Clinical Trials Update: August 2006
Company* | Product | Description | Indication | Status |
AUTOIMMUNE | ||||
Genentech Inc. | Rituxan (FDA-approved) | Rituximab; antibody that targets and depletes CD20- positive B cells | Multiple sclerosis | Phase II trial in 104 relapsing- remitting MS patients showed a statistically significant reduction in gadolinium enhancing T1 lesions (8/28) |
Kemia Inc.* | KC706 | Anti-inflammatory agent; inhibitor of p38 MAP kinase | Various indications | Phase I trial demonstrated safety, tolerability and bioavailability in healthy volunteers (8/23) |
La Jolla | Riquent | Abetimus sodium; B-cell toleragen; designed to reduce levels of antibodies to double-stranded DNA | Lupus | Began Phase III trial under FDA SPA that will evaluate time to renal flare in 600 patients from 10 countries (8/9) |
Millennium | MLN0415 | Selective, small-molecule inhibitor of IKK2 | Various indications | Began Phase I trial to evaluate safety, tolerability and pharmaco-kinetics in up to 72 healthy volunteers (8/30) |
CANCER | ||||
Abeille | AB-1001 | Transdermal patch that delivers a commercially available 5HT3-antagonist | Chemotherapy- induced nausea and vomiting | Began Phase II irritation and sensitization study in 240 healthy volunteers (8/9) |
Allos | PDX | Pralatrexate; next- generation antifolate | Advanced peripheral T-cell lymphoma | Began pivotal Phase II trial under FDA SPA to evaluate responses and survival in 100 patients (8/29) |
Argos | AGS-003 | Second-generation, dendritic cell-based personalized immunotherapy | Advanced kidney cancer | Began Phase I/II trial to evaluate activity and safety in 26 patients with newly diagnosed, metastatic disease (8/17) |
Avantogen Oncology Inc. | RP101 | Antiviral drug marketed in Europe that downregulates the STAT3 and APEX genes | Advanced pancreatic cancer | Phase I trial with gemcitabine in patients established the dose for further testing (8/17) |
Celldex | CDX-110 | Epidermal growth factor receptor variant III peptide | Ovarian and prostate cancers | Phase I trial demonstrated tolerability and induction of specific immune responses in patients expressing the mutant receptor (8/23) |
Cell | Xyotax | Paclitaxel linked to a biodegradable polyglutamate polymer | Esophageal and gastric cancers | Four of 12 patients in Phase I trial with radiation had a complete response, and seven had a partial response (8/23) |
Cell | Pixantrone | Anthracycline agent designed to have improved properties | Relapsed non- Hodgkin's lymphoma | Phase III EXTEND trial will continue after encouraging response data were seen in a review of the first 40 patients (8/8) |
CuraGen | PXD101 | Small-molecule histone deacetylase inhibitor | Aggressive B-cell lymphoma | Began Phase II trial to evaluate tolerability and responses in up to 40 refractory patients (8/24) |
CuraGen | PXD101 | Small-molecule histone deacetylase inhibitor | Advanced solid tumors | Began Phase I trial to evaluate safety, tolerability and pharmacocokinetics, as well as antitumor activity (8/10) |
CuraGen | PXD101 | Small-molecule histone deacetylase inhibitor | Advanced hematologic malignancies | Began Phase I trial to evaluate safety and tolerability in combination with azacitidine in up to 15 patients (8/8) |
Enzon | Oncaspar (FDA-approved) | PEG-enhanced version of the naturally occurring enzyme L-asparaginase | Advanced solid tumors and lymphomas | Began Phase I trial to evaluate safety and dosing in combination with Gemzar (8/1) |
Exelixis Inc. | XL647 | Small-molecule inhibitor of the HER2, EGF, VEGF and EphB4 receptor tyrosine kinases | Metastatic non- small-cell lung cancer | Began Phase II trial in advanced patients to evaluate safety and tolerability, as well as responses and survival (8/1) |
GPC Biotech | Satraplatin | Oral platinum compound | Advanced non- small-cell lung cancer | Began Phase II trial to evaluate drug with Tarceva as a first-line therapy in patients 70 years of age and older (8/2) |
Hana | Marqibo | Vincristine sulfate liposomes injection | Advanced acute lymphoblastic leukemia | Began Phase II trial with pulse dexamethasone to evaluate efficacy in up to 44 patients (8/17) |
Hana | Sphingosome Encapsulated Vinorelbine | Targeted liposomal formulation of the microtubule inhibitor vinorelbine | Advanced solid tumors | Began Phase I trial to evaluate safety, tolerability and preliminary efficacy in patients (8/3) |
Medarex Inc. | MDX-1106 (ONO-4538) | Fully human anti-PD-1 antibody | Cancer | Starting Phase I trial to evaluate pharmacokinetics and safety in up to 39 patients (8/1) |
MethylGene | MGCD0103 | Oral histone deacetylase inhibitor | Advanced Hodgkin's lymphoma | Began single-agent Phase II trial to evaluate responses and other endpoints in up to 35 patients who failed other therapies (8/22) |
Oncolys | Telomelysin | Adenovirus carrying human telomerase gene promoter sequence | Solid tumors | Is starting Phase I trial in patients resistant to other treatments (8/28) |
Raven | RAV12 | High-affinity IgG1 chimeric antibody | Adeno- carcinomas | Interim Phase I data from 15 patients showed a partial remission in one patient and stable disease in three patients (8/15) |
SGX | Troxatyl | Troxacitabine; nucleoside analogue | Acute myelogenous leukemia | Monitors in Phase II/III trial evaluating drug in third-line setting said drug was unlikely to show efficacy; the trial was discontinued (8/28) |
YM BioSciences | Tesmilifene | Small molecule designed to augment the activity of chemotherapy agents | Advanced breast cancer | Monitors in pivotal Phase III trial of drug with chemotherapy recommended continuation of study, following their second safety and efficacy review(8/22) |
CARDIOVASCULAR | ||||
Cytokinetics | CK-1827452 | Oral formulation; direct cardiac myosin activator | Heart failure | Began Phase I trial to evaluate pharmacokinetics in healthy volunteers (8/23) |
Decode | DG051 | Small-molecule inhibitor of leukotriene A4 hydrolase | Prevention of heart attack | Began Phase I trial to evaluate safety, tolerability and pharmacokinetics (8/14) |
Icagen Inc. | ICA-17043 | Small-molecule ion channel inhibitor | Sickle cell disease | Monitors recommended ending enrollment in Phase III trial for those not on concurrent hydroxyurea therapy, about half the patients, due to lack of efficacy (8/4) |
Inspire | INS50589 Antiplatelet | Inhibitor of the platelet P2Y12 adenosine diphosphate receptor | For use in cardiac surgery | Phase II trial was terminated after interim analysis showed bleeding complications (8/7) |
Predix | PRX-08066 | Small-molecule serotonin 5-HT2B antagonist | Pulmonary hypertension | Began Phase II trial to evaluate efficacy and safety in 72 patients with PH associated with chronic obstructive pulmonary disease (8/7) |
CENTRAL NERVOUS SYSTEM | ||||
Accera Inc.* | Ketasyn (AC-1202) | Neuronal cell-targeting agent that acts to increase mitochondrial activity | Age-associated memory impairment | Began Phase IIa trial to evaluate safety and efficacy in patients ages 50 to 85 (8/23) |
Adolor | ADL5859 | Oral Delta opioid receptor agonist | Pain | Began Phase I trial to evaluate safety, tolerability and pharmaco-kinetics in healthy volunteers (8/30) |
BioDelivery | BEMA LA | Long-acting formulation of an undisclosed analgesic | Pain | Proof-of-concept study in normal volunteers produced positive pharmacokinetic results (8/16) |
Corcept | Corlux | Mifepristone; oral GR-II antagonist with potential mitigating effects on cortisol | Psychotic major depression | First of three Phase III trials failed to demonstrate efficacy vs. placebo for both primary and secondary endpoints (8/25) |
NovaDel | Zolpidem oral spray | Formulation of the active ingredient in Ambien | Insomnia | Began bioequivalance trial to evaluate pharmacokinetic profile vs. Ambien in healthy volunteers (8/8) |
Pain | PTI-202 | Long-acting, abuse- resistant opioid agent | Pain | Began Phase I trial to evaluate the agent in humans (8/2) |
Pharmos | Cannabinor (PRS-211,375) | CB2-selective synthetic cannabinoid agent | Pain | Began second Phase IIa trial to evaluate analgesic activity and safety in healthy subjects with capsaicin-induced pain (8/10) |
TorreyPines | NGX426 | Oral prodrug of tezampanel, an AMPA/ kainate receptor antagonist | Migraine and neuropathic pain | Began Phase I trial to evaluate safety, tolerability and pharmaco-kinetics in 30 healthy volunteers (8/7) |
Transition | AZD-103 | Scyllo-cyclohexanehexol; oral agent that acts on beta amyloid | Alzheimer's disease | Is starting Phase I trial to evaluate safety, tolerability and pharmaco-kinetics in healthy volunteers (8/1) |
DIABETES | ||||
Advanced Viral | AVR118 | Peptide-nucleic acid complex with immuno-modulatory properties | Type II diabetes | Interim Phase I data demonstrated safety; no effects were seen on blood glucose levels in those receiving oral hypoglycemic therapies (8/10) |
Alantos | ALS 2-0426 | Small-molecule inhibitor of dipeptidyl peptidase IV | Type II diabetes | Began Phase I trial to evaluate safety, tolerability and pharmaco-kinetics in 50 healthy volunteers (8/16) |
INFECTION | ||||
Advancis | Amoxicillin Pulsys | Amoxicillin formulation delivered with once-daily pulsatile Pulsys technology | Pharyngitis/ tonsillitis | Phase III trial in patients with strep throat achieved its desired microbiological and clinical end-points (8/10) |
Ambrilia | PPL-100 | HIV protease inhibitor | HIV | Phase I trial demonstrated safety, tolerability and a favorable pharmacokinetic profile (8/17) |
Anacor | AN2690 | Small-molecule, boron-based topical agent | Onychomycosis | No drug was detected in the bloodstream of 15 patients in a Phase I trial (8/9) |
Cerexa Inc.* | Ceftaroline | Next-generation, broad- spectrum, injectable cephalosporin agent | Complicated skin and skin structure infections | Phase II trial in 100 patients showed a clinical cure rate of 96.8% vs. 88.9% for those treated with vancomycin (8/2) |
Cubist | Cubicin (FDA-approved) | Daptomycin for injection; a bactericidal antibiotic | S. aureus bacteremia and endocarditis | Phase III trial vs. dual-therapy standard of care demonstrated non-inferiority (8/16) |
Enzo | StealthVector HGTV43 | Retrovirus-based vector used to transfer three antisense genes | HIV | Data from Phase I trial showed long- erm presence of the engineered CD34+ stem cells and engineered CD4+ immune cells; Phase I/II trial is under way (8/15) |
GenVec Inc. | -- | HIV vaccine using GenVec's adenoviral vector technology with a DNA prime vaccine | HIV | The Vaccine Research Center began the first human studies, which will evaluate safety and immunogenicity in 15 patients (8/22); data from Phase I trials showed favorable safety and immunogenicity results (8/31) |
Genzyme | Tolevamer | Non-antibiotic polymer therapy | C. difficile- associated diarrhea | Phase II trial demonstrated a similar treatment outcome as vancomycin in time to resolution of diarrhea, and a trend toward reduced recurrence (8/15) |
Iomai Corp. | -- | Vaccine patch | Travelers' diarrhea | Began Phase II trial to evaluate safety and frequency of E. coli infection in volunteers traveling to Mexico and Guatemala (8/15) |
Tanox Inc. | TNX-355 | Humanized anti-CD4 monoclonal antibody; viral entry inhibitor | HIV | Phase II trial with optimized background regimen produced a sustained virologic response through 48 weeks (8/17) |
Theravance | Telavancin | Lipoglycopeptide; bactericidal injectable antibiotic | Complicated skin and skin structure infections | Phase III ATLAS I and ATLAS II trials in 1,867 patients demonstrated better rates of cure and microbio- logical eradication than vancomycin (8/22) |
VaxGen Inc.(PK:VXGN) | rPA102 | Recombinant protective antigen anthrax vaccine | Anthrax infection | Phase I trial showed a relationship between the dose and the immune response; no toxicity was seen (8/14) |
Vical Inc. | -- | Vaccine based on Vical's DNA delivery technology, boosted by an adenoviral vector vaccine | HIV | A Phase I trial sponsored by the National Institutes of Health was initiated (8/22); Phase I trial in 40 patients demonstrated safety, tolerability and induction of T-cell immune responses (8/31) |
ViRexx | HepaVaxx B Vaccine | Vaccine containing a a hepatitis B viral antigen and part of a murine monoclonal antibody | Hepatitis B | No significant adverse events were seen in a Phase I trial in 15 healthy volunteers (8/9) |
ViroPharma | HCV-796 | Oral hepatitis C virus polymerase inhibitor | Hepatitis C | Preliminary data from Phase Ib trial with pegylated interferon showed encouraging activity (8/28) |
MISCELLANEOUS | ||||
Athernagen | ATG003 | Eye-drop formulation of the anti-angiogenic agent mecamylamine | Age-related macular degeneration | Began a Phase I trial to evaluate ocular tolerability and safety (8/21) |
Critical | Zileuton I.V. | Intravenous formulation of zileuton (Zyflo), a 5-lipoxygenase inhibitor | Asthma | Phase I/II trial demonstrated tolerability and improvements in lung function vs. placebo (8/23) |
Halozyme | Enhanze | Delivery platform based on recombinant human PH20 hyaluronidase | Agent designed to enhance absorption of large molecules | Began trial to evaluate pharmaco-kinetics, safety and tolerability of a protein drug injected both with and without rHuPH20 (8/8) |
Isotechnika | TAFA93 | Small-molecule mTOR inhibitor; prodrug of rapamycin | Various indications | Phase I trials demonstrated safety and tolerability in healthy volunteers (8/21) |
MAP | MAP0010 | Nebulized formulation of budesonide for inhalation | Asthma | Began Phase II trial to evaluate asthma control in 225 pediatric and adolescent patients (8/2) |
Nastech | PYY(3-36) | Nasal spray formulation of the Y2 receptor agonist | Obesity | Began study in 12 obese subjects to evaluate pharmacokinetics, safety and efficacy measures (8/14) |
Omrix Bio- | Omr-IgG-am | Intravenous immunoglobulin | Primary immune deficiency | Partner FFE Enterprises Inc. is starting a Phase III trial (8/17) |
OrthoLogic | Chrysalin (TP508) | Synthetic peptide that mimics certain attributes of the thrombin molecule | Wrist fractures | Interim data from 240 patients in Phase IIb trial did not show benefit vs. placebo; secondary end- points also were missed; the trial was discontinued (8/29) |
Palatin | Bremelanotide | Melanocortin receptor agonist | Female sexual arousal disorder | Phase IIa pilot study in post- menopausal women showed 73% of drug patients reported an increased level of genital arousal vs. 23% for placebo (8/3) |
PDL | Terlipressin | Peptide derived from the natural hormone lysine-vasopressin | Type 1 hepatorenal syndrome | Phase III trial showed a trend toward improvement but did not achieve statistical significance for primary endpoint of treatment success (8/3) |
Progenics | Oral methylnaltrexone | Peripheral mu opioid receptor antagonist | Opioid-induced constipation | Began Phase II trial to evaluate efficacy and dosing in 200 to 400 patients (8/22) |
Serono SA | -- | Recombinant human growth hormone | HIV-associated adipose redistribution syndrome | Phase III HARS trial met all primary and major secondary efficacy endpoints; a supplemental NDA is on file with the FDA (8/17) |
Sirna | Sirna-027 | Chemically modified short interfering RNA targeting vascular endothelial growth factor receptor-1 | Age-related macular degeneration | Phase I trial demonstrated safety and tolerability, and all 26 patients showed visual acuity stabilization eight weeks after a single injection (8/10) |
Vyteris | Actyve | Transdermal drug delivery technology | Infertility | Phase I trials demonstrated the delivery of therapeutic levels of a peptide used to treat infertility (8/10) |
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; FSE = Frankfurt Stock Exchange; NYSE = New York Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; PK = Pink Sheets; TSE = Toronto Stock Exchange. | ||||