Anti Fibrotic LOXL2 Program Set To Commence Phase 1 Trials in Q4 2017
Pharmaxis has consolidated its position as a significant competitor in the NASH market as the LOXL2 inhibitor joins its SSAO inhibitor in the clinic. Pharmaxis and its collaborator UK biotechnology company Synairgen plc recently announced completion of the preclinical development stage of their anti-fibrotic Lysyl Oxidase type 2 (LOXL2) inhibitor program allowing the first compound to commence human clinical phase I studies in Q4 2017.
The Pharmaxis drug discovery group has developed a number of selective small molecule inhibitors to the LOXL2 enzyme utilising the same amine oxidase platform that delivered PXS-4728A, an anti-inflammatory drug that was acquired by Boehringer Ingelheim in 2015. The LOXL2 enzyme is fundamental to the fibrotic cascade that follows chronic inflammation in the liver disease NASH, cardiac fibrosis, kidney fibrosis, and idiopathic pulmonary fibrosis (IPF), and it also plays a role in some cancers.
The extensive pre‐clinical program performed on the program compounds confirmed that they have all the characteristics of a successful once a day, oral drug showing excellent efficacy in several different in vivo fibrosis models including fibrosis of the liver, lung, kidney and heart. In regulatory toxicity studies, our compounds have been well tolerated and shown a good safety profile.
NASH is a growing and valuable market for the future and we anticipate that our LOXL2 program will be attractive to many of the multinational pharmaceutical companies that are building a portfolio approach to treating NASH and some other fibrotic diseases such as IPF.
Pharmaxis Earns A$15 Million Milestone Payment as Boehringer Ingelheim Clinical Trial in Diabetic Retinopathy Doses 1st Patient
Pharmaxis Ltd has announced dosing of the first patient in Boehringer Ingelheim’s Phase 2a clinical trial in patients with diabetic retinopathy (DR), triggering a €10 million (~A$15 million) milestone payment to Pharmaxis. DR is the second disease to be targeted with BI 1467335 originally discovered by Pharmaxis. BI 1467335 is also in an ongoing Phase 2a clinical trial for non-alcoholic steatohepatitis (NASH). Both studies are due to report in the second half of 2018.
The Phase 2a clinical trial of BI 1467335 in diabetic retinopathy will randomise 100 patients to either drug or placebo for a 12-week treatment period with an additional 12-week follow-up period. A subsequent Phase 2b study will seek to confirm and extend these findings.
Diabetic retinopathy is the leading cause of vision-loss in adults aged 20-74. Of an estimated 285 million people with diabetes mellitus worldwide, approximately one third have signs of the disease.
Boehringer Ingelheim’s two-pronged clinical program with BI 1467335, an oral inhibitor of amine oxidase, copper containing 3 (AOC3) discovered by Pharmaxis, is focused on tackling the common diseases related to the complications of diabetes which affect millions of people worldwide. The drug was acquired by Boehringer Ingelheim in 2015 with an upfront payment of A$41m to initially study the chronic liver condition NASH.
This milestone payment of $15 million for the commencement of a phase 2a trial in a second disease brings the total payments received by Pharmaxis from Boehringer to €57 million (~A$86 million). The total of all potential development milestones payable to Pharmaxis if both indications in the deal are approved is approximately A$625 million.
Both development plans attract the same total development payments through to approval, with the 2nd 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 a total of €62 million in milestones.
 Also known as vascular adhesion protein-1 (VAP-1) or semicarbazide-sensitive amine oxidase (SSAO)
Details of the DR clinical trial can be found here.
Watch a 5 minute video with Pharmaxis CEO Gary Phillips discussing the clinical program with Boehringer: https://www.youtube.com/watch?v=OSB0fGkW5Kk
Pictured above : Pharmaxis CEO Gary Phillips and Boehringer Ingelheim MD Australia & NZ Wes Cook mark the start of a new clinical tril in diabetic retinopathy.
Chairman Malcolm McComas’ Address to the 2017 Pharmaxis Annual General Meeting
Welcome to the Pharmaxis 2017 Annual General Meeting, a meeting that follows another successful year for the Company in terms of revenue, drug development initiatives and shareholder value.
On 22 September 2017, something we had pointed to for some time happened as expected. It was however something that is not common in the pharmaceutical research sector. Without going out to the stock market and asking shareholders for new money, Pharmaxis banked $27m cash. This was the second payment received from Boehringer Ingelheim and together with their initial $41m payment received in May 2015, total cash received to date is $68m. This is equivalent to approximately 21 cents per share.