VITA™ Technology Presented at ICAAC
Cambridge, MA, September 24, 1998 -- Cubist Pharmaceuticals, Inc. (Nasdaq: CBST) announced today that it will be presenting data on its proprietary VITA™ (Validation In Vivo of Targets and Assays for Antiinfectives) drug discovery technology at the 38th Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC) in San Diego on Sunday, September 27. The VITA technology couples the validation of drug targets during an established infection in a mouse model system with assay development and screening for the discovery of novel drug leads. The data to be presented demonstrates the validation of a target for antiinfective drug discovery by the regulated inhibition of the target in a pathogen during a systemic infection.
In collaboration with Novalon Pharmaceutical Corporation, Cubist will also be presenting data on the identification of potent inhibitor molecules discovered in a new high throughput screening competitive binding assay that overcomes obstacles inherent in the screening of novel targets.
"The genomics explosion is doing an excellent job of solving the problem of identifying potential protein targets, but it has created a new technical challenge of how to harvest this wealth of information for drug discovery," said Scott M. Rocklage, Ph.D., President and Chief Executive Officer of Cubist. "We recognized early on that innovative technologies would be needed to overcome the rate-limiting steps between protein identification, target validation and the discovery of new drug leads. We believe our data demonstrate the ability of our VITA platform technology to triage candidate drug targets and enable an assessment of a greater number and diversity of targets while providing valuable information on potential efficacy within the context of an actual infection. VITA seamlessly integrates the power of genomics into the discovery process. Additionally, in our collaboration with Novalon we have developed an assay that can identify quality screening hits that bind to functionally relevant sites on protein targets."
In a study, titled "1VITA: Validation in vivo of Targets for Antiinfectives," data will be presented describing the successful inhibition of bacterial growth in vitro and the protection of mice against a lethal infection by the regulated intracellular expression of a peptide inhibitor of an essential target.
"Other target validation technologies, such as gene knock-out studies, antisense inhibition of RNA and ribozyme technologies are indirect methods for assaying the role of protein targets during an infection and do not provide a direct path for assaying a target once it is validated," said Francis Tally, M.D., Executive Vice President, Scientific Affairs at Cubist and senior author of the abstract. "Since VITA simulates how a potential antiinfective might affect a drug target in an animal infection model and immediately enables an automated high throughput assay for the target, valuable data facilitating acceleration of the discovery process is obtained. With VITA, we intracellularly express a peptide that binds to a microbial drug target, thereby inhibiting its function. If a target is essential to a pathogen's growth, then its inhibition should allow infected mice to survive an infection by that pathogen, when they would normally die. By observing direct results in the animal, we can prove that there are no other mechanisms that can negate the effect of inhibition of the candidate essential target."
Dr. Tally continued, "The peptide is utilized to assay the target in high throughput screening using a competitive binding assay and chemical compound libraries, obviating the need to develop specific functional assays for each target. Novel targets retractable to other screening technologies such as those encoded by open reading frames and genes of unknown function can now be assayed using the technology."
In a separate study done in collaboration with scientists from Novalon Pharmaceutical Corporation, titled "2Platform Assay Development Strategy: Active Site Directed Peptides as Tools for HTS," researchers describe the identification of small molecule inhibitors to protein targets using a new, platform competitive binding assay. Using phage display technology, specific peptide ligands were identified to approximately 90% of targets tested from a diverse protein target collection. A subset of the peptides was shown to be competitive inhibitors of the targets. Potent, nM level inhibitors that displaced the bound peptide were identified in a competitive binding screening assay. These same inhibitors were also shown to inhibit the enzymatic function of the target.
"Working closely with Cubist, we have developed an assay to facilitate the identification of quality hits for drug discovery," said Dana Fowlkes, MD., Ph.D., CEO and Chief Scientific Officer of Novalon Pharmaceutical Corporation. "The applicability of the assay expands the horizon of screenable targets across broad, therapeutic areas."
Cubist Pharmaceuticals, Inc. is a biopharmaceutical company focused on the research, development and commercialization of novel antimicrobial drugs to combat emerging strains of drug resistant bacteria and fungi. Cubist is engaged in strategic partnerships with Merck & Co., Inc. and Bristol-Myers Squibb for the discovery and development of novel antiinfective products, and has formed biotechnology alliances with ArQule, Inc., Genzyme Corporation, Neurogen Corporation, Novalon Pharmaceutical Corporation and Pharmacopeia, Inc.
1: VITA: Validation in vivo of Targets for Antiinfectives. (1998). J. Tao, T. Li, G. Connelly, X. Shen, J.Silverman, F. Houman, P. Wendler, F.P. Tally. Abstracts of the 38th ICAAC, San Diego, CA.
2: Platform Assay Development Strategy: Active Site Directed Peptides as Tools for HTS. (1998). P. Wendler, P. Gallant, J. Kranz, A. Lim, M. Namchuk, J. Zhang, S. Rocklage, R. Hyde-Deruyscher, L. Page, N. Hyde-Deruyscher, P. Hamilton, Z. Fredericks, D. Fowlkes. Abstracts of the 38th ICAAC, San Diego, CA.
Cubist Safeharbor Statement
Statements contained herein that are not historical facts may be forward-looking statements (within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934) that are subject to a variety of risks and uncertainties. There are a number of important factors that could cause actual results to differ materially from those projected or suggested in any forward-looking statements made by the Company. These factors include, but are not limited to: (i) the Company's ability to successfully complete product research and development, including pre-clinical and clinical studies and commercialization; (ii) the Company's ability to obtain required governmental approvals; (iii) the Company's ability to attract and/or maintain manufacturing, sales, distribution and marketing partners; and (iv) the Company's ability to develop and commercialize its products before its competitors. Additional factors that would cause actual results to differ materially from those projected or suggested in any forward-looking statements is contained in the Company's filings with the Securities and Exchange Commission, including those factors discussed under the caption "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 1997.
VITA ™ is a trademark of Cubist Pharmaceuticals, Inc.
For additional information, visit the Company's Internet web site at http://www.cubist.com.
Editor's Note: For a copy of these papers or to receive additional information on Cubist, please call Neil Cohen at 415-677-4455.