Company |
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. |