Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that
14-month median follow-up data from the pivotal Phase 1/2
LINKER-MM1 trial of linvoseltamab in patients with
relapsed/refractory (R/R) multiple myeloma (MM) were shared during
an oral presentation at the European Hematology Association (EHA)
Congress 2024 and published in the Journal of Clinical Oncology.
These longer-term results show a deepening of responses following
the 11-month median follow-up data presented at the American
Association for Cancer Research Annual Meeting in April.
Linvoseltamab is an investigational 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.
“Previous results from LINKER-MM1 have demonstrated that
linvoseltamab has compelling efficacy characterized by deep and
durable responses. With 14-months of median follow-up, 50% of
patients achieved a complete response or better, despite their
cancer being refractory to or relapsing on standard therapies,”
said Suzanne Lentzsch, MD, PhD, Director of the Multiple Myeloma
and Amyloidosis Program at Columbia University. “Additionally, a
study using US-based electronic health record data to indirectly
compare linvoseltamab to real-world standard-of-care treatment also
support the overall body of evidence for this investigational
medicine in heavily pretreated multiple myeloma. Collectively,
these presentations underscore the exciting potential of
linvoseltamab as we await decisions from regulatory
authorities.”
The 14-month median follow-up LINKER-MM1 data for linvoseltamab
among patients treated at the 200 mg dose (N=117) reinforce the
durability and increasing depth of response shown in previous data
cuts. Per the presentation and publication, results showed:
- 71% objective response rate (ORR),
with 50% of patients achieving a complete response (CR) or
better and 63% achieving a very good partial response
(VGPR) or better, as determined by an independent review
committee.
- Median duration of response (DoR) of 29 months for all
responders, while median DoR was not reached for those who achieved
a CR or better. In analyses that were not pre-specified,
there was an 81% and 95% estimated probability of maintaining a
response at 12 months after achieving a partial response or better
among all patients and those who achieved a CR or better,
respectively.
- Median progression-free survival (PFS) was not
reached. There was a 70% estimated probability of being
progression free at 12 months among all patients; the estimated
probability was 96% among those who achieved a CR or better, per an
analysis that was not pre-specified.
- Median overall survival (OS) of 31 months for
all patients (95% CI: 22 months to NE). In analyses that were not
pre-specified, the median OS was not reached for patients who
achieved a CR or better, and there was a 75% and 100% estimated
probability of survival at 12 months among all patients and those
who achieved a CR or better, respectively.
- High rates of CRs or better in prespecified
subgroups, including 55% (17 of 31 patients) among those
aged 75 years or older, 48% (22 of 46 patients) among those with
high cytogenetic risk, 45% (9 of 20 patients) among Black or
African American patients, and 28% (10 of 36 patients) among those
with plasmacytomas (including extramedullary and
paramedullary).
Safety data at the 14-month median follow-up was generally
consistent with those at the 11-month median follow-up. Cytokine
release syndrome (CRS) was the most commonly occurring
treatment-emergent adverse event (TEAE) and was observed in 46% of
patients; 35% were Grade 1, 10% were Grade 2 and one case (1%) was
Grade 3. Adjudicated immune effector cell-associated neurotoxicity
syndrome (ICANS) events of any grade occurred in 8% of patients,
including three cases that were Grade 3 and no cases that were
≥Grade 4. Infections occurred in 74% of patients – including
36% that were Grade 3 or 4 – and decreased in frequency and
severity after 6 months. The most common Grade 3 or 4 TEAEs (≥20%)
were neutropenia (42%) and anemia (31%). Six deaths considered due
to TEAEs by investigators occurred on treatment or within 30 days
of the last treatment dose; five were due to infection, and one was
due to renal failure.
Also shared at EHA was a retrospective study comparing outcomes
of linvoseltamab 200 mg Phase 2 patients (N=105) in LINKER-MM1 at
14-months of median follow-up to those of real-world external
control patients (N=101) who received standard-of-care (SOC)
treatment in clinical practice (approximately 80 varied regimens).
Patients receiving SOC treatment also met similar
inclusion/exclusion criteria to the LINKER-MM1 trial. Comparing
linvoseltamab to SOC treatment, the ORR was 70% versus 32% (odds
ratio [OR] 5.4), median PFS was 20 months versus 3 months (hazard
ratio [HR]: 0.23), and median OS was not reached versus 12 months
(HR: 0.40).
In the U.S., linvoseltamab has been granted Fast Track
Designation and was accepted for Priority Review for the treatment
of R/R MM by the U.S. Food and Drug Administration with a target
action date of August 22, 2024. Linvoseltamab is also under review
for R/R MM by the European Medicines Association.
The Phase 3 confirmatory trial (LINKER-MM3) for linvoseltamab in
patients with R/R MM is ongoing. Linvoseltamab is currently under
clinical development, and its safety and efficacy have not been
fully evaluated by any regulatory authority.
About Multiple MyelomaAs the second most common
blood cancer, there are over 176,000 new cases of MM diagnosed
globally, and 35,000 cases are diagnosed in the U.S. every year. In
the U.S., there are approximately 8,000 people who have MM that has
progressed after three lines of therapy, and 4,000 whose disease
has progressed after four or more therapies. The disease is
characterized by the proliferation of cancerous plasma cells (MM
cells) that crowd out healthy blood cells in the bone marrow,
infiltrate other tissues and cause potentially life-threatening
organ injury. Despite treatment advances, MM is not curable and
while current treatments are able to slow progression of the
cancer, most patients will ultimately experience cancer progression
and require additional therapies.
About the Linvoseltamab Phase 1/2 Trial and Clinical
Development ProgramThe ongoing, open-label, multicenter
Phase 1/2 dose-escalation and dose-expansion LINKER-MM1 trial is
investigating linvoseltamab in 282 enrolled patients with
relapsed/refractory MM. The Phase 1 dose-escalation portion of the
trial – which is now complete – primarily assessed safety,
tolerability and dose-limiting toxicities across nine dose levels
of linvoseltamab and explored different administration regimens.
The ongoing Phase 2 dose expansion portion is assessing the safety
and anti-tumor activity of linvoseltamab, with the primary endpoint
of ORR. Key secondary endpoints include DoR, PFS, rate of minimum
residual disease (MRD) negative status and OS.
Eligibility in the Phase 2 portion requires patients to have
received at least three prior lines of therapy or have triple-class
refractory MM. Linvoseltamab is administered with an initial
step-up dosing regimen followed by the full 200 mg dose
administered weekly. At week 16, all patients transition to every
two-week dosing. A response-adapted regimen further enables
patients to shift to every four-week dosing if they achieve a VGPR
or better and have completed at least 24 weeks of therapy. The
regimen requires a total of two 24-hour hospitalizations for safety
monitoring.
The broader linvoseltamab clinical development program includes
additional trials in earlier lines of therapy and stages of disease
that are planned or underway. They include a Phase 1/2 trial in
first-line MM, a Phase 2 trial in high-risk smoldering MM, and a
Phase 2 trial in monoclonal gammopathy of undetermined
significance. A Phase 1 trial of linvoseltamab in combination with
a Regeneron CD38xCD28 costimulatory bispecific in MM is also
planned. For more information, visit the Regeneron clinical trials
website, or contact via clinicaltrials@regeneron.com or
844-734-6643.
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 various
combinations 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, and investigational
RNA-approaches focused on depleting abnormal proteins or blocking
disease-causing cellular signaling.
About Regeneron Regeneron (NASDAQ: REGN) is a
leading biotechnology company that invents, develops and
commercializes life-transforming medicines for people with serious
diseases. Founded and led by physician-scientists, our unique
ability to repeatedly and consistently translate science into
medicine has led to numerous approved treatments and product
candidates in development, most 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, neurological
diseases, hematologic conditions, infectious diseases, and rare
diseases.
Regeneron pushes the boundaries of scientific discovery
and accelerates drug development using our proprietary
technologies, such as VelociSuite®, which produces optimized fully
human antibodies and new classes of bispecific antibodies. We are
shaping the next frontier of medicine with data-powered insights
from the Regeneron Genetics Center® and pioneering genetic medicine
platforms, enabling us to identify innovative targets and
complementary approaches to potentially treat or cure diseases.
For more information, please visit www.Regeneron.com or follow
Regeneron on LinkedIn, Instagram, Facebook or X.
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,”
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possible success and therapeutic applications of products marketed
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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 any potential regulatory approval of
linvoseltamab for the treatment of relapsed/refractory multiple
myeloma (“R/R MM”) by the U.S. Food and Drug Administration (the
“FDA”) (including the timing of enrollment of patients in the Phase
3 confirmatory trial for linvoseltamab in patients with R/R MM
referenced in this press release (the “R/R MM Confirmatory Trial”),
whether any beneficial regulatory designations previously granted
by the FDA and referenced in this press release will positively
impact the timing for potential FDA approval, and whether any such
approval will be obtained by the FDA’s target action date
referenced in this press release) or the European Medicines Agency;
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 (such as the R/R MM
Confirmatory Trial), 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,
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ability of Regeneron to manage supply chains for multiple products
and product candidates; safety issues resulting from the
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coverage and reimbursement determinations by such payers and new
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extent to which the results from the research and development
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Injection), other litigation and other proceedings and government
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District of Massachusetts), the ultimate outcome of any such
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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, 2023 and its Form 10-Q for the quarterly
period ended March 31, 2024. Any forward-looking statements are
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including without limitation any financial projection or guidance,
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Contacts:Media
RelationsTammy AllenTel: +1
914-306-2698tammy.allen@regeneron.com |
Investor RelationsVesna TosicTel:
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