1. No category

Developing Innovative Therapies for Patients Suffering
from Life-threatening Diseases
NasdaqCM: LJPC
Jefferies 2015 Global Healthcare Conference
June 1, 2015
Forward-Looking Statements
These slides contain "forward-looking" statements within the meaning of the Private
Securities Litigation Reform Act of 1995. These statements may be identified by the use of
forward-looking terminology such as "anticipate", "believe", "continue", "could", "estimate",
"expect", "intend", "may", "might", "plan", "potential", "predict", "should" or "will" and
include statements regarding La Jolla Pharmaceutical’s product candidates and clinical
trial progress and results. These forward-looking statements are based on our current
expectations, speak only as of the date of this presentation and involve risks and
uncertainties, many of which are outside of our control, that can cause actual results to
differ materially from those in the forward-looking statements. Potential risks and
uncertainties include, but are not limited to, our ability to commence and complete clinical
trials within projected time periods, anticipated regulatory and patent exclusivity, the ability
to manufacture clinical or commercial product, issues arising in the regulatory process, the
ability to out-license programs, the ability to successfully develop our product candidates,
including the results of ongoing and future clinical trials (including product safety issues
and efficacy results) and expected duration of the company’s operating runway based on
current cash resources. Further information is included in La Jolla Pharmaceutical’s
periodic reports filed with the SEC at www.sec.gov. We disclaim any duty to update any
forward-looking statements.
2
Overview of LJPC
LJPC-501 (Angiotensin II) for CRH
LJPC-401 (Hepcidin) for Iron Overload
LJPC-30Sa/b (Gentamicin Derivatives) for
Bacterial Infections and Rare Genetic
Disorders
Financial Position
LJPC Corporate Highlights
•  Focused on de-risked product opportunities
§  Naturally occurring peptides with well-understood biological functions
§  Derivative components of FDA-approved products
•  LJPC-501 (angiotensin II) for catecholamine-resistant hypotension (CRH)
§  Phase 3 registration study actively enrolling
–  SPA agreement with FDA in place
–  Data expected end of 2016
•  LJPC-401 (hepcidin) for iron overload
§  Phase 1 data in 2015
•  LJPC-30Sa/b (gentamicin derivatives) for bacterial infections & rare
genetic disorders
§  Plan to initiate Phase 1 study, following positive pre-IND meeting
4
Product Pipeline
Indication
IND
Phase 1
Phase 2
Q1
2015
CRH
LJPC-501
Angiotensin II
HRS
LJPC-401
Hepcidin
LJPC-30Sa &
LJPC-30Sb
Gentamicin Derivatives
Phase 1/2
Iron Overload
Mid2015
End
2015
Bacterial
Infections
Rare Genetic
Disorders
Other R&D
Various
Galectin-3
Inhibitor Program
Various
Completed
5
Phase 3
Out-licensing
Underway
Planned
H2
2016
Overview of LJPC
LJPC-501 (Angiotensin II) for CRH
LJPC-401 (Hepcidin) for Iron Overload
LJPC-30Sa/b (Gentamicin Derivatives) for
Bacterial Infections and Rare Genetic
Disorders
Financial Position
LJPC-501 for CRH: Overview
•  LJPC-501 is a proprietary formulation of angiotensin II, a naturally occurring
regulator of blood pressure
•  Catecholamine-resistant hypotension (CRH) is an acute, life-threatening
condition in which blood pressure drops to dangerously low levels and is
unresponsive to current treatments
•  LJPC-501 has been shown to raise blood pressure in a pilot RPC clinical trial in
CRH, as well as animal models of hypotension
•  Special Protocol Assessment (SPA) agreement reached with FDA
§  Agreement reached that blood pressure can be the primary endpoint for approval
•  Phase 3 trial actively enrolling
•  Multiple points of potential proprietary protection
§  Potential Orphan Drug Designation
§  Proprietary formulation developed, LJPC IP filed, IP licensed from George Washington University
7
High Dose Catecholamine Increases
Mortality
•  Catecholamines cause
cardiac toxicity, digital
necrosis and metabolic
complications leading
to higher mortality
80
70
60
Percentage
•  Blocking the cardiac
toxicity of
norepinephrine
improves outcome
90
50
Alive
40
Dead
30
20
10
0
Low Dose
Hi Dose
Norepinephrine Dose
References: Sviri, S., et al. 2014 J. of Crit Care 29, 157-160. JAMA 2013 Oct23/30 310:1683-1691.
8
Norepinephrine Dose Decreases with Angiotensin II
Surrogate Effect on Blood Pressure
§  Catecholamine dose
sparing; surrogate for
BP effect
•  Published October
2014 in Critical Care
•  Strong proof-ofconcept that
angiotensin II
increases blood
pressure in CRH
100% of Angiotensin II Treated Patients
Experienced an Increase in BP
Norepinephrine Dose (mcg/min)
•  Randomized,
placebo-controlled,
double-blind pilot trial
•  Primary efficacy
endpoint:
Reference: Busse, LW, et al. 2014, Critical Care 18(Suppl 1), 160
9
50
placebo group
Placebo
45
angiotensin II
AT-II Arm
group
40
Angio
dose II
angiotensin
dose
35
30
25
20
15
Catecholamine
Resistance
10
5
p<0.05
0
Pre2 Pre1
Hr0
Hr1
Hr2
Hr3
Hr4
Hr5
Hr6 Post1 Post2
Phase 3 Trial of LJPC-501 in CRH
SPA Agreement with FDA Reached, Trial Enrolling
•  ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial
initiated in March 2015
•  Randomized, placebo-controlled, double-blind Phase 3 trial
•  Patient population: catecholamine-resistant, based on amount of
catecholamine required
•  Primary endpoint: blood pressure at 3 hours
•  Secondary endpoint: change in CV SOFA* score
•  Size: 300 patients, 25-35 sites
•  Projected results: end of 2016
*Cardiovascular Sequential Organ Failure Assessment
10
LJPC-501: Market Opportunity
Total Annual Hospitalized
U.S. Population with
Severe Hypotension*
555,479*
Applicable Severe
Hypotension: 65%
361,684
Catecholamine-Treated
Patients: 92%
332,189
Catecholamine-Resistant**
Patients: 38%
126,232
LJPC-501 Target
Market
*Informed by annualized IMS data from 650+ hospitals and secondary research.
**Catecholamine-resistant hypotension is defined as requiring a norepinephrine equivalent dose > 0.2 mcg/kg/min
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Source(s): IMS Data; DR Consulting Physician Interviews, 2014-2015; AHA Hospital Statistics, 2014; Angus et al, 2001; Kumar et al 2011;
Annane et al., 2002
1
1
LJPC-501: Market Opportunity
Attractive Pricing Potential
Prices of Hospital-Based Drugs Reimbursed under DRGs
12
Drug Name
Indication
Cost per
Treatment
Course
Panhematin
Acute intermittent porphyria
$40,235
Acthar
Infantile spasms
$99,780
Acthar
Acute exacerbations of multiple sclerosis
$125,500
Neulasta
Febrile neutropenia
$4,253
Xigris
Severe sepsis
$13,426
Angiomax
Anticoagulant
$4,019
Cubicin
Skin and blood infection
$3,000 (skin)
$9,000 (blood)
LJPC-501: Market Opportunity
New Market Research Indicates Strong Interest
•  P&T committee members, representing 40% of physicians
surveyed, show strong support for broad use of LJPC-501
% Physicians Who Would Initiate Use of LJPC-501
100%
% Physicians
80%
60%
P&T Committee
Members
40%
Non-P&T Committee
Members
20%
0%
0.2
0.3
0.4
Replace Rescue
Vasopressor or
Methylene Blue
Replace Third
Catecholamine
Replace First or Second
Catecholamine
0.5
0.6
0.7
0.8
Norepinephrine Equivalent Catecholamine Dose (mcg/kg/min)
13
0.9
Summary of LJPC-501 in CRH
•  LJPC-501 is a proprietary formulation of angiotensin II, a naturally
occurring regulator of blood pressure
•  Catecholamine-resistant hypotension (CRH) is an acute, life-threatening
condition in which blood pressure drops to dangerously low levels and is
unresponsive to current treatments
•  LJPC-501 has been shown to raise blood pressure in a pilot RPC clinical
trial in CRH, as well as animal models of hypotension
•  Special Protocol Assessment (SPA) agreement reached with FDA
§  Agreement reached that blood pressure can be the primary endpoint for approval
•  Phase 3 trial actively enrolling
•  Multiple points of potential proprietary protection
§  Potential Orphan Drug Designation
§  Proprietary formulation developed, LJPC IP filed, IP licensed from George Washington
University
14
Overview of LJPC
LJPC-501 (Angiotensin II) for CRH
LJPC-401 (Hepcidin) for Iron Overload
LJPC-30Sa/b (Gentamicin Derivatives) for
Bacterial Infections and Rare Genetic
Disorders
Financial Position
LJPC-401: Overview
• LJPC-401 is a formulation of hepcidin, which is an
endogenous peptide hormone that controls and regulates
iron metabolism
• Iron overload damages the heart, pancreas and other vital
organs
• Several key indications
§  Acquired iron overload (e.g. beta thalassemia)
–  Currently treated with chelators; 50% of patients are intolerant to or fail
–  Approximately 25,000 patients in the U.S.
§  Hereditary hemochromatosis
–  Disorder of hepcidin production
–  1,000,000 patients in the U.S.; 5% fail or are intolerant to phlebotomy
–  Juvenile variant is more severe
16
LJPC-401: Hepcidin
•  Hepcidin: the insulin of
iron metabolism
§  Regulates iron absorption
and disposition in all organs
§  Rapid and sustained
lowering of iron levels
•  Progress
§  SQ formulation developed
–  No findings
•  Phase 1 data in 2015
§  Including serum iron data
Serum Iron (ug/dL) §  Completed toxicology
400.0 300.0 Placebo 200.0 LJPC-­‐401 100.0 0.0 0 hr 17
4 hr 24 hr Time 48 hr Day 15 LJPC-401: Phase 1 Development Plan
• 
Population: normal
healthy volunteers
• 
Iron studies:
• 
• 
serum iron;
ferritin;
transferrin
TIBC; UIBC
N = 21 to 30
4 Cohorts Escala@ng Doses Cohorts 1 , 2, 3
0.24, 0.48, 0.96 mg/
kg
3 to 6 subjects each
Cohort 4
1.92 mg/kg
12 subjects
OBJECTIVES: determine safety of escala@ng doses of LJPC-­‐401 in NHV; evaluate PK; evaluate effect on iron levels Duration of Study: single dose
•  Key endpoint includes effect on serum iron levels
§  Early insight into LJPC-401’s efficacy using clinically relevant endpoint
§  Study to start in mid-2015 – results will be available by end of 2015
•  Future plan: move directly into two parallel POC studies in
hemochromatosis and thalassemia
18
LJPC-401: Advisory Board
Recommendations
KOL Advisory Board Expanded in April 2015
Paul Adams, M.D. – Chief of Gastroenterology, University of Western Ontario
Hemochromatosis and iron overload
Victor Gordeuk, M.D. – Hematology/Oncology, University of Illinois, Chicago
Sickle cell disease
Ashutosh Lal, M.D. – Director of Thalassemia Program, UCSF/Children's Hospital Oakland
Iron overload and hemoglobinopathies
Gordon McLaren, M.D. – Hematology/Oncology, UCI/VA Long Beach
Hemochromatosis and iron overload
•  Advisory board recommended two development strategies:
§  Hemochromatosis, adult or juvenile
–  Goal = reduce number of needle sticks and phlebotomy procedures
§  Anemia, thalassemia major or intermedia
–  Goal = reduce chelation needs, increase Hgb by 1 g/dL
19
Overview of LJPC
LJPC-501 (Angiotensin II) for CRH
LJPC-401 (Hepcidin) for Iron Overload
LJPC-30Sa/b (Gentamicin Derivatives) for
Bacterial Infections and Rare Genetic
Disorders
Financial Position
LJPC-30Sa/b: Overview
•  LJPC-30Sa and LJPC-30Sb are purified derivatives of
gentamicin, which retain biologic activity but lack traditional
kidney toxicity
•  Gentamicin FDA-approved, standard-of-care for serious Gramnegative bacterial infections
§  Mixture of several distinct but closely related chemical entities
§  >3 million vials of gentamicin used in the U.S. in 2014
§  Use is limited due to kidney toxicity, believed to be due to certain constituent components
•  Two parallel development paths
§  Bacterial infections: aminoglycosides = $500+ million market in the U.S.*
§  Rare genetic disorders: gentamicin’s mechanism may be leveraged for rare genetic
disorders; proof-of-concept data exists in cystic fibrosis
•  Recent positive FDA feedback on Phase 1 proposal
21
*Adjusted for branded pricing of comparable hospital antimicrobials
LJPC-30Sa/b: Mechanism of Action
Gentamicin induces errors in the translation of genes into proteins
Bacteria
Stops bacterial protein
synthesis leading to cell death
22
Humans
Can be leveraged to read
through genetic mutations
LJPC-30Sa/b: Strong Scientific Rationale
Next-generation improved gentamicin derivative
1. Retain activity
23
K.pneumonieae (kill)
K.pneumonieae (inhib)
16.0
12.0
8.0
4.0
0.0
placebo
next-generation
gentamicin
derivative
gentamicin
0.7
Serum Creatinine mg/dL
Inhibition/kill zone (mm)
B.subtilus
2. Improve safety
0.6
placebo
0.5
0.4
0.3
0.2
0.1
0
nextgeneration
gentamicin
derivative
gentamicin
LJPC-30Sa/b: Large Established Market
•  Antibiotic opportunity = $500+ million per year in U.S.
§  Market could expand with a safer alternative
–  Increased duration of therapy, increased penetration, and/or new indications
U.S. Aminoglycoside Market (2014)
7,000,000
3,000,000
6,000,000
2,500,000
5,000,000
2,000,000
4,000,000
1,500,000
3,000,000
1,000,000
2,000,000
500,000
1,000,000
-­‐
-­‐
# of Vials
All Aminoglycosides
Days of Treatment
Gentamicin
Cubicin
•  Other large potential market opportunity in rare genetic disorders, such as
cystic fibrosis
24
Source: Source Healthcare Analytics
LJPC-30Sa/b: Market Exclusivity
• Strong IP & regulatory exclusivity position
• Patents optioned from IU and UAB provide protection
beyond 2026
§  LJPC expected to supplement with own patent applications
• Potential regulatory exclusivity
§  Antibiotic: 8+ years including Hatch-Waxman + GAIN (QIDP)
exclusivity
§  Rare genetic disorders: 7 years of orphan drug exclusivity
25
Overview of LJPC
LJPC-501 (Angiotensin II) for CRH
LJPC-401 (Hepcidin) for Iron Overload
LJPC-30Sa/b (Gentamicin Derivatives) for
Bacterial Infections and Rare Genetic
Disorders
Financial Position
Financial Position
Condensed Balance Sheet Data
As of Mar 31, 2015
(in millions)
Cash
$42.7
Total liabilities
$2.6
Total shareholders’ equity
$43.0
Cash resources expected to fund Company through 2016
Fully Diluted, As-Converted
Shares Outstanding*
*Includes common stock, preferred stock & outstanding equity awards as of March 31, 2015
27
24,019,500
LJPC Investment Summary
•  Focused on de-risked product opportunities
§  Naturally occurring peptides with well-understood biological functions
§  Derivative components of FDA-approved products
•  LJPC-501 (angiotensin II) for catecholamine-resistant hypotension
(CRH)
§  Phase 3 registration study actively enrolling
–  SPA agreement with FDA in place
–  Data expected end of 2016
•  LJPC-401 (hepcidin) for iron overload
§  Phase 1 data in 2015
•  LJPC-30Sa/b (gentamicin derivatives) for bacterial infections & rare
genetic disorders
§  Plan to initiate Phase 1 study, following positive pre-IND meeting
28
Thank You