Company (Location)

Product

Description

Indication

Status (Date)#

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)

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)

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)

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)

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)

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)

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.