Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Cymabay Therapeutics Inc. (Newark, Calif.)

MBX-8025

Seladelpar

Primary biliary cholangitis

Initiated a new phase II study

12/8/16

Galapagos NV (Mechelen, Belgium)

Filgotinib

JAK1 inhibitor

Crohn's disease

Data from the phase II study showed that it met the primary endpoint of clinical remission at 10 weeks: the percentage of patients achieving a Crohn's Disease Activity Index (CDAI) score below 150 was significantly higher in patients treated with filgotinib vs. patients receiving placebo

12/16/16

Lycera Corp. (New York)

LYC-30937-EC

Enteric coated

Psoriasis

Started a phase II trial

12/8/16

Medicinova Inc. (La Jolla, Calif.)

MN-166

Ibudilast

Progressive multiple sclerosis

Said the external data and safety monitoring board (DSMB) for the ongoing phase IIb trial has reviewed the results of the interim efficacy analysis and made the recommendation to the National Institute of Neurological Diseases and Stroke (NINDS) that the trial should continue as planned and this recommendation was accepted by NINDS

12/20/16

Redhill Biopharma Ltd. (Tel Aviv, Israel)

RHB-104

Antibiotic combination therapy

Relapsing-remitting multiple sclerosis (MS)

Reported that the percentage of MS patients who were relapse-free in the modified intent-to-treat population of its phase IIa proof-of-concept study compared favorably with previously reported studies of Rebif and Avonex; the annualized relapse rate (ARR) at both 24 and 48 weeks in the per-protocol population was zero and the ARR in the modified intent-to-treat population, was 0.288 and 0.29, respectively, comparing favorably with previously reported studies of Avonex (0.67) and Rebif (0.87-0.91)

12/13/16

Takeda Pharmaceutical Co. Ltd. (Osaka, Japan)

Entyvio

Vedolizumab therapy; an integrin antagonist that targets gut-specific α4β7

Moderately to severely active Crohn's disease

An analysis based on pre-specified and post-hoc exploratory outcomes of GEMINI II and GEMINI III data demonstrated higher rates of response in patients receiving the drug as a first-line biologic after conventional therapy failure vs. patients who had previously experienced tumor necrosis factor (TNF)-alpha antagonist therapy failure

12/12/16

CANCER

Abbvie Inc. (Chicago)

Veliparib

Poly-ADP ribose polymerase inhibitor

Locally recurrent or metastatic breast cancer with BRCA1 or BRCA2 mutations

Supplementing veliparib to carboplatin and paclitaxel chemotherapy improved the overall response rate without increasing adverse events, according to data from a phase II clinical trial

12/8/16

Adaptimmune Therapeutics plc (Oxford, U.K.)

NY-ESO SPEAR

Engineered T cells

Myxoid/round cell liposarcoma

Started a study in up to 15 patients

12/6/16

Aptevo Therapeutics Inc. (Seattle)

Otlertuzumab

Anti-CD37 protein therapeutic

Relapsed chronic lymphocytic leukemia

Phase II data show an improved overall response rate and progression-free survival with combination otlertuzumab/bendamustine therapy

12/19/16

Ariad Pharmaceuticals Inc. (Cambridge, Mass.)

Brigatinib

Anaplastic lymphoma kinase (ALK) inhibitor

ALK-positive non-small-cell lung cancer

Data from the pivotal ALTA trial in patients who experienced disease progression on crizotinib (Xalkori, Pfizer Inc.) showed that, as of May 31, 55% of patients on the 180-mg regimen achieved the primary endpoint of confirmed objective response (OR); the median progression-free survival (PFS) was 15.6 months, and 67% of patients with measurable brain metastases achieved confirmed intracranial OR, and intracranial PFS was 18.4 months among patients with any brain metastases at baseline

12/13/16

Boehringer Ingelheim GmbH (Ingelheim, Germany)

Vargatef

Nintedanib

Unresectable malignant pleural mesothelioma

The LUME-Meso phase II trial met its primary endpoint demonstrating a meaningful clinical benefit compared to placebo plus pemetrexed/cisplatin, with a significantly improved progression-free survival (9.4 vs 5.7 months, HR = 0.56 and p value =0.0174)

12/8/16

Calithera Biosciences Inc. (San Francisco)

CB-839

A first-in-class glutaminase inhibitor

Advanced/metastatic triple negative breast cancer

Clinical data from its lead product candidate showed that among evaluable patients treated with CB-839 doses of at least 600 mg bid (n=16), there are five partial responses (31%) and disease control (response or stable disease) in 11 patients (69%)

12/7/16

Eisai Ltd. (Hatfield, U.K.)

Halaven

Eribulin

Unresectable advanced or metastatic liposarcoma

German Federal Joint Committee (G-BA) confirmed additional benefit for Halaven compared to dacarbazine to treat unresectable advanced or metastatic liposarcoma; the G-BA found the data for mortality, symptoms and quality of life, which showed an additional benefit over the appropriate comparator, to be decisive, declining to follow the recommendation of the German Institute for Quality and Efficiency in Health Care, issued in September, to withhold an additional benefit based on formal grounds

12/6/16

Five Prime Therapeutics Inc. (South San Francisco)

FPA144

Experimental immunotherapy for tumors that overexpress FGFR2b

Bladder cancer

Translational data suggest that FGFR2b protein is expressed in a subset of bladder cancers; a patient with bladder cancer that expressed FGFR2b achieved a complete response after treatment with FPA144; data showed positive FGFR2b immunohistochemistry staining in 11.6% of 387 archival primary bladder cancer tumor samples it tested

12/5/16

Galena Biopharma Inc. (San Ramon, Calif.)

Neuvax

Nelipepimut-S

Ductal carcinoma in situ

Opened for enrollment its phase II trial

12/15/16

Gtx Inc. (Memphis, Tenn.)

GTX-024

Enobosarm

Advanced, estrogen receptor positive, androgen receptor positive breast cancer

Initial top-line data from an ongoing open-label phase II trial showed that for the nine patients achieving the primary efficacy endpoint in the 9 mg cohort, two patients demonstrated a partial response with a mean reduction in tumor size of 44.4% from baseline and seven patients exhibited stable disease

12/9/16

Heat Biologics Inc. (Durham, N.C.)

HS-410

Vesigenurtacel-L; non-muscle invasive vaccine

Bladder cancer

Top-line phase II data of the vaccine added to standard-of-care treatment, Bacillus Calmette-Guerin, showed it failed to make a statistically significant impact on the proportion of patients achieving recurrence-free survival at one year vs. BCG plus placebo; in one arm of the trial, 76.9% of patients treated with BCG and a placebo achieved one-year recurrence-free survival; in both of the arms that added '410 to the mix, the one-year recurrence-free survival rate was 65.4%

12/1/16

Ignyta Inc. (San Diego)

Entrectinib

Orally available, CNS-penetrant tyrosine kinase inhibitor, targeting tumors that harbor TRK, ROS1 or ALK fusions

Mammary analog secretory carcinoma

A patient with mammary analog secretory carcinoma with an NTRK3 fusion, treated with a combination of entrectinib and Mekinist (trametinib; Novartis AG), achieved a 22% reduction in tumor volume and remained on the combination regimen for nearly seven months

12/5/16

Immunomedics Inc. (Morris Plains, N.J.)

IMMU-132

Sacituzumab govitecan

Metastatic triple-negative breast cancer

A single-arm, phase II study achieved the planned enrollment of 100 patients

12/28/16

Kadmon Holdings Inc. (New York)

Tesevatinib

Blood-brain-barrier penetrant oral tyrosine kinase inhibitor

Epidermal growth factor receptor mutation-positive non-small-cell lung cancer

Phase II data showed a partial response in the brain in an MRI taken after 29 days of tesevatinib therapy and a partial response in both brain metastases and peripheral disease after 57 days of therapy; 11 of the first 13 enrolled patients treated with tesevatinib 300 mg did not have subsequent central nervous system (CNS) progression

12/7/16

Medicenna Biopharma Inc. (Houston)

MDNA55

Immunotherapy based on its Empowered Cytokine platform

Recurrent glioblastoma

Started a phase II trial

12/14/16

Merrimack Pharmaceuticals Inc. (Cambridge, Mass.)

MM-302

HER2 antibody-targeted liposomal doxorubicin

HER2-positive metastatic breast cancer

Halted the phase II HERMIONE study in patients previously treated with the Genentech Inc./Roche Holding AG agents trastuzumab (Herceptin), pertuzumab (Perjeta) and ado-trastuzumab emtansine (T-DM1, Kadcyla); the company based its decision on a recommendation by the independent data and safety monitoring board (DSMB) and a subsequent futility analysis confirming the trial wasn't likely to show a benefit for MM-302 over comparators

12/22/16

Morphosys AG (Martinsried, Germany)

MOR208

Fc-engineered monoclonal antibody targeting CD19

Relapsed or refractory chronic lymphocytic leukemia or small lymphocytic leukemia

The first patient was dosed in the phase II COSMOS trial testing MOR208 in combination with PI3K delta inhibitor Zydelig (idelalisib, Gilead Sciences Inc.)

12/14/16

Oncbiomune Pharmaceuticals Inc. (Baton Rouge, La.)

Proscavax

Vaccine

Prostate cancer

About 30 patients have been pre-screened for the phase II/III trial in Mexico; the trial is being conducted through a joint venture between Oncbiomune and the its Mexico-based acquisition target, Vitel Laboratorios SA de C.V.

12/29/16

Oncoquest Inc. (Edmonton, Calif.)

Oregovomab

Monoclonal antibody that binds to CA125

Advanced epithelial ovarian, adnexal or peritoneal carcinoma

Interim results from a phase IIb study showed statistically significant differences in time to clinical relapse, recurrence-free survival and overall survival between patients in the chemoimmunotherapy combination arm vs. the arm where patients were solely treated with chemotherapy

12/1/16

Prima Biomed Ltd. (Sydney)

IMP321

An antigen-presenting cell activator

Metastatic breast cancer

Interim data from the AIPAC phase IIb trial of IMP321 plus paclitaxel chemotherapy demonstrated it is safe and well-tolerated at both the 6-mg and 30-mg dosage levels; immune monitoring data has also confirmed it is working to generate the desired immune responses; data demonstrated activation and an increased level of blood monocytes, dendritic cells and CD8 T-cells

12/23/16

TG Therapeutics Inc. (New York)

TG-1101

Ublituximab; glycoengineered anti-CD20 monoclonal antibody

Chronic lymphocytic leukemia

Data from a phase II study showed that the combination of TG-1101 and Imbruvica (ibrutinib, Abbvie Inc. and Johnson & Johnson), an oral BTK inhibitor, was well-tolerated, with limited grade 3/4 adverse events observed in patients; an 88% overall response rate (ORR) was reported at month six for all patients treated, with a 95% ORR observed in patients with high-risk CLL (presence of a 17p or 11q deletion or a TP53 mutation)

12/19/16

Tocagen Inc. (San Diego)

Toca 511 and Toca FC

Vocimagene amiretroprepvec and extended-release 5-fluorocytosine

Glioblastoma or anaplastic atrocytoma

Its phase II/III study, Toca 5, is now enrolling patients in Israel, in addition to the U.S. and Canada; the study is comparing cancer-selective virus Toca 511 in combination with Toca FC to standard of care G963

12/14/16

CARDIOVASCULAR

Aobiome LLC (Cambridge, Mass.)

B244

Live bacteria

Hypertension

Launched a phase II trial exploring the discovery of a correlation between the application of its ammonia oxidizing bacteria and blood pressure during a recent phase Ib/IIa acne study, for which blood pressure was tracked as a safety endpoint

12/19/16

Arena Pharmaceuticals Inc. (San Diego)

Ralinepag

Oral, selective IP receptor agonist targeting the prostacyclin pathway

Pulmonary arterial hypertension

Completed enrollment in the phase II trial

12/8/16

Humacyte Inc. (Research Triangle Park, N.C.)

Humacyl

Experimental human acellular vessel (HAV)

Peripheral arterial disease

Initiated a phase II arterial bypass trial in which Humacyl will be surgically implanted in the above-the-knee, femoral-to-popliteal arterial position in the legs of patients, with the goal of assessing whether the vessel performs in the arterial bypass position and is usable and suitable for repairing human arterial blood vessels

12/30/16

Mast Therapeutics Inc. (San Diego)

AIR001

Sodium nitrite solution for intermittent inhalation via nebulization

Heart failure with preserved ejection fraction

The first patient has been enrolled in an investigator-sponsored phase II study

12/7/16

Reneuron plc

CTX cells

Allogeneic human neural stem cell therapy

Disability caused by stroke

Phase II trial did not meet the primary endpoint, which was that two patients would show a two point or more improvement in performing part two of ARAT (Action Research Arm Test), a measure of upper limb function, at three months post-treatment; however, 15 of 21 patients had a clinically significant response on at least one of several measures of efficacy

12/6/16

CENTRAL NERVOUS SYSTEM

AC Immune SA (Lausanne, Switzerland) and Genentech (South San Francisco; unit of Roche AG)

Crenezumab

An anti-A-beta antibody

Alzheimer's disease

Phase Ib data support the binding and increased dosing of crenezumab

12/12/16

Acadia Pharmaceuticals Inc. (San Diego)

Pimavanserin

Selective serotonin inverse agonist

Major depressive disorder

Initiated the phase II CLARITY trial

12/2/16

Acadia Pharmaceuticals Inc. (San Diego)

Pimavanserin

Selective serotonin inverse agonist preferrentially targeting 5-HT2A receptors

Alzheimer's disease psychosis

Phase II exploratory study showed it met the primary endpoint with a statistically significant reduction in AD psychosis vs. placebo as measured by the Neuropsychiatric Inventory-Nursing Home Psychosis score at week six of dosing (p=0.0451)

12/21/16

Anavex Life Sciences Corp. (New York)

ANAVEX 2-73

Targets cellular homeostasis

Mild-to-moderate Alzheimer's disease

Disclosed a positive 57-week update from its phase IIa study showing it met both primary and secondary endpoints

12/9/16

Axovant Sciences Ltd. (Hamilton, Bermuda)

RVT-101

Intepirdine; 5-HT6 receptor antagonist

Mild-to-moderate Alzheimer's disease

Phase IIb trial of intepirdine combined with donepezil compared with donepezil plus placebo suggested treatment was associated with reduced progression in dependence, based on the Dependence Scale; the company said findings suggested that individuals in the study who received treatment with it spent more time in earlier, less dependent stages of the disease

12/13/16

Biohaven Pharmaceutical Holding Co. Ltd. (New Haven, Conn.)

BHV-4157

Formulation of a glutamate-modulating agent

Hereditary spinocerebellar ataxia

Enrolled the first patient in its potentially pivotal phase IIb/III trial assessing the efficacy and safety

12/23/16

Brainstorm Cell Therapeutics Inc. (Hackensack, N.J.)

Nurown

Cell therapy platform

Amyotrophic lateral sclerosis

completed an end-of-phase-II meeting with the FDA, which agreed the company could proceed with phase III

12/20/16

Cerecor Inc. (Baltimore)

CERC-301

Fast-acting antidepressant; selective NMDA receptor subunit 2B antagonist

Moderate to severe depression

Failed to help patients see significant improvement on the primary endpoint for mean improvement in Bech-6, a subset of the Hamilton Depression Scale, in a phase II study

12/1/16

Cerecor Inc. (Baltimore)

CERC-501

Oral, selective kappa opioid receptor antagonist

Nicotine withdrawal

Phase II data showed it did not meet its primary objective

12/6/16

EIP Pharma LLC (Cambridge, Mass.)

Neflamapimod

Previously VX-745; oral small molecule inhibitor of the intra-cellular enzyme p38 mitogen-activated protein kinase alpha

Alzheimer's disease

Phase IIa data showed statistically significant improvement in tests of episodic memory and learning as assessed by Wechsler Memory Scale (WMS) immediate and delayed recall composite measures in study 302 and by Hopkins Verbal Learning Test-Revised in Study 303

12/9/16

Eisai Ltd. (Hatfield, U.K.)

Zebinix

Eslicarbazepine acetate; voltage-gated sodium channel blocker

Seizures

Reduced seizure frequency and seizure freedom with a favorable adverse event profile and an improved global clinical impression of change and severity when used as adjunctive therapy

Living Cell Technologies Ltd. (Sydney)

Ntcell

Microcapsules

Parkinson's disease

Completed treatment of all six patients in group two of the phase IIb trial; there are no safety issues in any of the six patients

12/23/16

Minerva Neurosciences Inc. (Waltham, Mass.)

MIN-101

Candidate with equipotent affinities for sigma 2 and 5-hydroxytryptamine-2A and lower affinity at a1-adrenergic receptors

Schizophrenia

Phase IIb data demonstrate broad internal consistency across multiple endpoints, supporting the direct effect of this compound in treating negative symptoms in schizophrenia

12/6/16

Neuroderm Ltd. (Rehovot, Israel)

ND0612

Continuous, subcutaneously delivered levodopa/carbidopa liquid formulation

Parkinson's disease

It intends to pursue a comparative bioavailability regulatory path for lead product candidate ND0612 based on comparative pharmacokinetic data in place of data from phase III clinical trials

12/6/16

Neuroderm Ltd. (Rehovot, Israel)

ND0612H

Continuously administered levodopa/carbidopa solution

Advanced Parkinson's disease

Completed patient enrollment and treatment in trial 006, a 28-day international, multicenter, parallel-group, single blind (rater-blinded), randomized pilot phase II experiment

12/23/16

Pharnext SA (Paris)

PXT864

A fixed dose combination of baclofen and acamprosate

Alzheimer's disease

Phase II trial showed the drug to be well-tolerated, with a compliance rate above 90%; results showed it may slow the progression of cognitive disability in patients with mild AD, with the primary endpoint – AD Assessment Scale Cognitive Subscale test, or ADAS-Cog11 – appearing to decline less in the pooled PXT864 dose one and two groups

12/14/16

Revance Therapeutics Inc. (Newark, Calif.)

RT002

Daxibotulinum-toxinA injectable

Moderate to severe isolated cervical dystonia

Interim results from its U.S. phase II open-label, dose-escalating trial showed that RT001 appeared generally safe and well-tolerated at all three dose cohorts

12/14/16

Sage Therapeutics Inc. (Cambridge, Mass.)

SAGE-547

Allosteric modulator of synaptic and extra-synaptic GABAA receptors

Postpartum depression

FDA officials said the current program, along with previous phase II data, can support an NDA if ongoing experiments prove successful

12/7/16

Sage Therapeutics Inc. (Cambridge, Mass.)

SAGE-217

Oral GABAA receptor modulator

Postpartum depression and major depressive disorder

Initiated a phase II study

12/20/16

Tonix Pharmaceuticals Holding Corp. (New York)

TNX-102

Sublingual tablets

Military-related post-traumatic stress disorder

The Atease study, a 12-week, randomized, double-blind, placebo-controlled phase II trial, confirmed that patients with combat-related PTSD and entry CAPS-5 ≥ 33, treated with TNX-102 showed improvement on total CAPS-5 severity, in addition to certain items in CAPS-5 cluster scores, such as reckless and self-destructive behavior

12/9/16

Zogenix Inc. (Emeryville, Calif.)

ZX008

Low-dose fenfluramine

Lennox Gastaut syndrome and Dravet syndrome

New data demonstrate effectiveness and cardiovascular-related safety for patients treated with ZX008 as an adjunctive therapy for seizures associated with Lennox Gastaut Syndrome (LGS), and continued effectiveness and safety for the on-going open-label patients with Dravet syndrome

12/6/16

DIABETES

Adoxia SAS (Lyon, France) and Eli Lilly and Co. (Indianapolis)

Biochaperone Lispro

An ultra-rapid formulation of insulin lispro

Type 1 diabetes

Completed an insulin pump study showing Biochaperone Lispro consistently delivered ultra-rapid insulin absorption compared to Humalog in the two insulin pumps tested

12/19/16

Allegro Ophthalmics LLC (San Juan Capistrano, Calif.)

Luminate

An integrin peptide therapy

Diabetic macular edema

Completed enrollment in the second stage of its DEL MAR trial testing Luminate in combination and as an adjunctive therapy with anti-VEGF drugs

12/16/16

Biodelivery Sciences International Inc. (Raleigh, N.C.)

Clonidine

Topical gel

Diabetic neuropathy

Phase IIb data failed to show a statistically significant difference in pain relief between Clonidine and placebo

12/14/16

Lexicon Pharmaceuticals Inc. (The Woodlands, Texas)

Sotagliflozin

A dual SGLT1 and SGLT2 inhibitor

Type 1 diabetes

Top-line results from a phase II clinical study showed the A1C primary endpoint numerically favored sotagliflozin, with a placebo-adjusted reduction of 0.35%, but did not reach statistical significance

12/7/16

Sihuan Pharmaceutical Holdings Group Ltd. (Hong Kong)

Imigliptin dihydrochloride

Oral hypoglycemic agent; a DPP-4 inhibitor

Type 2 diabetes

Received approval from the CFDA to run phase II/III trials

12/15/16

Thrombogenics NV (Leuven, Belgium)

THR-409

Ocriplasmin

Nonprolif-erative diabetic retinopathy

Amended the protocol of the phase II CIRCLE trial, which is testing multiple doses to induce a total posterior vitreous detachment in patients with nonproliferative diabetic retinopathy, to allow for the inclusion of patients with less severe NPDR, resulting in a broader patient pool for recruitment

12/27/16

Vtv Therapeutics Inc. (Bagsvaerd, Denmark)

TTP273

Oral GLP-1 receptor agonist

Type 2 diabetes

Helped type 2 diabetics on stable doses of metformin achieve mean placebo-subtracted HbA1c differences of up to -0.86% during a phase II study

12/16/16

GASTROINTESTINAL

Galapagos NV (Mechelen, Belgium)

Filgotinib

Janus kinase 1 inhibitor

Ulcerative colitis

Dosed the first patient in the SELECTION phase IIb/III study

12/12/16

Protalix Biotherapeutics Inc. (Carmiel, Israel)

OPRX-106

Plant cell-expressed recombinant human tumor necrosis factor receptor II fused to an IgG1 Fc domain

Ulcerative colitis

The first patient has been enrolled in its phase II trial

12/1/16

INFECTION

Achillion Pharmaceuticals Inc. (New Haven, Conn.)

JNJ-4178

A 3DAA combination of NS5A inhibitor odalasvir, NS3/4A hepatitis C virus protease inhibitor simeprevir (Olysio, Janssen Products LP) and uridine nucleotide analogue AL-335

Chronic hepatitis C virus without cirrhosis

Patient dosing in the global phase IIb open-label OMEGA-1 study of JNJ-4178 has been initiated by Janssen Research & Development LLC.; the international, multicenter study is expected to enroll about 300 HCV patients

12/1/16

Aicuris Anti-infective Cures GmbH (Wuppertal, Germany)

Pritelivir

A small-molecule helicase-primase inhibitor

Recurrent labial herpes

Enrolled the first subject in a phase II study

12/2/16

Arbutus Biopharma Corp. (Vancouver, British Columbia)

ARB-1467

RNAi candidate

Chronically infected hepatitis B virus

Results from the multiple-dose cohort of its ongoing phase II study showed a significant reduction of HBsAg

12/14/16

Arrowhead Pharmaceuticals Inc. (Pasadena, Calif.)

ARC-520

Designed with the EX1 liver-targeted, intravenously given vehicle

Chronic hepatitis B virus

Company is discontinuing all pipeline candidates designed with the EX1 liver-targeted, intravenously given vehicle, including, ARC-520, ARC-521 and ARC-AAT; the decision follows nonclinical trouble in November with deaths at the highest dose, causing the FDA to place the phase II trial on clinical hold

12/1/16

Contravir Pharmaceuticals Inc. (Edison, N.J.)

Tenofovir exalidex (TXL, formerly CMX157)

Prodrug analog of tenofovir

Hepatitis B virus

Exhibited increased antiviral activity at the highest planned dose of 100 mg in the ongoing phase IIa multiple ascending-dose trial comparing TXL to Viread (tenofovir disoproxil fumarate [TDF], Gilead Sciences Inc.)

12/20/16

Matinas Biopharma Holdings Inc. (Bedminster, N.J.)

MAT2203

Orally administered, encochleated formulation of the broad-spectrum fungicidal medication amphotericin B

Refractory mucocutaneous candidiasis infection

Patient dosing has commenced in the NIH-sponsored phase IIa study

12/6/16

Shionogi & Co. Ltd. (Osaka, Japan)

S-033188

A cap-dependent endonuclease inhibitor

Influenza

Phase II data showed that 40-mg was associated with a shorter time to alleviation of systemic symptoms compared with placebo, such as feverishness/chills (median 23 hours vs. 28.8 hours, p=0.0216), headache (37.9 hours vs. 43.7 hours, p=0.0304), fatigue (31.1 hours vs. 42.7 hours, p=0.0463) and aches and pains (25.4 hours vs. 41.9 hours, p=0.0145) and provided more rapid resolution of fever (28.9 hours vs. 45.3 hours, p<0.0001)

12/2/16

INFLAMMATORY

C3J Therapeutics Inc. (previously C3 Jian Inc.; Los Angeles)

C16G2

Peptide-based drug

To prevent dental caries

Phase II trials conducted this year showed that a dental varnish formulation containing C16G2 reduced Streptococcus mutans

12/13/16

Centrexion Therapeutics Inc. (Boston)

CNTX-4975

TRPV1-targeting drug

Knee osteoarthritis pain

Top-line data from its phase IIb study showed that its TRPV1-targeting drug met the primary endpoint of reduction in pain with walking through 12 weeks and demonstrated a duration of effect of at least 24 weeks after a single dose

12/14/16

Ocera Therapeutics Inc. (Palo Alto, Calif.)

OCR-002

Ornithine phenylacetate

Hepatic encephalopathy

Completed enrollment in its phase IIb STOP-HE study

12/8/16

Orthotrophix Inc. (Oakland, Calif.)

TPX-100

A novel 23-amino-acid peptide

Bilateral osteoarthritis of the knee

Results of a phase II proof-of-principle study showed excellent safety and tolerability; demonstrated both statistically significant and clinically meaningful improvements in the TPX-100-treated knees as compared to the placebo-treated knees

12/16/16

MISCELLANEOUS

Acceleron Pharma Inc. (Cambridge, Mass.)

ACE-083

A locally acting muscle agent

Facioscapulo-humeral muscular dystrophy

The first patient was treated in a phase II trial

12/27/16

Alexion Pharmaceuticals Inc. (New Haven, Conn.)

Soliris

Eculizumab

Delayed graft function after kidney transplantation

Results from the PROTECT Study, a phase II/III registration trial showed the primary endpoint of incidence of DGF with a two-dose regimen of the drug compared with placebo did not reach statistical significance

12/23/16

Bellus Health Inc. (Laval, Quebec)

Kiacta

Eprodisate

AA amyloidosis

Partner Auven Therapeutics Management L.P. decided to terminate the AA program; Auven continues to evaluate "the conduct" of a phase II/III study with Kiacta in sarcoidosis

12/22/16

Biophytis SA (Romainville, France)

Sarconeos

From a new class based on the activation of the MAS receptor

Sarcopenia

Completed its SARA-PK clinical study; the results confirmed Sarconeos' appropriate safety profile, with no serious adverse events; the company will next conduct a phase IIb study in 2017

12/1/16

Cellceutix Corp. (Beverly, Mass.)

Brilacidin

Small-molecule, defensing-mimetic antibiotic

Ulcerative proctitis/ulcerative proctosigmoiditis

Treatment has begun for the second of three six-patient cohorts enrolled in its phase II study

12/8/16

Cytori Therapeutics Inc. (San Diego)

Cytori Cell Therapy

ECCI-50 therapy, comprising periurethral injection of autologous adipose derived regenerative cells, and a mixture of ADCRs and adipose tissue

Stress urinary incontinence after prostatic intervention for prostate cancer or benign prostatic hypertophy

More than half of 45 patients in the ADRESU trial in Japan have been enrolled and treated

12/15/16

Dimerix Ltd. (Melbourne, Australia)

DMX-200

A new chronic kidney disease treatment

Chronic kidney disease

Completed recruitment of patients into part A (dose escalation) of its phase II trial

12/7/16

Eiger Biopharma-ceuticals Inc. (Palo Alto, Calif.)

Subcutaneous exendin 9-39

31-amino acid peptide that selectively targets and blocks GLP-1 receptors

Post-bariatric surgical patients who experience post-bariatric hypoglycemia

Interim results from an ongoing phase II study showed that all doses led to an increase in glucose nadir (reduced magnitude of hypoglycemia) during OGTT on the last day of treatment; doses ≥ 0.2 mg/kg showed a mean decrease of 51% in peak postprandial insulin levels

12/13/16

Factor Therapeutics Ltd. (Brisbane, Australia)

VF001

A recombinant fusion protein consisting of an engineered vitronectin motif linked to IGF-1

Venous leg ulcers

Enrolled the first patient for its U.S. phase II trial

12/21/16

Hansa Medical AB (Malmo, Sweden)

IdeS

Immunoglobulin G-degrading enzyme of Streptococcus pyogenes

Kidney transplant

The second Swedish open label phase II study was completed; the ascending dose study, in which 10 sensitized kidney patients were given IdeS prior to transplantation, met its primary and secondary objectives

12/22/16

Kiadis Pharma NV (Amsterdam)

ATIR101

Allodepleted T-cell immunotherapeutics

Graft-vs.-host disease

Second phase II trial data showed that none of the 10 patients suffered from grade 3/4 graft-vs.-Host-disease (GVHD) upon infusion of the first dose; with a second infusion, some of six patients subsequently suffered from various grades of GVHD, including grade 3/4 GVHD

12/22/16

Neothetics Inc. (San Diego)

LIPO-202

An injectable formulation of salmeterol xinafoate

To reduce submental subcutaneous fat

Started a phase II study, LIPO-202-CL-31, that will enroll 150 patients and will assess the safety and efficacy of two doses of LIPO-202 vs. placebo

12/27/16

Noveome Biotherapeutics Inc. (Pittsburgh)

ST266

Secretome

Allergic conjunctivitis

Started a phase II trial

12/6/16

Ultragenyx Pharmaceutical Inc. (Novato, Calif.)

UX007

Triheptanoin

Long-chain fatty acid oxidation disorder

78-week data from the phase II study showed frequency and duration of major medical events were reduced significantly during treatment, and patients demonstrated improved exercise tolerance and quality of life during the study; there was a 48.1% reduction (p=0.0208) in the mean annualized rate of MCEs and a 50.3% reduction (p=0.0284) in the mean annualized duration of all MCEs after 78 weeks of treatment

12/1/16

Wilson Therapeutics AB (Stockholm)

WTX101

Bis-choline tetrathiomolybdate; Decuprate; first-in-class copper modulating agent

Wilson disease

Phase II study met its primary endpoint; 79% of patients achieved or maintained normalized levels of free copper, or experienced a reduction of at least 25% in free copper in blood from baseline, after 24 weeks of treatment with WTX101

12/6/16

Zealand Pharma A/S (Copenhagen)

Dasiglucagon

A glucagon analogue

Hypoglycemia

Dosed the first patients in its phase IIa trial

12/14/16

RESPIRATORY

Asit Biotech SA (Brussels, Belgium)

hdm-ASIT+

Product candidate for house dust mite rhinitis

House dust mite rhinitis

Recruited the last patient in its phase IIa trial

12/1/16

Bavarian Nordic A/S (Copenhagen)

MVA-BN RSV

A broad-spectrum vaccine candidate

Respiratory syncytial virus

Completed enrollment of 400 subjects in a phase II trial

12/15/16

Concert Pharmaceuticals Inc. (Lexington, Mass.)

CTP-656

Deuterated ivacaftor; next-generation CFTR potentiator

Cystic fibrosis

Initiated a U.S. phase II trial

12/22/16

Corbus Pharmaceuticals Holdings Inc. (Norwood, Mass.)

Resunab

JBT-101

Cystic fibrosis

Completed its phase II study

12/30/16

Galapagos NV (Mechelen, Belgium)

GLPG1837

Potentiator

Cystic fibrosis

Top-line results from its phase II SAPHIRA 1 study showed a statistically significant dose-dependent decrease in sweat chloride concentration; at the 500-mg twice-daily dose, sweat chloride decreased from a mean value of 98 mmol/L at baseline to 66 mmol/L (p <0.0001)

12/22/16

Protalix Biotherapeutics Inc. (Carmiel, Israel)

Air Dnase

PRX-110; a plant cell-derived recombinant form of human deoxyribonuclease I

Cystic fibrosis

Enrolled the last patient in the phase II trial

12/23/16

Pulmagen Therapeutics Ltd. (Slough, U.K.) and Teijin Pharma Ltd. (Tokyo)

PTR-36

Eosinophilic asthma candidate

Asthma

Reported observing a statistically significant difference between two doses of PTR-36 and placebo during an exploratory phase II study

12/15/16

Respirerx Pharmaceuticals Inc. (Glen Rock, N.J.)

CX1739

AMPAKINE molecule

Opioid-induced respiratory depression

Data from a phase IIa study showed it was found to be safe and well-tolerated and showed statistically significant effects at the low and high doses in overcoming the respiratory depression induced by short-acting opioid remifentanil (remi) in two models of opioid use

12/16/16

Notes

Public biopharmaceutical 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.