Cardiome Pharma Corp., of Vancouver, British Columbia, said the Therapeutic Products Directorate of Health Canada has approved the Aggrastat (tirofiban hydrochloride) 25 mcg/kg (high) dose bolus regimen, as requested under the company's supplemental new drug submission. The regimen will now become the recommended dose to reduce the rate of refractory ischemic conditions, new myocardial infarction and death in high-risk patients with non-ST-elevation acute coronary syndrome who undergo early percutaneous coronary intervention.

Daiichi Sankyo Europe GmbH, the European headquarters of Daiichi Sankyo Co. Ltd., of Tokyo, said the European Committee for Medicinal Products for Human Use (CHMP) has recommended approval of a label update for the company's oral, once-daily direct factor Xa inhibitor, Lixiana, to provide guidance on its use in patients undergoing transesophageal echocardiography-guided and delayed cardioversion (treatment to restore a normal heart rhythm). The update is based on results from the ENSURE-AF study, which enrolled 2,199 patients, and compared once-daily Lixiana with enoxaparin/warfarin. Those data support the use of the drug as an effective and safe alternative to the best possible conventional treatment with enoxaparin and warfarin.

Dicerna Pharmaceuticals Inc., of Cambridge, Mass., said it presented new preclinical data suggesting the potential utility of DCR-PHXC, a GalXC-based investigational therapy, for treating all forms of primary hyperoxaluria (PH) at the Workshop on Primary Hyperoxaluria for Professionals, Patients and Families in Tenerife, Spain. The research from animal models demonstrated how DCR-PHXC inhibits the lactate dehydrogenase A (LDHA) gene, which the company has identified as potentially being an optimal therapeutic target in patients with PH, a group of severe, rare, inherited disorders of the liver that often result in kidney failure. The company also presented an update from the Primary HYperoxaluria Observational Study (PHYOS), in patients with PH1 that is collecting data on key biochemical parameters implicated in the pathogenesis of PH1 to better understand the baseline disease state, knowledge that will help guide long-term drug development plans. The study's primary objective is to measure changes in oxalate, glycolate and other metabolites over a six-month period in patients with PH1. PHYOS investigators reported data from 20 enrolled patients with a median age at screening of 21 years (range 12-61 years). Over the observation period, the variability (coefficient of variation) between 24-hour urine measurements of oxalate at different time points was 28 percent. Those data will help design a study using 24-hour urinary oxalate excretion as a surrogate marker for clinical benefit, the company said.

Ember Therapeutics Inc., of New York, said scientists validated a molecular mechanism that regulates scar formation in the heart, a common manifestation of aging and nearly every form of heart disease. Work also confirmed that a protein, bone morphogenetic protein 7 (BMP-7), can prevent the generation of fibrotic signals by cardiomyocytes and may be able to serve as a therapy for cardiac fibrosis. Data were published in Circulation. Ember holds patents covering the use of BMP-7 in fibrosis and other indications.

Ionis Pharmaceuticals Inc., of Carlsbad, Calif., said it earned a $10 million milestone payment from Biogen Inc., of Cambridge, Mass., for the validation of an undisclosed neurological disease target. Ionis will continue to evaluate that newest target using its antisense technology with the goal of advancing the program into development. Biogen has the option to license each antisense program at a particular stage in development.

Ligand Pharmaceuticals Inc., of San Diego, said it entered a commercial license and supply agreement granting Amgen Inc., of Thousand Oaks, Calif., rights to use its Captisol technology, a chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs in the formulation of AMG-330, an anti-CD33 and anti-CD3 (BiTE) bispecific antibody construct being investigated as a treatment for acute myeloid leukemia. The agreement replaces a prior one, which allowed Amgen to evaluate AMG-330 with Captisol in preclinical and early clinical studies. Amgen now receives exclusive worldwide rights to combine Captisol with AMG-330 for use in humans for a wide variety of therapeutic indications. In addition to an undisclosed up-front payment, Ligand is entitled to potential milestone payments, royalties and revenue from future sales of AMG-330 formulated using Captisol.

Nemus Bioscience Inc., of Costa Mesa, Calif., said data being presented at the Society of Neuroscience meeting in Washington will show the superiority of its analogue of cannabidiol (CBD), NB-2222, vs. plant-derived CBD in ameliorating pain in a validated mouse model of chemotherapy-induced peripheral neuropathy using an opioid as an active comparator.

Norgine BV, of Amsterdam, said it closed the merger with Merus Labs International Inc., of Toronto, acquiring a product portfolio of 12 products.

Perrigo Co. plc, of Dublin, said it has received final FDA approval for its AB-rated abbreviated new drug application referencing North Chicago-based Abbvie Inc.'s Androgel topical gel, 1.62 percent packets, indicated for replacement therapy in males for conditions associated with a deficiency or absence of endogenous testosterone.

Prima Biomed Ltd., of Sydney, said it will receive a second undisclosed significant clinical milestone payment from Basel, Switzerland-based Novartis AG, relating to its IMP-701 LAG-3 antibody (also referred to as LAG-525) under their collaboration and licensing agreement. The antibody is being evaluated in clinical trials together with Novartis' PD-1 inhibitor PDR-001 for the treatment of cancer. Novartis has full responsibility for the continued development of the antibody program and Prima is eligible to receive further potential development-based milestone payments and royalties on sales following commercialization of the products.

Renova Therapeutics Inc., of San Diego, presented preclinical data regarding safety and efficacy of intravenous delivery of an adeno-associated virus 8 vector encoding urocortin 2 (AAV8.UCn2) in the treatment of mice with insulin resistance, a key feature of type 2 diabetes. Urocortin 2 is a member of the corticotropin releasing factor (CRF) family of peptides and elicits its pharmacologic action by selectively binding to and activating the CRF type 2 receptor. In murine models of heart failure, insulin resistance or type 2 diabetes, gene transfer of urocortin 2 and/or urocortin 3 – peptides with paracrine activity – was associated with significant and long-lasting beneficial effects on heart function, glucose disposal, weight loss and other benefits, including reduction in fatty liver infiltration. Preliminary data in mice indicate that high doses of AAV8.UCn2 are not associated with behavioral or marked laboratory abnormalities, or serious or severe adverse effects. Data were presented at the American Heart Association's Basic Cardiovascular Sciences Scientific Sessions in Portland, Ore. Renova, which is developing gene therapy RT-200, is planning a first-in-human trial in type 2 diabetes in 2018.

Spark Therapeutics Inc., of Philadelphia, said the FDA accepted for filing its BLA and granted priority review for voretigene neparvovec, a potential one-time gene therapy candidate for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). The gene therapy, which has the proposed trade name Luxturna, has a PDUFA date of Jan. 12, 2018.

Sutro Biopharma Inc., of South San Francisco, reported research showing that STRO-001, its antibody-drug conjugate (ADC) targeting the CD74 cell surface protein in hematologic B-cell malignancies, has been shown to eradicate tumors in human xenograft models of non-Hodgkin lymphoma and multiple myeloma. Findings show that STRO-001 eliminated tumors or significantly delayed tumor growth in diffuse large B-cell lymphoma and mantle cell lymphoma xenograft models and prolonged survival in the disseminated Mino mantle cell lymphoma xenograft model compared to tumor models treated with vehicle, which developed advanced disease with palpable neck and abdominal tumors. The ADC also suppressed tumor growth in a diffuse large B-cell lymphoma tumor model, SU-DHL-6, when administered with standard-of-care chemotherapy. Sutro is planning a phase I trial for early 2018.

Therapeuticsmd Inc., of Boca Raton, Fla., said it participated in a type A post-action meeting with the FDA's Division of Bone, Reproductive, and Urologic Products last month to discuss the complete response letter previously issued for TX-004HR, its applicator-free estradiol vaginal softgel capsule for the treatment of moderate to severe vaginal pain during sexual intercourse (dyspareunia). The company received the minutes from the meeting and, per the FDA's request, has submitted new information for consideration related to the NDA.

Valeant Pharmaceuticals International Inc., of Laval, Quebec, said certain affiliates of the company entered an agreement to sell its Obagi Medical Products skincare business for $190 million in cash to Haitong International Zhonghua Finance Acquisition Fund I LP. Valeant will use proceeds from the sale to permanently repay term loan debt under its senior secured credit facility. The transaction is expected to close in the second half of 2017.

Voyager Therapeutics Inc., of Cambridge, Mass., said Nature Neuroscience published preclinical data showing that a second-generation AAV capsid provided up to a 100-fold increase in the transduction of the CNS in an adult model over the historical standard, AAV9, as compared with the first-generation capsid reported last year showing a more than 40-fold improvement over AAV9. Voyager obtained a co-exclusive license to the AAV capsids from Caltech, along with intellectual property and related technology, in September 2016.