jefferies global healthcare conference...uveitis eyeguard-b + eyeguard-a and/ or eyeguard-c ~150,000...
TRANSCRIPT
Jefferies Global Healthcare Conference November 20, 2014
Forward-looking Statements
Certain statements contained herein including, but not limited to, statements related to
anticipated timing of initiation and completion of clinical trials, anticipated size of clinical trials,
therapeutic and market potential of XOMA’s product candidates, continued sales of approved
products, regulatory approval of unapproved product candidates, sufficiency of our cash
resources and anticipated levels of cash utilization, or that otherwise relate to future periods
are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933
and Section 21E of the Securities Exchange Act of 1934.
These statements are based on assumptions that may not prove accurate, and actual results
could differ materially from those anticipated due to certain risks inherent in the biotechnology
industry and for companies engaged in the development of new products in a regulated
market. Potential risks to XOMA meeting these expectations are described in more detail in
XOMA's most recent filing on Form 10-K and in other SEC filings. Consider such risks
carefully when considering XOMA's prospects. Any forward-looking statement herein
represents XOMA’s views only as of the date of this presentation and should not be relied upon
as representing its views as of any subsequent date. XOMA disclaims any obligation to update
any forward-looking statement, except as required by applicable law.
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XOMA: Value Creation and Value Capture
Create value by focusing on lead asset ─ gevokizumab
• Potent, fully humanized allosteric-modulating monoclonal antibody
• Once-a-month, subcutaneous injection
• Modulates inflammatory response to cytokine interleukin-1 beta (IL-1 β)
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Disruption of
disease-causing
inflammatory cascade
IL-1β binds to
IL-1β Receptor
Inflammatory diseases
induced through
over-produced IL-1β by
multiple Immune
system cells
Gevokizumab
binds to IL-1β
and
down-modulates
IL-1β signaling
XOMA: Value Creation and Value Capture
Create value by developing gevokizumab broadly across multiple
inflammatory indications
• Execute ongoing Phase 3 EYEGUARD™ studies in non-infectious uveitis (NIU)
– Report top-line results from EYEGUARD-B, Behçet's disease uveitis patients,
approximately 6 weeks after achieving the target event
– EYEGUARD-A and EYEGUARD-C studies in broad NIU population - results will follow
• Initiated Phase 3 study in second pivotal indication: pyoderma gangrenosum
• Use expansive proof-of-concept (POC) program to let gevokizumab lead us to
next diseases for pivotal development
– Multiple indications in POC testing at XOMA
– Leveraging Servier’s development in additional POC indications
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XOMA: Value Creation and Value Capture
Capture value for XOMA by commercializing gevokizumab
directly in the U.S.
• Deal with Servier retains XOMA U.S. and Japanese commercial rights
– Select indications served by specialty physicians to enable direct commercialization
by XOMA in the U.S.
• Leverage Servier commercialization outside the U.S. and Japan
– Milestones and royalties to XOMA
• License gevokizumab development and commercialization in Japan
Use gevokizumab success to advance additional XOMA
discoveries
• 30 years of leadership in antibody discovery and development
• Sizeable portfolio of potential antibodies to explore
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Gevokizumab: Pipeline in a Product
IndicationPre-
clinicalPhase 1 Phase 2 Phase 3
Upcoming
Milestones
XOMA’s Development Programs
Non-infectious uveitis (NIU) – EYEGUARD™-A & -C*2015:
Ph 3 Endpoints
Behçet's disease uveitis (BDU) – EYEGUARD-USQ3 14:
Initiated Ph 3 Trial
Pyoderma gangrenosum (PG)Q4 14:
Initiated Ph 3 Trial
Multiple POC Programs As Occurring
Servier’s Development Programs
Behçet's disease uveitis - EYEGUARD-BQ1 15:
Ph 3 Data
Cardiovascular Ongoing
Multiple POC programs As Occurring
6*Worldwide development by both XOMA and Servier
Gevokizumab: Behçet's Disease Uveitis (BDU)& Non-infectious Uveitis (NIU)
Gevokizumab: Three EYEGUARD™ Phase 3 Studies Ongoing in Non-infectious Uveitis
Inflammation of the uvea which can
occur in front, middle and/or back of
the eye
• Can be infectious or non-infectious
• Gevokizumab targeted for non-infectious
patients where back of the eye is affected
150,000 NIU patients in U.S. is
significant market opportunity yet
retains orphan status (granted)
Multiple etiologies
• Behçet's disease uveitis is representative of
multiple forms
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Gevokizumab: Clinical Activity in XOMA’s First Phase 2 Trial of Behçet's Disease Subset of NIU
7 of 7 patients showed rapid treatment effect for intraocular inflammatory attack
5 of 5 responded to second dose
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Day 0: Day 1: Day 4:
Flare score: 467 ph/msec *
Anterior chamber cell: 4+
Vitreous haze: 3+
Fundus score: 8
Flare score: 45 ph/msec
Anterior chamber cell: 4+
Vitreous haze: 3+
Fundus score: 4
Flare score: 62 ph/msec
Anterior chamber cell: 4+
Vitreous haze: 2+
Fundus score: 4
hypopyon off-center iris * photon counts per millisecond
Gevokizumab: Phase 2 Behçet's Disease Uveitis Studies Clearance of Vitreous Haze
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Day 0 - Vit. Haze: 3+
Servier’s study in 21 patients with Behçet's disease uveitis patients
included 15 patients with acute disease
• All responded to gevokizumab - most within 1 week
• Of 11 acute patients with a baseline vitreous haze score of ≥ 2+, 8 showed at least a
2-unit reduction at Day 70, remaining 3 had at least a 1-unit reduction
Day 1 - Vit. Haze: 3+ Day 4 - Vit. Haze: 2+
Day 7 - Vit. Haze: 1+ Day 14 - Vit. Haze: 0.5+ Day 21 - Vit. Haze: 0
Patient from XOMA 7-patient Phase 2 study
Gevokizumab: EYEGUARD-B Study Design and Status
Strong Phase 2 data in Behçet's disease uveitis patients (BDU)
led to Servier run EYEGUARD-B study
Ex-U.S. BDU patients, randomized, double-masked
Receive 60mg gevokizumab or placebo subcutaneously monthly
• Randomized 1:1
Patients must be stable on corticosteroids prior to randomization
• Steroids further tapered according to a pre-set fixed schedule
Primary endpoint is time to first exacerbation which is
interpolated from Kaplan-Meier survival analysis
• Hazard ratio of 0.3 gives 90% power at p≤ 0.05
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Gevokizumab: Driving Toward First BLA in Behçet’s Disease Uveitis
EYEGUARD-B results are expected first
• Waiting for final pre-specified exacerbations
Use EYEGUARD-B results to request pre-BLA Meeting with FDA
Perform statistical analysis of XOMA and Servier Phase 2 results
to support EYEGUARD-B results
Initiated EYEGUARD-US study in up to 28 BDU patients to further
supplement overall results
• May not be necessary for approval
• Interim results may be used for informational purposes
• Pivotal results may be required for approval
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Gevokizumab: EYEGUARD-US Behçet’s Disease Uveitis Supplemental Study
Patients can have active Behçet’s disease uveitis
• Patients with active disease are brought under control with gevokizumab
(open label), or
Patients can be under control with other therapies
• Treatment control transferred to gevokizumab
– From anti-TNF-αs
– From Prednisone and/or other immunosuppressives
Once patients are controlled on gevokizumab for 28 days -
• Randomized 1:1to continue to receive monthly 60 mg subcutaneous
treatment with gevokizumab or placebo
Primary Endpoint is time to first ocular exacerbation
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Gevokizumab: Regulatory Submission Strategy
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EYEGUARD-B
+
Two Phase 2
Trials
Supplemented w/
EYEGUARD-US
~7,500 U.S.
Patients
Non-infectious
uveitis
EYEGUARD-B
+
EYEGUARD-A
and/ or
EYEGUARD-C
~150,000 U.S.
Patients
Other follow-on
indications:
Ophthalmology
Scleritis
Dermatology
Pyoderma
Gangrenosum(Phase 3 Q4 2014)
Other Neutrophilic
Dermatoses
Behçet's disease
uveitisNon-infectious
uveitis
Other follow-on
indications
Gevokizumab: Pyoderma Gangrenosum (PG)
Pyoderma Gangrenosum: XOMA’s Next Phase 3 Indication
Severe inflammatory, ulcerative disease of the skin with
undetermined cause
• One of several indications collectively known as neutrophilic dermatoses
• Claims data over past 3 years indicate U.S. pyoderma gangrenosum patient
population of 11,000 - 14,000
• Typically takes >11 months to fully heal with corticosteroids*
• Orphan Drug Designation granted by FDA: February 2014
6 patients enrolled in pilot study at 60mg dose
• 5 patients showed improvement in ulcer size
by Day 28
– 4 patients had total resolution of ulcer by Day 84
• All patients demonstrated pain improvement
16 *Bennett ML et al. “Pyoderma gangrenosum. A comparison of typical and atypical forms with an emphasis on time to remission. Case review of 86 patients
from 2 institutions.” Medicine (Baltimore) Jan;79(1):37-46. PMID: 10670408
DAY 0 DAY 84
1
Gevokizumab: Pyoderma Gangrenosum Subject 2300
Day 0 Day 84
ID Area (cm2) Area (cm2) D%
1 5.2 0 100
Ulcer Size:Pain
(0 - 10) Investigators Assessment of Target Ulcer
Day 0 10
Day 84 70: Total resolution of target ulcer with no sign
of active PG
1
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Gevokizumab: Pyoderma Gangrenosum Phase 3 Program Study Designs
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Pyoderma Gangrenosum
Phase 3 Studies
U.S. Only
(Study 172)
U.S. & x-U.S.
(Study 173)
# Patients 58 58
Gevokizumab doses
(Monthly)60mg : Placebo 60mg : Placebo
Randomization 1:1 1:1
Primary endpoint Complete healing of target ulcer at approximately Day 124
Secondary endpoints Multiple, including time to ulcer closure and pain
Statistical Powering 80%; p=0.05 80%; p=0.05
XOMA Metabolic (XMet) Program
Creating Value from XMet Discovery Program
XMet A: selective insulin receptor modulator (SIRM)
• May provide a long-acting alternative to basal insulin*
• Data from non-human primates should be predictive and is being used for
partnering discussions
XMet S: insulin sensitizing antibodies
• May enable more effective use of own insulin for early stage diabetics
• Mouse model has shown a 20x increase in sensitivity to insulin
XOMA 358: antagonist (deactivator) of the insulin receptor
• 3 potential rare disease indications to be evaluated:
– Congenital Hyperinsulinism (CHI)
– Insulinomas
– Hyperinsulinemic Hypoglycemia Post Gastric Bypass Surgery
• Patients concentrated in a few centers nationwide - development could be
rapid
• Phase 1 study initiated October 2014
20*Vigneri, R., Squatrito, S., Frittitta, L., Diabetes May 2012
Building Value Through Development Activities
Compound IndicationPre-
clinicalPhase 1 Phase 2 Phase 3 Marketed
XOMA’s Development Programs
Gevokizumab Non-infectious uveitis (NIU) – EYEGUARD-A & -C
Behçet's disease uveitis (BDU) – EYEGUARD-US
Pyoderma gangrenosum (PG)
Erosive osteoarthritis of the hand (EOA)
Moderate-to-severe acne
Non-infectious scleritis
Autoimmune inner ear disease (AIED)
XOMA 358 Excess insulin – Rare disease indications
Servier’s Development Programs
Gevokizumab Behçet's disease uveitis - EYEGUARD-B
Cardiovascular
Polymyositis/dermatomyositis
Schnitzler syndrome
Giant cell arteritis (GCA)
XOMA Development Activities for Licensure
XMet A, S Type 1 & 2 diabetes mellitus
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Financial Highlights
$59.1 million cash at September 30, 2014
Approximately 107 million shares outstanding
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Investment Thesis –Committed to Become a U.S. Commercial Company
Complete gevokizumab global Phase 3 program in non-infectious
uveitis (NIU) clinical trials
• EYEGUARD-B data may allow a “Behçet's first” BLA submission
• EYEGUARD-US study to supplement Behçet's disease uveitis BLA
submission
• EYEGUARD-A and -C may expand label to broader NIU population
Second gevokizumab pivotal program with studies in pyoderma
gangrenosum
Assess gevokizumab’s potential in additional inflammatory
indications through POC program
Advance proprietary allosteric modulating antibody pipeline
towards an IND and commercialization or licensure, particularly
XOMA 358
Use gevokizumab to build XOMA into a fully integrated company
with a commercial capability
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