Arrowhead Presents New Clinical Data on ARO-AAT at Alpha-1 National Education Conference
Key new clinical data to be presented include the following:
- In the AROAAT1001 Phase 1 clinical study, a single, open-label dose of 100 mg of ARO-AAT in four subjects achieved 93% maximum serum AAT knockdown and 87% mean maximum serum AAT knockdown. At 8 weeks post-dose, mean serum AAT knockdown remained at 83%.
- The single 100 mg dose of ARO-AAT equates to an average dose of 1.4 mg/kg (range 1.0-1.6 mg/kg) in the subjects studied, who had an average weight of 72.9 kg (range 61.8-98.9 kg)
ARO-AAT appeared to be generally well-tolerated and as of the data
June 11, 2018, the following safety measures were observed in 40 subjects (24 received ARO-
AAT and 16 received placebo):
- No serious or severe adverse events (AEs)
- Most AEs reported were mild (one moderate gastroenteritis)
2 cases of injection site erythema at 100 mg after 1st dose
- Both were classified as mild and resolved within 48 hours
- No clinically meaningful adverse changes in BUN, creatinine, ALT, AST or total bilirubin
- No dose-related pattern of adverse laboratory changes seen
Preclinical data previously presented showed:
- Treatment with ARO-AAT for 8 weeks led to reductions in Z-AAT monomer and polymer content, and prevention of globule accumulation in the livers of young PiZ mice
- Two doses of 3 mg/kg of ARO-AAT led to 92% maximum serum AAT knockdown that was sustained for over 7 weeks following the second dose in nonhuman primates
A copy of the presentation slides can be accessed now on the Events and Presentations page under the Investors section of the Arrowhead website.
Arrowhead’s presentation is part of a session titled, “Driving New
Therapies and Tools in Alpha-1 – Presentation & Panel Discussion,”
AROAAT1001 (NCT03362242) is a Phase 1 single- and multiple-ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers. The study includes 7 cohorts in which 16 subjects receive placebo and 28 subjects receive single or multiple doses of ARO-AAT at doses of 35, 100, 200, or 300 mg. Additional cohorts were planned at a dose of 400 mg, but were deemed unnecessary based on the observed activity at lower doses.
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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 future success of our scientific studies, our ability to successfully develop 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