The latest chapter in the long saga of Repros Therapeutics Inc.'s quest to get an estrogen blocker approved for secondary hypogonadism in overweight men gave investors cause for both optimism and concern, as the FDA's acceptance for filing of the new drug application (NDA) brought questions about the timing of an advisory committee meeting – and what U.S. gatekeepers might want next.
The shake-up caused by convincing phase Ib data from Dyax Corp. with its subcutaneously delivered kallikrein inhibitor DX-2930 for preventing attacks of hereditary angioedema (HAE) left followers of Shire plc mulling the future of that firm's marketed C1-esterase inhibitor Cinryze, given intravenously, and may have sweetened the prospects for Biocryst Pharmaceuticals Inc.'s early stage, oral, kallikrein-targeter BCX-4161.
Thrasos Therapeutics Inc.'s $21 million series D financing will let the firm push further into chronic kidney disease (CKD) with a separate compound while pursuing acute kidney injury (AKI) in patients undergoing major surgery with lead compound THR-184.
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.
