Company*
(Country;
Symbol)

Product

Description

Indication

Status (Date)


AUTOIMMUNE

Antisense
Therapeutics
Ltd.
(Australia;
ASX:ANP)

ATL1102

Second-generation antisense inhibitor of VLA-4, an immune system protein

Multiple sclerosis

Received approval to restart trial in Europe, a Phase IIa study to assess safety and activity in 80 patients(1/18)

Biogen Idec
Inc.
(BIIB) and
Fumapharm
AG*
(Switzerland)

BG-12

Oral fumarate derivative with an immunomodulatory mechanism of action

Multiple sclerosis

Phase II trial in 250 patients in Europe demonstrated statistically significant reductions in gadolinium-enhancing brain lesions (1/9)

Novagali
Pharma*
(France)

Nova22007

Ophthalmic formulation of cyclosporine A

Sjogren syndrome

Phase II trial demonstrated safety, as well as efficacy trends, in patients suffering SS associated with keratoconjunctivitis sicca (1/11)

CANCER

Affymax Inc.*

Hematide

Synthetic, peptide-based erythropoiesis-stimulating agent

Anemia in cancer patients

Began Phase II trial in Europe to evaluate safety and pharmacokinetics in cancer patients (1/30)

AmpliMed
Corp.*

Amplimexon

Imexon injection; a cyanoaziridine compound believed to disrupt mitochondria

Ovarian cancer

The product was granted orphan designation in Europe in that indication; the drug is in Phase I/II trials (1/11)

Ark
Therapeutics
Group plc
(UK; LSE:AKT)

Vitor

Small molecule originally developed for high blood pressure

Muscle wasting in cancer patients

Full data from 200-patient trial showed statistical significance was reached in colorectal and non-small-cell lung cancers but not in pancreatic cancer(1/16)

Genta Inc.
(GNTA)

Genasense

Oblimersen sodium; inhibits function of bcl-2 protein

Advanced malignant melanoma

Completed MAA filing in Europe seeking approval for use in combination with chemotherapy (1/3)

Proacta Inc.*

PR-104

Hypoxia-targeted small-molecule prodrug

Hypoxic tumors

Began Phase I trial in Australia and New Zealand to evaluate the agent (1/24)

SuperGen Inc.
(SUPG)

Orathecin

Rubitecan; topoisomerase-I inhibitor in capsule formulation

Pancreatic cancer

Withdrew MAA seeking approval in Europe after discussions with the EMEA; future plans will depend on ongoing trials (1/20)

Vion Pharmaceuticals
Inc.
(VION)

Cloretazine

Sulfonylhydrazine DNA alkylating agent

Acute myeloid leukemia

The product was granted orphan designation in Europe in that indication (1/23)

Xanthus
Life Sciences
Inc.*

Symadex (C-1311)

Next-generation product based on the existing cancer drug mitoxantrone

Metastatic colorectal cancer

Began trial in Europe to evaluate responses in 49 patients who failed oxaliplatin and/or irinotecan regimens (1/17)

Xanthus
Life Sciences
Inc.*

Symadex (C-1311)

Next-generation product based on the existing cancer drug mitoxantrone

Metastatic breast cancer

Began trial in Europe in 49 women who relapsed following treatment with an anthracycline and taxane regimen (1/3)

CARDIOVASCULAR

Gentium SpA
(Italy; AMEX:GNT)

efibrotide

Single-stranded DNA designed to protect vascular endothelial cells

Veno-occlusive disease

Began Phase II/III trial in Europe and Israel to evaluate the product in 270 pediatric patients under-going stem cell transplantation (1/20); data from study in Japan showed three of four patients responded to therapy; two remained alive at 100 days (1/30)

GPC Biotech
AG
(Germany;
GPCB)

1D09C3

Major histocompatibility complex class II monoclonal antibody

Chronic lymphocytic leukemia

The product was granted orphan designation in Europe in that indication for which Phase I trials are ongoing (1/27)

Speedel Holding
Ltd.
(Switzerland;
SWX:SPPN)

SPP100 (aliskiren; Rasilez)

Oral renin inhibitor

Hypertension

Partner Novartis AG reported positive Phase III data of drug when used as a monotherapy and with a calcium channel blocker and an ACE inhibitor (1/19)

United
Therapeutics
Corp.
(UTHR)

Remodulin (FDA-approved)

Treprostinil sodium injection

Pulmonary arterial hypertension

Israeli officials expanded the label there to include treatment of PAH associated with connective tissue disorders (1/10)

CENTRAL NERVOUS SYSTEM

Trophos*
(France)

TRO19622

Compound believed to promote the survival of a range of neurons

Neuro- degenerative diseases

Phase I trial in France showed the agent was safe, well tolerated and achieved the desired dose (1/31)

Vectura
Group plc
(UK; LSE:VEC)

VR040

Inhaled formulation of apomorphine

Parkinson's disease

The product was granted orphan designation in Europe for treating "off episodes" in PD patients (1/18)

YM BioSciences
Inc.
(Canada;
TSE:YM)

AeroLEF

Aerosolized liposome- encapsulated fentanyl

Pain

YM subsidiary Delex Therapeutics Inc. began Phase IIb trial in Canada in 120 post-surgical patients following orthopedic surgeries (1/26)

DIABETES

Generex
Biotechnology
Corp.
(Canada;
GNBT)

Oral-lyn

Oral insulin buccal spray formulation

Type I diabetes

Began trial in 27 juvenile patients in Ecuador (1/9); began trial in 60 patients in Europe to evaluate metabolic efficacy vs. regular insulin therapy

Nektar
Therapeutics
Inc.
(NKTR) and
Pfizer Inc.

Exubera

Dry-powder, inhaled insulin formulation that uses Nektar technology

Type I and II diabetes

The product was approved in the European Union, where it will be marketed by Pfizer (1/26)

INFECTION

Chiron Corp.
(CHIR) and
Cubist
Pharmaceuticals
Inc.
(CBST)

Cubicin (FDA-approved)

Daptomycin for injection; a bactericidal antibiotic

Complicated skin and soft-tissue infections

The product was approved in Europe for treating cSSTIs caused by Gram-positive bacteria (1/23)

Intercell AG
(Austria; VSE:ICLL)

IC41

Hepatitis C vaccine

Hepatitis C

Optimization trial demonstrated improved immunogenicity when delivered via optimized route (1/31)

Intercell AG
(Austria; VSE:ICLL)

--

Inactivated vaccine for immunizing against Japanese encephalitis virus

Japanese encephalitis virus

The vaccine was granted orphan designation in the European Union (1/30)

Intercell AG
(Austria; VSE:ICLL)

--

Inactivated vaccine for immunizing against Japanese encephalitis virus

Japanese encephalitis virus

Monitors saw no safety concerns in Phase III trial, allowing for completion of recruitment for the program (1/12)

MISCELLANEOUS

BioMarin
Pharmaceutical
Inc.
(BMRN)

Naglazyme (FDA-approved)

Galsulfase; an enzyme- replacement therapy

Mucopoly- saccharidosis-VI

The product was approved in the European Union, where it has orphan status (1/30)

Genzyme
Corp.
(GENZ)

Myozyme

Recombinant human acid alpha-lucosidase enzyme

Pompe disease

A regulatory committee in the European Union recomended full approval of the drug (1/27)

Inspire
Pharmaceuticals
Inc.
(ISPH)

Denufosol tetrasodium

P2Y2 receptor agonist

Cystic fibrosis

The EMEA granted orphan designation to the product in Europe (1/10)

ISTA
Pharmaceuticals
Inc.
(ISTA)

Vitragan (Vitrase; FDA- approved)

Ovine hyaluronidase formulation

Vitreous hemorrhage

The EMEA accepted for review the MAA seeking European market approval (1/12)

LAB
International
Inc.
(Canada;
TSE:LAB)

LAB CGRP

Calcitonin gene related peptide; vasodilative agent

Asthma

Phase I trial demonstrated safety, with no adverse events (1/24)

Serono SA
(Switzerland;
NYSE:SRA)

Gonal-f (FDA-approved)

Recombinant human follicle-stimulating hormone

Hypogonadism

The product was approved in Japan for inducing spermatogenesis in men with hypogonado- tropic hypogonadism (1/24)


Notes:

* Privately held.

MAA = Marketing authorization application; MRP = Mutual Recognition Procedure; EMEA = European Medicines Agency.

Unless otherwise noted, the stock symbols listed for public companies are on the Nasdaq market.

AMEX = American Stock Exchange; ASX = Australian Stock Exchange; LSE = London Stock Exchange; NYSE = New York Stock Exchange; SWX = Swiss Stock Exchange; TSE = Toronto Stock Exchange ; VSE = Vienna Stock Exchange.

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