Trial data showed a two-week course delayed the onset of diabetes by as much as two years in clinical patients.
Shares of Provention Bio have soared more than 355% this morning after the New Jersey-based company announced its immunotherapy treatment to delay the onset of type 1 diabetes hit the mark in a clinical trial.
The mid-stage study, conducted by TrialNet, was aimed at developing a therapy that could delay or prevent the onset of type 1 diabetes. Trial results showed that a 14-day course of PRV-031 (teplizumab), anti-CD3 monoclonal antibody, significantly delayed the onset and diagnosis of clinical T1D, as compared to placebo, by a median of two years in children and adults considered to be at high risk. The median time to clinical diagnosis of T1D for placebo participants was just over 24 months, Provention said in its announcement. In comparison, the median time for PRV-031-treated participants to clinical diagnosis of T1D was just over 48 months. The trial included 76 participants ages 8 to 49 who were “at-risk” because they had two or more type 1 diabetes autoantibodies and abnormal glucose metabolism (dysglycemia).
During the trial, 72% of participants who took placebo developed clinical diabetes compared to only 43% of the PRV-031 group. PRV-031 was well tolerated and the safety data were consistent with prior studies in newly diagnosed patients.
Eleanor Ramos, chief medical officer and chief operating officer at Provention, said the trial results show immunotherapy can be used to prevent or delay the onset of clinical type 1 diabetes by at least two years in individuals who are nearly certain to develop the disease.
“More importantly, approximately 60% of subjects in the study did not develop T1D following only one course of PRV-031 therapy, double the placebo group. Teplizumab is the first immune modulator to show a delay in the clinical onset of type 1 diabetes,” Ramos said in a statement.
Provention Chief Executive Officer Ashleigh Palmer said the company is evaluating a path to seek regulatory approval of PRV-031 in at-risk individuals. Palmer expressed his delight at the results of the trial, which he said reinforces confidence not only in PRV-031, but also in Provention’s strategic intent to intercept and prevent immune-mediated disease. Provention is also assessing PRV-031 in newly-diagnosed T1D patients in the Phase III PROTECT study, which launched in April.
“The ability to delay the onset of clinical T1D is an enormous breakthrough, given that a recent study indicated the life expectancy for patients diagnosed with T1D before the age of ten is reduced by as much as 16 years on average,” Palmer said. “Our broader goal for PRV-031 is to address the continuum of T1D and provide therapeutic options for this life-impacting and life-threatening autoimmune disease that, until now, has seen no disease-modifying innovation since the development of insulin a century ago.”
Kevan Herold, a professor of immunobiology and medicine at Yale University and lead author of the study said delaying the onset of type 1 diabetes may mean the burden of the disease could be postponed until a time when a patient is better able to manage the disease, such as after infancy, or their formative years in school.
“With PRV-031 we may now be able to intervene and fundamentally change the progression of T1D for these at-risk subjects. In addition, we look forward to learning more as we observe patients during the study’s follow-up period, which will also evaluate the long-term outcomes for those in whom the diagnosis of disease has been delayed to see if they will be diagnosed with T1D or are protected,” Herold said in a statement.
