(The annual meeting was held Dec. 4-7 in Orlando, Fla.)

Company

(Country)

Product

Description

Indication

Status (Date)#

Abbott and Bristol-Myers Squibb Co.

Elotuzumab

Targets a cell-surface glycoprotein that is present on multiple myeloma cells

Multiple

myeloma

Interim Phase II data of elotuzumab plus lenalidomide and low-dose dexamethasone showed that 90% of 31 patients achieved an objective response (12/8)

Aeterna Zentaris Inc. (Canada) and Keryx Biopharma-ceuticals Inc.

Perifosine

An oral Akt inhibitor

Chronic lymphocytic leukemia and relapsed or refractory Hodgkin's lymphoma

Phase II data showed it achieved objective response and a high rate of stable disease as a single-agent therapy in CLL and in combination with Nexavar in Hodgkin's lymphoma (12/7)

Alder Biopharma-ceuticals Inc.

ALD518

Interleukin-6-targeting antibody

Advanced non-small-cell lung cancer

Phase II data showed that 58% of advanced cancer patients with anemia experienced hemoglobin level increases from less than 11 g/dL to more than 12 g/dL vs. no patients experiencing that increase in the placebo arm at 12 weeks of treatment (12/8)

Alexion Pharmaceuticals Inc.

Samalizumab

Humanized monoclonal antibody

Advanced stage B-cell chronic lymphocytic leukemia or multiple myeloma

Interim data from the first Phase I/II trial showed it was well tolerated at all doses studied, exhibited a dose-dependent biological and pharmacokinetic response and exhibited initial evidence of antitumor activity (12/7)

Alexion Pharmaceuticals Inc.

Soliris

Eculizumab

Paroxysmal nocturanl hemoglobinuria

Long-term data showed that patients achieved survival comparable to that of an age- and gender-matched normal population when treated with Soliris (12/8)

Allos Therapeutics Inc.

Folotyn

Pralatrexate

injection

Peripheral T-cell lymphoma

Phase II data demonstrated the ability of Folotyn to achieve responses in patients in the second-line treatment setting (12/7)

Amgen Inc.

Nplate

Romiplostim

Adult chronic immune thrombocytopenic purpura

Five-year, open-label extension study results showed platelet cells were safely maintained between 50,000 and 200,000 per microliter in a majority of patients for up to 277 weeks (12/7)

Ariad Pharmaceutical Inc.

AP24534

Ponatinib

Chronic myelogenous leukemia

About two-thirds of patients who were resistant to two or three receptor tyrosine kinase inhibitors had a major cytogenetic response, and half achieved a complete response (12/7)

Array BioPharma Inc.

ARRY-520

Kinesin spindle protein inhibitor

Multiple

myeloma

Phase I data showed it was well tolerated and had encouraging results (12/7)

Baxter Healthcare Corp.

rVWF

Recombinant von Willebrand factor

von Willebrand disease

Phase I data showed it may be safe and well tolerated (12/7)

BioCryst Pharmaceuticals Inc.

Forodesine

Purine nucleoside phosphorylase inhibitor

Chronic

lymphocytic leukemia

Phase II data confirmed clinical activity (12/7)

Biovest International Inc.

BiovaxID

Personalized vaccine

Non-Hodgkin's lymphoma

Phase III data suggested that improved disease-free survival rates following administration with BiovaxID depend on an integral tumor protein fragment administered as part of each patient's vaccine (12/7)

Bristol-Myers Squibb Co. and Otsuka Pharmaceutical Co. Ltd. (Japan)

Sprycel

Dasatinib

Chronic myeloid leukemia

An 18-month follow up from its Phase III trial of Sprycel vs. Gleevec was consistent with data at 12 months, showing Sprycel produced higher and faster rates of complete cytogenic response (12/7)

Calistoga Pharmaceuticals Inc.

CAL-101

Oral, delta-isoform-selective P13K inhibitor

Hematologic malignancies

Phase I data demonstrated the clinical potential of CAL-101 as a single agent and in combination with other standard-of-care therapies (12/7)

Celator Pharmaceuticals Inc.

CPX-351

Cytarabine: daunorubicin

Acute myeloid leukemia

Phase II data showed that elderly patients with newly diagnosed disease had a higher aplasia rate, a higher remission rate, lower mortality, improved median event-free survival and improved median overall survival than patients receiving conventional cytarabine and daunorubicin (12/8)

Celgene Corp.

Revlimid

Lenalidomide

Multiple myeloma; indolent non-Hodgkin's lymphoma; myelodysplastic syndromes

Phase III data showed a 60% reduction in the risk of disease progression and a median time to progression of 42.3 months for Revlimid vs. 21.8 months for placebo; another trial showed a 50% reduction in the risk of disease progression, with median progression-free survival of 42 months vs. 24 months for placebo; in newly diagnosed patients, those 65 and older had an 85% two-year survival on low-dose and 72% on high dose, while those younger than 65 had 92% and 86%, respectively (12/7); Phase II data showed an average 58% overall response rate with Revlimid and low-dose dexamethasone plus rituximab, and a second study in relapsed or refractory patients showed an overall response rate of 76%; in myelodysplastic syndromes with a del(5q) chromosome abnormality, Phase III data showed the three-year overall survival rate was 56%

Celgene Corp.

Pomalidomide

IMiD compound

Multiple myeloma

Phase II data of pomalidomide plus low-dose dexamethasone showed an overall response rate of 42% and 39% in patients receiving the therapy for 21 and 28 days, respectively (12/8)

Cell Therapeutics Inc.

Pixuvri

Pixantrone dimaleate

Relapsed or refractory aggressive non-Hodgkin's lymphoma

Phase III data showed continuous improvement in primary and secondary endpoints; complete response was 24% in the pixantrone arm vs. 7% in the comparator arm (12/7)

ChemGenex Pharmaceuticals Ltd. (Australia)

Omapro

Omacetaxine mepesuccinate

Chronic myeloid leukemia

Clinical data showed it produced durable hematologic and cytogenetic responses in a significant proportion of chronic phase patients (12/7)

Cytheris SA (France)

CYT107

Recombinant human interleukin-7

Hematological malignancies

Phase I data showed a median increase in CD4 T cells exhibiting a naive or central memory phenotype of 69% over baseline in eight evaluable post-transplant patients (12/8)

Emergent BioSolutions Inc.

TRU-016

Humanized anti-CD37 small modular immunopharmaceutical candidate

B-cell malignancies

Phase I data showed favorable response rates and it was generally well tolerated (12/7)

EntreMed Inc.

ENMD-2076

An Aurora A/angiogenic kinase inhibitor

Relapsed/refractory multiple myeloma; relapsed/refractory leukemia

Interim Phase I data showed it was well tolerated with three of nine evaluable patients showing stable disease and one achieving a partial response; data from 20 evaluable patients showed that daily administration was associated with reductions in bone marrow blast counts of 11%, 14% and 65% (12/8)

Gentium SpA (Italy)

Defibrotide

A DNA-based drug

Veno-occlusive disease

Clinical data showed that patients who were not on dialysis or were not ventilator-dependent at study entry had a 41% complete response rate compared to a 23% response rate in patients who were (12/8)

Geron Corp.

GRNVAC1

An autologous dendritic cell vaccine targeting telomerase

Acute myelogenous leukemia

Phase II data said it may prolong remission duration in some patients with high-risk AML (12/7)

HemaQuest Pharmaceuticals Inc.

HQK-1001

An orally administered small chain fatty acid derivative

Sickle cell disease and beta thalassemia

Data showed the drug was well tolerated with significant drug-related adverse events in both trials (12/8)

ImmunoGen Inc.

IMGN901

Designed to target and kill cancer cells that express the CD56 antigen

Multiple

myeloma

Phase I data were positive (12/7)

Immunomedics Inc.

Milatuzumab and veltuzumab

A humanized anti-CD74 antibody and a humanized anti-CD20 antibody

Non-Hodgkin's lymphoma

Combination treatment resulted in therapeutic responses in heavily pretreated and Rituxan-refractory patients (12/7)

Immunomedics Inc.

Y-90 epratuzumab

Yttrium-90 labeled epratuzumab

Diffuse B-cell lymphoma

Conslidation treatment with fractionated Y-90, after induction therapy with rituximab and CHOP chemotherapy, improved remission status of adult patients (12/7)

Incyte Corp.

INCB18424

A selective, oral JAK1 and JAK2 inhibitor

Advanced polycythemia vera and essential thrombocythemia

Phase II data showed long-term clinical activity, including reduction in spleen size, phelebotomy independence and improvement in blood counts lasting up to 27 months (12/7)

MethylGene Inc. (Canada)

MGCD0103

Mocetinostat

Hodgkin

lymphoma

Phase II data showed a complete response for two out of 43 patients, partial response for 12 patients and durable disease for at least six months for one patient (12/7)

Micromet Inc.

MT103

Blinatumomab

Acute lymphoblastic leukemia

Phase II data demonstrated that it produced prolonged remissions in patients with ALL (12/7)

Millennium (unit of Takeda Pharmaceutical Co. Ltd.)

MLN4924

A small-molecule inhibitor of the NEDD8-activating enzyme

Acute myeloid leukemia or high-grade myelodysplastic syndromes

Phase I data showed that four patients achieved a complete response (12/8)

Millennium Pharmaceuticals Inc. and Janssen-Cilag (Belgium)

Velcade

Bortezomib

Relapsed multiple myeloma; relapsed or refractory follicular lymphoma

Data from a 222-patient study comparing subcutaneous vs. intravenous administration showed the efficacy was similar in both administrations with one-year overall survival of 72.6% in the subcutaneous group and 76.7% in the intravenous group (12/7); Phase III data of Velcade and rituximab showed an improvement in progression-free survival and prolonged the time to next therapy over rituximab alone (12/9)

Novartis Pharmaceutical Corp.

LBH589

Panobinostat

Hodgkin lymphoma

Pivotal Phase II data showed that it demonstrated substantial disease control and tumor reduction in pretreated patients who had relapsed or become refractory after an autologous stem cell transplant (12/7)

Novartis Pharmaceutical Corp.

Tasigna

Nilotinib

Chronic myeloid leukemia

Phase III data showed that patients treated with a 300 mg dose twice daily had a lower incidence of progression to accelerated phase and blast crisis, compared to Gleevec 400 mg once daily (12/7)

Onconova Therapeutics Inc.

Estybon

ON 01910 Na

Acute myeloid leukemia

Data from 48 higher risk patients who failed or became resistant to previous drug treatments showed they had increases in overall survival when treated with Estybon (12/7)

Onyx Pharmaceuticals Inc.

Carfilzomib

Next-generation proteasome

inhibitor

Multiple myeloma

Phase IIb data demonstrated that in a subset analysis of 128 patients refractory to Velcade showed an overall response rate of 19% (12/8)

Pharmacyclics Inc.

PCI-32765

Btk-selective inhibitor

Chronic lymphocytic leukemia; B cell malignancies

Phase I data of 54 patients showed it was well tolerated (12/7); Phase Ia study showed an overall response rate of 45%, with the highest response rates in mantle cell lymphoma (78%) and chronic/small lymphocytic leukemia (56%) (12/8)

PharmaMar SA (Spain)

Aplidin

Plitidepsin

Peripheral T-cell lymphoma

Phase II data showed it was effective and safe, with two full responses and four partial responses in the 32 patients treated (12/8)

PolyMedix Inc.

PMX-60056

A synthetic, small molecule designed to reverse the anticoagulant activity of heparin and low-molecular-weight heparin

For use during cardiac surgery

Phase Ib/II data showed clinicians can accurately predict a single reversing dose of the drug that will safely and effectively neutralize the anticoagulation effects of heparin (12/8)

Roche AG

GA101

Monoclonal

antibody

Relapsed and refractory non-Hodgkin's lymphoma

Phase II data showed that a third of patients who had tried three previous therapies responded (12/7)

S*BIO Pte. Ltd. (Singapore)

SB1518

JAK2 inhibitor

Symptomatic myelofibrosis and enlarged spleens

Phase I and II data indicated clinical efficacy and good tolerability (12/7)

S*BIO Pte. Ltd. (Singapore)

SB939

Oral histone deacetylase inhibitor

Advanced hematologic malignancies

Phase I data showed tolerability and safety (12/8)

Seattle Genetics Inc.

SGN-35

Brentuximab vendotin

Relapsed Hodgkin's lymphoma; systemic anaplastic large cell lymphoma

Data showed that in 102 patients who had been failed by a median of 3.5 prior treatments, almost all patients, or 94%, achieved some sort of tumor reduction, and a third went into complete remission (12/7); Phase IIb data showed a 97% tumor volume reduction, an 86% objective response and a 53% complete response (12/8)

Spectrum Pharmaceuticals Inc.

Zevalin

Ibritumomab tiuxetan

Indolent follicular non-Hodgkin's lymphoma; advanced B-cell lymphoma

Phase III data showed that patients treated with a single-dose infusion of Zevalin after partial or complete response to first-line chemotherapy had a nearly three-year advantage in median progression-free survival compared to patients treated with chemotherapy alone or chemotherapy plus Rituxan (12/7); results showed non-myeloablative allogeneic transplant can provide prolonged remissions, and that its use is safe, feasible and can lead to early objective responses and prolonged disease control in certain patients (12/8)

Stemline Therapeutics Inc.

SL-401

A cancer stem cell-targeting agent

Acute myeloid leukemia and myelodysplastic syndromes

Phase I data showed efficacy at tolerable doses (12/8)

Synta Pharmaceuticals Corp.

STA-9090

A small-molecule Hsp90 inhibitor

Acute myeloid leukemia

Phase I/II trial showed early signs of clinical activity, including instances of long-term disease control (12/8)

Telik Inc.

Telintra

Ezatiostat hydrochloride

Myelodysplastic syndromes

Phase II data showed a significant response rate among various patient groups (12/7)

YM BioSciences Inc. (Canada)

CYT387

JAK1/JAK2 inhibitor

Myelofibrosis

Interim Phase I/II data showed an overall response rate of 62% (12/7)

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.