Eterna Therapeutics (US:ERNA)2023-03-19 16:00Intellectual 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].