In conversation with Samumed CEO Osman Kibar: Drugging Wnt, restoring youth and unconventional capital
Straight A’s for MarzAA
Last month at the 2019 Congress of the International Society on Thrombosis and Haemostasis (ISTH), Catalyst Biosciences Inc. presented Phase 2 data regarding its subcutaneous (SQ) Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA) for prophylaxis in patients with hemophilia A or B with inhibitors. The study met its primary endpoint of significantly reducing the annualized bleed rate, as well as all secondary endpoints of safety, tolerability and lack of anti-drug antibody or inhibitor formation.
ProQR’s retinitis pigmentosa candidate gets investigational NDA clearance
The FDA has cleared an investigational new drug application for QR-1123, an investigational oligonucleotide treatment candidate for vision loss with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene, according to a press release from ProQR Therapeutics.
Inside the Arena Makeover | Kevin Lind, CFO, Arena Pharmaceuticals
Up until about three years ago Kevin Lind would likely have been identified as yet another gifted private equity executive – capable of issuing business remedies from the tip of his tongue. At least in the minds of his CFO peers, who are accustomed to listening to PE pundits routinely hand down such remedies for ailing businesses. For Lind the CFO office at Arena Pharmaceuticals is a game changer, where he no longer hands down remedies but serves as a finance leader – an individual tasked with summoning others forward and building trust across an organization even as he or she sometimes completes necessary layoffs.
Initial Reports Positive for Plasma Treatment of Alzheimer Disease
Officials are optimistic about the initial results of a phase 2 clinical trial for a new plasma-based treatment for mild-to-moderate Alzheimer disease (AD). California-based Alkahest announced that GRF6019, a proprietary plasma fraction comprised of about 400 proteins, was both safe and well-tolerated with no cognitive decline as a treatment for AD.
Alzheimer’s patients didn’t decline after getting a cocktail derived from young blood, but big questions remain
Alzheimer’s patients who received an experimental protein cocktail derived from young blood plasma maintained their performance on measures of cognition and function after six months, the biotech company behind the therapy, Alkahest, said on Monday.
NeuBase takes antisense oligos to more targets in more places
Newcomer NeuBase Therapeutics is re-engineering antisense oligos to access a broader range of targets, while simultaneously solving the modality’s dosing and delivery issues. With three approvals since the start of 2016, antisense oligonucleotides (ASOs) have started to hit their stride, yet first-generation technologies are limited on multiple fronts.
