Arrowhead Nominates ARC-HIF2 As First RNAi Therapeutic Candidate Using Extrahepatic DPC™ Delivery System

Data to be presented September 27 at the European Cancer Conference 2015 in Vienna

PASADENA, Calif.--Arrowhead Research Corporation (ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it has nominated ARC-HIF2 as its first therapeutic candidate delivered using a new Dynamic Polyconjugate™ (DPC™) designed to target tissues outside of the liver. Arrowhead believes that ARC-HIF2, which uses RNA interference to silence transcription factor hypoxia-inducible factor 2α (HIF-2α), is a promising new candidate for the treatment of clear cell renal cell carcinoma (ccRCC). The company will present preclinical data at the European Cancer Congress 2015 (ECC2015) in Vienna on September 27 in a session starting at 16:45 CEST. In a poster titled “HIF-2α targeting with a novel RNAi delivery platform as therapy for renal cell carcinoma,” (abstract #353), Arrowhead scientists will show data suggesting that HIF-2α inhibition through RNA interference may significantly impact late stage ccRCC progression. The company is in the process of manufacturing scale up to allow for initiation of IND-enabling studies. Timing for anticipated regulatory submission will be announced in the future.

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Medicure Announces Filing of sNDA for New AGGRASTAT® Indication

Seeks Addition of STEMI Indication for AGGRASTAT

WINNIPEG, Sept. 10, 2015  - Medicure Inc. ("Medicure" or the "Company") (TSXV:MPH, OTC:MCUJF), a specialty pharmaceutical company, is pleased to announce that it has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) to expand the label for AGGRASTAT (tirofiban HCl) to include the treatment of patients presenting with ST segment elevation myocardial infarction (STEMI).  AGGRASTAT is currently approved by the FDA for treatment of patients presenting with non-ST segment elevation acute coronary syndrome (NSTE ACS). If approved for STEMI, AGGRASTAT would be the first in its class of Glycoprotein IIb/IIIa Inhibitors (GPI) to receive such a label in the United States.

"We have received substantial feedback from physicians that there is a need for more potent antiplatelet therapy when treating STEMI patients undergoing percutaneous coronary intervention (PCI)," stated Dawson Reimer, President and Chief Operating Officer of Medicure Inc. "We believe the STEMI indication would increase the utility of AGGRASTAT for physicians, and thereby increase hospital adoption and demand.  We look forward to hearing from the FDA on this sNDA submission."

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MGCD265 Demonstrates Clinical Efficacy with Confirmed Responses in NSCLC Patients with MET and AXL Gene Amplificaiton

- First Ever Confirmed Response in NSCLC Patient, with Axl Gene Amplification, Treated with an Orally Administered Small Molecule Inhibitor of MET and Axl to be Presented at IASLC 16th World Conference on Lung Cancer
- Company Announces a Confirmed Response in a NSCLC Patient with MET Gene Amplification and Provides Interim Update on Ongoing MGCD265 Phase 1b Expansion Cohort

DENVER, Sept. 9, 2015 -- Mirati Therapeutics, Inc. ("Mirati") (MRTX), an oncology company focusing on genetic and epigenetic drivers of cancer, today announced it will present data at the International Association of Lung Cancer (IASLC) 16th World Conference on Lung Cancer on the first non-small cell lung cancer (NSCLC) patient with AXL gene amplification enrolled in the MGCD265 Phase 1b expansion cohort. Data will be presented showing the patient had a confirmed Partial Response (PR) based on RECIST criteria. Additionally, the Company announced a confirmed PR in a NSCLC patient with MET gene amplification who was enrolled in the MGCD265 expansion cohort.

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Vitae Pharmaceuticals Announces Positive Top-Line Results From Initial Phase 1 Study of First-in-Class RORyt Inhibitor VTP-43742 in Autoimmune Disorders

  • Single ascending doses of VTP-43742 safe and generally well-tolerated, demonstrated once-daily pharmacokinetics
  • Robust ex vivo biomarker response, suppressing pro-inflammatory IL-17A by more than 90 percent

FORT WASHINGTON, Pa., Sept. 8, 2015  -- Vitae Pharmaceuticals, Inc. (VTAE), a clinical-stage biotechnology company, today announced positive top-line results from its Phase 1 single ascending dose clinical study of VTP-43742 in autoimmune disorders. VTP-43742 is Vitae's first-in-class, wholly owned RORγt inhibitor being developed for the treatment of a range of autoimmune disorders, potentially including psoriasis, psoriatic arthritis, rheumatoid arthritis, multiple sclerosis and irritable bowel disease (IBD), as well as numerous orphan diseases.

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Akebia Announces Positive Top-Line Results from its Phase 2 Study of Vadadustat in Dialysis Patients with Anemia Related to Chronic Kidney Disease

-Treatment with Vadadustat Successfully Maintained Mean Hemoglobin Levels Following Conversion from rESA Therapy-
-Vadadustat Demonstrated a Favorable Safety Profile with Once Daily and Three Times per Week Dosing-
-Conference Call at 5:00 PM Eastern Time Today-

CAMBRIDGE, Mass.--- Akebia Therapeutics, Inc. (AKBA), a biopharmaceutical company focused on delivering innovative therapies to patients with kidney disease through the biology of hypoxia inducible factor (HIF), today announced positive top-line results from its Phase 2 study of vadadustat (formerly AKB-6548) in dialysis patients with anemia related to chronic kidney disease (CKD). The study achieved its primary objective, indicating that vadadustat maintained stable hemoglobin (HGB) levels throughout the 16-week treatment period following conversion from recombinant erythropoiesis-stimulating agent (rESA) therapy. Vadadustat demonstrated a favorable safety profile with no drug-related serious adverse events and no deaths. The results highlight the potential of vadadustat, dosed either once daily or three times per week, to safely and predictably manage and sustain HGB levels in CKD patients undergoing dialysis.

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