Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Aurinia Pharmaceuticals Inc. (Victoria, British Columbia) |
Voclosporin |
Calcineurin inhibitor |
Active lupus |
In the first seven patients that have reached at least eight weeks of therapy, 100% have achieved at least a 25% reduction in proteinuria compared to study entry |
2/9/16 |
Celgene Corp. (Summit, N.J.) |
Ozanimod |
A selective S1P 1 and 5 receptor modulator |
Relapsing multiple sclerosis |
Radiance phase II data at week 72 showed patients on either dose had similar results to week 24; the trial had met its primary efficacy endpoint - reduction in the cumulative number of total gadolinium-enhancing lesions; for placebo patients switched after week 24 to either dose of ozanimod (0.5 mg or 1 mg), the mean number of GdE lesions was decreased by 90% to 95% compared with that at week 24 |
2/19/16 |
Fast Forward Pharmaceuticals BV (Utrecht, the Netherlands) |
FFP104 |
A monoclonal antibody targeting the CD40 molecule |
Primary biliary cirrhosis and Crohn's disease |
Initiated multicenter phase II trials |
2/4/16 |
Mesoblast Ltd. (New York) |
MPC-300-IV |
Rexlemestrocel-L |
Rheumatoid arthritis |
Top-line phase II data showed a single intravenous infusion of the lower dose was safe and resulted in early and sustained clinical responses; the ACR20 endpoint was achieved by 47% of all treated patients at 12 weeks, and by 60% of treated patients who failed one to two biologics vs. 25% and 17% of matched placebo controls |
2/17/16 |
Rigel Pharmaceuticals Inc. (South San Francisco) |
Fostamatinib |
Oral SYK inhibitor |
Chronic autoimmune hemolytic anemia |
Started a phase II trial that will enroll patients who previously received treatment but have relapsed, with stage one of the study enrolling 17 patients and stage two enrolling an additional 20 patients |
2/26/16 |
Swedish Orphan Biovitrum AB (Stockholm) |
Kineret |
Anakinra |
Acute gout and Still's disease |
Initiated two clinical programs with Kineret in North America; the acute gout program will include a dose-finding phase II study, followed by a planned phase III study; the Still's disease study is a phase III |
2/24/16 |
Synthetic Biologics Inc. (Rockville, Md.) |
Trimesta |
An oral estriol drug |
Relapsing-remitting multiple sclerosis |
Independent third-party analysis of a phase II trial showed that Trimesta plus Copaxone (glatiramer acetate injection) did not demonstrate statistically significant differences compared to the Copaxone plus placebo at either 12 or 24 months |
2/3/16 |
Xenon Pharmaceuticals Inc. (Burnaby, British Columbia) |
XEN801 |
A stearoyl co-A desaturase-1 inhibitor |
Moderate to severe acne |
Started dosing patients in a phase II trial |
2/11/16 |
Ziarco Pharma Ltd. (Sandwich, U.K.) |
ZPL-389 |
A selective histamine H4 receptor antagonist |
Moderate to severe psoriasis |
Started a phase IIa proof-of-concept |
2/3/16 |
CANCER | |||||
Amgen Inc. (Thousand Oaks, Calif.) |
AMG319 |
P13 kinase delta inhibitor |
Squamous cell carcinoma of the head and neck |
Launched a phase II trial that will enroll about 54 patients |
2/24/16 |
Biotime Inc. (Alameda, Calif.) and subsidiary Asterias Biotherapeutics Inc. |
AST-VAC1 |
Cancer immunotherapy |
Acute myeloid leukemia |
Completed an end-of-phase II meeting with the FDA, which indicated general agreement with the proposed development plan for registration of AST-VAC1 through a single phase III trial to support an accelerated development pathway and BLA filing |
2/25/16 |
Cleveland Biolabs Inc. (Buffalo, N.Y.) |
Entolimod |
Neo-adjuvant therapy |
Primary colorectal cancer |
Started a phase II study of entolimod as a neo-adjuvant therapy in treatment-naïve patients who are recommended for surgery |
2/3/16 |
Coherus Biosciences Inc. (Redwood City, Calif.) |
CHS-1701 |
A proposed biosimilar of Neulasta (pegfilgrastim, Amgen Inc.) |
Cancer |
Met both primary endpoints in a double-blind, randomized, two-period, parallel-arm study in 303 healthy subjects |
2/3/16 |
Heat Biologics Inc. (Durham, N.C.) |
HS-410 |
Vesigenurtacel-L |
Non-muscle invasive bladder cancer |
FDA placed the phase II trial on partial clinical hold due to a misidentified cell line; FDA lifted the partial clinical hold on the phase II trial of HS-410 either alone or in combination with standard of care bacillus Calmette-Guérin; It will no longer enroll patients in its phase II monotherapy arm; Heat added the monotherapy trial arm in response to the intermittent global shortage of standard of care Bacillus Calmette-Guérin, or BCG, in early 2015, and that shortage has since been resolved; the 16 patients currently enrolled will continue receiving HS-410 monotherapy |
2/4/16; 2/11/16; 2/26/16 |
Lion Biotechnologies Inc. (Los Angeles) |
LN-144 |
Derived from the patient's own tumor, LN-144 is based on adoptive cell therapy regimen |
Refractory metastatic melanoma |
FDA supported the start of a phase II trial; a single-arm study will enroll about 20 participants |
2/2/16 |
Nymox Pharmaceutical Corp. (Hasbrouck Heights, N.J.) |
NX-1207 |
Fexapotide triflutate |
Prostate cancer |
Results from the completion of the U.S. 40-month phase II NX03-0040 trial, which enrolled 147 patients with low grade localized prostate cancer, showed it successfully met its endpoints, with cancer progression clinical outcomes significantly improved |
2/10/16 |
OBI Pharma Inc. (Taipei, Taiwan) |
OBI-822/821 |
Immunotherapy |
Metastatic breast cancer |
Top-line phase II/III data showed it did not meet the primary efficacy endpoint of progression-free survival |
2/23/16 |
Pharmamar SA (Madrid, Spain) |
PM184 |
Microtubule inhibitor |
Hormone-receptor positive, HER2-negative, locally advanced and/or metastatic breast cancer |
Started an open-label, two-stage phase II trial, expected to enroll 106 patients who have experienced progression following earlier treatment with anthracyclines and taxanes |
2/29/16 |
Rexahn Pharmaceuticals Inc. (Rockville, Md.) |
Archexin |
Antisense drug |
Metastatic renal cell carcinoma |
Identified a maximum tolerated dose and completed stage 1 of a dose-escalation phase Iia study of archexin in combination with everolimus; also initiated enrollment in stage 2 of the trial |
2/9/16 |
CARDIOVASCULAR | |||||
Athersys Inc. (Cleveland) |
Multistem |
Cell therapy |
Ischemic stroke |
Phase II data showed that 23% of people who suffered an ischemic stroke achieved a clinically rated "excellent outcome" a year after treatments vs. just 8.2% of patients who received placebo |
2/19/16 |
Bellerophon Therapeutics Inc. (Warren, N.J.) |
Inopulse |
Inhaled nitric-oxide product |
Pulmonary arterial hypertension |
Phase II data extension data turned up a sustained benefit to patients who received Inopulse 75 mcg/kg ideal body weight per hour therapy for at least 12 hours per day combined with long-term oxygen therapy; in part 1, patients showed a mean improvement in six minute walk distance of 52.4 meters after 16 weeks; in part 2, after 16 to 32 months, the patients who remained on Ino 75 for at least 12 hours a day combined with LTOT continued to maintain a mean increase in 6MWD of 55.2 meters |
2/10/16 |
Biocryst Pharmaceuticals Inc. |
Avoralstat |
Oral, liquid-filled soft gel candidate |
Hereditary angioedema |
The phase IIb OPuS-2 study with avoralstat given three times daily failed to show a benefit on the primary endpoint of preventing attacks |
2/9/16 |
Juventas Therapeutics Inc. (Cleveland) |
JVS-100 |
Gene therapy encoding human stromal cell-derived factor-1 |
Advanced peripheral artery disease |
Is enrolling its STOP-PAD trial, a 120-patient, placebo-controlled, randomized, double-blind phase II study |
2/5/16 |
Mast Therapeutics Inc. (San Diego) |
AIR001 |
Nebulized AIR001 attenuates the hemodynamic derangements of cardiac failure |
Heart failure with preserved ejection fraction |
Top-line phase IIa data showed that the sodium nitrate solution statistically significantly improved the pre-specified primary endpoint: change in pulmonary capillary wedge pressure (PCWP) at 20 Watts exercise after drug treatment relative to PCWP at 20 Watts exercise in the initial assessment prior to drug treatment, compared to placebo-treated patients |
2/2/16 |
Opko Health Inc. (Miami) |
Factor VIIa-CTP |
Long-acting factor VIIa |
Bleeding episodes in hemophilia A or B |
Dosed the first patient in a phase IIa dose-escalation study |
2/24/16 |
Remedy Pharmaceuticals Inc. (New York) |
Cirara |
A high-affinity inhibitor of Sur1-Trmp4 channels |
To prevent edema in large hemispheric stroke patients |
Reported additional efficacy data from its phase II trial, including results showing that adjudicated neurological mortality was reduced from 25% in placebo to 7% in Cirara-treated patients (p=0.03) |
2/23/16 |
CENTRAL NERVOUS SYSTEM | |||||
Alkermes plc (Dublin) |
Aristada |
Aripiprazole lauroxil extended-release injectable suspension |
Schizophrenia |
Top-line data from a pharmacokinetic study evaluating a two-month dosing interval showed the 1,064-mg dose achieved therapeutically relevant plasma concentrations of aripiprazole with a PK profile that supports dosing once every two months |
2/26/16 |
Braeburn Pharmaceuticals Inc. (Princeton, N.J.) and Camurus AB (Lund, Sweden) |
CAM2038 |
Weekly and monthly subcutaneous buprenorphine injections |
Opioid-dependent patients with chronic pain |
The first patients were dosed with CAM2038 in a pharmacokinetic phase II study |
2/23/16 |
Capnia Inc. (Redwood City, Calif.) |
Carbon dioxide nasal CO2 |
Non-inhaled treatment |
Trigeminal neuralgia |
Enrolled the first patient in a phase II trial; the primary endpoint is pain relief as reported on a visual analogue scale in a subject diary, assessed through 24 hours from initial dose |
2/26/16 |
Cerecor Inc. (Baltimore) |
CERC-501 |
A selective kappa opioid receptor antagonist |
Tobacco use disorder |
Enrolled the first subject in a phase II trial |
2/3/16 |
Endo International plc (Dublin) |
Xiaflex |
Collagenase clostridium histolyticum |
Edematous fibrosclerotic panniculopathy |
Started a 350-patient, phase IIb study |
2/18/16 |
Fibrogen Inc. (San Francisco) |
Roxadustat |
Oral agent |
Anemia in patients with chronic kidney disease |
Phase II data showed roxadustat maintained hemoglobin levels among patients on hemodialysis previously treated with and switched from epoetin alfa |
2/17/16 |
Living Cell Technologies Ltd. (Auckland, New Zealand) |
Ntcell |
A choroid plexus cell product |
Neurodegen-erative diseases |
Received approval for a phase IIb trial in New Zealand |
2/4/16 |
Minerva Neurosciences Inc. (Waltham, Mass.) |
MIN-117 |
An antagonist on 5-HT1A and 5-HTT |
Major depressive disorder |
Completed patient enrollment in a randomized, placebo-controlled, double-blind European phase IIa trial |
2/23/16 |
Neurocrine Biosciences Inc. (San Diego) |
NBI-98854 |
Valbenazine; a selective small-molecule vesicular monoamine transporter 2 inhibitor |
Tourette syndrome |
Started a phase II trial that will enroll up to 90 subjects |
2/3/16 |
Neurotrope Inc. (Newark, N.J.) |
Bryostatin-1 |
PKC modulator |
Alzheimer's disease |
The first patients have been dosed in the company's phase IIb trial |
2/12/16 |
Pfizer Inc. (New York) |
PF-05212377 |
5-hydroxytryptamine6 serotonin receptor antagonist |
Alzheimer's disease |
Phase II fizzled due to differences in dosing, duration and the disability of patients |
2/3/16 |
Tobira Therapeutics Inc. (South San Francisco) |
Cenicriviroc |
An oral, once-daily, immunomodulator that blocks two chemokine receptors, CCR2 and CCR5 |
Obesity with pre-diabetes or diabetes and suspected non-alcoholic fatty liver disease |
Completed recruitment for the phase IIa ORION study |
2/10/16 |
Tonix Pharmaceuticals Holdings Corp. (New York) |
TNX-201 |
Dexisometheptene mucane |
Episodic tension-type headache |
Top-line results from its phase II study showed that it did not achieve its primary efficacy endpoint of achieving headache pain-free status at two hours after dosing; it also did not achieve two other primary endpoints: the proportion of participants with at least a 70% reduction in pain from baseline on the Visual Analog Scale at two hours after dosing, and an increase of the mean change from baseline to two hours post-dose in the VAS score |
2/17/16 |
Zafgen Inc. (Cambridge, Mass.) |
Beloranib |
MetAP2 inhibitor |
Severe obesity complicated by type II diabetes |
Phase IIb ZAF-203 data showed it achieved its primary endpoint, as treatment with the 1.8-mg and the 1.2-mg doses of beloranib resulted in 12.7% (p<0.0001) and 13.5% (p<0.0001) reductions in body weight, respectively, compared with a reduction of 3.1% for placebo |
2/19/16 |
DIABETES | |||||
Aerpio Therapeutics Inc. (Cincinnati) |
AKB-9778 |
A first-in-class human protein tyrosine phosphatase-beta (HPTP-beta) inhibitor |
Diabetic retinopathy |
Data from the randomized, double-blind, placebo-controlled phase IIa TIME-2 study suggested that it improved underlying retinopathy in the study eye at month three, alone and in combination with Lucentis (ranibizumab injection, 0.3 mg intravitreally, Genentech Inc./Roche AG) by two or more steps on the diabetic retinopathy severity scale |
2/11/16 |
Transition Therapeutics Inc. (Toronto) |
TT401 |
Once-weekly administered oxyntomodulin analog with dual GLP-1 and glucagon agonist activity |
Type 2 diabetes |
Phase II study results demonstrated HbA1c improvements of up to -1.43% (similar to the exenatide arm); all TT401 dose arms and the exenatide arm were statistically significant relative to the placebo arm at weeks 12 and 24, and TT401 produced dose dependent weight loss (up to -3.3 kg); the weight loss observed in the highest dose arm (50 mg TT401) was statistically significant relative to both the placebo and exenatide arms at weeks 12 and 24 |
2/2/16 |
Vtv Therapeutics Inc. (High Point, N.C.) |
TTP399 |
Oral, liver-selective glucokinase activator |
Type 2 diabetes |
Completed patient enrollment in the company's AGATA study, a phase IIb trial |
2/11/16 |
Zealand Pharma A/S (Copenhagen) |
ZP4207 |
A stable glucagon analogue for liquid formulation |
Hypoglycemia associated with diabetes |
The first patients were dosed in a phase II trial |
2/5/16 |
GASTROINTESTINAL | |||||
Zealand Pharma A/S (Copenhagen) |
ZP1848 |
A long-acting GLP-2 analogue |
Short bowel syndrome |
Dosed the first patients in a phase II trial |
2/17/16 |
INFECTION | |||||
Biota Pharmaceuticals Inc. (Atlanta) |
BTA074 |
A potent and selective inhibitor of the interaction between two viral proteins from HPV6 and HPV11 |
Condyloma, or anogenital warts, caused by human papillomavirus types 6 & 11 |
The first patient has been dosed in a phase II, double-blind, randomized, placebo-controlled trial |
2/9/16 |
Citius Pharmaceuticals Inc. (Maynard, Mass.) |
Hydrocortisone acetate and lidocaine hydrochloride |
As single agents and in combination |
Grade I and II hemorrhoids |
Top-line phase IIa data showed that, within the first few days of treatment, the highest concentration of the HC+L candidate was directionally superior to placebo, measured by the number of subjects experiencing a minimum of two levels of improvement from baseline on the Global Score of Disease Severity scale |
2/2/16 |
Genocea Biosciences Inc. (Cambridge, Mass.) |
GEN-003 |
T-cell-directed immunotherapy designed to elicit both a T-cell and B-cell immune response |
Genital herpes |
Started dosing in a 135-subject phase IIb trial |
2/1/16 |
Inflarx GmbH |
IFX-1 |
First-in-class complement 5a-targeting antibody |
Early stage sepsis-associated organ dysfunction |
Hit the primary endpoint of a phase IIa trial of 72 intensive care unit patients; the primary endpoints were associated with the pharmacokinetic and pharmacodynamic profile, but the company also saw trends toward efficacy on secondary endpoints |
2/1/16 |
Janssen Sciences Ireland UC (unit of Johnson & Johnson; New Brunswick, N.J.) and Viiv Healthcare Ltd. (London) |
Rilpivirine and cabotegravir |
Long-acting intramuscular formulations of HIV medicines |
HIV |
Ongoing phase IIb 32-week data of rilpivirine and cabotegravir, given together every four or eight weeks, showed comparable antiviral activity to three-drug oral antiretroviral therapy (investigational cabotegravir and two nucleoside reverse transcriptase inhibitors) |
2/24/16 |
Matinas Biopharma Holdings Inc. (Bedminster, N.J.) |
MAT2203 |
Anti-infective candidate |
Refractory mucocutaneous candidiasis infection |
Patient screening and enrollment are underway in a National Institutes of Health-sponsored phase IIa study |
2/9/16 |
Pharmassentia Corp. (Taipei, Taiwan) |
Ropeginterferon alfa-2b |
P1101 |
Chronic hepatitis C virus genotype 2 infection |
Phase II data support transition into a phase III trial testing P1101 and ribavirin targeting genotype 2 infection, especially in Asian countries |
2/24/16 |
Regulus Therapeutics Inc. (San Diego) |
RG-101 |
MicroRNA antagonist |
Hepatitis C virus |
Interim phase II data suggested that the length of direct-acting antiviral treatment regimens for many HCV cases could be halved by adding RG-101 |
2/18/16 |
Viiv Healthcare Ltd. (London) |
Cabotegravir and rilpivirine (Johnson & Johnson) |
Two-drug regimen |
HIV |
Results from the phase IIb LATTE-2 study showed that, following 32 weeks of maintenance treatment, viral suppression rates for patients dosed every eight weeks (95%) or every four weeks (94%) were comparable to the rate observed in patients continuing with a three-drug regimen (91%) comprising cabotegravir and two nucleoside reverse transcriptase inhibitors; data from the 41-week phase IIa ÉCLAIR study evaluating cabotegravir as a monotherapy for pre-exposure prophylaxis showed that adverse events during the injection phase occurred in 98% and 90% of cabotegravir and placebo group participants, respectively |
2/25/16 |
INFLAMMATORY | |||||
Cytori Therapeutics Inc. (San Diego) |
ECCO-50 |
Cellular therapeutic |
Chronic knee pain due to osteoarthritis |
Top-line phase II ACT-OA data evaluating the safety and feasibility at 48 weeks of a single intra-articular knee injection showed it did not obtain statistical significance, although Cytori said ACT-OA was not sized or powered for statistical significance in any of the endpoints |
2/9/16 |
MISCELLANEOUS | |||||
Ablynx NV (Ghent, Belgium) |
Caplacizumab |
An anti–von Willebrand factor humanized single-variable-domain immunoglobulin |
Thrombotic thrombocytopenic purpura |
Results of the company's phase II Titan study, a single-blinded, randomized, placebo-controlled study, showed it led to a 40% reduction in median time to platelet count normalization (p=0.005) during the Titan study while reducing the use of daily plasma exchange and preventing further consumption of platelets in microthrombi and small blood vessel occlusion |
2/12/16 |
Allena Pharmaceuticals Inc. (Newton, Mass.) |
ALLN-177 |
An orally administered recombinant oxalate-degrading enzyme |
Hyperoxaluria and kidney stones |
Patient treatment has started in its 28-day phase II study |
2/11/16 |
Axovant Sciences Ltd. (Hamilton, Bermuda) |
RVT-101 |
A 5-hydroxytryptamine6 (5-HT6) serotonin receptor antagonist |
Lewy body dementia |
A 24-week phase IIb (HEADWAY-DLB) study is under way in which daily doses of 35 mg and 70 mg of RVT-101 will be evaluated; also initiated a cross-over phase II study in patients with dementia with Lewy bodies or Parkinson's disease dementia suffering from visual hallucinations |
2/11/16 |
Bone Therapeutics SA (Gosselies, Belgium) |
Allob |
An allogeneic differentiated osteoblastic cell therapy product |
Symptomatic degenerative lumbar disc |
Treated 12 patients in its Allob phase IIa spinal fusion trial without any safety concerns, and only one more cohort of four patients remains to be treated in the study |
2/18/16 |
Bone Therapeutics SA (Gosselies, Belgium) |
Allob |
Stem cells mixed with bioceramic granules |
Lumbar spinal fusion |
Early data from a pilot phase IIa trial showed the presence of bone bridges between the first treated patient's vertebrae, as demonstrated by CT scans, as early as six months after treatment; X-rays also revealed the absence of motion of the vertebral bodies, indicating a successful fusion |
2/25/16 |
Insys Therapeutics Inc. (Phoenix) |
Pharmaceutical cannabidiol |
A synthetic version of the plant-derived cannabinoid |
Infantile spasms |
The company has enrolled the first patient in a phase II study |
2/10/16 |
Mediwound Ltd. (Yavne, Israel) |
Escharex |
Based on proteolytic enzyme technology used in Nexobrid |
Dead or damaged tissue in chronic and other hard-to-heal wounds |
Disclosed positive top-line results from the second phase II trial |
2/4/16 |
Novaliq GmbH (Heidelberg, Germany) |
Cyclasol |
Ciclosporin |
Moderate to severe dry eye disease |
Started enrolling patients in a phase II trial |
2/17/16 |
Regentree LLC (joint venture of Regenerx Biopharmaceuticals Inc., Rockville, Md., and G-treebnt Co. Ltd., Korea) |
RGN-259 |
Thymosin beta 4; sterile, preservative-free eye drop |
Dry eye |
Completed enrollment in a phase IIb/III trial |
2/1/16 |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Enclomiphene |
An estrogen receptor antagonist |
Obese secondary hypogonadal men |
Fully randomized a phase II double-blind, placebo-controlled proof-of-concept study of enclomiphene in conjunction with rigorous diet and exercise; the study enrolled its target of 45 subjects |
2/18/16 |
RESPIRATORY | |||||
Allergy Therapeutics plc (Worthing, U.K.) |
Grassmatampl |
An ultra-short course subcutaneous allergen-specific immunotherapy |
Grass pollen allergy |
Completed enrollment of 250 patients for its phase IIb dose-finding study, G204, in the U.S. |
2/19/16 |
Galapagos NV (Mechelen, Belgium) |
GLPG1837 |
A CFTR potentiator drug |
Cystic fibrosis |
Started dosing in its phase II exploratory program of GLPG1837 |
2/18/16 |
|
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. | |||||