Company (Location)

Product

Description

Indication

Status (Date)#

Adaptimmune Ltd. (Oxford, UK)

T cell therapy

Uses engineered, increased affinity T cell receptor proteins to strengthen natural patient T cell responses

Multiple myeloma

Opened a Phase I/II trial that will enroll 12 patients over the next two years who have received treatment and are eligible for an autologous stem cell transplant (5/27)

Athersys Inc. (Cleveland)

MultiStem

Cell therapy product

Leukemia

Phase I data in patient undergoing allogeneic hematopoietic stem cell transplants showed it was well tolerated at all dose levels (5/26)

BioInvent International AB (Lund, Sweden), ThromboGenics NV (Leuven, Belgium) and Roche AG (Basel, Switzerland)

TB-403

Antibody; RG7334

Glioblastoma multiforme

Dosed the first patient in a Phase Ib/II study (5/17)

Boehringer Ingelheim Pharmaceuticals Inc. (Ridgefield, Conn.)

BIBW 2992

Afatinib

Non-small-cell lung cancer

Phase Ib data showed that combining afatinib with cetuximab at the maximum tested dose controlled disease in all treated epidermal growth factor receptor-mutation positive patients (5/26)

Can-Fite BioPharma Ltd. (Petah-Tikva, Israel)

CF102

Inhibits NS5 RNA polymerase and induces apoptosis

Liver cancer and Child-Pugh cirrhosis

Phase I/II interim analysis showed a favorable safety profile (5/12)

Exelixis Inc. (South San Francisco)

Cabozant-inib

A dual MET/VEGFR2

Advanced solid tumors in lymphoma

Phase I data showed that of the 35 patients with measurable medullary thyroid cancer, 10 achieved partial responses (5/25)

ImmunoCellular Therapeutics Ltd. (Los Angeles)

ICT-107

A dendritic cell-based cancer vaccine

Glioblastoma multiforme

Phase I data showed that out of 16 patients, six had no tumor recurrence and three have been disease-free for four years; three others have been disease-free for 2.5 years (5/6)

Inovio Pharmaceuticals Inc. (Blue Bell, Pa.)

VGX-3100

A DNA vaccine delivered by Cellectra electroporation

Cervical dysplasia

Showed significant T-cell response during four months of treatment in a Phase I trial in 18 adult females (5/10)

Oxigene Inc. (South San Francisco)

OXi4503

Dual-mechanism, second-generation vascular disrupting agent

Acute myelogenous leukemia or myelodysplastic syndrome

Started a Phase I study (5/13)

Peregrine Pharmaceuticals Inc. (Tustin, Calif.)

Bavituximab

Monoclonal antibody

Castration-resistant prostate cancer

Began a Phase I/II trial (5/26)

Juventas Therapeutics Inc. (Cleveland)

JVS-100

Designed to encode stromal cell-derived factor-1

Class III heart failure

Phase I data showed that it met its safety endpoints and was tolerated at all doses (5/25)

Nile Therapeutics Inc. (San Mateo, Calif.)

Cenderitide

Natriuretic peptide

Heart failure and renal disease

Began dosing in a Phase I trial (5/4)

PolyMedix Inc. (Radnor, Pa.)

PMX-60056

A synthetic small molecule

To reverse heparin in patients undergoing percutaneous coronary intervention procedures

Phase Ib/II data suggested that both heparin and tinzaparin appear to bind to a one-to-one molecular basis (5/4)

Santaris Pharma AS (Hoersholm, Denmark)

SPC5001; and SPC4955

A mRNA-targeted drug; and a locked nucleic acid compound targeted at apolipoprotein B via mRNA

High cholesterol

Advanced it into Phase I trials (5/5); advanced SPC4955 into Phase I (5/12)

Selexys Pharmaceuticals (Oklahoma City)

SelG1

A humanized anti-P-selectin antibody

Vasoocclusive crisis

Started enrollment in a Phase I study (5/5)

Anavex Life Sciences Corp. (Hoboken, N.J.)

ANAVEX 2-73

An oral disease-modifying drug

Alzheimer's disease

The safety review committee unanimously approved skipping the 5-mg dose and moving directly to the 10-mg dose cohort (5/24)

KemPharm Inc. (North Liberty, Iowa)

KP201

Opioid-based drug candidate

Pain

Phase I data showed it is cleaved in man as predicted, releasing the active pharmaceutical compound, hydrocodone, into the bloodstream at amounts equivalent to the reference listed drug (5/25)

Oxford BioMedica plc (Oxford, UK)

ProSavin

Gene therapy

Parkinson's disease

Phase I/II data showed that patients in the third cohort at six months had an average motor function improvement of 43%, with a maximum of 61% in one patient (5/24)

Repligen Corp. (Waltham, Mass.)

RG3039

A potential treatment for spinal muscular atrophy

Spinal muscular atrophy

Received FDA approval to begin a Phase I trial (5/20)

Achillion Pharmaceuticals Inc. (New Haven, Conn.)

ACH-2684

A pan-genotypic protease inhibitor

Chronic hepatitis C virus infection

Began dosing in a Phase I trial (5/26)

Affinium Pharmaceuticals Inc. (Austin, Texas)

AFN-1252

Selective-spectrum, anti-staphylococcal agent

Serious staphylococcal infections

Phase I data showed that various doses met or exceeded exposures necessary for treatment (5/3)

Atox Bio Inc. (Ness Ziona, Israel)

AB103

An immunomodulator

Severe bacterial infections and sepsis

Successfully completed a Phase I study (5/13)

AVI BioPharma Inc. (Bothell, Wash.)

AVI-6002 and AVI-6003

Lead drug candidates

Ebola virus and Marburg virus

Started dosing in two Phase I studies (5/5)

Intercell AG (Vienna, Austria)

VEP

Vaccine enhancement patch

Influenza

Started a trial investigating the patch containing LT in combination with GlaxoSmithKline plc's H5N1 pandemic antigen (5/5)

Lytix Biopharma A/S (Oslo, Norway)

Lytixar

LYT-109

Methicillin-resistant Staphylococcus aureus and Gram-positive skin infections

Completed two Phase I/IIa studies showing it to be tolerated and safe, with negligible systemic uptake and demonstrating proof of concept (5/27)

Mymetics Corp. (Epalinges, Switzerland)

MYM-V101

Vaccine

HIV

Vaccine was well tolerated when administered intramuscularly and intranasally in a Phase I trial (5/27)

Profectus BioSciences Inc. (Baltimore)

Vaccine

Multi-antigen HIV plasmid DNA vaccine administered with GENEVAX IL-12 pDNA adjuvant

HIV

Immunized the first subject in a U.S. Phase I trial (5/26)

Protea Biosciences Inc. (Morgantown, W. Va.)

MS1819

Recombinant lipase product

Exocrine pancreatic insufficiency in chronic pancreatitis

Completed a Phase I/IIa trial (5/27)

Sinovac Biotech Ltd. (Beijing)

Vaccine

Vaccine against human enterovirus 71

Enterovirus 71 , which causes hand, foot and mouth disease

Showed favorable results for safety and tolerance for adult, child and infant age groups (5/26)

Aladagen Inc. (Durham, N.C.)

ALD-301

A stem cell therapy

Critical limb ischemia

Phase I/II data showed patients had significant improvements in three efficacy endpoints compared to baseline at 12 weeks (5/20)

Ambrx Inc. (San Diego)

ARX201

A growth hormone

Hormone deficiency

Phase I/II data demonstrated safety, efficacy and dosing frequency of ARX201 compared to native hormone therapy (5/26)

Amira Pharmaceuticals Inc. (San Diego)

AM152

An oral selective antagonist of the LPA1 receptor

Systemic

sclerosis

Phase I data was positive (5/3)

Esperion Therapeutics Inc. (Plymouth, Mich.)

ETC-1002

A small-molecule metabolic regulator of imbalances in lipid and carbohydrate metabolism

Mild dyslipidemia

Phase I data showed it is well tolerated and significantly reduces LDL-C levels (5/3)

Promedior Inc. (Malvern, Pa.)

PRM-151

Recombinant human pentraxin-2

Idiopathic pulmonary fibrosis

Phase I data showed it was safe and well tolerated across all study participants and showed activity against efficacy biomarkers (5/17)

QLT Inc. (Vancouver, British Columbia)

QLT091001

Oral, synthetic retinoid

Leber congenital amaurosis

Phase Ib data showed improvements in visual acuity and visual fields in eight out of 12 subjects (5/4)

Xoma Ltd. (Berkeley, Calif.)

XOMA 3AB

Antibody for botulism

Botulism poisoning

The National Institute of Allergy and Infectious Diseases is beginning a Phase I trial (5/4)

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.