Kyowa Hakko Kirin Co Ltd:The company has decided the future development direction in Japan for bardoxolone methyl (RTA 402), a small-molecule compound licensed from Reata Pharmaceuticals, Inc.Decided to continue developing this compound for CKD patients with type 2 diabetes with a particular emphasis on patient safety, and plans to evaluate both the safety and efficacy of bardoxolone methyl in a new Phase 2 clinical study to be performed in Japan, under the new direction.
Amgen Inc receives FDA breakthrough therapy designation for investigational BiTE Antibody Blinatumomab In Acute Lymphoblastic Leukemia
Amgen Inc:US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to investigational bispecific T cell engager (BiTE) antibody blinatumomab.For adults with Philadelphia-negative (Ph-) relapsed/refractory B-precursor acute lymphoblastic leukemia (ALL), rapidly progressing cancer of blood and bone marrow1.Breakthrough therapy designation was based on results of Phase 2 trial of 189 adult patients with Ph- relapsed/refractory B-precursor ALL treated with blinatumomab.FDA states that breakthrough therapy designation is intended to expedite development and review of drugs for serious or life-threatening conditions.Criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates drug may have substantial improvement on at least one clinically significant endpoint over available therapy.Breakthrough Therapy Designation conveys all of fast-track program features, more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review2.
Genocea Biosciences announces positive top-line 12-month follow-up data from phase 1/2a clinical trial for HSV-2 immunotherapy GEN-003
Genocea Biosciences Inc:Says positive top-line 12-month follow-up data from a Phase 1/2a study of GEN-003, the company's immunotherapy candidate against herpes simplex virus type 2 (HSV-2).
Aeolus Pharmaceuticals Inc announces additional data demonstrating efficacy of AEOL 10150 as Medical Countermeasure
Aeolus Pharmaceuticals Inc:Presentation of data confirming the efficacy of AEOL 10150 as medical countermeasure sulfur mustard gas inhalation and nitrogen mustard gas skin exposure.Additionally, an update on progress under the NIH CounterACT nerve agent grant was provided.Studies were run and verified earlier reported efficacy of AEOL 10150 in rats exposed to lethal sulfur mustard gas.AEOL 10150 treatment significantly (p < 0.0001) improved survival at 24 hours after sulfur mustard exposure from 42 pct vs. 94 pct and at 48hrs from 35 pct to 88 pct.AEOL 10150 treatment significantly (p < 0.0001) improved lung function 25 pct to normal levels in sulfur mustard exposed rats which correlated with arterial blood gas improvements in pO2, pCO2 and pH.AEOL 10150 treatment significantly (p < 0.0001) improved clinical scores 60 pct in sulfur mustard exposed rats.AEOL 10150 treatment reduces airway cast formation at 24 hours (p < .017) and 48 hours (p < .0071).Natural history studies reveal that the 1.4mg/kg sulfur mustard gas exposure is 100 pct lethal by 20 days.AEOL 10150 treatment increased the median sulfur mustard survival from 2.8 days to 16 days (p < 0.05) with survival of AEOL 10150 treated animals past 28 days.
Ipsen SA:Says that it has submitted a Supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) for Somatuline Depot 120mg injection for the treatment of gastroenteropancreatic neuroendocrine tumors.
Vertex Pharmaceuticals Inc submits supplemental new drug application (sNDA) to U.S. food and drug administration
Vertex Pharmaceuticals Inc:Submits supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration (FDA) for approval of KALYDECO in people with cystic fibrosis (CF) ages 18 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.In United States, KALYDECO is currently approved for use in people with CF ages 6 and older who have one nine mutations, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D.CF is caused by defective or missing CFTR protein that results from mutations in the CFTR gene.
Allergan Inc:Receives approval from U.S. Food and Drug Administration (FDA) for OZURDEX(reg)(dexamethasone intravitreal implant) 0.7 mg as treatment option for diabetic macular edema (DME) in adult patients who have an artificial lens implant (pseudophakic) or who are scheduled for cataract surgery (phakic).
Uni-Bio Science Group Ltd:Says in a phase II trial sponsored by the Group, Uni-PTH has successfully shown to increase lumbar spine bone mass density after 6 months of treatment.The objective of the phase III is to further validate the efficacy and safety results of the drug from the phase II clinical trial.Mode of administration and dosage is consistent to that of the phase II clinical trial.The phase III clinical trial results confirm that the Uni-PTH is safe and efficacious in post-menopausal women with osteoporosis. Uni-PTH met primary endpoint of increasing bone mass density of lumbar spine after 12 months of treatment.
Merck & Co Inc:Results from global, investigational Phase 3 study to evaluate the safety and efficacy of EMEND (aprepitant) in prevention of chemotherapy-induced nausea and vomiting (CINV) in pediatric cancer patients, aged 6 months to 17 years.In this study in pediatric cancer patients undergoing very highly, highly, or moderately emetogenic (vomit-inducing) chemotherapy.Says use of EMEND regimen for CINV prevention was significantly more effective than control regimen in achieving Complete Response, defined as no vomiting or retching and no use of rescue medication for nausea and vomiting, in all phases of CINV (acute, delayed, and overall).Nausea and vomiting are common complications of cancer chemotherapy and can be particularly distressful and debilitating to pediatric cancer patients.Based on these data, Merck plans worldwide regulatory submissions for EMEND (aprepitant), beginning in the United States, for use in the prevention of CINV in pediatric and adolescent cancer patients (ages 6 months to 17 years).In the United States, Merck plans to submit a New Drug Application (NDA) for a new pediatric formulation (powder for suspension) and a supplemental NDA for use of the current formulation (capsules).Both filings are planned for the second half of 2014.
Genzyme:Argentina's National Administration of Drugs, Food and Medical Technology (ANMAT) has approved Lemtrada (alemtuzumab) for adult patients with relapsing remitting multiple sclerosis (RRMS) with active disease defined by clinical or imaging features.Lemtrada is supported by comprehensive and extensive clinical development program that involved nearly 1,500 patients and 5,400 patient-years of follow-up.In addition to Argentina, Lemtrada is approved in European Union, Australia, Canada, Mexico, Brazil and Guatemala.Lemtrada is not approved in the United States.Genzyme recently announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's resubmission of its application seeking approval of Lemtrada.Genzyme expects FDA action on application in fourth quarter.Lemtrada clinical development program included two randomized Phase III studies comparing treatment with Lemtrada to high-dose subcutaneous interferon beta-1a in patients with RRMS who had active disease.Says were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II), as well as ongoing extension study.In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates; the difference observed in slowing disability progression did not reach statistical significance.Genzyme, a Sanofi SA company.