Deciphera Pharmaceuticals Inc. CEO Steven Hoerter told BioWorld Asia the FDA approval of Turalio (pexidartinib) from Daiichi Sankyo Co. Ltd. last week "leaves a lot of headroom for efficacy," given the response rate of about 40%. "One of the most significant concerns that the FDA had was hepatotoxicity, which is an off-target effect," he added, so the approval came with "some fairly onerous monitoring requirements."
Tokyo-based Daiichi Sankyo Co. Ltd.'s Turalio (pexidartinib) won FDA clearance for adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not responsive to improvement with surgery.
Deciphera Pharmaceuticals Inc. CEO Steven Hoerter told BioWorld the FDA approval of Turalio (pexidartinib) from Daiichi Sankyo Co. Ltd. last week "leaves a lot of headroom for efficacy," given the response rate of about 40%.
Tokyo-based Daiichi Sankyo Co. Ltd.'s Turalio (pexidartinib) won FDA clearance for adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not responsive to improvement with surgery.
With data from the phase III, 341-patient Reset trial due soon, investors in Glycomimetics Inc. eagerly await the findings to be unveiled by partner Pfizer Inc. with rivipansel to treat vaso-occlusive crisis (VOC) in sickle cell disease (SCD).
Aztherapies Inc.'s new phase III approach in Alzheimer's disease (AD), ALZT-OP1, combines two well-characterized small molecules in optimized form – inhaled mast cell stabilizer cromolyn and orally given ibuprofen – for a multimodal attack of the condition at an early stage, tamping down "the big fire that's burning in the brain," Chief Medical Officer (CMO) Karen Reeves told BioWorld. Formerly with New York-based Pfizer Inc., Reeves was not the first to characterize AD as a "ticking time bomb."
Trial investigator Neal Shore told BioWorld that Bayer AG's just-approved oral androgen receptor inhibitor (ARI), Nubeqa (darolutamide), for non-metastatic castration-resistant prostate cancer (CRPC) brings a pronounced advantage in adverse events (AEs) over two other approved oral drugs in the class.
Calling the tie "magic," Pfizer Inc. CEO Albert Bourla defended his firm's decision to spin off its Upjohn unit and merge in an all-stock deal with Mylan N.V., creating a new entity to be named later with pro forma revenues as high as $20 billion. "We have a commercial footprint that is very much focused on China, on emerging markets," he said, which will help Mylan.
What the phase III multiple sclerosis (MS) win scored by Biogen Inc. and partner Alkermes plc might mean in the marketplace for diroximel fumarate – tested against their approved MS therapy Tecfidera (dimethyl fumarate) – became fodder for speculation as investors weighed the fine points of side-effect profiles and would-be reimbursement hitches.
