Core Insights - Eterna Therapeutics Inc. has successfully completed a comprehensive financial restructuring aimed at accelerating its developmental activities and long-term success [1][2] Financial Restructuring - The company has reduced its liabilities by discontinuing a longstanding lease, resulting in estimated savings of $72 million and a reduction of approximately $700,000 in monthly cash outflow [2] - With shareholder approval, Eterna has completed transactions that significantly reduced its balance sheet debt and secured a new round of $5 million in financing through a PIPE investment [2] Strategic Focus - The financial restructuring allows Eterna to execute its strategic mission to develop innovative off-the-shelf cellular therapies, both internally and in collaboration with academic partners, with greater financial agility [3] Product Development - Eterna is advancing its lead therapeutic candidate, ERNA-101, which is designed to deliver IL-7/IL-15 selectively to tumors, focusing initially on triple-negative breast cancer (TNBC) and platinum-resistant, TP53-mutant ovarian cancer [4] - The company aims to be well-positioned for IND-enabling studies and targets IND submissions by 2026 [4] Company Overview - Eterna Therapeutics is a publicly traded, preclinical-stage company focused on innovative cell therapies for advanced solid tumors, particularly TNBC and platinum-resistant, TP53-mutant ovarian cancer [5] - The lead product, ERNA-101, utilizes a best-in-class approach to engineer allogenic induced pluripotent stem cells (iPSCs) to express genes of interest and efficiently differentiate into mesenchymal stem cells [5]

Core Viewpoint - Eterna Therapeutics Inc. and Factor Bioscience Limited have entered into an exclusive license and collaboration agreement to accelerate the development of advanced cell therapy candidates targeting oncology, rare diseases, and autoimmune disorders [1] Group 1: Agreement Details - Eterna has secured a worldwide, exclusive, non-transferable, royalty-bearing license to develop and market certain induced pluripotent stem cell (iPSC)-based cell therapy products, specifically iPSC-derived mesenchymal stem cells (iMSC) engineered to express certain cytokines [1] - The collaboration aims to generate data demonstrating the efficacy of the licensed drug candidates for development towards Investigational New Drug (IND) applications by Eterna or third-party sublicensees [2] - Factor Bioscience will receive milestone payments per product candidate and post-commercialization royalties as part of the agreement [2] Group 2: Company Focus and Pipeline - Eterna Therapeutics is a preclinical-stage company focused on innovative off-the-shelf cell therapies for advanced solid tumors, particularly triple-negative breast cancer (TNBC) and platinum-resistant, TP53-mutant ovarian cancer [3] - The lead product, ERNA-101, is designed to selectively deliver pro-inflammatory cytokines IL-7 and IL-15 to the tumor microenvironment to enhance anti-tumor immunity [3] - Eterna's technology utilizes a best-in-class approach to engineer allogenic iPSCs to express genes of interest and efficiently differentiate into iMSCs [3] Group 3: Factor Bioscience Overview - Factor Bioscience, founded in 2011, is a leading cell engineering platform technology company focused on harnessing cell engineering to treat diseases and improve health [4] - The company is privately held and headquartered in Cambridge, MA [4]

Revenue and Loss - Revenue for the three months ended March 31, 2024, was $47,000, compared to $0 for the same period in 2023, reflecting a $47,000 increase [141] - The net loss for Q1 2024 was $6,647,000, compared to a net loss of $5,416,000 in Q1 2023, representing an increase in loss of $1,231,000 [141] - The company incurred a net loss of $6.6 million for the three months ended March 31, 2024, and used $3.7 million in operating activities [150] Expenses - Cost of revenues for Q1 2024 was $61,000, up from $50,000 in Q1 2023, resulting in a gross loss of $14,000, an improvement of $36,000 year-over-year [141] - Research and development expenses decreased by approximately $216,000 to $1,458,000 in Q1 2024, primarily due to reduced professional fees and other miscellaneous expenses [144] - General and administrative expenses increased by $723,000 to $4,315,000 in Q1 2024 compared to $3,592,000 in Q1 2023 [141] - General and administrative expenses increased by approximately $0.7 million to $4.3 million for the three months ended March 31, 2024, compared to $3.6 million in the same period of 2023 [145] - The company recognized an increase in interest expense of approximately $0.8 million for the three months ended March 31, 2024, primarily due to interest related to convertible notes [148] Cash and Financing - Cash and cash equivalents as of March 31, 2024, were approximately $9.2 million, with $4.1 million classified as restricted cash [150] - A private placement of convertible notes raised $9.2 million, with $7.8 million received on December 15, 2023, and $1.4 million on January 11, 2024 [131] - The company has an outstanding amount of $18.5 million under convertible notes, with $9.0 million related to July 2023 notes and $9.5 million to December 2023 notes [165] - Cash used in operating activities decreased by approximately $2.3 million to $3.7 million for the three months ended March 31, 2024, compared to $6.0 million in the same period of 2023 [158] - The company plans to raise additional funds to support working capital needs through various means, including public or private equity offerings and debt financings [152] Operational Plans - The company aims to transition from a preclinical to a clinical-stage company by initiating its first-in-human study and diversifying its pipeline of product candidates [125] - The company plans to leverage its mRNA technology platform through strategic partnerships and out-licensing arrangements to generate revenue [133] Lease Obligations - The company has incurred approximately $2.3 million in past due rent obligations under its sublease in Somerville, Massachusetts [132] - The company has entered into a sublease for approximately 45,500 square feet of office and laboratory space with estimated base rent obligations of approximately $63.0 million over 10 years [161] - As of May 3, 2024, the company has past due rent payments of approximately $2.3 million related to the sublease [162] Technology - The mRNA gene-editing technology is designed to enable precise editing of DNA sequences, potentially leading to new therapeutic approaches for cancer treatment [128] Tax Liabilities - The company expects to incur state income tax liabilities in 2024 and has established a full valuation allowance for all deferred tax assets [149]

CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Eterna Therapeutics Inc. (NASDAQ:ERNA) ("Eterna" or the "Company"), a preclinical-stage biopharmaceutical company, committed to realizing the potential of mRNA cell engineering to provide patients with transformational new medicines, today announces the appointment of Mahendra Rao, PhD, to its scientific advisory board. "We're honored to have Dr. Mahendra Rao join our Scientific Advisory Board," said Sanjeev Luther, President and CEO of Eterna. "His deep sc ...

Intellectual Property and Licensing - The company has in-licensed a portfolio of over 100 patents covering key mRNA cell engineering technologies, including mRNA delivery and gene editing [22]. - The company plans to derive revenue in the short term by leveraging its core intellectual property portfolio through licensing and co-development arrangements [24]. - An amended and restated exclusive license agreement was entered into with Factor Limited on November 14, 2023, which includes a term expiring on November 22, 2027, with a potential five-year extension if $6.0 million in sublicense fees are paid [35]. - The company will pay Factor Limited 20% of any sublicense fee received during the term of the agreement and a monthly maintenance fee of approximately $0.4 million starting September 2023 [35]. - As of February 6, 2024, the company has in-licensed 16 patent families, including 138 granted patents and 100 pending applications, focusing on mRNA technology [37]. - The patent portfolio includes various families such as FAB-001, FAB-003, and FAB-005, which cover methods for transfecting cells and expressing proteins [40]. - The company is obligated to pay expenses incurred by Factor Limited for maintaining the Factor Patents and bear all costs associated with enforcing these patents [35]. - Patent terms generally last for 20 years from the earliest effective filing date, with potential extensions for regulatory delays, but cannot exceed 14 years from FDA approval [42]. - The company may face challenges in obtaining and maintaining intellectual property rights, which could materially impact its business [44]. Business Strategy and Goals - The mid-term goal is to transform from a preclinical stage company to a clinical-stage company by achieving IND approval and initiating first-in-human studies [25]. - The long-term aspiration is to become a therapeutics company with multiple approved gene and cellular therapy products across various indications, including oncology and rare diseases [26]. - The company has a strategy to develop therapeutic products through strategic partnerships, leveraging its mRNA technology platform [37]. - The company is focusing on strategic partnerships to deploy its mRNA technology platform for preclinical and clinical development of product candidates [47]. Product Development and Clinical Trials - The company has ceased the development of its former product candidate IRX-2 and does not currently plan to develop any product candidates [27]. - The company anticipates generating allogeneic CAR-T therapies for cancer treatment using its mRNA gene-editing technology [31]. - The mRNA gene-editing technology is designed to delete, insert, and repair DNA sequences in living cells, potentially correcting disease-causing mutations [29]. - The mRNA delivery technology aims to enhance the uptake of nucleic acids by cells, addressing limitations of conventional delivery systems [28]. - The company is developing several innovative therapies, including mesenchymal stem cell therapies and engineered immune cell therapies [46]. - The company is also exploring the use of circular RNA structures for gene editing and therapeutic applications [46]. - The regulatory framework for drug and biologic approvals in the U.S. requires substantial time and financial resources, impacting the company's operations and financial condition [48]. - The FDA's approval process for drugs and biologics involves multiple stages, including preclinical tests, IND submission, and clinical trials, which can take years [63]. - The company must ensure compliance with current good manufacturing practices to maintain product quality and safety during production [61]. - The FDA may impose additional requirements or deny applications based on safety and efficacy evaluations, affecting the timing of product launches [64]. - The company is required to conduct Phase 4 clinical trials post-approval to gain additional experience and document clinical benefits [58]. - The FDA may require post-marketing testing and surveillance, which can impact the market potential and profitability of approved products [71]. Financial Condition and Challenges - The company faces substantial capital requirements and has incurred significant losses since inception, making it difficult to assess future prospects [17]. - The company must navigate complex foreign regulatory requirements for marketing authorization, which vary by country [75]. - The company is subject to annual program fees and user fees that typically increase annually as part of the FDA approval process [64]. - The company faces intense competition from major pharmaceutical and biotechnology firms, which may have greater resources and strategic partnerships [82]. - The company is unable to predict future legislative changes that may affect the healthcare industry and its business operations [81]. Employee and Ethical Standards - The company has eight full-time employees, including four in research and development and four in administrative roles [83]. - The company emphasizes competitive compensation and benefits to attract and retain talent, aligning employee interests with stockholder interests [84]. - The company is committed to ethical business practices, emphasizing integrity and fairness in its Code of Conduct [88]. - The company has implemented extensive safety measures for employees during the COVID-19 pandemic, including on-site testing and flexible remote work options [89]. - The company’s employee compensation strategy includes performance-based cash and equity compensation to align employee interests with stockholder interests [86]. - The company is committed to maintaining high ethical standards and promoting employee well-being, especially during the COVID-19 pandemic [88].

PART I – FINANCIAL INFORMATION [Financial Statements](index=4&type=section&id=Item%201.%20Financial%20Statements) The unaudited condensed consolidated financial statements for the period ended September 30, 2023, are presented [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets and liabilities increased significantly due to new lease obligations, while stockholders' equity declined **Condensed Consolidated Balance Sheet Summary (in thousands)** | Account | Sep 30, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash | $4,551 | $11,446 | | Total current assets | $7,031 | $13,681 | | Right-of-use assets - operating leases | $34,860 | $1,030 | | **Total assets** | **$52,248** | **$22,279** | | **Liabilities & Equity** | | | | Total current liabilities | $9,626 | $7,654 | | Convertible notes payable, net | $3,452 | $0 | | Operating lease liabilities, non-current | $34,998 | $887 | | **Total liabilities** | **$48,686** | **$10,172** | | **Total stockholders' equity** | **$3,562** | **$12,107** | [Condensed Consolidated Statements of Operations](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) The company reported its first revenue and a reduced net loss for Q3 2023 compared to the prior-year period **Condensed Consolidated Statements of Operations Summary (in thousands, except per share amounts)** | Metric | Q3 2023 | Q3 2022 | Nine Months 2023 | Nine Months 2022 | | :--- | :--- | :--- | :--- | :--- | | Revenue | $51 | $0 | $51 | $0 | | Research and development | $1,457 | $4,963 | $4,710 | $8,430 | | General and administrative | $3,979 | $3,341 | $10,081 | $14,060 | | Impairment of IPR&D | $0 | $0 | $0 | $5,990 | | Loss from operations | $(5,505) | $(8,304) | $(15,370) | $(28,480) | | **Net loss** | **$(5,591)** | **$(7,316)** | **$(15,515)** | **$(20,090)** | | Net loss per share | $(1.03) | $(2.49) | $(2.94) | $(7.04) | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operations remained stable while financing activities provided less cash than the prior year **Cash Flow Summary for the Nine Months Ended September 30 (in thousands)** | Cash Flow Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(15,747) | $(15,541) | | Net cash used in investing activities | $0 | $(176) | | Net cash provided by financing activities | $8,852 | $11,986 | | **Net decrease in cash and cash equivalents** | **$(6,895)** | **$(3,731)** | [Notes to Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Key disclosures include a going concern warning, recent financing, an asset acquisition, and a new facility sublease - Management concluded there is **substantial doubt about the Company's ability to continue as a going concern**, as it does not expect to have sufficient cash to fund operations for the next twelve months[34](index=34&type=chunk) - In July 2023, the company closed a financing for approximately **$8.7 million in convertible promissory notes** and warrants to purchase 6.1 million shares of common stock[33](index=33&type=chunk)[55](index=55&type=chunk) - In April 2023, the company acquired intellectual property assets from Exacis Biotherapeutics Inc for **$460,000**, which was expensed as in-process research and development (IPR&D)[36](index=36&type=chunk)[42](index=42&type=chunk) - The company recognized its **first revenue of less than $0.1 million** for cell line customization activities in Q3 2023[43](index=43&type=chunk)[51](index=51&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=27&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses reduced net losses, recent financing, and expresses substantial doubt about its going concern status [Results of Operations](index=30&type=section&id=Results%20of%20Operations) The net loss for the nine-month period decreased by $4.5 million, driven by lower operating expenses **Comparison of Operating Results (in thousands)** | Item | Nine Months 2023 | Nine Months 2022 | Change | | :--- | :--- | :--- | :--- | | Revenue | $51 | $0 | $51 | | Research and development | $4,710 | $8,430 | $(3,720) | | General and administrative | $10,081 | $14,060 | $(3,979) | | Impairment of IPR&D | $0 | $5,990 | $(5,990) | | **Loss from operations** | **$(15,370)** | **$(28,480)** | **$13,110** | | **Net loss** | **$(15,515)** | **$(20,090)** | **$4,575** | - The decrease in R&D expenses was primarily due to a reduction in MSA fees paid to Factor Bioscience and lower payroll and stock-based compensation expense from employee terminations[167](index=167&type=chunk) - The decrease in G&A expenses was due to lower payroll, reduced professional fees, and the non-recurrence of a ROU asset impairment and loss on disposal of fixed assets from 2022[170](index=170&type=chunk) [Liquidity and Capital Resources](index=34&type=section&id=Liquidity%20and%20Capital%20Resources) The company holds $4.6 million in unrestricted cash but lacks sufficient funds for the next twelve months - The company had cash, cash equivalents, and restricted cash of $8.6 million, of which **$4.6 million was unrestricted** as of September 30, 2023[181](index=181&type=chunk) - Management states that based on current financial conditions, they **do not have sufficient funds for operations for the next twelve months**, raising substantial doubt about the ability to continue as a going concern[190](index=190&type=chunk) - In July 2023, the company closed a financing of **$8.7 million in aggregate principal amount of Convertible Notes** and issued warrants[187](index=187&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=37&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, Eterna Therapeutics Inc is exempt from providing this information - The company is a smaller reporting company and is not required to provide the information otherwise required by this item[202](index=202&type=chunk) [Controls and Procedures](index=37&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls were not effective due to a previously identified material weakness - Management concluded that **disclosure controls and procedures were not effective** as of the end of the period covered by the report[206](index=206&type=chunk) - The ineffectiveness stems from a material weakness identified due to errors in prior financial statements, which management attributed to accounting personnel's **lack of technical proficiency in complex matters**[208](index=208&type=chunk) - Remediation plans include enhancing review processes for complex transactions, providing additional training, and consulting with an accounting advisor[215](index=215&type=chunk) PART II – OTHER INFORMATION [Legal Proceedings](index=38&type=section&id=Item%201.%20Legal%20Proceedings) The company is involved in ongoing litigation with former employees and faces a trademark infringement lawsuit - The company's subsidiary, Novellus, Inc, is in a legal dispute with former employees involving claims and counterclaims related to conduct that took place before Eterna's acquisition of Novellus[102](index=102&type=chunk)[103](index=103&type=chunk) - On July 31, 2023, eTheRNA Immunotherapies NV and eTheRNA Inc filed a complaint against Eterna Therapeutics Inc alleging **federal and state trademark infringement** and unfair competition[106](index=106&type=chunk) [Risk Factors](index=39&type=section&id=Item%201A.%20Risk%20Factors) New material risks arise from restrictive covenants and potential cash redemption requirements of recent convertible notes - The Convertible Notes contain **restrictive covenants** that limit the company's ability to, among other things, create liens, pay dividends, and incur additional indebtedness[216](index=216&type=chunk) - The company is **required to redeem the Convertible Notes for cash** under certain circumstances, and failure to do so could lead to default, acceleration of debt, and potential bankruptcy or liquidation[218](index=218&type=chunk)[221](index=221&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=39&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities were reported during the period that were not previously disclosed - There were no unregistered sales of equity securities to report for the period[219](index=219&type=chunk) [Exhibits](index=39&type=section&id=Item%206.%20Exhibits) This section lists key agreements related to the July 2023 financing and other required filings - Key exhibits filed include agreements for the July 2023 financing and an amendment to the license agreement with Factor Bioscience Limited[220](index=220&type=chunk)

Financing Activities - The company has entered into a private placement of $8.7 million in convertible notes, with an interest rate of 6% per annum, maturing on July 14, 2028[129][130]. - The company has entered into a Standby Equity Purchase Agreement allowing for the sale of up to $10 million in common stock, with gross proceeds of approximately $0.3 million recognized to date[140]. - The company closed a Private Placement of $8.7 million in aggregate principal amount of Notes, intending to use the net proceeds for general working capital purposes[169]. - Net cash provided by financing activities for the six months ended June 30, 2023, included $0.3 million of gross proceeds received under the SEPA with Lincoln Park[181]. Revenue Generation - The company has not generated any revenues from product sales to date, as it is still in the pre-clinical stage[142]. - A cell line customization and license agreement with Lineage Cell Therapeutics may lead to additional revenues, although no assurances can be made[142]. - The company received a $0.3 million upfront, nonrefundable payment under the Lineage Agreement for an option right to obtain a sublicense of intellectual property, with potential future payments based on customer requests[165]. Research and Development - Research and development expenses are recognized as incurred, with major components including preclinical study costs and clinical trial expenses[144][145]. - The company acquired intellectual property assets from Exacis Biotherapeutics, enhancing its mRNA technology platform[138]. - The mRNA technology platform includes over 100 patents, focusing on mRNA cell reprogramming and gene editing technologies[121][122]. - The company anticipates developing product candidates for neurological indications and acute myeloid leukemia using its mRNA technology platform[122]. - The company expensed approximately $0.5 million for the Purchased License acquired in the Exacis Acquisition during the three and six months ended June 30, 2023[155]. Operating Expenses - Total operating expenses for the three months ended June 30, 2023, were $4.549 million, a decrease of $9.331 million compared to $13.880 million in the same period of 2022[149]. - Research and development expenses decreased to $1.579 million for the three months ended June 30, 2023, down from $1.685 million in 2022, reflecting a reduction of $106,000[152]. - General and administrative expenses significantly decreased to $2.510 million for the three months ended June 30, 2023, compared to $6.205 million in 2022, a reduction of $3.695 million[153]. Financial Performance - The net loss for the three months ended June 30, 2023, was $4.508 million, compared to a net loss of $3.398 million in the same period of 2022, an increase in loss of $1.110 million[149]. - Cash, cash equivalents, and restricted cash totaled approximately $5.9 million as of June 30, 2023, with $4.1 million classified as restricted cash[163]. - The change in fair value of warrant liabilities resulted in a credit of $0.191 million for the three months ended June 30, 2023, compared to a credit of $10.792 million in the same period of 2022[157]. - The company recognized a contingent consideration liability of $0.2 million for future payments related to the Exacis Acquisition, with a credit of $0.1 million recognized for the change in fair value as of June 30, 2023[158]. - Loss on non-controlling investment in NoveCite was approximately $8,000 for the three months ended June 30, 2023, compared to $296,000 in the same period of 2022[159]. Future Outlook - The company expects to continue incurring operating losses as it develops product programs and operates as a publicly traded company[175]. - Future funding requirements will depend on various factors, including the cost of regulatory approvals and the scope of clinical trials[176]. - The company may need to raise substantial additional funds to pursue product development, which could result in dilution to stockholders[176]. - The increase in cash used in operating activities was primarily driven by MSA fees and accrued severance payments for the six months ended June 30, 2023[179]. Lease Agreement - The company entered into a sublease agreement for approximately 45,500 square feet of office and laboratory space in Somerville, Massachusetts, with a total base rental payment of approximately $63.0 million over a 10-year term[164]. Licensing Agreements - An amendment to the Exclusive Factor License Agreement has expanded the field of use to include veterinary applications and reduced sublicense fees from 30% to 20%[136].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _____________ Commission file number: 001-11460 Eterna Therapeutics Inc. (Exact name of registrant as specified in its charter) Delaware 31-11034 ...

TABLE OF CONTENTS As filed with the Securities and Exchange Commission on April 14, 2023 Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 ETERNA THERAPEUTICS INC. (Exact name of registrant as specified in its charter) | Delaware | 2836 | 31-1103425 | | --- | --- | --- | | (State or other jurisdiction of incorporation | (Primary Standard Industrial Classification | (I.R.S. Employer | | or organizatio ...

Intellectual Property and Patents - Eterna Therapeutics has in-licensed a portfolio of over 100 patents covering key mRNA cell engineering technologies, including mRNA cell reprogramming and gene editing [20]. - The company is substantially dependent on intellectual property licensed from Factor Limited, which is critical for its strategic partnerships and product development [19]. - As of March 20, 2023, the company had in-licensed approximately 12 patent families, including 125 granted patents and 64 published patent applications related to its mRNA technology platform [56]. - The company owns or controls approximately 10 patent families related to IRX-2, including 94 granted patents, 12 pending, and 6 published applications [62]. - The Exclusive Factor License Agreement allows the company to receive sublicensing fees, with a 20% royalty on fees received before the initial expiration date and 30% during the renewal term [47]. - The term of the Exclusive Factor License Agreement expires on November 22, 2027, with an automatic extension if the company receives at least $100 million in sublicensing fees [47]. - The anticipated expiration dates for various patents indicate a long-term strategy for maintaining competitive advantages in the market [62]. - The company is actively pursuing patent applications in multiple jurisdictions, including pending applications in Australia and Europe for several technologies [62]. - The patent protection strategy includes jurisdictions such as the United States, Europe, Australia, and Japan, ensuring broad coverage for its technologies [62]. - The company has published patents related to nucleic acid-based therapeutics, cationic lipids, and transfection methods, indicating ongoing innovation in these areas [63]. - The company has a total of 10 patent families related to IRX-2, with 94 granted patents and 12 pending applications, focusing on novel compounds and methods of treatment [62]. - The anticipated expiration date for key patents related to IRX-2 is April 14, 2029, for the modified manufacturing process and December 30, 2030, for methods of reversing immune suppression [62]. - The patent family FAB-012 focuses on cationic lipids and transfection methods, which are crucial for delivering nucleic acids to cells [63]. - The company has published applications for various patent families, including FAB-011 and FAB-018, which relate to nucleic acid-based therapeutics and circular RNA, respectively [63][64]. - The company is exploring vaccine immunotherapy for advanced prostate cancer, utilizing a cytokine mixture to enhance immune responses [72]. - The company has three in-licensed patents currently under re-examination by the USPTO, which may impact its ability to protect intellectual property rights [71]. Business Operations and Financial Outlook - Eterna expects to incur significant losses for the foreseeable future and may never achieve or maintain profitability [19]. - The company plans to develop therapeutic products through strategic partnerships, focusing on hypoimmune induced pluripotent stem cell-derived candidates for neurological indications [30]. - Eterna's operations may be adversely affected by risks such as reliance on intellectual property and potential business disruptions from events like the COVID-19 pandemic [19]. - The company anticipates that initial product candidates will include immune-modulating cells for indications such as acute myeloid leukemia and solid tumors [30]. - Eterna is focusing on immunotherapy methods to increase immunological effects in patients, including treatments for tumors and immune prophylaxis [72]. - The company must ensure that all marketing claims comply with FDA-approved labeling, as violations can lead to enforcement actions [95]. - The pricing of pharmaceutical products is under scrutiny, and potential legislative changes could significantly affect the company's business prospects [108]. - The company may need to conduct pharmacoeconomic studies to demonstrate the value of its products to third-party payors, which is increasingly important for reimbursement [104]. - Eterna expects to incur significant losses for the foreseeable future and may require substantial additional capital to fund operations [19]. Regulatory Environment - Regulatory approval processes for drugs and biologics require substantial time and financial resources, with strict compliance to FDA regulations necessary for market entry [77]. - The FDA's approval process for drugs and biologics involves multiple stages, including pre-clinical tests and human clinical trials, which are time-consuming and uncertain [78]. - The FDA may impose clinical holds on trials if safety concerns arise, which could delay the development process [86]. - The FDA approval process for a marketing application (MA) can take years and involves a comprehensive review of clinical studies, manufacturing processes, and compliance with cGMP requirements [89]. - For FDA fiscal year 2023, the application fee for a marketing application may exceed $3 million, with an annual program fee of $393,933 per prescription drug product [90]. - The FDA may require additional testing or information before accepting a marketing application for filing, which can delay the approval process [90]. - Post-approval, the FDA mandates ongoing compliance with cGMP and may require Phase 4 clinical trials to monitor the long-term effects of approved products [100]. - The FDA may grant orphan drug designation to drugs intended for rare diseases affecting fewer than 200,000 individuals in the U.S., providing a seven-year exclusivity period upon first approval [111]. - The European Orphan Drug Regulation offers ten years of marketing exclusivity for drugs treating conditions affecting five or fewer per 10,000 people in the EU, with a potential two-year extension [113]. - Under the fast-track program, the FDA must determine if a product qualifies for fast-track designation within 60 days of the request, facilitating expedited review for serious conditions [114]. - Products approved under accelerated approval regulations must provide meaningful therapeutic benefits over existing treatments, based on surrogate endpoints [116]. - The FDA's priority review process allows for a six to eight-month review timeframe for products addressing serious conditions or unmet medical needs [119]. - The company is subject to foreign regulatory requirements, which vary widely and must be satisfied before marketing products in those countries [101]. Research and Development - Eterna's mRNA delivery technology aims to enhance the uptake of nucleic acids by cells, addressing limitations of conventional delivery systems [32]. - The company is in the pre-clinical testing stage for its pharmaceutical candidates, which includes laboratory evaluations and animal studies before human clinical trials can commence [80]. - Human clinical trials are conducted in three phases, with Phase 1 focusing on safety and Phase 2 evaluating efficacy for specific indications [84]. - The company is developing methods to reverse immune suppression in patients, focusing on restoring T-cell immunity and promoting dendritic cell maturation [72]. - The company is exploring engineered immune cell therapies and mesenchymal stem cell therapies as part of its innovative product development strategy [63]. - The company has developed a modified manufacturing process for primary cell-derived biologics, which includes multiple steps such as removing contaminating cells and producing a clarified supernatant [67]. - A method for treating human papillomavirus (HPV) has been established, which involves administering a primary cell-derived biologic to induce an immune response against HPV [67]. - The company is focusing on mRNA gene-editing technology, which aims to improve efficiency and reduce unwanted mutagenesis compared to conventional methods [34]. - The mRNA gene-editing technology aims to delete, insert, and repair DNA sequences, potentially enabling new therapeutic approaches for cancer treatment [34]. - The company anticipates that its mRNA gene-editing technology can generate allogeneic CAR-T therapies, which may prevent graft-versus-host disease (GvHD) by inactivating the endogenous T-cell receptor [34]. Corporate Structure and Changes - The company completed the acquisition of Novellus, Inc. on July 16, 2021, which focuses on developing engineered mesenchymal stem cell therapies using mRNA-based technologies [22]. - A cell line customization and license agreement was entered into with Lineage Cell Therapeutics, which includes a $250,000 non-refundable upfront payment and potential future royalty payments [24]. - The company executed a reverse stock split at a ratio of 1-for-20 on October 16, 2022, affecting the number of shares but not ownership percentages [27]. - Eterna's common stock was transferred from The Nasdaq Global Market to the Nasdaq Capital Market on January 11, 2023, trading under the symbol "ERNA" [26]. - The company changed its name from Brooklyn ImmunoTherapeutics, Inc. to Eterna Therapeutics Inc. on October 17, 2022, and its stock symbol from "BTX" to "ERNA" [26]. - As of March 20, 2023, the company had nine full-time employees, including four in research and development and five in administrative roles [134]. - The company faces competition from major pharmaceutical firms with greater resources, which may impact its ability to secure patent protection and market approval [131]. - The company emphasizes the importance of ethical standards and integrity in its business conduct [138]. - The company is committed to employee well-being, offering competitive compensation and benefits [137].