Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Amgen Inc. (Thousand Oaks, Calif.) |
AMG 416 |
Intravenous etelcalcetide; a calcimimetic agent that suppresses the secretion of parathyroid hormone |
Secondary hyperpara-thyroidism |
A head-to-head phase III study comparing AMG 416 with oral cinacalcet showed it met the primary endpoint of non-inferiority |
11/10/15 |
Amgen Inc. (Thousand Oaks, Calif.) |
ABP 501 |
Biosimilar Humira (adalimumab, Abbvie Inc.) |
Moderate to severe rheumatoid arthritis |
Findings from a head-to-head phase III study comparing ABP 501 with Humira showed it met the primary endpoint, which was achievement of ACR20 at week 24, with 74.6% of ABP 501 patients and 72.4% of adalimumab patients meeting the response criteria |
11/11/15 |
Boehringer Ingelheim GmbH (Ingelheim, Germany) |
BI695501 |
Biosimilar Humira (adalimumab, Abbvie Inc.) |
Moderate to severe rheumatoid arthritis |
Completed enrollment in the pivotal phase III study |
11/11/15 |
Coherus Biosciences Inc. (Redwood City, Calif.) and Baxalta Inc. (Bannockburn, Ill.) |
CHS-0214 |
Biosimilar of Enbrel (etancercept) |
Moderate-to-severe chronic plaque psoriasis |
Met its primary endpoints in a confirmatory, double-blind, randomized, controlled, two-part phase III study |
11/10/15 |
Dermira Inc. (Menlo Park, Calif.) |
Cimzia |
Certolizumab pegol; a tumor necrosis factor-alpha inhibitor |
Moderate to severe chronic plaque psoriasis |
Completed patient enrollment for the CIMPAI-1 trial |
11/20/15 |
Incyte Corp. (Wilmington, Del.) and Eli Lilly and Co. (Indianapolis) |
Baricitinib |
JAK1/2 inhibitor |
Rheumatoid arthritis |
Pivotal RA-BEAM trial data, the fourth phase III study, demonstrated statistical superiority for baricitinib over Humira (adalimumab, Abbvie Inc.) after 12 weeks based on several measures of RA disease activity |
11/10/15 |
Sanofi SA (Paris) and Regeneron Pharmaceuticals Inc. (Tarrytown, N.Y.) |
Sarilumab |
Human antibody against the IL-6 receptor |
Rheumatoid arthritis |
Pivotal phase III results showed it met both its co-primary endpoints of improvements in signs and symptoms and improvements in physical function, as well as secondary efficacy endpoints |
11/10/15 |
CANCER | |||||
Adaptimmune Therapeutics plc (Oxford, UK) |
NY-ESO |
T-cell therapy |
Synovial sarcoma |
Data from an extended follow-up of a clinical study showed that 50% of patients treated with NY-ESO responded, and 75% remain alive and on long-term follow-up |
11/6/15 |
Arqule Inc. (Burlington, Mass.) |
Tivantinib |
MET inhibitor |
Non-small-cell lung cancer |
Exploratory subanalysis of the MARQUEE trial showed that tivantinib, added to erlotinib (Tarceva, Roche AG and Astellas Pharma Inc.), increased progression-free survival to 13 months compared to 7.5 months in the erlotinib plus placebo arm |
11/10/15 |
Celyad SA (Mont-Saint-Guibert, Belgium) |
NKG2D |
CAR T-cell therapy |
Cancer |
Completed the 30-day safety follow-up of the final patient enrolled in the first cohort of the company's phase I trial; no treatment-related safety issues were reported |
11/11/15 |
Genentech Inc. (South San Francisco) |
Cotellic |
Cobimetinib |
Metastatic melanoma |
Pivotal study data showed that Cotellic, in combination with Zelboraf (vemurafenib) in people with BRAF V600E and V600K mutation-positive unresectable or MM, reduced the risk of death by 30% compared to Zelboraf alone and helped people live a median of nearly two years (median overall survival of 22.3 months vs. 17.4 months, hazard ratio=0.70, 95% CI: 0.55-0.90, p = 0.005) |
11/24/15 |
Genmab A/S (Copenhagen) |
Ofatumumab |
Subcutaneous formulation |
Non-Hodgkin lymphoma |
Said the phase III study of ofatumumab compared to Rituxan (rituximab, Biogen Inc. and Roche AG) in patients with follicular NHL that has relapsed at least six months after completion of treatment with a rituximab-containing regimen will be stopped early after an interim analysis by an independent data monitoring committee showed it was unlikely that ofatumumab would show superiority if the trial was to be completed as planned |
11/25/15 |
Gilead Sciences Inc. (Foster City, Calif.) |
Zydelig |
Idelalisib |
Chronic lymphocytic leukemia |
Its phase III study 115 evaluating Zydelig added to standard therapy will be unblinded early due to a predefined interim analysis indicating a statistically significant benefit in efficacy for progression-free survival and overall survival |
11/17/15 |
Infinity Pharmaceuticals Inc. (Cambridge, Mass.) |
Duvelisib |
An oral, dual inhibitor of P13K-delta and P13K-gamma |
Relapsed or refractory chronic lymphocytic leukemia |
Reached target enrollment of 300 patients in DUO, a randomized, phase III, monotherapy study of duvelisib compared to Arzerra (ofatumumab, Genmab A/S) |
11/18/15 |
Oasmia Pharmaceutical AB (Uppsala, Sweden) |
Paclical |
Micellar paclitaxel |
Metastatic breast cancer |
Analysis of previously published findings from a head-to-head study of Paclical with Abraxane (nab-paclitaxel, Celgene Corp.) confirmed the formulations were bioequivalent with regard to drug concentrations |
11/6/15 |
Oryzon Genomics SA (Barcelona, Spain) |
ORY-1001 |
Potent and selective inhibitor of Lysine Specific Demethylase 1 (LSD1) |
Relapsed or refractory acute leukemia |
Dosed the first patient in the extension cohort of its ORY-1001 phase I trial |
11/11/15 |
Sunesis Pharmaceuticals Inc. (South San Francisco) |
Vosaroxin |
Quinolone derivative |
Acute myeloid leukemia |
Phase III VALOR study missed the primary endpoint, though subset data hit statistical significance; of 570 patients alive at the 60-day mark, the complete response rate was 33% for the voasroxin/cytarabine arm vs. 15.4% for the placebo/cytarabine arm |
11/6/15 |
Xbiotech Inc. (Austin, Texas) |
Xilonix |
Blocks inflammation involved in tumor growth and metastasis |
Colorectal cancer |
Said data cleaning of its phase III European study revealed a fewer number of per-protocol patients available for primary endpoint evaluation; it found 25 patients dropped off study prior to receiving any dosing with drug or placebo, 14 patients erroneously received either placebo or study drug, 33 patients completed the study but failed to receive scheduled DEXA scans, properly complete EORTC evaluation or both |
11/25/15 |
CARDIOVASCULAR | |||||
Astrazeneca plc (London) |
Brilinta |
Ticagrelor |
Myocardial infarction |
Results of a subanalysis of the PEGASUS-TIMI 54 study showed that in patients who stayed on therapy, Brilinta reduced the rate of the composite efficacy endpoint of cardiovascular death, MI or stroke at three years, consistent with the results of the overall population of the PEGASUS study |
11/11/15 |
Cerenis Therapeutics SA (Toulouse, France) |
CER-001 |
An engineered HDL-mimetic |
Atherosclerosis |
Cerenis showed that it induced atherosclerosis regression as measured by coronary intravascular ultrasound in patients with a baseline percent atheroma volume of 30% or greater; investigators reported a statistically significant decrease in PAV vs. placebo |
11/11/15 |
Dyax Corp. (Burlington, Mass.) |
DX-2930 |
Fully human monoclonal antibody inhibitor of plasma kallikrein |
Hereditary angioedema |
Phase Ib data showed that DX-2930 was well tolerated at all dose levels, with pharmacokinetics showing a linear, dose-dependent exposure to drug and a mean elimination half-life of about 14 days |
11/11/15 |
Portola Pharmaceuticals Inc. (South San Francisco) |
Andexanet alfa |
Antidote to anticoagulant effects of Fxa inhibitors |
To reverse anticoagulation |
Results of its phase III ANNEXA studies showed that both ANNEXA Part 1 (bolus only) and Part 2 (bolus plus continuous infusion) met all primary and secondary efficacy endpoints, including the measurement of reversal Anti-Xa activity (p<0.0001) for both rivaroxaban and apixaban |
11/12/15 |
Regeneron Pharmaceuticals Inc. (Tarrytown, N.Y.) and Sanofi SA (Paris) |
Praluent |
Alirocumab |
High cholesterol |
A post-hoc analysis of six phase III trials showed that 74% of patients reached their pre-specified low-density lipoprotein cholesterol targets within eight weeks of adding Praluent to their standard-of-care treatment, including statins; completed enrollment in a global phase III trial, which is evaluating the potential cardiovascular benefits of Praluent injection after an acute coronary syndrome |
11/12/15 |
Resverlogix Corp. (Calgary, Alberta) |
RVX-208 |
Apabetalone |
Coronary artery disease and type 2 diabetes mellitus |
The first patient was randomized and dosing has started in the phase III trial, called BETonMACE |
11/12/15 |
Scpharmaceuticals Inc. (Lexington, Mass.) |
Furosemide |
Subcutaneous administration |
Heart failure |
Phase III pivotal results indicated that the subcutaneous administration of the furosemide formulation was as effective as the traditional intravenous administration in getting furosemide into the bloodstream and in achieving diuresis |
11/5/15 |
CENTRAL NERVOUS SYSTEM | |||||
Acelrx Pharmaceuticals Inc. (Redwood City, Calif.) |
Zalviso |
Sublingual sufentanil |
Moderate to severe acute pain |
Company is moving forward with another phase III study to satisfy the FDA, which issued a complete response letter in 2014 |
11/2/15 |
Acelrx Pharmaceuticals Inc. (Redwood City, Calif.) |
Zalviso |
Sufentanil sublingual tablet system 15 mcg |
Obese postsurgical patients |
Three phase III studies showed it provides a faster onset of pain relief than intravenous patient-controlled analgesia with morphine |
11/5/15 |
Agenebio Inc. (Baltimore) |
AGB101 |
Extended-release, once-daily formulation |
Amnestic mild cognitive impairment |
Results of a crossover food effect study showed that the proprietary dose and formulation are suitable for use in the upcoming HOPE4MCI phase III trial |
11/9/15 |
Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.) |
AVP-786 |
Deuterium-modified dextromethorphan compounds |
Alzheimer's disease |
Started enrollment into a phase III trial |
11/17/15 |
Forum Pharmaceuticals Inc. (Waltham, Mass.) |
Encenicline |
A selective partial agonist of the α7 nicotinic receptor |
Schizophrenia |
FDA removed a partial clinical hold on the phase III program for cognitive impairment |
11/12/15 |
Neos Therapeutics Inc. (Dallas) |
Cotempla XR-ODT |
Methylphenidate XR orally disintegrating tablet product candidate |
Attention deficit hyperactivity disorder |
Phase III data showed a statistically significant improvement in symptom control compared to placebo across the classroom day |
11/3/15 |
DIABETES | |||||
Alimera Sciences Inc. (Atlanta) |
Iluvien |
Fluocinolone acetonide |
Diabetic retinopathy |
A new analysis of patients from the FAME study demonstrated a significant slowing of the progression; at month 36, statistically significantly fewer Iluvien-treated eyes experienced progression of PDR (17%) vs. sham-treated eyes (31%) |
11/17/15 |
Derma Sciences Inc. (Princeton, N.J.) |
DSC127 |
Aclerastide |
Diabetic foot ulcers |
A committee monitoring a phase III recommended stopping trial enrollment based on futility; company is stopping development also for scar reduction and radiation dermatitis |
11/13/15 |
New Haven Pharmaceuticals Inc. (New Haven, Conn.) |
Durlaza |
Low-dose, extended-release aspirin |
Diabetes |
Data from the DURATION trial showed that Durlaza delivered sustained antiplatelet control for a full 24-hour period in high-risk patients |
11/12/15 |
GASTROINTESTINAL | |||||
Albireo Ltd. (Gothenburg, Sweden) and Ajinomoto Pharmaceuticals Co. Ltd. (Tokyo) |
Elobixibat |
IBAT inhibitor |
Chronic constipation |
Started a phase III study in Japan |
11/11/15 |
INFECTION | |||||
Cymabay Therapeutics Inc. (Newark, Calif.) |
MBX-8025 |
An orally administered selective PPAR-delta agonist |
Primary biliary cholangitis |
Started a phase II study that will enroll about 75 patients who have had an inadequate response to ursodiol |
11/11/15 |
Novavax Inc. (Gaithersburg, Md.) |
RSV F vaccine |
Respiratory syncytial virus F-protein nanoparticle vaccine candidate |
Respiratory syncytial virus |
Started a phase III trial, called Resolve |
11/10/15 |
Paratek Pharmaceuticals Inc. (Boston) |
Omadacycline |
Broad-spectrum aminomethylcycline antibiotic candidate |
Community-acquired bacterial pneumonia |
Started a pivotal phase III trial |
11/10/15 |
MISCELLANEOUS | |||||
Aeterna Zentaris Inc. (Quebec City) |
Macrilen |
Macimorelin; orally active ghrelin agonist |
Adult growth hormone deficiency |
The first patient was enrolled in a confirmatory phase III efficacy study |
11/20/15 |
Alexion Pharmaceuticals Inc. (Cheshire, Conn.) |
Soliris |
Eculizumab |
Atypical hemolytic uremic syndrome |
Data from a long-term follow-up study showed treated patients had a 74% lower rate of thrombotic microangiopathy events than those who discontinued Soliris therapy |
11/10/15 |
Auris Medical Holding AG (Zug, Switzerland) |
AM-111 |
Contains the synthetic peptide D-JNKI-1 |
Idiopathic sudden sensorineural hearing loss |
Enrolled the first patient into the pivotal HEALOS trial |
11/16/15 |
Genentech Inc. (unit of Roche AG; Basel, Switzerland) |
Esbriet |
Pirfenidone |
Idiopathic pulmonary fibrosis |
Pirfenidone's efficacy at 12 months in patients who were stratified by lung function using forced vital capacity or gender, age and physiology stage showed that a higher proportion of placebo patients with FVC <80% and GAP II-III stage had a >/= 50-meter decline in six-minute walk distance or death or a ≥20-point change in the dyspnea |
11/17/15 |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) and subsidiary Akcea Therapeutics |
Volanesorsen |
Antisense drug designed to reduce the production of apoC-III |
Familial partial lipodystrophy |
Started a phase III study |
11/6/15 |
Ocular Therapeutix Inc. (Bedford, Mass.) |
Dextenza |
Sustained-release dexamethasone |
Allergic conjunctivitis |
The first patients were enrolled in a second phase III trial to evaluate the safety and efficacy |
11/25/15 |
Ohr Pharmaceutical Inc. (New York) |
OHR-102 |
Squalamine lactate ophthalmic solution, 0.2 percent |
Neovascular age-related macular degeneration |
Submitted a special protocol assessment request to the FDA on the detailed design of the phase III program |
11/13/15 |
Opko Health Inc. (Miami) |
Rayaldee |
Calcifediol modified-release capsules; oral vitamin D prohormone |
Secondary hyperpara-thyroidism in chronic kidney disease |
Phase III data showed that plasma levels of intact parathyroid hormone continued to fall as serum levels of 25-hydroxyvitamin D rose above 30 ng/mL, suggesting patients with stage III or IV CKD require higher levels of serum 25D than previously thought in order to control elevated iPTH |
11/6/15 |
Otonomy Inc. (San Diego) |
Otiprio |
Formerly known as Auripro |
Otitis media |
The first pediatric patients have been enrolled in an open-label phase IIIb trial in patients requiring tympanostomy tubes |
11/4/15 |
Otonomy Inc. (San Diego) |
OTO-104 |
A sustained exposure form of dexamethasone |
Meniere's disease |
Enrolled the first patients in the U.S. phase III trial |
11/19/15 |
Proteon Therapeutics Inc. (Waltham, Mass.) |
PRT-201 |
Vonapanitase |
Chronic kidney disease |
Completed enrollment in the first of two phase III studies |
11/4/15 |
Rexahn Pharmaceuticals Inc. (Rockville, Md.) |
Archexin |
Cancer candidate |
Metastatic kidney disease |
Interim phase IIa data showed the drug, in combination with Afinitor (everolimus, Novartis AG) appears to be safe and well tolerated at all doses tested to date, with early evidence of clinical activity at low doses |
11/6/15 |
Sophiris Bio Inc. |
PRX302 |
topsalysin |
Benign prostatic hyperplasia |
Phase III data showed it met the primary endpoint of statistically significant improvement of BPH symptoms over 12 months in final data; those treated with PRX302 saw a 7.6-point improvement, while those treated with saline solution vehicle had a 6.58-point overall improvement (p = 0.043) |
11/11/15 |
Spark Therapeutics Inc. (Philadelphia) |
SPK-RPE65 |
Gene therapy |
RPE65-mediated inherited retinal dystrophies |
Additional secondary endpoint data from the pivotal phase III trial showed that full-field light sensitivity threshold testing and change in bilateral mobility testing for the assigned first eye showed highly statistically significant improvement |
11/17/15 |
Tonix Pharmaceuticals Holding Corp. (New York) |
Tonmya |
TNX-102 SL; cyclobenzaprine HCl sublingual tablets, 2.8 mg |
Fibromyalgia |
Additional results from its 12-week, 205-patient, phase IIb BESTFIT study showed that those patients who reported the greatest improvement in sleep quality were the most likely to experience pain relief |
11/11/15 |
ZS Pharma Inc. (San Mateo, Calif.) |
ZS-9 |
Sodium zirconium cyclosilicate |
Hyperkalemia |
Interim phase III results suggest that, in addition to its ability to rapidly lower potassium levels to the normal range, ZS-9 was shown to maintain normal potassium levels over a 12-month period |
11/10/15 |
RESPIRATORY | |||||
PTC Therapeutics Inc. (South Plainfield, N.J.) |
Translarna |
Ataluren |
Nonsense mutation cystic fibrosis |
Completed enrollment of ACT CF, the second phase III trial |
11/20/15 |
Vectura Group plc (Chippenham, UK) and Novartis AG (Basel, Switzerland) |
Ultibro Breezhaler |
Once-daily indacaterol/glycopyrronium |
Chronic obstructive pulmonary disease |
Results from the phase III FLAME head-to-head trial showed it met its primary endpoint of noninferiority and also demonstrated superiority to twice-daily Seretide (salmeterol/fluticasone, Glaxosmithkline plc) |
11/18/15 |
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. |