Clinical Trials Update: October 2003
Company* | Product | Description | Indication | Status |
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AUTOIMMUNE | ||||
Amgen | Enbrel (FDA-approved) | Etanercept; fully | Rheumatoid arthritis; psoriatic arthritis and ankylosing spondylitis | Amgen presended data at ACR from the TEMPO trial showing 37% of RA patients taking Enbrel and methotrexate achieved clinical remission at one year, and 80% of patients had no progression of joint damage (10/24); other studies showed Psoriatic patients improved after one year, and that 57% of ankylosing spondylitis patients reported a 20% improvement at six months (10/24) |
Aspreva Pharmaceuticals Corp.* | CellCept | Mycophenolate | Lupus nephritis | Lupus Foundation presented 24-week data at ACR from 140 patients showing it had equivalent efficacy to cyclophosphamide with potentially fewer side effects (10/27) |
AtheroGenics Inc. (AGIX) | AGIX-4207 | Modulator of TNF-alpha-induced inflammatory genes | Rheumatoid | Company began a Phase II trial named OSCAR in which 220 patients will be given one of three doses or placebo (10/21) |
Centocor Inc. | Remicade | Infliximab; antibody | Ankylosing | Results of the ASSERT trial showed Remicade reduced signs and symptoms of the disease (10/25); another study showed the drug increased quality of life and decreased disease burden (10/28) |
Biogen Inc. (BGEN) | Amevive (FDA-approved) | Alefacept; targets the CD45RO+ subset of T cells | Rheumatoid | Study combining Amevive and methotrexate in 36 patients showed disease improvement (10/26) |
Genentech Inc. (NYSE:DNA) and IDEC Pharmaceuticals | Rituxan | Rituximab; antibody | Rheumatoid | Follow-up results from the Phase II trial presented at ACR showed a short course of treatment improved symptoms at 48 weeks when compared to methotrexate alone (10/26) |
Human Genome Sciences Inc. (HGSI) | LymphoStat-B | Human antibody to B-lymphocyte | Systemic lupus erythematosus | Company said clinical and pre- clinical results to date support continued development (10/28) |
Peptimmune Inc.* | PI-0824 | Synthetic peptide | Pemphigus | Company began a Phase I trial to test safety (10/7) |
NPS Pharmaceuticals | ALX-0600 | Teduglutide; derivative of protein that regulates gastrointestinal processes | Crohn's | NPS began a proof-of concept study examining dosing and efficacy in 100 patients (10/7) |
Regeneron Pharmaceuticals | IL-1 Trap | Designed to block interleukin-1 | Rheumatoid | Phase II trial in 200 patients missed primary endpoint of ACR-20 responses, though company said it demonstrated some evidence of efficacy (10/7) |
CANCER | ||||
Antigenics Inc. (AGEN) | Oncophage (HSPPC-96) | Personalized cancer vaccine | Cancer | Immunological Phase II data published in The Journal Of Immunology showed a cancer-specific response (10/1) |
Aton | SAHA | Histone | Multiple | Enrollment began in Phase I trial that will assess safety and dosing, as well as anticancer effects and activity (10/13) |
Bioenvision Inc. (AMEX:BIV) | Clofarabine | Purine nucleoside antimetabolite | Leukemia | Company said Phase I data published in Blood showed a 32% response rate in pediatric patients with advanced disease (10/29) |
Bioenvision Inc. (AMEX:BIV) | Gossypol | Isoform of the cancer agent | Cancer | Company said it will begin Phase I testing to determine dosin (10/8) |
Celgene Corp. (CELG) | CC-8490 | Cancer compound | Brain cancer | Celgene and the National Cancer Institute began clinical testing of the drug, which is being developed under a CRADA (10/31) |
Celgene Corp. (CELG) | Actimid (CC-4047) | Immunomodulatory | Prostate cancer | Initiated open-label Phase II trial in 19 patients to study safety and efficacy as a single agent for hormone-refractory disease (10/30) |
Cell Therapeutics Inc. (CTIC) | Xyotax | Paclitaxel linked to biodegradable polyglutamate | Non-small-cell | Company is reducing the dose in its pivotal STELLAR 4 trial follow-recommendation from a data monitoring committee due to toxicities (10/20) |
Cerus Corp. (CERS) | ACIT | Allogeneic cellular immune therapy | Use in stem cell tranplants | Product to improve transplant outcomes in patients with hema- tologic malignancies will be tested in Phase Ib trial involving three doses and up to 42 patients (10/23) |
Endo Pharmaceuticals | EN3247 | 0.1% triclosan | Oral mucositis resulting from chemotherapy | The Phase III trial in 355 patients failed to demonstrate efficacy (10/24) |
GlycoGenesys | GCS-100 | Cancer compound | Advanced solid | Phase I results in 12 patients showed it was well tolerated with no dose-limiting toxicity; five patients achieved stable disease (10/22) |
Hybridon Inc. (OTC BB: HYBN) | GEN231 | Antisense | Cancer | Data from 14-patient safety study were published in Clinical Cancer Research; a dose was selected for Phase II study (10/2) |
Genentech Inc. (NYSE:DNA) and OSI Pharmaceuticals Inc. (OSIP) | Tarceva | Erlotinib; small-molecule EGFR inhibitor | Metastatic non-small-cell | Data from two Phase III studies of Tarceva and standard chemotherapy showed drug did not meet primary endpoint of improving overall survival (10/1) |
ILEX Oncology | ILX-651 | Synthetic analogue of dolastatin; targets tubulin | Recurrent or | ILEX began a Phase II study to measure efficacy and tolerability at 19 sites in the U.S. (10/7) |
Millennium Pharmaceuticals | MLN2704 | Targeted antibody delivering maytansinoid chemotherapeutic agent DM1 | Metastatic androgen- | Millennium began an escalating-dose Phase I/II study in about 46 patients (10/9) |
Novuspharma SpA (Italy; Nuovo Mercato:NOV) | Pixantrone (BBR 2778) | Anthracycline agent | Aggressive non-Hodgkin's lymphom | Company began a Phase II trial, expected to include 75 patients, to test Pixantrone as part of a chemotherapy salvage regimen (10/22) |
Onyx Pharmaceuticals | BAY 43-9006 | Signal transduction inhibitor designed to block the Ras/MEK/ERK pathway | Advanced | Companies initiated international Phase III trial in more than 800 patients who failed prior therapy; they also signed a special protocol agreement with the FDA (10/25) |
OSI | OSI-7904L | Liposomal thymidylate synthase inhibitor | Advanced gastric cancer | OSI began a Phase II study in 50 reviously untreated patients; the primary endpoint is response rate (10/24) |
Progen Industries Ltd. (Australia; PGLAF) | PI-88 | Anti-angiogenesis | Advanced cancers | Progen presented early data from Phase I/II trial highlighting some encouraging results (10/8**) |
Telik | Telcyta (TLK286) | Small-molecule | Refractory or resistant ovarian cancer | Telik reported positive interim results from Phase I/IIa trial of Telcyta in combination therapy (10/21) |
Telik | Telcyta (TLK286) | Small-molecule | Non-small-cell | Telik reached agreement with FDA on special protocol assessment for Phase III trial of Telcyta; the ASSIST-2 study will include about 500 third-line patients (10/1) |
CARDIOVASCULAR | ||||
AryX Therapeutics Inc.* | ATI-2042 | Amiodarone | Atrial fibrillation | Company initiated first clinical study of drug, a Phase I trial to evaluate safety and pharmaco-kinetic profile (10/22) |
Avant Immunotherapeutics Inc. (AVAN) | CETi-1 | Vaccine for | Cholesterol maintenance | Results from 203-patient Phase II trial failed to demonstrate a statistically significant improvement over placebo in increasing HDL levels (10/22) |
Corentech Inc.* | CGT003 | EF2 decoy; oligonucleotide that inactivates E2F transcription factor | Graft failure following | Company completed enrollment in second Phase III trial, this one in 1,400-patient PREVENT study (10/2) |
CV Therapeutics Inc. (CVTX) | CVT-3146 | Selective A2A-adenosine | Stress agent in cardiac perfusion imaging studies | CV initiated a pivotal Phase III trial of drug, triggering $3M milestone from partner Fujisawa Healthcare Inc. (10/28) |
Medicure Inc. (Canada; TSE:MPH) | MC-1 | Cardioprotective | For use in coronary artery bypass graft surgery | Company filed for approval in Canada and the U.S. to begin Phase II trial in up to 900 patients to assess effects in patients undergoing high-risk CABG (10/15) |
United | Remodulin (FDA-approved) | Treprostinil sodium injection | Pulmonary arterial hypertension | Sixteen-month data on 19 patients showed the drug was effective on a long-term basis (10/2) |
XOMA Ltd. (XOMA) | Neuprex | Modified form of fragment of bactericidal/ | Pediatric patients with abnormalties requiring open-heart surgery associated with cardio-pulmonary bypass | XOMA said Children's Medical Center of Dallas started an open-label Phase I/II study (10/2) |
XOMA Ltd. (XOMA) and Millennium Pharmaceuticals | MLN2201 | Humanized | Conditions related to inflammation of the heart and blood vessels | XOMA said it was discontinuing development following a Phase I trial that failed to meet predetermined criteria (10/10) |
CENTRAL NERVOUS SYSTEM | ||||
Cortex Pharmaceuticals | Ampakine CX516 | Compound designed | Excessive daytime sleepiness | Preiminary results of Phase I trial in 10 healthy men showed positive dose-related responses (10/21) |
Cypress Bioscience Inc. (CYPB) | Milnacipran | Norepinephrine | Fibromylasia syndrome | Data presented at meeting from Phase II trial showed drug improved syndrome in multiple measures of clinical pain (10/28) |
Cypress Bioscience Inc. (CYPB) | Milnacipran | Norepinephrine | Fibromylasia syndrome | Cypress began a Phase III program in the U.S. expected to take about two years (10/21) |
Guilford Pharmaceuticals Inc. (GLFD) | GPI 1485 | Neuroimmunophilin ligand | Parkinson's | Company completed enrollment of 212 patients in Phase II trial (10/14) |
Durect Corp. (DRRX) | Chronogesic pain product | Osmotic implant that delivers sufentanil | Chronic pain | Durect said data from a preclinical model indicated a potential problem with the delivery mechanism, a result expected to delay the restart of the Phase III program (10/16) |
Ligand Pharmaceuticals | Avinza (FDA-approved) | Extended-release morphine | Chronic back pain | Company said product provided stable analgesia for one year without an increase in dose or use of rescue medicines; data were from open-label extension study in 128 patients (10/14) |
NeorogesX Inc.* | NGX-410 | Capsaicin dermal delivery system | Neuropathic pain associated with HIV and AIDS | NeurogesX began a Phase II/III study comparing analgesic effect compared with low-dose capsaicinin in up to 300 patients (10/8) |
Neurobiological Technologies Inc. (NTII) and Forest Laboratories Inc. | Namenda | Memantine; NMDA receptor antagonist | Neuropathic pain | Forest said it would continue to study the drug after analyzing Phase III results that did not demonstrate efficacy in treating nocturnal pain (10/22) |
Pain Therapeutics Inc. (PTIE) | Oxytrex | Small-molecule | Severe chronic pain | Company said Phase II results in osteoarthritic patients demonstrated statistically significant results in primary endpoint of prospectively defined reduction in pain scores at three weeks; study also met secondary endpoints (10/16) |
Sepracor | Estorra | Eszopiclone; non-benzodiazepine agent | Chronic insomnia | Published Phase III data showed statistically significant improvement; drug is on file with FDA (10/21) |
Targacept Inc.* | TC-1734 | Neuronal nicotinic receptor agonist | Cognitive and memory disorders | Company said Phase I trial showed no safety concerns and a dose-specific effect on surrogate measures of disease (10/30) |
DIABETES | ||||
AmCyte Inc.* | -- | Replacement islets for transplantation | Diabetes | Company said a Phase I/II trial was initiated that will evaluate islet transplantion in three patients (10/30) |
Generex Biotechnology | Oralin | Oral insulin spray formulation | Type II diabetes | Generex presented results from studies pointing to efficacy (10/7) |
Keryx Biopharmaceuticals Inc. (KERX) | KRX-101 | Sulodexidel; oral heparinoid compound | Diabetic nephropathy | Keryx began the Phase II portion of its Phase II/III program; this portion of the study will include about 135 patients with Type II diabetes (10/21) |
INFECTION | ||||
Amarillo | Interferon-alpha | Low-dose version formulated in lozenge | Oral warts in HIV patients | Amarillo filed an IND for a pivotal Phase III study in 90 patients (10/14) |
AVI BioPharma | AVI-420 | Neugene | West Nile virus | AVI said it would being a second study in West Nile virus to allow expanded access of the drug (10/8) |
Biosynexus Inc.* | BSYX-L210 | Intranasal cream containing lysostaphin | Eradication of S. aureus nasal carriage | Company said Phase I/II trial met endpoints for safety and micro-biological activity, and data will be submitted for publication (10/14) |
Chiron | Tifacogin | Recombinant form of tissue factor pathway inhibitor | Severe | Chiron said it was initiating plans for a Phase III trial; it also said it acquired all of Pfizer Inc.'s interest in the drug (10/29) |
Genzyme Corp. (GENZ) | -- | Tolevamer sodium | Clostridium difficile-associated diarrhea | Genzyme presented preliminary data from a Phase II trial in 289 patients showing non-inferiority to vancomysin in time to diarrhea resolution (10/20) |
Gilead | Hespera (FDA-approved) | Adefovir dipivoxil; nucleoside analogue | Hepatitis B | Gilead presented data from open-label Study 460i showing drug was associated with sustained reduction of HBV through 144 weeks in lamivudine-resistant patients co-infected with HIV (10/28) |
Gilead | Viread (FDA-approved) | Tenofovir disoproxil fumarate; nucleotide analogue reverse transcriptase inhibitor | HIV | One presentation from extension of pivotal study showed low incidence of adverse events for up to four years; another, from the1,350- patient RECOVER study, suggested drug in place of stavudine may improve dyslipidemia (10/28) |
Human Genome Sciences Inc. (HGSI) | Albuferon | Long-acting form of interferon-alpha | Hepatitis C | Interim results from Phase I/II trial showed drug was well tolerated, had a prolonged half-life and was biologically active (10/28) |
Idenix Pharmaceuticals Inc.* | Telbivudine (LdT) | Antiviral agent | Chronic hepatitis B | 52-week data from Phase IIb trial in 104 patients showed better suppresion of HBV and normalization of ALT when compared to lamivudine monotherapy (10/28) |
Idun Pharmaceuticals Inc.* | IDN-6556 | Inhibitor of caspase enzymes that mediate apoptosis | Liver impairment caused by hepatitis C infection | Dose-ranging study showed the drug normalized liver enzymes after two weeks of treatment; data are based on results from 41 patients (10/27) |
InterMune Inc. (ITMN) | Infergen | Interferon alfacon-1 | Hepatitis C | Company said an investigator-sponsored study showed drug with ribavarin produced positive results (10/29) |
MedImmune Inc. (MEDI) | Numax | Next-generation antibody | Respiratory syncytial virus | MedImmune submitted IND for first trial of Numax to assess safety and tolerability in healthy adult volunteers (10/1) |
MedImmune Inc. (MEDI) and Wyeth | -- | Intranasal, cold-adapted trivalent vaccine | Influenza | Companies presented encouraging data from five studies totaling 5,500 patients (10/13) |
Samaritan Pharmaceuticals Inc. (OTC BB:SPHC) | -- | Neuroimmuno- | HIV | Company submitted Phase II clinical report to FDA showing data demonstrating statistical significance for viral load and quality of life, and said it is developing a Phase III regimen (10/28) |
SciClone Pharmaceuticals Inc. (SCLN) | Zadaxin | Synthetic thymosin alpha-1 | Hepatitis C | Results from 23 patients in pilot trial showed triple therapy with ribavarin and pegylated interferon-alpha produced a 12-week early virologic response in 61% of non-responder patients (10/27) |
Trimeris Inc. (TRMS) and F. Hoffmann La-Roche Ltd. (Switzerland) | Fuzeon (FDA-approved) | Enfuvirtide; HIV fusion inhibitor | HIV | New data presented at AIDS conference showed Fuzeon as part of combination therapy provided virologic and immunologic benefit through 48 weeks in patients with less-advanced HIV (10/27) |
Vertex Pharmaceuticals | Lexiva (FDA-approved) | Fosamprenavir | HIV infection | Data from Phase III study showed HIV patients taking drug boosted with ritonavir had increased levels of HDL cholesterol at 48 weeks (10/28) |
Vertex Pharmaceuticals Inc. (VRTX) | VX-497 | Merimepodib; small- molecule inhibitor of the enzyme inosine guanosine triphosphate | Hepatitis C | Vertex said the drug met its primary endpoint of safety and tolerability in an interim analysis from a Phase II study in 31 patients; it also showed clinical activity (10/17) |
Vicuron Pharmaceuticals | Dalbavancin | Semi-synthetic glycopeptide | Skin and | Vicuron started a Phase III study comparing its drug to vancomycin in up to 150 patients (10/8) |
MISCELLANEOUS | ||||
Adolor | Entereg | Alvimopan; mu opioid antagonist | Postoperative ileus | Top-line results from 519-patient Phase III study showed the drug generally was well tolerated; other Phase III trials are ongoing (10/22) |
Altus Biologics Inc.* | TheraCLEC | Oral enzyme replacement therapy | Malabsorption resulting from pancreatic insufficiency | Altus said a Phase I trial demonstrated a favorable safety and clinical activity profile (10/22) |
Avanir Pharmaceuticals Inc. (AMEX:AVN) | AVP 13358 | Molecule designed to inhibit production of IgE | Allergy and asthma | Avanir said preliminary results show product was well tolerated at single rising doses in a Phase I trial (10/21) |
AVI BioPharma Inc. (AVII) | AVI-4557 | Neugene antisense drug | To affect drug metabolism | AVI initiated Phase I trial of oral drug (10/10) |
BioSante Pharmaceuticals Inc. (AMEX:BPA) | LibiGel | Gel formulation of testosterone | Female sexual dysfunction | BioSante said interim analysis of Phase II trial showed statistically significant improvement in sexual desire and activity (10/21) |
Corus Pharma Inc.* | Corus 1020 | Aztreonam formulated for inhalation | Cystic fibrosis | Corus initiated Phase II trial to evaluate safety, tolerability and efficacy (10/15) |
ICOS Corp. (ICOS) and Eli Lilly and Co. | Cialis | Talafidil; PDE 5 inhibitor dysfunction | Erectile Phase III data showing efficacy | Companies' joint venture reported of Cialis in men who have undergone a form of prostatectomy (10/10) |
Genaera Corp. (GENR) | Lomucin | Talniflumate; an anti- inflammatory drug | Cystic fibrosis | Genaera and Cystic Fibrosis Foundation Therapeutics presented Phase II data showing safety and maintenance of existing lung function in CF patients (10/16) |
Genaera Corp. (GENR) | Squalamine | Anti-angiogenic drug | Choroidal neo-vascularization related to age- related macular degeneration | Company said updated Phase II results in 40 patients were positive (10/7) |
Idun Pharmaceuticals Inc.* | IDN-6556 | Designed to protect liver cells from apoptosis | Liver damage in transplantation | Twelve transplant centers are expected to participate in the 100-patient Phase II study (10/8) |
Indevus Pharmaceuticals Inc. (IDEV) | Trospium | Muscarinic receptor antagonist | Overactive bladder | Indevus presented data showing that early patient response was an accurate predictor of long-term therapeutic success (10/14) |
Indevus Pharmaceuticals Inc. (IDEV) | Trospium | Muscarinic receptor antagonist | Urgency severity, a symptom of overactive bladder | Indevus presented data from controlled, 523-patient study showing reduced urgency severity (10/7) |
Neurotech SA* (France) | NT-501 | Intraocular polymer containing cells modified to secrete ciliary neurotrophic factor | Retinitis | Company initiated Phase I trial in 10 patients with late-stage disease; trial will be conducted at U.S. National Eye Institute (10/8) |
NexMed Inc. | Femprox | Alprostadil cream arousal disorder | Female sexual showed positive dose-related | Phase II data published in journal (NEXM) trends (10/7) |
NPS Pharmaceuticals Inc. (NPSP) | Preos | Recombinant, full-length parathyroid hormone | Osteoporosis | NPS completed enrollment of 2,700 women in Phase III TOP trial (10/7) |
Progenics Pharmaceuticals Inc. (PGNX) | Methylnaltrexone | Opioid-receptor antagonist | Opioid-induced urinary retention | Company said Phase II data showed drug relieved condition without reversing CNS effects of the opiod (10/10) |
RegeneRx Biopharmaceuticals Inc. (OTC BB:RGRX) | Thymosin beta-4 | Naturally occurring peptide | Wound healing | RegeneRx completed Phase I trial in 15 volunteers, showing it was well tolerated (10/8) |
Theratechnologies Inc. (Canada; TSE:TH) | ThGRF | Growth hormone-releasing factor analogue | Chronic wasting | Preliminary results from Phase II study in patients with disease secondary to chronic obstructive pulmonary disease suggested improvement in condition (10/21) |
Xenova Group plc (UK; XNVA) | TA-CD | Therapeutic vaccine designed to produce anti-cocaine antibodies | Cocaine addiction | Xenova initiated Phase IIb trial in up to 132 methadone-dependent addicts who will be given drug or placebo; trial is expected to last two years (10/24) |
Xenova Group plc (UK; XNVA) | TA-NIC | Therapeutic vaccine | Smoking cessation | Xenova began a second Phase I trial to further establish safety and tolerability, and to determine dosing (10/8) |
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Notes: | ||||
* Privately held. | ||||
** Date item was published in BioWorld International. | ||||
ACR = American College of Rheumatology meeting | ||||
Unless otherwise noted, the stock symbols listed for public companies are on the Nasdaq market. | ||||
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TSE = Toronto Stock Exchange. | ||||