Amplia Therapeutics receives FDA orphan drug designation for pancreatic cancer

Amplia Therapeutics ASX ATX FDA Amplia orphan drug designation pancreatic cancer
Amplia plans to initiate its first phase two clinical study of AMP945 in cancer patients in 2021.

Pharmaceutical company Amplia Therapeutics (ASX: ATX) has received regulatory approval from the US Food and Drug Administration (FDA) for its Focal Adhesion Kinase inhibitor (FAKi) AMP945 – a novel new treatment for pancreatic cancer.

The company said the drug is a “pure play” inhibitor with superior specificity and will potentially make progress into stage two trials next year.

More broadly, Amplia is advancing a pipeline of FAK inhibitors for cancer and fibrosis. FAK is an increasingly important target in the field of cancer immunology and the company has a particular development focus in pancreatic and ovarian cancer.

Earlier today, Amplia told the market the FDA has awarded what’s known as an ‘orphan drug designation’ for AMP945, which effectively means the pharma company will qualify for fee exemptions, clinical trial protocol assistance and a raft of other incentives to assist progress with the new drug.

Orphan drugs are typically developed to treat rare medical conditions, referred to as ‘orphan diseases’, which means developing such drugs can be financially precarious without government and regulatory assistance.

If the FDA approves AMP945 for the treatment of pancreatic cancer, Amplia would also qualify for seven years of market exclusivity in FDA-administered markets.

Rare but commercially viable

Amplia said it intends to conduct a phase one clinical trial of AMP945 in healthy volunteers later this year to confirm the drug is well tolerated and safe.

If the trial is successful, the company plans to initiate its first phase two clinical study of AMP945 in cancer patients in 2021.

Amplia claims AMP945 has multiple modes of action that make it an appealing candidate for incorporation into treatment regimes for hard-to-treat solid fibrotic cancers such as pancreatic, ovarian, breast and lung.

Despite the recent success of immuno-oncology (I-O) drugs, their use in fibrotic cancers has been met with limited success.

Recent research shows that FAK is significantly upregulated in these highly fibrotic tumours and therefore, drugs targeting FAK have the potential to “sensitise” the tumour micro-environment to both I-O and chemotherapies.

Inhibiting FAK can also directly boost the number of immune cells responsible for attacking tumours, thereby further amplifying the effect of I-O treatment, according to Amplia.

“Pancreatic cancer is a seriously unmet medical need and FDA’s designation of AMP945 as an orphan-drug underlines global regulatory agencies’ interest in supporting novel treatments for this deadly disease,” Amplia Scientific Advisory Board member Professor Paul Timpson said.

Concerning scaling up its activities within FAK inhibitors, Amplia said drug manufacturing scale-up chemistry has already been developed in partnership with a leading international commercial contract manufacturer.

Furthermore, the production methods are suitable for GMP production of clinical trial material and due to the common synthetic route for both molecules, large-scale manufacturing should be “highly cost-effective”, according to Amplia.

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