Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Active Biotech AB (Lund, Sweden)

Nerventra

Laquinimod

Relapsing-remitting multiple sclerosis

CHMP issued a negative opinion, concluding that the risk-benefit profile was not favorable at this time

1/27/14

CANCER

Arqule Inc. (Woburn, Mass.)

Tivantinib

Tyrosine kinase inhibitor

Hepatocellular carcinoma

A data monitoring committee recommended the phase III trial continue with a lower dose of 120 mg twice daily, which showed a reduced incidence of neutropenia; top-line phase III data in Asia of tivantinib and Tarceva in patients with advanced or metastatic nonsquamous non-small-cell lung cancer with wild-type epidermal growth factor receptor indicated that overall survival favored the treatment arm, compared to the erlotinib-only control arm; the result, however, was not statistically significant

1/21/14

Biovest International Inc. (Minneapolis)

BiovaxID

Personalized cancer vaccine

Non-Hodgkin's lymphoma

EMA accepted the MAA

1/15/14

Celgene International Sarl (Boudry, Switzerland; a unit of Celgene Corp.)

Abraxane

Nab-paclitaxel

First-line metastatic adenocarcinoma of the pancreas or metastatic pancreatic cancer

European Commission approved it for use in combination with gemcitabine

1/8/14

Cell Therapeutics Inc. (Seattle)

Pactrinib

Oral JAK2/FLT3 inhibitor

Relapsed acute myeloid leukemia

Started an international cooperative group phase II trial of pacritinib in adult patients with mutations of the FLT3 gene; approximately 80 patients at sites in England and Wales will be enrolled and, if an encouraging response rate is observed, a pacritinib arm may be adopted in the first-line therapy study

1/31/14

Celltrion Inc. (Incheon, South Korea)

Herzuma

Biosimilar monoclonal antibody trastuzumab, a biosimilar of Herceptin

Advanced HER2-positive breast cancer and stomach cancer

Received approval in Korea

1/16/14

Cel-Sci Corp. (Vienna, Va.)

Multikine

Leukocyte interleukin, injection

Head and neck cancer

Received approval from Serbian regulators allowing it to start enrolling patients in its ongoing phase III head and neck cancer study

1/24/14

Halozyme Therapeutics Inc. (San Diego)

Mabthera

Rituximab

Non-Hodgkin's lymphoma

CHMP recommended that the European Commission approve the subcutaneous formulation

1/27/14

Helix Biopharma Corp. (Aurora, Ont.)

L-DOS47

An immunoconjugate candidate

Inoperable, locally advanced, recurrent or metastatic, nonsquamous stage IIIB/IV non-small-cell lung cancer

Opened patient screening for the sixth dose level cohort of its ongoing phase I/II trial in Poland, following completion of the first treatment cycle of the three patients enrolled in the fifth cohort, in which therapy was well tolerated

1/24/14

Novocure (Haifa, Israel)

NovoTTF

Anti-mitotic treatment

Locally advanced and metastatic pancreatic adenocarcinoma

The first patient was enrolled in the open-label, pilot EF-20 Study, designed to examine NovoTTF therapy plus gemcitabine as first-line therapy; the study plans to recruit 20 patients from centers in Spain, Germany and Switzerland

1/27/14

Prima Biomed Ltd. (Sydney)

Cvax

An autologous dendritic cell-based product

Epithelial ovarian cancer

The amended CAN-004 phase II protocol was approved by Belgian regulators, and it will enroll a new cohort of 210 patients

1/21/14

Seattle Genetics Inc. (Bothell, Wash.) and Takeda Pharmaceutical Co. Ltd. (Osaka, Japan)

Adcetris

Brentuximab vedotin

CD30-positive relapsed or refractory Hodgkin’s lymphoma and anaplastic large cell lymphoma

Received approval in Japan

1/21/14

Synta Pharmaceuticals Corp. (Lexington, Mass.)

Ganetespib

Inhibits the heat shock protein 90 chaperone protein

Metastatic ovarian cancer

Launched GANNET53, a pan-European randomized trial evaluating its combination with paclitaxel vs. paclitaxel alone in more than 200 patients

1/13/14

CARDIOVASCULAR

Aegerion Pharmaceuticals Inc. (Cambridge, Mass.)

Juxtapid

Lomitapide

To reduce cholesterol in homozygous familial hypercholes-terolemia

Mexican authorities approved it as an adjunct treatment to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol, total cholesterol, apolipoprotein B and non-high-density lipoprotein cholesterol

1/22/14

INFECTION

Actelion Ltd. (Allschwil, Switzerland)

Zavesca

Miglustat

Niemann-Pick Type C

Company filed an application for approval in China

1/8/14

Cell Medica Ltd. (London)

Cytovir ADV

T-cell immunotherapy

Adenovirus infections following allogeneic hematopoietic stem cell transplant

European Commission for Orphan Medicinal Products issued a positive opinion on the application

1/8/14

Cytos Biotechnology AG (Zurich, Switzerland)

gH1-Qbeta

Vaccine

Influenza

Met the primary endpoint for immunogenicity (seroconversion based on hemaglutination inhibition titers) in a phase I trial in healthy Asian volunteers

1/30/14

Gilead Sciences Inc. (Foster City, Calif.)

Sovaldi; nucleotide analogue polyermase inhibitor

Sofosbuvir 400 mg tablets; once-daily oral treatment

Chronic hepatitis C virus

European Commission granted marketing authorization

1/21/14

Viiv Healthcare Ltd. (Middlesex, UK)

Tivicay

Dolutegravir; an integrase inhibitor

HIV

European Commission approved Tivicay for use in combination with other antiretroviral medicinal products to treat HIV-infected adults and adolescents older than 12 years

1/22/14

MISCELLANEOUS

Biolinerx Ltd. (Jerusalem)

BL-5010P

A disposable, pen-like applicator containing BL-5010, an aqueous solution

For nonsurgical removal of benign skin lesions

Received approval in Germany

1/22/14

Cytonet GmbH & Co. KG (Weinheim, Germany)

N/A

Liver cell therapy

Urea cycle disorders

EMA validated the MAA

1/17/14

Finox Biotech AG (Burgdorf, Switzerland)

Bemfola

Follitropin alfa solution for injection in pre-filled pens

Infertility

CHMP adopted a positive opinion

1/24/14

Keryx Biopharma-ceuticals Inc. (New York) and Japan Tobacco Inc. (Tokyo)

Zerenex

Ferric citrate coordination complex

Hyperphos-phatemia in patients with chronic kidney disease

Received approval in Japan

1/21/14

NPS Pharmaceuticals Inc. (Bedminster, N.J.)

Natpara

Recombinant human parathyroid hormone

Hypopara-thyroidism

European Commission granted orphan drug designation

1/6/14

Proteon Therapeutics Inc. (Waltham, Mass.)

PRT-201

A recombinant elastase

To prevent arterovenous access dysfunction in hemodialysis patients

Received orphan drug designation in the European Union

1/9/14

PTC Therapeutics Inc. (South Plainfield, N.J.)

Ataluren

A protein restoration therapy

Duchenne muscular dystrophy

CHMP adopted a negative opinion on the MAA for conditional approval

1/27/14

Scioderm Inc. (Durham, N.C.)

SD-101

Topical cream

Epidermolysis bullosa

European Commission granted orphan drug designation

1/23/14

Tengion Inc. (Winston-Salem, N.C.)

Neo-Kidney Augment

Uses a patient's own cells to augment or replace renal function

To prevent or delay dialysis and transplantation in patients with advanced chronic kidney disease (CKD)

Neo-Kidney Augment was implanted in five CKD patients in an ongoing phase I trial in Sweden; the product is safe and well tolerated, and the company plans to expand enrollment in the trial to 15 patients

1/14/14


Notes

BLA = Biologics license application; CHMP = European Committee for Medicinal Products for Human Use; CMA = Continuous marketing application; EMA = European Medicines Agency; FDA = Food and Drug Administration; IND = Investigational new drug application; MAA = Marketing authorization application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment.

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.