Tzield Efficacy and Safety

By preserving beta-cell function, Tzield significantly delays the onset of Stage 3 by median ~5 years (vs 2yrs placebo), with some subjects exceeding 7.5 years without symptoms.¹

Stage 2 Study: A significant delay was observed in the Tzield treated group compared to placebo in the onset of clinical T1D 

Primary analysis with median 2 years of follow-up

Tzield’s ability to delay onset of Stage 3 is linked to near-term improvements in quality of life and long-term morbidity and mortality benefits¹

^*Estimates and p-value are based on an MMRM model with treatment group, visit, age group at randomization, screening peak C-peptide category, baseline score, and a treatment by visit interaction term as fixed effects. Least-squares mean difference = Tzield – placebo. PedsQL scores range from 0 to 100 where a higher score indicates a better outcome. Minimal clinically important difference scores in PedsQL were 5.27 for child/teen and 4.54 for parents.11 HFS scores range from 0 to 4 where a lower score indicates a better outcome. Signs have been adjusted for graphical representation. DTSQ scores range from 0 to 48 where a higher score indicates a better outcome. CI, confidence interval; DTSQ, Diabetes Treatment Satisfaction Questionnaire; HFS, Hypoglycemic Fear Survey; LS, least squares; PedsQL, Pediatric Quality of Life Inventory.

Quality of Life²

Tzield demonstrated statistically significant improvement in perceived diabetes control score and treatment satisfaction via DTSQ instrument used in TN-10 study 

• Perceived diabetes control scores in teens decreased from baseline to week 78 from 9.2 to 7.8 in the Tzield group and from 9.1 to 7.3 in the placebo group (LSM difference: 1.1; 95% Cl: 0.0, 2.1)
• Treatment satisfaction scores in parents increased from baseline to week 78 from 35.7 to 38.5 in the Tzield group and from 34.6 to 36.3 in the placebo groups (LSM difference: 2.4; 95% Cl: 0.3, 4.5)

The Tzield group experienced less decline in perceived diabetes control at Week 78 Parents indicated greater satisfaction with their teen's treatment at Week 78

Long-term safety data has been collected from over 1000 children & adults who have participated in clinical trials since 2001. Adverse events typically resolve over a short period of time.^1,2

•  Safety was consistent with previous experience; no new safety signals
• The most common adverse events leading to study drug discontinuation were protocol defined LFT elevations (Tzield 6.9%; placebo 2.7%) and cytokine release syndrome (CRS) (Tzield 1.8%; placebo 0%)
• Similar rates of SAEs observed in Tzield 5.5% (CRS 1.4%) and placebo 5.4% (infections 2.7%)
• EBV reactivation was detected in 8 Tzield treated patients and was asymptomatic in 6; there was one new case of EBV in each treatment group. 

All events resolved without antiviral treatment

Safety data from 791 teplizumab-treated participants (children & adults) across Stage 2 and Stage 3 autoimmune T1D clinical trials show that teplizumab has a favorable safety profile characterized by self-limited AEs.¹⁵

• Most AEs were Grade 1 (mild) or 2 (moderate) in severity and typically occurred during or immediately following the dosing period and resolved without intervention.¹⁵
• AEs leading to permanent discontinuation of teplizumab were reported in 14.3% and 3.7% of participants in the teplizumab and control groups, respectively, across five clinical trials.¹⁵

^*Data from study included in pooled efficacy analysis.^14 
†Data from study included in pooled efficacy and safety analyses.^12 
‡Study period was 2 years. A 7-year follow-up study was conducted at the conclusion of the clinical trial.^5,15 
§Terminated early due to futility on its primary endpoint.^15
║Terminated prematurely with 254 subjects (64% of the planned 400 subjects) enrolled after Protégé failed to demonstrate efficacy on the primary endpoint.^15
¶Data from study included in pooled safety analysis.¹⁴ 
These data from 791 teplizumab-treated individuals represent 1500 patient-years of follow-up, the most extensive dataset in autoimmune T1D disease modification with a single agent to date.¹⁵