Vical's TransVax(TM) CMV Vaccine Achieves Statistical Significance on Key Clinical Endpoints in Phase 2 Trial
Conference Call and Webcast Concurrent With ICAAC Presentation at 11:15 a.m. EDT Tuesday
SAN DIEGO, Sep 13, 2010 (GlobeNewswire via COMTEX) -- Vical Incorporated announced today the achievement of key efficacy, immunogenicity and safety results in a Phase 2 trial, establishing its TransVax(TM) cytomegalovirus (CMV) vaccine as the first to provide evidence of protection in immunocompromised hematopoietic cell transplant (HCT) recipients, and defining a potential pathway for further development.
"The TransVax(TM) vaccine has demonstrated the ability to drive effective immune responses in the inherently difficult therapeutic setting of immunocompromised patients," said CMV and organ transplant expert Mark D. Pescovitz, M.D. "In addition to providing evidence of protective efficacy in HCT recipients, the TransVax(TM) Phase 2 results have identified clinically relevant endpoints that may be appropriate for further development."
"We look forward to finalizing our approach through discussions with CMV and transplant experts, and reviewing our resulting plans with the regulatory agencies," said Richard T. Kenney, M.D., Vical's Vice President of Clinical Development, who will present the data at 11:15 a.m. EDT Tuesday at the 50th Annual Interscience Conference on Antimicrobial Agents and Chemotherapy (Boston -- September 12-15). Dr. Pescovitz, Professor of Surgery and Microbiology & Immunology, and Vice Chair of Research in Surgery at the Indiana University School of Medicine, will join Vical management on a concurrent conference call and webcast for the investment community.
The TransVax(TM) Phase 2 trial was a 1:1 randomized, double-blind, placebo-controlled, multi-center study in 80 patients scheduled to receive HCTs for their underlying leukemias or lymphomas. Through 12 months post-transplant, significant reductions were achieved for key viral reactivation metrics:
-- The incidence of CMV viremia (percentage of subjects with greater then
or equal500 copies of CMV virus per mL of blood by a central lab assay)
was 32% in the TransVax(TM) group, and 62% in the placebo group
-- The median time to initial viremia was >365 days (i.e., median not
reached during the 12-month study period) for the TransVax(TM) group,
and 109.5 days for the placebo group (p=0.003);
-- The number of CMV viremia episodes (0-4 per subject) was significantly
lower for the TransVax(TM) group than for the placebo group (p=0.017);
-- The duration of viremia was lower for the TransVax(TM) group than for
the placebo group (mean of 10.6 days vs. mean of 19.5 days,
respectively; p=0.069), and significantly lower as a percentage of the
time subjects spent on study (mean of 4.9% vs. mean of 7.7%,
respectively; p=0.042); and
-- The prevalence of CMV viremia episodes over the trial period was
significantly lower for the TransVax(TM) group than for the placebo
Antiviral therapy use was triggered by site-specific treatment practices based on local lab assays, which impeded comparability among the 16 enrolling U.S. sites. Overall, 48% of subjects in the TransVax(TM) group vs. 62% of subjects in the placebo group received antiviral drugs for CMV during the study. The median duration of antiviral treatment was approximately 30 days in the TransVax(TM) group vs. approximately 40 days in the placebo group. Because antiviral drugs are used preemptively to control CMV outbreaks, CMV-associated disease is rare and the study was not powered to detect a difference in this endpoint. Only three of 40 subjects (8%) in the TransVax(TM) group and four of 34 subjects (12%) in the placebo group developed CMV-associated disease. Antiviral drug usage and CMV-associated disease are not practical endpoints for future studies.
Published data have shown that T-cell responses are critical for controlling CMV reactivation; antibody responses may provide additional protection. The HCT recipients in the study all had preexisting CMV infections and therefore would be expected to have baseline immune responses against CMV. They were also immunosuppressed as a result of treatments for their underlying leukemias or lymphomas. Despite these challenges:
-- T-cell responses to phosphoprotein 65 (pp65) were significantly higher
in the TransVax(TM) group than in the placebo group over the one-year
trial (p=0.003); and
-- T-cell and antibody responses to glycoprotein B (gB) were higher in the
TransVax(TM) group than in the placebo group and trending toward greater
separation over the one-year trial.
The TransVax(TM) vaccine was well tolerated in the Phase 2 trial in immunocompromised HCT recipients, and no safety concerns were raised during the trial. There was no significant difference in the number of serious adverse events (SAEs) between the TransVax(TM) and placebo groups. There also were fewer deaths in the TransVax(TM) group (18%) than in the placebo group (32%) during the trial.
Phase 2 Trial Design
The Phase 2 trial evaluated the potential for TransVax(TM) to control CMV reactivation in immunosuppressed CMV-seropositive HCT recipients, which could reduce antiviral usage and CMV-associated disease. Subjects were randomized by site, transplant donor CMV-seropositive or -seronegative status, and donor/recipient genetic match (matching either 5 of 6 or 6 of 6 human leukocyte antigen alleles) and received either the TransVax(TM) vaccine or placebo shortly before the transplant and again at approximately 1, 3 and 6 months post-transplant. The study was conducted at 16 U.S. sites, and the endpoints evaluated for 12 months post-transplant included safety, immunogenicity, use of CMV antiviral therapy, and viral load.
TransVax(TM) is a bivalent DNA vaccine containing plasmids (closed loops of DNA) encoding CMV pp65 and gB antigens for induction of both cellular and humoral immune responses. TransVax(TM) is formulated with a proprietary poloxamer-based delivery system. TransVax(TM) has received orphan drug designation in the United States for HCT and solid organ transplant patients.
Vical will conduct an audio-only conference call and audio/slide webcast at 11:15 a.m. EDT Tuesday to discuss details of the results with invited analysts and institutional investors. The call is open on a listen-only basis to any interested parties. The webcast audio and slides also will be available live and archived through the Events page of the company's website at www.vical.com. To listen to the conference call, dial in approximately ten minutes before the scheduled call to (888) 205-6648, or (913) 981-5543 for international participants, and reference confirmation code 4384312. A replay of the call will be available for 48 hours beginning about two hours after the call. To listen to the replay, dial (888) 203-1112, or (719) 457-0820 for international participants, and enter replay passcode 4384312. For further information, contact Vical's Investor Relations department by phone at (858) 646-1127 or by e-mail at [email protected]
Vical researches and develops biopharmaceutical products based on its patented DNA delivery technologies for the prevention and treatment of serious or life-threatening diseases. Potential applications of the company's DNA delivery technology include DNA vaccines for infectious diseases or cancer, in which the expressed protein is an immunogen; cancer immunotherapeutics, in which the expressed protein is an immune system stimulant; and cardiovascular therapies, in which the expressed protein is an angiogenic growth factor. The company is developing certain infectious disease vaccines and cancer therapeutics internally. In addition, the company collaborates with major pharmaceutical companies and biotechnology companies that give it access to complementary technologies or greater resources. These strategic partnerships provide the company with mutually beneficial opportunities to expand its product pipeline and address significant unmet medical needs. Additional information on Vical is available at www.vical.com.
The Vical Incorporated logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=5768
This press release contains forward-looking statements subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements about Vical's technologies, the TransVax(TM) vaccine effectiveness in reducing key CMV reactivation metrics, as well as the company's focus, collaborative partners, and independent and partnered product candidates. Risks and uncertainties include whether Vical or others will continue development of TransVax(TM) or any other product candidates; whether endpoints identified in or as a result of the Phase 2 trial will be acceptable to the relevant regulatory agencies; whether Phase 2 results will be predictive of results in any future studies; whether Vical or others will advance TransVax(TM) to Phase 3 testing; whether such testing, if conducted, will be successful; whether Vical or its collaborative partners will seek or gain approval to market TransVax(TM) or any other DNA-based human vaccine or therapeutic product candidates; whether Vical or its collaborative partners will succeed in marketing any product candidates; and additional risks set forth in the company's filings with the Securities and Exchange Commission. These forward-looking statements represent the company's judgment as of the date of this release. The company disclaims, however, any intent or obligation to update these forward-looking statements.
RTI Biologics and Athersys Announce Collaboration in the Orthopedic Market
Applying Complementary Stem Cell and Regenerative Medicine Technologies
RTI Biologics and Athersys Announce Collaboration in the Orthopedic Market
Applying Complementary Stem Cell and Regenerative Medicine Technologies
ALACHUA, Fla. & CLEVELAND, Sep 13, 2010 (BUSINESS WIRE) -- RTI Biologics Inc. (RTI) /quotes/comstock/15*!rtix/quotes/nls/rtix (RTIX 2.25, +0.05, +2.27%) , a leading provider of orthopedic and other biologic implants, and Athersys Inc. (Athersys) /quotes/comstock/15*!athx/quotes/nls/athx (ATHX 2.92, +0.07, +2.40%) , a leader in regenerative medicine and cell therapy research and development, announced today an agreement under which Athersys will provide RTI access to its Multipotent Adult Progenitor Cell (MAPC) technologies.
Under the agreement, RTI has licensed Athersys' technology to isolate and preserve cells from organ and tissue donors. This will enable RTI to develop and commercialize MAPC technology-based biologic implants exclusively for certain orthopedic applications. With this license, RTI expands its capabilities for accessing the fastest growing segment of the bone graft substitutes market, while Athersys extends the application of its robust stem cell technology platform to an important segment of the orthopedic market.
"We are very excited about our collaboration with Athersys and the potential to apply its MAPC and related technologies in the orthobiologics market," said Brian K. Hutchison, RTI's chairman and CEO. "After significant research into stem cells and the evaluation of multiple technologies, we have determined that the MAPC technology offers the greatest potential to create high quality, innovative implants for our surgeons and their patients. Licensing this technology is an important step in enhancing and further differentiating RTI's orthobiologics offering, an area of strategic focus for the company."
"We are enthusiastic about our relationship with RTI and this application of our proprietary stem cell technologies to the orthopedic market," said William Lehmann, Jr., president and COO of Athersys. "Our primary focus is the development and commercialization of expanded, 'off-the-shelf' cell products, such as MultiStem(R), for the treatment of certain cardiovascular, central nervous system-related, inflammatory and immune system disorders, diseases and conditions. This collaboration allows for the utilization of our already developed stem cell technologies in an additional area. Further, it provides potential near term revenues from the orthopedic market."
Under the agreement, Athersys will receive a $3 million license fee and is also eligible to receive payments up to a cumulative total of $37.5 million as follows: $2 million contingent upon successful achievement of certain development and commercialization milestones, and an additional maximum total of $35.5 million contingent upon achievement of certain cumulative revenue milestones, which will reflect the ultimate commercial success of the product in this fast growing market. In addition, Athersys will receive tiered royalties from the distribution of implants using Athersys' technologies.
RTI plans to make MAPC technology-based biologic implants available to its customers for use in orthopedic surgeries in the first half of 2012.
About RTI Biologics Inc.
RTI Biologics Inc. is a leading provider of sterile biological implants for surgeries around the world with a commitment to advancing science, safety and innovation. RTI prepares human donated tissue and bovine tissue for transplantation through extensive testing and screening, precision shaping and using proprietary, validated processes. These allograft and xenograft implants are used in orthopedic, dental, hernia and other specialty surgeries.
RTI's innovations continuously raise the bar of science and safety for biologics -- from being the first company to offer precision-tooled bone implants and assembled technology to maximize each gift of donation, to inventing validated sterilization processes that include viral inactivation steps. Two such processes -- the BioCleanse(R) Tissue Sterilization Process and the Tutoplast(R) Tissue Sterilization Process -- have a combined record of more than two million implants distributed with zero incidence of allograft-associated infection. These processes have been validated by tissue type to inactivate or remove viruses, bacteria, fungi and spores from the tissue while maintaining biocompatibility and functionality.
RTI's worldwide corporate headquarters are located in Alachua, Fla., with international locations in Germany and France. The company is accredited by the American Association of Tissue Banks in the United States.
RTI Forward Looking Statement
This communication contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on management's current expectations, estimates and projections about our industry, our management's beliefs and certain assumptions made by our management. Words such as "anticipates," "expects," "intends," "plans," "believes," "seeks," "estimates," variations of such words and similar expressions are intended to identify such forward-looking statements. In addition, except for historical information, any statements made in this communication about anticipated financial results, growth rates, new product introductions, future operational improvements and results or regulatory approvals or changes to agreements with distributors also are forward-looking statements. These statements are not guarantees of future performance and are subject to risks and uncertainties, including the risks described in public filings with the U.S. Securities and Exchange Commission (SEC). Our actual results may differ materially from the anticipated results reflected in these forward-looking statements. Copies of the company's SEC filings may be obtained by contacting the company or the SEC or by visiting RTI's website at www.rtix.com or the SEC's website at www.sec.gov.
About Athersys Inc.
Athersys is a clinical stage biopharmaceutical company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The company is developing MultiStem(R), a patented, adult-derived "off-the-shelf" stem cell product platform for multiple disease indications, including damage caused by myocardial infarction, bone marrow transplantation and oncology treatment support, ischemic stroke, and inflammatory bowel disease. The company is also developing a portfolio of other therapeutic programs, including orally active pharmaceutical product candidates for the treatment of metabolic and central nervous system disorders, utilizing proprietary technologies, including Radom Activation of Gene Expression (RAGE(R)). Athersys has forged strategic alliances and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research institutions in the United States and Europe to further develop its platform and products. More information is available at www.athersys.com.
Athersys Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. We have attempted to identify forward-looking statements by using such words as "anticipates," "believes," "can," "continue," "could," "estimates," "expects," "intends," "may," "plans," "potential," "should," "will," or other similar expressions. These forward-looking statements are only predictions and are largely based on our current expectations. A number of known and unknown risks, uncertainties, and other factors could affect the accuracy of these statements. Some of the more significant known risks that we face are the risks and uncertainties inherent in the process of discovering, developing, and commercializing products that are safe and effective for use as human therapeutics, the uncertainty regarding market acceptance of any MAPC technology-based biologic implants in orthopedic applications under the agreement with RTI and our ability to reach milestones and realize any additional payments under such agreement. These risks may cause our actual results, levels of activity, performance, or achievements to differ materially from any future results, levels of activity, performance, or achievements expressed or implied by these forward-looking statements. You should not place undue reliance on forward-looking statements contained in this press release, and we undertake no obligation to publicly update forward-looking statements, whether as a result of new information, future events or otherwise.
SOURCE: RTI Biologics Inc.
RTI Biologics Inc.
Robert Jordheim, Chief Financial Officer
Wendy Crites Wacker, APR, Corporate Communications, 386-418-8888
William (B.J.) Lehmann, J.D., President and Chief Operating Officer, 216- 431-9900
Lisa M. Wilson, 917-543-9932
Pure Communications, Inc.
Dan Budwick, 973-271-6085
This news release was distributed by GlobeNewswire, www.globenewswire.com
SOURCE: Vical Incorporated
CONTACT: Vical Incorporated
Alan R. Engbring