Vertex Pharmaceuticals Inc. is targeting third quarter filings in the U.S. and Europe after reporting positive data from two phase III studies testing oral CFTR corrector tezacaftor (VX-661) plus approved CFTR potentiator Kalydeco (ivacaftor) in cystic fibrosis (CF), a combination expected to broaden the swath of patients eligible for therapy.
Even as Tesaro Inc. prepares to launch newly approved Zejula (niraparib) late next month in ovarian cancer, the Waltham, Mass.-based company already is setting sights on an expanded clinical development program for the PARP inhibitor, which is not restricted to patients with BRCA mutations.
The advent of checkpoint inhibitors yielded a big win for the burgeoning immuno-oncology space, supercharging activated T cells to attack cancer cells, and the success with drugs targeting CTLA-4 and PD-1 has translated into efforts chasing other checkpoint mechanisms, all in the hopes of expanding the stunning results into broader patient populations.
BARCELONA, Spain – With drug pricing in the political crosshairs and the first gene therapies advancing toward the market, the health care sector will have to figure how to put a price tag on therapies that provide durable, curative responses.
BARCELONA, Spain – The new U.S. president has been in office for two months. During that time, he has – among other moves – pledged to reduce regulatory hurdles during a meeting with biopharma execs, sent biopharma stocks falling on a tweet on drug pricing, nominated an industry-friendly Scott Gottlieb as FDA commissioner and last week released a proposed White House budget for fiscal 2018 that calls for a $15.1 billion bite out of the Health and Human Services budget, including nearly $6 billion from the NIH.
Egenesis Inc., a 2015 startup that has helped put xenotransplantation back on the table as a potentially viable option for organ transplant thanks to its founders' work using CRISPR gene editing technology, closed its first substantial financing, a $38 million series A expected to take development efforts through preclinical proof of concept.
To say that the discovery leading to Aobiome LLC's approach to treat disease by targeting the microbiome came via an unconventional method is an understatement. As the story goes, founder David Whitlock, a Massachusetts Institute of Technology chemist, during a stroll at a local farm, began wondering why horses rolled around in the dirt.
NEW YORK – The Affordable Care Act (ACA) will be repealed, PDUFA VI will almost certainly be passed and the pressure on drug pricing will not go away in the coming year. While panelists during a panel on policy outlook under the new Trump administration largely agreed on those predictions, some noted that gaining a bit of certainty could be the biggest boon for the industry in 2017.
NEW YORK – The 21st Century Cures Act signed into U.S. law last year included a provision for $500 million in funding for prevention and treatment programs targeting the opioid epidemic.
Despite all the advances in drug research and development, the human proteome remains largely untapped – of the estimated 20,000 proteins, only about 600 are currently targeted by FDA-approved drugs. That leaves a lot of opportunity on the table for Vividion Therapeutics Inc., a San Diego-based firm launching Thursday with a $50 million series A investment co-led by Arch Venture Partners and Versant Ventures.
