Company*
(Country; Symbol)

Product

Description

Indication

Status (Date)


AUTOIMMUNE

Acorda
Therapeutics
Inc.*

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
Inc.
(BIIB) and Fumapharm AG* (Switzerland)

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.
(CHIR) and Berlex

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.
(RGEN)

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
(Switzerland; NYSE:SRA)

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
Response Corp.
(IMNR)

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.
(Canada; TSE:AOM)

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
(UK; LSE:ASM) and Hoffmann- La Roche Inc.

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
Inc.
(AVII)

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.
(Canada; BIOM) and Merck KGaA (Germany)

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.
(Canada; BIOM) and Merck KGaA (Germany)

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.
(CELG)

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
Inc.
(CTIC)

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
Inc.
(CTIC)

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
BioSciences
(subsidiary of Theratechnologies Inc.; Canada; TSE:TH)

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.
(CRGN)

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.
(ENMD)

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
(Denmark; CSE: GEN)

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
Therapeutics
Inc. (INGN)

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
Therapeutics
Inc. (INGN)

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
Edwards Inc.
(MSHL)

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

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
BioMedica plc
(UK; LSE:OXB)

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
Pharmaceuticals
Inc. (REGN)

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
Pharmaceuticals
Inc. (SPPI)

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
(France; TRGNY)

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
Inc.
(XCYT)

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
Inc.
(ZGEN)

--

Recombinant human interleukin-21

Cancers

Got FDA OK for Phase I trial testing safety and pharmacokinetics (4/5)

CARDIOVASCULAR

Acorda
Therapeutics
Inc.*

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
Pharmaceuticals
Inc. (ALXN)

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.
(AMEX:ALT)

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
Inc.
(CVTX)

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
Inc.
(CVTX)

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.
(OTC BB:EVSC)

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*
(Germany)

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.
(Canada; TSE:MPH)

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
Inc.
(Canada; TSE:TH)

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
Inc.
(ZGEN)

--

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
Sciences Inc.
(BSLI)

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.
(CEPH)

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
Pharmaceuticals
Inc.*

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
Inc.
(MYGN)

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
Technologies
Inc. (NTII)

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.
(Canada;

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
Biosciences
Inc. (NBIX)

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
(UK; LSE:PYM)

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
Biotechnology
Ltd. (Australia; PRAN)

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
Pharmaceuticals
Inc.*

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.
(ADGM)

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.
(Canada; TSE:CJC)

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
(UK; ACAM)

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
Biotherapy
Inc. (OTC BB:ADVB)

--

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
Pharmaceuticals
Inc. (AMEX:ANX)

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-
therapeutics
Inc. (AVAN)

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
Inc.
(GILD)

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
Pharmaceuticals
Inc.*

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
Sciences Corp.*

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.
(VXGN)

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
Pharmaceuticals
Inc. (VRTX)

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
Magnetics Inc.
(AMEX; AVM)

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
Laboratories
Inc. (Canada; AELA)

Cetrorelix

Luteinizing hormone-
releasing hormone antagonist

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
Laboratories
Inc. (Canada; AELA)

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.
(AMGN)

Sensipar (FDA-approved)

Cinacalcet HCl; oral calcium calcimimetic

Secondary hyperpara-
thyroidism

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
Therapeutics
Inc.*

ATI-7505

Analogue of cisapride, a product removed from the market in 2000 due to safety problems

Gastro-
esophageal reflux disorder and gastroparesis

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.
(CRXA)

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.
(GENR)

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.
(GNVC)

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
Pharmaceutical

Co. Inc.
(INKP)

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
Pharmaceuticals
Inc.
(ISPH)

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
Pharmaceuticals
Inc.
(ISPH)

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
orp.
(MCHM)

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-
pharmaceuticals
Inc. (NABI)

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
Inc.
(Canada; NRMX)

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
Pharmaceuticals
Inc.
(NPSP)

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
Pharmaceuticals Inc.
(RIGL)

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.
(VVUS)

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)


Notes:

* Privately held

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