Results from its pivotal TN-10 teplizumab clinical study have Provention Bio thinking that their candidate might end up being the most significant therapeutic breakthrough for type 1 diabetes since the invention of insulin in 1921.
RED BANK, NJ—Biopharmaceutical company Provention Bio Inc. recently announced that extended follow-up data from the pivotal TN-10 teplizumab clinical study had the potential to be a game-changer for treating at-risk type 1 diabetes (T1D)—suggesting that it could possibly be the most significant therapy for T1D since the invention of insulin in 1921.
The new Phase 2 data, published in Science Translational Medicine, showed that just a single 14-day infusion course of new investigational drug, teplizumab (PRV-031), delayed insulin dependence and the onset of clinical disease in at-risk T1D patients by nearly three years.
More than 1.6 million Americans have T1D, an autoimmune disease caused by the destruction of beta cells. Currently, the standard of care for these patients is insulin.
Teplizumab is the first disease-modifying investigational drug with data showing an ongoing delay to insulin-dependent T1D, and researchers are observing closely to determine whether this observed delay could extend even further over time. This drug candidate is an anti-CD3 monoclonal antibody currently under review by the FDA for the delay and prevention of clinical T1D in individuals at risk for the disease, with a PDUFA date of July 2, 2021.
“These data give us great hope for the T1D community,” Eleanor L. Ramos, chief medical officer, Provention Bio told DDN. “The potential to delay the onset of clinical T1D and insulin-dependence could mean that patients have more time to learn how to manage their disease progression with their doctors. We believe any incremental increase in the delay increases the maturity level to understand how to handle the intense management of the disease. This is profoundly relevant for patients facing a lifetime of insulin dependency, glucose monitoring and lifestyle challenges to survive.”
Ramos noted that the company is exploring whether disease onset could be delayed even further, in addition to studying teplizumab in a Phase 3 study of newly diagnosed patients.
“We understand the urgency in fundamentally changing the progression of T1D in early-stage disease before clinically relevant beta cell loss occurs. We remain steadfast in our commitment to bring teplizumab to the T1D community as quickly as possible,” she added. “If approved by the FDA, we expect to launch teplizumab in the United States in the second half of 2021. With up to 2.3 million people at risk of T1D worldwide, we also believe there is also a significant global market opportunity for teplizumab.”
“One of our goals as a company is to address T1D across the continuum,” Ramos continued. “In addition to pursuing FDA approval of teplizumab for the delay or prevention of insulin-dependent T1D in at-risk (Stage 2) patients, we are also assessing teplizumab in children and adolescents recently diagnosed with T1D in the ongoing Phase 3 PROTECT study. We expect to complete enrollment in the PROTECT study in the second half of 2021. In the future, we also intend to study combinations of teplizumab with other agents and with beta cell transplantation, which could address established T1D disease.”
The TN-10 Study was conducted through the Type 1 Diabetes TrialNet, an international research collaboration aimed at discovering ways to delay or prevent T1D.
The median time to clinical T1D was approximately five years in teplizumab-treated patients, compared to slightly over two years in the placebo group, according to the study. At this median follow-up of 2.5 years, twice as many teplizumab-treated patients remained free of clinical T1D compared to patients in the placebo group, 50 percent vs. 22 percent, respectively, (HR=0.457 p=0.01).
“These data embolden our enthusiasm surrounding the potential impact teplizumab may have on the lives of T1D patients, families, and caregivers,” remarked Ashleigh Palmer, CEO and co-founder of Provention Bio.
Seventy-six patients were enrolled in the study—ages 8 to 49, with 72 percent under the age of 18—and randomized to receive a single course of either teplizumab or placebo. Patients were followed in a blinded fashion until 40 of them developed clinical-stage T1D, and then indefinitely after the analysis of the randomized period data.
The psychological impact of T1D is hard to quantify, but a diagnosis is life-altering, and regular monitoring and maintenance can be extremely stressful, according to researchers. T1D typically takes more than a decade off a person’s life, and life expectancy is reduced by 16 years on average for people diagnosed with T1D before the age of 10. Insulin is the current T1D treatment and is necessary to keep patients alive, but it is a constant effort for patients. No disease-modifying treatments for T1D are currently available.
More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab consistently demonstrated the ability to preserve beta-cell function, a measure of endogenous insulin production.
Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration.
Provention Bio Inc. https://proventionbio.com/