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Type 1 diabetes prevention promise sends Provention Bio soaring

Remarkable results prompt firm to consider regulatory pathway

Provention Bio

Many patients who are at risk of developing type 1 diabetes could have their disease onset delayed or even prevented by a new treatment, in what could be a remarkable breakthrough.

The share price of Provention Bio doubled yesterday after it released data which showed its immunotherapy PRV-031 (teplizumab) could delay the onset of of type 1 diabetes in children and adults by a median of two years.

Shares in the US biotech rose 217% to $13.79 thanks to the phase 2 trial in 76 patients at risk of developing Type 1 diabetes (T1D) who were given PRV-031 significantly delayed the onset and diagnosis of Type 1 diabetes compared to placebo, by a median of 2 years in children and adults considered to be at high risk.

The results were even more impressive for a subgroup of patients, around 60%, who did not develop the condition at all in the study period.

The US biotech firm was established just three years ago, and acquired rights to teplizumab from MacroGenics, which suspended its development of the drug in 2010 after a clinical trial run with Lilly showed it failed to show sufficient efficacy in T1D patients.

Jeffrey Bluestone, a distinguished diabetes researcher and a director of Provention Bio had worked on the drug for over 30 years, and was instrumental in in-licensing.

Provention Bio’s approach is to intercept the T1D process at an early stage of the autoimmune condition, which involves a set of autoantibodies being created in the body which eventually destroy all the insulin cells in the pancreas.

Teplizumab works by binding to the pro-inflammatory T cell co-receptor CD3, which in turn prevents them from activating the T cells that attack and kill the pancreas’ beta cells.

The "At Risk" Study enrolled 76 participants ages 8 to 49 who had two or more T1D autoantibodies and abnormal glucose metabolism (dysglycaemia); 72% of participants were under the age of 18.  Subjects were randomised to receive either PRV-031 (teplizumab) or placebo.

Results showed that a single 14-day course of PRV-031 delayed onset and diagnosis of clinical T1D by a median of 2 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.

In comparison, the median time for PRV-031 (teplizumab)-treated participants to clinical diagnosis of T1D was just over 48 months. During the trial, 72% in the placebo group developed clinical diabetes compared to only 43% of the PRV-031 group, and the drug was well tolerated with no new safety signals.

"This groundbreaking study demonstrates that we can use immunotherapy, specifically PRV-031 (teplizumab), to prevent or significantly delay the onset of clinical type 1 diabetes by at least two years in individuals who will almost certainly progress to clinical disease," said Dr. Eleanor Ramos, Provention's chief medical officer and chief operating officer.

"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."

Dr. Kevan Herold, M.D., professor of Immunobiology and Medicine at Yale University, lead author of the study, said: “Delaying the onset of clinical T1D may mean the disease burden could be postponed to a point at which patients are better able to manage their disease such as after infancy, elementary school, high school or even college. With PRV-031 (teplizumab), we may now be able to intervene and fundamentally change the progression of T1D for these at-risk subjects.”

He added that patients will be observed closely in the follow-up period, with the hope that at least some patients could be permanently free of developing the T1D, and those who have seen it delayed also benefitting.

The company recently initiated a phase 3 trial in patients with T1D, with enrolment to be completed in 2020, and complettion scheduled for May 2022.

Provention has also seized the opportunity to raise capital by announcing an underwritten public offering of 5.5m shares.

Jeffrey Bluestone

Jeffrey Bluestone

Jeffrey Bluestone PhD, A.W. is Mary Margaret Clausen distinguished professor of metabolism and endocrinology at the UC San Francisco (UCSF) Diabetes Center, president, CEO of the Parker Institute for Cancer Immunotherapy, and a director of Provention Bio.

His decision to keep faith in the drug looks to have paid off.

“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."

The company is now investigating a regulatory path for PRV-031 in at-risk individuals, and is also assessing the treatment in newly-diagnosed T1D patients in our Phase 3 PROTECT study.

Article by
Andrew McConaghie

20th June 2019

From: Healthcare



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