Company (Location) |
Product |
Description |
Indication |
Status (Date)# |
AUTOIMMUNE | ||||
Anthera Pharmaceuticals Inc. (Hayward, Calif.) |
Blisibimod |
A peptibody drug designed to act as a broad inhibitor of B-cell activating factor |
Systemic lupus erythematosus |
Phase IIb data showed that weekly and monthly subcutaneous doses of blisibimod resulted in statistically significant reductions of B cells (12/2) |
Metabolex Inc. (Hayward, Calif.) and Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) |
Arhalofenate |
A PPAR gamma agonist; a uricosuric agent |
Gout |
Open-label clinical pharmacology study showed that the higher dose of arhalofenate plus Takeda's Uloric increased response rate by 73%; the combination was well tolerated (12/9) |
Neovacs SA (Paris) |
TNF-Kinoid |
Targets TNF-mediated chronic inflammatory diseases |
Crohn's disease |
Completed enrollment in the first phase of its double-blind, placebo-controlled, international Phase II trial (12/16) |
XOMA Ltd. (Berkeley, Calif.) |
XOMA 052 |
Gevokizumab |
Acne vulgaris |
XOMA began dosing patients in a Phase II proof-of-concept trial (12/22) |
CANCER | ||||
Acacia Pharma Ltd. (Cambridge, Mass.) |
APD209 |
An oral formulation of a currently marketed drug |
Cancer cachexia |
Phase IIa data showed six of seven patients achieved a major response, which included an increase in quadricep muscle size of at least 4% (12/7) |
AstraZeneca plc (London) |
Olaparib |
PARP inhibitor |
Ovarian cancer |
Will not progress into Phase III after an interim analysis of a Phase II study indicated a progression-free survival benefit is unlikely to translate into an overall survival benefit (12/21) |
AVEO Pharmaceuticals Inc. (Cambridge, Mass.) |
Tivozanib |
VEGF inhibitor |
Advanced metastatic colorectal cancer |
Started a Phase II trial comparing VEGF inhibitor tivozanib plus modified FOLFOX6 to Avastin plus mFOLFOX6 as first-line therapy (12/27) |
Celldex Therapeutics Inc. (Needham, Mass.) |
CDX-011 |
Glembatamumab vedotin |
Metastatic breast cancer |
Completed accrual for its Phase IIb trial (12/22) |
Cyclacel Pharmaceuticals Inc. (Berkeley Heights, N.J.) |
Sapacitabine |
A nucleoside analogue |
Non-small-cell lung cancer; breast, ovarian, pancreatic and other cancers |
Interim topline Phase II data in lung cancer, and Phase I data in combination with seliciclib in the other cancers, showed partial responses and stable disease; there also were responders in the Phase I trial (12/8) |
CytRx Corp. (Los Angeles) |
INNO-206 |
Tumor-targeting doxorubicin conjugate |
Soft tissue sarcoma |
Started a Phase IIb trial comparing INNO-206 to native doxorubicin (12/27) |
Emergent BioSolutions Inc. (Rockville, Md.) |
TRU-016 |
Humanized anti-CD37 mono-specific protein therapeutic |
Relapsed chronic lymphocytic leukemia |
Started a Phase II study in combination with bendamustine (12/29) |
Endocyte Inc. (West Lafayette, Ind.) |
EC145 |
A small-molecule conjugate combining vitamin folate with vinca alkaloid |
Ovarian cancer |
Data showed it lost statistical significance in the intent-to-treat population, and statistical significance was maintained only in a subset of patients whose lesions all tested positive for folate receptor (12/14) |
Galena Biopharma Inc. (Lake Oswego, Ore.) |
NeuVax |
Vaccine |
Breast cancer |
Five-year efficacy data from an ongoing Phase II trial showed it is safe and well tolerated, and demonstrated efficacy in preventing breast cancer recurrence (12/8) |
Geron Corp. (Menlo Park, Calif.) |
GRN1005 |
An LRP-directed peptide-drug conjugate that combines paclitaxel with peptide Angiopep-2 |
Breast cancer-derived brain metastases; non-small-cell lung cancer |
Started a Phase II trial (12/13); began a Phase II in non-small-cell lung cancer patients with brain metastases (12/27) |
Jennerex Inc. (San Francisco) |
JX-594 |
Adjuvant therapy |
Colorectal cancer metastasized to the liver |
Treated its first patient in a Phase IIa trial (12/22) |
Marinus Pharmaceuticals Inc. (Branford, Conn.) |
Ganaxolone |
Neurosteroid |
Partial onset seizures |
Phase II data demonstrated an overall decrease of 23.2% in median weekly seizure frequency from baseline (12/22) |
Oncolytics Biotech Inc. (Calgary, Alberta) |
Reolysin |
Intravenously administered |
Advanced pancreatic cancer |
Interim Phase II data showed Reolysin in combination with Gemzar met the study's primary endpoint (12/5) |
Peregrine Pharmaceuticals Inc. (Tustin, Calif.) |
Bavituximab |
Targets the blood vessels of tumors as opposed to a binding site on the cancer cell |
Non-small-cell lung cancer |
Phase II data showed a 50% improvement in overall response rate (12/7) |
SymBio Pharmaceuticals Ltd. (Tokyo) |
SyB L-0501 |
Bendamustine hydrochloride |
Non-Hodgkin's lymphoma and mantle cell leukemia |
Began an open-label Phase II trial (12/28) |
CARDIOVASCULAR | ||||
Furiex Pharmaceuticals Inc. (Morrisville, N.C.) |
PPD-10558 |
A novel statin |
Dyslipidemia in statin-associated myalgia |
Top-line Phase II data showed it did not meet its primary efficacy endpoint (12/12) |
Oxigene Inc. (South San Francisco) |
Zybrestat |
Intravenous injection |
Polypoidal choroidal vasculopathy |
Phase II data showed it was well tolerated, with a decrease in polyp activity and reduction of subretinal fluid and retinal edema compared to placebo (12/8) |
Pearl Therapeutics Inc. (Redwood City, Calif.) |
PT001 |
Long-acting muscarinic agonist drug glycopyrrolate via metered dose inhaler |
Chronic obstructive pulmonary disease |
Phase IIb data showed statistically significant improvements in lung function compared to placebo; PT001 was safe and well tolerated, and at some doses was noninferior to Atrovent HFA Inhalation aerosol (12/8) |
Sygnis Pharma AG (Heidelberg, Germany) |
AX200 |
A recombinant version of the naturally occurring protein granulocyte colony-stimulating factor |
Acute ischemic stroke |
The drug failed to demonstrate efficacy in a 328-patient Phase IIb trial (12/20) |
ThromboGenics NV (Leuven, Belgium) and BioInvent International AB (Lund, Sweden) |
TB-402 |
Long-acting anticoagulant |
Venous thromboembolism |
Completed enrollment in a 632-patient Phase IIb trial (12/19) |
ViroPharma Inc. (Exton, Pa.) and Halozyme Therapeutics Inc. (San Diego) |
Cinryze |
Subcutaneous version of the C1 esterase inhibitor |
Hereditary angioedema |
Top-line data from a Phase II trial showed the addition of Halozyme's Enhanze deliver technology led to maximum levels and greater systemic exposure of functional and antigenic C1 inhibitor for both doses tested as compared to subcutaneous administration of Cinryze alone (12/7) |
CENTRAL NERVOUS SYSTEM | ||||
Aestus Therapeutics Inc. (East Windsor, N.J.) |
ATx08-001 |
Selective PPAR modulator |
Neuropathic pain therapeutic |
Positive Phase II data showed it met the primary objective of demonstrating significant analgesia and showed an excellent safety profile (12/14) |
Ceregene Inc. (San Diego) |
CERE-120 |
A gene therapy product |
Parkinson's disease |
Completed enrollment in a Phase IIb trial (12/2) |
Cytokinetics Inc. (South San Francisco) |
CK-2017357 |
A selective fast skeletal muscle troponin complex activator |
Amyotrophic lateral sclerosis |
Phase II data showed the drug was well tolerated when dosed daily as a single agent for two weeks (12/1) |
EnVivo Pharmaceuticals Inc. (Watertown, Mass.) |
EVP-6124 |
An oral nicotinic alpha-7 agonist |
Schizophrenia |
Phase IIb data showed statistically significant and clinically meaningful effects on both cognition and functional symptoms including global cognitive function (12/6) |
Prana Biotechnology Ltd. (Melbourne, Australia) |
PBT2 |
A metal-protein attenuating compound |
Alzheimer's disease |
Began to recruit and screen patients for a 12-month Phase II imaging trial testing PBT2 compared to placebo (12/16) |
Shire plc (Dublin, Ireland) |
Vivanse |
Lisdexamfetamine dimeslate; adjunctive therapy |
Major depressive disorder |
Phase II data showed the study met its primary endpoint of change from baseline (12/9) |
Targacept Inc. (Winston-Salem, N.C.) |
TC-5619 |
A selective modulator of the alpha 7 neuronal nicotinic receptor |
Schizophrenia and ADHD |
Started two trials, a Phase IIb study to test it for negative symptoms and cognitive dysfunction in schizophrenia, and a Phase II study in attention deficit hyperactivity disorder (12/6) |
Theravance Inc. (South San Francisco) |
TD-9855 |
A norepinephrine and serotonin reuptake inhibitor |
Attention deficit hyperactivity disorder |
Started a Phase II study (12/13) |
XenoPort Inc. (Santa Clara, Calif.) |
XP21279 |
A transported prodrug of levodopa, co-formulated with carbidopa |
Advanced Parkinson's disease |
Phase II trial fell short of showing statistically significant improvements over Sinemet (12/6) |
DIABETES | ||||
ChemoCentryx Inc. (Mountain View, Calif.) |
CCX140 |
A CCR2 antagonist |
Diabetic kidney disease |
Began a Phase II trial (12/9) |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Androxal |
Oral therapy |
Hypogonadal men with Type II diabetes |
Phase II data showed it significantly increased testosterone levels at both the low and high doses vs. placebo; there was a trend toward improved glycemic control in the men on the higher dose (12/14) |
Zealand Pharma A/S (Copenhagen, Denmark) and Sanofi SA (Paris) |
Lyxumia |
Lixisenatide; GLP-1 agonist |
Type II diabetes |
Phase II data dhowed it produced significantly greater postprandial glucose lowering than Victoza (12/6) |
INFECTION | ||||
Anacor Pharmaceuticals Inc. (Palo Alto, Calif.) |
AN2728 and AN2898 |
Boron-based phosphodiesterase-4 inhibitors |
Atopic dermatitis |
Phase IIa data showed each achieved their primary endpoint (12/14) |
BioMarin Pharmaceutical Inc. (Novato, Calif.) |
GALNS |
N-acetylgalactos-amine 6-sulfatase |
Mucopoly-saccharidosis IVA |
Started a Phase II study (12/1) |
Biotron Ltd. (Sydney, Australia) |
BIT225 |
Designed to target the p7 protein |
Hepatitis C virus |
Phase IIa trial showed that 87% of BIT225 subjects achieved a complete early viral response, defined as virus levels in the blood below the level of detection, compared to 63% of SOC patients (12/9) |
Hatchtech Pty Ltd. (Melbourne, Australia) |
DeOvo |
Topical formulation; an inhibitor of metalloproteinases |
Head lice infestation |
Phase IIb data suggested the compound is safe and effective following a single application (12/16) |
Pharmasset Inc. (Princeton, N.J.) |
PSI-938 |
Guanine nucleotide analogue |
Hepatitis C virus |
Pharmasset dropped PSI-938 from its Phase IIb QUANTUM trial after routine monitoring unveiled laboratory abnormalities associated with liver function (12/19) |
PolyMedix Inc. (Radnor, Pa.) |
PMX-30063 |
Antibiotic |
Skin infections caused by S. aureus |
Interim Phase II analysis showed it was safe and effective in all dosing arms, with a combined clinical cure rate of 92% (12/8) |
Rib-X Pharmaceuticals Inc. (New Haven, Conn.) |
Delafloxacin |
Antibiotic |
Acute bacterial skin and skin structure infections |
Top-line Phase IIb data indicated it met or exceeded primary and secondary efficacy endpoints when compared to Zyvox (12/16) |
MISCELLANEOUS | ||||
Amicus Therapeutics Inc. (Cranbury, N.J.) |
AT2220 |
Duvoglustat HCl |
Pompe disease |
Treated the first patient in an open-label Phase II drug-drug interaction study of AT2220 co-administered with enzyme replacement therapy (12/2) |
Apeiron Biologics AG (Vienna, Australia) and GlaxoSmithKline plc (London) |
GSK2586881 |
Recombinant human Angiotensin Converting Enzyme 2 |
Acute lung injury |
Started a Phase IIa study (12/20) |
Auxilium Pharmaceuticals Inc. (Malvern, Pa.) |
Xiaflex |
Collagenase clostridium histolyticum |
Idiopathic adhesive capsulitis of the shoulder |
The first patient was dosed in its Phase IIa trial (12/2) |
Chelsea Therapeutics International Ltd. (Charlotte, N.C.) |
Northera |
Droxidopa |
Fibromyalgia |
Phase II trial showed the highest dose tested of droxidopa, 600 mg TID, resulted in a 6.2-point improvement from a baseline and a 3.2-point pain improvement over placebo in a questionnaire (12/27) |
Impax Pharmaceuticals (Hayward, Calif.) |
IPX159 |
An oral controlled-release formulation of a small molecule |
Severe restless legs syndrome |
Started a Phase IIb trial (12/20) |
NovaBay Pharmaceuticals Inc. (Emeryville, Calif.) |
NVC-422 |
Aganocide compound |
Spinal cord injury |
Advanced its Phase II program to treat urinary catheter blockage and encrustation in patients with spinal cord injury after Part A of the study met its goal of establishing primary proof of concept (12/1) |
Protox Therapeutics Inc. (San Diego) |
PRX302 |
A genetically engineered version of the pore forming protein proaerolysin designed to disrupt membrane integrity |
Benign prostatic hyperplasia |
Completed dosing of the second cohort in the transrectal study (12/27) |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Androxal |
Oral therapy designed to act at the level of the hypothalamic-pituitary axis |
Secondary hypogonadism |
Top-line Phase II data showed statistically significant and clinically relevant improvement in testosterone production compared to placebo and equivalent to topical testosterone product Testim; Androxal maintained sperm counts while Testim reduced counts (12/28) |
Soligenix Inc. (Princeton, N.J.) |
orBEC |
Oral beclomethasone dipropionate |
Graft-vs.-host disease |
Phase II data showed the drug was well tolerated but did not achieve statistical significance in reducing graft-vs.-host disease following hematopoietic cell transplantation (12/13) |
Taris Biomedical Inc. (Lexington, Mass.) |
LiRIS |
A product designed to deliver lidocaine over an extended period directly to the bladder |
Interstitial cystitis |
Started a Phase II study (12/1) |
|
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. | ||||