acorda therapeutics: preclinical pipeline

1 | Preclinical Programs November 2008

Acorda Therapeutics:Preclinical Pipeline

March 6, 2009

2 | Preclinical Programs November 2008

Forward Looking Statements

This presentation includes forward-looking statements within the meaning of the Private

Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts,

regarding management's expectations, beliefs, goals, plans or prospects should be considered

forward-looking. These statements are subject to risks and uncertainties that could cause actual

results to differ materially, including delays in obtaining or failure to obtain FDA approval of

Fampridine-SR, the risk of unfavorable results from future studies of Fampridine-SR, Acorda

Therapeutics' ability to successfully market and sell Fampridine-SR, if approved, and Zanaflex

Capsules, competition, failure to protect its intellectual property or to defend against the

intellectual property claims of others, the ability to obtain additional financing to support Acorda

Therapeutics' operations, and unfavorable results from its preclinical programs. These and other

risks are described in greater detail in Acorda Therapeutics' filings with the Securities and

Exchange Commission. Acorda Therapeutics may not actually achieve the goals or plans

described in its forward-looking statements, and investors should not place undue reliance on

these statements. Acorda Therapeutics disclaims any intent or obligation to update any forward-

looking statements as a result of developments occurring after the date of this presentation.

2 | Corporate Overview November 2008

3 | Corporate Overview March 2009

Acorda Overview

• Commercial stage biopharmaceutical company focused on restoring function in people with multiple sclerosis (MS), spinal chord injury (SCI) and related conditions of the central nervous system (CNS).

• Zanaflex operations were cash flow positive in 2008, expected to be similar for 2009

• Specialty sales force composed of 65 sales professionals • Late stage product candidate (Fampridine-SR)

– Two positive Phase 3 clinical trials completed under SPA– NDA submitted January 2009

• Compelling preclinical pipeline– Multiple approaches to regeneration and repair of the CNS – Experienced preclinical/clinical team with strong academic collaborations– Target filing an IND in 2009 and others to follow


Product Pipeline

5 | Preclinical Programs March 2009

Preclinical Pipeline

Neuregulins

ChondroitinaseRemyelinatingAntibodies


• Large family of proteins, structurally related to EGF

• Bind to ErbB receptors

• Diverse bioactivities and potential applications

– Promote growth of oligodendrocytes MS, SCI

– Protect cardiomyocytes and promote differentiation of stem cells to cardiomyocytes CHF, Cardiotoxicity, MI

– Protect neurons from stressors Stroke, TBI

• GGF2 selected as lead candidate

Neuregulins


Market Opportunities

• MS - 400,000 people in the US1

• SCI - 200,000 people in the US2

• TBI - 1.4 million new cases in the US annually3 • Stroke - 750,000 new cases in the US annually4

• CHF – 5.3 million people in the US; 400,000 new cases annually5

• MI – 7.5 million people in the US; 920,000 new cases annually6

• Cardiotoxicity – Occurs in up to 30% of patients treated with chemotherapies7

• Sources: 1- Atlas MS Resources in the World – WHO 2008; 2,3 – CDC; 4,5, 6 & 7 – AHA


Highlights of Neurological Applications

• Reduces relapses and disease severity in EAE model of MS (Cannella et al)

• Reduces infarct size and improves function in transient ischemia model of stroke (Xu et al.)

• Improves function with wide therapeutic window in permanent ischemia model of stroke (Acorda)

• Reduces neuron loss and enhances dopamine production in models of Parkinson’s Disease (Yurek et al., Seroogy et al.)

• Others: peripheral nerve injury, SCI


Neuroprotection: Data Highlights

Forelimb Placing Test

(Seroogy, U Cincinnati)

Rat 6-OHDA Model

(Acorda Therapeutics)

Rat Permanent MCAO Model


2009 Neuregulin Milestones

• Expect to complete cGMP manufacturing process and produce clinical material

–CMC ICOS in Bothwell, WA

• Expect to complete toxicology program–Rodent and non-human primate studies underway

• Expect to file IND for CHF – late 2009– Pending satisfactory animal safety data

• Conduct parallel efficacy studies –Expand possible indications - neurological–Clarifying dosing and routes of administration–Determine therapeutic window


Remyelinating Monoclonal Antibodies

• Human IgM antibodies identified by Moses Rodriguez at Mayo Clinic

• Protect oligodendrocytes and promote remyelination• Compelling preclinical data in 3 animal models, effects

include:– Protection of oligodendrocytes

– Promotion of remyelination

– Functional benefit

• Lead Selected – rHIgM22• Exclusive worldwide license from Mayo Clinic


rHIgM22 Mechanism of Action

MYELIN GENE% of Control Expression

T TEST

Proteolipid protein 137 ± 15 p = 0.05

DM-20 PLP 147 ± 3 p = 0.02

Myelin basic protein 124 ± 0.4 p = 0.03

Myelin-associated glycoprotein 107 ± 9 p = 0.3

Myelin oligodendrocyte basic protein

191 ± 19 p = 0.005

Myelin oligodendrocyte glycoprotein

140 ± 17 p = 0.04

Data are shown as percent of myelin gene levels measured in infected animals treated only with PBS. Values shown are means of three independent experiments ± SEM.

Bind and Oligomerize Targets within Lipid Rafts

Protect Oligodendrocytes and Enhance Myelin Production


rHIgM22 Stimulates Remyelination

rHIgM14

rHIgM22

0

5

10

15

20

25

14 22 PBS

Human mAbs

AR

EA

OF

CN

S R

EM

YE

LIN

AT

ION

(% O

F A

RE

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** P < 0.05

TMEV model


Future rHIgM22 Milestones

• Complete cGMP manufacturing process and purification

• Complete toxicology studies–Studies underway

• Completed Pre-IND meeting


Chondroitinase Program

• Enzyme that degrades growth inhibitors in the CNS

• Shown to promote CNS regeneration and plasticity

• Functional recovery in multiple models of CNS damage– Brain and spinal cord

– Different species

– Results replicated in at least 6 laboratories

• Evidence for improvements in:– Locomotor and forelimb motor function

– Sensory function

– Autonomic (bladder) function

– Visual function


Acorda Study:Chondroitinase improves locomotor function in rat model of spinal cord injury*

*Journal of Neurotrauma, Volume 22 Number 2 February 2005

Effect of Chondroitinase ABC I on locomotor activity following contusive spinal cord injury

0

2

4

6

8

10

0 20 40 60 80

Days post injury

BB

B s

core

(mea

ns

+ S

EM

)

aCSF

Penicillinase

Chase ABC I


Acorda Study:Chondroitinase improves bladder function in rat model of spinal cord injury

Residual Urine Volumes: Moderate

0

2

4

6

8

10

12

0 10 20 30 40 50 60 70

Days post injury

Me

an

Da

ily U

rin

e V

olu

me

s (

mill

ilite

rs)

Penicillinase

Chondroitinase


Chondroitinase Milestones

• Demonstrate efficacy in larger animal models of traumatic injury

• Advance lead Chondroitinase from research to preclinical development


2009 Preclinical Milestones

• Neuregulins – Expect to file IND with FDA in late 2009

• rHIgM22 – Establish cGMP manufacturing and continue safety program

• Chondroitinase – Enhance animal POC data

End of Presentation

acorda therapeutics: preclinical pipeline

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