Acorda Therapeutics: Preclinical Pipeline
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Transcript of 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
4 | Corporate Overview March 2009
Product Pipeline
5 | Preclinical Programs March 2009
Preclinical Pipeline
Neuregulins
ChondroitinaseRemyelinatingAntibodies
6 | Preclinical Programs March 2009
• 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
7 | Corporate Overview March 2009
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
8 | Preclinical Programs March 2009
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
9 | Preclinical Programs March 2009
Neuroprotection: Data Highlights
Forelimb Placing Test
(Seroogy, U Cincinnati)
Rat 6-OHDA Model
(Acorda Therapeutics)
Rat Permanent MCAO Model
10 | Preclinical Programs March 2009
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
11 | Preclinical Programs March 2009
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
12 | Preclinical Programs March 2009
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
13 | Preclinical Programs March 2009
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
A)
** P < 0.05
TMEV model
14 | Preclinical Programs March 2009
Future rHIgM22 Milestones
• Complete cGMP manufacturing process and purification
• Complete toxicology studies–Studies underway
• Completed Pre-IND meeting
15 | Preclinical Programs March 2009
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
16 | Preclinical Programs March 2009
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
17 | Preclinical Programs March 2009
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
18 | Preclinical Programs March 2009
Chondroitinase Milestones
• Demonstrate efficacy in larger animal models of traumatic injury
• Advance lead Chondroitinase from research to preclinical development
19 | Preclinical Programs March 2009
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