Clinical Trials Update: October 2006
Company* | Product | Description | Indication | Status |
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AUTOIMMUNE | ||||
Array | ARRY-438162 | Small-molecule inhibitor of the protein kinase MEK | Inflammatory diseases | Phase I trial in 20 healthy volunteers met its primary objectives; studies in patients were being planned (10/19) |
Aspreva | CellCept | Mycophenolate mofetil; an immunosuppressant | Myasthenia gravis | 80-patient trial showed no difference in efficacy between drug and prednisone vs. prednisone alone (10/11); 176-patient Phase III trial failed to meet either primary or secondary endpoints (10/26) |
Biogen Idec | Tysabri | Natalizumab; humanized monoclonal antibody that inhibits adhesion molecules on immune cells | Crohn's disease | Data showed drug maintained remission for longer than two years (10/24) |
Biogen Idec | Tysabri (FDA-approved) | Natalizumab; humanized monoclonal antibody that inhibits adhesion molecules on immune cells | Multiple sclerosis | Data from Phase III AFFIRM trial showed drug significantly reduced corticosteroid use and hospitalizations, and decreased disease activity (10/6) |
ChemoCentryx | Traficet-EN (CCX282-B) | Oral anti-inflammatory agent that targets the CCR9 chemokine receptor | Crohn's disease | Phase II trial showed a drop in disease activity and in blood levels of C-reactive protein (10/24) |
Osiris | Prochymal | Intravenous formulation of mesenchymal stem cells | Crohn's disease | Pilot Phase II study in those who didn't respond to other therapies showed a statistically significant decrease in disease activity (10/19) |
CANCER | ||||
Alfacell Corp. | Onconase | Ranpirnase; cytotoxic ribonuclease | Malignant mesothelioma | Further analysis of Phase IIIa trial showed greater median survival times for drug vs. doxorubicin (10/24) |
Allos | Efaproxyn | Efaproxiral; small molecule designed to sensitize hypoxic areas of tumors during radiation therapy | Brain metastases originating from breast cancer | Monitors recommended pivotal Phase III ENRICH trial continue to completion, following a second interim analysis (10/23) |
Antisoma plc | AS1404 | Small molecule derived from xanthenone acetic acid; vascular disrupting agent | Ovarian cancer | Phase II trial showed a response rate of 75% vs. 63% for patients receiving chemotherapy alone (10/15) |
AVEO | AV-412 | Next-generation, oral tyrosine kinase inhibitor of EGFR/HER2 | Solid tumors | Began Phase I trial to evaluate safety, tolerability and dosing in patients (10/23) |
Biogen Idec | Zevalin (FDA-approved) | Ibritumomab tiuxetan; monoclonal antibody targeting CD20, combined with radioisotope | Diffuse large-B- cell lymphoma | Began Phase III ZEAL trial to evaluate survival in patients taking Zevalin after chemotherapy regimen (10/4) |
Biovest | BiovaxID | Personalized vaccine; tumor antigen conjugated to KLH administered with GM-CSF | Non-Hodgkin's follicular lymphoma | 20 of 25 indolent patients in Phase II trial developed an anti-idiotype immune response; second complete responses were significantly longer than first responses (10/19) |
Celgene | Revlimid (FDA-approved) | Lenalidomide; derivative of Thalomid (thalidomide) | Advanced non- Hodgkin's lymphoma | Seven of 22 evaluable patients in open-label Phase II trial exhibited an objective response (10/2) |
Cellgate Inc.* | CGC-11047 | Polyamine analogue that targets proliferating cells | Advanced solid tumors and lymphomas | Began Phase Ib trial to evaluate safety and pharmacokinetics with individual chemotherapeutics, in 70 patients (10/2) |
Cerus Corp. | CRS-100 | Attenuated strain of Listeria monocytogenes; immunotherapy | Cancers that metastasized to the liver | Began Phase I trial to evaluate dosing, safety and tolerability in patients (10/10) |
Cougar | CB7630 | Abiraterone acetate; oral inhibitor of the steroidal enzyme 17 alpha- hydroxylase/C17,20 lyase | Advanced prostate cancer | Eight of 12 patients in Phase I trial experienced reduced PSA levels of greater than 50%; treatment was well tolerated (10/8) |
CuraGen Corp. | PXD101 | Small-molecule histone deacetylase inhibitor | Cancers | Preliminary data from a Phase II trial and two Phase Ib/II trials, in solid tumors and multiple myeloma, showed encouraging results (10/26) |
EntreMed | MKC-1 | Small-molecule cell-cycle inhibitor | Non-small-cell lung cancer | Began Phase I/II trial to evaluate safety and dosing with pemetrexed (Alimta); Phase II portion will assess antitumor activity and progression- free survival in up to 60 patients (10/25) |
Genmab A/S | HuMax-EGFr | Zalutumumab; human antibody that targets the epidermal growth factor receptor | Advanced head and neck cancer | Began Phase I/II trial in 36 patients to evaluate first-line treatment in combination with radiotherapy and cisplatin (10/24) |
Genta Inc. | G4460 | Antisense agent that targets the oncogene c-myb | Advanced hematologic cancers | Began Phase I trial to evaluate dosing, safety, activity and down-regulation of c-myb in patients(10/13) |
Gloucester | Romidepsin (FK228) | Depsipeptide; histone deacetylase inhibitor | Metastatic hormone- refractory prostate cancer | Data from 22 patients in ongoing Phase II trial showed one partial response, two with stable disease and seven with disease stabilization(10/4) |
Hana | Talvesta (PT-523) | Talotrexin; nonclassical antifolate; analogue of aminopterin | Advanced acute lymphoblastic leukemia | Began Phase II trial to evaluate remission rates, as well as other efficacy endpoints (10/31) |
ImmunoGen | AVE1642 | Antibody that binds to the insulin-like growth factor 1 receptor | Cancers | Partner Sanofi-Aventis SA began clinical testing of the antibody (10/4) |
Innovive | INNO-305 | WT1 peptide therapeutic vaccine designed to stimulate both CD8 and CD4 T cells | Hematologic malignancies and solid tumors | Began Phase I trial to study safety, tolerability, pharmacokinetics and preliminary efficacy in WT1-expressing cancers (10/27) |
MethylGene | MGCD0103 | Class I-specific histone deacetylase inhibitor | Solid tumors | Began Phase I/II trial to evaluate the agent with Gemzar, first testing safety and dosing; the Phase II portion will assess responses in up to 60 patients (10/25) |
Northwest | DCVax-Brain | Dendritic cell-based immunotherapy made from patients' cells | Glioblastoma multiforme | Updated trial data showed 44% of drug patients were progression-free vs. 11% for historical controls; survival at two years was improved (10/30) |
Novacea Inc. | Asentar (DN-101) | High-dose oral formulation of calcitriol | Cancers | Phase I trial demonstrated safety and tolerability (10/30) |
Rexahn Corp.* | RX-0201 | Signal inhibitor that blocks production of the protein kinase Akt | Cancers | Successfully concluded Phase I trial in patients with advanced cancers (10/17) |
Vion | Cloretazine (VNP40101M) | Sulfonylhydrazine DNA alkylating agent | Advanced small- cell lung cancer | Initial data on 36 patients from single-agent Phase II trial showed a response rate of 32% (10/26) |
Ziopharm | ZIO-101 | Organic arsenic agent | Solid tumors | Data from ongoing Phase I trial showed benefit in eight of 29 patients (10/3) |
ZymoGenetics | -- | Interleukin-21 | Metastatic stage IV renal cell cancer | Began Phase I/II trial to evaluate safety and activity of the agent in combination with the approved drug Nexavar (sorafenib) (10/17) |
CARDIOVASCULAR | ||||
Amgen Inc. | AMG 531 | Peptibody protein designed to stimulate the thrombopoietin receptor | Immune thrombocytopenic purpura | Phase I/II trial demonstrated increased blood platelet counts (10/19) |
AVI | Resten-MP | Intravenous delivery of AVI-4126, an antisense drug targeting the c-myc transcription factor | Cardiovascular indications | Preliminary data from Phase II APPRAISAL trial demonstrated encouraging safety and delivery results (10/31) |
AVI | AVI-5126 | New generation of antisense drug | For use in coronary artery bypass grafting | Began pivotal trial to evaluate the product in 600 patients under-going CABG procedures (10/18) |
CardioVascular | -- | Formulation containing Cardio Vascu-Grow, a stimulator of angiogenesis | Peripheral artery disease | Began Phase I trial to evaluate safety and the growth of new blood vessels in the legs of 24 patients (10/24) |
CoGenesys | Cardeva | Long-acting form of B-type natriuretic peptide | Heart failure | Began Phase I/II trial to evaluate safety and tolerability in up to 80 stable patients with Class II or III heart failure (10/11) |
Corautus | VEGF-2 | Vascular endothelial growth factor in the form of naked plasmid DNA | Severe angina | Final data from GENASIS Phase IIb trial failed to show statistical significance in improving exercise tolerance time in any dose group (10/10) |
DeCode | DG031 (veliflapon) | Inhibitor of 5-lipoxygenase activating protein, or FLAP | Prevention of heart attack | Voluntarily suspended Phase III trial due to an unexpected formulation problem with the tablets being used in the trial (10/5) |
Metabasis | MB07811 | Liver-targeting thyroid hormone receptor agonist | Hyperlipidemia | Began Phase I trial to evaluate safety and tolerability in healthy volunteers (10/30) |
Momenta | M118 | Anticoagulant agent that binds to anti-thrombin III and thrombin | Acute coronary syndromes | Began Phase I trial in up to 36 healthy adult males to evaluate safety, tolerability and pharmaco- kinetics (10/10) |
Omrix Bio- | -- | Topical, human plasma- derived thrombin | For use in general surgeries | Pivotal Phase III trial demonstrted equivalence to bovine thrombin; a BLA filing was planned for November (10/16) |
Paion AG* | Desmoteplase | Genetically engineered version of a clot-dissolving protein found in the saliva of the vampire bat | Acute ischemic stroke | Monitors put Phase IIb/III DIAS-2 trial on hold pending further data analysis (10/25); patient enrollment was resumed with no modification of the protocol (10/28) |
Portola | -- | Intravenous formulation of its ADP receptor antagonist; an antiplatelet agent | Cardiovascular indications | Began a Phase I trial of the product (10/31) |
Renovis Inc. | NXY-059 | Neuroprotectant with free radical-trapping properties | Acute ischemic stroke | Pivotal Phase III SAINT II trial did not demonstrate a statistically significant reduction on stroke- related disability vs. placebo; secondary endpoints also were missed (10/26) |
Titan | DITPA | Analogue of thyroid hormone (T3) | Congestive heart failure | Discontinued further enrollment in Phase II trial; resources are being focused elsewhere (10/25) |
CENTRAL NERVOUS SYSTEM | ||||
Ceregene | CERE-120 | Gene therapy consisting of an adeno-associated virus vector carrying the gene for neurturin | Parkinson's disease | Phase I trial in 12 patients showed a 40% reduction in symptoms; treatment was well tolerated (10/10) |
Durect Corp. | Memryte | Durin implant technology with leuprolide acetate | Alzheimer's disease | Voyager ended Phase III trials in order to get an earlier look at potential efficacy; 600 patients had been accrued (10/19) |
Medivation | Dimebon | Neuroprotectant agent that's been on the market in Russia since 1983 | Huntington's disease | Began Phase I/IIa trial to evaluate safety, tolerability and preliminary efficacy in 75 patients (10/19) |
Neurogen | NG2-73 | Selective modulator of gamma aminobutyric (GABA) receptors | Insomnia | Began Phase II trial that will evaluate the efficacy and safety of five different dose and formulation profiles of the drug (10/30) |
NeurogesX | Transacin (NGX-4010) | High-concentration trans- capsaicin dermal patch | Post-herpetic neuralgia | Phase III trial in 420 patients demonstrated statistically significant pain reduction vs. a control patch (10/18) |
Neurologix | -- | Adeno-associated virus delivering gene encoding glutamic acid decarboxylase | Parkinson's disease | One-year data from Phase I trial in 12 patients showed statistically significant improvement from baseline; safety and tolerability also were demonstrated (10/17) |
Santhera | SNT-MC17 | Idebenone; small molecule optimized to facilitate the transport of electrons within mitochondria | Friedreich's ataxia | Phase II trial in 48 patients demonstrated safety and a trend toward improved neurological parameters (10/6) |
Somaxon | Silenor | Low dose of the approved depression drug doxepin | Transient insomnia | Phase III trial in 565 patients demonstrated statistically significant improvements in sleep onset vs. placebo, and on other sleep-related endpoints (10/23) |
Titan | Probuphine | Product designed to continuously deliver the approved drug buprenorphine | Opioid dependence | Began Phase III trial to evaluate safety and efficacy vs. placebo in 150 patients (10/25) |
TorreyPines | Tezampanel | AMPA/kainate receptor antagonist | Acute migraine | Began Phase IIb trial to evaluate pain relief and other endpoints in 300 patients (10/16) |
DIABETES | ||||
Emisphere | -- | Insulin delivered with company's oral eligen technology | Type II diabetes | Phase II trial met the company's safety and efficacy objectives (10/30) |
Sirtris | SRT501 | Agent that targets SIRT1, a member of the human sirtuin family of enzymes | Type II diabetes | Phase I trial in healthy volunteers demonstrated safety and tolerability; began Phase Ib trial in 90 patients to evaluate safety and pharmacokinetics (10/4) |
INFECTION | ||||
Advanced | Cethromycin | Second-generation ketolide antibiotic agent | Acute bacterial sinusitis | Presented data from studies that supported selection of 300-mg dose for further studies (10/13) |
Alnylam | ALN-RSV01 | RNAi agent designed to silence the RSV nucleo- capsid "N" gene; inhaled formulation | Respiratory syncytial virus | Began Phase I trial to evaluate safety, tolerability and pharmacokinetics in healthy adult volunteers (10/11) |
AVI | AVI-4065 | Neugene antisense compound | Hepatitis C | Exploratory study in 12 patients failed to show efficacy; a change in protocol was planned (10/4) |
Crucell NV | -- | AdVac-based tuberculosis vaccine; uses Ad35 adenovirus vector | Tuberculosis | Began Phase I trial to evaluate safety, tolerability and immuno-genicity in 24 healthy volunteers (10/25) |
GlobeImmune | GI-5005 | Targeted immunotherapy expressing a NS3-Core fusion protein | Hepatitis C | Phase Ib trial showed generation of cellular immune responses in 12 of 29 patients, and elicited a statistically significant improvement in alanine amino transferase levels (10/30) |
Hollis-Eden | Neumune | Immune-regulating hormone | Infections and acute radiation syndrome | Phase I trial in healthy volunteers demonstrated positive safety and hematopoietic data (10/13) |
Human | Albuferon | Long-acting form of interferon alpha, created with albumin fusion technology | Hepatitis C | Interim data from Phase IIb trial with ribavarin showed positive quality-of-life results through week 12 (10/16); Phase II data showed comparable efficacy to pegylated interferon in treatment-naïve patients (10/31) |
Idenix | Tyzeka | Telbivudine; oral, once-daily nucleoside analogue | Hepatitis B | Presented favorable data from the second year of the GLOBE study and from other clinical trials (10/27) |
Idenix | Valopicitabine (NM283) | Oral nucleoside analogue | Hepatitis C | Phase IIb trial with pegylated interferon showed favorable viral suppression at 24 weeks in treatment-naïve patients (10/27) |
MedImmune | CAIV-T | Trivalent vaccine; next generation of FluMist; refrigerator-stable formulation | Influenza | Trial in 52 children demonstrated higher antibody responses than the traditional injectable trivalent inactivated flu vaccine (10/13) |
Pharmasset | R7128 | Polymerase inhibitor; prodrug of the agent PSI-6130 | Hepatitis C | Began Phase I trial to evaluate safety and pharmacokinetics in healthy volunteers, and antiviral activity in patients (10/27) |
PowderMed | DNA-based trivalent vaccine delivered via needle-free injection device | Influenza | Is starting Phase I trial to evaluate safety, tolerability and immuno-genicity (10/2) | |
Sequella Inc.* | SQ109 | Inhibitor of cell wall synthesis in certain microorganisms | Tuberculosis | Began Phase I trial to evaluate safety and pharmacokinetics in 46 healthy volunteers (10/3) |
Starpharma | VivaGel | Vaginal microbicide gel | Prevention of genital herpes | Began Phase I trial that will evaluate safety in 60 volunteers (10/24) |
Theravance | Telavancin | Lipoglycopeptide; bactericidal injectable antibiotic | Complicated skin and skin-structure infections | Additional data from Phase III ATLAS 1 and ATLAS 2 trials demonstrated non-inferiority to vancomycin (10/10) |
Vertex | VX-950 | Telaprevir; oral HCV protease inhibitor | Hepatitis C | Phase Ib trial with pegylated interferon showed suppression of both wild-type HCV and resistant variants; also, trial showed 24 of 26 patients who took drug plus peg-FN and ribavirin had undetectable HCV RNA (10/27) |
ViroPharma | HCV-796 | Orally dosed hepatitis C viral polymerase inhibitor | Hepatitis C | Began Phase II trial to evaluate safety, tolerability, pharmacokinetics and antiviral activity when used with pegylated interferon alfa-2b plus ribavirin (10/24) |
MISCELLANEOUS | ||||
Adenosine | Apadenoson (ATL146e) | Selective adenosine A2A agonist; anti-inflammatory agent | Various conditions | Began Phase Ib trial to evaluate safety, dosing and anti-inflammatory proof of principle (10/25) |
Advanced | Ferumoxytol | Intravenously administered bioavailable iron | Iron replacement in anemic chronic kidney disease | Monitors found no safety concerns in review of data from 1,200 patients in Phase III trial (10/23) |
Amylin | Symlin (FDA-approved) | Pramlintide acetate; an analogue of human amylin | Obesity | 52-week data from Phase II trial showed a 7% to 8% weight reduction vs. 1% reduction for placebo; company plans several studies testing drug with neurohormones for obesity (10/24) |
Auxilium | AA4500 | Injectable enzyme product | Peyronie's disease | 89% of patients in Phase II trial who received three treatments achieved clinical success (10/25) |
Auxilium | TestoFilm | Testosterone replacement transmucosal film product | Hypogonadism | Development of the formulation, which was in Phase III comparative trials, was discontinued due to insufficient commercial viability (10/18) |
Collegium | -- | Agent incorporating both a sedating and nonsedating antihistamine | Allergic rhinitis | Began proof-of-concept study of the combination product (10/4) |
Critical | -- | Controlled-release formulation of zileuton (Zyflo), a 5-lipoxygenase inhibitor | Asthma | 613-patient trial produced favorable safety and efficacy data vs. placebo and Zyflo (10/25) |
Discovery | Surfaxin | Engineered version of natural human lung surfactant | Broncho- pulmonary dysplasia | Phase II trial in 136 premature infants demonstrated safety and tolerability, and encouraging signs of efficacy (10/11) |
Dynavax | AIC | Toll-like receptor 9 agonist linked to ragweed allergen | Ragweed allergy | Pilot study showed treatment reduced allergic responses and sustained that effect more than 12 months (10/4) |
EPIX | PRX-07034 | Highly selective 5-HT6 receptor antagonist | Obesity | Began Phase I trial to evaluate safety, tolerability and pharmacokinetics in otherwise healthy obese adults; earlier Phase I trial demonstrated tolerability (10/24) |
Genaera | Evizon (squalamine) | Anti-angiogenesis agent; vascular endothelial growth factor inhibitor | Wet age-related macular degeneration | Data from Phase II trial showed no significant increase in systolic or diastolic blood pressure (10/16) |
Inspire | -- | Epinastine nasal spray formulation | Seasonal allergic rhinitis | Began Phase II program that will evaluate formulations and dosing (10/10) |
Manhattan | Oleoylestrone | Oral, synthetic form of Oleoylestrone | Obesity | Began Phase IIa trial to evaluate safety and efficacy in 24 morbidly obese male subjects (10/12) |
LAB | LAB CGRP | Calcitonin gene-related peptide; vasodilative agent | Asthma | Crossover Phase II study in 12 patients showed statistically significant broncho-protective effects vs. placebo (10/4) |
Microbia Inc.* | Linaclotide (MD-1100) | Peptide designed to act on guanylate cyclase-C, a receptor found on intestinal cells | Chronic constipation | Phase IIa trial demonstrated tolerability and safety, and improvements in bowel function (10/24) |
Nabi Bio- | PhosLo (FDA-approved) | Calcium acetate; phosphate binder | Stage IV kidney disease | EPICK study achieved its primary endpoint of superior serum phosphorus control vs. placebo; secondary endpoints also were met (10/16) |
NicOx SA | HCT 3012 (naproxcinod) | Nitric oxide-donating derivative of naproxen | Osteoarthritis of the knee | Phase III trial demonstrated statistical significance vs. placebo on all three co-primary efficacy endpoints (10/27) |
Orexigen | Excalia | Combination of bupropion, a dopamine and norepinephrine reuptake inhibitor, and the approved anticonvulsant zonisamide | Obesity | Phase IIa trial in 127 nonsmokers demonstrated increased weight loss vs. placebo (10/22) |
Regeneron | IL-1 Trap | Agent designed to attach to and neutralize interleukin-1 | CIAS1-associated periodic syndromes | Both Phase III trials met their primary endpoints of improving disease activity (10/30) |
Repligen | RG1068 | Synthetic human secretin | Pancreatic disease | Began study to evaluate the diagnostic utility of the product in MRI of the pancreas in 45 healthy vol-unteers and patients (10/30) |
PTC | PTC124 | Oral agent that targets nonsense mutations | Duchenne muscular dystrophy | Preliminary data from Phase II trial indicated increases in dystrophin and improvements in muscle enzymes (10/21) |
Tanox Inc. | TNX-650 | Humanized monoclonal antibody that targets interleukin-13 | Asthma | Began Phase I trial to evaluate safety, tolerability and pharmacokinetics in 32 healthy volunteers (10/25) |
Theravance | TD-5108 | Selective 5-HT4 receptor agonist | Chronic constipation | Began Phase II trial to evaluate safety and efficacy of a range of doses in 350 patients (10/9) |
Trine | Crofelemer | Agent extracted from the rain- forest plant Croton lechleri | Diarrhea- predominant irritable bowel syndrome | Began Phase IIb trial to evaluate efficacy and safety in 240 women with D-IBS (10/30) |
Urigen NA* | U101 | Intravesical formulation of FDA- approved components | Pelvic pain of bladder origin | Phase II trial did not meet primary endpoint of improved pain and urgency; positive trends were seen (10/30) |
Vivus Inc. | Qnexa | Agent incorporating low doses of the approved products phentermine and topiramate | Obesity | Phase II trial demonstrated a statistically significant weight loss and a reduction in waist size; lipid levels also were reduced (10/23) |
XenoPort | XP19986 | Transported prodrug of the generic spasticity drug R-baclofen | Gastro- esophageal reflux disease | Phase IIa trial demonstrated tolerbility and statistically significant reductions in reflux episodes and reflux-associated heartburn symptoms (10/22) |
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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; CSE = Copenhagen Stock Exchange; LSE = London Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; TSX = Toronto Stock Exchange. | ||||