Company
(Location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Apitope International NV (Bristol, UK)

ATX-MS-1467

Designed to work with the immune system to treat the underlying cause of disease by restoring immunological balance

Relapsing multiple sclerosis

Completed the second Phase I trial, with MRI results showing a significant decrease in the number of contrast-enhancing brain lesions in patients treated by intradermal injection; the same effect was not seen in the subcutaneously dosed group

9/4/13

Xenoport Inc. (Santa Clara, Calif.)

XP23829

A fumaric acid ester compound

Relapsing-remitting multiple sclerosis

Phase I data showed both formulations tested produced monomethyl fumarate in the plasma but with different pharmacokinetic profiles, and the drug was generally well tolerated

9/11/13

CANCER

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-221

Oral, selective inhibitor of the mutated IDH2 protein

Advanced hematologic malignancies

Dosed the first patient in a Phase I study

9/24/13

Cerulean Pharma Inc. (Cambridge, Mass.)

CRLX101

Nanoparticle-based, camptothecin-loaded

Metastatic renal cell carcinoma

Phase Ib/IIa trial established the combinability of CRLX101 and Avastin; three of nine patients experienced partial responses

9/27/13

Constellation Pharmaceuticals Inc. (Cambridge, Mass.)

CPI-0610

A BET protein bromodomain inhibitor

Lymphomas

Started a Phase I trial

9/11/13

Cyclacel Pharmaceuticals Inc. (Berkeley Heights, N.J.)

Sapacitabine

Oral nucleoside analogue

Ovarian cancer

Data showed that CNDAC, the active metabolite of sapacitabine, was active against 75% of primary ovarian cancer samples isolated from patients

9/23/13

Debiopharm Group (Lausanne, Switzerland)

Debio1347

A selective FGFR 1, 2, 3 inhibitor

Advanced solid tumors

Started a Phase I trial

9/4/13

Gamida Cell Ltd. (Jerusalem)

NiCord

Umbilical cord blood unit

Blood cancers

First patient was successfully transplanted

9/10/13

Kiadis Pharma BV (Amsterdam, the Netherlands)

ATIR

Cell therapy

Hematopoietic stem cell transplants (non-matching) in leukemia patients

Phase I/II data showed an overall survival of 78% at one year and 67% at five years

9/11/13

Oncomed Pharmaceuticals Inc. (Redwood City, Calif.)

OMP-21M18

Demcizumab

Ovarian cancer

Started a Phase Ib/II trial

9/20/13

Oncomed Pharmaceuticals Inc. (Redwood City, Calif.)

Vantictumab

Monoclonal antibody

Advanced solid tumors

Phase I data showed it was well tolerated and had pharmacodynamics effects on the WNT pathway in patient samples

9/24/13

Oncothyreon Inc. (Seattle)

ONT-380

An oral, reversible and selective small-molecule HER2 inhibitor; also called ARRY-380

HER2-positive breast cancer

Started a Phase I study of ONT-380 in combination with Herceptin

9/4/13

To-BBB Technologies BV (Leiden, the Netherlands)

2B3-101

Brain-targeted doxorubicin liposomes

Brain cancer

Phase I portion of the Phase I/IIa study showed no signs of drug-related neurotoxicity or cardiotoxicity, but it showed signs of antitumor activity

9/27/13

Tracon Pharmaceuticals Inc. (San Diego)

TRC105

A monoclonal antibody to endoglin

Metastatic renal cell carcinoma

Started dosing in a Phase Ib clinical trial the combination of TRC105 and axitinib

9/24/13

Verastem Inc. (Cambridge, Mass.)

Defactinib

An inhibitor of focal adhesion kinase

Mesothelioma; advanced solid tumors

Phase I/Ib study of defactinib in combination with paclitaxel showed one complete response in a patient that was platinum-resistant; started a Phase I trial in advanced solid tumors

9/23/13; 9/25/13

CARDIOVASCULAR

Bioheart Inc. (Sunrise, Fla.)

Myocell

A muscle-derived cell therapy

Heart damage

Phase I data showed an average improvement in exercise capacity or a six-minute walk test of 47 meters

9/30/13

Sanbio Inc. (Mountain View, Calif.)

SB623

Allogeneic cell therapy candidate

Stroke

Completed enrollment in its Phase I/II trial

9/5/13

CENTRAL NERVOUS SYSTEM

Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.)

AVP-825

A device delivering sumatriptan powder intranasally

Acute migraine

The study in 20 healthy subjects found that it gave 27% higher peak exposure and 75% more systemic exposure in the first 15 minutes than liquid nasal spray

9/23/13

Biomarin Pharmaceutical Inc. (San Rafael, Calif.)

BMN 190

A recombinant human tripeptidyl peptidase 1

Batten disease

Dosed the first patient in the Phase I/II trial

9/24/13

GW Pharmaceuticals plc (London)

GWP42006

A nonpsychoactive cannabinoid

Epilepsy

Started a Phase I trial

9/19/13

Lightlake Therapeutics Inc. (London)

Naloxone

Intranasal

Drug abuse

Started a two-week trial of the intranasal version in 14 healthy volunteers

9/25/13

Nupathe Inc. (Conshohocken, Pa.)

Zecuity

Sumatriptan iontophoretic transdermal system

Migraines

Began dosing patients in a Phase I trial

9/24/13

Omeros Corp. (Seattle)

OMS824

Lead compound in its phosphodiesterase 10 program

Huntington’s disease and schizophrenia

Phase I data was positive and the compound will be advanced into Phase II programs

9/13/13

Rhythm Pharmaceuticals Inc. (Boston)

RM-493

Melanocortin 4 receptor agonist

Obesity

Started the first Phase Ib trial of a series of clinical trials

9/25/13

Zalicus Inc. (Cambridge, Mass.)

Z944

Oral T-type calcium channel blocker

Neuropathic pain

Initiated a Phase Ib study

9/10/13

INFECTION

Affinium Pharmaceuticals Inc. (Austin, Texas)

AFN-1720

A prodrug of its active antibiotic, AFN-1252

Acute bacterial skin and skin structure infections

Initiated dosing in a Phase I trial

9/6/13

Argos Therapeutics Inc. (Durham, N.C.)

AGS-004

Dendritic cell-based immunotherapy

HIV

Study confirmed antiviral cytotoxic T-cell immunity can be induced without generalized systemic immune activation, but AGS-004 did not induce changes in the proportion of CD4 and CD8 T-cell subsets

9/17/13

Imaxio SA (Lyon, France)

IMX313

Proimmunogenic technology

Tuberculosis

Administered for the first time in humans in a tuberculosis vaccine Phase I trial

9/4/13

ISA Pharmaceuticals BV (Leiden, the Netherlands)

ISA101

Synthetic long peptide vaccine

HIV/anal intraepithelial neoplasia

Started a Phase I/II study

9/17/13

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

NDV-3

Vaccine

Vulvovaginal candidiasis

Started a Phase Ib/IIa trial

9/27/13

Profectus Biosciences Inc. (Baltimore)

MAg-pDNA

Multi-antigen HIV plasmid DNA vaccine administered with Genevax interleukin-12 pDNA adjuvant

HIV

Started a Phase I trial

9/18/13

INFLAMMATORY

Cellular Biomedicine Group Inc. (Palo Alto, Calif.)

haMPC

Human adipose derived mesenchymal precursor cell therapy

Knee osteoarthritis

Phase I/IIa data showed improvement from baseline and no serious adverse events

9/9/13

RESPIRATORY

Celtaxsys Inc. (Atlanta)

CTX-4430

Small-molecule inhibitor of leukotriene A4 hydrolase

Cystic fibrosis

Completed the first two trials in its Phase I program

9/18/13

Kalobios Pharmaceuticals Inc. (South San Francisco)

KB001

A first-generation Pseudomonas aeruginosa Type III secretion system antibody

Cystic fibrosis

Phase I data showed a single intravenous dose had an acceptable safety profile and demonstrated a trend toward a short-term reduction in markers of inflammation, particularly neutrophil elastase

9/20/13

MISCELLANEOUS

Acorda Therapeutics Inc. (Ardsley, N.Y.)

AC105

Magnesium formulation

Spinal cord injury

Enrolled the first patient

9/26/13

Alnylam Pharmaceuticals Inc. (Cambridge, Mass.)

ALN-TTRsc

Subcutaneously delivered RNAi therapeutic that targets the transthyretin gene

TTR-mediated amyloidosis

Phase I data showed administration of the therapeutic led to robust, consistent and statistically significant knockdown of serum TTR protein levels of up to 94%

9/24/13

AM-Pharma (Bunnik, the Netherlands)

Bovine alkaline phosphatase

New recombinant human version of the anti-inflammatory enzyme

Acute kidney injury

Entered the clinic again with 50 healthy volunteers

9/23/13

Antares Pharma Inc. (Ewing, N.J.)

Testosterone enanthate

Administered weekly by subcutaneous injection via the Vibex Quickshot auto injector device

Testosterone deficiency

The first patients have been dosed in a clinical study

9/17/13

Ascendis Pharma A/S (Copenhagen)

ACP-001

A once-weekly human growth hormone prodrug

Growth hormone-deficiency

Started dosing in a trial that will compare three dose levels of weekly ACP-001 to daily growth hormone treatment

9/20/13

Bioaxone Biosciences Inc. (Cambridge, Mass.)

Cethrin

A biologic drug

Spinal cord

injury

Phase I/IIa data showed motor recovery scores

9/26/13

Eleven Biotherapeutics Inc. (Cambridge, Mass.)

EBI-005

Topically administered IL-1 protein

Dry eye disease

Phase Ib/IIa trial data showed it had activity against the signs and symptoms of the disease

9/23/13

Iceutica Inc. (Philadelphia)

ICE 1201

A submicron version of metaxalone; a muscle relaxant

Acute, painful musculoskeletal conditions

Phase I data showed improved oral bioavailability, decreased pharmacokinetic variability, more rapid absorption and decreased food effect, when compared to Skelaxin

9/13/13

Isis Pharmaceuticals Inc. (Carlsbad, Calif.)

ISIS-SMNRx

Designed to work by altering the splicing of closely related gene SMN2 to increase production of fully functional SMN protein

Spinal muscular atrophy

Phase I data showed most SMA children receiving the two highest doses (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection

9/20/13

Prosensa Holding NV (Leiden, the Netherlands)

PRO053

Exon-skipping compound

Duchenne’s muscular dystrophy

Dosed the first patient in its Phase I/II trial

9/9/13

Stemcells Inc. (Newark, Calif.)

HuCNS-SC cells

Purified human neural stem cells

Dry age-related macular degeneration

The first high-dose patient was dosed in the Phase I/II trial

9/13/13

Synageva Biopharma Corp. (Lexington, Mass.)

SBC-102

Sebelipase alfa

Lysosomal acid lipase deficiency

Phase I/II data showed reductions in liver damage and improvement in the dyslipidemia associated with LAL deficiency

9/5/13

Ultragenyx Pharmaceutical Inc. (Novato, Calif.)

UX007

Triheptanoin

Long-chain fatty acid oxidation disorders

Data showed that treatment appeared to reduce the frequency and severity of hospitalizations but did not completely eliminate them

9/17/13


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.