This week's sponsor is Premier Research. | | New Insights in the Battle Against Alzheimer’s Thanks to awareness efforts like World Alzheimer’s Day this Friday, there’s a relentless focus on finding breakthrough therapies for this ravaging disease. Our new white paper looks at how we’re combating this growing epidemic. Premier Research. It’s what we do. Best. | Today's Rundown With positive phase 2 data, Akcea, Ionis could pocket $150M from Novartis Sarepta DMD gene therapy hold lifted in ‘record time’ FDA lifts hold on Epizyme cancer drug, shares surge Nightstar posts early-phase data on retinal gene therapy AnaptysBio’s lead IL-33 antibody clears midstage test in asthma Former Merck clinical research VP Arthur Santora joins Entera Bio as CMO Foundation, Roche launch new 70-gene cancer blood test following June buyout CRISPR screen identifies Zika-fighting gene Featured Story | Monday, September 24, 2018 Akcea Therapeutics reported topline data showing its hyperlipoproteinemia drug lowered lipoprotein(a) levels in a phase 2 study in patients with cardiovascular disease. If partner Novartis licenses the drug, Akcea and Ionis would get a $150 million payout. |
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| Top Stories Monday, September 24, 2018 Two months after Sarepta was hit with a clinical hold by the FDA, the rare disease biotech has seen this lifted after sorting out a quality blip. Monday, September 24, 2018 The FDA has lifted a partial clinical hold from Epizyme’s tazemetostat. Epizyme got the green light to resume enrollment in trials of the EZH2 inhibitor after showing the FDA data on the risk of secondary malignancies. Monday, September 24, 2018 Nightstar Therapeutics has presented early clinical data on its gene therapy treatment for X-linked retinitis pigmentosa. Around half of the patients in higher-doses cohorts experienced vision improvements, encouraging Nightstar to move NSR-RPGR into the larger phase 2 portion of the trial. Monday, September 24, 2018 Next-generation antibody company AnaptysBio has positive data for its lead pipeline drug etokimab in a third indication, adding asthma to its earlier wins in peanut allergy and atopic dermatitis. Monday, September 24, 2018 Former Merck clinical research leader Arthur Santora, M.D., Ph.D., is coming out of retirement to serve as the new chief medical officer of Entera Bio to help the company develop its oral hormone-based treatments for osteoporosis and hypoparathyroidism. Monday, September 24, 2018 Roche and Foundation Medicine have launched a new blood test in the U.S. that analyzes 70 genes known to drive solid tumor growth, as well as microsatellite instability high status, to help guide the use of precision cancer treatments and immunotherapies. Monday, September 24, 2018 CRISPR gene editing is being widely adopted as a tool for genome-scale screening, and a team at the University of Texas Southwestern Medical Center just used it to pinpoint a gene that helps human cells resist infection from flaviviruses like Zika. This week's sponsor is ExL Events. | | | Resources Presented by: Catalent Learn the best strategies for attracting investors in early phases of development and for overcoming formulation challenges. Sponsored by: Amazon Web Services Download our new eBook to learn how Healthcare and Life Sciences organizations are taking advantage of solutions from AWS and the AWS Partner Network to obtain value from critical data. Sponsored by: Reprints Desk, Inc. Go from Search to Full-Text Scientific Papers in a Single Click! Sponsored by: Covance FSPx Covance FSPx boasts one of the world’s largest dedicated FSP groups in the contract research organization space with ~4200 staff in 60+ countries. Sponsored by: Covance FSPx Covance FSPx boasts one of the world’s largest dedicated FSP groups in the contract research organization space with ~4200 staff in 60+ countries. Sponsored by: Docusign Life sciences companies must immediately identify and realize productivity gains to drive down operational costs—or risk seeing more nimble competitors disappear into the distance. Where to begin? Sponsored by: Veeva A top 20 pharma and growing biotech re-engineer regulatory processes. Sponsored by: Sciformix Are automation, big data and artificial intelligence (AI) a key part of your company’s pharmacovigilance strategy? Read this whitepaper to know what they should be! Sponsored by: Biotech Primer The Biopharma industry is moving at lightning speed and it can be a challenge to keep pace. Here at Biotech Primer we spend hours each week researching, writing, and editing original content for the Biotech Primer WEEKLY with one goal in mind: to help everyone better understand the latest science and technology driving today’s healthcare industry. Sponsored by: Patheon, part of Thermo Fisher Scientific When developing a parenteral, pharmaceutical scientists are faced with a variety of choices – choices that can be conflicting or present differing advantages. Sponsored by: Baxter Baxter BioPharma Solutions’ whitepaper offers a method to address the challenges of tech transfer for lyophilized products from lab-scale to commercial-scale. Sponsored by: Blue Matter A whitepaper outlining the key commercial challenges facing rare disease products, and how biopharmaceutical companies can overcome them. Sponsored by: AWS and Tableau Inova is using the capabilities of Tableau on AWS to identify biomarkers for personalized treatment intended to improve health outcomes for children. Sponsored by: AWS and Reltio Reltio and IQVIA provide holistic data management solutions for traditional pharma, emerging biotech, and consumer health companies, on AWS. Presented by: AWS and Druva Life Sciences organizations are embracing cloud-based Master Data Management (MDM) to modernize the organization of critical data. Sponsored by: Catalent Develop a basic understanding of principles and concepts of bioavailability of oral drugs. Drug Approval, Manufacturing Quality & Regulation 2018 | Online Drug Development Boot Camp® 2018 – Registration closes 30 October - Register Now! November 14-15, 2018 | Boston, MA | Registration closes on the 30 of October 2018 due to the need for participants to complete pre-Boot Camp preparations. Register now and start preparing for this intensive training in drug development. AI World Conference and Expo 2018 Monday, December 3, 2018 | 10/5/18 – Early Deadline 2018 Latin America Healthcare Compliance Certification Program September 24-27, 2018 | Sao Paulo, Brazil FierceBiotech 3rd Drug Development Forum October 1–3, 2018 | Hilton Boston Logan Airport Hotel | Boston, MA Compliance Certification Program at Seton Hall Law October 8-11, 2018 | Newark, NJ BioImmersion: Biotech for the Non-Scientist October 16-18, 2018 | Chicago, IL Fierce Innovation Awards: Life Sciences Edition 2018 Welcome to the inaugural Neuromuscular Drug Development Summit October 29-31, 2018 | Boston, MA, USA FierceBiotech Executive Summit: London October 30, 2018 | Royal Society of Medicine Understanding Commercialization Within Biopharma Nov 7-8, 2018 | Boston, MA Drug Development Immersion December 5-6, 2018 | San Diego, CA Understanding Drug Pricing and Reimbursement December 12, 2018 | Boston, MA |