Company
(location)

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.