Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Arena Pharmaceuticals Inc. (San Diego)

APD334

Oral candidate designed to target sphingosine 1-phosphate subtype receptor

Autoimmune diseases

Started dosing in a phase Ib trial

6/4/14

Protalex Inc. (Florham Park, N.J.)

PRTX-100

Incorporates a highly purified form of Staphyloccal protein A

Active rheumatoid arthritis

Phase Ib data of PRTX-100 in combination with methotrexate or leflunomide indicated that the drug was generally safe and well tolerated; data from the unblinded analysis of 41 patients also revealed positive effects of treatment on certain measures of disease activity, though those effects did not reach statistical significance

6/4/14

CANCER

Advanced Cancer Therapeutics Inc. (Louisville, Ky.)

PFK-158

Small-molecule candidate that inactivates a cancer metabolism target by inhibiting 6-phosphofructo-2-kinase/fructose-2, 6-biphosphatase 3

Solid tumors

Started phase I testing

6/5/14

Bio-Path Holdings Inc. (Houston)

BP-100-1.01

Liposomal Grb-2

Blood cancers

Company is resuming its phase I trial now that sufficient drug quantities are in place to complete cohort six of the trial

6/20/14

Curis Inc. (Lexington, Mass.)

CUDC-427

An oral small molecule

Advanced and/or refractory solid tumors or lymphomas

Re-initiated dosing in the phase I single-agent trial

6/6/14

Deciphera Pharmaceuticals LLC (Cambridge, Mass.)

DCC-2701

MET/TIE2/VEGFR2/TRK inhibitor altiratinib

Solid tumors

Started a phase I trial

6/2/14

Genspera Inc. (San Antonio)

G-202

Activated by the prostate-specific membrane antigen enzyme

Hepatocellular carcinoma

Interim results from a phase Ib and ongoing phase II study in patients who had previously progressed on or who were intolerant of Nexavar showed that 80% had stable disease at two months and 50% exhibited stable disease at four months

6/26/14

Glycomimetics Inc. (Gaithersburg, Md.)

GMI-1271

An E-selectin antagonist

Acute myeloid leukemia

The first healthy volunteer was dosed in its phase I study designed to test the safety, tolerability and pharmacokinetics of GMI-1271

6/20/14

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

HS-410

Vesigenpuma-tucel-L

Bladder cancer

Dose escalation committee has approved the expansion of the first cohort to full enrollment in its ongoing phase I/II study

6/2/14

Immune Design Corp. (Seattle)

LV305

An immune-oncology agent from the firm's Dcvex lentiviral vector platform

Locally advanced, relapsed or metastatic breast cancer, melanoma, non-small-cell lung cancer, ovarian cancer or sarcoma

Treated the first patient in a phase I trial that will enroll up to 36 patients

6/6/14

Inovio Pharmaceuticals Inc. (Blue Bell, Pa.)

INO-3112

Immunotherapy; a combination of Inovio's lead immunotherapy, VGX-3100, and its immune activator expressing interleukin-12

Human papillomavirus-associated head and neck cancer

Started a phase I/IIa trial

6/11/14

Isis Pharmaceuticals Inc. (Carlsbad, Calif.) and Astrazeneca plc (London)

ISIS-ARRx

An antisense drug that inhibits the production of the androgen receptor

Various cancers

Initiated a phase I study of ISIS-ARRx (AZD5312); the open-label, dose-escalation study will test the drug in patients with advanced solid tumors, including metastatic castrate-resistant prostate, breast, bladder and ovarian cancers, where the AR pathway is potentially a contributing factor

6/11/14

Kadmon Corp. LLC (New York)

KD019

Orally bioavailable small-molecule kinase inhibitor of Src, HER2, EGFR and VEGFR2/KDR

HER2-positive breast cancer metastasized to the brain

Started a phase Ib/IIa study

6/5/14

Macrogenics Inc. (Rockville, Md.)

MGD006

A humanized Dual-Affinity Re-Targeting bispecific antibody-based molecule

Relapsed or refractory acute myeloid leukemia

The first patient was dosed in a phase I study

6/20/14

Mirna Therapeutics Inc. (Austin, Texas)

MRX34

A microRNA mimic

Cancer

Enrolled the first patient in the hematological malignancy cohort of its ongoing phase I trial

6/11/14

Northwest Biotherapeutics Inc. (Bethesda, Md.)

Dcvax-Direct

Personalized immune therapy

Inoperable solid tumors

In an ongoing phase I/II trial, all nine patients who have received four of the six planned injections are showing tumor cell death, tumor shrinkage, substantial immune cell accumulation in their tumors and/or stabilization of their advanced cancers

6/12/14

Oncomed Pharmaceuticals Inc. (Redwood City, Calif.)

Vantictumab and Fzd8-Fc

Monoclonal antibodies targeting the Wnt pathway

Cancer

Company halted six phase Ib trials testing two Bayer AG-partnered drugs due to "on-target mild-to-moderate" bone-related adverse events

6/16/14

CARDIOVASCULAR

Bluebird Bio Inc. (Cambridge, Mass.)

Lentiglobin BB305

Gene therapy

Beta-thalassemia major

Phase I/II data from a study in Paris showed that at 4.5 months following autologous transplant, one patient had total hemoglobin of 10.1 g/dL, of which 6.6 g/dL was therapeutic betaAT87Q-globin 9, and at two months the second post-transplant patient showed total hemoglobin of 11.6 g/dL, of which 4.2 g/dL was betaAT87Q-globin; Lentiglobin was well tolerated

6/17/14

Capstone Therapeutics Inc. (Tempe, Ariz.)

AEM-28

Apo E mimetic peptide

Refractory hypercholes-terolemia

Started dosing for the phase Ib/IIa trial in refractory hypercholesterolemic subjects who are already on optimal cholesterol lowering therapy but are unable to reach target cholesterol levels

6/12/14

Edge Therapeutics Inc. (Berkeley Heights, N.J.)

EG-1962

Polymeric microparticle form of the calcium channel blocker nimodipine

Aneurysmal subarachnoid hemorrhage

Is advancing into the second cohort of a phase I/II trial after a data safety monitoring committee recommended increasing the dose to 200 mg, since no unexpected drug-related serious adverse events showed up with the 100-mg dose in the first cohort

6/6/14

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

REG2 anticoagulation system

Two-component system consisting of a subcutaneous depot formulation of pegnivacogin and anivamersen

Venous thrombosis indications such as DVT and VTE prophylaxis

Reported results from a phase I trial demonstrating the dose- and concentration-dependent attenuation of thrombin generation and subsequent restoration of thrombin following reversal associated with REG2

6/24/14

CENTRAL NERVOUS SYSTEM

Cara Therapeutics Inc. (Shelton, Conn.)

CR845

Peripherally selective kappa opioid agonist

Acute and chronic pain

Dosed the first subjects in a phase Ia/Ib trial

6/19/14

Coronado Biosciences Inc. (Burlington, Mass.)

CNDO-201

Trichuris suis ova; oral therapy

Autism spectrum disorder

Data from a pilot study showed it failed to reach statistical significance, though benefits of TSO over placebo were observed in several subscales, including the Montefiore-Einstein Rigidity Scale, ABC Irritability, RBS-R Sameness and RBS-R Restricted Behavior

6/27/14

Elite Pharmaceuticals Inc. (Northvale, N.J.)

ELI-200

An abuse deterrent product

Pain

The first subject was dosed in the treatment phase of a human abuse liability clinical study for ELI-200

6/10/14

Enteris Biopharma Inc. (Boonton, N.J.) and Cara Therapeutics Inc. (Shelton, Conn.)

CR845

Peripherally selective kappa opioid agonist; tablet formulation

Acute and chronic pain

Dosed the first subjects in a further phase Ia/Ib trial of a tablet formulation

6/23/14

Sage Therapeutics Inc. (Cambridge, Mass.)

SAGE-547

An allosteric modulator of GABAA receptors

Super-refractory status epilepticus

Preliminary data from its ongoing open-label phase I/II trial showing that the first three patients met both the primary endpoint of safety and tolerability and the key secondary endpoint of being successfully weaned off their anesthetic agents while SAGE-547 was being administered

6/6/14

Trevena Inc. (King of Prussia, Pa.)

TRV734

Mu-opioid receptor biased ligand

Acute and chronic pain

Results from a phase I trial demonstrated that it is pharmacologically active at a range of safe and well-tolerated doses

6/24/14

Trevena Inc. (King of Prussia, Pa.)

TRV130

G protein-biased ligand at the mu-opioid receptor

Acute pain

Phase Ib data suggest that it may reduce pain more effectively, act more quickly and produce less severe gastrointestinal side effects, reducing the risk of respiratory depression compared to existing opioids, improving the margin of safety for dosing

6/25/14

DIABETES

Prometic Life Sciences Inc. (Laval, Quebec)

PBI-4050

Oral anti-fibrotic drug

Diabetes

Completed a phase I trial in 40 healthy volunteers and found the oral anti-fibrotic drug to be safe and well tolerated, without any serious adverse events reported in any of the five cohorts tested

6/19/14

Rhythm Pharmaceuticals Inc. (Boston)

RM-493

Melanocortin 4 receptor (MC4R) agonist

Obesity

Results from a crossover study in 12 obese individuals suggested that RM-493 increased resting energy expenditure significantly

6/24/14

Targacept Inc. (Winston-Salem, N.C.)

TC-6499

Small molecule that modulates the activity of the alpha3beta4 and other neuronal nicotinic receptors

Diabetic gastroparesis

Started a phase I/II exploratory study

6/26/14

INFECTION

Achillion Pharmaceuticals Inc. (New Haven, Conn.)

ACH-3422

A uridine-analogue nucleotide polyermase inhibitor

Genotype 1 chronic hepatitis C virus infection

Started dosing for seven days in a phase I trial

6/11/14

Basilea Pharmaceutica Ltd. (Basel, Switzerland)

BAL30072

Gram-negative antibiotic

Infections

Started a phase I study

6/13/14

Calimmune Inc. (San Diego)

Cal-1

Gene-based stem cell therapy

HIV

The data safety monitoring board for a phase I/II trial confirmed no serious adverse events or dangerous side effects in the first group of patients, indicating that the firm can begin treating a second cohort

6/26/14

Debiopharm Group (Lausanne, Switzerland)

Debio 1450

An anti-infective agent selectively active against a large number of Staphylococcus species

Methicillin-resistant S. aureus and vancomycin-intermediate S. aureus

Launched a phase I study

6/20/14

Dilaforette AB (Stockholm)

Sevuparin

Malaria treatment

Malaria

The drug was found to be safe and well tolerated as adjunct treatment in adults with uncomplicated falciparum malaria but did not achieve statistical significance in the primary endpoint

6/2/14

Genocea Biosciences Inc. (Cambridge, Mass.)

GEN-004

Vaccine

To prevent infections from all serotypes of pneumococcus, or Streptococcus pneumoniae

Results from a phase I study met its safety, tolerability and immunogenicity goals, including measurable increases in the blood of T helper 17 (TH17) cells; serum IgG titers increased in a dose-dependent manner to each of the antigens included in GEN-004, and measurable increases in peripheral TH17 responses were seen among subjects receiving the highest dose (100 mcg) with adjuvant

6/24/14

Themis Bioscience GmbH (Vienna)

Vaccine

Prophylactic vaccine candidate

Chikungunya fever

Induced a significant neutralizing immune response and was confirmed as safe, based on interim results from a phase I study

6/13/14

INFLAMMATORY

Cellular Biomedicine Group Inc. (Palo Alto, Calif.)

Rejoin

Human adipose-derived mesenchymal precursor cell therapy

Knee osteoarthritis

Six-month follow-up data analysis of its phase I/IIa trial showed an increase in cartilage volume of whole joint as early as three months after therapy, as measured via MRI Quantitative Assessment, and showed reduction of bone marrow lesions in some patients

6/20/14

MISCELLANEOUS

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-348

An orally available activator of pyruvate kinase-R

Pyruvate kinase deficiency

A multiple ascending dose phase I trial in healthy volunteers has started

6/10/14

Bone Therapeutics SA (Gosselies, Belgium)

Allob

Allogeneic osteoblastic (bone-forming) cell therapy

Delayed union fractures

The first patient was dosed in a phase I/IIa trial

6/26/14

Isis Pharmaceuticals Inc. (Carlsbad, Calif.)

ISIS-DMPKRx

Designed to improve the underlying genetic defect that causes DM1

Myotonic dystrophy type 1

Initiated a phase I study

6/10/14

Ligand Pharmaceuticals Inc. (San Diego)

Lasofoxifene

Selective estrogen receptor modulator

To increase circulating testosterone and gonadotropin levels

Data from the first-in-human study, performed by Pfizer Inc., showed a single dose of lasofoxifene to increase circulating testosterone and gonadotropin levels in healthy young men; single oral doses of lasofoxifene of 1 mg, 3 mg, 30 mg and 100 mg all increased levels of testosterone, with the 30-mg and 100-mg doses raising levels by more than 80%

6/24/14

Medgenics Inc. (Wayne, Pa.)

MDGN-201

Epodure

End-stage renal disease or chronic kidney disease

The first patient was enrolled in its phase I/II study, with the aim of validating the potential of the firm's Biopump platform using a second-generation vector

6/6/14

Stemcells Inc. (Newark, Calif.)

HUCNS-SC

Human neural stem cells

Dry age-related macular degeneration

Closed enrollment in its phase I/II trial with interim data showing a 70% reduction in the rate of geographic atrophy (GA) vs. the control eye and a 65% reduction in the rate of GA vs. the natural history of the disease, following a single dose

6/23/14

Synageva Biopharma Corp. (Lexington, Mass.)

SBC-102

Sebelipase alfa

Lysosomal acid lipase deficiency

Data showed that sebelipase alfa continues to improve disease-related abnormalities at two years in an ongoing phase I/II trial

6/4/14

Ultragenyx Pharmaceutical Inc. (Novato, Calif.)

KRN23 (US023)

Anti-FGF23 antibody

X-linked hypophos-phatemia

Data from a phase I/II study demonstrated that blocking excess FGF23 increases urinary phosphorus reabsorption, serum phosphorus levels and 1,25 dihydroxy vitamin D levels, and repeat doses over four months led to an increase in serum phosphorus in 100% of patients, with about 89% reaching the low end of the normal range

6/26/14


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.