FDA approval of patisiran, branded Onpattro, to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) represents several firsts for Alnylam Pharmaceuticals Inc., not least of which is the distinction of bringing the initial RNAI interference (RNAi) drug to market. Although the Cambridge, Mass.-based company also beat competitor Akcea Therapeutics Inc., which is developing Tegsedi (inotersen), to first approval in the hATTR indication, Alnylam now faces the challenge of wooing physicians and patients to a drug in a class without precedent. Read More
Researchers at Memorial Sloan-Kettering Cancer Center (MSKCC) have reported both preclinical data and a case study of a patient treated under a compassionate use protocol indicating that the tropomyosin receptor kinase (TRK) fusion inhibitor larotrectinib (LOXO-101, Loxo Oncology Inc.) is effective in hematological malignancies as well as in solid tumors, where it is currently in late-stage clinical trials. Read More
Co-Diagnostics Inc., of Salt Lake City, closed a nonconvertible debt instrument of $2 million that it plans to use for working capital, to expand distribution of its infectious disease testing products and to accelerate its plan to develop multiplex screens for liquid biopsy cancer screening, blood-bank screening and SNP detection. Read More
Puma Biotechnology Inc., of Los Angeles, reported second-quarter sales of $50.8 million for its adjuvant breast cancer treatment, Nerlynx (neratinib), a 41 percent increase over the first quarter. Read More
A1M Pharma AB, of Lund, Sweden, said the company's Rosgard shows a "strong protective effect" against acute kidney injury in a preclinical study conducted in collaboration with Indiana University, results that are in line with a similar European study. Read More
A collaborative study by Harvard University and Chinese researchers has identified a previously unrecognized and clinically prevalent class of Mycobacterium tuberculosis variants that alter propionate metabolism and mediate multidrug tolerance, thereby driving drug resistance. Read More
Gemphire Therapeutics Inc.'s gemcabene has been given to about 1,200 adult patients in 25 phase I and phase II trials for up to 12 weeks without any drug-related severe adverse events, but that winning safety streak came to an abrupt and puzzling end as the data and safety monitoring board (DSMB) overseeing a phase IIa study in pediatric subjects recommended the study be stopped because patients apparently were getting sicker. Read More
Like popcorn in a pan, data with late-stage drugs for nonalcoholic steatohepatitis (NASH) will burst forth from a series of drug developers, and Wall Street is hard pressed to figure out which treatment – or combination thereof – might work best. But that hasn't stopped them from trying. Read More
LONDON – Artios Pharma Ltd. raised $84 million in an oversubscribed series B, attracting leading corporate venture funds to invest in its next-generation small-molecule inhibitors of DNA damage response (DDR). Read More
HONG KONG – Chinese firm Cure Genetics Co. Ltd., of Suzhou, has secured $17 million in series A financing to advance its gene-editing and delivery platform to treat easy-to-relapse or hard-to-treat cancers and genetic diseases. Read More
