Amneal Pharmaceuticals LLC, of Bridgewater, N.J., and Intrexon Corp., of Germantown, Md., formed an exclusive channel collaboration to develop an improved production process for a complex active pharmaceutical ingredient (API). The goal of the collaboration is to develop a biological process for a targeted API production that may reduce the cost of production, leveraging Intrexon’s Ultravector platform and the company’s suite of integrated synthetic biology technologies.
Anavex Life Sciences Corp., of New York, said it was encouraged by the findings of a research report published in Neuroscience Letters, suggesting the results point to potential for ANAVEX 2-73 to treat amyotrophic lateral sclerosis (ALS). The data showed that sigma-1 receptor (S1R) agonists are effective in suppressing motor neuron degeneration and symptom progression in ALS animal models. One mechanism by which motor neuronal injury is caused involves inhibiting specific components of the mitochondrial electron transfer chain. Therapeutic measures aimed at protecting mitochondrial respiratory chain function may be useful in related familial and possibly other forms of ALS. Although ANAVEX 2-73 was developed to treat Alzheimer’s through potential disease modification, the compound may have a similar effect on respiratory cell mitochondria, according to Anavex, which said it will initiate preclinical studies of ANAVEX 2-73 in ALS models.
Argenta Discovery Ltd., of Harlow, UK, a unit of Galapagos NV, of Mechelen, Belgium, said it achieved a milestone in its collaboration partner Antabio, of Labège, France, in an antibacterial drug discovery project, triggering the drawdown of a €1.7 million (US$2.4 million) tranche of their seeding drug discovery award from the Wellcome Trust. The companies identified a lead series of pan-metallo beta-lactamase inhibitors that, in combination with carbapenems, restores antibacterial activity against clinical isolates whilst exhibiting no toxicity or developmental issues. The collaboration began in February 2013.
Bergenbio AS, of Bergen, Norway, said it was awarded a NOK13 million (US$2.2 million) grant from the Research Council of Norway’s user-driven research-based innovation program to help fund research into therapeutics to inhibit tumor epithelial-mesenchymal transition, or EMT, a driver of metastasis and a mechanism of drug resistance. The project is designed to implement state-of-the-art R&D strategies to identify and develop drug candidates with the potential to prevent and reverse acquired cancer drug resistance. In developing BGB324, its first-in-class selective Axl kinase inhibitor, Bergenbio showed that inhibiting the Axl/EMT pathway is effective against acquired drug resistant and aggressive cancers and identified new approaches to inhibiting EMT. (See BioWorld Today, Feb. 6, 2014.)
Biothera Inc., of Eagan, Minn., will present data at the Keystone Symposia on Inflammation, Infection and Cancer in Whistler, British Columbia, demonstrating a threshold presence of anti-beta glucan antibodies (ABA), IgG and IgM, are predictive of binding of the company’s cancer immunotherapy Imprime PGG to neutrophils and monocytes, which can then recognize and kill cancer cells. Previous research has demonstrated that opsonized Imprime PGG binds to complement receptor 3 on human neutrophils and monocytes and is dependent on the ABA-initiated classical pathway of complement activation. Studies also have shown that the serum of healthy donors exhibits varying levels of ABA, specifically immunoglobulin G (IgG), and that higher levels of IgG are associated with binding of Imprime PGG to innate immune cells. The data demonstrated that a threshold of ABA IgM is also predictive of binding. Significant levels of complement activation proteins C4a, C5a and SC5b-9 were found in subjects with high ABA IgG levels. These proteins were not detected in subjects with low anti-beta-glucan antibody levels.
Cevec Pharmaceuticals GmbH, of Cologne, Germany, disclosed data from an evaluation program for production of respiratory syncytial virus (RSV) vaccine in CAP cells. The data were obtained in a collaborative project with Paragon Bioservices Inc., of Baltimore, a contract manufacturer, and show that CAP cells were able to produce very high titers of RSV. Moreover and in contrast to other production systems, RSV produced in CAP cells show high-level of functional G-protein resulting in a very effective RSV vaccine with positive impact on attenuated-vaccine studies, Cevec said.
The FDA released a draft guidance intended to help drugmakers develop therapies to treat chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME). The draft, to be published in Tuesday’s Federal Register, focuses on specific development and trial design issues that are unique to CFS/ME. Although CFS/ME is a debilitating disease that affects up to 6 million people in the U.S., no drugs have been approved for the indication. The guidance discusses potential efficacy endpoints while encouraging sponsors to propose other clinically meaningful endpoints that may better assess the efficacy of their products. Since treatment for the disease will be prolonged, sponsors will need long-term safety data, the FDA said. Comments on the draft are due by May 10.
Heat Biologics Inc., of Chapel Hill, N.C., will present the results of its preclinical research study, “Comparative Combination Cancer Immunotherapy with Vaccination and TNFRSF Stimulation,” at the Keystone Symposia on Molecular and Cellular Biology: Immune Evolution in Cancer (X2) in Whistler, British Columbia. The study highlights the complementary activity of vaccination and direct T-cell co-stimulation and suggests that combination immunotherapy may be an effective treatment strategy for antitumor immunity, Heat said.
Hemispherx Biopharma Inc., of Philadelphia, said along with its partner in Latin America, GP Pharm SA, of Gava, Spain, they are planning on making applications in Chile, Peru and Uruguay for regulatory approval of Ampligen to treat chronic fatigue syndrome.
Janssen-Cilag Ltd., of High Wycombe, UK, part of Johnson & Johnson, said that the Scottish Medicines Consortium (SMC) recommended the use of Stelara (ustekinumab) for the treatment of active psoriatic arthritis in adult patients in NHS Scotland. Ustekinumab is the first in a class of biologics with a mode of action that targets interleukin-12 (IL-12) and interleukin-23 (IL-23), which are naturally occurring proteins that are important in regulating immune responses. In September 2013 the European Commission approved the use of ustekinumab, alone or in combination with methotrexate, for the treatment of active psoriatic arthritis in adult patients when the response to previous nonbiological disease-modifying anti-rheumatic drug therapy has been inadequate.
Keryx Biopharmaceuticals Inc., of New York, submitted a marketing authorization application to the European Medicines Agency, seeking the approval of Zerenex (ferric citrate coordination complex) as a treatment for hyperphosphatemia in patients with chronic kidney disease (CKD), including dialysis- and nondialysis dependent CKD. In the U.S., a new drug application already has been submitted and bears a PDUFA data of June 7, 2014. (See BioWorld Today, Aug. 9, 2013.)
Ligand Pharmaceuticals Inc., of San Diego, said its Captisol licensee H. Lundbeck AS, of Copenhagen, received FDA review acceptance of the new drug application for the investigational therapy intravenous carbamazepine, an intravenous formulation of the anti-epileptic drug carbamazepine. An action letter is anticipated before the end of 2014. Carbella Injection is the proposed U.S. trade name. With acceptance of the NDA filing, Ligand earns a $200,000 milestone payment.
Medicinova Inc., of San Diego, entered into an agreement with Research Foundation for Mental Hygiene to supply ibudilast (MN-166) for a clinical study of the compound in marijuana-dependent subjects. The study will be led by investigators from Columbia University. Ibudilast, which Medicinova is studying in multiple sclerosis, is a glial attenuator that suppresses pro-inflammatory cytokines IL-1ß, TNF-a and IL-6, and up-regulates the anti-inflammatory cytokine IL-10. It is also a functional antagonist of toll-like receptor 4.
Neurovive Pharmaceutical AB, of Stockholm, said its Hong Kong-based subsidiary, Neurovive Pharmaceutical Asia Ltd., signed a financial advisory agreement with Taiwan’s Yuanta Securities Co. Ltd., which marks the first phase of a process with the objective of an initial public offering (IPO) of the subsidiary on the Taiwanese stock exchange within two to three years. The company said the motivation for initiating the IPO process is to increase the development rate of its drug candidates in mitochondrial medicine and specific viral diseases by using capital from the Asian market. An IPO also would create a secure base in Asia for Neurovive group’s continued growth in that region.
Neostem Inc., of New York, and subsidiary Progenitor Cell Therapy LLC (PCT) said PCT expanded both its Allendale, N.J., and Mountain View, Calif., current good manufacturing practice facilities to significantly increase its engineering and process development laboratories and controlled environment space available for client needs.
Northwest Biotherapeutics Inc. (NW Bio), of Bethesda, Md., said it received approval from the Paul Ehrlich Institute in Germany for a “hospital exemption” early access program under Section 4b of the German Drug Law, allowing the company to provide its personalized immune therapy Dcvax-L to patients for the treatment of any glioma brain cancers (both glioblastoma multiforme and lower-grade gliomas), both newly diagnosed and recurrent, outside of the company’s clinical trial and allowing the firm to charge full price. The patients may be from Germany or elsewhere. The approval has a term of five years and can be re-applied for and re-issued at the end of that period. NW Bio also said the German reimbursement authority determined that Dcvax-L treatments for glioma brain cancers are eligible for reimbursement from the Sickness Funds of the German health care system. Dcvax-L currently is being tested in a phase III trial. Shares of NW Bio (NASDAQ:NWBO) gained $1.79, or 29.6 percent, to close Monday at $7.85.
Omeros Corp., of Seattle, reported positive data using OMS721, the lead human monoclonal antibody in its mannan-binding lectin-associated serine protease-2, or MASP-2, program in ex vivo studies of endothelial activation relevant to the pathophysiology of human atypical hemolytic uremic syndrome (aHUS), a form of thrombotic microangiopathy (TMA). In February, Omeros reported positive results from its OMS721 phase I trial and, earlier this month, the company disclosed submission of an investigational new drug application to the FDA to initiate a phase II trial in patients with TMA, including patients with aHUS. The preclinical data were derived from an experimental model based on the finding that serum samples from aHUS patients cause complement deposition and thrombus formation when incubated with human microvascular endothelial cells. The data showed that OMS721 significantly inhibited complement deposition in the model using serum samples from aHUS patients obtained during the acute phase of disease and during remission compared to untreated controls.
Regado Biosciences Inc., of Basking Ridge, N.J., said the FDA designated REG1 for anticoagulant therapy to be used in patients with coronary artery disease during percutaneous coronary interventions as a fast-track development program. The therapy is in phase III testing. Shares of Regado (NASDAQ:RGDO) gained $3.12, or 38 percent, to close Monday at $11.30.
Seattle Biomed, of Seattle, said it received a seven-year Integrated Preclinical/Clinical AIDS Vaccine Development grant from the National Institute of Allergy and Infectious Diseases to develop a vaccine that would elicit broadly neutralizing antibodies against HIV-1. Seattle Biomed will lead a consortium comprising Rockefeller University, the University of Washington, Seattle Children’s Hospital and the Fred Hutchinson Cancer Research Center. The investigators will receive $9.8 million over seven years to fund the initial phase of the project, which will include the optimization and preclinical evaluation of two vaccine candidates. The second phase will include production of those vaccines and evaluation of their safety and immunogenicity in a phase I trial.
Venter Pharma SL, of Madrid, and Grupo Ferrer International SA, of Barcelona, Spain, reached an agreement for Ferrer’s acquisition of an 18 percent stake in Venter Pharma for a total of €3.5 million ($US5 million). With approval by the boards of both companies, Ferrer has made an initial payment of €1.25 million.