Company* |
Product | Description |
Indication | Status |
| | ||||
AUTOIMMUNE | ||||
Antisense |
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. |
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 |
HuMax-Inflam/ |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
Abraxane |
Nanoparticle albumin-bound taxane |
Metastatic breast cancer |
Phase II trial produced long-term disease control in taxane-refractory patients (12/13) |
AnorMED Inc. |
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. |
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. |
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. |
ARQ 501 |
Activated Checkpoint Therapy molecule designed to selectively kill cancer cells |
Advanced cancers |
Began Phase Ib trial with Taxotere (12/7) |
AVAX |
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 |
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 |
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. |
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 |
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 |
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 |
Revlimid (lenalidomide) |
Derivative of Thalomid (thalidomide) |
Myelodysplatic syndromes |
Reported preliminary positive results from ongoing Phase II trial in patients with 5q deletion (ASH) |
Celgene |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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. |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
Velcade |
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 |
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 |
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. |
Skeletal Targeted |
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 |
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 |
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 |
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 |
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. |
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 |
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 |
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. |
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. |
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. |
Telintra |
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 |
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 |
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 |
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 |
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 |
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 |
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. |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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. |
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 |
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 |
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. |
-- |
Biodegradable implant leuprolide acetate treatment |
Alzheimer's disease |
Began trial to evaluate safety and tolerability in healthy volunteers (12/20) |
Helicon |
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 |
-- |
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 |
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 |
-- |
Lingual spray formulation of zolpidem (Sanofi-Aventis' Ambien) |
Sleep induction |
Began pilot pharmacokinetic feasibility study in humans (12/7) |
Pain |
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 |
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 |
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 |
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 |
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 |
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 |
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.* |
SYR619 |
Human dipeptidyl peptidase IV inhibitor |
Type II diabetes |
They filed an IND to begin clinical testing of the product (12/22) |
INFECTION | ||||
Advancis |
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. |
-- |
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 |
-- |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
Opterone |
Testosterone in cream form delivered with SEPA technology |
Hypogonadism |
Began bioavailability study to evaluate absorption of different doses (12/8) |
Medical |
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 |
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 |
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 |
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. |
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) |
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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. | ||||
