Aetion Inc., of New York, and McKesson Corp., of Irving, Texas, said they established a collaboration focused on advancing the use of real-world evidence in cancer research. The joint solutions combine the Aetion evidence platform with data from McKesson's Iknowmed oncology electronic health record system to power regulatory-grade outcomes research. They will first be made available to the team of researchers at Brigham and Women's Hospital who are leading the FDA demonstration project, RCT DUPLICATE, in order to replicate oncology randomized controlled trials with real-world data. Aetion and McKesson are also both part of the Friends of Cancer Research Real-World Evidence pilot project to assess several front-line treatment regimens in real-world patients with advanced non-small-cell lung cancer, which will inform the FDA's acceptance of real-world evidence for drug approvals.

Botanix Pharmaceuticals Ltd., of Sydney, said it was awarded an AU$50,000 (US$33,788) Innovation Connections Grant from the Australian government to accelerate its medicinal chemistry program targeting the creation of synthetic analogues of cannabidiol and the investigation of cannabidiol's antimicrobial activity. The program is being conducted in collaboration with The University of Queensland's Institute for Molecular Bioscience.

The Critical Path Institute, or C-Path, and CDISC said they released a global foundational standard that describes how to present data for natural history and efficacy studies conducted in animals that are submitted to applications under the FDA Animal Rule, which provides a regulatory framework for the approval of drugs and biological products when human efficacy studies are not ethical or feasible. The standard is designed to streamline data reporting to the agency to enhance learnings from animal studies that may accelerate the development of human therapies. The standard, released in the form of an implementation guide and model for data managers, statisticians, programmers and study managers, is freely available on the CDISC website.

Crispr Therapeutics AG, of Zug, Switzerland, and KSQ Therapeutics Inc., of Cambridge, Mass., said they inked a license agreement giving Crispr Therapeutics access to KSQ intellectual property (IP) for editing certain gene targets in its allogeneic oncology cell therapy programs and giving KSQ access to Crispr Therapeutics' IP for editing gene targets identified by KSQ as part of its engineered tumor infiltrating lymphocyte, or eTIL, cell programs. Gene targets within the scope of the agreement were identified using KSQ's Crispromics drug discovery engine with the goal of identifying genetic edits that could enhance the functionality and quality of adoptive cell therapies in oncology. Financial terms of the agreement were not disclosed.

Diamyd Medical AB, of Stockholm, said it formed an agreement with the Norwegian University of Science and Technology for an investigator-sponsored phase II trial of the diabetes vaccine candidate Diamyd administered directly into the lymph node in approximately 15 adults newly diagnosed with latent autoimmune diabetes and not yet on insulin therapy. Diamyd Medical will contribute the study drug and internal expertise. The application for the trial, which is expected to be conducted at clinics in Norway and Sweden, was submitted to the Norwegian Medicines Agency. The trial will evaluate safety along with immunological and clinical response over one year.

Eicosis LLC, of Davis, Calif., said it was awarded a grant from the National Institute of Drug Abuse (NIDA) in relation to the NIH initiative Helping to End Addiction Long-Term, or HEAL, that will help to support phase I and II studies of a non-opioid compound that showed effectiveness in treating pain when tested preclinically. EC-5026 is designed to inhibit soluble epoxide hydrolase, an enzyme in the metabolism of fatty acids, consequently treating pain by stabilizing natural analgesic and anti-inflammatory mediators. The work originated in the laboratory of company founder and CEO Bruce Hammock, professor of entomology and of the Comprehensive Cancer Center at the University of California, Davis. The grant is associated with a cooperative agreement between Eicosis and NIDA and awarded in two parts, with $6 million in funding over the first two years to establish specific milestones and a potential $9 million over three additional years based on the achievement of the milestones.

Eleusis Benefit Corp. PBC, of New York, said research published in Elsevier's Life Sciences demonstrated the anti-inflammatory properties of serotonin 2A receptor (5-HT2A) agonists in a translational model of chronic asthma, a disease characterized by chronic allergic inflammatory response. Eleusis is pursuing the therapeutic potential of 5-HT2A agonists, also known as psychedelics, to treat inflammatory diseases. The study highlighted the capacity of 5-HT2A agonists to reverse airway hyperresponsiveness and to reduce associated pulmonary inflammation and mucus overproduction and also suggested the capability of 5-HT2A agonists to reverse airway structural remodeling. In the mouse model of chronic asthma, the study observed a 70% reduction in asthma-induced collagen deposition, associated with pulmonary fibrosis.

Enterprise Therapeutics Ltd., of Brighton, U.K., said it was awarded up to £5.7 million (US$7 million) in milestone-related funding under the Therapeutics Development Award program from the Cystic Fibrosis (CF) Foundation to support development up to the end of phase II for ETD-002, a TMEM16A potentiator targeting the treatment of individuals with CF independent of their transmembrane conductance regulator mutation type. By enhancing the activity of TMEM16A, ETD-002 is designed to increase anion and fluid flow into the airways, thinning the mucus and increasing its clearance from the airways. (See BioWorld, April 13, 2018.)

Generex Biotechnology Corp., of Miramar, Fla., said it plans a merger transaction with a public company to list the common stock of its subsidiary, Nugenerex Immuno-Oncology, as an independent, publicly traded company. Generex said an annual report on Form 10-K will include audited financial statements for the legal documentation and filing for the merger, with shares of Nugenerex expected to be quoted on a public exchange by the end of November. The merger is designed to help advance the company's Ii-Key technology and AE-37 immunotherapeutic vaccine program, currently enrolling a phase II trial to establish the recommended dose in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) to enhance tumor-specific immune response and show efficacy in individuals with advanced triple-negative breast cancer.

Ichnos Sciences Inc., of Paramus, N.J., a spin-off of Glenmark Holding SA, said it has launched and will focus on its pipeline that includes five clinical-stage assets in oncology, autoimmune disease and pain by deploying its bispecific antibodies based on the T-cell receptor platform. The company has two research centers, a GMP biologics manufacturing facility and 350 employees. Its CD38xCD3 bispecific antibody, GBR-1342, is under development for relapsed and refractory multiple myeloma. Also, GBR-1302, a HER2xCD3 bispecific antibody, is being evaluated for HER2-positive breast cancer. Data readouts for both candidates are expected in 2021. Also in the pipeline is GBR-830, an anti-OX40 monoclonal antibody in phase IIb development for the treatment of moderate to severe atopic dermatitis, and GRC-27864, a non-opioid, potent, selective and orally bioavailable inhibitor of microsomal prostaglandin E synthase-1 being evaluated in phase IIb studies for osteoarthritic pain. GRC-17536, a non-opioid TRPA1 antagonist, has completed a phase IIa proof-of-concept study in patients with painful diabetic peripheral neuropathy.

Oncolytics Biotech Inc., of Calgary, Alberta, said the results of a metanalysis of 13 clinical studies of the company's systemically delivered oncolytic reovirus, pelareorep, were presented at the International Oncolytic Virus Conference. The analyses examined the effectiveness of viral replication within the tumors of patients treated systemically with pelareorep. The data demonstrated that, unlike other oncolytic viruses that require intratumoral delivery, intravenous systemic delivery of pelareorep resulted in 81% of patient tumor samples across multiple types of cancer testing positive for virus replication, with no infection in normal tissue. Those results are from studies across a broad range of solid and liquid tumors, including metastatic disease.

Pliant Therapeutics Inc., of South San Francisco, reported the publication of a study evaluating the use of a positron emission tomography (PET) tracer for the detection of idiopathic pulmonary fibrosis through the recognition of protein integrin avb6. The results of the pilot clinical study encourage evaluation of PET tracers for diagnosis, staging of disease, patient stratification in trials, treatment selection and monitoring of therapy. The study is published in Nature Communications.

Plus Therapeutics Inc., of Austin, Texas, said it received a $4.6 million payment from the Biomedical Advanced Research and Development Authority (BARDA) to reimburse the company for work performed during fiscal years 2012 through 2019, based on retrospective changes in indirect cost rates under a contract with BARDA. The company's nanotechnology platform is centered around the enhanced delivery of a variety of drugs using liposomal encapsulation technology. Its lead product candidate, Doceplus, is a protein-stabilized pegylated liposomal formulation of docetaxel.

New preclinical proof-of-concept data from Pneumagen Ltd., of St. Andrews, Scotland, showed its carbohydrate binding molecule significantly inhibited tumor growth in an orthotopic mouse xenograft model of human ovarian cancer. Pneumagen's platform generates engineered oligomers of monomeric carbohydrate-binding modules derived from carbohydrate-active enzymes. The technology is being used to find a universal treatment for respiratory tract infections and to research glycan-targeted cancer therapeutics.

Sanofi SA, of Paris, recently inaugurated its first digitally enabled, continuous manufacturing facility in Framingham, Mass., for biologics-based therapies. The facility, featuring paperless and data-driven technology, is designed to reduce time taken to move products from development labs to the manufacturing plant. Sanofi is also designing similar initiatives for Canada, Brazil, Ireland, France and Belgium.

Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, will divest a portfolio of about 30 over-the-counter and prescription pharmaceutical products it sells in the Near East, Middle East and Africa to Basel, Switzerland-based Acino International AG for more than $200 million. Acino acquires the rights, title and interest to products exclusive to the area and Takeda will manufacturing them. The deal is expected to close in the first quarter of next year. This is Takeda's third transaction in fiscal year 2019 since acquiring rare disease specialist Shire plc in January for $62 billion. (See BioWorld, Jan. 11, 2019.)

Preclinical results from Foster City, Calif.-based Terns Pharmaceuticals Inc. show its farnesoid X receptor (FXR) agonist, TERN-101, to treat nonalcoholic steatohepatitis (NASH) was preferentially delivered to the liver, enabling an effect on FXR and potentially mitigating non-liver-mediated adverse effects. TERN-101 and TERN-201, a semicarbazide-sensitive amine oxidase inhibitor, are being developed to treat NASH and were discovered and developed by Eli Lilly and Co., of Indianapolis.

Tel Aviv, Israel-based Therapix Biosciences Ltd.'s preclinical data evaluating THX-120, which contains nonpsychoactive cannabinoid cannabidiol (CBD) and palmitoylethanolamide (PEA), showed potential synergy between the compounds during in vitro tests to treat epilepsy and inflammatory conditions. The study was performed in a hepatocytes model of fat accumulation, where each was tested in a range of concentrations. In those tests, PEA showed no effect of its own, while the effect of CBD was enhanced by adding PEA, lowering the required effective concentration of CBD. "This is the first time that PEA was shown to exert a synergy toward CBD in an in vitro model," said Adi Zuloff-Shani, Therapix's chief technology officer.

No Comments