Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Epirus Biopharmaceuticals Inc. (Boston)

BOW015

Infliximab, reference biologic Remicade

Active rheumatoid arthritis

Initiated its global registration study for BOW015; the company plans to enroll more than 500 patients in the 58-week UNIFORM study, which will be conducted at sites in Europe, North America and Latin America

2/10/16

Novan Inc. (Durham, N.C.)

SB204

Topical nitric-oxide gel

Acne

Kicked off its phase III program

2/24/16

CANCER

Amgen Inc. (Thousand Oaks, Calif.)

Blincyto

Blinatumomab

Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia

Results of a prespecified interim analysis showed that the primary endpoint of improved overall survival was met in a phase III study, called Tower

2/8/16

Amgen Inc. (Thousand Oaks, Calif.)

Aranesp

Darbepoetin alfa

Myelodysplastic syndromes

The phase III Aranesp ARCADE trial met its primary endpoint of reducing the incidence of red blood cell transfusions in anemic patients with low and intermediate-1 risk myelodysplastic syndromes at the end of the blinded 25-week study period

2/17/16

Biofrontera AG (Leverkusen, Germany)

BF-200 ALA

Topical treatment

Actinic keratosis

Data from three pivotal phase III trials demonstrated patient clearance rates of 85% to 91% in the topical treatment of actinic keratosis

2/12/16

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

Vyxeos

Cytarabine, daunorubicin liposome for injection; CPX-351

High-risk acute myeloid leukemia

The phase III trial reached its prespecified number of events required for the analysis of overall survival

2/17/16

Cel-Sci Corp. (Vienna, Va.)

Multikine

Leukocyte interleukin injection

Advanced primary head and neck cancer

Enrolled 29 patients in an ongoing phase III trial, bringing the total to 697

2/2/16

Chugai Pharmaceutical Co. Ltd. (Tokyo) and Genentech Inc. (South San Francisco)

Alecensa

Alectinib

Non-small-cell lung cancer

An independent data monitoring committee suggested stopping a phase III trial comparing Alecensa and Xalkori (crizotinib, Pfizer Inc.) early, as the study met its endpoint in the 207-patient Japanese trial, called J-ALEX

2/11/16

Eisai Co. Ltd. (Tokyo)

Havalen

Eribulin

Unresectable locally advanced liposarcomas and leiomyosarcomas

Phase III data demonstrated that Havalen improved median overall survival compared to the chemotherapy dacarbazine

2/12/16

Exelixis Inc. (South San Francisco)

Cabozantinib

Multityrosine kinase inhibitor

Second-line renal cell carcinoma

Added increased overall survival vs. Afinitor (everolimus, Novartis AG) to the list of benefits; the data, from a second interim analysis of the company's phase III METEOR trial, adds to earlier information that showed cabozantinib besting Afinitor on progression-free survival and objective response measures in July and also hinting at what is now statistically significant

2/2/16

Ferring Pharmaceuticals SA (Saint Prex, Switzerland)

Degarelix

GnRH receptor antagonist

Prostate cancer and cardiovascular disease

Initiated the phase IIIb PRONOUNCE trial to compare, over a one-year treatment period, the occurrence of major adverse cardiovascular events in patients receiving degarelix to patients receiving the GnRH receptor agonist, leuprolide

2/9/16

Helsinn Group (Lugano, Switzerland)

Anamorelin

Ghrelin receptor agonist

Cancer anorexia-cachexia in non-small-cell lung cancer

Pivotal ROMANA phase III trials showed that anamorelin improved, in respect to placebo, lean body mass and body weight in addition to symptom burden, including appetite

2/24/16

Incyte Corp. (Wilmington, Del.)

Jakafi

Ruxolitinib; a first-in-class JAK1/JAK2 inhibitor

Advanced or metastatic pancreatic cancer

Discontinued the phase III study (JANUS 1) of ruxolitinib or placebo in combination with capecitabine for second-line treatment, following a planned inteirm analysis that did not show a sufficient level of efficacy to warrant continuation

2/12/16

Navidea Biopharmaceuticals Inc. (Dublin, Ohio)

Lymphoseek

Technetium Tc 99m tilmanocept

Cervical cancer

The first patient has been enrolled in a study evaluating Lymphoseek injection

2/12/16

OBI Pharma Inc. (Taipei, Taiwan)

OBI-822/821

Active immunotherapy

Metastatic breast cancer

Top-line global phase II/III data showed it missed its primary endpoints, but the phase II part of the study showed a proof-of-concept immune response

2/29/16

Ose Pharma SA (Paris)

Tedopi

Immunotherapy designed to work by activating T lymphocytes

Non-small-cell lung cancer

Initiated in the U.S. its phase III trial; treated the first patients in its Atalante 1 phase III trial

2/5/16; 2/23/16

Peregrine Pharmaceuticals Inc. (Tustin, Calif.)

Bavituximab

Targets phosphatidylserine

Non-small-cell lung cancer

Halted the phase III SUNRISE trial evaluating bavi plus docetaxel compared to docetaxel alone after the bavi group showed insufficient improvement in overall survival to warrant continuation; the analysis was conducted after 33% of targeted events (patient deaths) in the study were reached

2/29/16

Spectrum Pharmaceuticals Inc. (Henderson, Nev.)

SPI-2012

Eflapegrastim; long-acting G-CSF

Chemotherapy-induced neutropenia in breast cancer

Started the registrational trial, ADVANCE, as part of an SPA; it will enroll 580 newly diagnosed, early stage patients

2/1/16

Steba Biotech (Luxembourg)

Tookad

Padeliporfin di-potassium

Localized prostate cancer and other solid tumors

Positive top-line results from PCM304, its Latin American phase III trial, showed that the primary endpoint has been met, demonstrating that Tookad increased the rate of cancer-free patients at 12 months

2/2/16

VBL Therapeutics Inc. (Tel Aviv, Israel)

VB-111

Ofranergene obadenovec

Recurrent glioblastoma

Enrolled the first patient in Israel, as part of the GLOBE phase III pivotal trial

2/23/16

CARDIOVASCULAR

Amgen Inc. (Thousand Oaks, Calif.)

Repatha

Evolocumab

High cholesterol

The phase III GAUSS-3 trial in patients who cannot tolerate statins met its co-primary endpoints: mean percent reductions from baseline in low-density lipoprotein cholesterol (LDL-C) at weeks 22 and 24, and the percent reduction from baseline in LDL-C at week 24

2/8/16

Jazz Pharmaceuticals plc (Dublin)

Defibrotide

Branded as Defitelio in the EU

Hepatic veno-occlusive disease

Pivotal phase III data showed that defibrotide use in patients with hepatic veno-occlusive, also known as sinusoidal obstruction syndrome, with multi-organ failure post-hematopoietic stem cell transplantation was associated with a statistically significant improvement in day +100 survival and in rate of complete response by day +100, compared with historical controls

2/2/16

Mast Therapeutics Inc. (San Diego)

MST-188

Veploxamer

Sickle cell disease experiencing vaso-occlusive crisis

Completed enrollment in its phase III study

2/23/16

Tenax Therapeutics Inc. (Morrisville, N.C.)

Levosimendan

Calcium sensitizer

Cardiac surgery

Provided an update for its ongoing phase III LEVO-CTS trial; the independent data and safety monitoring board completed its review of the first 254 patients enrolled and recommended that Tenax continue with the trial as planned without any alterations

2/2/16

CENTRAL NERVOUS SYSTEM

Abbvie Inc. (North Chicago) and Neurocrine Biosciences Inc. (San Diego)

Elagolix

A selective, orally active, short-duration nonpeptide antagonist of the gonadotropin-releasing hormone receptor

Endometriosis

Top-line results from the second of two pivotal phase III trials show that after six months of continuous treatment, both doses of Elagolix met the study's co-primary endpoints reducing scores of menstrual pain and non-menstrual pelvic pain associated with endometriosis

2/11/16

Acelrx Pharmaceuticals Inc. (Redwood City, Calif.)

ARX-04

A non-invasive candidate consisting of 30 mcg sufentanil tablets delivered sublingually via a disposable, pre-filled, single-dose applicator

Moderate to severe acute pain associated with trauma or injury

Interim results from the single-arm, open-label phase III study showed a substantial clinical reduction in pain intensity, resulting in a SPID1 value that is similar to previous studies of sublingual sufentanil in postoperative patients; patients treated with one dose of ARX-04 experienced a mean decrease from baseline of 2.7 on a 0 – 10 numeric rating scale for pain intensity one hour after dosing

2/26/16

Algobate AG (Basel, Switzerland)

NLX PRT

Prolonged-release, once-daily, oral tablet formulation of naloxone

Opioid-induced constipation in chronic noncancer pain

Completed an end-of-phase II/pre-phase III meeting with the FDA and said it believes the results of the two proposed phase III trials, together with other data in the NLX PRT development program and relevant reference products, could form the basis of a new drug application via a 505(b)(2) pathway; it's preparing to start the phase III program this year

2/29/16

Alkermes plc (Dublin)

ALKS 3831

Combines olanzapine with the opioid antagonist samidorphan

Schizophrenia

Launched the second of two phase III studies, ENLIGHTEN-2

2/11/16

Cara Therapeutics Inc. (Shelton, Conn.)

CR845

Intravenous therapy

Postoperative pain

FDA protocol hold of its phase III adaptive pivotal trial was triggered by a pre-specified, conservative measure of previously seen sodium levels

2/29/16

Histogenics Corp. (Waltham, Mass.)

Neocart

A cartilage-like, tissue-engineered implant created from a patient's own cartilage cells

Full thickness knee cartilage defects

Enrolled 123, or just over half, of the 245 patients required to complete enrollment of its ongoing phase III trial being conducted under an SPA with the FDA

2/4/16

Recro Pharma Inc. (Malvern, Pa.)

N1539

Intravenous meloxicam, a long-acting, preferential COX-2 inhibitor

Acute postoperative pain following bunionectomy surgery

The first patient was dosed in the pivotal phase III trial

2/2/16

Shionogi & Co. Ltd. (Osaka, Japan)

Naldemedine

Oral, peripherally acting mu-opioid receptor antagonist

Opioid-induced constipation in chronic noncancer pain

Pivotal phase III COMPOSE I study showed that once-daily treatment improved opioid-induced constipation compared to placebo; 47.6% of patients taking naldemedine once-daily at 0.2 mg were considered responders, compared to 34.6% of patients on placebo over 12 weeks; it was generally well tolerated

2/22/16

DIABETES

Novo Nordisk A/S (Bagsvaerd, Denmark)

Tresiba

Insulin degludec

Type 2 diabetes

Headline results from SWITCH2, the first of 2x32-week, randomized, double-blind, crossover, treat-to-target trials testing Tresiba against insulin glargine showed, from a mean baseline of 7.6%, noninferiority in HbA1c reduction, fulfill the requirements for objectively comparing hypoglycemia rates between the two treatments

2/1/16

Novo Nordisk A/S (Bagsvaerd, Denmark)

Semaglutide

GLP-1 therapy

Type 2 diabetes

Top-line data from the fifth phase IIIa trial, SUSTAIN 5, which enrolled 397 patients, achieved its primary objective by demonstrating that individuals treated with 0.5 mg and 1.0 mg semaglutide achieved a statistically significant and superior improvement in HbA1c of 1.4% and 1.8%, respectively, from a mean baseline HbA1c of 8.4%, compared with an improvement in HbA1c of 0.1% for those who received placebo

2/24/16

Novo Nordisk A/S (Bagsvaerd, Denmark)

Tresiba

Insulin degludec

Type 1 diabetes

Top-line SWITCH 1 data comparing Tresiba to Lantus (insulin glargine, Sanofi SA) showed the trial met the primary endpoint by showing noninferiority in HbA1c reduction

2/24/16

GASTROINTESTINAL

Atlantic Healthcare plc (Cambridge, U.K.)

Alicaforsen

Antisense drug; targets intercellular adhesion molecule 1

Ulcerative colitis with pouchitis

Began recruitment onto a pivotal phase III trial

2/11/16

INFECTION

Bristol-Myers Squibb Co. (New York)

Daclatasvir and asunaprevir

A pan-genotypic NS5A complex inhibitor and an NS3/4A protease inhibitor

Genotype 1b hepatitis C virus

Reported that 91% of patients from China treated in a phase III trial for 24 weeks achieved a sustained virologic response after treatment

2/23/16

Cellceutix Corp. (Beverly, Mass.)

Brilacidin

Single-dose antibiotic, an HDP mimic

Acute bacterial skin and skin structure infections caused by gram-positive bacteria

Submitted a special protocol assessment request, along with a final protocol, to the FDA for a phase III trial

2/29/16

Cempra Inc. (Chapel Hill, N.C.)

Solithromycin

Oral capsules

Community-acquired bacterial pneumonia

Pivotal phase III results showed it met the primary objective of statistical noninferiority to oral moxifloxacin to treat CABP, with a treatment success rate 72 hours after the first dose of study drug of 78.2% for solithromycin and 77.9% for moxifloxacin

2/8/16

Chimerix Inc. (Durham, N.C.)

Brincidofovir

Oral nucleotide analog

To prevent cytomegalovirus

Phase III SUPPRESS data showed that it did not meet the primary endpoint of prevention of clinically significant CMV infection at Week 24 following hematopoietic cell transplantation; however, a clear antiviral effect was seen at the end of the on-treatment period at Week 14, with patients who received brincidofovir experiencing fewer clinically significant CMV infections than patients in the placebo group (24% vs. 38%, p=0.002)

2/23/16

Gilead Sciences Inc. (Foster City, Calif.)

F/TAF

Fixed-dose combination of emtricitabine and tenofovir alafenamide

HIV

Phase III 48-week data showed that regimens containing F/TAF (200/10 mg and 200/25 mg) were found to be statistically noninferior to the regimens containing emtricitabine and tenofovir disoproxil fumarate (F/TDF; Truvada); data also demonstrated statistically significant improvements in renal and bone laboratory parameters among patients receiving F/TAF-based regimens

2/24/16

Leafbio Inc. (San Diego; commercial arm of Mapp Biopharmaceutical Inc.)

Zmapp

Cocktail of monoclonal antibodies

Ebola virus disease

Results from the Prevail II trial conducted in Liberia, Sierra Leone, Guinea and the U.S. over the course of nearly a year showed that while the study ultimately did not enroll enough patients to produce definitive results, researchers said Zmapp was well tolerated and showed promise; mortality in the Zmapp-treated participants was 40% lower (eight of 36; 22% mortality) than the mortality in participants receiving standard of care alone (13 of 35; 37% mortality)

2/25/16

Merck & Co. Inc. (Kenilworth, N.J.)

Isentress

Integrase inhibitor; raltegravir

HIV

Top-line phase III pivotal data of Isentress, to be given as 2 x 600 mg once daily, showed it met its primary efficacy endpoint: 1,200 mg was statistically non-inferior to the marketed formulation approved dose of Isentress 400 mg twice daily, each in combination therapy with Truvada (emtricitabine/tenofovir disoproxil fumarate, Gilead Sciences Inc.), as assessed by the proportion of patients achieving HIV-1 RNA <40 copies/mL at week 48

2/23/16

Siga Technologies Inc. (New York)

ST-246

Tecovirimat; Tpoxx; antiviral

Orthopoxvirus-related diseases

Received FDA concurrence on a dose for continuing the pivotal study; Siga plans to complete the phase III expanded safety study in human subjects for the oral formulation

2/25/16

INFLAMMATORY

Anges MG Inc. (Japan)

Oligodeoxy-nucleotide

NF-kB decoy

Atopic dermatitis

Completed phase III testing in Japan; full results will be announced in the first half of this year

2/26/16

Carbylan Therapeutics Inc. (Palo Alto, Calif.)

Hydros-TA

Formulation of hyaluronic acid which entraps low-dose corticosteroid triamcinolone acetonide

Osteoarthritis of the knee

Top-line results from COR1.1, its first phase III trial, showed it met the first of its two primary endpoints, demonstrating a statistically significant improvement from baseline in the WOMAC A pain score at week two vs. Hydros (hyaluronic acid alone) and maintained a significant reduction in pain from baseline over 26 weeks; it missed its second primary endpoint, as patients in the TA arm continued to show an unexpected significant reduction in pain through 26 weeks

2/3/16

Flexion Therapeutics Inc. (Burlington, Mass.)

Zilretta

FX006; injected intra-articular sustained-release, non-opioid, non-steroid treatment

Moderate to severe osteoarthritis knee pain

Phase III data showed it achieved clinically meaningful and highly statistically significant separation from placebo at each measured time point in weeks one through 16, meeting the primary endpoint at week 12 with a "p" value of <0.0001

2/18/16

MISCELLANEOUS

Aerie Pharmaceuticals Inc. (Irvine, Calif.)

Rhopressa

Netarsudil ophthalmic solution

Glaucoma or ocular hypertension

Phase III Rocket2 data demonstrated successful 12-month interim safety results; previously reported it had achieved its primary 90-day efficacy endpoint of demonstrating noninferiority of IOP lowering for Rhopressa once per day compared to timolol twice per day

2/19/16

Amgen Inc. (Thousand Oaks, Calif.) and UCB SA

Romosozumab

AMG785, CDP7851

To reduce the risk of fracture in osteoporosis

Phase III FRAME data showed it hit the primary endpoint of reducing the risk of new vertebral fracture over 12 and 24 months, but the effect size was deemed inferior to that attained by abaloparatide, Radius Health Inc.'s synthetic peptide analogue of human parathyroid-hormone-related protein, in an earlier trial

2/23/16

Cumberland Pharmaceuticals Inc. (Nashville, Tenn.)

Vaprisol

Conivaptan

Hyponatremia

An open label study evaluating 20 mg/day and 40 mg/day doses of Vaprisol in 251 patients found both doses to be efficacious in increasing patients' sodium concentrations over four days of treatment, with no observed increase in the frequency of adverse events using the higher dose

2/11/16

GW Pharmaceuticals plc (London)

Epidiolex

Cannabidiol

Dravet syndrome and tuberous sclerosis complex

Its three phase III studies have been fully recruited above original target sample sizes and are on track to produce initial data starting in March

2/11/16

Mesoblast Ltd. (Melbourne, Australia)

MSC-100-IV

Mesenchymal stem cell product candidate remestemcel-L

Steroid-refractory acute graft-vs.-host disease

A new analysis of long-term data showed that children treated with the cells demonstrated clinically meaningful responses and significantly increased survival, with an overall response rate of 65% at 28 days after treatment; the company is entering a phase III study that will test the therapy in 60 patients

2/23/16

RESPIRATORY

Boehringer Ingelheim GmbH (Ingelheim, Germany)

Ofev

Nintedanib

Idiopathic pulmonary fibrosis

Pooled analysis from the TOMORROW and INPULSIS trials confirmed that Ofev reduces the risk of acute exacerbations by about 50%; findings also showed that Ofev slowed disease progression by about 50% across the range of patient types in the clinical program and reduced the risk of death; Ofev was approved in 2014

2/26/16

Theravance Biopharma Inc. (South San Francisco) and Mylan NV (Hertfordshire, U.K.)

TD-4208

Revefenacin; a long-acting muscarinic antagonist

Chronic obstructive pulmonary disease

Started a phase III program

2/11/16


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.