Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Anacor Pharmaceuticals Inc. (Palo Alto, Calif.)

Crisaborole

Topical ointment 2% (formerly AN2728), a nonsteroidal anti-inflammatory phosphodeisterase-4 inhibitor

Mild to moderate atopical dermatitis

Top-line results from the long-term safety study showed it was well tolerated and demonstrated a safety profile consistent with that seen in the positive phase III pivotal studies reported in July

11/2/15

Five Prime Therapeutics Inc. (South San Francisco)

FPA008

Anti-CSF1R antibody that blocks the binding of CSF1 and IL-34 ligands to CSF1R

Rheumatoid arthritis

Preliminary data from part 3 of Five Prime's ongoing phase I trial showed it was well tolerated up to 6 mg/kg x 2 doses

11/12/15

Pfenex Inc. (San Diego)

PF530

A biosimilar candidate of Betaseron (interferon beta-1b, Bayer AG)

Multiple sclerosis

Phase I trial enrolled 12 subjects; data showed no statistically significant differences in PK and PD between the groups administered PF530 vs. the reference product

11/16/15

Qu Biologics Inc. (Vancouver, British Columbia)

QBECO SSI

Derived from components of inactivated E. coli

Crohn's disease

Completed enrollment of the QBECO-01 phase I/II trial

11/25/15

Vitae Pharmaceuticals Inc. (Fort Washington, Pa.)

VTP-43742

Orally active RORgammat inhibitor

Moderate to severe psoriasis

Top-line phase I data showed it was safe and generally well tolerated at all dose levels assessed

11/20/15

Xencor Inc. (Monrovia, Calif.)

XmAb5871

Fc-engineered antibody

Rheumatoid arthritis

Phase Ib/IIa data demonstrated it was generally well tolerated and showed trends in improvement in RA disease activity by multiple disease activity measures and across multiple dose groups

11/10/15

CANCER

Adaptimmune Therapeutics plc (Oxford, U.K.)

Affinity enhanced T-cell therapy

Targets the NY-ESO-1 cancer antigen

Non-small-cell lung cancer (NSCLC)

Started a phase I/II trial of its affinity-enhanced T-cell therapy targeting the NY-ESO-1 cancer antigen in patients with stage IIIb or stage IV NSCLC

11/25/15

Advaxis Inc. (Princeton, N.J.)

ADXS-HPV

Axalimogene filolisbac

Persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix

Phase I data showed it may be safely administered with prophylactic antibiotics up to 1 x 1010 CFU, a 10-fold increase

11/10/15

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-120

Oral selective inhibitor of mutant isocitrate dehydrogenase-1

Advanced solid tumors

First data from the dose-escalation portion of the ongoing phase I study showed no dose-limiting toxicities

11/10/15

Aptose Biosciences Inc. (San Diego)

APTO-253

Small molecule that induces a gene that expresses the Krüppel-like factor 4 (KLF4) master transcription factor

Hematologic cancers

FDA placed a clinical hold on the phase Ib trial, following the company's voluntary suspension of dosing due to an operational difficulty with an intravenous infusion pump

11/23/15

Beigene Co. Ltd. (Beijing)

BGB-290

Poly-ADP ribose polymerase inhibitor

Solid tumors

A first-in-human dose-escalation trial enrolled 29 patients that were treated; at the 80 mg twice daily, the maximum dose has not been reached, and dose escalation is ongoing; responses were seen even at the lowest dose tested; of 15 ovarian cancer patients, seven responded to the drug, with one complete and six partial responses

11/10/15

Calithera Biosciences Inc. (South San Francisco)

CB-839

Selective, orally bioavailable glutaminase inhibitor

Renal cell carcinoma

Phase I data support earlier findings of the clinical activity, tolerability and unique mechanism of action, as well as show one partial response, and stable disease across a variety of tumor types

11/10/15

Cellceutix Corp. (Beverly, Mass.)

Kevetrin

Small molecule

Solid tumors

Completed enrollment in the phase I study

11/12/15

Cerulean Pharma Inc. (Cambridge, Mass.)

CRLX101

Nanoparticle-based, camptothecin-loaded

Advanced gastric cancers

Pre- and post-tumor treatment biopsies show the presence of CRLX101 and its anticancer payload in tumors and an almost exclusive absence of both from surrounding normal tissue; inhibition of the molecular targets of CRLX101 was demonstrated in post-treatment biopsies

11/10/15

Chemocentryx Inc. (Mountain View, Calif.)

CCX872

A CCR2 inhibitor

Pancreatic cancer

Phase Ib data showed that, after a single dose, across four patients in part A of the study, the pharmacokinetic profile was favorable and pharmacodynamic assays demonstrated excellent receptor coverage after 12 hours

11/5/15

Cortice Biosciences Inc. (New York)

TPI287

A microtubule stabilizing agent

Recurrent glioblastoma

Phase I/II data showed that of 11 evaluable patients, one achieved a complete response and three achieved stable disease; median progression-free survival is 1.4 months, and median overall survival is 4.7 months

11/23/15

Essa Pharma Inc. (Houston)

EPI-506

Intended to block a new target on the androgen receptor, the N-terminal domain

Metastatic castration-resistant prostate cancer

The first patient was enrolled in its phase I/II study

11/30/15

Five Prime Therapeutics Inc. (South San Francisco)

FPA144

An isoform-selective antibody

Gastric cancer

The dose-escalation part of the ongoing phase I trial has been completed and dose expansion has begun at a selected dose in new cohorts of patients whose tumors overexpress FGFR2b

11/23/15

Galena Biopharma Inc. (San Ramon, Calif.)

GALE-302

E39 peptide

Ovarian and breast cancer

Data from a recent phase Ib trial showed it achieved a statistically significant increase in local reactions and delayed type hypersensitivity when delivering GALE-302 after E39 (GALE-301) in the primary vaccine series (p<0.001)

11/10/15

Genocea Biosciences Inc. (Cambridge, Mass.)

ATLAS

Rapid antigen identification screening system

Cancer

Findings from a pilot study involving a retrospective analysis of 10 checkpoint inhibitor-treated patients' T-cell responses to 23 known tumor-associated antigens support the potential of ATLAS, which successfully identified the cancer antigens to which either (or both) CD4+ or CD8+ T cells became activated

11/5/15

Halozyme Therapeutics Inc. (San Diego) and Janssen Biotech Inc. (unit of Johnson & Johnson; New Brunswick, N.J.)

Daratumumab

Subcutaneously delivered with Halozyme's Enhanze technology

Multiple myeloma

Dosed the first patient in a phase Ib trial

11/5/15

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

HS-410

Vesigenurtacel-L

High-risk, non-muscle-invasive bladder cancer

Phase I results of HS-410 after standard-of-care Bacillus Calmette-Guerin showed the drug had a positive safety profile and was well tolerated with no patients discontinuing the trial due to adverse events

11/9/15

Helix Biopharma Corp. (Aurora, Ontario)

L-DOS47

An immunoconjugate-based drug

Cancer

Opened patient screening for the 14th dose level cohort of its ongoing phase I/II study in Poland; during the 13th cohort, one patient experienced a serious adverse event that was a dose-limiting toxicity and was withdrawn from further dosing and later recovered from the event

11/5/15

Ignyta Inc. (San Diego)

RXDX-105

Orally available, small molecule multikinase inhibitor

Cancer

Interim results from the company's ongoing phase I trial showed it was generally well tolerated

11/10/15

Immunogen Inc. (Waltham, Mass.)

Mirvetuximab soravtansine

Folate receptor alpha-targeting antibody-drug conjugate candidate

Platinum-resistant ovarian cancer

Phase I data in 20 efficacy-evaluable patients identified an association between the amount of FRα present on patient cancer cells and response to treatment with the drug; it found nine of 10 patients with high levels of FRα had an objective response on treatment

11/10/15

Immuodulon Therapeutics Ltd. (London)

IMM-101

A bacterially derived systemic immunomodulator administered intradermally

Stage IV metastatic melanoma

The latest five-year survival results showed that of the 10 patients who entered the open-label, long-term follow-up study after the original phase I study in 2010, six are alive and still being treated

11/20/15

Incyte Corp. (Wilmington, Del.)

Epacadostat

Selective indoleamine 2,3-dioxygenase inhibitor

Advanced cancers

Results from an ongoing proof-of-concept phase I/II study of epacadostat in combination with anti-PD-1 drug Keytruda (pembrolizumab, Merck & Co. Inc.) indicated a 79% disease control rate in evaluable patients

11/4/15

Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.)

INO-3112

DNA-based immunotherapy

Head and neck cancer associated with human papillomavirus types 16 and 18

Interim data from a phase I/IIa study showed that it generated specific T-cell responses and was well tolerated

11/6/15

Loxo Oncology Inc. (Stamford, Conn.)

LOXO-101

A selective inhibitor of tropomyosin receptor kinase signaling molecules

Solid tumors

Phase I data showed all three evaluable patients achieved an objective response at the first response assessment

11/9/15

Mirna Therapeutics Inc. (Austin, Texas)

MRX34

MiR-34 replacement therapy

Solid tumors

Interim results from its ongoing phase I trial has a safety profile manageable with standard interventions or tests used by oncologists, and demonstrates the therapeutic potential

11/10/15

Molecular Partners AG (Zurich-Schlieren, Switzerland)

MP0250

Multi-Darpin-blocking vascular endothelial growth factor and hepatocyte growth factor

Solid tumors

Preliminary phase I data showed that MP0250 was well tolerated in 15 patients with solid tumors, with favorable pharmacokinetics and sustained exposure over multiple applications

11/9/15

Newlink Genetics Corp. (Ames, Iowa)

Indoximod

Indoleamine-(2,3)-dioxygenase pathway inhibitor

Glioblastoma

Phase Ib data showed that indoximod in combination with temozolomide demonstrated preliminary efficacy, including a six-month progression-free survival rate of 25%

11/24/15

Oncoceutics Inc. (Philadelphia)

ONC-201

Small molecule with an active angular structure

Solid tumors

Phase I data showed the study advanced through all five intended dose levels, 125 mg through 625 mg, with only one patient per dose level, and identified a recommended phase II dose

11/3/15

Oncoceutics Inc. (Philadelphia)

ONC201

Small-molecule drug

Multiple myeloma

Began enrolling patients with various solid tumors in a dose-escalation trial

11/30/15

Oncolytics Biotech Inc. (Calgary, Alberta)

Reolysin

Reovirus variant

Relapsed or refractory multiple myeloma

Initiated a phase Ib study of Reolysin in combination with standard doses of bortezomib (Velcade, Millennium: The Takeda Oncology Co.) and dexamethasone

11/19/15

Onxeo SA (Paris)

Belinostat

Pan-histone deacetylase inhibitor

Peripheral T-cell lymphoma

Phase I data of belinostat in combination with CHOP chemotherapy regimen demonstrated it to be well-tolerated with an objective response rate of 89% based on 18 evaluable patients (16/18), with the vast majority, 72%, achieving a complete response (13/18) and 17% achieving a partial response (3/18)

11/10/15

Oxigene Inc. (South San Francisco)

CA4P

Combretastatin A4 Prodrug

Advanced recurrent ovarian cancer

Initial phase Ib/II data of CA4P, in combination with Votrient (pazopanib, Glaxosmithkline plc), showing that, of nine patients, two showed partial responses, five had stable disease and two had progressive disease

11/9/15

Polaris Group (San Diego)

ADI-PEG 20

Pegylated arginine deiminase

Malignant plural mesothelioma and nonsquamous non-small-cell lung carcinoma

Phase I preliminary results showed no dose-limiting toxicity observed in the nine patients enrolled; seven patients had partial responses and two patients had stable disease as best response, for a 100% disease control rate

11/12/15

Seattle Genetics Inc. (Bothell, Wash.)

SGN-CD33A

Vadastuximab talirine

Relapsed or refractory acute myeloid leukemia

Started a phase I/II trial

11/24/15

Targovax AS (Lysaker, Norway)

ONCOS-102

Adenovirus-based cancer immunotherapy

Advanced tumors

Immune biomarker data from a phase I study suggested that local immunotherapy with ONCOS-102 has the potential to activate immunologically silent tumors and reduce local immune suppression

11/6/15

Tocagen Inc. (San Diego)

Toca 511 in combination with Toca Fc

Injectable RRV and extended-release version of 5-FC

Glioma and other cancers

Phase I and preclinical data demonstrated a favorable safety profile and extended overall survival in patients

11/24/15

Tracon Pharmaceuticals Inc. (San Diego)

TRC105

A monoclonal antibody to endoglin

Advanced or metastatic renal cell carcinoma

Phase Ib data of TRC105 with Inlyta (axitinib, Pfizer Inc.) showed an overall response rate in 29% of those receiving the combination treatment, with 59% showing stable disease, for an overall disease control rate of 88%

11/9/15

Virttu Biologics Ltd. (Glasgow, UK)

Seprehvir

Oncolytic immunotherapeutic

Mesothelioma

Ongoing phase I/IIa clinical trial data identified a robust Th1 cytokine response and the appearance of antitumor IgG responses in most treated patients, confirming Seprehvir's potential for immunological efficacy

11/6/15

Ziopharm Oncology Inc. (Boston)

Veledimex

Ad-RTS-hIL-12+ orally administered veledimex; a viral gene therapy candidate for the controlled expression of IL-12

Recurrent or progressive glioblastoma or grade III malignant glioma

Results from an ongoing phase I dose-escalation study showed IL-12 was detectable in peripheral blood along with downstream interferon-gamma, indicating it crossed the blood-brain barrier

11/20/15

CARDIOVASCULAR

Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) and The Medicines Co. (Parsippany, N.J.)

ALN-PCSsc

Subcutaneous administration

High cholesterol

Results from the ongoing phase I trial showed that subcutaneous administration resulted in an up to 83% lowering of LDL-C, with an up to 64 ± 5 percent mean maximum reduction

11/12/15

Capricor Therapeutics Inc. (Los Angeles)

CAP-1002

Allogeneic, cardiosphere-derived cell therapy

Advanced heart failure

Multi-vessel intracoronary infusion of CAP-1002 in subjects with dilated cardiomyopathy was shown to be safe in this study with no major adverse cardiac events reported at one month or at six months post-infusion; the six-month data demonstrated encouraging and congruent preliminary efficacy signals in multiple parameters

11/10/15

Ironwood Pharmaceuticals Inc. (Cambridge, Mass.)

IW-1973 and IW-1701

Soluble guanylate cyclase stimulators

Cardiovascular diseases, fibrotic diseases, muscular dystrophy and other disorders

Started a phase Ib study of IW-1973 and a phase Ia study of IW-1701

11/4/15

CENTRAL NERVOUS SYSTEM

Aequus Pharmaceuticals Inc. (Vancouver, British Columbia)

AQS1301

Once-weekly transdermal patch

Bipolar disorder, schizophrenia, major depressive disorder and irritability associated with autistic disorder

Will initiate a phase I trial following receipt of a no objection letter from Health Canada

11/17/15

Alzheon Inc. (Framingham, Mass.)

ALZ-801

A prodrug of tramiprosate

Alzheimer's disease

Started two studies, a phase Ib multiple dose-ranging trial and a phase Ib tablet bioequivalence study

11/9/15

Neurocrine Biosciences Inc. (San Diego)

NBI-640756

A vesicular monoamine transporter 2 inhibitor

Essential tremor

Started a sequential dose-escalation phase I safety and pharmacokinetics study exploring NBI-640756 in up to 32 healthy volunteers

11/5/15

Ocera Therapeutics Inc. (Palo Alto, Calif.)

OCR-002

Ornithine phenylacetate

Neurocognitive symptoms of acute hepatic encephalopathy

Phase I study showed the oral formulation in healthy subjects exhibited encouraging extended-release properties, demonstrated a desirable pharmacokinetic profile and was well tolerated

11/18/15

Otonomy Inc. (San Diego)

OTO-311

A sustained-exposure formulation of the N-Methyl-D-Aspartate receptor antagonist, gacyclidine

Tinnitus

Enrolled the first subjects in a phase I safety trial

11/17/15

Portage Biotech Inc. (Toronto)

BHV-0223

A glutamate modulating agent

Mood and anxiety disorders

Preliminary results showed a phase I trial met its study objectives and supports advancing it into later stages of development

11/23/15

Rhythm Pharmaceuticals Inc. (Boston)

RM-493

Setmelanotide; melanocortin-4 receptor

MC4R heterozygous deficiency obesity

Phase Ib trial showed that it reduced weight after four weeks of treatment, with good tolerability; it demonstrated strong trends for placebo-subtracted weight loss ( 2.62 kg; p=0.088), waist circumference ( 5.1 cm; p = 0.188) and daily caloric intake ( 351 kCal/day; p=not significant), without clinically important effects on heart rate or blood pressure

11/9/15

Turing Pharmaceuticals AG (unit of Turing Pharmaceuticals LLC; New York)

TUR-004

Oral formulation

Epileptic encephalopathies

The first subjects were dosed in the single ascending dose study

11/17/15

Zosano Pharma Corp. (Fremont, Calif.)

ZP-Triptan

Zolmitriptan-coated microneedle patch

Migraine

Appears to be well tolerated and produced no safety issues during a phase I study

11/4/15

DIABETES

Adocia SAS (Lyon, France)

Biochaperone Combo

A formulation combining insulin glargine and insulin lispro

Type 2 diabetes

Top-line phase Ib data showed it was significantly superior to Humalog Mix75/25 and exhibited no difference compared to the separate injections of Lantus and Humalog

11/30/15

GASTROINTESTINAL

Hutchison China Meditech Ltd. (Hong Kong)

HMPL 523

A selective small-molecule inhibitor targeting spleen tyrosine kinase

Hypertension and severe diarrhea

Completed its phase I study in healthy volunteers in Australia; no material off-target toxicities were observed and it exhibited a linear pharmacokinetic profile and a dose-dependent suppression of B-cell activation

11/2/15

INFECTION

Alios Biopharma Inc. (South San Francisco)

ALS-008176

Cytidine nucleoside analogue

Respiratory syncytial virus

In a randomized, double-blind study, 62 healthy volunteers were inoculated with RSV and randomized to ALS-008176 or placebo, in which the treatment resulted in a greater reduction (73% to 88% under the curve) of viral load and faster viral clearance compared to placebo

11/20/15

Arsanis Inc. (Waltham, Mass.)

ASN100

Combination of two monoclonal antibodies

Staphylococcus aureus

The first cohort was initiated in the phase I trial

11/24/15

Cidara Therapeutics Inc. (San Diego)

CD101

Intravenous therapy

Candidemia

Proved well tolerated systemically and at infusion sites across entire dose ranges during a phase I single ascending-dose study in healthy volunteers

11/18/15

Nitto Denko Corp. (Osaka, Japan)

ND-L02-s0201

Targeted siRNA lipid nanoparticle

Non-alcoholic steatohepatitis and hepatitis C virus liver fibrosis

Phase Ib/II studies showed it was well tolerated, with histological improvement of fibrosis observed

11/23/15

Scynexis Inc. (Jersey City, N.J.)

SCY-078

Oral glucan synthase inhibitor

Invasive and life-threatening fungal infections

Started a phase I trial

11/13/15

Transgene SA (Paris)

TG1050

Immunotherapy candidate

Chronic hepatitis B virus infection

The first patient was dosed in a phase I/Ib study

11/5/15

INFLAMMATORY

Complexa Inc. (Pittsburgh)

CXA-10

Endogenous nitro-fatty acid modulator of Nrf2 and NF-κB

Inflammation and fibrosis

Completed an extensive phase I development program

11/13/15

Galapagos NV (Mechelen, Belgium)

GLPG1972

First-in-class candidate

Osteoarthritis

Began dosing in a phase I study

11/23/15

MISCELLANEOUS

Allergan plc (Dublin)

Bimatoprost

Sustained-release

Glaucoma

Six-month interim results from a 24-month phase I/II trial of 75 patients showed that a single dose lowered intraocular pressure in 92% of patients at four months and 71% at six months while demonstrating a favorable efficacy and safety profile

11/17/15

Arqule Inc. (Burlington, Mass.)

ARQ 092

Orally available, small molecule multikinase inhibitor

Proteus syndrome

Enrolled the first patient in the first clinical trial

11/18/15

Biolinerx Ltd. (Tel Aviv, Israel)

BL-8040

Synthetic peptide that functions as a high-affinity antagonist of CXCR4

Bone marrow failure conditions hypoplastic myelodysplastic syndrome (hMDS) and aplastic anemia (AA)

Started a phase I/II trial

11/24/15

Bluebird Bio Inc. (Cambridge, Mass.)

Lentiglobin BB305

Gene therapy

Beta-thalassemia

Company reported that transfusion independence was achieved in patients with beta-thalassemia major with non-beta-zero genotypes followed for at least six months in the Northstar and HGB-205 studies

11/6/15

Eyegate Pharmaceuticals Inc. (Waltham, Mass.)

EGP-437

A combination of reformulated corticosteroid dexamethasone phosphate delivered using the company's Eyegate II delivery system

Macular edema

Interim data from an ongoing phase Ib/IIa pilot trial showed positive responses in some of the patients, with pseudophakic eyes; the therapy also showed no serious treatment-emergent adverse effects, including no increase in ocular pressure even at three times the iontophoretic dose

11/6/15

Ix Biopharma Ltd. (Singapore)

Pheonix

Sildenafil

Erectile dysfunction

Completed a pilot bioavailability study comparing the pharmacokinetics of the sildenafil in Pheonix with that in Viagra (Pfizer Inc.), demonstrating Pheonix has a similar rate and extent of drug absorption as a Viagra tablet

11/30/15

Kadmon Corp. LLC (New York)

Tesevatinib

Oral tyrosine kinase inhibitor

Autosomal dominant polycystic kidney disease

Phase Ib/IIa data demonstrated the tolerability of tesevatinib at 50 mg, 100 mg and 150 mg QD, with rash occurring in the 150-mg QD dose cohort; the phase IIa portion evaluated tesevatinib 150 mg administered twice or three times weekly and found that the tolerability of the intermittent dosing schedules was improved over 150-mg QD, but rash still occurred

11/3/15

Miragen Therapeutics Inc. (Boulder, Colo.)

MRG-201

Synthetic microRNA mimic (promiR) to microRNA-29b

Cutaneous scleroderma

Initiated a phase I study

11/13/15

Novabay Pharmaceuticals Inc. (Emeryville, Calif.)

NVC-422

Antimicrobial compound auriclosene

To reduce urinary catheter blockage and encrustation

Completed a 34-patient study evaluating it in patients with long-term indwelling urinary catheters

11/5/15

Panoptica Inc. (Bernardsville, N.J.)

PAN-90806

Topical, anti-VEGF eye drop

Neovascular age-related macular degeneration

Preliminary results from the monotherapy arm of a phase I/II study showed signals of safety and anti-VEGF biological activity

11/13/15

Prometic Life Sciences Inc. (Laval, Quebec)

Plasma-derived plasminogen

Replacement therapy

Plasminogen-deficiency

Phase I therapy was used to treat an infant in critical condition in an intensive care unit at the Altona Children's Hospital in Hamburg, Germany; the administration of plasma was not successful in raising his plasminogen to an effective level, so the company's plasminogen was provided and within a few days, physicians observed reduction of the lesions

11/5/15

Retrotope Inc. (Los Altos, Calif.)

RT001

Designed as a stabilized fatty-acid aimed at shutting down the degradation of lipids in mitochondrial and cellular membranes

Friedreich's ataxia

Opened the second site for the ongoing, 28-day phase I/II study, testing ascending doses of orally dosed RT001

11/23/15

Rxi Pharmaceuticals Corp. (Marlborough, Mass.)

RXI-109

An sd-rxRNA

Retinal scarring

Initiated a phase I/II trial

11/6/15

RESPIRATORY

Concert Pharmaceuticals Inc. (Lexington, Mass.)

CTP-656

Deuterium-modified ivacaftor

Cystic fibrosis

Began a phase I multiple ascending-dose trial

11/13/15

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.