As Biomarin Pharmaceutical Inc. unveiled a data analysis comparing the efficacy of Kyndrisa (drisapersen) in similar patients across three randomized studies, the FDA let fly with briefing documents for Tuesday's meeting of the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee (adcom).
Lightlake Therapeutics Inc., a small New York-based biopharma, gained some big interest following FDA approval of Narcan (naloxone hydrochloride) for emergency treatment of known or suspected opioid overdose. Lightlake developed the nasal spray, which will be marketed by partner Adapt Pharma Ltd., of Dublin.
Although it was founded in 2006, the roots of Swedish biotech Pledpharma AB go back almost 20 years earlier, to the discovery of a class of compounds known as pyridoxyl ethyldiamine, or "PLED," derivatives, which were discovered in the U.S. in the late 1980s by Salutar Inc. and used as MRI contrast agents.
With Biogen Inc. veteran Doug Williams at its helm as founding president and CEO and a scientific platform from the University of Texas MD Anderson Cancer Center powering its engines, Codiak Biosciences Inc. collected the first installment of a combined series A/B financing that's expected to exceed $80 million.
In a stunning turn for its oncology drug, rociletinib (CO-1686), Clovis Oncology Inc. reported that the FDA wants additional clinical data to evaluate the efficacy of the 500 mg and 625 mg dose groups in the treatment of NSCLC patients with initial activating epidermal growth factor receptor mutations, as well as the dominant resistance mutation T790M.
Baxalta Inc. expanded its hemophilia franchise with FDA approval of Adynovate (antihemophilic factor [recombinant], pegylated) to treat hemophilia A. The therapy, which was built around the company's existing treatment, Advate (octocog alfa, or antihemophilic factor [recombinant]), with pegylation technology from Nektar Therapeutics Inc., extends treatment times to twice-weekly dosing, compared to conventional therapy of three to four doses per week.
Astrazeneca plc moved AZD9291 (osimertinib) across the finish line in rapid fashion, gaining accelerated approval from the FDA a little more than 30 months after advancing the drug into the clinic in March 2013.
The decade-old drumbeat for clinical trials transparency grew louder with release of a paper in BMJ Open about the dearth of voluntary data reporting – sometimes even from late-stage trials – on drugs that were subsequently approved.
Days after luring Sanofi SA to a potential multi-billion-dollar diabetes deal, the pipeline of metabolic assets at Hanmi Pharmaceuticals Co. Ltd. caught the eye of another big pharma. Janssen Pharmaceuticals Inc., a unit of Johnson & Johnson, of New Brunswick, N.J., gained exclusive global rights outside Korea and China to develop and commercialize Hanmi's oxyntomodulin-based therapies, including HM12525A (LAPSGLP/GCG), a GLP-1/glucagon receptor dual agonist.
It's been a long road for regenerative medicine pioneer Ocata Therapeutics Inc., founded in 1994 as Advanced Cell Technology Inc. (ACT), where one of its earliest deals was a $10 million collaboration with Genzyme Transgenics Corp. to create cloned cows capable of producing human serum albumin in their milk. (See BioWorld Today, Oct. 9, 1997.)
