Profile: Novavax Inc (NVAX.O)

Novavax, Inc. (Novavax), incorporated in 1987, is a clinical-stage biopharmaceutical company focused on creating differentiated, value-added vaccines that improve upon preventive options for a range of infectious diseases. These vaccines leverage its virus-like particle (VLP) platform technology coupled with a disposable production technology. VLPs are genetically engineered three-dimensional nanostructures, which incorporate immunologically important lipids and recombinant proteins. Novavax’s VLPs resemble the virus but lack the genetic material to replicate the virus. Its production technology uses insect cells rather than chicken eggs or mammalian cells. The Company’s product targets include vaccines against the H5N1 and other subtypes of avian influenza with pandemic potential, human seasonal influenza, Varicella Zoster, which causes shingles, and respiratory syncytial virus (RSV). This RSV vaccine was announced on October 30, 2008.

Research and Development Technology and Activities

Novavax develops and produces biopharmaceutical proteins for use as vaccines against pandemic and seasonal influenza and other infectious diseases, and as tolerogens to prevent inflammatory responses in the initiation and progression of stroke and other illnesses. The Company's lead vaccine technology platform is based on VLPs, which are self-assembling protein structures that resemble viruses. These are noninfectious particles that, for many viral diseases, have been shown in animal and human studies to make effective vaccines. VLPs closely mimic natural virus particles with repeating protein structures that can elicit antibody and cellular immune responses, but lack the genetic material required for replication. Novavax has several ongoing development programs involving VLP vaccines that address urgent medical needs, including pandemic and seasonal influenza, Varicella Zoster Virus, RSV and other infectious diseases.

Proof-of-concept of the VLP approach in H5N1 pandemic influenza has been demonstrated by the Phase I/IIa human clinical trial interim results released by the Company in December 2007. Novavax reported that its VLP vaccine for H5N1 influenza is immunogenic, that elicited immune responses at both 15 and 45 mcg doses. The Company began subject enrollment in the second portion of the Phase I/IIa trial in March 2008, to gather additional subject immunogenicity and safety data and determine a final dose through the completion of this clinical trial. In August 2008, it reported favorable results from this clinical trial, which demonstrated strong neutralizing antibody titers across all three doses tested. In February 2009, the Company announced that the vaccine induced robust hemagglutination inhibition (HAI) responses, which have been shown to be important for protection against influenza disease.

In April 2008, Novavax announced that it received positive results from an immunogenicity study in ferrets inoculated with its trivalent seasonal influenza vaccine candidate. In September 2008, the Company began Phase II clinical trials to evaluate the safety and immunogenicity of different doses of its seasonal influenza vaccine. In November 2008, it announced a delay of its seasonal influenza dose ranging study in the elderly (>65 years of age), pending top line safety and immunogenicity results from its ongoing seasonal influenza study in healthy adults. Novavax had observed a slightly different safety profile (non-serious adverse events) from its Phase IIa trial of its pandemic VLP vaccine, and decided to review and analyze the dose response curve, as well as the safety data from the healthy adult seasonal trial prior to commencing a study in the elderly. In December 2008, Novavax announced favorable safety and immunogenicity results from its Phase IIa seasonal study in healthy adults.

In September 2007, the Company announced a new discovery-phase product indication target for the prevention of a disease associated with Varicella Zoster virus in older patients, commonly referred to as Shingles. Shingles, a skin rash often with painful blisters, is caused by the same virus that causes chickenpox.

On December 9, 2008, Novavax and the University of Massachusetts Medical School announced results from a preclinical study of an RSV vaccine candidate. Novavax has licensed exclusive worldwide rights from the University of Massachusetts Medical School to certain technology for the development and commercialization of Paramyxovirus vaccines incorporating certain VLPs. This vaccine candidate is the Company’s first recombinant VLP for the prevention of RSV disease. The preclinical study evaluated the immunogenicity and efficacy of the RSV VLP vaccine candidate in mice. The RSV VLP vaccine induced antibody responses against RSV, protected mice against RSV replication in the lungs, and did not lead to enhanced inflammation of the airways.

Other VLP Projects

The Company is working on certain other vaccine projects with sponsoring organizations. These projects are funded and controlled by other parties. As is typical with these research contracts, Novavax does not have commercial rights to these products, as of December 31, 2008.

Under a five-year National Institutes of Health (NIH) grant that was awarded in 2003, Novavax is working in collaboration with the University of Alabama - Birmingham, Emory University and Harvard Medical School in the development of a second-generation acquired immunodeficiency syndrome (AIDS) vaccine. In January 2007, the Company announced that it has enhanced both the quality and purity of its VLP vaccine for human immunodeficiency virus (HIV)/AIDS. This second generation AIDS vaccine is based on the HIV-1 viral envelope with a natural three-dimensional structure to trigger a protective immune response. Preclinical studies are ongoing using the improved HIV-1 vaccine, and planning has begun to advance this new vaccine to human clinical trials in collaboration with the United States government potentially as early as 2009.

In collaboration with the National Institute of Neurological Disorders and Stroke (NINDS), Novavax has been developing E-selectin-based, molecularly-derived products for the prevention of strokes. In September 2002, a published report provided experimental evidence on prevention of stroke in stroke-prone rats. These results provided supportive evidence that E-selectin tolerization may help in the prevention of strokes and other illness where inflammatory and immune responses are involved in the initiation and progression of disease. The Company was awarded a government contract for the formulation development and manufacture of E-selectin for Phase I clinical trials to be run by the NINDS and the NIH.

The Company competes with sanofi pasteur, Inc., Astra-Zeneca, Inc., GlaxoSmithKline Biologicals, Novartis, Inc., and Merck & Co.