AC Immune SA, of Lausanne, Switzerland, said it received a milestone payment from its partner, Life Molecular Imaging, in connection with the start of a phase II study of the tau positron-emission tomography tracer PI-2620, developed using the company's Morphomer discovery platform in a research collaboration with Life Molecular Imaging. It is designed to bind to tau deposits which, along with beta-amyloid plaques, are believed to represent a critical pathological hallmark of Alzheimer's disease. Tau deposits also play an important role in other neurodegenerative diseases. The U.K. phase II longitudinal study is expected to last approximately three years, with the overall goal being to evaluate PI-2620 as a targeted radiopharmaceutical for the detection of tau deposits in the human brain. The data generated are intended to be used for obtaining U.S. and European regulatory approval.
Adaptive Biotechnologies Corp., of Seattle, said it entered a global agreement with Amgen Inc., of Thousand Oaks, Calif., for the use of its next-generation sequencing-based Clonoseq assay to assess minimal residual disease (MRD) across multiple drug development programs within the Amgen hematology portfolio. Under the terms of the four-year agreement, Adaptive will receive annual development fees in addition to sequencing payments and regulatory milestones in exchange for providing MRD testing and analysis for ongoing and future clinical trials.
Anabios Corp., of San Diego, said it will receive an FDA grant to further develop its drug discovery platform utilizing cardiomyocytes. Funds from the grant will be used to develop a preclinical biomarker to identify the pro-arrhythmia risk of potential drugs based on contractility measurements in human adult primary cardiomyocytes. The company said its cardiomyocyte research has yielded highly translational information about cellular properties and drug-induced variations in cardiac function. Anabios' human cardiomyocyte contractility model simultaneously evaluates pro-arrhythmia risks from the measurement of drug-induced changes in several parameters. It plans to further combine the parameters into a single biomarker that can be used to predict pro-arrhythmia risk in advance of clinical studies. The outcome of the research project should provide critical information for drug developers and regulators with respect to the cardiac risk of drugs at the preclinical stages of drug development, the company said.
Asklepios Biopharmaceutical Inc. (Askbio), of Research Triangle Park, N.C., acquired the tech assets of St. Cloud, Minn.-based Rovermed Biosciences LLC, which develops a platform for nanotechnology cargo delivery of therapeutics into the nucleus of diseased cells. Askbio will integrate the company's technology and assume all of Rovermed's assets. Terms were not disclosed. Askbio recently landed $235 million in fresh funding. (See BioWorld, April 12, 2019.)
Bioq Pharma Inc., of San Francisco, entered a development, supply and distribution agreement with Alpharetta, Ga.,-based Avanos Medical Inc. to gain approvals and then commercialize a self-contained, infusible, non-opioid, postoperative pain product in the U.S., Canada and Mexico. Avanos develops, manufactures and markets its products in more than 90 countries. Terms were not disclosed.
Clene Nanomedicine Inc., of Salt Lake City, received a $1.37 million grant from the Fight Motor Neuron Disease Foundation of Australia. The grant will be used to fund a phase II trial assessing the efficacy and safety of Clene's nanocatalyst, CNM-Au8, designed to slow the disease progression of amyotrophic lateral sclerosis. The study will be conducted at two sites in Sidney.
Crispr Therapeutics AG, of Zug, Switzerland, and Cambridge, Mass.,-based Viacyte Inc. released positive in vitro clinical data showing the CyT49 pluripotent stem cell line can be successfully edited with CRISPR. The line is used to generate islet progenitors for clinical trials investigating type 1 diabetes. The companies are jointly working to develop and commercialize products targeting type 1 diabetes.
Eleusis Ltd., of New York, published preclinical data in Scientific Reports on serotonin 2A receptor (5-HT2A) agonists. In an apolipoprotein E knockout mouse model of cardiovascular disease, the 5-HT2A receptor agonists reduced expression of pro-inflammatory biomarkers – IL-6, vcam1 and TNF-alpha mRNA – in vascular tissues.
Evoke Pharma Inc., of Solana Beach, Calif., manufactured commercial scale batches of its nasal spray, Gimoti (metoclopramide), for gastroparesis with its partner, Thermo Fisher Scientific Inc., of Waltham, Mass. Evoke plans to collect chemistry, manufacturing and controls data from the batches that were requested in the complete response letter from the FDA. The company plans to resubmit the NDA for Gimoti in the fourth quarter of this year. (See BioWorld, April 3, 2019.)
Fujifilm Corp., of Tokyo, has exercised its license option from a deal with Cynata Therapeutics Ltd., of Melbourne, Australia, in graft-vs.-host disease. Fujifilm now has exclusive, worldwide license to develop and commercialize Cynata's lead mesenchymal stem cell product, CYP-001, which just completed a phase I trial in which all safety and efficacy endpoints were reached. Cynata received $3 million in cash up front and Fujifilm will pay for further product development activities along with assuming responsibility for regulatory submissions and commercialization costs. The initial agreement was reached three years ago. (See BioWorld, Sept. 15, 2016.)
Happify Inc., of New York, and Sanofi SA, of Paris, are co-developing a digital therapeutic to address key co-morbidities, including depression and anxiety, of patients with multiple sclerosis, which will be based on Happify's digital platform. Financial terms of the deal weren't disclosed.
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa., received a $4.6 million grant from the NIH to support its preclinical development of therapies for multidrug-resistant infections using its DNA-encoded monoclonal antibodies platform.
Knopp Biosciences LLC, of Pittsburgh, reported results of preclinical experiments demonstrating that KB-3061, the lead molecule in its ion channels platform, restored the function of Kv7 potassium channels in cells engineered to express gene variants that cause the rare, neonatal disease KCNQ2 epileptic encephalopathy. Cells transfected to express three highly recurrent missense mutations in the KCNQ2 gene demonstrated strong suppression of function, as measured by significantly reduced potassium current through the mutated Kv7.2 channels. When treated with KB-3061, the function of the mutated channels was fully restored, producing normal Kv7.2 channel current density. Data were presented at the Epilepsy Precision Medicine Conference in Washington.
Perspectum Inc., of Dallas, was awarded $250,000 from the FDA to qualify its biomarkers for nonalcoholic steatohepatitis (NASH) in collaboration with Naim Alkhouri at the Texas Liver Institute. The study will qualify the imaging markers cT1 and PDFF as drug development tools in NASH, initially as diagnostic screening biomarkers to identify patients for inclusion in clinical trials, then as pharmacodynamic efficacy biomarkers to detect clinically meaningful change, with the ultimate objective being approval as non-invasive surrogate endpoints.
Procarta Biosystems Ltd., of Stevenage, U.K., received an award of $2.2 million up front, with another $7 million tied to project milestones, from Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator (CARB-X). The funds will be used to support Procarta's oligonucleotide-based antimicrobial platform, including its lead preclinical asset, PRO-202, being developed for complicated urinary tract infections and complicated intra-abdominal infections.
Sensorion SA, of Montpellier, France, reported preclinical data at the American Academy of Otolaryngology, Head and Neck Surgery/Foundation in New Orleans showing a strong correlation between the amplitude loss of Distortion Product OtoAcoustic Emissions and changes in circulating Prestin levels in an animal model of variable and mild noise-induced hearing loss. Those data further support potential of the inner ear-specific protein Prestin as a circulating biomarker. Sensorion is developing seliforant (SENS-111), a histamine type 4 receptor antagonist, for inner ear diseases.
Sienna Biopharmaceuticals Inc., of Westlake Village, Calif., filed a voluntary petition to allow restructuring under Chapter 11 of the bankruptcy code. (See BioWorld, July 31, 2018.)
Tonix Pharmaceuticals Holding Corp., of New York, licensed TNX-1700 (recombinant trefoil family factor 2) for the treatment of gastric and pancreatic cancers from Columbia University. Financial terms of the deal weren't disclosed.
Trialspark and Pfizer Inc., both of New York, established a multiyear collaboration to increase the use of community doctors who have not previously been study investigators in Pfizer's clinical trials. Trialspark will provide the training, technology, resources and research staff to establish clinical trial sites that allow patients to join a clinical trial under the continued care of their own doctor.
University of California, of Berkeley, Calif., University of Vienna and Emmanuelle Charpentier were awarded a new CRISPR/Cas9 patent from the U.S. Patent and Trademark Office, covering compositions comprising single-molecule DNA-targeting RNAs or nucleic acids encoding single molecule DNA-targeting RNAs as well as methods of targeting and binding a target DNA, modifying a target DNA, or modulating transcription from a target DNA with a complex that comprises a Cas9 protein and single-molecule DNA-targeting RNA.
Vesselon Inc., of Norwalk, Conn., said it secured an exclusive worldwide license agreement with Temple University for technology that enables viral therapy to be delivered repeatedly by systemic injection. The technology was developed by Pier Paolo Claudio, while serving as director of the Molecular Therapeutic Program at Temple, who identified a means by which Vesselon's microbubbles could serve as an effective drug complex with genes loaded within safe viral particles to treat specific tissue sites for therapeutic purposes.
Zymeworks Inc., of Vancouver, British Columbia, plans to delist from the Toronto Stock Exchange in Canada on or around Oct. 1. Shares of the company will continue to be traded on the New York Stock Exchange.