NEXT GENERATION RNA MEDICINES January 2026 JP Morgan Healthcare Conference Forward Looking Statements This presentation contains forward-looking statements. These statements relate to future events and involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future performances or achievements expressed or implied by the forward-looking statements. Each of these statements is based only on current inf ...

Company Overview - Arcturus Therapeutics (ARCT) shares increased by 7.7% to $23.16 in the last trading session, with a higher-than-average trading volume [1] - The stock has gained 2.3% over the past four weeks [1] Pipeline and Development - The positive investor sentiment is linked to ARCT's pipeline, which includes two mRNA therapeutic candidates: ARCT-810 for ornithine transcarbamylase deficiency and ARCT-032 for cystic fibrosis, both in mid-stage development [2] - Arcturus has partnered with CSL Seqirus to develop Kostaive, the world's first sa-mRNA COVID-19 vaccine, which is approved in Japan and the EU [2] Financial Expectations - The company is expected to report a quarterly loss of $1.09 per share, reflecting a year-over-year decline of 319.2% [3] - Projected revenues for the upcoming quarter are $16.67 million, down 60% from the same quarter last year [3] Earnings Estimates and Stock Performance - The consensus EPS estimate for Arcturus has remained unchanged over the last 30 days, indicating a lack of upward revisions [4] - The stock's price typically does not continue to rise without trends in earnings estimate revisions, suggesting the need for monitoring future performance [4] - Arcturus currently holds a Zacks Rank of 1 (Strong Buy) [4] Industry Comparison - In the same industry, Septerna, Inc. (SEPN) saw a 5% decrease in its stock price, closing at $21.31, but has returned 40.3% over the past month [4] - Septerna's consensus EPS estimate has remained unchanged at $0.19, representing a 102.3% increase from the previous year, and it holds a Zacks Rank of 3 (Hold) [5]

Core Insights - Protara Therapeutics is a clinical-stage biotech company focused on developing treatments for cancer and rare diseases, with its lead program TARA-002 targeting non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [2][8] - TARA-002 utilizes a novel approach derived from a genetically distinct cell bank, functioning through dual mechanisms of directly killing tumor cells and stimulating an immune response [1][8] - The company is also advancing IV Choline Chloride for patients requiring parenteral nutrition, addressing a significant unmet need [10] TARA-002 Development - TARA-002 is currently being evaluated in a mid-stage study called ADVANCED-2 for NMIBC, showing promising early results with a 67% complete remission rate in BCG-unresponsive patients at six months [8][12] - In the Phase 2 STARBORN-1 trial for lymphatic malformations, two out of three pediatric patients achieved complete response after one injection, indicating a favorable safety profile [9][12] - Protara plans to deliver interim results for both trials in 1Q26 and 4Q25, respectively [8][9] Market Potential and Analyst Sentiment - Analysts project TARA-002 represents a potential market opportunity exceeding $500 million, with a strong buy consensus rating and a price target suggesting an 840% upside [12][13] - The average price target from analysts for TARA shares is $24.20, indicating a potential upside of approximately 659% [13] Financial Performance of Alpha Teknova - Alpha Teknova reported $10.3 million in revenue for 2Q25, a 7.3% year-over-year increase, with a reaffirmed full-year revenue guidance of $39-42 million [17] - The company is positioned to benefit from growth in the bioprocessing market, with expectations of over 20% revenue growth and 30%+ adjusted EBITDA margins in the long term [19][20] Analyst Ratings for Alpha Teknova - Alpha Teknova holds a strong buy consensus rating, with a price target of $12 indicating a potential one-year upside of approximately 159% [20]

Company Overview - Arcturus Therapeutics (ARCT) shares increased by 8.7% to close at $19.62, with trading volume significantly higher than usual, contrasting with a 4% gain over the past four weeks [1][2] Pipeline and Product Development - The recent stock uptick is driven by positive investor sentiment regarding ARCT's pipeline, which includes two mRNA therapeutic candidates: ARCT-810 for ornithine transcarbamylase deficiency and ARCT-032 for cystic fibrosis, both in mid-stage development [2] - Arcturus has partnered with CSL Seqirus to develop Kostaive, the world's first sa-mRNA COVID-19 vaccine, which is currently approved in Japan and the EU [2] Financial Performance Expectations - The company is expected to report a quarterly loss of $1.09 per share, reflecting a year-over-year decline of 319.2%, with revenues projected at $16.67 million, down 60% from the previous year [3] - The consensus EPS estimate for the upcoming quarter has been revised 13% lower over the last 30 days, indicating a negative trend in earnings estimate revisions, which typically does not lead to price appreciation [4] Industry Context - Arcturus Therapeutics is part of the Zacks Medical - Biomedical and Genetics industry, where Anika Therapeutics (ANIK) also operates, having closed 3.2% higher at $8.94, with a 3.5% return over the past month [4] - Anika's consensus EPS estimate for its upcoming report remains unchanged at $0.02, representing a year-over-year increase of 108%, and it holds a Zacks Rank of 2 (Buy) [5]

Summary of Arcturus Therapeutics KOL Presentation on ARCT-810 Phase II Interim Data for OTC Deficiency Company Overview - **Company**: Arcturus Therapeutics - **Headquarters**: San Diego - **Focus**: mRNA medicines, specifically targeting rare liver diseases like ornithine transcarbamylase (OTC) deficiency [5][6] Industry Context - **Industry**: Biotechnology, specifically in the development of mRNA therapeutics for rare diseases - **Condition**: OTC deficiency is the most common urea cycle disorder with significant unmet medical needs [6][7] Key Points from the Presentation 1. **ARCT-810 Overview**: - ARCT-810 is an mRNA therapeutic designed to replace dysfunctional OTC enzymes, improving urea cycle activity, detoxifying ammonia, and potentially eliminating the need for liver transplants [6][7][8] - It utilizes Arcturus' proprietary lunar delivery platform for effective delivery to hepatocytes [7] 2. **Regulatory Designations**: - ARCT-810 has received multiple designations: orphan drug designation, orphan medicinal product designation, fast track designation, and rare pediatric disease designation [8] 3. **Phase II Study Design**: - Two Phase II studies were conducted: one in the US and one in Europe, focusing on safety, tolerability, and biomarker assessments [11][12] - The US study enrolled patients with more severe disease, while the European study included patients with stable disease [12] 4. **Biomarker Results**: - **Plasma Glutamine**: - In the European study, mean glutamine levels decreased from high to normal during treatment and began to rise again after four weeks post-treatment [13][14] - In the US study, glutamine levels normalized after three doses and remained normal for approximately twenty days [14] - **Ureagenesis Function**: - The new N15 assay showed significant increases in relative ureagenesis function (RUF) post-treatment, with a mean increase of 14.7% [16][17] - Two subjects achieved RUF levels above 50%, indicating clinically meaningful improvements [17] - **Ammonia Levels**: - Ammonia levels remained stable and within normal ranges after treatment, supporting the favorable glutamine and ureagenesis data [18] 5. **Safety Profile**: - The safety database included 40 participants, indicating that ARCT-810 was generally safe and well-tolerated [19] - No serious infusion-related reactions were reported, and adverse events were manageable [19][20] 6. **Next Steps**: - Arcturus plans to complete the ongoing Phase II US study and engage with regulatory agencies for a multi-biomarker driven pivotal trial [68][69] Additional Insights - **Clinical Implications**: - The KOLs emphasized the importance of normalizing diet and reducing the need for ammonia scavengers as key success metrics for OTC therapies [78][80] - mRNA therapies are viewed as a potential alternative to liver transplants, especially for severe cases [100][101] - **Comparison with Other Therapies**: - ARCT-810 is positioned as a more effective solution compared to existing ammonia scavengers, which do not restore urea cycle function [87][88] Conclusion - The interim data for ARCT-810 demonstrates promising results in reducing glutamine levels and improving urea cycle function, with a favorable safety profile. The company is poised to advance its clinical development and regulatory strategy to address the significant unmet needs in OTC deficiency treatment [67][68]

Core Insights - CytomX Therapeutics, in collaboration with Moderna, has announced promising preclinical data for an mRNA-encoded masked IL-12 therapeutic, which demonstrates significant anti-tumor activity and enhanced tolerability [1][2] Company Overview - CytomX Therapeutics is a clinical-stage biopharmaceutical company focused on developing conditionally activated, masked biologics aimed at localized treatment within the tumor microenvironment [3] - The company utilizes its proprietary PROBODY therapeutic platform to create safer and more effective cancer therapies [3] - CytomX's pipeline includes various therapeutic candidates such as CX-2051, a masked ADC targeting EpCAM, and CX-801, a masked interferon alpha-2b PROBODY cytokine [3] Collaboration and Technology - The collaboration with Moderna combines CytomX's PROBODY masking technology with Moderna's mRNA technology to create a selectively activated IL-12 therapeutic [2] - The new mRNA therapeutic is designed to minimize systemic activity while maximizing anti-tumor efficacy within the tumor microenvironment [2] Presentation Details - The preclinical data will be presented at the American Association for Cancer Research (AACR) Annual Meeting on April 28, 2025, under the title "An mRNA-encoded masked IL-12 improves systemic tolerability while maintaining anti-tumor efficacy in preclinical studies" [3]

TABLE OF CONTENTS As filed with the U.S. Securities and Exchange Commission on May 19, 2023. Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 UNDER THE SECURITIES ACT OF 1933 TRANSCODE THERAPEUTICS, INC. (Exact name of Registrant as specified in its charter) Delaware (State or other jurisdiction of incorporation or organization) 2834 (Primary Standard Industrial Classification Code Number) FORM S-1 REGISTRATION STATEMENT 81-1065054 (I.R.S. Employer Identification ...