Action Required: Password Reset on Next Login

As part of our recent security and system improvements, a password reset will be required the next time you log in. This applies to username/password users only; SSO users are not affected.

To reset your password, please visit the Reset Password page.

We appreciate your patience during this transition and are available to assist with any questions. For support, please call us at +1 650-552-4224 or email us at support@biocentury.com.

BioCentury
ARTICLE | Regulation

Jan. 9 Quick Takes: Arrowhead falls on unexpected high placebo response in AAT study

Plus updates from BioMarin, Santhera-ReveraGen, Day One and more

January 10, 2023 12:25 AM UTC

Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) lost about $750 million in value on Monday after the biotech and its partner Takeda Pharmaceutical Co. Ltd. (Tokyo:4502; NYSE:TAK) reported top-line data from the Phase II SEQUOIA study of fazirsiran (TAK-999/ARO-AAT) for liver disease associated with alpha-1 antitrypsin deficiency (AATD-LD). While treatment of 16 AATD patients with the RNAi therapy showed an impressive median reduction of 94% of liver Z-AAT expression, there was an unexpectedly high number of placebo responders on the fibrosis endpoint. In the study, seven (50%) of 14 treated patients showed an improvement of fibrosis of at least one point by METAVIR stage, while 38% (3/8 patients) showed fibrosis improvement on placebo. The partners said Takeda plans to start a Phase III study this month of fazirsiran in 160 AAT patients. Shares of Arrowhead were off $7.10 (19%) to $30.28 on Monday. 

With three years of follow up from the Phase III GENEr8-1 study, factor VIII levels in severe hemophilia A patients who received in vivo gene therapy valoctocogene roxaparvovec from BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) remained above the threshold for moderate disease, but activity continued to decline over time. BioMarin reported that the gene therapy led to median FVIII activity of 8.4 IU/dL in year three in the 134-patient study, and 7.4 IU/dL among the 17 patients with four years of follow-up, off from 11.8 IU/dL at two years. Though annualized bleeding rates requiring treatment remained relatively consistent at a mean of 1.0, annualized FVIII utilization more than doubled to 8.4 from two to three years in the study. Valoctocogene roxaparvovec has a March 31 PDUFA date, but the company previously said the Phase III data report requested by FDA may represent a major amendment that pushes the date back by three months...

Get Unlimited Access
Continue reading with a free trial.
Or Purchase This Article