Fibrosis can occur in many tissues of the body as a result of inflammation or damage. As a result, collagen build up occurs which can result in scarring of vital organs such as the lung, liver, skin, eye heart and kidney leading to irreparable damage and eventual organ failure. It has been estimated that fibrosis is prevalent in 45% of all diseases; and this condition represents a large unmet clinical need.
There is no clinically satisfactory therapeutic approach to fibrosis.
Lungs – Idiopathic Pulmonary Fibrosis (IPF)
A specific form of lung fibrosis is Idiopathic pulmonary fibrosis (IPF), which is a chronic and ultimately fatal disease, where tissue deep in the lungs becomes scarred, over time, resulting in a progressive decline in lung function and shortness of breath.
There is currently no cure for IPF with most people living only three to five years after diagnosis. The rate at which the disease progresses is highly variable, with some patients remaining stable for several years while others may deteriorate rapidly, 50% of patients die within two to three years of diagnosis.
IPF is rare, but still affects over 135, 000 people in the United States (US), with about 48,000 new cases being diagnosed annually. In the US 40,000 people die each year from IPF; the same mortality as breast cancer. In addition IPF affects about 100,000 people in Europe and 5,000 people in Australia. Because this condition affects less than 200,000 patients within the United States or Europe, any products or drugs addressing this indication are referred to as orphan drugs.
In 2017, AdAlta applied for and achieved what’s known as “Orphan Drug Designation” with the United States Food and Drug Administration (FDA.) Access to the Orphan Drug Designation scheme means that AdAlta gets increased access to the FDA, new drug application fee waivers, a potentially faster route to market and an additional seven years of exclusivity once the drug is on market.
An overview of IPF can be downloaded here, which includes a summary of AdAlta’s unique approach for the treatment of IPF with AD-214.
Respiratory Physician at The Alfred hospital, Dr Glen Westall spoke at AdAlta’s inaugural fibrosis investor briefing February 2017, where he provided an overview of IPF and currently available treatments. The full-length presentation is available on AdAlta’s website as well as a video highlight of the day.
Liver fibrosis is a major global problem driven by increasing obesity and diabetes. Major causes of liver fibrosis are chronic hepatitis B virus (HBV) and hepatitis C virus (HCV) infection along with the metabolic disorders non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH).
Kidney fibrosis may be caused when the kidneys stop working and eventually transplantation is required. Fibrosis may occur at any stage from chronic kidney disease (CKD) to end-stage renal disease (ESRD).
AdAlta is currently working with the Kolling Institute to investigate the anti-fibrotic effects of AD-114, for the treatment of kidney fibrosis, research that has recently been granted a $768,000 NHMRC grant. Learn more about the work AdAlta and the Kolling Institute are completing in kidney fibrosis through presentations from Professor Carol Pollock and Dr Muh Geot Wong at AdAlta’s special investor/analyst briefings.
Infection or inflammation of the eye results in impairment of visual function and can ultimately lead to fibrosis. Eye fibrosis diseases include diabetic retinopathy and age related macular degeneration (AMD).
AMD is the commonest cause of severe visual impairment in people over the age of 50 years in the developed world. It is estimated that in 2010, there were 1.023 million Australians and 2.07 million Americans with AMD and these figures are expected to double by 2050. In Australia, AMD is the most common cause of blindness contributing to 50% of all blindness.
Globally, diabetic retinopathy is one of the most significant causes of visual loss and a principal cause of impaired vision in patients aged between 25 and 74 years of age. According to the US National Institutes of Health National Eye Institute, it was estimated that there were 7.7 million Americans affected by diabetic retinopathy in 2010.
The numbers of people with these diseases is predicted to increase due to demographic ageing.
Optometrist and Professor at University of Melbourne, Professor Erica Fletcher spoke at AdAlta’s inaugural fibrosis investor briefing February 2017, where she provided an overview of eye fibrosis with a focus on wet-AMD and currently available treatments. The full-length presentation is available on AdAlta’s website as well as a video highlight of the day.
Cardiac fibrosis causes the thickening and loss of flexibility in the heart muscle, due to deposition of collagen, that eventually may lead to heart failure.
Scarring is a result of an imbalance in the production of collagen in a healing wound. Scarring may continue to thicken for up to six months or may overgrow the site of the wound, even after the wound has healed.
AdAlta has demonstrated that the lead candidate, i-body AD-214, has broad anti-fibrotic effects in the lung, eye, skin, kidney and the liver.