Company* |
Product | Description | Indication |
Status |
AUTOIMMUNE | ||||
Alba |
AT1001 |
Oral zonulin receptor antagonist |
Celiac disease |
Phase Ib trial in 21 patients demonstrated positive responses vs. placebo (3/13) |
Biogen Idec |
Tysabri |
Natalizumab; humanized monoclonal antibody that inhibits adhesion molecules on immune cells |
Multiple sclerosis |
Began monotherapy safety extension trial in patients who participated in Phase III MS trials and subsequent safety evaluation (3/29) |
Celgene |
CC-10004 |
Small molecule designed to inhibit production of multiple pro-inflammatory mediators |
Severe plaque psoriasis |
Open-label Phase II trial in 19 patients produced encouraging results (3/6) |
Centocor Inc. |
Remicade (FDA-approved) |
Infliximab; monoclonal antibody that targets tumor necrosis factor-alpha |
Severe plaque psoriasis |
Phase III trial showed rapid, significant improvements and long-term responses (3/3) |
Genmab A/S |
HuMax-CD20 |
Human antibody that binds to the CD20 antigen |
Rheumatoid arthritis |
73% of patients who received two doses of drug in Phase I/II trial achieved ACR20, vs. none for placebo (3/8); additional data from the 26 patients showed 38% achieved ACR50 and 15% ACR70 (3/22) |
Neurocrine |
APL-MS |
Altered peptide ligand technology |
Multiple sclerosis |
Phase II trial in 157 patients did not demonstrate efficacy; the APL-MS program was discontinued (3/8) |
PDL |
Visilizumab |
Monoclonal antibody designed to target and block the action of T cells |
Severe ulcerative colitis |
Began Phase II/III RESTORE 1 trial to evaluate safety and efficacy in intravenous steroid- refractory UC (3/27) |
Targeted |
tgAAC94 |
Product using an adeno- associated viral vector to deliver a soluble TNF- alpha receptor protein |
Inflammatory arthritis |
Amended Phase I trial to include a higher dose and increase the number of patients from 40 to 120; it now is a Phase I/II trial (3/16) |
Vertex |
VX-702 |
Oral, p38 MAP kinase inhibitor |
Rheumatoid arthritis |
Phase II trial in 315 patients showed tolerability, and demonstrated statistically significant effects on signs and symptoms of RA (3/8) |
CANCER | ||||
AmpliMed |
Amplimexon |
Imexon injection; a cyanoaziridine compound believed to disrupt mitochondria |
Advancing multiple myeloma |
Began Phase Ib trial to evaluate drug as stand-alone therapy in patients whose disease is progressing despite prior treatments (3/28) |
Antigenics |
Oncophage |
Personalized cancer vaccine based on heat- shock protein technology |
Kidney cancer |
Top-line data from Phase III trial showed a trend in favor of drug in recurrence-free survival and a trend against it for overall survival; neither was statistically significant (3/24) |
Arrowhead |
IT-101 |
Nanotechnology-based agent incoporating the cancer drug camptothecin |
Cancers |
Subsidiary Insert Therapeutics Inc. began Phase I trial to evaluate safety and tolerability in 24 to 48 patients (3/14) |
BiPar Sciences |
BSI-201 |
Inhibitor of poly-adenyl- ribose polymerase |
Solid tumors |
Began Phase I trial to evaluate safety, pharmacokinetics and activity in patients (3/7) |
CancerVax |
Canvaxin |
Therapeutic whole-cell vaccine that expresses more than 20 antigens |
Advanced melanoma |
Data from discontinued Phase III trial in Stage IV disease showed median survival of 31.5 months vs. 38.7 months for placebo; trial in Stage III disease showed median overall survival for both groups of more than 69 months (3/24) |
Cell |
Pixantrone |
Anthracycline agent designed to have improved properties |
Advanced non- Hodgkin's lymphoma |
Phase I/II trial showed an overall response rate of 71% when pixantrone replaced doxorubicin in the standard CHOP regimen (3/22) |
CuraGen Corp. |
PXD101 |
Small-molecule histone deacetylase inhibitor |
Advanced malignancies |
Began Phase I trial with Velcade to evaluate the drug in up to 36 patients with advanced solid tumors and lymphomas (3/29) |
CuraGen Corp. |
PXD101 |
Small-molecule histone deacetylase inhibitor |
Advanced multiple myeloma |
Began Phase Ib/II trial to evaluate the drug with Velcade in patients who have failed at least two prior lines of therapy (3/22) |
Cytokinetics |
Ispinesib (SB-715992) |
Small-molecule inhibitor of kinesin spindle protein |
Non-small-cell lung cancer |
Phase II trial failed to satisfy the criteria for advancement to the next stage of development (3/30) |
GenVec Inc. |
TNFerade |
Adenovector with TNF- alpha gene and a radiation- responsive promoter |
Metastatic melanoma |
Began Phase II trial with radiation that will evaluate responses in 29 patients with Stage III or IV disease (3/29) |
GenVec Inc. |
TNFerade |
Adenovector with TNF- alpha gene and a radiation- responsive promoter |
Advanced pancreatic cancer |
Expanded Phase II trial into Phase II/III trial with chemotherapy and radiation to evaluate 12-month survival in 330 patients (3/15) |
Gloucester |
Depsipeptide (FK228) |
Histone deacetylase inhibitor |
Metastatic hormone- refractory prostate cancer |
Interim Phase II data showed encouraging activity and tolerability (3/1) |
Hana |
Talotrexin (PT-523) |
Non-classical antifolate; analogue of aminopterin |
Advanced non- small-cell lung cancer |
Began Phase II trial to evaluate survival and other endpoints in relapsed or refractory patients (3/31) |
Hana |
Zensana |
Ondansetron oral spray; 5-HT3 antagonist |
Chemotherapy- induced nausea and vomiting |
Pivotal studies confirmed bioequivalence, and quicker delivery, vs. commercially available ondansetron, Zofran tablets (3/9) |
Introgen |
Advexin |
Adenoviral vector containing the p53 tumor-suppressor gene |
Advanced esophageal cancer |
Phase I/II trial demonstrated safety and feasibility; 9 of 10 patients had local tumor responses of stable disease (3/16) |
Keryx Bio- |
KRX-0401 (perifosine) |
Oral agent that modulates AKT and other signal transduction pathways |
Leukemia |
Began Phase II trial to evaluate responses and other endpoints of drug used as a single agent in relapsed or refractory acute leukemias (3/2) |
Medarex Inc. |
MDX-010 |
Ipilimumab; fully human antibody against CTLA-4, a molecule on T cells |
Metastatic melanoma |
Began pivotal trial to evaluate use as a monotherapy in 150 patients with unresectable Stage III or IV disease who have progressed despite prior therapy (3/31) |
MGI Pharma |
Gliadel Wafer (FDA-approved) |
Wafer comprised of a biodegradable polymer incorporating carmustine |
High-grade malignant glioma |
Follow-up data showed patients treated with drug and radiation therapy had a significant survival advantage at three years vs. placebo (3/8) |
Millennium |
Velcade (FDA-approved) |
Bortezomib; proteasome inhibitor |
Advanced non- small-cell lung cancer |
Began Phase II trial with pemetrexed that will evaluate responses in 135 patients (3/6) |
RxKinetix Inc.* |
RK-0202 |
The antioxidant N- |
Oral mucositis in cancer patients |
Phase II trial demonstrated reduced oral mucositis vs. placebo, and improvements in a number of other symptoms (3/1) |
Sunesis |
SNS-595 |
Small-molecule cell-cycle modulator designed to induce apoptosis |
Small-cell lung cancer |
Began Phase II trial to examine safety and efficacy of the drug in patients who have failed first- line therapy (3/20) |
SuperGen |
Nipent (FDA-approved) |
Pentostatin for injection |
Chronic lymphocytic leukemia |
Updated data from trial demonstrated that combination therapy with pentostatin, cyclophos- phamide and rituximab is an active regimen (3/14) |
YM |
TheraCIM |
Nimotuzumab; anti- EGFr monoclonal antibody |
Non-small-cell lung cancer |
Began Phase I/II trial to evaluate the drug with radiation in patients with Stage IIB and III disease (3/23) |
CARDIOVASCULAR | ||||
Amgen Inc. |
Aranesp (FDA-approved) |
Darbepoetin alfa, a recombinant erythropoietic protein |
Heart failure |
Phase II trial showed drug was well tolerated, raised hemoglobin and improved symptoms; began Phase III RED-HF trial to evaluate the effect on morbidity and mortality in patients with symptomatic heart failure (3/13) |
Corautus |
VEGF-2 |
Vascular endothelial growth factor-2 in the form of naked plasmid DNA |
Severe angina |
Suspended treatment in Phase IIb GENASIS trial after seeing three cases of pericardial effusion; 295 patients have been treated (3/14); the FDA placed a hold on the trial (3/20) |
CoTherix Inc. |
Ventavis (FDA-approved) |
Inhaled formulation of iloprost, a prostacyclin analogue |
Pulmonary arterial hypertension |
Began Phase III VISION trial to evaluate safety and efficacy in combination with sildenafil in 180 patients (3/8) |
Critical |
CTI-01 |
Ethyl pyruvate; small molecule believed to inhibit release of cytokines |
Complications of cardiac surgery |
Discontinued Phase II trial due to stability issues that could affect the integrity of drug supplies; 102 of a planned 150 patients had been treated (3/15) |
Emisphere |
-- |
Oral heparin formulation using eligen technology |
Anticoagulant |
Trial demonstrated the oral heparin was chemically identical to heparin delivered by injection (3/31) |
Encysive |
TBC3711 |
Next-generation, selective endothelin receptor antagonist |
Hypertension |
Placed trials on hold due to a finding from an animal study; initial trials were just getting under way (3/23) |
Gentium SpA |
Defibrotide |
Single-stranded DNA designed to protect vascular endothelial cells |
Veno-occlusive disease |
Began Phase III trial to evaluate survival in 80 patients with VOD with multiple organ failure as a complication of stem cell transplantation (3/28) |
Isis |
ISIS 301012 |
Oral formulation of second-generation antisense inhibitor of ApoB-100 |
Cholesterol management |
Additional data from trials showed agent did not interact with lipid-lowering drugs simvastatin and ezetimibe (3/13) |
Neurobiological |
Viprinex (ancrod) |
Thrombin-like enzyme |
Acute |
Began second Phase III trial, ASP-II; together they will enroll 1,300 patients; it will evaluate patient function 90 days post-stroke and other endpoints (3/22) |
NitroMed Inc. |
BiDil |
Nitric oxide-enhancing oral agent; combination of isosorbide dinitrate and hydralazine |
Heart failure in African-Americans |
Additional data from A-HeFT trial showed decreased systolic blood pressure in those with higher baseline SBP but not in those with lower baseline SBP (3/13); post-approval analysis of A-HeFT trial showed similar positive outcomes for both men and women (3/14) |
PR |
PulmoLAR |
2-methoxyestradiol (2ME), an endogenous non- estrogenic metabolite of estradiol |
Pulmonary arterial hypertension |
Began Phase I trial to evaluate safety, tolerability and pharmaco- kinetics in healthy volunteers (3/29) |
Surface |
SLx-4090 |
Microsomal triglyceride transfer protein inhibitor |
Dyslipidemia |
Began Phase I trial to evaluate safety, tolerability, pharmacoki- netics and preliminary efficacy in healthy male subjects (3/14) |
Synthetic Blood |
Oxycyte |
Perfluorocarbon-based blood substitute and therapeutic oxygen carrier |
Blood substitute |
Began Phase II proof-of-concept trial to evaluate safety and bio- logical effects in 8 patients with traumatic brain injury (3/16) |
The Medicines |
Angiomax |
Bivalirudin; direct thrombin inhibitor |
Anticoagulant |
ACUITY trial in 13,800 patients showed improved outcomes when heparin was replaced with Angiomax (3/12) |
Titan |
DITPA |
Analogue of thyroid hormone (T3) |
Elevated cholesterol |
Began Phase II trial to evaluate product in combination with statin therapy (3/7) |
Vasogen Inc. |
Celacade |
Immune-modulation therapy |
Peripheral arterial disease |
Phase III SIMPADICO trial in 553 patients did not meet primary endpoint in treadmill walking distance; the trial did show reduced high-sensitivity C- reactive protein (3/12) |
CENTRAL NERVOUS SYSTEM | ||||
Acadia |
ACP-103 |
Selective 5-HT2A receptor inverse agonist |
Parkinson's disease |
Phase II trial in PD patients suffering from treatment- induced psychosis met primary endpoint of not worsening motor function; secondary efficacy endpoints also were met (3/23) |
Avicena |
PD-02 |
Agent that incorporates ultra-pure form of creatine |
Parkinson's disease |
Phase II trial in 200 early PD paitents showed that both PD-02 and the antibiotic minocycline warrant further study (3/14) |
Clinical Data |
Vilazodone |
Selective serotonin reuptake inhibitor and a 5HT1A partial agonist |
Depression |
Began pivotal Phase III trial to assess safety and efficacy in 400 adult patients with major depressive disorder (3/30) |
Corgentech |
ALGRX 4975 |
Non-opioid, VR1 agonist that acts as a C-neuron anesthetic |
Pain |
Phase II trial showed drug demonstrated sustained, statistically significant pain relief vs. placebo in elbow tendinitis patients (3/9) |
Cortex |
CX717 |
Ampakine analogue; designed to improve brain cell signaling |
Attention deficit hyperactivity disorder |
Phase IIa trial in 49 adult patients showed a positive trend in the higher dose group (3/6) |
Humanetics |
NIC5-15 |
Agent designed to prevent or slow the progression of beta amyloid plaques |
Alzheimer's disease |
Began human trials that will test the compound in early stage AD patients (3/30) |
MGI Pharma |
Aquavan |
Water-soluble prodrug of propofol |
Sedation |
Began pivotal program consisting of two Phase III trials and an open-label safety study; one Phase III trial will include 300 colonoscopy patients; one will enroll 250 bronchoscopy patients (3/20) |
Myriad |
Flurizan |
Selective amyloid beta-42 lowering agent |
Alzheimer's disease |
Additional data from Phase II trials showed continued benefit through 21 months (3/13) |
Renovis Inc. |
NXY-059 |
Neuroprotectant with free radical-trapping properties |
Intracerebral hemorrhage |
Phase IIb CHANT trial showed safety and tolerability similar to placebo, with comparable mortality rates and no difference on stroke outcomes (3/14) |
Repligen |
RG2417 |
Oral formulation of |
Bipolar |
Began Phase II trial to evaluate safety and effectiveness vs. placebo in 80 patients (3/9) |
Theratech- |
TH9507 |
Stabilized analogue of growth hormone- releasing factor |
Mild cognitive impairment |
Began SMART trial to evaluate the drug on cognitive function in healthy older adults (3/28) |
XenoPort Inc. |
XP13512 |
Transported prodrug of gabapentin |
Restless legs syndrome |
Began Phase III trial (Study XP052) to evaluate safety and efficacy in 200 patients (3/14) |
DIABETES | ||||
Amylin |
Byetta (FDA-approved) |
Long-acting form of the approved agent Byetta; exenatide; incretin mimetic |
Type II diabetes |
Began long-term comparator trial comparing safety and efficacy to twice-daily Byetta (3/24) |
MannKind |
Technosphere Insulin |
Dry powder formulation of insulin delivered by company's inhaler |
Types I and II diabetes |
Began two pivotal Phase III trials; Study 009 will evaluate efficacy with a basal insulin regimen in 500 Type I patients; Study 102 in Type II patients will evaluate the drug in 500 patients under an FDA SPA (3/6) |
INFECTION | ||||
AnorMED Inc. |
AMD070 |
Agent targeting CXCR4 chemokine receptor; HIV entry inhibitor |
HIV |
Preliminary data from open-label XACT trial showed drug was active, generally safe and well tol- erated in patients (3/17) |
Arpida Ltd. |
Iclaprim |
Capsule formulation of broad-spectrum antibiotic |
Skin and skin structure infections |
Monitors in Phase III ASSIST-1 trial recommended continuation after reviewing safety data from 40% of the patients to be enrolled (3/21) |
BioBalance |
Probactrix |
Single strain of non- pathogenic E. coli M-17 |
Pouchitis |
Began a Phase I/II trial to evaluate the product in 63 antibiotic- dependent patients (3/27) |
BioCryst |
Peramivir |
Influenza neuraminidase inhibitor |
Influenza |
Began Phase I to evaluate phar- macokinetics and safety of intra- venous formulation in healthy volunteers (3/9) |
Coley |
Actilon (CPG 10101) |
Agent designed to target and stimulate Toll-like receptor 9 |
Hepatitis C |
Interim data in relapsed patients showed benefit of using drug with pegylated interferon and ribavirin vs. those drugs alone (3/21) |
Elusys |
Anthim |
Monoclonal antibody that targets the protective antigen component of anthrax |
Anthrax infection |
Phase I trial in healthy volunteers demonstrated safety and tolera- bility, both alone and with cipro- floxacin (3/29) |
Enzo |
HGTV43 |
Gene contruct; in this trial it will be used to transfer three antisense genes |
HIV |
Phase I trial demonstrated safety in five subjects and the ability of the engineered cells to continue to function in vivo; another trial was approved and close to getting under way (3/1) |
Human Genome |
Albuferon |
Long-acting form of interferon alpha that uses albumin fusion technology |
Hepatitis C |
Interim data from Phase II trials with ribavirin demonstrated safety, tolerability and antiviral activity (3/14) |
Idenix |
NM283 |
Valopicitabine; oral nucleoside analogue |
Hepatitis C |
Modified ongoing Phase IIb trial by reducing dosing level, due to dose-related gastrointestinal side effects (3/23) |
Immtech |
DB289 |
Pafuramidine maleate; oral, dicationic antiviral agent |
Pneumocystis pneumonia |
Began pivotal Phase III trial under FDA SPA to evaluate the drug against TMP-SMX in 270 HIV/AIDS patients with PCP (3/27) |
Metabasis |
Pradefovir |
Prodrug of the approved HBV drug adefovir (Gilead Sciences Inc.'s Hepsera) |
Hepatitis B |
Partner Valeant Pharmaceuticals Inc. presented 48-week Phase II data showing a significant decline in viral load. vs. Hepsera (3/16) |
Nabi Bio- |
Gram- positive programs |
Products including StaphVAX and Altastaph |
Infections |
Said it will continue development of products despite Phase III failure of StaphVAX in November; a panel said there were differences in the product vs. that used in previous Phase III trial (3/21) |
PowderMed |
pdpSC18 |
Dual-antigen encoding immunotherapeutic |
Hepatitis B |
Is starting a Phase I trial in the U.S. and Southeast Asia to evaluate safety and tolerability, with its needle-free device (3/29) |
Samaritan |
SP01A |
Oral entry inhibitor |
HIV |
Began second Phase II monotherapy trial to evaluate drug in treatment-experienced patients who are failing current therapy (3/9) |
Stem Cells |
HuCNS-SC |
Human neural stem cell formulation isolated from the fetal brain |
Batten disease |
Is starting a Phase I trial to evaluate safety and preliminary efficacy in patients (3/9) |
ViroPharma |
Maribavir |
Benzimidazole compound; oral inhibitor of cytomegalovirus |
Prevention of CMV infection in transplant patients |
Phase II trial in 111 patients who had allogeneic stem cell transplantations demonstrated a statistically significant reduction in CMV reactivation (3/29) |
VirXsys Corp.* |
VRX496 |
T cells are removed from patient, treated with an HIV lentiviral vector, then reintroduced to patient |
HIV |
Each of three patients in safety study reached three-month post- infusion visit with no serious adverse events (3/17) |
MISCELLANEOUS | ||||
Amarillo |
-- |
Low-dose, oral interferon alpha |
Idiopathic pulmonary fibrosis |
Data from ongoing trial in 20 patients demonstrated tolerability and a reduction in cough (3/6) |
Aradigm |
-- |
Aerosolized formulation of hydroxychloroquine |
Asthma |
Began Phase II trial to evaluate safety and efficacy vs. aerosolized placebo in 100 patients (3/23) |
BioMarin |
Phenoptin |
Sapropterin hydrochloride; form of the enzyme co- factor 6R-BH4 |
Phenylketonuria |
Phase III trial in 89 patients met primary and secondary endpoints, showing a statistically significant reduction in blood phenylalanine levels (3/15) |
BioMimetic |
GEM OS1 Bone Graft |
Recombinant platelet- derived growth factor combined with a synthetic bone matrix |
Foot and ankle fusions; unstable distal radius fractures |
Began separate studies to evaluate the product in foot and ankle fusion procedures and in treating distal radius fractures (3/20) |
BioSante |
Bio-E-Gel |
Estradiol transdermal gel |
Hot flashes |
Phase III trial in 484 symptomatic menopausal women demonstrated statistical significance vs. placebo (3/10) |
Corgentech |
Avrina |
NF-kappaB decoy; inhibitor of the NF-B transcription factor |
Atopic dermatitis (eczema) |
Phase I/II trial demonstrated safety and tolerability and positive data from the lowest dose (3/6) |
Discovery |
Surfaxin |
Engineered version of natural human lung surfactant |
Acute respiratory distress syndrome |
Phase II trial in 124 patients demonstrated improved oxygenation, through an increase in the P/F ratio (3/30) |
Dyax Corp. |
DX-88 |
Recombinant small protein designed to inhibit human plasma kallikrein |
Hereditary angioedema |
Phase II EDEMA2 trial showed drug was well tolerated and successful in treating all types of attacks (3/6) |
Dynavax |
Tolamba |
Dynavax's ISS molecule linked to Amb a 1, the major allergen of ragweed |
Ragweed allergy |
Additional data from Phase II/III trial showed statistically significant reductions in nasal symptoms vs. placebo in both the first and second years of trial (3/6) |
Dynogen |
DDP225 |
Noradrenaline reuptake inhibitor and a serotonin type 3 receptor antagonist |
Chronic functional vomiting |
Began Phase II trial that will evaluate multiple symptom-based end-points (3/14) |
Exelixis Inc. |
XL784 |
Small-molecule inhibitor of the ADAM-10 metallo-protease enzyme |
Proteinuria |
Began Phase II trial in 130 patients with diabetes; the primary end-point is a reduction in proteinuria, a marker for renal damage (3/27) |
Genaera |
Evizon (squalamine) |
Anti-angiogenesis agent; vascular endothelial growth factor inhibitor |
Wet age-related macular degeneration |
24-week interim data from Phase II Study 209 showed agent was safe, well tolerated and stabilized vision (3/1); subset analysis from Phase II data showed benefit in patients whose vision had already significantly deteriorated (3/29) |
Genta Inc. |
Ganite (FDA-approved) |
Gallium nitrate injection |
Cancer-related hypercalcemia |
Phase II trial in 64 patients showed drug was effective, but not with statistical significance, vs. Novartis AG's Aredia (3/2) |
Indevus |
Nebido |
Long-acting injectable testosterone preparation |
Hypogonadism |
Is starting pharmacokinetic trial designed to support marketing application (3/9) |
MediciNova |
MN-246 |
Selective Beta3-adrenergic receptor agonist |
Urinary incontinence |
Began Phase I trial to evaluate safety, tolerability and pharmacokinetics (3/30) |
Neurotech SA* |
NT-501 |
Encapsulated cell technology used to deliver ciliary neurotrophic factor |
Retinitis pigmentosa |
Phase I trial showed NT-501 can be safely implanted into the eye and was well tolerated; vision improvements were seen (3/14) |
Nuvo |
Pennsaid |
Topical non-steroidal anti-inflammatory agent |
Osteoarthritis |
Initial data from long-term trial in 300 patients confirmed safety; a full analysis was being conducted (3/1); published data from four trials demonstrated efficacy vs. placebo (3/2) |
OrthoLogic |
Chrysalin (TP508) |
Synthetic peptide that mimics certain attributes of the thrombin molecule |
Wrist fractures |
Phase III trial did not demonstrate statistical significance vs. placebo in the primary endpoint of time to removal of immobilization; one secondary endpoint was met, but others were not (3/15) |
Notes: | ||||
* Privately held. | ||||
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; OSK = Osaka Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; SWX = Swiss Stock Exchange; TSE = Toronto Stock Exchange. | ||||
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