Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Anacor Pharmaceuticals Inc. (Palo Alto, Calif.) |
A27278 |
Boron-based phosphodiesterase-4 inhibitor |
Mild to moderate atopic dermatitis |
Phase II data disclosed positive results showing that 74% of patients achieved an Investigator Static Global Assessment (ISGA) score of zero or 1 after four weeks of treatment, and 35% achieved an ISGA score of zero or 1 with a minimum two-grade improvement after four weeks |
12/12/12 |
Idera Pharmaceuticals Inc. (Cambridge, Mass.) |
IMO-3100 |
A selective antagonist of Toll-like receptors 7 and 9 |
Moderate to severe plaque psoriasis |
48% of patients demonstrated improvements in Psoriasis Area Severity Index (PASI) scores of 35% to 90% from baseline at the completion of a 44-patient Phase IIa study testing two dose levels of the drug administered for four weeks, with a four-week follow-up period |
12/20/12 |
Rigel Pharmaceuticals Inc. (South San Francisco) and AstraZeneca plc (London) |
Fostamatinib |
An oral inhibitor of spleen tyrosine kinase (Syk) |
Rheumatoid arthritis |
Phase IIb data showed that the drug was superior to placebo at six weeks, but it fell short of a secondary goal of noninferiority against Humira |
12/13/12 |
XBiotech Inc. (Austin, Texas) |
MABp1 |
True Human monoclonal antibody candidate |
Moderate to severe acne vulgaris |
Phase II data demonstrated that it produced continual improvement in lesions over the course of therapy, with up to a 42% reduction in eight weeks |
12/13/12 |
CANCER | |||||
AVEO Oncology Inc. (Cambridge, Mass.) and Astellas Pharma Inc. (Tokyo) |
Tivozanib |
A drug designed to inhibit all three vascular endothelial growth factor receptors |
Triple-negative breast cancer |
Began a Phase II study |
12/4/12 |
Cell Therapeutics Inc. (Seattle) |
Brostallicin |
Cancer therapy |
Metastatic triple-negative breast cancer |
Phase II data of brostallicin in combination with cisplatin showed nine patients had a partial response and one had a confirmed response |
12/7/12 |
Geron Corp. (Menlo Park, Calif.) |
GRN1005 |
A peptide-drug conjugate; targets the low-density lipoprotein receptor-related protein LRP-1 |
Brain cancer |
Discontinued development after an interim analysis of its Phase II study showed no intracranial responses in the first 30 evaluable patients |
12/5/12 |
Gliknik Inc. (Baltimore) |
GL-0817 |
MAGE-A3 immunomodulator |
Multiple |
Interim Phase II data showed a 180-day rate of complete or partial response of 96% |
12/21/12 |
Inovio Pharmaceuticals Inc. (Blue Bell, Pa.) |
WT1 DNA vaccine |
Delivered with Inovio's electroporation delivery system |
Leukemia |
Phase II data showed vaccine-specific antibody responses |
12/7/12 |
OncoGenex Pharmaceuticals Inc. (Bothell, Wash.) |
OGX-427 |
A heat-shock protein 27 inhibitor |
Metastatic castrate-resistant prostate cancer |
Started a Phase II study, dubbed PACIFIC, in patients who are experiencing a rising prostate-specific antigen (PSA) while receiving Zytiga |
12/20/12 |
Spectrum Pharmaceuticals Inc. (Henderson, Nev.) |
Belinostat |
A pan-histone deacetylase (HDAC) inhibitor |
Relapsed/refractory peripheral T-cell lymphoma |
Hit the primary endpoint in a Phase II trial |
12/26/12 |
Viralytics Ltd. (Melbourne, Australia) |
Cavatak |
Virotherapy candidate |
Melanoma |
Achieved an interim efficacy milestone of three objective responses in its ongoing U.S.-based Phase II trial; the goal was hit with only 13 patients recruited |
12/14/12 |
CARDIOVASCULAR | |||||
Inspiration Biopharma-ceuticals Inc. (Cambridge, Mass.) |
FVIII (OBI-1) |
Intravenous recombinant porcine |
Hemophilia A |
Phase II/III interim data showed successful control of serious hemorrhage at 24 hours after the initial dose, and subsequent resolution of bleeding |
12/14/12 |
Inspiration Biopharma-ceuticals Inc. (Cambridge, Mass.) |
IB1001 |
Recombinant Factor IX |
Hemophilia B |
A study showed more rapid metabolism of Factor IX and a shorter terminal half-life in pediatric patients compared with previous findings in adults |
12/14/12 |
Portola Pharmaceuticals Inc. (South San Francisco) |
PRT4445 |
Universal antidote for Factor Xa inhibitor anticoagulants |
To reverse anticoagulants |
Started a Phase II study |
12/11/12 |
ViroPharma Inc. (Exton, Pa.) and Halozyme Therapeutics Inc. (San Diego) |
Cinryze |
C1 esterase inhibitor [human] in combination with Halozyme's Enhanze technology |
Hereditary angioedema |
Started a Phase IIb dose-ranging study to test the subcutaneous administration |
12/20/12 |
CENTRAL NERVOUS SYSTEM | |||||
Biotie Therapies Corp. (Turku, Finland) |
SYN115 |
Tozadenant; adenosine A2a antagonist |
Parkinson's disease |
Phase IIb trial showed it met the primary endpoint of decreasing the "off" time in Parkinson's patients experiencing levodopa-related end-of-dose wearing off vs. placebo; the drug also demonstrated efficacy across multiple secondary endpoints |
12/12/12 |
Naurex Inc. (Evanston, Ill.) |
GLYX-13 |
A partial agonist of the N-Methyl-D-Aspartate (NMDA) receptor |
Depression |
Phase IIa data showed statistically significant reductions in depression scores in patients who had failed treatment with one or more antidepressant agents |
12/7/12 |
Neurocrine Biosciences Inc. (San Diego) |
NBI-98854 |
Vesicular monoamine transporter 2 compound |
Moderate to severe tardive dyskinesia and underlying mood disorders, gastrointestinal disorders and schizophrenia or schizoaffective disorder |
Started a Phase IIb trial |
12/20/12 |
Spinifex Pharmaceuticals Pty. (Melbourne, Australia) |
EMA401 |
Angiotensin II Type II receptor antagonist |
Chemotherapy-induced peripheral neuropathy |
The first patients were treated in its Phase II study |
12/11/12 |
Transcept Pharmaceuticals Inc. (Point Richmond, Calif.) |
TO-2061 |
A low-dose form of ondansetron |
Obsessive compulsive disorder |
Top-line Phase II data showed it failed to meet its primary efficacy endpoint of improving OCD symptoms compared to placebo |
12/26/12 |
DIABETES | |||||
Tranzyme Pharma Inc. (Research Triangle Park, N.C.) |
TZP-102 |
Oral gastrointestinal motility drug that targets the ghrelin receptor |
Diabetic gastroparesis |
A second Phase IIb trial is being discontinued after an interim futility analysis showed a large placebo effect and no treatment effect |
12/18/12 |
INFECTION | |||||
Cempra Inc. (Chapel Hill, N.C.) |
CEM-102 |
A loading dose formulation of fusidic acid |
Prosthetic joint infections |
Started a Phase II trial |
12/17/12 |
Infectex (Moscow) |
SQ109 |
Diamine compound |
Multidrug-resistant tuberculosis |
Enrolled the first patients in a Phase II/III trial |
12/20/12 |
Medivir AB (Huddinge, Sweden) |
GS7977 |
An interferon-free regimen combining simeprevir and sofosbuvir |
Hepatitis C virus |
Began a second cohort in a Phase II trial, which will include genotype 1 treatment-naive and null responders with advanced hepatic fibrosis |
12/14/12 |
MISCELLANEOUS | |||||
Flexion Therapeutics Inc. (Woburn, Mass.) |
FX006 |
Intra-articular sustained-release steroid |
Osteoarthritis |
Maintained therapeutic concentrations in the knee joint significantly longer than the most commonly prescribed immediate-release steroid, triamcinolone acetonide, in a Phase II pharmacokinetic study |
12/18/12 |
Noxxon Pharma AG (Berlin) |
NOX-H94 |
Anti-hepcidin Spiegelmer |
Anemia associated with chronic disease |
Treated the first patients in a Phase IIa trial |
12/5/12 |
Pharmalink AB (Stockholm, Sweden) |
Nefecon |
Nefigan |
IgA nephropathy |
Began a Phase IIb trial |
12/19/12 |
Sarepta Therapeutics Inc. (Cambridge, Mass.) |
Eteplirsen |
Exon-skipper |
Duchenne's muscular dystrophy |
Phase IIb extension data showed that patients treated with eteplirsen for 62 weeks and evaluable on ambulatory measures (modified intent-to-treat population) maintained a statistically significant clinical benefit on the primary clinical outcome measure, the six-minute walk test, compared to patients who received placebo for 24 weeks followed by 38 weeks of eteplirsen treatment |
12/10/12 |
Synergy Pharmaceuticals Inc. (New York) |
Plecanatide |
A synthetic analogue of uroguanylin |
Constipation-predominant irritable bowel syndrome |
Dosing has commenced in a Phase IIb trial |
12/28/12 |
Unigene Laboratories Inc. (Boonton, N.J.) |
PTH |
Oral parathyroid hormone |
Osteoporosis in postmenopausal women |
Complete efficacy and safety findings from the Phase II study showed clinically relevant mean increases in bone mineral density at the lumbar spine of 2.2% (p<0.001) at week 24 compared to baseline |
12/13/12 |
Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. The date indicated refers to the BioWorld Today issue in which the news item can be found. |