Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Cytos Biotechnology AG (Schlieren, Switzerland) |
CYT003 |
Therapeutic vaccine |
Allergic asthma |
Failed in a phase IIb trial |
4/15/14 |
Galapagos NV (Mechelen, Belgium) and Glaxosmithkline plc (London) |
GSK2586184 |
A selective JAK1 inhibitor |
Psoriasis |
GSK2586184 met the primary endpoint in phase IIa psoriasis study, with patients given the drug achieving a 75% or greater improvement from baseline in their Psoriasis Area Severity Index scores at week 12 |
4/18/14 |
Gene Signal International SA (Lausanne, Switzerland) |
GS-101 |
Aganirsen; an antisense oligonucloetide |
Psoriasis |
A phase IIa study demonstrated that topical application of GS-101 reduced the size of psoriatic lesions and inflammation compared to placebo |
4/11/14 |
Geneuro SA (Geneva) |
GNbAC1 |
Monoclonal antibody that targets the MSRV-Env protein |
Multiple sclerosis |
The one-year open-label extension of its phase IIa study in 10 patients was completed, with results showing that the treatment was well tolerated over the long term and that pharmacodynamic responses could be observed |
4/18/14 |
Phosphagenics Ltd. (Melbourne, Australia) |
Tretinoin |
Formulated with Phosphagenics' transdermal delivery technology TPM |
Acne |
Completed enrollment in a phase II trial |
4/24/14 |
Synthetic Biologics Inc. (Rockville, Md.) |
Trimesta |
Oral estriol; oral, once-daily treatment |
Relapsing-remitting multiple sclerosis |
Topline data from the phase II trial demonstrated that Trimesta given with first-line RRMS therapy Copaxone (glatiramer acetate injection) resulted in reduced annualized relapse rate and improved cognitive function at 12 months of treatment as compared to placebo plus Copaxone |
4/30/14 |
CANCER | |||||
Ambit Biosciences Corp. (San Diego) |
Quizartinib |
Selective FLT3 inhibitor |
FLT3-ITD positive acute myeloid leukemia |
Started a phase II cohort of a phase I/II study of quizartinib in combination with either 5-azacitidine or low-dose cytarabine |
4/8/14 |
Dendreon Corp. (Seattle) |
Provenge |
Sipuleucel-T |
Biochemically recurrent prostate cancer |
Phase II STAND data suggested tumor-specific T-cell responses appear to be enhanced and sustained when Provenge is given following androgen deprivation therapy |
4/14/14 |
Halozyme Therapeutics Inc. (San Diego) |
PEGPH20 |
Pegylated recombinant human hyaluronidase |
Pancreatic cancer |
Halted its phase II trial following an unexpected imbalance in thromboembolic event rate between treatment and control groups; FDA placed a clinical hold on patient enrollment and dosing |
4/7/14; 4/10/14 |
Oncosec Medical Inc. (San Diego) |
Immunopulse |
DNA IL-12 DNA-based immunotherapy |
Cutaneous T-cell lymphoma |
Company is relaunching a phase II trial |
4/8/14 |
Progenics Pharmaceuticals Inc. (Tarrytown, N.Y.) |
PSMA ADC |
Comprises a fully human monoclonal antibody selectively targeting prostate-specific membrane antigen linked to a chemotherapeutic drug |
Prostate cancer |
Completed enrollment in the chemotherapy-naïve cohort in its phase II trial of PSMA ADC; that cohort of 36 patients, all of whom progressed on hormonal therapies, was added to the phase II study following positive response to PSMA ADC in patients in the chemotherapy-experienced setting |
4/25/14 |
Puma Biotechnology Inc. (Los Angeles) |
PB272 |
Neratinib; a pan-HER tyrosine kinase inhibitor |
Metastatic breast cancer |
Phase II data showed that the neratinib-containing regimen graduated, based on a high probability of success in phase III with a signature of HER2-positive/HR-negative; neratinib achieved an estimated pCR rate of 55.6% compared to the control arm, which had an estimated pCR rate of 32.6% |
4/9/14 |
Spectrum Pharmaceuticals Inc. (Henderson, Nev.) |
Melphalan |
Captisol-enabled propylene glycol-free |
Multiple myeloma |
Met the primary endpoints in a pivotal phase II trial |
4/24/14 |
Ventirx Pharmaceuticals Inc. (Seattle) |
VTX-2337 |
A small molecule that targets Toll-like receptor 8 |
Recurrent or persistent epithelial ovarian, fallopian tube or primary peritoneal cancer |
Completed enrollment in GOG-3003 (NCT01666444), a randomized, placebo-controlled phase II trial of VTX-2337 in combination with pegylated liposomal doxorubicin |
4/18/14 |
CARDIOVASCULAR | |||||
Arca Biopharma Inc. (Westminster, Colo.) |
Gencaro |
Bucindolol hydrochloride |
To prevent atrial fibrillation in patients with heart failure or left ventricular dysfunction |
Genetically screened the first patient in its phase IIb/III trial |
4/18/14 |
Glycomimetics Inc. (Gaithersburg, Md.) |
GMI-1070 |
Rivipansel |
Vaso-occlusive crisis in people with sickle cell disease |
Phase II data showed it improved efficacy outcomes independent of hydroxyurea |
4/15/14 |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) |
ISIS-APOCIIIRx |
An antisense drug |
High triglycerides |
Phase II data showed patients treated achieved average reductions of as much as 71% in apolipoprotein C-III a component of very low density lipoprotein, up to 64% in triglycerides, and average increases of up to 52% in high-density lipoprotein cholesterol |
4/1/14 |
CENTRAL NERVOUS SYSTEM | |||||
Alder Biopharmaceuticals Inc. (Bothell, Wash.) |
ALD403 |
Genetically engineered monoclonal antibody that targets calcitonin gene-related peptide |
To prevent migraine |
A randomized, double-blind, placebo-controlled proof-of-concept trial showed it met the primary endpoint of the study by significantly reducing mean migraine days per month vs. placebo during weeks five to eight |
4/24/14 |
Ampio Pharmaceuticals Inc. (Greenwood Village, Colo.) |
Ampion |
Single intra-articular injection |
Inflammation-associated pain in symptomatic osteoarthritis of the knee |
In a subgroup of patients with moderate to severe OAK, there were statistically significant improvements in WOMAC pain (p = 0.005) and function scores (p = 0.04) over 20 weeks |
4/10/14 |
Brainstorm Cell Therapeutics Inc. (New York and Petach Tikvah, Israel) |
Nurown |
Autologous mesenchymal stem cells secreting neurotrophic factors |
Amyotrophic lateral sclerosis |
FDA approved commencement of its phase II trial |
4/29/14 |
Civitas Therapeutics Inc. (Chelsea, Mass.) |
CVT-301 |
An inhaled formulation of levodopa |
Parkinson's disease |
Reported positive results from a phase IIb trial of CVT-301 showing the study met its primary endpoint |
4/29/14 |
Cytokinetics Inc. (South San Francisco) |
Tirasemtiv |
Fast skeletal muscle troponin activator |
Amyotrophic lateral sclerosis |
Failed in a phase IIb trial |
4/28/14 |
Genervon Biopharmaceuticals LLC (Pasadena, Calif.) |
GM604 |
Experimental therapy |
Amyotrophic lateral sclerosis |
Phase IIa data showed that at 10 weeks following completion of dosing clinical measurements of ALS progression remained the same in two of eight patients, while the rates of degradation of those clinical measurements had slowed in five of the remaining six treated patients |
4/29/14 |
Intra-Cellular Therapies Inc. (New York) |
ITI-007 |
5-HT2A receptor antagonist |
Schizophrenia |
Early data from a phase II study reveal that low doses of the drug show it acts as a potent 5-HT2A receptor antagonist, and that, as the dose is increased, it gradually engages other key brain receptors with regional selectivity |
4/8/14 |
Prana Biotechnology Ltd. (Melbourne, Australia) |
PBT2 |
Imaging agent |
Alzheimer's disease |
The phase II Imaging trial, IMAGINE, did not meet its primary endpoint of a statistically significant reduction in the levels of beta-amyloid plaques |
4/1/14 |
DIABETES | |||||
Adocia SAS (Lyon, France) |
Biochaperone Lispro |
Ultra-fast formulation of insulin |
Diabetes |
Phase IIa data showed that in comparison to Humalog (Eli Lilly and Co.) commercial insulin, it met the primary endpoint and showed a significant increase in Biochaperone Lispro bioavailability in the first half-hour compared to Humalog |
4/10/14 |
Concert Pharmaceuticals Inc. (Lexington, Mass.) |
CTP-499 |
A multi-subtype selective inhibitor of phosphodiesterases |
Diabetic kidney disease |
Missed the primary endpoint in a phase II trial of CTP-499 in patients with diabetic kidney disease, but the 48-week results showed some promise in a longer treatment duration |
4/28/14 |
Islet Sciences Inc. (Raleigh, N.C.) and BHV Pharma Inc. (Research Triangle Park, N.C.) |
Remogliflozin etabonate |
A selective SGLT2 inhibitor |
Type 2 diabetes and nonalcoholic steatohepatitis |
Two 12-week phase IIb studies showed that at week 12, twice-daily dosing of remogliflozin etabonate produced a statistically significant trend in dose response for change from baseline with changes in HbA1c ranging from -1.0 to -1.4%; once-daily dosing demonstrated a trend in dose response for change from baseline in HbA1c above the lowest dose with changes in HbA1c ranging from -0.5 to -0.8% |
4/17/14 |
Lexicon Pharmaceuticals Inc. (The Woodlands, Texas) |
LX4211 |
Sodium glucose transporter inhibitor |
Type 1 diabetes |
Phase II data showed it met the primary endpoint of reducing mealtime insulin use as well as several secondary endpoints |
4/15/14 |
Noxxon Pharma AG (Berlin) |
NOX-E36 |
Emapticap pegol |
Diabetic nephropathy |
Phase IIa proof-of-concept data showed statistically significant reductions in urinary albumin excretion and improved glycemic control |
4/7/14 |
Oramed Pharmaceuticals Inc. (Jerusalem) |
OMRD-0801 |
An orally ingestible insulin capsule |
Type 2 diabetes |
Phase IIa data showed that the oral insulin appeared to be safe and well tolerated for the dosing regimen tested |
4/25/14 |
Xeris Pharmaceuticals Inc. (Austin, Texas) |
G-Pump |
Stable liquid glucagon in an Insulet Corp. Omnipod infusion pump |
Type 1 diabetes |
Dosed the first subject in a phase II study designed to treat hypoglycemia |
4/28/14 |
GASTROINTESTINAL | |||||
Athersys Inc. (Cleveland) |
Multistem |
Stem cell product |
Treatment-refractory ulcerative colitis |
Product failed to moderate the severity of disease in a phase II study of 128 patients; it did not show statistically significant improvement compared to placebo |
4/29/14 |
INFECTION | |||||
Arrowhead Research Corp. (Pasadena, Calif.) |
ARC-520 |
RNAi therapeutic |
Chronic hepatitis B infection |
The first cohort of eight patients was fully enrolled and dosed in a phase IIa trial |
4/2/14 |
Gilead Sciences Inc. (Foster City, Calif.) |
Sovaldi |
Sofosbuvir; nucleotide analogue polymerase inhibitor |
Hepatitis C virus |
Phase II data of 400 mg of Sovaldi with the NS5A inhibitor ledipasvir 90 mg, with and without ribavirin twice-daily, showed 100% of treatment-naïve genotype 3 patients who got 12 weeks of the combo plus ribavirin, and 64% of the same patients without ribavirin achieved SVR 12 weeks after treatment |
4/11/14 |
Medivir AB (Stockholm) |
Simeprevir |
Protease inhibitor |
Genotype 1 hepatitis C virus infection |
Phase II data from cohort 2 in the interferon-free COSMOS study showed that 93% of prior null responder and treatment-naïve patients achieved sustained virologic responses 12 weeks after the end of treatment with simeprevir and Sovaldi (sofosbuvir, Gilead Sciences Inc.) |
4/15/14 |
MISCELLANEOUS | |||||
Akebia Therapeutics Inc. (Cambridge, Mass.) |
AKB-6548 |
A hypoxia-inducible factor prolyl hydroxylase inhibitor |
Anemia associated with chronic kidney disease |
Completed enrollment in its ongoing 200-patient phase IIb study |
4/16/14 |
Auris Medical AG (Basel, Switzerland) |
AM-101 |
An N-methyl-d-aspartate (NMDA) receptor antagonist |
Acute inner ear tinnitus |
Phase IIb data demonstrated it was well tolerated and safe and established proof of concept |
4/1/14 |
Celgene Corp. and Acceleron Pharma Inc. (Cambridge, Mass.) |
Sotatercept |
Formerly known as ACE-011; targets TGF-beta molecules |
Anemia in end-stage renal disease |
Interim phase IIa data showed dose-dependent increases in hemoglobin in patients on hemodialysis |
4/25/14 |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) |
ISIS-SMNRx |
An antisense drug |
Spinal muscular atrophy |
Phase II data showed that of seven infants, five are alive without the need for permanent ventilation; in another phase II study in children with SMA encouraging preliminary results were also observed in two additional functional tests: the six-minute walk test and the upper limb module test |
4/30/14 |
Ohr Pharmaceutical Inc. (New York) |
Squalamine |
An anti-angiogenic small molecule that counteracts multiple growth factors |
Wet form age-related macular degeneration |
Completed enrollment of its phase II trial with 142 patients |
4/30/14 |
Rxi Pharmaceuticals Corp. (Marlborough, Mass.) |
RXI-109 |
An sd-rxRNA compound that targets connective tissue growth factor |
Abnormal dermal scars |
The first patient has enrolled in a phase IIa study |
4/30/14 |
Scioderm Inc. (Durham, N.C.) |
SD-101 |
A topical therapy |
Blisters and lesions in patients with epidermolysis bullosa |
Completed enrollment in a phase IIb study (SD-003) to evaluate the safety and efficacy of SD-101; the study enrolled a total of 48 subjects, ages 6 months and older |
4/2/14 |
RESPIRATORY | |||||
Anergis SA (Epalinges, Switzerland) |
Allert |
Birch pollen allergy vaccine |
Birch pollen allergy |
The long-term efficacy trial is fully enrolled, including 196 patients enrolled that participated in the 2013 field-based phase IIb trial of Allert |
4/24/14 |
DBV Technologies SA (Bagneux, France) |
Viaskin |
Epicutaneous immunotherapy |
Peanut allergy |
A patient enrolled in the phase II ARACHILD study who received 18-month epicutaneous immunotherapy with Viaskin remained desensitized to peanut allergy after one year off-treatment, with a strict peanut diet |
4/17/14 |
Novavax Inc. (Gaithersburg, Md.) |
RSV-F |
Protein nanoparticle vaccine candidate |
Respiratory syncytial virus |
Phase II data in 720 healthy women of childbearing age showed it was well tolerated with no serious adverse events; it also demonstrated significant increases in RSV-F antibody levels across all doses and formulations, as well as clear increases in RSV neutralizing antibodies |
4/30/14 |
Theravance Inc. (South San Francisco) |
TD-4208 |
A nebulized aqueous solution |
Moderate-to-severe chronic obstructive pulmonary disease |
Started a dose-ranging phase IIb study |
4/14/14 |
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. |