Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Anacor Pharmaceuticals Inc. (Palo Alto, Calif.) |
AN2728 |
Boron-based phospho-diesterase-4 inhibitor |
Atopic dermatitis |
Enrolled the first patient in a pair of pivotal phase III trials |
4/1/14 |
Biogen Idec Inc. (Cambridge, Mass.) |
Plegridy |
Peginterferon beta-1a |
Relapsing multiple sclerosis |
Two-year phase III data from the ADVANCE trial demonstrated favorable results on relapse rates, magnetic resonance imaging findings and disease progression when dosed every two weeks |
4/30/14 |
Catalyst Pharmaceutical Partners Inc. (Coral Gables, Fla.) |
Firdapse |
Amifampridine phosphate |
Lambert-Eaton myasthenic syndrome |
Reached patient enrollment target for its pivotal phase III |
4/2/14 |
CANCER | |||||
Agenus Inc. (Lexington, Mass.) and Glaxosmithkline plc (London) |
MAGE-A3ii |
Cancer immuno-therapeutic that contains QS-21 Stimulon adjuvant |
Non-small-cell lung cancer |
Companies are terminating the phase III MAGRITi study after it missed both the first and second co-primary endpoints |
4/3/14 |
Ambit Biosciences Inc. (San Diego) |
Quizartinib |
An oral, once-daily selective inhibitor of FLT3 |
Relapsed/refractory acute myeloid leukemia |
Initiated the phase III QUANTUM-R study comparing quizartinib as a monotherapy to chemotherapy regimens in patients with the FMS-like tyrosine kinase-3 (FLIT3)-ITD mutation |
4/9/14 |
Amgen Inc. (Thousand Oaks, Calif.) |
T-vec |
Cancer immunotherapy talimogene laherparepvec |
Melanoma |
Phase III secondary endpoint data on overall survival showed a "strong trend" despite a statistical miss; the trend in favor of T-vec achieved a "p" value of 0.051 |
4/7/14 |
Celsion Corp. (Lawrenceville, N.J.) |
Thermodox |
Heat-sensitive liposome anticancer platform |
Cancer |
Final trial results from a 701-patient trial suggest that it may significantly improve overall survival, compared to a control, in patients whose lesions undergo the radiofrequency ablation treatment for 45 minutes or more |
4/11/14 |
Heron Therapeutics Inc. (Redwood City, Calif.) |
Sustol |
Formerly APF530; 5-HT3 receptor antagonist |
To prevent chemotherapy-induced nausea and vomiting |
Started a phase III label expansion study |
4/1/14 |
Oncogenex Pharmaceuticals Inc. (Bothell, Wash.) and Teva Pharmaceutical Industries Ltd. (Jerusalem) |
Custirsen |
A drug designed to block production of clusterin |
Metastatic castrate-resistant prostate cancer |
Failed to perform better than standard chemotherapy in the pivotal Synergy trial; adding custirsen to docetaxel and prednisone did not significantly improve overall survival for the 1,022 men |
4/29/14 |
Oncolytics Biotech Inc. (Calgary, Alberta) |
Reolysin |
Oncolytic virus candidate |
Second-line, platinum-refractory, taxane-naïve head and neck cancers |
The phase III REO 018 study testing Reolysin in combination with carboplatin and paclitaxel showed a statistically significant improvement (p = 0.0072); an analysis of overall survival also demonstrated a statistically significant improvement in the Reolysin arm vs. control arm (p = 0.0146) |
4/9/14 |
Oncothyreon Inc. (Seattle) and Merck KGaA (Darmstadt, Germany) |
Tecemotide |
Cancer vaccine |
Unresectable, locally advanced stage III non-small-cell lung cancer |
Started a phase III trial |
4/8/14 |
Onyx Pharmaceuticals Inc. (South San Francisco) and Bayer Healthcare Pharmaceuticals Inc. (Whippany, N.J.) |
Nexavar |
Sorafenib tablets |
Locally recurrent or metastatic, progressive, differentiated thyroid carcinoma |
Results from the phase III DECISION trial demonstrated that Nexavar tablets significantly extended the time patients lived without their disease worsening |
4/25/14 |
Sorrento Therapeutics Inc. (San Diego) |
Cynviloq |
Paclitaxel polymeric micelle |
Metastatic breast cancer and non-small-cell lung cancer |
The first patient was dosed in the pivotal trial, TRIBECA (TRIal designed to evaluate bioequivalence between Cynviloq and Abraxane) |
4/2/14 |
CARDIOVASCULAR | |||||
Amgen Inc. (Thousand Oaks, Calif.) |
Evolocumab |
Protein convertase subtilisin/kexin type 9 inhibitor |
Cholesterol |
Phase III data confirmed phase II results showing evolocumab brought down low-density lipoprotein cholesterol by 50-60% |
4/1/14 |
Armetheon Inc. (Sunnyvale, Calif.) |
Tecarfarin |
Oral anticoagulant therapy |
Prosthetic heart valves |
Reached an agreement with the FDA on an SPA for the final pivotal trial of tecarfarin |
4/30/14 |
Biogen Idec Inc. (Cambridge, Mass.) and Swedish Orphan Biovitrum AB (Stockholm) |
Eloctate |
Recombinant factor VIII Fc fusion protein |
Hemophilia A |
Positive top-line results of the Kids A-LONG phase III trial showed it was generally well tolerated and no neutralizing antibodies that might interfere with its activity were detected |
4/11/14 |
Portola Pharmaceuticals Inc. (South San Francisco) |
Betrixaban |
Once-daily Factor Xa anticocagulant |
Critical cardiovascular conditions |
Launched a global, pivotal phase III trial to determine whether extended preventive treatment can prevent blood clots in the leg and lung that can occur in hospitalized cardiovascular disease patients |
4/15/14 |
Regado Biosciences Inc. (Basking Ridge, N.J.) |
REG1 |
Antithrombotic drug |
For patients undergoing percutaneous coronary intervention, or angioplasty |
Company is expanding enrollment in the phase III trial comparing REG1 to Angiomax (bivalirudin, the Medicines Co.) after achieving its 1,000-patient enrollment milestone |
4/4/14 |
CENTRAL NERVOUS SYSTEM | |||||
Alkermes plc (Dublin) |
Aripiprazole lauroxil |
Long-acting injectable antipsychotic |
Schizophrenia |
Favorable phase III data demonstrated statistically significant reductions from baseline in Positive and Negative Syndrome Scale total scores at week 12, compared to placebo |
4/9/14 |
Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.) |
Nuedexta |
Dextromethorphan HBr and quinidine sulfate |
Pseudobulbar affect |
Phase III data showed that 19% of treated patients achieved remission from symptoms after one week; 50% of patients achieved remission by the end of the 12-week trial |
4/29/14 |
Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.) |
AVP-825 |
A breath-powered powder sumatriptan intranasal treatment |
Severe migraine pain |
Migraine relief for some patients began as early as 15 minutes following treatment, and a statistically significant greater number of treated patients experienced headache relief compared to placebo at all time points from 30 minutes through two hours |
4/29/14 |
Cytokinetics Inc. (South San Francisco) |
Tirasemtiv |
A novel skeletal muscle activator |
Amyotrophic lateral sclerosis |
Results from BENEFIT-ALS showed that of the 711 patients enrolled in the trial, 605 were randomized to double-blind treatment with either tirasemtiv or placebo for 12 weeks; the trial did not achieve its primary efficacy endpoint,, but did result in a statistically significant and potentially clinically meaningful reduction in the decline of slow vital capacity |
4/30/14 |
Ironshore Pharmaceuticals & Development Inc. (subsidiary of Highland Therapeutics Inc.; Toronto) |
HLD-200 |
Formulation of the stimulant methylphenidate |
Attention-deficit hyperactivity disorder |
Began enrollment in a phase III study of HLD-200 |
4/29/14 |
Navidea Biopharmaceuticals Inc. (Dublin, Ohio) |
Lymphoseek |
Technetium Tc 99m tilmanocept |
Pain |
Navidea is testing Lymphoseek injection in a head-to-head study with radiolabeled sulfur colloid to see which drug causes patients less pain |
4/21/14 |
DIABETES | |||||
Biodelivery Sciences International Inc. (Raleigh, N.C.) |
Clonidine |
Topical gel |
Pain associated with diabetic neuropathy |
Enrolled the first patient in a phase III trial |
4/4/14 |
Evoke Pharma Inc. (Solana Beach, Calif.) |
EVK-001 |
An intranasal formulation of metoclopramide |
Acute and recurrent diabetic gastroparesis |
Enrolled the first patient in the phase III trial |
4/23/14 |
INFECTION | |||||
Abbvie Inc. (Chicago) |
ABT-450 |
Ritonavir; fixed-dose combination of ABT-450 (150/100 mg) co-formulated with ombitasvir (ABT-267) 25 mg, dosed once daily, and dasabuvir (ABT-333) 250 mg with or without RBV, dosed twice daily |
Hepatitis C virus infection |
Phase III data showed patients achieved sustained virologic response rates of 91.8% and 95.9% in the 12-week and 24-week treatment arms, respectively |
4/15/14 |
Durata Therapeutics Inc. (Chicago) |
Dalvance |
Dalbavancin |
Acute bacterial skin and skin structure infections caused by susceptible gram-positive bacteria |
Durata began enrollment of a phase IIIb trial to evaluate the efficacy and safety of Dalvance for injection, in a single 1,500-mg dose infused over 30 minutes |
4/25/14 |
Dynavax Technologies Corp. (Berkeley, Calif.) |
Helisav-B |
Vaccine that combines hepatitis B surface antigen with a Toll-like receptor 9 agonist |
Hepatitis B virus |
Started a phase III trial |
4/16/14 |
Emergent Biosolutions Inc. (Rockville, Md.) |
Biothrax |
Anthrax vaccine adsorbed post-exposure prophylaxis |
Anthrax |
Completed the last licensure-enabling study in its Biothrax post-exposure prophylaxis program; it met both its primary and secondary endpoints |
4/17/14 |
Enanta Pharmaceuticals Inc. (Watertown, Mass.) and Abbvie Inc. (Chicago) |
ABT-450, ABT-267 and ABT-333 |
Three direct-acting antiviral regimen, boosted protease inhibitor ABT-450/ritonavir, NS5A inhibitor ABT-267 and non-nucleoside polymerase inhibitor ABT-333 |
Chronic genotype 1 hepatitis C virus infection |
Pivotal phase III SAPPHIRE-I and II study data showed patients achieved sustained virologic response rates 12 weeks post-treatment of 96.2% (n = 455/473) and 96.3% (n = 286/297), respectively |
4/14/14 |
Gilead Sciences Inc. (Foster City, Calif.) |
Sovaldi |
Sofosbuvir |
Genotype 2 chronic hepatitis C virus infection |
Positive top-line results from a phase III trial of Sovaldi with ribavirin showed it met its primary efficacy endpoint of superiority compared to a predefined historical control sustained virologic response rate |
4/4/14 |
Medivir AB (Stockholm) |
Simeprevir |
NS3/4A protease inhibitor |
Chronic genotype 1 hepatitis C virus |
Two phase III trials are recruiting patients to study simeprevir in combination with sofosbuvir in treatment-naïve and treatment-experienced patients with and without cirrhosis |
4/3/14 |
Alexion Pharmaceuticals Inc. (Cheshire, Conn.) |
Soliris |
Eculizumab |
Relapsing neuromyelitis optica and refractory generalized myasthenia gravis |
Initiated separate multinational, placebo-controlled trials to evaluate the safety and efficacy of eculizumab in patients |
4/25/14 |
MISCELLANEOUS | |||||
Amicus Therapeutics Inc. (Cranbury, N.J.) |
Migalastat |
The oral, small-molecule chaperone for alpha-galactosidase A |
Fabry disease |
Phase III data showed that subjects who switched from placebo to migalastat after month six turned up a statistically significant reduction in kidney interstitial capillary GL-3 at month 12 (p = 0.013), and those who stayed on migalastat for 12 months showed a durable reduction |
4/30/14 |
Auxilium Pharmaceuticals Inc. (Chesterbrook, Pa.) and Swedish Orphan Biovitrum AB (Stockholm) |
Xiaflex |
Collagenase clostridium histolyticum (CCH) |
Peyronie's disease |
Additional phase III data showed improved clinical outcomes; adverse events were mostly localized to the penis, non-serious and resolved without intervention before the next injection |
4/15/14 |
Biomarin Pharmaceutical Inc. (San Rafael, Calif.) and Merck KGaA (Darmstadt, Germany) |
Kuvan |
Sapropterin dihydrochloride |
Phenylketonuria |
Phase IIIb SPARK data showed it met its primary endpoint |
4/25/14 |
Intercept Pharmaceuticals Inc. (New York) |
OCA |
Obeticholic acid |
Primary biliary cirrhosis |
Phase III POISE trial data showed it met its primary endpoint (p < 0.0001) |
4/15/14 |
Lipocine Inc. (Salt Lake City) |
LPCN 1021 |
Oral testosterone undecanoate |
Hypogonadal men with low testosterone |
Completed enrollment of its SOAR (Study of Oral Androgen Replacement) pivotal phase III study |
4/30/14 |
Pacira Pharmaceuticals Inc. (Parsippany, N.J.) |
Exparel |
Bupivacaine liposome injectable suspension |
Total knee arthroplasty |
Phase III data supported the safety and efficacy to achieve a femoral nerve block in patients; there was a 24% reduction in total opioid use |
4/8/14 |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Androxal |
Enclomiphene |
Testosterone deficiency |
Reached its enrollment goal of 120 subjects in the first of two head-to-head studies comparing Androxal to the leading U.S. testosterone replacement therapy |
4/15/14 |
Revo Biologics Inc. (Framingham, Mass.) |
Atryn |
Antithrombin, recombinant |
Preeclampsia |
Started a phase III program in pregnant women during the 24th to 28th week of pregnancy |
4/1/14 |
Xoma Corp. (Berkeley, Calif.) |
XOMA 052 |
Gevokizumab |
Pyoderma gangrenosum |
Based on its meeting with the FDA, plans are being finalized for a gevokizumab phase III program expected to include two double-blind, placebo-controlled clinical studies |
4/29/14 |
RESPIRATORY | |||||
Theravance Inc. (South San Francisco) and Glaxosmithkline plc (London) |
Fluticasone furoate and vilanterol |
Combination of inhaled corticosteroid fluticasone furoate and long-acting beta2 agonist vilanterol |
Chronic obstructive pulmonary disease |
Started a phase III study testing the combination treatment, FF/VI 100/25 mcg once daily, compared to VI 25 mcg once daily, administered via the Ellipta inhaler |
4/23/14 |
|
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. | |||||