Albireo Pharma Inc., of Boston, said lead candidate A4250 was granted access to the EMA’s Priority Medicines, or PRIME, program for the treatment of progressive familial intrahepatic cholestasis, or PFIC.
Centrexion Therapeutics Inc., of Baltimore, said the FDA granted fast track designation to CNTX-4975 to treat Morton’s neuroma, a rare nerve disorder in the foot. The company completed a phase IIb study in patients with the condition, and an open-label, multiple-dose extension study is ongoing. (See BioWorld Today, March 31, 2016.)
Cynata Therapeutics Ltd., of Melbourne, Australia, said it received an R&D tax incentive refund from the Australian government of approximately A$1.75 million (US$1.3 million) for the 2015-16 financial year.
Da Volterra, of Paris, said it joined the European Consortium COMBACTE-NET (Combatting Bacterial Resistance in Europe) alongside Medimmune, a unit of Astrazeneca plc, of London, and The Medicines Co., of Parsippany, N.J. The consortium was established by the European Commission and the European Federation of Pharmaceutical Industries and Associations within the framework of the Innovative Medicines Initiative (IMI) to execute trials enabling the registration of therapeutic agents to treat antibiotic-resistant bacterial infections. Within the IMI framework, Da Volterra is launching an epidemiological study on 1,000 patients recruited in 30 European centers, who will be followed over three months to detect any occurrence of Clostridium difficile infections following antibiotic treatment and to discern populations for which preventing the pathology are most effective. A therapeutic solution, designated as DAV132, aims to prevent C. difficile infections in those high-risk patients.
Juniper Pharmaceuticals Inc., of Boston, inked an agreement with Allergan plc, of Dublin, to monetize future royalty payments for U.S. sales of Crinone (progesterone gel). Under the agreement, Juniper will receive a one-time payment of $11 million from Allergan. Juniper sold U.S. rights to Crinone to Allergan in July 2010 and has since received a 10 percent royalty on U.S. sales. Juniper will continue to supply Crinone exclusively for all ex-U.S. markets and to receive revenue from those sales from Merck KGaA, of Darmstadt, Germany, which holds the product’s marketing authorization in more than 90 countries. Crinone is indicated as part of assisted reproductive technology treatment for infertile women with progesterone deficiency.
Kamada Ltd., of Ness Ziona, Israel, signed a collaboration agreement with Yissum Research Development Co. of the Hebrew University of Jerusalem for the development of a eukaryotic expression system for recombinant human alpha 1 antitrypsin (AAT), an effort focused on maximizing protein yields and functionality. Upon completion of the work with Yissum, Kamada intends to begin GMP manufacturing scale-up activities. The main advantage of recombinant AAT over the plasma-derived AAT at the heart of Kamada’s flagship product, Glassia, is its potentially higher availability at lower price per raw material, the company has said.
Marinus Pharmaceuticals Inc., of Radnor, Pa., disclosed preclinical data showing that the combination of its central nervous system-selective GABAA modulator, ganaxolone, plus diazepam administered intravenously (I.V.) produced a synergistic effect in blocking pilocarpine-induced seizures in a benzodiazepine-refractory model of status epilepticus (SE). Marinus is developing ganaxolone I.V. for the condition, a life-threatening medical emergency associated with high mortality and limited treatment options. The data were presented during a poster presentation at Neuroscience 2016 in San Diego. Subtherapeutic doses of diazepam and ganaxolone when administered in combination 15 minutes after onset of status epilepticus produced a partial or complete block of treatment-resistant status epilepticus in a rat model of SE, a clinically translatable model of that condition, the company said.
Midatech Pharma plc, of Oxford, U.K., selected its wholly owned candidate, MTR104, to take into a formal investigational new drug application-enabling program during 2017, followed by clinical trials planned for 2018. The program, which employs a nanoparticle-based conjugate, is focused on advanced liver cancer. (See BioWorld Today, Nov. 26, 2014.)
Neovacs SA, of Paris, acquired from Pilar, Argentina-based Amegabiotech a manufacturing license for interferon-alpha, a key component of Neovacs’ lead therapeutic candidate, IFN-alpha Kinoid, which is composed of inactivated IFN-alpha coupled with a carrier protein, keyhole limpet hemocyanin. Financial terms of the license were not disclosed.
OSE Immunotherapeutics SA, of Nantes, France, signed service agreements with Selexis SA, of Geneva, gaining access to research cell banks from the Selexis Suretechnology platform. The agreements are designed to help OSE advance two preclinical products based on immune activation and regulation: Effi-DEM is a new-generation checkpoint inhibitor that blocks the signals regulatory protein-alpha receptor expressed by myeloid suppressor cells and tumor-associated macrophages, and Effi-7 is a humanized monoclonal antibody targeting the CD127 receptor.
Pharmaxis Ltd., of Sydney, reported that the National Health and Medical Research Council has awarded it a research grant of A$421,545 (US$318,560) for development and testing of its Orbital Inhaler with a dry powder formulation of the antibiotic tobramycin for the treatment of cystic fibrosis. The company will work with the Sydney-based Woolcock Institute of Medical Research.
Sciclone Pharmaceuticals Inc., of Foster City, Calif., received an unsolicited, nonbinding proposal from a consortium led by GL Capital Management GP Ltd. and ABG Management Ltd. to acquire the company for $11.18 per share in cash. The proposal is subject to a number of contingencies, including financing, due diligence and documentation, it said.
Uni-Bio Science Group Ltd., of Hong Kong, established a strategic partnership with Shanghai-based Luqa Pharmaceuticals Trading Co. Ltd., a China-focused specialty pharmaceutical company, to conduct co-promotion and co-marketing of dermatology drugs in mainland China, something Uni-Bio said would enable it to immediately expand its income stream and generate cash flow. Under the terms of the alliance, products for co-promotion and co-marketing cover drugs or medical devices for the treatment of dermatological disorders, including Uni-Bio Science’s and Luqa’s current and future products in the dermatology field. The three-year promotion agreement includes an option to renew for two additional years. While the agreement is in force, Uni-Bio Science will co-promote selected products in hospitals or qualified institutions, drug stores and retail channels in certain territories, it said. Uni-Bio Science will additionally support Luqa with market access in the territories, including provincial and national reimbursement expansion of products under collaboration. Financial details of the arrangements were not disclosed.
Uniqure NV, of Amsterdam, completed a companywide strategic review aimed at refocusing its pipeline, consolidating its manufacturing and enhancing overall execution. The company expects to realize €5 million to €6 million (US$5.4 million to $6.4 million) of annualized cost savings in personnel and other related operating expenses as a result of the elimination of approximately 50 to 60 positions, or 20 percent to 25 percent of its global headcount, by the end of 2017. Additionally, the company expects to further reduce planned operating expenses by €11 million to €15 million over the next two years through the focusing of its pipeline. Existing cash resources will be sufficient to fund operations into 2019, it said. The company will prioritize programs in hemophilia B, Huntington’s disease and those associated with its collaboration with Bristol-Myers Squibb Co., of New York, in cardiovascular disease. Additionally, the company will restructure its R&D organization in the Netherlands and consolidate manufacturing in the U.S. The actions are expected to reduce operating expenses and create a more efficient company focused on the development of gene therapies, it said.
Ziopharm Oncology Inc., of Boston, highlighted the publication of data demonstrating enhanced persistence of genetically modified T cells targeting leukemia through utilization of its nonviral Sleeping Beauty system to co-express membrane-bound IL-15 (mbIL15) and a CD19-specific chimeric antigen receptor (CAR). The article, “Tethered IL-15 augments antitumor activity and promotes a stem-cell memory subset in tumor-specific T cells,” was published in the Nov. 14, 2016, issue of the Proceedings of the National Academy of Sciences.