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Core Insights - Arcturus Therapeutics Holdings Inc. held its Q2 2025 Earnings Conference on August 11, 2025, featuring key executives including the CEO and CFO [1][2][3]. Company Overview - The conference call was led by Joseph E. Payne, President and CEO, and Andrew H. Sassine, CFO, with Dr. Pad Chivukula, CSO and COO, participating in the Q&A session [3]. Financial Update - The call was part of Arcturus Therapeutics' quarterly financial update and pipeline progress, indicating ongoing developments in their business operations [3]. Investor Relations - Neda Safarzadeh, Vice President and Head of Investor Relations, Public Relations, and Marketing, facilitated the conference, highlighting the company's commitment to transparency with investors [2][3].

Company Performance - Arcturus Therapeutics reported a quarterly loss of $0.34 per share, significantly better than the Zacks Consensus Estimate of a loss of $1.11, representing an earnings surprise of +69.37% [1] - The company posted revenues of $28.3 million for the quarter ended June 2025, exceeding the Zacks Consensus Estimate by 20.84%, although this is a decline from $49.86 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 two times [2] Stock Performance - Arcturus Therapeutics shares have declined approximately 29.8% since the beginning of the year, contrasting with the S&P 500's gain of 8.6% [3] - The current Zacks Rank for Arcturus is 3 (Hold), indicating that the shares are expected to perform in line with the market in the near future [6] Future Outlook - The consensus EPS estimate for the upcoming quarter is -$1.12 on revenues of $20.92 million, and for the current fiscal year, it is -$3.73 on revenues of $70.59 million [7] - The outlook for the Medical - Biomedical and Genetics industry, where Arcturus operates, is currently in the bottom 41% of over 250 Zacks industries, which may impact the stock's performance [8]

Financial Data and Key Metrics Changes - Revenue for Q2 2025 was $28 million, a decrease of $22 million compared to Q2 2024, while revenue for the first half of 2025 was $58 million, down $30 million year-over-year [19] - Total operating expenses for Q2 2025 were $40 million, down from $71 million in Q2 2024, and for the first half of 2025, operating expenses were $86 million compared to $139 million in the prior year [19][20] - The net loss for Q2 2025 was approximately $9.2 million or $0.34 per diluted share, compared to a net loss of $17.2 million or $0.64 per diluted share in Q2 2024 [22] Business Line Data and Key Metrics Changes - The ARCT032 program for cystic fibrosis is advancing with enrollment in a Phase 2 trial, focusing on participants who do not benefit from existing CFTR modulator therapies [6][8] - The ARCT810 program for ornithine transcarbamylase deficiency showed positive interim data, with decreases in glutamine levels observed in participants [9][10] - The company is preparing for regulatory meetings regarding its partnered COVID-19 vaccine, with applications filed in multiple regions [12][13] Market Data and Key Metrics Changes - The company has streamlined its operations to focus on the OTC and cystic fibrosis programs, extending its financial runway into 2028 [18] - Cash, cash equivalents, and restricted cash were reported at $253.4 million as of June 30, 2025, down from $293.9 million at the end of 2024 [22] Company Strategy and Development Direction - The company is focusing on its mRNA therapeutics and vaccines pipeline, with plans to provide interim data from the Phase 2 CF study in September 2025 [23] - The company aims to engage with the FDA regarding pivotal trial designs and biomarker strategies for its therapeutic programs [39][40] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the safety and tolerability of its inhaled mRNA therapeutics, addressing historical challenges in the field [36] - The company anticipates that achieving safety and a positive measurable FEV will allow for further development of its cystic fibrosis program [28][46] Other Important Information - The company appointed Dr. Monsef Slawi as Chairman of the Board effective July 1, 2025 [17] - The restructuring plan is nearing completion, consolidating operations to enhance focus on key therapeutic areas [18] Q&A Session Summary Question: Can you provide a refresher on the success criteria for the CF program? - Management indicated that a 3% threshold for FEV improvement is expected to advance the program, with a focus on safety and tolerability [27][30] Question: What safety or efficacy data do you have on a blinded basis? - Management discussed the importance of addressing toxicology and tolerability issues in inhaled RNA therapeutics, emphasizing their advancements in delivery systems [36][38] Question: Will you proceed to a higher dose cohort for CF? - The current plan includes a 12-subject trial with three doses, and management confirmed flexibility in the dosing strategy [59] Question: What is the timing for the end of Phase II meeting with the FDA? - Management expects to complete Phase II enrollment this year and engage with the FDA shortly thereafter, aiming for a Phase III initiation in 2026 [78] Question: How will you discern treatment effects without a placebo arm? - Management noted that a placebo strategy could be implemented in Phase III, leveraging historical FEV data for self-control [88] Question: What are the expectations for the OTC program? - Management indicated that decisions regarding the higher dose cohort will influence the timeline for data release, with ongoing discussions with regulatory agencies [104]

[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section provides the unaudited condensed consolidated financial statements for Arcturus Therapeutics, including balance sheets, statements of operations, and cash flows, along with detailed notes [Item 1. Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited condensed consolidated financial statements for Arcturus Therapeutics as of June 30, 2025, and for the three and six-month periods then ended [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2025, total assets decreased to **$309.3 million** from **$344.1 million** at December 31, 2024, primarily due to reduced cash and cash equivalents Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $196,467 | $237,028 | | Total current assets | $274,503 | $305,979 | | **Total assets** | **$309,270** | **$344,069** | | **Liabilities & Equity** | | | | Total current liabilities | $46,515 | $65,489 | | **Total liabilities** | **$78,217** | **$103,091** | | **Total stockholders' equity** | **$231,053** | **$240,978** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For Q2 2025, total revenues were **$28.3 million** with a net loss of **$9.2 million**, a reduction from the prior year's **$49.9 million** revenue and **$17.2 million** net loss Q2 2025 vs Q2 2024 Statement of Operations (in thousands, except per share data) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Total revenue | $28,301 | $49,859 | | Total operating expenses | $39,917 | $70,985 | | Loss from operations | $(11,616) | $(21,126) | | **Net loss** | **$(9,180)** | **$(17,216)** | | Net loss per share | $(0.34) | $(0.64) | Six Months Ended June 30, 2025 vs 2024 Statement of Operations (in thousands, except per share data) | Metric | H1 2025 | H1 2024 | | :--- | :--- | :--- | | Total revenue | $57,683 | $87,871 | | Total operating expenses | $86,125 | $139,409 | | Loss from operations | $(28,442) | $(51,538) | | **Net loss** | **$(23,256)** | **$(44,033)** | | Net loss per share | $(0.86) | $(1.64) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to **$40.9 million** for the six months ended June 30, 2025, resulting in a **$40.6 million** net decrease in cash Cash Flow Summary for Six Months Ended June 30 (in thousands) | Activity | 2025 | 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(40,893) | $(35,699) | | Net cash used in investing activities | $(137) | $(568) | | Net cash provided by financing activities | $469 | $4,591 | | **Net decrease in cash, cash equivalents and restricted cash** | **$(40,561)** | **$(31,676)** | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail significant accounting policies, revenue recognition, balance sheet components, and the impact of new tax laws, including a **$20.0 million** milestone from CSL Seqirus - The company's COVID-19 vaccine, KOSTAIVE® (ARCT-154), received marketing authorization in Japan in 2023 and became commercially available in September 2024, marking it as the world's first approved self-amplifying RNA (sa-mRNA) vaccine[24](index=24&type=chunk) - During Q2 2025, the company achieved **$20.0 million** in development milestones related to the CSL Collaboration Agreement[38](index=38&type=chunk) Revenue Breakdown by Source (in thousands) | Revenue Source | Q2 2025 | Q2 2024 | H1 2025 | H1 2024 | | :--- | :--- | :--- | :--- | :--- | | CSL Seqirus | $24,427 | $45,911 | $49,900 | $78,292 | | BARDA | $3,791 | $3,883 | $7,696 | $9,297 | | **Total Collaboration Revenue** | **$24,510** | **$45,976** | **$49,987** | **$78,574** | | **Total Grant Revenue** | **$3,791** | **$3,883** | **$7,696** | **$9,297** | - The One Big Beautiful Bill Act (OBBBA), signed into law on July 4, 2025, is expected to impact the company's effective tax rate and deferred tax liability, with changes to be reflected starting in Q3 2025[67](index=67&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's transition to a commercial-stage entity following the Japanese launch of KOSTAIVE®, detailing business updates, financial results, liquidity, and future funding requirements [Business Updates](index=23&type=section&id=Business%20Updates) The company highlights significant progress across its vaccine and therapeutic programs, including European marketing authorization for KOSTAIVE® and advancements in influenza, OTC, and cystic fibrosis pipelines - KOSTAIVE® (ARCT-154) received marketing authorization from the European Commission in February 2025, valid in all 27 EU member states and 3 EEA countries[89](index=89&type=chunk) - CSL Seqirus submitted a marketing authorization application for KOSTAIVE® to the UK's MHRA in June 2025[90](index=90&type=chunk) - The pandemic avian influenza (H5N1) vaccine candidate, ARCT-2304, was granted Fast Track Designation by the U.S. FDA in April 2025[104](index=104&type=chunk) - Positive multiple-dosing data from two Phase 2 studies of ARCT-810 for OTC deficiency were announced on June 30, 2025, showing a significant decrease in glutamine levels and an increase in ureagenesis function[111](index=111&type=chunk)[112](index=112&type=chunk) [Results of Operations](index=29&type=section&id=Results%20of%20Operations) Revenue decreased by **43%** in Q2 2025 and **34%** for the first six months, primarily due to reduced CSL collaboration revenue, while total operating expenses fell significantly due to lower R&D and G&A costs Revenue Comparison (in thousands) | Period | 2025 | 2024 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Three Months Ended June 30 | $28,301 | $49,859 | $(21,558) | -43% | | Six Months Ended June 30 | $57,683 | $87,871 | $(30,188) | -34% | Operating Expense Comparison (in thousands) | Period | Expense Category | 2025 | 2024 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | :--- | | **Q2** | Research & Development, net | $29,579 | $58,669 | $(29,090) | -50% | | | General & Administrative | $10,338 | $12,316 | $(1,978) | -16% | | **H1** | Research & Development, net | $64,471 | $112,242 | $(47,771) | -43% | | | General & Administrative | $21,654 | $27,167 | $(5,513) | -20% | - The decrease in R&D expenses was primarily driven by lower manufacturing and clinical costs for the LUNAR-COVID program, reflecting its transition from development to commercial phase[122](index=122&type=chunk)[123](index=123&type=chunk) [Liquidity and Capital Resources](index=32&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2025, the company held **$253.4 million** in cash, cash equivalents, and restricted cash, with management expecting current funds to cover operations for at least the next twelve months, though additional capital will be required for long-term plans - The company's cash, cash equivalents, and restricted cash balance was **$253.4 million** as of June 30, 2025[132](index=132&type=chunk) - The company has achieved a total of approximately **$509.7 million** in upfront payments and milestones from CSL Seqirus through Q2 2025[132](index=132&type=chunk) - Management expects current cash to be sufficient for at least the next twelve months, but will need to raise additional capital to fund long-term development[151](index=151&type=chunk) Cash Flow Summary for Six Months Ended June 30 (in thousands) | Activity | 2025 | 2024 | | :--- | :--- | :--- | | Cash used in Operating activities | $(40,893) | $(35,699) | | Cash used in Investing activities | $(137) | $(568) | | Cash provided by Financing activities | $469 | $4,591 | | **Net decrease in cash** | **$(40,561)** | **$(31,676)** | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=39&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity on its cash and cash equivalents, which management considers not material - The company's main market risk is interest rate changes affecting interest income on its cash and cash equivalents, which is not considered a material risk[156](index=156&type=chunk) [Item 4. Controls and Procedures](index=39&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were not effective as of June 30, 2025, due to a material weakness in internal control over financial reporting related to information technology general controls (ITGCs) - Management concluded that disclosure controls and procedures were not effective as of June 30, 2025, due to a material weakness in internal control over financial reporting[157](index=157&type=chunk) - The material weakness relates to information technology general controls (ITGCs), including deficiencies in user access controls, program change management, and monitoring of critical IT operations[161](index=161&type=chunk) - A remediation plan is in progress, involving enhancing IT policies, limiting system access, improving user access reviews, and hiring an internal audit manager, with completion expected in fiscal year 2025[162](index=162&type=chunk)[163](index=163&type=chunk)[164](index=164&type=chunk) [PART II. OTHER INFORMATION](index=42&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section covers legal proceedings, risk factors, equity sales, other information, and exhibits [Item 1. Legal Proceedings](index=42&type=section&id=Item%201.%20Legal%20Proceedings) The company initiated arbitration against CSL Seqirus on May 30, 2025, to seek a milestone payment related to KOSTAIVE®'s European marketing authorization, which CSL Seqirus disputes - On May 30, 2025, Arcturus initiated an arbitration against CSL Seqirus to seek payment of a milestone under their collaboration agreement following the European marketing authorization of KOSTAIVE®[168](index=168&type=chunk) - CSL Seqirus submitted an Answer on July 25, 2025, contending it does not have a current obligation to pay the milestone, and the arbitration is ongoing[168](index=168&type=chunk) [Item 1A. Risk Factors](index=42&type=section&id=Item%201A.%20Risk%20Factors) No material changes have occurred from the risk factors previously disclosed in the company's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 - No material changes have occurred from the risk factors described in the Annual Report on Form 10-K for the year ended December 31, 2024[170](index=170&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=42&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period - None[171](index=171&type=chunk) [Item 5. Other Information](index=42&type=section&id=Item%205.%20Other%20Information) During the three months ended June 30, 2025, none of the company's directors or officers adopted or terminated a Rule 10b5-1 trading arrangement - No directors or officers adopted or terminated a Rule 10b5-1 trading arrangement during the second quarter of 2025[174](index=174&type=chunk) [Item 6. Exhibits](index=43&type=section&id=Item%206.%20Exhibits) This section provides an index of all exhibits filed with or incorporated by reference into this Quarterly Report on Form 10-Q, including sales agreements, corporate governance documents, material contracts, and officer certifications

[Corporate and Pipeline Highlights](index=1&type=section&id=Corporate%20and%20Pipeline%20Highlights) Arcturus is advancing its mRNA pipeline, with key progress in its Cystic Fibrosis (ARCT-032) and OTC Deficiency (ARCT-810) programs, announcing positive interim data for ARCT-810 and expecting Phase 2 data for ARCT-032 in September 2025, while progressing regulatory filings for KOSTAIVE® globally and reporting positive Phase 1 results for its seasonal flu vaccine (ARCT-2138) with Phase 1 results for its pandemic flu vaccine (ARCT-2304) expected in 2025 [Cystic Fibrosis (CF) Program (ARCT-032)](index=1&type=section&id=Cystic%20Fibrosis%20(CF)%20Program%20(ARCT-032)) The Phase 2 trial for ARCT-032 is advancing, with enrollment expected to be complete by year-end 2025, interim data from the first nine participants will be presented in September 2025, and the company plans to meet with regulatory agencies in H1 2026 to discuss pivotal trial plans * Phase 2 interim data from the first nine participants (N=3 @ 5 mg; N=6 @ 10 mg) is scheduled to be presented in **September 2025**[1](index=1&type=chunk)[3](index=3&type=chunk) * The Phase 2 trial is expected to complete enrollment by the **end of 2025**[1](index=1&type=chunk)[3](index=3&type=chunk) * Meetings with the FDA and other regulatory agencies to discuss Phase 3 plans are anticipated in the **first half of 2026**, with Phase 3 initiation planned for **2026**[3](index=3&type=chunk) [OTC Deficiency Program (ARCT-810)](index=1&type=section&id=OTC%20Deficiency%20Program%20(ARCT-810)) Positive interim data from two Phase 2 studies showed that ARCT-810 led to decreases in glutamine levels to within the normal range and maintained stable ammonia levels in participants, with the treatment generally safe and well-tolerated across 40 participants to date, and the company expects to align with regulatory agencies on the Phase 3 trial design in the first half of 2026 * Positive interim Phase 2 data showed decreases in glutamine to normal range and stable ammonia levels in participants on standard of care therapy[3](index=3&type=chunk) * Data from a newly developed assay suggested improvement of urea cycle function in all three participants in the Phase 2 U.S. study[3](index=3&type=chunk) * The company plans to align with regulators on the Phase 3 trial design and pediatric studies in **H1 2026**[1](index=1&type=chunk)[5](index=5&type=chunk) [KOSTAIVE® (COVID-19 Vaccine) Regulatory Updates](index=2&type=section&id=KOSTAIVE%C2%AE%20(COVID-19%20Vaccine)%20Regulatory%20Updates) Regulatory submissions for KOSTAIVE® are progressing in the UK, Japan, and the U.S., with approval in the UK expected by September 2025, Japanese approvals anticipated in Q3/Q4 2025, and a U.S. BLA filing on track for Q3 2025 * UK (MHRA) approval is expected by **September 2025**[5](index=5&type=chunk) * Japan (PMDA) approvals for the lyophilized vaccine and the 2025-2026 variant update are anticipated in **Q3/Q4 2025**[5](index=5&type=chunk) * U.S. (FDA) BLA filing remains on track for **Q3 2025**, with an approval decision expected in **2026**[5](index=5&type=chunk) [Influenza Vaccine Programs (ARCT-2138 & ARCT-2304)](index=1&type=section&id=Influenza%20Vaccine%20Programs%20(ARCT-2138%20%26%20ARCT-2304)) The seasonal flu vaccine (ARCT-2138) showed positive Phase 1 results, demonstrating immunogenicity against all four strains in both young and older adults with a good safety profile, while the pandemic flu vaccine (ARCT-2304), supported by BARDA, has received FDA Fast Track Designation, and Phase 1 immunogenicity results are expected in Q4 2025 * Seasonal flu vaccine (ARCT-2138) Phase 1 study showed immunogenicity against all four influenza strains at doses as low as **2 μg** and was well-tolerated[5](index=5&type=chunk) * Pandemic flu vaccine (ARCT-2304) received U.S. FDA Fast Track Designation[5](index=5&type=chunk) * Phase 1 immunogenicity results for the pandemic flu vaccine (ARCT-2304) are expected in **Q4 2025**, with no safety concerns raised from available data[5](index=5&type=chunk) [Financial Results (Q2 2025)](index=3&type=section&id=Financial%20Results%20for%20the%20three%20months%20ended%20June%2030%2C%202025) For Q2 2025, Arcturus reported revenues of **$28.3 million** and a net loss of **$9.2 million**, or **($0.34)** per share, representing a decrease in revenue but a smaller net loss compared to Q2 2024, primarily due to reduced R&D spending as key programs mature, with the company's cash position remaining strong at **$253.4 million** and a projected cash runway into 2028 [Statement of Operations Analysis](index=3&type=section&id=Statement%20of%20Operations%20Analysis) Revenue for Q2 2025 was **$28.3 million**, a decrease from **$49.9 million** in Q2 2024, primarily due to lower revenue from the CSL collaboration as KOSTAIVE® progresses towards commercialization, while operating expenses also decreased significantly to **$39.9 million** from **$71.0 million** year-over-year, driven by lower R&D costs, resulting in a reduced net loss of **$9.2 million** compared to **$17.2 million** in the prior year's quarter Statement of Operations (in thousands) | Metric (in thousands) | Q2 2025 | Q2 2024 | YTD 2025 | YTD 2024 | | :--- | :--- | :--- | :--- | :--- | | **Total Revenue** | **$28,301** | **$49,859** | **$57,683** | **$87,871** | | R&D Expenses | $29,579 | $58,669 | $64,471 | $112,242 | | G&A Expenses | $10,338 | $12,316 | $21,654 | $27,167 | | **Net Loss** | **$(9,180)** | **$(17,216)** | **$(23,256)** | **$(44,033)** | | Net Loss per Share | $(0.34) | $(0.64) | $(0.86) | $(1.64) | * The decrease in revenue was primarily driven by reduced activity and lower amortization from the CSL collaboration as KOSTAIVE® nears commercialization[6](index=6&type=chunk) * The decrease in R&D expenses was mainly due to lower manufacturing and clinical costs for the KOSTAIVE and LUNAR-FLU programs, partially offset by higher clinical costs for the cystic fibrosis Phase 2 trials[8](index=8&type=chunk)[9](index=9&type=chunk) [Balance Sheet and Cash Position](index=4&type=section&id=Balance%20Sheet%20and%20Cash%20Position) As of June 30, 2025, Arcturus had **$253.4 million** in cash, cash equivalents, and restricted cash, down from **$293.9 million** at year-end 2024, with total assets at **$309.3 million** and total liabilities at **$78.2 million**, and the company projects its current cash runway extends into 2028 Balance Sheet (in thousands) | Metric (in thousands) | June 30, 2025 | Dec 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents & restricted cash | $253,352 | $293,913 | | Total Assets | $309,270 | $344,069 | | Total Liabilities | $78,217 | $103,091 | | Total Stockholders' Equity | $231,053 | $240,978 | * The company's cash runway is expected to extend into **2028** based on the current pipeline and programs[12](index=12&type=chunk) [Company Overview and Forward-Looking Statements](index=4&type=section&id=Company%20Overview%20and%20Forward-Looking%20Statements) Arcturus Therapeutics, founded in 2013 and based in San Diego, is a commercial mRNA medicines company with proprietary LUNAR® delivery and STARR® mRNA technologies, having developed KOSTAIVE®, the first approved sa-mRNA COVID vaccine, and maintaining a pipeline focused on rare diseases like OTC deficiency and cystic fibrosis, as well as infectious disease vaccines, while the press release contains forward-looking statements regarding clinical trial timelines, regulatory approvals, and financial projections, which are subject to substantial risks and uncertainties * Arcturus is a commercial mRNA company with key technologies including LUNAR® lipid-mediated delivery and STARR® self-amplifying mRNA[13](index=13&type=chunk) * The company's pipeline includes RNA therapeutic candidates for OTC deficiency and cystic fibrosis, along with partnered mRNA vaccine programs for COVID-19 and influenza[13](index=13&type=chunk) * The report includes forward-looking statements concerning clinical trial timelines, regulatory approvals, and financial performance, which are subject to significant risks as detailed in SEC filings[14](index=14&type=chunk)

ARCT-810 Overview - Arcturus is a commercial mRNA medicines company with a pipeline of multiple therapeutic candidates in advanced clinical trial development[7] - ARCT-810 aims to restore OTC enzyme function, potentially preventing neurological damage and the need for liver transplantation in Ornithine Transcarbamylase (OTC) Deficiency patients[9] - ARCT-810 has received Orphan Drug Designation (FDA), Orphan Medicinal Product Designation (EMA), Fast Track Designation (FDA), and Rare Pediatric Disease Designation (FDA)[14, 15, 16, 17] Clinical Trial Results - European Phase 2 study (N = 8; 6 ARCT-810 / 2 placebo) is completed, and the U S Phase 2 study (N = 3 completed to date) is ongoing[20] - Combined analysis of both Phase 2 studies showed mean glutamine levels decreased significantly (N = 8, p-value = 0 0055)[25] - In the Phase 2 Open-label U S Study, mean glutamine levels decreased significantly (N = 3, p-value = 0 004), with all subjects achieving normal levels after three administrations[28] - Interim Phase 2 data from the U S Open Label Study (N = 3) showed a mean RUF (relative ureagenesis function) increase of +14 7% from baseline to 28 days post-fifth dose, from 29 0% (SD 9 1%) to 43 7% (SD 21 7%), which is statistically significant (p-value = 0 026)[31] Safety and Tolerability - ARCT-810 was generally safe and well-tolerated in Phase 2 studies at all tested dose levels, comprising 40 participants to date, including 20 OTC deficient participants[34, 35] KOL Insights - Glutamine rises earlier than ammonia in OTC deficiency and has lower variability, making it a reliable indicator of urea cycle stress[47] - 15N-Ureagenesis assay directly reflects cycle function, providing a clear readout of therapeutic efficacy[47]

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 Viewpoint - Arcturus Therapeutics (ARCT) has seen a 7.5% increase in share price over the past four weeks, closing at $12.81, with analysts suggesting a potential upside of 371.4% based on a mean price target of $60.38 [1][12]. Price Targets and Analyst Estimates - The mean price target is derived from eight short-term estimates, with a standard deviation of $33.97, indicating variability among analysts [2]. - The lowest price target of $32 suggests a 149.8% increase, while the highest target of $140 indicates a potential surge of 992.9% [2]. - A low standard deviation among price targets suggests a strong consensus among analysts regarding the stock's price direction [9]. Earnings Estimates and Analyst Agreement - Analysts show strong agreement in revising earnings estimates upward, which is a positive indicator for potential stock upside [4][11]. - The Zacks Consensus Estimate for the current year has increased by 42.1% over the past month, with four estimates rising and no negative revisions [12]. - ARCT holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors [13]. Caution on Price Targets - While price targets are a common metric for investors, relying solely on them can be misleading, as they often do not accurately predict stock price movements [3][7][10]. - Analysts may set overly optimistic price targets due to business incentives, which can inflate expectations [8].

Core Viewpoint - Arcturus Therapeutics (ARCT) shows significant upside potential based on Wall Street analysts' short-term price targets, with a mean target of $65.43 indicating a 503.6% increase from the current price of $10.84 [1] Price Targets and Estimates - The mean estimate consists of seven short-term price targets with a standard deviation of $34.14, indicating variability among analysts. The lowest estimate of $44 suggests a 305.9% increase, while the highest estimate predicts a surge of 1191.5% to $140 [2] - A low standard deviation in price targets indicates a high degree of agreement among analysts regarding the stock's price movement direction and magnitude [9] Earnings Estimates and Analyst Sentiment - Analysts are optimistic about ARCT's earnings prospects, as indicated by a positive trend in earnings estimate revisions. The Zacks Consensus Estimate for the current year has increased by 28.3% over the last 30 days, with four estimates moving higher and no negative revisions [11][12] - ARCT holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates, suggesting a strong potential upside in the near term [13] Caution on Price Targets - While the consensus price target is a commonly referenced metric, relying solely on it for investment decisions may not be prudent due to historical inaccuracies in analysts' price targets [3][7][10] - Analysts often set optimistic price targets influenced by business relationships, which can lead to inflated expectations [8]