Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Galapagos NV (Mechelen, Belgium)

GLPG0634

A selective JAK1 inhibitor

Active Crohn's disease

The first clinical centers have been opened for enrollment in the phase II study with 180 patients

1/30/14

CANCER

Aduro Biotech Inc. (Berkeley, Calif.)

CRS-207 and Gvax Pancreas

Cancer immunotherapy candidates

Metastatic pancreatic cancer

Phase II was halted early after the study met the primary endpoint

1/15/14

Agenus Inc. (Lexington, Mass.)

Prophage

Checkpoint-inhibitor vaccine

Melanoma

Started a randomized phase II trial with the vaccine combined with Bristol-Myers Squibb Co.’s Yervoy (ipilimumab) for the treatment of stage III and stage IV disease

1/15/14

Apogenix GmbH (Heidelberg, Germany)

APG101

A fusion protein comprising the extracellular domain of the CD95 receptor fused to the Fc domain of an IgG1 antibody

Glioblastoma

Phase II data showed that biomarker-positive patients who received APG101 plus radiotherapy had a median overall survival of 16.1 months, whereas biomarker-positive patients who received radiotherapy only had a median overall survival of 6.5 months; the result was statistically significant (p = 0.003)

1/14/14

Aveo Oncology Inc. (Cambridge, Mass.) and Astellas Pharma Inc. (Tokyo)

Tivozanib

An inhibitor of all three vascular endothelial growth factor (VEGF) receptors

Locally recurrent or metastatic triple negative breast cancer

Discontinued the BATON phase II study due to insufficient enrollment

1/31/14

Biothera Inc. (Eagan, Minn.)

Imprime PGG

Immunotherapeutic

Advanced non-small-cell lung cancer

Phase II data in 90 patients treated with Imprime PGG, Erbitux, carboplatin and paclitaxel compared with the monoclonal antibody and chemotherapy combination alone showed a statistically significant improvement in objective response rate of 48% in the Imprime PGG treated group compared to 23% in the control group; the objective overall response rate was 67% for biomarker-positive patients, with median overall survival of 16.5 months compared with 11.2 months for the control group

1/9/14

California Stem Cell Inc. (Irvine, Calif.)

Ovapuldencel-T

A combination of autologous dendritic cells loaded with irradiated autologous tumor cells in GM-CSF

Stage III or stage IV ovarian, fallopian tube or primary peritoneal cancer

Submitted a phase II protocol to the FDA for a study to evaluate the cells

1/24/14

Cerulean Pharma Inc. (Cambridge Mass.)

CRLX101

Nanoparticle-based, camptothecin-loaded

Relapsed ovarian cancer

Achieved its primary efficacy endpoint in an ongoing open-label single-arm phase II monotherapy trial

1/14/14

Cortice Biosciences Inc. (New York)

TPI 287

An abeotaxane, a taxoid derivative

Recurrent glioblastoma

Enrollment has begun in the first stage of a phase II open-label trial designed to evaluate the safety and efficacy of TPI 287, in combination with standard-of-care Avastin for treatment of patients who have not received prior anti-angiogenic treatment for their disease

1/10/14

Cytrx Corp. (Los Angeles)

Aldoxorubicin

An improved albumin-binding form of doxorubicin

Unresectable glioblastoma multiforme

The first patient has been dosed in a phase II trial with aldoxorubicin; the trial is expected to enroll up to 28 patients

1/15/14

Cytrx Corp. (Los Angeles)

Aldoxorubicin

An improved, albumin-binding form of doxorubicin

HIV patients with Kaposi's sarcoma

Started a phase II trial with up to 30 patients

1/24/14

Entremed Inc. (Rockville, Md.)

ENMD-2076

An oral Aurora A/angiogenic kinase inhibitor

Advanced ovarian clear cell carcinoma

Submitted a new drug global clinical trial application with China’s Food and Drug Administration (CFDA) to expand its phase II trial

1/14/14

Erytech Pharma SA (Lyon, France)

Eryasp

Also known as Graspa, and consists of red blood cells loaded with the enzyme asparaginase

Second-line pancreatic cancer

Launched a phase II study

1/8/14

Galena Biopharma Inc. (Portland, Ore.)

GAL-301

A folate receptor alpha-derived peptide-based cancer immunotherapy

High-risk endometrial and ovarian cancer patients

The first patient was enrolled in the phase II study of GAL-301 in combination with granulocyte macrophage-colony stimulating factor as an adjuvant treatment to prevent recurrences

1/8/14

Generex Biotechnology Corp. (Toronto)

AE37

Breast cancer vaccine

Breast cancer

Enrollment was completed in the phase II trial being conducted by its subsidiary, Antigen Express Inc.

1/14/14

Gtx Inc. (Memphis, Tenn.)

GTx-758

An oral estrogen receptor alpha agonist

Metastatic castrate-resistant prostate cancer

The 125-mg arm in the phase II trial completed enrollment of 38 patients

1/30/14

Mei Pharma Inc. (San Diego)

Pracinostat

Oral histone deacetylase inhibitor

Myelodysplastic syndrome

The first patient has been dosed in a phase II trial, which is expected to enroll up to a total of 76 patients

1/15/14

Nymox Pharmaceutical Corp. (Hasbrouck Heights, N.J.)

NX-1207

Injected by a urologist under ultrasound guidance directly into the area of the prostate to be treated

Prostate cancer

Closed recruitment for its phase II study

1/14/14

Progenics Pharmaceuticals Inc. (Tarrytown, N.Y.)

PSMA ADC

Prostate-specific membrane antigen antibody drug conjugate

Taxane-refractory metastatic castration-resistant prostate cancer

Phase II data showed that circulating tumor cells were reduced by more than 50% in more than 70% of treated patients and said the 2.3 mg/kg dose was well tolerated

1/31/14

Rexahn Pharmaceuticals Inc. (Rockville, Md.)

Archexin

An Akt1 protein kinase inhibitor

Metastatic renal cell carcinoma

Initiated a phase IIa proof-of-concept trial to study the safety and efficacy of Archexin

1/15/14

Tokai Pharmaceuticals Inc. (Cambridge, Mass.)

TOK-001

Galeterone

Castration-resistant prostate cancer

Interim phase II ARMOR2 study results showed that in 39 patients followed for at least 12 weeks across all doses, 79% achieved a PSA decline of at least 30% and 67% achieved a PSA decline of at least 50%; galeterone was well tolerated

1/30/14

Transgene SA (Strasbourgh, France)

TG4010

An immunotherapy

Non-small-cell lung cancer

Phase IIb/III data validated the French company’s predictive biomarker and confirmed earlier phase II results

1/10/14

Viralytics Ltd. (Sydney)

Cavatak

Coxsackievirus A21; intratumoral

Melanoma

The 54th late-stage melanoma patients was injected, achieving the target enrollment milestone for the phase II CALM study

1/9/14

CARDIOVASCULAR

Cerenis Therapeutics SA (Toulouse, France)

CER-001

HDL mimetic

Post-acute coronary syndrome

Phase IIb CHI-SQUARE study did not reach its primary endpoint

1/3/14

Dezima Pharma BV (Naarden, the Netherlands)

TA-8995 (DEZ-001)

CETP inhibitor

Dyslipidemia

Completed enrollment of its phase IIb TULIP study

1/14/14

D-Pharma Ltd. (Rehovot, Israel)

THR-18

Corresponds to a fragment of plasminogen activator inhibitor-1

Acute stroke

Completed enrollment of patients in the first tier of its phase II trial

1/22/14

Prosonix Ltd. (Oxford, UK)

PSX1002

A particle-engineered drug-only suspension formulation of long-acting muscarinic antagonist glycopyrronium bromide

Moderate to severe chronic obstructive pulmonary disease

Top-line results from its phase II study demonstrated statistically significant separation from placebo for all doses, with a clear progression of effect by dose, meeting the primary endpoint

1/24/14

Regado Biosciences Inc. (Basking Ridge, N.J.)

REG1

A direct-acting, antidote-controlled anticoagulant

For use during cardiac catheterization

Phase IIb RADAR trial data demonstrated the safety of early sheath removal in patients treated with REG1

1/30/14

Resverlogix Corp. (Calgary, Alberta

RVX-208

A BET inhibitor

Atherosclerosis

Major adverse cardiac event (MACE) data from two trials were combined, demonstrating that treatment with RVX-208 led to a significant reduction in MACE; RVX-208-treated-patients had less cumulative events of 6.74% vs. 15.09% (p = 0.02) in the placebo-treated group

1/16/14

Trevena Inc. (King of Prussia, Pa.)

TRV027

A beta-arrestin biased ligand of the angiotensin II type 1 receptor

Acute heart failure

Started dosing in BLAST-AHF, the company’s randomized, multicenter phase IIb trial

1/10/14

CENTRAL NERVOUS SYSTEM

Labrys Biologics Inc. (San Mateo, Calif.)

LBR-101

Formerly RN-307; a subcutaneous once-monthly treatment

To prevent frequent migraines

Patients are now being enrolled in two separate phase IIb studies

1/9/14

Neurovance Inc. (Cambridge, Mass.)

EB-1020 SR

A triple reuptake inhibitor that modulates norepinephrine (NE), dopamine (DA) and serotonin (5-HT)

Attention deficit hyperactivity disorder

Neurovance reported interim results from its phase IIa pilot study indicating a statistically significant improvement in ADHD symptoms

1/10/14

Omeros Corp. (Seattle)

OMS824

Phosphodiesterase 10 inhibitor

Schizophrenia

Phase IIa data showed the drug was well tolerated and demonstrated comparable systemic pharmacokinetics when administered alone and concomitantly with approved antipsychotic agents

1/30/14

Zafgen Inc. (Cambridge, Mass.)

Beloranib

A selective inhibitor of methionine aminopeptidase 2

Prader-Willi syndrome

Phase IIa data showed improvements in body weight, hunger-related behaviors and body composition, including reductions in body fat content and preserved lean body mass following four weeks of treatment

1/16/14

DIABETES

Adocia SAS (Lyon, France)

Lispro

Fast-acting formulation of insulin Lispro using its Biochaperone technology

Type 1 diabetes

Launched a phase IIa trial

1/7/14

Evoke Pharma Inc. (San Diego)

EVK-001

Metoclopramide

Diabetic gastroparesis

Phase IIb data showed that intranasal delivery of metoclopramide was more effective in managing symptoms of diabetic gastroparesis compared to the marketed oral tablet formulation of metoclopramide

1/23/14

Oramed Pharmaceuticals Inc. (Jerusalem)

ORMD-0801

Oral insulin capsule

Type 2 diabetes

Phase IIa data showed it met all primary and secondary endpoints

1/31/14

GASTROINTESTINAL

Altheus Therapeutics Inc. (Oklahoma City)

Zoenasa

Rectal gel

Ulcerative colitis

Completed enrollment for a six-week, double-blind, active-controlled phase II trial of the efficacy and safety of Zoenasa in 120 adult patients with left-sided, or distal, ulcerative colitis

1/10/14

Rebiotix Inc. (Roseville, Minn.)

RBX2660

Microbiota suspension

Recurrent Clostridium difficile-associated diarrhea

Completed enrollment in the PUNCH CD phase II trial to evaluate a non-antibiotic, live microbial drug product conducted using the same protocol at all sites

1/14/14

Salix Pharmaceuticals Ltd. (Raleigh, N.C.)

Fulyzaq

Crofelemer

Noninfectious diarrhea caused by antiretroviral therapy in HIV

Clinical data showed Fulyzaq provided significant improvement in the symptomatic relief of noninfectious diarrhea with a safety profile similar to placebo and with no negative impact on clinical immune parameters (HIV viral load and CD4+ cell count)

1/15/14

INFECTION

Cytodyn Inc. (Vancouver, Wash.)

PRO 140

A monoclonal CCR5 antibody

Human immuno-deficiency virus Type I

Received approval from the FDA to begin patient screening for a phase IIb study

1/9/14

Idenix Pharmaceuticals Inc. (Cambridge, Mass.)

Samatasvir (IDX719)

Once-daily pan-genotypic NS5A inhibitor

Hepatitis C virus

Interim data from its phase II 12-week HELIX-1 trial of samatasvir in combination with simeprevir (TMC435), from Janssen R&D Ireland (a unit of Johnson & Johnson and Medivir AB), showed it was well tolerated

1/14/14

Intercept Pharmaceuticals Inc. (New York)

OCA

Obeticholic acid

Non-alcoholic steatohepatitis

Company stopped the FLINT phase IIb trial since it hit the primary endpoint

1/10/14

Vertex Pharmaceuticals Inc. (Cambridge, Mass.)

VX-135

Nucleotide analogue hepatitis C virus polymerase inhibitor

Hepatitis C virus

Phase IIa data from an intent-to-treat analysis showed the sustained viral response rate four weeks after the completion of treatment (SVR4) was 83% (10 of 12) in treatment-naïve genotype 1 patients who received 200 mg of VX-135 in combination with daclatasvir

1/10/14

Xbiotech Inc. (Austin, Texas)

Antibody

Anti-IL-1a therapeutic antibody

Pyoderma gangrenosum

Launched a phase II study

1/17/14

MISCELLANEOUS

Amgen Inc. (Thousand Oaks, Calif.) and UCB SA (Brussels, Belgium)

AMG 785/CDP7851

Romosozumab

Postmenopausal women with low bone mineral density

Phase II data demonstrated that, compared with placebo, romosozumab treatment for 12 months significantly increased BMD at the lumbar spine, total hip and femoral neck

1/3/14

Biomarin Pharmaceutical Inc. (San Rafael, Calif.)

BMN 111

An analog of c-type natriuretic peptide

Achondroplasia

Dosed the first child in the phase II trial with BMN 111

1/15/14

D-Pharm Ltd. (Rehovot, Israel)

Dp-b99

A cell protective drug

Acute high-risk pancreatitis

The first patient has been recruited into its phase II trial; the study is enrolling 30 patients to be treated, twice daily for two days, with either DP-b99 or placebo

1/15/14

La Jolla Pharmaceutical Co. (San Diego)

GCS-100

A complex, naturally occurring polysaccharide derived from pectin

Chronic kidney disease

La Jolla started a phase II extension study of GCS-100, which will enroll CKD patients, including a maximum of 115 patients at five sites, with the primary objective of determining the safety and tolerability of extended dosing

1/16/14

Neurocrine Biosciences Inc. (San Diego)

NBI-98854

Vesicular monoamine transporter 2 (VMAT2) inhibitor

Tardive dyskinesia

Company hit its endpoints in a phase IIb study, causing a significant change from baseline in the abnormal involuntary movement scale; showed an excellent safety profile and a clinically meaningful reduction in symptoms in up to 12 weeks of continuous dosing

1/8/14

Ocera Therapeutics Inc. (Palo Alto, Calif.)

OCR-002

Ornithine phenylacetate

Liver cirrhosis and hepatic encephalopathy

The first patient has been enrolled in its multicenter phase IIb study, the “Stop-HE” or OCR002-HE209 trial

1/10/14

Pharmalink AB (Stockholm)

Nefecon

Nefigan

IgA nephropathy

Completed recruitment of patients into a phase IIb NEFIGAN study, designed to assess the efficacy and safety of two different doses of Nefecon in 90 IgA-nephropathy patients at risk of end-stage renal failure, despite optimized standard-of-care therapy

1/14/14

Promedior Inc. (Lexington, Mass.)

PRM-151

A recombinant form of human pentraxin-2 protein

Myelofibrosis

Completed enrollment in the first stage of its adaptive phase II trial

1/9/14

Raptor Pharmaceutical Corp. (Novato, Calif.)

RP103

Cysteamine bitartrate delayed-release

Non-alcoholic fatty liver disease in children

The phase II trial to evaluate the safety and potential efficacy of the drug met its enrollment target

1/14/14

Ritter Pharmaceuticals Inc. (Los Angeles)

RP-G28

An oligosaccharide

Lactose intolerance

Phase II data showed it dramatically reduced the pain and symptoms of lactose-intolerant patients

1/9/14

Sarepta Therapeutics Inc. (Cambridge, Mass.)

Eteplirsen

Exon-skipping candidate

Duchenne muscular dystrophy

Data through week 120 from Study 202, a phase IIb open-label extension study showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the six-minute walk test

1/17/14

RESPIRATORY

Advanced Inhalation Therapies Ltd. (Rehovot, Israel)

Noxcurect

Specialized formulation of inhaled nitric oxide delivered using the firm's drug delivery system

Cystic fibrosis

Started enrolling patients in a phase II trial

1/8/14

Alios Biopharma Inc. (South San Francisco)

AL-8176

Nucleoside analog

Respiratory syncytial virus (RSV) infection

Starting a phase IIa study designed to evaluate the safety, pharmacokinetics and antiviral activity of multiple doses of the drug against RSV infection in a virus challenge model

1/14/14

Kalobios Pharmaceuticals Inc. (South San Francisco)

KB003

Anti-GM-CSF monoclonal antibody

Severe asthma

Phase II study missed the primary endpoint of improving FEV1 in patients with severe asthma, compared to placebo; it was generally safe and well tolerated

1/31/14


Notes

BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; 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.