Cornerstone Pharmaceuticals, Inc.

Cyclo Therapeutics’ TransportNPC™ Phase 3 clinical trial for Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1, a rare and fatal genetic disease, is fully enrolled and results from the 48-week interim analysis are expected in the middle of 2025. NEWARK, N.J. and GAINESVILLE, Fla., Aug. 22, 2024 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL), and Cyclo Therapeutics, Inc. (Nasdaq: CYTH) today announced that they have entered into a definitive merger agreement to combine the two companies to focus on the development of Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1. On consummation of the merger, Rafael Holdings will issue shares of its Class B common stock to Cyclo Therapeutics’ shareholders, based on an exchange ratio valuing Cyclo Therapeutics shares at $.95 per share and Rafael Holdings at its cash value combined with the value of its marketable securities and certain other investments less certain current liabilities. In addition, the cash value will take into account the funding of Cyclo’s operations by Rafael with convertible notes through closing. Following the closing, Rafael Holdings intends to fund the TransportNPC™ clinical trial to its 48-week interim analysis. The boards of directors of Rafael Holdings and Cyclo Therapeutics have approved this transaction and expect it to close in late 2024, pending approval of the companies’ shareholders, the effectiveness of a registration statement to register the shares of Class B common stock of Rafael Holdings to be issued in the transaction and other customary closing conditions. Rafael Holdings made its first strategic investment in Cyclo Therapeutics in March 2023 to help drive treatment innovation for patients with the debilitating diagnosis of Niemann-Pick Disease Type C1. Rafael Holdings led another financing round in the fall of 2023 and has continued to support Cyclo Therapeutics via convertible debt financings in 2024. “The proposed merger with Cyclo Therapeutics is a major step forward in our strategy to invest in, develop and commercialize clinical stage assets in areas of high unmet medical need,” said Bill Conkling, President and CEO of Rafael Holdings. Bill added, “Cyclo Therapeutics continues to make substantial progress in advancing its lead asset, Trappsol® Cyclo™, announcing the completion of enrollment in its pivotal TransportNPC™ Phase 3 clinical study for the treatment of Niemann-Pick Disease Type C1 at the end of May 2024. We are impressed with the execution by the Cyclo Therapeutics team in fully enrolling a comprehensive clinical trial in NPC and we eagerly await the 48-week interim analysis in the middle of 2025. Rafael Holdings is excited to join forces with Cyclo Therapeutics to make Trappsol® Cyclo™ our lead clinical program. We are committed to the program and will leverage our resources to help bring this much needed treatment option to NPC patients.” N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, commented, “Our partnership with Rafael Holdings during the last year and a half has enabled Cyclo to get to where we are today. We are extremely pleased to announce our merger agreement with Rafael Holdings and believe that the strength of Rafael’s balance sheet and its strong management team will solidify our commitment to deliver the results of the TransportNPC™ trial for our shareholders and patients.” Cassel Salpeter & Co. is acting as financial advisor to Cyclo Therapeutics in connection with the transaction. Schwell Wimpfheimer & Associates is serving as legal advisor to Rafael Holdings and Fox Rothschild LLP is serving as legal advisor to Cyclo Therapeutics. About Rafael Holdings, Inc. Rafael Holdings is a holding company with interests in clinical and early-stage pharmaceutical companies including an investment and planned merger with Cyclo Therapeutics, Inc. (Nasdaq: CYTH), a clinical-stage biotechnology company dedicated to developing life-changing medicines for patients and families living with challenging diseases through its lead therapeutic asset, Trappsol® Cyclo™, a majority investment in Cornerstone Pharmaceuticals, Inc., formerly known as Rafael Pharmaceuticals Inc., a cancer metabolism-based therapeutics company, a majority equity interest in LipoMedix Pharmaceuticals Ltd., a clinical stage pharmaceutical company, the Barer Institute Inc., a wholly-owned preclinical cancer metabolism research operation, a majority investment in Day Three Labs, Inc., a company which reimagines existing cannabis offerings with pharmaceutical-grade technology and innovation like Unlokt™ to bring to market better, cleaner, more precise and predictable products in the cannabis industry, and a majority interest in Rafael Medical Devices, LLC, an orthopedic-focused medical device company developing instruments to advance minimally invasive surgeries. Rafael’s primary goal has been to expand its investment portfolio through opportunistic and strategic investments including therapeutics which address high unmet medical needs. Upon closing of the merger transaction with Cyclo Therapeutics, Rafael intends to focus its efforts on making Trappsol® Cyclo™ its lead clinical program. About Cyclo Therapeutics, Inc. Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with disease. The Company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is the subject of four formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (www.ClinicalTrials.gov NCT02939547, NCT02912793, NCT03893071 and NCT04860960). The Company is conducting a Phase 2b clinical trial using Trappsol® Cyclo™ intravenously in early Alzheimer’s disease (NCT05607615) based on encouraging data from an Expanded Access program for Alzheimer’s disease (NCT03624842). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the Company’s website: www.cyclotherapeutics.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans regarding the further evaluation of clinical data in the success of the potential combination with Cyclo Therapeutics; and the potential of our pipeline. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements Among other things, these risks include that there can be no guarantee that the proposed business combination will be completed in the anticipated timeframe or that the conditions required to complete the proposed combination will be met. In addition, these risks include, but are not limited to, those disclosed under the caption “Risk Factors” in Rafael’s Annual Report on Form 10-K for the year ended July 31, 2023, and Cyclo’s Annual Report on Form 10-K for the year ended December 31, 2023 and the companies’ other filings with the SEC. All these factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent the companies’ management’s estimates as of the date of this press release. While they may elect to update such forward-looking statements at some point in the future, the companies disclaim any obligation to do so, even if subsequent events cause views to change. No Offer or Solicitation This press release is not a proxy statement or solicitation of a proxy, consent or authorization with respect to any securities or in respect of the proposed business combination and shall not constitute an offer to sell or a solicitation of an offer to buy any securities, nor shall there be any sale of securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. No offer of securities shall be made except by means of a prospectus meeting the requirements of the Securities Act. Important Additional Information Will Be Filed with the SEC Rafael Holdings plans to file with the SEC a Registration Statement on Form S-4 in connection with the transactions and both Rafael Holdings and Cyclo Therapeutics plan to file with the SEC and mail to their respective stockholders a Joint Proxy Statement/Prospectus in connection with the transactions. Investors and security holders are urged to read the Registration Statement and the Joint Proxy Statement/Prospectus and other relevant documents filed with the SEC in connection with the proposed transaction or incorporated by reference into the Joint Proxy Statement/Prospectus (if any) carefully when they are available before making any voting or investment decision with respect to the proposed transactions. The Registration Statement, the Joint Proxy Statement/Prospectus and other documents filed with the SEC in connection with the proposed transaction or incorporated by reference into the Joint Proxy Statement/Prospectus (if any) will contain important information about Rafael Holdings, Cyclo Therapeutics, the transactions and related matters. Investors and security holders will be able to obtain free copies of the Registration Statement and the Joint Proxy Statement/Prospectus and other documents filed with the SEC by Rafael Holdings and Cyclo Therapeutics through the web site maintained by the SEC at www.sec.gov. In addition, investors and security holders will be able to obtain free copies of the Registration Statement and the Joint Proxy Statement/Prospectus from Rafael Holdings by contacting david.polinsky@rafaelholdings.com or Cyclo Therapeutics by contacting cyth@jtcir.com. Participants in the Solicitation Rafael and Cyclo and their respective directors and executive officers may be deemed to be participants in the solicitation of proxies in respect of the transactions contemplated by the merger agreement. Information regarding Rafael’s directors and executive officers is contained in Rafael proxy statement dated November 20, 2023, which is filed with the SEC. Information regarding Cyclo’s directors and executive officers is contained in Cyclo’s Annual Report on Form 10-K for the year ended December 31, 2023, which is filed with the SEC. Additional information regarding the persons who may be deemed participants in the proxy solicitation and a description of their direct and indirect interests in the proposed business combination will be available in the Registration Statement and the Joint Proxy Statement / Prospectus.

CAMBRIDGE, Mass., Dec. 20, 2023 (GLOBE NEWSWIRE) -- Eterna Therapeutics Inc. (Nasdaq: ERNA) (“Eterna” or the “Company”), a life science company committed to realizing the potential of mRNA cell engineering to provide patients with transformational new medicines, has appointed Sanjeev Luther as President and Chief Executive Officer and a member of the company's Board of Directors, effective January 1, 2024. Mr. Luther will replace Matt Angel, Ph.D., the current President and Chief Executive Officer. James Bristol, Ph.D., Chairman of Eterna’s Board of Directors, said, “We are excited to have Sanjeev take on the role of President and CEO of Eterna as we continue our work to support the development of next-generation therapies using mRNA cell engineering. Sanjeev has an impressive track record in leadership, strategic planning, and business development across various global organizations in the pharmaceutical and healthcare sectors. The Board looks forward to working with Sanjeev as Eterna continues to capitalize on its in-licensed portfolio of patented mRNA cell engineering technologies.” Mr. Luther stated, “It’s an honor to join Eterna’s team at this exciting time. I look forward to working closely with Eterna's scientists, management team, and Board to deliver sustained value to our shareholders and to further the reach and promise of mRNA cell engineering.” Mr. Luther is a seasoned pharmaceutical executive having spent over 30 years in leadership roles across public and private biotechnology and global pharmaceutical companies. Most recently, he served as President, CEO, and Board Member of Cornerstone Pharmaceuticals, where he oversaw the evolution of the company’s pipeline for rare cancers and secured strategic partnership, in-licensing, and out-licensing agreements. At Cornerstone, he also held the roles of Chief Operations Officer and Chief Business Officer. Prior to Cornerstone, Mr. Luther led business-building and fundraising strategies for Fortune 500 corporations and start-ups in pharmaceuticals, biotech, medical devices, and diagnostics, including Bristol-Myers Squibb, Novartis, Bausch and Lomb, and GE Healthcare. He has experience across a range of therapeutic areas and stages, from R&D through commercialization. Mr. Luther holds an MBA in Marketing and a B.S. in Marketing/Business Administration from the State University of New York at Buffalo. About Eterna Therapeutics Inc.Eterna Therapeutics is a life science company committed to realizing the potential of mRNA cell engineering to provide patients with transformational new medicines. Eterna has in-licensed a portfolio of over 130 patents covering key mRNA cell engineering technologies, including technologies for mRNA cell reprogramming, mRNA gene editing, the NoveSlice™ and UltraSlice™ gene-editing proteins, and the ToRNAdo™ mRNA delivery system from Factor Bioscience. NoveSlice™, UltraSlice™, and ToRNAdo™ are trademarks of Factor Bioscience. For more information, please visit www.eternatx.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are intended to be covered by the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are any statements that are not statements of historical fact and may be identified by terminology such as “believe,” “could,” “estimate,” “anticipate,” “expect,” “plan,” “possible,” “potential,” “project,” “will” or other similar words and the negatives of such words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those stated or implied in any forward-looking statement as a result of various factors, including, but not limited to, uncertainties related to: (i) the evolution of Eterna’s business model into a platform company focused on mRNA, induced pluripotent stem (iPS) cell and gene editing technologies; (ii) Eterna’s ability to successfully, cost-effectively and efficiently develop its technology and products; (iii) Eterna’s ability to successfully commence clinical trials of any products on a timely basis or at all; (iv) Eterna’s ability to successfully fund and manage the growth of its development activities; and (v) Eterna’s ability to obtain regulatory approvals of its products for commercialization. You should not rely upon forward-looking statements as predictions of future events. The forward-looking statements made in this communication speak only as of the date on which they were made, and Eterna does not undertake any obligation to update the forward-looking statements contained herein to reflect events that occur or circumstances that exist after the date hereof, except as required by applicable law. Factors that may cause Eterna’s actual results to differ from those expressed or implied in forward-looking statements contained in this press release are more fully disclosed in Eterna’s periodic public filings with the U.S. Securities and Exchange Commission, particularly under the heading “Risk Factors” in Eterna’s Annual Report on Form 10-K for the year ended December 31, 2022, as well as under similar headings in Eterna’s subsequently filed Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Investor Relations Contact:investors@eternatx.com Media Contact:EternaPR@westwicke.com

[09-October-2023] NEW YORK, Oct. 9, 2023 /PRNewswire/ -- A new combination of treatments safely decreased growth of pancreatic cancer in mice by preventing cancer cells from scavenging for fuel, a new study finds. Led by researchers at NYU Grossman School of Medicine, its Department of Radiation Oncology, and the Perlmutter Cancer Center, the work builds on prior discoveries at NYU Langone that revealed how pancreatic cancer cells, to avert starvation and keep growing, find alternate fuel sources. Normally supplied by the bloodstream, oxygen, blood sugar, and other resources become scarce as the increasing density of fast-growing pancreatic tumors cuts off their own blood supply. In this environment, the ability to switch fuels contributes to the deadliness of pancreatic cancers. Published online October 9 in Nature Cancer, the new study involves a drug designed to prevent pancreatic ductal adenocarcinoma (PDAC) cells from one such switch. PDAC cells use the enzyme glutaminase to convert the amino acid glutamate into glutamine, a form that can be burned for fuel to sustain rapid tumor growth. Drugs designed to block glutaminase, however, have been shown to cause cancer cells to switch to still other scavenging pathways. For this reason, the field next looked at experimental treatments like DRP-104, designed by Dracen Pharmaceuticals, a new "prodrug" form of the compound 6-Diazo-5-oxo-L-norleucine (DON) that is preferentially activated within tumors to overcome toxicity issues seen with DON. DON was designed to starve cancer cells by mimicking glutamine, which unlike glutaminase blockers, broadly inhibits all metabolic pathways that use glutamine. Currently in clinical trials against non-small cell lung cancer, DRP-104 cannot be burned as fuel, but clings to the same enzymes as glutamine. In the current study, DRP-104 treatment alone decreased PDAC growth in mouse models of pancreatic cancer. Importantly, the current team also found that PDAC cells pushed into metabolic crisis by DRP-104 increase signaling through a protein called extracellular signal-regulated kinase or ERK, to make up for their loss of glutamine metabolism. When the team combined DRP-104 with an existing drug that blocks the ERK signaling pathway, trametinib, it further improved survival in pancreatic cancer mouse models compared to DRP-104 treatment alone. "Despite a decade of advances in understanding how cancer cells switch fuel sources, we have not yet effectively translated this into clinically relevant therapies," says corresponding study author Alec Kimmelman, MD, PhD, the Anita Steckler and Joseph Steckler Chair of Radiation Oncology at NYU Langone Health. "Broad antagonism of metabolic pathways with glutamine analogs may provide another mode of attack against these highly resistant tumor cells. The fact that such drugs are already being tested in the clinic makes us hopeful that we may finally see patient outcomes improve, if this approach proves to be effective in clinical trials." Moving forward, the research team will seek to understand how glutamine antagonism impacts other adaptive nutrient scavenging mechanisms in pancreatic cancers and whether these could be targeted as well. The success of such approaches will depend on careful balancing of improved therapeutic efficacy and toxicity from the potential effects on normal tissues, Kimmelman adds. Along with Kimmelman, authors from Perlmutter Cancer Center and the Department of Radiation Oncology at NYU Langone were Joel Encarnación-Rosado, Albert Sohn, Douglas Biancur, Elaine Lin, Victoria Osorio-Vasquez, and Diana González-Baerga. Also Perlmutter authors were Ende Zhao and Diane Simeone. Also authors of the study were Tori Rodrick and Drew Jones from the Division of Advanced Research Technologies at NYU Grossman School of Medicine; Seth Parker from the Department of Biochemistry & Molecular Biology at University of British Columbia in Vancouver, and Yumi Yokoyama and Robert Wild from Dracen Pharmaceuticals, Inc., of San Diego. Funding for the study came from Perlmutter Cancer Center support grant P30CA016087 and National Cancer Institute grants P01A1080192, P01CA117969, R35CA232124, P30CA016087-38, 1R01CA251726-01A1; as well as from the Lustgarten Foundation, Stand Up to Cancer, and the Howard Hughes Medical Gilliam Fellowships. Kimmelman has financial interests in Vescor Therapeutics, is on the scientific advisory board of Rafael/Cornerstone Pharmaceuticals and OncoRev, and has been a consultant for Deciphera and Abbvie. These relationships are being managed according with the policies of NYU Langone Health. Yokoyama and Wild are employees of, and have ownership in, Dracen Pharmaceuticals, maker of DRP-104. Contact: Gregory Williams gregory.williams@nyulangone.org