Company (Location) |
Product |
Description |
Indication |
Status (Date)# |
AUTOIMMUNE | ||||
Adeona Pharmaceuticals Inc. (Ann Arbor, Mich.) |
Trimesta |
Oral estriol |
Relapsing-remitting multiple sclerosis |
Enrolled the 150th patient in its Phase II study (9/20) |
Vertex Pharmaceuticals Inc. (Cambridge, Mass.) |
VX-509 |
JAK3 inhibitor |
Rheumatoid arthritis |
Showed positive results in a Phase IIa trial (9/7) |
CANCER | ||||
4SC AG (Planegg-Martinsried, Germany) |
Resminostat |
Histone deacetylase inhibitor |
Hodgkin's lymphoma |
Attained the primary endpoint of a Phase II trial (9/7) |
Aeterna Zentaris Inc. (Quebec City) |
AEZS-108 |
Targeted cytotoxic luteinizing hormone-releasing hormone analogue |
Advanced endometrial cancer |
Final Phase II data showed that, in 43 evaluable patients, there were two complete responses, 10 partial responses and 17 patients with disease stabilization, for an estimated overall response rate of 30.8% and a clinical benefit rate of 74.4% (9/15) |
Alchemia Ltd. (Brisbane, Australia) |
HA-Irinotecan |
Topoisomerase I inhibitor |
Small-cell lung cancer |
Began recruitment for a Phase II trial (9/13); Phase II data showed tumor shrinkage in the first two patients dosed (9/29) |
Apogenix GmbH (Heidelberg, Germany) |
APG101 |
Inhibitor of CD95 ligand |
Glioblastoma |
Completed patient recruitment for a Phase II trial (9/21) |
BioAlliance Pharma SA (Paris) |
Livatag |
Liver cancer product |
Liver cancer |
Showed increased survival in a Phase II trial (9/7) |
BioAlliance Pharma SA (Paris) |
AMEP |
Biotherapy designed to target integrins; delivered via intramuscular administration |
Metastatic melanoma |
Phase I data showed it was safe at the 0.5-mg and 1-mg doses assessed, and stabilization of tumor growth was obtained in 60% of lesions treated (9/14) |
Jennerex Inc. (South San Francisco) |
JX-594 |
Liver cancer candidate |
Liver cancer |
JX-594 followed by sorafenib induced tumor responses that were maintained for up to 15 months (9/7) |
Nektar Therapeutics Inc. (San Francisco) |
NKTR-102 |
A topoisomerase I inhibitor-polymer conjugate |
Breast cancer |
Showed a high response rate and strong clinical benefit in a Phase II trial, even in subsets of patients with a poor prognosis (9/13) |
OncoGenex Pharmaceuticals Inc. (Bothell, Wash.) |
OGX-011 |
Clusterin inhibitor custirsen |
Metastatic castrate-resistant prostate cancer |
Phase II data showed the drug in combination with docetaxel retreatment resulted in overall survival of 15.8 months (9/7) |
Oncothyreon Inc. (Seattle) |
PX-866 |
Targets phosphatidylinositol-3-kinase |
Prostate cancer |
Enrolled its first patient in a Phase II trial (9/13) |
Peregrine Pharmaceuticals Inc. (Tustin, Calif.) |
Bavituximab |
A phosphatidylserine-targeting monoclonal antibody |
Non-small-cell lung cancer |
Completed enrollment of a targeted 86 patients in its Phase II trial of bavituximab plus carboplatin and paclitaxel (9/9) |
Rexahn Pharmaceuticals Inc. (Rockville, Md.) |
Archexin |
An Akt protein kinase inhibitor |
Metastatic pancreatic cancer |
Completed enrollment in its Phase II trial (9/29) |
Syndax Pharmaceuticals Inc. (Waltham, Mass.) |
Exemestane |
Hormone therapy |
Metastatic breast cancer |
Phase II data of exemestane, with and without the company's HDAC inhibitor entinostat, hit the primary endpoint of progression-free survival (9/7) |
Telik Inc. (Palo Alto, Calif.) |
Telintra |
Oral ezatiostat; a GST P1-1 inhibitor |
Myelodysplastic syndrome |
Phase II data showed a 63% hematologic improvement-erythroid response rate in a group of 19 patients who were transfusion-dependent and Dacogen and Vidaza naive (9/30) |
CARDIOVASCULAR | ||||
Aastrom Biosciences Inc. (Ann Arbor, Mich.) |
Ixmyelocel-T |
A patient-specific multicellular therapy |
Dilated cardiomyopathy |
12-month follow-up data from the 40-patient Phase II trial showed treatment was well tolerated and efficacy observations were consistent with improved function of imparied myocardium (9/20) |
Action Pharma A/S (Aarhus, Denmark) |
AP214 |
Designed to target the melanocortin receptors |
For patients undergoing major cardiac surgery |
Top-line data for its Phase IIb trial indicated it is safe, reduces kidney injury and improves 90-day outcomes on a composite end point (9/27) |
HemaQuest Pharmaceuticals Inc. (San Diego) |
HQK-1001 |
An oral drug |
Sickle cell disease |
Completed enrollment in a Phase II study (9/13) |
ProFibrix BV (Leiden, the Netherlands) |
Fibrocaps |
Based on a mixture of blood-clotting proteins fibrinogen and thrombin |
Surgical hemostasis |
Completed patient enrollment in its two Phase II studies (9/14) |
Resverlogix Corp. (Calgary, Alberta) |
RVX-208 |
Small molecule |
To raise apolipoprotein A-1 production |
Phase II data showed it enhanced biomarkers of reverse cholesterol transport, thus reflecting robust cholesterol clearance from arterial wall plaques via that metabolic pathway (9/2); opened sites for the Phase IIb trial (9/27); initiated dosing in the Phase IIb (9/28) |
Theratechnol-ogies Inc. (Montreal) |
Tesamorelin |
A stabilized analogue of the growth hormone-release factor |
Muscle wasting in chronic obstructive pulmonary disease |
Began patient screening for a Phase II trial (9/7) |
ViroPharma Inc. (Exton, Pa.) and Halozyme Therapeutics Inc. (San Diego) |
Cinryze |
C1 esterase inhibitor |
Hereditary angioedema |
Started a Phase II study (9/14) |
CENTRAL NERVOUS SYSTEM | ||||
Adamas Pharmaceuticals Inc. (Emeryville, Calif.) |
ADS-5102 |
Amantadine HCL extended release |
Levodopa-induced dyskinesia in Parkinson's disease |
Began a Phase II/III trial (9/29) |
Alcobra Ltd. (Jerusalem) and Teva Pharmaceutical Industries Ltd. (Jerusalem) |
MG01CI |
An extended-release formulation of metadoxine |
Attention deficit hyperactivity disorder |
Top-line Phase II data showed it met its primary efficacy endpoint by significantly improving ADHD symptom scores compared to placebo (9/8) |
Innocoll Inc. (Ashburn, Va.) |
XaraColl |
A biodegradable and fully resorbable collagen/bupivacaine matrix formulated and manufactured using Innocoll's collagen-based delivery platform |
Postoperative pain |
Phase II data demonstrated a statistically significant reduction in the total use of opioid medication through 24 hours and 48 hours postsurgery, as well as a statistically significant increase in the time before patients took any opioid medication (9/14) |
Iroko Pharmaceuticals LLC (Philadelphia) |
Diclofenac |
A nanotechnology-based formulation |
Osteoarthritis |
It relieved the pain of osteoarthritis at a lower dose than standard in a Phase II trial (9/16) |
Spinifex Pharmaceuticals Pty. Ltd. (Victoria, Australia) |
EMA401 |
An angiotensin II Type II receptor antagonist |
Postherpetic neuralgia |
The first patients have been treated in its Phase II trial (9/20) |
Transition Therapeutics Inc. (Toronto) |
ELND005 |
An oral inhibitor of amyloid beta aggregation |
Alzheimer's disease |
Phase II data showed trends in the area of cognition for the mild Alzheimer's group, although none of the primary endpoints reached significance (9/28) |
DIABETES | ||||
ChemoCentryx Inc. (Mountain View, Calif.) |
CCX140-B |
A CCR2 antagonist |
Type II diabetes |
Phase II data showed it significantly decreased HbA1c after four weeks and decreased fasting glucose in a Phase II trial (9/16) |
ConjuChem LLC (Los Angeles) |
CJC-1134-PC |
GLP-1 receptor agonist drug |
Type II diabetes |
Started patient enrollment for the Phase II trial (9/15) |
Dr. Reddy's Laboratories Ltd. (Hyderabad, India) |
DRL-17822 |
An oral inhibitor of cholesteryl ester transfer protein |
Type II diabetes |
Started a Phase II trial (9/6) |
Intarcia Therapeutics Inc. (Hayward, Calif.) |
ITCA 650 |
DUROS subcutaneous continuous delivery of exenatide |
Type II diabetes |
Phase II data showed patients experienced reductions in HbA1C, fasting plasma glucose and body weight during the 48 weeks of treatment at all doses tested (9/15) |
Kuros Biosurgery AG (Zurich, Switzerland) |
KUR211 |
A topical formulation consisting of a modified variant of platelet-derived growth factor incorporated into a fibrin sealant |
Diabetic foot ulcers |
Completed recruitment in a Phase IIb study (9/2) |
Metabolic Solutions Development Co. (Kalamazoo, Mich.) |
MSDC-0160 |
Insulin sensitizer |
Type II diabetes |
Lowered HbA1c levels in a 28-day Phase IIa trial (9/9) |
Tranzyme Pharma Inc. (Research Triangle Park, N.C.) |
TZP-102 |
Ghrelin agonist |
Gastroparesis in diabetes |
Data from a Phase II trial showed the drug significantly improved symptoms of the disease and was well tolerated with no effect on weight or glucose control (9/13) |
TWi Biotechnology Inc. (Taipei, Taiwan) |
AC-201 |
An orally available small molecule designed to modulate the cytokine synthesis of IL-1 beta |
Type II diabetes |
Completed recruitment of 240 patients in a Phase IIb trial (9/8) |
INFECTION | ||||
AiCuris GmbH & Co. (Wuppertal, Germany) |
AIC316 |
DNA polymerase |
Herpes simplex virus 2-positive |
Phase II data showed no emergence of viruses with any recognized resistance mutations (9/7) |
Bionor Pharma ASA (Oslo, Norway) |
Vacc-4x |
Therapeutic HIV vaccine based on four synthetic slightly modified peptide sequences |
HIV |
Phase IIb data showed significant viral load reduction compared to pre-treatment levels (9/15) |
Cempra Pharmaceuticals Inc. (Chapel Hill, N.C.) |
CEM-101 |
Fourth-generation macrolide solithromycin |
Community-acquired bacterial pneumonia |
CEM-101 demonstrated efficacy comparable with levofloxacin and a favorable safety and tolerability profile in a Phase II trial (9/16) |
Circassia Ltd. (Oxford, UK) |
ToleroMune |
Vaccine |
Hay fever |
Improved allergy symptoms in a Phase II trial, with a 30% reduction in eye responses compared to placebo (9/28) |
Gilead Sciences Inc. (Foster City, Calif.) |
GS9190 |
Non-nucleoside polymerase inhibitor |
Hepatitis C virus |
Dropped dosing of GS9190 in combination with interferon and ribavirin following incidences of pancytopenia in the Phase II trial (9/7) |
Hatchtech Pty. Ltd. (Melbourne, Australia) |
DoOvo |
A topical metalloprotease inhibitor |
Head lice |
Enrolled the first subjects in a Phase IIb study (9/1) |
Inhibitex Inc. (Atlanta) |
INX-189 |
A nucleotide inhibitor targeting NS5b |
Hepatitis C virus |
Started dosing in a 90-patient Phase II trial of INX-189 in combination with pegylated interferon and ribavirin (9/20) |
Intercell AG (Vienna, Austria) and Biological E. Ltd. (Hyderabad, India) |
Vaccine |
Vaccine for Japanese encephalitis |
Japanese encephalitis |
Completed a Phase II/III trial in 456 subjects (9/19) |
Medicago Inc. (Quebec City) |
Vaccines |
H5N1 influenza vaccine and H1N1 VLP-based vaccines |
Influenza |
Vaccines were well tolerated with no adverse events and they induced a strong antibody response in a Phase II trial (9/13) |
Peregrine Pharmaceuticals Inc. (Tustin, Calif.) |
Bavituximab |
A phosphatidylserine-targeting monoclonal antibody |
Hepatitis C virus infection |
Completed patient enrollment in its second Phase II trial (9/27) |
Pharmasset Inc. (Princeton, N.J.) |
PSI-7977; and PSI-938 |
Polymerase inhibitor; nucleotide analogue polymerase inhibitors |
Hepatitis C virus |
Reported sustained virologic response results from its Phase IIb study (9/7); started a Phase IIb study of PSI-7977 and PSI-938 (9/14) |
PolyMedix Inc. (Radnor, Pa.) |
PMX-30063 |
A defensin-mimetic antibiotic |
Acute bacterial skin and skin structure infections |
Started a Phase I trial (9/13) |
Theraclone Sciences Inc. (Seattle) |
TCN-032 |
A fully human monoclonal antibody |
Influenza A virus |
Dosed the first subjects in a Phase I trial (9/22) |
MISCELLANEOUS | ||||
Alexion Pharmaceuticals Inc. (Cheshire, Conn.) |
Soliris |
Eculizumab |
Severe and refractory generalized myasthenia gravis |
Phase II data in 14 patients showed a strong disease improvement signal as measured by the Quantitative Myasthenia Gravis disease severity score (9/16) |
Amgen Inc. (Thousand Oaks, Calif.) |
Prolia |
Denosumab |
Osteoporosis |
Long-term data showed that Prolia for up to eight years was associated with a continued increase in bone mineral density (9/20) |
Ascendis Pharma A/S (Copenhagen, Denmark) |
ACP-001 |
A once-weekly human growth hormone prodrug |
Growth-hormone deficiency |
Phase II data showed it met all primary and secondary endpoints (9/14) |
Furiex Pharmaceuticals Inc. (Morrisville, N.C.) |
MuDelta |
An oral agent |
Diarrhea-predominant irritable bowel syndrome |
Phase II data demonstrated tolerability, safety and efficacy; it was statistically superior to placebo (9/8) |
Lpath Inc. (San Diego) |
iSONEP |
Ocular formulation of a monoclonal antibody that targets sphingosine-1 phosphate |
Retinal pigment epithelium detachment |
Began dosing patients in a human proof-of-concept trial (9/30) |
Neurocrine Biosciences Inc. (San Diego) and Abbott (Abbott Park, Ill.) |
Elagolix |
A next-generation gonadotropin-releasing hormone receptor antagonist |
Uterine fibroids |
Started a Phase II trial (9/14) |
Neurocrine Biosciences Inc. (San Diego) |
NBI-98854 |
Vesicular mono-amine transporter 2 compound |
Tardive dyskinesia |
Started a second Phase II trial (9/20) |
NovImmune SA (Plan-Les-Ouates, Switzerland) |
NI-0801 |
A fully human monoclonal antibody neutralizing the CXCL 10/Interferon-gamma-inducible protein-10 chemokine |
Primary biliary cirrhosis |
Started a proof-of-concept study (9/23) |
QLT Inc. (Vancouver, British Columbia) |
Latanoprost |
Punctal Plug Delivery System |
Ocular hypertension and open-angle claucoma |
Phase II data showed that, after two weeks of treatment, intraocular pressure showed a statistically significant mean change from baseline of -6.2 mmHg, with 73% of subjects showing an IOP reduction vs. baseline of 5 mmHg or greater (9/1) |
RegeneRx Biopharma-ceuticals Inc. (Rockville, Md.) |
RGN-259 |
Topical eye drop |
Dry eye syndrome |
Treatment and follow-up are completed on 69 patients in a Phase II trial (9/26) |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Proellex |
A selective progesterone receptor blocker |
Endometriosis |
Started dosing of the final cohort in its Phase II low-dose trial (9/22) |
Tonix Pharmaceuticals Inc. (New York) |
TNX-102 |
Low dose cyclobenzaprine |
Fibromyalgia |
Phase IIa data showed it improved fibromyalgia (9/8) |
Trevi Therapeutics Inc. (Sandy Hook, Conn.) |
T111 |
Oral formulation of an existing compound |
Chronic uremic pruritus |
Began Phase II development (9/28) |
YM BioSciences Inc. (Mississauga, Ont.) |
CYT387 |
JAK1/JAK2 inhibitor |
Myelofibrosis |
Enrolled the first subjects in its Phase II trial (9/29) |
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. |