Dinesh Khanna, MD, Professor of Rheumatology and Medicine at the University of Michigan, discusses Horizon Therapeutics’ phase 2b clinical trial of HZN-825, to treat patients with diffuse cutaneous systemic sclerosis (dcSSc).
dcSSc is a subtype of systemic scleroderma (also known as systemic sclerosis) that is characterized by fibrosis and affects multiple body systems. The disease can occur at any age but most often affects women between 30 and 50 years of age. Symptoms include Raynaud’s phenomenon; skin fibrosis beginning on the fingers and face that rapidly becomes generalized; telangiectasias on the thorax, face, lips, tongue, and fingers; gastroesophageal reflux; and dysphagia along with weight loss, vomiting, diarrhea or constipation. Dry mouth and dental involvement can occur. Joint pain, muscular pain, weakness, cramps, and destruction of the tips of the fingers or toes are frequent. Severe lung problems (e.g., pulmonary arterial hypertension, interstitial lung disease) may occur, as well as kidney problems. The exact cause of the condition is unknown. There is currently no approved targeted therapy for dcSSc. As such, management of these patients is dependent on specific symptoms rather than prevention.
Horizon Therapeutics recently recruited and randomized the first patient in their phase 2b clinical trial of HZN-825, an oral selective LPAR1 antagonist. As Dr. Khanna explains, this trial was inspired by encouraging efficacy and safety data in a Phase 2a trial in patients with early dcSSc.
As Dr. Khanna notes, approximately 300 subjects who meet the trial eligibility criteria will be randomized in a 1:1:1 ratio to receive HZN-825 at 300 mg once daily, HZN-825 at 300 mg twice daily, or placebo for 52 weeks. The primary endpoint is change in forced vital capacity (FVC) percent after 52 weeks of treatment. The secondary endpoints include the Health Assessment Questionnaire‐Disability Index, or HAQ-DI, and other patient and physician-reported outcome measures used to evaluate the efficacy of the medicine. All subjects who complete the 52-week study will be eligible to enter a 52-week extension trial.
Data from this trial is estimated to be available late 2024/early 2025.
To learn more about dcSSc and other autoimmune conditions, visit checkrare.com/diseases/autoimmune/
