On 2 November, Provention Bio announced the completion of a rolling Biologic License Application (BLA) submission to the Food and Drug Administration (FDA) for teplizumab for the delay or prevention of type 1 diabetes (T1D) in at-risk individuals (individuals with relatives who have diagnosed T1D and / or presence of two or more T1D-related autoantibodies and dysglycemia). Teplizumab is an Fc receptor-nonbinding anti-CD3 monoclonal antibody that works to modulate the response of the T-lymphocytes that mediate the destruction of the insulin-producing beta cells in the islets of the pancreas. GlobalData expects that the approval of teplizumab will force the treatment paradigm of type 1 diabetes to shift by placing a greater emphasis on screening for T1D and by driving the T1D market toward precision medicine.

Teplizumab was granted breakthrough therapy designation in August 2019, and Provention has requested a priority review in conjunction with the completion of the final submission. The priority review designation indicates that the FDA will take action on the application within six months (compared to ten months under standard review). If approved by the FDA, teplizumab will be the first disease-modifying therapy for T1D and could be brought to the market by the mid-to-end of 2021.

Key opinion leaders (KOLs) have consistently stressed the considerably high unmet need for disease-modifying therapies. The potential approval for teplizumab is not likely to be a final treatment for T1D, but it will encourage larger players in the T1D market to focus on novel therapeutics and look further into addressing the underlying causes of T1D. In addition, another key unmet identified by KOLs is an earlier diagnosis as poor management of blood sugar levels during the early onset of T1D can advance the likelihood of life-threatening complications. Currently, patients with T1D are ultimately diagnosed once symptoms of T1D occur. By the time symptoms of T1D occur, more than 80%–90% of insulin-producing beta cells will have been destroyed, but evidence of beta-cell autoimmunity through the presence of autoantibodies would have been present for many years prior. A US KOL had previously said they thought ‘the only thing keeping us from [screening] the population at large is that we don’t have an effective prevention therapy’. Teplizumab is one of the first therapeutics to demonstrate a disease-modifying effect in clinical trials and, as it is a prevention therapy, will encourage early screening, and thus, earlier diagnosis.

Teplizumab has had a contentious development history with its first iteration of trials conducted by Eli Lilly and MacroGenics. In October 2010, Lilly and MacroGenics terminated their collaboration agreement after concluding that the primary efficacy endpoint of teplizumab’s Phase III Protégé trial was not met. Provention is claiming success where Lilly and MacroGenics failed by using the change in C-peptide as the primary endpoint, rather than HbA1c or insulin use. Phase II clinical data has shown that a two-week course of teplizumab delayed T1D progression by a median of three years in high-risk pre-symptomatic patients. In addition, teplizumab was shown to significantly reverse the decline of C-peptide levels, which suggests that teplizumab can not only delay the destruction of beta cells but also restore insulin production in dysfunctional beta cells.

While advancements have been made in our understanding of biomarkers and critical T1D genes, there is no routine pre-screening for T1D. According to the 2020 America Diabetes Association’s Standard of Care guidelines, there is currently a lack of accepted screening programmes, and screening typically occurs only in the setting of a clinical research study. There is no question that the prevalence of T1D is increasing, as in 2016, the diagnosed prevalent cases of T1D across the eight major markets (US, 5EU, Japan, Canada) were 3,396,254, and GlobalData expects that number to grow to 4,725,990 by 2026 at an annual growth rate of 3.92%. Increased screening and early treatments like teplizumab could be exactly what the market needs to drive preventative and patient-specific care in T1D.

Provention is currently enrolling a Phase III trial for Teplizumab in newly diagnosed T1D patients.

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