Adynxx Inc. used the same measure to select trial patients as other pain drug developers have used to weed them out – the Pain Catastrophizing Scale (PCS) – and arrived at what the company is calling a phase II success that greases the skids for a non-opioid phase III in a hard-to-treat patient sector.
In February, when Biogen Inc. said it was adding to the phase III effort with aducanumab in Alzheimer's disease (AD), language by the firm's chief medical officer (CMO), Alfred Sandrock, might have raised a red flag, or at least a pink one. "We did see more variability on the primary endpoint than assumed when we did the original sample size estimation, so we decided to increase the sample size" by 510 patients in the two studies, he said during a Leerink Partners health care conference.
Verastem Inc. CEO Robert Forrester told BioWorld that the company seeks to share the upper atmosphere's "rarefied air" with small-to-mid cap biotechs that have reached the market with new oncology drugs to fly the same skies as big pharma. "These things don't happen very often," he noted. "If we are fortunate, we will be the 11th" such smaller firm since 2010 to win clearance for a promising cancer compound.
With multiple first-time clinical data readouts due this year, some of which – in the view of Raymond James analyst Reni Benjamin – could "transform the company," immuno-oncology (I-O) player Leap Therapeutics Inc. is readying for the first round at the American Association of Cancer Research (AACR) meeting in Chicago starting Saturday.
Last August, after Merck & Co. Inc. disclosed positive but not especially compelling results with its cholesteryl ester transfer protein (CETP) inhibitor, anacetrapib, Credit Suisse analyst Vamil Divan concluded that the company made the right decision to quit, although drugs in the class "may still have a role in patients with certain genetic predispositions."
Alexion Pharmaceuticals Inc. gained air time in mid-March for proving in phase III the long-acting, intravenous (I.V.) C5 complement inhibitor ALXN-1210 non-inferior to the company's own Soliris (eculizumab) in complement inhibitor treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). But the company's hardly the only game in town.
Sensitive investors put a dent in shares of Rigel Pharmaceuticals Inc. after finding out about phase II data with fostamatinib in IgA nephropathy (IgAN), but others kept the faith as the PDUFA date nears for the same compound in chronic or persistent immune thrombocytopenia (ITP).
Next steps with FDA-stalled ALKS-5461 in major depressive disorder (MDD) won't be known until Alkermes plc sits down with regulators, but maybe data from the ongoing phase IIIb study 217 with the oral, once-daily therapy can satisfy the demand for more data.
With Fibrogen Inc. and U.S. partner Astrazeneca plc, of London, set to roll out data in the fourth quarter on roxadustat (roxa) for anemia associated with chronic kidney disease (CKD) – and with a filing for approval possible in the first half of next year – speculation has begun about the oral hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor's odds for success.
As Edge Therapeutics Inc. sifted through the wreckage of its phase III trial with EG-1962 (nimodipine microparticles), analysts wanted an explanation of how the study could have passed the futility analysis – which just happened in December – by the data monitoring committee (DMC), and fail the interim peek.
