| Company* (Country; Symbol) | Product | Description | Indication | Status (Date) |
| AUTOIMMUNE | ||||
| Chelsea Therapeutics International Ltd. (CHTP) | CH-4051 | Orally available, non-metabolized antifolate | Rheumatoid arthritis | Initiated dosing in volunteers in the single ascending dose study of its Phase I program (11/13) |
| CANCER | ||||
| Adherex Technologies Inc. (AMEX:ADH) | Eniluracil | Oral dihydropyrimidine dehydrogenase inhibitor | Liver cancer | Suspended further enrollment in Phase I/II trials (11/12) |
| Antigenics Inc. (AGEN) | Oncophage | Vitespen | Recurrent, high-grade glioma | Phase I data showed that Oncophage vaccination following brain cancer surgery increased overall median survival to about 10.5 months, compared to a historical median survival of 6.5 months (11/24) |
| Bionovo Inc. (BNVI) | BZL101 | Causes energy collapse in cancer cells by inhibiting glycolysis | Metastatic breast cancer | Phase Ib data showed it continued to be safe and well tolerated, with early signs of clinical efficacy (11/20) |
| Celldex Therapeutics Inc. (CLDX) | CDX-1307 | A dendritic cell-targeted immunotherapy, combined with granulocyte macrophage colony-stimulating factor | Breast, bladder, pancreatic or colorectal cancer | Phase I data showed it was well tolerated at all doses via both intradermal and intravenous injection without any dose-limiting toxicity (11/3) |
| Cougar Biotechnology Inc.(CGRB) | CB7630 | Abiraterone acetate; an orally active inhibitor of the steroidal enzyme 17alpha-hydroxylase/C17,20 lyase | Advanced breast cancer | First patient was enrolled in a Phase I/II trial (11/20) |
| CytRx Corp. (CYTR) | INNO-406 | Orally available dual Bcr-Abl and Lyn-kinase inhibitor | Chronic myeloid leukemia | Demonstrated clinical responses in patients who were intolerant to or resistant to Gleevec and, in some cases, another Bcr-Abl inhibiting tyrosine kinase inhibitor (11/11) |
| Cytopia Ltd. (Australia; ASX:CYT) | CYT997 | A vascular-disrupting anticancer agent | Glioblastoma multiforme | Commenced dosing in a Phase Ib/II study (11/17) |
| Dendreon Corp. (DNDN) | Neuvenge | Lapuleucel-T, an active cellular immunotherapy | HER2/neu-positive cancer | Phase I data showed the treatment was generally well tolerated and the median T-cell proliferative stimulation index increased (11/6) |
| EntreMed Inc. (ENMD) | MKC-1 | An orally active cell-cycle inhibitor | Non-small-cell lung cancer | MKC-1 in combination with pemetrexed met the primary endpoint of tumor response in the open-label Phase I/II study (11/10) |
| Lexicon Pharmaceuticals Inc. (LXRX) | LX1032 | An orally delivered small-molecule drug candidate | To manage gastrointestinal symptoms associated with carcinoid syndrome | Completed a Phase Ib trial, demonstrating a potent reduction in both blood serotonin levels and urinary 5-HIAA (11/13) |
| Medarex Inc. (MEDX) | MDX-1106 | Human anti-PD-1 antibody | Cancer | Started a Phase Ib trial (11/24) |
| Pharmacyclics Inc. (PCYC) | PCI-27483 | Small-molecule Factor VIIa inhibitor | Cancer | Phase I data showed the drug induced therapeutic levels of anticoagulation in a dose-dependent manner without adverse events (11/25) |
| Poniard Pharmaceuticals Inc. (PARD) | Picoplatin | Oral formulation | Solid tumors | Phase I data showed it achieves linear and dose-dependent plasma exposure when given by the oral route (11/6) |
| Provectus Pharmaceuticals Inc. (OTC BB:PVCT) | PV-10 | Provecta | Melanoma | Phase I data showed a continued statistically significant difference in survival between those subjects achieving an objective response of their injected melanoma lesions, with a median survival of 30.7 months, vs. patients who failed to respond to PV-10 treatment, with a median survival of 12.3 months (11/20) |
| SuperGen Inc. (SUPG) | SGI-1776 | An orally administered inhibitor of Pim kinases | Solid tumors | Received clearance to initiate a Phase I trial (11/20) |
| Sunesis Pharmaceuticals Inc. (SNSS) | Voreloxin | Formerly SNS-595 | Acute leukemias | Phase I data showed it was well tolerated, with its most frequently observed dose-limiting toxicity being reversible grade 3/4 oral mucositis; in a Phase Ib/II study three of 11 patients achieved a complete remission (11/6) |
| Synta Pharmaceuticals Corp. (SNTA) | Elesclomol | An oxidative stress inducer | Metastatic hormone-refractory prostate cancer | Treated the first patient in a Phase I/II trial (11/20) |
| Ziopharm Oncology Inc. (ZIOP) | Zymafos | Palifosfamide | Soft tissue sarcoma | Phase I data showed it was well tolerated in combination with doxorubicin (11/17) |
| CARDIOVASCULAR | ||||
| Celladon Corp.* | Mydicar | A recombinant adeno-associated viral vector | Heart failure | Demonstrated an acceptable safety profile in the first nine patients treated in a Phase I trial (11/10) |
| Cerus Corp. (CERS) | Intercept | Intercept Blood System for red blood cells | For use in blood transfusions | Started a Phase I trial (11/24) |
| NicOx SA (France; Paris: COX) | Naproxcinod | A first in the cycloxygenase-inhibitng nitric oxide donator | Blood pressure | Clinical data were in favor of naproxcinod vs. naproxen (11/12) |
| Resverlogix Corp. (Canada; TSX:RVX) | RVX-208 | A novel small-molecule therapeutic that facilitates endogenous ApoA-1 production | Atherosclerosis and vascular disorders | Data from an 80-patient Phase Ia trial showed that RVX-208 increased the change for ApoA-1 by 11% in treated subjects compared to placebo following administration for seven days (11/13); successfully completed the second arm of its Phase Ib/IIa study (11/28) |
| CENTRAL NERVOUS SYSTEM | ||||
| Oxford BioMedica plc (UK; LSE:OXB) | ProSavin | A gene-based therapeutic | Parkinson's disease | Phase I/II data showed that the three patients treated with the low dose indicated ProSavin is safe and well tolerated, with all three patients ambulatory within 48 hours (11/14) |
| Paion AG (Germany; LSE:PAI) | CNS 7056 | A short-acting sedative and anesthetic | For use as a sedative | Completed a double-blind, placebo-controlled, 81-patient Phase I trial (11/19) |
| Phosphagenics Ltd. (Australia; LSE:PSG) | TPM | Drug delivery system for the targeted delivery of a leading non-steroidal anti-inflammatory drug, diclofenac | Inflammation | Started a Phase I trial (11/12) |
| DIABETES | ||||
| Living Cell Technologies Ltd. (Australia; PK:LVCLF) | DiabeCell | An encapsulated porcine insulin-producing cell product | Type I diabetes | Phase I/IIa data showed that of two patients followed for a year, one maintained HbA1c at the ideal level of 6.7% with less insulin and patient two did not require insulin for five months after the first implant and now uses 36% less insulin daily than before the implant (11/6) |
| Metabolex Inc.* | MBX-2982 | Agonist of GPR119, a G-protein-coupled receptor | Type II diabetes | Phase Ia data showed it had excellent exposure and exhibited a half-life consistent with once-daily dosing (11/12) |
| INFECTION | ||||
| Anadys Pharmaceuticals Inc. (ANDS) | ANA598 | Oral, Toll-like receptor 7 agonist prodrug ANA773 | Hepatitis C virus | Phase I data showed that it was well tolerated with no serious adverse events (11/3) |
| Inovio Biomedical Corp. (AMEX:INO) and Tripep AB (Sweden; SSE:TPEP) | ChronVac-C | DNA-based vaccine | Hepatitis C virus | Two of three subjects in the highest dose cohort of the Phase I/II study demonstrated reductions in viral load of 93% and 99.7% (11/17) |
| InterMune Inc. (ITMN), Pharmasset Inc. (VRUS) and F. Hoffmann-La Roche Ltd. (Switzerland) | R7227 and R7128 | A protease inhibitor and a polymerase inhibitor | Hepatitis C virus | The first patients have been dosed in a trial to investigate the combination of two oral antiviral molecules in the absence of interferon (11/10) |
| NasVax Ltd. (Israel; TASE:NSVX) | VaxiSome | Adjuvanted influenza vaccine | Influenza | Started a second Phase I/IIa study (11/3) |
| PhotoCure ASA (Norway; OSLO: PHO) | Cevira | Photodynamic therapy | Precancerous lesions of the cervix induced by human papilloma virus infections | Phase I/II data from a trial in Norway showed that 73% of patients were treated successfully (11/17) |
| RegeneRx Biopharmaceuticals Inc. (AMEX:RGN) | RGN-259 | A sterile eye-drop formulation of TB4 | Ophthalmic wounds and related disorders caused by the herpes zoster virus | In all four patients, the eye ulcers either completely healed or demonstrated significant improvement by treatment day 28 (11/17) |
| MISCELLANEOUS | ||||
| Aldagen Inc.* | ALD-301 | A stem cell therapy | Critical limb ischemia | Phase I/II data showed the compound was well tolerated (11/20) |
| Ambrx Inc.* | ARX201 | A long-acting recombinant human growth hormone drug candidate | Growth hormone deficiencies | Phase I/II data demonstrated that ARX201 normalized insulin-like growth factor 1 levels while delivering an acceptable safety and tolerability profile in adults (11/13) |
| Mesoblast Ltd. (Australia; ASX:MSB) | | Allogeneic mesenchymal precursor cells | Hematologic malignancies undergoing bone marrow transplantation | Started a Phase I/II trial (11/5) |
| Ophthotech Corp.* | Volociximab | A monoclonal antibody targeting alpha4beta1 integrin | Wet age-related macular degeneration | Treated the first patient in a Phase I trial (11/10) |
| Potentia Pharmaceuticals Inc.* | POT-4 | A complement inhibitor | Wet and dry age-related macular degeneration | Interim Phase I data showed it was safe with no drug-related toxicity thus far (11/13) |
| Notes: | ||||
| * Privately held. | ||||
| 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; OTC BB = Over-the-Counter Bulletin Board; PK = Pink Sheets; SSE = Stockholm Stock Exchange; TASE = Tel Aviv Stock Exchange; TSX = Toronto Stock Exchange. | ||||
