Company* |
Product | Description | Indication |
Status (Date) |
AUTOIMMUNE | ||||
Active Biotech |
RhuDex |
Small-molecule CD80 antagonist |
Rheumatoid arthritis |
Phase I trials of the product began (5/9) |
Astralis Ltd. |
Psoraxine |
Protein-based immunostimulator |
Psoriasis |
Further data from Phase II trial that failed to meet primary end-point identified factors that could have led to that result (5/6) |
Enzo Biochem |
Alequel |
A complex of autologous colon-derived antigens administered orally |
Crohn's disease |
Phase IIb trial produced clinical remission rates of 58% for drug patients vs. 29% for placebo (5/16) |
Medarex Inc. |
MDX-1100 |
Fully human monoclonal antibody that targets the chemokine IP-10 (CXCL10) |
Ulcerative colitis |
Began Phase I trial in up to 32 patients to collect safety and initial efficacy data (5/11) |
Synta |
STA-5326 |
Small-molecule, selective inhibitor of the IL-12 cytokine family |
Crohn's disease |
Phase IIa trial in 73 patients demonstrated meaningful clinical activity at all dose levels except the lowest (5/18) |
CANCER | ||||
Adherex |
ADH-1 (Exherin) |
Cadherin antagonist; small peptide, tumor/vascular-targeting agent |
Advanced chemotherapy-resistant cancer |
Phase I data showed ADH-1 was well tolerated over a 20-fold dose range and has displayed evidence of antitumor activity (ASCO) |
Adherex |
ADH-1 (Exherin) |
Cadherin antagonist; small peptide, tumor/vascular-targeting agent |
Advanced cancers |
Began Phase Ib/II trial in 40 patients whose tumors express N-cadherin, to evaluate safety, tolerability and antitumor activity (5/12) |
Adventrx |
CoFactor |
Form of folic acid designed to enhance the antitumor effects of 5-FU |
Metastatic colorectal cancer |
Phase II trial with 5-FU showed a 63% overall clinical benefit and a 38% objective response rate (5/13) |
AEterna |
Neovastat |
Agent believed to inhibit VEGF signaling, MMP activity and induction of endothelial-cell apoptosis |
Stage III non- mall-cell lung cancer |
Data from 341 patients in NCI Phase III trial demonstrated that blinded overall toxicity appeared acceptable (ASCO) |
Agennix Inc.* |
Talactoferrin |
Oral, recombinant human lactoferrin |
Advanced non- small-cell lung cancer |
Phase II data showed drug enhanced patient responses to chemotherapy on all tumor-related endpoints (ASCO) |
Amgen Inc. (AMGN) and |
Panitumumab |
Fully human monoclonal antibody that targets the epidermal growth factor receptor |
Metastatic colorectal cancer |
Updated data from Phase II single- agent trial demonstrated a 9% overall response rate (ASCO) |
Amgen Inc. |
Neulasta (FDA-approved) |
Pegfilgrastim; white-blood-cell stimulator |
Solid tumors |
Trial in older patients showed those receiving Neulasta in the first chemotherapy cycle had reductions in febrile neutopenia and related complications, compared to those who received it in later chemotherapy cycles (ASCO) |
Amgen Inc. |
Aranesp (FDA-approved) |
Darbepoetin alfa; a recombinant erythropoietic protein |
Chemotherapy-induced anemia |
Phase III data demonstrated that 200 mcg of Aranesp every two weeks is as effective as 40,000 U of Epoetin alfa once a week in boosting hemoglobin levels and reducing red-blood-cell transfusions (ASCO) |
Antisoma plc |
AS1411 (formerly AGRO100) |
Anti-nucleolin aptamer |
Spreading cancers |
Seven patients in Phase I trial showed stable disease for two months or more, and one had a near-complete response (ASCO) |
Aphton Corp. |
IGN101 |
Vaccine designed to trigger an immune response to epithelial cell-adhesion molecule |
Solid tumors |
Phase II data showed the median number of circulating tumor cells had been reduced by about 50% (ASCO) |
Ariad Pharmaceuticals |
AP23573 |
Oral form of agent designed to inhibit cell-signaling protein mTOR |
Refractory/relapsed cancers |
Began Phase Ib trial to evaluate safety, activity and pharmaco-kinetics in up to 150 patients (5/26) |
Ariad |
AP23573 |
Intravenous form of agent designed to inhibit cell-signaling protein mTOR |
Advanced sarcoma |
Phase II data showed clinical benefits and symptomatic improvements (ASCO) |
Ariad |
AP23573 |
Small molecule designed to inhibit cell-signaling protein mTOR |
Refractory prostate cancer |
Began Phase II single-agent trial to evaluate the clinical benefit in up to 35 patients (5/3) |
ArQule Inc. |
ARQ 501 |
Activated Checkpoint Therapy molecule designed to selectively kill cancer cells |
Advanced solid tumors |
Phase I data demonstrated clinical tolerability, favorable pharmacokinetics and evidence of anti-tumor activity (ASCO) |
Callisto |
Atiprimod |
Drug designed to inhibit VEGF and interleukin-6 |
Relapsed multiple myeloma |
No dose-limiting toxicities were observed through three patient cohorts in Phase I/IIa trial (5/4) |
CancerVax |
Canvaxin |
Therapeutic whole-cell vaccine that expresses more than 20 antigens |
Melanoma |
Phase II trial in 13 patients showed treatment induced antibody responses to several melanoma-associated antigens (ASCO) |
CancerVax |
SAI-EGF |
Immunotherapy agent that targets the epidermal growth factor receptor signaling pathway |
Advanced non-small- cell lung cancer |
Phase II trial showed agent was well tolerated, stimulated an antibody response to EG, and reduced circulating EGF (ASCO) |
Celgene Corp. |
Revlimid |
Lenalidomide; derivative of Thalomid (thalidomide) |
Chronic lymphocytic leukemia; myelodysplastic syndrome; multiple myeloma |
Clinical data showed that 12 of 14 relapsed or refractory CLL patients responded to treatment; Revlimid provided evidence it could be used to treat MDS patients with deletion 5q chromosomal abnormalities; Phase III data showed Revlimid plus dexamethasone more than doubled the response rate in MM patients (ASCO) |
Cell |
GVAX |
Whole-cell vaccine comprised of tumor cells irradiated and modified to secrete GM-CSF |
Advanced prostate cancer |
Additional data from 22 patients in Phase II trial showed final median survival will be no less than 24.1 months (ASCO) |
Cell |
GVAX |
Whole-cell vaccine comprised of tumor cells irradiated and modified to secrete GM-CSF |
Acute myelogenous leukemia |
Follow-up data from Phase II trial showed GVAX generally was well tolerated and may reduce residual leukemic cells (ASCO) |
Cell |
Xyotax |
Paclitaxel linked to a biodegradable polyglutamate polymer |
Ovarian cancer (Stage III/IV); esophageal and gastric cancer; non- small-cell lung cancer |
Phase II data of Xyotax with carboplatin showed a major tumor response in 98% of patients; Phase I data of Xyotax with radiation demonstrated complete disappearance of the tumor in 36% of patients; Phase III trial showed a 40% improvement in overall survival compared with vinorelbine (ASCO) |
Cell |
Xyotax |
Paclitaxel linked to a biodegradable polyglutamate polymer |
Non-small-cell lung cancer |
Phase III STELLAR 2 and 4 trials missed their primary endpoints of superior overall survival, but did show equivalent survival with reduced side effects (5/2) |
Cel-Sci Corp. |
Multikine |
Mixture of human cytokines designed to stimulate an immune response |
Advanced oral squamous-cell carcinoma |
Phase II data showed an overall response rate of 42%, with a 12% complete response rate (5/20) |
Coley |
CPG 7909 |
Toll-like receptor 9 agonist |
Advanced non-small-cell lung cancer |
Phase II data of CPG 7909 with standard chemotherapy showed an objective response rate of 37%, compared with 19% in those receiving chemotherapy alone (ASCO) |
CuraGen Corp. |
CG53135 |
Growth factor that promotes both epithelial- and mesenchymal-cell proliferation |
Oral mucositis in cancer patients |
Final Phase I data suggested the drug was well tolerated following intravenous administration (ASCO) |
Cytogen Corp. |
Quadramet (FDA-approved) |
Samarium-153 bound to a small-molecule, bone- seeking phosphonate |
Progressive hormone- refractory prostate cancer |
Phase II data of Quadramet with docetaxel demonstrated good palliative effect lasting five months (ASCO) |
Cytokinetics |
SB-743921 |
Small-molecule inhibitor of kinesin spindle protein |
Cancers |
Phase I data showed the compound appeared to have an acceptable tolerability profile (ASCO) |
Dendreon |
Provenge |
Immunotherapy that uses a recombinant form of the prostatic acid phosphatase antigen |
Early stage prostate cancer |
Open-label D9905 Phase II trial suggested single-agent treatment may lead to improved PSA doubling time (5/24) |
Dendreon |
Provenge |
Immunotherapy that uses a recombinant form of the prostatic acid phosphatase antigen |
Asymptomatic, metastatic androgen-independent prostate cancer |
Provenge improved survival when compared to patients receiving placebo (ASCO) |
Genentech Inc. |
Herceptin (FDA-approved) |
Trastuzumab; monoclonal antibody against HER2/neu protein |
Early stage breast cancer |
Phase III data showed that HER2-positive patients had a 52% reduction in the risk of disease recurrence compared to those receiving chemotherapy alone (ASCO) |
Genentech Inc. |
Avastin (FDA-approved) |
Bevacizumab; antibody designed to inhibit vascular endothelial growth factor |
First-line, non-squamous, non-small-cell lung cancer |
A Phase III trial of Avastin plus paclitaxel and carboplatin met its primary endpoint, with treated patients having a 30% improvement in overall survival (ASCO) |
Genentech Inc. |
Tarceva (FDA-approved) |
Erlotinib HCl; small- molecule HER1/EGFR inhibitor |
Advanced pancreatic cancer |
Phase III data showed a 23.5% improvement in overall survival when Tarceva was used with gemcitabine (ASCO) |
Genentech Inc. |
Omnitarg |
Pertuzumab |
Ovarian, breast and prostate cancers |
Phase II data showed Omnitarg had limited activity as a single agent, but the company plans to pursue further study in ovarian cancer (ASCO) |
Genentech Inc. |
-- |
Antagonist of the Hedgehog signaling pathway |
Basal-cell |
Starting a Phase I trial of an agent for the topical treatment of BCC (5/4) |
Genmab A/S |
HuMax-EGFr |
Human antibody that targets the epidermal growth factor receptor |
Refractory head and neck cancer |
Additional data from Phase I/II trial showed seven of 18 patients achieved partial responses and four had stable disease(ASCO) |
Genta Inc. |
Genasense |
Oblimersen sodium; inhibits function of bcl-2 protein |
Relapsed/refractory chronic lymphocytic leukemia |
Updated data from positive Phase III trial showed significantly superior duration of responses (5/13) |
Genta Inc. |
Genasense |
Oblimersen sodium; inhibits function of bcl-2 protein |
Solid tumors |
Combination trial showed tolerability in children with solid tumors; 14 of 27 prostate cancer patients achieved a major reduction in PSA levels; Phase I trials in gastrointestinal cancer produced safety data (ASCO) |
Genta Inc. |
Genasense |
Oblimersen sodium; inhibits function of bcl-2 protein |
Advanced malignant melanoma |
Phase III data showed a significant increase in durable response and a near-significant trend toward increased overall survival (ASCO) |
GenVec Inc. |
TNFerade |
Adenovector with TNF-alpha gene and a radiation-responsive promoter |
Rectal cancer |
Company got OK from FDA to resume enrollment in Phase II trial; a hold had been placed on the study in October 2004 (5/25) |
GenVec Inc. |
TNFerade |
Adenovector with TNF-alpha gene and a radiation-responsive promoter |
Advanced pancreatic cancer |
Phase II data showed dose-dependent improvements in tumor shrinkage and resectability and in patient survival (ASCO) |
Geron Corp. |
GRN163L |
Telomerase inhibitor |
Chronic lymphocytic leukemia |
Starting a Phase I/II trial to assess safety, tolerability and pharmacokinetics in patients with advanced CLL (5/23) |
Gloucester |
FK228 |
Depsipeptide; histone deacetylase inhibitor |
Cutaneous T-cell lymphoma and solid tumors |
Objective responses were seen in five of 19 patients with advanced CTCL in Phase II trial; drug was relatively well tolerated in Phase I trial in pediatric patients with solid tumors (ASCO) |
GTx Inc. |
Acapodene |
Toremifene citrate; small- molecule non-steroidal selective estrogen receptor modulator |
Prostate cancer |
Phase IIb data showed it significantly reduced the risk of prostate cancer by about 48% among men treated for a year (ASCO) |
Guilford |
Gliadel Wafer (FDA-approved) |
Wafer comprised of a biodegradable polymer incorporating carmustine |
High-grade malignant glioma |
Trial of drug and temozolomide determined the combination to be safe (ASCO) |
Hana |
Talotrexin (PT-523) |
Non-classical antifolate; analogue of aminopterin |
Acute lymphoblastic leukemia |
Began Phase I/II single-agent trial in relapsed or refractory patients that initially will assess dosing, safety, tolerability and pharmacokinetics (5/12) |
Human |
HGS-ETR2 and HGS-ETR1 |
Agonistic human monoclonal antibody to TRAIL receptors 2 and 1, respectively |
Advanced solid tumors |
Ongoing Phase I trials demonstrated tolerability of both agents, and support further evaluation in Phase II trials (ASCO) |
Hybridon Inc. |
Imoxine |
Immunomodulatory oligonucleotide; TLR9 agonist |
Renal-cell carcinoma |
Phase I data showed it was well tolerated and had immunopharmacological activity (ASCO) |
ImClone |
Erbitux (FDA-approved) |
Cetuximab; antibody that blocks the epidermal growth factor receptor |
Colorectal cancer |
Data showed a 37% partial response in irinotecan-refractory metastatic colorectal cancer patients when Erbitux was used with Avastin and irinotecan, and it showed a 10% complete response and a 71% partial response in patients with non-resectable, EGFR-expressing metastatic colorectal cancer treated with Erbitux and Folfox- (ASCO) |
Immuno- |
Mepact |
Immune system stimulant designed to activate macrophages |
High-grade osteosarcoma |
Six-year data from 793 patients suggested drug added to chemotherapy improves disease-free and overall survival in those with nonmetastatic disease (ASCO) |
ImmunoGen |
huN901-DM1 |
Anti-CD56 antibody huN901 combined with the cell-killing agent, DM1 |
Small-cell lung cancer |
Initial Phase II data demonstrated evidence of anticancer activity, triggering expansion of the study (ASCO) |
Inex |
Marqibo |
Vincristine encapsulated in Inex's TCS (liposomal) drug delivery technology |
Various malignancies |
Phase II trials totaling 211 patients with non- Hodgkin's lymphoma demonstrated clinically meaningful responses; Phase II follow-up data in relapsed Hodgkin's disease showed agent was well tolerated; Phase II trial in 30 patients with pediatric malignancies concluded further study was warranted (ASCO) |
Introgen |
INGN 225 |
Product using Advexin to stimulate dendritic cells, which are then used as a therapeutic vaccine |
Advanced small-cell lung cancer |
Phase II data showed that 67% of patients had objective responses following INGN 225 treatment (ASCO) |
Keryx Bio- |
KRX-0401 |
Perifosine; oral AKT inhibitor |
Biochemically recurrent hormone-sensitive prostate cancer |
Phase II data demonstrated the tolerability and potential efficacy of KRX-0401 (ASCO) |
Keryx Bio- |
KRX-0401 |
Perifosine; oral AKT inhibitor |
Breast cancer |
Began two Phase II trials, one in combination with Herceptin and one with endocrine therapy (5/11) |
Kosan |
KOS-862 |
Epothilone D, a |
Advanced non-small-cell lung cancer |
Four of 50 patients in a single-agent Phase II trial had partial responses, and 13 had disease stabilization (ASCO) |
Kosan |
KOS-953 |
17-AAG; designed to inhibit heat-shock protein 90 |
Relapsed or refractory multiple myeloma |
Phase I data showed anticancer |
Ligand |
Targretin (FDA-approved) |
Bexarotene capsules; selective retinoid X receptor modulator |
Advanced non-small-cell lung cancer |
Pivotal Phase III trials did not meet their endpoints of improved overall survival and projected two- ear survival; separately, positive data were reported from other studies (ASCO) |
Ligand |
Ontak (FDA-approved) |
Denileukin diftitox |
Advanced non-small-cell lung cancer and CLL |
Phase II trial showed disease control in 16 of 40 NSCLC patients; Phase II trial in 16 patients with B-cell chronic lymphocytic leukemia showed moderate activity with manageable toxicities (ASCO) |
Marshall |
Phenoxodiol |
Agent designed to induce apoptosis by removing intracellular proteins XIAP and c-FLIP |
Ovarian cancer |
Phase IIa trial in 40 patients resistant to cisplatin and/or paclitaxel showed an overall disease control rate of 77% when drug was added to the regimen (5/10) |
MedImmune |
Siplizumab |
Humanized monoclonal antibody that binds to the CD2 receptor on T cells and natural killer cells |
T-cell lymphomas and leukemias |
Preliminary Phase I data demonstrated safety and tolerability; partial disease remissions also were noted (ASCO) |
MedImmune |
Vitaxin |
Monoclonal antibody that targets the alpha-v beta-3 integrin |
Stage IV meta- static melanoma |
Phase II data showed a 12.7- month median survival for those treated with Vitaxin alone, and a 9.4-month median survival of patients treated with Vitaxin plus dacarbazine (ASCO) |
MGI Pharma |
Dacogen |
Decitabine for injection; a hypomethylating agent |
Myelodysplastic syndromes |
Dacogen demonstrated a 44% overall response rate in a dosing study and a 17% overall response rate in the MDS trial (ASCO) |
Millennium |
Velcade (FDA-approved) |
Bortezomib; proteasome inhibitor |
Lymphoma, lung cancer and multiple myeloma |
Median survival had not been reached at 10.5 months in mantle- cell lymphoma patients, who had an overall response rate of 42%; lung cancer patients showed a response rate comparable with approved agents; MM front-line data show potential benefits in earlier lines of therapy (ASCO) |
Millennium |
MLN2704 |
Monoclonal antibody (MLN591) directed at PSA conjugated to DM1 |
Prostate cancer |
Phase I/II data showed four of six patients receiving a high dose achieved a reduction in prostate-specific antigen (ASCO) |
NeoPharm Inc. |
LErafAON- ETU |
Liposomal formulation of an antisense oligonucleotide complementary to the c-raf mRNA sequence |
Advanced cancers |
Reported on successful initial clinical dosing in a Phase I trial (ASCO) |
NeoPharm Inc. |
IL13-PE38QQR |
Tumor-targeting agent designed to deliver bacterial cytotoxic PE38 |
Brain cancer |
Combined data from 45 patients in Phase I trials showed median survival of 44 weeks, vs. 28 weeks for approved therapies (5/6); combined data from Phase I trials showed advantages for peritumoral vs. intratumoral infusion (ASCO) |
NeoRx Corp. |
Skeletal Targeted |
Bone-targeting molecule DOTMP with radioactive substance holmium-166 |
Various cancers |
NeoRx discontinued clinical development of STR for multiple myeloma and other indications, after the FDA sought changes for Phase III trials (5/6) |
Novacea Inc.* |
DN-101 |
Contains calcitriol, a naturally occurring hormone and the biologically active form of vitamin D |
Prostate cancer |
Data demonstrated that prostate-specific antigen response occurred more frequently in patients receiving DN-101 plus Taxotere than those in the control group, but the difference was not statistically significant (ASCO) |
Onyx |
Sorafenib (BAY 43-9006) |
RAF kinase and VEGF inhibitor |
Advanced renal-cell carcinoma |
Phase III data showed sorafenib improved progression-free survival from a median value of 12 weeks up to 24 weeks (ASCO) |
Onyx |
Sorafenib (BAY 43-9006) |
RAF kinase and VEGF inhibitor |
Advanced metastatic melanoma |
Began Phase III trial with carboplatin and paclitaxel that will assess progression-free survival in more than 200 patients (5/13) |
OSI |
Tarceva (FDA-approved) |
Erlotinib HCl; small- molecule HER1/EGFR inhibitor |
Non-small-cell lung cancer |
Analysis of a registration study showed a greater, but not significant, survival benefit from Tarceva in all subsets of patients whose tumors expressed EGFR protein; Phase II monotherapy trials showed encouraging signs of antitumor activity, and combination studies outside NSCLC also were encouraging (ASCO) |
OxiGene Inc. |
CA4P |
Combretastatin A4 prodrug; a vascular targeting agent |
Advanced cancer |
Phase Ib/II data showed it was well tolerated when combined with radiotherapy (ASCO) |
OxiGene Inc. |
CA4P |
Combretastatin A4 prodrug; a vascular targeting agent |
Advanced cancers |
Antitumor activity and no unexpected toxicities were seen in Phase Ib portion of Phase Ib/II combination trial (ASCO) |
Pharmacyclics |
Xcytrin |
Motexafin gadolinium; small molecule from the texaphyrin class designed to induce apoptosis |
Advanced renal-cell carcinoma |
Interim data from Phase II trial in 22 patients showed clinical benefit in five patients; three patients showed tumor responses (ASCO) |
Pharmacyclics |
Xcytrin |
Motexafin gadolinium; small molecule from the texaphyrin class designed to induce apoptosis |
Advanced refractory tumors |
Interim data from Phase I trial with Taxotere showed partial responses in six of 12 patients; two more patients achieved stabilization of their tumors (ASCO) |
Point |
Talabostat |
Small molecule designed to stimulate proliferation of hematopoietic progenitor cells |
Advanced non-small-cell lung cancer |
Five of 36 patients patients demonstrated a clinical response to combination treatment with docetaxel in a Phase II trial (ASCO) |
Point |
Talabostat |
Small molecule designed to stimulate proliferation of hematopoietic progenitor cells |
Stage IV melanoma |
Phase II data showed two patients had at least a 30% reduction in tumor size in response to talabostat, with one achieving a complete response (ASCO) |
Praecis |
Plenaxis (FDA-approved) |
Abarelix for injection, depot formulation; gonadotropin-releasing hormone antagonist |
Palliative treatment of advanced prostate cancer |
As part of a restructuring, the firm discontinued promotional activities and the sale of Plenaxis for new patients in the U.S. (5/20) |
Pro- |
Davanat |
Carbohydrate compound designed for targeted delivery |
Metastatic colorectal cancer |
Began Phase II trial to evaluate efficacy and safety with 5-FU as as third- or fourth-line therapy in up to 38 patients (5/9); Phase I trial showed disease stabilization in 14 of 26 patients (ASCO) |
Reata |
RTA 744 |
Anthracycline derivative that crosses the blood- brain barrier |
Advanced brain cancers |
Starting trials of the drug in patients (5/6) |
Regeneron |
VEGF Trap |
Intravenous product designed to block vascular endothelial growth factor |
Advanced solid malignancies |
Phase I trial in 27 patients showed the agent generally was well tolerated, and showed evidence of efficacy in some patients (ASCO) |
Regeneron |
VEGF Trap |
Intravenous product designed to block vascular endothelial growth factor |
Advanced cancers |
Began trial to assess safety, tolerability, pharmacokinetics and responses in combination with the Folfox-4 regimen (5/12) |
Seattle |
SGN-30 |
Monoclonal antibody that targets CD30+ hematologic malignancies |
Systemic anaplastic large-cell lymphoma |
SGN-30 was well tolerated and demonstrated objective anti-tumor activity (ASCO) |
Seattle |
SGN-40 |
Humanized monoclonal antibody that targets the CD40 antigen |
Multiple myeloma |
SGN-40 showed evidence of biological activity (ASCO) |
Sonus |
Tocosol |
Formulation of paclitaxel using Sonus' delivery system |
Cancers |
Data showed a single dose administered over 15 minutes delivered 67% more active paclitaxel compared to an identical dose of Taxol (ASCO) |
Spectrum |
SPI-153 |
Fourth-generation luteinizing hormone- releasing hormone |
Hormone-dependent prostate cancer |
Starting a Phase I/II trial in patients (5/31) |
Structural |
Troxatyl |
Troxacitabine; anti- cancer agent |
Acute myelogenous leukemia |
Phase I/II trial in 48 patients demonstrated tolerability in relapsed patients, and an overall response rate of 17% (ASCO) |
SuperGen Inc. |
Orathecin |
Rubitecan; topoisomerase-I inhibitor in capsule formulation |
Pancreatic cancer |
Phase III data indicate patients with progressive disease following gemcitabine treatment can benefit from Orathecin (ASCO) |
Synta |
STA-4783 |
Small-molecule compound designed to induce expression of heat-shock protein 70 tumor cell surfaces |
Various cancers |
Phase II trials with paclitaxel in non-small-cell lung cancer and soft-tissue sarcoma supported advancement to the second stage of the trials; Phase I trial with pacli- taxel in various cancers demonstrated tolerability and some responses (ASCO) |
Tapestry |
TPI 287 |
Taxane designed to overcome the resistance to other taxanes |
Recurrent/ refractory malignancies |
Began Phase I trial to evaluate safety and pharmacokinetics in about 48 patients (5/23) |
Telik Inc. |
Telcyta (TLK286) |
Small-molecule, tumor-activated drug designed to induce apoptosis |
Advanced non-small-cell lung cancer |
Phase I/IIa trial with carboplatin and paclitaxel in 13 patients showed a 62% objective response rate; trial with cisplatin showed a 36% objective response rate and 86% rate for disease stabilization (ASCO) |
Therion |
Prostvac-VF |
Therapy designed to stimulate the immune system to target and destroy cancer cells expressing epitopes of PSA |
Metastatic androgen- insensitive prostate cancer |
Phase II trial in 30 patients confirmed the safety profile and showed declines in PSA in several patients (ASCO) |
Therion |
Panvac-VF |
Vaccine designed to stimulate the immune system to target and destroy cells expressing CEA and mucin-1 |
Pancreatic cancer |
Phase I data suggest the treatment was well tolerated and resulted in prolonged overall survival (ASCO) |
Transgene SA |
MVA-MUC1-IL2 |
Recombinant MVA virus containing sequences encoding for the human MUC1 antigen and the cytokine IL-2 |
Prostate cancer and non- small-cell lung cancer |
Phase II data showed that in 63% of patients MVA-MUC1-IL2 induced a significant increase in the time it took for PSA to double; a tumor response rate of 37% was seen in a 44-patient combination NSCLC trial (ASCO) |
Vical Inc. |
Allovectin-7 |
DNA plasmid/lipid complex containing the DNA sequences encoding HLA-7 and ß2 microglobulin |
Metastatic melanoma |
Phase II data showed Allovectin-7 appeared to benefit from having at least two cycles of treatment (ASCO) |
Vion |
Cloretazine (VNP40101M) |
Sulfonylhydrazine DNA alkylating agent |
High-risk acute myelogenous leukemia and high-risk myelo-dysplasia |
Cloretazine showed significant single-agent activity in previously untreated elderly patients (ASCO) |
Xanthus Life |
Xanafide |
A new salt formulation of the cancer compound amonafide |
Acute myeloid leukemia |
Phase I trial with cytosine arabinoside showed 38% of 26 patients went into complete remission (ASCO) |
Xcyte Therapies |
Xcellerated T-Cells |
T cells from patient stimulated to carry out immune functions |
Chronic lymphocytic leukemia |
Discontinued development of product in CLL after inability to reach agreement with FDA on an acceptable pathway for approval (5/16) |
YM |
Norelin |
Vaccine targeting the gonadotropin-releasing hormone |
Hormone- dependent prostate cancer |
The majority of patients developed antibodies to GnRH in a proof-of-concept study (5/25) |
YM |
Tesmilifene(DPEE) |
Small molecule designed to augment the activity of chemotherapy agents |
Metastatic breast cancer |
Updated data from Phase III trial showed a 143% overall survival improvement in the 191 patients whose cancer spread or recurred within 36 months of diagnosis (ASCO) |
ZioPharm Inc.* |
ZIO-101 |
Small-molecule organic arsenic |
Solid tumors |
Began a Phase I trial to assess safety and dosing in up to 40 patients with solid tumors (5/19) |
ZymoGenetics |
-- |
Recombinant human interleukin-21 |
Cancer |
Interleukin-21 showed evidence of antitumor activity and was reasonably well tolerated in a Phase I trial (ASCO) |
CARDIOVASCULAR | ||||
Alteon Inc. |
Alagebrium |
A.G.E. crosslink breaker |
Various conditions |
Data from the initial group in a Phase IIa trial demonstrated increased arterial elasticity and improved endothelial function (5/23) |
Arginox |
Tilarginine Acetate Injection |
Agent designed to inhibit the production of nitric oxide |
Cardiogenic shock |
Began Phase III TRIUMPH trial to test effectiveness on improved survival in heart attack patients who develop CS (6/23) |
CoTherix Inc. |
Ventavis (FDA-approved) |
Inhaled formulation of iloprost, a prostacyclin analogue |
Pulmonary arterial hypertension |
Follow-up data from 52 patients in a Phase III trial demonstrated long-term safety and efficacy in NYHA Class III and IV patients (5/23) |
Encysive |
Thelin |
Sitaxsentan; small molecule designed to block endothelin |
Pulmonary arterial hypertension |
Phase III STRIDE-2 trial met the primary endpoint of improved six- minute walk distance; also, six poster presentations on the drug were made (5/22 and 5/23) |
Myogen Inc. |
Ambrisentan |
Type-A selective |
Pulmonary |
Presented additional and follow- up data from various trials that showed positive safety and efficacy results (5/23) |
Myogen Inc.(MYOG) |
Ambrisentan |
Type-A selective endothelin receptor antagonist |
Pulmonary arterial hypertension |
Began trial to assess safety, tolerability and efficacy in 30 patients who discontinued bosentan or sitaxsentan therapy due to liver function test abnormalities (5/19) |
Nuvelo Inc. |
rNAPc2 |
Recombinant nematode anticoagulant protein c2 originally isolated from hookworms |
Acute coronary syndrome |
Phase IIa ANTHEM trial showed an acceptable safety and tolerability profiles in patients with non-ST- elevation ACS (5/24) |
Predix |
PRX-08066 |
Selective 5-HT2B receptor antagonist |
Pulmonary hypertension |
Began a Phase I trial to evaluate safety, tolerability and pharmaco-kinetics (5/30) |
Renovis Inc. |
Cerovive (NXY-059) |
A neuroprotectant with free radical-trapping properties |
Stroke |
First analysis of Phase III SAINT I trial involving more than 1,700 patients showed a statistically significant reduction vs. placebo on disability; partner AstraZeneca plc ran the trial (5/4) |
CENTRAL NERVOUS SYSTEM | ||||
Alkermes Inc. |
Vivitrex |
Long-acting, injectable form of naltrexone |
Alcohol dependence |
12-month extension of Phase III trial showed drug and counseling led to a sustained reduction in heavy drinking over an 18-month period (5/23) |
Cephalon Inc. |
CEP-1347 |
Small-molecule inhibitor of stress-activated protein kinase pathway |
Parkinson's disease |
Phase II/III trial was stopped after monitors concluded the data were unlikely to provide evidence of significant effect (5/11) |
Cephalon Inc. |
OraVescent Fentanyl |
Fentanyl formulated with transmucosal delivery technology |
Breakthrough cancer pain |
Phase III trial in 123 patients showed a statistically significant benefit; an NDA filing is planned for 3Q:05 (5/5) |
Helicon |
HT-0712 (IPL455,903) |
Oral inhibitor of phosphodiesterase-4 |
Learning and memory disorders |
Phase I trial showed the drug was safe and well tolerated at all doses tested (5/11) |
Metaphore |
M40403 |
Superoxide dismutase mimetic |
Post-surgical pain |
Began a Phase II trial with morphine to compare analgesia to morphine alone in 240 bunionectomy patients (5/2) |
Neurocrine |
Indiplon |
Capsule form of a non-benzodiazepine agent that targets the GABA-A receptor |
Insomnia |
Phase III trial in 702 patients showed significant and sustained improvement in sleep onset and sleep maintenance (5/25) |
NeurogesX |
NGX-4010 |
High-concentration trans-capsaicin dermal patch |
HIV-associated sensory neuropathy |
Phase III trial in 307 patients demonstrated statistically signifi- cant pain reduction over 12 weeks (5/18) |
New River |
NRP104 |
Prodrug of amphetamine |
Attention deficit hyperactivity disorder |
Each dose in a Phase III trial in 285 children demonstrated efficacy vs. placebo on both primary and secondary endpoints(5/23) |
Praecis |
Apan |
Agent that may mobilize beta- myloid and facilitate its clearance |
Alzheimer's disease |
Company discontinued development of the early stage compound as part of a cost-cutting move (5/20) |
Sepracor |
Lunesta (FDA-approved) |
Eszopiclone tablets; a non-benzodiazepine agent |
Insomnia |
Positive data were reported from Phase III/IV trial in patients with major depressive disorder and insomnia, and from trials in the elderly and adults (5/26) |
DIABETES | ||||
Coremed Inc.* |
Intesulin |
Oral insulin |
Diabetes |
Initial trial of single gel capsule in patients demonstrated safety (5/9) |
Keryx Bio- |
KRX-101 |
Sulodexidel; oral heparinoid compound |
Diabetic nephropathy |
Interim Phase II data in 149 patients showed better success in drug group than placebo (5/6) |
INFECTION | ||||
Acambis plc |
ChimeriVax-West Nile |
Vaccine that uses a live weakened yellow fever virus to create chimeric viruses |
West Nile virus |
Phase I trial in 80 subjects demonstrated development of neutralizing antibodies and high antibody titers (5/11) |
Coley |
Actilon (CPG 10101) |
Agent designed to target and stimulate Toll-like receptor 9 |
Hepatitis C |
Phase Ib trial in 42 nonresponders showed good safety and tolerability, as well as antiviral activity (5/17) |
Hemispherx |
Alferon LDO |
Low-dose, oral alpha- interferon cocktail |
Avian flu and other viral diseases |
Began trials in volunteers to determine whether new delivery format can resuscitate antiviral and immunostimulatory genes (5/16) |
Idenix |
Telbivudine |
Selective, oral nucleoside agent |
Hepatitis B |
Data from an extended-treatment Phase IIb trial demonstrated continuing advantages vs. lamivudine across efficacy endpoints through two years of treatment (5/16) |
Immtech |
DB289 |
Oral, dicationic antiviral agent |
Infectious diseases |
Safety trial comparing three different formulations of drug led to selection of tablet formulation for use in pivotal Phase III trials (5/4) |
Inhibitex Inc. |
Aurexis |
Humanized monoclonal antibody based on MSCRAMM protein platform |
S. aureus infections |
Phase II trial with antibiotic therapy in 60 patients demonstrated positive results in primary and secondary endpoints (5/9) |
InterMune |
Infergen (FDA-approved) |
Interferon alfacon-1 |
Hepatitis C |
Presented positive data from a trial with ribavirin and from a retrospective analysis evaluating drug and Actimmune in PEG-non- responders (5/16) |
MedImmune |
Numax |
Third-generation variant of the anti-RSV antibody Synagis |
Respiratory syncytial virus |
Phase I/II trial in 217 high-risk infants demonstrated safety and tolerability, with an acceptable pharmacokinetic profile(5/14) |
MedImmune |
FluMist (FDA- approved) and CAIV-T |
Intranasal influenza vaccine and next-generation, trivalent vaccine |
Influenza |
Presented data from various studies of both products that showed broad protection against the flu (5/14) |
Nabi Bio- |
-- |
Vaccine designed to prevent Staphylococcus epidermidis infections |
S. epidermidis infections |
Began a Phase l trial to evaluate safety and immune responses in healthy volunteers (5/25) |
Nabi Bio- |
StaphVAX |
Staphylococcus aureus polysaccharide conjugate vaccine |
To prevent S. aureus infections |
Began bridging and consistency studies in healthy adults to compare three commercial lots of the vaccine (5/18) |
Peregrine |
Tarvacin |
Chimeric monoclonal antibody that binds to the phospholipid phosphatidylserine |
Hepatitis C |
Began a Phase I trial to evaluate safety, pharmacokinetics and viral load in up to 32 relapsed or nonresponsive patients (5/31) |
Samaritan |
SP01A |
Oral entry inhibitor agent |
HIV |
Began Phase II trial in 92 patients that will assess reductions in viral load (5/11) |
Vertex |
VX-950 |
Oral protease inhibitor |
Hepatitis C |
Phase Ib trial in 34 patients demonstrated tolerability and potent antiviral activity (5/10 and 5/17) |
ViroPharma |
HCV-796 |
Polymerase inhibitor |
Hepatitis C |
Began Phase Ib trial to assess antiviral activity, safety and pharmacokinetics in 96 treatment-naïve patients (5/24) |
MISCELLANEOUS | ||||
Acologix Inc.* |
AC-100 (Dentonin) |
Synthetic peptide derived from a protein produced by bone and dental cells |
For use in dental procedures |
Began a Phase I trial of the product (5/5) |
Acuity |
Cand5 |
Small interfering RNA therapeutic designed to shut down VEGF |
Age-related macular degeneration |
Interim safety data from Phase I trial were positive, with no drug- related adverse events (5/3) |
Arena |
APD356 |
Small-molecule, selective 5HT2C serotonin receptor agonist |
Obesity |
Phase II trial in 352 obese people showed a statistically significant average weight loss of 2.9 pounds vs. 0.7 pounds in the placebo group (5/11) |
Ascend |
4-OHT |
4-hydroxytamoxifen gel |
Cyclic |
Phase II trial demonstrated improved signs and symptoms as well as reduced breast pain (5/11) |
Bone Care |
LR-103 |
Vitamin D hormone |
Secondary hyperparathyroidism |
Starting a Phase I/II trial in patients with Stage V chronic kidney disease or end-stage renal disease (5/5) |
Carrington |
GelVac |
Powder intranasal delivery system for vaccines |
Vaccine delivery |
Phase I trial demonstrated safety and tolerability, and delivery to the nasal cavity (5/19) |
Corgentech |
E2F Decoy (edifoligide) |
Oligonucleotide designed to inactivate the cell-cycle transcription factor E2F |
Atopic dermatitis (eczema) |
Began a Phase I/II trial to evaluate safety and the feasibility of repeated skin applications (5/24) |
Depomed |
Furosemide GR |
Controlled-release formulation of the diuretic furosemide |
Edema |
Extended data from Phase II trial showed variable urinary urgency and frequency; development in the indication was stopped (5/13) |
Discovery |
Surfaxin |
Engineered version of natural human lung surfactant |
Respiratory distress syndrome in premature babies |
Long-term data from Phase III trials showed survival benefits continued through one year of life (5/18) |
Eyetech |
Macugen |
Pegaptanib sodium injection; pegylated anti-VEGF aptamer |
Diabetic macular edema |
Retrospective analysis of patients showed improvements in various measures of diabetic retinopathy (5/5) |
Eyetech |
Macugen (FDA-approved) |
Pegaptanib sodium injection; pegylated anti-VEGF aptamer |
Wet age-related macular degeneration |
No new safety concerns were identified in the second year of a trial (5/2) |
Genaera |
Evizon (squalamine) |
Anti-angiogenesis agent; vascular endothelial growth factor inhibitor |
Wet age-related macular degeneration (5/3) |
Phase II trial with photodynamic therapy in 46 subjects showed safety and improved visual acuity |
Genaera |
Evizon (squalamine) |
Anti-angiogenesis agent; vascular endothelial growth factor inhibitor |
Wet age- related macular degeneration |
Data from 18 patients in a Phase II trial showed improved visual acuity (5/2) |
Genentech |
Lucentis |
Ranibizumab; humanized antibody fragment designed to bind and inhibit VEGF-A |
Wet age- related macular degeneration |
Phase III trial met its primary endpoint; 95% of patients maintained or improved vision at one year vs. 62% for placebo (5/23) |
ICOS Corp. |
Cialis (FDA-approved) |
Tadalafil; oral PDE5 inhibitor |
Erectile dysfunction |
Data from open-label trial showed drug improved erections in more than 75% of men with ED and one or more co-morbid conditions (5/23) |
InterMune |
Actimmune (FDA-approved) |
Interferon gamma-1b |
Idiopathic pulmonary fibrosis |
Data from 70 patients on the drug an average of 2.5 years showed relatively stable lung function (5/23) |
Isotechnika |
ISA247 |
Immunosuppressive agent |
For use in kidney transplants |
Starting a Phase IIb trial that will compare drug to tacrolimus in 332 newly transplanted kidney patients (5/12) |
MediciNova |
MN-001 |
Leukotriene receptor antagonist and inhibitor of of phosphodiesterases III and IV, 5-lipoxygenase and thromboxane A2 |
Interstitial cystitis |
Began Phase II trial; details were not disclosed (5/24) |
Microbia Inc.* |
MD-1100 |
Superagonist of the guanylate cyclase-C receptor |
Irritable bowel syndrome |
Phase Ia trial in 30 healthy volunteers showed drug was safe and well tolerated, with no detectable systemic absorption (5/17) |
Nabi Bio- |
PhosLo (FDA-approved) |
Calcium acetate; phosphate binder |
Stage IV kidney disease |
Began EPICK study in 86 patients to evaluate efficacy in controlling elevated phosphorus levels (5/10) |
Neurotech SA* |
NT-501 |
Encapsulated cell technology used to deliver ciliary neurotrophic factor |
Retinitis pigmentosa |
Phase I trial demonstrated safety and tolerability, and showed improvements in visual acuity (5/1) |
Regeneron |
VEGF Trap |
Intravenous product designed to block vascular endothelial growth factor |
Age-related macular degeneration |
Phase I trial in 25 patients met its prespecified efficacy endpoint (5/3) |
Sangamo |
SB-509 |
Injectable formulation of plasmid DNA that encodes a zinc finger DNA-binding protein transcription factor designed to up-regulate the VEGF-A gene |
Peripheral neuropathy |
Began a Phase I trial to evaluate safety in diabetics with mild to moderate diabetic peripheral sensory motor neuropathy in the legs (5/5) |
Sirna |
Sirna-027 |
Chemically modified short interfering RNA targeting vascular endothelial growth factor receptor-1 |
Age-related macular degeneration |
Interim data from Phase I trial showed the agent to be safe and well tolerated at all doses tested (5/4) |
Vivus Inc. |
MUSE (FDA-approved) |
Alprostadil urethral suppository |
Erectile dysfunction |
Study in 91 patients who developed ED due to a bilateral nerve-sparing radical prostatectomy showed improved erections vs. placebo (5/23) |
Vivus Inc. |
Avanafil |
Oral phosphodiesterase type-5 inhibitor |
Erectile dysfunction |
Open-label trial in 13 subjects showed no accumulation of drug in plasma following twice-daily dosing (5/4) |
| | ||||
Notes: | ||||
* Privately held. | ||||
ASCO = Data were presented at the American Society of Clinical Oncology meeting May 14-17. | ||||
BLA = Biologics license application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; SPA = Special protocol assessment. | ||||
Unless otherwise noted, the stock symbols listed for public companies are on the Nasdaq market. | ||||
AMEX = American Stock Exchange; CSE = Copenhagen Stock Exchange; LSE = London Stock Exchange; NYSE = New York Stock Exchange; OSE = Osaka Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; TSE = Toronto Stock Exchange. | ||||
