Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Alexar Therapeutics Inc. (Malvern, Pa.) |
ALX-101 |
LXR agonist |
Mild to moderate atopic dermatitis |
Started a phase IIa trial to evaluate the safety, tolerability, dose-response and pharmacokinetics of two concentrations of ALX-101 gel |
11/24/15 |
Aquinox Pharmaceuticals Inc. (Vancouver, British Columbia) |
AQX-1125 |
A SHIP1 activator |
Mild to moderate dermatitis |
Company is halting development after the phase II KINSHIP trial failed to demonstrate efficacy |
11/4/15 |
Canbex Therapeutics Ltd. (London) |
VSN16R |
Oral candidate |
Spasticity caused by multiple sclerosis |
Entered a phase II trial that will enroll 142 subjects |
11/3/15 |
Cymabay Therapeutics Inc. (Newark, Calif.) |
Arhalofenate |
A PPAR gamma agonist |
Gout, hyperuricemia and a history of flares |
A double-blind, placebo-controlled phase IIb study in 239 patients showed a 46% reduction in flare rate (p=0.0056); it was well tolerated and appeared safe as a monotherapy and in combination with febuxostat |
11/6/15 |
Galapagos NV (Mechelen, Belgium) |
Filgotinib |
An orally available, selective inhibitor of JAK1 |
Rheumatoid arthritis |
Phase IIb data showed filgotinib in combination with MTX demonstrated consistent efficacy on signs and symptoms of disease with a rapid onset of action |
11/10/15 |
CANCER | |||||
Aadi LLC (Pacific Palisades, Calif.) |
ABI-009 |
Targeted nanoparticle albumin-bound version of mTOR inhibitor sirolimus or rapamycin |
Advanced perivascular epitheliod cell tumors |
Started a registration trial, a phase II study that will enroll about 35 patients |
11/3/15 |
Aduro Biotech Inc. (Berkeley, Calif.) |
CRS-207 |
Immunotherapy |
Pancreatic cancer |
Seven patients treated in the phase IIa trial of CRS-207, in combination with GVAX Pancreas, survived for more than three years |
11/10/15 |
Angiochem Inc. (Montreal) |
ANG1005 |
A paclitaxel-peptide drug conjugate |
Breast cancer with brain metastases |
Ongoing phase II data demonstrated that patients, including a subset with leptomengeal carcinomatosis, achieved encouraging responses; of 21 heavily pre-treated patients, five achieved a partial response and 11 had stable disease as best intracranial response |
11/23/15 |
Bind Therapeutics Inc. (Cambridge, Mass.) |
AZD2811 |
A selective inhibitor of Aurora B kinase |
Solid and hematological tumors |
Started patient dosing in a phase I trial |
11/19/15 |
Biolinerx Ltd. (Tel Aviv, Israel) |
BL-8040 |
Short peptide that functions as a high-affinity antagonist for CXCR4 |
Relapsed or refractory acute myeloid leukemia |
Data from the dose-escalation portion of the phase II trial showed the drug, as a single agent and in combination with cytarabine, was safe and well tolerated at all doses tested up to and including the highest dose level of 1.5 mg/kg, with no major adverse events |
11/6/15 |
Caladrius Biosciences Inc. (South San Francisco) |
CLBS20 |
Immunotherapy |
Metastatic melanoma |
Phase II data of CLBS20 vs. a control group of irradiated tumor cells alone concluded that CLBS20 was associated with improved survival |
11/10/15 |
Casi Pharmaceuticals Inc. (Rockville, Md.) |
ENMD-2076 |
An orally active Aurora A/angiogenic kinase inhibitor |
Advanced fibrolamellar carcinoma |
Dosed the first patient in a phase II trial |
11/5/15 |
Casi Pharmaceuticals Inc. (Rockville, Md.) |
ENMD-2076 |
A selective angiogenic kinase inhibitor |
Soft tissue sarcoma |
Phase II data showed it prompted partial response or stable disease for more than six months in 17% of patients studied |
11/9/15 |
Celldex Therapeutics Inc. (Hampton, N.J.) |
Rintega |
Rindopepimut |
EGFRvIII-positive, recurrent glioblastoma |
Mature survival data from its phase II ReACT study continues to show a marked benefit (p=0.0137), with a long-term survival benefit clearly seen in the Rintega arm |
11/23/15 |
Cinfa Biotech SL (Pamplona, Spain) |
B12019 |
A biosimilar version of Neulasta (pegilgrastim, Amgen) |
Chemotherapy-induced neutropenia |
The first subjects have been dosed in a trial |
11/17/15 |
Cytrx Corp. (Los Angeles) |
Aldoxorubicin |
Tumor-targeted doxorubicin conjugate |
Unresectable glioblastoma multiforme |
Achieved target enrollment of 28 patients in its phase II trial |
11/24/15 |
Delmar Pharmaceuticals Inc. (Vancouver, British Columbia) |
VAL-083 |
Dianhydrogalactitol |
Refractory glioblastoma multiforme |
Phase II data show it was well tolerated and exhibited a favorable safety profile |
11/24/15 |
Genspera Inc. (San Antonio) |
Mipsagargin |
G-202, a prostate-specific membrane antigen-activated prodrug targeting the tumor endothelium |
Recurrent or progressive glioblastoma |
Phase II data observed in the first 12 glioblastoma patients exceeded criteria required to expand enrollment and commence the second stage of the phase II trial; interim data showed clinical benefit in a subset of patients with high levels of PSMA expression in the primary tumor |
11/16/15 |
Genspera Inc. (San Antonio) |
G-202 |
Mipsagargin; PSMA-activated prodrug targeting endothelial cells |
Glioblastoma multiforme |
Phase II data showed that three of 11 evaluable patients demonstrated at least stable disease at the first disease assessment, one of which has met the primary endpoint of six-month progression-free survival |
11/23/15 |
Immune Design Corp. (Seattle) |
CMB305 |
Prime-boost cancer immunotherapy |
Soft tissue sarcoma |
Started a randomized phase II study |
11/12/15 |
Immunocellular Therapeutics Ltd. (Los Angeles) |
ICT-107 |
A dendritic cell-based immunotherapy |
Newly diagnosed glioblastoma |
Phase II results indicate a survival advantage in the ICT-107-treated group compared to the control group and also showed a significant association between immune response and survival, especially in HLA-A2-positive patients |
11/23/15 |
Immunomedics Inc. (Boston) |
IMMU-132 |
Sacituzumab govitecan; antibody-drug conjugate |
Metastatic triple-negative breast cancer |
Phase II data showed patients enrolled lived for a median of seven months without tumor progression after receiving at least three doses |
11/10/15 |
Kite Pharma Inc. (Santa Monica, Calif.) |
KTE-C19 |
Therapy in which a patient's T cells are genetically modified to express a chimeric antigen receptor designed to target the antigen CD19 |
Refractory, aggressive non-Hodgkin lymphoma |
Opened enrollment in the phase II portion of a phase I/II trial |
11/3/15 |
Kite Pharma Inc. (Santa Monica, Calif.) |
KTE-C19 |
ZUMA-2 |
Relapsed or refractory mantle cell lymphoma |
Initiated a phase II study |
11/10/15 |
Momenta Pharmaceuticals Inc. (Cambridge, Mass.) |
M402 |
Necuparanib |
Metastatic pancreatic cancer |
Put a temporary hold on patient enrollment in its ongoing phase II portion of a phase I/II study of M402 in combination with Abraxane (nab-paclitaxel, Celgene Corp.) and gemcitabine, so the company can amend its protocol as a results of specific toxicities, including thrombocytopenia, risk of bleeding and thromboembolic events |
11/16/15 |
Morphotek Inc. (subsidiary of Eisai Inc.; Woodcliff Lake, N.J.) |
MORAb-009 |
Amatuximab; monoclonal antibody targeting mesothelin |
Malignant pleural mesothelioma |
Enrolled the first patient in the randomized, double-blind ARTEMIS trial evaluating the efficacy and safety of MORAb-009 |
11/19/15 |
Oncoceutics Inc. (Philadelphia) |
ONC201 |
Small-molecule drug |
Multiple myeloma |
Began enrolling patients with various solid tumors in a dose-escalation trial |
11/30/15 |
VBL Therapeutics Inc. (Tel Aviv, Israel) |
VB-111 |
Targeted anti-angiogenic gene therapy |
Recurrent glioblastoma |
Phase II data showed the overall survival rate for patients treated with VB-111 in combination with bevacizumab upon disease progression was 57% at 12 months, compared with an overall 12-month survival of 28% (range 16% to 38%) in the pooled data from the four Avastin studies (p = 0.007) |
11/20/15 |
Virttu Biologics Ltd. (Glasgow, Scotland) |
Seprehvir |
Oncolytic virus |
Non-central nervous system solid tumors and high-grade gliomas |
Two clinical trials suggest the combination of PD-1 and oncolytic herpes virotherapy may be an effective treatment strategy for some cancers |
11/10/15 |
CARDIOVASCULAR | |||||
Cytokinetics Inc. (South San Francisco) and Amgen Inc. (Thousand Oaks, Calif.) |
Omecamtiv mecarbil |
A cardiac myosin activator |
Chronic heart failure |
Phase II expansion phase data showed it met its primary pharmacokinetic endpoints as well as leading to statistically significant improvements in several measures of cardiac function, including systolic ejection time, stroke volume and N-terminal-pro-brain natriuretic peptide, at 20 weeks following randomization |
11/10/15 |
Medicinova Inc. (La Jolla, Calif.) |
MN-001 |
Tipelukast |
Hypertriglyceri-demia in nonalcoholic steatohepatitis |
Started a phase IIa trial |
11/20/15 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Vericiguat |
A soluble guanylate cyclase stimulator |
Worsening chronic heart failure |
The primary endpoint was not achieved in a phase II trial of vericiguat compared to placebo, patients receiving vericiguat 10 mg daily experienced a greater reduction in NT-proBNP, greater improvement in left ventricular ejection fraction and fewer clinical events |
11/10/15 |
Protalex Inc. (Florham Park, N.J.) |
PRTX-100 |
Highly purified form of Staphylococcal protein A |
Persistent/chronic immune thrombo-cytopenia |
Dosed the first patient in its U.S. phase I/II study |
11/24/15 |
Tigenix NV (Leuven, Belgium) |
AlloCSC-01 |
A suspension of allogeneic human cardiac stem cells |
Acute myocardial infarction |
Completed enrollment of its phase II study |
11/20/15 |
Quark Pharmaceuticals Inc. (Fremont, Calif.) |
QPI-1007 |
siRNA-based neuroprotective drug |
Acute non-arteritic anterior ischemic optic neuropathy |
Started the U.S. arm of a phase II/III pivotal study |
11/13/15 |
CENTRAL NERVOUS SYSTEM | |||||
Stemcells Inc. (Newark, Calif.) |
HuCNS-SC cells |
Human neural stem cells |
Cervical spinal cord injury |
Six-month interim results from the first cohort in its ongoing phase II Pathway Study, which showed motor improvements in both strength and function |
11/20/15 |
Vistagen Therapeutics Inc. (San Francisco) |
AV-101 |
Orally available |
Treatment-resistant major depressive disorder |
The first patient was dosed in a phase IIa study |
11/4/15 |
DIABETES | |||||
Aerpio Therapeutics Inc. (Cincinnati) |
AKB-9778 |
A small molecule designed to inhibit human protein tyrosine phosphatase beta; acts as a negative regulator of the Tie2 receptor |
Diabetic macular edema |
Data from its phase IIa study showed the combination of AKB-9778 and Lucentis (ranibizumab, Roche AG) provided a clinically significant benefit in reduction of macular edema compared to Lucentis alone at month two (p=0.02) and at month three (p=0.008) |
11/18/15 |
Biodel Inc. (Danbury, Conn.) |
BIOD-531 |
Biphasic insulin formulation |
Type 2 diabetes |
Started a phase IIa trial designed to elevate glucose control achieved with BIOD-531 |
11/5/15 |
Chemocentryx Inc. (Mountain View, Calif.) |
CCX140 |
Chemokine receptor CCR2 inhibitor |
Diabetic nephropathy |
Additional data from a successful phase II study showed statistically significant improvement in levels of protein in the urine across the entire patient population tested, with particularly marked improvements in the pre-specified patient group who had the highest proteinuria when enrolled in the study |
11/6/15 |
Phasebio Pharmaceuticals Inc. (Malvern, Pa.) |
PE0139 |
Super-long-acting basal insulin |
Hyperglycemia associated with diabetes |
Started a phase IIa trial |
11/10/15 |
Poxel SA (Lyon, France) |
Imeglimin |
Targets the mitochondria bioenergetics |
Type II diabetes |
Phase II data showed imeglimin improved glucose sensing, leading to an increase in insulin secretion, and improved insulin sensitivity as shown by several biomarkers |
11/24/15 |
Remd Biotherapeutics Inc. (Camarillo, Calif.) and subsidiary Cosci-Remd Biotherapeutics |
REMD-477 |
Fully human antibody to the glucagon receptor |
Type 2 diabetes |
A phase II trial is enrolling about 72 patients |
11/20/15 |
GASTROINTESTINAL | |||||
Ironwood Pharmaceuticals Inc. (Cambridge, Mass.) and Allergan plc (Dublin) |
Linaclotide |
Orally administered colonic-release formulations of guanylate cyclase agonist |
Irritable bowel syndrome with constipation |
Started a phase IIb trial that will enroll up to 520 patients |
11/3/15 |
INFECTION | |||||
Immuron Ltd. (Melbourne, Australia) |
IMM-124E |
Oral formulations of hyperimmune bovine colostrum powder |
Non-alcoholic steatohepatitis |
Its phase IIb trial has successfully passed a 25% recruitment milestone with 35 patients randomized into either arms; no significant adverse events have been reported |
11/19/15 |
Rebiotix Inc. (Roseville, Minn.) |
RBX2660 |
Microbiota suspension |
Multi-recurrent Clostridium difficile infection |
Completed enrollment in the phase IIb PUNCH CD 2 study with 117 patients across 20 sites in the U.S. and Canada |
11/19/15 |
Summit Therapeutics plc (Oxford, U.K.) |
Ridinilazole |
Antibiotic |
Clostridium difficile infection |
Phase II results showed that ridinilazole brought sustained clinical response rates of 66.7% vs. 42.4% with current standard of care vancomycin; noninferiority of ridinilazole compared to vancomycin in sustained clinical response, the primary endpoint, had a noninferiority margin of 15% (p = 0.0004) |
11/24/15 |
INFLAMMATORY | |||||
AB2 Bio Ltd. (Lausanne, Switzerland) |
Tadekinig alfa |
Recombinant interleukin-18-binding protein |
Severe systemic inflammation |
Obtained clinical proof of concept in a case involving an infant girl at risk of death from severe systemic inflammation; therapy was initiated on a compassionate-use basis in June and by September she was reported to be in full remission |
11/18/15 |
Capgenesis Therapeutics Inc. (Bethesda, Md.) |
CGS-125 |
Topical formulation of the TRPV1 channel agonist trans-capsaicin |
Osteoarthritic knee pain |
Dosed the first patient in a randomized, double-blind exploratory study |
11/24/15 |
Generon Corp. (Shanghai) |
F-652 |
Recombinant fusion protein containing human interleukin 22 |
Alcoholic hepatitis |
FDA cleared a phase IIa investigational new drug application for a study to assess the safety and efficacy |
11/24/15 |
Idera Pharmaceuticals Inc. (Cambridge, Mass.) |
IMO-8400 |
TLR7, 8 and 9 antagonist |
Dermatomyositis |
Started enrolling patients in a phase II trial |
11/10/15 |
Samumed LLC (San Diego) |
SM04690 |
A small-molecule inhibitor of the Wnt pathway |
Moderately to severely symptomatic osteoarthritis |
Started enrolling patients in a phase II study |
11/9/15 |
MISCELLANEOUS | |||||
Akebia Therapeutics Inc. (Cambridge, Mass.) |
Vadadustat |
Designed to stabilize HIF, a transcription factor that regulates the expression of genes involved with red blood cell production in response to changes in oxygen levels |
Anemia related to chronic kidney disease |
Phase II study achieved its primary endpoints of maintaining stable hemoglobin levels over 16 weeks of treatment in three cohorts of patients converting from recombinant erythropoiesis-stimulating agents |
11/10/15 |
Allena Pharmaceuticals Inc. (Newton, Mass.) |
ALLN-177 |
Recombinant oxalate-degrading enzyme |
Secondary hyperoxaluria in patients with a history of calcium oxalate kidney stones |
Results from the first phase II trial showed a statistically significant reduction of urinary oxalate excretion in patients with secondary hyperoxaluria (p = 0.0084) |
11/13/15 |
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
Patisiran and revusiran |
RNAi candidates targeting transthyretin |
TTR-mediated amyloidosis |
Phase II open-label extension data of patisiran provided additional evidence, after 18 months of dosing, that the candidate offers the potential to halt neuropathy progression and was generally well tolerated in FAP out to nearly two years; phase II OLE study data of revusiran showed robust and sustained knockdown of serum TTR, and the candidate was generally well tolerated in most patients with TTR cardiac amyloidosis |
11/4/15 |
Angion Biomedica Corp. (Uniondale, N.Y.) |
BB3 |
A small molecule with hepatocyte growth factor-like therapeutic properties |
Renal transplantation |
Phase II data showed BB3 administered as late as 24 hours after renal transplantation, met the primary and secondary endpoints and improved outcome in patients with delayed graft function |
11/3/15 |
Auven Therapeutics (Ft. Lauderdale, Fla.) |
Seciera |
OTX-101; a nanomicellar formulation of cyclosporine |
Dry eye disease |
Results from its phase IIb/III trial found that both concentrations (0.05% and 0.09% of Seciera demonstrated statistical superiority over placebo vehicle for the co-primary efficacy endpoint of change from baseline at week 12 in total conjunctival lissamine green staining |
11/16/15 |
DBV Technologies SA (Bagneux, France) |
Viaskin milk |
Patch based on epicutaneous immunotherapy |
Milk allergy-induced eosinophilic esophagitis |
The first patient has been enrolled in SMILEE, a phase IIa trial |
11/16/15 |
Ohr Pharmaceutical Inc. (New York) |
OHR-102 |
Squalamine lactate ophthalmic solution, 0.2% combination therapy |
Wet age-related macular degeneration |
Additional data from the phase II IMPACT study showed that the size of occult choroidal neovascularization was most important in predicting treatment success in the combination of OHR-102 plus Lucentis |
11/17/15 |
Protalix Biotherapeutics Inc. (Carmiel, Israel) |
PRX-102 |
A plant cell-expressed, chemically modified recombinant alpha-galactosidase-A enzyme |
Fabry disease |
End-of-phase II meeting with the FDA indicated the agency's acceptance of the proposed phase III trial to support a full BLA approval; it will begin next year |
11/17/15 |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Proellex |
Telapristone acetate |
Symptomatic uterine fibroids |
Completed randomization for its phase IIb trial |
11/16/15 |
Samumed LLC (San Diego) |
SM04554 |
Topical small-molecule compound |
Androgenetic alopecia (AGA), or male pattern baldness |
Completed a phase II trial in which no serious adverse events were observed |
11/24/15 |
Viking Therapeutics Inc. (San Diego) |
VK5211 |
Nonsteroidal selective androgen receptor modulator |
Hip fracture |
Dosing has begun in a phase II trial |
11/4/15 |
RESPIRATORY | |||||
Afferent Pharmaceuticals Inc. (San Mateo, Calif.) |
AF-219 |
P2X3 antagonist |
Idiopathic pulmonary fibrosis with cough |
Started a phase II trial that will enroll 30 patients |
11/12/15 |
Aimmune Therapeutics Inc. (Brisbane, Calif.) |
AR101 |
Oral immunotherapy |
Peanut allergy |
Phase II data confirmed efficacy results in patients who rolled over from placebo and no treatment-related serious adverse events occurred |
11/12/15 |
Discovery Laboratories Inc. (Warrington, Pa.) |
Aerosurf |
Aerosolized KL4 surfactant |
To prevent respiratory distress syndrome |
Top-line phase IIa data in premature infants suggest it is generally safe and well tolerated and may decrease nCPAP failure and the need for intubation; it is now moving into phase IIb |
11/13/15 |
Invion Ltd. (Brisbane, Australia) |
INV102 |
Nadolol |
Mild asthma |
Completed enrollment in its phase II study |
11/3/15 |
Verona Pharma plc (Cardiff, UK) |
RPL554 |
An inhaled PDE3/PDE4 inhibitor |
Asthma and chronic obstructive pulmonary disease |
Completed patient enrollment for the phase IIa dose-finding study in asthma and the phase IIa combination study in COPD |
11/6/15 |
|
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. For more information about individual companies and/or products, see Thomson Reuters Cortellis. | |||||