RED BANK, N.J., Jan. 28, 2021 /PRNewswire/ -- Provention
Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to
intercepting and preventing immune-mediated diseases, today
reported results from a pre-clinical proof-of-concept study for
PRV-3279, a DART® (bispecific antibody-based molecule)
targeting the B cell surface proteins CD32B and CD79B, conducted in
a murine model of gene therapy for Pompe disease. A PRV-3279 mouse
surrogate was tested in mice transgenic for human CD32B, which
received gene therapy with an adeno-associated virus (AAV) vector
AAV9 encoding for the enzyme acid-alpha-glucosidase (GAA)
gene. Errors in the GAA gene cause the serious human glycogen
storage disease type II (Pompe disease).
![(PRNewsfoto/Provention Bio, Inc.) (PRNewsfoto/Provention Bio, Inc.)](https://mma.prnewswire.com/media/720849/Provention_Bio_Logo.jpg)
In the study, the PRV-3279 surrogate reduced anti-AAV9 vector
antibody levels in a dose-dependent fashion. Anti-AAV9 antibodies
have been linked to reduced efficacy, safety concerns and the
inability to re-dose patients, and thus, based on these and other
study data, we believe PRV-3279 co-administration with gene therapy
products has the potential to improve the safety and efficacy of
this therapeutic modality. The PRV-3279 surrogate in combination
with sirolimus increased skeletal muscle levels of GAA enzyme
expression. Consistent with prior results from clinical trials in
healthy human subjects, the PRV-3279 surrogate decreased IgM
production and was well tolerated.
"As the field of gene therapy advances, patients' immune
responses to the viral vectors and the transgene products remain a
key challenge negatively impacting the safety, efficacy and ability
to deliver additional courses systemically," stated Francisco Leon, M.D., Ph.D., chief scientific
officer, Provention Bio. "One of the current mitigation strategies
to overcome these immune responses is pharmacological modulation of
the patients' antibody immune responses with the B cell depleting
agent rituximab in combination with the immune-suppressive agent
sirolimus. Prolonged use of rituximab has been associated with
certain adverse events. The use of PRV-3279, a non-depleting B cell
inhibitor, is a potential strategy to address this unmet need in
serious genetic diseases."
"A critical challenge for the success of gene therapy is the
host immune responses to both the vector capsid and transgene
product, which pose ongoing concerns regarding the safety,
longevity, extent of gene expression and ability to re-dose,"
stated Professor Barry Byrne,
director of the Powell Gene Therapy Center at the University of Florida. "PRV-3279's mechanism of
action, inhibiting B cell activation without depleting these
important cells, has the potential to provide a unique opportunity
to be used as an adjunctive therapy with gene therapy products. We
look forward to collaborating with Provention Bio and other
potential partners in forthcoming clinical studies."
"We believe PRV-3279 has the potential to intercept and prevent
the immunogenicity of life-saving gene therapy products and other
biotherapeutics," stated Ashleigh
Palmer, CEO and co-founder, Provention Bio. "Administration
of PRV-3279 has been well-tolerated and pharmacodynamically
effective in Phase 1 studies, with linear PK and dose-dependent
reduction in B cell activation in the absence of depletion.
PRV-3279 has also been shown to reduce B cell responses to viral
antigens using experimental vaccine challenge in Phase 1. Given
these promising clinical data and the novel pre-clinical data in
gene therapy, we look forward to opportunities to work with
academic and industry experts to combine PRV-3279 with gene therapy
products to further our mission of preventing and intercepting
devastating immune-mediated conditions."
The company plans to submit the data from this study for
presentation at an upcoming medical conference later in 2021.
About PRV-3279:
PRV-3279 is a humanized diabody (a bispecific DART molecule)
targeting the B cell surface proteins, CD32B and
CD79B. Simultaneous engagement of the CD32B and CD79B
receptors triggers inhibition of B cell function and suppression of
autoantibody production, thereby regulating B cells without causing
depletion. Provention is initially developing PRV-3279 for the
interception of systemic lupus erythematosus (SLE), a chronic
autoimmune disorder characterized by an abnormal overactivation of
B cells and subsequent pathologic production of
auto-antibodies. PRV-3279 also has the potential to prevent or
reduce the immunogenicity of biotherapeutics, including but not
limited to gene therapy vectors and transgenes.
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 diseases. The Biologics License
Application (BLA) for teplizumab, its lead investigational drug
candidate, for the delay or prevention of clinical type 1 diabetes
in at-risk individuals has been filed by the U.S. Food and Drug
Administration (FDA). The Company's pipeline includes additional
clinical-stage product candidates that have demonstrated in
pre-clinical or clinical studies proof-of-mechanism and/or
proof-of-concept in other autoimmune diseases, including 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 Company's
studies, the potential safety, health benefits of and planned
research and development efforts for PRV-3279. 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 delays in, or failure to obtain FDA
approvals or clearances and noncompliance with FDA regulations; 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; 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, 2019, our quarterly reports on
form 10-Q, 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. The information set forth herein speaks only as of the
date hereof.
Partnering:
Alex
Rabiee, SVP, Business Development & Program
Management
arabiee@proventionbio.com
908-698-4612 (EXT-118)
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:
Lori Rosen, LDR
Communications
lori@ldrcommunications.com
917-553-6808
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SOURCE Provention Bio, Inc.