ProMetic’s Scleroderma Treatment PBI-4050 to Enter Phase 2 Clinical Trial

ProMetic’s Scleroderma Treatment PBI-4050 to Enter Phase 2 Clinical Trial

ProMetic Life Sciences  recently announced the initiation of a Phase 2 clinical trial on PBI-4050 for the treatment of scleroderma, a chronic connective tissue disease for which there is currently no cure.

PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles confirmed in several in vivo experiments targeting fibrosis. Fibrosis is a complex process where continued inflammation causes vital organs to lose their function, and normal tissue is replaced by fibrotic scar tissue.

“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,” Dr. Lyne Gagnon, ProMetic’s vice president of research and development, said in a press release. “The most recent results have shown that even in mice genetically programmed to develop scleroderma, PBI-4050 prevented the over production of collagen and the formation of fibrotic scarring.”

The 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,” said Dr. John Moran, chief medical officer of ProMetic, which is based in Quebec, Canada.

“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,” Moran said.

“ProMetic is focused on and committed to targeting unmet medical needs where fibrosis is the underlying process leading to the medical complications,” said Pierre Laurin, ProMetic’s president and CEO. “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 causes an overproduction of collagen and abnormal growth of connective tissue, leading to scarring (fibrosis) of the skin. Limited scleroderma involves cutaneous manifestations, which mainly affect the hands, arms, and face.

Diffuse scleroderma, on the other hand, affects a large area of the skin, and one or more internal organs, usually the kidneys, esophagus, heart, or lungs. This form of scleroderma can be quite disabling. Approximately 300,000 individuals in North America alone are affected by the disease.

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