Cereno Scientific (CRNO) CMD 2024 summary

Strategic focus and portfolio evolution

• Emphasis on rare diseases with high unmet medical need, targeting pulmonary arterial hypertension (PH), idiopathic pulmonary fibrosis (IPF), and rare thrombotic disorders.

• CS1 demonstrated safety and promising efficacy in phase IIa for PH, with 43% of patients improving REVEAL Risk Score, translating to a 23% reduction in 12-month mortality risk.

• CS014, a novel HDAC inhibitor, is now focused on IPF, a fatal lung disease with limited treatment options; phase I is ongoing, with phase II planned.

• CS585 targets thrombosis without increased bleeding risk, with preclinical data supporting its potential in rare diseases like APS; clinical entry expected by 2025.

• Orphan drug strategy offers regulatory and commercial advantages, including exclusivity and favorable pricing.

Clinical and preclinical results

• CS1 phase IIa trial met safety and tolerability endpoints, with no serious drug-related adverse events and positive exploratory efficacy signals in REVEAL Risk Score, functional class, and CardioMEMS data.

• 70% of patients showed stabilization or improvement in REVEAL Risk Score; 86% were stable or improved in functional class; CardioMEMS indicated reduced mean pulmonary artery pressure.

• Preclinical and clinical data support CS1 and CS014's ability to reverse vascular remodeling and fibrosis, with rapid effects observed in animal models and select patients.

• CS585 demonstrated high selectivity, long-lasting antithrombotic effects, and potential to address microvascular thrombosis in APS, with no increased bleeding risk in preclinical models.

Business development and future plans

• Multiple collaborations with leading academic and industry partners, including FLUIDDA for advanced imaging and Abbott for device integration.

• Expanded access program for CS1 is ongoing, with strong patient and investigator interest.

• Regulatory engagement with FDA and EMA planned for next-stage trials, including phase IIb/III for CS1 and phase II for CS014.

• Commercial focus on rare diseases leverages orphan drug incentives and positions the portfolio for potential partnerships or acquisitions.

• Three clinical assets expected in the clinic within the next year, with pivotal trials and further expansion anticipated by 2026.