Janssen Announces Investigational CAR-T Therapy JNJ-68284528 Granted PRIME Designation by the European Medicines Agency

April 4, 2019 Off By BusinessWire

PRIME (PRIority MEdicines) designation based on clinical study
results evaluating safety and efficacy of novel CAR-T therapy in the
treatment of patients with advanced relapsed or refractory multiple
myeloma

BEERSE, Belgium–(BUSINESS WIRE)–The Janssen Pharmaceutical Companies of Johnson & Johnson today
announced that the European Medicines Agency (EMA) has granted a PRIME
(PRIority MEdicines) designation for the company’s investigational
B-cell maturation antigen (BCMA) chimeric antigen receptor T-cell
(CAR-T) therapy, JNJ-68284528 (JNJ-4528). PRIME offers enhanced
interaction and early dialogue to optimise development plans and speed
up evaluation of cutting-edge, scientific advances that target a high
unmet medical need.¹

“The PRIME designation of this novel BCMA CAR-T therapy highlights the
value of regulatory innovation in the European Union,” said Sjaak Bot,
Vice President, Head EMEA Regulatory Affairs at Janssen Biologics B.V.
“We hope to bring this important advance to patients as quickly as
possible and this PRIME designation, the first for Janssen, marks an
important milestone towards potential market approval.”

The PRIME designation is based on results from the Phase 1/2 LEGEND-2
study (NCT03090659)
evaluating LCAR-B38M CAR-T cells, sponsored by Nanjing Legend Biotech
Co.,² and the Phase 1b/2 CARTITUDE-1 study (NCT03548207)
evaluating JNJ-4528, sponsored by Janssen and being conducted in
collaboration with Legend Biotech USA Inc.³ Results from the
LEGEND-2 study were presented
at the American Society of Hematology (ASH) 2018 annual meeting.⁴
Results from the CARTITUDE-1 study will be presented in the future.

“CAR-T therapy is an exciting therapeutic platform that harnesses the
patient’s immune system to attack tumour cells,” said Sen Zhuang, M.D.,
Ph.D., Vice President, Oncology Clinical Development, Janssen Research &
Development, LLC. “We continue to advance this novel BCMA targeted CAR-T
therapy through clinical studies globally as we strive to bring it to
the patients with multiple myeloma around the world.”

JNJ-4528 is currently being investigated for the treatment of patients
with multiple myeloma who have received at least three prior regimens,
including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD),
and an anti-CD38 antibody, and have documented disease progression
within 12 months of starting the most recent therapy, or are double
refractory to an IMiD and PI.³ These patients have few
available treatment options and are often faced with poor outcomes.⁵

In December
2017, Janssen entered into a worldwide collaboration and licence
agreement with Legend Biotech to jointly develop and commercialise
LCAR-B38M in multiple myeloma.⁶ In China, the Phase 2
CARTIFAN-1 confirmatory trial (NCT03758417),
sponsored by Nanjing Legend Biotech Co. Ltd. and registered with the
Center for Drug Evaluation (CTR20181007), is actively recruiting to
further evaluate LCAR-B38M in patients with advanced relapsed or
refractory multiple myeloma.⁷

About LEGEND-2

LEGEND-2 (NCT03090659)
is an ongoing single-arm, open-label Phase 1/2 study being conducted at
four participating hospitals in China evaluating the efficacy and safety
of LCAR-B38M for the treatment of relapsed or refractory multiple
myeloma.²

About CAR-T and BCMA

CAR T-cells are an innovative approach to eradicating cancer cells by
harnessing the power of a patient’s own immune system. BCMA is a protein
that is highly expressed on myeloma cells.⁸ By targeting BCMA
via this approach, CAR-T therapies may have the potential to redefine
treatment for multiple myeloma.

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer that starts in the bone
marrow and is characterised by an excessive proliferation of plasma
cells.⁹In Europe, more than 48,200 people were diagnosed
with multiple myeloma in 2018, and more than 30,800 patients died.¹⁰
Almost 40 percent of patients with multiple myeloma do not reach
five-year survival.¹¹

Although treatment may result in remission, unfortunately, patients will
most likely relapse as there is currently no cure.¹²
Refractory multiple myeloma is when a patient’s disease is
non-responsive or progresses within 60 days of their last therapy.^13,14
Relapsed myeloma is when the disease has returned after a period of
initial, partial or complete remission and does not meet the definition
of being refractory.¹⁵ While some patients with multiple
myeloma 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.¹⁶Patients who
relapse after treatment with standard therapies, including PIs and
IMiDs, have poor prognoses and few treatment options available.¹⁷

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 Biologics B.V. and Janssen Research &
Development, LLC 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
product development and the potential benefits and treatment impact of
LCAR-B38M and JNJ-68284528. 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
projections of Janssen Biologics B.V., Janssen Research & Development,
LLC, any of the Janssen Pharmaceutical Companies of Johnson & Johnson
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 31, 2017, including in the sections
captioned “Cautionary Note Regarding Forward-Looking Statements” and
“Item 1A. Risk Factors,” and in the company’s subsequent Quarterly
Reports on Form 10-Q and other 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. None of the Janssen Pharmaceutical
Companies nor Johnson & Johnson undertakes to update any forward-looking
statement as a result of new information or future events or
developments.

###

References

¹ European Medicines Agency. PRIME Factsheet. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines
Last accessed March 2019.

² ClinicalTrials.gov. LCAR-B38M-02 cells in treating
relapsed/refractory (R/R) multiple myeloma (LEGEND-2). NCT03090659.
Available at: https://clinicaltrials.gov/ct2/show/NCT03090659
Last accessed March 2019.

³ ClinicalTrials.gov. A Study of JNJ-68284528, a Chimeric
Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell
Maturation Antigen (BCMA) in Participants With Relapsed or Refractory
Multiple Myeloma. NCT03548207. Available at: https://clinicaltrials.gov/ct2/show/NCT03548207
Last accessed March 2019.

⁴ Zhao WH, Liu J, Wang BY, et al. Updated analysis of a phase
1, open-label Study of LCAR-B38M, a chimeric antigen receptor T-cell
therapy directed against B-cell maturation antigen, in patients with
relapsed/refractory multiple myeloma. Presented at 60th Annual Meeting
and Exposition of the American Society of Hematology (ASH), San Diego,
CA, USA, 1-4 December 2018: Abstract 955.

⁵ Castella, M., Fernández de Larrea, C. and Martín-Antonio,
B., 2018. Immunotherapy: a novel era of promising treatments for
multiple myeloma. International journal of molecular sciences, 19(11),
p.3613.

⁶ Johnson & Johnson. Janssen enters worldwide collaboration
and license agreement with Chinese company Legend Biotech to develop
investigational CAR-T anti-cancer therapy. Press release December 21,
2017. Available at: https://www.jnj.com/media-center/press-releases/janssen-enters-worldwide-collaboration-and-license-agreement-with-chinese-company-legend-biotech-to-develop-investigational-car-t-anti-cancer-therapy
Last accessed March 2019.

⁷ ClinicalTrials.gov. A Study of LCAR-B38M CAR-T Cells, a
Chimeric Antigen Receptor T-cell (CAR-T) Therapy Directed Against B-cell
Maturation Antigen (BCMA) in Chinese Participants With Relapsed or
Refractory Multiple Myeloma. NCT03758417. Available at: https://clinicaltrials.gov/ct2/show/NCT03758417
Last accessed March 2019.

⁸ Cho SF, Anderson KC, Tai YT. Targeting B-cell maturation
antigen (BCMA) in multiple myeloma: potential uses of BCMA-based
immunotherapy. Front Immunol. 2018;9:18-21.

⁹ American Society of Clinical Oncology. Multiple myeloma:
introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction
Last accessed March 2019.

¹⁰ 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 March 2019.

¹¹ 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.

¹² Abdi J, Chen G, Chang H, et al. Drug resistance in
multiple myeloma: latest findings and new concepts on molecular
mechanisms. Oncotarget. 2013;4:2186–2207.

¹³ National Cancer Institute. NCI dictionary of cancer terms:
refractory. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245
Last accessed March 2019.

¹⁴ 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.

¹⁵ National Cancer Institute. NCI dictionary of cancer terms:
relapsed. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866
Last accessed March 2019.

¹⁶ American Cancer Society. Multiple myeloma: early
detection, diagnosis and staging. Available at: https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf
Last accessed March 2019.

¹⁷ 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.

CP-80772

April 2019