Company* | Product | Description | Indication | Status (Date) |
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AUTOIMMUNE | ||||
Acorda | Fampridine-SR | Selective neuronal potassium channel blocker | Multiple sclerosis | Phase II study showed positive trends but did not reach statistical significance in the primary endpoint, improvement in walking speed (4/14) |
Biogen Idec | BG-12 | An oral fumarate | Psoriasis | Phase II results of 144 patients with severe psoriasis showed 42% on highest dose achieved PASI improvement of 75% at 12 weeks vs. 11% on placebo (4/29) |
Chiron Corp. | Betaseron (FDA-approved) | Interferon beta-1b | Multiple sclerosis | Initial data from BEYOND study showed that increasing the dose significantly improved responses Laboratories Inc. in patients with persisting disease on the standard dose (4/28) |
Repligen Corp. | CTLA4-Ig | Soluble form of T-cell regulatory protein | Multiple sclerosis | Initial data from Phase I study support the safety and novel mechanism of action of drug in MS (4/29) |
Serono SA | Rebif | Interferon beta-1a | Multiple sclerosis | Long-term data support use of high-dose, high-frequency treatment in reducing long-term accumulation of brain lesion volume in relapsing-remitting patients (4/28) |
The Immune | NeuroVax | T-cell receptor peptide vaccine; combines three TCR peptides with an adjuvant | Multiple sclerosis | Phase I/II trial in 37 patients produced a peptide-specific immune response in 94% of those treated (4/29) |
CANCER | ||||
AnorMED Inc. | AMD3100 | Drug designed to block receptor-triggering movement of stem cells out of bone marrow | Stem cell transplantation in cancer patients | Began Phase II trial to optimize the schedule for administering drug in combination with G-CSF (4/1) |
Antisoma plc | R1549 | Mouse antibody linked to yttrium-90 that binds to MUC1 protein | Ovarian cancer | Phase III SMART trial begun in 1998 failed to beat the comparator arm of standard care alone; trial included 420 patients in 17 countries (4/26) |
AVI BioPharma | AVI-4126 | Neugene antisense drug targeting the oncogene c-myc | Cancer | Phase I data showed the drug concentrated in breast and prostate tumor tissues after a single dose (4/1) |
Biomira Inc. | BLP25 | Therapeutic vaccine encapsulating a sequence of the MUC1 cancer mucin in a liposomal delivery system | Stage IIIb and IV non-small-cell lung cancer | Phase IIb trial showed increased survival of 4.4 months vs. control group, a result that was not statistically significant; companies are considering a plan for pivotal program (4/1) |
Biomira Inc. | BLP25 | Therapeutic vaccine encapsulating a sequence of the MUC1 cancer mucin in a liposomal delivery system | Prostate cancer | 16-patient pilot Phase II trial did not demonstrate a reduction or stabilization of serum PSA levels (4/1) |
Celgene Corp. | Revimid | Immunomodulatory drug | Melanoma | Pivotal Phase III trial was stopped following analysis that showed endpoint would not be met; other trials of the drug were not affected (4/28) |
Cell Therapeutics | CT-2106 | Polyglutamate-20S- camptothecin conjugate | Colorectal cancer | Began Phase I trial in combination with 5-FU in refractory patients to examine maximum dose, dose-limiting toxicities, safety and anti-tumor activity (4/20) |
Cell Therapeutics | Trisenox (FDA-approved) | Arsenic trioxide | Acute promyelocytic leukemia | Published data showed drug and ATRA induction therapy resulted in better results than either agent alone; results were from 61 newly diagnosed patients (4/7) |
Celmed | Theralux | Ex vivo photodynamic therapy designed to destroy cancer cells in bone marrow or blood | Non-Hodgkin's lymphoma | Ten of 13 patients in open-label trial received grafts without detectable levels of cancer cells after Theralux therapy (4/29) |
CuraGen Corp. | CG53135 | Growth factor believed to promote epithelial and mesenchymal cell proliferation | Oral mucositis in cancer patients | Began Phase I trial to evaluate the safety and potential response in about 15 patients undergoing bone marrow transplantation (4/21) |
EntreMed Inc. | Panzem | 2-methoxyestradiol; naturally occurring metabolite of endogenous estrogen | Cancers | Phase I trial of new formulations of drug showed higher peak blood levels than original formulation (4/27) |
FeRx Inc.* | MTC-DOX | Doxorubicin combined with MagneTarg delivery system | Primary liver cancer | Enrollment in Phase II/III MAGNET trial was stopped after determination that survival endpoint against intravenous doxorubicin would not be met (4/30) |
Genmab A/S | HuMax-CD4 | Human antibody that targets the CD4 receptor on T lymphocytes | Cutaneous T-cell lymphoma | Phase II trial showed benefit of higher doses and helped set up planned pivotal trial (4/28) |
Introgen | Advexin | Adenoviral vector containing the p53 tumor-suppressor gene | Stage III or IV squamous cell carcinoma of the oral cavity | Southwest Oncology Group began a Phase II study to assess the feasibility, efficacy and safety of Advexin given before chemotherapy and radiation (4/21) |
Introgen | INGN 241 | Agent containing the tumor-suppressor gene Mda-7/IL-24 | Metastatic melanoma | Began Phase II study to assess activity and other endpoints in up to 25 patients with at least three regional lesions (4/7) |
Marshall | Phenoxodiol | Agent designed to induce apoptosis by removing intracellular proteins (XIAP, c-FLIP) | Ovarian cancer | Began trial to assess the agent's ability to restore the sensitivity of ovarian cancer to standard chemotherapies (4/21) |
OSI | Tarceva | Erlotinib HCl; small- molecule HER1/EGFR inhibitor | Non-small-cell lung cancer | Phase III trial in refractory patients met its primary endpoint of improving overall survival, and met secondary endpoints; NDA filing planned for summer 2004 (4/26) |
Oxford | TroVax | Vaccine that delivers an antigen (5T4) using a pox virus vector | Metastatic renal cancer | Got FDA approval for combination trial with interleukin-2 to test safety and immunogenicity, as well as clinical benefit (4/29) |
Regeneron | VEGF Trap | Intravenous product designed to block vascular endothelial growth factor | Cancer | Regeneron and Aventis SA began Phase I trial to assess safety and tolerability (4/14) |
Spectrum | Elsamitrucin | Antibiotic designed to inhibit topoisomerase I and II | Non-Hodgkin's lymphoma | Began Phase II study to evaluate drug as single agent in about 80 refractory patients (4/8) |
Transgene SA | Ad-IL2 | Adeno-interluekin-2; immunotherapy | Melanoma and other solid tumors | Presented Phase I/II data showing treatment was tolerated and produced responses in some patients (4/14) |
Xcyte Therapies | Xcellerated T Cells | T cells from patient stimulated to carry out immune functions | Multiple myeloma | Began Phase II trial in 30 patients with relapsed disease to assess safety, as well as the biologic and therapeutic activity (4/15) |
ZymoGenetics | -- | Recombinant human interleukin-21 | Cancers | Got FDA OK for Phase I trial testing safety and pharmacokinetics (4/5) |
CARDIOVASCULAR | ||||
Acorda | Fampridine-SR | Selective neuronal potassium channel blocker | Spinal cord injury | Neither of two Phase III trials demonstrated statistical significance in the primary endpoint, although one showed a positive trend in reducing muscle spasticity (4/14) |
Alexion | Eculizumab | Humanized monoclonal antibody designed to block complement protein C5 | Paroxysmal nocturnal hemoglobinuria | One-year data from 11 patients in Phase Ib trial showed statistically significant reductions in the need for blood transfusions and in hemolysis (4/20) |
Alteon Inc. | Alagebrium (ALT-711) | Alagebrium chloride; A.G.E. crosslink breaker | Heart failure | Began Phase II PEDESTAL trial in 20 patients to evaluate diastolic function and ventricular mass (4/20) |
CV Therapeutics | Regadenoson (CVT-3146) | Selective A2A-adenosine receptor agonist | Cardiac perfusion imaging | Began second international Phase III trial of the pharmacologic stress agent (4/26) |
CV Therapeutics | Ranexa (ranolazine) | Partial inhibitor of fatty- acid oxidation | Chronic angina | Published Phase III MARISA data showed a statistically significant increase in symptom-limited exercise duration, the primary endpoint (4/20) |
Endovasc Inc. | Liprostin | Lisposomal formulation of prostaglandin E1 | Critical limb ischemia | The first patients from a Phase II trial were showing significant improvement in transcutaneous oxygen pressure levels (4/13) |
Jerini AG* | Icatiban (JE049) | Bradykinin-2 receptor antagonist | Hereditary angioedema | Company will move forward with pivotal studies with subcutaneous formulation after positive bioavailability studies (4/14) |
Medicure Inc. | MC-1 | Small-molecule cardioprotective agent | Hypertension | Phase II trial showed positive trend and supported move into expansion of program for MC- 4232, which combines MC-1 with an ACE inhibitor (4/7) |
Neurochem Inc. (Canada; NRMX) | Cerebril | Small molecule designed to prevent formation of amyloid fibrils | Hemorrhagic stroke | Phase II trial identified safe and well-tolerated doses for Phase IIb study (4/27) |
Theratechnologies | ThGFR | Analogue of growth hormone-releasing factor | HIV-associated lipodystrophy | Phase II trial showed a good safety profile, a positive effect on body composition and a clinically relevant reduction in visceral fat (4/15) |
ZymoGenetics | -- | Recombinant human thrombin | Control of bleeding associated with surgery | Began Phase II trial in patients undergoing liver resection, the first of four planned indications for Phase II trials (4/19) |
CENTRAL NERVOUS SYSTEM | ||||
Boston Life | Altropane | Radioimaging agent | Parkinson's disease diagnosis | Reached agreement with FDA on pivotal trial in at least 500 patients to distinguish Parkinsonian from non-Parkinsonian tremors (4/1) |
Cephalon Inc. | Gabitril | Tiagabine hydrochloride; selective GABA reuptake inhibitor | Anxiety | Moving forward with Phase III program after 260-patient Phase II trial produced statistically significant results; trial expected to start in 2H:04 (4/13) |
Ceregene Inc.* | -- | Adeno-associated viral gene delivery system carrying the nerve growth factor gene | Alzheimer's disease | Phase I results on six patients showed a potential reduction in disease advancement and an increase in brain metabolic activity (4/29) |
Metaphore | M40403 | Small molecule that mimics the activity of superoxide dismutase | Pain | Phase II trial in 350 patients showed the addition of M40403 to morphine improved the analgesic profile of morphine (4/27) |
Myriad Genetics | Flurizan | Acts on NFkB, a transcriptional activator | Alzheimer's disease | Safety monitors reported no serious concerns after five months and recommended the Phase II trial continue (4/20) |
Neurobiological | Xerecept | Synthetic mimic of the corticotropin-releasing factor hormone | Peritumoral cerebral edema | Began first of two pivotal trials, in about 200 patients who require chronic treatment with steroids; the primary endpoint is a 50% reduction of steroid use at three weeks (4/20) |
Neurochem Inc. | Alzhemed | Small molecule designed to interfere with association between glycosaminoglycans and A-beta protein | Alzheimer's disease | Additional interim Phase II data in 23 patients with mild AD NRMX) showed 13 had stabilized or improved cognitive function after 16 months of treatment (4/14) |
Neurocrine | Indiplon | Non-benzodiazapine agent that acts on a specific site of the GABA-A receptor | Insomnia | Reported additional positive Phase III results of immediate- and modified-release formualtions in elderly patients (4/7) |
Phytopharm plc | PYM50018 | Product based on Asian plant extract believed to have neuroprotective effects | Amyotrophic lateral sclerosis | Phase I trial was positive in terms of safety, tolerability and bio-availability (4/28) |
Prana | PBT-1 (clioquinol) | Small molecule that binds metal ions; designed to remove zinc bound to amyloid-beta | Alzheimer's disease | Extended Phase II data in nine patients showed a slowing of the decline in cognitive function vs. historical controls (4/16) |
Saegis | SGS742 | Oral, selective GABA-B receptor antagonist | Mild cognitive impairment | Phase II trial in 110 patients demonstrated no improvement in cognitive performance but did reach other endpoints (4/28) |
DIABETES | ||||
Aradigm Corp. | AERx iDMS | Pulmonary insulin delivery system | Diabetes | Twelve-month analysis of first Phase III trial showed delayed post-meal plasma glucose suppression in Type I diabetics, leading partner Novo Nordisk A/S to end the trial early (4/30) |
ConjuChem Inc. | DAC:GLP-1 | Insulinotropic hormone GLP-1 created with firm's DAC technology | Type II diabetes | Interim data from 80 patients in Phase II monotherapy trial produced statistically significant reduction of A1c levels, as well as similar benefit on other endpoints (4/12) |
INFECTION | ||||
Acambis plc | ACAM2000 | Second-generation vaccine | Smallpox | Enrollment was stopped in Phase III trial comparing vaccine to Dryvax after cases of myopericarditis were seen in both arms |
Advanced | -- | Inhibitor of tumor necrosis factor-alpha | HIV | Began a Phase I trial in 10 patients who are in virologic failure to test safety and possible clinical effect (4/19) |
Adventrx | BlockAide/CR | Peptide-based drug designed to block viral entry | HIV | Began Phase Ib/IIa trial to assess safety, pharmacokinetics and effect on viral load in patients with increasing viral load (4/15) |
Avant Immuno- | CholeraGarde | Live, attenuated cholera vaccine (Peru 15) | Cholera | The vaccine was well tolerated in a Phase II trial in 70 adult volunteers, and no safety concerns were seen in toddler portion of the trial (4/28) |
CollaGenex Pharmaceuticals Inc. (CGPI) | Metastat (Col-3) | Orally active angiogenesis inhibitor | HIV-related Kaposi's sarcoma | Interim Phase II data showed a response rate of 38% in the low-dose group and 26% in the high-dose group (4/30) |
Cubist Pharmaceuticals Inc. (CBST) | Ceftriaxone | Various oral forms of a broad-spectrum antibiotic known collectively as OCTX | Infections | Human studies did not meet the bioavailability thresholds, and development of the product was ended (4/29) |
Cubist Pharmaceuticals Inc. (CBST) | Cubicin (formerly Cidecin) | Daptomycin for injection | Endocarditis and bacteremia caused by S. aureus | Monitors performed second safety review and recommended continuation of Phase III trial; data are expected in 1H:05 (4/1) |
Gilead Sciences | Hepsera (FDA-approved) | Adefovir dipivoxil | Hepatitis B | 144-week data from Study 438 showed progressive reductions in serum HBV DNA replication and sustained ALT normalization (4/15) |
Idenix | Telbivudine | Oral nucleoside agent | Hepatitis B | One-year data from Phase IIb trial showed reduced HBV replication and higher rates of ALT normalization compared with lamivudine monotherapy (4/16) |
Peninsula Pharmaceuticals Inc.* | Doripenem (S-4661) | Agent from the carbapenem class of beta-lactam antibiotics | Urinary tract infections | Began second pivotal trial in complicated UTIs; the primary endpoint is microbiological response (4/15) |
Protein | FluBIOk | Influenza vaccine derived from recombinant hemagglutinin | Influenza | Phase IIb trial met primary endpoints; vaccine was safe and more immunogenic against H3 strain than current vaccine (4/28) |
Valeant Pharmaceuticals Inc. (NYSE:VRX) | Viramidine | Oral nucleoside analogue | Hepatitis C | Phase II trial comparing drug to ribavirin, both in combination with pegylated interferon, demonstrated similar efficacy with a lower incidence of anemia in the Viramidine group (4/19) |
VaxGen Inc. | rPA102 | Recombinant protective antigen anthrax vaccine | Anthrax infection | Began Phase II trial at 12 centers in the U.S.; the work is being done under an NIH contract (4/15) |
Vertex | Merimepodib (VX-497) | Small-molecule inhibitor of the enzyme inosine mono- phosphate dehydrogenase | Hepatitis C | Data from 11 patients in extension of Phase IIa trial in combination with pegylated interferon alpha and ribavirin support advancing the compound (4/16) |
Vicuron Pharmaceuticals Inc. (MICU) | Anidulafungin | Broad-spectrum agent from the echinocandin class | Infections | Variety of data presented at European meeting were encouraging for the late-stage product (4/28) |
MISCELLANEOUS | ||||
Advanced | Ferumoxytol | Intravenous iron replacement therapeutic | Anemic chronic kidney disease | Began two Phase III studies in patients not yet on dialysis (4/27); Phase II trial showed drug effectively supplied iron in anemia patients on hemodialysis (4/30) |
AEterna | Cetrorelix | Luteinizing hormone- | Endometriosis, uterine myomas and benign prostatic hyperplasia | Phase II results from six Phase II trials in the three indications demonstrated statistical significance (4/29) |
AEterna | EP-1572 | Orally available peptidomimetic; growth hormone secretagogue | Growth hormone and other disorders | Began dose-ranging study to evaluate the safety and pharmacokinetics (4/21) |
Amgen Inc. | Sensipar (FDA-approved) | Cinacalcet HCl; oral calcium calcimimetic | Secondary hyperpara- | Published data from 741 patients showed reduced parathyroid hormone levels in patients on dialysis with secondary HPT and lowered elevated calcium and phosphorus levels (4/8) |
ARYx | ATI-7505 | Analogue of cisapride, a product removed from the market in 2000 due to safety problems | Gastro- | Began Phase I trial of the selective 5HT4 receptor agonist (4/29) |
CollaGenex Pharmaceuticals Inc. (CGPI) | Periostat (FDA-approved) | Doxycyline hycalte | Adult periodontitis | Began Phase III trial to evaluate once-daily Periostat MR formulation (4/5) |
Corixa Corp. | CRX-675 | Toll-like receptor 4 agonist | Seasonal allergic rhinitis | Began Phase I trial to test safety and immune response in 64 adults allergic to ragweed pollen (4/26) |
Genaera Corp. | Squalamine | Anti-angiogenesis agent; VEGF inhibitor | Wet age- related macular degeneration | Began pharmacokinetic and safety study of three doses in 18 patients; said Phase III trials planned for early 2005 (4/22) |
GenVec Inc. | AdPEDF | Product that uses an adenovector to produce pigment-epithelium- derived factor | Wet age-related macular degeneration | Phase I trial in patients with severe disease showed the product was well tolerated at all doses (4/28) |
InKine | Visicol (FDA-approved) | Tablet form of sodium phosphate | Chronic constipation | Phase IV study in 40 patients showed nearly 100% effectiveness in increasing bowel movements; controlled trials are planned (4/19) |
Inspire | INS37217 Respiratory | P2Y2 agonist designed to enhance mucosal hydration and clearance | Cystic fibrosis | Phase II trial in 90 patients showed significantly better lung function at four weeks in the drug group (4/27) |
Inspire | INS37217 Ophthalmic | P2Y2 receptor agonist delivered by intravitreal injection | Rhegmatogenous retinal detachment | Began a Phase II trial vs. placebo in about 160 patients (4/21) |
MacroChem | Topiglan | Topical cream combining alprostadil with Macro- Chem's SEPA absorption enhancer | Erectile dysfunction | A Phase II study failed to meet primary clinical endpoint of penile rigidity (4/14) |
Nabi Bio- | PhosLo | Calcium acetate | Control of blood phosphorus levels in kidney patients on dialysis | Published data from CARE study comparing PhosLo to Genzyme Corp.'s Renagel showed PhosLo should be first-line treatment, Nabi said (4/19) |
Neurochem | Fibrillex | Glycosaminoglycan mimetic with anti- amyloid properties | Amyloid A amyloidosis | Monitoring board issued fourth recommendation to continue the Phase II/III trial, which includes 183 patients worldwide (4/26) |
NPS | Teduglutide | Analogue of glucagon- like peptide 2 | Short-bowel syndrome | Began pivotal Phase III trial in 80 patients; primary endpoint is the reduction in the use of intravenous feeding (4/6) |
Rigel | R112 | Intranasal agent designed to interrupt the signal from the IgE receptor | Allergic rhinitis | Began Phase II “Park“ study in 300 patients in two cities who have experienced the condition during the spring pollen season (4/20) |
Savient Pharmaceuticals Inc. (SVNT) | Puricase | Polyethylene glycol conjugate of uricase | Gout | Began Phase II study to assess the effect of a range of doses and regimens on uric acid levels, and safety and tolerability (4/7) |
Transkaryotic Therapies Inc. (TKTX) | GA-GCB | Human glucocerebrosidase; enzyme replacement therapy | Gaucher's disease | Began trial in 12 patients with Type I disease who will be treated for nine months (4/26) |
Vivus Inc. | Avanafil (TA-1790) | Oral phosphodiesterase type-5 inhibitor | Erectile dysfunction | Began crossover-design Phase II trial in 250 patients to evaluate safety and efficacy (4/22) |
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Notes: | ||||
* Privately held | ||||
Unless otherwise noted, the stock symbols listed for public companies are on the Nasdaq market. | ||||
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