Medicinova, Inc. · NASDAQ:MNOV
Medicinova — Investor Presentation (July 2012)
57 slides from Medicinova's investor presentation, Jul 5, 2012
Slide 1: Accelerating the global development and commercialization of innovative pharmaceuticals
Exhibit 99.1 Accelerating the global development and commercialization of innovative pharmaceuticals
Slide 2: Forward-Looking Statements
Forward-Looking Statements Statements in this presentation that are not historical in nature constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include statements regarding M
Slide 3: Corporate Overview: MediciNova, Inc.
Corporate Overview: MediciNova, Inc. MediciNova Overview: • Founded in September 2000 • Headquartered in San Diego, CA, with an office in Tokyo, Japan • Dual listing on NasdaqGM as MNOV and Osaka Securities Exchange as 4875 • $37.5 million market cap (NasdaqGM) as of 6/26/2012 (aggregate value of 18
Slide 4: Business Model
Business Model: Return On Investment In-License: • Novel, small-molecule product candidates with significant clinical or preclinical data packages and attractive market opportunities Conduct Proof-of-Concept Clinical Trials: • Conduct Phase 1 and Phase 2 clinical trials to demonstrate safety and eff
Slide 5: Commercially-Attractive Diversified Portfolio
Commercially-Attractive Diversified Portfolio Product Candidates Preclinical Phase 1 Phase 2 Phase 3 Bedoradrine Sulfate (MN-221) Program Acute Exacerbations of Asthma Exacerbations of COPD Preterm Labor Ibudilast (MN-166) Program Progressive Multiple Sclerosis Neuropathic Pain Drug Addiction Non-Co
Slide 6: Significant Milestones
Significant Milestones Milestone: Timeline*: Receive Use Patent for Ibudilast in Progressive MS Patients1Q, 2012 Results from Phase 2b MN-221-CL-007 Acute Asthma Trial 2Q, 2012 Top-line Results from Phase 1b Multi-Dose Trial in COPD 3Q, 2012 Plan to Announce Phase 2 Clinical Program for Ibudilast (A
Slide 7: MN-221: Acute Exacerbations of Asthma, Exacerbations of COPD
MN-221: • Acute Exacerbations of Asthma • Exacerbations of COPD
Slide 8: Acute Exacerbations of Asthma (AEA)
Acute Exacerbations of Asthma (AEA) Definition: • Long-lasting and severe asthma episode that is not responsive to initial bronchodilator or corticosteroid therapy Market Opportunity*: • Approximately 1.9 million annual emergency room visits in the U.S. • ~500,000 annual hospitalizations in U.S. (~5
Slide 9: COPD Exacerbations
COPD Exacerbations A COPD exacerbation is a sustained worsening of the Hospitalization rates amongst patient's condition, from the stable state and beyond Asthma and COPD patients normal day-to-day variations, that is acute in onset. Thousands COPD exacerbations are associated with a significant 1,9
Slide 10: MN-221: A New Approach to Treating Acute Exacerbations
MN-221: A New Approach to Treating Acute Exacerbations (Asthma & COPD) MN-221:A novel,highlyselective adrenergic receptor agonist Potential advantages over current therapy: 1. Improved Efficacy • Route of administration (IV vs. inhalation) • Facilitates bronchodilation and onset clinical improvement
Slide 11: Acute Asthma Treatment Flow in Emergency Departments in the U.S.
Acute Asthma Treatment Flow in Emergency Departments (EDs) in the U.S. Input: 1ststline therapy in ED: 2nd ndline therapy in ED: Hospitalization: 1,900,000 patients Patients receive SOC, Patients who do not Patients who do not with acute many while in the initially respond continue respond to SOC ar
Slide 12: MN-221 for Treatment of AEA: Pivotal Trial Development Strategy
MN-221 for Treatment of AEA: Pivotal Trial Development Strategy End-of-Phase 2 Meeting with the FDA Scheduled for Monday, October nd 22nd, 2012 Pivotal Trial Design Modifications from Phase 2 Clinical Trial Based on these Core Principles*: 1. PrimaryEndpointshouldbeFEV1 improvement atHour1 (delta)or
Slide 13: MN-221-CL-007: Phase 2 Trial Goals
MN-221-CL-007: Phase 2 Trial Goals Goals of the Phase 2b Clinical Trial: 1. Assess Efficacy adjunctive to SOC treatment 2. Establish Safety 3. Validate Proof-of-concept (POC) and Determine Pivotal Trial Design 4. Develop a basis for a successful End-of-Phase 2 Meeting with the FDA © MediciNova, Inc.
Slide 14: MN-221-CL-007: Study Design
MN-221-CL-007: Phase 2 Trial Study Design • Randomized, placebo-controlled, double-blind, multi-center Phase 2 clinical trial • 175 patients enrolled with acute exacerbations of asthma at multiple US ED sites • 164 patients in the Efficacy Evaluable (EE) population; some patients early- terminated f
Slide 15: Potential New Model for Treating AEA in the ER
MN-221: Potential New Model for Treating AEA in the ER Time (Hours) 0 1 2 3 4 5 6+ Admit Current Model Repeat Repeat Repeat Repeat “Wait & See” SOC SOC SOC SOC SOC Patient Arrives Discharge New Model Discharge “Early Intervention Of MN-221” MN-221 SOC + SOC Patient Arrives Admit ER cost per hour per
Slide 16: MN-221-CL-007: Efficacy Endpoints for FEV1
MN-221-CL-007: Efficacy Endpoints for FEV1 AUC of Change in 1FEV (mL) MN-221 EE Population, MM* 33% Improvement Placebo 54% Improvement 56% Improvement 100% Improvement p=0.066 100% 250% Improvement Improvement p=0.065 p=0.046 *EE = Efficacy Evaluable Population; MM= Mixed Model was used for statist
Slide 17: MN-221-CL-007: Change in FEV1 at Hour 1
MN-221-CL-007: Change in FEV1 at Hour 1 100% Change in FEV 1 (mL) MN-221 Population: EE Improvement Placebo 160% Improvement Hour 1 Mean Hour 1 Median 17
Slide 18: MN-221-CL-007: Improvements in Clinical Symptoms
MN-221-CL-007: Improvements in Clinical Symptoms Change in Dyspnea Index Score Change in Respiratory Rate Mean AUC(0-3) Mean AUC (0-3) Population: EE* Population: EE* p=0.039 Baseline: Baseline: 68.64% 64.57% Units are dyspnea index score Units are breaths/min. (0-10 scale; 5=severe, 3=moderate, 2=s
Slide 19: MN-221-CL-007: Analysis of High Responders
MN-221-CL-007: Analysis of High Responders Analysis of High Responders: FEV1 Improvement at Any Time Point during the Treatment Period EE population* FEV1 Placebo (%) MN-221 (%) P-value Improvement > 200mL 37 / 77 (48%) 48 / 78 (62%) 0.09 > 10% pred. 25 / 77 (32%) 34 / 78 (44%) 0.15 *EE = Efficacy E
Slide 20: MN-221-CL-007: Pharmacoeconomic Benefits
MN-221-CL-007: Phase 2 Trial Pharmacoeconomic Benefits The addition of MN-221 resulted in a 17% A higher percentage of patients in the placebo reduction in hospital admissions* group returned to the ER within 7 days *EE = Efficacy Evaluable Population; Performing Sites analysis includes patients fro
Slide 21: MN-221 Met its Goals of the Phase 2 Program
MN-221-CL-007: Phase 2 Trial MN-221 Met its Goals of the Phase 2 Program 1. Assess Efficacy adjunctive to SOC treatment MN-221 group showed improvement over placebo group in measurements of lung function MN-221 showed a notable reduction in hospital admissions in performing sites and a notable reduc
Slide 22: MN-221-CL-012: Ongoing Phase 1b/2a COPD Trial
MN-221-CL-012: Ongoing Phase 1b/2a COPD Trial • Randomized, placebo-controlled, double-blind, Phase 1b/2a clinical trial • ~ 20 stable moderate-to-severe COPD patientsFEV (30% 1 80%) • Two treatment groups • 1,200µg infusions ofMN-221every12/24hours(15pts.)over4 days • Placebo (5 pts.) • Primary obj
Slide 23: MN-221: Market Opportunity, Patent Summary, Next Steps
MN-221: • Market Opportunity • Patent Summary • Next Steps
Slide 24: MN-221 Market Opportunity
MN-221 Market Opportunity* COPD Market Acute Asthma Exacerbations US $375-400 million $380-420 million Europe $200-300 million $200-300 million Rest of World $150-250 million $150-250 million Worldwide $725-950 million $730-970 million MN-221 Sales Potential Combined Worldwide MN-221 Sales Potential
Slide 25: MN-221 Patent Summary
MN-221 Patent Summary • The U.S. patent for MN-221 has composition of matter and method of use claims and is set to expire no earlier than February 2017 • Corresponding composition of matter patents in various other countries • U.S. patent expiration does not include Waxman-Hatch patent term restora
Slide 26: MN-221 Next Steps
MN-221 Next Steps 1. Complete COPD Phase 1b/2a Clinical Trial Important Safety data of multiple infusions Validation of hand-held spirometry device 2. Market Analysis Collaborate with leading market research firm to quantify the value of reduced hospital admissions Quantify the value of reduced ER v
Slide 27: Ibudilast (MN-166/AV-411)
Ibudilast (MN-166/AV-411): • Progressive Multiple Sclerosis • Neuropathic Pain • Addiction
Slide 28: Ibudilast (MN-166) Overview
Ibudilast (MN-166) Overview Ibudilast (MN-166) • Oral administration • Safe and well tolerated • Approved in Japan/Korea over 3.2M patient exposures • >420 subjects treated with ibudilast • Dosing up to 100 mg daily doses • Mechanism(s) of action primarily: • Inhibition of macrophage Migration Inhib
Slide 29: Ibudilast (MN-166) Program
Ibudilast (MN-166) Program: Ongoing and Future Development 1Q 2012 2Q 2012 3Q 2012 4Q 2012 Ibudilast (MN-166) Program Progressive MS* / Neuropathic Pain* *Pending Grant / Phase 2b Trial (POC) Partner Funding Opioid Dependence Columbia University Phase – 2a Trial Medication Overuse Headache Pain Univ
Slide 30: Strategy for Ibudilast's Development
Strategy for Ibudilast’s Development • Sustain NIDA-sponsored Drug Addiction Development • Potential for Phase 2 POC Trial support • Potential for Gov’t and MS Society consortium funding of Phase 2 Progressive MS POC Trial • Collaboration with Development Partner: 1. Advance to Phase 2b Proof-of-Con
Slide 31: Patent/Commercial Overview
Patent/Commercial Overview Method of Use Composition of Matter nd Neuropathic MIF Inh. 2 Generation MS Addiction Progressive MS AV1013 Pain screen Analogs Exp. 2018 Exp. 2025 Exp. 2027 Exp. 2026 Exp. 2029 Exp. 2027 AV1013 Ibudilast + Enantiomer Acute & Sub- AnxietyTraumatic - chronic Pain MOH Pain B
Slide 32: Management Team with Global Experience
Management Team with Global Experience Years Leadership Background Experience Yuichi Iwaki, MD, PhD 35 Professor at USC, formerly Professor at University CEO & President of Pittsburgh; Advisor to JAFCO, Tanabe Michael Coffee 26 Avigen, Amarin Corp., Elan Pharmaceuticals, N.A., Chief Business Officer
Slide 33: Financial Overview
Financial Overview Financial Resources: • $11.0millionincash& cashequivalents asof3/31/2012 • Including $10 million raised through equity sale and non-dilutive funding by Kissei • $7.5 million raised in private stock sale to Kissei Pharmaceutical Co., Ltd. • $2.5 million additional non-dilutive R&D
Slide 34: MediciNova Corporate Summary
MediciNova Corporate Summary Translational Medicine & Clinical development expertise Strong international presence, especially Japan Large and small pharma/biotech experience 1. Announced Results from CL-007 Q2 Trial - 2. Announce Results from CL-012 Q3 Trial - 3. Announce Phase 2 Trial(s) for Ibudi
Slide 35: Addendum
Addendum Data from Completed Trials MN-221 Ibudilast (MN-166/AV411)
Slide 36: MN-221: Data from Completed Trials
MN-221: Data from Completed Trials Asthma Program: CL-004, CL-005, CL-006, CL-007 COPD Program: CL-010 Safety Review
Slide 37: MN-221 Clinical Development
MN-221 Clinical Development Acute Asthma Program: COPD Program: • • ofMultiple doses tested at 1 hour infusion Multiple doses tested at infusion length 15min, 1hr, and 2hr • Completed 1 trial in COPD patients with • Completed 2 trials in asthmatics with stable disease stable disease • CL-010(48patie
Slide 38: MN-221 Clinical Results
MN-221 Clinical Results Improved Lung Function at Different Dosing Levels: Stable Asthmatics CL-005: Stable Moderate/Severe Asthmatics CL-004: Stable Mild/Moderate Asthmatics © MediciNova, Inc. 2012 38
Slide 39: MN-221 Clinical Results
MN-221 Clinical Results Improved Lung Function and Clinical Outcome Above and Beyond Standard of Care (SOC) CL-006: Patients Suffering from Acute Exacerbation of Asthma in Emergency Department Mean FEV 1Change from Baseline (Hour 5) MN-221 (all dose groups) + SOC vs. SOC + placebo 16% 14% 14.0% 12%
Slide 40: MN-221-CL-007: Subject Populations
MN-221-CL-007: Phase 2 Trial Subject Populations Placebo MN-221 Enrolled subjects 86 89 Safety population 84 83 EE population 83 81 Performing Sites population 70 72 Definitions ofNote: EE = Efficacy Evaluable Population Performing Sites Population: analysis includes patients from sites completing m
Slide 41: MN-221-CL-007: Efficacy Endpoints for FEV1
MN-221-CL-007: Phase 2 Trial Efficacy Endpoints for FEV1 AUC of Change in 1FEV (mL) EE Population, MM; Performing Sites* 39% Improvement p=0.066 81% Improvement 68% p=0.050 Improvement 172% Improvement p=0.065 p=0.043 119% 225% Improvement Improvement *EE = Efficacy Evaluable Population; MM= Mixed M
Slide 42: MN-221-CL-007: Improvements in Clinical Symptoms
MN-221-CL-007: Phase 2 Trial Improvements in Clinical Symptoms Change in Dyspnea Index Score Change in Respiratory Rate AUC (0-3) Mean Mean AUC (0-3) Population: EE; Performing Sites* Population: EE; Performing Sites* p=0.055 Baseline: 69.35% *EE = Efficacy Evaluable Population; Performing Sites ana
Slide 43: MN-221 Clinical Results
MN-221 Clinical Results Improved Lung Function at Different Dosing Levels: COPD Patients © MediciNova, Inc. 2012 43
Slide 44: MN-221 Safety Review
MN-221 Safety Review • MediciNova has preclinical data and clinical data which demonstrates the safety of MN-221. • In summary, we have not seen clinically significant safety concerns with MN-221 and it has been tested in over 400 subjects to date. • According to interviews of emergency room physici
Slide 45: Human Beta-Adrenergic Receptor Selectivity
Human -Adrenergic Receptor Selectivity Test Drug 2-Adrenoceptor Selectivity 1IC50(M) 2IC50(M) (IC50for 1 / IC50 for 2) Levalbuterol 7.40E-06 1.40E-06 5.3 Albuterol 9.40E-06 1.60E-06 5.9 Terbutaline 6.00E-05 6.50E-06 9.2 MN-221 5.90E-06 1.40E-07 42.4 © MediciNova, Inc. 2012 45
Slide 46: MN-221: CHEST Posters (Nov. 2010)
MN-221: CHEST Posters (Nov. 2010)* • MN-221-CL-004: Evaluation of MN-221(bedoradrine), a Novel,HighlySelectiveBeta2-Adrenergic Receptor Agonist in Mild to Moderate Asthma via Intravenous Infusion (Poster #145) • MN-221-CL-005:Comparisonof Administration Ratesof MN-221(bedoradrine), a Novel,Highly Se
Slide 47: Ibudilast (MN-166): Data from Completed Trials
Ibudilast (MN-166): Data from Completed Trials Multiple Sclerosis Program Neuropathic Pain Program Drug Abuse/Addiction Program Safety Review
Slide 48: Multiple Sclerosis Clinical Study: MN-166-CL-001
Multiple Sclerosis Clinical Study: MN-166-CL-001 Placebo-controlled, Randomized, Double-blind Phase II Study: • Year 1 P- lacebo, 30 mg/day, 60mg/day • Year 2 3- 0 mg/day, 60mg/day • 297 patients (~100 patients/group) @ 25 sites in Serbia, Ukraine, Belarus, Bulgaria and Romania Key Inclusion Criteri
Slide 49: MN-166-CL-001 Study Results
MN-166-CL-001 Study Results Indicative of Potential Neuroprotective Effect: • Reduced brain volume loss P-Value: 0.035 P-Value: 0.004 • Reduced conversion of acute lesions to persistent black holes P-Value:0.026 • Sustained disability progression was significantly less likely (~50%) Acute Clinical B
Slide 50: MN-166-CL-001: Efficacy Review (One Year)
MN-166-CL-001: Efficacy Review (One Year) Endpoints Indicative of Disease Modifying Effect (Chronic aspects of MS): Brain Volume Loss Progression to PBH EDSS progression Dose (mg/d) Dose (mg/d) Dose (mg/d) Endpoints Relating to Acute Clinical Benefit: : Statistically Time to 1st Relapse % Relapse Fr
Slide 51: Secondary Progressive MS: Subset Analysis
Secondary Progressive MS: Subset Analysis Treatment Group (patient n) 30 mg/day 60 mg/day Placebo Low Dose Group High Dose Group % Brain % Brain % Brain Magnitude of Magnitude of Subset of Volume Volume Volume Effect Effect MS Patients: Change Change Change RRMS -1.2 (81) -1.1 (69) 8% less -0.8 (75)
Slide 52: Diabetic Peripheral Neuropathic Pain Study: AV411-010
Diabetic Peripheral Neuropathic Pain Study: AV411-010 Design: Two-center (Australian), Phase 1b/2a, randomized, double-blind, placebo-controlled, parallel-group study. Subjects: • Patients, aged 18 toyears, 75 with painful diabetic peripheral neuropathy (DPN) or complex regionalpainsyndrome (CRPS)of
Slide 53: Reduction Observed in Opioid Usage
Reduction Observed in Opioid Usage 20 25 15 20 10 Mean Median 15 5 0 10 -5 5 -10 0 -15 -20 -5 Placebo AV411 © MediciNova, Inc. 2012 53
Slide 54: Greater % of Responders Above Ibudilast Plasma Thresholds
Greater % of “Responders” Above Ibudilast Plasma Thresholds VAS ‘Responder’ Plasma Ibudilast Parameter % > 1000 ng*hr/mL 60% AUC0-24h < 1000 ng*hr/mL 25% > 60 ng/mL 64% Cmax < 60 ng/mL 14% > 27 ng/mL 55% Cmin < 27 ng/mL 29% Next Steps for Neuropathic Pain: Twelve week Phase 2 DPN trial. Potential fi
Slide 55: Investigator-Led Development
Investigator-Led Development • Recent validation of CNS action in opioid withdrawal & analgesia • Ongoing Methamphetamine interaction Phase 1b • Opioid self-administration Phase 2a initiating • Ongoing Phase 2a Medication Overuse Headache Pain trial • Randomized, double-blind, placebo-controlled, in
Slide 56: Ibudilast (MN-166): Neurological Indications and Translational Record
Ibudilast (MN-166): Neurological Indications and Translational Record Indication Preclinical Clinical Validation Comment Validation Progressive MS Phase MS + (EAE) + * MN-166-Cl-001 2b indicated Neuropathic Pain + (multiple models) + AV411-010 Phase 2b enabled Opioid Dependence + (multiple models) +
Slide 57: Ibudilast - References
Ibudilast - References • Barkhof, F. et al. Ibudilast in Relapsing-Remitting Multiple Sclerosis: a Neuroprotectant? Neurology, Mar 30 2010. • Fox, R. Primary Neuroprotection: the Holy Grail of Multiple Sclerosis Therapy. Neurology, Mar 30 2010. • Kagitani-Shimono K. and Mohri I. J Neuroinflammation.