Advanced Accelerator Applications SA, of Saint-Genis-Pouilly, France, said the FDA has accepted the company’s NDA and granted priority review for Lutathera, a Lu-177-labeled somatostatin analogue peptide currently under development for the treatment of gastroentero-pancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors in adults. The Prescription Drug User Fee Act (PDUFA) target action date is Dec. 28, 2016. The application is based on the results of a pivotal phase III study, NETTER-1 that compared treatment using Lutathera with a double dose of Octreotide LAR in patients with inoperable midgut NETs progressive under Octreotide LAR treatment and overexpressing somatostatin receptors. The study met its primary endpoint by demonstrating that treatment with Lutathera was associated with a statistically significant and clinically meaningful risk reduction of 79 percent in disease progression or death versus a treatment with a double dose of Octreotide LAR.
Agilis Biotherapeutics LLC, of Cambridge, Mass., said it has been selected by the National Center for Accelerating Translational Sciences (NCATS) as an awardee of a cooperative research and development agreement (CRADA) under the National Institutes of Health’s (NIH) Therapeutics for Rare and Neglected Diseases (TRND) program. The agreement will facilitate development activities in support of registration of the company’s gene-therapy candidate for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. This gene therapy program was licensed from National Taiwan University, which treated 18 patients. Following a single administration of the gene therapy, treated patients have shown substantial, durable gains in motor and cognitive function over multiple years, exhibited de novo production of dopamine as visualized by F-DOPA PET imaging, and realized improvements in metabolic biomarkers. The Agilis-TRND partnership will conduct toxicology, process development, and manufacturing work necessary for overall development of this therapeutic, and registration in the U.S. and abroad.
Bristol-Myers Squibb Co. (BMS), of New York, and the Angola Sickle Cell Initiative, of Houston, which is a public-private partnership of the Angola Ministry of Health, the Baylor College of Medicine International Pediatric AIDS Initiative, Texas Children’s Hospital and Chevron Corp., said they have begun a program to provide medical treatment for children in Angola who suffer from sickle cell disease. The five-year cooperative endeavor will distribute Droxia (hydroxyurea) to those who need the medication. BMS will donate its product to treat up to 1,200 children in the first two years and will increase the number of participants to 4,100 after that period. The program will begin in Luanda and Cabinda and expand later to additional sites. Last year, the company made an initial donation of Droxia to the Angola Sickle Cell Initiative.
Bristol-Myers Squibb Co., of New York, said it received breakthrough therapy status designation from the FDA for Opdivo as treatment for unresectable locally advanced or metastatic urothelial carcinoma that has progressed during or following a platinum-containing regimen. The submission was partially based on phase II data from CA209-275. The drug has already been granted that status for previously treated recurrent or metastatic squamous cell carcinoma of the head and neck, Hodgkin lymphoma after failure of autologous stem cell transplant and brentuximab-vedotin, previously treated advanced melanoma, previously treated non-squamous non-small-cell lung cancer, and previously treated advanced or metastatic renal cell carcinoma.
Eisai Co. Ltd., of Tokyo, and University College London (UCL) said the first scientific milestone was reached in the development of a treatment for neurodegenerative disorders. Eisai and UCL are conducting research as part of a collaboration that was established with Eisai’s Neurology Business Group open innovation strategy. The collaboration began in 2012 to discover treatments for Alzheimer’s, Parkinson’s and other related neurodegenerative disorders. No other specifics were released.
Emergent Biosolutions Inc., of Gaithersburg, Md., said it received a task order from the Biomedical Advanced Research and Development Authority (BARDA) to develop and manufacture three lots of a Zika vaccine to use in a phase I trial. The task order is valued at up to $21.9 million. BARDA will provide a base vaccine candidate and Emergent will produce and conduct other processes required using current good manufacturing practice regulations.
Ferring International Center SA, of Saint Prex, Switzerland, said it received approval for a specific dosing regimen on the label of Picoprep (sodium picosulfate, magnesium oxide, citric acid), a prep solution for colonoscopies, in mutual recognition procedure countries in the EU. The approval is based on data from OPTIMA, Ferring’s study that showed Picoprep’s tailored dosing provided more effective colon cleansing and provided better visibility as compared to the current dosing directions.
Five Prime Therapeutics Inc., of South San Francisco, said it will collaborate with the University of Minnesota Medical School on a research project to identify ways to generate hematopoietic stem cells (HSCs) capable of being transferred in vivo and regenerating the hematopoietic system. The research, funded by Regenerative Medicine Minnesota, will use Five Prime’s library to screen more than 5,700 human extracellular proteins to identify those that promote the in vitro formation of human from inducible pluripotent stem cell line created by investigators.
Janssen Inc., of Toronto, a unit of Johnson & Johnson, said Health Canada approved Invega Trinza (paliperidone palmitate), an antipsychotic medication for schizophrenia, which provides a dosing regimen option administered only four times per year. The three-month injection is indicated for treatment only after Invega Sustenna, a once-monthly version, has been established as adequate treatment for at least four months.
Jazz Pharmaceuticals plc, of Dublin, said the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 has expired, with respect to the firm’s proposed acquisition of Celator Pharmaceuticals Inc., of Ewing, N.J. The companies agreed to the $1.5 billion deal late last month. (See BioWorld Today, June 1, 2016.)
Medimetriks Pharmaceuticals Inc., of Fairfield, N.J., said it submitted a new drug application to the FDA seeking approval of ozenoxacin cream, 1 percent, a nonfluorinated, quinolone, for the treatment of impetigo. Medimetriks licensed exclusive U.S. commercialization rights to ozenoxacin from Grupo Ferrer Internacional SA, of Barcelona, in March 2014 and announced the completion of the second positive phase III pivotal trial in July 2015.
Merck & Co. Inc., of Kenilworth, N.J., said the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending approval of PD-1 inhibitor Keytruda (pembrolizumab) to treat locally advanced or metastatic non-small-cell lung cancer in adults whose tumors express PD-L1 and who have received at least one prior chemotherapy regimen.
Newron Pharmaceuticals SpA, of Milan, Italy, said it is sponsoring a study to evaluate the disease burden experienced by patients with Rett syndrome and their families. Rett syndrome is a rare disease characterized by severe neurological, respiratory, cognitive and behavioral symptoms. The study will comprise two global surveys, developed in accordance with regulatory guidance.
Onxeo SA, of Paris, announced plans to further develop AsiDNA, its first-in-class signal interfering DNA molecule which breaks the cycle of tumor DNA repair to induce cancer cell death. In March, Onxeo completed the acquisition of DNA Therapeutics for its DNA repair inhibitor technology platform and lead compound AsiDNA. The technology has already demonstrated an increase in the efficacy of radiotherapy, radiofrequency ablation and chemotherapy in a variety of preclinical animal models. A first-in-human phase I/IIa trial performed in metastatic melanoma further demonstrated that AsiDNA molecules showed good tolerance and safety when administered intra-tumorally and subcutaneously around the tumors, with no evidence of inflammatory phenomena. Preclinical programs have been initiated to further define the pharmacokinetic/pharmacodynamic profile following an intravenous administration. Results are expected in Q3/Q4 2016.
OSE Immunotherapeutics SA, of Paris, presented preclinical efficacy results for Effi-7, an antagonist of the interleukin 7 (IL-7) receptor in regulation immunotherapy, at the Federation of Clinical Immunology Societies. The preclinical results showed efficacy of Effi-7, an antagonist of the IL-7 receptor, in ulcerative colitis (UC) models, an autoimmune inflammatory bowel disease of the colon. Efficacy was observed in parallel with an innovative mechanism of action of Effi-7 in the prevention of the infiltration of human T lymphocytes, responsible for the inflammatory damage to the colon mucosa.
Puma Biotechnology Inc., of Los Angeles, submitted a marketing authorization application to the EMA for neratinib seeking approval to market the drug for the extended adjuvant treatment of HER2-positive early stage breast cancer that has previously been treated with trastuzumab-based adjuvant therapy. The submission is based upon the Extenet phase III study, which reached its primary endpoint whereby neratinib demonstrated a statistically significant 33 percent reduction of risk of invasive disease recurrence or death vs. placebo (hazard ratio = 0.67, p = 0.009).
Sanofi SA, of Paris, and Ingelheim, Germany-based Boehringer Ingelheim GmbH signed contracts securing the exchange of Sanofi’s animal health business, Merial and Boehringer’s consumer healthcare business, first announced in December 2015. The contract signing marks a major milestone before closing of the transaction, which is expected by year-end 2016, the companies said. The deal, which remains subject to approval by regulatory authorities in different territories, would help Sanofi enhance its position in several areas, including pain, allergy, cough and cold, feminine care, digestive health and vitamins, minerals and supplements.
Servier Canada, of Laval, Quebec, struck a deal with Tokyo-based Daiichi Sankyo Co. Ltd. under which Servier will market the oral, once-daily anti-coagulant edoxaban in Canada, if approved by Health Canada. Daiichi filed a new drug submission for edoxaban with the Health Products and Food Branch of the regulator in August 2015. Under the agreement, Daiichi Sankyo will receive an up-front payment of undisclosed value, payments based on regulatory and commercial milestones and royalties on net product sales. Further financial details were not disclosed.
Swedish Orphan Biovitrum AB, of Stockholm, said that the Swiss Agency for Therapeutic Products, Swissmedic, has approved Elocta (rFVIIIFc) for the treatment of hemophilia A. The approval covers both on-demand and prophylaxis treatment of people with hemophilia A of all ages. The Swiss approval was based on data from Elocta’s pivotal, phase III A-LONG study, which demonstrated the efficacy, safety and pharmacokinetics of rFVIIIFc in previously treated males 12 years of age and older with severe hemophilia A, and from the phase III Kids A-LONG clinical study, which demonstrated the efficacy and safety of rFVIIIFc in previously treated boys with hemophilia A younger than 12. Sobi has final development and commercialization rights in the Sobi territory (Europe, North Africa, Russia and most Middle Eastern markets). Biogen Inc., of Cambridge, Mass., leads development and manufacturing for Elocta and has commercialization rights in North America and all other regions in the world excluding Sobi’s territory. Eloctate, Biogen’s name for the drug, was approved by the FDA in June 2014. (See BioWorld Today, June 10, 2014.)
Tarix Orphan LLC said that the company’s lead compound, TXA127, has shown positive preclinical results in the C7-hypomorphic mouse model of recessive dystrophic epidermolysis bullosa (DEB), a rare and often severe genetic skin disorder. Researchers at the University of Freiburg, Germany showed that four weeks of daily subcutaneous administration of TXA127, a pharmaceutical formulation of the natural angiotensin (1-7) peptide, substantially reduced the fusion of digits, a symptom analogous to mitten deformity common in patients with DEB. In addition, immunohistology showed that TXA127 significantly reduced tenascin c, a marker of dermal fibrosis, the company said. Tarix Orphan intends to investigate TXA127 further in preclinical models of DEB with the ultimate goal of testing the drug in clinical trials. The company initially studied the compound in stem cell engraftment. (See BioWorld Today, Oct. 6, 2011.)