La Jolla Pharmaceutical 8K Regulation FD Disclosure | LJPC 13 Jul 12

Corporate Presentation

July 2012

Exhibit 99.1

Mission

La Jolla Pharmaceutical is Committed to

Discovering and Developing Novel Therapies

Designed to Modulate Immune Function

to Treat Organ Failure and Cancer

Corporate Highlights

Technology

•

Immune therapy platform targeting, Galectin-3, an emerging key with a

demonstrated role in organ failure and cancer via immune regulation

•

Clear path to proof-of-concept

Pipeline

•

Product

candidate:

GCS-100

Leading,

clinical

stage

galectin-3

antagonist

May prevent or reverse organ failure by mediating fibrosis via galectin-3 sequestration

Binds galectin-3 and reverses T-cell suppression

Extensive clinical data with clear single agent activity and favorable safety profile

Management and Investors

•

Experienced and driven

Milestones

•

Near-term, cost-efficient clinical and preclinical milestones to drive value

Robust Product Pipeline

Products

Program/Indication

R&D

IND

Phase 1

Phase 2

Phase 3

GCS-100

Cancer with Renal Insufficiency

Cancer Immunotherapy

Prevention of Chemotherapy Toxicity

End-Stage Renal Disease

LJPC-101

Renal Transplantation

End-Stage Renal Disease

LJPC-201

Heart Failure

Hepatic Fibrosis

LJPC-301

Cancer –

Solid Tumors

Galectins and Galectin-3

•

Galectins are proteins that can

bind to sugars proteins to modulate

function and communication

•

Normally present at low concentration, but up-regulated in cancer and organ

failure

GCS-100: The Lead Galectin-3 Antagonist

•

GCS-100 is a well-characterized, complex sugar

•

GCS-100 binds to and neutralizes galectin-3. Binding activity

is localized to the galactose containing side-branches

•

Patented manufacturing process required for biologic activity;

unmodified pectin has reduced biologic activity

GCS-100

Galectin-3 and Organ Failure

•

Galectin-3 knockout mice develop significantly less kidney

fibrosis

and

failure

after

damage

compared

normal

mice

•

Galectin-3 knockout mice do not develop liver fibrosis when

exposed

toxin

•

Galectin-3 serum assay is FDA approved to identify patients at

risk

for

death

due

heart

failure

•

Serum galectin-3 levels identify patients with end-stage renal

disease

who

are

highest

risk

for

death

1,2

The American Journal of Pathology, 2008; Vol. 172, No. 2: 288-298.

Transplantation International, 2008; Vol. 21, No. 10: 999-1007.

Proceedings of the National Academy of Sciences, 2006; Vol. 103, No. 13: 5060-5065.

Annals of Medicine, 2011; 43: 60–68.

Galectin-3 and Outcomes in Patients with End-Stage Renal Disease: Data from the German Diabetes and Dialysis Study, presented by Rudolf de Boer, MD, PhD,

Associate Professor of Cardiology at the University of Groningen, the Netherlands; American Heart Association Scientific Presentation,

November 2011.

Galectin-3 in ESRD: 2011 AHA Presentation

Department of Cardiology, University Medical Center Groningen, The Netherlands

Department of Medicine, Division of Nephrology, University Hospital Würzburg, Germany

Galectin-3 and Cardiovascular Outcomes in Patients

with End-Stage Renal Disease

Data from the German Diabetes and Dialysis Study

Rudolf

Boer

Christoph

Wanner

Katja

Blouin

MSc

Christiane

Drechsler

Galectin-3 in ESRD:

2011 AHA Presentation Conclusions

•

Galectin-3 levels are extremely elevated in patients with ESRD

on hemodialysis

•

High galectin-3 in independently associated with stroke, CV

events, all-cause and infectious mortality in dialysis patients

Galectin-3: Promotes Organ Failure

via Scar Formation

•

Mice genetically altered to lack

galectin-3 produce much less

scar tissue in the kidney after

injury. Normal (wild-type, WT)

mice or galectin-3 knockout mice

were either left alone (-) or

surgically injured by obstructing

the outflow of urine from the

kidney (UUO). The amount of

collagen and procollagen

produced is a measure of scar

formation. As indicated, galectin-

3 knockout mice produced much

less scaring.

The American Journal of Pathology, 2008; Vol. 172, No. 2: 288-298.

Galectin-3 and Cancer

•

Over-expression of galectin-3 correlates with aggressiveness

and relapse in multiple human cancers

1,2

•

Galectin-3 expression via transfection increases metastasis in

animal models

1,2

•

Galectin-3

suppresses

anti-tumor

T-cell

activity

•

Lung tumorigenesis is reduced in galectin-3-/-

vs. wild-type

mice

•

Galectin-3

knockdown

reduces

proliferation

and

tumor

growth

Reviewed in: Liu, F., et al. 2005. Galectins as Modulators of Tumour Progression. Nature Reviews Cancer. 5:29-41proliferation ane

Reviewed in: Takenaka, Y., et al. 2004. Galectin-3 and Metastasis. Glycoconj J. 19:543-549

Demotte, N. et al. 2008. Restoring the Association of the T Cell Receptor with CD8 Reverses Anergy in Human Tumor-Infiltrating Lymphocytes. Immunity 28, 414-424

Abdel-Aziz, H., et al. 2008. Targeted Disruption of the Galectin-3 Gene Results in Decreased Susceptibility to NNK-Induced Lung Tumorigenesis:

An Oligonucleotide Microarray Study. J Cancer Res Clin Oncol. 134:777-788

Peng W, et al. 2008. Tumor Associated Galectin-3 Modulates the Function of Tumor Reactive T-cells. Cancer Research 68:7228-7236.

Galectin-3 Expression Correlates with

Reduced Patient Survival

•

Survival curve of colorectal cancer

patients with positive and negative

galectin-3 expression. The

prognosis of patients was

significantly worse in patients with

galectin-3-positive expression

(purple line) than in galectin-3-

negative patients (blue line)

(p=0.0027).

Anticancer Research 25: 3117-3122 (2005)

Galectin-3 Expression is a Potential

Prognostic Marker in Colorectal Cancer

Galectin-3 Suppresses T-Cell Activation

Source: Pierre van der Bruggen, Ludwig Institute for Cancer Research

Galectin-3

Galectin-3 binding to cell surface glycoproteins blocks

co-receptor association and cell signal transmission

GCS-100 Reverses Galectin-3

T-Cell Suppression

Source: Pierre van der Bruggen, Ludwig Institute for Cancer Research

Galectin-3

GCS-100

GCS-100 Reverses Galectin-3

T-Cell Suppression

•

T-cells were collected from 4 ovarian cancer patients and tested for their

ability to kill the tumor (T-cell Activity). Untreated cells have little activity.

After treating the T-cells with either an antibody to galectin-3 (purple) or with

GCS-100 (blue), the T-cells regain their activity and ability to kill the tumor.

Source: Dermotte et al. A Galectin-3 Ligand Corrects the Impaired Function of Human CD4 and CD8 Tumor-Infiltrating Lymphocytes and

Favors Tumor Rejection in Mice, Cancer Research, Oct 1, 2010 70 (19), pgs. 7476-7488

GCS-100 Improves Melanoma

Cancer Vaccine

•

Animals treated with GCS-100 in addition to a vaccine against the tumor

live much longer than untreated animals or animals treated with vaccination

or GCS-100 alone

Source: Dermotte et al. A Galectin-3 Ligand Corrects the Impaired Function of Human CD4 and CD8 Tumor-Infiltrating Lymphocytes and

Favors Tumor Rejection in Mice, Cancer Research, Oct 1, 2010 70 (19), pgs. 7476-7488

GCS-100 Clinical Summary

•

>140 patients dosed in 9 Phase 1 and Phase 2 clinical trials

Significant clinical activity in multiple cancers, including chronic

lymphocytic leukemia (CLL), multiple myeloma (MM), renal cell

carcinoma

•

Pharmacokinetic parameters established

Single-

and

multiple-dose

administration

30-160

mg/m

–

Once daily for 5 consecutive days (21-day cycle)

–

Effective half-life of 36 hours

•

Demonstrated single-agent activity

•

Broad-based synergy in vitro with both chemotherapy and

other targeted agents

GCS-100 Side Effects Consistent with

Immune Modulation

•

Doses

160

mg/m

well

tolerated

24-patient

solid

tumor

study

No hematological toxicity

Only drug-related grade 3 AEs = rash (8%), arthralgia (8%) and myalgia

(4%), all consistent with immune effects

•

Dose-Limiting Toxicity

Grade 3 rash

•

Rash

Immune-mediated leucoclastic vasculitis

Responds to systemic steroid therapy

Not associated with additional organ involvement

Develops during, or within 1-2 days after completion of, dosing during a

treatment cycle

No clinical reports of skin desquamation, necrosis, or ulceration

Did not preclude additional treatment cycles with GCS-100

Phase 2a Single-Agent CLL Study: Design

•

Enrolled 24 patients with one or two prior therapies

Median age 67 years old; 15 patients > 67 years, 4 > 80 years

Prior therapies include Fludara-, Rituxan-

and chlorambucil-

containing regimens

•

Dosing regimen

160

mg/m

i.v.

daily

for

days

21-day

cycle

Regimen from Phase 1 solid tumor study

Short (~1 hour) infusion

No steroid prophylaxis

•

Primary objectives

Evaluate the short-term effect of GCS-100 on markers of cell death

Evaluate decreases in peripheral leukocyte count

Phase 2a Single-Agent CLL Study: Results

•

GCS-100 was well tolerated

Minimal hematologic toxicity

1 patient discontinued due

to grade 3 rash

No drug-related SAEs

•

In vivo evidence of caspase activation and apoptotic cell death

observed

•

Preliminary data indicate 6/24 patients (25%) achieved PR and

12/24

(50%)

achieved

SD,

for

75%

disease

control

rate

3 Patients >50% decrease in LN size

1 Patient >50% decrease in WBC with LN shrinkage

2 Patients >50% decrease in WBC

Cotter F. et al. 2008. 10th International Conference on Malignant Lymphoma

Cotter F. et al. 2009. 45th American Society of Clinical Oncology Annual Meeting

GCS-100 Clinical Summary

•

Clinical activity in multiple cancers, including chronic

lymphocytic leukemia (CLL), multiple myeloma (MM), and

renal cell

•

Well tolerated and has low toxicity based on >140 patients

treated

•

Broad-based synergy in vitro with both chemotherapy and

other targeted agents

•

Single agent activity observed

•

Rash, arthralgias, myalgias and increase in neutrophil count

consistent with immune effects

Next Steps: Clinical Trial Rational

•

Galectin-3 is is increased in patients with ESRD and the level

correlates to overall survival

•

Galectin-3 knockout mice develop less kidney scar after injury

•

Patients with ESRD have no existing therapeutic options

outside of renal transplantation

•

ESRD patients have a high mortality rate and biomarkers

correlate with survival

Albumin

Galectin-3

Preservation of urine output

Next Steps: Clinical Trial Rational

•

GCS-100 has shown single-agent activity in cancer

•

Galectin-3 is is increased in patients with renal failure

•

Galectin-3 knockout mice develop less kidney scar after injury

•

Cancer therapies often cause kidney injury and scar formation

with reduced renal function

•

There are no approved therapies for treating renal insufficiency

in cancer patients

Next Steps: Phase 1/2 Study in Cancer

Patients with Renal Insufficiency

•

Advanced-stage cancer patients (n=18)

•

GCS-100

alone

160

mg/m

weekly

•

Compare baseline to post-treatment:

Serum galectin-3

Renal function

Tumor response

Immune parameters

Intellectual Property Position

Title

Status

Coverage

Expiration

Modified Pectins,

Compositions and

Methods Related Thereto

Issued

US 8,128,966

Methods for making biologically active

modified or unmodified pectin using tangential

flow filtration having a molecular weight

between 10 and 250 kD.

2028

Modified Pectins,

Compositions and

Methods Related Thereto

Alllowed

US 13/357,325

Biologically active pectin (MW 10-250 kD)

from modified or unmodified pectin using

tangential flow filtration.

2025

Modified Pectins,

Compositions and

Methods Related Thereto

Pending

US 13/400,007

Highly bioactive forms of modified pectin and

methods of making modified pectin of high

molecular weights and/or lacking low

molecular weight materials

2025

Compositions and Uses

of Galectin Antagonists

Pending

US 11/803,150

Methods

for

reducing

the

rate

cancer

growth

by treating with a galectin inhibitor, such as

modified pectin, and a topoisomerase inhibitor

2027

Corporate Highlights

Technology

•

Pipeline

•

May prevent or reverse organ failure by mediating fibrosis via galectin-3 sequestration

Binds galectin-3 and reverses T-cell suppression

Extensive clinical data with clear single agent activity and favorable safety profile

Management and Investors

•

Milestones

•

Immune therapy platform targeting, Galectin-3, an emerging key with a

demonstrated role in organ failure and cancer via immune regulation

Clear path to proof-of-concept

Product

candidate:

GCS-100

Leading,

clinical

stage

galectin-3

antagonist

Experienced and driven

Near-term, cost-efficient clinical and preclinical milestones to drive value

Thank You

La Jolla Pharmaceutical (LJPC) 8-KRegulation FD Disclosure