National Institute of Allergy & Infectious Diseases

Gritstone Bio, a US company, has announced that the Biomedical Advanced Research and Development Authority (BARDA) has awarded the company a contract valued up to $433m to evaluate its mRNA vaccine, samRNA, as a candidate for COVID-19. The agreement was awarded as part of the US Department of Health and Human Services (HHS) initiative, Project NextGen, to advance new, innovative vaccines and therapeutics to provide broader and more durable protection against COVID-19. As part of the agreement, the clinical-stage biopharmaceutical company, Gritstone, will conduct a phase 2b double-blinded study, the CORAL programme, which will include 10,000 participants, to compare the efficacy, safety and immunogenicity of its COVID-19 vaccine candidate with an approved COVID-19 vaccine. The CORAL programme was designed to address the limitations of protection against SARS-CoV-2 variants by inducing durable neutralising antibodies and T cell-based immunity against current and future strains. The samRNA vaccine, which incorporates both spike and other viral targets, has previously shown potent immune responses as well as the potential to drive broad and durable clinical protection against COVID-19. In comparison to other traditional mRNA vaccines, samRNA creates several copies of the antigen RNA once in the cell, potentially providing an extended duration and magnitude of antigen expression. The study will be fully funded by BARDA and will take place in the US in collaboration with the COVID-19 Prevention Network (CoVPN), a network supported by the National Institute of Allergy and Infectious Diseases (NIAID). Ahead of the phase 2b study, Andrew Allen, co-founder, president and chief executive officer of Gritstone said that the company is “excited… to work alongside BARDA and look forward to initiating the phase 2b study (CORAL-BARDA) in the first quarter of 2024”. "With CORAL moving into a randomised phase 2 study alongside our personalised cancer vaccine program (GRANITE), Gritstone now sits at the precipice of unlocking the full potential of our novel vaccine platforms in both oncology and infectious diseases,” he added.

-New data to be presented from Phase 3 CERTAIN-1 study with Cefepime-Taniborbactam -MD Anderson Cancer Center analysis of Cefepime-Taniborbactam in vitro activity against clinically significant gram-negative bacteria isolated from patients with cancer MALVERN, Pa.--(BUSINESS WIRE)-- Venatorx Pharmaceuticals, a private, clinical-stage pharmaceutical company focused on improving health outcomes for patients with multidrug-resistant bacterial infections and hard-to-treat viral infections, today announced seven presentations during IDWeek 2023, October 11-15, 2023, in Boston, MA for its novel antibiotics cefepime-taniborbactam and ceftibuten-ledaborbactam, including: Presentation Details Title: Oral presentation: Ceftibuten-Ledaborbactam Date/Time: October 14, 2023, 8:00 am – 9:00 am Session Title: New Antimicrobials in the Pipeline Part 2 Session Location: 104 ABC Presenting Author: Paul McGovern (Venatorx) Title: CERTAIN-1 Subgroup Analysis: A Phase 3 Study of Cefepime-Taniborbactam Efficacy in the Treatment of Complicated Urinary Tract Infections (cUTI) (#2513) Date/Time: October 14, 2023, 12:15 pm – 1:30 pm Session Title: New Antimicrobial Drug Development Session Location: Hall B + C Presenting Author: Mary Beth Dorr (Venatorx) Title: Outcomes by Resistance Phenotype and Genotype among Baseline Pathogen in Patients with Complicated Urinary Tract Infection (cUTI) in the Phase 3 CERTAIN-1 Study (#2523) Date/Time: October 14, 2023, 12:15 pm – 1:30 pm Session Title: New Antimicrobial Drug Development Session Location: Hall B + C Presenting Author: Greg Moeck (Venatorx) Title: In Vitro Activity of Cefepime-Taniborbactam and Comparators Against Genotypically Characterized Carbapenem-Resistant Enterobacterales (CRE) and Carbapenem-Resistant Pseudomonas aeruginosa (CRPA) from the United States, 2018-2021 (#2150) Date/Time: October 14, 2023, 12:15 pm – 1:30 pm Session Title: Antibiotic Novel Agents Session Location: Hall B + C Presenting Author: Mark Wise (IHMA) Title: Antimicrobial Activity of Cefepime in Combination with Taniborbactam Against Resistant Clinical Isolates from the United States 2018-2021 (#2129) Date/Time: October 14, 2023, 12:15 pm – 1:30pm Session Title: Antimicrobial Novel Agents Session Location: Hall B + C Presenting Author: Meredith Hackel (IHMA) Title: In vitro activity of aztreonam-avibactam (ATM-AVI), cefiderocol (FDC), and cefepime-taniborbactam (FEP-TAN) against multi-species, NDM-producing Enterobacterales causing a local outbreak (#2185) Date/Time: October 14, 2023, 12:15 pm – 1:30pm Session Title: Antimicrobial Resistance Mechanisms Session Location: Hall B + C Presenting Author: Ellen G. Kline (University of Pittsburg) Title: In Vitro Activity of Cefepime-Taniborbactam Against Clinically Significant Gram-Negative Bacteria Isolated from Patients with Cancer (#2122) Date/Time: October 14, 2023, 12:15 pm – 1:30pm Session Title: Antimicrobial Novel Agents Session Location: Hall B + C Presenting Author: Bahgat Gerges (MD Anderson UT) About the CERTAIN-1 Phase 3 Clinical Trial CERTAIN-1 (Cefepime Rescue with Taniborbactam in cUTI) was a global, randomized, double-blind, active-controlled non-inferiority Phase 3 study evaluating the efficacy, safety, and tolerability of cefepime-taniborbactam compared to meropenem in adults with cUTI, including acute pyelonephritis. The trial enrolled 661 adult patients who were randomized 2:1 to receive cefepime-taniborbactam 2.5g q8h or meropenem 1g q8h for 7 days (up to 14 days for patients with bacteremia). The primary efficacy endpoint evaluated the composite clinical and microbiologic response (i.e., bacterial eradication) at the Test of Cure (TOC) visit (Day 19-23) in the microbiological intent-to-treat (microITT) population. Cefepime-taniborbactam met the primary efficacy endpoint of statistical noninferiority (NI) to meropenem in the microITT population at TOC with composite microbiologic and clinical success occurring in 70.6% of cefepime-taniborbactam treated patients and 58.0% of meropenem treated patients (treatment difference 12.6; 95% CI, 3.1, 22.2). A prespecified superiority assessment was conducted following confirmation of NI. The superiority of cefepime-taniborbactam was concluded as the lower bound of the 95% CI for noninferiority was greater than zero. The pre-specified superiority test (two-sided p-value = 0.0088) demonstrated the strength of evidence associated with the superiority conclusion for the composite endpoint at TOC. The superiority of cefepime-taniborbactam was sustained for the composite microbiologic and clinical response at the Late-Follow-Up (Day 28-35) visit. Rates of treatment-emergent adverse events (TEAEs) were 35.5% for cefepime-taniborbactam and 29.0% for meropenem. Serious TEAEs occurred in 2.0% and 1.8% of cefepime-taniborbactam and meropenem treated patients, respectively. Treatment discontinuations due to TEAEs occurred in 3.0% of cefepime-taniborbactam patients and 0.9% of meropenem treated patients. There was one death in the cefepime-taniborbactam treatment group, which was unrelated to study treatment as assessed by the investigator. About Cefepime-Taniborbactam Cefepime-taniborbactam is an investigational intravenous (IV) beta-lactam/beta-lactamase inhibitor (BL/BLI) antibiotic combination being developed for the treatment of complicated urinary tract infections (cUTIs), including pyelonephritis, and hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP). The New Drug Application for cefepime-taniborbactam was accepted for review by the US FDA for cUTI, including pyelonephritis with a PDUFA date of February 22, 2024. Cefepime, a fourth-generation cephalosporin, is a widely used beta-lactam (BL) antibiotic with more than two decades of proven safety and clinical utility against susceptible gram-negative and gram-positive bacteria. Taniborbactam is a beta-lactamase inhibitor (BLI) that, in combination with cefepime, is being studied as a potential treatment option for patients with serious bacterial infections caused by antibiotic resistant gram-negative bacteria, most notably extended spectrum beta-lactamase (ESBL)-expressing Enterobacterales, carbapenem-resistant Enterobacterales (CRE), and multidrug-resistant (MDR) Pseudomonas aeruginosa (MDR-PA), which can include carbapenem-resistant P. aeruginosa (CRPA). Cefepime-taniborbactam has been granted Qualified Infectious Disease Product (QIDP) and Fast Track designations by the U.S. Food and Drug Administration (FDA). About Ceftibuten-Ledaborbactam Etzadroxil Ceftibuten, an oral third-generation, highly bioavailable cephalosporin antibiotic, is approved for the treatment of upper and lower respiratory tract infections and urinary tract infections. Ledaborbactam etzadroxil is a novel broad-spectrum boronic acid beta-lactamase inhibitor (BLI). In vitro and in vivo studies demonstrated that ledaborbactam restores the activity of ceftibuten against strains of Enterobacterales expressing Ambler class A ESBLs, class C cephalosporinases, and class A & D serine carbapenemases (KPC and OXA-48, respectively) as well as multidrug-resistant (MDR) Enterobacterales. Ceftibuten-ledaborbactam etzadroxil may offer a new treatment option for outpatient therapy to treat serious bacterial infections caused by MDR Enterobacterales that are resistant to current standard-of-care oral and intravenous antibiotics, including fluoroquinolones, trimethoprim-sulfamethoxazole, cephalosporins and carbapenems. Ceftibuten-Ledaborbactam etzadroxil has been granted Qualified Infectious Disease Product (QIDP) and Fast Track designations by the U.S. Food and Drug Administration (FDA). Funding Partners and Collaborators Development of cefepime-taniborbactam began with federal funds from the National Institute of Allergy and Infectious Diseases, National Institutes of Health, Department of Health and Human Services under contract number HHSN272201300019C, and Wellcome Trust under award number 360G-Wellcome-101999/Z/13/Z, and continues with federal funds from the Biomedical Advanced Research and Development Authority, Administration for Strategic Preparedness and Response, Department of Health and Human Services under contract numbers HHSO100201900007C and 75A50122C00080. In September 2018, Venatorx entered into an exclusive license agreement with Everest Medicines to support the development, registration, and commercialization of cefepime-taniborbactam in People’s Republic of China, Macau, Hong Kong, Taiwan, South Korea, and select countries in Southeast Asia (the “Territory”). In April 2020, Venatorx and GARDP announced a collaboration to accelerate the development of, and access to, cefepime-taniborbactam for adult and pediatric populations. Venatorx has granted GARDP exclusive rights to distribute and sub-distribute cefepime-taniborbactam, once it is approved for clinical use, in low- and lower middle-income countries. Ceftibuten-ledaborbactam etzadroxil is funded in whole or in part by the National Institute of Allergy and Infectious Diseases, National Institutes of Health, Department of Health and Human Services under contract number HHSN272201600029C. About Venatorx Pharmaceuticals, Inc. Venatorx is a private, pre-commercial pharmaceutical company focused on improving health outcomes for patients with difficult-to-treat drug resistant gram-negative bacterial infections and viral infections. Venatorx’s lead asset, cefepime-taniborbactam, is an investigational antibiotic that completed a Phase 3 study (NCT03840148) in adults with complicated urinary tract infections (cUTI), including pyelonephritis and is under FDA review with a PDUFA action date of February 22, 2024. In October 2022, BARDA awarded a contract of up to $318M to Venatorx for development and procurement of cefepime-taniborbactam for the treatment of melioidosis and multidrug-resistant infections. As part of its broader pipeline, Venatorx is also developing an oral antibacterial, ceftibuten-ledaborbactam etzadroxil, that recently completed multiple Phase 1 clinical studies and is expected to begin global Phase 3 testing in 2Q2025. For more information about Venatorx and its anti-infectives portfolio, please visit .

Vaccines stable for at least two years when stored at 40 degrees Celsius / 104 degrees Fahrenheit Only subunit (protein) vaccine platform shown to protect against potentially lethal Ebola and Marburg viruses No cold chain storage and transport required as compared to other filovirus vaccines PRINCETON, N.J., Sept. 25, 2023 /PRNewswire/ -- Soligenix Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today two-year stability of its thermostabilized bivalent and trivalent filovirus vaccine candidates at temperatures of 40 degrees Celsius (104 degrees Fahrenheit) when formulated in a single vial, needing reconstitution only with sterile water immediately prior to use. This follows the previous successful demonstration of 100% protection of non-human primates (NHPs) against lethal Sudan ebolavirus and Marburg marburgvirus challenge with the bivalent vaccine. This important milestone is part of an ongoing collaboration with the University of Hawaiʻi at Mānoa (UHM), demonstrating the successful presentation of one or more antigen(s) within the same formulation while maintaining full potency and thermostability, including vaccines for Sudan ebolavirus (SuVax™) and Marburg marburgvirus (MarVax™). It further demonstrates the broad applicability of the heat stable vaccine platform, and its potential role in the United States (U.S.) Administration's initiative for pandemic preparedness. "Filoviruses such as Zaire ebolavirus, Sudan ebolavirus and Marburg marburgvirus are some of the most lethal viruses known, and they are endemic in areas of the world where the power supply and distribution network can be uncertain. There are no licensed vaccines for either Sudan or Marburg viruses, while vaccines for Zaire ebolavirus are constrained by cold chain logistics. A heat stable vaccine in a single vial format would significantly enhance any public health preparedness or response to a new outbreak, at its source," stated Axel Lehrer, PhD, Associate Professor, Department of Tropical Medicine, Medical Microbiology and Pharmacology, John A. Burns School of Medicine, UHM. "Our work to date has demonstrated the feasibility of rapid and efficient manufacturing, as well as the ability to thermostabilize multiple antigens that can then be stored at temperatures exceeding 100 degrees Fahrenheit. Having a vaccine platform available such as this has the potential to accelerate worldwide vaccination campaigns addressing future health emergencies, including another global pandemic." "Our next generation combined vaccine platform includes three major components: a robust protein manufacturing process that has been demonstrated on multiple protein antigens, a novel nano-emulsion adjuvant which induces broad immunity and a formulation procedure which enables thermostabilization of the combination of adjuvant and antigen in a single vial," stated Oreola Donini, PhD, Senior Vice President and Chief Scientific Officer of Soligenix. "Elements of this vaccine platform have also been utilized in our ricin toxin (RiVax®) and COVID-19 vaccine (CiVax™) candidates, indicating its broad applicability, including at least one-year thermostability at 40 degrees Celsius (104 degrees Fahrenheit). The ability to stably package more than one vaccine candidate in a single vial platform further adds to their developability, whether as a multivalent or individual monovalent vaccine, particularly against Marburg marburgvirus and Sudan ebolavirus where there are currently no available vaccines." Under the Company's Public Health Solutions business segment, ongoing collaborations with Dr. Lehrer, as well as work conducted by Theodore Randolph, PhD, Professor, Center for Pharmaceutical Biotechnology, Department of Chemical and Biological Engineering at the University of Colorado, Boulder have demonstrated the feasibility of developing thermally-stable subunit protein vaccine formulations for filoviruses. The thermostabilized filovirus vaccine program is continuing to advance with the support of a National Institutes of Health (NIH) grant (#R01-AI132323; awarded to the University of Hawaiʻi) and a Small Business Innovation Research grant (#1R44AI157593-01; awarded to Soligenix, Inc.). Work to date has demonstrated the compatibility of lyophilizing both antigen and adjuvant in the same vial, with reconstitution using sterile water for injection immediately prior to vaccination. This simple delivery format, as well as the compatibility with ambient storage, enables vaccines that significantly reduce the logistical hurdles that have been required for addressing the current pandemic or deployment of other Ebola virus vaccines in recent outbreaks in Central and West Africa. The antigens and adjuvants used in our filovirus vaccines represent the only recombinant subunit vaccines to date that have demonstrated full protection against challenge with Zaire ebolavirus, Sudan ebolavirus and Marburg marburgvirus in NHPs. Lyophilization of the antigens within monovalent vaccine formulations has also been demonstrated to thermostabilize the antigens at temperatures as high as 40 degrees Celsius (104 degrees Fahrenheit) for up to 12 weeks, enabling transport and storage at ambient temperature, even under challenging conditions. No currently licensed lyophilized vaccine that contains adjuvant is presented in a single vial format and there are few reports of successfully using nano-emulsions in lyophilized formulations. Previous work has demonstrated the use of a single vial platform to co-lyophilize antigen(s) and a nano-emulsion adjuvant, CoVaccine HT™, maintaining key adjuvant stability characteristics including particle size and colloidal stability, as well as maintaining immunogenicity. This most recent milestone confirms the robust thermostabilization provided by this vaccine platform. About Filovirus Infection Ebola Virus Disease is caused by one of six species of Ebolavirus, four of which are known to cause disease in humans, including its best-known member, Zaire ebolavirus (Ebola virus), with Sudan ebolavirus being the second-most common cause of human infection in this family. All species of ebolavirus belong to the Filoviridae family, a family that further contains the equally human pathogenic Marburg virus. Filoviruses are believed to be harbored in various animal species in Africa, particularly bats, although the specific reservoir host for many of these viruses is still unknown. There have been several known Ebola and Marburg Virus Disease outbreaks since 1967, with the largest outbreak starting in 2014 in Western Africa, and involved over 26,000 confirmed/probable/suspected cases with an estimated death toll of over 11,000 people according to the Centers for Disease Control and Prevention (CDC). These numbers also include some cases of virus introduction and limited spread in Europe and the United States. Transmission of filoviruses requires direct contact with bodily fluids from an infected person or contact with infected animals. The mortality rates following filovirus infections are extremely high, and, in the absence of wide availability of effective therapeutics, are affected by the quality of supportive care available with a focus on early initiation of treatment. Resolution of the disease largely depends on the patient's own immune system. There are limited treatment options for Ebola Virus Disease and no available treatments for Sudan Virus or Marburg Virus Disease, although steady progress has also been made in development of immunotherapeutics for filoviruses beyond Zaire ebolavirus. There are approved vaccines for Ebola virus (Zaire ebolavirus), requiring stringent ultra-low cold-chain storage, but no efficacious vaccines are yet available for Marburg virus (Marburg marburgvirus) or Sudan virus (Sudan ebolavirus). Filoviruses are one of the virus families identified as having the ability to cause pandemics. On the heels of the COVID-19 pandemic the U.S. government is accelerating its investment in pandemic preparedness, including having "the ability to rapidly make vaccines effective against any virus family". Specific initiatives have been spear-headed by the White House and Biden-Harris administration, as evidenced by the "American Pandemic Preparedness: Transforming Our Capabilities" white paper released in September 2021. About John A. Burns School of Medicine, University of Hawaiʻi at Mānoa The John A. Burns School Medicine (JABSOM) at the University of Hawaiʻi at Mānoa is one of the leading medical institutions and one of the most ethnically diverse institutions in the United States. For more than a decade, JABSOM has ranked in the top 10% of allopathic medical schools for graduate retention with one of our UH-sponsored residency programs. Hawaiʻi's cultural diversity and geographical setting affords JABSOM a unique research environment to excel in research directed at eliminating diseases that disproportionately affect people in Hawaii and the Pacific region. JABSOM faculty bring in extramural funds of $46 million into the state, annually. In addition, JABSOM was the first U.S. medical school to create a clinical department dedicated to the health and well-being of an indigenous population, Native Hawaiians. About Soligenix, Inc. Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte™ (SGX301 or synthetic hypericin sodium) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). With a successful Phase 3 study completed, regulatory approval is being sought and commercialization activities for this product candidate are being advanced initially in the U.S. Development programs in this business segment also include expansion of synthetic hypericin (SGX302) into psoriasis, our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn's disease (SGX203). Our Public Health Solutions business segment includes active development programs for RiVax®, our ricin toxin vaccine candidate, as well as our vaccine programs targeting filoviruses (such as Marburg and Ebola) and CiVax™, our vaccine candidate for the prevention of COVID-19 (caused by SARS-CoV-2). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agency (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA). For further information regarding Soligenix, Inc., please visit the Company's website at and follow us on LinkedIn and Twitter at @Soligenix_Inc. This press release may contain forward-looking statements that reflect Soligenix, Inc.'s current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations and clinical trial enrollment. Statements that are not historical facts, such as "anticipates," "estimates," "believes," "hopes," "intends," "plans," "expects," "goal," "may," "suggest," "will," "potential," or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, and include the expected amount and use of proceeds from the offering and the expected closing date of the offering. Soligenix cannot assure you that it will be able to successfully develop, achieve regulatory approval for or commercialize products based on its technologies, particularly in light of the significant uncertainty inherent in developing therapeutics and vaccines against bioterror threats, conducting preclinical and clinical trials of therapeutics and vaccines, obtaining regulatory approvals and manufacturing therapeutics and vaccines, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further funding to support product development and commercialization efforts, including grants and awards, maintain its existing grants which are subject to performance requirements, enter into any biodefense procurement contracts with the U.S. Government or other countries, that it will be able to compete with larger and better financed competitors in the biotechnology industry, that changes in health care practice, third party reimbursement limitations and Federal and/or state health care reform initiatives will not negatively affect its business, or that the U.S. Congress may not pass any legislation that would provide additional funding for the Project BioShield program. In addition, there can be no assurance as to the timing or success of any of its clinical/preclinical trials. Despite the statistically significant result achieved in the HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma, there can be no assurance that a marketing authorization from the FDA or EMA will be successful. Notwithstanding the result in the HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma and the Phase 2a clinical trial of SGX302 for the treatment of psoriasis, there can be no assurance as to the timing or success of the clinical trials of SGX302 for the treatment of psoriasis. Further, there can be no assurance that RiVax® will qualify for a biodefense Priority Review Voucher (PRV) or that the prior sales of PRVs will be indicative of any potential sales price for a PRV for RiVax®. Also, no assurance can be provided that the Company will receive or continue to receive non-dilutive government funding from grants and contracts that have been or may be awarded or for which the Company will apply in the future. These and other risk factors are described from time to time in filings with the Securities and Exchange Commission (the "SEC"), including, but not limited to, the Company's preliminary prospectus (Registration No. 333-271049) filed with the SEC on May 4, 2023, and Soligenix's reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events. Company Codes: NASDAQ-SMALL:SNGX