Slingshot members are tracking this event:

Arrowhead Pharmaceuticals (ARWR) Makes Poster Presentations of New Data on ARC-AAT

Do you think this event is important to the companies below? How will it affect their stock price?

Related Companies
High Medium Low
Impact on Stocks
ARWR Community voting in process

Additional Information

Clinical Data The poster titled, "RNA interference (RNAi) with ARC-AAT provides deep and prolonged knockdown of alpha-1 antitrypsin levels in healthy volunteers," publication LB-24 in the late-breaking poster session, describes data from a Phase 1, multi-center, randomized, placebo-controlled, double-blind, single dose-escalation first-in-human study to evaluate the safety, tolerability, pharmacokinetics of ARC-AAT and the effect on circulating alpha-1 antitrypsin (AAT) levels. Key findings from the study include the following: Š Dose-dependent reductions in serum AAT of up to 90% were observed Š Duration of effect indicates that monthly, or less frequent, dosing is likely Š Pharmacokinetic (PK) parameters were linear across dose levels with a constant half-life Š There have been no drop outs due to adverse events (AE), no clinically significant changes in ECGs, DLCO or FEV1, and one serious adverse event (SAE) in a placebo subject Š No clinically significant transaminase (ALT, AST) elevations were reported Š The most frequently reported ARC-AAT related AEs were headache, nausea and rigor (each, 3 events in 36 [8%] subjects) The oral presentation titled, "RNA interference therapeutic ARC-AAT prevents production of Z-alpha1 antitrypsin polymers and reverses liver disease phenotype in PiZ mouse model," publication 124 in the session Parallel 19: Pediatric and Metabolic Liver Diseases: Basic and Translational, describes data from a 33-week study of ARC-AAT in the PiZ mouse model. Key findings from the study include the following: Š Cleared Z-hAAT protein from the cytoplasm and reduced by > 90% in serum Š Prevented and reversed polymer accumulation, with Z-hAAT monomer reduced by 87% and polymer by 42% at week 33 Š Halted accumulation of Z-hAAT globules in the liver, with 61% less in ARC-AAT treated compared to saline controls and 24% less than at baseline Š Improved liver health and prevented further damage based on histopathology improvements compared to baseline and saline controls Š Prevented liver inflammation with fewer inflammatory foci and reduced total area of inflammation Š Prevented liver tumors Š Normalized gene expression associated with liver disease 
Slingshot Insights Explained
Catalyst Date
Occurred on:
Nov 14, 2016
Related Projects Image
  • Don’t see a project related to the catalyst you care about?

Related Keywords Arc-aat, The Liver Meeting, Liver Disease