Company*
(Country;
Symbol)

Product

Description

Indication

Status
(Date)


AUTOIMMUNE

Antisense
Therapeutics
Ltd.
(Australia;
ASX:ANP) and Isis
Pharmaceuticals
Inc.
(ISIS)

ATL1102

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

Multiple sclerosis

Began Phase IIa trial to evaluate preliminary efficacy in 60 patients with relapsing-remitting MS (12/21)

Biogen Idec Inc.
(BIIB) and Elan
Corp. plc (Ireland)

Tysabri (FDA-approved)

Natalizumab (formerly Antegren); humanized monoclonal antibody that inhibits adhesion molecules on immune cells

Multiple sclerosis

Began head-to-head STARS study to compare safety and efficacy to Rebif, Serono SA's interferon beta-1a product, in more than 1,000 patients worldwide (12/21)

Genmab A/S
(Denmark; CSE:GEN)
and Medarex
Inc.
(MEDX)

HuMax-Inflam/
MDX-018

High-affinity fully human antibody

Undisclosed autoimmune indication

16 of 28 patients in Phase I/II trial achieved a 50% or more reduction in disease activity one week after final dosing (12/21)

Genmab A/S
(Denmark; CSE:GEN)

HuMax-CD20

Human antibody that binds to the CD20 antigen

Rheumatoid arthritis

Got FDA OK to start a Phase I/II trial to evaluate safety and efficacy in patients who have failed a prior treatment (12/13)

InKine
Pharmaceutical
Co. Inc.
(INKP)

Colirest

Oral steroid molecule; Fc receptor technology

Crohn's disease

Company returned rights to the drug after a Phase IIb trial did not demonstrate sufficient efficacy (12/8)

Isis
Pharmaceuticals
Inc.
(ISIS)

ISIS 2302

Alicaforsen; an antisense inhibitor of intercellular adhesion molecule-1

Crohn's disease

Phase III trials involving 330 patients failed to demonstrate statistically significant induction of clinical remissions compared to placebo (12/2)

Isis
Pharmaceuticals
Inc.
(ISIS)

ISIS 2302

Alicaforsen; an antisense inhibitor of intercellular adhesion molecule-1

Ulcerative colitis

Phase II trials including more than 300 patients demonstrated durable responses and set the dose for Phase III trials (12/2)

Rigel
Pharmaceuticals
Inc.
(RIGL)

R406 and B cells

Oral syk kinase inhibitor that blocks activation of mast cells, macrophages

Rheumatoid arthritis

Began Phase I trial to evaluate safety and pharmacokinetics in 60 volunteers (12/21)

CANCER

Advanced Viral
Research Corp.
(OTC BB:ADVR)

AVR118

Peptide-nucleic acid complex with immuno-modulatory properties

Advanced malignancies

Is starting Phase II trial to test safety, tolerability and efficacy in advanced patients whose disease is progressing (12/27)

Allos
Therapeutics
Inc.
(ALTH)

Efaproxyn (efaproxiral; RSR13)

Small molecule designed to sensitize hypoxic areas of tumors prior to radiation

Brain metastases from breast cancer

Presented updated survival data from REACH trial, which is testing the drug as an adjunct to whole-brain radiation therapy (12/9)

American
BioSciences
Inc.
(APPX)

Abraxane

Nanoparticle albumin-bound taxane

Metastatic breast cancer

Phase II trial produced long-term disease control in taxane-refractory patients (12/13)

AnorMED Inc.
(Canada; TSE:AOM)

AMD3100

Agent designed to block receptor that triggers movement of stem cells out of bone marrow

Stem cell transplantation in cancer patients

All 20 patients treated in a Phase II trial with G-CSF achieved the target number of cells for transplantation; other data showed increased CD34+ cell counts (ASH)

AnorMED Inc.
(Canada; TSE:AOM)

AMD3100

Agent designed to block receptor that triggers movement of stem cells out of bone marrow

Stem cell transplantation in cancer patients

Began two pivotal Phase III trials under an FDA SPA, in non-hodgkin's lymphoma and in multiple myeloma; each will compare use with G-CSF to G-CSF alone in 300 patients (12/2)

AnorMED Inc.
(Canada; TSE:AOM)

AMD3100

Agent designed to block receptor that triggers movement of stem cells out of bone marrow

Stem cell transplantation in cancer patients

Began two Phase II trials, one in multiple myeloma and the other, with a mobilization regimen, in Hodgkin's disease (12/1)

ArQule Inc.
(ARQL)

ARQ 501

Activated Checkpoint Therapy molecule designed to selectively kill cancer cells

Advanced cancers

Began Phase Ib trial with Taxotere (12/7)

AVAX
Technologies
Inc.
(OTC BB:AVXT)

M-Vax

Autologous, hapten-modified melanoma vaccine

Melanoma

Got FDA OK for Phase I trial to test safety and immune responses in 65 patients; it is the first human study since the re-engineering of the manufacturing process (12/16)

BioCryst
Pharmaceuticals
Inc.
(BCRX)

Forodesine (BCX-1777)

Purine nucleoside phosphorylase inhibitor

Leukemias and lymphomas

Data from Phase I, I/II and IIa trials demonstrated the agent was well tolerated and produced encouraging efficacy signs (ASH)

Biogen
Idec Inc.
(BIIB)

Zevalin (FDA-approved)

Ibritumomab tiuxetan; monoclonal antibody targeting CD20 combined with radioisotope

Lymphomas

Drug was effective in trial of older patients with relapsed diffuse large B-cell lymphoma; drug with rituximab produced complete responses in 5 of 8 patients with previously untreated low-grade follicular non-Hodgkin's lymphoma; positive long-term data also were reported (ASH)

Biomira Inc.
(Canada; BIOM)
and Merck KGaA
(Germany)

BLP25

Therapeutic vaccine encapsulating a sequence of the MUC1 cancer mucin in a liposomal delivery system

Non-small-cell lung cancer

Median survival for 171 Stage IIIB patients in the vaccine arm in a Phase IIb trial had not been reached at 23 months, vs. 13.3 months in the control arm (12/3)

Celgene
Corp.
(CELG)

Thalomid (FDA-approved)

Thalidomide

Multiple myeloma

Phase II trial in 250 newly diagnosed patients showed a statistically significant difference in event-free survival with melphalan and prednisone vs. those agents alone (ASH)

Celgene
Corp.
(CELG)

Thalomid (FDA- approved)

Thalidomide

Multiple myeloma

Phase III trial with dexamethasone in 207 newly diagnosed patients showed statistically significant response rates vs. dexamethasone alone (ASH)

Celgene
Corp.
(CELG)

Revlimid (lenalidomide)

Derivative of Thalomid (thalidomide)

Myelodysplatic syndromes

Reported preliminary positive results from ongoing Phase II trial in patients with 5q deletion (ASH)

Celgene
Corp.
(CELG)

Revlimid (lenalidomide)

Derivative of Thalomid (thalidomide)

Multiple myeloma

25 of 30 patients achieved an objective response in the Phase II trial combining drug with dexamethasone and low-dose aspirin (ASH)

Cell
Genesys
Inc.
(CEGE)

GVAX

Whole-cell vaccine comprises tumor cells irradiated and modified to secrete GM-CSF

Acute myelogenous leukemia

Data on 19 patients from ongoing Phase II trial showed vaccine, given after chemotherapy, was well tolerated and may reduce residual leukemic cells (ASH)

Cell
Therapeutics
Inc.
(CTIC)

Pixantrone

Anthracycline agent designed to have improved properties

Non-Hodgkin's lymphoma

Complete responses were seen in 41% of patients who had failed the CHOP regimen, when drug was substituted for doxorubicin in the regimen (ASH)

CuraGen
Corp.
(CRGN)

CG53135

Fibroblast growth factor that promotes both epithelial and mesenchymal cell proliferation

Oral mucositis due to chemotherapy

Phase I trial showed the drug was well tolerated and supported move into Phase II (ASH)

Cytokinetics
Inc.
(CYTK)

SB-715992

Small-molecule inhibitor of kinesin spindle protein

Advanced ovarian cancer

Partner GlaxoSmithKline plc began Phase II trial to test safety and efficacy as a second-line treatment in 35 patients (12/16)

Dendreon
Corp.
(DNDN)

APC8024

Immunotherapy; vaccine that targets tumors expressing the HER2/neu marker

Advanced breast cancer

Analysis of 30 patients from Phase I trial showed a statistically significant T-cell immune response in HER2/neu-positive patients (12/13)

FibroGen
Inc.*

FG-2216

Small-molecule inhibitor of hypoxia-inducible factor-prolyl hydroxylase

Anemia

Phase I trial showed the drug was safe and well tolerated and stimulated production of erythropoietin and reticulocytes (ASH)

Gamida-Cell
Ltd.*
(Israel)

StemEx

Ex vivo expanded cord blood stem/progenitor cells with non-expanded cells from the same unit

Leukemia

Phase I/II trial in 10 patients undergoing StemEx transplants showed the therapy was safe (ASH)

Genentech
Inc.
(NYSE:DNA)
and Biogen
Idec Inc.
(BIIB)

Rituxan (FDA-approved)

Rituximab; antibody that binds to the CD20 antigen on the surface of normal and malignant B cells

Non-Hodkin's lymphoma and other cancers

More than 250 abstracts citing Rituxan were reported, including a trial showing advantages when drug is added to CHOP regimen (ASH)

Genmab A/S
(Denmark; CSE:GEN)

HuMax-CD20

Human antibody that binds to the CD20 antigen

Non-Hodgkin's lymphoma

Six of 11 patients with refractory or relapsed disease achieved a clinical response in a Phase I/II trial (ASH)

Genmab A/S
(Denmark; CSE:GEN)

HuMax-EGFr

Human antibody that targets the epidermal growth factor receptor

Head and neck cancer

Six of 15 patients in a Phase I/II trial showed a partial metabolic response and 3 patients showed stable metabolic disease (12/1)

Genta Inc.
(GNTA)

Genasense

Oblimersen sodium; inhibits function of bcl-2 protein

Relapsed/refractory chronic lymphocytic leukemia

Phase III trial in 241 patients showed drug plus fludarabine and cyclophosphamide significantly increased major responses vs. Flu/Cy alone (ASH)

Genta Inc. (GNTA)

Genasense

Oblimersen sodium; inhibits function of bcl-2 protein

Relapsed myeloma

18 of 30 patients in a Phase I/II trial of Genasense plus thalidomide and high-dose dexamethasone achieved a major response (ASH)

Genta Inc. (GNTA)

Genasense

Oblimersen sodium; inhibits function of bcl-2 protein

Acute myeloid leukemia

12 of 39 patients achieved complete remission in Phase II trial of Genasense plus Mylotarg in relapsed patients 60 and older (ASH)

Genta Inc. (GNTA)

Ganite (FDA-approved)

Gallium nitrate injection

Advanced non- Hodgkin's lymphoma

Phase II trial in 88 patients demonstrated activity in various subtypes of NHL (ASH)

ILEX
Oncology Inc.
(merged with
Genzyme Corp.;
GENZ)

Clofarabine

Second-generation purine nucleoside analogue

Acute leukemias in children

Additional data from Phase II trial showed a 30% response rate in refractory or relapsed lymphoblastic leukemia and 26% in myeloid leukemia (ASH)

Introgen
Therapeutics
Inc.
(INGN)

Advexin

Adenoviral vector containing the p53 tumor-suppressor gene

Unresectable head and neck cancer

Tumor growth control was achieved in 41% of patients, with an objective response rate of 15%; a rolling BLA filing is being planned (12/23)

Introgen
Therapeutics
Inc.
(INGN)

Advexin

Adenoviral vector containing the p53 tumor-suppressor gene

Breast cancer

Phase II trial of drug with neoadjuvant therapy in women with locally advanced disease showed a more than 50% reduction in tumor size in all patients (12/9)

Kosan
Biosciences
Inc.
(KOSN)

KOS-1584

A second-generation epothilone

Cancers

Began Phase I trial in advanced malignancies to test dosing, safety, pharmacokinetics and potential efficacy (12/8)

Ligand
Pharmaceuticals
Inc.
(LGND)

Ontak (FDA-approved)

Denileukin diftitox

Relapsed/ refractory T-cell non-Hodgkin's lymphoma

Data from 17 patients in a Phase II trial showed a 53% response rate, with another 29% of patients with stable disease; also, 4 of 10 patients responded in Phase II trial in fludarabine-refractory B- cell chronic lymphocytic leukemia (ASH)

Maxim
Pharmaceuticals
Inc.
(MAXM)

Ceplene

Histamine dihydrochloride in combination with interleukin-2

Acute myeloid leukemia

Additional data from Phase III trial in AML patients in complete remission included an updated p-value of 0.0096 for leukemia-free survival (ASH)

Medarex
Inc.
(MEDX)

MDX-060

Fully human antibody that targets CD30

Relapsed/refractory CD30-positive lymphomas

43% of 56 patients in Phase I/II trial experienced disease remission or stabilization (ASH)

MethylGene
Inc.
(Canada;
TSE:MYG)

MGCD0103

Isotypic-selective small-molecule inhibitor of histone deacetylase

Blood cancers

Began first of two Phase I trials; each is expected to enroll 18 to 25 patients (12/20)

Millennium
Pharmaceuticals
Inc.
(MLNM)

Velcade (FDA-approved)

Bortezomib; proteasome inhibitor

Multiple myeloma

Positive data were reported from a number of trials exploring the drug in treating all stages of MM, both alone and in combination regimens (ASH)

Millennium
Pharmaceuticals
Inc.
(MLNM)

Velcade
(FDA-approved)

Bortezomib; proteasome inhibitor

Indolent subtypes of non-Hodgkin's lymphoma

The overall response rates with single-agent treatment in three Phase II trials ranged from 60% to 17% in follicular lymphoma and from 54% to 39% in mantle cell lymphoma (ASH)

Myriad
Genetics Inc.
(MYGN)

MPC-2130 (formerly MPI-176716)

Broad-acting agent designed to induce apoptosis

Advanced cancers

Filed IND for Phase I trial to test safety and pharmacokinetics in patients with metastatic tumors or blood cancers, as well as refractory cancers (12/28)

Myriad
Genetics Inc.
(MYGN)

MPC-6827

Pro-apoptotic drug candidate

Advanced cancer

Filed IND for Phase I trial to test safety and pharmacokinetics in patients with advanced solid tumors (12/10)

NeoRx Corp.
(NERX)

Skeletal Targeted
Radiotherapy

Bone-targeting molecule DOTMP with radioactive substance holmium-166

Multiple myeloma

Updated data from Phase I/II trial in 10 patients showed a four-year survival rate of 70% and a complete response rate of 40% (ASH)

OxiGene
Inc.
(OXGN)

CA4P

Combretastatin A4 prodrug; a vascular targeting agent

Cancers

Began Phase II trial with carboplatin and paclitaxel to test safety, dosing and antitumor effects (12/16)

Pharmacyclics
Inc.
(PCYC)

Xcytrin

Motexafin gadolinium; small molecule from the texaphyrin class designed to induce apoptosis

Refractory chronic lymphocytic leukemia

Phase I trial in 13 patients showed evidence of antitumor activity in single- agent treatment (ASH)

Point
Therapeutics
Inc.
(POTP)

Talabostat (PT-100)

Small molecule designed to stimulate proliferation of hematopoietic progenitor cells

Non-Hodgkin's lymphoma

Phase I trial in 20 patients with indolent NHL demonstrated the safety and activity in combination with rituximab (ASH)

Procyon
Biopharma Inc.
(TSE:PBP)

PCK3145

Synthetic peptide analogue of prostate secretory protein

Metastatic prostate cancer

Filed IND for pilot study to assess dosing in 20 patients (12/14)

Seattle Genetics Inc.
(SGEN)

SGN-30

Monoclonal antibody that targets CD30+ hematologic malignancies

Anaplastic large cell lymphoma and Hodgkin's disease

Data from ongoing Phase II trial demonstrated drug was well tolerated and had antitumor activity (ASH)

Seattle
Genetics Inc.
(SGEN)

SGN-40

Humanized monoclonal antibody that targets the CD40 antigen

Non-Hodgkin's lymphoma

Began a Phase I trial to evaluate tolerability, pharmacokinetics and antitumor activity in 20 patients (12/2); showed agent was well tolerated in low doses (ASH)

SuperGen
Inc.
(SUPG)

Nipent (FDA-approved)

Pentostatin for injection

Leukemias

Presented various data, including regimen with cyclophosphamide and rituximab that demonstrated clinical activity in B-cell chronic lymphocytic leukemia with modest toxicity (ASH)

SuperGen Inc.
(SUPG) and
MGI Pharma
Inc.
(MOGN)

Dacogen

Decitabine for injection; a hypomethylating agent

Chronic myelogenous leukemia

Phase II data from 35 Gleevec-refractory patients showed an overall hematologic response rate of 65% (ASH)

SuperGen Inc.
(SUPG) and
MGI Pharma
Inc.
(MOGN)

Dacogen

Decitabine for injection; a hypomethylating agent

Myelodysplastic syndromes

Final data from Phase III trial showed an overall response rate of 17% with a median response duration of 266 days (ASH)

Telik Inc.
(TELK)

Telintra
(TLK199)

Glutathione analogue inhibitor of GST P1-1

Myelodysplatic syndromes

Data from ongoing Phase II trial showed 16 of 26 patients had clinically significant improvement in one or more blood cell lineages (ASH)

Therion
Biologics Corp.*

CEA-TRICOM

Therapeutic cancer vaccine

Cancers

Phase I trial in 58 patients with advanced CEA-expressing tumors showed disease stabilization for at least four months in 40% of the patients, and for more than six months in 24% (12/22)

Threshold
Pharmaceuticals
Inc.*

Glufosfamide

Small molecule targeting abnormal glucose metabolism

Cancers

Began Phase I trial to evaluate the drug with gemcitabine in advanced solid tumors, to be followed by a Phase II portion of the trial in pancreatic cancer (12/20)

TransMolecular
Inc.*

131I-TM-601

Tumor-targeting peptide derived from scorpion venom conjugated with iodine-131

Recurrent glioma

Based on favorable data from a Phase I/II trial, the company began a Phase II trial in 66 patients (12/7)

Vion
Pharmaceuticals
Inc.
(VION)

Triapine

Inhibitor of ribonucleotide reductase (inhibits a step in DNA synthesis)

Pancreatic cancer

Data from 60 patients in Phase II trial taking drug and gemcitabine showed an objective response rate of 11%, median progression-free survival of 4.7 months and median survival of 7.1 months (12/14)

Xcyte Therapies
Inc.
(XCYT)

Xcellerated T Cells

T cells from patient stimulated to carry out immune functions

Multiple myeloma

31 of 36 patients in a Phase I/II trial had a 50% or greater decrease in the serum M-protein; Phase II trial showed agent led to increases in both T cells and natural killer cells (ASH)

Xcyte Therapies
Inc.
(XCYT)

Xcellerated T Cells

T cells from patient stimulated to carry out immune functions

Chronic lymphocytic leukemia

12 of 17 patients in a Phase I/II trial demonstrated a 50% or more decrease in the size of their enlarged lymph nodes (ASH)

CARDIOVASCULAR

Actelion Ltd.
(Switzerland;
SWX:ATLN)

Clazosentan

Intravenous endothelin receptor antagonist

Vasospasm subsequent to subarachnoid hemorrhage

Began Phase II/III CONSCIOUS-1 trial to assess efficacy in 400 patients worldwide (12/22)

Amylin
Pharmaceuticals
Inc.
(AMLN)

AC2592

Continuous infusion of glucagon-like peptide-1

Congestive heart failure

Began Phase II trial in 180 patients to assess peak oxygen consumption and secondary endpoints (12/5)

Cardiome
Pharma Corp.
(Canada; CRME)

RSD1235

Oral formulation of drug designed to selectively block ion channels in the heart

Atrial arrhythmia

416-patient Phase III ACT-1 trial demonstrated statistical significance vs. placebo in converting patients to normal heart rhythm (12/20)

Cardiome
Pharma Corp.
(Canada; CRME)

Oxypurinol

Xanthine oxidase inhibitor that is the active metabolite of allopurinol

Congestive heart failure

Monitors recommended continuation of Phase II OPT-CHF trial after second of three planned safety analyses (12/2)

Corgentech
Inc.
(CGTK)

E2F Decoy (edifoligide)

Oligonucleotide designed to inactivate the cell-cycle transcription factor E2F

Peripheral artery bypass graft failure

PREVENT III, the first of two Phase III trials, failed to improve vein graft failure over the 12 months following surgery vs. placebo (12/6)

Icagen Inc.*

ICA-17043

Ion channel blocker

Sickle cell anemia

Phase II trial in 90 patients demonstrated a dose dependent and, at one dose, statistically significant increase in hemoglobin levels from baseline (ASH)

Inotek
Pharmaceuticals
Corp.*

INO-1001

Inhibitor of the nuclear cell death enzyme poly (ADP-ribose) polymerase

Cardiac necrosis

Began Phase II trial to test the drug in 40 patients undergoing emergency angioplasty after acute myocardial infarction (12/20)

Inspire
Pharmaceuticals
Inc.
(ISPH)

INS50589 Cardiovascular

Platelet aggregation inhibitor

Cardiovascular indications

Began a Phase I trial to test safety, tolerability and biological activity in at least 28 healthy volunteers (12/16)

Nuvelo Inc. (NUVO)

Alfimeprase

Modified fibrolase delivered via catheter; thrombolytic agent

To restore function in occluded central venous catheters

Phase II trial showed drug has the potential to restore function and that it was well tolerated in that setting (ASH)

Penwest
Pharmaceuticals
Co.
(PPCO)

PW2101

Low-dose formulation of a beta blocker

Hypertension and angina

Pivotal trial involving 110 hyper-tensive patients failed to meet its primary endpoint (12/2)

ProMetic
Life Sciences
Inc.
(Canada; TSE:
PLI)

PBI-1402

Orally active, low- molecular-weight synthetic compound

Anemia

Phase I trial demonstrated an increase in reticulocytes in healthy individuals without any significant adverse effects (12/14)

RegeneRx Biopharmaceuticals
Inc.
(OTC BB:RGRX)

Thymosin beta 4

Naturally occurring peptide that regulates actin, a cell-building protein

Venous stasis ulcers

Intends to begin a Phase II trial, the second of five planned in wound-healing indications (12/20)

The Medicines
Co.
(MDCO)

Angiomax (FDA-approved)

Bivalirudin; direct thrombin inhibitor

Anticoagulant

Phase III EVOLUTION-On trial demonstrated drug is a safe alternative anticoagulant to heparin with protamine reversal in on-pump cardiac surgery (12/28)

The Medicines
Co.
(MDCO)

Clevelox

Clevidipine; intravenous blood pressure control agent

For use in cardiac surgery

Cardiac surgery patients with high blood pressure achieved a 15% reduction in blood pressure 92.5% of the time vs. 17.3% for placebo in pivotal Phase III ESCAPE-1 trial (12/16)

Titan
Pharmaceuticals
Inc.
(AMEX:TTN)

DITPA

Analogue of thyroid hormone (T3)

Congestive heart failure

Began Phase IIb trial to evaluate the drug in 150 patients with advanced CHF and low serum T3 levels (12/14)

CENTRAL NERVOUS SYSTEM

Avigen Inc.
(AVGN)

AV201

AAV vector containing the gene for AADC; designed to restore normal levels of dopamine

Parkinson's disease

Began Phase I/II trial to evaluate safety and preliminary efficacy in patients with mid- to later-stage PD (12/16)

Catalyst
Pharmaceutical
Partners Inc.*

CPP-109

Designed to inhibit an enzyme that normally breaks down gamma amino butyric acid

Cocaine addiction

Filed IND for Phase I trial to test safety of the interaction of CPP- 109 and cocaine (12/8)

DOV
Pharmaceuticals
Inc.
(DOVP)

Bicifadine

Non-narcotic analgesic

Lower back pain

Began fourth of seven planned Phase III trials, a study that will evaluate safety for up to one year in 1,550 patients (12/15)

Durect Corp.
(DRRX) and
Voyager
Pharmaceuticals
Inc.

--

Biodegradable implant leuprolide acetate treatment

Alzheimer's disease

Began trial to evaluate safety and tolerability in healthy volunteers (12/20)

Helicon
Therapeutics
Inc.*

HT-0712

Oral inhibitor of phosphodiesterase-4

Memory indications

Began Phase I trial to test safety and pharmacokinetics in normal healthy volunteers (12/10)

Neurocrine
Biosciences Inc.
(NBIX)

--

Corticotropin releasing factor 1 receptor antagonist

CNS diseases

Partner GlaxoSmithKline plc began a Phase I trial to evaluate safety and pharmacokinetics in healthy volunteers (12/14)

Neurogen
Corp.
(NRGN)

NG2-73

Selective modulator of gamma aminobutyric (GABA) receptors

Insomnia

Began Phase I trial to test safety and pharmacokinetics in healthy volunteers (12/20)

NovaDel
Pharma Inc.
(AMEX:NVD)

--

Lingual spray formulation of zolpidem (Sanofi-Aventis' Ambien)

Sleep induction

Began pilot pharmacokinetic feasibility study in humans (12/7)

Pain
Therapeutics
Inc.
(PTIE)

Remoxy

Abuse-resistant form of long-acting oxycodone

Pain

Trial in 10 volunteers demonstrated drug was significantly less abusable than Oxycontin (12/6); began Phase III trial to assess efficacy in 200 patients with moderate to severe osteoarthritic pain (12/23)

Pharmos
Corp.
(PARS)

Dexanabinol

A tricyclic dextro-cannabinoid; synthetic analogue of marijuana

Severe traumatic brain injury

Phase III trial in 861 patients failed to demonstrate efficacy vs. placebo as measured by the Extended Glasgow Outcome Scale (12/20)

Predix
Pharmaceuticals
Inc.*

PRX-03140

Serotonin 4 (5-HT4) receptor agonist

Memory and cognitive disorders

Began Phase I trial to evaluate safety, tolerability and pharmaco-kinetics in healthy volunteers (12/20)

DIABETES

Amylin
Pharmaceuticals
Inc.
(AMLN)

Pramlintide (AC137)

Analogue of human amylin

Obesity in diabetics

Trial in 204 patients showed the drug was well tolerated and produced weight loss vs. placebo (12/5)

Amylin
Pharmaceuticals
Inc.
(AMLN)

Exenatide

Incretin mimetic

Type II diabetes

Open-label extension of pivotal trial showed sustained reductions in both blood sugar and body weight through 18 months (12/5)

ConjuChem
Inc.
(Canada;
TSE:CJC)

DAC:GLP-1

Insulinotropic hormone GLP-1 created with firm's DAC technology

Type II diabetes

Phase II clinical with Metformin in 85 patients demonstrated significantly reduced blood glucose levels in patients on diabetic drugs, but not in glycemic control (12/15)

MannKind
Corp.
(MNKD)

Technosphere Insulin

Pulmonary insulin formulation delivered via company's inhaler

Diabetes

Phase II trial showed a statistically significant reduction of HbA1c levels vs. placebo (12/22)

Syrrx Inc.*
and PPD Inc.

SYR619

Human dipeptidyl peptidase IV inhibitor

Type II diabetes

They filed an IND to begin clinical testing of the product (12/22)

INFECTION

Advancis
Pharmaceutical
Corp.
(AVNC)

Amoxicillin Pulsys

Amoxicillin formulation delivered with once-daily pulsatile Pulsys technology

Pediatric pharyngitis

Began Phase III trial in 500 children with acute pharyngitis/tonsillitis due to Group A streptococcal infections (12/17)

Aerogen Inc.
(AEGN)

--

Aerozolized formulation of amikacin

Ventilator- associated pneumonia

Began Phase II trial in 108 patients to evaluate dosing, safety, tolerability and preliminary efficacy (12/30)

Dynavax
Technologies
Corp.
(DVAX)

--

Prophylactic vaccine based on immunostimulatory sequences

Hepatitis B

Interim analysis of Phase II/III trial showed statistically significant superiority in protective effects vs. GlaxoSmithKline's Engerix-B in an older adult population (12/9)

MediGene AG
(Germany; FSE:
MDG)

Polyphenon E Ointment

Product from green tea leaves designed to block virus binding to cells

Anogenital and perianal warts

Phase III trial in more than 500 patients demonstrated statistical significance; NDA filing is planned for mid-2005 (12/7)

Panacos
Pharmaceuticals
Inc.*

PA-457

Small-molecule maturation inhibitor designed to block a step in processing of the HIV Gag protein

HIV

Began Phase IIa trial to test the antiviral potency in patients not on other antiretroviral therapy (12/10)

SinusPharma
Inc.*

SPRC-AB01

A formulation of an antibiotic for nasal inhalation

Chronic rhinosinusitis

Began Phase II trial to test safety and efficacy in patients who have had sinus surgery but continue to have bacterial infections (12/15)

Theravance
Inc.
(THRX)

Telavancin (TD-6424)

Lipoglycopeptide antibiotic believed to have multiple mechanisms of action

Gram-positive skin and skin-structure infections

Phase II FAST 2 trial showed clinical cure rates of 96.1% vs. 93.5% for patients treated with standard therapy (12/7)

Vertex
Pharmaceuticals
Inc.
(VRTX)

Lexiva (FDA-approved)

Fosamprenavir calcium; protease inhibitor

HIV

Study APV30005 combining drug with GlaxoSmithKline plc's ritonavir demonstrated sustained viral suppression and safety over 120 weeks (12/15)

MISCELLANEOUS

Arena
Pharmaceuticals
Inc.
(ARNA)

APD356

Small-molecule, selective 5HT2C serotonin receptor agonist

Obesity

Began Phase II trial to evaluate safety and preliminary efficacy in 400 obese volunteers (12/22)

Barrier
Therapeutics
Inc.
(BTRX)

Sebazole

Topical formulation of 2% ketoconazole, an antifungal agent

Seborrheic dermatitis

Confirmatory Phase III pivotal trial in 459 patients showed success in 25.8% of drug patients vs. 13.9% in vehicle-only arm (12/16)

BioMarin
Pharmaceutical
Inc.
(BMRN)

Phenoptin

Sapropterin hydrochloride; form of the enzyme co-factor 6R-BH4

Phenylketonuria

Began Phase II trial that will screen up to 400 patients for a response; responders will be eligible for a Phase III trial to follow (12/23)

ConjuChem
Inc.
(Canada;
TSE:CJC)

DAC:GRF

Chemically modified form of growth hormone-releasing factor

Growth hormone deficiency

Phase I trial demonstrated safety and tolerability, and a response on GH and IGF-1 levels (12/9)

Discovery
Laboratories Inc.
(DSCO)

Surfaxin

Engineered version of natural human lung surfactant

Respiratory distress syndrome in premature babies

Additional data from Phase III SELECT trial showed increased survival vs. comparator therapies; reduced chronic lung disease also was seen (12/13)

Discovery
Laboratories
Inc.
(DSCO)

Surfaxin

Engineered version of natural human lung surfactant

Acute respiratory distress syndrome

Preliminary data from 78 adults in a Phase II trial showed a 31% relative improvement in overall mortality at Day 28 vs. standard of care (12/7)

DOR BioPharma
Inc.
(AMEX:DOR)

orBec

Oral formulation of beclomethasone dipropionate; a corticosteroid

Graft-vs.-host disease

Phase III trial in 129 patients did not achieve statistical significance in primary endpoint of time to treatment failure, but did show a benefit on mortality (12/30)

Dynavax
Technologies
Corp.
(DVAX)

AIC

Anti-allergy immunotherapy

Ragweed allergy

One-year analysis of two-year Phase II/III trial showed positive trends on nasal symptom scores, as well as secondary endpoints (12/20)

Eyetech
Pharmaceuticals
Inc.
(EYET)

Macugen (FDA-approved)

Pegaptanib sodium injection; pegylated anti-VEGF aptamer

Wet age-related macular degeneration

Published data from VISION study showed a statistically significant reduction of vision loss (12/29)

FibroGen
Inc.*

FG-2216

Small-molecule inhibitor of hypoxia-inducible factor-prolyl hydroxylase

Anemia in chronic kidney disease

Phase IIa trial showed the drug produced significant increases in levels of circulating hemoglobin (ASH)

Inspire
Pharmaceuticals
Inc.
(ISPH)

INS37217 Respiratory

P2Y2 agonist designed to enhance mucosal hydration and clearance

Cystic fibrosis

Began a second Phase II trial to test safety and tolerability in a broader population of CF patients (12/21)

MacroChem
Corp.
(MCHM)

Opterone

Testosterone in cream form delivered with SEPA technology

Hypogonadism

Began bioavailability study to evaluate absorption of different doses (12/8)

Medical
Discoveries Inc.
(OTC BB:MLSC)

MDI-P

Electrolyzed solution of free radicals

Cystic fibrosis

Filed IND for a Phase I trial to test the product as an adjunct to tobramycin in late-term CF in adults (12/1)

PTC
Therapeutics
Inc.*

PTC124

Oral agent that targets nonsense mutations

Cystic fibrosis and Duchenne muscular dystrophy

Began Phase I trial to evaluate escalating doses in healthy volunteers (12/23)

Symbollon
Pharmaceuticals
Inc.
(OTC BB:SYMBA)

IoGen

Iodine-based oral drug

Breast pain associated with symptomatic fibrocystic breast disease

Began pivotal Phase III trial to evaluate clinical effects in 175 euthyroid, premenopausal women who are unresponsive to conservative treatment (12/22)

Topigen
Pharmaceuticals
Inc.*
(Canada)

ASM8

Inhalation drug designed to attack multiple mediators of the inflammatory mechanism

Asthma

Plans to begin a Phase I trial to evaluate safety and tolerability in volunteers (12/8)

Vivus Inc.
(VVUS)

Evamist

A transdermal formulaiton of estradiol

Vasomotor symptoms of menopause

Reached agreement with FDA on pivotal Phase III trial (12/16); began the trial in up to 500 patients (12/21)


Notes:

* Privately held

ASH = Data were presented at the American Society of Hematology meeting.

BLA = Biologics license application; FDA = Food and Drug Administration; IND = Investigational new drug application;

NDA = New drug application; SPA = Special protocol assessment.

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

AMEX = American Stock Exchange; ASX = Australian Stock Exchange; CSE = Copenhagen Stock Exchange; FSE = Frankfurt Stock Exchange; NYSE = New York Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; SWX = Swiss Stock Exchange; TSE = Toronto Stock Exchange.

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