OLDWICK, N.J., Oct. 22, 2019 /PRNewswire/ -- Provention Bio,
Inc. (Nasdaq: PRVB), a clinical stage biopharmaceutical company
dedicated to intercepting and preventing immune-mediated disease,
today announced top-line results from its randomized,
placebo-controlled Phase 2a PRINCE clinical trial evaluating
twice-daily PRV-6527, an oral Colony Stimulating Factor-1 Receptor
(CSF-1R) small molecule inhibitor.
The PRINCE study enrolled 93 moderate-to-severe Crohn's disease
patients who were either naïve to biologic therapy (~70%) or who
had previously failed at least one biologic drug (~30%). The
primary efficacy endpoint of the study was the change in the
Crohn's Disease Activity Index (CDAI) score at week 12. While
PRV-6527 demonstrated a substantial improvement in this symptom
driven score at week 12, it did not differentiate from placebo.
This high placebo response is deemed to be related to the
background medication used (~85%) in the study's predominantly
biologic-naïve population.
PRV-6527 was associated with improvements in several key
secondary objective endpoints in the steroid-free population (75%
of study subjects), including mucosal endoscopy (as assessed by the
Simple Endoscopic Score for Crohn's Disease, SES-CD) and tissue
histology (as measured by the Global Histological Activity Score,
GHAS). Analysis of exploratory serum and tissue biomarkers showed
that patients treated with PRV-6527 had significant reductions in
circulatory inflammatory monocytes, as well as macrophages,
dendritic cells and the CSF1 gene signature in colonic tissue,
providing proof of mechanism in the interception of inflammatory
myeloid cells.
PRV-6527 was found to be generally safe and well tolerated, with
no drug-related serious adverse events.
"Despite the PRINCE study's high placebo effect, single dose
level, limited 12-week duration, and relatively small sample
size, the initial top-line results highlight how PRV-6527's
CSF-1R inhibition intercepts the migration of inflammatory cells to
the gut. This rapid go/no-go, signal finding trial indicates a
potential role for PRV-6527 as a well-tolerated, oral, first-line
therapy in early Crohn's and, potentially, for the prevention of
relapse," stated Ashleigh Palmer,
CEO of Provention Bio. "Under the terms of our in- license
agreement, Janssen now has 90 days to exercise its option to
re-acquire this asset to support further development in
inflammatory bowel disease. Meanwhile, we will continue to devote
all of our efforts to advancing the rest of our portfolio,
especially our lead Breakthrough Therapy program PRV-031
(teplizumab) for the prevention or delay of type 1 diabetes."
Pursuant to its licensing agreement with Provention, Janssen has
the right to buy back PRV-6527 for a one-time payment of
$50 million and single-digit
royalties on future net sales. In the event that Janssen does not
take back PRV-6527, Provention is free to sublicence the program on
a worldwide basis to another partner in the field of inflammatory
bowel disease.
Conference Call and Webcast Information
Provention Bio
will discuss these results via conference call on Tuesday,
October 22, 2019 at 8:30 am ET. A webcast presentation
will also be available on the Investors page of the Company's
website, www.proventionbio.com. To access the call,
please dial 1-877-870-4263 (domestic) or 1-412-317-0790
(international) five minutes prior to the start time and ask to be
connected to the "Provention Bio Call". A webcast replay of the
call will be available beginning at 10:30 am ET on the
day of the call.
About PRV-6527
PRV-6527 is a highly potent and
selective small-molecule oral inhibitor of CSF-1R. It was initially
developed by Janssen Pharmaceuticals and has been evaluated in over
200 subjects to date. Proof of mechanism was demonstrated based on
inhibition of CSF-1R signaling and myeloid cell counts in blood.
CSF-1 binds to its receptor (CSF-1R) on myeloid cells and drives
the differentiation and maturation of these cells into inflammatory
dendritic cells and macrophages, which then populate the gut and
other tissues. In the gut, these differentiated myeloid cells
present antigens from intestinal bacteria (the microbiome) to white
blood cells and trigger inflammatory processes.
About Crohn's Disease
Crohn's disease is a chronic,
immune-mediated inflammatory bowel disease (IBD) characterized by
inflammation of the gastrointestinal (GI) tract. Myeloid
cells, a species of antigen-presenting cells, are believed to play
a central role in Crohn's disease by presenting microbiome antigens
to white blood cells in the gut. CSF-1R drives myeloid cell
differentiation in the bone marrow resulting in the maturation of
inflammatory dendritic cells and macrophages, which then populate
the gut and other tissues and trigger inflammatory processes.
About Provention Bio, Inc.
Provention Bio, Inc.
(Nasdaq: PRVB) is a clinical-stage biopharmaceutical company
leveraging a transformational drug development strategy that is
focused on the prevention or interception of immune-mediated
disease. Provention's mission is to in-license, transform
and develop therapeutic candidates targeting the high morbidity,
mortality and escalating costs of autoimmune and inflammatory
diseases including: type 1 diabetes (T1D), Crohn's disease, celiac
disease, and lupus. Provention's diversified portfolio includes
advanced-stage product development candidates that have undergone
clinical testing by other companies.
Forward Looking Statements
Certain statements in this
press release are forward-looking within the meaning of the Private
Securities Litigation Reform Act of 1995. These statements may be
identified by the use of forward-looking words such as
"anticipate," "believe," "forecast," "estimate," "expect," and
"intend," among others. These forward-looking statements are based
on Provention'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 failure to obtain FDA approvals or
clearances and noncompliance with FDA regulations; uncertainties of
patent protection and litigation; limited research and development
efforts and dependence upon third parties; substantial competition;
our need for additional financing and the risks listed under "Risk
factors" in our annual report on Form 10-K for the year
ended December 31, 2018 and any subsequent filings with
the Securities and Exchange Commission (SEC). 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. The information set forth
herein speaks only as of the date hereof.
Investors:
Sam Martin, Argot Partners
sam@argotpartners.com
212-600-1902
Media:
David Rosen, Argot Partners
david.rosen@argotpartners.com
212-600-1902
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SOURCE Provention Bio, Inc.