BioNTech Announces Positive Topline Phase 2 Results for mRNA
Immunotherapy Candidate BNT111 in Patients with Advanced Melanoma
- Primary endpoint met in a Phase 2 trial evaluating the
investigational mRNA immunotherapy BNT111 in combination with the
PD-1 checkpoint inhibitor cemiplimab
- Data demonstrated a statistically significant improvement of
overall response rate (“ORR”) compared to historical control in
patients with anti-PD-(L)1 relapsed/refractory advanced
melanoma
- BioNTech and Regeneron plan to present data from this trial
at a forthcoming medical conference; the BNT111 program received a
Fast Track designation and an Orphan Drug designation from the U.S.
Food and Drug Administration (“FDA”) in 2021
- BNT111 is based on BioNTech’s fully owned FixVac platform
and proprietary uridine mRNA-LPX technology
MAINZ, Germany, July 30, 2024 (GLOBE
NEWSWIRE) -- BioNTech SE (Nasdaq: BNTX, “BioNTech” or “the
Company”) today announced positive topline data from the ongoing
Phase 2 clinical trial (EudraCT No.: 2020-002195-12; NCT04526899)
in patients with unresectable stage III or IV melanoma whose
disease had progressed following anti-PD-(L)1-containing treatment.
The randomized trial evaluates the clinical activity and safety of
the investigational mRNA cancer immunotherapy BNT111 in combination
with Libtayo® (cemiplimab), an anti-PD-1 monoclonal
antibody being developed by Regeneron, and assesses the two single
agents alone.
The trial met its primary efficacy outcome
measure, demonstrating a statistically significant improvement in
ORR in patients treated with BNT111 in combination with cemiplimab
as compared to historical control in this indication and treatment
setting. Both randomized monotherapy arms showed clinical activity.
The ORR in the cemiplimab monotherapy arm was in line with the
historical control of anti-PD-(L)1 or anti-CTLA-4 treatments in
this patient group. The treatment was well tolerated and the safety
profile of BNT111 in combination with cemiplimab in this trial was
consistent with previous clinical trials assessing BNT111 in
combination with anti-PD-(L)1-containing treatments. The Phase 2
trial will continue as planned to further assess the secondary
endpoints, which were not mature at the time of the primary
analysis.
“These Phase 2 results mark a significant step
towards our vision of personalized cancer medicine. We envision
mRNA as a centerpiece in future treatment paradigms for cancer,
helping to address unmet medical needs, such as for patients with
anti-PD-(L)1 refractory or resistant melanoma,” said Prof. Özlem
Türeci, M.D., Chief Medical Officer and Co-Founder at BioNTech.
“These data are a proof of concept for us in three dimensions:
First, for our decade-long improved mRNA cancer vaccine technology
that uses uridine mRNA chemistry, a non-coding backbone that is
engineered for optimal translational performance and our
proprietary lipoplex formulation for delivery. Second, for our
computational approaches for selecting suitable tumor antigens for
our cancer indication-specific FixVac platform candidates. Third,
for our strategy to combine synergistic modalities, in this case
BNT111 with an established immune checkpoint treatment.”
BioNTech and Regeneron plan to present data from
this trial at a forthcoming medical conference. Further, the
companies also intend to submit these data for publication in a
peer reviewed scientific journal.
BNT111 is based on BioNTech’s fully owned FixVac
platform that utilizes a fixed combination of four mRNA-encoded,
tumor-associated antigens designed to trigger an innate and
tumor-antigen-specific immune response against cancer cells
expressing one or more of the respective tumor antigens. In 2021,
BNT111 in combination with cemiplimab received Fast Track
designation by the FDA for the treatment of
anti-PD-1-refractory/relapsed, unresectable Stage III or IV
melanoma. In the same year, the FDA granted Orphan Drug designation
for BNT111 the treatment of stage IIB through IV melanoma.
About BioNTech’s oncology mRNA
platforms
BioNTech has developed a range of mRNA platforms to establish a
novel class of therapeutics and vaccines aimed at improving the
health of people worldwide. In oncology, BioNTech utilizes five
mRNA platforms. Each platform is designed with the aim to address
unique challenges in oncology. BioNTech’s fully owned FixVac (Fix
Combination Vaccine) platform candidates target specific cancer
indications focusing on tumor-associated antigens which are shared
by many cancer patients, while iNeST (Individualized Neoantigen
Specific Immunotherapy) platform candidates are personalized
immunotherapies tailored to the patient’s individual tumor profile.
Both platforms utilize BioNTech’s proprietary optimized uridine
mRNA (“uRNA”) technology and the lipoplex (“LPX”) delivery
technology that the scientific founders and the researchers at
BioNTech have pioneered over decades of scientific discoveries and
technological advancements. These technologies are optimized for
immunotherapy applications aiming to boost the immunostimulatory
effect of the investigational immunotherapies and to trigger
targeted immune responses against cancer cells expressing one or
more of the respective encoded tumor antigens. Currently, six
programs based on the Company’s FixVac and iNeST platforms are
being evaluated in randomized Phase 2 trials in various solid tumor
indications.
In addition to the FixVac and iNeST platforms,
BioNTech is leveraging mRNA to deliver the building plan for
targeted antibody, cytokine or immunomodulating protein approaches
directly to the patient based on the Company’s RiboMab,
RiboCytokine and Intratumoral Immunotherapy platforms aiming to
help the body to produce its own therapeutic.
About advanced melanoma
Melanoma is amongst the leading causes of cancer-related deaths
globally, responsible for roughly 58,000 deaths yearly.
1 Anti-PD-1 refractory/relapsed unresectable Stage III
or IV melanoma is an aggressive form of melanoma, which remains
particularly lethal. Current standard of care includes checkpoint
inhibitor therapies that substantially improve the life expectancy
of patients with melanoma.2,3 Despite
advances in treatment, a high proportion of patients exhibit
resistance to approved therapies, leading to limited options for
those who progress on targeted or immunotherapy.4 The
5-year survival rate for patients with distant metastatic melanoma
is approximately 35%.5 This underscores the significant
unmet medical need.
About BNT111
BNT111 is an mRNA-based off-the-shelf cancer immunotherapy
candidate for intravenous administration encoding a fixed set of
four non-mutated melanoma-associated antigens (NY-ESO-1, MAGE-A3,
tyrosinase, and TPTE) delivered as uridine mRNA-lipoplex
formulation. Over 90% of patients with cutaneous melanomas express
at least one of these antigens.6 Data of the Lipo-MERIT
Phase 1 clinical trial have shown that BNT111 alone or in
combination with blockade of the checkpoint inhibitor PD-1 induces
novel antigen-specific anti-tumor immune responses and enhances
pre-existing immune responses against the encoded
melanoma-associated antigens in checkpoint inhibitor-experienced
patients with unresectable melanoma.7 Further, these
data have shown that the candidate can prime and activate T cells
against the vaccine antigens that persisted for more than one year
under continuous monthly vaccination.7 BNT111 is one of
three clinical-stage FixVac product candidates within BioNTech’s
development pipeline. The candidate is currently being evaluated in
a Phase 2 clinical trial in combination with cemiplimab, in
patients with anti-PD-1 refractory/relapsed unresectable Stage III
or IV melanoma.
About the BNT111-01 trial
The
BNT111-01 trial (EudraCT No.: 2020-002195-12; NCT04526899) is an
open-label, randomized Phase 2 trial evaluating the efficacy of
BNT111 and cemiplimab and the contribution of the single components
in patients with anti-PD-1/PD-L1-refractory or relapsed,
unresectable Stage III or IV cutaneous melanoma. Conducted across
approximately 60 sites in 7 countries, this multi-site trial aims
to demonstrate anti-tumor activity and ORR of the combination
therapy as well as each agent alone. Additional endpoints include
duration of response (DOR), disease control rate (DCR), overall
survival (OS), safety, and tolerability. Patients were randomized
in a 2:1:1 ratio to Arm 1 (BNT111 + cemiplimab), Arm 2 (BNT111
monotherapy), and Arm 3 (cemiplimab monotherapy), with up to 24
months of active treatment. More information on this trial can be
found at clinicaltrials.gov or www.clinicaltrialsregister.eu.
About BioNTech
Biopharmaceutical New Technologies (BioNTech) is a global next
generation immunotherapy company pioneering novel therapies for
cancer and other serious diseases. BioNTech exploits a wide array
of computational discovery and therapeutic drug platforms for the
rapid development of novel biopharmaceuticals. Its broad portfolio
of oncology product candidates includes individualized and
off-the-shelf mRNA-based therapies, innovative chimeric antigen
receptor (CAR) T cells, several protein-based therapeutics,
including bispecific immune checkpoint modulators, targeted cancer
antibodies and antibody-drug conjugate (ADC) therapeutics, as well
as small molecules. Based on its deep expertise in mRNA vaccine
development and in-house manufacturing capabilities, BioNTech and
its collaborators are developing multiple mRNA vaccine candidates
for a range of infectious diseases alongside its diverse oncology
pipeline. BioNTech has established a broad set of relationships
with multiple global and specialized pharmaceutical collaborators,
including Biotheus, DualityBio, Fosun Pharma, Genentech, a member
of the Roche Group, Genevant, Genmab, MediLink, OncoC4, Pfizer and
Regeneron.
For more information, please visit www.BioNTech.com.
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995, as amended, including, but not be limited to,
statements concerning: the initiation, timing, progress and results
of BioNTech’s research and development plans in oncology, including
its collaboration with Regeneron and FixVac program candidates
BNT111, BNT113 and BNT116; the nature and characterization of and
timing for release of clinical data across BioNTech’s platforms,
including any data readouts of the Phase 2 trial of BNT111 in
combination with cemiplimab in patients with anti-PD-(L)1
refractory/relapsed unresectable Stage III or IV melanoma, which is
subject to peer review, regulatory review and market
interpretation; the planned next steps in BioNTech’s pipeline
programs, including, but not limited to, statements regarding
timing or plans for initiation or enrollment of clinical trials, or
submission for and receipt of product approvals and potential
commercialization with respect to BioNTech’s product candidates;
the ability of BioNTech’s mRNA technology to demonstrate clinical
efficacy outside of BioNTech’s infectious disease platform; and the
potential safety and efficacy of BioNTech’s product candidates. In
some cases, forward-looking statements can be identified by
terminology such as “will,” “may,” “should,” “expects,” “intends,”
“plans,” “aims,” “anticipates,” “believes,” “estimates,”
“predicts,” “potential,” “continue,” or the negative of these terms
or other comparable terminology, although not all forward-looking
statements contain these words.
The forward-looking statements in this press
release are based on BioNTech’s current expectations and beliefs of
future events, and are neither promises nor guarantees. You should
not place undue reliance on these forward-looking statements
because they involve known and unknown risks, uncertainties, and
other factors, many of which are beyond BioNTech’s control, and
which could cause actual results to differ materially and adversely
from those expressed or implied by these forward-looking
statements. These risks and uncertainties include, but are not
limited to: the uncertainties inherent in research and development,
including the ability to meet anticipated clinical endpoints,
commencement and/or completion dates for clinical trials,
regulatory submission dates, regulatory approval dates and/or
launch dates, as well as risks associated with preclinical and
clinical data, including the data discussed in this release, and
including the possibility of unfavorable new preclinical, clinical
or safety data and further analyses of existing preclinical,
clinical or safety data; the nature of clinical data, which is
subject to ongoing peer review, regulatory review and market
interpretation; the ability to produce comparable clinical results
in future clinical trials; the timing of and BioNTech’s ability to
obtain and maintain regulatory approval for its product candidates;
discussions with regulatory agencies regarding timing and
requirements for additional clinical trials; BioNTech’s and its
counterparties’ ability to manage and source necessary energy
resources; BioNTech’s ability to identify research opportunities
and discover and develop investigational medicines; the ability and
willingness of BioNTech’s third-party collaborators to continue
research and development activities relating to BioNTech’s
development candidates and investigational medicines; unforeseen
safety issues and potential claims that are alleged to arise from
the use of products and product candidates developed or
manufactured by BioNTech; BioNTech’s and its collaborators’ ability
to commercialize and market, its product candidates, if approved;
BioNTech’s ability to manage its development and expansion;
regulatory developments in the United States and other countries;
BioNTech’s ability to effectively scale its production capabilities
and manufacture its products and product candidates; risks relating
to the global financial system and markets; and other factors not
known to BioNTech at this time.
You should review the risks and uncertainties
described under the heading “Risk Factors” in BioNTech's Report on
Form 6-K for the period ended March 31, 2024, and in subsequent
filings made by BioNTech with the SEC, which are available on the
SEC’s website at www.sec.gov. These forward-looking statements
speak only as of the date hereof. Except as required by law,
BioNTech disclaims any intention or responsibility for updating or
revising any forward-looking statements contained in this press
release in the event of new information, future developments or
otherwise.
CONTACTS
Investor Relations
Victoria Meissner, M.D.
+1 617 528 8293
Investors@biontech.de
Media Relations
Jasmina Alatovic
+49 (0)6131 9084 1513
Media@biontech.de
1 Sung H, Ferlay J, Siegel RL, Laversanne M,
Soerjomataram I, Jemal A, Bray F. CA Cancer J Clin.2021 Feb 4. Epub
ahead of print.
2 Nogrady B. Nature 580, S14-S16 (2020). Available at
https://www.nature.com/articles/d41586-020-01038-9
3 Huang AC, Zappasodi R. Nat Immunol 23, 660–670
(2022).
4 Hassel JC, et al. Cancers (Basel). 2023 Jul; 15(13):
3448.
5 National Cancer Institute. Cancer Stat Facts: Melanoma
of the Skin. Available from:
https://seer.cancer.gov/statfacts/html/melan.html. Last accessed
15.07.2024
6 Data on file.
7 Sahin, U., Oehm, P., Derhovanessian, E. et al. Nature
585, 107–112 (2020). Available from:
https://www.nature.com/articles/s41586-020-2537-9
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