Summary of Arcturus Therapeutics FY Conference Call Company Overview - **Company**: Arcturus Therapeutics (NasdaqGM:ARCT) - **Industry**: Biotechnology, specializing in messenger RNA (mRNA) medicines and therapeutics - **Founded**: 2013 - **Location**: San Diego - **Employee Count**: Approximately 100 - **Key Products**: COVID vaccine Kostaive, H5N1 pandemic flu funding from BARDA [2][3] Core Products and Pipeline Kostaive COVID Vaccine - Approved in over 30 countries, including Japan, the UK, and the EU [2] - Partnered with CSL for distribution in Japan and BARDA for pandemic efforts in the U.S. [3] mRNA Therapeutic Candidates 1. **Ornithine Transcarbamylase (OTC) Deficiency** - Flagship liver program with a focus on replacing the missing OTC enzyme [19][20] - Phase 1 and 1B studies completed, transitioning to Phase 2 trials [20][21] - Received orphan drug designation and fast track designation from the FDA [21] 2. **Cystic Fibrosis (CF)** - Inhaled mRNA therapeutic candidate targeting Class I CF patients [9][10] - Phase 2 study ongoing with a focus on safety, tolerability, and preliminary efficacy [11][12] - CF Foundation has committed approximately $25 million to this asset [7] Key Milestones and Data Cystic Fibrosis Program - **Phase 2 Study**: - Initiating a fourth cohort study with 20 CF participants planned for the first half of 2026 [11][12] - Significant mucus plug reduction observed in the second cohort, with reductions of 28%-33% in volume [14] - High-resolution CT scans used to evaluate lung structure changes, which are predictors of lung function improvements [13] OTC Deficiency Program - **Phase 2 Trials**: - Focus on safety, tolerability, and biomarker responses (glutamine, ammonia, ureogenesis) [21][22] - Significant reduction in glutamine levels observed, indicating drug efficacy [22][23] - Ammonia levels maintained within normal range, supporting the drug's safety profile [23] Regulatory and Market Considerations - **Regulatory Clarity**: - Need for alignment with FDA on pivotal trial strategies for both pediatric and adult populations for OTC deficiency [24] - Importance of demonstrating clinical activity and safety to facilitate potential product approval [25] Financial Position - **Cash Runway**: Extended into 2028, providing flexibility to pursue clinical milestones and continue innovation on the LUNAR platform [34] - **Operational Decisions**: Restructuring and expense reduction have strengthened the balance sheet, allowing focus on key value-creating milestones [34][35] Conclusion - Arcturus Therapeutics is positioned to leverage its innovative mRNA delivery technology and strong financial position to advance its clinical programs in cystic fibrosis and OTC deficiency, with significant unmet medical needs in both areas. The upcoming regulatory discussions and clinical data will be critical for the company's future growth and market opportunities [24][35]

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 ...

Core Viewpoint - Arcturus Therapeutics is positioned as a high-potential biotech stock under $10, with expectations of a 404% increase by 2026 due to promising clinical data and a clear development pipeline [1]. Company Overview - Arcturus Therapeutics (ARCT) focuses on developing mRNA-based medicines and vaccines, aiming to address root causes of diseases rather than just symptoms [2]. - The company is currently valued at $194.3 million and experienced a 64% decline in stock value last year, contrasting with a 16% gain in the S&P 500 Index, although the stock has risen by 10% this year [2]. Product Development and Pipeline - Arcturus has developed the world's first approved self-amplifying mRNA Covid-19 vaccine, KOSTAIVE, and is collaborating on mRNA vaccines for Covid-19 variants and pandemic influenza [3]. - The company is advancing ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis (CF), with interim Phase 2 data showing safety and reductions in mucus burden in patients [4]. - A 12-week safety and preliminary efficacy study for ARCT-032 is planned for the first half of 2026, with a higher dose cohort being enrolled to refine the dose-response profile [5]. Additional Therapeutic Developments - Arcturus is also developing ARCT-810 for ornithine transcarbamylase (OTC) deficiency, with plans to collaborate with regulatory agencies on trial designs in 2026 [6]. - The company's mRNA platform is validated through its vaccine programs, with significant immune responses and safety profiles observed in ongoing trials for Covid-19 and pandemic influenza candidates [6].

Core Viewpoint - Arcturus Therapeutics Holdings Inc. is set to present at the 44th Annual J.P. Morgan Healthcare Conference, highlighting its focus on messenger RNA medicines for rare diseases and infectious disease vaccines [1] Company Overview - Arcturus Therapeutics is a commercial company specializing in messenger RNA medicines [1] - The company is focused on developing therapeutics for liver and respiratory rare diseases, as well as vaccines for infectious diseases [1] Event Details - Joseph Payne, the President & CEO of Arcturus, will present at the conference on January 12, 2026, at 5:15 p.m. Pacific Time [1]

Group 1: Ark Invest's Bitcoin Strategy - Ark Invest has increased its investment in the ARK 21Shares Bitcoin ETF (BATS:ARKB) despite Bitcoin's price volatility and regulatory changes [1][2] - The firm purchased a total of 55,000 shares of ARKB, valued at $1.7 million, through its ARK Next Generation Internet ETF (BATS:ARKW) and ARK Blockchain & Fintech Innovation ETF (BATS:ARKF) [2] - Bitcoin's price is currently trading at $92,269.08, reflecting a 2.49% increase over 24 hours, as market expectations for a Federal Reserve rate cut grow [5] Group 2: Market Dynamics and Regulatory Environment - Recent market analysis indicates that aggressive high-frequency trading strategies have contributed to Bitcoin's price fluctuations, particularly during U.S. trading sessions [3] - The Commodity Futures Trading Commission's new framework allows approved intermediaries to accept major tokens and stablecoins as margin, enhancing Bitcoin's integration into traditional finance [4] - Crypto prediction markets suggest that Bitcoin has roughly even odds of surpassing $100,000 by the end of 2025, influenced by regulatory support and macroeconomic factors [5] Group 3: Other Notable Trades by Ark Invest - Ark Invest has made several adjustments across its sector-focused funds, including reducing its position in Ibotta (IBTA) and satellite operator Iridium Communications Inc. (IRDM) [6][8] - The firm sold 90,807 shares of Adaptive Biotechnologies Corp (ADPT) while increasing its holdings in Arcturus Therapeutics Holdings Inc. (ARCT) and GeneDx Holdings Corp (WGS) [8] - Ark also reduced its semiconductor testing exposure by selling shares of Teradyne Inc. (TER), aligning with its strategy to shift capital towards higher-growth innovation themes [8]

Core Insights - Arcturus Therapeutics (ARCT) shares have dropped 59.4% over the past three months due to mixed interim results from a mid-stage study of its inhaled mRNA therapy, ARCT-032, for cystic fibrosis (CF) patients [2][6] Study Results - ARCT-032 utilizes a proprietary LUNAR lipid-mediated aerosolized platform to deliver CFTR mRNA to the lungs of CF patients who do not respond to existing treatments [3] - Interim data from the Phase II study showed no significant improvement in Forced Expiratory Volume in 1 second (FEV1) from day 1 to day 28 [4] - A post hoc exploratory analysis indicated modest lung-function gains in four out of six Class-I CF patients, with mean absolute and relative increases of 3.8% and 5.1% in percent predicted FEV1, but these changes are within the expected natural variability [5] - AI-assisted high-resolution computed tomography (HRCT) scans showed reductions in mucus plugs and volume in four of six patients by day 28, suggesting potential efficacy in reducing mucus burden [6] Safety Profile - ARCT-032 demonstrated a favorable safety profile, with treatment-related adverse events similar to those in the Phase I study, and one serious adverse event deemed unrelated to the treatment [8] Future Plans - The third cohort of the Phase II study is currently enrolling up to six patients to assess dose-escalation at 15 mg, with plans for a 12-week study involving up to 20 CF patients in 2026 [9] Market Context - Cystic fibrosis is a serious genetic disease caused by CFTR mutations, leading to severe respiratory complications, and existing CFTR modulators do not benefit all patients, creating a need for alternative treatments [10][11]

Financial Performance - Total revenue for Q3 2025 was $17.151 million, a decrease of 58.8% compared to $41.673 million in Q3 2024[16] - Collaboration revenue for the nine months ended September 30, 2025, was $64.140 million, down 45.4% from $117.389 million in the same period of 2024[16] - Net loss for Q3 2025 was $13.448 million, compared to a net loss of $6.903 million in Q3 2024, representing a 94.0% increase in losses year-over-year[16] - Total collaboration revenue for the three months ended September 30, 2025, was $14.2 million, a decrease of 63.5% compared to $38.8 million for the same period in 2024[35] - Total grant revenue for the nine months ended September 30, 2025, was $10.7 million, compared to $12.2 million for the same period in 2024, reflecting a decrease of 12.3%[35] - For the three months ended September 30, 2025, revenues were $17,151,000, a decrease from $41,673,000 in the same period of 2024, representing a decline of approximately 59%[81] - For the nine months ended September 30, 2025, total revenues were $74,835,000, down from $129,544,000 in 2024, indicating a decrease of about 42%[81] Cash and Assets - Cash and cash equivalents decreased to $180.360 million as of September 30, 2025, down 24.0% from $237.028 million at December 31, 2024[14] - Total assets decreased to $282.343 million as of September 30, 2025, down 17.9% from $344.069 million at December 31, 2024[14] - Total stockholders' equity as of September 30, 2025, was $224.559 million, a decrease of 6.8% from $240.978 million at December 31, 2024[14] - The company’s total current liabilities decreased to $31.699 million as of September 30, 2025, down 51.7% from $65.489 million at December 31, 2024[14] - Remaining available funding under the BARDA Contract as of September 30, 2025, was $30.3 million[48] - As of September 30, 2025, the company's cash and cash equivalents, including restricted cash, totaled $237.2 million[137] Research and Development - Research and development expenses for the nine months ended September 30, 2025, were $87.736 million, a decrease of 42.1% from $151.376 million in the same period of 2024[16] - Total research and development expenses for the three months ended September 30, 2025, were $23,265,000, compared to $39,134,000 in 2024, reflecting a reduction of approximately 40%[81] - Total research and development expenses for the nine months ended September 30, 2025, were $87.7 million, down 42% from $151.4 million in the same period in 2024[126] - Research and development expenses decreased to $87.7 million for the nine months ended September 30, 2025, from $151.4 million in the same period of 2024, primarily due to lower costs associated with the LUNAR-COVID program transitioning to commercial phase[129] Operating Expenses - Operating expenses for the three months ended September 30, 2025, were $33.7 million, down 36% from $52.4 million in the same period in 2024[125] - General and administrative expenses were $10.4 million for the three months ended September 30, 2025, down from $13.3 million in 2024, and $32.1 million for the nine months ended September 30, 2025, compared to $40.4 million in 2024, mainly due to reduced share-based compensation and headcount[134] - Share-based compensation expense for the three months ended September 30, 2025, was $5.7 million, down from $9.5 million for the same period in 2024, a decrease of approximately 40%[67] Collaborations and Partnerships - The Company received a $200 million upfront payment under the CSL Collaboration Agreement and is eligible for over $1.3 billion in development milestones[38] - The Company recognized $4.1 million in revenue related to the CSL Collaboration Agreement during the third quarter of 2025[38] - The company is collaborating with CSL Seqirus on the development of mRNA vaccines for COVID-19, influenza, and other infectious diseases[91] - The company has achieved approximately $513.8 million in total upfront payments and milestones from CSL Seqirus, including $4.1 million in the current quarter[137] Legal and Regulatory Matters - The company is involved in legal proceedings, including a lawsuit for trade secret misappropriation against AbbVie Inc. and Capstan Therapeutics, with a response deadline set for December 1, 2025[176] - A prolonged U.S. federal government shutdown could adversely affect the company's business operations and financial condition[175] - Reports indicate that CSL Limited may separate CSL Seqirus, which could disrupt the company's collaboration and adversely affect its business[177] Internal Controls and Governance - There is a material weakness in internal control over financial reporting as of September 30, 2025, related to information technology general controls[165] - Remediation efforts for the identified material weakness are ongoing, with expectations to complete activities in fiscal year 2025[169] - Management has implemented enhanced internal control procedures to address the material weakness identified[168] Future Outlook - The company anticipates continued losses and will require additional capital to fund operations and support long-term plans[157] - The company expects to continue incurring additional losses in the long term and will need to execute on milestones within the CSL Collaboration Agreement to transition to profitability[151] - Future guidance indicates projected revenue growth of 30% for the next fiscal year, aiming for $65 million[186] Miscellaneous - The company has a revolving credit line of $50 million with Wells Fargo, with no amounts drawn down during the three months ended September 30, 2025[62] - The company has not adopted or terminated any Rule 10b5-1 trading arrangements during the three months ended September 30, 2025[182] - The company has 1,586,213 shares available for future issuance under the 2019 Omnibus Equity Incentive Plan as of September 30, 2025[65] - The company has initiated a merger discussion with a complementary biotech firm to enhance its product pipeline[192]

Core Insights - Arcturus Therapeutics Holdings Inc. reported better-than-expected third-quarter earnings for 2025, showcasing resilience in a challenging market environment [1][6] Financial Performance - The company reported an earnings per share (EPS) of -$0.49, significantly better than the estimated EPS of -$0.80, resulting in a 60.8% earnings surprise compared to the Zacks Consensus Estimate of a loss of $1.25 per share [2][6] - Revenue for the quarter was $17.15 million, exceeding the estimated $16.03 million and surpassing the Zacks Consensus Estimate by 50.63%, although this represents a decline from $41.67 million in the same quarter last year [3][6] Financial Metrics - The company has a negative price-to-earnings (P/E) ratio of approximately -3.96, indicating negative earnings, while the price-to-sales ratio is about 2.03, suggesting investors are willing to pay $2.03 for every dollar of sales [4] - The enterprise value to sales ratio is 0.55, indicating a relatively low valuation compared to its sales, and the debt-to-equity ratio of 0.10 shows a low level of debt [4] - A current ratio of 5.90 indicates strong liquidity, reflecting the company's ability to meet short-term obligations [4] Strategic Engagement - During the Q3 2025 earnings call, key figures from the company discussed financial performance and strategic direction, highlighting initial findings of the ARCT-032 clinical activity presented to the cystic fibrosis community [5]

Financial Performance - Arcturus Therapeutics reported a quarterly loss of $0.49 per share, significantly better than the Zacks Consensus Estimate of a loss of $1.25, representing an earnings surprise of +60.80% [1] - The company posted revenues of $17.15 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 50.63%, although this is a decline from $41.67 million in the same quarter last year [2] - Over the last four quarters, Arcturus has surpassed consensus EPS estimates three times and topped consensus revenue estimates three times as well [2] Stock Performance - Arcturus Therapeutics shares have declined approximately 48.6% since the beginning of the year, contrasting with the S&P 500's gain of 14.4% [3] - The stock currently holds a Zacks Rank 4 (Sell), indicating expectations of underperformance in the near future [6] Future Outlook - The current consensus EPS estimate for the upcoming quarter is -$1.49 on revenues of $9.77 million, and for the current fiscal year, it is -$3.49 on revenues of $73.55 million [7] - The earnings outlook and estimate revisions will be crucial for assessing the stock's future performance [4][5] Industry Context - The Medical - Biomedical and Genetics industry, to which Arcturus belongs, is currently ranked in the top 36% of over 250 Zacks industries, suggesting a favorable industry outlook [8] - The performance of Arcturus may also be influenced by the overall industry trends and the performance of peer companies, such as TScan Therapeutics, which is expected to report a quarterly loss of $0.35 per share [9]

Financial Data and Key Metrics Changes - Revenues for Q3 2025 were $17.2 million, a decrease of $24.5 million compared to Q3 2024, while revenues for the nine months ended September 30, 2025, were $74.8 million, down $54.7 million year-over-year [14] - Total operating expenses for Q3 2025 were $33.7 million, down from $52.4 million in Q3 2024, and for the nine months ended September 30, 2025, total operating expenses were $119.8 million compared to $191.8 million in the prior year [14][15] - The net loss for Q3 2025 was approximately $13.5 million or $0.49 per diluted share, compared to a net loss of $6.9 million or $0.26 per diluted share in Q3 2024 [16] Business Line Data and Key Metrics Changes - The ARCT-032 program for cystic fibrosis is progressing with a planned 12-week study to begin in the first half of 2026, following positive interim data from the ongoing phase 2 clinical trial [5][9] - The ARCT-810 program for ornithine transcarbamylase deficiency is preparing for regulatory meetings in 2026 to discuss pivotal trial strategies for both pediatric and adult populations [9][10] Market Data and Key Metrics Changes - The Costave COVID-19 vaccine program has faced delays in the BLA filing due to regulatory changes by the FDA, impacting commercial visibility in the U.S. [13] - The Japanese partner, Meiji Seika Pharma, has launched a two-dose vial of Costave updated for the JN.1 variant in Japan, marking the first distribution of this presentation [10] Company Strategy and Development Direction - The company is focused on advancing its mRNA therapeutics and vaccines pipeline, with a commitment to addressing the unmet medical needs in cystic fibrosis and OTC deficiency [9][18] - Cost reductions are being implemented to extend the runway for the cystic fibrosis and OTC programs, while expecting continued support from CSL for commercialization in Asia and Europe [13][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing clinical trials and the potential for ARCT-032 to address significant unmet medical needs in cystic fibrosis [7][8] - The company remains in a strong financial position with a cash runway extended into 2028, allowing for the achievement of near-term milestones [17] Other Important Information - The company plans to add additional sites for the upcoming 12-week study to accelerate enrollment, particularly targeting countries with higher prevalence of class one CF patients [78] - The FDA's stance on CT imaging as a potential endpoint for approval is still under discussion, with management indicating that it may serve as a supportive endpoint rather than a primary one [54] Q&A Session Summary Question: Have you been able to do some PK/PD modeling for the third-dose cohort? - Management confirmed that the third cohort is being evaluated similarly to the first two cohorts, with additional data collection planned for the 12-week study [21] Question: What are the optionalities for the OTC pivotal programs? - Management indicated that separate discussions with regulatory agencies will be held for adult and pediatric populations, focusing on different biomarkers for each group [24] Question: Do you expect improvements in mucus over time in the CF study? - Management noted that mucus plugs form in smaller airways and that improvements are expected over time, with the 12-week study designed to assess this [28] Question: What metrics will drive the go/no-go decision for the 15-mg dose? - Management stated that safety and tolerability will be key metrics, along with observing a dose response in mucus plug reduction [37] Question: When should we expect to see data from the 15-mg cohort? - Data from the 15-mg cohort is expected in the first quarter of next year, with plans to transition quickly to the 12-week study thereafter [44] Question: What is the age range for the pediatric population in the OTC program? - Management indicated that the age cutoff for pediatric patients will be determined in discussions with regulatory agencies [80]