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.
Fibrotic diseases addressable market
IPF market is underserved today
– Two existing therapies generated US$4.3b in 2022
– They slow but do not halt progression and do not significantly extend life expectancy
– Their side effects result in 30-50% of patients discontinuing therapy after one year
IPF market is projected to grow
– 2% pa growth in prevalence
– 4-6% growth in market size
– US$5.1b market by 2029
– US$136,000 pa cost of treatment in US
Kidney Fibrosis
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).
Other Indications
Fibrosis can affect almost every organ system in the body, resulting in loss of organ function. This is believed to be a contributor to up to 45% of deaths in western countries. The biological target for AD-214 is also implicated in more than 23 different cancers where it may contribute to cancer proliferation and metastases.
AD-214 and its predecessor molecules have been studies in animal models of eye and liver fibrosis and cancer.