Arrowhead Pharmaceuticals Receives Fast Track Designation for ARO-AAT for Treatment of Alpha-1 Liver Disease
“With no currently approved agents to treat AATD-associated liver disease, alpha-1 patients and their physicians have an urgent need for new therapeutic options,” said
Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier.
A drug that receives fast track designation is eligible for some or all of the following:
More frequent meetings with
FDAto discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval
More frequent written communication from
FDAabout such things as the design of the proposed clinical trials and use of biomarkers
- Eligibility for accelerated approval and priority review, if relevant criteria are met
Rolling Review, enabling a drug company to submit completed sections of its New Drug Application (NDA) for review by
FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. NDA review usually does not begin until the drug company has submitted the entire application to the FDA.1
SEQUOIA (NCT03945292) is a placebo-controlled, adaptive design Phase 2/3 study to evaluate the safety, efficacy, and tolerability of ARO-AAT administered subcutaneously to 120 patients with AATD-associated liver disease. Doses will be administered on day 1, 29, and approximately every 12 weeks thereafter. The four-arm placebo-controlled Part A component of the study will feed seamlessly into a two-arm placebo-controlled Part B component. The primary objective for Part A is to select a single dose level for use in Part B of the study based on a combined evaluation of safety and pharmacodynamic dose response in each Part A cohort using change from baseline in soluble liver mutant AAT (Z-AAT), insoluble liver Z-AAT, and serum AAT levels as pharmacodynamic metrics. The primary objective for Part B is to evaluate efficacy, as assessed by the proportion of ARO-AAT treated patients relative to placebo achieving a 2-point improvement in a histologic grading scale of AATD-associated liver disease, and no worsening of liver fibrosis on end of study biopsy.
AROAAT2002 (NCT03946449) is a pilot open-label, multi-dose Phase 2 study to assess changes in a histologic grading scale in response to ARO-AAT administration in up to 12 patients with AATD-associated liver disease. Doses will be administered on day 1, 29, and approximately every 12 weeks thereafter. The histologic grading score will be assessed at weeks 24 and 72 for cohort 1 and weeks 48 and 96 for cohort 2. Multiple secondary and exploratory objectives will also be assessed throughout the study.
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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