Apeiron Biologics AG, of Vienna, which is developing therapies to treat cancer and respiratory conditions, said it has selected Domainex Ltd. to provide integrated lead optimization services to advance the development of inhibitors for the E3 ubiquitin ligase Cbl-b through small-molecule drug discovery. To date, Domainex has provided the company with its expertise in hit identification, both fragment-based and virtual screening, by establishing and deploying a suite of biophysical assays against variants of the E3 ligase Cbl-b and other members of that target class, to determine the binding affinity, mechanism and selectivity of promising compounds.
Applied Therapeutics Inc., of New York, presented preclinical data, at the American Heart Association Scientific Sessions, demonstrating that aldose reductase (AR) inhibition, using the AR inhibitor AT-001, normalizes cardiac energy metabolism and prevents cardiac structural and functional abnormalities in a mouse model of diabetic cardiomyopathy. AT-001-treated mice exhibited decreased cardiac fatty acid oxidation rates and decreased cardiac oxygen consumption, resulting in an improved cardiac efficiency. AT-001 is presently being studied in ARISE-HF, a global phase III pivotal study in diabetic cardiomyopathy patients at high risk of progression to overt heart failure.
Arcturus Therapeutics Inc., of San Diego, said it received new manufacturing financial support and potential vaccine purchases up to $220 million from the Singapore’s Economic Development Board (EDB) that includes a limited recourse loan of $45 million and terms for ARCT-021 vaccine purchases of up to $175 million. The loan will be repaid through royalties on future ARCT-021 commercial sales. If ARCT-021 does not obtain regulatory approval, the loan will be forgiven.
Biomed X GmbH, of Heidelberg, Germany, said it added a further research program in the field of immunology to the existing collaboration with Janssen Research & Development LLC, one of the Janssen Pharmaceutical Cos. of New Brunswick, N.J.-based Johnson & Johnson. The new project will focus on the development of novel methodologies to systematically profile and dissect complex tissue microenvironments. The goal is to identify components that induce pro-resolution responses in tissue-resident innate immune cells to restore homeostasis in autoinflammatory diseases such as psoriasis, systemic lupus erythematosus, rheumatoid arthritis or inflammatory bowel disease.
Bridge Biotherapeutics Inc., of Seongnam, South Korea, reported it has mutually agreed with Boehringer Ingelheim GmbH, of Ingelheim, Germany, on the termination of their July 2019 collaboration and license agreement to develop BBT-877 for the treatment of idiopathic pulmonary fibrosis and various fibrotic diseases. Bridge Biotherapeutics will regain all rights to BBT-877.
Cannabics Pharmaceuticals Inc., of Tel Aviv, Israel, said it completed a preclinical study using its RCC-33 formulation on human biopsies, obtained under a Helsinki Committee-approved protocol. The results show an antitumor synergistic effect of the active compounds, confirming the company' previously obtained data on the antitumor properties of the drug candidate when screened on colorectal cell lines.
Capricor Therapeutics Inc., of Los Angeles, said in collaboration with researchers at Johns Hopkins University, positive data from a preclinical study for a multivalent exosome-based mRNA vaccine for COVID-19 has been posted on the bioRxiv preprint server and will be submitted for publication. The data show that the multiplexed exosome-RNA vaccine expresses viral antigens that induce cellular immunity and antibody responses to multiple proteins of SARS-CoV-2. The company said it is planning to meet with the FDA to discuss next steps for a clinical development strategy for the vaccine.
Cellink AB, of Goteborg, Sweden, will take part in the Triankle consortium, made up of 12 European organizations, which aims to develop 3D bioprinted personalized scaffolds for tissue regeneration of ankle joints. The research project in regenerative medicine has an overall budget of €5.9 million (US$6.9 million) and will be developed over four years, starting in January 2021. Cellink´s portion of the grant is €622,000. Specifically, the consortium aims to create personalized collagen- and gelatin-based implants manufactured with 3D technology for patients with tendinopathies such as Achilles tendon partial ruptures and cartilage injuries.
Galmed Pharmaceuticals Ltd., of Tel Aviv, Israel, and Mybiotics Pharma Ltd., of Rehovot, Israel, signed a research and development collaboration agreement to identify and optimize the selected microbiome repertoire associated with the response to Galmed’s investigational Aramchol (arachidyl amido cholanoic acid). The research will also focus on development of a standalone microbiome-based treatment for nonalcoholic steatohepatitis and fibrosis. Terms were not disclosed.
Homology Medicines Inc., of Bedford, Mass., said Pfizer Inc., of New York, agreed to make a $60 million equity investment in Homology. Pfizer will purchase 5 million shares of Homology’s common stock at a price of $12 each as part of the Pfizer Breakthrough Growth Initiative, made public earlier this year. The purchase is expected to close on or about Nov. 10.
Intelgenx Corp., of Saint Laurent, Quebec, said International Journal of Clinical Pharmacy published a study on montelukast Versafilm’s effect on neurological aging. The study builds on a similar study conducted in 2017 that suggested that montelukast, now at the phase IIa stage for Alzheimer’s disease, could alleviate the cognitive decline associated with human aging, the company said. Montelukast is a leukotriene receptor antagonist.
Lift Biosciences Ltd., of London, said its cell therapy, Neutrophil Only Leukocyte Infusion Therapy (N-LIfT), has demonstrated migration to the human pancreatic tumor site, successfully infiltrating the orthotopically grown tumor in nude mice. The tumor was grown over two weeks before mice were given a single dose of 2 billion N-LIfT cells via the tail-vein, the company said. N-LIfT is produced from the stem cells of exceptional cancer killing donors.
Medison Pharma Ltd., of Petach Tikva, Israel, signed an exclusive distribution agreement with Deciphera Pharmaceuticals Inc., of Waltham, Mass, to commercialize Qinlock, a switch-control tyrosine kinase inhibitor, in Canada and Israel for the treatment of fourth-line gastrointestinal stromal tumor (GIST). The product has been approved in Canada as of June for the treatment of adult patients who have received prior treatments and is expected to be launched by Medison Canada in the fourth quarter of this year. Medison Israel is planning to submit Qinlock for registration during the first quarter of next year and commence an early access program for Israeli patients immediately after. Qinlock has also been approved by the FDA for fourth-line GIST.
Ossianix Inc., of Philadelphia, disclosed the results of a nonhuman primate study demonstrating best-in-class blood-brain barrier (BBB) delivery by single-domain antibodies. The Ossianix VNAR shuttle TXP-1 showed efficient transfer into all six regions of the brain examined, with the hippocampus showing a more than 30-fold increase in levels of the molecule compared to the control. The TXP-1 shuttle was administered at a dose of 1.35 mg/kg by intravenous injection and reached a concentration of 4 nM in the brain after 20 hours. The data will be presented at the Virtual PEGS Summit Europe meeting on Nov. 11.
Phoremost Ltd., of Cambridge, U.K., and Oxford Biomedica plc, of Oxford, U.K., signed a discovery collaboration to develop next-generation CAR T-cell therapies. Financial details of the agreement were not disclosed. Phoremost will deploy its in-house expertise and next-generation phenotypic screening platform, Siteseeker, to identify therapeutic candidates for Oxford Biomedica’s Lentivector gene therapy delivery system.
Stembiosys Inc., of San Antonio, Texas, and Cartox Inc., of Ann Arbor, Mich., disclosed the publication of research in Nature Scientific Reports demonstrating that Cellvo Matrix Plus, an extracellular matrix technology derived from human perinatal stem cells, supports rapid functional and structural maturation of human induced pluripotent stem cell-derived cardiomyocytes in culture and enables high throughput cardiotoxicity screening using mature human cardiomyocytes. The paper is titled "Human perinatal stem cell derived extracellular matrix enables rapid maturation of hiPSC-CM structural and functional phenotypes."
Taigen Biotechnology Co. Ltd., of Taipei, signed an exclusive agreement with GPCR Therapeutics Inc., of Seoul, South Korea, for the continued development of burixafor worldwide and the commercialization of Taigexyn (nemonoxacin) in South Korea. Burixafor is a CXCR4 inhibitor currently under clinical development. It can be used as a stem cell mobilizer for hematopoietic stem cell transplantation and a chemosensitizer in hematological and solid tumors. Taigexyn is an antibiotic for the treatment of bacterial infections, including those caused by drug-resistant bacteria. Under the terms of the agreement, GPCR will be wholly responsible for the development, registration and commercialization of Taigexyn in South Korea and burixafor worldwide. Apart from up-front fees, Taigen will receive shares of GPCR as well as potential milestone and royalty payments.
Ultragenyx Pharmaceutical Inc., of Novato, Calif., disclosed plans to build a new large-scale gene therapy manufacturing facility in Bedford, Mass. The facility will enable in-house manufacturing of the company’s pipeline of clinical-stage adeno-associated virus-based gene therapies, including DTX-301 for ornithine transcarbamylase deficiency, DTX-401 for glycogen storage disease type Ia, and UX-701 for Wilson disease, as well as other preclinical programs. The company said it will continue to leverage some contract manufacturing organizations in addition to its own manufacturing facility.