SCIENTIFIC WEBINAR 2025 PC111 - A New Drug Candidate for Rare Skin Diseases: Pemphigus and SJS/TEN Pincell Acquisition Option 1 Agenda Ø Overview of Pemphigus and SJS/TEN Symptoms, implications and patho-mechanisms Ø Overview of Current Treatment Landscape Ø PC111 Deep Dive: -- Mechanism of Action -- Achievements to Date -- Comparison to Current SOC -- Safety Profile -- Combination with Existing Therapies (Risks/Benefits) -- Development Plan -- Challenges in Conducting Rare Disease Clinical Trials Ø Commerc ...

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.

Summary of Scinai Immunotherapeutics (SCNI) Conference Call Company and Industry Overview - **Company**: Scinai Immunotherapeutics (SCNI) - **Industry**: Biotechnology, specifically focusing on treatments for rare autoimmune diseases such as pemphigus and Stevens Johnson syndrome (SJS) Key Points and Arguments 1. **Acquisition Announcement**: Scinai has entered into a binding option agreement to acquire the Italian biotech company, Pincell, and submitted a grant application for €12 million to develop PC-one hundred eleven [1][2] 2. **Overview of Diseases**: The call discussed pemphigus, SJS, and toxic epidermal necrolysis (TEN), highlighting their symptoms, implications, and current treatment landscape [8][9] 3. **Medical Need**: There is a significant unmet medical need for effective treatments for pemphigus and SJS, with pemphigus affecting approximately 2 million people globally and SJS being a rare but severe condition [14][19] 4. **Current Treatments**: Current therapies for pemphigus include glucocorticoids and rituximab, which have severe side effects and a high relapse rate. There are no approved therapies for SJS [21][22][44] 5. **PC-one hundred eleven Mechanism**: PC-one hundred eleven is a fully human monoclonal antibody targeting soluble Fas ligand, which plays a critical role in the pathogenesis of pemphigus and SJS. It blocks keratinocyte apoptosis and acantholysis, potentially preventing blister formation [26][27][43] 6. **Efficacy Comparison**: PC-one hundred eleven is expected to have a rapid onset of action and a better safety profile compared to current treatments like rituximab, which has a medium time to remission of around six months [41][43] 7. **Development Plans**: Scinai plans to conduct parallel development for PC-one hundred eleven, focusing on chronic pemphigus and acute SJS, with an expected approval timeline of three to four years [52][53] 8. **Market Size**: The addressable patient population for pemphigus in target countries is around 164,000, with a focus on relapsing refractory patients. For SJS, the addressable population is estimated at 15,000 [56][58] 9. **Pricing and Revenue Potential**: Potential peak revenues for PC-one hundred eleven are estimated at around $500 million, with an upside of over $1 billion for both pemphigus and SJS indications [63][64] 10. **Partnership Opportunities**: There is significant interest from pharmaceutical companies in smaller indications with transformational efficacy, which could lead to partnerships for PC-one hundred eleven [66][67] Additional Important Content - **Challenges in Clinical Trials**: The company plans to address challenges in conducting clinical trials for rare diseases by collaborating with scientific societies and patient advocacy groups to identify suitable trial sites [55] - **Safety Considerations**: The non-immunosuppressive nature of PC-one hundred eleven is expected to reduce the risk of infections and complications associated with current treatments, making it a safer option for patients [70] - **Combination Therapy Potential**: PC-one hundred eleven could be used in combination with existing therapies like rituximab for pemphigus and could enhance supportive care for SJS patients [49][51] This summary encapsulates the critical insights and developments discussed during the conference call, emphasizing the potential of PC-one hundred eleven in addressing significant medical needs in the treatment of rare autoimmune diseases.

Financial Performance - As of December 31, 2024, the company had cash and cash equivalents of $1.9 million, down from $4.9 million as of December 31, 2023[41]. - The company reported an operating loss of $8.6 million for the twelve months ended December 31, 2024, compared to a loss of $9.7 million for the same period in 2023[35]. - The accumulated deficit as of December 31, 2024, was $117.6 million, slightly improved from $122.5 million as of December 31, 2023[40]. - The current cash resources are insufficient to fund projected cash requirements for at least the next 12 months[41]. - The company has implemented a cost-saving plan, including layoffs and postponing capital expenditures[36]. - The company has not paid any cash dividends on the ADSs since inception and does not anticipate doing so in the foreseeable future[209]. Business Strategy and Development - The company has suspended further development of its COVID-19 NanoAb program due to decreased market interest and funding[47]. - The focus has shifted to developing NanoAbs targeting Interleukin-17 (IL-17) for conditions like plaque psoriasis and psoriatic arthritis, with a licensing agreement established in June 2023[47]. - The company is exploring an acquisition of the Italian biotech company Pincell srl, which owns a fully human monoclonal antibody[47]. - The option agreement for acquiring Pincell includes a requirement to secure $3 million by December 31, 2025, to fund the development of PC111, a monoclonal antibody[55]. - The company plans to commence a pre-clinical toxicology study for a novel VHH antibody for psoriasis in 2026[180]. - The company has established a CDMO business unit to leverage its laboratory and manufacturing capacity at its cGMP facility in Jerusalem[227]. Regulatory and Compliance Challenges - The company is focusing on the development of IL-17 NanoAb for treating psoriasis and psoriatic arthritis, facing significant regulatory challenges due to the novelty of the technology[51]. - The company may face delays in regulatory approvals due to the extensive requirements set by the FDA and other authorities, which can significantly impact commercialization timelines[59]. - Regulatory authorities may impose ongoing requirements even after approval, which could lead to loss of approvals if not complied with[62]. - The company must navigate complex regulatory environments, which may lead to increased costs and delays in product development and approval[60]. - The company is subject to extensive government regulation, which increases the cost and risk of developing and selling products[96]. - The company must comply with various federal and state healthcare laws, with non-compliance potentially resulting in significant penalties and operational restrictions[100]. Market and Competitive Landscape - The company faces competition from other products targeting the same conditions, including biosimilars, which could affect market share[64]. - The company has limited marketing capabilities and may struggle to develop its own sales force, impacting the commercialization of its product candidates[50]. - Market acceptance and sales of product candidates depend heavily on coverage and reimbursement policies from government authorities and third-party payors[89]. - In the U.S., reimbursement for pharmaceutical products varies significantly among third-party payors, impacting sales potential[90]. - The company faces significant competition from fully integrated pharmaceutical companies and smaller biotech firms, which may have greater financial resources and experience[120]. Clinical Trials and Development Risks - The company acknowledges the high rate of attrition in clinical trials, indicating that success in early trials does not guarantee later success[59]. - Participant enrollment in clinical trials is critical, and delays could significantly increase development costs and hinder timely regulatory approvals[80]. - The company may face challenges in achieving primary/secondary endpoints in clinical trials, which could necessitate additional studies and further delay commercialization[68]. - If clinical trials produce negative results, the company may be required to conduct additional trials, impacting the ability to obtain regulatory approval[77]. - The FDA may not accept data from clinical trials conducted outside the U.S., potentially leading to costly and time-consuming additional trials[74]. Operational and Financial Risks - The ability to continue operations is highly dependent on obtaining additional financing, which may not be available under favorable terms[41]. - The company faces challenges in identifying potential product candidates due to research methodology limitations[88]. - The company may struggle to find partners for the development of its COVID-19 NanoAb program, which is currently suspended due to low interest and funding[78]. - The company is exposed to potential product liability risks, which could result in substantial liabilities and affect its ability to market current and future product candidates[126]. - The company may face challenges in obtaining adequate insurance coverage, which could adversely affect its financial condition and ability to recruit qualified personnel[130]. Intellectual Property and Legal Risks - The company may face challenges in protecting its intellectual property rights, which could diminish its competitive position and future revenues[151]. - The company may need to engage in costly litigation to protect its intellectual property rights, which could divert management resources and attention[163]. - There is a risk that the company may be unable to obtain exclusive licenses for co-owned patents, potentially allowing competitors to market similar products[159]. - The company’s reliance on proprietary know-how and confidentiality agreements, but these may not provide adequate protection against unauthorized use or disclosure[161]. - The company may face significant expenses and liabilities from claims alleging violation of third-party intellectual property rights, which could harm its business[165]. External Factors and Market Conditions - The emergence of new COVID-19 variants poses ongoing risks to the company's operations and financial stability, potentially disrupting supply chains and access to capital[105]. - The ongoing conflict may disrupt the company's operations and supply chain, impacting business continuity[186]. - Moody's downgraded Israel's credit rating from A1 to A2, with a negative outlook, which may affect the company's ability to raise capital[185]. - The political and security situation in Israel may lead to delays in business agreements and affect supply chain logistics[178]. - The company is exposed to currency fluctuation risks due to operational expenses incurred in currencies other than the U.S. Dollar[198]. Nasdaq Compliance and Shareholder Issues - Failure to meet Nasdaq's continued listing requirements could result in delisting, adversely affecting market liquidity and share price[199]. - On November 1, 2023, the company received a notice of non-compliance from Nasdaq regarding the minimum bid price requirement of $1.00 per share, with a 180-day period to regain compliance[200]. - The company appealed the delisting determination and approved a reverse split of 1 for 10, increasing the number of Ordinary Shares represented by each ADS from 400 to 4,000, which allowed it to regain compliance with the Minimum Price Rule[200]. - On November 20, 2023, the company announced it had regained compliance with the Minimum Stockholders' Equity Rule after receiving formal notification from Nasdaq[201]. - A delisting from Nasdaq would likely negatively impact the price of the ADSs and impair shareholders' ability to trade their securities[205].

Core Viewpoint - Scinai Immunotherapeutics Ltd. has filed for a Euro 12 million grant to support the development of its monoclonal antibody PC111, which targets severe dermatological conditions, and has entered into an option agreement to acquire the Italian biotech company Pincell srl [1][2][7]. Company Overview - Scinai Immunotherapeutics Ltd. specializes in inflammation and immunology biological products and offers CDMO services through its Scinai Bioservices unit [1][10]. - Pincell srl focuses on developing first-in-class anti-inflammatory therapies for rare and severe skin diseases, founded by experts in dermatology [11]. Product Development - PC111 is a fully human monoclonal antibody that blocks the activation of apoptosis in skin cells, addressing significant unmet medical needs in skin blistering disorders [3][5]. - The antibody has shown potential in preclinical studies to block blister formation in pemphigus without the use of steroids, indicating its promise as a targeted therapy [5][7]. Grant Application - The grant application submitted by Scinai's Polish subsidiary is part of the European Funds for a Modern Economy (FENG) program, with a decision expected by mid-July to early August [2]. - The funding will primarily be non-dilutive, requiring only Euro 3 million from Scinai's capital, which is one-fifth of the total budget [8]. Market Need - Pemphigus, Stevens-Johnson Syndrome (SJS), and Toxic Epidermal Necrolysis (TEN) are severe conditions with high mortality rates (5-10% for pemphigus and SJS, 30-40% for TEN), highlighting the urgent need for effective treatments [6][7]. Strategic Partnership - The acquisition of Pincell is expected to enhance Scinai's capabilities in developing PC111, leveraging Pincell's expertise in dermatology and research [8][9]. - The management team from Pincell will integrate into Scinai's operations, further strengthening the collaboration [9].

Core Insights - Scinai Bioservices, a CDMO unit launched by Scinai in 2024, operates from a facility in Jerusalem, Israel, featuring 1,850 square meters of clean rooms and laboratories compliant with cGMP standards [2][3] - The CDMO unit has engaged in drug development projects for various biotech companies, including upstream and downstream process development, scale-up, and drug manufacturing for clinical trials [3] - Scinai's U.S. subsidiary, Scinai Bioservices Inc., aims to support early-stage biotech companies in the U.S. by providing essential CDMO services that are currently in short supply [4] Company Overview - Scinai Immunotherapeutics Ltd. (NASDAQ: SCNI) operates two business units: one focused on developing biological therapeutic products for inflammation and immunology, and the other providing CDMO services to early-stage biotech companies [5] - The company is actively seeking partnerships with potential pharma partners in the fields of inflammation and immunology, as well as institutional and private investors interested in its value proposition [7]

Core Viewpoint - Scinai Immunotherapeutics Ltd. has entered into a Standby Equity Purchase Agreement (SEPA) with Yorkville Advisors, allowing the company to sell up to $10 million of its American Depository Shares (ADSs) over a three-year period to support its R&D and CDMO business expansion [1][2][5]. Group 1: SEPA Details - The SEPA allows Scinai to sell ADSs at a 3% discount to the lowest daily volume-weighted average price over three consecutive trading days following an advance notice [3]. - Scinai retains full control over the timing and amount of sales under the SEPA, with no obligation to utilize the full $10 million [4]. - The agreement includes a beneficial ownership cap of 9.99% of Scinai's share capital at any one time, along with other restrictions [2]. Group 2: Use of Proceeds - Proceeds from the potential offering of ADSs under the SEPA will be used to further develop NanoAbs programs, support the CDMO business, and for general corporate purposes including working capital and regulatory matters [5]. Group 3: Company Overview - Scinai Immunotherapeutics is focused on developing inflammation and immunology biological products and offers CDMO services through its Scinai Bioservices unit [1][7]. - The company has a pipeline of nanosized VHH antibodies (NanoAbs) targeting diseases with significant unmet medical needs [7].

Company Overview - Scinai Immunotherapeutics Ltd. is a biopharmaceutical company listed on Nasdaq under the ticker SCNI, focusing on inflammation and immunology (I&I) biological therapeutic products and providing CDMO services for early-stage biotech drug development [1][2] Business Units - The company operates two complementary business units: one dedicated to the in-house development of innovative nanosized VHH antibodies (nanoAbs) targeting diseases with significant unmet medical needs, and the other offering a boutique CDMO service that includes biological drug development, analytical methods development, clinical cGMP manufacturing, and trial design and execution [1] Recent Developments - Scinai has released a year-end video showcasing its key accomplishments and achievements for 2024, highlighting its progress and future plans [1] Target Audience - The company is engaging with institutional and private investors, potential pharmaceutical partners in the inflammation and immunology field, and prospective clients for its comprehensive biologics CDMO services [3]

Core Viewpoint - Scinai Immunotherapeutics Ltd. has established a U.S.-based subsidiary, Scinai Bioservices Inc., to enhance its Contract Development and Manufacturing Services (CDMO) offerings, addressing the growing demand for these services in the U.S. biotech sector [1][6]. Company Overview - Scinai Immunotherapeutics is a biopharmaceutical company focused on developing inflammation and immunology biological products and providing CDMO services through its Scinai Bioservices business unit [1][8]. - The company launched its CDMO business unit in 2024, operating from a facility in Jerusalem, Israel, which includes 1,850 square meters of clean rooms and laboratories compliant with cGMP conditions [2][8]. CDMO Business Development - The CDMO unit has executed drug development projects for nine biotech companies, covering various aspects such as process development, scale-up, and drug manufacturing for pre-clinical and clinical supplies [3][8]. - Scinai has partnered with Ayana Pharma to provide specialized liposomal encapsulated drug development services [3]. Market Demand and Trends - There is a growing demand for CDMO services for biological early-stage drugs in the U.S., driven by improved molecular biological understanding, accelerated market approvals, and an increasing number of novel drug candidates [4]. - The complexity of biologics manufacturing is leading to a heightened demand for high-quality development and manufacturing capacities that meet stringent regulatory standards [4]. Challenges in the Industry - Despite the growing demand for CDMO services, there are concerns regarding the capacity to meet this demand, particularly for early-stage projects due to complex manufacturing routes associated with biologics [5]. - The BIOSECURE Act, introduced in January 2024, may impact U.S. biopharmaceutical drugs in development by companies partnered with Chinese CDMOs, potentially affecting over 120 drugs, with many in clinical-stage development [5]. Strategic Initiatives - The new U.S. subsidiary aims to serve early-stage biotech companies by providing much-needed CDMO services, allowing these companies to utilize U.S. government grant funding without concerns related to the BIOSECURE Act [6]. - Scinai's CDMO business unit is focused on rapid growth, acquiring new clients, and enhancing brand awareness, with expectations of significant revenue increases in the coming years [7][8]. Leadership Commentary - The CEO of Scinai expressed enthusiasm about the new U.S. subsidiary, highlighting the signing of the first contract with Serpin Pharma for clinical manufacturing, which is expected to enhance the prospects of the CDMO unit [8].

Company Overview - Scinai Immunotherapeutics Ltd. is a biopharmaceutical company listed on Nasdaq (SCNI) with two main business units: one focused on the in-house development of inflammation and immunology (I&I) biological therapeutic products, and the other serving as a boutique CDMO for biological drug development and clinical trial services [4]. Key Appointments - Prof. Michael Schön has joined Scinai's Scientific Advisory Board, bringing extensive expertise in dermatology, particularly in chronic inflammatory skin diseases such as psoriasis and atopic dermatitis. His background includes over 440 scientific publications and significant experience in drug development [3]. - Dr. Jonathan Sadeh has been appointed as Chief Scientific Officer and Chief Medical Officer of Bausch Health, marking a notable change within Scinai's advisory team [3]. Innovation and Pipeline - Scinai is developing a pipeline of innovative nanosized VHH antibodies (nanoAbs) aimed at addressing diseases with significant unmet medical needs. The company emphasizes the potential of its targeted immunological therapeutic approaches in precision inflammation medicine [3][4].