Arrowhead Research Corporation (NasdaqGS: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it recently filed an application for approval to begin a Phase 1 clinical trial of its RNAi-based therapeutic candidate, ARC-AAT, for the treatment of liver disease associated with alpha-1 antitrypsin deficiency.

Pending approval, Arrowhead intends to proceed with a double blind, placebo-controlled dose escalation, Phase 1 study to determine the safety, tolerability, pharmacokinetics and effect on circulating alpha-1 antitrypsin levels of ARC-AAT in healthy volunteers and patients with alpha-1 antitrypsin deficiency. The study is planned to start in Australia in healthy volunteers and will dose escalate until predetermined levels of alpha-1 antitrypsin reduction are reached. Once these levels of protein knockdown are achieved, the study will transition into a population of patients with ZZ genotype alpha-1 antitrypsin deficiency to further evaluate escalating doses of ARC-AAT. The study is designed to enroll up to 48 subjects, including healthy volunteers and alpha-1 antitrypsin deficiency patients.

The application was submitted to an ethics committee in compliance with the Clinical Trial Notification process of the Australian Department of Health and Ageing, Therapeutic Goods Administration. 

TapImmune, Inc. (OTCQB: TPIV), is pleased to announce a new collaborative research agreement with The Vaccine & Gene Therapy Institute of Florida (VGTI Florida), a leading, non-profit biomedical research institute, forming a partnership to advance TapImmune's proprietary,  cancer vaccines into  Phase II human clinical trials for the treatment of breast and ovarian cancers. 

These highly promising therapeutic cancer vaccine candidates were developed by VGTI Florida's Director of Cancer Vaccines and Immune Therapies Program, Keith Knutson, Ph.D.  The goal is to prevent breast and ovarian cancer recurrence for patients who achieve remissions following standard-of-care treatment.  The vaccine works by enabling the immune system to target and eradicate any residual cancer cells or newly arising cancer cells that express the antigens delivered by the vaccine.   TapImmune has already announced interim data on both clinical programs that exceeded its expectations for immune responses in the treated patients.

VGTI Florida will work with TapImmune to design and execute the Phase II clinical programs.  This includes the design of the clinical protocols, selection of clinical trial sites, recruitment of key opinion leaders as clinical advisors, and selection of external manufacturing and clinical resources.

TapImmune had previously announced the licensing of these vaccines technologies for the treatment of HER2/neu breast cancer and ovarian and breast cancer developed in the laboratory of Dr. Keith Knutson while at the Mayo Clinic.

TapImmune's CEO, Glynn Wilson commented: "We are delighted to establish this collaboration with the Center for the Diseases of Aging at VGTI Florida to expand our relationship with Dr. Keith Knutson and leverage their unique experience in breast and ovarian cancer vaccine development and clinical trial design as we move our clinical programs into Phase II trials."