 asset to Boehringer.”
Boehringer Ingelheim phase 2 NASH trial
Boehringer acquired PXS-4728A in May 2015 to develop initially as a treatment for non-alcoholic steatohepatitis (NASH).
Under the terms of the agreement, Boehringer has total responsibility for the development program and is required to make milestone payments to Pharmaxis as the drug progresses towards approval as well as other sales related payments post approval.
Boehringer has advised that a phase 2 NASH trial has commenced recruitment and dosing of the first patient will trigger a milestone payment to Pharmaxis of €18 million (~A$26 million).
Boehringer has now advised that a phase 2 trial in a second indication will commence in the second half of 2017, triggering the payment of a €10 million (~$14 million).
LOXL2 inhibitor program
Pharmaxis is working with its collaborator, UK biotechnology company Synairgen plc (LSE: SNG) to commence a phase 1 study in the second half of 2017.
The Pharmaxis drug discovery group has developed a number of selective inhibitors to the lysyl oxidase type 2 enzyme (LOXL2).
LOXL2 is a key enzyme in the formation of excessive collagen depositions and important in the liver disease NASH, cardiac fibrosis, kidney fibrosis, the fatal lung disease idiopathic pulmonary fibrosis (IPF) and also plays a role in some solid cancers.
Large pharma company interest
The role of LOXL2 in fibrotic diseases such as NASH and pulmonary fibrosis is of significant interest to many large pharma companies.
The annual US BIO conference in June 2017 provided an opportunity to share updates with them on Pharmaxis’s scientific progress and the timing of its partnering process which it plans to initiate in the second half of 2017 and conclude with a partnering deal in 2018.
Other irons in the fire
Pharmaxis has other drugs in its development pipeline including:
- A LOX inhibitor program which has potential anti-fibrotic application in scarring;
- The SSAO/MPO program which is developing a dual inhibitor with potential anti-inflammatory application in respiratory and cardiovascular disease; and
- The SSAO/MAOB inhibitor program has potential anti-inflammatory application in a number of indications.
Bronchitol for cystic fibrosis
Bronchitol® is an inhaled dry powder for the treatment of cystic fibrosis and has been the subject of three large scale global clinical trials conducted by Pharmaxis.
The product is approved and marketed in Europe, Russia, Australia and several other countries.
The study reported in June paves the way for the company’s partner Chiesi to conclude the US application to market the product for adult cystic fibrosis patients.
This step also completes the change in Pharmaxis’s business model as it reduces exposure to late stage clinical development and commercialisation risk and generates increasing value from early stage clinical development and partnering.