Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Biogen Idec Inc. (Weston, Mass.)

Daclizumab

Once-monthly, subcutaneous daclizumab

Relapsing-remitting multiple sclerosis

Phase III DECIDE data showed it hit the study's primary endpoint, demonstrating a statistically significant 45% reduction in annualized relapse rate (p < 0.0001) compared to the company's once-weekly intramuscular Avonex (interferon beta-1a)

6/17/14

Can-Fite Biopharma Ltd. (Petach Tikva, Israel)

A3 adenosine receptor agonist

CF101

Psoriasis

Finalized enrollment in its phase II/III trial

6/18/14

Celgene Corp. (Summit, N.J.)

Otezla

Apremilast; oral, selective inhibitor of phosphodiesterase 4

Psoriatic arthritis

Phase III data demonstrated that treatment with Otezla improved measures of psoriatic arthritis disease activity, including tender and swollen joints, compared to placebo at 16 weeks, with all measurement of disease activity showing sustained improvements through week 52 for patients continuously treated with Otezla

6/12/14

Coherus Biosciences Inc. (Redwood City, Calif.)

CHS-0214

Etanercept biosimilar

Rheumatoid arthritis

Started a phase III trial

6/25/14

Epirus Pharmaceuticals Inc.

BOW015

Biosimilar to the antitumor necrosis factor therapy Remicade (infliximab, Johnson & Johnson)

Rheumatoid arthritis

Phase III results showed that BOW015 compared favorably to the branded drug in a phase III equivalence trial

6/13/14

Genmab A/S (Copenhagen) and Glaxosmithkline plc (London)

Ofatumumab

Subcutaneous formulation of CD2-targeting monoclonal antibody

Relapsing-remitting multiple sclerosis and neuromyelitis optica

Decided to start additional pivotal studies, including a phase III in MS, and a pivotal study in NMO

6/2/14

Hospira Inc. (Lake Forest, Ill.)

Inflectra

Monoclonal antibody; biosimilar infliximab

Rheumatoid arthritis and ankylosing spondylitis

Data showed it demonstrated comparable and sustained reductions in disease activity to European reference product Remicade (infliximab, Johnson & Johnson) over one year

6/16/14

Redhill Biopharma Ltd. (Tel-Aviv, Israel)

RHB-104

An antibiotic combination therapy in oral capsule formulation

Crohn's disease

Started a long-term population pharmacokinetic study as part of the ongoing RHB-104 MAP U.S. study; product is in phase III testing

6/6/14

Regeneron Pharmaceuticals Inc. (Tarrytown, N.J.) and Sanofi SA (Paris)

Sarilumab

Fully human monoclonal antibody directed against the alpha subunit of the IL-6 receptor complex

Rheumatoid arthritis

Phase III data showed it increased major clinical response rates, defined as achieving an ACR70 for at least 24 consecutive weeks, and resulted in sustained improvement in signs and symptoms of RA after 52 weeks, meeting secondary endpoints of the trial

6/13/14

Veloxis Pharmaceuticals A/S (Horsholm, Denmark)

Envarsus XR

Tacrolimus extended-release tablets

Kidney transplant

Reported two-year results of its phase III study, Study 3002, testing in de novo kidney transplant patients, which continued to demonstrate noninferiority compared to Prograf

6/24/14

CANCER

Astellas Pharma US Inc. (subsidiary of Astellas Pharma Inc.; Tokyo) and Medivation Inc. (San Francisco)

Enzalutamide

Cancer treatment

Metastatic prostate cancer

Phase III PREVAIL trial showed that men treated with enzalutamide demonstrated a statistically significant delay in cancer progression and reduction in the risk of death compared with placebo

6/3/14

Auspex Pharmaceuticals Inc. (La Jolla, Calif.)

SD-809

Deutetrabenazine

Chorea associated with Huntington's disease

Results from an interim subgroup analysis of the ARCH-HD Switch study indicated that SD-809 maintained chorea control in those subjects; the conversion was generally well tolerated, with no adverse events of loss of chorea control and no serious adverse events reported

6/25/14

Celator Pharmaceuticals Inc. (Ewing, N.J.)

CPX-351

Cytarabine; daunorubicin liposome injection

High-risk acute myeloid leukemia

Achieved 75% enrollment in its ongoing phase III trial; completed safety review and study will continue as planned

6/5/14

Clovis Oncology Inc. (Boulder, Colo.)

CO-1686

Next-generation EGFR inhibitor

Lung cancer

Early data from 72 patients treated with CO-1686 showed that hyperglycemia and impaired glucose tolerance was found to be the most common adverse effect, with 16 patients (22%) experiencing grade 3 effects requiring treatment; the company attributed it to a metabolite; dosed the first patient in the TIGER2 study

6/3/14; 6/24/14

Galena Biopharma Inc. (Portland, Ore.)

GALE-301

A folate-binding protein peptide immunotherapy

Ovarian and endometrial cancer

Completed enrollment in its phase IIa trial

6/18/14

Genmab A/S (Copenhagen) and Glaxosmithkline plc (London)

Arzerra

Ofatumumab

Bulky fludarabine-refractory chronic lymphocytic leukemia

Phase III data showed it did not meet its primary endpoint of progression-free survival (PFS); the median PFS, as assessed by the Independent Review Committee, was 5.36 months for the ofatumumab group and 3.61 months for physicians' choice (p = 0.267)

6/30/14

Ligand Pharmaceuticals Inc. (San Diego) and Glaxosmithkline plc (London)

Promacta in combination with Vidaza

Eltrombopag and azacitidine

Myelodysplastic syndromes

Started a phase III study to evaluate the platelet supportive care effects of the combo, the current standard of care, vs. placebo plus Vidaza in intermediate-1, intermediate-2 or high-risk patients

6/26/14

Merrimack Pharmaceuticals Inc. (Cambridge, Mass.)

MM-398

A nanoliposomal encapsulation of irinotecan

Pancreatic cancer

Data from the NAPOLI-1 phase III trial showed that the combination of MM-398 plus 5-fluorouracil(5-FU) and leucovorin extended overall survival (OS), achieving OS of 6.1 months vs. 4.2 months in the control arm of 5-FU and leucovorin alone (p = 0.012.)

6/27/14

Navidea Biopharmaceuticals Inc. (Dublin, Ohio)

Lymphoseek

Agent used for sentinel lymph node mapping

Multiple tumor types

Phase III data indicated that the agent's sensitivity for sentinel lymph node mapping was consistent across the tumor type studies, regardless of whether surgery was conducted on the same day as, or on the day after injection of Lymphoseek

6/12/14

Northwest Biotherapeutics Inc. (Bethesda, Md.)

Dcvax-L

Dendritic cell-based vaccine

Prostate cancer

Started the phase III trial in Germany; to date, the trial has been enrolling in parallel in the U.S. and Europe

6/11/14

Psioxus Therapeutics Ltd. (Oxford, UK)

Enadeno-tucirev

Oncolytic vaccine; previously ColoAd1

Platinum-resistant ovarian cancer

Dosed the first patient in the OCTAVE (Ovarian Cancer Treated with Adeno Vaccine Enadenotucirev) phase I/II study

6/18/14

Tesaro Inc. (Waltham, Mass.)

Rolapitant

Neurokinin-1 receptor antagonist

To prevent chemotherapy-induced nausea and vomiting

Data from the 1,369-patient phase III trial, in which about half of patients were treated with anthracycline-based regimens for breast cancer, 71.3% of rolapitant-treated patients met the primary endpoint of complete response, defined as no emesis and no use of rescue medication during the delayed phase following administration of chemotherapy vs. 61.6% in the control group (p = 0.001)

6/30/14

CARDIOVASCULAR

Actelion Ltd. (Allschwil, Switzerland)

Selexipag

A first-in-class selective oral prostacyclin IP receptor agonist

Pulmonary arterial hypertension

Met the primary endpoint of a phase III outcomes trial in 1,156 patients; those on the drug had a 39% reduction in risk of experiencing a morbidity or mortality event vs. those on placebo; the result was highly statistically significant (p < 0.0001)

6/17/14

Amarin Corp. plc (Dublin)

Vascepa

Icosapent ethyl capsules

High cholesterol

Results of a retrospective analysis of patient cases that examined the drug's effect on lipids in hyperlipidemic patients who were switched from Lovaza (omega-3-acid ethyl esters, Glaxosmithkline plc) capsules to Vascepa showed that 12 patients experienced a decrease in triglyceride and low-density lipoprotein cholesterol levels and 13 patients experienced a decrease in triglyceride and non-high-density lipoprotein cholesterol levels

6/12/14

Amgen Inc. (Thousand Oaks, Calif.)

AMG 145

Cholesterol-lowering medication, evolocumab

Homozygous familial hypercholes-terolemia and premature cardiovascular disease

Phase III TESLA data showed that adding evolocumab 420 mg subcutaneous monthly to a stable dose of statin therapy and other lipid-lowering medications significantly reduced LDL-C by 31% (p < 0.001) from baseline at week 12 compared to placebo

6/3/14

Chelsea Therapeutics International Ltd. (Charlotte, N.C.)

Northera

Droxidopa

Neurogenic orthostatic hypotension

Results from its pivotal phase III 301 study showed how the drug demonstrated a statistically significant difference in efficacy compared to placebo for improving symptoms

6/23/14

Mast Therapeutics Inc. (San Diego)

MST-188

A purified form of a nonionic, triblock copolymer (poloxamer 188)

 

Sickle cell disease

Started patient enrollment in a substudy within EPIC, its pivotal phase III study, to investigate the effect of the drug on tissue oxygenation, which will be measured using a non-invasive, FDA-approved device

6/17/14

Taxus Cardium Pharmaceuticals Group Inc. (San Diego)

Generx

Alferminogene tadenovec, Ad5FGF-4

Myocardial ischemia

Safety and efficacy data showed that eight weeks following administration, patients had significant improvements in myocardial blood flow under stress, and the degree of improvement was of a magnitude similar to that observed following coronary interventions such as bypass surgery or angioplasty and stenting

6/25/14

CENTRAL NERVOUS SYSTEM

Adamas Pharmaceuticals Inc. (Emeryville, Calif.)

ADS-5102

An extended-release formulation of generic drug amantadine

Parkinson's disease

Started a pivotal phase III study

6/11/14

Alkermes plc (Dublin)

ALKS 5461

Once-daily, oral adjunctive treatment

Major depressive disorder

Started the FORWARD-3 and FORWARD-4 trials, two of the three planned phase III core efficacy studies

6/11/14

Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.)

AVP-825

A drug-device combination product consisting of low-dose sumatriptan powder delivered intranasally using Breath Powered delivery technology

Acute migraines

COMPASS, its phase IIIb trial comparing the efficacy and safety of AVP-825 22 mg to sumatriptan 100-mg tablets, met its primary endpoint; results showed that migraine sufferers achieved greater pain relief within 30 minutes of treatment with AVP-825 vs. sumatriptan (p < 0.0001) and achieved greater pain freedom in a greater proportion of migraine attacks at 15 minutes, 30 minutes, 45 minutes, 60 minutes and 90 minutes post-dose (p < 0.05)

6/11/14

Neuralstem Inc. (Germantown, Md.)

NSI189

Neurogenic compound

Major depressive disorder

Reported two sets of clinical data, showing significantly increased brain wave patterns in the hippocampal region of the brain and increased electrical coherence in the prefrontal cortical region; data also showed that the combined treatment group had statistically significant improvement (p = 0.02) after 28 days of the drug treatment compared to the placebo control group

6/26/14

Neurim Pharmaceuticals Ltd. (Tel Aviv, Israel)

Circadin

Prolonged-release melatonin 2 mg

Alzheimer's disease

Data showed that patients treated with Circadin for six months had significantly better cognitive performance vs. those on placebo

6/20/14

Orexo AB (Uppsala, Sweden)

Zubsolv

A sublingual formulation of buprenorphine and naloxone

Opioid dependence

Data from a phase III study demonstrated that Zubsolv is as effective of Suboxone film

6/26/14

DIABETES

Ampio Pharmaceuticals Inc. (Greenwood Village, Colo.)

Optina

Based on the low dose of the weak androgen, low-molecular-weight lipophilic steroid danazol

Diabetic macular edema

Informed the FDA's Transplant and Ophthalmology Division of its intent to reduce the patient sample size in the OptimEyes Study, since the present enrollment of more than 355 patients provides an adequate power of 88% which the company said it believes is more than sufficient for statistical evaluation

6/26/14

Biodelivery Sciences International Inc. (Raleigh, N.C.)

Clonidine

Topical gel

Diabetic neuropathy

Completed patient enrollment for its initial phase III trial

6/27/14

Nephrogenex Inc. (Research Triangle Park, N.C.)

Pyridoxamine dihydro-chloride

Inhibits formation of harmful advanced glycation end-products; derivative of vitamin B6

Diabetic nephropathy

Entered a pivotal program

6/20/14

GASTROINTESTINAL

Index Pharmaceuticals AB (Stockholm)

Kappaproct

A Toll-like receptor 9 agonist

Ulcerative colitis

Phase III data showed it missed the primary endpoint, the induction of clinical remission according to the Clinical Activity Index; it hit several key secondary endpoints, including a reduction in the colectomy rate, symptomatic remission – reduced blood in stool and reduced number of stools – and what the company called "registration remission," which combines elements of the CAI and the Disease Activity Index (DAI)

6/30/14

INFECTION

Amvac AG (Zug, Switzerland)

Gynevac

Vaccine

Bacterial vaginosis

Started a phase III study

6/18/14

Cubist Pharmaceuticals Inc. (Lexington, Mass.)

Sivextro

Tedizolid phosphate

Acute bacterial skin and skin structure infections and complicated skin and soft tissue infections

Positive results from ESTABLISH-2, a pivotal phase III trial met the primary and secondary endpoints defined by the FDA and European Medicines Agency

6/9/14

Durata Therapeutics Inc. (Chicago)

Dalvance

Dalbavancin

Acute bacterial skin and skin structure infections

Phase III data from its DISCOVER program highlight the benefits of dalbavancin and its potential to change the treatment paradigm

6/6/14

Medimetriks Pharmaceuticals Inc. (Fairfield, N.J.) and Grupo Ferrer Internacional SA (Barcelona, Spain)

Ozenoxacin

A bactericidal nonfluorinated quinolone

Impetigo

Initiated the second phase III trial comparing Ozenoxacin 1% cream vs. placebo; it will enroll more than 400 patients

6/13/14

Tetraphase Pharmaceuticals Inc. (Watertown, Mass.)

Eravacycline

Intravenous and oral formulations

Complicated urinary tract infections

Completed patient enrollment in the lead-in portion of its IGNITE 2 trial, a two-part phase III study

6/20/14

The Medicines Co. (Parsippany, N.J.)

Orbactiv

Oritavancin; antibiotic

Acute bacterial skin and skin structure infections

Results from its SOLO I phase III trial showed that a single 1,200 mg intravenous dose was noninferior to twice-daily intravenous dosing of vancomycin given for seven to 10 days

6/6/14

INFLAMMATORY

Iroko Pharmaceuticals LLC (Philadelphia)

Solumatrix

Meloxican 5 mg and 10 mg; a low-dose nonsteroidal anti-inflammatory drug

Osteoarthritis

Phase III data showed treated patients had significantly greater pain relief compared to placebo

6/13/14

MISCELLANEOUS

Alexion Pharmaceuticals Inc. (Cheshire, Conn.)

Soliris

Eculizumab

Atypical hemolytic uremic syndrome

In three separate subgroup analyses, it resulted in clinically meaningful improvements in key hematologic markers of complement-mediated thrombotic microangiopathy and reversal of renal damage in patients with aHUS, regardless of kidney transplant history or baseline dialysis status

6/3/14

Clarus Therapeutics Inc. (Northbrook, Ill.)

Rextoro

Testosterone undecanoate

Male hypogonadism

Phase III data from two studies showed it met its endpoints in both studies, and both trials demonstrated testosterone replacement and with the revised dose-titration algorithm the Cmax was comparable to other testosterone replacement therapies

6/24/14

Cytonet GmbH & Co. KG (Weinheim, Germany)

LCT

Liver cell therapy

Urea cycle disorders

New data found it may help temporarily stabilize pediatric patients while they await liver transplantation; Cytonet has two ongoing multicenter clinical trials in U.S. and Canada (SELICA III), and in Germany (SELICA V)

6/3/14

Hyperion Therapeutics Inc. (Brisbane, Calif.)

Ravicti

Glycerol phenylbutyrate oral liquid

Urea cycle disorders

EMA completed technical and content validation and accepted the filing of a an MAA

6/27/14

Kala Pharmaceuticals Inc. (Waltham, Mass.)

KPI-121

Nanotechnology-based loteprednol etabonate MPP candidate

Inflammation and pain associated with cataract surgery and dry eye disease

Started two trials, including a phase III study (KPI-121-C-001) designed to evaluate the safety and efficacy of the drug in managing inflammation and pain associated with cataract surgery, and a phase II trial (KPI-121-C-002) in patients with dry eye disease

6/20/14

Ophthalix Inc. (subsidiary of Can-Fite Biopharma Ltd.; Petach Tikva, Israel)

CF101

An A3 adenosine receptor agonist

Dry eye syndrome

Terminated development of CF101 based on a lack of correlation between patients' response to CF101 and overexpression of the drug target, and following last year's phase III trial failure, in which the drug met neither primary nor secondary efficacy endpoints

6/10/14

Raptor Pharmaceutical Corp. (Novato, Calif.)

Procysbi

Gastroresistant hard capsules of cysteamine (as mercaptamine bitartrate)

Nephropathic cystinosis

Its open-label extension study demonstrated that patients treated for two years were able to sustain optimal cystine control in their white blood cells and preserved kidney function over the long term, and had significant improvements in social, school and total functioning

6/25/14

Recro Pharma Inc. (Malvern, Pa.)

Dex-IN

An intranasal formulation of dexmedetomidine

For patients undergoing bunionectomy surgery

Dosed the first patient in a phase IIb

6/18/14

Uniqure NV (Amsterdam)

Glybera

Gene therapy; alipogene tiparvovec

Lipoprotein lipase deficiency

Full analysis from the six-year follow-up data demonstrated that after a single administration of Glybera, patients have experienced reductions in both the frequency and severity of pancreatitis; specifically, data showed that those patients had a range of about 40% to 50% lower post-treatment documented pancreatitis events and abdominal pain events consistent with pancreatitis, with no severe pancreatitis up to six years post-treatment

6/4/14

ZS Pharma Inc. (Coppell, Texas)

ZS005

Designed to maintain normal serum potassium levels

Hyperkalemia

Started enrolling patients in ZS005, a phase III open-label, 12-month, long-term maintenance study

6/27/14

RESPIRATORY

Alk-Abello A/S (Copenhagen) and Torii Pharmaceutical Co. Ltd. (Tokyo)

SLIT-tablet

Sublingual allergy immunotherapy tablet

House dust mite allergy-induced respiratory disease

Completed its second of two phase III trials confirming that the treatment was well tolerated; the study did not meet its primary endpoint of showing statistically significantly lower risk of moderate to severe asthma exacerbations when compared to patients receiving placebo

6/17/14

Aradigm Corp. (Hayward, Calif.)

Pulmaquin

Inhaled ciprofloxacin

Noncystic fibrosis bronchiectasis

The first patient was dosed in ORBIT-4, the second of two pivotal phase III trials

6/24/14

Theravance Inc. (South San Francisco) and Glaxosmithkline plc (London)

Incruse Ellipta

Umeclidinium 62.5 mcg or umeclidinium 125 mcg

Chronic obstructive pulmonary disease

Phase III data showed it hit the primary endpoint

6/12/14

Vertex Pharmaceuticals Inc. (Boston)

Combination of VX-809 and Kalydeco

Lumacaftor and ivacaftor

Cystic fibrosis

Phase III data showed marked improvements in lung function, and it hit multiple secondary endpoints

6/25/14

Vertex Pharmaceuticals Inc. (Boston)

Kalydeco

Ivacaftor

Cystic fibrosis

Phase III data showed that treatment with Kalydeco reduced the annual loss of lung function by half over three years compared to similar untreated CF patients

6/13/14


Notes

SPA = Special protocol assessment.

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.