Company
(Location)

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
myeloma

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
myeloma

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
myeloma

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
myeloma

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.