AB SCIENCE (AB) Status Update summary

Clinical development update for masitinib

• Masitinib targets mast cells and microglia, showing promise in ALS, Alzheimer's, and progressive MS, with robust preclinical and clinical evidence supporting its neuroprotective effects.

• In ALS, masitinib demonstrated significant benefits in slowing functional decline and improving survival, with long-term data showing some patients surviving over five years, outperforming predictive models.

• EMA rejected conditional approval for ALS, but a new confirmatory study (AB23005) with improved design and broader inclusion criteria has been approved by both FDA and EMA, aiming for robust registration data.

• In progressive MS, masitinib showed a 37% risk reduction in disability progression, competitive with other emerging therapies, and confirmatory studies are authorized.

• For Alzheimer's, masitinib is differentiated by targeting the innate immune system and is the only drug with positive data in moderate patients; future strategies include combination and adjuvant therapies.

Intellectual property and market positioning

• Masitinib benefits from orphan drug status, robust use patents (extending to 2037–2041), and data exclusivity, ensuring strong protection in key markets.

• The addressable markets in ALS, progressive MS, and Alzheimer's are substantial, with masitinib positioned as a potential blockbuster if successful in any indication.

• Multiple failed competitor programs have left masitinib as one of the most advanced candidates in its fields.

Financing and partnership strategy

• Recent fundraising secured €5 million, providing 12 months of financial visibility; additional funding for future phases is expected from equity or partnerships.

• Partnership discussions are ongoing, with regulatory clarity expected to facilitate deals, especially for phase III development.

• Debt restructuring discussions are underway to prioritize R&D investment.