Agex Therapeutics Inc., of Alameda, Calif., said it formed a research collaboration with The Ohio State University to use the company’s brown adipocyte tissue (BAT) cell therapy candidate, AGEX-BAT1, in mice to determine whether transplantation of the BAT cells may improve diet-induced obesity, metabolic health, including glucose metabolism, and cardiac function. BAT researcher Kristin Stanford, associate professor in physiology & cell biology and associate director of the Diabetes and Metabolism Research Center at Ohio State’s Wexner Medical Center, will oversee the project.
Benitec Biopharma Inc., of Hayward, Calif., reported findings from an interim analysis of the pilot dosing study of its gene therapy candidate, BB-301, in dogs. The study evaluated the safety and biological activity of BB-301 at concentrations of 1+E13 vg/mL and 3+E13 vg/mL across three doses – 1+E13 vg/mL (low) and 3+E13 vg/mL with low injection volume (intermediate) and 3+E13 vg/mL with high injection volume (high) – following direct intramuscular injection into the hypopharyngeus (HP) and thyropharyngeus (TP) muscles. In the HP muscle, low-dose BB-301 achieved a vector copy number of 1.7 copies per cell while the intermediate dose achieved 3.5 copies per cell and the high dose achieved 7.2 copies per cell. In the TP muscle, the low dose achieved a vector copy number of 2 copies per cell, intermediate achieved 2.3 copies per cell and high achieved 8.6 copies per cell. In the HP muscle, the low dose achieved siRNA13, siRNA17 and coPABPN1 copy numbers of 161,358 copies per cell, 26,652 copies per cell and 21 copies per cell, respectively, while the intermediate dose achieved 256,928, 47,944 and 24 copies per cell, respectively, and the high dose achieved 374,324, 57,126 and 52 copies per cell, respectively. In the TP muscle, low-dose BB-301 achieved 195,182, 40,106 and 15 copies per cell, respectively, while the intermediate dose achieved 293,597, 57,969 and 43 copies per cell, respectively, and the high dose achieved 751,484, 173,211 and 100 copies per cell, respectively. The study also showed evidence of wtPABPN1 silencing activity, which may serve as a surrogate for activity anticipated in the presence of mutant PABPN1. Benitec is advancing BB-301 to treat oculopharyngeal muscular dystrophy.
Cocrystal Pharma Inc., of Bothell, Wash., said it extended through August 2023 its drug discovery collaboration formed in 2017 with Hitgen Inc., of Chengdu, China, and Interx Inc., of Walnut Creek, Calif. Scientists from the companies are applying Hitgen’s DNA-encoded library technology platform, Cocrystal’s structure-based drug discovery platform technology and Interx’s computational science to develop antiviral candidates, with a joint steering committee overseeing the collaboration.
Cure Pharmaceutical Holding Corp., of Oxnard, Calif., said it added two development programs to its pipeline. In an antiviral development program, the company will seek to load a known active pharmaceutical ingredient (API) onto its thin film drug delivery platform, Curefilm, designed to optimize the absorption and metabolism of therapeutic compounds. Cure will use a similar bioequivalence approach to formulate delivery of a known antiseizure API for adult and pediatric indications. The programs will leverage existing preclinical and clinical safety and toxicity data while advancing the candidates.
Cytoagents Inc., of Pittsburgh, said it was awarded a $2 million U.S. NIH grant, its second in a year, to accelerate development of lead candidate GP-1681 (esuberaprost sodium), a cytokine receptor antagonist that targets hypercytokinemia, or cytokine storm, to treat cytokine release syndrome in CAR T-cell therapy and in COVID-19.
Enveric Biosciences Inc., of Naples, Fla., said it acquired an exclusive, perpetual license from Diverse Biotech Inc., of Doylestown, Pa., to five molecules, four dermatology-focused and one pain-focused. Enveric will also gain access to Diverse's scientists and formulators to help synthesize and validate the drugs. Through the conjugation of cannabidiol with existing, standard-of-care drugs via Diverse’s drug delivery platform, Enveric said its goal is to alleviate treatment side effects experienced by cancer patients.
ERS Genomics Ltd., of Dublin, said it granted a nonexclusive license agreement to Setsuro Tech, a startup from the University of Tokushima, giving it access to ERS’ CRISPR/Cas9 patent portfolio for Japan. Setsuro Tech generates genetically engineered mice at low cost and in a short timeframe, according to ERS, and applies CRISPR/Cas9 technology to create genome-edited cell and animal models, based on end-user requirements. Financial details were not released.
I-Mab Biopharma Co. Ltd., of Shanghai, said it signed collaborations with Complix NV, of Hasselt, Belgium, and Affinity Biopharmaceutical Technology Co. Ltd., of Shanghai, to gain access to technology platforms comprising cell penetrating alphabodies (CPABs) against two immuno-oncology intracellular drug targets and masked antibodies for targeted tumor-site activation, respectively. In the arrangement with Complix, development of resulting CPABs will be managed jointly with I-Mab, which will have an exclusive license to commercialize the CPABs in greater China. Commercialization rights in the rest of the world will be owned equally by the companies. Complix is entitled to an up-front payment and potential development milestones fees, as well as tiered royalties, and Affinity is set to receive undisclosed milestone fees from I-Mab. Additional details were not disclosed.
Immunitybio Inc., of Culver City, Calif., and Nantkwest Inc., of El Segundo, Calif., have completed their 100% stock-for-stock merger. The combined company will operate as Immunitybio and its shares of common stock began trading on Nasdaq on March 10 under the new ticker symbol IBRX. The company’s platform activates the innate and adaptive immune systems to treat cancer and infectious diseases.
New data from Intellia Therapeutics Inc., of Cambridge, Mass., demonstrated proof of concept for CRISPR-based in vivo editing of bone marrow and hematopoietic stem cells in mice. Intellia’s nonviral delivery system for in vivo genome editing of tissues outside the liver has application to treat inherited blood disorders such as sickle cell disease, the company said. Intellia said there was durable, multidose editing of whole bone marrow and hematopoietic stem cells in mouse models at therapeutically relevant levels. The data were presented at the virtual Keystone Esymposium.
Medidata, of New York, said it expanded its partnership with Karyopharm Therapeutics Inc., of Newton, Mass., to leverage 10 solutions from Medidata in more than 15 clinical studies of hematologic malignancies and solid tumors. Medidata said it has the first end-to-end scientific and business platform, from research to commercialization.
Lipigon Pharmaceuticals AB, of Chengdu, China, said it expanded its agreement with Hitgen Inc., also of Chengdu, to include a second target in treating cardiometabolic disease. The two companies have collaborated to develop molecules enhancing lipoprotein lipase activity since May. They will use Hitgen’s screening platform to identify small molecules and jointly develop a candidate. Lipigon will be responsible for clinical development and out-licensing of commercialization rights. Hitgen is entitled to a starting fee and revenue sharing for any candidate resulting from the collaboration.
Longeveron Inc., of Miami, expanded its collaboration with the University of Miami to include IND applications and the associated research data for allogeneic human mesenchymal stem cells for aging frailty and for hypoplastic left heart syndrome (HLHS). The deal also includes a patent application for methods for treating endothelial dysfunction and an exclusive option to obtain a license to additional intellectual property related to treatment of HLHS and dilated cardiomyopathy. The expanded collaboration modified the milestone payments and payment deadlines from Longeveron to the university.
Medicinova Inc., of La Jolla, Calif., will receive funding from the U.S. Biomedical Advanced Research and Development Authority for preclinical proof-of-concept studies testing MN-166 (ibudilast) as a potential medical countermeasure against chlorine gas-induced lung damage, such as acute respiratory distress syndrome and acute lung injury.
Modus Therapeutics AB, of Stockholm, plans to focus on developing its polysaccharide drug, sevuparin, as a treatment for sepsis/septic shock and possibly for inflammatory complications caused by major trauma and surgery, autoimmunity and viral infection. The company plans to start clinical testing of the drug in the new indication around the end of 2021.
Neuren Pharmaceuticals Ltd., of Melbourne, Australia, completed manufacturing of NNZ-2591, a synthetic analogue of cyclic glycine proline, for the company's phase II study in children with Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome. The company is preparing to submit an IND to the FDA for the study.
Uni-Bio Science Group Ltd., of Hong Kong, and Dotbio Pte. Ltd., of Singapore, are collaborating to develop drugs for retinal diseases, such as age-related macular degeneration, diabetic macular edema, retinal vein occlusion and myopic choroidal neovascularization. Dotbio will use its Dotbody technology to generate multiple multivalent and/or bispecific stabilized and humanized single-domain antibody candidates for various targets. Uni-Bio will be responsible for manufacturing, clinical development and commercialization of the products. Dotbio will receive undisclosed up-front and research payments and is eligible for milestone payments and royalties. Dotbio has the option to obtain additional stakes in the products by participating in the later-stage development.
UPM Biomedicals, of Helsinki, and Cellink AB, of Gothenburg, Sweden, are collaborating to develop 3D bioprinted products for tissue repair or replacement. The companies will combine UPM's nanocellulose biomaterials with Cellink's bio-ink technology.
VBI Vaccines Inc., of Ottawa, Ontario, will receive up to $33 million from the Coalition for Epidemic Preparedness Innovations to develop its enveloped virus-like particle vaccine candidates against SARS-CoV-2 variants, including the B.1.351 variant. The funds will support development of the vaccine for the B.1.351 variant through phase I development, as well as preclinical funding for multivalent vaccine candidates.
Venatorx Pharmaceuticals Inc., of Malvern, Pa., selected VNRX-9945 as a clinical candidate that it plans to develop for the treatment of chronic hepatitis B virus infection. The company expects to start clinical development of VNRX-9945, which inhibits nucleocapsid assembly and formation of cccDNA, in the second quarter of 2021.
Virpax Pharmaceuticals Inc., of Berwyn, Pa., contracted with Charles River Laboratories International Inc., of Wilmington, Mass., to run IND-enabling tests on Epoladerm, Virpax's anti-inflammatory topical spray film for acute musculoskeletal pain. Virpax plans to file its IND in the first half of 2021.