Company |
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. |