Company* (Country; Symbol)




Status (Date)


Genelabs Technologies Inc. (GNLB)


Prasterone; GL701; the synthetic equivalent of dehydroepiandrosterone

Systemic lupus erythematosus

Clinical data showed Prestara improved or stabilized overall SLE disease activity and symptoms without deterioration (4/3)

Ester Neurosciences* (Israel)


A lead compound in Ester's disease-modifying platform technology for the pre-expression control of a specific variant of the AChE protein

Myasthenia gravis

Phase Ib data showed it was an effective and safe therapy (4/1)

Human Genome Sciences Inc. (HGSI)


A human monoclonal antibody to B-lymphocytestimulator, BlyS

Systemic lupus erythematosus

Phase I data demonstrated that LymphoStat-B was well tolerated and biologically active (4/21)

SangStat Medical Corp. (SANG)


Anti-inflammatory peptide

Crohn's disease

Phase II data showed that only 66% of patients treated with 200 mg a day achieved response (4/9)

Industries Ltd.

(Israel; TEVA)

Copaxone (FDA-approved)

Glatiramer acetate injection

Multiple sclerosis

Clinical data showed Copaxone doubled the time relapsing-remitting multiple sclerosis patients took to accumulate disability (4/8)


Active Biotech AB (Sweden; SSE:ACTI)


Tumor Targeted Super-antigens

Non-small-cell lung cancer

Clinical results show positive survival results with a median survival time of 8.1 months (4/23)

Allos Therapeutics Inc. (ALTH)


Efaproxiral; a synthetic allosteric modifier of hemoglobin

Brain metastases

Phase III data showed it failed to meet a statistical endpoint, but it did demonstrate a survival trend in breast cancer patients (4/23)

Antigenics Inc. (AGEN)


A personalized heat-shock protein 70

Chronic myelogenous leukemia

Company began a Phase II trial testing it with Novartis AG's Gleevec (4/9)

Applied Genetics Inc.*


T4N5 liposome lotion; an approach to preventing skin cancer by repairing DNA damage

To prevent skin cancer

Clinical data showed dimericine reduces suppression of the immune system caused by solar ultraviolet rays (4/28)

CancerVax Corp.*


Therapeutic cancer vaccine; allogeneic, whole-cell vaccine that expresses more than 20 antigens


Company resumed enrollment in a Phase III trial after the FDA halted it a year ago due to production questions (4/15)

Cell Genesys Inc. (CEGE)


An oncolytic virus therapy

Prostate cancer

Company initiated a Phase I/II trial (4/22)

Cell Genesys Inc. (CEGE)


Lung cancer vaccine

Non-small-cell lung cancer

Company began enrollment in a Phase II trial (4/1)

Favrille Inc.*


Vaccine that stimulates the body's immune response against the tumor

Non-Hodgkin's lymphoma

Company expanded the Phase II trial of Favid in combination with Rituxan to include patients who have not received prior treatment for low-grade or follicular non-Hodgkin's lymphoma (4/24)

Geron Corp.

Telomerase immunotherapy

Antigen for cancer prostate cancer


Phase I data showed it was well tolerated and resulted in the generation of an anti-telomerase immune response (4/7)

Human Genome Sciences Inc. (HGSI)


Keratinocyte growth factor-2, KGF-2

Cancer therapy-induced mucositis

Phase II data showed it was well tolerated and had efficacy (4/2)

Isis Pharmaceuticals Inc. (ISIS)


Inhibitor of protein kinase C-alpha expression

Non-small-cell lung cancer

Phase III data showed it did not meet its primary endpoint (4/2)

Lorus Therapeutics Inc. (OTC BB:LORFF)


Immunotherapeutic drug that stimulates macrophages, monocytes and natural killer cells to attack tumor cells

Advanced pancreatic cancer

A pivotal Phase III trial is being expanded to include about 50 sites in North America and Latin America (4/16)

MethylGene Inc.* (Canada)


A second-generation antisense compound designed to inhibit the expression of DNA methyltransferase, an enzyme implicated in tumor growth


Phase I data showed that MG98 was well tolerated, produced evidence of objective tumor response or disease stabilization in cancer patients and has predictable human pharmacokinetics (4/3)

NeoPharm Inc. (NEOL)


Tumor-targeting agent; a recombinant protein designed to detect and bind to IL-13 receptors, then deliver PE38, a bacterial cytotoxic agent, to destroy tumor cells

Malignant glioma

Phase I/II data showed evidence of cytotoxic effects against malignant glioma tumor cells (4/30)

OncoGenex Technologies Inc.* (Canada)


An antisense drug designed to inhibit clusterin and therefore help the effectiveness of anticancer agents

Solid tumors

Company initiated a second Phase I trial (4/7)



Vaccine consisting of three inactivated cell lines that target a range of prostate cancer-associated antigens

Prostate cancer

Company completed patient recruitment in a Phase IIa trial (4/23**)

Salmedix Inc.*


L-alanosine; anticancer agent

Non-small-cell lung cancer, mesothelioma, pancreatic cancer, osteosarcoma and soft tissue sarcoma

Company began a Phase II trial (4/2)

SuperGen Inc. (SUPG)


Pentostatin for injection

Chronic lymphocytic leukemia

Phase II data showed an overall response rate of 74% (4/1)


Xcellerated T Cells technology

T cells produced using Xcyte's Xcellerate

Multiple myeloma

Company began a Phase I/II trial (4/3)


Avant Immunotherapeutics Inc. (AVAN)


Complement inhibitor

Patients under-going adult cardiac surgery

Phase II data showed a treatment benefit to male patients, but no significant benefit to females (4/29)

Theratechnologies Inc. (Canada; TSE:TH)


A growth hormone-releasing factor analogue

A catabolic state secondary to chronic obstructive pulmonary disease

Company completed enrollment in a Phase II trial (4/23)

United Therapeutics Corp. (UTHR)

Remodulin (FDA-approved)

Treprostinil sodium injection

Severe pulmonary hypertension

Clinical data showed long-term therapy improves New York Heart Association function class and exercise tolerance and improves long-term survival (4/14)


Ascend Therapeutics Inc.*


4-hydroxytamoxifen gel

Cyclical breast pain

Company began a Phase II trial (4/30)

Celltech Pharmaceuticals Inc. (arm of Celltech Group plc; UK; NYSE:CLL)

Metadate CD (FDA-approved)

Methylphenidate HCl extended-release capsules, 20 mg

Attention deficit hyper-activity disorder

Clinical results showed a comparable treatment effect to Ritalin 10 mg given twice daily (4/22)

Forest Laboratories Inc. (NYSE:FRX)


A moderate-affinity, uncompetitive N-methyl D-aspartate receptor antagonist that modulates glutamate levels

Alzheimer's disease

Phase III data showed that memantine enhanced cognitive function when treated in combination with Aricept (4/4)

Neurocrine Biosciences Inc. (NBIX)


Capsules; acts on the GABA-A receptor


Phase III data showed an immediate-release formulation of the drug achieved statistically significant results in primary and secondary endpoints of sleep initiation over about a month (4/22)

Pain Therapeutics Inc. (PTIE)


Next-generation version of oxycodone, a painkiller

Severe osteoarthritic pain

Company completed patient enrollment in a Phase II trial (4/22)

Pharmos Corp. (PARS)


Non-psychotropic, synthetic analogue of marijuana

Post-surgical cognitive impairment

Company began dosing in a Phase IIa study (4/8)

Titan Pharmaceuticals Inc. (AMEX:TTP)


Cell therapy in development that uses normal human cells attached to microcarriers and is designed to enhance brain levels of dopamine

Parkinson's disease

Clinical data showed Spheramine produced on average 41% improvement in motor function (4/2)

Vernalis Group plc (UK; LSE:VER)

Frovatriptan (FDA-approved)

5-HT 1B/D agonist

Menstrually associated migraines

Phase IIIb data showed that it was effective in preventing migraines when administered for six days during the perimenstrual period (4/2)


Regeneron Pharmaceuticals Inc. (REGN)


Genetically reengineered version of a naturally occurring human protein known as ciliary neurotrophic factor

Obesity in diabetics

Phase II data showed statistically significant weight loss in obese people with diabetes (4/14)


Chiron Corp. (CHIR)


A conjugate vaccine against Neisseria meningitidis sero group C

To prevent meningococcal C disease

Company began a Phase III trial (4/16)

Gilead Sciences Inc. (GILD)

Hepsera (FDA-approved)

Adefovir dipivoxil 10 mg

Hepatitis B

Clinical data showed more than 70% of patients treated with Hepsera showed persistent suppression of HBV DNA viral replication, continued histological improvements and sustained improvements in liver function through 96 weeks (4/10)

Hemispherx Biopharma Inc. (AMEX:HEB)


Immunotherapeutic; double-stranded synthetic RNA compound poly 1; poly C124


Phase IIb data showed that patients taken off highly active anti-retroviral therapy but given Ampligen continued to show virus levels below 5,000 copies/ml for a mean time of 25 weeks and counting, while the control group had an HIV rebound with a mean of 13 weeks (4/29)

Immtech International Inc. (OTC BB:IMMT)


Oral anti-infective; dicationic pharmaceutical compound

AIDS patients with pneumocystis carinii pneumonia

Company completed a pilot Phase IIa trial that showed improved lung function in 90% of patients (4/10)

Inhibitex Inc.*


Humanized monoclonal antibody

Staphylococcus aureus infections

Company will begin a Phase I trial (4/30)

InSite Vision Inc. (AMEX:ISV)


Combines InSite's sustained-release DuraSite technology with a fourth-generation fluoroquinolone

Ocular bacterial infections

Company began a Phase I trial (4/30)

SciClone Pharmaceuticals Inc. (SCLN)


Thymalfasin, thymosin alpha 1

Hepatitis B

Clinical results showed a 71% long-term sustained response rate achieved after one year of combination therapy with Zadaxin and interferon alpha (4/9)

Trimeris Inc. Hoffmann-La Roche Inc.


Enfuvirtide; HIV fusion inhibitor


Phase III data showed responses (TRMS) and seen at 24 weeks were maintained at 48 weeks (4/28)




Small-molecule opioid receptor antagonist

Post-operative ileus

Phase III data showed it hit its primary endpoint (4/2)

Biovitrum AB* (Sweden)


A 5-HT2C receptor agonist

To suppress appetite

Company started a Phase IIb study (4/7)

CollaGenex Pharmaceuticals Inc. (CGPI)

Periostat (FDA-approved)

Doxycycline hyclate tablets, 20 mg

Moderate facial acne

Phase II data showed that Periostat significantly reduced the number of inflammatory and non-inflammatory lesions, was well tolerated, had no detectable antimicrobial effect on the skin flora and did not increase resistant organisms or overgrowth of opportunistic pathogens (4/22)

Connetics Corp. (CNCT)


New drug formulation of 2% ketoconazole in its foam delivery system

Seborrheic dermatitis

Phase III data showed a 50% patient response for Extina, a 44% response for Nizoral, a 40% response for placebo foam and a 26% response for placebo cream (4/24)

Copernicus Therapeutics Inc.*


A nonviral gene transfer system that tightly binds strands of DNA to pass through a cell membrane and into the nucleus

Cystic fibrosis

Phase I data showed positive results without significant side effects and a well-tolerated treatment (4/29)

Cytos Biotechnology AG* (Switzerland)



Smoking addiction

Company began a Phase I trial (4/23)

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


A PDE5 inhibitor

Erectile dysfunction

Clinical data showed 79% of U.S. men of diverse ethnic origin reported improved erections after treatment with Cialis, compared to 19% for placebo (4/28)

Immusol Inc.*


Inhibits the growth of cells taken from multiple keloid biopsies as compared to the same keloidal cells treated with a control

Keloids and hypertrophic scars

The first patient was treated in a Phase I trial (4/15)

Neurochem Inc. (Canada; TSE:NRM)


Anti-amyloid drug

Secondary AA amyloidosis

The company received a unanimous recommendation to con- tinue its pivotal Phase II/III trial from an independent Data Safety Monitoring Board (4/10)

NicOx SA (France; Nouveau Marche:NICOX)


Nitric oxide-donating derivative of budesonide


Company began a Phase I trial (4/1)

Pain Therapeutics Inc. (PTIE)


Opioid antagonist

Irritable bowel syndrome

Company completed enrollment in a pilot study (4/29)

Palatin Technologies Inc. (AMEX:PTN)


Peptide analogue of (alpha)- MSH ([alpha]-melanocyte- stimulating hormone)

Erectile dysfunction

Phase IIa data showed a statistically significant improvement in erectile function across a range of patients with no significant adverse effects (4/28)

Progenics Pharmaceuticals Inc. (PGNX)


Designed to reverse the side effects of opioids without interfering with pain relief


Phase II data showed 60% of patients who received at least a 5 mg mg dose laxated within four hours as compared to less than 8% at lower doses (4/30)

RegeneRx Bio-pharmaceuticals Inc. (OTC BB:RGRX)

Thymosin beta 4

A naturally occurring 43-amino-acid peptide

Chronic dermal wounds

Company enrolled the first patients in clinical trials (4/18)

SangStat Medical Corp. (SANG)


Anti-inflammatory peptide designed to inhibit the synthesis of TNF-alpha, gamma interferon and interleukin-12

Ulcerative colitis

Phase II data showed a peak response rate of 77% and a 71% remission rate - both statistically significant (4/9)

Sucampo Pharmaceuticals


A chloride channel activator

Constipation- predominant irritable bowel syndrome

Company began a clinical study (4/28)


* Privately held

** Denotes the date the item ran in BioWorld International.

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