8-K
Ernexa Therapeutics Inc. (ERNA)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): August 2, 2023
Eterna Therapeutics Inc.
(Exact Name of Registrant as Specified in its Charter)
| Delaware | 001-11460 | 31-1103425 |
|---|---|---|
| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (IRS Employer Identification No.) |
| 10355 Science Center Drive, Suite 150 | ||
| --- | --- | |
| San Diego, CA | 92121 | |
| (Address of Principal Executive Offices) | (Zip Code) |
Registrant’s telephone number, including area code: (212) 582-1199
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ☐ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
|---|---|
| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| --- | --- |
| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| --- | --- |
| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
| --- | --- |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading symbol | Name of each exchange on which registered |
|---|---|---|
| Common Stock, par value $0.005 per share | ERNA | The Nasdaq Stock Market LLC |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 or Rule 12b-2 of the Securities Exchange Act of 1934:
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 7.01 | Regulation FD Disclosure. |
|---|
On August 2, 2023, Eterna Therapeutics Inc., a Delaware corporation (the “Company”, “we”, “us”, or “our”), made available a new presentation for investors. A copy of this presentation is attached hereto as Exhibit 99.1 and incorporated by reference in this Item 7.01. A copy of the presentation is also available on our website at https://investor.eternatx.com.
Use of our Website to Distribute Material Company Information
We use our website as a channel of distribution for important Company information. We routinely post on our website
important information, including press releases, investor presentations and financial information, which may be accessed by clicking on the “Investor Relations” section of www.eternatx.com. We may also use our website to expedite public
access to time-critical information regarding our Company in advance of or in lieu of distributing a press release or a filing with the Securities and Exchange Commission \(the “SEC”\)
disclosing the same information. Therefore, investors should look to the “Investor Relations” section of our website for important and time-critical information. Visitors to our website can also register to receive automatic e-mail and other
notifications alerting them when certain new information is made available on our website. Information contained on, or accessible through, our website is not a part of and is not incorporated by reference in this Current Report on Form 8-K
or in any of our other filings with the SEC.
The information contained in this Item 7.01, including in Exhibit 99.1 attached hereto, is “furnished” and not “filed” for purposes of Section 18 of the Securities and Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section. Such information shall not be incorporated by reference in another filing under the Exchange Act or the Securities Act of 1933, as amended, except to the extent such other filing specifically incorporates such information by reference.
| Item 9.01 | Financial Statements and Exhibits. |
|---|
(d) Exhibits.
| Exhibit<br><br> Number | Description |
|---|---|
| 99.1 | Eterna Therapeutics Investor Presentation – August 2023. |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, hereunto duly authorized.
| Eterna Therapeutics Inc. | ||
|---|---|---|
| Dated: August 2, 2023 | By: | /s/ Matthew Angel |
| Matthew Angel | ||
| Chief Executive Officer and President |
Exhibit 99.1
Eterna TherapeuticsInvestor Presentation August 2023 1 © Eterna Therapeutics Inc.
This presentation (this “Presentation”) and any oral statements made in connection with this Presentation are for informational purposes only and do not constitute an offer to sell, a solicitation of an offer to buy, or a recommendation to purchase any equity, debt or other securities of Eterna Therapeutics Inc., a Delaware corporation (including its consolidated subsidiaries and affiliates, the “Company”). The information contained herein does not purport to be all inclusive. The data contained herein is derived from various internal and external sources believed to be reliable, but there can be no assurance as to the accuracy or completeness of such information. Any data on past performance contained herein is not an indication as to future performance. Except as required by applicable law, the Company assumes no obligation to update the information in this Presentation. Nothing herein shall be deemed to constitute investment, legal, tax, financial, accounting or other advice. The communication of this Presentation is restricted by law, and it is not intended for distribution to, or use by any person in, any jurisdiction where such distribution or use would be contrary to local law or regulation. Forward-Looking Statements This Presentation includes certain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements may address, among other things, the Company’s expected plans and prospects, including without limitation, the Company’s views with respect to the potential for mRNA Cell Engineering, its expectations with respect to timing of regulatory filings and the reporting of initial data from any clinical trial(s), the potential therapeutic opportunities for mRNA Cell Engineering, its expectations regarding its product strategies, and its plans regarding commercialization of mRNA Cell Engineering. These forward-looking statements are distinguished by use of words such as “anticipate,” “aim,” “believe,” “continue,” “can,” “could,” “designed to,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “will,” “would” and the negative of these terms, and similar references to future periods. These statements are based on management’s current expectations and are subject to uncertainty and changes in circumstances. Actual results may differ materially from these expectations due to, among other things, the Company’s ability to discover and develop novel drug candidates and delivery approaches, the Company’s ability to successfully demonstrate the efficacy and safety of its drug candidates, the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates, the actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, the Company’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, obtaining regulatory approval for products, competition from others using technology similar to the Company’s and others developing products for similar uses, the Company’s ability to manage operating expenses, the Company’s ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, the Company’s dependence on third parties for development, manufacture, marketing, sales and distribution of products, the outcome of litigation, and unexpected expenditures. Detailed information about these factors and additional important factors can be found in the documents that the Company files with the Securities and Exchange Commission, such as Form 10-K, Form 10-Q and Form 8-K. Forward-looking statements speak only as of the date the statements were made. The Company does not undertake an obligation to update forward-looking information, except to the extent required by applicable law. Legal Notices 2 © Eterna Therapeutics Inc.
Investment Highlights 3 © Eterna Therapeutics Inc. Novel technologies primed to generate value through collaborations and partnerships, and compete in growing, multi-billion dollar markets Gene editing and mRNA delivery technology instrumental for R&D in cell engineering Technology is protected by a diligently prosecuted patent portfolio with over 130 granted patents Corporate strategy designed to maximize value of technologies and runway, while ensuring continued progress for key activities
Primed to Compete in Growing, Multi-billion $ Markets Eterna’s technology is designed to potentially help solve a variety of industry problems, including efficient editing and delivery *Verified Market Research;**Nova1Advisor CAGR 4.7% CAGR 16.52%
5 © Eterna Therapeutics Inc. Each platform is supported by a significant intellectual property estate with patents designed to both support and foster candidate development and trademarks designed to provide additional brand exclusivity and recognition The Eterna Platform RNA ToRNAdo™ RNA Delivery Gene Editing Platforms Technology RNAGene Editing RNA Cell Reprogramming Applications Library of Ionizable Lipids Eterna: An Innovative RNA Cell Engineering Company
Substantial Shortcomings Remain with Industry Standard Gene Editing Technologies 6 © Eterna Therapeutics Inc. Other Technologies in Use Key Shortcomings TALENs CRISPR Can be complex or can require expensive customization Immunogenic Potential cause of autoimmune diseases Potential for undesired mutations (“off-target effects”) Potential for undesired genome disruption Delivery challenges
7 © Eterna Therapeutics Inc. Eterna’s platform is novel and differentiated with the potential to overcome the challenges of industry standard cell engineering technologies The Eterna Platform RNA ToRNAdo™ RNA Delivery Gene Editing Platforms Technology Library of Ionizable Lipids Potential Advantages Immunogenicity not expected Issues with off-target effects not expected Issues with genomic disruption not expected Efficient delivery Potential Advantages of Eterna’s Novel RNA Technologies with Gene Editing and Cell Reprogramming Applications
8 © Eterna Therapeutics Inc Potential for high-value therapeutics CORE POTENTIAL MODALITIES POTENTIAL THERAPEUTICS Gene editing Blood cancers Gene replacement Solid tumors Cell replacement Ophthalmology, cardiology, neurology, etc. Reprogramming, Gene Editing, Delivery Cell Therapy Oncology Pipeline ISO Class 7 Cleanroom Facility RNA Research Lab, Equipment, and Staff Foundational Patent Portfolio Partnership Strategy Offers Multiple Potential Paths to Revenue Upfront license payments, milestones & royalties Partner-funded product co-development Partner-funded joint ventures
RiboSlice™: Non-Viral, DNA-Free Targeted Gene Editing 9 © Eterna Therapeutics Inc. RiboSlice™: mRNA construct that encodes gene editing proteins mRNA transiently expresses high levels of gene editing proteins within cells Does not rely on viruses or DNA-based vectors Potential to enable efficient and effective single or multiplexed editing Potential RiboSlice™ Application: Allogeneic CAR-T Example application: Allogeneic CAR-T RiboSlice™ used to knock out endogenous TCR to prevent therapeutic T cells from causing graft-versus-host disease (GvHD) RiboSlice™ mRNA can be delivered via ToRNAdo™ or electroporation CAR can be inserted using RiboSlice™ or traditional lentiviral approaches High-efficiency gene editing of TRAC and PD1 in human cells Eterna Gene-Editing Technologies
NoveSlice™: high specificity gene-editing endonuclease Exhibits high efficiency on-target cutting and enhanced sensitivity to the chromatin context of the target site Designed with an array of DNA-binding repeat sequences connected by flexible linkers Designed to target cutting activity to actively expressed genes, with the potential to: reduce off-target effects minimize cellular toxicity enable enhanced safety for future potential therapeutics High-efficiency gene editing of the AAVS1 genomic safe harbor locus in human iPSCs © Eterna Therapeutics Inc. 10 NoveSlice™: Chromatin Context Sensitive Gene-Editing Endonuclease Eterna Gene-Editing Technologies
The Eterna Difference: Eterna’s LNP Delivery is Customizable Through Its Library of Novel Ionizable Lipids 11 © Eterna Therapeutics Inc. Designed to effectively transfect cells Customizable constructs allow Eterna to vary the lipid head group, tail, and spacer link to fine tune the lipid transfection ability Exploring tropism for traditionally difficult to target tissues: heart, solid tumors, spleen, kidney Lipid Technology
Ionizable Lipids Have Demonstrated Robust Transfection 12 © Eterna Therapeutics Inc. Transfection efficiency may be increased by fine tuning with only a few highly targeted edits to the structure Eterna LNPs and lipoplexes exhibited greater in vitro transfection efficiency of THP-1 monocytes than commercially-available controls Transfection of Jurkat T cells Lipoplex Screen in THP-1 Monocytes Jurkat T Cell Transfection Lipid Technology
Eterna’s mRNA Delivery Capabilities to Target the Lungs 13 © Eterna Therapeutics Inc. Transgene expression observed as early as 4 h after transfection in BEAS2B cells treated with both nebulized and non-nebulized complexes and was maintained to at least 48 h(Figs. A–E) Fluorescent images of nebulized formulated mRNA 48 h after administration to the rat lung showed a clear pattern of GFP fluorescence in epithelial cells (Fig. D) and quantitative assessment showed GFP at a concentration of 35,000 pg/g of homogenized lung tissue Robust expression even at 48 h (Fig. E) Eterna’s semisynthetic mRNA technology to reduce ARDS severity Aerosolized Pulmonary Delivery of mRNA Constructs Attenuates Severity of Escherichia coli Pneumonia in the Rat, Sean D. McCarthy, Christopher B. Rohde, Matt Angel, Claire H. Masterson, Ronan MacLoughlin, Juan Fandiño, Héctor E. González, Declan Byrnes, John G. Laffey, and Daniel O'Toole. Nucleic Acid Therapeutics 2023 33:2, 148-158 Lipid Technology
Eterna’s mRNA Delivery Capabilities to Target the Lungs 14 © Eterna Therapeutics Inc. IkBα and SOD3 expression significantly increased in vitro and in vivo in pulmonary cells administered respective complexes compared to those receiving scrambled mRNA control A B C D Aerosolized Pulmonary Delivery of mRNA Constructs Attenuates Severity of Escherichia coli Pneumonia in the Rat, Sean D. McCarthy, Christopher B. Rohde, Matt Angel, Claire H. Masterson, Ronan MacLoughlin, Juan Fandiño, Héctor E. González, Declan Byrnes, John G. Laffey, and Daniel O'Toole. Nucleic Acid Therapeutics 2023 33:2, 148-158 Lipid Technology
Platform to Address Challenges of Cell Reprogramming 15 © Eterna Therapeutics Inc. Allogeneic “off-the-shelf” cell therapies can be a very powerful tool in treating a variety of diseases Eterna’s technology aims to address the challenges facing allogeneic “off-the-shelf” cell therapies including increasing the expansion potential and reducing the in vivo persistence by minimizing host immune rejection Cell Reprogramming Challenges and Eterna Approach Cell Reprogramming
16 © Eterna Therapeutics Inc. iPSC Markers for Confirmation Clonal iPSC Lines Generated in 2 Weeks mRNA Transfections with Eterna Technology Verified iPSC Differentiation Potential Mesenchymal Stem Cells Adipocytes Oil Red O stain Chondrocytes Alcian Blue stain Osteoblasts Alizarin Red S stain Functional Cardiomyocytes Neurons β-tubulin Retinal Pigment Epithelial Cells Natural Killer Cells Macrophages Eterna Technology Efficiently Reprograms Cells with mRNA to iPSCs that can Differentiate into Multiple Tissues and Cell Types Cell Reprogramming
Combined mRNA Gene Editing and Cell Reprogramming Creation of pluripotent stem cell lines containing defined deletions in the CCR5 gene Demonstrated gene editing and dedifferentiation of human somatic cells in preclinical studies Successful demonstration of capability to generate clonal engineered pluripotent stem cell lines 17 © Eterna Therapeutics Inc. Cell Reprogramming
Summary Highlights 18 © Eterna Therapeutics Inc. Novel technologies primed to generate value through collaborations and partnerships, and compete in growing, multi-billion dollar markets Partnership strategy is well positioned to maximize several potential revenue streams including upfront payments, milestones, royalties, and partner funded co-development and joint ventures Gene editing and mRNA delivery technology instrumental for R&D in cell engineering Technology is protected by a diligently prosecuted patent portfolio with over 130 granted patents Corporate strategy designed to maximize value of technologies and runway, while ensuring continued progress for key activities