PRNewswire - Friday October 16, 1998

Agenerase has been designated as a fast-track product by the FDA, thereby affording the potential for an expeditious review of the NDA. Applications to market Agenerase are expected to be submitted in Europe and Canada in the coming weeks. In connection with the U.S. NDA filing, Vertex earned a $3 million milestone payment from Glaxo Wellcome.

Clinical results to date suggest that Agenerase has significant antiviral activity and can be used in combination in a variety of two-, three-, and four-drug regimens. Agenerase has been tested clinically in combination with numerous approved antiretroviral therapies, including the nucleoside reverse transcriptase inhibitors, non-nucleoside reverse transcriptase inhibitors, and other protease inhibitors.

"The NDA submitted today contains a broad range of information about the drug's efficacy, safety and pharmacokinetic properties as observed in data from 20 clinical trials involving more than 1,500 patients," said Lynn Smiley, M.D., Vice President of Antiviral Clinical Research at Glaxo Wellcome. "The development of Agenerase underscores Glaxo Wellcome's sustained leadership and commitment to making innovative HIV and antiviral therapies available to patients."

"The NDA submission, the first for a Vertex-discovered product, is a major achievement for the Company and our partner Glaxo Wellcome. Agenerase is the most advanced product in Vertex's portfolio, and its clinical results to date confirm the ability of our structure-based approach to produce compounds with qualities critical to successful pharmaceuticals," commented Dr. Joshua Boger, Chairman, President, and CEO of Vertex. "Clinical studies suggest that Agenerase will fit well into the current standard of care for HIV treatment while addressing the need for simpler regimens."

Preliminary Phase III data presented three weeks ago at the Interscience Conference on Antimicrobial Agents and Chemotherapy showed that in an as- treated analysis(A), 88 percent of patients (65 of 74) who received Agenerase+ Epivir(R) (lamivudine; also known as 3TC(R))+Retrovir (zidovudine; AZT) for 16 weeks achieved viral loads below the limits of detection of standard assays (less than 400 copies/mL) compared to 19 percent of patients who received Epivir and Retrovir. Furthermore, using an observed data analysis(B), 59 percent of patients (52 of 88) in the Agenerase arm achieved viral load below the limit of detection of an investigational assay (less than 50 copies/mL). A separate intent-to-treat data analysis(C), considered to be the most conservative and stringent statistical analysis, showed that 59 percent of patients (66 of 112) receiving Agenerase + Epivir + Retrovir had less than 400 copies/mL at 16 weeks, compared to 17 percent of patients who received Epivir + Retrovir.

Agenerase is dosed as 1200 mg (eight 150 mg capsules) twice daily and, in clinical trials, was taken without regard to food or water. The dose studied in children has been 20 mg/kg of body weight twice daily. A 50 mg pediatric formulation of Agenerase has been developed and is included in the NDA submission. A liquid formulation of Agenerase will be submitted for approval within the next several months.

A unique early access program for Agenerase was announced in mid-September which makes the drug available to patients through three treatment protocols, including two for patients who have already failed a protease inhibitor- containing regimen. Physicians interested in enrolling patients into this program can call 1-800-248-9757 for more information.

In clinical trials to date, Agenerase appears to be generally well- tolerated with few treatment-limiting adverse events. The most commonly reported adverse events in clinical trials have included nausea, diarrhea, headache, fatigue, vomiting, rash and perioral paresthesia.

Agenerase was discovered by scientists at Vertex Pharmaceuticals of Cambridge, MA. Glaxo Wellcome has been responsible for product formulation and manufacture of Agenerase, design and implementation of clinical trials, and regulatory submissions to the FDA. Glaxo Wellcome will also lead the commercialization efforts for Agenerase, with co-promotion assistance from Vertex Pharmaceuticals.

Glaxo Wellcome is the pharmaceutical industry leader in HIV research and therapies. The company currently manufactures and markets Epivir, Retrovir and Combivir(R), which combines Epivir and Retrovir into one tablet. The company also has submitted an application to the FDA to market the investigational new drug Ziagen(TM) (abacavir sulfate).

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX - news) is engaged in the discovery, development and commercialization of novel, small molecule pharmaceuticals for the treatment of diseases for which there are currently limited or no effective treatments. The Company is a leader in the use of structure-based drug design, an approach to drug discovery that integrates advanced biology, biophysics and chemistry. The Company is concentrating on the discovery and development of drugs for the treatment of viral diseases, multidrug resistance in cancer, autoimmune and inflammatory diseases, and neurodegenerative diseases.

There can be no assurance that clinical trials will continue, that initial results will be predictive of any future results, that drugs under development by Vertex or its partners will receive marketing approval from the U.S. Food and Drug Administration or other regulatory authorities, or that drugs, if any, which receive such approval will be marketed successfully. Investors are also directed to consider other risks and uncertainties discussed in Vertex documents filed with the Securities and Exchange Commission.

NOTE: (A) As-treated analyses evaluate only those patients who remain on study medication through the entire study period and do not include patients who discontinued the study. Many physicians prefer as-treated analyses as they provide a more practical basis for success if patients can be continued on a particular treatment regimen over time.

NOTE: (B) Observed data analyses are a common method of analysis in HIV clinical trials. Only patients with data available at the time point of analysis are included. Missing information, regardless of the reason, is not counted. NOTE: (C) Intent-to-treat analyses, which form the bases on which investigational anti-HIV drugs are reviewed by the FDA, include patients who discontinued a study by considering them treatment failures. Intent-to-treat analyses are considered to be valuable as they offer a projection of a regimen's performance across a population of patients.

SOURCE: Vertex Pharmaceuticals Incorporated
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