Company (Location)

Product

Description

Indication

Status

Date

CANCER

Advaxis Inc. (Princeton, N.J.)

ADXS-HPV

Immunotherapy candidate

Human papillomavirus-positive head and neck cancer

Two patients have been enrolled into a Phase I/II study

5/9/12

Areva Med (Bethesda, Md.)

212Pb-TCMC-Trastuxumab

A monoclonal antibody carrying a radioisotope

Intra-abdominal cancers

Enrolling patients for the Phase I trial

5/4/12

AVEO Pharmaceuticals Inc. (Cambridge, Mass.)

AV-203

ErbB3-binding antibody

Advanced solid tumors

Started a Phase I study

5/24/12

Bionomics Ltd. (Adelaide, Australia)

BNC105

Vascular-disrupting agent

Ovarian cancer

Launched a Phase I/II trial

5/25/12

Bio-Path Holdings Inc. (Houston)

BP-100-1.01

Liposomal Grb-2

Blood cancers

Completed treatment of the third dosage cohort of three patients in its Phase I trial

5/30/12

Cancer Research UK (London)

Caprelsa

Vandetanib

Non-small-cell lung cancer

Opened the first trial testing the combination of Caprelsa and selumetinib

5/17/12

Celtic Pharmaceutical Holdings LP (New York)

Xerecept

Corticorelin acetate

Peritumoral brain edema

Completed a Phase I/II study in pediatric patients who are dependent on the steroid Decadron; results showed Xerecept can reduce or eliminate the need for Decadron use and provide significant quality of life improvements

5/23/12

Galectin Therapeutics Inc. (Newton, Mass.)

GM-CT-01

Carbohydrate-based galectin inhibitor

Advanced metastatic melanoma

The first patient was dosed in a Phase I/II trial

5/16/12

Marshall Edwards Inc. (San Diego)

ME-344

Mitochondrial inhibitor

Refractory solid tumors

First patients were dosed in a Phase I trial

5/30/12

MorphoSys AG (Martinsried, Germany) and Xencor Inc. (Monrovia, Calif.)

MOR208

An anti-CD19 antibody with a modification to the Fc portion

B-cell malignancies

Completed patient enrollment in a Phase I trial

5/23/12

Novelos Therapeutics Inc. (Madison, Wis.)

I-131-CLR1404

Cancer-targeted molecular radiotherapeutic compound

Advanced solid tumors

Completed the first cohort in its Phase Ib dose-escalation trial

5/16/12

Oncos Therapeutics Ltd. (Helsinki, Finland)

CGTG-102

An oncolytic adenovirus

Solid tumors

Started a Phase I study

5/30/12

CARDIOVASCULAR

AesRx LLC (Newton, Mass.)

Aes-103

Anti-sickling agent

Sickle cell disease

Started a Phase I/IIa trial

5/15/12

Alnylam Pharmaceuticals Inc. (Cambridge, Mass.)

ALN-TTR01

An RNAi therapeutic

TTR-mediated amyloidosis

Phase I data showed that use of the drug resulted in statistically significant reductions in serum TTR protein levels

5/11/12

Spectrum Pharmaceuticals Inc. (Henderson, Nev.)

RenaZorb

A lanthanum-based nanotechnology compound with phosphate-binding properties

Hyperphos-phatemia

Started patient enrollment in a Phase I study

5/30/12

CENTRAL NERVOUS SYSTEM

Alder BioPharmaceuticals Inc. (Bothell, Wash.)

ALD403

Targets the calcitonin gene-related peptide

Migraine

Started a Phase I study

5/8/12

Alkermes plc (Dublin, Ireland)

AKLS 5461

A combination of buprenorphine and opioid modulator ALKS 33

Major depressive disorder

Phase I/II data showed it significantly reduced depressive symptoms

5/31/12

Pharmanext AB (Stockholm, Sweden)

SHACT

Topical

For pain relief from intrauterine device insertion

First patient was dosed in a 15-patient, open-label Phase I study

5/1/12

Pozen Inc. (Chapel Hill, N.C.)

PA32540

A coordinated-delivery tablet containing immediate-release omeprazole layers around pH-sensitive aspirin

Pain

Phase I data showed it provided faster protection compared with delayed-release enteric-coated omeprazole as measured by mean time to gastric pH

5/22/12

Zalicus Inc. (Cambridge, Mass.)

Z160

Oral N-type calcium channel blocker

Neuropathic pain

Selected the most promising formulation of Z160 from a Phase I trial that showed it was well tolerated and demonstrated a sixfold improvement in Z160's bioavailability

5/3/12

DIABETES

MonoSol Rx LLC (Warren, N.J.) and Midatech Ltd. (Oxford, UK)

Midaform Insulin PharmFilm

Nanoformulated transbuccal insulin film product

Diabetes

Phase I data showed positive bioavailability and pharmacokinetic results in 27 healthy volunteers

5/4/12

Rhythm Pharmaceuticals Inc. (Boston)

RM-131

Its ghrelin agonist

Diabetic gastroparesis

Phase Ib data showed that it greatly accelerated gastric emptying, with emptying time reduced by 66%

5/23/12

INFECTION

Achillion Pharmaceuticals Inc. (New Haven, Conn.)

ACH-3102

A second-generation pan-genotypic NS5A inhibitor

Hepatitis C virus infection

Started dosing in a Phase I trial

5/10/12

Achillion Pharmaceuticals Inc. (New Haven, Conn.)

ACH-2684

A second-generation protease inhibitor

Chronic hepatitis C virus genotype 1

Phase Ib data showed it achieved a mean maximum 3.73 log10 reduction in HCV RNA after three-day 400-mg monotherapy with once-daily dosing

5/22/12

Bionor Pharma ASA (Oslo, Norway), Eurocine Vaccines AB (Solna, Sweden) and Oslo University Hospital

Vacc-4X and Endocine

Vaccine and adjuvant

Infection

A trial produced vaccine-related immune responses in a nasal immunization study in human patients

5/11/12

ChemoCentryx Inc. (Mountain View, Calif.)

CCX168

An oral C5aR inhibitor

Anti-neutrophil cytoplasmic antibody-associated renal vasculitis

Phase I data established a targeted dosing level, and showed the compound was well tolerated and caused no serious adverse events

5/30/12

GeoVax Labs Inc. (Atlanta)

HVTN 094

Second-generation vaccine that includes granulocyte-macrophage colony-stimulating factor as an adjuvant

HIV

Opened enrollment for a Phase I trial

5/23/12

Hemispherx Biopharma Inc. (Philadelphia)

Ampligen

Rintatolimod

Influenza

Ampligen was administered intranasally in conjunction with FluMist to healthy volunteers in a study at the University of Alabama

5/1/12

Inovio Pharmaceuticals Inc. (Blue Bell, Pa.)

SynCon

Avian influenza vaccine

Avian influenza

Generated protective hemagglutination inhibition titers against six different unmatched strains of H5N1 in a Phase I trial

5/17/12

Isconova AB (Uppsala, Sweden)

Matrix M

Vaccine adjuvant

Influenza

Phase I data showed it met both the primary and secondary endpoints when tested in a group of elderly subjects administered Vaxigrip

5/15/12

Mucosis BV (Groningen, the Netherlands)

FluGEM

Mimopath mucosal vaccines

Influenza

Phase I provided proof-of-concept data that the vaccines are safe and well tolerated, and able to produce balanced immune responses in circulating blood and the respiratory tract

5/11/12

NovaDigm Therapeutics Inc. (Grand Forks, N.D.)

NDV-3

A prophylactic vaccine containing recombinant AIs3

Candida albicans

Phase I data showed a single dose of NDV-3 was safe and well tolerated, with or without alum adjuvant, and induced strong antibody and T-cell immune responses

5/8/12

Novavax Inc. (Rockville, Md.)

VLP

Monovalent virus-like particle vaccine candidate

A/H5N1 avian influenza viruses

Started enrollment in a Phase I trial; began a second Phase I trial

5/3/12

Theraclone Sciences Inc. (Seattle)

TCN-032

A fully human monoclonal antibody

Influenza A infections

Phase I data showed it was well tolerated with no dose-limiting toxicities or serious adverse events

5/16/12

MISCELLANEOUS

Acucela Inc. (Seattle)

ACU-4429

Small-molecule visual cycle modulator

Advanced dry age-related macular degeneration/geographic atrophy

Interim Phase IIa data showed that ACU-4429 produced a dose-dependent response and modulated rod visual cycle for doses up to 10 mg

5/9/12

Catalyst Pharmaceutical Partners Inc. (Coral Gables, Fla.)

CPP-115

A GABA aminotransferase inhibitor and vigabatrin analogue

Infantile spasms

Phase Ia data showed it was well tolerated at all six doses administered

5/23/12

Medivir AB (Stockholm, Sweden)

MIV-711

A cathepsin K inhibitor

Bone disorders such as osteoporosis

Phase I trial has been initiated

5/30/12

Oxford BioMedica plc (Oxford, UK) and Sanofi SA (Paris)

RetinoStat

A gene-based treatment

Wet age-related macular degeneration

Phase I data showed that samples from six patients confirmed substantial increases in expression of endostatin and angiostatin proteins in the eye

5/9/12

Promethera Biosciences SA (Mont-Saint-Guibert, Belgium)

Promethera HepaStem

A cell-based therapy

Urea cycle disorders and Crigler-Najjar syndrome

Treated the first two patients in a Phase I/II trial

5/15/12

StemCells Inc. (Newark, Calif.)

HuCNS-SC

purified human neural stem cells

Spinal cord injury

Phase I/II data showed it was well tolerated

5/18/12

Summit Corp. plc (Oxford, UK)

SMT C1100

A small-molecule utrophin up-regulator

Duchenne's muscular dystrophy

Dosed the first cohort of patients in a Phase I study

5/29/12

Ultragenyx Pharmaceutical Inc. (Novato, Calif.)

UX001

Sialic acid

Hereditary inclusion body myopathy

Phase I data showed it was well tolerated

5/2/12

Versartis Inc. (Redwood City, Calif.)

VRS-317

A once-monthly form of recombinant human growth hormone

Growth hormone deficiency

Completed patient enrollment in a Phase I trial

5/3/12


Notes:

Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.

The date indicated refers to the BioWorld Today issue in which the news item can be found.