About Arrowhead Research

Overview

Arrowhead Research Corporation is a clinical stage nanomedicine company developing innovative therapies at the interface of biology and nanoengineering. The Company has clinical programs in small RNA therapeutics and obesity. Arrowhead's programs are based on its proprietary drug development and delivery platforms, each capable of creating multiple therapeutic products. This strategy seeks to enhance the return potential for investors and reduce risk compared to traditional biotech companies. Arrowhead's world-class capabilities and intellectual property covering nucleic acid delivery, siRNA chemistry, and tissue targeting allow it to optimize therapeutic agents for a wide range of diseases.

The company's lead products include:

  • CALAA-01, an oncology drug candidate based on the gene silencing RNA interference (RNAi) mechanism
  • Adipotide, and anti-obesity peptide that targets and kills the blood vessels that feed white adipose tissue
  • RNAi development programs for hepatitis B virus and targeting HIF-2a for solid tumors

Arrowhead is leveraging its proprietary Dynamic Polyconjugate (DPC), Liposomal Nanoparticle (LNP), and RONDEL delivery platforms to support its own pipeline of preclinical and clinical candidates, and to secure external partnerships and collaborations with biotech and pharmaceutical companies. Arrowhead believes this comprehensive suite of delivery solutions, with its broad license to the three primary siRNA structures, positions the company as the partner of choice for small RNA therapeutics.

Arrowhead's current portfolio includes wholly-owned Arrowhead Madison, an innovator in delivery and development of RNA therapeutics acquired from Roche in 2011, majority holdings in Calando Pharmaceuticals, a leader in delivering small RNAs for gene silencing, and Ablaris Therapeutics, an anti-obesity therapeutics company. The Company also holds minority interests in Nanotope, a regenerative medicine company, and Leonardo Biosciences, a next generation drug delivery company initially targeting treatment for cancer.

Arrowhead is headquartered in Pasadena, CA with research facilities in Madison, WI.



Wholly and Majority-Owned Subsidiaries

Arrowhead Madison, Inc.

Arrowhead Madison was acquired in 2011 from Roche and includes a purpose-built Madison, Wisconsin research facility, world-class scientists, and proprietary RNAi technologies that form the core of our integrated discovery and development efforts. Formerly Mirus Bio and subsequently acquired by Roche in 2009 for $125 million, our Madison scientists are key inventors of an RNAi delivery system called Dynamic Polyconjugates (DPCs) which are well tolerated and have demonstrated unprecendented single-dose therapeutic indices of >10 in non-human primates (indicating that there is a ten-fold safety margin between the effective dose and the toxic dose). Arrowhead Madison is a wholly owned subsidiary.

Calando Pharmaceuticals, Inc. is a clinical stage company focused on nano-enabled drug discovery, development, and delivery. Based on Calando's RONDEL delivery system, Calando's siRNA therapeutic, currently undergoing Phase I clincial testing, is the first to be used to deliver siRNA systemically in humans. RNAi (through siRNA) is a potentially revolutionary new way of treating diverse disease classes; however, the lack of a proven method of delivering siRNA in humans has held the new field back. Calando is a majority-owned subsidiary of Arrowhead.

Ablaris Therapeutics, Inc. is commercializing a new pharmaceutical approach to fighting obesity — a widespread and growing problem worldwide. Based on technology developed at the University of Texas MD Anderson Cancer Center, the Ablaris platform consists of a suite of proprietary peptides that specifically target and kill the blood vessels that feed white fat tissue. Deprived of nutrition, the fat cells die and are broken down by the body. Independent studies across multiple species, including non-human primates, have demonstrated substantial and rapid weight loss in overweight animals. This is accomplished directly by reducing fat stores and indirectly by reducing appetite. Ablaris' first drug candidate has been cleared by the FDA to enter the clinic this year. Ablaris is a majority-owned subsidiary of Arrowhead.



Minority Investments

Arrowhead has minority investments in two early-stage nanomedicine companies, Nanotope, Inc. and Leonardo Biosystems, Inc. Along with our ownership interests, we provide financial, administrative, corporate and strategic resources.

Nanotope is a regenerative medicine company developing a highly flexible and customizable platform out of Northwestern University. Nanotope is developing a suite of products, each customized to regenerate specific tissues; including neuronal, vascular, bone, myocardial, and cartilage. The products are injectable compounds that work with surviving cells in and around the point of damage to initiate and support tissue regeneration and growth. Once regeneration is complete, the compounds are safely broken down and removed by the body. Nanotope entered into its first commercialization corporate partnership in 2010.

Leonardo Biosystems has a multi-stage delivery platform that has been shown in animal models to be highly effective in targeting delivery of siRNA and small molecule drugs.