Company*
(Country;
Symbol)

Product

Description

Indication

Status (Date)


AUTOIMMUNE

Active Biotech
AB*
(Sweden)
and Avidex Ltd.*
(UK)

RhuDex

Small-molecule CD80 antagonist

Rheumatoid arthritis

Phase I trials of the product began (5/9)

Astralis Ltd.
(OTC BB:ASTR)

Psoraxine

Protein-based immunostimulator

Psoriasis

Further data from Phase II trial that failed to meet primary end-point identified factors that could have led to that result (5/6)

Enzo Biochem
Inc.
(NYSE:ENZ)

Alequel

A complex of autologous colon-derived antigens administered orally

Crohn's disease

Phase IIb trial produced clinical remission rates of 58% for drug patients vs. 29% for placebo (5/16)

Medarex Inc.
(MEDX)

MDX-1100

Fully human monoclonal antibody that targets the chemokine IP-10 (CXCL10)

Ulcerative colitis

Began Phase I trial in up to 32 patients to collect safety and initial efficacy data (5/11)

Synta
Pharmaceuticals
Corp.*

STA-5326

Small-molecule, selective inhibitor of the IL-12 cytokine family

Crohn's disease

Phase IIa trial in 73 patients demonstrated meaningful clinical activity at all dose levels except the lowest (5/18)

CANCER

Adherex
Technologies
Inc.
(AMEX:ADH)

ADH-1 (Exherin)

Cadherin antagonist; small peptide, tumor/vascular-targeting agent

Advanced chemotherapy-resistant cancer

Phase I data showed ADH-1 was well tolerated over a 20-fold dose range and has displayed evidence of antitumor activity (ASCO)

Adherex
Technologies
Inc.
(AMEX:ADH)

ADH-1 (Exherin)

Cadherin antagonist; small peptide, tumor/vascular-targeting agent

Advanced cancers

Began Phase Ib/II trial in 40 patients whose tumors express N-cadherin, to evaluate safety, tolerability and antitumor activity (5/12)

Adventrx
Pharmaceuticals
Inc.
(AMEX:ANX)

CoFactor

Form of folic acid designed to enhance the antitumor effects of 5-FU

Metastatic colorectal cancer

Phase II trial with 5-FU showed a 63% overall clinical benefit and a 38% objective response rate (5/13)

AEterna
Zentaris Inc.
(Canada; AEZS)

Neovastat

Agent believed to inhibit VEGF signaling, MMP activity and induction of endothelial-cell apoptosis

Stage III non- mall-cell lung cancer

Data from 341 patients in NCI Phase III trial demonstrated that blinded overall toxicity appeared acceptable (ASCO)

Agennix Inc.*

Talactoferrin

Oral, recombinant human lactoferrin

Advanced non- small-cell lung cancer

Phase II data showed drug enhanced patient responses to chemotherapy on all tumor-related endpoints (ASCO)

Amgen Inc. (AMGN) and
Abgenix Inc.
(ABGX)

Panitumumab

Fully human monoclonal antibody that targets the epidermal growth factor receptor

Metastatic colorectal cancer

Updated data from Phase II single- agent trial demonstrated a 9% overall response rate (ASCO)

Amgen Inc.
(AMGN)

Neulasta (FDA-approved)

Pegfilgrastim; white-blood-cell stimulator

Solid tumors

Trial in older patients showed those receiving Neulasta in the first chemotherapy cycle had reductions in febrile neutopenia and related complications, compared to those who received it in later chemotherapy cycles (ASCO)

Amgen Inc.
(AMGN)

Aranesp (FDA-approved)

Darbepoetin alfa; a recombinant erythropoietic protein

Chemotherapy-induced anemia

Phase III data demonstrated that 200 mcg of Aranesp every two weeks is as effective as 40,000 U of Epoetin alfa once a week in boosting hemoglobin levels and reducing red-blood-cell transfusions (ASCO)

Antisoma plc
(UK; LSE:ASM)

AS1411 (formerly AGRO100)

Anti-nucleolin aptamer

Spreading cancers

Seven patients in Phase I trial showed stable disease for two months or more, and one had a near-complete response (ASCO)

Aphton Corp.
(APHT)

IGN101

Vaccine designed to trigger an immune response to epithelial cell-adhesion molecule

Solid tumors

Phase II data showed the median number of circulating tumor cells had been reduced by about 50% (ASCO)

Ariad Pharmaceuticals
Inc.
(ARIA)

AP23573

Oral form of agent designed to inhibit cell-signaling protein mTOR

Refractory/relapsed cancers

Began Phase Ib trial to evaluate safety, activity and pharmaco-kinetics in up to 150 patients (5/26)

Ariad
Pharmaceuticals
Inc.
(ARIA)

AP23573

Intravenous form of agent designed to inhibit cell-signaling protein mTOR

Advanced sarcoma

Phase II data showed clinical benefits and symptomatic improvements (ASCO)

Ariad
Pharmaceuticals
Inc.
(ARIA)

AP23573

Small molecule designed to inhibit cell-signaling protein mTOR

Refractory prostate cancer

Began Phase II single-agent trial to evaluate the clinical benefit in up to 35 patients (5/3)

ArQule Inc.
(ARQL)

ARQ 501

Activated Checkpoint Therapy molecule designed to selectively kill cancer cells

Advanced solid tumors

Phase I data demonstrated clinical tolerability, favorable pharmacokinetics and evidence of anti-tumor activity (ASCO)

Callisto
Pharmaceuticals
Inc.
(AMEX:KAL)

Atiprimod

Drug designed to inhibit VEGF and interleukin-6

Relapsed multiple myeloma

No dose-limiting toxicities were observed through three patient cohorts in Phase I/IIa trial (5/4)

CancerVax
Corp.
(CNVX)

Canvaxin

Therapeutic whole-cell vaccine that expresses more than 20 antigens

Melanoma

Phase II trial in 13 patients showed treatment induced antibody responses to several melanoma-associated antigens (ASCO)

CancerVax
Corp.
(CNVX)

SAI-EGF

Immunotherapy agent that targets the epidermal growth factor receptor signaling pathway

Advanced non-small- cell lung cancer

Phase II trial showed agent was well tolerated, stimulated an antibody response to EG, and reduced circulating EGF (ASCO)

Celgene Corp.
(CELG)

Revlimid

Lenalidomide; derivative of Thalomid (thalidomide)

Chronic lymphocytic leukemia; myelodysplastic syndrome; multiple myeloma

Clinical data showed that 12 of 14 relapsed or refractory CLL patients responded to treatment; Revlimid provided evidence it could be used to treat MDS patients with deletion 5q chromosomal abnormalities; Phase III data showed Revlimid plus dexamethasone more than doubled the response rate in MM patients (ASCO)

Cell
Genesys Inc.
(CEGE)

GVAX

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

Advanced prostate cancer

Additional data from 22 patients in Phase II trial showed final median survival will be no less than 24.1 months (ASCO)

Cell
Genesys Inc.
(CEGE)

GVAX

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

Acute myelogenous leukemia

Follow-up data from Phase II trial showed GVAX generally was well tolerated and may reduce residual leukemic cells (ASCO)

Cell
Therapeutics
Inc.
(CTIC)

Xyotax

Paclitaxel linked to a biodegradable polyglutamate polymer

Ovarian cancer (Stage III/IV); esophageal and gastric cancer; non- small-cell lung cancer

Phase II data of Xyotax with carboplatin showed a major tumor response in 98% of patients; Phase I data of Xyotax with radiation demonstrated complete disappearance of the tumor in 36% of patients; Phase III trial showed a 40% improvement in overall survival compared with vinorelbine (ASCO)

Cell
Therapeutics
Inc.
(CTIC)

Xyotax

Paclitaxel linked to a biodegradable polyglutamate polymer

Non-small-cell lung cancer

Phase III STELLAR 2 and 4 trials missed their primary endpoints of superior overall survival, but did show equivalent survival with reduced side effects (5/2)

Cel-Sci Corp.
(AMEX:CVM)

Multikine

Mixture of human cytokines designed to stimulate an immune response

Advanced oral squamous-cell carcinoma

Phase II data showed an overall response rate of 42%, with a 12% complete response rate (5/20)

Coley
Pharmaceutical
Group Inc.*

CPG 7909

Toll-like receptor 9 agonist

Advanced non-small-cell lung cancer

Phase II data of CPG 7909 with standard chemotherapy showed an objective response rate of 37%, compared with 19% in those receiving chemotherapy alone (ASCO)

CuraGen Corp.
(CRGN)

CG53135

Growth factor that promotes both epithelial- and mesenchymal-cell proliferation

Oral mucositis in cancer patients

Final Phase I data suggested the drug was well tolerated following intravenous administration (ASCO)

Cytogen Corp.
(CYTO)

Quadramet (FDA-approved)

Samarium-153 bound to a small-molecule, bone- seeking phosphonate

Progressive hormone- refractory prostate cancer

Phase II data of Quadramet with docetaxel demonstrated good palliative effect lasting five months (ASCO)

Cytokinetics
Inc.
(CYTK) and
GlaxoSmithKline
plc (UK)

SB-743921

Small-molecule inhibitor of kinesin spindle protein

Cancers

Phase I data showed the compound appeared to have an acceptable tolerability profile (ASCO)

Dendreon
Corp.
(DNDN)

Provenge

Immunotherapy that uses a recombinant form of the prostatic acid phosphatase antigen

Early stage prostate cancer

Open-label D9905 Phase II trial suggested single-agent treatment may lead to improved PSA doubling time (5/24)

Dendreon
Corp.
(DNDN)

Provenge

Immunotherapy that uses a recombinant form of the prostatic acid phosphatase antigen

Asymptomatic, metastatic androgen-independent prostate cancer

Provenge improved survival when compared to patients receiving placebo (ASCO)

Genentech Inc.
(NYSE:DNA)

Herceptin (FDA-approved)

Trastuzumab; monoclonal antibody against HER2/neu protein

Early stage breast cancer

Phase III data showed that HER2-positive patients had a 52% reduction in the risk of disease recurrence compared to those receiving chemotherapy alone (ASCO)

Genentech Inc.
(NYSE:DNA) and
F. Hoffmann-La
Roche Ltd.
(Switzerland)

Avastin (FDA-approved)

Bevacizumab; antibody designed to inhibit vascular endothelial growth factor

First-line, non-squamous, non-small-cell lung cancer

A Phase III trial of Avastin plus paclitaxel and carboplatin met its primary endpoint, with treated patients having a 30% improvement in overall survival (ASCO)

Genentech Inc.
(NYSE:DNA) and
OSI Pharmaceuticals
Inc.
(OSIP)

Tarceva (FDA-approved)

Erlotinib HCl; small- molecule HER1/EGFR inhibitor

Advanced pancreatic cancer

Phase III data showed a 23.5% improvement in overall survival when Tarceva was used with gemcitabine (ASCO)

Genentech Inc.
(NYSE:DNA)

Omnitarg

Pertuzumab

Ovarian, breast and prostate cancers

Phase II data showed Omnitarg had limited activity as a single agent, but the company plans to pursue further study in ovarian cancer (ASCO)

Genentech Inc.
(NYSE:DNA) and
Curis Inc.
(CRIS)

--

Antagonist of the Hedgehog signaling pathway

Basal-cell
carcinoma

Starting a Phase I trial of an agent for the topical treatment of BCC (5/4)

Genmab A/S
(Denmark; CSE:
GEN)

HuMax-EGFr

Human antibody that targets the epidermal growth factor receptor

Refractory head and neck cancer

Additional data from Phase I/II trial showed seven of 18 patients achieved partial responses and four had stable disease(ASCO)

Genta Inc.
(GNTA)

Genasense

Oblimersen sodium; inhibits function of bcl-2 protein

Relapsed/refractory chronic lymphocytic leukemia

Updated data from positive Phase III trial showed significantly superior duration of responses (5/13)

Genta Inc.
(GNTA)

Genasense

Oblimersen sodium; inhibits function of bcl-2 protein

Solid tumors

Combination trial showed tolerability in children with solid tumors; 14 of 27 prostate cancer patients achieved a major reduction in PSA levels; Phase I trials in gastrointestinal cancer produced safety data (ASCO)

Genta Inc.
(Genta)

Genasense

Oblimersen sodium; inhibits function of bcl-2 protein

Advanced malignant melanoma

Phase III data showed a significant increase in durable response and a near-significant trend toward increased overall survival (ASCO)

GenVec Inc.
(GNVC)

TNFerade

Adenovector with TNF-alpha gene and a radiation-responsive promoter

Rectal cancer

Company got OK from FDA to resume enrollment in Phase II trial; a hold had been placed on the study in October 2004 (5/25)

GenVec Inc.
(GNVC)

TNFerade

Adenovector with TNF-alpha gene and a radiation-responsive promoter

Advanced pancreatic cancer

Phase II data showed dose-dependent improvements in tumor shrinkage and resectability and in patient survival (ASCO)

Geron Corp.
(GERN)

GRN163L

Telomerase inhibitor

Chronic lymphocytic leukemia

Starting a Phase I/II trial to assess safety, tolerability and pharmacokinetics in patients with advanced CLL (5/23)

Gloucester
Pharmaceuticals
Inc.*

FK228

Depsipeptide; histone deacetylase inhibitor

Cutaneous T-cell lymphoma and solid tumors

Objective responses were seen in five of 19 patients with advanced CTCL in Phase II trial; drug was relatively well tolerated in Phase I trial in pediatric patients with solid tumors (ASCO)

GTx Inc.
(GTXI)

Acapodene

Toremifene citrate; small- molecule non-steroidal selective estrogen receptor modulator

Prostate cancer

Phase IIb data showed it significantly reduced the risk of prostate cancer by about 48% among men treated for a year (ASCO)

Guilford
Pharmaceuticals
Inc.
(GLFD)

Gliadel Wafer (FDA-approved)

Wafer comprised of a biodegradable polymer incorporating carmustine

High-grade malignant glioma

Trial of drug and temozolomide determined the combination to be safe (ASCO)

Hana
Biosciences Inc.
(OTC BB:HNAB)

Talotrexin (PT-523)

Non-classical antifolate; analogue of aminopterin

Acute lymphoblastic leukemia

Began Phase I/II single-agent trial in relapsed or refractory patients that initially will assess dosing, safety, tolerability and pharmacokinetics (5/12)

Human
Genome
Sciences
Inc.
(HGSI)

HGS-ETR2 and HGS-ETR1

Agonistic human monoclonal antibody to TRAIL receptors 2 and 1, respectively

Advanced solid tumors

Ongoing Phase I trials demonstrated tolerability of both agents, and support further evaluation in Phase II trials (ASCO)

Hybridon Inc.
(AMEX:HBY)

Imoxine

Immunomodulatory oligonucleotide; TLR9 agonist

Renal-cell carcinoma

Phase I data showed it was well tolerated and had immunopharmacological activity (ASCO)

ImClone
Systems Inc.
(IMCL) and
Bristol-Myers
Squibb Co.

Erbitux (FDA-approved)

Cetuximab; antibody that blocks the epidermal growth factor receptor

Colorectal cancer

Data showed a 37% partial response in irinotecan-refractory metastatic colorectal cancer patients when Erbitux was used with Avastin and irinotecan, and it showed a 10% complete response and a 71% partial response in patients with non-resectable, EGFR-expressing metastatic colorectal cancer treated with Erbitux and Folfox- (ASCO)

Immuno-
Designed
Molecules
SA*
(France)

Mepact

Immune system stimulant designed to activate macrophages

High-grade osteosarcoma

Six-year data from 793 patients suggested drug added to chemotherapy improves disease-free and overall survival in those with nonmetastatic disease (ASCO)

ImmunoGen
Inc.
(IMGN)

huN901-DM1

Anti-CD56 antibody huN901 combined with the cell-killing agent, DM1

Small-cell lung cancer

Initial Phase II data demonstrated evidence of anticancer activity, triggering expansion of the study (ASCO)

Inex
Pharmaceuticals
Corp.
(Canada;TSE:IEX)

Marqibo

Vincristine encapsulated in Inex's TCS (liposomal) drug delivery technology

Various malignancies

Phase II trials totaling 211 patients with non- Hodgkin's lymphoma demonstrated clinically meaningful responses; Phase II follow-up data in relapsed Hodgkin's disease showed agent was well tolerated; Phase II trial in 30 patients with pediatric malignancies concluded further study was warranted (ASCO)

Introgen
Therapeutics
Inc.
(INGN)

INGN 225

Product using Advexin to stimulate dendritic cells, which are then used as a therapeutic vaccine

Advanced small-cell lung cancer

Phase II data showed that 67% of patients had objective responses following INGN 225 treatment (ASCO)

Keryx Bio-
pharmaceuticals
Inc.
(KERX)

KRX-0401

Perifosine; oral AKT inhibitor

Biochemically recurrent hormone-sensitive prostate cancer

Phase II data demonstrated the tolerability and potential efficacy of KRX-0401 (ASCO)

Keryx Bio-
pharmaceuticals
Inc.
(KERX)

KRX-0401

Perifosine; oral AKT inhibitor

Breast cancer

Began two Phase II trials, one in combination with Herceptin and one with endocrine therapy (5/11)

Kosan
Biosciences
Inc.
(KOSN)

KOS-862

Epothilone D, a
polyketide

Advanced non-small-cell lung cancer

Four of 50 patients in a single-agent Phase II trial had partial responses, and 13 had disease stabilization (ASCO)

Kosan
Biosciences
Inc.
(KOSN)

KOS-953

17-AAG; designed to inhibit heat-shock protein 90

Relapsed or refractory multiple myeloma

Phase I data showed anticancer
activity and tolerability (ASCO)

Ligand
Pharmaceuticals
Inc.
(LGND)

Targretin (FDA-approved)

Bexarotene capsules; selective retinoid X receptor modulator

Advanced non-small-cell lung cancer

Pivotal Phase III trials did not meet their endpoints of improved overall survival and projected two- ear survival; separately, positive data were reported from other studies (ASCO)

Ligand
Pharmaceuticals
Inc.
(LGND)

Ontak (FDA-approved)

Denileukin diftitox

Advanced non-small-cell lung cancer and CLL

Phase II trial showed disease control in 16 of 40 NSCLC patients; Phase II trial in 16 patients with B-cell chronic lymphocytic leukemia showed moderate activity with manageable toxicities (ASCO)

Marshall
Edwards Inc.
(MSHL)

Phenoxodiol

Agent designed to induce apoptosis by removing intracellular proteins XIAP and c-FLIP

Ovarian cancer

Phase IIa trial in 40 patients resistant to cisplatin and/or paclitaxel showed an overall disease control rate of 77% when drug was added to the regimen (5/10)

MedImmune
Inc.
(MEDI)

Siplizumab

Humanized monoclonal antibody that binds to the CD2 receptor on T cells and natural killer cells

T-cell lymphomas and leukemias

Preliminary Phase I data demonstrated safety and tolerability; partial disease remissions also were noted (ASCO)

MedImmune
Inc.
(MEDI)

Vitaxin

Monoclonal antibody that targets the alpha-v beta-3 integrin

Stage IV meta- static melanoma

Phase II data showed a 12.7- month median survival for those treated with Vitaxin alone, and a 9.4-month median survival of patients treated with Vitaxin plus dacarbazine (ASCO)

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

Dacogen

Decitabine for injection; a hypomethylating agent

Myelodysplastic syndromes

Dacogen demonstrated a 44% overall response rate in a dosing study and a 17% overall response rate in the MDS trial (ASCO)

Millennium
Pharmaceuticals
Inc.
(MLNM)

Velcade (FDA-approved)

Bortezomib; proteasome inhibitor

Lymphoma, lung cancer and multiple myeloma

Median survival had not been reached at 10.5 months in mantle- cell lymphoma patients, who had an overall response rate of 42%; lung cancer patients showed a response rate comparable with approved agents; MM front-line data show potential benefits in earlier lines of therapy (ASCO)

Millennium
Pharmaceuticals
Inc.
(MLNM)

MLN2704

Monoclonal antibody (MLN591) directed at PSA conjugated to DM1

Prostate cancer

Phase I/II data showed four of six patients receiving a high dose achieved a reduction in prostate-specific antigen (ASCO)

NeoPharm Inc.
(NEOL)

LErafAON- ETU

Liposomal formulation of an antisense oligonucleotide complementary to the c-raf mRNA sequence

Advanced cancers

Reported on successful initial clinical dosing in a Phase I trial (ASCO)

NeoPharm Inc.
(NEOL)

IL13-PE38QQR

Tumor-targeting agent designed to deliver bacterial cytotoxic PE38

Brain cancer

Combined data from 45 patients in Phase I trials showed median survival of 44 weeks, vs. 28 weeks for approved therapies (5/6); combined data from Phase I trials showed advantages for peritumoral vs. intratumoral infusion (ASCO)

NeoRx Corp.
(NERX)

Skeletal Targeted
Radiotherapy

Bone-targeting molecule DOTMP with radioactive substance holmium-166

Various cancers

NeoRx discontinued clinical development of STR for multiple myeloma and other indications, after the FDA sought changes for Phase III trials (5/6)

Novacea Inc.*

DN-101

Contains calcitriol, a naturally occurring hormone and the biologically active form of vitamin D

Prostate cancer

Data demonstrated that prostate-specific antigen response occurred more frequently in patients receiving DN-101 plus Taxotere than those in the control group, but the difference was not statistically significant (ASCO)

Onyx
Pharmaceuticals
Inc.
(ONXX) and
Bayer Pharma-
ceuticals Corp.

Sorafenib (BAY 43-9006)

RAF kinase and VEGF inhibitor

Advanced renal-cell carcinoma

Phase III data showed sorafenib improved progression-free survival from a median value of 12 weeks up to 24 weeks (ASCO)

Onyx
Pharmaceuticals
Inc.
(ONXX) and
Bayer Pharma-
ceuticals Corp.

Sorafenib (BAY 43-9006)

RAF kinase and VEGF inhibitor

Advanced metastatic melanoma

Began Phase III trial with carboplatin and paclitaxel that will assess progression-free survival in more than 200 patients (5/13)

OSI
Pharmaceuticals
Inc.
(OSIP)

Tarceva (FDA-approved)

Erlotinib HCl; small- molecule HER1/EGFR inhibitor

Non-small-cell lung cancer

Analysis of a registration study showed a greater, but not significant, survival benefit from Tarceva in all subsets of patients whose tumors expressed EGFR protein; Phase II monotherapy trials showed encouraging signs of antitumor activity, and combination studies outside NSCLC also were encouraging (ASCO)

OxiGene Inc.
(OXGN)

CA4P

Combretastatin A4 prodrug; a vascular targeting agent

Advanced cancer

Phase Ib/II data showed it was well tolerated when combined with radiotherapy (ASCO)

OxiGene Inc.
(OXGN)

CA4P

Combretastatin A4 prodrug; a vascular targeting agent

Advanced cancers

Antitumor activity and no unexpected toxicities were seen in Phase Ib portion of Phase Ib/II combination trial (ASCO)

Pharmacyclics
Inc.
(PCYC)

Xcytrin

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

Advanced renal-cell carcinoma

Interim data from Phase II trial in 22 patients showed clinical benefit in five patients; three patients showed tumor responses (ASCO)

Pharmacyclics
Inc.
(PCYC)

Xcytrin

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

Advanced refractory tumors

Interim data from Phase I trial with Taxotere showed partial responses in six of 12 patients; two more patients achieved stabilization of their tumors (ASCO)

Point
Therapeutics
Inc.
(POTP)

Talabostat

Small molecule designed to stimulate proliferation of hematopoietic progenitor cells

Advanced non-small-cell lung cancer

Five of 36 patients patients demonstrated a clinical response to combination treatment with docetaxel in a Phase II trial (ASCO)

Point
Therapeutics
Inc.
(POTP)

Talabostat

Small molecule designed to stimulate proliferation of hematopoietic progenitor cells

Stage IV melanoma

Phase II data showed two patients had at least a 30% reduction in tumor size in response to talabostat, with one achieving a complete response (ASCO)

Praecis
Pharmaceuticals
Inc.
(PRCS)

Plenaxis (FDA-approved)

Abarelix for injection, depot formulation; gonadotropin-releasing hormone antagonist

Palliative treatment of advanced prostate cancer

As part of a restructuring, the firm discontinued promotional activities and the sale of Plenaxis for new patients in the U.S. (5/20)

Pro-
Pharmaceuticals
Inc.
(AMEX:PRW)

Davanat

Carbohydrate compound designed for targeted delivery

Metastatic colorectal cancer

Began Phase II trial to evaluate efficacy and safety with 5-FU as as third- or fourth-line therapy in up to 38 patients (5/9); Phase I trial showed disease stabilization in 14 of 26 patients (ASCO)

Reata
Pharmaceuticals
Inc.*

RTA 744

Anthracycline derivative that crosses the blood- brain barrier

Advanced brain cancers

Starting trials of the drug in patients (5/6)

Regeneron
Pharmaceuticals
Inc.
(REGN)

VEGF Trap

Intravenous product designed to block vascular endothelial growth factor

Advanced solid malignancies

Phase I trial in 27 patients showed the agent generally was well tolerated, and showed evidence of efficacy in some patients (ASCO)

Regeneron
Pharmaceuticals
Inc.
(REGN)

VEGF Trap

Intravenous product designed to block vascular endothelial growth factor

Advanced cancers

Began trial to assess safety, tolerability, pharmacokinetics and responses in combination with the Folfox-4 regimen (5/12)

Seattle
Genetics
Inc.
(SGEN)

SGN-30

Monoclonal antibody that targets CD30+ hematologic malignancies

Systemic anaplastic large-cell lymphoma

SGN-30 was well tolerated and demonstrated objective anti-tumor activity (ASCO)

Seattle
Genetics
Inc.
(SGEN)

SGN-40

Humanized monoclonal antibody that targets the CD40 antigen

Multiple myeloma

SGN-40 showed evidence of biological activity (ASCO)

Sonus
Pharmaceuticals
Inc.
(SNUS)

Tocosol

Formulation of paclitaxel using Sonus' delivery system

Cancers

Data showed a single dose administered over 15 minutes delivered 67% more active paclitaxel compared to an identical dose of Taxol (ASCO)

Spectrum
Pharmaceuticals
Inc.
(SPPI)

SPI-153

Fourth-generation luteinizing hormone- releasing hormone

Hormone-dependent prostate cancer

Starting a Phase I/II trial in patients (5/31)

Structural
GenomiX Inc.*

Troxatyl

Troxacitabine; anti- cancer agent

Acute myelogenous leukemia

Phase I/II trial in 48 patients demonstrated tolerability in relapsed patients, and an overall response rate of 17% (ASCO)

SuperGen Inc.
(SUPG)

Orathecin

Rubitecan; topoisomerase-I inhibitor in capsule formulation

Pancreatic cancer

Phase III data indicate patients with progressive disease following gemcitabine treatment can benefit from Orathecin (ASCO)

Synta
Pharmaceuticals
Corp.*

STA-4783

Small-molecule compound designed to induce expression of heat-shock protein 70 tumor cell surfaces

Various cancers

Phase II trials with paclitaxel in non-small-cell lung cancer and soft-tissue sarcoma supported advancement to the second stage of the trials; Phase I trial with pacli- taxel in various cancers demonstrated tolerability and some responses (ASCO)

Tapestry
Pharmaceuticals
Inc.
(TPPH)

TPI 287

Taxane designed to overcome the resistance to other taxanes

Recurrent/ refractory malignancies

Began Phase I trial to evaluate safety and pharmacokinetics in about 48 patients (5/23)

Telik Inc.
(TELK)

Telcyta (TLK286)

Small-molecule, tumor-activated drug designed to induce apoptosis

Advanced non-small-cell lung cancer

Phase I/IIa trial with carboplatin and paclitaxel in 13 patients showed a 62% objective response rate; trial with cisplatin showed a 36% objective response rate and 86% rate for disease stabilization (ASCO)

Therion
Biologics
Corp.*

Prostvac-VF

Therapy designed to stimulate the immune system to target and destroy cancer cells expressing epitopes of PSA

Metastatic androgen- insensitive prostate cancer

Phase II trial in 30 patients confirmed the safety profile and showed declines in PSA in several patients (ASCO)

Therion
Biologics
Corp.*

Panvac-VF

Vaccine designed to stimulate the immune system to target and destroy cells expressing CEA and mucin-1

Pancreatic cancer

Phase I data suggest the treatment was well tolerated and resulted in prolonged overall survival (ASCO)

Transgene SA
(France; TRGNY)

MVA-MUC1-IL2

Recombinant MVA virus containing sequences encoding for the human MUC1 antigen and the cytokine IL-2

Prostate cancer and non- small-cell lung cancer

Phase II data showed that in 63% of patients MVA-MUC1-IL2 induced a significant increase in the time it took for PSA to double; a tumor response rate of 37% was seen in a 44-patient combination NSCLC trial (ASCO)

Vical Inc.
(VICL)

Allovectin-7

DNA plasmid/lipid complex containing the DNA sequences encoding HLA-7 and ß2 microglobulin

Metastatic melanoma

Phase II data showed Allovectin-7 appeared to benefit from having at least two cycles of treatment (ASCO)

Vion
Pharmaceuticals
Inc.
(VION)

Cloretazine (VNP40101M)

Sulfonylhydrazine DNA alkylating agent

High-risk acute myelogenous leukemia and high-risk myelo-dysplasia

Cloretazine showed significant single-agent activity in previously untreated elderly patients (ASCO)

Xanthus Life
Sciences Inc.*

Xanafide

A new salt formulation of the cancer compound amonafide

Acute myeloid leukemia

Phase I trial with cytosine arabinoside showed 38% of 26 patients went into complete remission (ASCO)

Xcyte Therapies
Inc.
(XCYT)

Xcellerated T-Cells

T cells from patient stimulated to carry out immune functions

Chronic lymphocytic leukemia

Discontinued development of product in CLL after inability to reach agreement with FDA on an acceptable pathway for approval (5/16)

YM
BioSciences Inc.
(Canada; TSE:YM)

Norelin

Vaccine targeting the gonadotropin-releasing hormone

Hormone- dependent prostate cancer

The majority of patients developed antibodies to GnRH in a proof-of-concept study (5/25)

YM
BioSciences Inc.
(Canada; TSE:YM)

Tesmilifene(DPEE)

Small molecule designed to augment the activity of chemotherapy agents

Metastatic breast cancer

Updated data from Phase III trial showed a 143% overall survival improvement in the 191 patients whose cancer spread or recurred within 36 months of diagnosis (ASCO)

ZioPharm Inc.*

ZIO-101

Small-molecule organic arsenic

Solid tumors

Began a Phase I trial to assess safety and dosing in up to 40 patients with solid tumors (5/19)

ZymoGenetics
Inc.
(ZGEN)

--

Recombinant human interleukin-21

Cancer

Interleukin-21 showed evidence of antitumor activity and was reasonably well tolerated in a Phase I trial (ASCO)

CARDIOVASCULAR

Alteon Inc.
(AMEX:ALT)

Alagebrium
(ALT-711)

A.G.E. crosslink breaker

Various conditions

Data from the initial group in a Phase IIa trial demonstrated increased arterial elasticity and improved endothelial function (5/23)

Arginox
Pharmaceuticals*

Tilarginine Acetate Injection

Agent designed to inhibit the production of nitric oxide

Cardiogenic shock

Began Phase III TRIUMPH trial to test effectiveness on improved survival in heart attack patients who develop CS (6/23)

CoTherix Inc.
(CTRX)

Ventavis (FDA-approved)

Inhaled formulation of iloprost, a prostacyclin analogue

Pulmonary arterial hypertension

Follow-up data from 52 patients in a Phase III trial demonstrated long-term safety and efficacy in NYHA Class III and IV patients (5/23)

Encysive
Pharmaceuticals
Inc.
(ENCY)

Thelin

Sitaxsentan; small molecule designed to block endothelin

Pulmonary arterial hypertension

Phase III STRIDE-2 trial met the primary endpoint of improved six- minute walk distance; also, six poster presentations on the drug were made (5/22 and 5/23)

Myogen Inc.
(MYOG)

Ambrisentan

Type-A selective
endothelin receptor
antagonist

Pulmonary
arterial
hypertension

Presented additional and follow- up data from various trials that showed positive safety and efficacy results (5/23)

Myogen Inc.(MYOG)

Ambrisentan

Type-A selective endothelin receptor antagonist

Pulmonary arterial hypertension

Began trial to assess safety, tolerability and efficacy in 30 patients who discontinued bosentan or sitaxsentan therapy due to liver function test abnormalities (5/19)

Nuvelo Inc.
(NUVO)

rNAPc2

Recombinant nematode anticoagulant protein c2 originally isolated from hookworms

Acute coronary syndrome

Phase IIa ANTHEM trial showed an acceptable safety and tolerability profiles in patients with non-ST- elevation ACS (5/24)

Predix
Pharmaceuticals
Inc.*

PRX-08066

Selective 5-HT2B receptor antagonist

Pulmonary hypertension

Began a Phase I trial to evaluate safety, tolerability and pharmaco-kinetics (5/30)

Renovis Inc.
(RNVS)

Cerovive (NXY-059)

A neuroprotectant with free radical-trapping properties

Stroke

First analysis of Phase III SAINT I trial involving more than 1,700 patients showed a statistically significant reduction vs. placebo on disability; partner AstraZeneca plc ran the trial (5/4)

CENTRAL NERVOUS SYSTEM

Alkermes Inc.
(ALKS)

Vivitrex

Long-acting, injectable form of naltrexone

Alcohol dependence

12-month extension of Phase III trial showed drug and counseling led to a sustained reduction in heavy drinking over an 18-month period (5/23)

Cephalon Inc.
(CEPH) and
H. Lundbeck
A/S (Denmark)

CEP-1347

Small-molecule inhibitor of stress-activated protein kinase pathway

Parkinson's disease

Phase II/III trial was stopped after monitors concluded the data were unlikely to provide evidence of significant effect (5/11)

Cephalon Inc.
(CEPH)

OraVescent Fentanyl

Fentanyl formulated with transmucosal delivery technology

Breakthrough cancer pain

Phase III trial in 123 patients showed a statistically significant benefit; an NDA filing is planned for 3Q:05 (5/5)

Helicon
Therapeutics
Inc.*

HT-0712 (IPL455,903)

Oral inhibitor of phosphodiesterase-4

Learning and memory disorders

Phase I trial showed the drug was safe and well tolerated at all doses tested (5/11)

Metaphore
Pharmaceuticals
Inc.*

M40403

Superoxide dismutase mimetic

Post-surgical pain

Began a Phase II trial with morphine to compare analgesia to morphine alone in 240 bunionectomy patients (5/2)

Neurocrine
Biosciences
Inc.
(NBIX)

Indiplon

Capsule form of a non-benzodiazepine agent that targets the GABA-A receptor

Insomnia

Phase III trial in 702 patients showed significant and sustained improvement in sleep onset and sleep maintenance (5/25)

NeurogesX
Inc.*

NGX-4010

High-concentration trans-capsaicin dermal patch

HIV-associated sensory neuropathy

Phase III trial in 307 patients demonstrated statistically signifi- cant pain reduction over 12 weeks (5/18)

New River
Pharmaceuticals
Inc.
(NRPH)

NRP104

Prodrug of amphetamine

Attention deficit hyperactivity disorder

Each dose in a Phase III trial in 285 children demonstrated efficacy vs. placebo on both primary and secondary endpoints(5/23)

Praecis
Pharmaceuticals
Inc.
(PRCS)

Apan

Agent that may mobilize beta- myloid and facilitate its clearance

Alzheimer's disease

Company discontinued development of the early stage compound as part of a cost-cutting move (5/20)

Sepracor
Inc.
(SEPR)

Lunesta (FDA-approved)

Eszopiclone tablets; a non-benzodiazepine agent

Insomnia

Positive data were reported from Phase III/IV trial in patients with major depressive disorder and insomnia, and from trials in the elderly and adults (5/26)

DIABETES

Coremed Inc.*

Intesulin

Oral insulin

Diabetes

Initial trial of single gel capsule in patients demonstrated safety (5/9)

Keryx Bio-
pharmaceuticals
Inc.
(KERX)

KRX-101

Sulodexidel; oral heparinoid compound

Diabetic nephropathy

Interim Phase II data in 149 patients showed better success in drug group than placebo (5/6)

INFECTION

Acambis plc
(UK; ACAM)

ChimeriVax-West Nile

Vaccine that uses a live weakened yellow fever virus to create chimeric viruses

West Nile virus

Phase I trial in 80 subjects demonstrated development of neutralizing antibodies and high antibody titers (5/11)

Coley
Pharmaceutical
Group Inc.*

Actilon (CPG 10101)

Agent designed to target and stimulate Toll-like receptor 9

Hepatitis C

Phase Ib trial in 42 nonresponders showed good safety and tolerability, as well as antiviral activity (5/17)

Hemispherx
Biopharma
Inc.
(AMEX:HEB)

Alferon LDO

Low-dose, oral alpha- interferon cocktail

Avian flu and other viral diseases

Began trials in volunteers to determine whether new delivery format can resuscitate antiviral and immunostimulatory genes (5/16)

Idenix
Pharmaceuticals
Inc.
(IDIX)

Telbivudine

Selective, oral nucleoside agent

Hepatitis B

Data from an extended-treatment Phase IIb trial demonstrated continuing advantages vs. lamivudine across efficacy endpoints through two years of treatment (5/16)

Immtech
International
Inc.
(AMEX:IMM)

DB289

Oral, dicationic antiviral agent

Infectious diseases

Safety trial comparing three different formulations of drug led to selection of tablet formulation for use in pivotal Phase III trials (5/4)

Inhibitex Inc.
(INHX)

Aurexis

Humanized monoclonal antibody based on MSCRAMM protein platform

S. aureus infections

Phase II trial with antibiotic therapy in 60 patients demonstrated positive results in primary and secondary endpoints (5/9)

InterMune
Inc.
(ITMN)

Infergen (FDA-approved)

Interferon alfacon-1

Hepatitis C

Presented positive data from a trial with ribavirin and from a retrospective analysis evaluating drug and Actimmune in PEG-non- responders (5/16)

MedImmune
Inc.
(MEDI)

Numax

Third-generation variant of the anti-RSV antibody Synagis

Respiratory syncytial virus

Phase I/II trial in 217 high-risk infants demonstrated safety and tolerability, with an acceptable pharmacokinetic profile(5/14)

MedImmune
Inc.
(MEDI)

FluMist (FDA- approved) and CAIV-T

Intranasal influenza vaccine and next-generation, trivalent vaccine

Influenza

Presented data from various studies of both products that showed broad protection against the flu (5/14)

Nabi Bio-
pharmaceuticals
(NABI)

--

Vaccine designed to prevent Staphylococcus epidermidis infections

S. epidermidis infections

Began a Phase l trial to evaluate safety and immune responses in healthy volunteers (5/25)

Nabi Bio-
pharmaceuticals
(NABI)

StaphVAX

Staphylococcus aureus polysaccharide conjugate vaccine

To prevent S. aureus infections

Began bridging and consistency studies in healthy adults to compare three commercial lots of the vaccine (5/18)

Peregrine
Pharmaceuticals
Inc.
(PPHM)

Tarvacin

Chimeric monoclonal antibody that binds to the phospholipid phosphatidylserine

Hepatitis C

Began a Phase I trial to evaluate safety, pharmacokinetics and viral load in up to 32 relapsed or nonresponsive patients (5/31)

Samaritan
Pharmaceuticals
Inc.
(AMEX:LIV)

SP01A

Oral entry inhibitor agent

HIV

Began Phase II trial in 92 patients that will assess reductions in viral load (5/11)

Vertex
Pharmaceuticals
Inc.
(VRTX)

VX-950

Oral protease inhibitor

Hepatitis C

Phase Ib trial in 34 patients demonstrated tolerability and potent antiviral activity (5/10 and 5/17)

ViroPharma
Inc.
(VPHM)
and Wyeth

HCV-796

Polymerase inhibitor

Hepatitis C

Began Phase Ib trial to assess antiviral activity, safety and pharmacokinetics in 96 treatment-naïve patients (5/24)

MISCELLANEOUS

Acologix Inc.*

AC-100 (Dentonin)

Synthetic peptide derived from a protein produced by bone and dental cells

For use in dental procedures

Began a Phase I trial of the product (5/5)

Acuity
Pharmaceuticals
Inc.*

Cand5

Small interfering RNA therapeutic designed to shut down VEGF

Age-related macular degeneration

Interim safety data from Phase I trial were positive, with no drug- related adverse events (5/3)

Arena
Pharmaceuticals
Inc.
(ARNA)

APD356

Small-molecule, selective 5HT2C serotonin receptor agonist

Obesity

Phase II trial in 352 obese people showed a statistically significant average weight loss of 2.9 pounds vs. 0.7 pounds in the placebo group (5/11)

Ascend
Therapeutics
Inc.*

4-OHT

4-hydroxytamoxifen gel

Cyclic
breast pain

Phase II trial demonstrated improved signs and symptoms as well as reduced breast pain (5/11)

Bone Care
International
Inc.
(BCII)

LR-103

Vitamin D hormone

Secondary hyperparathyroidism

Starting a Phase I/II trial in patients with Stage V chronic kidney disease or end-stage renal disease (5/5)

Carrington
Laboratories
Inc.
(CARN)

GelVac

Powder intranasal delivery system for vaccines

Vaccine delivery

Phase I trial demonstrated safety and tolerability, and delivery to the nasal cavity (5/19)

Corgentech
Inc.
(CGTK)

E2F Decoy (edifoligide)

Oligonucleotide designed to inactivate the cell-cycle transcription factor E2F

Atopic dermatitis (eczema)

Began a Phase I/II trial to evaluate safety and the feasibility of repeated skin applications (5/24)

Depomed
Inc.
(DEPO)

Furosemide GR

Controlled-release formulation of the diuretic furosemide

Edema

Extended data from Phase II trial showed variable urinary urgency and frequency; development in the indication was stopped (5/13)

Discovery
Laboratories
Inc.
(DSCO)

Surfaxin

Engineered version of natural human lung surfactant

Respiratory distress syndrome in premature babies

Long-term data from Phase III trials showed survival benefits continued through one year of life (5/18)

Eyetech
Pharmaceuticals
Inc.
(EYET)

Macugen
(FDA-approved)

Pegaptanib sodium injection; pegylated anti-VEGF aptamer

Diabetic macular edema

Retrospective analysis of patients showed improvements in various measures of diabetic retinopathy (5/5)

Eyetech
Pharmaceuticals
Inc.
(EYET)

Macugen (FDA-approved)

Pegaptanib sodium injection; pegylated anti-VEGF aptamer

Wet age-related macular degeneration

No new safety concerns were identified in the second year of a trial (5/2)

Genaera
Corp.
(GENR)

Evizon (squalamine)

Anti-angiogenesis agent; vascular endothelial growth factor inhibitor

Wet age-related macular degeneration (5/3)

Phase II trial with photodynamic therapy in 46 subjects showed safety and improved visual acuity

Genaera
Corp.
(GENR)

Evizon (squalamine)

Anti-angiogenesis agent; vascular endothelial growth factor inhibitor

Wet age- related macular degeneration

Data from 18 patients in a Phase II trial showed improved visual acuity (5/2)

Genentech
Inc.
(NYSE:DNA)

Lucentis

Ranibizumab; humanized antibody fragment designed to bind and inhibit VEGF-A

Wet age- related macular degeneration

Phase III trial met its primary endpoint; 95% of patients maintained or improved vision at one year vs. 62% for placebo (5/23)

ICOS Corp.
(ICOS) and Eli
Lilly and Co.

Cialis (FDA-approved)

Tadalafil; oral PDE5 inhibitor

Erectile dysfunction

Data from open-label trial showed drug improved erections in more than 75% of men with ED and one or more co-morbid conditions (5/23)

InterMune
Inc.
(ITMN)

Actimmune (FDA-approved)

Interferon gamma-1b

Idiopathic pulmonary fibrosis

Data from 70 patients on the drug an average of 2.5 years showed relatively stable lung function (5/23)

Isotechnika
Inc.
(Canada;
TSE:ISA)

ISA247

Immunosuppressive agent

For use in kidney transplants

Starting a Phase IIb trial that will compare drug to tacrolimus in 332 newly transplanted kidney patients (5/12)

MediciNova
Inc.
(OSE:4875)

MN-001

Leukotriene receptor antagonist and inhibitor of of phosphodiesterases III and IV, 5-lipoxygenase and thromboxane A2

Interstitial cystitis

Began Phase II trial; details were not disclosed (5/24)

Microbia Inc.*

MD-1100

Superagonist of the guanylate cyclase-C receptor

Irritable bowel syndrome

Phase Ia trial in 30 healthy volunteers showed drug was safe and well tolerated, with no detectable systemic absorption (5/17)

Nabi Bio-
pharmaceuticals
(NABI)

PhosLo (FDA-approved)

Calcium acetate; phosphate binder

Stage IV kidney disease

Began EPICK study in 86 patients to evaluate efficacy in controlling elevated phosphorus levels (5/10)

Neurotech SA*
(France)

NT-501

Encapsulated cell technology used to deliver ciliary neurotrophic factor

Retinitis pigmentosa

Phase I trial demonstrated safety and tolerability, and showed improvements in visual acuity (5/1)

Regeneron
Pharmaceuticals
Inc.
(REGN)

VEGF Trap

Intravenous product designed to block vascular endothelial growth factor

Age-related macular degeneration

Phase I trial in 25 patients met its prespecified efficacy endpoint (5/3)

Sangamo
BioSciences
Inc.
(SGMO)

SB-509

Injectable formulation of plasmid DNA that encodes a zinc finger DNA-binding protein transcription factor designed to up-regulate the VEGF-A gene

Peripheral neuropathy

Began a Phase I trial to evaluate safety in diabetics with mild to moderate diabetic peripheral sensory motor neuropathy in the legs (5/5)

Sirna
Therapeutics
Inc.
(RNAI)

Sirna-027

Chemically modified short interfering RNA targeting vascular endothelial growth factor receptor-1

Age-related macular degeneration

Interim data from Phase I trial showed the agent to be safe and well tolerated at all doses tested (5/4)

Vivus Inc.
(VVUS)

MUSE (FDA-approved)

Alprostadil urethral suppository

Erectile dysfunction

Study in 91 patients who developed ED due to a bilateral nerve-sparing radical prostatectomy showed improved erections vs. placebo (5/23)

Vivus Inc.
(VVUS)

Avanafil

Oral phosphodiesterase type-5 inhibitor

Erectile dysfunction

Open-label trial in 13 subjects showed no accumulation of drug in plasma following twice-daily dosing (5/4)


Notes:

* Privately held.

ASCO = Data were presented at the American Society of Clinical Oncology meeting May 14-17.

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; CSE = Copenhagen Stock Exchange; LSE = London Stock Exchange; NYSE = New York Stock Exchange; OSE = Osaka Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; TSE = Toronto Stock Exchange.

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