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Celgene Presents Data from a Phase 1/2 Clinical Study of Iberdomide in Combination with Dexamethasone in Patients with Relapsed and Refractory Multiple Myeloma at ASCO 2019

Investigational treatment demonstrates favorable early safety and
efficacy data in patients who had received a median five prior
treatments including immunomodulatory agents, proteasome inhibitors and
anti-CD38 agents

SUMMIT, N.J.–(BUSINESS WIRE)–Celgene Corporation (NASDAQ: CELG) today announced the first clinical
results evaluating iberdomide (CC-220) in combination with dexamethasone
in patients with relapsed and refractory multiple myeloma from the
ongoing phase 1/2 CC-220-MM-001 study during an oral presentation at the
2019 American Society of Clinical Oncology (ASCO) Annual Meeting in
Chicago.

The results included preliminary safety and efficacy data from the
ongoing multicenter, open-label, dose-escalation study, which aims to
determine the maximum tolerated dose and the recommended phase 2 dose of
iberdomide in combination with dexamethasone. Iberdomide is Celgene’s
proprietary cereblon E3 ligase modulator (CELMoD®) compound
with enhanced tumoricidal and immune stimulatory effects demonstrated in
preclinical studies. The phase 1/2 study is expected to enroll
approximately 300 participants.

“Nearly half a million people globally are affected by multiple myeloma,
a cancer of plasma cells. The management of patients with late relapsed
or refractory multiple myeloma continues to be challenging due to the
complex nature of the disease’s pathophysiology. Despite the
introduction of newer agents, patients continue to experience disease
relapse therefore new therapeutic options are needed for patients who
have failed multiple prior treatments,” said Sagar Lonial, MD, Chief
Medical Officer at Winship Cancer Institute of Emory University. “The
early data on iberdomide in combination with dexamethasone in these
heavily pretreated patients show promising activity, and we look forward
to advancing our understanding of this combination’s potential in this
patient population.”

As of April 2019, 66 patients at a median age of 65 received the
iberdomide plus dexamethasone combination, with iberdomide being
administered in 8 incremental doses ranging from 0.3 mg to 1.3 mg.
Escalating doses of iberdomide were given on days 1 through 21 in
combination with dexamethasone (40 mg; 20 mg in patients older than 75)
on days 1, 8, 15, and 22 of each 28-day cycle. Patients had a median of
five prior multiple myeloma treatment regimens, which could have
included stem cell transplant, immunomodulatory drugs including
lenalidomide and pomalidomide, proteasome inhibitors and daratumumab.

Grade 3-4 adverse events (AE) reported included neutropenia (29%),
infection (26%), anemia (24%), thrombocytopenia (12%), pulmonary
embolism (1.5%) and peripheral sensory neuropathy (1.5%). Six patients
(9%) discontinued treatment due to adverse events.

Of the 66 patients who received the iberdomide plus dexamethasone
combination, 59 were evaluable for response. The overall response rate
was 32% (19/59), with 29% (17/59) achieving a partial response and two
patients achieving a very good partial response.

Patients (n=51) who were refractory to IMiD® compounds, which
included lenalidomide and pomalidomide, had an overall response rate of
35% (18/51) with 33% (17/51) of patients achieving a partial response
and one patient achieving a very good partial response. Further,
patients who were refractory to both daratumumab and pomalidomide (n=27)
had an overall response rate of 29% (8/27), with 25% (7/27) achieving a
partial response and one patient achieving a very good partial response.
Maximum tolerated dose and the recommended phase 2 dose have not yet
been determined.

“While we have made tremendous progress in treating multiple myeloma,
there is still a significant need for new options to address the
heterogeneous nature of the disease. We are focused on exploring the
potential of our next generation CELMoD compounds to help fill this
gap,” said Dr. Alise Reicin, President, Global Clinical Development for
Celgene. “The preliminary clinical activity and favorable safety data
observed with the combination of iberdomide and dexamethasone are
encouraging, particularly in patients who have failed multiple lines of
therapy including patients who were refractory to lenalidomide,
pomalidomide and/or daratumumab.”

The phase 1/2 study is also evaluating iberdomide as monotherapy and in
combination with daratumumab or bortezomib or carfilzomib. Iberdomide is
investigational and has not been approved in any country.

About Iberdomide (CC-220)

Iberdomide is an investigational cereblon E3 ligase modulator (CELMoD®)
compound that induces degradation of transcription factors Aiolos and
Ikaros, thereby inhibiting growth of myeloma cells in vitro. In
pre-clinical models, iberdomide has demonstrated ability to destroy
tumor cells, stimulate an immune response, overcome resistance to
immunomodulatory drugs, and synergize with dexamethasone, daratumumab
and bortezomib.

About CC-220-MM-001

The open-label phase 1/2 CC-220-MM-001 dose escalation study
(NCT02773030) seeks to determine the maximum tolerated dose and
recommended phase 2 dose of iberdomide (CC-220) as monotherapy and in
combination with dexamethasone, as well as further evaluate safety and
preliminary efficacy in patients with relapsed/refractory multiple
myeloma. The study consists of a dose-escalation portion (Part 1) as
well as an expansion of these two cohorts at the recommended phase 2
dose to further evaluate safety and estimate preliminary efficacy (Part
2). The study also seeks to establish the maximum tolerated dose and
recommended phase 2 dose of iberdomide when administered in combination
with daratumumab or bortezomib or carfilzomib.

About Multiple Myeloma

Multiple myeloma is a life-threatening blood cancer that is
characterized by tumor proliferation and suppression of the immune
system.1 It is a rare but deadly disease – more than 32,000
new cases will be diagnosed in the United States in 2019, and
approximately 13,000 deaths will occur in 2019, representing about 2.1%
of all cancer-related deaths.2 The typical multiple myeloma
disease course includes periods of symptomatic myeloma followed by
periods of remission, and eventually, the disease becomes refractory
(nonresponsive).

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an
integrated global biopharmaceutical 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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company is unable to retain key personnel.

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1 Palumbo A and Anderson K. Multiple myeloma. N Engl J Med.
2011;364:1046-1060.
2 National Cancer Institute. Cancer
Stat Facts: Myeloma. Available at: https://seer.cancer.gov/statfacts/html/mulmy.html.
Accessed April 2019.

Contacts

Celgene Corporation
Investors:
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ir@celgene.com
or

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