Cereno Scientific (CRNO) investor relations material

Strategic focus and business development

• Emphasis on pioneering epigenetic modulation via HDAC inhibition for cardiopulmonary diseases, targeting rare conditions with high unmet need and aiming for disease modification and remission rather than symptom management.

• Shifted CS014's initial indication from IPF to pulmonary hypertension with interstitial lung disease to optimize market entry, cost, and probability of success; IND submission planned for this year.

• Actively pursuing partnerships with pharma, aiming for a deal between phase IIb approval and data readout, with ongoing discussions at global, regional, and local levels.

• Analyst-estimated deal value for CS1 after a successful phase IIb is around $2 billion, with peak sales potential estimated at $2–4 billion annually.

• Biotech innovation is highlighted as the main driver for new drug development, with pharma primarily launching rather than innovating, reinforcing the importance of strategic partnerships.

Clinical progress and trial design

• CS1 is advancing to a global phase IIb trial in pulmonary arterial hypertension, with first patient in targeted for Q2 and top-line data expected in 2028.

• The trial is designed for disease modification, featuring a 9-month primary endpoint (pulmonary vascular resistance), medication withdrawal period, and comprehensive secondary endpoints including quality of life and functional assessments.

• Fast-track and orphan drug designations from FDA and EMA provide regulatory advantages and closer authority interaction.

• Expanded access program and phase IIa data show promising safety, tolerability, and early efficacy signals, including improvements in right heart function and quality of life.

• A world-class steering committee and CRO are engaged to ensure high-quality execution and global reach.

Market opportunity and value proposition

• The global PH market is projected to reach $13 billion by 2031, with CS1 expected to capture a significant share due to its oral, once-daily, well-tolerated profile and potential for early use in the treatment algorithm.

• Disease modification and remission are positioned as the key differentiators, with the potential to expand into additional indications and create a broader franchise.

• Analyst and management estimates for CS1 and CS014 suggest multi-billion dollar peak sales, with additional upside from platform expansion into other rare and thrombotic disorders.

• The company has experienced strong analyst valuation growth, though retail market valuation lags, creating a potential value gap for investors.

• Strategic focus for the year includes executing clinical milestones, securing regulatory approvals, expanding the investor base, and closing a major pharma partnership.