Company
(Location)

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
myeloma

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.