Abeona Therapeutics Inc., of New York, said it has retained Jefferies LLC as its financial advisor to assist with a strategic options review focused on advancing the company's mission and maximizing stakeholder value.
Adamis Pharmaceuticals Corp., of San Diego, said it completed pharmacokinetic studies comparing its naloxone injection product candidate, Zimhi (naloxone) injection, to a generic naloxone comparator. In June, the company amended the new drug application for the product to remove any reference to Evzio (Kaleo Inc.) and withdrew the Paragraph IV certification relating to that product. The data for the study has been recorded and is complete and the database will be forwarded to the FDA this month.
Amarin Corp. plc, of Dublin, said the European Society of Cardiology and the European Atherosclerosis Society have updated their clinical practice guidelines for the management of dyslipidemias. This 2019 update incorporates findings from the REDUCE-IT cardiovascular outcomes study and includes the recommendation that icosapent ethyl, 2 g twice a day, should be considered for patients with cardiovascular disease who have triglyceride levels 135 mg/dL to 499 mg/dL despite statin treatment, which places them at high risk of cardiovascular events, such as heart attack, stroke or death.
Batavia Biosciences BV, of Leiden, the Netherlands, signed a license agreement to utilize Cambridge, U.K.-based Horizon Discovery Group plc's GS knockout CHO K1 cell line expression system for the development of high-yield antibody-expressing cell lines. Initially, Batavia will deploy the system for production of a Zika virus neutralizing antibody, working in collaboration with Vanderbilt University Medical Center and Idbiologics Inc., of Nashville, Tenn., focused on developing human antibodies for the prevention, treatment and cure of infectious diseases. Terms were not disclosed.
Brickell Biotech Inc., of Boulder, Colo., completed its merger with Vical Inc., of San Diego, following approval by Vical's stockholders. Brickell commenced trading Tuesday on Nasdaq under the ticker BBI, with shares closing at $4.69. Vical contributes about $35 million to the combined company in addition to a research and development financing arrangement with Novaquest Capital Management that provides $25 million in funding.
Bristol-Myers Squibb Co., of New York, and Pfizer Inc., of New York, disclosed findings from NAXOS (EvaluatioN of ApiXaban in strOke and Systemic embolism prevention) in patients with nonvalvular atrial fibrillation (NVAF) in the real-life setting in France, the largest real-world data analysis on oral anticoagulant (OAC) effectiveness and safety in Europe among patients with NVAF. NAXOS is a retrospective cohort analysis including nearly all patients in France ages 18 or older with NVAF, newly initiating one of the OACs between 2014 and 2016 (n=321,501). In the analysis, Eliquis (apixaban, Bristol-Myers Squibb Co.) use was associated with a lower rate of major bleeding compared to a vitamin K antagonist (hazard ratio [HR]: 0.49, 95% confidence interval [CI]: 0.46-0.52), rivaroxaban (Xarelto, Johnson & Johnson; HR: 0.63, 95% CI: 0.58-0.67) and dabigatran (Pradaxa, Boehringer Ingelheim GmbH; HR: 0.85, 95% CI: 0.76-0.95). The data were featured as a late-breaking oral presentation at the European Society of Cardiology Congress 2019 in Paris.
Clinigen Group plc, of Burton Upon Trent, U.K., and Cheplapharm Arzneimittel GmbH, of Mesekenhagen, Germany, signed an exclusive distribution agreement for chemotherapy products Etopophos (etoposide phosphate) and Vepesid (etoposide) in Australia and New Zealand. Cheplapharm acquired the global rights to the products from Bristol-Myers Squibb Co., of New York, in August 2018. Cheplapharm has global capabilities in manufacturing and distribution and will continue to supply and provide access to those medicines for patients.
Cocrystal Pharma Inc., of Bothell, Wash., presented preclinical data with CC-42344 at the ISIRV: Options X for the Control of Influenza Conference in Singapore. Cocrystal's broad-spectrum anti-influenza preclinical lead molecule, CC-4234, targets the cap-binding PB2 domain and is active against a panel of seasonal, pandemic and Tamiflu (oseltamivir phosphate, Roche Holding AG)-resistant influenza A strains. The firm also offered in vitro characterization and mechanism of action of novel PB2 inhibitors.
Correvio Pharma Corp., of Vancouver, British Columbia, disclosed results from the Spectrum study evaluating Brinavess (vernakalant hydrochloride, intravenous), the company's antiarrhythmic drug for the rapid conversion of recent onset atrial fibrillation, at the European Society of Cardiology Congress in Paris. Spectrum was conducted as part of the follow-up measures agreed to with the EMA in 2010. In the prospective and retrospective, international, multicenter, observational registry, 1,778 patients with 2,009 treatment episodes were enrolled to describe patients receiving Brinavess and to characterize normal conditions of use and dosing, and quantify possible medically significant risks associated with the use of Brinavess in real-world clinical practice. The data for Spectrum were provided by 53 participating hospitals in the EU and demonstrated that treatment with Brinavess successfully converted 70.2% of all treated episodes. Treatment with Brinavess also showed a median time to conversion of 12 minutes from start of first infusion among patients who converted. Cardioversion to sinus rhythm was 70.4% and 65.4% of treatment episodes in non-surgery and post-cardiac surgery patients, respectively. Brinavess was administered in the emergency department in 64.2% of cases, with a median stay of 7.5 hours in that setting.
Daiichi Sankyo Co. Ltd., of Tokyo, reported through its European arm one-year outcomes results from a study of 24,962 patients with nonvalvular atrial fibrillation (NVAF) treated with Lixiana (edoxaban), including elderly NVAF patients and those with and without a history of intracranial hemorrhage. One-year follow-up data from the ETNA-AF (Edoxaban Treatment in routiNe clinical prActice) study were presented at the European Society of Cardiology Congress in Paris. A new analysis, which reported the outcomes of 24,962 Lixiana-treated patients with NVAF at one year follow-up, supports the treatment's safety and efficacy profile in elderly and very elderly AF patients. The majority of patients were 65 or older, the company noted.
Emergent Biosolutions Inc., of Gaithersburg, Md., was awarded a contract valued at about $2 billion over 10 years by the U.S. Department of Health and Human Services to continue supplying Acam2000, a live smallpox vaccine, to the Strategic National Stockpile. The contract, awarded by the Office of the Assistant Secretary for Preparedness and Response, includes a one-year base period of performance valued at approximately $170 million and nine option years. In October 2017, Emergent acquired Acam2000, the only FDA-approved smallpox vaccine, from Paris-based Sanofi SA and assumed fulfilling an existing 10-year contract for Acam2000 with the CDC valued at up to $425 million.
Helsinn Group, of Lugano, Switzerland, and Blanver Farmoquímica e Farmacêutica SA, of Sao Paulo, Brazil, signed distribution and license agreements for pracinostat, a pan histone deacetylase inhibitor, in South America. Pracinostat is in a phase III trial as combination therapy with azacitidine for treating adults with newly diagnosed acute myeloid leukemia who are unfit for intensive induction chemotherapy. It's also in an open-label phase II study in combination with azacitidine for treating naïve patients with high-risk myelodysplastic syndromes.
ITM Isotopen Technologien Munchen AG, of Munich, Germany, and Duchembio Co. Ltd., of Seoul, South Korea, agreed to license and develop Solucin targeted radionuclide therapy in South Korea. The companies plan to recruit patients for a local clinical study for Solucin next year to compare it to everolimus in patients with inoperable, progressive, somatostatin receptor-positive neuroendocrine tumors of gastroenteric or pancreatic origin. The study's primary endpoint is progression-free survival. Solucin targets tumor cells with ionizing radiation.
Kamada Ltd., of Rehovot, Israel, extended its strategic supply agreement with Takeda Pharmaceutical Ltd., of Tokyo, for Glassia, a liquid alpha1-proteinase inhibitor, through 2021. Kamada projects revenues of $155 million to $180 million for this year and next. Takeda will pay Kamada 12% on net sales through August 2025 and 6% afterward until 2040, with a minimum of $5 million annual for each year from 2022 to 2040. Glassia is for treating adults with clinically evident emphysema due to severe congenital alpha-1 antitrypsin deficiency.
Lineage Cell Therapeutics Inc., of Carlsbad, Calif., said preclinical data published in Stem Cells and Development demonstrated that human pluripotent stem cell-derived retinal tissue was successfully introduced and delivered into the subretinal space of a large eye model following a pars plana vitrectomy. Researchers optimized the immunosuppression regimen to enable the grafts to survive for several weeks. In addition, the team demonstrated tumor-free maturation of the transplanted stem cell-derived retinal tissue and establishment of graft-host axonal connectivity and graft-host synaptic connectivity.
Obseva SA, of Geneva, reported results from a mechanism of action trial of its oral oxytocin receptor antagonist, nolasiban, assessing its effects on uterine contractions and endometrial blood flow, both of which are known to be associated with uterine receptivity and embryo implantation, and ultimately pregnancy and live birth following in vitro fertilization. A single oral dose produced measurable and durable effects, with a reduction in the frequency of uterine contractions and a marked and sustained increase in endometrial blood flow. Within 24 hours of administration, statistically significant changes were observed in the expression of a set of endometrial genes that have been described as potentially important in endometrial receptivity and embryonic implantation. Obseva said the data represent the first in vivo demonstration of the effect of oxytocin receptor inhibition on endometrial blood flow and receptivity-related gene expression.
Phasebio Pharmaceuticals Inc., of Malvern, Pa., presented data from a pharmacodynamics evaluation of PB-2452, a reversal agent for antiplatelet drug ticagrelor, at the ESC Congress in Paris, showing the drug provided immediate and sustained reversal activity. Results from three different assays of platelet function, including Verifynow Prutest, demonstrated a high degree of correlation.
Preveceutical Medical Inc., of Vancouver, British Columbia, said it reached a milestone in its research program for development of non-addictive analgesics encompassing the design, synthesis and cell-based screening of lead peptide constructs intended for development as non-addictive painkilling medicines as a replacement for highly addictive analgesics. The company said it confirmed that the subset of peptides demonstrated potency in a well-accepted functional dose response assay that is on par with clinically available agents, such as morphine.
Protalix Biotherapeutics Inc., of Carmiel, Israel, said it received a deficiency letter from NYSE American LLC, stating that it is not in compliance with continued listing standards, as it reported a stockholders' equity deficiency as of June 30, 2019, and net losses in its five most recent fiscal years ending Dec. 31, 2018. The company said it intends to submit a detailed plan of compliance.
Roche Holding AG, of Basel, Switzerland, and Spark Therapeutics Inc., of Philadelphia, said Roche extended the offering period of its previously announced tender offer to purchase all the outstanding shares of Spark at $114.50 apiece to Oct. 1, at 5 p.m. EST. The extension was made to provide additional time for the U.S. FTC and the U.K. Competition and Markets Authority to complete reviews of the pending acquisition. The companies inked the potential $4.8 billion deal in February. (See BioWorld, Feb. 26, 2019.)
Sandoz AG, of Holzkirchen, Germany, a division of Novartis AG, said it entered a global commercialization agreement with Polpharma Biologics SA, of Gdańsk, Poland, for a proposed biosimilar of Tysabri (natalizumab, Biogen Inc.), for treating relapsing-remitting multiple sclerosis. Under the terms, Polpharma will maintain responsibilities for development, manufacturing and supply of proposed biosimilar natalizumab, while Sandoz will commercialize and distribute the medicine in all markets upon approval through an exclusive global license. Other specific terms of the agreement are confidential.
Seqirus, of Summit, N.J., the vaccines business of CSL Ltd., presented data at the Options for the Control of Influenza Conference in Singapore, demonstrating that circulating influenza B/Victoria (B/Vic) viruses are a closer match to cell-based B/Vic vaccine viruses compared with egg-based B/Vic vaccine viruses. The data build on a previous analysis that evaluated the level of match between circulating influenza A (H3N2) viruses and corresponding H3N2 cell and egg-based vaccine viruses, highlighting the potential role of egg-adaptation in varying vaccine effectiveness.
Sutrovax Inc., of Foster City, Calif., said it received an award from CARB-X for up to $15 million in nondilutive funding to develop a universal vaccine to prevent infections caused by Group A Strep bacteria, which include pharyngitis, impetigo and necrotizing fasciitis. The award commits initial funding of up to $1.6 million and up to $15 million in total funding available upon achievement of development milestones.
The Institute for Clinical and Economic Review is asking for comment and suggested refinements on a planned review of the comparative clinical effectiveness and value treatments for crizanlizumab and voxelotor, both for sickle cell disease. Crizanlizumab, from Basel, Switzerland-based Novartis AG, is a P-selectin inhibitor that is under FDA priority review. Global Blood Therapeutics Inc., of South San Francisco, said it will file an NDA for voxelotor, an HbS polymerization inhibitor, by the end of this year. Comments should be submitted to email@example.com by 5 pm on Sept. 20.
The U.S. Patent and Trademark Office awarded a CRISPR/Cas9 patent to the University of California, University of Vienna and Emmanuelle Charpentier covering compositions of single molecule DNA-targeting RNA and a Cas9 protein or nucleic acid encoding the Cas9 protein.
Valo Therapeutics Ltd., of Oxford, U.K., said it licensed from Helsinki University access to Peptienv technology that enables the coating of enveloped viruses by neoantigen-peptides. The technology is expected to improve the therapeutic response to enveloped oncolytic viruses in development for the treatment of cancer. Terms of the deal were not disclosed.
Velabs Therapeutics GmbH, of Heidelberg, Germany, said it entered an agreement with Alytas Therapeutics GmbH, of Jena, Germany, to develop modulatory and functional antibodies for an immune-based therapy against obesity. The collaboration will leverage Velabs' microfluidic-based technology for rapid functional antibody screening. Under the terms, Velabs will be entitled to royalties from the worldwide sales of products that result from the collaboration.
Vertex Pharmaceuticals Inc., of Boston, said it agreed to acquire privately held Semma Therapeutics Inc., of Cambridge, Mass., for $950 million in cash. Semma, which is developing stem cell-derived human islets as a potentially curative treatment for type 1 diabetes, will become a separate operating subsidiary of Vertex. The deal is set to close in the fourth quarter.
Viriom Inc., of San Diego, said it will be supplemented by an NIH/National Institute of Allergy and Infectious Diseases and Division of AIDS collaboration with LEAP (Long-Acting/Extended Release Antiviral Resource Program). As part of the program, Viriom will partner with the pharmacology core at the University of Liverpool in the U.K., which will apply advanced quantitative pharmacology methods to the analysis of the long-acting injection version of elsulfavirine (VM-1500A), as well as a once-weekly oral dose form of elsulfavirine.