Company* (Country; Symbol)




Status (Date)


Active Biotech AB (Sweden; SSE: ACTI)

Laquinimod (SAIK-MS)

Small-molecule immunomodulatory agent

Multiple sclerosis

Phase IIb trial in relapsing-remitting MS patients met its primary endpoint of reducing inflammatory disease activity (9/5)

Avidia Inc.*


Inhibitor of interleukin-6

Crohn's disease

Began Phase I trial in Australia to evaluate safety, tolerability and pharmacokinetics in adults with Crohn's disease (9/20)

Debiopharm Group* (Switzerland)

Debio-0513 (PTR-262)

Dual-altered ligand peptide analogue of the myasthenogenic epitopes of the acetylcholine receptor

Myasthenia gravis

The product was granted orphan designation in Europe in that indication (9/21)

Nitec Pharma AG* (Switzerland)


Modified-release tablet formulation of prednisone

Rheumatoid arthritis

Phase III trial in 288 patients in Europe showed significantly reduced morning stiffness; an MAA seeking European approval was filed in August (9/28)


Abraxis BioScience Inc. (ABBI)

Abraxane (FDA-approved)

Paclitaxel protein-bound particles for injection; albumin-bound

Breast cancer

The EMEA accepted for review the application seeking approval in Europe (9/28)

Active Biotech AB (Sweden; SSE:ACTI)


Tumor angiogenesis suppression by quinolines

Hormone-refractory prostate cancer

Interim data from Phase I trial in Sweden showed a reduced rate of PSA increase in all 10 patients evaluated (9/20)

Adherex Technologies Inc. (AMEX:ADH)


Oral dihydropyrimidine dehydrogenase inhibitor

Liver cancer

Began Phase I/II trial in patients in Asia to evaluate drug with 5-FU (9/28)

Antisoma plc (UK; LSE:ASM)


Small molecule derived from xanthenone acetic acid; vascular disrupting agent

Non-small-cell lung cancer

Final data from Phase II trial showed median survival of 14 months vs. 8.8 months in the chemotherapy-only arm (9/28)

Astex Therapeutics Ltd.* (UK)


Inhibitor of the Aurora A and B kinases


Began Phase I/IIa trial to evaluate safety, tolerability and preliminary efficacy (9/26)

BioAlliance Pharma SA (France; Euronext:BIO)

Doxorubicin Transdrug

Nanoparticle formulation of doxorubicin

Liver cancer

Phase I/II trial in 20 patients in France showed five partial responses and seven disease stabilizations (9/19)

Bioenvision Inc. (BIVN)

Evoltra (FDA-approved)

Clofarabine; second-generation purine nucleoside analogue

Pediatric acute lymphoblastic leukemia

New data from ongoing study in Europe confirmed a significant response rate in a difficult-to-treat patient population (9/21)

Gendux AB* (Sweden) and Introgen Therapeutics Inc. (INGN)


Adenoviral vector containing the p53 tumor-suppressor gene

Li-Fraumeni syndrome

The product was granted orphan designation in Europe in that indication (9/7)

lmmutep SA* (France)

ImmuFact IMP321

Human T-celI immuno-stimulatory factor


Began Phase I trial in Belgium to evaluate HLA-A2 melanoma peptide antigens with or without IMP321 and/or Montanide (9/25)

Peregrine Pharmaceuticals Inc. (PPHM)

Bavituximab (formerly Tarvacin)

Anti-phosphotidylserine immunotherapeutic


Is starting a Phase I trial in India to evaluate safety and tolerability with chemotherapy regimens (9/11)


Alexion Pharmaceuticals Inc. (ALXN)

Soliris (eculizumab)

Humanized monoclonal antibody designed to block complement protein C5

Paroxysmal nocturnal hemoglobinuria

Submitted MAA with the EMEA, seeking approval of the product in Europe (9/26)

Biopure Corp. (BPUR)


Oxygen therapeutic consisting of bovine hemoglobin

For use in surgery procedures

MAA filed in the UK in July was accepted for review; approval is being sought in acutely anemic adult orthopedic surgery patients younger than 80 (9/12)

Genfit SA* (France)


Activator of peroxisome proliferator-activated receptors

Cardiometabolic disease

Began a Phase I trial of the drug candidate (9/19)

Speedel Group (Switzerland; SWX:SPPN) and Novartis AG

SPP100 (Rasilez)

Oral renin inhibitor


The EMEA accepted for review Novartis' submission seeking European approval of drug as both a monotherapy and as part of combination therapies (9/28)

ThromboGenics NV (Belgium; Euronext:THR)


Truncated form of the human protein plasmin

Acute ischemic stroke

Began Phase IIa MITI IA trial in Germany to evaluate drug in up to 20 patients with vertebro-basilar artery occlusion (9/22)


EpiCept Corp. (EPCT)


Patch designed to provide sustained topical delivery of lidocaine


Phase III trial in Europe in 440 patients who underwent hernia repair surgery failed to demonstrate efficacy vs. placebo (9/5)

GW Pharmaceuticals plc (UK; LSE:GWP)


Spray formulation of cannabis extract

Spasticity in multiple sclerosis patients

Filed for approval under the decentralized procedure in the UK, Spain, Denmark and the Netherlands; the UK is the reference member state (9/5)

Lexicon Genetics Inc. (LEXG)


Small-molecule inhibitor of a membrane protein expressed in the central nervous system

Cognitive disorders

Began Phase I trial in the UK to evaluate safety, tolerability and pharmacokinetics in healthy volunteers (9/6)

Medivation Inc. (AMEX:MDV)


Neuroprotectant agent that's been on the market in Russia since 1983

Alzheimer's disease

Phase II trial in 183 patients in Russia demonstrated statistical significance on primary and all secondary efficacy endpoints (9/21)

Metabolic Pharmaceuticals Ltd. (Australia; ASX:MBP)


Inhibitor of neuronal nicotinic acetylcholine receptors; derived from Australian cone snail

Neuropathic pain

Began Phase IIa trial in Australia to evaluate safety and efficacy in 40 patients with neuropathic sciatic pain (9/27)

Trophos SA* (France)


Compound believed to promote the survival of a range of neurons

Amyotrophic lateral sclerosis

The product was granted orphan designation in Europe in that indication (9/5)

WEX Pharmaceuticals Inc. (Canada; TSX:WXI)


Tetrodotoxin; non-narcotic agent

Cancer-related pain

Canadian regulators approved company's request to resume clinical trials of the drug (9/6)

YM BioSciences Inc. (Canada; TSX:YM)


Aerosolized liposome-encapsulated fentanyl


Phase IIb trial will enroll the planned 99 patients after interim analysis on the first 67 did not demonstrate predefined significance (9/27)


Amylin Pharmaceuticals Inc. (AMLN) and Eli Lilly and Co.

Byetta (FDA-approved)

Exenatide; incretin mimetic

Type II diabetes

The CHMP of the EMEA recommended approval of the drug in that indication (9/21)

Amylin Pharmaceuticals Inc. (AMLN) and Eli Lilly and Co.

Byetta (FDA-approved)

Exenatide; incretin mimetic

Type II diabetes

One-year data from trial in Europe showed drug was as effective as biphasic insulin aspart 30/70 in improving blood sugar levels in those with unacceptable blood sugar control (9/14)

ConjuChem Biotechnologies Inc. (Canada; TSX:CJB)

PC-DAC: Exendin-4

Albumin-bound analogue of glucagon-like peptide-1

Type II diabetes

Final data from Phase I/II trial confirmed tolerability, positive efficacy on glucose reduction and extended duration of activity (9/12)

Diamyd Medical AB (Sweden; SSE:DIAM)


Agent containing GAD65, the dominating autoantigen in autoimmune diabetes

Type I diabetes

Presented further data from Phase II trial in Sweden, showing positive efficacy and safety (9/18)

NicOx SA (France; Euronext:NICOX)

NCX 4016

Nitric oxide-donating derivative of acetyl salicylic acid

Type II diabetes

Data from Phase II subgroup and Phase I trial in volunteers showed increase in glucose utilization and in glucose uptake, respectively (9/6)


Idenix Pharmaceuticals Inc. (IDIX) and Novartis AG (Switzerland)


Oral, once-daily nucleoside analogue

Hepatitis B

The drug was approved in Switzerland for treating chronic hepatitis B in compensated patients with viral replication and active liver inflammation (9/21)

PowderMed Ltd.* (UK)


H3-based DNA vaccine


Is starting trial in healthy volunteers in the UK to evaluate effectiveness against a flu challenge (9/21)

Nektar Therapeutics Inc. (NKTR)


Amphotericin B inhalation powder

Fungal infections

The product was granted orphan designation in Europe for preventing pulmonary fungal infections in at-risk patients (9/18)

Novexel* (France)


Small-molecule bacterial topoisomerase/gyrase inhibitor

Bacterial infections

Began Phase I development of the compound (9/26)



Modified form of fragment of bactericidal/permeability increasing protein

Meningococcal disease

The product was granted orphan designation in Europe in that indication (9/6)


Ardana plc (UK; LSE:ARA)


Testosterone cream


Open-label Phase II trial in 16 men showed product effectively restored testosterone levels to the normal range (9/11)

Ark Therapeutics Group plc (UK; LSE:AKT)


Agent designed to increase the ability of mitochondria to produce energy

Lipodystrophy in HIV patients

Extension data from Phase II trial showed improvements in metabolic risk factors (9/5)

Can-Fite BioPharma Ltd. (Israel; TEL:CFBI)


Agent that targets the A3 adenosine receptor

Dry-eye syndrome

Is starting a Phase II trial in Israel to evaluate the product in 50 patients (9/6)

Intercytex Ltd.* (UK)


Human dermal fibroblasts embedded in a fibrin gel

Diabetic foot ulcers

Began open-label Phase II trial in the UK to evaluate wound closure in up to 15 patients (9/5)

Neurochem Inc. (Canada; NRMX)

Kiacta (formerly Fibrillex)

Glycosaminoglycan mimetic with anti-amyloid properties

Amyloid A amyloidosis

The EMEA accepted for review application seeking approval of the product in Europe (9/28)


* Privately held.

CHMP = Committee for Medicinal Products for Human Use; EMEA = European Medicines Agency; MAA = Marketing authorization application; MRP = Mutual Recognition Procedure.

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; OTC BB = Over-the-Counter Bulletin Board; SSE = Stockholm Stock Exchange; SWX = Swiss Stock Exchange; TEL = Tel Aviv Stock Exchange; TSX = Toronto Stock Exchange.

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