Date: 2018-05-17
Type of
information: Presentation of results at a congress
phase: 1-2
Announcement: presentation of results at the International Investigative Dermatology conference
Company: Syntimmune (USA - MA)
Product: SYNT001
Action
mechanism:
- monoclonal antibody. SYNT001 is an investigational humanized IgG4 monoclonal antibody optimized to inhibit FcRn binding to IgG at both neutral and acidic pH. Studies have shown that SYNT001 rapidly facilitates clearance of IgG and IgG circulating immune complexes (CICs), with the potential to block innate immune responses induced by IgG and CIC, as well as inhibit T cell and B cell activation in response to CIC. Additionally, studies suggest that SYNT001 accomplishes its effects on IgG without destroying immune cells or impacting other types of immunoglobulin. SYNT001 has the potential to exert a rapid therapeutic effect in a wide range of IgG-mediated autoimmune diseases.
Disease: pemphigus (vulgaris or foliaceus)
Therapeutic
area: Autoimmune diseases - Rare diseases
Country: USA
Trial
details:
- SYNT001-103 is a multi-center, open-label Phase 1b study evaluating the safety and clinical effect of SYNT001 in patients with active pemphigus vulgaris (PV) or pemphigus foliaceus (PF). SYNT001 will be studied in three successive dosing cohorts, each treated with five weekly doses of SYNT001 administered intravenously (IV), with dose escalation between cohorts up to a maximum of 45 mg/kg. The study observation period covers 16 weeks, including an active treatment period from day 0-28 and follow-up through day 112. The primary endpoint of the study is safety and tolerability. Secondary endpoints include PDAI, total IgG, CIC and serum anti-desmoglein IgG (anti-DSG1 and anti-DSG3) levels (NCT03075904)
Latest
news:
- • On May 17, 2018, Syntimmune announced positive preliminary results from its Phase 1b proof-of-concept trial of SYNT001 in patients with pemphigus vulgaris and pemphigus foliaceus. The data showed clinically meaningful benefit of SYNT001, with a favorable safety and tolerability profile similar to that observed in the Phase 1a study. These results have been presented by Donna Culton, M.D., Ph.D., an assistant professor at the University of North Carolina School of Medicine, at the International Investigative Dermatology conference.
- The results reported include data from seven patients (six with PV and one with PF) enrolled in the first cohort who received five weekly infusions of 10 mg/kg SYNT001 and are in varying stages of follow-up.
- Results from First Cohort: Primary endpoint analysis revealed SYNT001 to be well tolerated in treated patients, with all study drug-related adverse events (AEs) characterized as mild or moderate. No severe or serious study drug-related AEs were reported.
- The secondary endpoint measures showed a reduction in mean PDAI score from severe to moderate, with clinical effect persisting beyond the treatment period. Rapid and clinically meaningful reductions in pharmacodynamic biomarkers were observed in all patients. At nadir, mean total IgG levels were reduced by 59% (day 30), mean CIC levels were reduced by 50% (day 33), mean anti-DSG1 levels were reduced by 22% (day 14) and mean anti-DSG3 levels were reduced by 24% (day 33).
- • On November 1, 2017, Syntimmune announced that the company is advancing a multicenter, open-label, safety, tolerability, and activity Phase 1b/2a study of SYNT001 in subjects with pemphigus (vulgaris or foliaceus).
Is
general: Yes
