News Release

Achillion and UMBC collaboration identifies new class of agents with potential to treat HIV

Research published today in Journal of Molecular Biology

Peer-Reviewed Publication

Achillion Pharmaceuticals

Baltimore, MD and New Haven, CT, April 1, 2003 – In a successful partnership between industry and academia, researchers from The University of Maryland, Baltimore County (UMBC) and Achillion Pharmaceuticals today announced the discovery of a new target on the HIV molecule that could potentially lead to a new class of anti-viral drugs to fight the virus that causes AIDS. The study, headed by Dr. Michael Summers, UMBC professor of chemistry/biochemistry and Howard Hughes Medical Institute (HHMI) Investigator, is featured on the cover of this week's Journal of Molecular Biology.

"The greatest challenge in treating HIV today is drug resistance brought on when the virus mutates and renders existing drugs ineffective at stopping viral replication," said Dr. Summers. "Our research has led to the identification of a new class of compounds that inhibit a novel target in HIV. These compounds disrupt the assembly of the HIV-1 capsid protein, which is a vital step in changing immature, non-infectious HIV into its mature, infectious form."

"Investigators around the world have been looking at the HIV-1 capsid protein as a potential drug target for more than a decade, and we are therefore particularly pleased to finally have solid leads to guide these efforts," said William G. Rice, Ph.D., Achillion's CEO. "While we are encouraged by the laboratory tests of the compounds we've identified so far, additional testing needs to be undertaken before this approach can be tested in humans."

Dr. Summers and his team identified the target at the UMBC HHMI Lab and discovered a number of compounds that bound to a specific area of the capsid protein thought to play a key role in the assembly process necessary for HIV to mature to its infectious form. The identification process leveraged the nuclear magnetic resonance (NMR) technology resources and target validation expertise at Summers' HHMI laboratory.

Once the new capsid assembly target and compounds inhibiting the target were identified using computer models, researchers from privately-held Achillion Pharmaceuticals tested the compounds in a number of biochemical assays, as well as in human cells infected with live HIV. These experiments demonstrated that the anti-HIV activity of inhibitors was indeed due to disruption of the HIV-1 capsid protein. Three patents have been filed based on the findings reported in today's publication.

"These findings offer exciting opportunities for the discovery of new drugs to treat HIV," said Dr. Rice. "These compounds are part of the portfolio of drug candidates we are building to treat infectious diseases, including antiviral drugs to treat hepatitis B and C viruses (HBV and HCV) and HIV. Achillion will continue to collaborate with Dr. Summers' team to identify additional candidates for optimization and clinical testing."

The discovery is all the more remarkable considering the youth of Summers' research team at UMBC, a group of two graduate and two undergraduate students. The team includes biochemistry graduate student Chun Tang; sixteen-year-old college sophomore and Presidential Fellow Erin Loeliger; Meyerhoff Scholar and HHMI Undergraduate Scholar Isaac Kinde; and former Meyerhoff Scholar Samson Kyere, now an MD/PhD student at the University of Maryland, Baltimore.

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About HIV/AIDS
HIV/AIDS continues to be a major worldwide health issue, with over 40 million people estimated by the World Health Organization to be living with HIV/AIDS worldwide in 2002, including over 900,000 individuals in the US. Drug resistance remains a major issue in the treatment of HIV/AIDS. The Centers for Disease Control estimates that over one-half of US HIV/AIDS patients are infected with strains that are resistant to at least one class of existing drugs, and that up to 16 percent of new HIV infections are due to drug-resistant strains. New classes of drugs working through novel mechanisms are needed to add to the combination therapy, or "drug cocktail", that is the standard of care for suppressing HIV replication in patients.

About the UMBC Research Team
Dr. Summers' previous work on the protein structure of HIV, particularly with undergraduate students, has received much national attention. Working from the only Howard Hughes Medical Institute lab at a public university in Maryland, Dr. Summers and his students have successfully solved three of the seven protein structures which make up HIV. Dr. Summers has also been nationally recognized for his work mentoring undergraduate and graduate student researchers in his lab through the Meyerhoff Scholars Program at UMBC. In 2000, Dr. Summers was one of ten national recipients of the 2000 Presidential Award for Excellence in Science, Mathematics and Engineering Mentoring. The award is administered and funded through the National Science Foundation (NSF).

About Achillion
Achillion (http://www.achillion.com) is a privately-held pharmaceutical company focused on the discovery, development and commercialization of innovative small molecule drugs that combat drug resistance in infectious diseases. Achillion's drug development pipeline is led by the product candidate elvucitabine (Beta-L-Fd4C or ACH-126,443), which is currently in human clinical trials for the treatment of infections caused by hepatitis B virus or human immunodeficiency virus (HIV). Achillion's drug discovery pipeline embodies an innovative program-based approach to identifying new infectious disease targets and designing small molecule drugs to attack these targets, with a particular emphasis on antiviral drugs to treat diseases caused by hepatitis B and C viruses (HBV and HCV), HIV, and new antimicrobial drugs.

This news release contains certain forward-looking statements that involve risks and uncertainties. Such statements are only predictions and Achillion's actual results may differ materially from those anticipated in these forward-looking statements. Factors that may cause such differences include the risk that products that appeared promising in early research and clinical trials do not demonstrate safety or efficacy in larger-scale clinical trials and the risk that the company will not obtain approval to market its products.

For Achillion:
Amy Enders
Achillion Pharmaceuticals, Inc.
203-401-3135
aenders@achillion.com

For University of Maryland, Baltimore County:
Charles Rose
410-455-5793
crose@umbc.edu

Beth Preston
Feinstein Kean Healthcare
617-577-8110
epreston@fkhealth.com


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