Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR) Focus On subQ and Extra-Hepatic RNAi Therapeutics

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Arrowhead Pharmaceuticals Inc

Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR) reported a planned redeployment of resources to boost the advancement of RNAi therapeutics that use the firm’s new proprietary subQ and extra-hepatic delivery mechanisms. The company will discontinue advancement of clinical phase drug candidates ARC-521, ARC-AAT and ARC-520, which use the or EX1, or DPCiv™ delivery vehicle.

The highlights

Arrowhead remains dedicated to finding therapeutic alternatives for patients with alpha-1 antitrypsin deficiency and chronic HBV infection. The firm plans to progress to the clinic two initially unreported AATD and HBV programs utilizing subQ platform. Arrowhead has known a great deal during previous AAT and HBV trials that will help lead the subQ plans efficiently.

Existing preclinical extra-hepatic and subQ plans such as ARC-AMG1 and ARC-LPA, which are associated with ARC-F12, ARC-HIF2, Amgen and other unreported plans are not impacted by this choice. Because of the discontinuation of its current clinical plans, the firm in minimizing its workforce by nearly 30%, while maintaining full resourcing required to support potential and current future partner-based plans and Arrowhead’s growing pipeline.

This more planned structure should allow the firm to continue to advance its programs rapidly, and is planned to spread its cash runway into 2019. The choice to discontinue advancement of EX1-containing plans was based mainly on 2 factors. First, in current talks with outside experts and regulatory agencies, it became evident that there would be considerable delays in all clinical plans that use EX1, while the firm further explored the deaths causes in a non-clinical toxicology trial in non-human primates.

Next, Arrowhead has made considerable advances in RNA chemistry and aiming resulting in considerable potency gains for subQ given and extra-hepatic RNAi-based advancement programs. In preclinical trials with the subQ platform, the firm has obtained duration and depth of target gene knockdown coming that of intravenously given EX1-containing runners, at lower dosages and with good safety margins.

Arrowhead believes it is wise to focus its advancement resources entirely on its extra-hepatic and subQ pipeline, which comprises programs in Factor 12, HIF-2alpha, HBV, AAT and other unannounced plans. The company is also focused on offering full resources to advance its association with Amgen and potential future associations for its extra-hepatic delivery and subQ systems.

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