Phase 2b clinical trial planned after earlier results showed lung function gains
by Lindsey Shapiro, PhD | September 24, 2024

FT011, Certa Therapeutics’ oral treatment candidate for systemic sclerosis (SSc), will now be known under the generic name asengeprast.

That decision was made by the World Health Organization (WHO), which is responsible for assigning International Non-Proprietary Names, or INNs, to pharmaceutical substances or active ingredients. INNs are globally recognized, unique identifiers for such substances, and may be understood as the generic name for an active ingredient in a medication. INNs are public property and aren’t owned by any particular company.

“The granting of an INN for our lead drug candidate asengeprast is another important step in [the] development of this important therapy,” Darren Kelly, PhD, CEO, founder, and managing director of Certa, said in a company press release. “​​We are continuing to drive the clinical development of asengeprast and believe it has the potential to address a critical need for people living with SSc, a debilitating condition with the highest mortality amongst rheumatic diseases.” Kelly is also a professor at the University of Melbourne in Australia.

The company plans to launch a confirmatory Phase 2b clinical trial with SSc patients after positive data from a Phase 2a clinical trial (NCT04647890) showed the treatment eased disease severity and improved lung function. It’s also working to develop biomarkers and gene signatures for identifying patients most likely to respond to the treatment.
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In SSc, the immune system launches harmful self-reactive attacks that cause fibrosis, or excessive scarring of the skin and internal organs.

Outcomes differ for patients of different races, meaning antibodies play a role
by Marisa Wexler, MS | September 17, 2024

Some types of disease-specific antibodies are more common in Black patients with scleroderma than white patients, a study shows.

Differences in antibody profiles may help explain why clinical outcomes tend to differ for patients of different races, but the data suggest these variations can’t fully account for all the clinical differences between races, meaning other factors must also factor in.

“Distinct autoantibody types are enriched in Black [scleroderma] patients compared with those observed in white patients; these specificities associate with more aggressive clinical manifestations of the disease. However, differences in autoantibody distributions explain only a fraction of the racial effects on clinical outcomes,” the researchers wrote.

The study, “Racial variability in immune responses only partially explains differential systemic sclerosis disease severity,” was published in the Annals of the Rheumatic Diseases.
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Scleroderma, also called systemic sclerosis, or SSc, is marked by uncontrolled fibrosis (scarring) that can affect various organs. Several specific types of autoantibodies, that is, antibodies that attack healthy tissue and are thought to be a main driver of the disease, have been linked to the disease. The biological consequences of individual autoantibody types are poorly understood, however.

Calcium channel blocker seen to ease SSc symptoms such as Raynaud's
by Margarida Maia, PhD | September 10, 2024

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Aisa Pharma’s Profervia (cilnidipine), an investigational oral calcium channel blocker also known as AISA-021, for the treatment of systemic sclerosis (SSc).

In its request package for the designation, the company included data from an ongoing Phase 2 clinical study called RECONNOITER (ACTRN12621000459820) in which Profervia is showing benefits in easing symptoms of SSc. Among the symptoms reduced is Raynaud’s phenomenon, which causes pain and tingling in the hands and fingers.

“We hope this designation will accelerate our development program for AISA-021, which is designed to provide a once-daily, well-tolerated, and economical treatment that we hope can improve the lives of patients with SSc,” Andrew Sternlicht, MD, Aisa’s CEO and founder, said in a company press release.
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The granting of orphan drug status provides an incentive for companies to develop products for rare diseases. The designation’s benefits include tax credits and a potential seven years of market exclusivity should the treatment ultimately be approved. According to Sternlicht, the company believes this is “the first time” a calcium channel blocker has been granted such a designation for an autoimmune disease.