A mid-stage trial testing the Galmed Pharmaceuticals Ltd. candidate aramchol (arachidyl amido cholanoic acid) over 12 weeks found it made no apparent difference in liver fat for people with HIV-associated lipodystrophy and non-alcoholic fatty liver disease (NAFLD). While company shares (NASDAQ:GLMD) fell by 45.4 percent on the news, Galmed's chief scientific officer, Liat Hayardeny, told BioWorld that substantial differences in the pathogenesis of fatty liver in HIV and non-alcoholic steatohepatitis (NASH) – the focus of another aramchol trial due to read out next quarter – should quell concerns about any potential read-through.
Bioxcel Therapeutics Inc., a company using artificial intelligence to repurpose existing compounds for new uses, has filed to raise up to $69 million in an initial public offering intended to help it advance two candidates through proof of concept (POC), and possibly further.
Abeona Therapeutics Inc., a company developing gene therapies for the two most common types of Sanfilippo syndrome, also known as mucopolysaccharidosis III (MPS III), has reported early progress on both programs. ABO-102 (AAV-SGSH), the more advanced candidate for MPS IIIA, showed time and dose-dependent responses in biomarkers and, following a recent FDA allowance, could soon be used to treat patients earlier in the course of their disease, the company said.
Rani Therapeutics Inc., the San Jose, Calif.-based developer of a pill designed to replace injectable drugs for chronic disease, has raised $53 million to invest in manufacturing and preparation for clinical trials expected to begin within 12 months.
Gilead Sciences Inc. reported that fourth-quarter sales of its hepatitis C virus (HCV) therapies halved since last year, falling to $1.5 billion from $3.2 billion in the fourth quarter of 2016 as new competition arrived on the scene and fewer patients started treatment.
Xoc Pharmaceuticals Inc., a CNS-focused startup developing drugs for migraine prevention and Parkinson's disease (PD) maintenance, has closed a tranched $30 million series A financing led by New Enterprise Associates. The Los Gatos, Calif.-based company, founded by a team of Map Pharmaceuticals Inc. alumni, said the funds will enable it to complete preclinical testing and take its two lead compounds, XC-101 and XC-130, into the clinic.
The first of two phase III trials seeking to prove Spectrum Pharmaceuticals Inc.'s Rolontis (eflapegrastim) noninferior to Neulasta (pegfilgrastim) for chemotherapy-induced neutropenia management achieved that goal, validating the company's decision to license the drug from Hanmi Pharmaceutical Co. Ltd. in 2014.
Amgen Inc. shares (NASDAQ:AMGN), dented by missed fourth-quarter earnings expectations, regained lost ground Friday, buoyed by expectations for a lower 2018 tax rate and its plans to repurchase up to $10 billion of its own shares through a Dutch auction, starting as early as this week.
Avrobio Inc. has completed a $60 million series B financing expected to support multiple gene therapies from the company's lentiviral platform, including its lead candidate, AVR-RD-01, currently in phase I for Fabry disease.
Three U.S.-based phase I trials of Bellicum Pharmaceuticals Inc.’s lead candidate, an adjunct T-cell therapy for bone marrow transplant patients, are on an FDA-ordered clinical hold after the company reported three cases of encephalopathy possibly related to the treatment.
