Canada-based ProMetic Life Sciences, a global biopharma, recently announced it will launch a double-blind, placebo-controlled Phase 2 clinical trial in patients with scleroderma to evaluate its lead product PBI-4050.
Drug candidate PBI-4050, taken orally, has confirmed safety and efficacy profiles through a series of in vivo studies in fibrosis. Fibrosis is a process where continuing inflammation leads to loss of function in vital organs, as healthy tissue is progressively replaced by fibrotic (scarred) tissue. PBI-4050’s proof of concept data confirms the compound’s anti-fibrotic action in a number of vital organs, including the heart, lungs, kidneys, and liver.
“PBI-4050 has been shown to significantly reduce fibrosis in several of the key organs in various preclinical models, irrespective of the cause of the injuries or whether acute or chronic in nature,” ProMetic’s vice president of preclinical research and development, Dr. Lyne Gagnon, said in a press release. “The most recent results have shown that even in mice genetically programmed to develop scleroderma, PBI-4050 prevented the overproduction of collagen and the formation of fibrotic scarring.”
“ProMetic is focused on and committed to targeting unmet medical needs where fibrosis is the underlying process leading to the medical complications,” said ProMetic President and CEO Pierre Laurin. “We are merely leveraging PBI-4050’s proven ability to downregulate the excessive production of scarring tissue, which in the case of systemic scleroderma, is often life threatening.”
Scleroderma is a chronic condition characterized by an abnormal growth of connective tissue and an overproduction of collagen, which causes scarring (fibrosis) of the skin and internal organs. From the Greek sclerosis – meaning hardness – and derma – meaning skin – the condition literally means “hard skin.” It’s also commonly called systemic sclerosis. It is a progressive disease and its most characteristic feature is the hard and thick skin, with lesions in small blood vessels.
“This accumulated data is very promising for patients affected by scleroderma in that, even though we do not understand the cause or causes of their condition, PBI-4050 addresses the underlying pathological process leading to the scarring of tissues and organs. This double-blind, placebo-controlled, Phase 2 clinical trial will investigate whether PBI-4050 can prevent or even reverse fibrosis in the skin and key target organs such as the lungs,” said ProMetic Chief Medical Officer Dr. John Moran.