1. POMALYST®
(pomalidomide)
Drug Showcase

2. Introduction
 Brand name: POMALYST®
 Generic name: pomalidomide
 Approval: 2013
 Manufacturer: Celgene Corp

3. Indication, Dosage & Administration

4. Indication
 POMALYST is indicated for the treatment of multiple myeloma in patients
who have:
◦ Received at least two prior therapies, including lenalidomide and
bortezomib, and
◦ Demonstrated disease progression on or within 60 days of completion of
the last therapy

5. Dosage and Administration
 Recommended Dose
◦ 4 mg orally once daily on Days 1-21 of repeated 28-day cycle
 Administration
◦ To be administered until disease progression
◦ Must be administered with dexamethasone
◦ Must be taken with water
◦ Should be taken without food (either 2 hours before or after a meal)
 Dose Modifications
◦ Please see full Prescribing Information for dose modification guidelines
associated with:
 Hematologic toxicities: neutropenia or thrombocytopena
◦ Grade 3 or 4 toxicities (other than neutropenia or thrombocytopenia)
 Hold POMALYST
 When toxicity has resolved to Grade 2 or less, restart at 1 mg less than
previous dose
 Discontinue if toxicities occur after dose reduction to 1 mg

6. Use in Specific Populations

7. Considerations for Special Populations
Patient Group Recommendations
Pregnancy Category X
• POMALYST is contraindicated during pregnancy
• If used during pregnancy or if patient becomes
pregnant during treatment, patient should be apprised
of the potential hazard to the fetus
• If pregnancy occurs during treatment, discontinue
drug
Nursing mothers • It is unknown whether or not POMALYST is excreted in
human milk
• Decision to discontinue nursing or discontinue therapy
should be made considering the importance of the
drug to the mother
Pediatric Safety and effectiveness has not been established in
patients <18 years of age

8. Considerations for Special Populations (cont.)
Patient Group Recommendations
Females of
Reproductive
Potential & Males
• Females of reproductive potential must avoid
pregnancy by abstaining or using two methods of
reliable birth control
• Contraception should begin 4 weeks before initiating
POMALYST, during therapy and 4 weeks after
discontinuing therapy
Geriatric • No dose adjustment is required based on age
• In the study, patients >65 years of age were more
likely to experience pneumonia
Renal Impairment • Safety and efficacy have not been evaluated
• Avoid use in patients with serum creatinine >3 mg/dL
Hepatic Impairment • Safety and efficacy have not been evaluated
• Avoid use in patients with serum bilirubin >2 mg/dL
and AST/ALT >3x upper limits of normal

9. Clinical Pharmacology

10. Mechanism of Action
 POMALYST is an immunomodulatory agent with antineoplastic activity
◦ Thalidomide analogue
 As shown in in vitro celullar assays
◦ Inhibited proliferation of lenalidomide-resistant multiple myeloma cell
lines
◦ Induced tumor cell apoptosis when combined with dexamethasone in
both lenalidomide-sensitive and lenalidomide-resistant cell lines
◦ Enhanced T cell- and natural killer cell-mediated activity
◦ Inhibited monocyte production of pro-inflammatory cytokines (eg, TNF-
alpha and interluekin-6)

11. Pharmacokinetics
Parameters Values
Time to Peak 2-3 hours
Volume of distribution 62L-138L (steady state)
Protein binding • 12% to 44%
• Not concentration dependent
Metabolism • Hepatic
• Primarily via CYP1A2, CYP3A4
Half-life elimination • 9.5 hours (healthy patients)
• 7.5 hours (multiple myeloma patients)
Excretion • Feces: 15%
• Urine: 73%

12. Drug Interactions
 No formal studies have been completed
 CYP3A, CYP1A2, P-gp inhibitors
◦ Co-administration with strong CYP3A, CYP1A2, or P-gp inhibitors could
increase exposure to POMALYST
◦ Combination should be avoided
 CYP3A, CYP1A2, P-gp inducers
◦ Co-administration with strong CYP3A, CYP1A2, or P-gp inducers could
decrease POMALYST exposure
◦ Combination should be avoided
 Dexamethasone
◦ Did not affect POMALYST pharmacokinetics
 Smoking
◦ May reduce effectiveness of POMALYST

13. Warnings & Precautions

14. Contraindications, Warnings and Precautions
 Contraindications
◦ POMALYST is contraindicated in women who are pregnant
◦ If drug is used during pregnancy or patient becomes pregnant, patient should
be apprised of the risk to the fetus
 Embryo-Fetal Toxicity
◦ POMALYST is a thalidomide-analogue; thalidomide causes severe birth defects
or embryo-fetal death
◦ Pregnancy should be avoided during POMALYST therapy and at least 4 weeks
after completing therapy
◦ POMALYST is present in male semen
 Males must always use latex or synthetic condoms during sexual contact
with females of reproductive potential during and 28 days after therapy
 Males taking POMALYST should not donate sperm
◦ Blood donation
 Blood should not be donated during treatment and up to 1 month after
completing treatment since exposed blood may be transferred to a pregnant
female

15. POMALYST REMS™ Program
 Due to embyro-fetal risk, POMALYST is only available through POMALYST
REMS
 Requirements include:
◦ Prescribers must be certified with the REMS program
◦ Patients must sign a Patient-Prescriber agreement and comply with
REMS requirements
 Includes pregnancy testing and contraception requirements
◦ Pharmacist must be certified with the REMS program
 May only dispense POMALYST to patients authorized to receive
POMALYST
15

16. Warnings and Precautions
 Venous Thromboembolism
◦ Consider concomitant anti-
coagulation prophylaxis based on
patient’s risk
 Hematologic Toxicity
◦ Neutropenia, anemia, and
thrombocytopenia were most
commonly reported
◦ Monitor complete blood counts
weekly for first 8 weeks, then
monthly thereafter
◦ Dose may require adjustment or
holding
 Hypersensitivity
◦ Risk may be higher in patients
who experienced hypersensitivity
with thalidomide or lenalidomide
 Dizziness and Confusional State
◦ Patients should avoid any
medications or situations that
may cause dizziness or lead to
confusion without proper medical
advice
 Neuropathy
◦ Patients may experience
neuropathy or peripheral
neuropathy
 Second Primary Malignancy Risk
◦ Cases of acute myelogenous
leukemia have been reported in
patients receiving POMALYST
16

17. Clinical Safety & Efficacy

18. Trial 1 Information
 Objective
◦ Evaluate the safety and efficacy of POMALYST alone or in combination with low
dose dexamethasone (Low-dose Dex)
 Study Design
◦ Multicenter, randomized, open label, phase 2 study
 Patient Population and Treatment
◦ Patients enrolled had relapsed multiple myeloma, were refractory to their last
myeloma therapy and received lenalidomide and bortezomib
 Relapse was defined as achieving at least stable disease for at least one
treatment cycle to at least one prior regimen before disease progressed
 Refractory was defined as disease progression on or within 60 days of last
therapy
◦ Regimen: POMALYST 4mg, once daily for 21 days (of 28-day cycle), alone or
in combination with low dose dexamethasone (Low dose Dex)
 Low dose Dex schedule: Days 1, 8, 15 and 22 of 28-day cycle
 Patients ≤75 years of age received 40 mg daily; >70 years of age received
20 mg daily

19. Trial 1: Results
 Overall response rate did not differ based on prior anti-myeloma therapy
19
Response POMALYST*
(N=108)
POMALYST/Low dose
Dex (N=113)
Overall Response Rate,
n (%)
(95% CI)
8 (7.4%)
(3.3-14.1)
33 (29.2%)
(21.0-38.5)
Complete Response,
n (%)
0 (0%) 1 (0.9%)
Partial Response,
n (%)
8 (7.4%) 32 (28.3%)
Duration of Response,
Median (months),
(95% CI)
NE 7.4
(5.1-9.2)
*Results are prior to the addition of dexamethasone.
CI=confidence interval; NE=not established (median has not yet been reached).

20. Common Adverse Events, >25%
POMALYST
(N = 107)
POMALYST + Low dose Dex
(N=112)
• Fatigue and asthenia (55%)
• Neutropenia (52%)
• Anemia (38%)
• Thrombocytopenia (25%)
• Constipation (36%)
• Diarrhea (34%)
• Nausea (36%)
• Upper respiratory tract infection
(32%)
• Back pain (32%)
• Dyspnea (34%)
• Fatigue and asthenia (63%)
• Pyrexia (30%)
• Neutropenia (47%)
• Anemia (39%)
• Constipation (35%)
• Diarrhea (33%)
• Pneumonia (29%)
• Upper respiratory tract infection
(25%)
• Back pain (30%)
• Dyspnea (45%)

21. Drug Storage & Supply

22. Supply and Pricing Specifications
 How supplied
o 1-mg, 2-mg, 3-mg, and 4-mg capsules: 21-count and 100-
count bottle
 Storage and Handing
◦ Store at room temperature
◦ Capsules should not be opened or crushed
◦ If powder comes in contact with skin, wash immediately with
soap and water
◦ If powder comes in contact with mucous membranes, flush with
water
 Drug Pricing
o AWP/unit price: $596.88/capsule

23. References
 POMALYST® (pomalidomide) [package insert]. Celgene
Corporation. Summit, NJ.
http://pomalyst.com/docs/prescribing_information.pdf
 Truven Health Analytics. Red Book Online® Search. Micromedex.