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NAFLD Activity Score (NAS) % of fibrosis area Tipelukast MN 001 MediciNova, Inc. 2014 MediciNova, Inc. 2014 Next Steps Progressive MS 15 Q2 2013: Submit New IND Amendment and New Ph2 Protocol Q3 2013: FDA approval Study Initiation Q3 2013: Screening begins Q4 2013: First Patient In Q1 2015: Last Patient In Q1 2017: Last Patient Out 1H 2017: Results available Progressive Multiple Sclerosis Progressive MS MediciNova, Inc. 2014 MediciNova, Inc. 2014 Next Steps ALS 16 Amyotrophic Lateral Sclerosis (ALS) Lou Gehrig's Disease Q3 2014: Submit New ALS Protocol as IND Amendment Q3 2014: FDA Approval to Start Study Q3 2014: IRB Submission Site Activation Q3 2014: Study Start up Database Build Q4 2014: Began Enrollment TBD: Last Patient In MediciNova, Inc. 2014 MediciNova, Inc. 2014 Next Steps NASH 17 Q1 2014: Positive preclinical data in NASH Q3 2014: Positive preclinical data in Advanced NASH Q3 2014: Prepare IND submission Q4 2014: Present NASH data at JDDW ASAP: Submit IND and Protocol Nonalcoholic Steatohepatitis (NASH) Tipelukast MN 001 MediciNova, Inc. 2014 MediciNova, Inc. 2014 18 MN 166 Ibudilast Tipelukast MN 001 Timeline Summary MEDIC I NOVA
Genes Promoting Fibrosis LOXL2 Collagen Type 1 CCR2 MCP 1 TIMP 1 LOXL2 Cross linking of collagen and elastin fibrosis Collagen Type 1 CCR2 MCP 1 TIMP 1 MediciNova, Inc. 2014 MediciNova, Inc. 2014 13 How does MN 001 work? Tipelukast MN 001 MN 001 LOXL2 Cross linking of collagen and elastin fibrosis Collagen Type 1 CCR2 MCP 1 TIMP 1 MN 001 Reduces Gene Expression LOXL2 Collagen Type 1 CCR2 MCP 1 TIMP 1 MediciNova, Inc. 2014 MediciNova, Inc. 2014 More than 600 human subjects exposed to MN 001 Phase 2 study of MN 001 in asthma with positive results MN 001 was considered generally safe and well tolerated Pre clinical data shows anti fibrotic effect in a Dose Dependent Manner Improved NAFLD Activity Score (NAS) via a reduction in hepatocyte ballooning Reduced fibrosis area 14 What Do the Data Show?
MediciNova, Inc. 2014 MediciNova, Inc. 2014 11 What is Fibrosis? Fibrosis Fibrosis is the development of excess fibrous connective tissue in an organ Fibrosis is a result of inflammation, irritation, or healing (e.g. scar) Cross linking of collagen and elastin is the final step in fibrosis Cross linking of collagen and elastin fibrosis MediciNova, Inc. 2014 MediciNova, Inc. 2014 12 How does Fibrosis Develop?
MediciNova, Inc. 2014 MediciNova, Inc. 2014 MN 166 Phase 2 RRMS Data Significant attenuation of brain volume loss (p=0.035) Significant attenuation of conversion of acute lesions to persistent black holes (p=0.004) Sustained disability progression was significantly less likely (p=0.026) Significant improvement in time to first relapse (p=0.04) MN 166 Ongoing NIH funded Phase 2b study PPMS and SPMS study Results expected early 2017 10 What Do the Data Show?
National Digestive Diseases Information Clearinghouse (NDDIC) 2. Allied Market Research 3. Coalition for Pulmonary Fibrosis 4. Research and Markets 5. Esbriet prescribing information IPF prevalence about 128,000 in the U.S. 3 (Orphan indication) Two thirds of IPF patients die within 5 years 3 IPF Market forecast: $1 Billion in 2017 4 Approved Drugs: Esbriet (pirfenidone) approved in October 2014; Esbriet Phase 3 studies enrolled mild to moderate IPF; No survival benefit shown 5 MediciNova, Inc. 2014 8 Tipelukast MN 001 Developing Novel Therapeutics MEDIC I NOVA MN 166 Ibudilast MediciNova, Inc. 2014 MediciNova, Inc. 2014 GLIAL CELL ATTENUATION Role of Glia: Type of macrophage Increases in number during brain damage Glial activation leads to neurodegeneration PDE Inhibition: Increases cAMP, reducing inflammation MIF inhibition Linked to attenuated disease progression in animal models of MS 9 How does MN 166 work?
We believe Progressive MS market is at least as large as RRMS market. Approved Drugs: NONE APPROVED for long term treatment of Progressive MS Amyotrophic Lateral Sclerosis (ALS) Lou Gehrig's Disease 1. Source: National Multiple Sclerosis Society 2. Source: ALS Association 3. Source: Cowen Co. estimate 4. Cochrane Database of Systematic Reviews Fatal: ALS Life expectancy is 2 5 years 2 ALS affects up to 30,000 people in the U.S. 2 (Orphan indication) Market opportunity: an effective new drug for ALS could generate sales $1 billion per year 3 Approved Drugs: RILUZOLE increases survival by only 2 3 months 4 MediciNova, Inc. 2014 MediciNova, Inc. 2014 7 Fibrotic Diseases Idiopathic Pulmonary Fibrosis IPF Nonalcoholic Steatohepatitis NASH NASH prevalence in the U.S. is 2 5% 1 Additional 10 20% have fatty liver due to being overweight or obese 1 NASH Market forecast: $1.6 billion by 2020 2 Approved Drugs: NO TREATMENT APPROVED 1.
You are cautioned not to place undue reliance on these forward looking statements, which speak only as of October 17, 2014. MediciNova disclaims any intent or obligation to revise or update these forward looking statements. Forward Looking Statements MediciNova, Inc. 2014 MediciNova, Inc. 2014 Novel product candidates in clinical development with encouraging efficacy and safety data MN 166 (ibudilast) for the treatment of Progressive MS, ALS, and Drug Dependence Two large Phase 2b studies ongoing (Progressive MS and methamphetamine) Initiated clinical development in ALS in 2014 Patents cover Progressive MS and addiction MN 001 for the treatment of NASH (nonalcoholic steatohepatitis) and IPF (idiopathic pulmonary fibrosis) MN 221 for the treatment of acute exacerbations of asthma Well capitalized Experienced management team 3 MediciNova Highlights MediciNova, Inc. 2014 MediciNova, Inc. 2014 4 MediciNova: Active Programs in Clinical Development in Sep. 2013 Active Programs as of September 2013 MediciNova, Inc. 2014 MediciNova, Inc. 2014 5 MediciNova: Active Programs in Clinical Development in Oct. 2014 MediciNova, Inc. 2014 MediciNova, Inc. 2014 6 Neurodegenerative Diseases Progressive Multiple Sclerosis Progressive MS MS affects more than 400,000 people in the U.S. and 2.3 million worldwide 1 Patients experience a diminished quality of life (e.g. fatigue, walking difficulties, weakness, pain, cognitive changes, depression) 1 Market opportunity: Total sales of RRMS drugs were $16 billion worldwide in 2013.
Factors that may cause actual results or events to differ materially from those expressed or implied by these forward looking statements include, but are not limited to, risks of obtaining future partner or grant funding for development of MN 166, MN 221, MN 001, and MN 029 and risks of raising sufficient capital when needed to fund MediciNova's operations and contribution to clinical development, risks and uncertainties inherent in clinical trials, including the potential cost, expected timing and risks associated with clinical trials designed to meet FDA guidance and the viability of further development considering these factors, product development and commercialization risks, the uncertainty of whether the results of clinical trials will be predictive of results in later stages of product development, the risk of delays or failure to obtain or maintain regulatory approval, risks associated with the reliance on third parties to sponsor and fund clinical trials, risks regarding intellectual property rights in product candidates and the ability to defend and enforce such intellectual property rights, the risk of failure of the third parties upon whom MediciNova relies to conduct its clinical trials and manufacture its product candidates to perform as expected, the risk of increased cost and delays due to delays in the commencement, enrollment, completion or analysis of clinical trials or significant issues regarding the adequacy of clinical trial designs or the execution of clinical trials, and the timing of expected filings with the regulatory authorities, MediciNova's collaborations with third parties, the availability of funds to complete product development plans and MediciNova's ability to obtain third party funding for programs and raise sufficient capital when needed, and the other risks and uncertainties described in MediciNova's filings with the Securities and Exchange Commission, including its annual report on Form 10 K for the year ended December 31, 2013 and its subsequent periodic reports on Forms 10 Q and 8 K.
These forward looking statements may be preceded by, followed by or otherwise include the words believes, expects, anticipates, intends, estimates, projects, can, could, may, will, would, considering , planning or similar expressions. These forward looking statements involve a number of risks and uncertainties that may cause actual results or events to differ materially from those expressed or implied by such forward looking statements.
These forward looking statements include statements regarding MediciNova s clinical trials supporting the safety and efficacy of its product candidates and the potential novelty of such product candidates as treatments for disease, plans and objectives for clinical trials and product development, strategies, future performance, expectations, assumptions, financial condition, liquidity and capital resources.
MediciNova, Inc. 2014 Developing Novel Therapeutics for the Treatment of Serious Diseases with Unmet Medical Needs Exhibit 99.1 MediciNova, Inc. 2014 MediciNova, Inc. 2014 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.