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US FDA and EMA Accept Applications for Ozanimod for the Treatment of Relapsing Forms of Multiple Sclerosis

SUMMIT, N.J.–(BUSINESS WIRE)–$CELG #MS–Celgene Corporation (NASDAQ:CELG) today announced that the U.S. Food and
Drug Administration (FDA) has accepted for review the New Drug
Application for ozanimod for the treatment of people with relapsing
forms of multiple sclerosis (RMS) in the United States. The European
Medicines Agency (EMA) also accepted for review the Marketing
Authorization Application for ozanimod for the treatment of adults with
relapsing-remitting multiple sclerosis (RRMS) in the European Union.
Ozanimod is an oral, sphingosine 1-phosphate (S1P) receptor modulator
that binds with high affinity selectively to S1P subtypes 1 (S1P1)
and 5 (S1P5). Under the Prescription Drug User Fee Act, the
FDA has set its action date as March 25, 2020. A regulatory decision
from the EMA is expected in the first half of 2020.

Both applications are based primarily on ozanimod data from the SUNBEAM™
and RADIANCE™ Part B phase 3, multicenter, randomized, double-blind,
double-dummy, active-controlled trials.

“The U.S. Food and Drug Administration and European Medicines Agency
acceptances of our applications represent a crucial step forward in our
efforts to bring ozanimod to people with multiple sclerosis,” said Jay
Backstrom, M.D., Chief Medical Officer for Celgene. “We believe that
ozanimod has the potential to be an important option early in the
treatment of relapsing forms of MS and a best-in-class S1P receptor
modulator.”

Ozanimod is an investigational compound that is not approved for any use
in any country.

About SUNBEAM™

SUNBEAM is a pivotal, phase 3, multicenter, randomized, double-blind,
double-dummy, active-controlled trial evaluating the efficacy, safety
and tolerability of two doses of oral ozanimod (0.92 mg and 0.46 mg,
equivalent to 1 mg and 0.5 mg ozanimod HCI respectively) against weekly
intramuscular interferon beta-1a (Avonex®) for at least a
12-month treatment period. The study included 1,346 people living with
RMS across 152 sites in 20 countries.

The primary endpoint of the trial was annualized relapse rates (ARR)
during the treatment period. The secondary MRI endpoints included the
number of new or enlarging hyperintense T2-weighted brain MRI lesions
over 12 months, number of gadolinium-enhanced brain MRI lesions at month
12 and percent change from baseline in whole brain volume at month 12.
Cortical grey and thalamic volume changes were also prospectively
assessed versus active comparator.

An analysis of the time to onset of 3-month confirmed disability
progression was pre-specified using pooled data from both the SUNBEAM
and RADIANCE Part B phase 3 trials.

About RADIANCE™

RADIANCE Part B is a pivotal, phase 3, multicenter, randomized,
double-blind, double-dummy, active-controlled trial evaluating the
efficacy, safety and tolerability of two doses of oral ozanimod (0.92 mg
and 0.46 mg, equivalent to 1 mg and 0.5 mg ozanimod HCI respectively)
against weekly intramuscular interferon beta-1a (Avonex®)
over a 24-month treatment period. The study included 1,320 people living
with RMS across 150 sites in 21 countries.

The primary endpoint of the trial was ARR over 24 months. The secondary
MRI endpoints included the number of new or enlarging hyperintense
T2-weighted brain MRI lesions over 24 months, number of
gadolinium-enhanced brain MRI lesions at month 24 and percent change
from baseline in whole brain volume at month 24. Cortical grey and
thalamic volume changes were also prospectively assessed versus active
comparator.

An analysis of the time to onset of 3-month confirmed disability
progression was pre-specified using pooled data from both the SUNBEAM
and RADIANCE Part B phase 3 trials.

About Ozanimod

Ozanimod is an oral, sphingosine 1-phosphate (S1P) receptor modulator
that binds with high affinity selectively to S1P subtypes 1 (S1P1)
and 5 (S1P5). Ozanimod causes lymphocyte retention in
lymphoid tissues. The mechanism by which ozanimod exerts therapeutic
effects in multiple sclerosis is unknown, but may involve the reduction
of lymphocyte migration into the central nervous system.

Ozanimod is in development for immune-inflammatory indications including
RMS, ulcerative colitis and Crohn’s disease.

About Multiple Sclerosis

Multiple sclerosis (MS) is a disease in which the immune system attacks
the protective myelin sheath that covers the nerves. The myelin damage
disrupts communication between the brain and the rest of the body.
Ultimately, the nerves themselves may deteriorate — a process that’s
currently irreversible. Signs and symptoms vary widely, depending on the
amount of damage and the nerves affected. Some people living with MS may
lose the ability to walk independently, while others experience long
periods of remission during which they develop no new symptoms. MS
affects approximately 400,000 people in the U.S. and approximately 2.5
million people worldwide.

RMS is characterized by clearly defined attacks of worsening neurologic
function. These attacks — often called relapses, flare-ups or
exacerbations — are followed by partial or complete recovery periods
(remissions), during which symptoms improve partially or completely with
no apparent progression of disease. RMS is the most common disease
course at the time of diagnosis. Approximately 85 percent of patients
are initially diagnosed with RMS, compared with 10-15 percent with
progressive forms of the disease.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an
integrated global pharmaceutical company engaged primarily in the
discovery, development and commercialization of innovative therapies for
the treatment of cancer and inflammatory diseases through
next‐generation solutions in protein homeostasis, immuno‐oncology,
epigenetics, immunology and neuro‐inflammation. For more information,
please visit www.celgene.com.
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For inquiries, please contact:

Investors:
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Executive Director, Investor Relations
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