Category: The Company

The Company Overview

Contacts

Riku Rautsola, PhD
President and Chief Executive Officer
[email protected]

Gerard Fleury,
EVP and CFO
[email protected]

Gary McGarrity, PhD
EVP Scientific and Clinical Affairs
[email protected]
Address
VIRxSYS Corporation
200 Perry Parkway, Suite 1A
Gaithersburg, MD 20877
United States

Telephone
301-987-0480

Fax
301-987-0489

Year Founded
1998

Financial Summary
Fundraising and Strategic Outlook
The Company has raised more than $80 million in six previous private equity rounds of financing since 1999, through Signature Capital Securities, LLP. The most recent round of fundraising will fund the Company through the completion of Phase II clinical trials for VRX496, a novel gene delivery therapy for HIV.

Company Profile
The Company, founded in Gaithersburg, Maryland, in 1998, is a private biotechnology company whose mission is to develop gene-based therapies using its proprietary lentiviral vector-based delivery system. The first application of the Company’s novel gene delivery technology has been for the treatment of HIV, the virus that causes AIDS. After a successful first-in-class Phase I clinical trial that demonstrated the safety profile of the Company’s product VRX496, this new ex-vivo anti-HIV treatment using a lentivector is currently being evaluated in the first and only Phase II clinical trial approved by the FDA involving a lentiviral vector. The Company’s lentiviral vector platform is an ideal delivery system for the effective treatment of disease. The Company’s vector technology is also being investigated for applications in anti-cancer therapies and for the treatment of other various life-threatening diseases.

Technology
The Company has developed a proprietary HIV-based lentiviral vector platform for delivery of genetic therapeutic payloads. Its first product, VRX496, for treatment of HIV, may offer the patients a long-term alternative to antiretroviral drug regimens. Traditional drug regimens are toxic and produce drug resistance strains of HIV. The Company believes that its therapy is non-toxic and solves the resistance issue thus enabling patients to live drug free. This has the potential to represent a significant improvement in the quality of life for people with HIV compared to that with the current antiretroviral drug treatments. The Company’s manufacturing capabilities are unparalleled in terms of large-scale clinical grade vector production and cell processing (including purification of vector, and transduction and expansion of patient cells). The Company has developed a stable cell line, which will make clinical grade vector production more compatible with commercialization. The Company has several therapies in preclinical development that apply the Company’s technology to the treatment of other diseases, such as cancer.

VIRxSYS has taken proof-of-concept studies performed at The Johns Hopkins University, completed its Phase I clinical trials in humans, and has initiated Phase II clinical trials in less than seven years. Company milestones are as follows:

Date	Milestone
1998	VIRxSYS incorporated
1999	First round of fundraising completed
2000	Initial meeting with the FDA to discuss the VIRxSYS lentiviral vector, VRX496
2001
January 2001	Second round of financing completed
August 2001	Pre-clinical studies completed toward an Investigational New Drug (IND) application
September 2001	VIRxSYS Phase I protocol for VRX496 presented to the NIH Recombinant Advisory Committee (RAC) for public review
October 2001	VRX496 presented to the FDA Biological Response Modifier Advisory Committee (BRMAC) for formal protocol review
2002
April 2002	Third round of financing completed
August 2002	IND application submitted to the FDA for final review of VIRxSYS’s proposed Phase I clinical trial for VRX496
December 2002	VIRxSYS granted permission by the FDA to proceed with the Phase I clinical trial for VRX496 modified CD4 T cells as an anti-HIV therapy; this clinical trial is a first-in-class use of lentiviral vectors in humans for any indication
2003
January 2003	Final approval received from The Institutional Review Board (IRB) at the University of Pennsylvania for Phase I clinical trial to be held at that institution
July 2003	First patient in the Phase I clinical trial dosed with VRX496 modified CD4 T cells
2004
April 2004	VIRxSYS awarded $1.5 million by the NIH for construction of a cell processing center and completion of important safety studies for VRX496 insertion into CD4 T cells
July 2004	Fourth round of financing completed
July 2004	“Fast Track” status granted to VIRxSYS by the FDA for VRX496
September 2004	State-of-the-art cell processing center completed in Gaithersburg, MD
September 2004	Final patient of the Phase I trial dosed
2005
May 2005	All patients in the Phase I trial reached nine-month post infusion, the official end of the trial; participants continue to be monitored
September 2005	Phase II Trial to establish safety and tolerability of multiple infusions of VRX496 commenced
October 2005	Fifth round of financing completed
2006
July 2006	Multiple infusions of VRX496 completed for all patients receiving multiple doses in Phase II trial, demonstrating the safety and tolerability of multiple doses of VRX496
July 2006	Patient from Phase I trial shows safety of treatment and persistence of vector at three years post infusion
September 2006	Phase I/II Clinical trial commenced at the University of Pennsylvania to test the safety, tolerability, and efficacy of VRX496 in HIV-positive patients who are well-controlled by traditional drug regimens
September 2006	Phase I/II clinical trial approved for the  treatment of HIV positive patients who are not on drug therapy, to be conducted at Harvard University, The University of Pennsylvania, and The University of Amsterdam;  AIDS Clinical Trial Group funds received for clinical trial sites in the United States

VIRxSYS has raised over $80 million through six rounds of financing thanks to the support of our investors.  Without their continued support and enthusiasm, the Company would not be able to develop technology that delivers on the promise of genetic medicine, allowing for the treatment of serious human diseases.

Technology Advancement
VIRxSYS intends to develop technology internally, as well as acquire technology which fit an SSD strategy:

• Strategic – must help us drive toward our mission
• Synergistic – must take advantage of our core competency in lentiviral vectors
• Disruptive – must provide a significant and unique therapeutic advantage

Business Model
VIRxSYS’s strategy is to develop gene therapy treatments for serious diseases and advance each of those therapies into the market along one of three avenues:

1. Exclusive licensing to, or partnering with, a major bio-pharmaceutical company
2. Non-exclusive licensing to a bio-pharmaceutical company, retaining co-promotion or co-marketing rights for the United States
3. Go-it-alone strategy whereby VIRxSYS will take the lead in sales, marketing and distribution

The “go to market” track will depend on a number of factors including, but not limited to:

• Size of the market
• Capital requirements to take to market
• Complexity of the manufacturing and distribution channels
• Strength of potential partners

Each disease indication will be evaluated to determine the most appropriate track.

Initial Market Opportunity
Conventional drug therapies for HIV-positive people in the industrialized world are estimated to be more than $6 billion.  When ancillary medical costs are factored in, the cost of treating HIV/AIDS more than doubles to $13 billion each year.  Despite the enormous cost of these treatments, toxicities inherent in the current antiretroviral drugs available make them undesirable for long-term use.  Genetic medicine holds promise for well-tolerated, effective therapies to combat HIV.

In addition to HIV/AIDS, VIRxSYS is researching similar applications of the Company’s proprietary technology toward other life-threatening diseases such as cancer.  With the National Institutes of Health estimating the cost of cancer to be close to $190 billion each year in direct and indirect medical costs and lost productivity, there is a clear need for innovative solutions.

These examples represent a significant opportunity to deliver on the promise of genetic medicine for the millions of people suffering from HIV/AIDS and cancer, and with the technology being researched at VIRxSYS, the Company is poised to do just that.

If you are interested in finding out more information about VIRxSYS, contact: [email protected]

Medical Advisory Board
VIRxSYS has assembled a Medical Advisory Board comprised of experts in HIV medicine.  The Medical Advisory Board offers guidance in the design and execution of the Company’s HIV clinical trials, and guidance in positioning VRX496.  The members of the Medical Advisory Board include:

Member	Affiliation
Brigitte Autran, MD, PhD	Groupe Hospitalier Pitie-Salpetriere Paris, France
Steven Deeks, MD	University of California at San Francisco San Francisco, California
Carl June, MD	University of Pennsylvania Philadelphia, Pennsylvania
Christine Katlama, MD	Groupe Hospitalier Pitie-Salpetriere Paris, France
Daniel Kuritzkes, MD	Harvard University Cambridge, Massachusetts
Joep Lange, MD	Academic Medical Centre Amsterdam, The Netherlands
Julio Montaner, MD	British Columbia Centre for Excellence in HIV/AIDS Vancouver, British Columbia
Robert Murphy, MD	Northwestern University Evanston, Illinois
Richard Pollard, MD	University of California at Davis Sacramento, California
John Sullivan, MD	University of Massachusetts Medical Center Worcester, Massachusetts
Andrew Zolopa, MD	Stanford University Stanford, California