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.
Investor Contacts:
Robert Doody, VP of Investor
Relations
rdoody@proventionbio.com
484-639-7235
Sam Martin, Argot Partners
sam@argotpartners.com
212-600-1902
Media Contact:
Lori Rosen, LDR Communications
lori@ldrcommunications.com
917-553-6808
1 International Diabetes Foundation (IDF) Atlas
9th Edition 2019, https://www.diabetesatlas.org/en
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SOURCE Provention Bio, Inc.