Arrowhead Pharmaceuticals Presents Late-Breaking Clinical Data on ARO-AAT at Liver Meeting® 2018
- Three monthly doses of 300 mg ARO-AAT led to reductions in serum alpha-1 antitrypsin to below the level of quantitation in 100% of subjects
- Reductions were sustained for greater than 14 weeks indicating that quarterly or less frequent dosing appears feasible
- Single and multiple doses of ARO-AAT appear to be well-tolerated at all doses tested
In the AROAAT1001 study, 45 normal healthy volunteers (NHV) received a single dose of ARO-AAT (n=16), three monthly doses of ARO-AAT (n=12), or placebo (n=17). Key data presented include the following:
ARO-AAT at single- and multiple-doses produced robust and consistent
reductions in serum AAT levels
- Single-doses of 200 and 300 mg resulted in greater than 91% serum AAT reduction, with 3 of 4 subjects having concentrations below the level of quantitation (BLQ)
- In 200 and 300 mg single-dose cohorts, an average serum AAT reduction of greater than 90% was sustained for 6 weeks
- In the multiple-dose cohorts of 200 and 300 mg, for subjects receiving all 3 doses, an average of greater than 90% reduction in serum AAT was sustained for longer than 14 weeks
- The maximum NADIR reduction is 94%
- Monthly serum AAT follow up is ongoing with 9 of 10 subjects at BLQ in the multiple-dose cohorts, including 100% of subjects from the 300 mg cohort
- Duration of response indicates that quarterly or less frequent dosing appears feasible
ARO-AAT has been well tolerated at all doses tested (up to 300 mg)
given three times every 28 days
- The most common adverse events (AE) were upper respiratory tract infection (39%) and headache (32%)
“The ARO-AAT data being presented at The Liver Meeting demonstrate the
power of our Targeted RNAi Molecule (TRiMTM) platform, which
is the basis of our growing pipeline of RNAi therapeutics targeting a
diverse range of diseases,” said
A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the
Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum
Alpha-1 Antitrypsin levels in
- Publication Number: LB-9
- Session: Late-Breaking Poster Session
Session Date and Time:
November 12, 2018from 8:00 a.m. to 5:30 p.m. PT
Moscone Center North/South Building, Hall C
Christian Schwabe, et al.
A copy of the poster may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
ARO-AAT is designed to silence the production of the misfolded Z-AAT protein with the intent to:
- Prevent accumulation of the disease-causing protein in the liver
- Allow for clearance of the accumulated protein
- Prevent repeated cycles of cellular damage
- Reverse fibrosis associated with prior damage
AROAAT1001 (NCT03362242) is a Phase 1 randomized, double-blind, placebo controlled single-ascending dose (SAD) and multiple-ascending dose (MAD) study to evaluate the safety, tolerability, pharmacokinetics, and effect of subcutaneous doses of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers. The SAD portion of the study included four cohorts at dose levels of 35, 100, 200, and 300 mg and the MAD portion of the study included three cohorts at dose levels of 100, 200, and 300 mg. Additional cohorts were planned at a dose of 400 mg, but were deemed unnecessary based on observed activity at lower doses. AROAAT1001 enrolled 45 healthy volunteers.
For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.
Safe Harbor Statement under the Private Securities Litigation Reform Act:
This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the safety and efficacy of our product candidates, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, and the enforcement of our intellectual property rights. Our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
Investors and Media:
LifeSci Advisors, LLC