Today's Rundown Spark slides on hemophilia A data, aims for phase 3 U.K. commits to adopt EU trial regulation, minimize divergence after Brexit With patisiran launch in its sights, Alnylam plots a major hiring spree FDA notes Paratek antibiotic’s noninferiority as it heads to AdComm vote Akouos raises $50M to take hearing loss gene therapy into humans Ironwood abandons deal with AstraZeneca for lesinurad, once hoped to be a gout blockbuster Cha-ching: Small biotechs write some of America's biggest paychecks Featured Story | | | Tuesday, August 7, 2018 Spark Therapeutics’ hemophilia A gene therapy reduced bleeding by 97% in a phase 1/2 trial but sent one patient to the hospital, leading the biotech's stock to dip about 30% before the market opened. |
|
|
Top Stories Tuesday, August 7, 2018 The U.K. regulatory agency has detailed how it expects Brexit to affect its clinical trial regulations. Officials reiterated their intent for the U.K. to implement the incoming European Union Clinical Trials Regulation and expanded on the long-term effects of Brexit on research approval processes. Tuesday, August 7, 2018 Alnylam’s first drug approval could come any day now, and the company is bulking up to get ready—in more ways than one. The RNAi-focused biotech is expanding in Cambridge's Kendall Square and going on a hiring binge that will double its roster. Tuesday, August 7, 2018 Paratek Pharmaceuticals’ first drug, and the first in a new class of broad-spectrum antibiotics called aminomethylcyclines, has shown to be noninferior to current regimens for bacterial pneumonia and skin infections, FDA reviewers wrote ahead of an Aug. 8 advisory committee meeting. Tuesday, August 7, 2018 Akouos has raised $50 million to take a gene therapy treatment for hearing loss into the clinic. The Boston-based biotech unveiled the series A round alongside the appointment of a Spark Therapeutics co-founder and other organizational changes. Monday, August 6, 2018 Ironwood Pharmaceuticals has decided to walk away from its 2016 deal to license AstraZeneca’s billion-dollar gout treatment lesinurad in the U.S., following poor market performance of the drug, as Ironwood continues its march toward splitting into two separate public companies next year. Tuesday, August 7, 2018 Infinity, Savara and Sarepta are among the highest-paying companies in the U.S., a new report reveals. But the gap between CEO compensation and the pay the average employee brings home remains vast. Resources Sponsored by: Patheon, part of Thermo Fisher Scientific “Faster and better” has become the mantra for biopharmaceutical companies as they face intense pressure to get therapies to market faster. Quickly proving efficacy in first-in-human (FIH) trials is a make-or-break milestone for these cash-strapped companies whose hopes hinge upon one or two molecules. Read how CDMOs are responding to this pressure. Sponsored by: PharmaCord When was the last time your patient support program perfectly aligned with the patient journey, solving all the critical gaps in positioning your brand for success? Follow the 3-D approach to your solution design, and you will achieve a better outcome for your patients. Sponsored by: WCG Selecting the right sites is the most crucial decision you’ll make in your next clinical trial, and the single most important factor to consider in selecting a site is whether or not it can meet its enrollment goals. Sponsored by: Veeva Industry Research: See new results from one of the industry's largest, global clinical operations surveys. Sponsored by: Medidata Accelerate Clinical Research as Study Complexity Grows Sponsored by: Veeva Ready to modernize your RIM environment? Secure buy-in with these tips. Sponsored by: Patheon, part of Thermo Fisher Scientific Biologics, orphan drugs, and precision medicine are on the rise, and that means some big changes for drug development and manufacturing companies. What should pharma companies look for when navigating this new era of small-volume manufacturing? Here are some ideas to consider when developing a new manufacturing strategy. Sponsored by: Veeva See results from the Tufts eClinical Landscape Study – CRO Report on current data management practices, goals, and challenges for contract research organizations (CROs). Sponsored by: Catalent Develop a basic understanding of principles and concepts of bioavailability of oral drugs. Drug Approval, Manufacturing Quality & Regulation 2018 | Online The Life Science Women's Conference August 8-9, 2018 | Austin, Texas BIO Latin America 2018 September 4-5, 2018 | São Paulo, Brazil RESI HealthTech Week September 5-7, 2018 | Boston, MA 4th Annual Bio/Pharmaceutical Product Launch Summit September 25-26, 2018 | Cambridge, MA Drug Development Immersion September 26-27, 2018 | West Windsor, NJ 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 2018 BIO Investor Forum October 17-18, 2018 | Westin St. Francis, San Francisco, CA FierceBiotech Executive Summit: London October 30, 2018 | Royal Society of Medicine Drug Development Boot Camp® 2018 – Register Now! November 14-15, 2018 | Boston, MA | Register now and start your pre-Boot Camp preparation Field Service Amsterdam November 27-28, 2018 | NH Collection Amsterdam Grand Hotel Krasnapolsky | 