Company
(location)

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.