CI Scientists Remarks: 

• In 2010, Teplizumab failed a pivotal late-stage diabetes study sponsored by Eli Lilly and the drug maker returned the rights to MacroGenics. In 2018, Provention Bio in-licensed the drug from MacroGenics and used it as their lead drug
• Since then, Teplizumab has received breakthrough therapy and now priority review from U.S. FDA for delay or prevention in T1D
• EMA has also granted priority medicines (“PRIME”) eligibility to Teplizumab for the delay or prevention of clinical T1D
• Provention Bio initiated rolling NDA submission in Apr’20 and completed in Nov’20 based on results from Phase 2 TN-10 study
• Results from Pivotal TN-10 study (Teplizumab: N= 44, placebo: N=32)
  • Teplizumab clinical T1D delay = ~3 years
  • Median time to clinical T1D diagnosis: 59.6 months (Teplizumab) vs 24.4 months (placebo)
  • Single 2 week course of Teplizumab significantly delayed the clinical onset of T1D by 35.2 months
  • T1D diagnosis after treatment: 43% of the Teplizumab-treated participants vs 72% of the placebo-treated
  • C-peptide levels: Reversed/increased (p=.02) upon Teplizumab treatment; placebo-treated subjects kept declining (p=.002 for Teplizumab vs placebo)
• US KOL said, ‘the only thing keeping us from [screening] the population at large is that we don’t have an effective prevention therapy’. Teplizumab has demonstrated a disease-modifying effect in clinical trials and, as it is a prevention therapy, it will encourage early screening, and thus, earlier diagnosis.
  • Commercial preparations:
    • The company has recently hired market access, sales, analytics and forecasting, medical affairs, and regulatory affair teams
    • Provention Bio has a services agreement with AGC Biologics to manufacture and supply Teplizumab
  • Teplizumab Life cycle management:
    • Phase 3 PROTECT trial in newly diagnosed T1D patients is on-going
    • The company also plans to expand Teplizumab development in Psoriatic arthritis, Rheumatoid arthritis, Crohn’s disease, Celiac disease, Autoimmune hepatitis