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.