Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Can-Fite Biopharma Ltd. (Petach Tikva, Israel) |
CF101 |
An A3 adenosine receptor agonist |
Moderate to severe plaque psoriasis |
All patients enrolled in its phase II/III psoriasis trial have completed the study's 32-week treatment protocol, and the firm expects to publish top-line results by the end of March |
2/5/15 |
Cymabay Therapeutics Inc. (Newark, Calif.) |
Arhalofenate |
A PPAR gamma agonist; a uricosuric agent |
Gout |
Top-line results from its phase IIb study showed that the trial met its primary endpoint of demonstrating a reduction in gout flare rate (p = 0.0056); it was well tolerated, and the overall safety profile was favorable and consistent with results of earlier studies |
2/25/15 |
Galapagos NV (Mechelen, Belgium) |
GLPG0634 |
Filgotinib |
Rheumatoid arthritis |
The last patient in the DARWIN 1 phase II trial completed the 12-week visit, triggering final data collection from the 599 patients enrolled in the trial, followed by database lock and analysis; top-line data from 24 weeks of treatment are expected in July |
2/24/15 |
Medicinova Inc. (La Jolla, Calif.) |
MN-166 |
Ibudilast |
Amyotrophic lateral sclerosis |
Its ongoing trial has now enrolled 30 of the planned 60 subjects; the study includes a six-month treatment period followed by a six-month open-label extension |
2/18/15 |
VBL Therapeutics (Tel Aviv, Israel) |
VB-201 |
Autoimmune therapy |
Psoriasis and ulcerative colitis |
VB-201 proved safe and well tolerated in both trials; in psoriasis, data showed it had no effect compared to placebo on the primary or secondary endpoints at either dose level tested, with the PASI 50 score for VB-201 patients being 26.4% at 16 weeks vs. 38% for placebo patients; in ulcerative colitis, no statistically significant effect of VB-201 was observed compared to placebo on either the primary or secondary endpoints, and at 12 weeks, the remission rate for VB-201 was 10.5% vs. 15.1% in the placebo arm |
2/18/15 |
CANCER | |||||
AB Science SA (Paris) |
Masitinib |
Tyrosine kinase inhibitor |
Nonresectable, metastatic esophagogastric adenocarcinoma |
Efficacy and safety results from a phase II study showed that in the masitinib plus irinotecan treatment-arm, median overall survival (OS) and progression-free survival (PFS) were 13.4 and 3.9 months, respectively; in the masitinib plus FOLFIRI treatment-arm, median OS and PFS were 10.9 and 2.4 months, respectively; the masitinib plus 5-fluorouracil treatment-arm was closed early due to lack of efficacy on PFS |
2/3/15 |
Aduro Biotech Inc. (Berkeley, Calif.) |
Gvax Pancreas and CRS-207 |
Immuno-oncology candidates |
Metastatic pancreatic cancer |
An investigator-sponsored phase II trial is under way to evaluate Gvax Pancreas and CRS-207 in combination with Opdivo (nivolumab), a monoclonal antibody against programmed death-1 receptor, or PD-1, developed by Bristol-Myers Squibb Co. |
2/27/15 |
Bio-Path Holdings Inc. (Houston) |
BP-100-1.01 |
Liposomal Grb-2 |
Acute myeloid leukemia |
Began enrollment in the safety segment of its phase II study |
2/10/15 |
Genmab A/S (Copenhagen) |
Daratumumab |
A human CD38 monoclonal antibody |
Double-refractory multiple myeloma |
Phase II data showed an overall response rate of 29.2% in the 16 mg/kg dosing group and the median duration of response was 7.4 months as determined by an independent review committee |
2/4/15 |
Heat Biologics Inc. (Durham, N.C.) |
HS-410 |
Vesigenurtacel-L |
Non-muscle invasive bladder cancer |
Is adding a fourth monotherapy arm to its ongoing phase II study |
2/27/15 |
Immunomedics Inc. (Morris Plains, N.J.) |
Sacituzumab govitecan |
Antibody-drug conjugate (ADC) |
Small-cell lung cancer (SCLC) and non-small-cell lung cancer (NSCLC) |
Of patients, 33% with SCLC and 31% with NSCLC saw their tumors reduced in size by 30% or more after being treated with sacituzumab govitecan; including patients that reported stable disease as their best response, the ADC controlled the progression of the cancer in 75% and 56% of NSCLC and SCLC patients, respectively |
2/20/15 |
Lion Biotechnologies Inc. (Los Angeles) |
LN-144 |
Cell product of autologous tumor infiltrating lymphocytes |
Refractory metastatic melanoma |
Gained FDA approval to begin a phase II study |
2/3/15 |
Merrimack Pharmaceuticals Inc. (Cambridge, Mass.) |
MM-121 |
A fully human monoclonal antibody targeting ErbB3 |
Heregulin-positive, locally advanced or metastatic non-small-cell lung cancer |
Initiated a global, open-label, biomarker-selected, randomized phase II trial of MM-121 in combination with docetaxel or pemetrexed vs. docetaxel or pemetrexed alone |
2/20/15 |
Oncogenex Pharmaceuticals Inc. (Bothell, Wash.) |
Apatorsen |
Heat shock protein 27 inhibitor |
Non-squamous, non-small-cell lung cancer |
Completed patient enrollment in Spruce, an investigator-sponsored, randomized, placebo-controlled phase II trial evaluating apatorsen in combination with carboplatin and pemetrexed |
2/25/15 |
Oncolytics Biotech Inc. (Calgary, Alberta) |
Reolysin |
Reovirus variant |
Advanced or metastatic colorectal cancer |
Enrollment has been completed in a randomized phase II study |
2/19/15 |
Oncomed Pharmaceuticals Inc. (Redwood City, Calif.) |
OMP-21M18 |
Demcizumab; anti-DLL4 |
Advanced-stage non-small-cell lung cancer |
Patient dosing has begun in the double-blind, placebo-controlled, randomized phase II trial |
2/5/15 |
Qu Biologics Inc. (Vancouver, British Columbia) |
QBKPN |
Site-specific immunomodulator |
Non-small-cell lung cancer |
Enrolled the first two subjects in its phase IIa trial |
2/5/15 |
Spectrum Pharmaceuticals Inc. (Henderson, Nev.) |
Captisol-enabled melphalan |
Propylene glycol-free; an injectable formulation of the chemotherapy that incorporates Ligand Pharmaceuticals Inc.'s Captisol beta-cyclodextrin |
Multiple myeloma |
A phase II study supports the safety and efficacy of high-doses as a conditioning treatment prior to autologous hematopoietic stem cell transplantation in patients with the disease |
2/13/15 |
CARDIOVASCULAR | |||||
Mast Therapeutics Inc. (San Diego) |
AIR001 |
A sodium nitrite solution for intermittent inhalation via nebulizer |
Heart failure |
Patient dosing has started in two institutional-sponsored phase IIa studies of AIR001 for the treatment of patients experiencing heart failure with preserved ejection fraction |
2/5/15 |
Medicinova Inc. (La Jolla, Calif.) |
MN-001 |
Tipelukast |
Moderate to severe idiopathic pulmonary fibrosis |
FDA has approved the protocol for a clinical trial, a phase II study |
2/11/15 |
Omeros Corp. (Seattle) |
OMS721 |
Human monoclonal antibody |
Thrombotic microangio-pathies |
Completed dosing of the low-dose cohort of patients in its ongoing phase II study |
2/20/15 |
Trevena Inc. (King of Prussia, Pa.) |
TRV027 |
Targets the angiotensin II type 1 receptor |
Acute heart failure (AHF) |
The randomized, double-blind, standard of care-controlled BLAST-AHF trial is enrolling 400 patients to compare TRV027 plus standard heart failure therapy vs. placebo plus standard therapy; the primary objective is a composite of clinically important outcomes: mortality, worsening heart failure, hospital readmission rate, dyspnea and length of hospital stay |
2/4/15 |
CENTRAL NERVOUS SYSTEM | |||||
Amarantus Biosciences Holdings Inc. (San Francisco) |
Eltoprazine |
Small-molecule 5-HT1A/1B serotonin receptor agonist |
Parkinson's disease levodopa-induced dyskinesias |
Phase IIa data demonstrated that eltoprazine significantly reduced peak dose dyskinesia at both the 5-mg and 7.5-mg doses using the Combined Dyskinesia Rating Scale; the 5-mg dose also showed a significant anti-dyskinetic effect on other measures of dyskinesia, including the Rush dyskinesia rating scale; there were no adverse effects on levodopa efficacy at any dose level |
2/11/15 |
Forum Pharmaceuticals Inc. (Watertown, Mass.) |
FRM-0334 |
Potentially reverses the loss of protein function in FTD-GRN |
Frontotemporal dementia with granulin mutation |
Dosed the first patient in a phase IIa proof-of-mechanism trial |
2/11/15 |
Marinus Pharmaceuticals Inc. (Radnor, Pa.) |
Ganaxolone |
A synthetic analogue of the endogenous neurosteroid allopregnanolone |
Epilepsy |
Initiated a phase II trial in female children with epilepsy caused by a mutation of the protocadherin 19 gene |
2/4/15 |
Neurotrope Inc. (Newark, N.J.) |
Bryostatin-1 |
A protein kinase C epsilon modulator |
Alzheimer's disease |
Top-line results from its randomized, double-blind, placebo-controlled, single-dose phase IIa trial met its primary endpoint by demonstrating preliminary safety and tolerability; no serious adverse events or clinically significant laboratory abnormalities occurred, and bryostatin was well tolerated |
2/25/15 |
DIABETES | |||||
Adocia SA (Lyon, France) |
Hinsbet |
Recombinant human insulin |
Type 1 diabetes |
Results from a phase II trial of Hinsbet, in comparison to commercial products Humalog (insulin lispro) and Humulin (recombinant human insulin), both sold by Indianapolis-based Eli Lilly and Co., showed it met its primary endpoint, measuring the increase of recombinant human insulin bioavailability during the first hour, with data showing that Hinsbet has an early effect equivalent to that of a fast-acting insulin analogue, Humalog, and twice superior to that of regular recombinant human insulin, Humulin |
2/6/15 |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) |
ISIS-PTP1BRx |
Antisense therapeutics |
Type 2 diabetes |
Top-line results from a phase II study showed patients achieved statistically significant reductions in body weight and HbA1c, with a mean reduction in HbA1c of 0.7 percentage points from baseline achieved at 36 weeks, compared to a mean reduction of 0.2 percentage points for placebo-treated patients (p=0.03) |
2/4/15 |
Nuo Therapeutics Inc. (Gaithersburg, Md.) |
Aurix |
Biodynamic hematogel |
Diabetic foot ulcers, venous leg ulcers and pressure ulcers, or bed sores |
Recruiting patients for a newly initiated study of Aurix; patients will be treated using Aurix and standard care, and compared 1-to-1 with patients receiving "usual and customary care" |
2/20/15 |
GASTROINTESTINAL | |||||
Ardelyx Inc. (Fremont, Calif.) and Astrazeneca plc (London) |
Tenapanor |
The NHE3 sodium transporter inhibitor |
Constipation-predominant irritable bowel syndrome |
Met its primary endpoint: a statistically significant dose-related decrease in serum phosphate levels for patients on drug compared to those given placebo (p = 0.012); it showed higher-than-expected incidences of diarrhea, but the overall safety profile was described as consistent with previous trials |
2/3/15 |
Ironwood Pharmaceuticals Inc. (Cambridge, Mass.) |
IW-3718 |
A grastric retentive formulation of a bile acid sequestrant |
Refractory gastroeso-phageal reflux disease |
Top-line data from an exploratory, 93-patient phase IIa trial indicated that IW-3718 improved heartburn and certain other symptoms |
2/5/15 |
Zealand Pharma A/S (Copenhagen) and Helsinn Healthcare SA (Lugano, Switzerland) |
Elsiglutide |
A GLP-2 receptor agonist |
To prevent chemotherapy-induced diarrhea |
Started a phase IIb dose-finding study |
2/6/15 |
INFECTION | |||||
Abivax SAS (Paris) |
ABX464 |
A small molecule inhibiting HIV replication |
HIV |
Began enrollment and the first HIV-positive patient has been dosed in a phase IIa trial; 80 treatment-naïve patients will be enrolled into 10 cohorts with six patients receiving ABX464 and two participants receiving placebo in each cohort |
2/3/15 |
Abivax SAS (Paris) |
ABX203 |
A therapeutic vaccine formulated as a nasal spray solution |
Hepatitis B virus |
Dosed the first patient in a pivotal phase IIb/III trial that will recruit 230 patients through 50 clinical centers in seven Asia-Pacific countries |
2/27/15 |
Achillion Pharmaceuticals Inc. (New Haven, Conn.) |
ACH-3102 |
NS5A inhibitor |
Hepatitis C virus |
Phase II data showed that a combination of ACH-3102 and Sovaldi (sofosbuvir, Gilead Sciences Inc.) cleared HCV in 100% of 12 genotype 1 patients after six weeks of treatment |
2/10/15 |
Albireo AB (Gothenburg, Sweden) |
A4250 |
Designed to act locally in the gut to inhibit the ileal sodium dependent bile acid transporter |
Nonalcoholic steatohepatitis |
Initiated a phase II trial |
2/6/15 |
Contravir Pharmaceuticals Inc. (Edison, N.J.) |
FV-100 |
Fast-acting, low-dose, once-daily oral antiviral |
Varicella zoster virus that causes shingles |
FV-100 demonstrated antiviral activity against the varicella zoster virus that causes shingles, as well as the potential to reduce the incidence of postherpetic neuralgia, in a phase II study |
2/24/15 |
Eiger Biopharmaceuticals Inc. (Palo Alto, Calif.) |
Lonafarnib |
Orally active inhibitor of farnesyl transferase |
Chronic hepatitis delta viral infection |
Findings from a phase II study showed a decrease in HDV RNA viral levels after treatment for 28 days compared with placebo, including a statistically significant dose-dependent difference between the twice-daily 100 mg and 200 mg doses of lonafarnib compared with placebo; lonafarnib was well tolerated in the study, with mild to moderate nausea and diarrhea as the most common adverse events |
2/24/15 |
Savara Pharmaceuticals Inc. (Austin, Texas) |
Aerovanc |
An inhaled antibiotic |
Methicillin-resistant Staphylococcus aureus lung infection |
Top-line results from its phase II trial showed Aerovanc met the primary endpoint in the modified intent to treat population with a statistically significant reduction in MRSA density in sputum, compared to placebo |
2/24/15 |
Viamet Pharmaceuticals Holdings LLC (Research Triangle Park, N.C.) |
VT-1161 |
Orally available, potent and selective inhibitor of fungal CYP51 |
Recurrent vulvovaginal candidiasis |
Initiated the phase IIb REVIVE (REcurrent Vulvovaginal Candidiasis Inhibition; an Oral VT-1161 Tablet Evaluation) trial |
2/13/15 |
INFLAMMATORY | |||||
Cytori Therapeutics Inc. (San Diego) |
ECCO-50 |
Autologous adipose-derived therapy |
Osteoarthritis of the knee |
Treated the first patient in a randomized, double-blind, placebo-controlled phase II study |
2/18/15 |
Flexion Therapeutics Inc. (Burlington, Mass.) |
FX006 |
An intra-articular sustained-release steroid |
Osteoarthritis of the knee |
Completed enrollment in the pivotal phase IIb confirmatory trial |
2/25/15 |
MISCELLANEOUS | |||||
Afferent Pharmaceuticals Inc. (San Mateo, Calif.) |
AF-219 |
Selective, non-narcotic and orally administered P2X3 antagonist |
Interstitial cystitis/bladder pain syndrome |
Phase II results showed that AF-219 improved symptoms of pain and urinary urgency |
2/27/15 |
Dermira Inc. (Menlo Park, Calif.) |
DRM-04 |
Topical anticholinergic product |
Axillary hyperhidrosis |
Phase IIb data showed that average reduction in sweat production from baseline to week four ranged from 67.7% to 79.8% (72.7 to 105.3 mg per five minutes) in patients in the two arms treated with DRM04, compared to 48.7% (53.9 mg per five minutes) in patients who received vehicle |
2/9/15 |
Galectin Therapeutics Inc. (Norcross, Va.) |
GR-MD-02 |
Galectin-2 inhibitor |
Advanced fatty liver disease, or nonalcoholic steatohepatitis with cirrhosis |
The design of its planned phase II program to be conducted under a special protocol assessment, if approved, includes two studies beginning with NASH-CX, a multicenter, randomized, placebo-controlled, double-blind, parallel-group phase II trial |
2/25/15 |
Leading Biosciences Inc. (San Diego) |
LB1148 |
Broad-spectrum serine protease inhibitor |
Septic shock and multi-organ failure |
FDA cleared the company to proceed on an investigational new drug application to develop LB1148 in patients with septic shock and multi-organ failure; LBS plans to initiate enrollment in the second quarter in a randomized, double-blind, multicenter, placebo-controlled phase II safety and efficacy study known as SSAIL (Treatment of Septic Shock by Inhibiting Autodigestion and Preserving Gut Integrity with Enteric LB1148) in patients with septic shock |
2/3/15 |
Ocera Therapeutics Inc. (Palo Alto, Calif.) |
OCR-002 |
Ornithine phenylacetate |
Upper gastro-intestinal bleeding associated with liver cirrhosis |
Top-line results from a phase IIa study showed that OCR-002 resulted in greater absolute and relative reductions in median plasma ammonia vs. placebo at 12 hours after baseline (-19.35 microM/L and -19.62% vs. -2 microM/L and -3.25%, respectively), but the differences in plasma ammonia levels between treatment arms did not reach statistical significance, due in part to the higher than expected variability in plasma ammonia levels |
2/20/15 |
Stealth Biotherapeutics Inc. (Boston) |
Bendavia |
Targets the inner mitochondrial membrane |
Chronic kidney disease at risk of acute kidney injury |
Interim phase II results with Bendavia in its cardio-renal program demonstrated an improvement in long-term renal blood flow and cortical perfusion, correlating with a statistically significant decrease in serum creatinine |
2/24/15 |
RESPIRATORY | |||||
Anergis SA (Epalinges, Switzerland) |
Allert |
Birch pollen allergy immunotherapeutic |
Birch pollen allergy |
Phase IIb data demonstrated in a field-based, randomized, placebo-controlled, double-blind trial in 239 patients, who received either Allert (at 50 μg or 100 μg doses) or a placebo during a two-month immunotherapy regimen, that all main efficacy and safety endpoints were met |
2/24/15 |
Aquinox Pharmaceuticals Inc. (Vancouver, British Columbia) |
AQX-1125 |
Small-molecule activator of SHIP1 |
Chronic obstructive pulmonary disease |
Reached its target enrollment in the phase II FLAGSHIP trial |
2/3/15 |
Dbv Technologies SA (Bagneux, France) |
Viaskin Peanut |
Immunotherapy |
Peanut allergy |
A 250-mcg dose met the primary efficacy endpoint of a phase IIb study, with 50% of responders measured 12 months after treatment having a peanut allergy-eliciting dose 10-fold greater than baseline or achieving a post-treatment-eliciting dose of at least 1,000 mg. vs. 25% of with placebo (p = 0.0108) |
2/25/15 |
Nuvo Research Inc. (Mississauga, Ontario) |
WF10 |
Immune-modulating drug candidate |
Refractory allergic rhinitis |
Results of its 16-week, double-blind, placebo-controlled phase II study conducted in Germany to compare the safety and efficacy of WF10 and its main constituents (sodium chlorite and sodium chlorate) with saline showed that the WF10 arm demonstrated a reduction in Total Nasal Symptom Score (TNSS) over the course of the observation period, similar to the reduction in TNSS demonstrated in the WF10 arm in the company's previous 2010 phase II proof-of-concept study; however, the placebo arm demonstrated a greater reduction in TNSS than observed in the earlier study |
2/2/15 |
|
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. For more information about individual companies and/or products, see Thomson Reuters Cortellis. | |||||