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Phase 3 COLUMBA Study Investigating a Subcutaneous Formulation of Darzalex®▼ (daratumumab) Showed Non-Inferiority to Intravenous Administration in Patients with Relapsed/Refractory Multiple Myeloma

Subcutaneous formulation also showed reduced administration time and
lower rates of infusion-related reactions compared to intravenous
administration

BEERSE, Belgium–(BUSINESS WIRE)–The Janssen Pharmaceutical Companies of Johnson & Johnson announced
today results from the Phase 3 COLUMBA (MMY3012,
NCT03277105) study, investigating a subcutaneously (SC) administered
formulation of Darzalex® (daratumumab), co-formulated with
recombinant human hyaluronidase PH20 (rHuPH20) [Halozyme’s ENHANZE®
drug delivery technology], in patients with relapsed/refractory multiple
myeloma. The results showed non-inferior efficacy and pharmacokinetics
for the SC administered formulation of daratumumab compared to
intravenous (IV) administration, the only currently approved formulation
of daratumumab (Abstract
#8005
).1 The data presentation – the first for this Phase
3 study with SC formulation – is being featured in an oral session at
the 55th American Society of Clinical Oncology (ASCO) Annual
Meeting in Chicago, and was selected for the Best
of ASCO 2019 Meetings
.

“This study showed that the subcutaneous formulation of daratumumab
resulted in non-inferior pharmacokinetics and efficacy compared to the
current intravenous formulation, and also importantly offers the
potential for a fixed-dose administration, shorter infusion times and a
lower rate of infusion-related reactions,” said Maria-Victoria Mateos,
M.D., Ph.D., COLUMBA primary investigator and Director of the Myeloma
Unit at University Hospital of Salamanca-IBSAL, Salamanca, Spain.
“Daratumumab IV has proven to be an important medication in the
treatment of multiple myeloma, and a new subcutaneous formulation may
offer patients a different experience, including a shorter
administration time.”

At a median follow-up of 7.5 months, the overall response rate (ORR) was
41 percent for the SC administered formulation of daratumumab compared
to 37 percent for daratumumab IV (95 percent confidence interval [CI],
1.11 (0.89-1.37); P<0.0001).1 The ORR was similar across
all clinically relevant subgroups, including bodyweight.1 The
ratio of geometric means of Ctrough for SC daratumumab over
IV daratumumab was 108 percent (90 percent CI, 96 percent -122 percent).1
The progression-free survival was comparable between the SC administered
formulation of daratumumab and the current IV formulation of daratumumab
(Hazard Ratio [HR] = 0.99; 95 percent CI, 0.78-1.26; P<0.9258).1
The median duration for each SC injection was five minutes, compared to
more than three hours with IV infusions.1

The most common (>5%) Grade 3/4 treatment-emergent adverse events
(TEAEs) were thrombocytopenia (14 percent vs. 14 percent), anaemia (13
percent vs. 14 percent) and neutropenia (13 percent vs. 8 percent).1
A lower rate of infusion-related reactions was observed in the arm that
received the SC administered formulation of daratumumab compared to
daratumumab IV (13 percent vs. 35 percent, respectively) (Odds Ratio =
0.28; 95 percent CI (0.18-0.44); P<0.0001).1 The primary
reasons for treatment discontinuation included progressive disease (43
percent in the SC arm vs. 44 percent in the IV arm) and adverse events
(7 percent in the SC arm vs. 8 percent in the IV arm).1

“Our ambition in multiple myeloma has always focused on improving
outcomes, but also experience for patients, and we are therefore
incredibly pleased to see these results which confirm the potential for
a new, and shorter, route of administration,” said Dr Patrick Laroche,
Europe, Middle East and Africa (EMEA) Haematology Therapeutic Area Lead,
Janssen-Cilag France. “We look forward to submitting these data for
regulatory review in coming months, to extend the reach of daratumumab
to patients who could benefit from this novel formulation.”

ENDS

In Europe, daratumumab is indicated:2

About the COLUMBA Trial3

The randomised, open-label, multicentre Phase 3 study included 522
patients with multiple myeloma who had received at least three prior
lines of therapy including a proteasome inhibitor (PI) and an
immunomodulatory drug (IMiD), or whose disease was refractory to both a
PI and an IMiD (median age of 67). In the arm that received the SC
administered formulation of daratumumab (n=263), patients received a
fixed dose of daratumumab 1,800 milligrams (mg) co-formulated with
recombinant human hyaluronidase (rHuPH20) 2,000 Units Per millilitre
(U/mL), subcutaneously weekly for Cycles 1 – 2, every two weeks for
Cycles 3 – 6, and every four weeks for Cycle 7 and thereafter. In the
daratumumab IV arm (n=259), patients received daratumumab for
intravenous infusion 16 milligrams per kilogram (mg/kg) weekly for
Cycles 1 – 2, every two weeks for Cycles 3 – 6, and every four weeks for
Cycle 7 and thereafter. Each cycle was 28 days. Patients in both
treatment arms continued until disease progression or unacceptable
toxicity. Co-primary endpoints were ORR (analysed by Farrington-Manning
test, with non-inferiority = 60 percent retention of ORR) and pre-dose
C3D1 DARA Ctrough (non-inferiority = lower bound of 90
percent CI for the ratio of the geometric means [GM] ≥80%).

About daratumumab

Daratumumab is a first-in-class4 biologic targeting CD38, a
surface protein that is highly expressed across multiple myeloma cells,
regardless of disease stage.5 Daratumumab is believed to
induce tumour cell death through multiple immune-mediated mechanisms of
action, including complement-dependent cytotoxicity (CDC),
antibody-dependent cell-mediated cytotoxicity (ADCC) and
antibody-dependent cellular phagocytosis (ADCP), as well as through
apoptosis, in which a series of molecular steps in a cell lead to its
death.2 A subset of myeloid derived suppressor cells (CD38+
MDSCs), CD38+ regulatory T cells (Tregs) and CD38+ B cells (Bregs) were
decreased by daratumumab.2 Daratumumab is being evaluated in
a comprehensive clinical development programme across a range of
treatment settings in multiple myeloma, such as in frontline and
relapsed settings.6,7,8,9,10,11,12,13 Additional studies are
ongoing or planned to assess its potential in other malignant and
pre-malignant haematologic diseases in which CD38 is expressed, such as
smouldering myeloma.14,15 For more information, please see www.clinicaltrials.gov.

For further information on daratumumab, please see the Summary of
Product Characteristics at https://www.ema.europa.eu/documents/product-information/darzalex-epar-product-information_en.pdf.

In August
2012
, Janssen Biotech, Inc. and Genmab A/S entered a worldwide
agreement, which granted Janssen an exclusive licence to develop,
manufacture and commercialise daratumumab.16

About Multiple Myeloma

Multiple myeloma (MM) is an incurable blood cancer that starts in the
bone marrow and is characterised by an excessive proliferation of plasma
cells.17 In Europe, more than 48,200 people were diagnosed
with MM in 2018, and more than 30,800 patients died.18
Almost 60 percent of patients with MM do not survive more than five
years after diagnosis.19

Although treatment may result in remission, unfortunately, patients will
most likely relapse as there is currently no cure.20
Refractory MM is when a patient’s disease progresses within 60 days of
their last therapy.21,22 Relapsed cancer is when the disease
has returned after a period of initial, partial or complete remission.23
While some patients with MM have no symptoms at all, most patients are
diagnosed due to symptoms that can include bone problems, low blood
counts, calcium elevation, kidney problems or infections.24 Patients
who relapse after treatment with standard therapies, including
proteasome inhibitors and immunomodulatory agents, have poor prognoses
and few treatment options available.25

About the Janssen Pharmaceutical Companies of Johnson & Johnson

At Janssen, we’re creating a future where disease is a thing of the
past. We’re the Pharmaceutical Companies of Johnson & Johnson, working
tirelessly to make that future a reality for patients everywhere by
fighting sickness with science, improving access with ingenuity, and
healing hopelessness with heart. We focus on areas of medicine where we
can make the biggest difference: Cardiovascular & Metabolism,
Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and
Pulmonary Hypertension.

Learn more at www.janssen.com/emea.
Follow us at www.twitter.com/janssenEMEA
for our latest news. Janssen R&D, LLC, Janssen Biotech and Janssen-Cilag
France are part of the Janssen Pharmaceutical Companies of Johnson &
Johnson.

Cautions Concerning Forward-Looking Statements

This press release contains “forward-looking statements” as defined
in the Private Securities Litigation Reform Act of 1995 regarding the
benefits of daratumumab for the treatment of patients with multiple
myeloma. The reader is cautioned not to rely on these forward-looking
statements. These statements are based on current expectations of future
events. If underlying assumptions prove inaccurate or known or unknown
risks or uncertainties materialise, actual results could vary materially
from the expectations and of Janssen Research & Development, LLC.,
Janssen-Cilag France and any of the other Janssen Pharmaceutical
Companies and/or Johnson & Johnson. Risks and uncertainties include, but
are not limited to: challenges and uncertainties inherent in product
research and development, including the uncertainty of clinical success
and of obtaining regulatory approvals; uncertainty of commercial
success; manufacturing difficulties and delays; competition, including
technological advances, new products and patents attained by
competitors; challenges to patents; [product efficacy or safety concerns
resulting in product recalls or regulatory action; changes in behaviour
and spending patterns of purchasers of health care products and
services; changes to applicable laws and regulations, including global
health care reforms; and trends toward health care cost containment. A
further list and descriptions of these risks, uncertainties and other
factors can be found in Johnson & Johnson’s Annual Report on Form 10-K
for the fiscal year ended December 30, 2018, including in the sections
captioned “Cautionary Note Regarding Forward-Looking Statements” and
“Item 1A. Risk Factors,” and in the company’s most recently filed
Quarterly Report on Form 10-Q, and the company’s subsequent filings with
the Securities and Exchange Commission. Copies of these filings are
available online at
www.sec.gov,
www.jnj.com
or on request from Johnson & Johnson. Neither the Janssen Pharmaceutical
Companies of Johnson & Johnson nor Johnson & Johnson undertakes to
update any forward-looking statement as a result of new information or
future events or developments.

# # #

EHNANZE® is a registered trademark of Halozyme, Inc.

______________________

1 Mateos MV, Nahi H, Legiec W, et al. Efficacy and safety of
the randomized, open-label, non-inferiority, phase 3 study of
subcutaneous (SC) versus intravenous (IV) daratumumab (DARA)
administration in patients (pts) with relapsed or refractory multiple
myeloma (RRMM): COLUMBA. Presented at the Annual Meeting of the American
Society of Clinical Oncology, Chicago, IL, USA, 31 May – 4 June 2019.

2 European Medicines Agency. DARZALEX summary of product
characteristics, January 2019. Available at: https://www.ema.europa.eu/documents/product-information/darzalex-epar-product-information_en.pdf
Last accessed May 2019.

3 ClinicalTrials.gov. A Study of Subcutaneous Versus (vs.)
Intravenous Administration of Daratumumab in Participants With Relapsed
or Refractory Multiple Myeloma. NCT03277105. Available at: https://clinicaltrials.gov/ct2/show/NCT03277105
Last accessed May 2019.

4 Sanchez L, Wang Y, Siegel DS, Wang ML. Daratumumab: a
first-in-class CD38 monoclonal antibody for the treatment of multiple
myeloma. J Hematol Oncol. 2016;9:51.

5 Fedele G, di Girolamo M, Recine U, et al. CD38 ligation in
peripheral blood mononuclear cells of myeloma patients induces release
of protumorigenic IL-6 and impaired secretion of IFNgamma cytokines and
proliferation. Mediat Inflamm. 2013;2013:564687.

6 ClinicalTrials.gov. A study to evaluate daratumumab in
transplant eligible participants with previously untreated multiple
myeloma (Cassiopeia). NCT02541383. Available at: https://clinicaltrials.gov/ct2/show/NCT02541383
Last accessed May 2019.

7 ClinicalTrials.gov. A study comparing daratumumab,
lenalidomide, and dexamethasone with lenalidomide and dexamethasone in
relapsed or refractory multiple myeloma. NCT02076009. Available at: https://clinicaltrials.gov/ct2/show/NCT02076009
Last accessed May 2019.

8 ClinicalTrials.gov. Addition of daratumumab to combination
of bortezomib and dexamethasone in participants with relapsed or
refractory multiple myeloma. NCT02136134. Available at: https://clinicaltrials.gov/ct2/show/NCT02136134
Last accessed May 2019.

9 ClinicalTrials.gov. A study of combination of daratumumab
and Velcade (bortezomib) melphalan-prednisone (DVMP) compared to Velcade
melphalan-prednisone (VMP) in participants with previously untreated
multiple myeloma. NCT02195479. Available at: https://clinicaltrials.gov/ct2/show/NCT02195479
Last accessed May 2019.

10 ClinicalTrials.gov. Study comparing daratumumab,
lenalidomide, and dexamethasone with lenalidomide and dexamethasone in
participants with previously untreated multiple myeloma. NCT02252172.
Available at: https://clinicaltrials.gov/ct2/show/NCT02252172
Last accessed May 2019.

11 ClinicalTrials.gov. A study of Velcade (bortezomib)
melphalan-prednisone (VMP) compared to daratumumab in combination with
VMP (D-VMP), in participants with previously untreated multiple myeloma
who are ineligible for high-dose therapy (Asia Pacific region).
NCT03217812. Available at: https://clinicaltrials.gov/ct2/show/NCT03217812
Last accessed May 2019.

12 ClinicalTrials.gov. Comparison of pomalidomide and
dexamethasone with or without daratumumab in subjects with relapsed or
refractory multiple myeloma previously treated with lenalidomide and a
proteasome inhibitor daratumumab/pomalidomide/dexamethasone vs
pomalidomide/dexamethasone (EMN14). NCT03180736. Available at: https://clinicaltrials.gov/ct2/show/NCT03180736
Last accessed May 2019.

13 ClinicalTrials.gov. Study of carfilzomib, daratumumab and
dexamethasone for patients with relapsed and/or refractory multiple
myeloma (CANDOR). NCT03158688. Available at: https://clinicaltrials.gov/ct2/show/NCT03158688
Last accessed May 2019.

14 ClinicalTrials.gov. A study to evaluate 3 dose schedules
of daratumumab in participants with smoldering multiple myeloma.
NCT02316106. Available at: https://clinicaltrials.gov/ct2/show/NCT02316106
Last accessed May 2019.

15 ClinicalTrials.gov. An efficacy and safety proof of
concept study of daratumumab in relapsed/refractory mantle cell
lymphoma, diffuse large B-cell lymphoma, and follicular lymphoma.
NCT02413489. Available at: https://clinicaltrials.gov/ct2/show/NCT02413489
Last accessed May 2019.

16 Johnson & Johnson. Janssen Biotech announces global
license and development agreement for investigational anti-cancer agent
daratumumab. Press release August 30, 2012. Available at: https://www.jnj.com/media-center/press-releases/janssen-biotech-announces-global-license-and-development-agreement-for-investigational-anti-cancer-agent-daratumumab
Last accessed May 2019.

17 American Society of Clinical Oncology. Multiple myeloma:
introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction
Last accessed May 2019.

18 GLOBOCAN 2018. Cancer Today Population Factsheets: Europe
Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf
Last accessed May 2019.

19 De Angelis R, Minicozzi P, Sant M, et al. Survival
variations by country and age for lymphoid and myeloid malignancies in
Europe 2000-2007: results of EUROCARE-5 population-based study. Eur J
Cancer.
2015;51:2254-68.

20 Abdi J, Chen G, Chang H, et al. Drug resistance in
multiple myeloma: latest findings and new concepts on molecular
mechanisms. Oncotarget. 2013;4:2186–207.

21 National Cancer Institute. NCI dictionary of cancer terms:
refractory. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245
Last accessed May 2019.

22 Richardson P, Mitsiades C, Schlossman R, et al. The
treatment of relapsed and refractory multiple myeloma. Hematology Am
Soc Hematol Educ Program
. 2007:317-23.

23 National Cancer Institute. NCI dictionary of cancer terms:
relapsed. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866
Last accessed May 2019.

24 American Cancer Society. Multiple myeloma: early
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25 Kumar SK, Lee JH, Lahuerta JJ, et al. Risk of progression
and survival in multiple myeloma relapsing after therapy with IMiDs and
bortezomib: a multicenter international myeloma working group study. Leukemia.
2012;26:149-57.

June 2019

CP-94167

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