Clinical Trials Update: December 2004
Company* | Product | Description | Indication | Status |
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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. | ||||