TARRYTOWN, N.Y., Dec. 5, 2019 /PRNewswire/ --
Results from initial 6-patient cohort show pozelimab reduced
lactate dehydrogenase (LDH) to normal levels at week 8 in patients
with paroxysmal nocturnal hemoglobinuria (PNH), utilizing a weekly
subcutaneous dosing regimen
Second part of Phase 2 trial initiated; plans for Phase 3
program underway
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN)
today announced topline data from the pozelimab
(REGN3918) Phase 2 clinical program in paroxysmal nocturnal
hemoglobinuria (PNH), validating the weekly 800 mg subcutaneous
(SC) dosing regimen, following an initial intravenous (IV) loading
dose. Pozelimab reduced the abnormal destruction of red blood
cells, otherwise known as "hemolysis," with patients in the initial
cohort achieving normal levels of a blood biomarker of elevated
hemolysis called lactate dehydrogenase (LDH).
PNH is an ultra-rare, chronic, life-threatening disease where
genetic mutations cause hemolysis, resulting in a range of symptoms
including fatigue, shortness of breath and blood clots. Even with
existing therapies, which require regular intravenous infusions
administered at infusion centers or during a home visit by a health
professional, approximately 50% of newly-treated patients do not
achieve normal LDH levels.
"In our view, any new medicine for PNH must deliver real change
for patients, such as more patients achieving normal LDH levels, or
a reduced treatment burden that potentially allows for at-home
self-administration," said George D.
Yancopoulos, M.D., Ph.D., President and Chief Scientific
Officer at Regeneron. "We are encouraged by these early pozelimab
results, with patients achieving normal levels of LDH by week 8
using the subcutaneous dosing regimen. We look forward to speaking
with regulators about the next phase of our program for these
patients."
Pozelimab is a VelocImmune®-derived
fully-human monoclonal antibody that blocks the pathway leading to
red blood cell hemolysis, which was designed to reduce LDH levels
and the occurrence of breakthrough hemolysis, using a
self-administered subcutaneous regimen. All 6 patients in the
initial treatment cohort treated with pozelimab experienced rapid
and sustained reductions in LDH up to week 8. By week 2, LDH was
reduced to <1.5 times the upper limit of normal (ULN) in all
patients, and at the pre-specified week 8 evaluation timepoint, the
mean LDH level was 0.74 (x ULN); range: 0.62-0.91 (x ULN). One of
these patients had a known C5 variant resistant to existing
treatments.
In these 6 patients, no adverse events (AEs) were serious or led
to discontinuation. At the cut-off date of the analyses,
treatment-related AEs were reported in 3 patients (50%), including
headache (n=2), injection site reaction (n=1) and nausea (n=1). One
patient received a blood transfusion on day 50 due to underlying
bone marrow dysfunction. In the earlier Phase 1 trial, the only
serious AE was salpingitis in a single participant.
Patients in the trial initially received a 30 mg/kg IV loading
dose of pozelimab, followed by weekly 800 mg SC injections. Current
approved treatments are only available via IV infusion.
Data from the Phase 2 trial will be presented at an upcoming
medical meeting.
About Pozelimab
Pozelimab is an investigational,
fully-human monoclonal antibody designed to block complement factor
C5 and prevent the destruction of red blood cells (hemolysis) that
cause the symptoms of PNH and other diseases mediated by complement
pathway activity. It is an IgG4 antibody that binds with high
affinity to wild-type and variant human C5 and blocks its
activity.
Pozelimab was invented using Regeneron's proprietary
VelocImmune technology, which uses a unique
genetically-humanized mouse to produce optimized fully-human
antibodies. Pozelimab is currently under clinical development, and
its safety and efficacy have not been evaluated by any regulatory
authority.
About the Phase 2 Trial
The ongoing open-label,
single-arm, two-part trial will enroll patients with active
symptomatic PNH who are naïve to complement inhibitors or who have
not received treatment with a complement inhibitor within 6 months
prior to entering the trial. It consists of two cohorts: cohort A
(n=6), which is complete and achieved its objective of dose
confirmation; and cohort B (n=approx. 30), which is ongoing and
will focus on further evaluating efficacy and safety in a larger
PNH population.
Patients in the trial suffer from elevated hemolytic activity,
as reflected by baseline LDH levels of ≥2 times the ULN. Patients
are administered a single 30 mg/kg IV loading dose of pozelimab
followed by a once-weekly 800 mg SC dose.
This Phase 2 trial could potentially provide pivotal data
supporting approval for this orphan disease indication.
About Regeneron Pharmaceuticals,
Inc.
Regeneron (NASDAQ: REGN) is a leading biotechnology
company that invents life-transforming medicines for people with
serious diseases. Founded and led for 30 years by
physician-scientists, our unique ability to repeatedly and
consistently translate science into medicine has led to seven
FDA-approved treatments and numerous product candidates in
development, all of which were homegrown in our laboratories. Our
medicines and pipeline are designed to help patients with eye
diseases, allergic and inflammatory diseases, cancer,
cardiovascular and metabolic diseases, infectious diseases, pain
and rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our proprietary
VelociSuite® technologies, including
VelocImmune®, which uses a unique
genetically-humanized mouse to produce optimized fully-human
antibodies and bispecific antibodies, and through ambitious
research initiatives such as the Regeneron Genetics Center, which
is conducting one of the largest genetics sequencing efforts in the
world.
For additional information about the company, please visit
www.regeneron.com or follow @Regeneron on Twitter.
Regeneron Forward-Looking Statements and Use of Digital
Media
This press release includes forward-looking
statements that involve risks and uncertainties relating to future
events and the future performance of Regeneron Pharmaceuticals,
Inc. ("Regeneron" or the "Company"), and actual events or results
may differ materially from these forward-looking statements. Words
such as "anticipate," "expect," "intend," "plan," "believe,"
"seek," "estimate," variations of such words, and similar
expressions are intended to identify such forward-looking
statements, although not all forward-looking statements contain
these identifying words. These statements concern, and these risks
and uncertainties include, among others, the nature, timing, and
possible success and therapeutic applications of Regeneron's
products, product candidates, and research and clinical programs
now underway or planned, including without limitation pozelimab
(REGN3918); unforeseen safety issues resulting from the
administration of products and product candidates in patients,
including serious complications or side effects in connection with
the use of Regeneron's product candidates (such as pozelimab) in
clinical trials; the extent to which the results from the research
and development programs conducted by Regeneron or its
collaborators (including the ongoing Phase 2 trial evaluating
pozelimab discussed in this press release) may be replicated in
other studies and lead to therapeutic applications; the likelihood,
timing, and scope of possible regulatory approval and commercial
launch of Regeneron's late-stage product candidates and new
indications for marketed products; ongoing regulatory obligations
and oversight impacting Regeneron's marketed products, research and
clinical programs, and business, including those relating to
patient privacy; determinations by regulatory and administrative
governmental authorities which may delay or restrict Regeneron's
ability to continue to develop or commercialize Regeneron's
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candidates that may be superior to Regeneron's products and product
candidates; uncertainty of market acceptance and commercial success
of Regeneron's products and product candidates and the impact of
studies (whether conducted by Regeneron or others and whether
mandated or voluntary) on the commercial success of Regeneron's
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manufacture and manage supply chains for multiple products and
product candidates; the ability of Regeneron's collaborators,
suppliers, or other third parties (as applicable) to perform
manufacturing, filling, finishing, packaging, labeling,
distribution, and other steps related to Regeneron's products and
product candidates; the availability and extent of reimbursement of
the Company's products from third-party payers, including private
payer healthcare and insurance programs, health maintenance
organizations, pharmacy benefit management companies, and
government programs such as Medicare and Medicaid; coverage and
reimbursement determinations by such payers and new policies and
procedures adopted by such payers; unanticipated expenses; the
costs of developing, producing, and selling products; the ability
of Regeneron to meet any of its financial projections or guidance
and changes to the assumptions underlying those projections or
guidance; the potential for any license or collaboration agreement,
including Regeneron's agreements with Sanofi, Bayer, and Teva
Pharmaceutical Industries Ltd. (or their respective affiliated
companies, as applicable), to be cancelled or terminated without
any further product success; and risks associated with intellectual
property of other parties and pending or future litigation relating
thereto (including without limitation the patent litigation and
other related proceedings relating to Dupixent®
(dupilumab) and Praluent® (alirocumab)), other
litigation and other proceedings and government investigations
relating to the Company and/or its operations, the ultimate outcome
of any such proceedings and investigations, and the impact any of
the foregoing may have on Regeneron's business, prospects,
operating results, and financial condition. A more complete
description of these and other material risks can be found in
Regeneron's filings with the U.S. Securities and Exchange
Commission, including its Form 10-K for the fiscal year ended
December 31, 2018 and its Form 10-Q
for the quarterly period ended September 30,
2019. Any forward-looking statements are made based on
management's current beliefs and judgment, and the reader is
cautioned not to rely on any forward-looking statements made by
Regeneron. Regeneron does not undertake any obligation to update
publicly any forward-looking statement, including without
limitation any financial projection or guidance, whether as a
result of new information, future events, or otherwise.
Regeneron uses its media and investor relations website and
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Company, including information that may be deemed material to
investors. Financial and other information about Regeneron is
routinely posted and is accessible on Regeneron's media and
investor relations website (http://newsroom.regeneron.com)
and its Twitter feed
(http://twitter.com/regeneron).
Contacts:
Media Relations
Sarah Cornhill
Tel: +1 (917) 297-1522
Sarah.Cornhill@regeneron.com
Investor Relations
Justin Holko
Tel: +1 (914) 847-7786
Justin.Holko@regeneron.com
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SOURCE Regeneron Pharmaceuticals, Inc.