Pharmaceutical company Amplia Therapeutics (ASX: ATX) has received a second orphan drug designation from the US Food and Drug Administration for its focal adhesion kinase inhibitor AMP945.
The designation has been awarded for the use of AMP945 in the treatment of the chronic lung disease known as idiopathic pulmonary fibrosis and means that Amplia will qualify for waived FDA fees, clinical trial protocol assistance and other incentives.
Furthermore, if Amplia secures US regulatory clearance for AMP945 for the treatment of idiopathic pulmonary fibrosis, the drug will qualify for seven years’ exclusivity in FDA-administered markets.
Phase 1 trial
Amplia managing director Dr John Lambert said the company would start a Phase 1 clinical trial of the drug in healthy volunteers later this year to confirm it is well tolerated.
“This designation provides further validation of the pipeline of opportunities we are putting in place for our proprietary focal adhesion kinase inhibitors,” he said.
“Phase 1 will provide the safety, pharmacokinetic and exploratory pharmacodynamic data to enable AMP945 to progress into Phase 2 clinical trials in cancer and idiopathic pulmonary fibrosis patients next year.”
No known cause
Idiopathic (or “no known cause”) pulmonary fibrosis is a rare disease which causes the tissue around the lungs’ air sacs (or alveoli) to become thick and scarred.
The scarring stiffens the lungs, which makes it increasingly difficult for a patient to breathe deeply and slows the delivery of oxygen into the bloodstream for transport to the rest of the body.
Idiopathic pulmonary fibrosis is estimated to affect over 130,000 people in the US and more than three million people worldwide.
The disease can have a significant impact on a patient’s quality of life with a median prognosis of up to five years if left untreated.
Pirfenidone and nintedanib are the only two drugs to date which have been approved for use but while they can slow progression of the disease by up to 50%, they are not able to permanently treat it.
In pre-clinical studies using the industry standard, chemically-induced lung fibrosis model, AMP945 was successfully able to reduce and reverse the formation of fibrotic (or hardened) tissue.
Modes of action
As Amplia’s lead drug candidate, AMP945 has multiple modes of action which make it suitable for incorporation into treatment regimes for challenging cancers such as those affecting the pancreas, ovaries, breasts and lungs.
In March, Amplia received its first orphan drug designation for AMP945 to treat pancreatic cancer.
The rapid review of its USFDA application at the time highlighted a recognised need for new therapeutic options to combat the deadly disease.
That same month, Amplia lodged an international patent application covering the preferred salt form of AMP945 which will be used in future clinical trials.
If granted, the application will provide AMP945 with additional patent coverage through to 2039.