Phasebio Pharmaceuticals Inc., of Malvern, Pa., gained FDA orphan status for Vasomera (PB1046) injection for the treatment of cardiomyopathy associated with dystrophinopathies, including Duchenne muscular dystrophy, Becker muscular dystrophy and X-linked dilated cardiomyopathy. The company plans to initiate a phase IIa study of PB1046 in the first quarter of 2016. Read More
Remedy Pharmaceuticals Inc., of New York, said it completed a $9 million financing led by Vertical GP-5 LLC, an affiliate of The Vertical Group, and other new investors with participation from existing investors. The funds will be used to accelerate the company's pathway to a phase III study of its lead compound, Cirara, a high-affinity inhibitor of Sur1-Trpm4 channels, in severe stroke. Read More
Otonomy Inc., of San Diego, said it completed enrollment in a phase II trial of Otiprio in patients with acute otitis externa, also known as swimmer's ear, with preliminary results demonstrating clinical feasibility for that label expansion indication. The one-month, open-label phase II trial enrolled a total of 75 pediatric and adult patients with unilateral acute otitis externa. Read More
Adaptimmune Therapeutics plc, a U.K.-based immunotherapy developer, has agreed to pay Seattle-based start-up Universal Cells Inc. $5.5 million up front and up to $41 million in milestones for an exclusive global license to a gene editing technology that could lead to the creation of new allogeneic T-cell therapies. Read More
Induced pluripotent stem cells (iPSCs) are young cells created by reprogramming old cells into a stem cell-like state via various transcription factor cocktails. And for many applications – certainly for any involving transplantation – the rejuvenation of iPSCs is one of their selling points. But for studying age-related diseases, turning back the clock in such cells means giving up important information. Read More
LONDON – Kesios Therapeutics Ltd. has raised £19 million (US$28.5 million) in a series A round that will enable the company to take the lead program targeting the nuclear factor kappa B (NF-kB) pathway through to phase II in the treatment of multiple myeloma. Read More
NEW DELHI – There has been mixed reaction – by and large welcoming – among India's pharmaceutical and biotechnology industry to a proposal by the Indian government to simplify corporate tax laws and end a "sunset" clause for companies that receive tax benefits as part of setting up in special economic zones (SEZ). Read More
A historic three-day summit on gene editing has convened in Washington amid concern that the huge potential benefits of the technology in treating human disease will be sidelined by fears about its inappropriate use to alter the germline and change human inheritance. Read More
SHANGHAI – Glaxosmithkline plc has had a rough couple of years in China. But the big pharma is seeking now to emerge from the dust of a scandal that forced it to pay a $3 billion fine for giving bribes to boost drug sales and led to suspended prison sentences for five executives and the embarrassing deportation of GSK's China general manager. Read More
