LONDON – Horama SAS has closed a €19 million (US$22 million) series B round, providing the capital to start clinical development of its lead gene therapy program in the treatment of a rare inherited form of retinitis pigmentosa. Read More
The FDA is seeking White House Office of Management and Budget approval to collect information in support of a new draft guidance to help drug sponsors prepare voluntary target product profiles (TPPs). Read More
Alnylam Pharmaceuticals Inc., of Cambridge, Mass., started the phase III ENVISION trial testing givosiran, an RNAi therapeutic targeting aminolevulinic acid synthase 1, in approximately 75 patients with acute hepatic porphyrias, with a primary endpoint of annualized rate of porphyria attacks requiring hospitalization, urgent health care visit or hemin administration at home over the six-month treatment period. Read More
Milestone Pharmaceuticals USA Inc., of Charlotte, N.C., part of Milestone Pharmaceuticals Inc., of Montreal, named Lorenz Muller chief commercial officer. Read More
Archerdx, of Boulder, Colo., said it signed an agreement to develop and commercialize a next-generation sequencing-based oncology companion diagnostic (CDx) for Summit, N.J.-based Celgene Corp.’s investigational drug, CC-122, for indications in diffuse large B-cell lymphoma (DLBCL). Read More
PERTH, Australia – Companies in the regenerative medicine space have long been pushing the Therapeutic Goods Administration (TGA) to regulate autologous cell and tissue products in Australia, and they strongly support the agency’s moves toward stronger regulation. Read More
Oyster Point Pharmaceuticals Inc.’s $22 million series A financing will push two lead therapies in corneal surface disorders through phase II trials, but CEO Jeffrey Nau told BioWorld he was “not willing at the moment to comment on the mechanism of action. We want to make sure that we keep a competitive advantage for a little bit. Once we have our first clinical trial under our belt – and we’re very optimistic at the moment – we’ll share more information,” he said. Read More
An allogeneic whole cell vaccine for the treatment of non-small-cell lung cancer (NSCLC) developed by Pique Therapeutics Inc. helped people with late-stage, second-line disease achieve a median overall survival (OS) of 12.5 months during a phase II trial, an improvement of 4.1 months over the median OS of patients who received placebo. For the 71 nonsquamous patients enrolled in the study, median OS extended to 15.5 months, besting median OS in the placebo arm by nearly 10 months. Read More
BERLIN – Outsourcing has been one of the megatrends that has swept over the pharmaceutical industry in recent decades. Manufacturing, research and commercialization have become virtualized to varying degrees as companies seek to minimize capital expenditure and maximize the return on their pipeline assets. Read More
HONG KONG – With a series of advancements in the area of biosimilar development, China’s Shanghai Fosun Pharmaceutical Group Co. Ltd. is getting closer to becoming the first domestic company to market a biosimilar product in China. Read More
Treating aging rats with an experimental compound that prevented neuronal cell death protected the animals from both depressive symptoms that are often the first symptom of Alzheimer’s disease (AD), and the memory decline that follows. The compound did not, however, affect the amyloid beta (a-beta) plaques, tau phosphorylation and inflammation that are widely considered to be the toxic agents behind neuronal demise in AD. Read More
