Myovant Sciences Ltd. was thrilled with Tuesday's data announcement from the first of two phase III studies of relugolix, in which Liberty 1 met its primary efficacy endpoint and six secondary endpoints in women with uterine fibroids. The results keep the phase III races tight between relugolix, Abbvie Inc.'s elagolix and Obseva SA's OBE-2109. Read More
DUBLIN – Meiragtx Holdings plc remains on course to move its second-generation adeno-associated virus (AAV)-based gene therapy for inherited retinal disorders arising from RPE65 (retinal pigment epithelium-specific 65 KDa protein) deficiency into a pivotal trial shortly, following a six-month readout from a phase I/II trial in which the construct met the primary endpoint of safety and tolerability and achieved statistical significance on several measures of clinical activity. Read More
As expected, Tokyo-based Daiichi Sankyo Co. Ltd. faced rough sledding in its review by the FDA's Oncologic Drugs Advisory Committee (ODAC) of quizartinib tablets for adults with relapsed or refractory acute myeloid leukemia (AML) that is FMS-like tyrosine kinase (FLT) 3-internal tandem duplication (ITD) positive, as detected by an FDA-approved test. Read More
Instead of looking to D.C. for help with the hefty cost of prescription drugs, U.S. states are increasingly taking matters into their own hands, with some of them casting their eyes to the north and even further afield. Read More
SAN DIEGO – "Wherever I look, I see the ugly face of complement." That, complement researcher Jörg Köhl told the audience at the 2019 annual meeting of the American Association of Immunologists (AAI), was the comment of a research colleague who repeatedly stumbled across complement contributions to what had once seemed unrelated research. Read More
TEL AVIV, Israel – Generating all kinds of data that can feed artificial intelligence (AI) and machine learning engines is increasingly cheap and in many ways easy, but interpreting all those data and translating them into information that is useful to users, ranging from drug developers to patients, remains a significant challenge. Read More
Shares of Solid Biosciences Inc. took it on the chin again, after the company reported a serious adverse event (SAE) from the second cohort of its phase I/II Ignite DMD study testing gene therapy SGT-001 in Duchenne muscular dystrophy. The latest setback comes three months after Cambridge, Mass.-based Solid disclosed disappointing data from the first patient cohort and a year after the FDA lifted a clinical hold placed on the same trial after the first patient treated suffered an SAE. Read More
Isoplexis Corp., of Branford, Conn., which focuses on advancing single-cell biomarkers in oncology, said it raised $25 million in series C funding led by Northpond Ventures and including new and current investors Spring Mountain Capital, Ironwood Capital, North Sound Capital and Connecticut Innovations. Read More
Reform Biologics LLC, of Woburn, Mass., said it will perform feasibility studies for Bayer AG, of Leverkusen, Germany, using its technologies, in which viscosity-reducing excipients are applied to high-concentration biologic formulations to enable subcutaneous injection or device-assisted administration or to improve filtration unit operations. Financial terms were not disclosed. Read More
Gilead Science Inc.'s U.S. pricing of its HIV prevention drug, Truvada (emtricitabine/tenofovir disoproxil fumarate), led to company Chairman and CEO Daniel O'Day getting the sole invite to testify in a hearing before the House Oversight and Reform Committee Thursday. Read More
