Company |
Product |
Description |
Indication |
Status |
Date |
Acetylon Pharmaceuticals Inc. (Boston) |
ACY-1215 |
HDAC6 inhibitor |
Relapsed or relapsed/refractory multiple myeloma |
Phase I/II study showed that in combination with Velcade and dexamethasone it may provide a well-tolerated treatment option |
12/12/12 |
Aeterna Zentaris Inc. (Quebec City) |
Perifosine |
Oral AKT inhibitor |
Relapsed/refractory lymphomas |
Final Phase II data demonstrated that the combination of perifosine and Nexavar was well tolerated by heavily pretreated patients, and it showed promising clinical response activity in patients with classical Hodgkin lymphoma |
12/12/12 |
Ambit Biosciences Inc. (San Diego) |
Quizartinib |
Inhibits the Flt3-ITD kinase |
Acute myeloid leukemia |
Phase II data showed treatment led to increases in overall survival that were modest, but the drug did improve patients' overall states sufficiently to enable them to get stem cell transplants, thereby improving survival more noticeably |
12/11/12 |
AMAG Pharmaceuticals Inc. (Lexington, Mass.) |
Ferumoxytol |
Anemia therapy |
Iron deficiency anemia |
Phase III data in subjects who had failed or could not tolerate oral iron treatment ferumoxytol achieved both primary efficacy endpoints |
12/11/12 |
Ariad Pharmaceuticals Inc. (Cambridge, Mass.) |
Ponatinib |
Bcr-Abl inhibitor |
Resistant or refractory chronic myeloid leukemia or Philadelphia chromosome-positive acute lymphoblastic leukemia |
Pivotal study showed that 56% of chronic-phase CML patients in the trial, including 70% of patients with a T315I mutation, achieved a major cytogenetic response; 12-month follow-up data from its PACE trial showed that 57% of CML patients achieved major hematologic response, including 50% with the T315I mutation |
12/11/12; 12/12/12 |
Array BioPharma Inc. (Boulder, Colo.) |
ARRY-520 |
A kinesin spindle protein inhibitor |
Multiple |
Phase II data showed that patients with triple-refractory multiple myeloma and a median number of 10 prior treatment regimens demonstrated a 22% overall response rate when given ARRY-520 plus low-dose dexamethasone |
12/12/12 |
Astex Pharmaceuticals Inc. (Dublin, Calif.) |
SGI-110 |
A second-generation hypomethylation agent |
Relapsed/refractory intermediate or high-risk myelodysplastic syndromes or acute myelogenous leukemia |
Phase I data showed it had a differentiated pharmacokinetic profile resulting in an extended half-life and exposure to decitabine as delivered via subcutaneous administration |
12/11/12 |
Baxter International Inc. (Deerfield, Ill.) |
BAX 326 |
A recombinant Factor IX protein |
For bleeding episodes for patients with hemophilia B |
Pivotal study results showed that twice-weekly prophylactic treatment achieved a median annualized bleed rate of 1.99, with 43% of patients experiencing no bleeds |
12/11/12 |
Biothera Inc. (Eagan, Minn.) |
Imprime PGG |
Immunomod-ulatory drug |
High-risk chronic lymphocytic leukemia |
Phase I data of Imprime PGG, Campath and Rituxan combination therapy showed 64% achieved a complete response |
12/11/12 |
Boehringer Ingelheim GmbH (Ingelheim, Germany) |
Volasertib |
A small molecule inhibitor of the PLK1 protein |
Acute myeloid leukemia |
Phase II data showed higher rates of objective response and a trend for longer median event-free survival when given in combination with low-dose cytarabine vs. cytarabine alone |
12/11/12 |
Bristol-Myers Squibb Co. (New York) and Abbott (Abbott Park, Ill.) |
Elotuzumab |
Monoclonal antibody |
Multiple |
Phase II data of elotuzumab (10 mg/kg and 20 mg/kg) in combination with lenalidomide and low-dose dexamethasone showed that in the 10 mg/kg arm, median progression-free survival (PFS) was not reached after 20.8 months of follow-up (N = 36) and the objective response rate (ORR) was 92%; of patients who received elotuzumab at a dose of 20 mg/kg, median PFS was 18.6 months (N = 37) and ORR was 76% |
12/11/12 |
Celator Pharmaceuticals Inc. (Princeton, N.J.) |
CPX-351 |
A fixed-dose combination of the established cancer drugs cytarabine and daunorubicin |
Acute myeloid leukemia |
Phase Iib data showed it was highly active in all patient subgroups and had the greatest response rate in patients with secondary AML and adverse cytogenetics |
12/12/12 |
Celgene Corp. (Summit, N.J.) |
CC-486 |
Oral azacitidine |
Myelodysplastic syndromes |
Phase I data showed that oral, epigenetic therapy resulted in an overall response rate of 42.3% in the 14-day treatment arm, with a response rate of 37% (10 of 27 patients) in the 21-day treatment arm |
12/11/12 |
Celgene International Sarl (Boudry, Switzerland) |
Revlimid |
Lenalidomide |
Untreated acute myeloid leukemia |
A Phase II study of Revlimid plus Vidaza (azacitidine) in patients 60 or older showed an overall response rate of 41%, with 28% achieving a complete response |
12/12/12 |
Clavis Pharma ASA (Oslo, Norway) |
Elacytarabine |
A modified form of cytarabine |
Acute myeloid leukemia |
Phase II data of elacytarabine and idarubicin showed the combination elicited a complete response in 13 of 46 evaluable patients (28%) and an overall response rate of 43.5% |
12/11/12 |
Cyclacel Pharmaceuticals Inc. (Berkeley Heights, N.J.) |
Sapacitabine |
Oral capsules |
Newly diagnosed acute myeloid leukemia |
Updated survival data from its Phase III SEAMLESS trial showed 19 patients, or 41%, responded, with 10 complete responses, four partial responses and five major hematological improvements; median overall survival was 238 days, or about eight months, with 35% of patients surviving one year or longer |
12/11/12 |
Genmab A/S (Copenhagen, Denmark) |
HuMax(R)-CD38 |
Daratumumab |
Multiple |
Phase I/II data showed that two patients achieved a partial response and one achieved a minimal response |
12/11/12 |
Gentium SpA (Villa Guardia, Italy) |
Defibrotide |
The sodium salt of a complex mixture of single-stranded oligodeoxribonucleotides derived from procine mucosal DNA |
Hepatic veno-occlusive disease in hematopoietic stem cell transplant |
30% of patients achieved a complete response; 50% survived to day 100 |
12/14/12 |
Geron Corp. (Menlo Park, Calif.) |
Imetelstat |
Telomerase inhibitor |
Thrombo-cythemia |
Phase II data showed it produced rapid and durable hematologic and molecular responses |
12/11/12 |
Glycomimetics Inc. (Gaithersburg, Md.) |
GMI-1070 |
A glycomimetic inhibitor of E-, P- and L-selectins |
Sickle cell disease |
A pilot study showed that the drug affected biomarkers significant to sickle cell disease; the drug had a significant effect on biomarkers associated with cell adhesion, activation and coagulation |
12/12/12 |
ImmunoGen Inc. (Waltham, Mass.) |
IMGN901 |
A drug consisting of the company's DM1 cancer cell-killing agent attached to CD56-binding antibody lorvotuzumab |
Multiple |
Phase I data showed that IMGN901 in combination with Revlimid and dexamethasone in CD56-expressing relapsed or relapsed/refractory multiple myeloma produced a 64% clinical response, and 31% of patients had stable disease |
12/12/12 |
Immunomedics Inc. (Morris Plains, N.J.) |
Veltuzumab |
Single agent, subcutaneously administered; a second-generation humanized anti-CD20 antibody |
Relapsed immune thrombocytopenia |
Data demonstrated an overall objective response rate of 50%, with 12 patients (29%) having a complete response, meaning their platelet levels rose to or above 100,000 per uL |
12/12/12 |
Incyte Corp. (Wilmington, Del.) |
Jakafi |
JAK inhibitor ruxolitinib |
Myelofibrosis |
Data showed improved survival over placebo and best available therapy, suggesting an overall survival benefit in patients with intermediate or high-risk MF |
12/12/12 |
Infinity Pharmaceuticals Inc. (Cambridge, Mass.) |
IPI-145 |
Oral inhibitor of PI3K-delta and PI3K-gamma |
Advanced hematologic malignancies |
Phase I data showed the drug to be well tolerated and clinically active in both B-cell and T-cell malignancies |
12/11/12 |
Millennium: The Takeda Oncology Co. (Cambridge, Mass.) |
MLN9708 |
Once-a-week therapy |
Newly diagnosed multiple myeloma |
Phase I/II data of MLN9708 in combination with standard-dose lenalidomide and dexamethasone showed that two achieved a complete response and one achieved a VGPR |
12/11/12 |
Millennium: The Takeda Oncology Co. (Cambridge, Mass.) |
Velcade |
Bortezomib |
Newly-diagnosed multiple myeloma |
A meta-analysis of 23 Velcade-based studies showed that, across studies in which overall response rate (ORR) data were available, the pooled, weighted average ORR was 39%, including 57% in relapsed-only patients and 23% in refractory-only patients |
12/11/12 |
Millennium: The Takeda Oncology Co. (Cambridge, Mass.) |
MLN9708 |
Once-a-week proteasome inhibitor |
Relapsed or refractory systemic light-chain amyloidosis |
A Phase I trial determined a maximum-tolerated dose of 4 mg |
12/12/12 |
Millennium: The Takeda Oncology Co. (Cambridge, Mass.) |
Adcetris |
Brentuximab vedotin |
Newly diagnosed advanced stage Hodgkin lymphoma |
Phase I data of Adcetris in combination with ABVD (adriamycin, bleomycin, vinblastine and dacarbazine) showed a complete remission of 95%, while Adcetris with AVD showed a complete remission of 96% |
12/12/12 |
Novartis AG (Basel, Switzerland) |
Tasigna |
Nilotinib |
Philadelphia chromosome-positive chronic myelogenous leukemia |
Phase III data showed that patients achieved significantly deeper molecular responses vs. Glivec (imatinib) |
12/12/12 |
Onconova Therapeutics Inc. (Newtown, Pa.) |
Rigosertib |
Multikinase PI3K inhibitor |
Myelodysplastic syndromes and acute myeloid leukemia |
Phase I/II data showed that single-agent rigosertib was well tolerated and achieved effects that were in line with previous studies |
12/12/12 |
OxiGene Inc. (South San Francisco) |
OXi4503 |
Vascular disrupting agent |
Acute myelogenous leukemia |
Phase I showed an overall response of 40%, with one patient achieving a marrow complete remission and another achieving partial remission |
12/11/12 |
Pharmacyclics Inc. (Sunnyvale, Calif.) |
PCI-32765 |
Bruton's tyrosine kinase inhibitor ibrutinib |
Treatment-naive chronic lymphocytic leukemia or small lymphocytic lymphoma and for those with relapsed/refractory (RR) or high-risk disease |
Phase Ib/II data of 116 patients showed an overall response rate of 68% in elderly treatment-naive patients with an estimated 96% progression-free survival rate at 26 months |
12/11/12 |
Pharmacyclics Inc. (Sunnyvale, Calif.) |
PCI-32765 |
Bruton's tyrosine kinase inhibitor ibrutinib |
Relapsed or refractory mantle cell lymphoma |
Phase II data suggested that in Velcade-naïve and Velcade-exposed patients, the drug resulted in high and durable responses and was generally well tolerated |
12/12/12 |
Pharmacyclics Inc. (Sunnyvale, Calif.) |
PCI-32765 |
Bruton's tyrosine kinase inhibitor ibrutinib |
Relapsed and refractory chronic lymphocytic leukemia, small lymphocytic lymphoma, mantle cell lymphoma, diffuse large B-cell lymphoma, follicular lymphoma and multiple myeloma |
Showed prolonged progression-free survival with a favorable safety profile in a number of indications |
12/13/12 |
Portola Pharmaceuticals Inc. (South San Francisco) |
Betrixaban |
Oral Factor Xa inhibitor anticoagulant |
To prevent venous thromboembolism |
Phase III data demonstrated limited drug-drug interactions with betrixaban |
12/11/12 |
Sanofi SA (Paris) |
SAR302503 |
A selective JAK2 inhibitor |
Intermediate-2 or high-risk primary or secondary myelofibrosis |
Phase II data showed that treatment reduced spleen size and improved constitutional symptoms |
12/11/12 |
Seattle Genetics Inc. (Bothell, Wash.) |
Adcetris |
Brentuximab vedotin |
Newly diagnosed mature T-cell lymphoma |
Phase II data of Adcetris in combination with chemotherapy showed 100% of patients had an objective response, including 23 (88%) with a complete remission |
12/11/12 |
Seattle Genetics Inc. (Bothell, Wash.) |
Adcetris |
Brentuximab vedotin |
Mycosis fungoides and relapsed cutaneous T-cell lymphoma |
Phase II trials of patients with MF or Sezary syndrome showed that 14 of 20 patients (70%) achieved an objective response across all stages of disease; in CTCL patients, 67% achieved an objective response |
12/12/12 |
Spectrum Pharmaceuticals Inc. (Henderson, Nev.) |
Zevalin |
Yttrium-90-ibritumomab tiuxetan |
Cancer |
Phase II data showed Zevalin plus BEAM chemotherapy (carmustine, etoposide, cytarabine and melphalan) prior to autologous stem cell transplantation in refractory non-Hodgkin's diffuse large B-cell lymphoma produced a two-year overall survival rate of 65% and a progression-free survival rate of 63% after a median follow-up of 22.7 months; Phase II data of Zevalin following R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone) therapy showed that, for Stage I and Stage II patients with CD20-positive diffuse large B-cell lymphoma who completed Zevalin consolidation, 87% were in complete response (CR/Cru) and 89% were in functional CR; at four years, 88% of patients remained progression-free and 98% were alive |
12/14/12 |
Telik Inc. (Palo Alto, Calif.) |
Ezatiostat |
A glutathione S-transferase inhibitor |
Idiopathic chronic neutropenia (ICN) suffering Grade 4 neutropenia |
Phase II data showed it resulted in durable increases in the white blood cell levels, leading to clinically significant reductions in serious infections; ezatiostat was well tolerated and may be appropriate for long-term therapy |
12/14/12 |
YM BioSciences Inc. (Mississauga, Ont.) |
CYT387 |
JAK1/JAK2 inhibitor |
Myelofibrosis |
Phase I/II data showed a 68% durable 12-week transfusion independence response rate with a maximal duration of response approaching three years and ongoing |
12/11/12 |
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. |