Scinai Immunotherapeutics (SCNI) Update / Briefing Transcript

Summary of Scinai Immunotherapeutics (SCNI) Update / Briefing May 07, 2025 Company Overview - Company: Scinai Immunotherapeutics (SCNI) - Focus: Development of PC-one hundred eleven, a fully human monoclonal antibody targeting soluble Fas ligand for the treatment of pemphigus and Stevens Johnson syndrome (SJS) / toxic epidermal necrolysis (TEN) [2][3][27] Industry Context - Diseases Discussed: Pemphigus, Stevens Johnson syndrome, and toxic epidermal necrolysis - Medical Need: Significant unmet medical need for effective treatments in these rare but severe autoimmune diseases [22][25] Key Points and Arguments Disease Overview - Pemphigus: A rare autoimmune disease characterized by painful blisters and erosions on skin and mucous membranes, with a mortality rate of 5% to 15% if untreated [14][15] - SJS and TEN: Rare diseases with high mortality rates, often triggered by medications, leading to severe skin and mucous membrane reactions [20][21] Current Treatment Landscape - Pemphigus Treatments: Current therapies include systemic glucocorticoids and immunosuppressants like rituximab, which have significant side effects and risks [22][23] - SJS/TEN Treatments: No approved therapies exist; treatment is primarily supportive care and withdrawal of the triggering medication [24][25] PC-one Hundred Eleven (PC-111) - Mechanism of Action: Targets soluble Fas ligand, blocking keratinocyte apoptosis and preventing blister formation [27][28] - Efficacy: Expected to provide a rapid onset of action and better safety profile compared to current treatments [44][45] - Development Plans: Two parallel development plans for chronic pemphigus (subcutaneous) and acute SJS/TEN (intravenous) [53][54] Market Considerations - Market Size: Estimated addressable patient population for pemphigus is around 30,000 in target countries, with a potential peak revenue of approximately $500 million to over$1 billion [57][64] - Positioning: PC-111 is expected to be positioned as a first-line therapy for pemphigus and as a rapid intervention for SJS/TEN [62][63] Safety and Efficacy - Non-Immunosuppressive Profile: PC-111's unique mechanism is expected to reduce the risk of infections and complications associated with immunosuppressive therapies [71] - Combination Therapy Potential: Can be used in combination with existing therapies like rituximab to enhance therapeutic effects [50][51] Development Challenges - Clinical Trials: Identifying sites with adequate patient availability is crucial for conducting trials in rare diseases [56] Collaboration Opportunities - Pharma Partnerships: Increased interest from pharmaceutical companies in smaller indications with significant effect sizes, presenting opportunities for collaboration [66][68] Additional Important Insights - Regulatory Advantages: Smaller studies with high effect sizes may receive favorable regulatory attention, facilitating faster approvals [68][70] - Long-term Efficacy: Preliminary data suggests low immunogenicity for PC-111, indicating sustained efficacy without the need for increased dosages [48][49] This summary encapsulates the critical insights from the conference, highlighting the potential of PC-one hundred eleven in addressing significant medical needs in the treatment of pemphigus and SJS/TEN.