Seven new drugs launched this year are projected to achieve more than $1 billion in sales by 2023, according to a new report published by Clarivate Analytics, which owns BioWorld. Abbvie Inc.'s rheumatoid arthritis candidate, upadacitinib, and Novartis AG's Avexis-sourced gene therapy, Zolgensma (onasemnogene abeparvovec), for spinal muscular atrophy (SMA) could lead the way, raking in $2 billion each, while Fibrogen Inc.'s anemia medicine, roxadustat, isn't far behind with anticipated sales of $1.97 billion in 2023. The list, backed by analysis of data from Cortellis, is dominated by immune-related and genetic disorders. Its composition indicates a shift toward targeting niche patient populations bolstered by strong collaborations with big and specialty pharma companies, Clarivate said.
Shares of Dermira Inc. (NASDAQ:DERM) climbed 82.2 percent to $12.61 on Monday after new data from a midstage study of its anti-IL-13 candidate, lebrikizumab (lebri), in atopic dermatitis (AD) showed a monthly dose of the drug performing roughly in line with Regeneron Pharmaceuticals Inc.'s blockbuster, Dupixent (dupilumab), which is dosed every other week. Though no head-to-head test with Dupixent was included in Dermira's phase IIb study, three different doses of lebri outperformed a placebo on a standard measure of eczema severity, showing efficacy that analysts said nearly matched Regeneron's drug.
Redpin Therapeutics Inc., a New York-based preclinical gene therapy startup leveraging a chemogenetics approach to hard-to-treat CNS diseases, has highlighted a new paper in Science detailing the ion channel-based platform behind its nascent pipeline. The approach, the rights to which Redpin exclusively licensed from the Howard Hughes Medical Institute (HHMI), enables targeted cell activation or inhibition that can be controlled by low doses of the anti-smoking drug Chantix (varenicline), it said.
Emboldened by lessons learned from an earlier endpoint-missing study, Biohaven Pharmaceutical Holding Co. Ltd. is reprising its investigation of troriluzole in the rare neurodegenerative disorder spinocerebellar ataxia (SCA) with a new phase III study that recently enrolled its first patient. The study features a higher dose, an extended treatment period and an FDA-blessed modified functional rating scale as a registrational endpoint. It's informed by an encouraging readout from a long-term extension study and a still-unmet need among thousands of patients with the debilitating genetic disorder.
A little less than a year after launching Rhopressa (netarsudil), its first drug for lowering intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension, Aerie Pharmaceuticals Inc. has won FDA approval for Rocklatan, a once-daily eye drop that adds the widely prescribed prostaglandin analogue (PGA) latanoprost to the mix.
Akebia Therapeutics Inc. said two pivotal Japanese studies of its anemia drug, vadadustat, each with active controls, met their primary endpoints by helping people with chronic kidney disease (CKD) achieve mean hemoglobin levels noninferior to Aranesp (darbepoetin alfa). The results will support Akebia's commercial partner, Mitsubishi Tanabe Pharma Corp. (MTPC), in readying a new drug application seeking Japanese regulatory approval this year.
Turnaround bets placed by Axovant Sciences Ltd. on early stage gene therapies in the wake of its failed bid to tackle Alzheimer's disease (AD) showed hints of paying off, boosting company shares (NASDAQ:AXGT) by 14.2 percent to $1.69 on Monday. Positive interim results from two patients in a phase II study of AXO-Lenti-PD in Parkinson's disease (PD) and initial data on its Tay-Sachs disease candidate, AXO-AAV-GM2, put both programs on track for further development.
Formation Biologics Inc., a company more commonly called Forbius that's developing biologics for fibrosis and cancer, has dosed the first patient in a phase IIa study designed to evaluate its EGFR-targeting antibody-drug conjugate, AVID-100, in patients with EGFR-overexpressing squamous cell carcinoma of the head and neck (SCCHN).
Top-line data from three pivotal studies of Allergan plc's NMDA receptor modulator, rapastinel, in major depressive disorder (MDD) found adjunctive therapy with the candidate no better than a placebo in improving symptoms of the condition. An interim analysis of a fourth trial, in preventing relapses, appeared unlikely to meet its endpoints, either, the company said.
