Press release: TZIELD Phase 3 data presented at ISPAD shows potential to slow the progression of Stage 3 type 1 diabetes in newly diagnosed children and adolescents; full data simultaneously published in the NEJM

New data from TZIELD's (teplizumab-mzwv) PROTECT Phase 3 trial were presented on 18 Oct 2023 at the 49th Annual ISPAD Conference, in Rotterdam, The Netherlands. PROTECT studied the efficacy and safety of TZIELD, compared to placebo, to slow the loss of beta cells and preserve beta cell function as measured by C-peptide, in children and adolescents aged 8-17 years diagnosed in the preceding 6 weeks with Stage 3 autoimmune type 1 diabetes (T1D). The full data set has been simultaneously published in The New England Journal of Medicine. TZIELD met the study's primary endpoint, demonstrating superior beta cell preservation assessed by significantly slowing the decrease in mean C-peptide levels [area under the curve (AUC) after a 4-hour mixed meal tolerance test] at trial completion, compared to placebo. C-peptide is a biomarker for beta cell function. This significant difference indicates the potential of TZIELD to slow the progression of Stage 3 type 1 diabetes in this population. While the study's key secondary endpoints did not meet statistical significance, numerical trends favouring TZIELD were seen in relevant clinical parameters. On average, people on TZIELD required numerically fewer insulin units and had numerically higher time in range, compared to those on placebo. HbA1c reductions and the overall rates of clinically important low blood sugar (hypoglycaemic) events were similar among both study groups. The availability of the PROTECT data represents a key early milestone for Sanofi on TZIELD, following the acquisition of Provention Bio (a Sanofi Company) in Apr 2023. TZIELD is a strategic fit for Sanofi at the intersection of its growth in immune-mediated diseases and disease modifying therapies, and the company's expertise in diabetes. The PROTECT clinical trial was a randomized, double blind, placebo-controlled, multi-national trial. From baseline and through the trial's completion at 78 weeks, the following was observed for TZIELD vs placebo: Primary endpoint: Significantly less decrease in mean C-peptide levels [area under the concentration curve (AUC), following a 4-hour mixed-meal tolerance test (MMTT)]: difference in least-squares means (LSM) of 0.13 pmol/mL; (95% CI: 0.09, 0.17; P less than 0.001). 94.9% of participants in the TZIELD group maintained peak C-peptide levels at least 0.2 pmol/mL, compared with 79.2% of those who received the placebo (P less than 0.001). The safety results of the trial were consistent with previous data from TZIELD's currently approved FDA indication to delay the onset of Stage 3 type 1 diabetes in adults and children 8 years and older diagnosed with Stage 2 T1D, as well as other prior clinical studies with TZIELD. No new safety signals were identified. Adverse events of special interest (AESI) were prespecified and occurred in 29% of those on TZIELD vs 21.6% on placebo, the most frequent one being hypoglycaemia (TZIELD: 13.4%; placebo: 16.2%). Other common adverse events (AEs) were headache, nausea, rash, lymphopenia and vomiting. Serious adverse events (SAEs) were reported by 5.5% of participant who received TZIELD vs 5.4% on placebo; the most common SAEs were cytokine release syndrome. Original source: Sanofi, website: http://en.sanofi.com/, Copyright Sanofi 2023.
research and development; antidiabetics; teplizumab-mzwv; TZIELD; Provention Bio; [...]