Getting a drug through today's rigorous and costly development and regulatory processes – a 2014 Tufts study estimated more than a decade and $2.6 billion in expenses – might seem like the endgame. But the next step, getting that drug into the hands of doctors and patients, is just as crucial, and that's where a successful direct-to-consumer (DTC) marketing campaign could make the difference between a market leader and an also-ran.
A much-hyped January alliance with the MD Anderson Cancer Center bringing together a suite of technologies in the red hot CAR T-cell space caught the attention of Merck Serono, which inked a potential $941 million deal with Intrexon Corp.
Late 2013 start-up Aeglea Biotherapeutics Inc. attracted investments from big pharma venture arms Lilly Ventures and Novartis Venture Fund, which co-led the firm's $44 million series B round to support initial clinical work testing its amino acid-degrading approach in both a rare disease indication and cancer.
Teva Pharmaceutical Industries Ltd.'s decision last year to divest its R&D oncology assets ended up being Ignyta Inc.'s gain, with the latter picking up rights to four clinical and preclinical compounds in an asset purchase deal that triples the size of its pipeline and sets the stage for master protocol-designed trials of multiple targeted therapies.
Cidara Therapeutics Inc. became the latest firm to file for a proposed IPO, aiming to raise $69 million to advance its preclinical antifungal compounds into human testing, even as the rate of biotech IPO pricings looks to be dropping off.
Trying to translate discovery research and early clinical data into phase III success has proved the bane for drug developers working in Alzheimer's disease. But executives at Agenebio Inc., which is gearing up to start a phase III study in the second half of this year with lead product AGB101, think their approach will be up to the task.
CTI Biopharma Corp.'s pacritinib hit the primary endpoint in the first phase III study evaluating the next-generation JAK2/FLT3 multikinase inhibitor in myelofibrosis patients, with top-line data also showing benefit in a subset currently not helped by marketed JAK2 inhibitor Jakafi (ruxolitinib), findings that could provide crucial differentiation in the marketplace.
The latest foray in the oft-thorny RNAi patent landscape sees Arrowhead Research Corp. nabbing the entire RNAi portfolio of Novartis AG, picking up rights to a host of intellectual property (IP) and assets, including an existing license agreement with RNAi powerhouse Alnylam Pharmaceuticals Inc., a pipeline of three preclinical candidates and a patent estate the Pasadena, Calif.-based biotech said falls safely outside the scope of competitors' IP.
In the biopharma space, finding potential new uses for shelved drugs has become a business model unto itself, with proponents touting successes such as AZT – a drug that bombed in cancer trials only to return two decades later as the first approved therapy for HIV – and the National Institutes of Health even endorsing the strategy, setting up the National Center for Advancing Translational Sciences as a matchmaker to hook up researchers with failed compounds in 2012.
Rather than seeking a new partner after a worldwide licensing deal with Roche AG quietly terminated last year, privately held Chiasma Inc. decided to take its new drug application (NDA)-ready oral octreotide to market in the U.S. on its own.
