These people are related to 2 types of diabetes, which related to abnormal glucose (sugar) tolerance and autoantibodies (a protein made by an immune system). Subjects were randomised to receive either PRV-031 (teplizumab) or placebo.
Forty-four randomly assigned participants took teplizumab and 32 took a placebo.
In those who did progress to a diagnosis of type 1 diabetes, development was significantly delayed in those who received teplizumab compared with placebo, with a median time to diagnosis of 48.4 months versus 24.4 months, respectively.
Throughout the trial period, 72% of the people in the control group had thrived clinical diabetes with comparison to 43% of the teplizumab group. A Phase 3 study of teplizumab in diabetes patients with recent-onset disease failed in 2010, and Lilly handed back its rights to the drug to MacroGenics.
All participants regularly received glucose tolerance tests until the study was completed, or until they developed clinical Type 1 diabetes - whichever came first.
According to Dr. Kevan Herold, a professor of immunobiology and medicine at Yale, who presented the study in San Francisco, "every day you can delay this disease is important". With PRV-031 (teplizumab), we may now be able to intervene and fundamentally change the progression of T1D for these at-risk subjects.
"But it's particularly an issue for children, who represent about three-quarters of the participants in our study", he stated. "The rate of development of diabetes was reduced by half". "Just as we treat the asymptomatic presence of hypertension to prevent a heart attack or a stroke, these findings provide strong evidence we are approaching a future in which we can identify and treat type 1 diabetes long before symptoms occur".
It's at least a few years until this drug could possibly be approved for use outside of a clinical trial, said Jessica Dunne, senior director of research at JDRF (formerly the Juvenile Diabetes Research Foundation).
The TrialNet research has been funded by the US National Institutes of Health (NIH), primarily through the Special Diabetes Program, with additional support from JDRF. The researchers from Yale University involved 76 participants with the ages between 8 years and 49 years. A webcast presentation will also be available on the Investors page of the Company's website, www.proventionbio.com.
A drug that targets the immune system can delay type 1 diabetes for a median of two years in children and adults at high risk, according to findings from a mid-stage trial. The candidate has been the subject of multiple clinical studies involving more than 1,000 subjects with more than 800 patients receiving PRV-031 in those studies. In previous studies of newly diagnosed patients, PRV-031 has consistently demonstrated the capability of preserving beta cell function and reducing the need for exogenous insulin usage. Provention is now evaluating PRV-031 in patients with recent onset T1D (the Phase 3 PROTECT Study); additional information on the clinical trial is available at clinicaltrials.gov. Provention is also evaluating opportunities to advance development of teplizumab in relatives of T1D patients at risk for developing the disease. MacroGenics/Provention Bio donated the study drugs and provided funds for additional site monitoring.