Bob Duggan' Pharmacyclics' Presentation

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Pharmacyclics
Corporate Presentation
March 2014

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During the course of this presentation we will make statements that constitute
forward-looking statements. These statements may include operating expense
projections, the initiation, timing and results of pending or future clinical trials,
the actions or potential action of the FDA, the status and timing of ongoing
research, corporate partnering activities and other factors affecting
Pharmacyclics’ financial condition or operations. Such forward-looking
statements are not guarantees of future performance and involve risks,
uncertainties and other factors that may cause actual results, performance or
achievements to vary materially from those expressed or implied in such
statements. These and other risk factors are listed from time to time in reports
filed with the Securities and Exchange Commission (SEC), including but not
limited to, reports on Forms 10-Q and 10-K. Pharmacyclics does not intend to
update any forward-looking information to reflect actual results or changes in
the factors affecting the forward-looking information.
Safe Harbor Statement

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Making a difference for the betterment of patients
Our Mission
To build a viable biopharmaceutical company that
designs, develops and commercializes novel therapies
intended to improve quality of life, increase duration of life
and resolve serious medical healthcare needs.
To identify and control promising product candidates
based on exceptional scientific development and
administrational expertise, develop our products in a
rapid, cost-efficient manner and to pursue
commercialization and/or development partners when and
where appropriate.
We exist to make a difference for the better and these are
important times to do just that.

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PCYC Corporate Development
Jan 2006 to Dec 2008
$0
$1
$2
$3
$4
$5
$6
Share Price
$0.79
$5.07
$4.92
$1.45
04/10/06:
Assumption of
Celera compounds
BTK, FVIIa, HDAC
09/11/08:
PCYC Board &
Management
Transition
09/19/07:
Bob Duggan
joins PCYC
Board 05/01/08:
Offer to purchase
4M shares at
$1.05 /share
by RW Duggan
10/21/07:
FDA Non‐
approvable
letter for Xcytrin
02/22/07:
FDA Refusal to
file letter for
Xcytrin

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PCYC Corporate Development
Jan 2009 to Mar 2014
$0
$20
$40
$60
$80
$100
$120
$140
$160
Share Price
02/12/14:
IMBRUVICA
approved for CLL
pts w/ at least one
prior therapy.
$57.78
$3.14
$6.08
$14.828/5/2009
Rights Offering
22.5M shares
sold at $1.28
07/17/11:
Secondary
Offering
6.5M shares
sold at $8.85
11/13/13:
IMBRUVICA
approved for MCL
pts w/ at least one
prior therapy.
PCYC's first label.
12/31/08
Price: $0.79
Employees: 47
Mkt Cap: $20M
W.Cap: $7.2M
$6M loan by
R.W. Duggan
07/10/13: PCYC announced its first NDA filing
of ibrutinib, for treatment of patients with
R/R CLL and R/R MCL.
2/12/13: PCYC receives Breakthrough
Therapy Designation from the FDA for MCL
and WM. $70.37
12/8/11: PCYC
enters into
collaboration
agreement with
Janssen Biotech,
Inc.
6/21/10: PCYC
raises $50.8M
net proceeds in a
registered direct
offering.
$6.74

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IMBRUVICA Approved November 13, 2013 in MCL
and February 12, 2014 in CLL
4.5 years from 1st patient in to NDA filing 4.5 months from filing to first FDA approval

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“[IMBRUVICA] is a revolutionary
blood cancer drug.”
Headlines and Highlights….Approval Grabs Attention
of Top Tier Media
FDA speedily approves
Imbruvica, a treatment
for rare lymphoma
J&J-Pharmacyclics
Win U.S. Approval for
Breakthrough Drug

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IMBRUVICA (ibrutinib)
Leads Our Oncology Pipeline
Molecule & Program / Indication
Discovery /
Preclinical
Phase
I
Phase
II
Phase
III
Ibrutinib (PCI-32765): Bruton’s tyrosine kinase (BTK) inhibitor for Oncology *
Chronic lymphocytic leukemia
Mantle cell lymphoma
Diffuse large B-cell lymphoma
Multiple myeloma
Follicular lymphoma
Waldenstrom’s Macroglobulinemia
Abexinostat HCI (PCI-24781): Histone deacetylase (HDAC) inhibitor for Oncology **
Follicular lymphoma and mantle cell
lymphoma
PCI-27483: Factor VIIa Inhibitor for Oncology
Pancreatic cancer
BTK inhibitor for Autoimmune Diseases
Autoimmune disease
*Janssen Biotech: global partnership
** Servier: ex-U.S. partnership
APPROVED
APPROVED

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IMBRUVICA (Ibrutinib - PCI-32765)
• IMBRUVICA is an oral therapy that targets an important
pathway in B-cell malignancies. Over 2800 patients
treated in company sponsored clinical trials.
• IMBRUVICA was approved for the treatment of
patients with mantle cell lymphoma who have received
at least one prior therapy
• IMBRUVICA has demonstrated in clinical trials:
o Tumor reduction (response) in heavily pretreated patients
o Responses in patients previously treated with
chemotherapy and with aggressive disease
o Durable responses with many patients still on drug after
prolonged periods of time
o Patient tolerability demonstrated

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From: de Rooij et al, Blood 119: 2590-2594
Mechanism of BTK: IMBRUVICA (ibrutinib – PCI-32765)
Blocks Malignant B-cell Growth and Proliferation
Chronic Lymphocytic Leukemia Cell Lymph Node Peripheral Blood

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B-Cell Development and
Origin of B-cell Malignancies
Pre-B
Immature
B
Naïve
B
Germinal
Center B
Memory
B
Plasma
Cell
Malignant:
Leukemia
Chronic
Lymphocytic
Leukemia (CLL)
un-mutated Mantle Cell Lymphoma
(MCL)
Multiple
Myeloma
(MM)
Adopted from:
2012 Pan Pacific Lymphoma Conference
J Rubenstein, M.D., Ph.D.
Normal :
Immature antibody producing
Chronic
Lymphocytic
Leukemia
mutated
Follicular
Lymphoma (FL)
Diffuse Large B-Cell
Lymphoma (DLBCL)
Waldenstrom’s
Macroglobulinemia (WM)

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Patient Populations in
Major Hematology Malignancies
US All Major Markets*
Incidence Prevalence Incidence Prevalence
CLL/SLL 16,0001 114,5002 40,0001 259,0001
MCL 2,9001 11,3002 6,0001 37,0001
WM 1,5004 12,0002 6,0004 23,0004
DLBCL 25,0001 112,0002 53,0001 356,0001
FL 13,0001 63,0002 28,0001 240,0001
MM 20,0001 77,0001 48,0001 183,0001
TOTAL 82,500 390,0002 181,000 1,100,0001
1 © 2013 DR/Decision Resources, LLC. All rights reserved. Reproduction, distribution, transmission or publication is prohibited. Reprinted with permission. For the CLL diagnosed
incidence the US National Cancer Institute estimations were used
2 IMS patient claims estimates for July 2012‐June 2013. Note: This information is an estimate derived from the use of information under license from the following IMS Health Incorporated
information service: IMS Oncology Tracking Reports for the period July 2012 to June 2013. IMS expressly reserves all rights, including rights of copying, distribution and republication.
3 Major markets include: US, UK, Spain, Germany, France, Italy, and Japan 4 WM Foundation estimate
Pharmacyclics, Inc. makes no representation with respect to the accuracy or reliability of this information. Investors are advised to independently verify this information
before using it to make investment decisions.

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CLL/SLL & MCL US Patient Estimates
CLL/SLL MCL
Diagnosed Incidence 1 16,000 2,900
Prevalence 2 114,500 11,300
No Therapy 2 50,600 2,000
1L Therapy 2 23,200 3,900
2L Therapy 2 9,300 1,300
3L+ Therapy 2 6,100 800
*Other 2 (All these patients are between lines
of therapy; 1/3 received maintenance, 2/3 were
not on therapy in observation period)
25,300 3,300
1 © 2013 DR/Decision Resources, LLC. All rights reserved. Reproduction, distribution, transmission or publication is prohibited. Reprinted with permission. For the CLL
diagnosed incidence the US National Cancer Institute estimations were used.
2 IMS patient claims estimates for July 2012‐June 2013. Note: This information is an estimate derived from the use of information under license from the following IMS
Health Incorporated information service: IMS Oncology Tracking Reports for the period July 2012 to June 2013. IMS expressly reserves all rights, including rights of
copying, distribution and republication.
Pharmacyclics, Inc. makes no representation with respect to the accuracy or reliability of this information. Investors are advised to independently verify
this information before using it to make investment decisions.

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Breakthrough Therapy Designations
and Regulatory Progress
• IMBRUVICATM approved on November 13, 2013 for the treatment of
patients with MCL who have received at least one prior therapy and on
February 12, 2014 for the treatment of patients with CLL, who have
received at least one prior therapy.
• New Drug Application (NDA) submitted in relapsed/refractory MCL
and relapsed/refractory CLL on July 10, 2013. NDA was accepted on
August 27, 2013 - Priority Review was granted with a PDUFA of Feb
28, 2014.
• FDA approved Breakthrough Designations for IMBRUVICA in:
o Relapsed/Refractory Mantle Cell Lymphoma (MCL) Feb 2013
o Waldenstrom’s Macroglobulinemia (WM) Feb 2013
o Chronic Lymphocytic Leukemia (CLL) with deletion 17p Mar 2013

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Clinical Development Plan: Select Studies of
IMBRUVICA in CLL/SLL Patients
PCYC/JNJ
/ISTs
Phase Study ID Status
Line of
Therapy
# of
Patients
Trial 1st
Released
Study Design
CLL
I PCYC‐1108 compl RR 33 Feb‐11 i+FCR; i+BR
II
PCYC‐1103 active RR 200 Jun‐10 Roll Over Study
PCYC‐1117
RESONATE‐17
active RR 111 Jan‐13 Monotherapy in 17p
PCYC‐1102 compl TN/RR 133 May‐10 Monotherapy
Burger‐MDACC active RR 40 Feb‐12 i+R in high risk pts
Burger‐MDACC recruit RR 208 Dec‐13 i vs iR
III
PCYC‐1112
RESONATE
active RR 350 Jun‐12
i vs Ofa
(Cross‐over added 8/13/13)
PCYC‐1115
RESONATE‐2
active TN 272 Jan‐13 i vs Chlorambucil in Elderly
HELIOS recruit RR 580 Sept‐12 i+BR
CLL3002 not yet RR 150 Oct‐13 i vs R (in China)
Woyach not yet TN 523 Jun‐13 i vs iR vs BR in Elderly
CLL‐12 not yet TN 302 ASH ’13
i vs Placebo; Watch & Wait (German
Study Group)
ECOG not yet TN 519 ASH ’13 iR vs. FCR; Young Fit

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The Bruton’s Tyrosine Kinase (BTK) Inhibitor Ibrutinib (PCI-32765)
Monotherapy Demonstrates Long-Term Safety and Durability of
Response in Chronic Lymphocytic Leukemia/Small Lymphocytic
Lymphoma Patients in an Open-Label Extension Study (O’Brien, ASH 2013)
• A total of 148 patients TN or
R/R CLL/SLL received ibrutinib
monotherapy in a phase 1
multiple ascending dose study
(PCYC-04753)3 or phase 1b/2
continuous-dosing study
(PCYC-1102),4 and were
continued on a long-term
extension study for follow-up for
safety and efficacy with ibrutinib
monotherapy
• Median Duration of Response
(DOR) was not reached for
either Treatment Naïve (TN) or
Relapsed/Refractory (R/R)
responders achieving partial
response or better, after a
median follow-up of 28.1 and
23.9 months.
At 30 mos, 95.8% of TN and 69.7% of R/R
responders were alive without disease
progression.

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The Bruton’s Tyrosine Kinase (BTK) Inhibitor Ibrutinib (PCI-32765)
Monotherapy Demonstrates Long-Term Safety and Durability of
Response in Chronic Lymphocytic Leukemia/Small Lymphocytic
Lymphoma Patients in an Open-Label Extension Study (O’Brien, ASH 2013)
• The percentage of patients with
a serious Adverse Events grade
≥ 3 declined from 43% within
the first year of study treatment
to 32% after the 1st year of
treatment.
• Grade 3 AEs (severe) and serious
AEs (life threatening) declined from
24% to 7% and serious AEs
declined from 8 % to 0% from
the first year of treatment to
after the first year of treatment.
• AEs leading to ibrutinib
discontinuation occurred in
8.1% (12/148) of patients
within the first year of treatment
and declined to 5.5% (6/109) of
patients after the first year of
treatment.
Response Rate
n (%)
TN ≥ 65 years
(n = 31)
R/R
(n = 117)
Total
(N = 148)
ORR 25 (80.6%) 98 (83.8%) 123 (83.1%)
ORR + PR‐L
27 (87.1%) 104 (88.9%) 131 (88.5%)
Safety: Treatment-Emergent Related AE’s Grade ≥ 3 With Incidence ≥ 2%
Efficacy: Best Overall Response (ORR)
(Partial Response with
Lymphocytosis)
AEs Grade 3 = Severe Grade 4 = Life Threatening

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Single Agent Ibrutinib Achieves Equal Responses in CLL
Patients With and Without Deletion 17p (Farooqui, ASH 2013)
Patient Population: 53 Total Patients
• Normal (NL) 17p = 24 pts (8 pts TN/16 pts R/R)
• Del 17p = 29 pts (15 pts TN/14 pts R/R)
Evaluable at 6 Mo: 47 pts (only 1 PD)
• Est. Event Free Survival at 14 mo is 93%
• 4 pts (20%) had no evidence of 17p after 6 mos
• Del17p does not confer resistance to ibrutinib
Non-Heme Toxicity
Responses Progression Free Survival
(Median Follow‐up: 14 mos)

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Ibrutinib in combination with rituximab (iR) is well tolerated and induces
a high rate of durable remissions in patients with high-risk CLL: new,
updated results of a Phase II trial in 40 patients (Burger, ASH 2013)
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• i+R resulted in a ORR of 95% in high risk pts.
78% of patients were progression free after
18 mos.
• The ORR in the 20 pts with del17p or TP53
mutation was 90% (16 PR, 2 CR).
• Questionnaires revealed significantly
improved overall health and quality of life
after 6 months, which coincided with a
significant weight gain
at 3 and 6 months.
• Treatment generally was well tolerated, with
infectious complications (6 cases of
pneumonia and 3 cases of upper respiratory
infections) being the most common
complication. There were two Grade 3,
possibly related AEs: mucositis (n=1), and
peripheral neuropathy (n=1). Milder toxicities
included Grade 1-2 bruising (n=7), Grade 1
subdural hematoma (n=1), fatigue (n=2),
bone pain, myalgias, and arthralgia (n=5), or
diarrhea (n=1).

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• 31 treatment naïve pts
treated with ibrutinib
monotherapy.
• After median follow-up of
22.1 mos, ORR was 71%
(13% CR)
• Est. PFS: 96.3% at 24
mos (Figure A)
• Est. Overall Survival:
96.6% at 24 mos
(Figure B)
Ibrutinib as initial therapy for elderly patients with chronic
lymphocytic leukaemia or small lymphocytic lymphoma:an
open-label, multicentre, phase 1b/2 trial (O’Brien, Lancet 2013)

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• Toxicity was mainly of mild-to-moderate severity (grade 1–2). Three (10%)
• patients developed grade 3 infections, although no grade 4 or 5 infections
occurred. One patient developed grade 3 neutropenia, and one developed grade 4
thrombocytopenia.
• Improvements in hemoglobin, platelet counts, and absolute neutrophil counts
were observed in patients treated with ibrutinib
• Median serum IgA, IgM, and IgG levels also showed significant improvement.
Ibrutinib as initial therapy for elderly patients with chronic
lymphocytic leukaemia or small lymphocytic lymphoma:an
open-label, multicentre, phase 1b/2 trial (O’Brien, Lancet 2013)

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IMBRUVICA in MCL patients
PCYC/JNJ
/ISTs
Line of
Therapy
# of
Patients
Trial 1st
Released
Study Design
MCL
II
PCYC‐1104 active RR 115 Feb‐11 Monotherapy
SPARK active RR 120 Aug‐12 Monotherapy; Failed BR
Wang‐
MDACC
recruit RR 50 Jul‐13 i+R
III
RAY recruit RR 280 Dec‐12 Monotherapy vs. Temsirol
SHINE recruit TN 520 May‐13 i+BR in elderly
IV MCL4001 recruit RR 250 Apr‐13 Monotherapy

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Highlights EHA 2013: Phase II Monotherapy Trial in
Relapsed/Refractory MCL Patients-Long Term Follow
Up (Rule, EHA 2013)
PATIENT CHARACTERISTICS FOR ALL TREATED POPULATION
Bortezomib‐Naïve
(N = 63)
Bortezomib‐Exposed
(N = 48)
Total
(N = 111)
Median Age, yrs (Range) 66 (46‐83) 69 (40–84) 68 (40–84)
Gender: Male 46 (73%) 39 (81%) 85 (77%)
ECOG Status:
0 ‐ 1
2
> 2
53 (84%)
9 (14%)
1 (2%)
46 (96%)
2 (4%)
0 (0%)
99 (89%)
11 (10%)
1 (1%)
Prior Regimens:
Median (Range)
≥ 3 regimens
2 (1‐5)
31 (49%)
3 (1‐5)
30 (63%)
3 (1‐5)
61 (55%)
Median Months Since
Diagnosis (Range) 29 (3‐213) 48 (7‐223) 42 (3‐223)

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Hematological AE
Bleeding events ≥ grade 3 occurred in 5% of patients
Non‐Hematological AE
.
0% 10% 20% 30% 40% 50% 60%
Neutropenia
Thrombocytopenia
Anemia
0% 10% 20% 30% 40% 50% 60%
Diarrhea
Fatigue
Nausea
Oedema peripheral
Dyspnea
Constipation
Upper respiratory tract infection
Vomiting
Decreased appetite
Cough
Pyrexia
Abdominal pain
Contusion
Rash
Grade 1
Grade 2
Grade 3
Grade 4
Grade 5
Highlights EHA 2013: Treatment Related and
Unrelated AEs Occurring in >15% of MCL Patients
(Rule, EHA 2013)

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Relapsed/Refractory MCL – PFS and Duration of
Response (Rule, EHA 2013)
100
0
80
20
40
60
240 4 128 16 20
Progression‐Free Survival, %
Months From First Dose
All
Bortezomib‐Naïve
Censored
111 81 57 33 22 0
48 37 29 14 10 0
63 44 28 19 12 0
2
2
0
100
0
80
20
40
60
200 4 128 16
Months From First Response
75 56 40 24 6 0
32 26 17 9 3 0
43 30 23 15 3 0
All
Bortezomib‐Naïve
Censored
Patients Alive Without Progression, %
Est. median PFS = 13.9 mos
Est. median DOR = 17.5 mos

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Best Response
19 23 21
49 44 47
0
20
40
60
80
100
Bortezomib‐
Naïve
(n = 63)
Bortezomib‐
Exposed
(n = 48)
Total
(n = 111)
68 67 68
Efficacy Population n = 111, Estimated Median Follow‐up 15.3 months
CR
PR
Patients, %
48.7
53.2
50.5 47.8
46.0 47.3
0
20
40
60
80
100
2 4 6 9 12 15
Response Rate, %
Time, months
66.7 68
52.3
62.2 64 64.9
Improvement of Complete and Overall
Response Rates Over Time
Relapsed/Refractory MCL Patients-Long Term Follow
Up (Rule, EHA 2013)
3.6 9.0 13.5 17.1 20.7 20.7

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IMBRUVICA in DLBCL patients
PCYC/JNJ Phase Study ID Status
Line of
Therapy
# of
Patients
Trial 1st
Released
Study Design
DLBCL
II
DLB1002 active TN 32 Jun‐12 i+RCHOP; DLBCL, MCL, FL
PCYC‐1106 recruit RR 125 May‐11 Monotherapy
PCYC‐1123 Not yet RR 110 ASH ’13 i+R+Len vs. i+Len
PCYC‐1124 Not yet RR 56 ASH ’13 i+Len+DA‐EPOCH‐R
III DBL3001 recruit TN 800 Sept‐13 i+RCHOP vs. RCHOP

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Highlights EHA 2013: Phase II Monotherapy Trial
in Relapsed/Refractory DLBCL Patients
(Vos, EHA 2013)
Background:
• Sub-typed for activated B-cell
(ABC) or Germinal Center B
(GCB)
• Median of 3 prior therapies (1-7)
Results:
• ABC ORR 41%, CR 17%.
Additional Ongoing Studies:
• Phase Ib dose escalation with CHOP-R (ORR 100%)
• Randomized Phase III Frontline CHOP-R+/-
IMBRUVICA in non- GCB (800 patients)
Molecular Subtype Predicts Outcome with R‐CHOP

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Combining Ibrutinib With Rituximab, Cyclophosphamide, Doxorubicin,
Vincristine, and Prednisone (R-CHOP): Updated Results From a Phase
1b Study in Treatment-Naïve Patients With CD20-Positive B-cell Non-
Hodgkin’s Lymphoma (NHL) (Younes, ASH 2013)
Study Results:
• 560 mg + R-CHOP resulted in high responses in both GCB and non-GCB pts
• A Phase 3 trial of R-CHOP ± ibrutinib is ongoing in de novo non-GCB pts.

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IMBRUVICA in Other NHL patients
PCYC/JNJ/
ISTs
Line of
Therapy
# of
Patients
Trial 1st
Released
Study Design
NHL
I
Ujjani‐NCI recruit TN 33 Apr‐13 i+R+Len; FL
Blum‐OSU recruit RR 48 Dec‐11 i+BR; MZL, FL, WM, DLBCL, MCL
Christian‐
OSU
recruit RR 34 Oct‐13
i+Len; MZL, FL, WM, DLBCL,
MCL
II
PCYC‐1125 Not yet TN 80 Dec‐13 i+R; FL
PCYC‐1121 Not yet RR 60 Oct‐13 Monotherapy;  MZL
FLR2002 recruit RR 110 Apr‐13 Monotherapy;  FL
Bartlett‐NCI recruit RR 40 Apr‐13 Monotherapy; FL
III FLR3001 Not yet RR 400 Oct‐13 i+BR or i+RCHOP;  FL, MZL

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Highlights ASH 2012: Phase I Monotherapy Trial Subset
of Relapsed/Refractory Follicular Lymphoma Patients
(Fowler, ASH 2012)
Background:
• Prior chemoimmunotherapy
• Median of 3 prior therapies (1-5)
Results:
• 16 subjects enrolled: ORR=44%
• Trend for dose response
o 9 patients >5.0 mg/kg with ORR: 56% (3 CRs and 2 PRs) and median
estimated Progression Free Survival = 19.6 months
Study initiated by Janssen:
• Single arm monotherapy IMBRUVICA Phase II trial in relapsed / refractory
follicular patients
• Primary endpoint Overall Response Rate

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Background:
• Phase I trial, PCYC 04753, PR in 3 out of 4 WM patients
• Phase II collaboration with the Dana-Farber Cancer Institute
Phase II trial:
• Safety and efficacy of IMBRUVICA monotherapy in relapsed or refractory
WM patients (2 median priors)
• 420 mg q day until PD; 30 planned patients
• Trial expanded from 35 patients to 63 patients.
Updates:
• Breakthrough Therapy Designation February 2013
• PCYC to discuss further development with the FDA
Clinical Development Plan:
IMBRUVICA in Waldenstrom patients
IST Phase Study ID Status
Line of
Therapy
# of
Patients
Trial 1st
Released
Study Design
WM II Treon‐DFCI active RR 60 May‐12 Monotherapy

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Phase II Monotherapy Investigator sponsored Trial in
Relapsed/Refractory Waldenstrom’s Macroglobulinemia
Patients (Treon, ASH 2013)
IgM and Hemoglobin levels improved post ibrutinib treatment

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Phase II Monotherapy Investigator sponsored Trial in
Relapsed/Refractory Waldenstrom’s Macroglobulinemia
Patients (Treon, ASH 2013)
Efficacy Response:
• 83% ORR after a median of 9
cycles
Safety Response:
• 87.3% (55) patients continued on
therapy after a median of 9 cycles
• Serious AE’s (Grade ≥ 3) Events
Thrombocytopenia: 7
Neutropenia: 9
Anemia: 1
Pneumonic Infection: 1

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Highlights ASH 2012: Phase II Monotherapy Trial in Relapsed/Refractory MM Patients
(Vij, ASH 2012)
Background:
• Median of 4 prior treatments
• Prior bortezomib and lenalidomide
Results:
• Signals of biologic and clinical activity
• 5/13 patients had a reduction in paraprotein , 1 PR in combo with dexamethasone
• Decreases in biomarkers of bone metabolism, angiogenesis and chemotaxis were
observed
Ongoing Study:
• Cohorts 1 (Monotherapy, 420mg) and 2 (560 mg with dex) did not achieve desired
results, expansion of these cohorts is not planned.
• Expansion to explore IMBRUVICA administration to a 840 mg monotherapy dose and
a 840 mg dose in combination with dexamethasone (Cohorts 3&4) is continuing.
IMBRUVICA in Multiple Myeloma patients
PCYC/JNJ Phase Study ID Status
Line of
Therapy
# of
Patients
Trial 1st
Released
Study Design
MM
I PCYC‐1119 Not yet RR 176 Dec‐13 i+Carfilzomib
II PCYC‐1111 recruit RR 164 Mar‐12 Monotherapy or i+Dex

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Histone Deacetylase Inhibitor: Abexinostat
• Abexinostat is optimized for half-
life, oral bioavailability, and
potency and synergizes with
DNA-damaging agents
• Partnered ex-US with Servier, in
Phase I/II program in Europe
• Phase 2 PCYC study in
lymphoma completed and
presented at ASH 2012, further
updates presented at ICML in
Lugano, June 2013
• Combination therapies between
HDAC and IMBRUVICA are being
investigated.

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Factor VIIa Inhibitor: PCI-27483
• First small-molecule FVII-specific
inhibitor targeting the tissue factor
(TF) pathway
• Tissue factor is upregulated in
certain tumors. TF:VIIa complex
induce signaling pathways that lead
to increase in cancer cell migration
and invasion
• Phase II pancreatic cancer trial
completed, results provided at
ASCO, June 2013
• Further usage of PCI-27483 are
currently being investigated

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Worldwide collaboration to broaden and accelerate the development
of IMBRUVICA in oncology, signed in December 2011
• $150M upfront; milestones $250M for continued development progress (of
which $200M were earned as of May 1, 2013), $225M for regulatory progress
and $350M for approval.
• Global development plan defined, each company leading the development for
specific indications. Development costs shared 40% Pharmacyclics and 60%
Janssen for multiple phase III trials
• 50/50 profit split. Pharmacyclics will book sales and lead commercialization
strategy in the US; Janssen will be responsible for the same outside the US
• Development and commercialization activities managed through a shared
governance structure
Collaboration with Janssen Biotech
to Develop and Commercialize IMBRUVICA

41
BROAD PATENT COVERAGE:
Our lead product candidates have issued US and European
composition of matter patents and are covered by various
issued/pending patent applications in other major markets
• BTK Inhibitor, IMBRUVICA (ibrutinib - PCI-32765) covered by
issued/pending patents projected until 2026* (composition of matter;
genus around composition of matter; method of treatment; method of
manufacture; inhibition of Btk via specific, irreversible inhibitor)
• Factor VIIa Inhibitor, PCI-27483 covered by issued/pending patents
projected until 2023*
• HDAC Inhibitor, PCI-24781 covered by issued/pending patents projected
until 2025*
Strong Patent Portfolio
* which does not include projected patent term extensions in the US

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Key Corporate Data
GENERAL Current
- Founded
- Location
- Employees as of 12/31/2013
1991
Sunnyvale, CA
484
SELECT FINANCIAL INFORMATION
- Revenue Q4/2013 as of 12/31/2013 $123.6 M
- Non-GAAP Op. Ex Q4/2013 as of 12/31/2013 $28.5 M*
- Cash & Cash Equivalents as of 12/31/2013 $635.6 M**
- Basic Shares Outstanding as of 12/31/2013 74.2 M
Janssen Biotech, Inc. contractual milestones remaining:
Development Progress $ 50 million
Regulatory Progress $ 100 million
Approval $ 230 million
$ 380 million
(earned as of 02/27: upfront $150M, $200M in development
milestones, $125M in Regulatory Progress, $120M Approval)
* Non GAAP Expenses do not include
$9.6M in stock-based compensation
expense and also $50.2M in Excess
Amounts paid by Janssen.
** Cash does not include $52.0M due to
Company by Janssen under the
collaboration agreement.

43
Pharmacyclics
Making a difference for the
betterment of patients

Bob Duggan' Pharmacyclics' Presentation

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