MediciNova (MNOV) investor relations material

Investment highlights and business model

• Late-stage ALS program (MN-166) completed enrollment in Phase 2b/3 trial, targeting a $3B+ orphan market with limited treatment options.

• MN-166 and MN-001 have well-established safety profiles, Orphan Drug Designation, and Fast Track status.

• Multiple late-stage assets are positioned for commercial partnerships, with additional pipeline indications in neurological, fibrotic, and metabolic diseases.

• Capital-efficient model with $27.3M cash, no debt, and annual burn of $12-13M, supporting runway through key milestones.

• Academic and government partnerships fully fund non-core programs, providing non-dilutive revenue opportunities.

ALS program and clinical development

• MN-166 (ibudilast) is a CNS-penetrant small molecule with multiple mechanisms: MIF, PDE, and TLR4 inhibition, reducing neuroinflammation and promoting neuroprotection.

• Phase 2a ALS study showed more responders in MN-166 group for functional and quality-of-life measures.

• Ongoing Phase 2b/3 COMBAT-ALS trial (n=234) is fully enrolled, with topline data expected by end of 2026; primary endpoint is combined function and survival.

• NIH-funded SEA-NOBI-ALS study targets late-stage ALS patients, with $22M funding and 50% enrollment as of January 2026.

• MN-166 is differentiated by broad target population, oral administration, and multi-target mechanism, addressing high unmet need in ALS.

Metabolic and non-core pipeline

• MN-001 (tipelukast) is an oral small molecule with anti-inflammatory, anti-fibrotic, and lipid-lowering properties, targeting ASCVD, hyperlipidemia, T2DM, and NAFLD.

• Phase 2 NAFLD/NASH + HyperTG study showed significant reduction in serum triglycerides and increase in HDL-C, especially in T2DM patients.

• Ongoing Phase 2 study in HyperTG + T2DM + NAFLD (n=40) completed enrollment; topline data expected Q3 2026.

• Multiple non-core programs are externally funded and managed by academic partners, with MediciNova retaining commercialization rights.