On a mission to provide quality of life to patients suffering from rare CNS diseases Proinvestor Life Science Seminar, 27th March 2012

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uncertainties materialize, or should other risks or uncertainties not foreseen or not identified

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may be materially and adversely affected as compared the forward-looking statements

described in this presentation. NeuroSearch does not undertake to meet, or give any guarantee

that it will meet, the intentions or goals that may be described in this presentation.

 

Content

Introduction to NeuroSearch

Huntexil® - Clinical results

Huntexil® - Regulatory feedback

Huntexil® - The new phase III programme Prime-HD

Huntexil® - The commercial potential

Financials

March 20123

Company introduction

Established in 1989; went public in 1996

Restructuring of the company announced in September 2011 to fully focus on Huntexil® - reduction to 35 employees by mid 2013

~110 employees as of 1 March with headquarters in Denmark

Huntexil® for treatment of motor symptoms associated with Huntington’s disease as main, fully owned drug candidate

Cash position of DKK 221m (USD 39m) as of end December 2011

4 March 2012

Huntexil®

Highlights

Two large clinical studies, HART and MermaiHD, failed to meet their primary endpoints, but showed statistically significant improvement in motor function as measured by Total Motor Score (TMS) which was the secondary/tertiary endpoint *

The observed effect on TMS is calculated to correspond to approximately 6-8 months disease progression

A statistically significant dose-response relationship observed in HART

Safe and well-tolerated in the doses tested with no worsening of other disease symptoms

Designated orphan drug status with both the FDA and the EMA granting 7 and 10 years market exclusivity, respectively

All rights for Huntexil® are retained

5 March 2012

* TMS is the “golden standard” measure of motor function in HD

6

Content

Introduction to NeuroSearch

Huntexil® - Clinical results

Huntexil® - Regulatory feedback

Huntexil® - The new phase III programme Prime-HD

Huntexil® - The commercial potential

Financials

March 2012

What is Huntington’s disease?

Huntington’s disease has serious negative implications on quality of life for patients and their families

Fatal, autosomal dominant genetic disorder

−Mutant huntingtin gene with CAG expansion

−Neurodegeneration in several brain areas, especially in the striatum

−Glutamate and dopamine neurotransmissions are disrupted

Symptom onset typically around 30–50 years of age

−Motor dysfunction

−Cognitive impairment

−Behavioural changes

Continuous disease progression for 10–20 years after symptom onset

HD has a marked impact on patients’ daily functioning and ultimately leads to loss of independence and premature death

7 March 2012

Huntexil®

Belongs to the new class of dopidines

Huntexil® is able to enhance or inhibit dopamine-dependent functions

Dopamine D2 competitive antagonist with fast off-rate kinetics

Through its effects at D2 receptors and its downstream effects on glutamate transmission, Huntexil® can stabilise dysregulated psychomotor functions

Unique therapeutic activityUnique therapeutic activity

Neuroleptic: Psychomotor activityNeuroleptic: Psychomotor activity Huntexil®: Psychomotor activityHuntexil®: Psychomotor activity

High

Low

Normal

High

Low

Normal

8 March 2012

Huntexil®

Phase IIb/III trials

MermaiHD Randomised, double-blind and placebo-controlled phase III study

Conducted in HD centres in Austria, Belgium, France, Germany, Italy, Portugal, Spain and United Kingdom

Objective: Evaluate efficacy and safety of Huntexil® (45 mg once or twice daily) after 26 weeks with mMS as primary endpoint and TMS among other endpoints

437 patients were randomised

HART Randomised, double-blind and placebo-controlled phase IIb study

Conducted in HD centres in Canada and United States

Objective: Evaluate efficacy and safety of Huntexil® (10, 22.5 or 45 mg, all twice daily) after 12 weeks + establish dose-response relationship with mMS as primary endpoint and TMS among other endpoints

227 patients were randomised

9 March 2012

The Total Motor Score, TMS

Measures 15 items related to motor symptoms

Disease progression: ~4–5 points increase p.a.*

TMS is the most commonly used scale to assess movement disorders related to HD

mMS is a sub-scale of TMS, excluding eye and involuntary movements

TMS is part of the Unified Huntingtons Disease Rating scale (UHDRS)

*Mahant N, McCusker EA, Byth K, Graham S. Huntington’s disease: clinical correlates of disability and progression. Neurology 2003; 61: 1085–92: “Over a 12-month period, the natural progression in the UHDRS–TMS is approximately 4.6 points.”

10 March 2012

Huntexil®

Effect on TMS

11

The HART studyThe MermaiHD study

The MermaiHD study

Phase III study with 437 patients in eight European countries

Statistically significant effects on TMS after 26 weeks (-3.0 points (p = 0.004))

The primary endpoint (mMS) was not met

The HART study

Phase IIb study with 227 patients in the United States and Canada

Statistically significant effect on TMS after 12 weeks (-2.8 points (p = 0.039))

The primary endpoint (mMS) was not met

March 2012

Huntexil® The HART study – TMS dose response

12 March 2012

Gait and balance

Huntexil®

Effect on hand movements, gait and balance

13

The HART study

The MermaiHD study

The HART study

The MermaiHD study

Hand movements

March 2012

March 201214

GaitInstruction video by courtesy of Ralf Reilmann, MD

Huntexil®

Safety profile is similar to placebo

Received Treatment*

Placebo

Huntexil® HART

10 mg BID

Huntexil® HART

22.5 mg BID

Huntexil® MermaiHD

45 mg once daily

Huntexil® HART &

MermaiHD45 mg BID

Total, active groups Total

Total Number of Patients 202 56 55 148 203 462 664

Any treatment emergent AEs 125 (62%) 30 (54%) 25 (46%) 91 (62%) 139 (69%) 285 (62%) 410 (62%)

Any treatment emergent SAEs 12 (6%) 0 2 (4%) 10 (7%) 12 (6%) 24 (5%) 36 (5%)

Deaths 2 (1%) 0 0 1 (0.7%) 1 (0.5%) 2 (0.4%) 4 (0.6%)

Any treatment emergent AEs Inter/Stop

18 (9%) 5 (9%) 2 (4%) 13 (9%) 25 (12%) 45 (10%) 63 (10%)

Any treatment emergent AEs Related

83 (41%) 18 (32%) 14 (26%) 56 (38%) 89 (44%) 177 (38%) 260 (39%)

15 March 2012

*BID = Twice daily

Huntexil®

Post-study provision of drug

EU

− After the open-label phase of MermaiHD, patients and physicians expressed an interest in continuing treatment

− A compassionate use programme was initiated in 2009

− The programme has subsequently been locally adopted and approved in all eight countries where MermaiHD was conducted

− 130 patients were enrolled by December 2011

− >40% of all completing patients are now included in the programme

North America

− Post-study access to Huntexil® was not planned for after the 12-week HART study (phase IIb)

− An open-label extension study was launched

− First patient was enrolled in March 2011 and when completed by December 2011, 118 patients were enrolled

− >50% of all eligible patients are thus included in the programme

16 March 2012

17

Content

17

Introduction to NeuroSearch

Huntexil® - Clinical results

Huntexil® - Regulatory feedback

Huntexil® - The new phase III programme Prime-HD

Huntexil® - The commercial potential

Financials

March 2012

March 201218

Feedback from FDA and EMA following discussions regarding Huntexil® data package*

*Discussions in spring 2011; EMA advice on protocol assistance; FDA End-of-Phase II meeting **No formal requirement on statistical significance

Topic EMA FDA

Approvability on basis of HART, MermaiHD and earlier studies

• Additional confirmatory evidence is needed to support a MAA/NDA for Huntexil® in HD

• Additional confirmatory evidence is needed to support a MAA/NDA for Huntexil® in HD

Endpoint •TMS acceptable as primary endpoint • TMS acceptable as primary endpoint

Clinical relevance • Consistency** required between the TMS and other endpoints incl. responder rate

• Statistical significance required on global or functional scale (co-primary endpoint)

• Strong internal consistency between secondary outcomes

Dosing • Recommended to explore higher doses

Safety • Including Prime-HD, the safety database appear to be sufficient (if no new safety signals emerges)

• Including Prime-HD, the safety database appear to be sufficient

• Potential higher dose require 50 patients exposure for 1 year

19

Content

19

Introduction to NeuroSearch

Huntexil® - Clinical results

Huntexil® - Regulatory feedback

Huntexil® - The new phase III programme Prime-HD

Huntexil® - The commercial potential

Financials

March 2012

Prime-HD for Huntexil® Phase III efficacy study design

• Global study with 630 patients in three treatment arms

• Primary endpoint is TMS. The primary objective is to show efficacy of Huntexil® 45 mg twice daily on TMS

• Other endpoints include Clinical Global Impression (CGI), activities of daily living (ADCS-ADL) and non-motor scales from UHDRS

• A closed testing procedure will be applied for primary and key secondary endpoint (CGI)

• Prime-HD is powered (>90%) to detect a 3 points TMS difference with the 45 mg dose at a 5% confidence level

20 March 2012

Huntexil®

Phase III programme overview

21 March 2012

MAD has completed active phase and preliminary results support Prime-HD dosing regimen

TQT and bioEQ are performed in healthy volunteers

Abuse liability is performed in recreational drug abusers

MAD

TQT

Phase III: Prime-HD 45 mg or 67.5 mg both twice daily vs placebo, 26 weeks

Phase III extension: Open Prime-HD 67.5 mg twice daily, 26 weeks

Abuse liability

Bioequivalence

CL

INIC

AL

ST

UD

IES

Content

Introduction to NeuroSearch

Huntexil® - Clinical results

Huntexil® - Regulatory feedback

Huntexil® - The new phase III programme Prime-HD

Huntexil® - The commercial potential

Financials

22 March 2012

Huntington’s DiseaseLarge unmet medical need

(1) Huntington Disease Society for America; European Huntington’s disease associations.(2) Prevalence sources: Rawlins M. Lancet 2010;376:1372–3.

Large unmet medical need in HD

− Prevalence of symptomatic patients is estimated to be 1:10,000 in most Western countries (1)

− Approximately 110,000 symptomatic patients worldwide

Prevalence study published in The Lancet, 2010 (2)

− Minimum prevalence in England and Wales is 1.24: 10,000

− Earlier epidemiology studies are believed to have underestimated prevalence due to the ’stigma’ of the disease

Number of HD patientsNumber of HD patients(Patients: 1,000s)

c.34

c.42

c.35

North America Western Europe Eastern Europe and RoW

2323 March 2012

March 201224

Tetrabenazine (Xenazine®) is the only approved compound (only US)

− Indicated for chorea only

− Carries a premium price (~50,000 USD/pt/yr)

− ‘Black box’ warning for increased risk of depression and suicidality

Neuroleptics and antidepressants used off-label

Limited existing treatment

Health economy efforts

Cost-of-illness/Burden of disease (independent from NeuroSearch)

– To be included in the Core Value Dossier

– Includes data from all countries

Huntington Disease model (sponsored by NeuroSearch)

– To be included in the Core Value Dossier

Cost-effectiveness of Huntexil® (sponsored by NeuroSearch)

– To be included in the Local Value Dossier for HTA/P&R applications

– Includes data from country/countries relevant to local conditions

2525 March 2012

Selected findings from cost-of-illness study

The direct medical costs (hospital visits, nursing home etc) associated with

Huntington's disease in France amount to approx. EUR 24,000 per patient per year

– Indirect costs (loss of productivity etc) are even greater

A survey in Poland and Italy reported an average time spent by caregivers caring

for a patient of more than 20 hours/day

Among caregivers surveyed in the United States, 43% reported overall

dissatisfaction with their quality of life

Motor symptoms excl. chorea are the main cost drivers

Motor symptoms excl. Chorea

Chorea Behavioral symptoms*Cognitivesymptoms

Total cost to society**

Significant correlation(p < 0.01)

Not significant

Not significant

Not significant

Disease severity***Significant correlation

(p < 0.01)Not

significantNot

significantNot

significant

Preliminary results from France, adapted from Dorey et al 2010*) None of the three symptoms depression, impulsivity, psychotic disorder tested individually were significantly correlated with total cost or disease severity**) Includes direct medical costs, loss of productivity etc***) Measured on a self-reported version of the UHDRS Independence scale

2626 March 2012

27

Huntexil® Intellectual Property

Orphan Drug status with the EMA

− Orphan drug data exclusivity 10 years from approval

Orphan Drug status with the FDA

– Orphan drug data exclusivity 7 years from approval

Strong intellectual property status

− Composition-of-matter patent expires in December 2020

− Patent term extension to be granted according to normal standards

− Patent term extension to 2024 / 2025 in US and 2025 in EU, respectively

March 201227

28

MermaiHD study published in The Lancet Neurology

The MermaiHD fase III study of Huntexil® has been described in a scientific article accepted by the well-reputed journal The Lancet Neurology

Dr Andrew Feigin, The Feinstein Institute for Medical Research, New York, USA, says*:

"A well tolerated drug that produces even small benefits for patients with Huntington’s disease would be a very welcome addition to the currently available treatments for this debilitating disorder… Analysis of individual items within the UHDRS-TMS in the MermaiHD study also suggests that pridopidine might benefit features of HD for which there are currently no treatments (eye movements, hand coordination, dystonia, and gait or balance problems).“

*Press release from The Lancet Neurology

March 2012

Interviews with 24 US based clinicians treating HD patients

Test of Huntexil® -like profile (Product Q) based on hypothetical PrimeHD results

Increase understanding of US HD market

Findings confirm attractiveness of Huntexil® hypothetical profile

Positive reaction to efficacy data and appreciation of clinical relevance

Safety profile was a key benefit

Very high intention to prescribe

Clinician segments* rated Product Q from 5.5 to 6.3 on a scale to 7 being the most appealing profile

29

US Clinician Market Research performed by external consultancy

HD MARKET ASSESSMENT AND PRIDOPIDINE PROFILE EVALUATION (USA)

Conducted for NeuroSearch September 2011

Optio Biopharma Solutions, LLC

901 Mission StreetSuite 105San Francisco, CA 94103www.OptioLLC.com

*) HD experts, movement disorder specialists, general neurologists and primary care physicians

March 2012

Huntexil®

Blockbuster potential

Population and patients (example)Population and patients (example)High rate of diagnosis

Clear disease markers

Severity of the disease

Hereditary nature of disease

High treatment rate

No worsening of other symptoms

High compliance ensured by caregivers, nurses and family members

Limited other treatment options

Patients eligible

Treatment relevant from first motor symptoms until ambulatory care is no longer possible

Population and patientsNorth

AmericaWestern Europe

Eastern Europe and

RoW Total

Population 342 420 876  

HD prevalence 0,010% 0,010% 0,004%  

HD patients 34.200 42.000 35.040 111.240

Rate of diagnosis 90% 90% 60%  

Treatment rate 97% 97% 90%  

Patients eligible 70% 70% 70%  

  61% 61% 38%  

Patients (no.) for treatment 20.900 25.666 13.245 59.811

We estimate that ~60% of HD patients are potential for treatment with Huntexil®

March 201230

31

Content

Introduction to NeuroSearch

Huntexil® - Clinical results

Huntexil® - Regulatory feedback

Huntexil® - The new phase III programme Prime-HD

Huntexil® - The commercial potential

Financials

March 2012

Financial results – full year 2011

NeuroSearch Group (DKK million) 2011 2010

Revenue 0 0

Total costs *(383) (168)

Operating profit/(loss)

Net financial income/(expense)

Tax

(383)

34

0

(168)

22

47

Net result of continuing activities (349) (99)

Net result of discontinuing activities (329) (160)

Net result for Group (678) (259)

*Include DKK 170 million one-off costs regarding an impairment write-down of intangible assets and a provision for changed milestone payments to the sellers of Carlsson Research and additional DKK 99 million related to impairment charges on property and other assets

Liquidity and capital resources on 31 December 2011

• Cash and cash equivalents including securities totalled DKK 221 million

• Expected total payments for staff working on projects under the alliance with Janssen, DKK 38 million

• Remaining payments from the sale of Sophion Bioscience DKK 9 million

March 201232

Financial expectations for 2012

Financial expectations for 2012

● Operating loss on continuing operations of approximately DKK 75 million excluding

any possible costs related to the phase III programme on Huntexil®

● The Prime-HD study will not be initiated before financing is secured to finalise the

study. So far, no decision on preferred route of financing has been taken

● No further costs related to the discontinued operations are expected in 2012. The

cash flow effect in 2012 is expected to be in the region of DKK 70 million.

March 201233

Huntexil®

Highlights

Two large clinical studies, HART and MermaiHD, failed to meet their primary endpoints, but showed statistically significant improvement in motor function as measured by Total Motor Score (TMS) which was the secondary/tertiary endpoint*

The observed effect on TMS is calculated to correspond to approximately 6-8 months disease progression

A statistically significant dose-response relationship observed in HART

Safe and well-tolerated in the doses tested with no worsening of other disease symptoms

Designated orphan drug status with both the FDA and the EMA granting 7 and 10 years market exclusivity, respectively

All rights for Huntexil® are retained

34 March 2012

* TMS is the “golden standard” measure of motor function in HD

For more information, please visit; www.neurosearch.com

or write to; investor@neurosearch.com