Company |
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. |