DUBLIN – Shares in PTC Therapeutics Inc. dropped as much as 13% during premarket trading Feb. 6 on eagerly awaited 12-month data for its oral spinal muscular atrophy (SMA) drug risdiplam, which is licensed to Genentech. Although patients with type 2 or type 3 SMA demonstrated statistically significant improvements in motor function, the level of that improvement evidently disappointed some investors.
California's Amunix Pharmaceuticals Inc. said Roche Holding AG has agreed to license its therapeutic half-life extension technology, XTEN, for an effort to discover and develop new non-oncology therapies. The deal, which includes $40 million up front, plus up to $1.5 billion in development and sales milestones, builds on a previous technology evaluation agreement the companies first inked in 2013.
For the first time since Congress opened the door to biosimilars in 2010, the FDA approved nearly as many biosimilars in 2019 as it did new biologics. As the first decade of biosimilars came to a close, the agency’s Center for Drug Evaluation and Research (CDER) approved 10 biosimilars referencing seven blockbuster biologics, bringing the total number of approved biosimilars to 26.
In a move that Sarepta Therapeutics Inc.’s president and CEO, Doug Ingram, called “transformational” for the company and “the largest licensing transaction in cell or gene therapy history,” Sarepta granted Roche Holding AG exclusive commercial rights outside the U.S. for SRP-9001, its gene therapy for Duchenne muscular dystrophy (DMD).
Roche Holding AG took an interest in immunometabolism specialist Rheos Medicines Inc. even before its March 2018 launch. Now, less than two years later, it has tapped Cambridge, Mass.-based company, founded by Third Rock Ventures, to identify and develop an undisclosed number of new therapies for autoimmune and inflammatory disease.
ORLANDO, Fla. - Chimeric antigen receptor T (CAR T) cells have proved to be a major advance in treating patients with refractory B-cell malignancies so far. But, often, in approaching those issues "we solve one and we create another," Stephen Schuster, a doctor at the Abramson Cancer Center at the University of Pennsylvania in Philadelphia, told reporters at the 61st American Society of Hematology (ASH) annual meeting Saturday.
Any worries by Promedior Inc. investors after Bristol-Myers Squibb Co. (BMS) backed away last year from its option to acquire the firm were resolved in a big way by last week's news that Roche Holding AG is taking over the company in a transaction valued as high as $1.39 billion. Privately held, Lexington, Mass.-based Promedior's lead asset is PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that won breakthrough designation from the FDA earlier this year for idiopathic pulmonary fibrosis (IPF).
Roche Holding AG and PTC Therapeutics Inc. together with their partner, the SMA Foundation, reported success in part two of the pivotal Sunfish study testing their survival motor neuron-2 splicing modifier risdiplam in patients with type 2 or 3 spinal muscular atrophy (SMA).
RNAi specialist Dicerna Pharmaceuticals Inc.'s chief operating officer, James Weissman, told BioWorld that scientists inside and outside of the company have come to believe in what's called "the X hypothesis" in hepatitis B virus (HBV). "My view, though, as a person who has been in the pharmaceutical industry for decades, is who cares, anyway?" he said – as long as the drug works. He pointed out that, with the cholesterol therapy Lipitor (atorvastatin calcium) from New York-based Pfizer Inc., where he served in business development and marketing, "everybody had a different theory for why it lowered the incidence of heart disease. Nobody knew with certainty."
HONG KONG – In one of the latest combination efforts against cancer, Kahr Medical Ltd. is teaming up with Switzerland's Basel-based Roche Holding AG to study the use of the former's lead program, DSP-10, in combination with the latter's PD-L1-blocking checkpoint inhibitor, Tecentriq (atezolizumab), in patients with advanced non-small-cell lung carcinoma refractory to checkpoint inhibitors.
