2006Report HIVM EDICINES IN/DAIDSEVELOPMENT FOR PRESENTED BY AMERICA’ S PHARMACEUTICAL COMPANIES Researchers Are Testing 77 Medicines and Vaccines For HIV and Opportunistic Infections IV/AIDS is one of the world’s most devastating M EDICINES IN D EVELOPMENT FOR AIDS* diseases. To help fight it, biopharmaceutical Hresearchers are testing 77 medicines for HIV/AIDS Anticancers 4 and related conditions and intensifying their work toward Antifungals 1 the development of vaccines. The medicines now in the Anti-Infectives pipeline will add to the 88 already approved since the 2 AIDS virus was first identified more than 20 years ago. Antivirals 35 Vaccine research is crucial to the fight against AIDS. Gene Therapy 2 “A safe and effective HIV vaccine is critical to the control Immunomodulators 7 of HIV globally,” says Dr. Anthony Fauci, Director of the Vaccines 19 National Institute of Allergy and Infectious Diseases. Other Currently, 19 vaccines are in development. 9 According to the Joint United Nations Programme on *Some medicines are listed in more than one category. HIV/AIDS, over the last quarter century, nearly 65 million people were infected with HIV, and an estimated • A vaccine combines DNA snippets from the AIDS 25 million have died of AIDS-related illnesses. As many virus with a protein that boosts immune response. as 1.2 million U.S. residents are estimated to be living The vaccine may prevent infection, limit the damage with HIV infection. Today it is estimated that close to the virus causes, or both. 40 million people worldwide live with HIV—yet the vast • An antisense gene therapy uses two novel technolo- majority are unaware of their status. The increased use of gies to boost immune responsiveness against HIV. newer prescription medicines has helped in recent years One involves the insertion of a new type of genetic to substantially reduce the U.S. AIDS death rate. However, material into blood cells to inhibit the growth of the without a dramatic breakthrough about 45 million new virus. The second involves inserting new genes into people could be infected with the HIV virus by 2010. target cells and integrating the gene into the chro- In addition to the 19 vaccines there are, 35 antivirals, mosome of the cell. The cells containing the new two anti-infectives, four cancer treatments, seven genes are then transferred to the patient. immunomodulators, one antifungal, two gene therapies, While HIV/AIDS remains a formidable foe and and nine other medicines are now in human clinical worldwide scourge, research-based pharmaceutical and trials or before the Food and Drug Administration biotechnology companies are continuing their efforts to awaiting approval. develop novel and more effective therapies and vaccines Examples of AIDS medicines and vaccines in the to contain the disease and improve and lengthen the pipeline include: lives of patients. • One medicine, the first in a new class of drugs known as integrase inhibitors, has been shown to decrease viral load in patients with significant HIV drug resistance. • A medicine in development binds itself to a receptor Billy Tauzin protein found on the surface of human cells and President and CEO blocks the HIV virus from entering the cell. PhRMA Medicines in Development for HIV/AIDS A NTICANCERS Product Name Sponsor Indication Development Status* Avastin® National Cancer Institute AIDS-related Kaposi’s sarcoma Phase II bevacizumab Bethesda, MD NCI TRIAL Genentech (800) 4-CANCER South San Francisco, CA Nexavar® National Cancer Institute AIDS-related Kaposi’s sarcoma Phase I sorafenib Bethesda, MD NCI TRIAL Bayer HealthCare, (800) 4-CANCER Pharmaceuticals West Haven, CT Rituxan® National Cancer Institute AIDS-related lymphoma Phase II rituximab Bethesda, MD NCI TRIAL Biogen Idec (800) 4-CANCER Cambridge, MA Genentech South San Francisco, CA Thalomid® Celgene Kaposi’s sarcoma Phase II thalidomide Summit, NJ (see also other) (908) 673-9000 A NTIFUNGALS Product Name Sponsor Indication Development Status Noxafil® Schering-Plough treatment of oropharyngeal application submitted posaconazole Kenilworth, NJ candidiasis (908) 298-4000 A NTI-INFECTIVES Product Name Sponsor Indication Development Status pafuramidine Immtech International Pneumocystis carinii pneumonia Phase III (DB289) Vernon Hills, IL (PCP) (847) 573-0033 Ushercell® Polydex Pharmaceuticals HIV infection prevention Phase I cellulose sulfate Toronto, Ontario (416) 755-2231 A NTIVIRALS Product Name Sponsor Indication Development Status 364735 GlaxoSmithKline HIV infection treatment Phase I (integrase Philadelphia, PA (888) 825-5249 inhibitor) Rsch. Triangle Park, NC Shionogi USA Florham Park, NJ * For more information about a specific medicine in this report, please call the telephone number listed. 2 M EDICINES IN D EVELOPMENT FOR HIV/AIDS 2006 A NTIVIRALS Product Name Sponsor Indication Development Status Alferon LDO® Hemispherx Biopharma HIV infection treatment Phase II New Brunswick, NJ (see also immunomodulators) (888) 253-3766 Philadelphia, PA AMD070 AnorMed HIV-1 infection Phase I Langley, British Columbia (604) 530-1057 amdoxovir Emory University HIV infection treatment Phase II completed (DAPD) Atlanta, GA University of Georgia Atlanta, GA apricitabine Avexa HIV-1 infection Phase I (AVX754) Richmond, Australia www.avexa.com bevirimat Panacos Pharmaceuticals HIV infection treatment Phase II (PA-457) Watertown, MA (617) 926-1551 brecanavir GlaxoSmithKline HIV infection Phase II (640385, Philadelphia, PA (888) 825-5249 aspartyl protease Rsch. Triangle Park, NC inhibitor) Vertex Pharmaceuticals Cambridge, MA calanolide A Sarawak MediChem HIV-1 infection Phase I (NNRTI) Pharmaceuticals (630) 739-6744 Woodridge, IL CCR5 mAb Human Genome Sciences HIV infection treatment Phase I Rockville, MD (301) 309-8504 CYT 99007 Cytheris HIV infection Phase I (recombinant Rockville, MD www.cytheris.com interleukin-7) elvucitabine Achillion Pharmaceuticals HIV infection treatment Phase II (Beta-L-Fd4C) New Haven, CT (203) 624-7000 Vion Pharmaceuticals New Haven, CT etravirine Tibotec Pharmaceuticals HIV infection treatment Phase III (TMC125, Co. Cork, Ireland (609) 730-7500 NNRTI) Yardley, PA fozivudine tidoxil Heidelberg Pharma HIV infection treatment Phase II Ladenburg, Germany www.heidelberg-pharma.com Fuzeon® Roche HIV-1 infection Phase II enfuvirtide Nutley, NJ (once-daily dosing vs. (973) 235-5000 Trimeris twice-daily dosings) Durham, NC GS 9137 Gilead Sciences HIV infection treatment Phase II Foster City, CA (650) 574-3000 GS 9160 Gilead Sciences HIV infection Phase I Foster City, CA (650) 574-3000 HIV attachment Bristol-Myers Squibb HIV-1 infection Phase I inhibitor Princeton, NJ (212) 546-4000 M EDICINES IN D EVELOPMENT FOR HIV/AIDS 2006 3 A NTIVIRALS Product Name Sponsor Indication Development Status HRG214 Virionyx HIV infection treatment Phase II Auckland, New Zealand www.virionyx.com INCB 9471 Incyte HIV infection treatment Phase I/II Wilmington, DE (302) 498-6700 KP-1461 Koronis Pharmaceuticals HIV infection treatment Phase I Redmond, WA (425) 825-0240 maraviroc Pfizer HIV-1 infection treatment Phase III (UK-427,857, New York, NY (860) 732-5156 CCR5 coreceptor antagonist) MK-0518 Merck HIV-1 infection Phase III (integrase inhibitor) Whitehouse Station, NJ (800) 672-6372 nonakine Gryphon Therapeutics HIV infection treatment Phase I South San Francisco, CA (650) 952-7714 PBS119 Phoenix Biosciences HIV infection Phase I Hollywood, FL (954) 963-6647 PRO 140 Progenics Pharmaceuticals HIV infection treatment Phase I Tarrytown, NY (914) 789-2800 PRO 542 Progenics Pharmaceuticals HIV infection treatment Phase I (CD4-lgG2) Tarrytown, NY (914) 789-2800 PRO 2000 Indevus Pharmaceuticals HIV infection prevention Phase III Lexington, MA (intravaginal gel) (781) 861-8444 Racivir® Pharmasset HIV infection treatment Phase II (NRTI) Princeton, NJ (609) 613-4100 Reyataz® Bristol-Myers Squibb HIV infection application submitted atazanavir (ATV) Princeton, NJ (212) 546-4000 (300mg single- dose capsule) Reyataz® Bristol-Myers Squibb HIV infection (pediatric) Phase III atazanavir (ATV) Princeton, NJ (212) 546-4000 (oral powder) Reyataz® Bristol-Myers Squibb HIV infection in Phase III atazanavir Princeton, NJ antiretroviral-boosted (212) 546-4000 (400mg QD or naive patients 300mg QD with 100mg Norvir® ritonivir) (oral capsule) SP-01A Samaritan Pharmaceuticals HIV infection treatment Phase II Las Vegas, NV (702) 735-7001 Sustiva® Bristol-Myers Squibb HIV-1 infection (pediatric) Phase II efavirenz Princeton, NJ (212) 546-4000 (oral solution) VGX-410 VGX Pharmaceuticals HIV infection Phase II Blue Bell, PA (267) 440-4200 4 M EDICINES IN D EVELOPMENT FOR HIV/AIDS 2006 A NTIVIRALS Product Name Sponsor Indication Development Status vicriviroc Schering-Plough HIV-1 infection Phase II (CCR5 receptor Kenilworth, NJ (908) 298-4000 antagonist) G ENE T HERAPY Product Name Sponsor Indication Development Status HGTV-43™ Enzo Biochem HIV-1 infection Phase I gene medicine Farmingdale, NY (631) 755-5500 VRX496 VIRxSYS HIV infection treatment Phase II (modified Gaithersburg, MD (301) 987-0480 autologous T cells) I MMUNOMODULATORS Product Name Sponsor Indication Development Status Alferon LDO® Hemispherx Biopharma HIV infection treatment Phase II New Brunswick, NJ (see also antivirals) (888) 253-3766 Philadelphia, PA Ampligen® Hemispherx Biopharma HIV infection treatment Phase II/III New Brunswick, NJ (strategic treatment intervention) (215) 988-0080 Philadelphia, PA AMZ0026 Amazon Biotech HIV infection treatment Phase II New York, NY (212) 947-3363 anti-HIV-1 mAb