Southern Research Institute

Dive Brief:An experimental drug from Ionis Pharmaceuticals succeeded in a Phase 3 trial in the rare disease hereditary angioedema, positioning the company and partner Otsuka to seek regulatory approvals in the U.S. and Europe.The medicine, called donidalorsen, reduced the rates of swelling attacks associated with the disease when given every one or two months, Ionis said Monday. Results showed a statistically significant benefit over placebo on secondary measures, too. No serious adverse events were reported in treated patients, according to Ionis.Ionis disclosed only summary data, leaving unanswered questions about the magnitude of donidalorsens effect and how it might compare to other medicines. Later this year, Ionis will present detailed findings as well as results from another late-stage study that enrolled people who switched to Ionis drug after taking another medicine.Dive Insight:Mondays announcement marks a step in Ionis transformation. A pioneering developer of RNA-based medicines called antisense oligonucleotides, the company previously favored licensing out its products instead of bringing them to market itself.However, Ionis has shifted strategy under CEO Brett Monia, aiming instead to capitalize on the sales upside of its newer medicines. Those drugs use a technology known as LICA, which is meant to make them safer and more potent than previous antisense oligonucleotides.The approach has paid dividends, as three of Ionis rare disease treatments have succeeded in late-stage testing over the last year. One, sold as Wainua, has reached market. At about $50 apiece, Ionis shares are trading at their highest levels since early 2021.We are now well on our way to independently launching medicines from our wholly owned pipeline, said Monia, in a statement.Still, Ionis isnt consistently profitable. It recorded a net loss of $357 million through the first nine months of 2023 after posting $270 million in losses a year earlier. Wainua, a key asset for Ionis, competes with a rival Alnylam Pharmaceuticals medicine.Donidalorsen, meanwhile, is one of many being developed to prevent the swelling attacks associated with HAE, a rare, inherited condition estimated to affect one in 50,000 people around the world. While HAE drugs are available from Takeda, CSL and others, they dont ward off attacks completely.Its unclear whether Ionis drug is better than available, preventive medicines like Takedas injectable Takhzyro or Biocryst Pharmaceuticals pill Orladeyo. Ionis is also developing donidalorsen at a time when other preventive options are advancing through clinical testing, among them a new antibody drug from CSL, an oral treatment from Pharvaris and a gene editing therapy from Intellia Therapeutics.One of the questions donidalorsen faces is whether the Phase 3 results meet the high bar it achieved in mid-stage testing, according to Stifel analyst Paul Matteis.We'll need to see detailed donidalorsen data to fully understand differentiation, Matteis wrote in a Monday note to clients. The HAE space is highly competitive, he noted, adding that the market is fragmented: some patients are willing switchers whereas others are sticky and brand loyal.Ionis holds U.S. rights to the drug. Otsuka acquired European rights in December. '

R&D Facility in Frederick, MD is Expected to Provide Internal Capacity to Discover and Develop Vaccines and Antiviral Drugs Against COVID-19, its Variants and Other Infectious Diseases Facility, Currently Owned and Operated by Tonix Partner Southern Research, has Housed Research Relating to Tonix’s COVID-19 Vaccine Candidate, TNX-1800 and Smallpox and Monkeypox Vaccine Candidate, TNX-801 CHATHAM, N.J., July 27, 2021 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced the signing of a Purchase and Sale Agreement to acquire an approximately 48,000 square foot research and development (R&D) facility in Frederick, MD to support Tonix’s expanding infectious disease pipeline, including TNX-1800, a live replicating viral vaccine designed to protect against COVID-19, TNX-801, a live vaccine designed to protect against smallpox and monkeypox, and TNX-3500, a small molecule antiviral to inhibit replication of SARS-CoV-2. Tonix agreed to purchase the R&D facility from Southern Research, a research collaboration partner for TNX-1800 and TNX-801 development. The facility currently operates at biosafety level 2 (BSL-2) containment. Pending transfer and approval of relevant permits, Tonix expects the transaction to close and the facility to be operational in the fourth quarter of 2021. Southern Research plans to consolidate its research activities at its Birmingham, AL campus. Tonix and Southern Research plan to continue those aspects of their collaboration on the development of vaccines and antivirals that are ongoing at the Birmingham, AL campus. “The Fredrick facility will be a major expansion of our R&D capabilities,” stated Seth Lederman, M.D., President and Chief Executive Officer of Tonix. “We believe this facility will ensure adequate resources and capacity to support and grow our pipeline of vaccines and antiviral therapeutics. In addition, we view control of in-house facilities as a strategic capability to ensure the speed and efficiency with which we can develop vaccines and antiviral products in the future against known, emerging or novel pathogens.” Dr. Lederman continued, “While COVID-19 has the appearance of being controlled in certain geographic centers, reports of increasing infections in both unvaccinated and vaccinated individuals, primarily related to new variants, have led to new mask mandates and restrictions in parts of the U.S. as well as new lockdowns and other restrictions in Europe and elsewhere. These concerning trends point to an urgent need for more robust vaccine technology and better overall preparedness. The COVID-19 pandemic revealed weaknesses in the U.S. domestic capability to conduct infectious disease R&D and produce vaccines and therapeutics, particularly in the setting of an interrupted global supply chain. We believe our planned capabilities at the Frederick facility will provide greatly needed domestic resources. The facility is ideally located in Maryland’s ’biotech corridor’, which is rich in highly skilled talent, and is also close to the center of the U.S. biodefense research community.” Josh Carpenter, PhD, Chief Executive Officer of Southern Research stated, “We are delighted that Tonix will be acquiring the Frederick research campus. This is another chapter in a robust partnership that will be made even stronger. We have enjoyed partnering with Tonix on TNX-1800 and TNX-801 vaccine projects and look forward to continued collaboration on these projects, as well as TNX-3500 with work performed at our Birmingham campus.” Tonix’s Fredrick R&D facility will complement its Advanced Development Center (ADC) being constructed in New Bedford, MA and the Commercial Manufacturing Center (CMC) that Tonix is planning in Hamilton, MT. The ADC will house laboratories dedicated to process analytical development and pilot manufacturing of its vaccine candidates. The CMC is expected to support commercial scale manufacturing of vaccine products. Tonix Pharmaceuticals Holding Corp. Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing small molecules and biologics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is primarily composed of central nervous system (CNS) and immunology product candidates. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL1, is in mid-Phase 3 development for the management of fibromyalgia. Tonix’s immunology portfolio includes vaccines to prevent infectious diseases and biologics to address immunosuppression, cancer, and autoimmune diseases. Tonix’s lead vaccine candidate, TNX-18002, is a live replicating vaccine based on the horsepox viral vector platform to protect against COVID-19, primarily by eliciting a T cell response. Tonix reported positive efficacy data from animal studies of TNX-1800 in the first quarter of 2021. TNX-8012, live horsepox virus vaccine for percutaneous administration, is in development to protect against smallpox and monkeypox. TNX-35003 (sangivamycin) is a small molecule antiviral drug in the pre-IND stage of development. 1TNX-102 SL is an investigational new drug and has not been approved for any indication.2TNX-1800 and TNX-801 are investigational new biologics and have not been approved for any indication.3TNX-3500 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication. This press release and further information about Tonix can be found at . About Southern Research Founded in 1941, Southern Research (SR) is an independent, 501(c)(3) nonprofit affiliated with the University of Alabama at Birmingham. SR is an applied scientific research organization with more than 400 scientists and engineers working across four divisions: CRO services, Drug Discovery, Energy Storage, and Engineering. SR has supported the pharmaceutical, biotechnology, defense, aerospace, environmental, and energy industries. SR works on behalf of the National Institutes of Health, the U.S. Department of Defense, the U.S. Department of Energy, NASA and other major aerospace firms, utility companies, and other external academic, industry and government agencies. SR pursues entrepreneurial and collaborative initiatives to develop and maintain a pipeline of intellectual property and innovative technologies that positively impact real-world problems. SR has numerous ongoing drug discovery programs, which encompass drug discovery programs to combat various forms of cancer, Alzheimer’s, schizophrenia, opioid use disorder, human immunodeficiency virus, disease, Parkinson’s, tuberculosis, influenza, and others. SR’s strong history, which includes nearly 80 years of successful collaborations to solve complex problems, has led to the discovery of seven FDA-approved cancer drugs—a number rivaling any other U.S. research institute. Furthermore, experts at SR are well-equipped to assist with the challenging landscapes of drug design and development technologies and market viability. SR is headquartered in Birmingham, Alabama. Further information about SR can be found at Forward Looking Statements Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the development of R&D facilities, risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2020, as filed with the Securities and Exchange Commission (the “SEC”) on March 15, 2021, and periodic reports filed with the SEC on or after the date thereof. All Tonix's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof. Contacts Jessica Morris (corporate)Tonix Pharmaceuticalsinvestor.relations@tonixpharma.com(862) 904-8182 Olipriya Das, Ph.D. (media)Russo PartnersOlipriya.Das@russopartnersllc.com (646) 942-5588 Peter Vozzo (investors)Westwicke/ICRpeter.vozzo@westwicke.com(443) 213-0505

Photo Credit: Trillium Renewable Chemicals Capricorn Partners (Leuven, Belgium) and Southern Research (Birmingham, Ala., U.S.) have jointly created a new Alabama-based startup company Trillium Renewable Chemicals (Birmingham). Southern Research has exclusively licensed its patented chemical manufacturing platform to Trillium to accelerate commercial development of this new technology. According to both companies, the majority of the more than 350,000 chemical products in circulation are currently made from oil or natural gas. However, bolstered by scientific advances, changing consumer preferences and regulatory pressures, a growing number of chemicals will be made from renewable feedstocks in the future. Trillium Renewable Chemicals is poised to address this fast-growing market segment through commercialization of the technology invented by Southern Research. The technology converts renewable feedstocks, such as sugar or glycerol to chemical products like acrylonitrile, propylene glycol and acrylic acid. These products are currently manufactured from oil and gas and used to make many familiar consumer and industrial goods like clothing, toys, car parts, sporting goods, paints, soaps and other valuable materials. “Southern Research is excited to team up with Capricorn Partners to further the development of our patented technology,” says Michael Catalano, COO for Southern Research. “With the foundation of Trillium and focused management, we will be able to maximize the commercial potential, which is the ultimate goal and is in line with our strategy to monetize intellectual property for reinvestment in future scientific endeavors.” Trillium and Southern Research will continue to work jointly on the process development, while Trillium will team up with strategic partners for the scale up and deployment of the technology. Corey Tyree, CEO of Trillium Renewable Chemicals adds: “Trillium will demonstrate the commerciality of this technology. Local production from renewable resources can meet the growing demand for green chemicals and offer stable pricing and a more secure supply chain. The market interest is there. To address it, the industry needs enabling technologies such as the catalyst discovered by Southern Research.” According to Amit Goyal, lead inventor from Southern Research, the company has developed an innovative, thermo-catalytic process that uses biomass derived sugars or glycerol, allowing for the use of a variety of biomass feedstocks, to produce acrylonitrile and other co-products. Goyal says this process has the potential to improve economics and the overall environmental footprint of downstream products that use acrylonitrile as a raw material. Capricorn Partners has invested from its Capricorn Sustainable Chemistry Fund. The proceeds will be used to scale up the process for the manufacturing of acrylonitrile and glycols. “We spotted the Southern Research Technology over two years ago and have been following the development closely as well as exploring market opportunities in Europe and the U.S.,” says Wouter Van de Putte and Rob van der Meij, of Capricorn Partners. “This type of ‘green drop-in’ molecule technology has a great advantage over new ‘green’ molecule developments in that the applications are already fully developed.” The Capricorn Sustainable Chemistry Fund, with a committed capital of €86.5 million, is aiming to capture the growing opportunities emerging from novel feedstocks, innovative and sustainable processes, more performing functional materials and smarter use of precious raw materials. Capricorn invests in companies which combine sound financial returns with a material contribution to and a front-runner view on developments in sustainability and advanced materials, processes and products. The fund focuses on companies that are in or near revenue stage and provided that the technologies and products are disruptive and have the potential to significantly address global markets.