Pharmaxis updates on business plan after BI halts study


Disappointment for Australian company Pharmaxis with Boehringer Ingelheim discontinuing the development of its investigational compound BI 1467335 in a significant liver disease.

The German company acquired rights to the AOC3 inhibitor in 2015 with plans for its development in Non-Alcoholic Steatohepatitis (NASH).

NASH is the progressive form of non-alcoholic fatty liver disease which is the most common liver disorder in Western industrialised countries.

It has subsequently expanded its development to include diabetic retinopathy.

Pharmaxis announced yesterday that while the development of BI 1467335 in NASH has been discontinued, ongoing studies including in diabetic retinopathy will continue.

"The reason provided by BI for the discontinuation after a successful phase 2a study that met its safety and efficacy endpoints was the potential for drug interactions in NASH patients," said the company in a statement.

It continued, "A second study of the drug in diabetic retinopathy with associated milestone payments will continue with future development to be decided by BI following completion of the current phase 2a study due to report 2H 2020.  

"Boehringer Ingelheim’s decision has no impact on short term cash as the next milestone payment for NASH was not scheduled until the commencement of a phase 3 study in several years’ time."

In a business update, Pharmaxis said it ended the September quarter with $23 million cash, which was topped up in October by a $6.2 million cash refund under the federal government's R&D tax incentive.

"In addition, Pharmaxis anticipates receipt of a US$10 million milestone payment for Bronchitol from its US licensee Chiesi in Q3 2020 subject to FDA approval midyear," it said.

According to CEO Gary Phillips, “We are obviously disappointed by BI’s decision not to progress the compound acquired from Pharmaxis in NASH. This was unexpected.

"We understand the significant hurdles to overcome in the early stages of drug development. That is why the company has pursued a strategy of generating multiple research pipeline opportunities underpinned by our cash position and a partnering strategy which offlays development risk where appropriate.”

Pharmaxis also has a topical pan LOX inhibitor program with potential in scar revision. It said this program is currently completing late-stage pre‐clinical testing and is anticipated to enter a phase 1 clinical trial in healthy volunteers in 2020.

“The topical LOX inhibitor has significant market potential in both post‐surgical and cosmetic indications. The company will carefully consider the optimal development plan based on the financial resources required and the level of interest from pharma companies with an interest in dermatology,” added Mr Phillips.