Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced
updated data from two Phase 2 expansion dose cohorts evaluating
investigational linvoseltamab (formerly REGN5458) in patients with
heavily pre-treated, relapsed/refractory (R/R) multiple myeloma.
The results will be shared in an oral session at the 2023 American
Society of Clinical Oncology (ASCO) Annual Meeting and the
LINKER-MM1 trial will form the basis of planned submissions to
regulatory authorities, including to the U.S. Food and Drug
Administration (FDA) later this year. Linvoseltamab is an
investigational BCMAxCD3 bispecific antibody designed to bridge
B-cell maturation antigen (BCMA) on multiple myeloma cells with
CD3-expressing T cells to facilitate T-cell activation and
cancer-cell killing.
“Despite advances, new treatments are needed that drive
meaningful and durable responses to help patients with relapsed
and/or refractory multiple myeloma,” said Hans Lee, M.D., Associate
Professor and Director, Multiple Myeloma Clinical Research at The
University of Texas MD Anderson Cancer Center. “Treatment with
linvoseltamab at the recommended 200 mg dose in the LINKER-MM1
trial demonstrated impressive efficacy, with rapid, deep and
durable responses in patients with multiple myeloma that’s highly
refractory to standard therapies. Moreover, less than half of
patients experienced any grade cytokine release syndrome, which was
mostly Grade 1, with some Grade 2 and a single Grade 3 case. This
reinforces the potential of linvoseltamab as a promising treatment
option.”
The new data to be presented at ASCO 2023 are from patients
treated in the 50 mg (n=104) and 200 mg (n=117) cohorts of the
Phase 1/2 trial. Initial results were presented at the 64th
American Society of Hematology (ASH) Annual Meeting and Exposition
in December 2022. Among the 200 mg cohort, the median soluble BCMA
(sBCMA) was 377 ng/mL, 22% had bone marrow plasma cells ≥50% and
36% had high-risk cytogenetics, representing a patient population
with a high disease burden and poor prognosis. The primary endpoint
was objective response rate (ORR) assessed by independent review
committee, which will be available when the data are more mature.
The secondary endpoints included ORR and other efficacy measures
assessed by local investigator. With a median follow-up of 6
months, patients receiving the recommended 200 mg dose showed:
- 71% ORR, per local investigator.
- 59% achieved a very good partial response (VGPR) or
better, with 30% achieving a complete response (CR) or stringent
complete response (sCR), per local investigator. Based on
earlier results, responses may deepen with longer follow-up.
- Median time to onset of response was less than 1
month.
- 84% and 79% probability of maintaining a response at 6
and 12 months, respectively, per Kaplan-Meier
estimates.
- Median progression-free survival was not
reached.
Strong efficacy per ORR was consistently observed in the 200 mg
cohort across multiple subgroups, even in high risk patients such
as adults ≥75 years of age (n=31; 68%), patients with International
Staging System (ISS) stage II and III disease (n=44 and 22; 73% and
59% respectively), patients with extramedullary plasmacytomas
(defined as disease without bone association, n=16; 56%) as well as
patients with baseline sBCMA ≥400 ng/mL (n=51; 55%). Additionally,
among patients treated with 50 mg and 200 mg that achieved CR or
sCR with available minimal residual disease (MRD) data, 54% were
MRD negative at 10-5.
No new safety signals were identified with longer follow-up in
the Phase 1 or Phase 2 portions of the trial. Among all patients in
the 200 mg cohort, 79% experienced Grade ≥3 adverse events (AE).
Most commonly occurring AEs (in ≥20% of patients) were cytokine
release syndrome (CRS; 45%), neutropenia, cough, fatigue and
diarrhea (33% each), anemia (27%), arthralgia (26%), and headache
(23%). Discontinuations due to an AE occurred in 16% of patients.
Deaths due to treatment-emergent AEs, on-treatment or within 30
days post last dose, in the 200 mg cohort were reported in 6
patients. None of the deaths were considered related to treatment
per the treating physician. Among the 200 mg cohort, the majority
of CRS cases were mild or moderate, there was a single case of
Grade 3 CRS, and no cases of ≥Grade 4 CRS. The median time to first
CRS onset was 15 hours (range: 0-177 hours), with the median time
to resolution within 1 day (17 hours; range: 1-144 hours). Among
the 50 mg and 200 mg dose cohorts (n=221), there were 14 immune
effector cell-associated neurotoxicity syndrome events (ICANS, 6%
all Grades; 2% Grades 3-4).
“With these latest pivotal results, linvoseltamab demonstrated
notable response rates, providing encouraging evidence for this
bispecific antibody,” said L. Andres Sirulnik, M.D., Ph.D., Senior
Vice President, Translational and Clinical Sciences, Hematology at
Regeneron. “We designed linvoseltamab with patient needs at the
center and are proud that it has provided benefit across the
spectrum of relapsed refractory multiple myeloma patients, even
those with hard-to-treat disease. We look forward to sharing these
data with regulatory authorities with the goal of bringing this
medicine to patients with heavily pre-treated multiple myeloma as
soon as possible.”
Based on these data, the Phase 3 development program
investigating linvoseltamab in earlier stages of the disease has
been initiated. In the U.S., linvoseltamab has been granted Fast
Track Designation for multiple myeloma by the FDA. Linvoseltamab is
currently under clinical development, and its safety and efficacy
have not been fully evaluated by any regulatory authority.
About the Phase 1/2 TrialThe ongoing,
open-label, multicenter Phase 1/2 dose-escalation and
dose-expansion trial is investigating linvoseltamab in patients
with R/R multiple myeloma. Among the 282 patients enrolled, all
have received at least three prior lines of therapy or are triple
refractory. Patients were administered linvoseltamab utilizing a
step-up dosing regimen that was designed to help mitigate CRS.
The Phase 1 dose-escalation portion of the trial, which is now
complete, primarily assessed safety, tolerability and dose-limiting
toxicities across 9 dose levels of linvoseltamab exploring
different administration regimens. The fully-enrolled Phase 2 dose
expansion portion of the trial is further assessing the safety and
anti-tumor activity of linvoseltamab, with a primary objective of
ORR. Key secondary objectives include duration of response,
progression free survival, rate of minimal residual disease
negative status and overall survival.
About Multiple MyelomaMultiple myeloma is the
second most common blood cancer. Globally, there were 176,404 new
diagnoses in 2020 and 35,730 new diagnoses estimated for 2023 in
the U.S. It is characterized by the proliferation of cancerous
plasma cells (multiple myeloma cells) that crowd out healthy blood
cells in the bone marrow, infiltrate other tissues and cause
potentially life-threatening organ injury. Multiple myeloma is not
curable despite treatment advances, and while current treatments
are able to slow the progression of the cancer, most patients will
ultimately experience cancer progression and require additional
therapies. In addition, patients are at increased risk of frequent
infections, bone fracture and pain, reduced kidney function, and
anemia.
About Regeneron in HematologyAt Regeneron,
we’re applying more than three decades of biology expertise with
our proprietary VelociSuite® technologies to develop medicines for
patients with diverse blood cancers and rare blood disorders.
Our blood cancer research is focused on bispecific antibodies
that are being investigated both as monotherapies and in
combination with each other and emerging therapeutic modalities.
Together, they provide us with unique combinatorial flexibility to
develop customized and potentially synergistic cancer
treatments.
Our research and collaborations to develop potential treatments
for rare blood disorders include explorations in antibody medicine,
gene editing and gene-knockout technologies, as well as
investigational RNA-approaches focused on depleting abnormal
proteins or blocking disease-causing cellular signaling.
If you are interested in learning more about our clinical
trials, please contact us (clinicaltrials@regeneron.com or
844-734-6643) or visit our clinical trials website.
About Regeneron's VelocImmune
TechnologyRegeneron's VelocImmune technology
utilizes a proprietary genetically engineered mouse platform
endowed with a genetically humanized immune system to produce
optimized fully human antibodies. When Regeneron's co-Founder,
President and Chief Scientific Officer George D. Yancopoulos was a
graduate student with his mentor Frederick W. Alt in 1985, they
were the first to envision making such a genetically humanized
mouse, and Regeneron has spent decades inventing and developing
VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos
and his team have used VelocImmune technology to create a
substantial proportion of all original, FDA-approved or authorized
fully human monoclonal antibodies. This includes REGEN-COV®
(casirivimab and imdevimab), Dupixent® (dupilumab), Libtayo®,
Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza®
(evinacumab-dgnb) and Inmazeb® (atoltivimab, maftivimab and
odesivimab-ebgn).
About RegeneronRegeneron is a leading
biotechnology company that invents, develops and commercializes
life-transforming medicines for people with serious diseases.
Founded and led for 35 years by physician-scientists, our unique
ability to repeatedly and consistently translate science into
medicine has led to nine FDA-approved treatments and numerous
product candidates in development, almost 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, pain, hematologic conditions, infectious diseases and
rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our proprietary VelociSuite
technologies, such as VelocImmune, which uses unique genetically
humanized mice 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 more information, please visit www.Regeneron.com or follow
@Regeneron on Twitter.
Forward-Looking Statements and Use of Digital
MediaThis 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 products marketed
or otherwise commercialized by Regeneron and/or its collaborators
or licensees (collectively, “Regeneron’s Products”) and product
candidates being developed by Regeneron and/or its collaborators or
licensees (collectively, “Regeneron’s Product Candidates”) and
research and clinical programs now underway or planned, including
without limitation linvoseltamab; the likelihood, timing, and scope
of possible regulatory approval and commercial launch of
Regeneron’s Product Candidates and new indications for Regeneron’s
Products, such as linvoseltamab for the treatment of
relapsed/refractory multiple myeloma; uncertainty of the
utilization, market acceptance, and commercial success of
Regeneron’s Products and Regeneron’s Product Candidates and the
impact of studies (whether conducted by Regeneron or others and
whether mandated or voluntary), including the studies discussed or
referenced in this press release, on any of the foregoing or any
potential regulatory approval of Regeneron's Products and
Regeneron's Product Candidates (such as linvoseltamab); the ability
of Regeneron’s collaborators, licensees, suppliers, or other third
parties (as applicable) to perform manufacturing, filling,
finishing, packaging, labeling, distribution, and other steps
related to Regeneron’s Products and Regeneron’s Product Candidates;
the ability of Regeneron to manage supply chains for multiple
products and product candidates; safety issues resulting from the
administration of Regeneron’s Products and Regeneron’s Product
Candidates in patients, including serious complications or side
effects in connection with the use of Regeneron’s Products and
Regeneron’s Product Candidates in clinical trials; determinations
by regulatory and administrative governmental authorities which may
delay or restrict Regeneron’s ability to continue to develop or
commercialize Regeneron’s Products and Regeneron’s Product
Candidates; ongoing regulatory obligations and oversight impacting
Regeneron’s Products, research and clinical programs, and business,
including those relating to patient privacy; the availability and
extent of reimbursement of Regeneron’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; competing drugs and
product candidates that may be superior to, or more cost effective
than, Regeneron’s Products and Regeneron’s Product Candidates; the
extent to which the results from the research and development
programs conducted by Regeneron and/or its collaborators or
licensees may be replicated in other studies and/or lead to
advancement of product candidates to clinical trials, therapeutic
applications, or regulatory approval; 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, collaboration, or supply
agreement, including Regeneron’s agreements with Sanofi and Bayer
(or their respective affiliated companies, as applicable) to be
cancelled or terminated; the impact of public health outbreaks,
epidemics, or pandemics (such as the COVID-19 pandemic) on
Regeneron's business; 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 EYLEA® (aflibercept)
Injection, Praluent® (alirocumab), and REGEN-COV® (casirivimab and
imdevimab)), 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 year ended December 31,
2022 and its Form 10-Q for the quarterly period ended March 31,
2023. 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 or otherwise)
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
social media outlets to publish important information about the
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 Tammy
Allen Tel: +1 914-306-2698tammy.allen@regeneron.com |
Investor RelationsVesna TosicTel:
+1 914-847-5443vesna.tosic@regeneron.com |
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