Clinical Trials Update: January 2005
Company* | Product | Description | Indication | Status (Date) |
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AUTOIMMUNE | ||||
Barrier | Rambazole | Oral retinoic acid metabolism-blocking agent | Psoriasis | Phase IIa trial in 10 patients showed PASI scores were reduced by an average of 50% (1/12) |
ChemoCentryx | Traficet-EN (CCX282) | Oral anti-inflammatory agent that targets the CCR9 chemokine receptor | Inflammatory bowel disease | Began Phase II trial in 60 patients with Crohn's disease to evaluate safety, tolerability and certain indicators of clinical activity (1/6) |
Serono SA | Mylinax | Oral formulation of cladribine; purine nucleoside analogue | Multiple sclerosis | They plan to begin a Phase III trial in 1,200 patients with relapsing forms of multiple sclerosis (1/11) |
Trubion | TRU-015 | Agent designed to deplete B lymphocytes | Rheumatoid arthritis | Began Phase I trial to evaluate the safety of increasing doses (1/11) |
CANCER | ||||
Access | AP5346 | A DACH polymer platinate | Cancers | Began a Phase I trial with fluorouracil and leucovorin to evaluate drug safety and establish dosing (1/28) |
Adventrx | CoFactor | Form of folic acid designed to enhance the antitumor effects of 5-FU | Metastatic colorectal cancer | Phase II trial met primary endpoint for response rate and demonstrated safety in the first stage of two-stage trial (1/25) |
Allos | PDX | Pralatrexate; small-molecule inhibitor of dihydrofolate reductase | Advanced non- small-cell lung cancer | Began Phase I trial with vitamin B12 and folic acid supplementation in up to six patients with Stage IIIB-IV disease (1/20) |
ArQule Inc. | ARQ 501 | Activated Checkpoint Therapy molecule designed to selectively kill cancer cells | Advanced cancers | Began Phase Ib/II trial with gemcitabine that initially will assess dose escalation (1/26) |
Cytogen Corp. | Quadramet (FDA-approved) | Samarium-153 bound to a small-molecule, bone-seeking phosphonate | Pain associated with metastatic bone disease | Began trial to test drug with bisphosphonates in patients with recurrent or refractory multiple myeloma (1/11) |
Dendreon | Provenge | Immunotherapy that uses a recombinant form of the prostatic acid phosphatase antigen | Advanced prostate cancer | Phase III D9902A trial failed to demonstrate a statistically significant delay in time to disease progression in the overall group or in Gleason score subgroups (1/11) |
EntreMed | Panzem | 2-methoxyestradiol; naturally occurring metabolite of endogenous estrogen | Cancers | Began Phase Ib trial to evaluate pharmacokinetics and safety in advanced patients (1/5) |
Gemin X | GX15-070 | Small-molecule inhibitor of bcl-2 proteins | Chronic lymphocytic leukemia | Began Phase I/II trial to evaluate safety, tolerability and pharmacokinetics in CLL patients (1/6) |
Genentech | Avastin (FDA- approved) | Bevacizumab; antibody designed to inhibit vascular endothelial growth factor | Metastatic colorectal cancer | Phase III study with FOLFOX4 regimen improved overall survival vs. the regimen alone (1/27) |
Genentech | Avastin and Erbitux (both FDA-approved) | VEGF inhibitor and EGFR inhibitor, respectively | Advanced colon cancer | Drugs plus irinotecan produced benefits vs. the drugs alone (1/26) |
Genetronics | Medpulser DNA Delivery System | Electroporation-mediated method for delivering DNA-based therapies | Malignant melanoma | Began Phase I trial to measure the safety of delivering plasmid DNA encoding a cytokine directly to tumors (1/13) |
GTx Inc. | Acapodene | Toremifene citrate; small-molecule non-steroidal selective estrogen receptor modulator | Prostate cancer | Began pivotal Phase III trial that will evaluate the incidence of prostate cancer in patients with high-grade prostatic intraepithelial neoplasia (1/26) |
ImClone | IMC-1121B | Fully human antibody targeting the vascular endothelial growth factor receptor-2 | Solid tumors | Began Phase I trial to evaluate safety and pharmacology in 33 patients (1/5) |
Millennium | Velcade (FDA-approved) | Bortezomib; proteasome inhibitor | Multiple myeloma | Began Phase III VISTA trial with melphalan and prednisone vs. those drugs alone in patients with newly diagnosed MM who are not transplant candidates (1/10) |
Northwest | DCVax-Prostate | Cell-based dendritic-cell product | Hormone-independent prostate cancer | Started a Phase III trial that is expected to enroll about 600 patients (1/31) |
OSI | Tarceva (FDA-approved) | Erlotinib HCl; small- molecule HER1/EGFR inhibitor | Advanced pancreatic cancer | Phase III trial with gemcitabine demonstrated a statistically significant 23.5% improvement in overall survival vs. gemcitabine plus placebo (1/27) |
Peplin Ltd. | PEP005 Topical | Molecular entity sourced from a plant | Actinic keratosis | Phase I trial demonstrated safety and tolerability, and also showed clearance of skin lesions (1/10) |
Peregrine | Tarvacin | Chimeric monoclonal antibody that binds to the phospholipid phosphatidylserine | Advanced solid tumors | Starting a Phase I trial to test safety, tolerability and pharmaco-kinetics in up to 28 patients (1/27) |
Peregrine | Cotara | Monoclonal antibody attached to iodine-131; product from Tumor Necrosis Therapy platform | Glioblastoma multiforme | Beginning a trial to evaluate safety, radiation exposure and efficacy at the first or second relapse (1/21) |
Pro-Pharmaceuticals | Davanat | Carbohydrate compound designed for targeted delivery | Advanced solid tumors | Phase I data from 20 patients showed the drug with 5-FU was well tolerated (1/27) |
Protein Design | M200 | Anti-alpha5beta1 integrin chimeric monoclonal antibody; anti-angiogenesis agent | Renal-cell carcinoma | Began Phase II open-label trial, the first of up to five Phase II trials in solid tumors (1/12) |
Raven | RAV12 | High-affinity IgG1 chimeric antibody | Colon, stomach and pancreatic cancers | Began Phase I/II trial to evaluate the safety and tolerability in 30 patients with advanced cancers (1/11) |
Vertex | VX-680 | Small-molecule inhibitor of Aurora kinases | Solid tumors | Began Phase I trial to evaluate safety and tolerability in refractory patients (1/6) |
CARDIOVASCULAR | ||||
Alexion | Eculizumab | Humanized monoclonal antibody designed to block complement protein C5 | Paroxysmal nocturnal hemoglobinuria | Began treatment phase of Phase III SHEPERD trial under an FDA SPA; the trial will include 75 patients (1/10) |
AtheroGenics | AGI-1067 | Oral agent designed to inhibit the production of VCAM-1 and other molecules in the inflammatory process | Atherosclerosis | Company proposed increasing enrollment in Phase III ARISE trial, to 6,000 patients from 4,000, as well as other changes (1/3) |
Dyax Corp. | DX-88 | Recombinant small protein designed to inhibit human plasma kallikrein | Hereditary angioedema | Reported positive interim data from Phase II EDEMA2 trial based on 61 HAE attacks; Phase III trial is being planned (1/10) |
Synthetic Blood | Oxycyte | Perfluorocarbon-based blood substitute and therapeutic oxygen carrier | Blood substitute | Began Phase II trial to test safety and efficacy in hip-surgery patients who lose blood during surgery (1/6) |
TargeGen Inc.* | TG100-115 | Selective PI3-kinase inhibitor | Acute myocardial infarction | Began a Phase I/II trial to test the agent in 100 patients (1/4) |
CENTRAL NERVOUS SYSTEM | ||||
Allon | AL-108 | Vasoactive intestinal peptide agent, a neuroprotectant | Alzheimer's disease | Began Phase Ia trial in 30 healthy adults to evaluate safety and pharmacokinetics (1/25) |
Amarin Corp. | Miraxion (LAX-101c) | Compound that inhibits certain enzymes, including phospholipases and caspases | Depression | Phase IIa trials showed a significant benefit in a subgroup of patients with melancholic vegetative symptoms (1/12) |
Catalyst | CPP-109 | Designed to inhibit enzyme that normally breaks down gamma amino butyric acid | Cocaine addiction | Starting a Phase I trial to test the safety of the interaction of CPP-109 and cocaine (1/3) |
Cortex | CX717 | Ampakine analogue | Cognitive dysfunction | Starting a pilot Phase II trial that will use PET imaging to determine response of patients with Alzheimer's disease and healthy elderly volunteers (1/31) |
Memory | MEM 1003 | Neuronal L-type calcium channel modulator | Alzheimer's disease | Began a two-phase trial that will include up to 32 patients in the dose-escalation and treatment phases, to test safety and tolerability (1/12) |
Myriad | Flurizan (MPC-7869) | Modulator of gamma- secretase that selectively lowers levels of amyloid beta 42 | Alzheimer's disease | Began a Phase III trial to evaluate changes in cognitive function and activities of daily living in 750 patients with mild to moderate AD (1/12) |
Neuro3d* | ND7001 | Agent that acts on a target that never has been associated with depression or anxiety | Depression and anxiety | Began Phase I trial to evaluate safety, tolerability and pharmaco-kinetics (1/25) |
Somaxon | -- | Low dose of the approved depression drug doxepin | Insomnia | Product demonstrated statistical significance in primary and secondary endpoints in a Phase II trial in 61 patients (1/6) |
StemCells | HuCNS-SC | Human neural stem cells | Batten disease | Filed IND with FDA; if approved it would be the first FDA-approved trial to use a purified composition of human neural stem cells as the therapeutic agent (1/4) |
XenoPort | XP13512 | Transported prodrug of gabapentin (Pfizer Inc.'s Neurontin) | Restless legs syndrome | Phase IIa trial in 38 patients demonstrated statistically significant benefits vs. placebo (1/6) |
DIABETES | ||||
Keryx Bio- | KRX-101 | Sulodexidel; oral heparinoid compound | Diabetic nephropathy | Monitors recommended moving into Phase III portion of Phase II/III trial after interim analysis of safety and efficacy data (1/5) |
Metabasis | CS-917 | Fructose 1-6 bisphosphatase inhibitor | Type II diabetes | Began Phase IIb trial to evaluate effectiveness in lowering blood glucose levels in patients (1/5) |
INFECTION | ||||
Barrier | Azoline | Broad-spectrum, oral antifungal agent | Superficial fungal infections | Phase IIa trial in 67 patients showed positive results in treating various fungal infections of the skin (1/12) |
Biolex Inc.* | BLX-883 | Form of alfa interferon manufactured using firm's LEX System | Infectious diseases | Plans to start Phase I trials in the U.S. and UK to evaluate safety and pharmacokinetics (1/6) |
Coley | Actilon (CPG 10101) | Synthetic Toll-like receptor-9 agonist | Hepatitis C | Phase Ia trial demonstrated tolerability in healthy volunteers; Phase Ib trial in relapsed HCV patients produced a 1.0 log or more reduction in HCV RNA in one-third of patients (1/6) |
Corgentech | -- | NF-kappaB decoy; inhibitor of the NF-kB transcription factor | Atopic dermatitis (eczema) | Beginning a Phase I/II trial to evaluate safety and feasibility of repeated applications in 75 people (1/12) |
GenVec Inc. | -- | Vaccine developed using GenVec's adenovector technology and its 293- RF6 production cell line | HIV | The National Institute of Allergy and Infectious Diseases began the second Phase I trial of the vaccine in healthy volunteers (1/19) |
Hemispherx | Ampligen | RNA drug with potential action as an immuno-modulator and antiviral | HIV | Partner Esteve Laboratories began a Phase II trial in patients with HIV who also may have hepatitis C (1/3) |
Human Genome | CCR5 mAb | Fully human antibody that binds the chemokine receptor CCR5 | HIV | Began Phase I trial to evaluate safety, tolerability and pharmacology in patients (1/10) |
ID Biomedical | StreptAvax | Subunit, protein-based vaccine against Group A streptococcal diseases | Group A streptococcal diseases | Phase II trial in 90 healthy adults showed a statistically significant increase in serum antibodies to all 26 M protein serotypes (1/26) |
ID Biomedical | FluINsure | Nonliving, subunit vaccine for intranasal administration | Influenza | Final analysis from 2003-04 field efficacy trial in 1,349 healthy subjects showed efficacy and other positive results (1/13) |
ID Biomedical | -- | Recombinant subunit plague vaccine | Plague infection | Began Phase I trial to evaluate safety, tolerability and immuno-genicity in healthy volunteers (1/10) |
ID Biomedical | Fluviral | Influenza vaccine | Influenza | Began study in the U.S. to test safety and immunogenicity in 300 healthy adults (1/7) |
Idenix | NM283 | Valopicitabine; oral nucleoside analogue | Hepatitis C | Interim Phase IIa data showed a 99.94% reduction in viral load after 12 weeks in patients taking drug and pegylated interferon (1/10) |
Immtech | DB289 | Oral, dicationic antiviral agent | Infections | Began Phase I trial to compare the current capsule formulation to two new formulations (1/24) |
Inhibitex | Veronate | Antibody-based immune globulin | S. aureus infections in infants | Monitors recommended Phase III trial continue after reviewing safety and other data from the first 500 patients (1/7) |
Intercell AG* | IC51 | Purified, inactivated vaccine | Japanese encephalitis virus | Began Phase III program that includes a 800-person non-inferiority trial vs. JE-VAX and a safety study in 3,000 people (1/10) |
MedImmune | -- | Intranasal vaccine against respiratory syncytial virus and parainfluenza virus type-3 | Pediatric respiratory diseases | Filed IND to begin Phase I trial to evaluate safety, tolerability and immunogenicity in healthy adults (1/20) |
Nabi Bio- | Altastaph | Staphylococcus aureus immune globulin | S. aureus infections | Phase IIa trial showed a 36% reduction in time to hospital discharge vs. placebo (9 days vs. 14 days) (1/10) |
Nabi Bio- | StaphVAX | Staphylococcus aureus polysaccharide conjugate vaccine | Infections | Began study in orthopedic surgery patients with implanted devices to evaluate safety and antibody levels (1/6) |
Theravance | Telavancin | Lipoglycopeptide antibiotic believed to have multiple mechanisms of action | Hospital-acquired pneumonia | Began Phase III trial to evaluate safety and efficacy vs. vancomycin in treating pneumonia caused by Gramositive organisms (1/26) |
Vaxin Inc.* | -- | Adenovirus-vectored nasal and epicutaneous influenza vaccines | Influenza | Phase I trial showed the product was immunogenic and well tolerated by volunteers (1/11) |
MISCELLANEOUS | ||||
Advanced | Ferumoxytol | Intravenously administered bioavailable iron | Iron replacement in anemic chronic kidney disease | Began a Phase III trial comparing the agent to placebo (1/13) |
Alizyme plc | ATL-962 | Inhibitor of gastrointestinal lipases | Obesity | Starting a trial to evaluate fat levels, safety and tolerability in about 80 obese subjects (1/31) |
Alteon Inc. | Alagebrium chloride (ALT-711) | A.G.E. crosslink breaker | Erectile dysfunction | Began Phase II trial in 40 diabetic patients who achieve limited benefit from PDE5 drugs (1/18) |
Enzon | ATG- Fresenius S | Polyclonal antibody preparation used for T-lymphocyte suppression | Preventing rejection in lung transplants | Began trial to compare drug and standard post-transplant triple immunosuppressive regimen to the regimen alone (1/6) |
Discovery | Surfaxin | Engineered version of natural human lung surfactant | Respiratory disorders in premature infants | Began a Phase II trial to test the product for preventing broncho-pulmonary dysplasia, and began a Phase II pilot trial to evaluate an aerosolized formulation (1/6) |
Genaera | Squalamine | Anti-angiogenesis agent; vascular endothelial growth factor inhibitor | Age-related macular degeneration | Preliminary Phase II data showed preserved or improved vision at week three in all 12 eyes tested (1/10) |
Nastech | PYY3-36 Nasal Spray | High-affinity Y2 receptor agonist | Obesity | Partner Merck & Co. Inc began a Phase I trial in obesity (1/26) |
Procyon | Fibrostat | 4 diaminobutane dihydrochloride; topical cream | Hypertrophic scarring | Phase IIb trial in 195 patients failed to demonstrate efficacy vs. placebo on excessive scar formation (1/18) |
Progenics | Methylnaltrexone | Agent designed to block opioid receptors in the gastrointestinal tract | Post-operative bowel dysfunction | Phase II trial in 65 patients showed those who received drug following abdominal surgery had improved gastrointestinal recovery vs. placebo (1/20) |
Savient Pharmaceuticals Inc. (SVNT) | Puricase | Polyethylene glycol conjugate of uricase | Gout | Phase II trial in 41 patients demonstrated reductions in plasma uric acid levels in most patients (1/10) |
Theravance | TD-2749 | Selective 5-HT4 agonist | Gastrointestinal motility dysfunction | Began Phase I trial to evaluate safety, tolerability and pharmacokinetics (1/3) |
Vela | Dextofisopam | R-enantiomer of racemic tofisopam, an agent used for multiple indications outside the U.S. | Irritable bowel syndrome | Phase II trial in 141 patients showed the agent was well tolerated and provided relief to patients (1/4) |
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; ASX = Australian Stock Exchange; LSE = London Stock Exchange; NYSE = New York Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; TSE = Toronto Stock Exchange. | ||||