DUBLIN – Merck & Co. Inc. is paying €115 million (US$137 million) up front and up to €349 million more in milestones to acquire Rigontec GmbH, which is developing an RNA-based immuno-oncology therapy, based on turning on the retinoic acid-inducible gene I (RIG-I) innate immune pathway to achieve an antitumor effect. Read More
Voyager Therapeutics Inc.'s shares (NASDAQ:VYGR) rose 24.7 percent to $13.63 on Wednesday as the company announced plans to embark on a pivotal phase II/III program testing its adeno-associated virus 2 (AAV2) gene therapy, VY-AADC-01, in advanced Parkinson's disease (PD) later this year, encouraged by early stage tests in a small group of PD patients. Dosing of the first patient in the pivotal program is expected in the first half of 2018. Read More
LONDON – Bioverativ Inc. is teaming up with Bicycle Therapeutics Ltd. to develop new treatments for hemophilia and sickle cell disease, in a deal worth $424.2 million to Bicycle if two products make it to market. Read More
Eagerly awaited data from Verastem Inc.'s phase III DUO study should "more than satisfy the skeptics," CEO Robert Forrester told BioWorld as the firm unveiled positive top-line data with duvelisib for relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL). "This drug, as we saw from the [earlier] DYNAMO study, is clearly active," he added, referring to a phase II experiment in non-Hodgkin's lymphoma (NHL), results from which investors deemed less than thrilling. Read More
Chatter about a potential new drug application (NDA) filing for golodirsen (SRP-4053) started as soon as Sarepta Therapeutics Inc. reported that the phase I/II study (4053-101) of the exon 53 skipping candidate achieved statistical significance on its primary and secondary biological endpoints in boys with confirmed deletions of the gene, one of several associated with Duchenne muscular dystrophy (DMD). Read More
The Senate Appropriations subcommittee charged with the budget for the NIH passed a bill that would boost spending at the agency by $2 billion, and while both the chairman and the ranking member of the subcommittee said this is not a bill they would have preferred, the full committee hearing will allow committee members to propose amendments. Read More
The Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) filed suit in the U.S. District Court for the District of Nevada challenging sections of a Nevada state law that they said would violate patent rights and negate trade secret protection for certain diabetes drugs. Read More
Conatus Pharmaceuticals Inc., of San Diego, named Keith W. Marshall executive vice president, chief operating officer and chief financial officer. Read More
Poseida Therapeutics Inc., of San Diego, reported that P-BCMA-101, its B-cell maturation antigen (BCMA)-specific chimeric antigen receptor T-cell (CAR T) candidate for the treatment of multiple myeloma, typically reduced tumor burden to the limit of detection within six to nine days in what it called "an ultra-stringent" p53 knockout mouse xenograft model. Read More
Excision Biotherapeutics Inc., of Philadelphia, said it completed a $10 million seed round, led by Artis Ventures, which will enable the company to enter human trials with its HIV-1-targeted CRISPR platform. Read More
Rxi Pharmaceuticals Corp., of Marlborough, Mass., completed enrollment in its phase II RXI-SCP-1502 trial testing Samcyprone, an improved topical ointment formulation of DPCP, as a treatment for cutaneous warts. Read More