Company (Location) |
Product |
Description |
Indication |
Status (Date)# |
Aeterna Zentaris Inc. (Quebec City) and Keryx Biopharma-ceuticals Inc. (New York) |
Perifosine |
Akt inhibitor |
Refractory and relapsed Hodgkin lymphoma |
Phase II data showed when used with Nexaver it significantly increased median progression-free survival in patients with high phosphorylation levels of Erk and Akt (12/14) |
AMAG Pharmaceuticals Inc. (Lexington, Mass.) |
Feraheme |
Ferumoxytol |
Iron deficiency anemia |
Clinical data showed no serious adverse events and 61% of patients achieved a greater than 2 g/dL rise in hemoglobin by week eight with a single 1,020-mg dose rather than two 510-mg doses separated by three to eight days (12/13) |
Array BioPharma Inc. (Boulder, Colo.) |
ARRY-614 |
A small-molecule dual p38/Tie2 inhibitor |
Myelodysplastic syndromes |
Phase I data showed activity as measured by hematologic improvement; it was generally well tolerated (12/13) |
Ariad Pharmaceuticals Inc. (Cambridge, Mass.) |
Ponatinib |
Pan-BCR-ABL inhibitor |
Chronic myelogenous leukemia; chronic myeloid leukemia or Philadelphia-positive acute lymphoblastic leukemia |
Phase II data showed the most common serious adverse event was pancreatitis, and efficacy was strong with an overall major cytologic response rate of 46% for chronic phase patients and major hematologic response of 74% in accelerated phase patients (12/12); Phase II data demonstrated that 47% of chronic-phase CML patients achieved a major cytogenetic response, including 65% of those who have a T315I mutation (12/13) |
Ariad Pharmaceuticals Inc. (Cambridge, Mass.) |
Gene therapy |
Gene therapy |
Hemophilia B |
Phase I data showed levels were between 3% and 12% of normal levels after treatment, compared with less than 1% of normal before treatment (12/13) |
Astellas Pharma Inc. (Tokyo) and Ambit Biosciences Corp. (San Diego) |
AC220 |
Quizartinib |
Acute myeloid leukemia |
Phase II data showed the monotherapy had activity in refractory and relapsed FLT3-ITD-positive AML (12/14) |
Avila Therapeutics Inc. (Bedford, Mass.) |
AVL-292 |
Bruton's tyrosine kinase inhibitor |
Chronic lymphocytic leukemia |
Data from the first two patient cohorts in an ongoing Phase Ib trial showed the drug demonstrated biological activity in CLL patients (12/13) |
Biogen Idec Inc. (Cambridge, Mass.) and Roche AG (Basel, Switzerland) |
Rituxan |
Rituximab |
Asymptomatic low-tumor-burden follicular lymphoma |
Phase III data comparing two different doses found that, following an induction course of the drug, repeated treatment with Rituxan at the time of progression is just as effective in managing disease as ongoing maintenance therapy (12/13) |
Bio-Path Holdings Inc. (Houston) |
BP-100-1.01 |
Lipsomal Grb-2; a systemic liposomal antisense treatment |
Blood cancers |
Phase I data suggested the drug is well tolerated (12/14) |
Biovest International Inc. (Tampa, Fla.) |
BiovaxID |
Active immunotherapy |
Mantle cell lymphoma |
Phase II data showed vaccination following rituximab combination chemotherapy induced nearly universal immune responses, which strongly correlated with overall survival in treated patients (12/14) |
Celator Pharmaceuticals Inc. (Princeton, N.J.) |
CPX-351 |
Cytarabine:dauno-rubicin liposome injection |
Acute myeloid leukemia |
Phase IIb data showed a statistically significant benefit in overall survival favoring the drug in first relapse patients (12/13) |
Celgene International Sarl (Boudry, Switzerland) |
Istodax |
HDAC inhibitor; romidepsin |
Peripheral T-cell lymphomas |
Phase II data showed overall response rates were similar across three common stubtypes: 29% in PTCL not otherwise specified, 30% in angioimmunoblastic TCL and 24% in anaplastic lymphoma kinase-1 negative ALCL (12/14) |
Celgene International Sarl (Boudry, Switzerland) |
Pomalidomide |
Oral therapy |
Multiple myeloma |
Phase II data demonstrated a partial response or better in 34% of patients receiving pomalidomide and dexamethasone vs. 13% in the pomalidomide-only group (12/14) |
Celgene Corp. (Summit, N.J.) |
Revlimid |
Lenalidomide |
Multiple myeloma; myelodysplastic syndromes |
Data showed improvements in progression-free survival when used as a maintenance therapy and as an induction therapy in newly diagnosed patients (12/12); median progression-free survival was 31 months in patients 75 years or older taking continuous Revlimid therapy with MPR-R (12/13); a retrospective analysis showed that Revlimid-treated patients benefited from a reduction in the risk of death, while no risk of progression to AML was observed (12/13); overall response rate was 72% in Phase II trial of Revlimid and Vidaza (12/14); Phase III data in patients with high-risk asymptomatic smoldering multiple myeloma demonstrated that early treatment with Revlimid and dexamethasone followed by continous lenalidomide delayed time to symptomatic disease and demonstrated a projected survival advantage (12/14) |
Celgene Corp. (Summit, N.J.) |
Vidaza |
Oral azacitidine |
AML, myelodysplastic syndromes or chronic myelomonocytic leukemia |
Phase I data showed that four of 15 patients achieved hematologic improvement, including those who had complex cytogenetics and/or had failed prior therapy (12/13) |
Cell Therapeutics Inc. (Seattle) and Chroma Therapeutics Ltd. (Oxford, UK) |
Tosedostat |
An inhibitor of aminopeptidases |
Acute myeloid leukemia |
Phase II data showed 51% of patients achieved disease control, and 22% achieved major leukemic response (12/14) |
Clavis Pharma ASA (Oslo, Norway) |
Elacytrabine |
A lipid-conjugated form of cytarabine |
Acute myeloid leukemia |
Interim efficacy data from a Phase II trial showed that in combination with idarubicin it produced a complete remission rate in about 46% of patients (12/13) |
Cornerstone Pharmaceuticals Inc. (Cranbury, N.J.) |
CPI-613 |
Cancer metabolism inhibitor |
Relapsed acute myeloid leukemia and myelodysplastic syndromes |
Phase I data showed the activity of CPI-613, with several patients experiencing significant clinical benefit, including a sustained complete remission when treated with it as a single agent (12/13) |
Coronado Biosciences Inc. (Burlington, Mass.) |
CNDO-109 |
A biologic that activates natural killer cells |
Acute myeloid leukemia and solid tumors |
Phase I data showed patients experienced a longer complete remission after receiving CNDO-109 activated NK cells than their previouis CR (12/14) |
Cyclacel Pharmaceuticals Inc. (Berkeley Heights, N.J.) |
Sapacitabine |
Oral therapy |
Newly diagnosed acute myeloid leukemia |
Phase I/II data showed an overall response rate of 40% (12/13) |
CytRx Corp. (Los Angeles) |
Bafetinib |
An oral Bcr-Abl, Lyn and Fyn kinase inhibitor |
B-cell chronic lymphocytic leukemia |
Phase II data demonstrated clinical activity and preliminary safety (12/13) |
Daiichi Sankyo Co. Ltd. (Tokyo) |
Edoxaban |
A direct oral, once-daily Factor Xa inhibitor |
Venous thromboembolism |
Results of a pooled analysis showed it significantly reduced the risk of developing venous thromboembolism following total knee or hip arthroplasty when compared to enoxaparin (12/13) |
Eli Lilly and Co. (Indianapolis) |
LY2784544 |
Small-molecule Janus kinase 2 inhibitor |
Myelo-proliferative neoplasms |
Phase I data showed it reduced spleen length by at least 35% in 13 of 17 evaluable patients (12/13) |
Fate Therapeutics Inc. (San Diego) |
ProHema |
FT1050-enhanced umbilical cord blood |
Hematologic malignancies |
Phase Ib data showed it achieved its primary objective of demonstrating safety and tolerability based on patient engraftment by day 42 (12/14) |
Genmab A/S (Copenhagen, Denmark) |
HuMax-CD38 |
Daratumumab |
Multiple myeloma |
Phase I/II data showed a 49%, a 55% and a 61% reduction in the serum M-component in the three patients treated at the highest dose level examined (12/13) |
Immunomedics Inc. (Morris Plains, N.J.) |
Milatuzumab |
Anti-CD74 antibody |
B-cell non-Hodgkin's lymphoma |
Phase I/II data of milatuzumab combined with second-generation anti-CD20 antibody veltuzumab showed no dose-limiting toxicities (12/14) |
Immunomedics Inc. (Morris Plains, N.J.) |
Veltuzumab |
A subcutaneous formulation of an anti-CD20 antibody |
Immune thrombocytopenia |
Data showed that two doses produced an overall objective response rate of 67%, including an 18% durable complete response rate, in 39 evaluable patients (12/13) |
Incyte Corp. (Wilmington, Del.) |
Jakafi |
Ruxolitinib |
Myelofibrosis |
Phase III data showed consistent benefit of the drug over placebo in spleen volume reduction and symptom improvement across subgroups, as well as an overall survival advantage (12/13) |
Inspiration Biopharma-ceuticals Inc. (Laguna Niguel, Calif.) |
IB1001 |
A recombinant Factor IX product |
Hemophilia |
Data indicated no evidence of the development of inhibitors when used during prophylactic treatment in a subset of patients who underwent a second pharmacokinetic evaluation; the initial crossover study demonstrated it is at least as good as BeneFIX (12/13) |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) |
ISIS-FXIRx |
An antisense inhibitor of Factor XI |
Clotting disorders |
Phase I data demonstrated that it produced dose-dependent statistically significant reductions of up to 78% in Factor XI activity and was safe and well tolerated (12/13) |
Janssen-Cilag International NV (Beerse, Belgium) |
Velcade |
Bortezomib |
Multiple myeloma |
Five-year follow-up data from the VISTA trial showed that Velcade, in combination with melphalan and prednisone, delivered a significant long-term overall survival benefit of 13.3 months compared to treatment with melphalan and prednisone alone in previously untreated disease (12/13) |
Merrimack Pharmaceuticals Inc. (Cambridge, Mass.) |
MM-121 |
A fully human monoclonal antibody that targets ErbB3 |
Advanced cancers |
The first patient has been dosed in a Phase I study (12/14) |
Micromet Inc. (Bethesda, Md.) |
Blinatum-omab |
BiTE antibody |
Acute lymphoblastic leukemia |
Phase II data showed that it more than doubled the complete remission rate produced by standard therapies (12/13) |
Millennium (Cambridge, Mass.; unit of Takeda Pharmaceutical Co. Ltd.) |
MLN9708 |
Oral proteasome inhibitor |
Multiple myeloma |
Phase I data showed one of 46 patients achieved a complete response and five achieved partial responses (12/14) |
Novartis AG (Basel, Switzerland) |
INC424 |
JAK inhibitor; ruxolitinib |
Myelofibrosis |
Phase III data showed substantial improvement in health-related quality of life and myelofibrosis symtpoms compared with baseline (12/14) |
Onconova Therapies Inc. (Newtown, Pa.) |
Rigosertib |
A small-molecule inhibitor of PLK1 and PI3K |
Myelodysplastic syndrome |
Phase I/II data showed a substantial portion of patients with clinically significant reduction or stabilization in blasts, with a correlation evident between blast reduction and overall survival benefit (12/13) |
Onyx Pharmaceuticals Inc. (South San Francisco) |
Carfilzomib |
A proteasome inhibitor |
Multiple myeloma |
Phase II data showed an overall response rate of 52% and a clinical benefit rate of 64% (12/14) |
Pharmacyclics Inc. (Sunnyvale, Calif.) |
PCI-32765 |
Selective irreversible Bruton's kinase inhibitor |
Relapsed or refractory chronic lymphocytic leukemia; mantle cell lymphoma |
Phase Ib/II data showed a partial response rate of 70% (12/13); Phase II interim data demonstrated an overall response of 69% in patients with relapsed or refractory mantle cell lymphoma (12/13) |
ReGenX BioSciences LLC (Washington) |
NAV rAAV8 |
Vectors that encode genes for anti-HIV neutralizing antibodies |
Hemophilia B |
Phase I/II data demonstrated correction of hemophilia B using the vectors to express a missing clotting factor (12/16) |
S*BIO Pte Ltd. (Singapore) |
Pacritinib |
JAK2 inhibitor |
Myelofibrosis |
Phase II data demonstrated that it reduced splenomegaly, with minimal impact on existing cytopenias (12/13) |
Seattle Genetics Inc. (Bothell, Wash.) and Millennium (Cambridge, Mass.) |
Adcetris |
Brentuximab vedotin |
Relapsed or refractory systemic anaplastic large-cell lymphoma |
Pivotal data showed that 86% of patients achieved an objective response with a median duration of 13.2 months (12/13) |
Symphogen A/S (Copenhagen, Denmark) |
Rozrolimupab |
A polyclonal antibody |
Immune thrombocytopenic purpura |
Phase II data showed it induced a rapid increase in blood platelets (12/14) |
Syndax Pharmaceuticals Inc. (Waltham, Mass.) |
Entinostat |
A small-molecule inhibitor of Class I histone deacetylases |
Hodgkin lymphoma |
Phase II data showed activity as a single agent (12/13) |
Telik Inc. (Palo Alto, Calif.) |
Telintra |
A small molecule product candidate designed to inhibit the enzyme, glutathione S-transferase P1-1 (GSTP1-1) |
Myelodysplastic syndromes |
Phase I data showed 40% of patients had hematologic improvement in red blood cell levels at a dose of 2,000 mg Telintra and 10 mg Revlimid (12/14) |
TetraLogic Pharmaceuticals Inc. (Malvern, Pa.) |
TL32711 |
Smac mimetic |
Acute myeloid leukemia |
New data showed that it induced apoptosis in AML cells (12/13) |
YM Biosciences Inc. (Mississauga, Ontario) |
CYT387 |
JAK1/JAK2 inhibitor |
Myelofibrosis |
A trial in 163 patients showed a median duration of treatment of 6.6 months; it was well tolerated and had substantial clinical activity (12/12); a trial in 166 patients produced transfusion independence in more than half of the participants for at least 12 weeks with significant improvements in splenomegaly and constitutional symptoms (12/14) |
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. |