Amplia takes aim at billion-dollar pancreatic cancer drug market

For everyday punters looking at medical technology companies, the best opportunities often come from identifying a drug that makes other multi-billion-dollar treatments work better. That is the main idea behind Melbourne-based medical company Amplia Therapeutics.

While most cancer companies spend billions trying to make stronger, more toxic chemotherapies, Amplia is taking a different path, developing a “shield-breaking” treatment. By breaking down the protective walls around hard-to-treat solid tumours, especially pancreatic cancer, the company believes it can position itself as a vital partner for the world’s largest pharmaceutical companies.

Accelerating exactly that strategy and following feedback from the United States Food and Drug Administration (FDA), the company has launched a new Phase 2b study in pancreatic cancer. The trial will test a continuous daily dosing schedule across two potency levels in a bid to optimise the therapeutic performance of the company’s FAK protein-inhibiting drug, narmafotinib.

The trial will initially enrol 12 newly diagnosed patients split into two 6-patient cohorts, combined with a conventional schedule of gemcitabine and Abraxane, standard frontline chemotherapy drugs.

Patient enrolment will begin by Q4 2026 across 3 to 4 clinical sites in Australia, with safety, tolerability, and pharmacokinetic assessments slated for completion in Q2 2027.

The biotech junior’s lead drug candidate, narmafotinib, is designed to turn off a specific protein called Focal Adhesion Kinase (FAK). In aggressive tumours, cancer cells hijack the FAK protein, helping the tumour to grow, survive and resist treatment.

Pancreatic tumours are notoriously difficult to treat because they often surround themselves with a dense wall of scar-like tissue that acts as a biological fortress, blocking chemotherapy drugs and even the body’s immune system from penetrating the cancer core.

The FAK protein effectively works as a stress sensor inside the tumour. When chemotherapy attacks, FAK triggers surrounding cells to pump out even more fibrotic tissue, reinforcing the tumour’s defensive barrier and helping the cancer survive.

That protective shield can make it incredibly difficult to shrink tumours sufficiently for surgeons to perform potentially life-saving debulking surgery, one of the few treatment options that can improve long-term survival outcomes.

Amplia’s best-in-class FAK inhibitor narmafotinib is designed to dismantle that defence system by breaking down the fibrotic barrier around tumours. The strategy creates a clearer pathway for standard frontline chemotherapy drugs and the immune system to attack the cancer more effectively, potentially making tumours far more vulnerable to treatment.

Amplia’s quest for effective therapies to combat fibrous tumours started from a major strategic pivot in 2018. Founded in 2000 as Innate Immunotherapeutics, the company spent two decades developing therapies for multiple sclerosis.

However, faced with the harsh reality of slow progress in the MS field, the company made the decision to shift its focus towards targeted cancer drugs, which were starting to attract major interest from global investors.

In April 2018, the company bought private developer Amplia Therapeutics, gaining exclusive ownership of a promising group of FAK-inhibitor drugs -including narmafotinib – originally discovered by the Cancer Therapeutics CRC in Melbourne in 2007. By September of the same year, shareholders voted to change the name to Amplia Therapeutics, with the ASX ticker ATX.

To lead the company from laboratory testing to human clinical trials, Amplia hired veteran scientist Dr Christopher Burns as CEO and managing director. Chris has more than 30 years of pharmaceutical experience, having held top leadership roles at Pfizer and Cytopia.

Critically for investors looking for confidence in the company’s clinical pathway, Burns was also a co-inventor of momelotinib, a successful drug developed to treat the rare bone marrow cancer myelofibrosis. The therapy was ultimately acquired by pharmaceutical giant GSK before securing FDA approval, giving Amplia immediate technical credibility through Burns’ proven track record of turning laboratory discoveries into approved patient treatments.

Unlike older, historic drugs that caused severe heart or stomach side effects by accidentally hitting the wrong proteins, narmafotinib is highly precise.

Backing up the science, the standout result from Amplia’s clinical push so far has been its ACCENT Trial, a Phase 1b/2a human study combining narmafotinib with frontline pancreatic cancer drugs gemcitabine and abraxane.

When Amplia released the final data from this 64-patient study in March, the results thrashed historical industry averages across three major areas. Notably, the complete response rate hit 7.8% – 5 out of 64 patients saw detectable cancer signs completely disappear for more than two consecutive months – a vanishingly rare result in pancreatic cancer trials.

Adding to the good news, the objective response rate reached 35.9 per cent, proving deep, measurable reductions in tumour size across a wide group. Just as impressively, the trial’s median overall survival data showed patients lived for a median of 11.1 months, delivering a compelling 2.6-month or 30 per cent improvement on historic chemotherapy-only results, all without piling on additional toxic side effects.

In parallel with its Phase 2b trials on pancreatic cancer, the company has inked a deal with the Australia New Zealand Gynaecological Oncology Group (ANZGOG) to launch a new clinical study to evaluate its protein-inhibiting drug on ovarian cancer.

This study is actively recruiting 15 to 20 patients to test narmafotinib with standard paclitaxel chemotherapy for advanced, recurrent cases of the devastating disease.

Since the same FAK-driven scar wall protects advanced ovarian cancer as pancreatic cancer, success here may well prove narmafotinib can act as a universal platform to unlock many different types of stubborn solid tumours.

Separately, a massive multi-billion-dollar commercial opportunity has emerged from a new global wave of targeted cancer therapies known as KRAS inhibitors, with 64 separate treatments currently in development. The interest is enormous because mutations in the KRAS gene are believed to drive more than 90 per cent of all pancreatic cancers.

Proving the significant potential of KRAS inhibitors in the fight against cancer, US developer Revolution Medicines recently halted Phase 3 trials of its flagship drug, Daraxonrasib, early. The drug was found to almost double the overall survival for pancreatic cancer patients to an unprecedented 13.2 months, prompting the FDA to fast-track review pathways.

However, the new generation of KRAS inhibitors continues to slam into a major biological roadblock – drug resistance. When the KRAS pathway is blocked, cancer cells cleverly switch on an alternative survival mechanism that ramps up FAK levels, rebuilding the tumour’s protective shield and ultimately blunting the impact of the expensive therapies.

Rather than trying to compete in the crowded KRAS field, Amplia is carving out a potentially crucial supporting role for narmafotinib. By combining the drug with KRAS inhibitors, the company believes it can effectively slam shut the cancer’s escape hatch, preventing the FAK spike and leaving tumours exposed to attack.

The company unveiled the mechanism at a major oncology conference in Los Angeles in March, where laboratory studies showed narmafotinib successfully blocked the emergency survival pathways across pancreatic, lung and ovarian cancers.

To turn this into financial value, Amplia has expanded its research partnership with South Korean firm Next & Bio, testing narmafotinib alongside approved KRAS drugs on live cells taken from real pancreatic cancer patients. The outcomes will likely be keenly sought and may even place narmafotinib onto the watchlists of global pharmaceutical giants.

This corporate positioning is crucial as global interest in FAK technology reaches an all-time high. Although other clinical competitors, including China-based InxMed and US-based Verastem Oncology, are quickly muscling in on the fast-moving FAK inhibitor market, Amplia believes it has a distinct structural advantage. Narmafotinib was designed from scratch to be a highly selective binder that delivers deep shield dissolution without causing the dangerous side effects seen in older competitor molecules, giving it clear “best-in-class” potential.

The company is launching into its next clinical trial phase with a healthy A$27.9 million cash balance at the end of March and, with quarterly spending running at about A$3.5 million, the company says it is fully funded to continue advancing through the regulatory pathway.

The market appears to have already begun to take notice of Amplia, with its shares surging as much as 185 per cent on a single day following the release of the trial data in March.

Capitalising on this momentum, the company is pushing to uplist its US stock ticker to the prestigious US-based OTCQB Venture Market. This will remove compliance barriers, allowing North American institutional funds, healthcare family offices and everyday American retail investors to buy shares easily.

Amplia has the cash, it has the science, and it has the manufacturing locked down. Now, it is time for the global registration study to prove if this Melbourne-born player can permanently rewrite the multi-billion-dollar rules of cancer medicine and treatment.

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