Company (Location) |
Product |
Description |
Indication |
Status (Date)# |
CANCER | ||||
Bavarian Nordic A/S (Kvistgard, Denmark) |
CV-301 |
Off-the-shelf immunotherapy |
Metastatic breast or ovarian cancer |
A trial with 26 heavily pretreated patients who received monthly vaccinations showed a median time to progression of 2.5 months in the breast cancer group, and median overall survival was 13.7 months (11/10) |
Celldex Therapeutics Inc. (Needham, Mass.) |
CDX-1127 |
A co-stimulatory molecule on T cells |
Malignant solid tumors or hematologic cancers |
Started a Phase I study (11/9) |
CureVac GmbH (Tuebingen, Germany) |
CV9201 |
mRNA-based cancer vaccine |
Non-small-cell lung cancer |
Phase I/IIa data showed the drug was well tolerated and biologically active (11/8) |
CytRx Corp. (Los Angeles) |
INNO-206 |
A tumor-targeting conjugate of doxorubicin |
Sarcoma |
Phase Ib/II trial data determined a maximum tolerated dose of the drug delivering doxorubicin at the equivalent of three and a half times standard doxorubicin doses (11/1) |
Hutchison MediPharma Ltd. (London) |
Epitnib (HMPL-813) |
An orally active small molecule inhibitor targeting the epidermal growth factor receptor |
Cancer |
Started the first-in-human Phase I trial (11/7) |
Infinity Pharmaceuticals Inc. (Cambridge, Mass.) |
IPI-145 |
Oral inhibitor of phosphinositide-3-kinase delta and gamma |
Advanced hematologic malignancies |
Started two Phase I trials (11/1) |
Lentigen Corp. (Gaithersburg, Md.) |
LG631 |
Gene therapy |
Cancer |
Started a Phase I trial (11/15) |
Senesco Technologies Inc. (Bridgewater, N.J.) |
SNA01-T |
Intravenous infusion |
Multiple myeloma |
Initiated patient dosing in its Phase Ib/IIa study (11/4) |
Silence Therapeutics plc (London) |
Atu027 |
siRNA drug |
Solid tumors |
Phase I data showed 37% of patients had stable disease after treatment (11/9) |
CARDIOVASCULAR | ||||
Cardiome Pharma Corp. (Vancouver, British Columbia) and Merck & Co. Inc. (Whitehouse Station, N.J.) |
Vernakalant |
Oral therapy |
Atrial fibrillation |
Recently completed an additional multiple rising-dose Phase I study and data showed the drug was well tolerated (11/7) |
Nile Therapeutics Inc. (San Mateo, Calif.) |
Cenderitide |
Subcutaneous infusion |
Chronic heart failure |
Phase I data showed that weight-based doses achieved target PK levels when delivered by subcutaneous pump, and that it was well tolerated, bioavailable and reduced PK variability compared to a fixed-dose regimen (11/17) |
Pozen Inc. (Chapel Hill, N.C.) |
PA32540 |
A combination of 325 mg aspirin and 40 mg immediate-release omeprazole |
Gastrointestinal risk in patients who require dual antiplatelet therapy and gastroprotection |
Phase I data showed it was associated with greater platelet inhibition when dosed 10 hours apart from Plavix compared to synchronous administration of aspirin, clopidogrel and delayed-release omeprazole at day seven (11/15) |
CENTRAL NERVOUS SYSTEM | ||||
Acadia Pharmaceuticals Inc. (San Diego) |
AM-831 |
A small molecule that combines muscarinic m1 partial agonism with dopamine D2 and serotonin 5-HT2A antagonism |
Schizophrenia |
FDA cleared it to begin a Phase I trial (11/8) |
Anavex Life Sciences Corp. (Hoboken, N.J.) |
Anavex 2-73 |
The first of a new class of oral drugs |
Alzheimer's disease |
Completed a Phase I single ascending dose trial of Anavex, showing it was well tolerated below the 55 mg to 60 mg dose with only mild adverse events (11/15) |
Cara Therapeutics Inc. (Shelton, Conn.) |
CR845 |
A peptide-based kappa opioid agonist |
Acute postoperative pain |
Started its first Phase I trial of an oral formulation (11/23) |
Neuralstem Inc. (Rockville, Md.) |
Stem cell therapy |
Stem cell therapy used in the cervical region of the spine |
Amyotrophic lateral sclerosis |
Dosed its first patient in the trial (11/28) |
NeuroDerm Ltd. (Ness Ziona, Israel) |
ND0611 |
Administered subcutaneously by a dermal patch |
Parkinson's disease |
Met all primary and secondary endpoints in a Phase I/II trial (11/10) |
Probiodrug AG (Halle, Germany) |
PQ912 |
A glutaminyl cyclase inhibitor |
Alzheimer's disease |
Phase I data demonstrated it was safe and well tolerated (11/15) |
Selecta Biosciences Inc. (Watertown, Mass.) |
SEL-068 |
A nicotine vaccine |
Smoking cessation and relapse prevention |
Started a Phase I trial (11/22) |
DIABETES | ||||
Akebia Therapeutics Inc. (Cincinnati) |
AKB-9778 |
A human protein tyrosine phosphatase beta inhibitor |
Diabetic macular edema and diabetic retinopathy |
Started a Phase I trial (11/22) |
Concert Pharmaceuticals Inc. (Lexington, Mass.) |
CTP-499 |
Controlled-release version; analogue of 1-([S]-5-hydroxyhexyl)-3,7-dimethylxanthine |
Diabetic nephropathy |
Phase I data showed it was well tolerated at single doses up to and including 1,800 mg (11/15) |
Convergence Pharmaceuticals Ltd. (Cambridge, UK) |
CNV2197944 |
A calcium channel blocker |
Chronic pain |
Initiated a Phase I trial (11/22) |
DARA BioSciences Inc. (Raleigh, N.C.) |
DB959 |
A peroxisome proliferator activated receptor-delta/gamma agonist |
Type II diabetes |
Phase Ib data showed a safety profile comparable to placebo when given as once-daily doses for seven days and was safe and well tolerated throughout the 40-fold dose range tested (11/2) |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) |
ISIS-GCGRRx and ISIS-GCCRRx |
Antisense drugs |
Type II diabetes |
Started Phase I studies (11/3) |
PhaseBio Pharmaceuticals Inc. (Malvern, Pa.) |
Glymera |
A recombinant glucagon-like peptide-1 analogue |
Hyperglycemia in Type II diabetes |
Phase I/IIa data demonstrated statistically significant reductions in fasting glucose glycemic load following meal tolerance testing and in average daily glucose (11/16) |
INFECTION | ||||
AVIR Green Hills Biotechnology AG (Vienna, Austria) |
deltaFLU |
Intranasal flu vaccine |
Influenza |
The vaccine was well tolerated and significantly increased influenza-specific antibodies in a Phase I/II trial (11/8) |
Inhibitex Inc. (Atlanta) |
INHX-189 |
Monotherapy; nucleotide polymerase inhibitor |
Hepatitis C virus |
Data showed INX-189 dosed at 100 mg once daily for seven days in combination with ribavirin resulted in a median HCV RNA reduction from baseline of -0.379 log10 (11/30) |
Inovio Pharmaceuticals Inc. (Blue Bell, Pa.) |
SynCon |
Avian influenza vaccine |
Avian flu |
A single intradermal electroporation boost of its vaccine generated hemagglutination inhibition titers against six different unmatched strains of H5N1 and it generated a fourfold or greater rise in HAI titers in 50% of boosted subjects in a Phase I study (11/18) |
Novacta Biosystems Ltd. (London) |
NVB302 |
An oral treatment |
Hospital-acquired Clostridium difficile infections |
Started dosing the first healthy volunteers in a Phase I trial (11/3) |
MISCELLANEOUS | ||||
Aeolus Pharmaceuticals Inc. (Mission Viejo, Calif.) |
AEOL 10150 |
A broad-spectrum catalytic antioxidant designed to neutralize reactive oxygen and nitrogen species |
Acute radiation syndrome |
Pilot study results of AEOL 10150 and G-CSF drug Neupogen confirmed that it does not interefer with the positive effects of Neupogen on the hematopoietic syndrome (11/2) |
Akebia Therapeutics Inc. (Cincinnati) |
AKB-6548 |
Oral hypoxia-inducible factor-prolyl hydroxylase inhibitor |
Anemia associated with chronic kidney disease |
Phase Ib/IIa data showed it was well tolerated and enhanced erythropoiesis (11/16) |
AlloCure Inc. (Burlington, Mass.) |
AC607 |
A bone marrow-derived mesenchymal stem cell therapy |
Acute kidney injury |
Phase I data showed it was safe and well tolerated, and treated subjects experienced a lower incidence of AKI, reduced length of hospital stay and reduced hospital readmission rates compared to a cohort of historical controls (11/14) |
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
ALN-TTR01 |
An RNAi therapeutic targeting transthyretin |
TTR-mediated amyloidosis |
Phase I data showed statistically significant reductions in serum TTR protein levels in ATTR patients (11/22) |
Bellicum Pharmaceuticals Inc. (Houston) |
CaspCIDe |
Technology used to eliminate donor T cells |
Graft-vs.-host disease |
Results of the first clinical study showed it worked quickly and effectively without compromising the function of the cell (11/4) |
Chimerix Inc. (Durham, N.C.) |
CMX001 |
A lipid-antiviral-conjugate designed to deliver cidofovir-diphosphate |
Kidney disease |
Phase I/II data showed it was generally safe and well tolerated when administered to renal transplant and hematopoietic stem cell transplant patients (11/14) |
Geron Inc. (Menlo Park, Calif.) |
hESC therapy |
Human embryonic stem cell therapy |
Acute spinal injury |
Company is stopping recruitment to its Phase I trial due to economic conditions (11/16) |
Pluristem Therapeutics Inc. (Haifa, Israel) |
PLX-PAD |
Cells derived from the firm's PLX (PLacental eXpanded) platform |
Critical limb ischemia |
Phase I data showed that it met all the endpoints, demonstrating a safe immunologic profile at all dosage levels and showing potential efficacy (11/4) |
Provesica Ltd. (Cambridge, UK) |
XEN-D0501 |
An antagonist of TRPV1 |
Overactive bladder |
Phase I data showed it was safe and well tolerated (11/28) |
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. |