Teplizumab Awarded Innovation Passport in the United Kingdom (UK) for the Delay of Onset of Clinical Type 1 Diabetes in At-risk Individuals

RED BANK, N.J., July 12, 2021 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced that teplizumab, an anti-CD3 monoclonal antibody (mAb), was awarded an Innovation Passport for the delay of clinical type 1 diabetes (T1D) in at-risk individuals. Teplizumab is one of the first investigational medicines to receive this designation under the United Kingdom's (UK) Innovative Licensing and Access Pathway (ILAP) launched by The Medicines and Healthcare products Regulatory Agency (MHRA) in January 2021.

In the UK, approximately 400,000 people have T1D, including about 39,000 children 19 years and younger¹. The UK has one of the highest rates of T1D globally, and new diagnoses are increasing by about 4% each year. T1D is managed through insulin therapy and glucose monitoring to keep patients alive but still can reduce life expectancy for patients by up to a decade. Currently, there is no approved disease-modifying treatment that targets the underlying cause of T1D.

Teplizumab is being developed for the delay of clinical T1D in at-risk individuals. In the pivotal TN-10 Study, a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with Stage 2 T1D compared to placebo. The observed adverse events were mechanism-based, transient, and predictable, including lymphopenia, transaminase elevations, rash, and cytokine release events.  These results were published in the New England Journal of Medicine and simultaneously presented at the American Diabetes Association meeting in 2019.

"My patients sometimes ask me if they can ever have a day without insulin. Teplizumab is the first treatment to potentially offer this, delaying the need for insulin by a median of 2-3 years following a single course in the TN-10 study," stated Colin Dayan, MA, MBBS, FRCP, PhD, Professor of Clinical Diabetes and Metabolism, Cardiff University School of Medicine. "For newly diagnosed children and adults, these are years when they can eat and enjoy playing sports just like their peers, free from the fear of hypoglycemia, while at the same time accruing the long-term benefits of excellent blood glucose control. We are very excited at the potential of having this drug available for treatment of patients before onset of T1D in the UK."

ILAP was launched at the start of 2021 to accelerate the development and access to promising medicines in the UK, thereby facilitating and improving patient access to new medicines. The pathway, part of the UK's plan to attract life sciences development in the post-Brexit era, features enhanced input and interactions with the MHRA and other stakeholders, including the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC). The decision to award the Innovation Passport to the teplizumab program was made by the ILAP Steering Group, which is comprised of representatives from MHRA, NICE, and SMC. The process is also supported by such bodies as the National Health Service (NHS) England, the NHS Improvement, Health Research Authority and the National Institute for Health Research.

To receive an Innovation Passport, an experimental drug needs to meet the following public health and/or patient-centric criteria: (1) the condition is life-threatening or seriously debilitating; (2) the program fulfills at least one of the following: innovative medicine, clinically significant new indication or, it is intended for a special population; and (3) the medicine has the potential to offer benefits to patients. The Innovation Passport designation is the first step in the ILAP process. It triggers the MHRA and its partner agencies to create a target development profile to chart out a roadmap for regulatory and development milestones with the goal of early patient access in the UK. Other benefits of ILAP include a 150-day accelerated assessment, rolling review, and a continuous benefit-risk assessment.

"There have been no new therapeutic innovations in T1D since the development of insulin 100 years ago, despite the significant life-long burden of the disease and steady increase of new diagnoses," said Ashleigh Palmer, co-founder and CEO of Provention Bio. "We are pleased to be developing one of the first medicines granted an Innovation Passport. We look forward to working closely with the MHRA and the other partner agencies involved in the ILAP process to advance teplizumab towards being a therapeutic option for patients at risk of developing clinical-stage T1D in the UK."

The Unmet Need in Type 1 Diabetes in the United Kingdom: 

Approximately 400,000 people in the United Kingdom have type 1 diabetes (T1D), an autoimmune disease caused by the destruction of beta cells. Diagnosis of T1D usually occurs in children and young adults, but it can happen at any age after symptoms appear when a person cannot make enough insulin. However, T1D starts in the body long before any symptoms and can be detected through a blood test. The psychological impact of T1D is hard to quantify, but a diagnosis is life-altering, and regular monitoring and maintenance can be extremely stressful. 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 before the age of 10. Insulin therapy and glucose monitoring are currently the standard of care for treating clinical-stage T1D and are necessary to keep T1D patients alive. The constant monitoring and administration of insulin represents a significant life-long burden for patients. No disease-modifying treatments for T1D are currently available.

About Teplizumab (PRV-031):

Teplizumab is an investigational anti-CD3 monoclonal antibody (mAb) being developed for the delay of clinical type 1 diabetes (T1D) in at-risk individuals. In the pivotal TN-10 Study, a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with Stage 2 T1D compared to placebo. The observed adverse events were mechanism-based, transient, and predictable, including lymphopenia, transaminase elevations, rash, and cytokine release events.  These results were published in the New England Journal of Medicine and simultaneously presented at the American Diabetes Association meeting in 2019.  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 as shown by C-peptide, a measure of endogenous insulin production. It correspondingly reduced the need for insulin use. Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration. Provention is currently also evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT study).

About Provention Bio, Inc.:

Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated disease. The Company's pipeline includes clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in autoimmune diseases, including type 1 diabetes, celiac disease and lupus. Visit www.ProventionBio.com for more information and follow us on Twitter: @ProventionBio.

Internet Posting of Information:

Provention Bio, Inc. uses its website, www.proventionbio.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation F.D. Such disclosures will be included on the Company's website in the "News" section. Accordingly, investors should monitor this portion of the Company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

Forward-Looking Statements:  

Certain statements in this press release are forward-looking, including but not limited to, statements relating to the medical need in T1D at-risk patients, the potential therapeutic effects and safety of teplizumab in at-risk T1D patients, and the advancement of teplizumab towards being a therapeutic option for patients at risk of developing clinical-stage T1D in the UK. These statements may be identified by the use of forward-looking words such as "will," "potentially" and "may," among others. These forward-looking statements are based on the Company's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to delays in or failure to obtain FDA, MHRA or other applicable approvals for teplizumab or other Company product candidates and the potential for noncompliance with FDA, MHRA or other applicable regulations; any inability to successfully work with FDA to find a satisfactory solution to address its concerns in a timely manner or at all, including any inability to provide the FDA with PK/PD data from our ongoing Phase 3 PROTECT study or other data sufficient to support an approval of the BLA for teplizumab; an inability to satisfactorily address other matters cited in the complete response letter received from the FDA, including those relating to product quality, fill/finish manufacturer deficiencies identified in a general inspection, the safety update required by FDA or any other FDA requirements for an approval of teplizumab; the potential impacts of COVID-19 on our business and financial results; changes in law, regulations, or interpretations and enforcement of regulatory guidance;  uncertainties of patent protection and litigation; the Company's dependence upon third parties; substantial competition; the Company's need for additional financing and the risks listed under "Risk Factors" in the Company's quarterly report on Form 10-Q for the quarter ended March 31, 2021 and any subsequent filings with the Securities and Exchange Commission. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Provention does not undertake an obligation to update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law. The information set forth herein speaks only as of the date hereof.

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¹ International Diabetes Foundation (IDF) Atlas 9^th Edition 2019, https://www.diabetesatlas.org/en

SOURCE Provention Bio, Inc.