Company
(Location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Creabilis SA (Luxembourg)

CT327

A TrkA kinase inhibitor

Atopic dermatitis

Treated the first patients in its Phase IIb study

9/19/13

CANCER

Agenus Inc. (Lexington, Mass.)

Prophage Series G-100

An autologous cancer vaccine

Glioblastoma multiforme

Phase II data showed an almost 18-month median progression-free survival, a 160% increase vs. current standard of care alone

9/18/13

Biolinerx Ltd. (Jerusalem)

BL-8040

Selective antagonist of chemokine receptor CXCR4

Acute myeloid leukemia

FDA granted orphan drug designation; it is in a Phase II study

9/10/13

Dendreon Corp. (Seattle)

Provenge

Sipuleucel-T

Metastatic castrate-resistant prostate cancer

Phase II combination study demonstrated its potency and immunological prime-boost responses are maintained when administered concurrently or sequentially with Zytiga plus prednisone

9/30/13

Genmab A/S (Copenhagen, Denmark) and Janssen Biotech Inc. (unit of Johnson & Johnson; New Brunwick, N.J.)

Daratumumab

A CD38 monoclonal antibody

Multiple myeloma

Started a Phase II study

9/11/13

Infinity Pharmaceuticals Inc. (Cambridge, Mass.)

Retaspimycin

Heat-shock protein 90 inhibitor

Non-small-cell lung cancer

Missed its efficacy endpoints in the Phase II study, failing to demonstrate statistically significant improvement in overall survival

9/26/13

Oncolytics Biotech Inc. (Calgary, Alberta)

Reolysin

Reovirus variant for Ras-mutant cancers

Squamous cell carcinoma of the lung

Final tumor response data from the Phase II trial showed 23 of 25 patients exhibited overall tumor shrinkage

9/10/13

Pharmamar SA (subsidiary of Grupo Zeltia; Madrid)

PM01183

Designed to bind covalently to the minor groove of DNA

Platinum-resistant/refractory ovarian cancer

Phase II data showed 30.3% of patients had an objective response, while no patients in the topotecan arm responded

9/13/13

Prima Biomed Ltd. (Sydney, Australia)

Cvac

Cancer vaccine

Epithelial ovarian cancer

Top-line Phase II data showed no observed difference in estimated median progression-free survival compared to control

9/19/13

Transgene SA (Strasbourg, France)

Pexa-Vec

Cancer vaccine

Advanced liver cancer

Phase IIb data showed it failed to meet its primary endpoint of overall survival for Pexa-Vec plus best supportive care vs. BSC alone; Pexa-Vec was well tolerated

9/4/13

Verastem Inc. (Cambridge, Mass.)

VS-6063

Focal adhesion kinase inhibitor defactinib

KRAS-mutated non-small-cell lung cancer

Started a Phase II trial

9/27/13

Viralytics Ltd. (Sydney)

Cavatak

Coxsackievirus A21

Melanoma

Phase II data showed it met its primary endpoint of immune-related progression free survival at six months after the first dose

9/19/13

CARDIOVASCULAR

Amgen Inc. (Thousand Oaks, Calif.)

AMG145

A PCSK9 inhibitor

High cholesterol

Phase II data showed significant reductions in low-density lipoprotein cholesterol of up to 59%

9/4/13

Aesrx LLC (Newton, Mass.)

Aes-103

Small molecule anti-sickling agent

Sickle cell disease

Advanced it into a Phase II trial

9/9/13

Cardiome Pharma Corp. (Vancouver, British Columbia)

Vernakalant

Intravenous therapy

Atrial fibrillation

Positive data from an open-label study showed vernakalant achieved conversion to normal sinus rhythm in a median time of 12 minutes compared to 151 minutes for the propafenone group and 162 minutes for the flecainide group

9/24/13

Celladon Corp. (San Diego)

Mydicar

Genetically targeted enzyme replacement therapy aimed at restoring levels of SERCA2a

Advanced heart failure

The Phase IIa portion showed that the durability of reduced cardiovascular and terminal events previously observed at 12 months was maintained

9/27/13

Cytokinetics Inc. (South San Francisco) and Amgen Inc. (Thousand Oaks, Calif.)

Omecamtiv mecarbil

Oral; cardiac myosin activator

Acute heart failure

Did not meet its primary endpoint of shortness-of-breath response in a Phase II trial

9/4/13

Esperion Therapeutics Inc. (Plymouth, Mich.)

ETC-1002

Oral, once-daily therapy

Elevated levels of low-density lipoprotein cholesterol

Phase IIa data showed it achieved incremental LDL-C lowering of 22% at eight weeks, compared with 0% in the placebo group; the drug also was well tolerated

9/4/13

Isis Pharmaceuticals Inc. (Carlsbad, Calif.)

ISIS-APOCIIIRx

Monotherapy; antisense drug

High triglycerides

Interim Phase II data showed it achieved statistically significant mean reductions of up to 79% in apolipoprotein C-III and up to 75% in triglycerides

9/4/13

Pluristem Therapeutics Inc. (Haifa, Israel)

PLX-PAD

Cells comprised of a drug delivery platform designed to release a cocktail of therapeutic proteins

Intermittent claudication

FDA lifted the clinical hold on the dose-escalation trial

9/17/13

Resverlogix Corp. (Calgary, Alberta)

RVX-208

An oral BET protein inhibitor

Cardiovascular disease

Phase IIb data of RVX-208 using intravascular ultrasound showed that the overall study missed its endpoint, but a breakout analysis showed patients taking rosuvastatin plus RVX-208 had a highly statistically significant percent atheroma volume plaque regression of -1.43%

9/4/13

CENTRAL NERVOUS SYSTEM

Adynxx Inc. (San Francisco)

AYX1

A small synthetic, dsDNA molecule that blocks EGR1 function by mimicking the genomic sequence EGR1 normally binds

To reduce acute postsurgical pain

Enrollment was completed in its Phase II study

9/6/13

Civitas Therapeutics Inc. (Chelsea, Mass.)

CVT-301

An inhaled formulation of levodopa

Parkinson’s disease

Initiated a Phase IIb study

9/6/13

Metabolic Solutions Development Co. (Kalamazoo, Mich.)

MSDC-0160

mTOT-modulating insulin sensitizer

Alzheimer’s disease

Phase IIa data showed it maintained glucose metabolism in key regions of the brain associated with cognitive decline due to AD

9/10/13

Neuralstem Inc. (Rockville, Md.)

NSI-566

Spinal cord-derived neural stem cells

Amyotrophic lateral sclerosis

First patient was treated in its Phase II trial

9/11/13

Neurocrine Biosciences Inc. (San Diego)

NBI-98854

A small-molecule VMAT2 inhibitor

Tardive dyskinesia

Did not meet the primary endpoint in the Phase IIb trial

9/10/13

Neurovance Inc. (Cambridge, Mass.)

EB-1020 SR

Designed to modulate norepinephrine, dopamine and serotonin reuptake inhibition

Attention deficit hyperactivity disorder

Company started a Phase IIa pilot study

9/25/13

Omeros Corp. (Seattle)

OMS824

A phosphodiesterase 10 inhibitor

Schizophrenia

Started enrollment in a Phase II trial

9/20/13

Zalicus Inc. (Cambridge, Mass.)

Z160

An oral, state-dependent, selective N-type calcium channel blocker

Chronic neuropathic pain

Completed patient enrollment in two Phase II trials

9/4/13

DIABETES

Biodel Inc. (Danbury, Conn.)

BIOD-123

An ultra-rapid-acting mealtime insulin

Type I diabetes

Phase II data showed it was non-inferior to Humalog, but injection-site pain data did not match

9/10/13

Boston Therapeutics Inc. (Manchester, N.H.)

PAZ320

A complex carbohydrate-based drug

Type II diabetes

Phase II data showed that 45% of patients responded with a 40% reduction of postmeal glucose in the blood compared to baseline in a dose-dependent manner

9/5/13

Chemocentryx Inc. (Mountain View, Calif.)

CCX140

An inhibitor of chemokine receptor CCR2

Diabetic nephropathy

Phase II data showed treatment on top of standard of care appears well tolerated to date

9/11/13

Elcelyx Therapeutics Inc. (San Diego)

Newmet

Delayed-release formulation of generic metformin

Type II diabetes

Met its primary endpoint with statistically significant reductions in fasting plasma glucose at four weeks compared to placebo

9/5/13

Zealand Pharma A/S (Copenhagen)

Lyxumia

Lixisenatide

Type II diabetes

Phase II data of Lyxumia showed reductions in HbA1c and in body weight were greatest in diabetes’ patients who had well-controlled baseline fasting plasma glucose

9/25/13

INFECTION

Afferent Pharmaceuticals Inc. (San Mateo, Calif.)

AF-219

Oral P2X3 antagonist

Treatment-refractory chronic cough

Phase II data showed a 75% reduction of daytime cough frequency at week two of treatment

9/10/13

Chimerix Inc. (Durham, N.C.)

CMX001

Brincidofovir

Cytomegalo-virus

Phase II data showed it met its primary endpoint of reduction in CMV viremia or CMV disease compared to placebo

9/27/13

Conatus Pharmaceuticals Inc. (San Diego)

IDN-6556

Emricasan

Severe alcoholic hepatitis

Initiated dosing in the Phase II trial

9/12/13

Presidio Pharmaceuticals Inc. (San Francisco) and Boehringer Ingelheim GmbH (Ingelheim, Germany)

PPI-668

Pan-genotypic NS5A inhibitor

Hepatitis C virus

Completed patient enrollment for a Phase IIa trial testing an interferon-free, all-oral, direct-acting antiviral combination of Boehringer’s faldaprevir and non-nucleoside NS5B polymerase inhibitor in combination with PPI-668, with and without ribavirin

9/11/13

Theraclone Sciences Inc. (Seattle)

TCN-032

A monoclonal antibody

Influenza A

Phase IIa data showed significant reductions in clinical symptoms score as well as viral load for the TCN-032 arm as compared to placebo-treated subjects

9/10/13

Theraclone Sciences Inc. (Seattle)

TCN-202

Recombinant, fully human monoclonal antibody

To prevent human cytomegalovirus infection

Dosed the first patient in a Phase IIa trial

9/11/13

Viamet Pharmaceuticals Inc. (Research Triangle Park, N.C.)

VT-1161

A small-molecule selective, oral candidate

Cryptococcal meningitis; athlete’s foot

Started a Phase II trial; started a second Phase II trial in moderate to severe interdigital tinea pedis

9/6/13;

9/25/13

INFLAMMATORY

Coronado Biosciences Inc. (Burlington, Mass.)

TSO

Trichuris suis ova, CNDO-201

Ulcerative colitis

Started a Phase II trial

9/13/13

Nektar Therapeutics Inc. (San Francisco)

NKTR-181

A mu-opioid agonist

Osteoarthritis of the knee

Phase II trial missed its primary endpoint; however, only nine of 295 patients were unable to achieve meaningful pain relief

9/30/13

Tonix Pharmaceuticals Holding Corp. (New York)

TNX-102 SL

Sublingual therapy

Fibromyalgia

Started its Phase IIb trial

9/17/13

RESPIRATORY

Anergis (Epalinges, Switzerland)

AllerT

Allergy vaccine

Allergies

Phase IIb study showed it met the primary endpoint of reducing the combined symptom and medication score with AllerT 50 mcg during the birch pollen season

9/11/13

Circassia Ltd. (Oxford, UK)

Toleromune

Technology in which synthetic versions of epitopes identified from allergens are used to generate helper T cells

House dust mite allergy

Phase II data showed a significant reduction in symptoms at one year in subjects receiving the most effective dose

9/17/13

Cytos Biotechnology Ltd. (Zurich, Switzerland)

CYT003

A first-in-class immune modulator

Allergic asthma

Phase IIa data suggested that the efficacy is particularly evident in patients with allergic asthma in the higher bEos group

9/10/13

DBV Technologies (Bagneux, France)

Viaskin Peanut

An epicutaneous immunotherapy

Peanut allergy

The first patient has been enrolled in a Phase IIb study

9/5/13

Skyepharma plc (London) and Mundipharma Research Ltd. (Cambridge, UK)

Formoterol

Fluticasone propionate

Chronic obstructive pulmonary disease

Started a Phase III study

9/20/13

Theravance Inc. (South San Francisco)

TD-4208

A long-acting muscarinic antagonist

Moderate to severe chronic obstructive pulmonary disease

Phase IIb data showed it met the primary efficacy endpoint for all six doses studied and demonstrated a statistically significant change vs. placebo

9/6/13

MISCELLANEOUS

Alexion Pharmaceuticals Inc. (Cheshire, Conn.)

Asfotase alfa

Targeted enzyme replacement candidate

Hypophosphatasia

An ongoing study in infants and young children showed it met its primary endpoint of significant improvement in skeletal mineralization from baseline

9/24/13

Alexion Pharmaceuticals Inc. (Cheshire, Conn.)

Soliris

Eculizumab

To prevent acute antibody-mediated rejection in sensitized deceased donor kidney transplant recipients

Preliminary data from a single-arm Phase II trial showed a nine-week occurrence of post-transplantation treatment failure of 10.6% of 47 patients, including a 6.4% rate of AMR compared to an expected 30% rate of AMR

9/11/13

Dompe Group (Milan)

Reparixin

Shown to improve efficacy of transplantation of insulin-producing pancreatic islets

Autologous islet cell transplantation

Launched a Phase II/III trial

9/27/13

Edison Pharmaceuticals Inc. (Mountain View, Calif.)

EPI-743

An orally bioavailable small molecule

Disorders of energy utilization or oxidation-reduction

Started a Phase II trial

9/25/13

Isis Pharmaceuticals Inc. (Carlsbad, Calif.)

ISIS-APOCIIIRx

Monotherapy

Familial Chylomicro-nemia syndrome

Phase II data showed all three patients achieved a triglyceride level below 500 mg/dL, reducing the risk of an acute pancreatic event

9/24/13

Kythera Biopharma-ceuticals Inc. (Calabasas, Calif.)

ATX-101

A formulation of a purified synthetic version of deoxycholic acid

Submental fat

Phase III data showed it met all primary and secondary endpoints testing it vs. placebo

9/18/13

Novabay Pharmaceuticals Inc. (Emeryville, Calif.)

NVC-422

Auriclosene

To prevent urinary catheter blockage and encrustation of indwelling urinary catheters

Phase II data showed that the auriclosene irrigation solution reduced the average encrustation at the time of catheter removal from 77% encrusted (saline arm) to 22% encrusted (auriclosene arm)

9/17/13

Regentis Biomaterials Ltd. (Princeton, N.J.)

Gelrinc implant

Biodegradable hydrogel implant

Articular cartilage in the knee

Clinical data showed safety and efficacy

9/17/13

Sarepta Therapeutics Inc. (Cambridge, Mass.)

Eteplirsen

Exon-skipping drug candidate

Duchenne muscular dystrophy

Phase IIb data showed a continued stabilization of walking ability in treated patients nearly two years; it met the primary endpoint of increased novel dystrophin

9/27/13

Transition Therapeutics Inc. (Toronto)

ELND005

Scyllo-inositol

Down syndrome

First patient was dosed in a Phase IIa trial

9/5/13


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.