Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Amgen Inc. (Thousand Oaks, Calif.)

ABP 501

Humira biosimilar; adalimumab; a monoclonal antibody designed to block TNF-alpha

Moderate to severe rheumatoid arthritis

Phase III data showed it met the primary endpoint, demonstrating clinical equivalence when compared to Humira, as assessed by ACR20 measurements at week 24

2/5/15

Anthera Pharmaceuticals Inc. (Hayward, Calif.)

Blisibimod

A peptibody that targets B-cell activating factor

Lupus

Passed the interim futility analysis in the phase III trial CHABLIS-SC1, with findings that the trial should continue and has a "reasonable likelihood of success"

2/11/15

Celltrion Healthcare Co. Ltd. (Incheon, South Korea)

Remicade infliximab

Remicade (Johnson & Johnson) biosimilar

Crohn's disease and ulcerative colitis

Presented six prospective observational trials including 258 patients from Europe and South Korea, showing that its biosimilar is an effective and well-tolerated treatment; the clinical response to the treatment at eight weeks was greater than or equal to 87% in patients who had not been treated with anti-tumor necrosis factor (anti-TNF) therapy before and greater than or equal to 67% in patients who had switched from another anti-TNF therapy

2/23/15

Incyte Corp. (Wilmington, Del.) and Eli Lilly and Co. (Indianapolis)

Baricitinib

A once-daily, oral, selective JAK1/JAK2 inhibitor

Rheumatoid arthritis

Demonstrated a statistically significant improvement compared to placebo in a second consecutive phase III trial; the 684-subject RA-BUILD study, which included patients with moderately to severely active disease who had inadequate responses to, or were intolerant of, at least one conventional disease-modifying antirheumatic drug, met its primary endpoint of an improved ACR20 response rate compared to placebo after 12 weeks of treatment

2/24/15

CANCER

Cel-Sci Corp. (Vienna, Va.)

Multikine

Immunotherapy; leukocyte interleukin for injection

Head and neck cancer

Enrolled 24 patients into its global pivotal phase III trial in January; a total of 352 patients have been enrolled

2/3/15

Celsion Corp. (Lawrenceville, N.J.)

Thermodox

A heat-activated liposomal encapsulation of doxorubicin

Primary liver cancer

Updated results from its retrospective analysis of its 701-patient HEAT study demonstrated that in a large, well-bounded subgroup of patients (n=285, about 41% of the study patients), the combination of Thermodox and RFA provided a 59% improvement in overall survival compared to optimized RFA alone (p = 0.02)

2/24/15

Galena Biopharma Inc. (Portland, Ore.)

Neuvax

Nelipepimut-S; a HER2-directed cancer immunotherapy

Early stage, node-positive breast cancer

Reached the target enrollment of 700 patients in the randomized, double-blind, placebo-controlled phase III PRESENT (Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with Neuvax Treatment) trial

2/10/15

Genentech Inc. (South San Francisco)

Perjeta

Pertuzumab

HER2-positive metastatic breast cancer

Final overall survival (OS) results from the phase III CLEOPATRA study showed that adding Perjeta to Herceptin (trastuzumab) and chemotherapy extended the lives of people with previously untreated HER2-positive metastatic breast cancer by 15.7 months compared to Herceptin and chemotherapy (median OS: 56.5 vs. 40.8 months)

2/20/15

Pharmacyclics Inc. (Sunnyvale, Calif.)

Imbruvica

Ibrutinib

Relapsed/refractory high-risk chronic lymphocytic leukemia

Treatment with Imbruvica was associated with an 88% overall response rate, with a median time on study of 23.3 months, in 16 patients

2/18/15

CARDIOVASCULAR

Esperion Therapeutics Inc. (Ann Arbor, Mich.)

ETC-1002

Dual-action cholesterol-lowering pill

Hypercholes-terolemia

FDA has removed the peroxisome proliferator-activated receptor partial clinical hold on ETC-1002 that will now allow the company to conduct trials exceeding six months in duration

2/4/15

La Jolla Pharmaceutical Co. (San Diego)

LJPC-501

Peptide agonist of the renin-angiotensin system

Catecholamine-resistant hypotension

Reached agreement with the FDA on a special protocol assessment for its phase III study

2/10/15

Portola Pharmaceuticals Inc. (South San Francisco)

Betrixaban

An oral, once-daily Factor Xa inhibitor

To prevent venous thromboembolism or blood clots

The independent data monitoring committee recommended that the phase III APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) study of betrixaban continue as planned without modification based on the successful completion of a pre-specified futility analysis

2/9/15

CENTRAL NERVOUS SYSTEM

AB Science SA (Paris)

Masitinib

Tyrosine kinase inhibitor

Mild to moderate Alzheimer's disease

Reported the successful completion of a futility analysis related to the masitinib phase III trial; the independent data safety monitoring committee has recommended the continuation of the study

2/10/15

Biotie Therapies Corp. (Turku, Finland)

Tozadenant

Adenosine A2A receptor antagonist

Parkinson's disease

Plans to begin recruiting patients for a phase III study by mid-2015; the double-blinded study will include 882 patients experiencing levodopa-related end-of-dose "wearing-off," or motor fluctuations

2/23/15

Flexion Therapeutics Inc. (Burlington, Mass.)

FX006

A non-opioid, sustained-release, intra-articular formulation of triamcinolone acetonide

Pain associated with osteoarthritis of the knee

Dosed the first patient in the phase III trial

2/4/15

Nektar Therapeutics Inc. (San Francisco)

NKTR-181

An opioid analgesic molecule

Pain

It enrolled the first patient in SUMMIT-07, its initial phase III study

2/26/15

Xenon Pharmaceuticals Inc. (Burnaby, British Columbia) and Teva Pharmaceutical Industries Ltd. (Jerusalem)

TV-45070

A topically applied small-molecule inhibitor of the sodium channel Nav1.7 and other sodium channels

Postherpetic neuralgia

Plans to initiate a phase IIb trial, a double-blind, placebo-controlled study that will enroll approximately 330 patients

2/23/15

DIABETES

Dipexium Pharmaceuticals Inc. (New York)

Locilex

Pexiganan cream; broad-spectrum topical antibiotic peptide

Mild infections of diabetic foot ulcers

Pivotal phase III trials, OneStep-1 and OneStep-2, passed the 25% enrollment milestone

2/26/15

Regeneron Pharmaceuticals Inc. (Tarrytown, N.Y.)

Eylea

Aflibercept

Diabetic macular edema

The Protocol T study comparing Eylea with Roche AG unit Genentech's Lucentis and Avastin against moderate to severe vision loss in patients with DME; in the 660-patient study Eylea beat the other drugs specifically in patients whose eyesight is 20/50 or worse when visual acuity was measured at one year

2/20/15

GASTROINTESTINAL

Index Pharmaceuticals AB (Stockholm)

Kappaproct

Toll-like receptor 9 agonist

Moderate to severe chronic active ulcerative colitis

COLLECT, a phase III study, missed its primary endpoint of inducing clinical remission measured at week 12, defined by a clinical activity index score of ≤4, though Kappaproct showed statistically significant improvement on important secondary endpoints at weeks four and eight, including symptomatic remission (blood in stool, number of stools) and registration remission (clinical remission with concurrent endoscopic remission) as well as rate of colectomy at week 22; the drug also was well tolerated

2/4/15

INFECTION

Chimerix Inc. (Durham, N.C.)

Brincidofovir

Oral nucleotide analogue lipid-conjugate drug

Adenovirus infection

Preliminary data from the open-label phase III AdVise trial showed a short-term mortality rate of less than 40% for bone marrow transplant recipients with disseminated disease and for the patient population overall; high-risk transplant recipients who began brincidofovir with localized or asymptomatic adenovirus infection had a preliminary observed mortality rate of 11% (two of 18 subjects); a median 99% decrease in the amount of adenovirus in the blood was observed

2/13/15

Chimerix Inc. (Durham, N.C.)

Brincidofovir

A broad-spectrum antiviral that inhibits RNA polymerase

Cytomegalovirus infection in hematopoietic cell transplant

The phase III SUPPRESS trial is expected to be fully enrolled by midyear, with data expected to report early next year

2/27/15

Medivector Inc. (Boston)

Favipiravir

A broad-spectrum antiviral

Uncomplicated influenza

Completed enrollment in two FAVOR favipiravir phase III studies in adults; the trials enrolled 2,021 patients and are evaluating the time to alleviation of all primary influenza symptoms

2/18/15

Micrux Pharmaceuticals Inc. (Hayward, Calif.)

MRX-I

An oral oxazolidinone antibiotic

Gram-positive infections

Received FDA approval to initiate a phase II trial

2/26/15

Theravance Biopharma Inc. (South San Francisco)

Vibativ

Telavancin; lipoglycopeptide antibiotic

Staphylococcus aureus bacteremia

Enrolled the first patient in a phase III registrational study

2/25/15

INFLAMMATORY

Ampio Pharmaceuticals Inc. (Englewood, Colo.)

Ampion

Aspartyl-alanyl diketopiperazine

Moderate to severe osteoarthritis of the knee

STRUT study results showed that WOMAC A pain score, the primary endpoint for FDA approval, improved by 64% from baseline to 20 weeks in patients who received Ampion, by -1.41 (0.81) compared to saline vehicle control, where improvement was -0.85 (0.64) (p = 0.0231)

2/26/15

MISCELLANEOUS

Adma Biologics Inc. (Ramsey, N.J.)

RI-002

Intravenous immunoglobulin products

Primary immune deficiency disease

Pivotal phase III data showed it met its primary endpoint with zero serious bacterial infections

2/24/15

Amgen Inc. (Thousand Oaks, Calif.)

AMG 416

A fully human antibody created using Abgenix's Xenomouse technology

Hyperpara-thyroidism in chronic kidney disease

Findings from the head-to-head phase III study comparing AMG 416 with cinacalcet showed it met the primary endpoint of noninferiority

2/27/15

Amicus Therapeutics Inc. (Cranbury, N.J.)

Migalastat

Oral small-molecule chaperone

Fabry disease

Phase III data showed reductions in disease substrate, stability of kidney function and improvement in a key cardiac parameter in patients with amenable mutations

2/11/15

Auspex Pharmaceuticals Inc. (La Jolla, Calif.)

SD-809

A small molecule inhibitor of vesicular monoamine 2 transporter

Tardive dyskinesia

Completed target enrollment in its pivotal clinical trial

2/4/15

Chiasma Inc. (Jerusalem)

Octreotide

Capsules

Acromegaly

Results from a multicenter phase III study that enrolled 155 people diagnosed with acromegaly and managed on somatostatin analogs showed that 65% of patients who received octreotide capsules twice-a-day achieved disease control, as measured by circulating concentrations of insulin-like growth factor-1 and growth hormone concentrations, at seven months, meeting the primary endpoint and 62% achieved disease control at 13 months

2/10/15

Genzyme (unit of Sanofi SA; Paris)

Cerdelga

Eliglustat

Gaucher disease type 1

Phase III ENGAGE data demonstrated that patients who switched from placebo to eliglustat showed improvements similar to the eliglustat-treated patients during the primary analysis while the eliglustat-treated patients continued to show improvements during the extension period; further data demonstrated a statistically significant reduction from baseline in spleen size by a mean of 28% compared in Cerdelga patients with a mean increase of 2% in placebo patients, for an absolute difference of 30% (p<0.0001); during the 12-month extension period for ENCORE, patients who crossed over to eliglustat from imiglucerase (Cerezyme) remained stable, as did patients treated with eliglustat for 24 months

2/13/15

Mesoblast Ltd. (Melbourne, Australia)

MSC-100-IV

Mesenchymal cell therapy candidate

Severe steroid-resistant acute graft-vs.-host disease

Results from a study of 160 children "continue to show a clear and meaningful survival benefit among responding pediatric bone marrow transplant recipients," with overall response rate at day 28 or later of 64%; the response has been correlated with statistically significant improved survival compared to nonresponders at day 100 or later after an MSC-100-IV infusion (81% vs. 39%, p = 0.0001)

2/18/15

Vital Therapies Inc. (San Diego)

VTI-208

ELAD System, an extracorporeal bio-artificial liver therapy

Alcohol-induced liver decompensation

Achieved the 200-subject enrollment target in the phase III trial

2/2/15

RESPIRATORY

Sunovion Pharmaceuticals Inc. (Marlborough, Mass.)

SUN-101

Glycopyrrolate; nebulized long-acting muscarinic antagonist

Chronic obstructive pulmonary disease

Opened the final two studies in a pivotal program that will enroll 2,340 adults

2/26/15

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

FF/UMEC/VI

Triple combination of fluticasone furoate, umeclidinium and vilanterol

Chronic obstructive pulmonary disease (COPD)

Started a second global phase III study, known as FULFIL (Lung FUnction and quality of LiFe assessment in COPD with closed trIpLe therapy), that will enroll approximately 1,800 patients across some 180 centers

2/10/15

Notes

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The date indicated refers to the BioWorld Today issue in which the news item can be found.

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