While partisan squabbling holds hostage the 2019 appropriations for several U.S. departments and agencies, companies that must deal with those agencies are left scratching their heads about what's open and what's not. Read More
Myokardia Inc. CEO Tassos Gianakakos said officials at his company "anticipated and were prepared for" Sanofi SA's decision not to extend the partnership begun in 2014, instead returning rights to pivotal phase III-stage candidate mavacamten (previously known as MYK-461), an oral, allosteric modulator of cardiac myosin to treat obstructive hypertrophic cardiomyopathy (HCM), as well as MYK-491, which works similarly and is expected to yield phase IIa proof-of-concept data in dilated cardiomyopathy (DCM) before the end of this year. Read More
After nearly a decade of labor, PIN Pharma Inc. reported success in its first-in-human, open-label, repeat-dose trial of immunomodulating precision immune stimulant (PIN)-2 in participants with advanced solid tumors. The New York-based company said rapid onset of action was signaled by a surge in circulating tumor necrosis factor (TNF)-alpha six hours after injection of PIN-2, a derivative of the HIV-1 Tat protein that activates monocyte-derived dendritic cells. Circulating TNF-alpha levels subsequently returned to baseline. A similar but more pronounced finding was observed during a second cycle of treatment, confirming proof of mechanism for the immunomodulatory effect of PIN-2 and corroborating results seen in human monocytes in vitro. Read More
HONG KONG - Immuno-oncology company Ziopharm Oncology Inc. and cell therapy company Triarm Therapeutics Ltd. are set to establish a joint venture (JV), Eden Biocell Ltd., to develop and commercialize CAR T therapies targeting CD19 antigen in the greater China region and South Korea, with the use of a technology platform named Sleeping Beauty. Read More
HONG KONG – Chugai Pharmaceutical Co. Ltd., a Japanese subsidiary of Roche Holding AG, won approval from Japan's Ministry of Health, Labour and Welfare (MHLW) for Foundationone CDx, Foundation Medicine Inc.'s genomic profiling test for solid tumors and companion diagnostics for molecularly targeted drugs. Read More
Cybrexa Therapeutics, a New Haven, Conn.-based company developing a solid tumor-targeting technology to work with an already-approved PARP inhibitor, said it plans to submit an investigational new drug application for its lead candidate, CBX-11, by the fourth quarter of 2019. A phase I trial evaluating the drug is expected to begin in the first quarter of 2020. If preclinical findings hold, the approach could potentially enhance the therapeutic index of the PARP inhibitor, improve overall survival and limit the toxicities associated with combining PARP inhibitors and DNA-damaging chemotherapy. Read More
With the deadline looming for EU member states to implement the final piece of the 2011 Falsified Medicines Directive, the U.K.'s Medicines and Healthcare Products Regulatory Agency updated its guidance for prescription drug companies on how to comply with the safety features regulation. Read More
Novan Inc., of Morrisville, N.C., appointed Paula Brown Stafford president and chief operating officer, Carri Geer senior vice president and chief technology officer and Elizabeth Messersmith, chief development officer. Read More
Prana Biotechnology Ltd., of Melbourne, Australia, said it entered a securities purchase agreement for a lead investment by Life Biosciences LLC, of Boston, to raise up to approximately AU$44.5 million (US$31.4 million). It will initially invest $7.5 million, with the agreement allowing the company to raise an additional $2 million from other investors. Read More
Briacell Therapeutics Corp., of Berkeley, Calif. and Vancouver, British Columbia, said it has completed development of a new frozen formulation of its lead candidate, the cellular immunotherapy Bria-IMT. The new formulation will enable on-demand shipment to clinical sites to accommodate higher patient volumes at reduced per-dose costs vs. the old formulation, the company said. Read More
