A deuterium-driven breakthrough in the push for a safer, better therapy in Huntington’s disease (HD) may have come in the form of Auspex Pharmaceuticals Inc.’s SD-809 (deutetrabenazine), an altered form of black-boxed, standard-of-care Xenazine (tetrabenazine, H. Lundbeck A/S).
ust as speculation dwindled about the odds of Teva Pharmaceutical Industries Ltd. making a play for generic drug maker Mylan Inc., the Jerusalem-based pharma giant made known its plan to enliven the pipeline by way of a different deal: a $3.2 billion tender offer for all of orphan-drug firm Auspex Pharmaceuticals Inc.'s outstanding shares at $101 each.
Data-analysis methods and variability of patient response figured into the second-half phase II surprise for Ohr Pharmaceutical Inc. with its eye-drop combination therapy for wet age-related macular degeneration (AMD), but the company said lessons from the study – results of which are still being sorted – will optimize the phase III program, due to start a few months later than originally planned.
Flagship Ventures' latest $537 million raise for its fifth fund – the largest since the firm's founding in 2000 – will let its Venturelabs innovator unit, which has been churning out two new companies per year, generate four to five in the future.
For VBL Therapeutics Inc., positive interim top-line phase II data with its gene therapy for recurrent glioblastoma took the sting out of February's news with a separate compound targeting psoriasis and ulcerative colitis, also each in a phase II trial.
What CEO Daniel Gold called a "ticking time bomb" apparently went off in the 102-patient phase II trial testing MEI Pharma Inc.'s histone deacetylase (HDAC) inhibitor pracinostat in combination with Vidaza (azacitidine, Celgene Corp.) against previously untreated intermediate-2 or high-risk myelodysplastic syndrome (MDS), as top-line data show the pair performed no better than Vidaza by itself.
Unlike Alzheimer's disease (AD), which took much of the spotlight Friday, "there's not another gene to argue over" in Parkinson's disease (PD), said CEO Dale Schenk, commenting on Prothena Corp. plc's phase I success with PRX002, which takes aim at alpha-synuclein.
What the post-approval confirmatory trial might look like and the outcome of a likely FDA advisory committee meeting are among the questions that remain for investors in Amicus Therapeutics Inc., but news of a clearer – and potentially faster – regulatory path for its Fabry disease therapy in Europe as well as the U.S. provided Wall Street with plenty of cause for optimism.
Asklepion Pharmaceuticals LLC is collecting a $27 million up-front payment from Retrophin Inc., plus about 661,278 shares of stock and the potential for $37 million more in sales-milestone payments, along with tiered royalties on the back end, thanks to a January agreement for the rights to Cholbam (cholic acid) once the drug was approved by the FDA.
Kite Pharma Inc. has augmented its T-cell receptor (TCR) platform and is making itself known in Europe via the buyout of privately held T-Cell Factory B.V. for €20 million (US$21 million) up front to the Amsterdam-based firm's shareholders, licensors and employees.
