Ultragenyx Pharmaceutical Inc. said it believes data from a phase III study testing burosumab (KRN23), a recombinant fully human monoclonal IgG1 antibody, are sufficient to support a BLA filing this year in X-linked hypophosphatemia (XLH), a bone disease characterized by phosphate wasting due to excess activity of FGF23.
When going after macrophages, "in my view, you play directly with the immune system," said Maxime Ranger, president and CEO of 2016 startup Mperia Therapeutics Inc., which is advancing macrophage-targeting immunotherapies based on the CD36 scavenger receptor for diseases involving chronic inflammation such as dry age-related macular degeneration (AMD) and atherosclerosis.
Wave Life Sciences Ltd. boasted a balance sheet of $150.3 million as of Dec. 31, funds the company said during its fourth-quarter earnings should get the firm into 2019. But as always in the field of drug development, a little extra padding never hurts, and Cambridge, Mass.-based Wave Life priced a $100 million public offering Wednesday as it gets ready to move its first three candidates into the clinic targeting CNS and neurological disorders.
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.
