Amplia Therapeutics has received encouraging lab results which highlighted the use of its lead drug narmafotinib, saying it can attack several of the nastier features that make the cancer so hard to treat.

The research, led by Professor Paul Timpson and colleagues at Sydney’s Garvan Institute of Medical Research, was the results of extensive laboratory work on narmafotinib in pancreatic cancer models and was posted online on the biology preprint server bioRxiv.

Amplia says the work adds further evidence that narmafotinib, a focal adhesion kinase (FAK) inhibitor, may improve outcomes by targeting tumour fibrosis, chemotherapy resistance and metastatic spread - three big hurdles in a disease that remains one of cancer medicine’s toughest assignments.

FAK is a protein inside cells that acts like a control switch for movement, survival and attachment. It sits where cells anchor to their surrounding environment and helps tell them whether to grow, move or shut themselves down.

‘The research conducted by Professor Timpson’s team over recent years is now being shared with the broader scientific community.’Amplia Therapeutics chief executive office and managing director Dr Chris Burns

In many cancers, FAK becomes overactive or over-produced, helping tumour cells survive, move and spread. That has made it a target for cancer-fighting drugs such as narmafotinib.

Among the key findings, the paper reported reduced tumour fibrosis and enhanced anti-tumour activity when narmafotinib was combined with chemotherapy. Other results included reduced cancer dissemination and metastatic spread in a pancreatic cancer model and the down-regulation – or diminished activity - of multiple genes linked to chemotherapy resistance.

In this case, the drug appears to turn down genes that usually help cancer cells resist chemotherapy. The effect is to weaken the cancer cells’ defences, leaving chemotherapy better able to do its job and potentially lowering the number of cancer cells that remain alive after treatment.

Amplia Therapeutics chief executive officer and managing director Dr Chris Burns said: “These studies strengthen the scientific rationale for the clinical development of narmafotinib and provide further insight into the multifaceted mechanisms through which narmafotinib can act in the treatment of this devastating disease.”

The update follows a busy clinical run for the Melbourne-based biotech. In May, Amplia launched the first stage of a Phase 2b registration-enabling pancreatic cancer study that will test daily dosing of narmafotinib with the chemotherapy drugs gemcitabine and Abraxane in newly diagnosed advanced pancreatic cancer patients.

A registration-enabling study is a pivotal clinical trial designed to produce the core evidence needed to support an application for approval to market a new drug. Its job is to show that a treatment is safe at the proposed dose, that it helps patients and that the data package is strong enough for regulators such as the US Food and Drug Administration or Australia’s Therapeutic Goods Administration to assess.

The study is expected to enrol 12 patients across two dose cohorts at three or four Australian sites, with patient enrolment planned for the fourth quarter of this year.

Amplia’s June quarterly also underlined why the company is pressing ahead. Mature ACCENT trial data presented at the American Association for Cancer Research showed favourable safety and encouraging efficacy for narmafotinib, with a 36 per cent overall response rate and a 70 per cent disease control rate, compared with 50 per cent for chemotherapy alone.

The data also showed median overall survival of 11.1 months — an improvement of more than two months — and progression-free survival of 7.7 months when narmafotinib was used with standard chemotherapy.

The company ended the June quarter with $24 million in cash, giving it a handy buffer as it pushes narmafotinib further into pancreatic cancer and prepares to test the same drug in a second oncology target.

That expansion comes through PRROSE, a phase two trial being run under an agreement with the Australia New Zealand Gynaecological Oncology Group (ANZGOG) to evaluate narmafotinib alongside standard chemotherapy in 15 to 20 patients with high-grade serous ovarian cancer.

For Amplia, the latest paper is not a clinical result — but it is another useful building block. And in a field where every extra piece of biological support counts, the company will no doubt be happy to bank more evidence as it heads toward the next stage of its pancreatic cancer program.

Is your ASX-listed company doing something interesting? Contact: mattbirney@bullsnbears.com.au