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June 16, 1999 ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) ARIAD Presents Exciting Animal Data at 2nd Annual American Society of Gene Therapy Conference; Reiterate BUY Recommendation
Summary Investment Considerations Last Friday, ARIA presented exciting new data in an animal model for diabetes. Using a novel gene therapy technology developed in conjunction with Memorial Sloan-Kettering Cancer Center and the University of Geneva, ARIA demonstrated the ability to control the production of insulin, and several other secreted proteins, in mice using a novel small-molecule drug. While still early in development, we believe this represents a large potential opportunity for ARIA both short-term, in its ability to partner the technology, and longer-term, through the multiple therapeutic applications this technology opens up. We continue to believe that ARIA stock is undervalued at current prices and are reiterating our BUY recommendation for those investors tolerant of the risks associated with micro-cap equity investments. The Background: Unlike ARIAs ARGENT technology, which uses an oral dimerizer drug to control the production of proteins at the gene transcription stage, this newly presented technology demonstrated the ability to control the release of secreted proteins already produced and stored within the cell. In the case of insulin, ARIA scientists engineered cells to produce insulin bound to a specific inert protein that prevented the insulin from being secreted from a storage compartment within the cell. These engineered cells were injected into diabetic mice that lack the ability to break down blood sugar. These hyperglycemic mice were then given ARIAs small-molecule drug that cleaves the insulin from the binding protein and allows secretion of insulin into the bloodstream. Within thirty minutes, in response to the newly released insulin, the animals blood glucose levels began to fall, and within two hours, the mice were able to achieve normal glucose levels. The Advantages: While it is still early stage, this technology appears to enable control of protein release much more quickly than with the ARGENT system. This is particularly important for those proteins that require frequent dosing by injection. If successful in humans, this technology could require infrequent gene therapy injections, and then dosing with an oral or sublingual drug perhaps several times per day. ARIA is now optimizing the delivery system used to deliver the genes and hopes to test the technology in larger animals by the end of the year. The Implications: Although this new technology should be applicable to multiple types of secreted proteins, insulin control, we believe, would offer ARIA the largest commercial opportunity. With an estimated population in the U.S. of over 15 million diabetics, a technology such as this could easily open up licensing and partnering doors for ARIA and provide an additional source of funding to the company. Conclusion: We continue to be impressed with the depth of ARIAs technology. In addition, due to its multi-million deal with Hoechst-Marion Roussel in drug development, its involvement in the Hoechst-ARIAD Genomic Center, its ARGENT technology that we believe ARIA is close to partnering, and this additional gene therapy technology, we feel ARIA presents a unique investment opportunity at this time.
Risk Considerations This section of the document is provided to remind potential investors to undertake a prudent level of due diligence prior to making an investment in the securities of ARIAD Pharmaceuticals, Inc. For a complete description of risks and uncertainties to ARIAs business, see the "Risk Factors" section in ARIAs SEC filings, which can be accessed directly from the SEC Edgar filings at www.SEC.gov on the Internet. Other potential risks include:
Sources for Additional Information The following are website addresses offering related information, and links to other sources of information. www.ariad.com ARIADs corporate website www.hoechst.com Hoecsht Marion Roussel corporate website www.SmallCapsOnline.com SmallCaps Onlines site for company information and research www.FDA.gov US Food and Drug Administration homepage www.sec.gov US Securities and Exchange Commission, with links to EDGAR filings
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