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Archive - Jun 26, 2017

HUNTINGTON'S DISEASE: Enrollment in Phase 1/2a Study of IONIS-HTT Rx in Patients with HD Completed and Open-Label Extension Study to Open in 2H 2017; Anti-Sense Drug Designed to Reduce Production of All Forms of HTT, Including Mutant HTT

On June 22, 2017, Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced completion of enrollment in the Phase 1/2a randomized, placebo-controlled, dose escalation study of IONIS-HTTRx in patients with Huntington's disease (HD). Dosing in the final patient cohort continues, and Ionis plans to report top-line results from this study around year-end 2017. The safety and tolerability profile of IONIS-HTTRx in the completed cohorts of the Phase 1/2a study supports its continued development. Patients who complete the Phase 1/2a study will be eligible to participate in an open-label extension (OLE) study that Ionis plans to initiate in the next several months. Roche, Ionis' partner for this drug, continues to advance and support the program. IONIS-HTTRx is the first therapy in clinical development targeting the cause of HD by reducing the production of the toxic mutant huntingtin protein (mHTT) from the mutated huntingtin gene. "We are encouraged by the safety profile of IONIS-HTTRx we have observed to date in the completed dosing cohorts in the Phase 1/2a study. Upon completion and full analysis of this study, the next step for this program will be to conduct a study to investigate whether decreasing mutant huntingtin protein with IONIS-HTTRx can slow the progression of this terrible disease," said C. Frank Bennett, PhD, Senior Vice President of Research at Ionis Pharmaceuticals. "We believe that IONIS-HTTRx, which is designed to reduce the production of all forms of the huntingtin (HTT) protein – the known cause of HD, represents the most promising opportunity to address this significant unmet medical need.

FDA Grants Pre-Market Approval to Thermo Fisher Scientific for First NGS-Based Companion Diagnostic Test for Biomarkers Associated with Three FDA-Approved Therapies for Non-Small Cell Lung Cancer (NSCLC)

On June 22, 2017, Thermo Fisher Scientific announced that the U.S. Food and Drug Administration (FDA) had granted premarket approval to the company for the first next-generation sequencing (NGS)-based test that simultaneously screens tumor samples for biomarkers associated with three FDA-approved therapies for non-small cell lung cancer (NSCLC). Lung cancer is the leading cause of cancer-related deaths in the U.S., with NSCLC accounting for 85 percent of all lung cancers. The Oncomine Dx Target Test simultaneously evaluates 23 genes clinically associated with NSCLC. Following FDA approval, results from analysis of three of these genes can now be used to identify patients who may be eligible for treatment with one of the following: the combined therapy of Tafinlar® and Mekinist®, XALKORI®, or IRESSA®. With this test, physicians can now match patients to these therapies in days instead of several weeks, which it often takes when screening samples one biomarker at a time. "For people battling non-small cell lung cancer, time is critical and days matter," said Joydeep Goswami, President of Clinical Next-Generation Sequencing and Oncology at Thermo Fisher. "The Oncomine Dx Target Test rapidly guides oncologists toward the right targeted therapy, with the goal of improving patient outcomes and the cost-efficiency of treatments." LabCorp's Diagnostics and Covance Businesses, NeoGenomics Laboratories, and Cancer Genetics, Inc., are among the first laboratories that will offer the Oncomine Dx Target Test as a service to oncologists.