Cornerstone Pharmaceuticals, Inc.

Rafael Holdings will provide Cylo Therapeutics' shareholders with shares of its class B common stock based on valuing Cylo’s shares at 95 cents apiece. With just weeks to go before the FDA decides whether to approve the first treatment for a rare genetic condition, Rafael Holdings is merging with Cyclo Therapeutics to try to push their own candidate over the line.Cyclo’s lead asset is Trappsol Cyclo, a formulation of hydroxypropyl beta cyclodextrin that is being evaluated in a phase 3 trial in Niemann-Pick disease type C1 (NPC). The rare, genetic lysosomal storage disease prevents the body from moving and using cholesterol and other lipids in cells. This leads to a buildup of cholesterol and other lipids in the liver, spleen or lungs.After the deal closes, which is expected in late 2024, Rafael said it will continue to fund the phase 3 trial of Trappsol Cyclo through to its 48-week interim analysis. Enrollment in the study was completed in May, and Cyclo has previously said the top-line readout is likely in the first half of 2025.The expectation among Cyclo’s leadership is that this phase 3 data will be the evidence needed to get Trappsol Cyclo approved as “a much-needed treatment option for the treatment of NPC1,” they said in a second-quarter update last week. Rafael’s interest in Cyclo dates back to March 2023, when the holding company made its first investment in the biotech. Since then, Rafael scooped up $5 million worth of Cyclo’s stock in June 2023 and contributed to a $2.4 million raise via a private offering of Cyclo’s stock in October of that year.Now, the companies have announced a merger where Rafael will provide Cylo’s shareholders with shares of its class B common stock based on valuing the biotech's shares at 95 cents apiece. Cyclo's shares closed at $1.26 yesterday and were trading down about 17% before the market open Thursday in the wake of the news “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,” Rafael’s CEO Bill Conkling said in the Aug. 22 release.In this morning’s release, Cyclo’s CEO N. Scott Fine said the biotech’s partnership with Rafael over 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.” Beyond Cyclo, Rafael’s interests include a majority investment in cancer-metabolism-focused Cornerstone Pharmaceuticals and a majority equity interest in LipoMedix Pharmaceuticals, which is working on liposome-delivered oncology therapies.There are currently no FDA-approved therapies for NPC, although Johnson & Johnson’s Zavesca is approved in Europe, Japan and certain other countries. But the race has heated up this month after an FDA advisory committee gave its backing to Zevra Therapeutics’ arimoclomol ahead of a regulatory decision next month.Arimoclomol was originally submitted to the FDA in July 2020 but was rejected almost a year later because the agency questioned aspects of the single clinical trial used to support the application. Zevra returned to the FDA in December 2023 with a rescored scale and more results from the study, including data from an open-label expansion phase. 

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.

None  Welcome to this week's Chutes & Ladders, our roundup of hirings, firings and retirings throughout the industry. Please send the good word—or the bad—from your shop to Max Bayer or Gabrielle Masson, and we will feature it here at the end of each week.   Scorpion’s CEO, CMO out the door Scorpion Therapeutics It’s a new year and change is afoot at Scorpion Therapeutics, with both former CEO Axel Hoos, M.D., Ph.D., and CMO Michael Streit, M.D., hitting the exits.  Industry veteran Hoos has led the oncology company since July 2021, when he left GSK for Scorpion. At the time, Hoos had taken the reins from interim CEO Adam Friedman, M.D., Ph.D., who will now take back the top title, this time without the temporary designation. Friedman is part of Scorpion’s founding team and most recently served as the biotech’s president of corporate strategy and business development.    The Boston biotech is also replacing Streit, who was the first to fill the company’s chief medical officer position. Streit joined in 2022 from Sanofi, where he had most recently served as vice president and senior project head.  Now, Mark Chao, M.D., Ph.D., will take over as CMO. He joins Scorpion from TenSixteen Bio, where he served as co-founder and CEO.  But the changes don’t stop there. Scorpion has also promoted Erica Jackson, Ph.D., to chief discovery officer. She previously served as the company’s executive vice president of biology. Fierce Biotech  CRISPR hunts for new CMO  CRISPR Therapeutics CRISPR Therapeutics Chief Medical Officer Phuong Khanh Morrow, M.D., is resigning—a decision that was announced six days after the company secured FDA approval for its Vertex-partnered gene therapy Casgevy. The biotech has already kicked off the search for a new CMO, a spokesperson told Fierce Biotech. “CRISPR Therapeutics has a seasoned leadership team with extensive experience managing clinical development for CRISPR’s programs and is well positioned to successfully manage the transition,” a spokesperson said via email. Morrow, who has been with the company since May 2022, will officially depart Jan. 26. Her resignation was not the result of any disagreement with the gene-editing company, according to CRISPR Tx. Before joining the Swiss-American biotech, Morrow spent more than 10 years in various leadership roles at Amgen. Fierce Biotech  Long-time Evotec CEO steps down Evotec Evotec’s CEO Werner Lanthaler has resigned from his role before his expected end of term, which was set for March 2026, citing personal reasons.  Lanthaler, who has led the German biotech since 2009, will serve as a strategic advisor to Evotec’s board, which has launched a search for a permanent CEO.  Until a successor is chosen, board member Mario Polywka will take on the role of interim CEO. Ploywka had served as Evotec’s chief operating officer from 2006 to 2018. Release  > LianBio CEO Yizhe Wang, Ph.D., is headed for the exits in pursuit of new opportunities. Chief Investment Officer Adam Stone will be the interim chief while the search for a permanent replacement begins. Release > Ex-Biogen CEO Michel Vounatsos has joined the board of Quris-AI. Former Merck regional president Yossi Ben Amram was also named president of the company. Release > Vicore Pharma has hired Bertil Lindmark, Ph.D., M.D., to serve as chief medical officer, taking the reins from Rohit Batta. Lindmark joins from Galecto, where he also held the CMO role, and has previous experience as AstraZeneca’s global vice president of clinical development for respiratory and inflammation. Release > Applied Therapeutics has named Constantine Chinoporos as its new chief operating officer and chief business officer. He’s been a strategic advisor for Apollo Therapeutics since February 2023 after past c-suite roles at other biotechs. Disclosure > Innate Pharma CEO Mondher Mahjoubi has resigned to pursue a new gig at a larger pharmaceutical company, the European biotech announced. Chairman and co-founder Hervé Brailly will be the interim leader while the search for a replacement gets underway. Release > SpliceBio will bring on Aniz Girach, M.D., as chief medical officer. Girach was previously CMO of fellow gene editing biotech ProQR until March 2023. Release > New year, new gig for Nathan Dowden, who’s been promoted at Entrada from chief operating officer to president and COO. He’s been with the company since 2019 after working at Rubius. Release > Relmada CMO Cedric O’Gorman, M.D., is leaving the company after one year. He previously was the chief medical officer at Alpha Cognition. Release > After Zevra’s recent acquisition of Acer, Adrian Quartel, M.D., will become the new chief medical officer. He was the CMO of Acer and was the chief medical officer of Adamis before that. Release > ARIAD Pharmaceuticals founder Harvey Berger, M.D., has been hired as the new CEO of Kojin Therapeutics. Kojin is backed by the likes of Leaps By Bayer and AbbVie’s venture arm. Release > Included in Radionetics Oncology’s latest financing announcement was news that the biotech has brought on Paul Grayson as CEO. He was most recently president and CEO of fellow oncology company Tentarix Biotherapeutics. Release  > Mineralys Therapeutics has tapped Minji Kim, Ph.D., to serve as chief business officer. Before Mineralys, she held the same role at Affamed Therapeutics and has past experience at Jounce Therapeutics, Curis and Hoffmann-La Roche. Release > ProMIS Neurosciences is bringing on Neil Warma as interim CEO to replace Gail Farfel, Ph.D. Warma has been a director on the company’s board since 2022. Release > Brii is bringing aboard former HemoShear CSO Brian Alvin Johns, Ph.D., for the same role. Before HemoShear, he was VP of discovery at Viiv and GSK. Release > Heike Keilhack, Ph.D., is joining ROME Therapeutics as chief scientific officer. She previously was CSO and oversaw R&D of two now-clinical-stage PARP inhibitors at Ribon Therapeutics. Release > Diagnostics vet Brian Kim is the new CEO of genomics company Mission Bio, the company announced. He previously was CEO of Fortis Life Science and held other leadership roles at Biosearch Technologies and PerkinElmer. Release > SV Health Investors has appointed Nikola Trbovic as managing partner and Jamil Beg as partner. The two will help steer the firm’s biotech portfolio, which includes companies like Bicycle and Nimbus. Release > CymaBay Therapeutics and former Chief Commercial Officer Lewis Stuart mutually agreed that Stuart would leave the company on Dec. 29. CymaBay’s VP Liver Franchise Lead Ben Kozub will assume leadership of commercial organization. Release > Alaunos Therapeutics has tapped Dale Curtis Hogue, Jr., to serve as director of the immuno-oncology company. Hogue most recently founded Dune Lake Capital and previously worked as a senior analyst at Discovery Capital Management. Release > Viatris has chosen Philippe Martin to lead the company’s research and development as chief R&D officer. Martin has clocked in past experience at Celgene and Schering-Plough (acquired by Merck). Release > Noriyuki Kasahara, M.D., Ph.D., is moving from 4DMT’s board to its C-suite, being named chief scientific officer. Kasahara is a brain surgeon who previously worked at UCSF. Release > Scribe Therapeutics’ next chief scientific officer and head of research and development will be Aarif Khakoo, M.D., who joins the CRISPR genetic medicines company from Calico Life Sciences. Also joining the biotech is Maria Mirotsou, Ph.D., as vice president of discovery biology. Release > Eterna Therapeutics has picked Sanjeev Luther as its next CEO, as of Jan. 1. He succeeds Matt Angel, Ph.D. Luther has spent the past 30 years at various roles across biopharma, most recently as CEO at Cornerstone Pharmaceuticals. Release > Henrik Luessen is the new chief business officer for German biotech Eleva, which has a moss-based platform that develops complex proteins for treatment of diseases. Release > Ashvattha Therapeutics has named Mehdi Paborji, Ph.D., as senior vice president of technical operations to oversee CMC and manufacturing for the biotech’s nanomedicines. Release  > Deka Biosciences has tapped Stanley Frankel, M.D., to serve as senior clinical advisor, interim chief medical officer and scientific advisory board member. The hematologist-oncologist joins Deka with past experience as SVP, global drug development for cell therapy for BMS following the 2019 acquisition of Celgene. Release > HERVOLUTION Therapeutics has chosen J. Robert Coleman, Ph.D., to lead the biotech as CEO. Before joining HERVOLUTION, Coleman co-founded Codagenix and served as the company’s CEO for 12 years. Release > ImPact Biotech has appointed clinical radiologist Eyal Morag, M.D., to be the company’s chief medical officer. Morag joins from Microbot Medical, where he was also CMO, and currently serves in scientific and medical advisory roles at Aidoc Medical, Vortex Imaging and HighRad. Release