Treatment with FT011 led to clinically meaningful improvements in physical disability and lung function for some scleroderma patients given the experimental therapy in a Phase 2 clinical trial. Christopher Denton, MD, a professor at University College London, shared the results at the American College of Rheumatology (ACR) Convergence 2023 annual meeting, in a presentation titled, “FT011 for the Treatment of Systemic Sclerosis. Results from a Phase II Study.” “The Phase 2 trial demonstrates promising efficacy and safety data for FT011 after 12 weeks of treatment and certainly warrants a furth ..read more

The Scleroderma Research Foundation’s (SRF) annual “Cool Comedy – Hot Cuisine, A Tribute to Bob Saget” event, a recent evening of levity to raise scleroderma research funds and awareness, and to honor a longtime supporter, garnered more than $1.2 million. Some 500 people attended the luminary-laden event, presented as part of the 19th Annual New York Comedy Festival, which featured top names in comedy and music to raise funds for the SRF, according to a press release from the foundation. The event also celebrated the life of the late Bob Saget, a “relentless champion” for those affected by sc ..read more

Dosing has begun in a Phase 2 clinical trial evaluating aTyr Pharma’s efzofitimod, a first-in-class therapy for people with interstitial lung disease (ILD) associated with systemic sclerosis (SSc) — together known as SSc-ILD. Recruitment for the proof-of-concept EFZO-CONNECT study (NCT05892614), which aims to enroll up to 25 adult patients, is ongoing at sites in Ohio and Louisiana. Additional locations in California, Illinois, Oklahoma, South Carolina, and Texas are expected to open soon. “We are very pleased to begin patient dosing in EFZO-CONNECT,” Sanjay S. Shukla, MD, president and CEO o ..read more

Including intravenous immunoglobulin (IVIG) as part of routine care may help to ease muscle, skin, and digestive symptoms in people with systemic sclerosis (SSc), a Spanish study found. The experimental treatment was seen to be generally safe, with reported side effects being mostly mild or moderate. While these findings point to IVIG as a potential treatment option for scleroderma, “randomized clinical trials are required to assess the role of IVIG in the treatment of SSc manifestations,” its researchers wrote. The study, “Immunoglobulins in systemic sclerosis management. A large multicenter ..read more

Kyverna Therapeutics is launching a Phase 1/2 trial to evaluate its investigational cell therapy KYV-101 in adults with diffuse cutaneous systemic sclerosis (dcSSc). The move follows the therapy’s recent clearance of an investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA). The trial, called KYSA-5, is the third study of KYV-101 and joins the ongoing Phase 1 KYSA-1 trial in the U.S. and the Phase 1/2 KYSA-3 trial in Germany. Both are testing the therapy candidate for lupus nephritis, a lupus complication marked by kidney damage. “We are immensely proud ..read more

Boehringer Ingelheim has joined the CONQUEST platform trial, adding a second experimental medication to the multidrug study launched by the Scleroderma Research Foundation (SRF) to speed therapy development for interstitial lung disease (ILD) associated with scleroderma (SSc). In a platform trial, a master protocol is used to compare multiple treatment candidates against a shared control group. New medications can be added or discontinued over time, reducing trial costs and the time to complete clinical testing. These randomized clinical studies aim to identify candidates worthy of advancing ..read more

A prediction tool known as nomogram may be used to calculate the chance of a person with systemic sclerosis (SSc) and interstitial lung disease (ILD) developing progressive pulmonary fibrosis, or worsening lung scarring, a new study suggests. The nomogram essentially is a pictorial representation of a complex mathematical formula. Here, it crossed information about sex, blood levels of CXCL4 — a biomarker for ILD — and acid reflux to predict the likelihood of progressive pulmonary fibrosis in the two years following immunosuppressive treatment with mycophenolate mofetil or cyclophosphamide. I ..read more

Blood levels of interleukin (IL)-6, a pro-inflammatory signaling molecule, do not link with a risk of pulmonary involvement in people with systemic sclerosis (SSc), a study reports. As a result, IL-6 “could not be regarded as a potential therapeutic target,” the researchers wrote in the study, “Serum interleukin-6 level and its association with pulmonary involvement in progressive systemic sclerosis; a case-control study,” published in the journal Clinical and Molecular Allergy. “Due to the high prevalence of lung disease” in these people and diagnostic tests that can be difficult, “there is ..read more

Arsenic trioxide (ATO) lowered lung inflammation and disease-related alterations to blood vessels in the lungs in a mouse model of scleroderma (SSc), a study reports. “Our study provides compelling evidence of the positive effects of ATO treatment on lung function in a mouse model of SSc,” researchers wrote in “Arsenic trioxide demonstrates efficacy in a mouse model of preclinical systemic sclerosis,” which was published in Arthritis Research & Therapy. The work was funded by Medsenic, a part of the BioSenic group. The findings “will provide invaluable knowledge for BioSenic’s ATO ap ..read more

Antibodies designed to neutralize the protein galectin-3 (Gal-3) eased scarring and inflammation in a mouse model of systemic sclerosis (SSc), a recent study shows. Gene activity related to Gal-3 and the proteins it interacts with were found to be strongly associated with disease severity, lung and heart malfunction, and immune cell alterations in the blood of SSc patients. This, along with previous evidence implicating Gal-3, led the scientists to develop the therapeutic antibodies, which were also found in mice to counteract SSc-associated pathways and normalize abnormal gene activity patte ..read more