•© 1992 Nature Publishing Group http://www.nature.com/naturebiotechnology IN 'IH E NEWS/ FDA USER FEES GAIN MOMENTUM •The U.S.FoodandDrugAdministra­ tion the company has received regard­ its rHB1.1 recombinant hemoglobin tion (FDA, Bethesda, MD) and the drug ing its research on cystic fibrosis; at the product. The trials were successful. industry are near an agreement regard­ beginning of this year, recombinant rHB1.1 is designed to replace blood lost ing user fees to speed up the drug ap­ cystic fibrosis transmembrane conduc­ in surgery. proval process. Under the plan, FDA tance regulator was also named an or­ •Celtrix (Santa Clara, CA) completed proposes that drug companies pay sev­ phan drug. phase II trials of its BetaKine for treat­ eral fees: $150,000 for each drug appli­ •Bio-Technology General (New York) ment of macular holes, wounds of the cation, regardless of whether it is in a received approval to market its recom­ retina in the macular region of the eye. life-saving category; $50,000 per com­ binant human growth hormone in Is­ Unless successfully repaired, macular pany per year; $5,000 for each product rael for treatment ofTurner' s syndrome, holes may diminish visual acuity, lead­ on the market. Generic drugs and medi­ a genetic disorder in which girls are ing to legal blindness. The trials had cal devices would not yet be included in born with only one healthy X chromo­ positive results. The company is collect­ the plan. In return for the fees, FDA some. ing data in preparation of phase III must cut the average time it takes to •Bristol-Myers Squibb (New York) studies. review new drug applications from the filed a new drug application (NDA) •Lynx Therapeutics (Foster City, CA) more than 20 months currently to 12 with FDA to market the anti-cancer drug is preparing an investigational new drug months, and cut the average time of taxol. The company is relinquishing (IND) application for its antisense com­ review on life-saving drugs to six months orphan drug designation for taxol, ap­ pound that targets the messenger RNA from the current one year. To accom­ parently to market the drug as treat­ coded by the p53 gene. The compound plish this, the agency will agree to hire ment for a wider variety of cancers. will be used to treat myelogenous leuke­ some 600 more specialists to do the •Cytogen (Princeton, NJ) started mia, and may later be applied to other scientific work necessary on new appli­ phase II trials of its OncoRad ovarian, types of cancer. cations for drugs and biological agents. the company's cancer therapeutic prod­ •Immunex (Seattle, WA) started phase This plan would be phased in over five uct. I trials of soluble tumor necrosis factor years, beginning in the 1993 fiscal year. •RMLMedicalLaboratories (Toronto, (TNF) receptor for the treatment of •Schering-Plough (Madison, NJ) re­ Canada) is expanding it~ phase II trials sepsis. The initial study will test the ceived FDA approval for its recombi­ ofVirulizin in the treatment of cancer. ability of soluble TNF receptor to block nant interferon alpha-2b for treatment The company also plans to pursue regu­ systemic inflammation caused by bacte­ of chronic hepatitis B, an infectious and latory approval to start trials in the U.S. rial infection. sometimes fatal liver disease. The drug and Europe. Current trials ofVirulizin •Isis Pharmaceuticals (Carlsbad, CA) is a copy of a naturally occurring pro­ are focused on recurrent and meta­ completed initial phase I trials for its tein that acts as an antiviral agent in the static melanoma, cancer of the pan­ antisense compound Isis 2105. The com­ body, and is already marketed for treat­ creas and kidney, and recurrent stom­ pound is designed to treat genital warts ment of hairy cell leukemia, AIDS-re­ ach, rectum, and colon cancer. caused by the human papilloma virus. lated Kaposi's sarcoma, genital warts, •CytRx (Norcross, GA) began a pilot •T Cell Sciences (Cambridge, MA) and hepatitis C. Although there are phase II trial with its TherMax gel, a and SmithKline Beecham (Philadelphia, vaccines to prevent hepatitis B, this drug topical therapeutic for pain, inflamma­ PA) filed an IND to begin trials ofsCR1 is the first treatment for the disease. tion, and healing associated with mild for treatment of adult respiratory dis­ •Genzyme (Cambridge,MA) received to moderate burns. tress syndrome (ARDS). The initial tri­ approval to market Ceredase in Israel. •ProCyte (Kirkland, WA) conducted als will be conducted in burn patients at Ceredase is an enzyme replacement a study in France with peptide copper to risk of developing ARDS; after burn therapyforpatientswith type I Gaucher halt hair loss in balding men. The study injury, fluid and white blood cells may disease. The drug was approved last was successful, and the company, as infiltrate the lungs and lead to pulmo­ year for use in the U.S., and applica­ soon as it receives financial backing for nary failure. tions are pending in the European Com­ the compound, expects to start clinical •AmylinPharmaceuticals (San Diego, munity, Canada, and other countries. testing. CA) began the first phase of clinical Also, Genzyme is starting phase 1/II •ImClone Systems (New York) is ex­ trials to test its synthetic amylin hor­ trials for Vianain enzymatic panding pilot studies of its anti-cancer mone therapy for diabetes. Drugs the debridement agent. Studies so far have vaccine, MelVax, for treatment of ma­ company hopes to develop would re­ indicated that Vianain appears to be lignant melanoma. The new patient place amylin in young diabetics who rapid and effective in removing non­ group will receive MelVax and an adju­ have difficulties with insulin therapy viable tissue from burn sites. vant intended to boost immune re­ and where amylin appears to be defi­ Genzyme received orphan drug desig­ sponse against melanoma tumor cells. cient, and block the action of the hor­ nation for its cystic fibrosis gene therapy. •Somatogen (Boulder, CO) has com­ mone in older patients with adult onset This is the second orphan drug designa- pleted the first stage of phase I trials of diabetes where there is an excess of the 940 BID/TECHNOLOGY VOL 10 SEPTEMBER 1992 •© 1992 Nature Publishing Group http://www.nature.com/naturebiotechnology hormone. Phase I trials are aimed at each other's patents and patent appli­ addressing a treatment for the young. cations related to anti-endotoxin MAbs. Centocor also formed a strategic alli­ AIDS CONFERENCE ance with Eli Lilly (Indianapolis, IN) •Biocine (Emeryville, CA) reported that gives Centocor at least $1 00 million that 12 vaccinated subjects who received and will give Lilly a 5 percent stake in 3 immunizations with the vaccine for­ Centocor. Lilly acquires marketing mula of recombinant gp120 plus adju­ rights to Centocor'sHA-lA, as well as an vant produced neutralizing antibodies option to spend additional money for against HIV. rights to Centocor's MAb platelet ag­ •Genentech's (S. San Francisco, CA) gregation inhibitor, CentoRx. gp120 vaccine was given to 28 HIV­ •Immunex and Hoechst (Frankfurt, negative adult volunteers. Two weeks Germany) agreed to settle all contract after the third immunization, 7 of 10 and other claims in two lawsuits pend­ subjects who had been given 300 meg ing between them. The suits dealt with doses developed fusion inhibition anti­ granulocyte macrophage colony-stimu­ body, and neutralization antibody was lating factor-a drug Immunex manu­ found in 9 of l 0 subjects to the homolo­ factures that is used in autologous bone gous isolate IIIB. marrow transplants-and give •ImmunoAG's (Wien,Austria) vaccine, Immunex exclusive rights to the drug recombinantgpl60, stimulated HIV anti­ in the U.S., while giving Behringwerke bodies and was well tolerated in 60 HIV­ (Marburg, Germany), a subsidiary of negativevolunteers. Hoechst, significantly increased royal­ •Immune Response (San Diego, CA) ties. andRhone-Poulenc Rorer (Collegeville, •Pharmaceutical Proteins (Edinburgh, PA) presented the results of a dose­ U.K.) and Wyeth-Ayerst (Philadelphia, ranging study of their HIV immuno­ PA) have entered into a long-term col­ therapeutic. The study demonstrated laboration to develop special use products that the HIV immunotherapeutic sig­ for metabolic, geriatric, and pediatric ap­ nificantly stimulated antibody and cel­ plications. The products will utilize the lular immune responses in treated pa­ technology ofproduction ofprot eins in tients. the milk of transgenic animals. •Tanox Biosystems (Houston, TX), • British Bio-technology (BBT, Oxford, University Hospital of Ziirich (Switzer­ U.K.) and Glaxo (New York) agreed to • V~ral Antigens • V1ral 0 A • Pur1f1ed land), and Ciba-Geigy (Basel, Switzer­ jointly develop BB-882, BBT's anti-in­ Protems • Human AB Serum • aturalll 2 land) completed a study of a mono­ flammatory drug. The companies will • V1rus Isolation • V1ral 0 A Amphf1cauon clonal antibody against HIV. Antibody collaborate primarily on the use of the Pr1mers • V1ral 0 A Controls For PCR therapy aims at controlling HIVby pro­ drug in the treatment of asthma. BBT • Macrophage Cultures • Elu nated Human viding an infected person with antibod­ started phase I trials of the drug, and lymphocytes • Adult Fresh Cultured ies that can neutralize or block the Glaxo is funding the trials. Glaxo will Mononuclear Cells • Elutnated Human also fund phase II trials. virus. The results of the limited study Monocytes • Cultured Cord Blood seem to show the antibody was well •Oxford Glycosystems (Abingdon, tolerated by the volunteer AIDS pa­ U.K.) and SmithKline Beecham (Ep­ Mononuclear Cells • Electron Microscopy tients. som, U.K.) signed a research agree­ • Fresh Cultured PHA-st1mulated •Genelahs Technologies (Redwood ment focused on developing carbohy­ Mononuclear Cells • Fresh Heparimzed City, CA) presented results of the phase drate-based inhibitors of selectin-medi­ Cord Blood • PHA·s!lmulated Cord Blood 1B study of its anti-AIDS drug, GLQ223, ated cell adhesion.