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Calando, UCLA and City of Hope Plans to Conduct First Cancer Clinical Trial for RNAi Therapeutic

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Calando Pharmaceuticals, a majority-owned subsidiary of Arrowhead Research Corporation, together with City of Hope (COH) and the UCLA Jonsson Cancer Center (UCLA), has announced that Calando, UCLA and COH are planning a broad-based Phase I clinical trial to begin later this year for Calando’s lead drug candidate, CALAA-01, a nanoparticle containing non-chemically modified siRNA and a transferrin protein targeting agent formulated with Calando’s RONDEL™ (RNA/Oligonucleotide Nanoparticle Delivery) technology.

Calando has commenced IND-enabling toxicity studies in rats and monkeys and is actively engaged in the scale-up of therapeutic manufacturing to produce cGMP-grade clinical materials for the Phase I trial.

Calando will be responsible for filing an Investigational New Drug application with the US Food and Drug Administration, and upon regulatory approval, Calando, COH and UCLA plan to conduct an open-label, dose-escalation Phase I clinical trial in patients with unresectable or metastatic solid tumors.

The study will be led by Yun Yen, MD, Ph.D., at COH and Antoni Ribas, MD, at UCLA. Based upon the evaluation of the results from this study, Calando plans to conduct separate, disease-specific Phase II trials.

“This will be the first targeted, formulated siRNA therapeutic to be used in humans,” said John Petrovich, Calando’s CEO.

“Others are taking a non-targeted approach to cancer, but based upon our work and those that are published in the open literature, we believe that some form of targeting to cancer cells that provides for efficient intracellular uptake is essential for good efficacy. We at Calando are excited to be working with two world-class medical centers on this first-of-its-kind clinical trial.”

The formulation to be investigated contains Calando’s proprietary delivery technology and utilizes RNA interference in cancer cells with an siRNA duplex targeting the M2 subunit of ribonucleotide reductase. This duplex, developed at Calando, demonstrates potent anti-proliferative activity across multiple types of cancer both in vitro and in vivo.

The nanoparticle formulation has human transferrin protein (Tf) targeting agents decorating the surface. The transferrin receptor has long been known to be up-regulated in a broad spectrum of cancer cell types, and Tf binding to this receptor triggers the uptake of nanoparticles by cancer cells and release of the siRNA payload.