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.