April 2017
Developing Innovative Therapies for Patients Suffering
from Life-threatening Diseases
NASDAQ: LJPC
Corporate Presentation
Forward-Looking Statements
These slides contain forward-looking statements as that term is defined in the Private Securities
Litigation Reform Act of 1995. These statements relate to future events or the company’s future
results of operations. These statements are only predictions or statements of current
expectations and involve known and unknown risks, uncertainties and other factors, that may
cause actual results to be materially different from those anticipated by the forward-looking
statements. The company cautions readers not to place undue reliance on any such forward-
looking statements, which speak only as of the date they were made. Certain of these risks,
uncertainties, and other factors are described in greater detail in the company’s filings with the
U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on
the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the
timing of the NDA submission for LJPC-501 and prospects for approval of the NDA; risks that the
full data set from the ATHOS 3 study will not be consistent with the top-line results of the study;
risks relating to the scope of product label(s) (if approved) and potential market sizes, as well as
the broader commercial opportunity; the anticipated timing for regulatory actions; the success of
future development activities; potential indications for which the company’s product candidates
may be developed; and the expected duration over which the company’s cash balances will fund
its operations. Subsequent written and oral forward-looking statements attributable to the
company or to persons acting on its behalf are expressly qualified in their entirety by the
cautionary statements set forth in the company’s reports filed with the SEC. The company
expressly disclaims any intent to update any forward-looking statements.
2
• Overview of LJPC
• LJPC-501 (Synthetic Human Angiotensin II)
for CRH
• LJPC-401 (Synthetic Human Hepcidin) for
Iron Overload
• LJPC-30S Program (Gentamicin Derivatives)
for Bacterial Infections and
Rare Genetic Diseases
• Financial Position
Agenda
3
Mission Statement
La Jolla is dedicated to improving the lives of
patients suffering from life-threatening diseases
by discovering and developing innovative
therapies
4
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 (synthetic human angiotensin II) for catecholamine-resistant
hypotension (CRH)
§ Phase 3 registration study conducted under SPA, positive topline results
reported February 2017
§ New Drug Application planned for second half of 2017
• LJPC-401 (synthetic human hepcidin) for iron overload caused by
diseases such as hereditary hemochromatosis (HH), thalassemia, sickle
cell disease (SCD) and myelodysplasia (MDS)
§ Positive Phase 1 study results reported in September 2016
§ Agreement reached with EMA on pivotal study design
• LJPC-30S (gentamicin derivative) for bacterial infections and rare genetic
diseases
5
Product Pipeline
UnderwayCompleted/milestone achieved Planned
6
LJPC-501
Synthetic
Human
Angiotensin II
LJPC-401
Synthetic
Human
Hepcidin
Other R&D
Indication IND Phase 1 Phase 2 Phase 3
Catecholamine
Resistant
Hypotension
Sept.
2016
Various
Iron overload
due to HH,
Thalassemia,
SCD and MDS
Q4
2015
LJPC-30S
Gentamicin
Derivative
Bacterial
Infections/Rare
Genetic
Diseases
Successful
Pre-IND
Meeting
Mid-
2017
Pivotal Study
Pivotal Study
Positive topline
results
NDA
H2
2017
Filing
• Overview of LJPC
• LJPC-501 (Synthetic Human Angiotensin II)
for CRH
• LJPC-401 (Synthetic Human Hepcidin) for
Iron Overload
• LJPC-30S Program (Gentamicin Derivatives)
for Bacterial Infections and
Rare Genetic Diseases
• Financial Position
Agenda
7
LJPC-501: Overview
8
• LJPC-501 is a proprietary formulation of synthetic human angiotensin II, a
naturally occurring regulator of blood pressure being developed for
catecholamine-resistant hypotension (CRH), dangerously low blood
pressure
• CRH is deadly, costly and prevalent
• Phase 3 conducted under Special Protocol Assessment (SPA)
§ Agreement reached that blood pressure can be the primary endpoint for approval
• Positive top-line results announced in February 2017; detailed study
results to be published and presented later this year
• New Drug Application planned for second half of 2017
• Issued patent claims covering potential product to 2034
Shock
MORTALITY RATES COMPARED
Shock: Deadly, Costly and Prevalent
1. Vincent JL, De Backer D. N Engl J Med. 2013;369(18):1726-1734. 2. Sviri S, Hashoul J, Stav I, van Heerden PV. J Crit Care.
2014;29(1):157-160. 3. Readmissions and deaths-national. Data.Medicare.gov website. https://data.medicare.gov/Hospital-
Compare/Readmissions-and-Deaths-National/qqw3-t4ie/data. Accessed January 10, 2017
Abbreviations: AMI=acute myocardial infarction; CHF=congestive heart failure.
9
• A well-characterized syndrome1
§ Occurs when the organs and tissue of the body
do not receive an adequate flow of blood
(oxygen) due to a lack of blood pressure
(hypotension)
• Deadly
§ Mortality rate exceeds that of most acute
conditions requiring hospitalization2
§ Can kill old and young alike within hours2
• Costly
§ Estimated costs are 2-3 times greater
compared to other conditions
• Prevalent
§ Affects one-third of patients in the intensive
care unit1
30-day mortality rate3
14% 12%
16%
AMI CHF Pneumonia
≥50% mortality
in patients with
shock in the
ICU2
CMS Covered Charges for CRH Population Are Much
Greater Than for Other Acute Hospital Conditions
10
Source: CMS FY14 Inpatient Public Use File (IPUF)
$87,282
$42,243
$31,453 $30,702
CRH AMI CHF Pneumonia
Weighted Average CMS Covered Charges
Abbreviations: AMI=acute myocardial infarction; CHF=congestive heart failure.
U.S. Shock Patient Population and
Treatment Paradigm
11
1. 9.42MM annualized vials (785K vials sold in January 2017 X 12); Symphony Health Solutions, 2017. 97% of vials sold for hypotensive shock; estimate based on
medical literature. 13 vials used per patient; estimate based on Russell et al, N Engl J Med, 358:877-87, 2008 and Asfar et al, N Engl J Med, 370:1583-93, 2014
2. Wolters Kluwer PriceRx Pro, 2017
3. 3.01MM annualized vials (251K vials sold in January 2017 X 12); Symphony Health Solutions, 2017. 81% of vials sold for hypotensive shock; estimate based on
medical literature. 10 vials used per patient; estimate based on Dunser et al, Circulation, 107:2313-2319, 2003 and Gordon et al, Crit Care Med, 42(6):1325-1333,
2014
4. Decision Resources Group market research
First-Line
Standard-of-Care
Second-Line
Standard-of-Care
LJPC-501 Target
Patient Population
555,479
361,684
332,189Patients Who Do Not
Adequately Respond
to Norepinephrine
and Vasopressin
Vasopressin:
244,000 Patients per Year3
$1,385 per Patient2
$338MM Sales Run Rate
Norepinephrine:
703,000 Patients per Year1
$153 per Patient2
$108MM Sales Run Rate
196,000
Estimated
Patients4
Randomized Study of Vasopressin
12
VASST Overall Survival1
VASST=Vasopressin and Septic Shock Trial
1. Russell JA, Walley KR, Singer J, et al. for VASST Investigators. N Engl J Med. 2008;358(9):877-887
Day 28 HR=0.90
(95%CI: 0.75-1.08)
P=0.27
Day 90 HR=0.88
(95%CI: 0.76-1.03)
P=0.10
LJPC-501: The First Synthetic Human
Angiotensin II Tested in a Randomized Phase 3 Study
13
• LJPC-501 is a proprietary formulation of synthetic human angiotensin II,
a naturally occurring regulator of blood pressure
• Angiotensin II has been shown to raise blood pressure in a randomized,
placebo-controlled, pilot study in CRH1, as well as in animal models of
hypotension
• Special Protocol Assessment (SPA) agreement reached with FDA for
Phase 3 study design
§ Agreement reached that blood pressure can be the primary endpoint for approval
• ATHOS-3 enrollment completed in Q4 2016
1. Chawla et al. Critical Care 2014, 18:534
Three Systems Work in Harmony to
Regulate Blood Pressure
Existing Treatments for Shock Only Utilize Two Systems
THERAPIES AND ASSOCIATED
ADVERSE EVENTS
SYMPATHETIC
NERVOUS
ARGININE-
VASOPRESSIN
RENIN
ANGIOTENSIN-
ALDOSTERONE
14
CATECHOLAMINES1: SYMPATHETIC NERVOUS
Prolonged elevated heart rate, tachyarrhythmia, acute
cardiac arrest or death, pulmonary hypertension
VASOPRESSIN: ARGININE-VASOPRESSIN
Myocardial ischemia, decreases gut blood flow
RENIN ANGIOTENSIN-ALDOSTERONE
No current therapies
1. Catecholamines include: norepinephrine, epinephrine, dopamine, phenylephrine, ephedrine
Primary endpoint
Percentage of patients
achieving pre-specified
target blood pressure
response1
ATHOS-3 (Angiotensin II for the Treatment of
High-Output Shock) Study Design
15
1. Specifically, percentage of patients achieving a Mean Arterial Pressure (MAP) ≥ 75 mmHg OR a 10 mmHg increase from
baseline MAP at 3 hours following the initiation of study treatment without an increase in standard-of-care vasopressors.
The primary endpoint was agreed upon with FDA and is reflected in our Special Protocol Assessment (SPA) agreement
dated February 2, 2015 (before study initiation) and has never been changed. ATHOS-3 was conducted without any
amendment to any part of the clinical protocol, including the primary and all other endpoints.
Patient
population:
• Adult patients
with CRH
• N=344 enrolled
• N=321 treated
Study Conducted In 74 Centers Across 9 Countries
1:1
double-blind
randomization
Placebo
N=158
LJPC-501
N=163
ATHOS-3 Topline Results: Primary Endpoint
Primary Efficacy
Endpoint Placebo LJPC-501 P-Value
Percentage of patients
achieving pre-specified
target blood pressure
response1
23% 70% <0.00001
16
• Analysis of primary efficacy endpoint was highly statistically significant
1. Specifically, percentage of patients achieving a Mean Arterial Pressure (MAP) ≥ 75 mmHg OR a 10 mmHg increase from
baseline MAP at 3 hours following the initiation of study treatment without an increase in standard-of-care vasopressors.
The primary endpoint was agreed upon with FDA and is reflected in our Special Protocol Assessment (SPA) agreement
dated February 2, 2015 (before study initiation) and has never been changed. ATHOS-3 was conducted without any
amendment to any part of the clinical protocol, including the primary and all other endpoints.
ATHOS-3 Topline Results: Mortality
Estimated
Risk Reduction Hazard Ratio
1
95%
Confidence
Interval
P-Value
22% 0.78 0.57-1.07 0.12
1. Proportional hazards estimate (unadjusted) of mortality to Day 28 comparing placebo-treated patients to LJPC-501-treated patients
17
• Trend toward longer survival observed
ATHOS-3 Topline Results: Safety
Placebo LJPC-501
Percentage of patients experiencing
at least one adverse event 92% 87%
Percentage of patients discontinuing
treatment due to an adverse event 22% 14%
18
• Throughout the study, safety outcomes were followed by an independent Data
Safety Monitoring Board (DSMB)
§ The DSMB recommended that the study continue as originally planned
LJPC-501: Path Forward
19
• Phase 3 conducted under Special Protocol Assessment (SPA)
§ Agreement reached that blood pressure can be the primary endpoint for approval
• Phase 3 positive topline results: primary efficacy endpoint highly
statistically significant, trend toward longer survival
• ATHOS-3 study detailed results will be presented and published later
this year
• New Drug Application planned for second half of 2017
• Issued patent claims covering potential product to 2034
• Overview of LJPC
• LJPC-501 (Synthetic Human Angiotensin II)
for CRH
• LJPC-401 (Synthetic Human Hepcidin) for
Iron Overload
• LJPC-30S Program (Gentamicin Derivatives)
for Bacterial Infections and
Rare Genetic Diseases
• Financial Position
Agenda
20
LJPC-401: Overview
• LJPC-401 is a proprietary formulation of synthetic human hepcidin, a naturally
occurring regulator of iron absorption and distribution
• Primary iron overload
§ Hereditary hemochromatosis (HH) is characterized by a genetic deficiency of hepcidin
resulting in excessive iron accumulation
– Most common genetic disease in Caucasians
– Causes liver cirrhosis, liver cancer, heart disease and/or failure, and diabetes
• Secondary iron overload
§ Patients with thalassemia (including beta thalassemia), sickle cell disease (SCD) and
myelodysplasia (MDS) have physiologically low hepcidin levels and are treated with
blood transfusions, resulting in acquired iron overload
• LJPC-401 has been shown to be effective at reducing serum iron levels in
preclinical and Phase 1 human testing
• Scalable manufacturing process capable of producing a pure, properly folded,
stable hepcidin formulation developed
• Orphan Drug Designation granted (EU)
• Agreement reached with European Medicines Agency (EMA) on pivotal study
design
21
Hepcidin: The Insulin of Iron Metabolism
• Hepcidin: the insulin of iron
metabolism
§ Regulates iron absorption and
disposition in all organs
§ Rapid and sustained lowering
of iron levels
• Progress
§ SC formulation developed
§ Orphan Drug Designation
granted (EU)
§ Positive Phase 1 study results
§ Agreement with EMA on pivotal
study design
22
From: Blood Research Vol. 48, No. 1, p.10, March 2013
0
50
100
150
200
250
300
350
400
450
500
0 hr 4 hr 24 hr 48 hr Day 15
S
er
um
Ir
on
(u
g/
dL
)
Time Points
GLP Toxicology Study in Rats
25
mg/kg
PRIMARY ENDPOINT
Safety and tolerability
via review of: Treatment
Emergent Adverse Events
(TEAEs), changes in clinical
lab values, ECGs, vital sign
and physical exam data
SECONDARY ENDPOINT
Serum iron
LJPC-401: Phase 1 Study Design
• Population: Adult
patients at risk of iron
overload (e.g., HH,
thalassemia, SCD)
• Design: Phase 1, open-
label, dose-escalation,
study
• Study Duration: Single
SC dose, 7-day
observation
Escalating dose levels
3 to 6 subjects at each
dose level
23
Data Monitoring Committee (DMC)
made dose-escalation decisions
LJPC-401: Phase 1 Results Overview
• Fifteen patients dosed at escalating dose levels from 1 mg to 20 mg
§ Patient subtypes treated: HH = 10; SCD = 3; and thalassemia = 2
• Safety observations
§ No dose-limiting toxicities at any dose level
§ 1 SAE at 1 mg dose level unrelated to study drug
– Hospitalization for acute sickle cell crisis; fully resolved
§ 9 injection-site reactions – all were mild or moderate in severity, self-limiting,
and fully resolved
§ No significant changes in serum chemistries or hematology other than serum
iron parameters
• Pharmacodynamic results
§ Dose-dependent, statistically significant reduction in serum iron (p=0.008)
§ Maximum serum iron reduction observed at 8 hours post-dose
§ Durable effect observed through last observation on Day 7
24
Dose Response
p=0.008
Individual dose p-values for change from baseline not adjusted for a potential regression to the mean effect.
Dose response not adjusted for multiple comparisons.
LJPC-401: Percent Change in Serum Iron
Baseline to Hour 8
25
Nominal Time from Treatment (hours)
Pe
rc
en
t C
ha
ng
e
fro
m
B
as
el
in
e
(%
)
0 8 24 48 168
-100
-75
-50
-25
0
25
Serum Iron: Percent Change from Baseline
Change from baseline through day 7: -21%
LJPC-401: Percent Change in Serum Iron
Baseline through Day 7 for 20 mg Dose
26
• Agreement reached with European Medicines Agency (EMA) on pivotal
study design
• Randomized, controlled, multi-center study in beta thalassemia patients
suffering from iron overload
§ A major unmet medical need in an orphan patient population
• Primary endpoint is a clinically relevant measurement directly related to
iron overload
• Plan to initiate study mid-2017
LJPC-401: Update on Registration Plan
Agreement Reached on Pivotal Study
27
LJPC-401: Patient Need
• Hereditary Hemochromatosis (HH)
§ Most common genetic disease in Caucasians
§ Silent Killer - Iron accumulation can lead to liver cirrhosis, liver cancer, heart
disease and/or failure, and diabetes
§ No FDA-approved treatment
§ Current treatments don’t address the underlying disease pathology and/or can
have lethal side effects
– Iron chelators may cause kidney failure, liver failure or gastrointestinal hemorrhage
– Phlebotomy creates heavy patient burden with weekly procedures for >1 year
§ Significantly underdiagnosed despite simple, inexpensive and readily available
genetic and serum iron tests
§ ~250,000 people in U.S. have clinically significant iron overload due to HH
• Acquired Iron Overload: thalassemia, sickle cell disease (SCD) and
myelodysplasia (MDS)
§ Attractive treatment alternative for iron overload in lieu of chelation therapy
• EMA orphan designation for beta thalassemia and SCD
28
• Overview of LJPC
• LJPC-501 (Synthetic Human Angiotensin II)
for CRH
• LJPC-401 (Synthetic Human Hepcidin) for
Iron Overload
• LJPC-30S Program (Gentamicin Derivatives)
for Bacterial Infections and
Rare Genetic Diseases
• Financial Position
Agenda
29
LJPC-30S: Overview
• LJPC-30S is a purified derivative of gentamicin, which retains
biologic activity but lacks traditional kidney toxicity
• Gentamicin: FDA-approved, standard-of-care for serious Gram-
negative bacterial infections
§ Mixture of several distinct but closely related chemical entities
§ >3 million vials of gentamicin used in the U.S. in 2015
§ Use is limited due to kidney toxicity, which is believed to be associated only with
certain constituent components
• Two target market opportunities
§ Bacterial infections: aminoglycosides = $500+ million market in the U.S.1
§ Rare genetic diseases: gentamicin’s mechanism may be leveraged for rare genetic
diseases; proof-of-concept data exists in cystic fibrosis
• Multiple points of potential proprietary protection
§ Potential regulatory exclusivity and Orphan Drug Designation
§ Antibiotic exclusivity: 8+ years including Hatch-Waxman + GAIN (QIDP)
§ IP licensed from IU and UAB
30
15.7MM vials is an estimated 300K regimens X an average wholesale acquisition cost (WAC) of $2,491 per regimen for
branded, intravenous only antibiotics (n=7); Symphony Health Solutions, 2016, medical literature and Wolters Kluwer
PriceRx Pro, 2016
Next-generation improved gentamicin derivative
1. Retain activity
LJPC-30S: Potential for
Improved Clinical Profile
31
2. Improve safety
0.0
4.0
8.0
12.0
16.0
placebo next-generation
gentamicin
derivative
gentamicinIn
hi
bi
tio
n/
ki
ll
zo
ne
(m
m
)
B.subtilus K.pneumonieae (kill) K.pneumonieae (inhib)
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
S
er
um
C
re
at
in
in
e
m
g/
dL
placebo
next-
generation
gentamicin
derivative
gentamicin
LJPC-30S: Current Antibiotic Usage
• Antibiotic opportunity = $500+ million per year in U.S.1
§ Market could expand with a safer alternative
– Increased duration of therapy, increased penetration, and/or new indications
• Other large potential market opportunity in rare genetic diseases, such
as cystic fibrosis
32
15.7MM vials is an estimated 300K regimens X an average wholesale acquisition cost (WAC) of $2,491 per regimen for branded,
intravenous only antibiotics (n=7); Symphony Health Solutions, 2016, medical literature and Wolters Kluwer PriceRx Pro, 2016
2Magill SS, Edwards JR, Beldavs ZG, et al; Prevalence of Antimicrobial Use in US Acute Care Hospitals, May-September 2011.
JAMA. 2014;312(14):1438-1446.
.
All Aminoglycosides Gentamicin Cubicin
U.S. Aminoglycoside Market (2015)2
-
1,000,000
2,000,000
3,000,000
4,000,000
5,000,000
6,000,000
7,000,000
# of Vials
• Overview of LJPC
• LJPC-501 (Synthetic Human Angiotensin II)
for CRH
• LJPC-401 (Synthetic Human Hepcidin) for
Iron Overload
• LJPC-30S Program (Gentamicin Derivatives)
for Bacterial Infections and
Rare Genetic Diseases
• Financial Position
Agenda
33
Financial Position
34
Condensed Balance Sheet Data As of December 31, 2016
(in millions)
Cash1 $183.2
Total liabilities $9.8
Total shareholders’ equity1 $178.5
Fully Diluted, As-Converted
Shares Outstanding2 32,205,772
1 Includes the effect of $117.5 million net proceeds received from the March 28, 2017 common stock offering
2 Includes common stock, preferred stock (as-converted), outstanding equity awards as of December 31, 2016 and the
shares issued in the March 28, 2017 common stock offering
Cash resources expected to fund Company into second half of 2018
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 (synthetic human angiotensin II) for catecholamine-resistant
hypotension (CRH)
§ Phase 3 study under SPA: primary endpoint analysis highly statistically significant
(p<0.00001), trend toward longer survival
§ New Drug Application planned for second half of 2017
• LJPC-401 (synthetic human hepcidin) for iron overload caused by diseases
such as hereditary hemochromatosis (HH), thalassemia, sickle cell disease
(SCD) and myelodysplasia (MDS)
§ Positive Phase 1 results: statistically significant reduction in serum iron
§ Agreement reached with EMA on pivotal study design
• LJPC-30S (gentamicin derivatives) for bacterial infections and rare genetic
diseases
• Recent common stock offering strengthened balance sheet
35
Thank You