Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Acorda Therapeutics Inc. (Ardsley, N.Y.)

rHIgM22

A remyelinating antibody

Multiple sclerosis

Phase I data found no dose-limiting toxicities at any of the five dose levels studied

2/3/15

Akaal Pharma Pty Ltd. (Melbourne, Australia)

AKP-11

A topical sphingosine 1-phosphate receptor-1 modulator

Mild to moderate plaque psoriasis

Results from a randomized, double-blind, placebo-controlled phase I trial showed it was found to be safe, well tolerated and resulted in a significant reduction in plaque severity

2/6/15

Alkermes plc (Dublin)

ALKS 8700

Monomethyl fumarate molecule

Multiple sclerosis

Positive topline results from a three-part, randomized, double-blind phase I study showed it was generally well tolerated and provided MMF exposures comparable to Tecfidera, with less variability and with improved gastrointestinal (GI) tolerability; the most common adverse events (AE) were flushing and GI-related symptoms, and no serious AEs or discontinuations due to AEs were observed for ALKS 8700

2/10/15

Aurinia Pharmaceuticals Inc. (Victoria, British Columbia)

Luveniq

Voclosporin

Active lupus nephritis

Plans to initiate an open-label, exploratory study to assess the short-term predictors of response using voclosporin in combination with mycophenolate mofetil; the company expects to complete patient enrollment of the pilot study by the third quarter

2/10/15

Oncobiologics Inc. (Cranbury, N.J.)

ONS-3010

Adalimumab (Humira, Abbvie Inc.) biosimilar candidate

Rheumatoid arthritis

ONS-3010 met the primary endpoints in its first clinical study, a three-arm single-dose pharmacokinetic trial in healthy volunteers that compared ONS-3010 both to the U.S.- and EU-sourced Humira reference products, and the two reference products to each other

2/13/15

CANCER

Aileron Therapeutics Inc. (Cambridge, Mass.)

ALRN-6924

A potent and specific re-activator of the tumor suppressor protein known as wild type p53

Advanced hematologic and solid malignancies

Dosed the first patients in a phase I trial of ALRN-6924

2/13/15

Apeiron Biologics AG (Vienna)

APN401

Cellular therapy

Melanoma, pancreatic cancer and other metastatic solid tumors

The first patient was dosed in a phase I trial

2/27/15

Bavarian Nordic A/S (Kvistgaard, Denmark)

Prostvac

Immunotherapy

Genitourinary cancers

Patients treated with Prostvac, plus escalating doses of Yervoy (ipilimumab, Bristol-Myers Squibb Co.), in a phase I study, achieved median overall survival of 31.3 months for all dose cohorts and 37.2 months when treated at 10 mg/kg; about 20% of patients treated with a 10 mg/kg dose were alive at 80 months after initiating treatment

2/25/15

Celsion Corp. (Lawrenceville, N.J.)

GEN-1

Interleukin-12-producing immunotherapy

Advanced platinum-resistant and recurrent ovarian cancer

Phase Ib data of GEN-1 combined with Doxil demonstrated its power to effectively recruit a cellular immune system response

2/23/15

Dicerna Pharmaceuticals Inc. (Cambridge, Mass.)

DCR-MYC

Dicer substrate short interfering RNA

Advanced hepatocellular carcinoma

The first patient was dosed in a global phase Ib/II trial

2/3/15

Helix Biopharma Corp. (Aurora, Ontario)

L-DOS47

Immunoconjugate-based drug

Inoperable, locally advanced, recurrent or metastatic, nonsquamous, stage IIIb/IV non-small-cell lung cancer

Opened patient screening for the 11th dose level cohort in its ongoing phase I/II trial in Poland

2/18/15

Immune Design Corp. (Seattle)

G100

Endogenous antigen therapy

Metastatic sarcoma

Dosed the first patient in a phase I study of G100, in combination with radiation therapy

2/12/15

Merus AB (Utrecht, the Netherlands)

MCLA-128

An ADCC-enhanced, full-length IgG bispecific antibody

Solid tumors

Started a phase I/II study

2/5/15

Polaris Group (San Diego)

ADI-PEG 20

Designed to deplete the external supply of arginine

Advanced gastro-intestinal malignancies

The first patient was dosed in the phase I trial of ADI-PEG 20 in combination with folinic acid (leucovorin), fluorouracil and oxaliplatin

2/4/15

Seattle Genetics Inc. (Bothell, Wash.)

SEA-CD40

Immuno-oncology agent targeted to CD40

Advanced solid tumors

Started a phase I trial

2/18/15

CARDIOVASCULAR

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

EG-1962

A polymeric nimodipine microparticle administered directly into patients' brain ventricles

Subarachnoid hemorrhage from a ruptured brain aneurysm

Reported that its ongoing phase I/II study completed enrollment and yielded safety and 90-day functional outcome efficacy data for 24 patients

2/12/15

Isis Pharmaceuticals Inc. (Carlsbad, Calif.)

ISIS-PKKRx

RNA-targeted antisense drug

Hereditary angioedema

Results from a phase I study showed that healthy volunteers achieved dose-dependent reductions of up to 95% in prekallikrein (PKK)

2/25/15

CENTRAL NERVOUS SYSTEM

Alkermes plc (Dublin)

ALKS 7106

Opioid-based treatment

Pain

Company halted development following phase I data in which the drug did not meet pre-specified criteria

2/25/15

Bionomics Ltd. (Melbourne, Australia)

BNC210

Mechanism of action is by negative allosteric modulation of the alpha 7 nicotinic acetylcholine receptor

Anxiety and depression

Started a phase Ib trial in healthy volunteers; the drug's mechanism of action is by negative allosteric modulation of the alpha 7 nicotinic acetylcholine receptor.

2/3/15

Chromadex Corp. (Irvine, Calif.)

Niagen

Nicotinamide riboside; a form of Niacin

Cockayne syndrome and other age-related neuro-degenerative conditions

Met its primary endpoint in the company's first phase I trial

2/12/15

Relmada Therapeutics Inc. (New York)

Mepigel

Topical dosage form of local anesthetic mepivacaine

Neuropathic pain

Selected the formulations to advance into clinical studies, that will be tested in upcoming single- and multiple-dose phase I studies in healthy volunteers

2/5/15

Relmada Therapeutics Inc. (New York)

Butab

Oral, enteric-coated buprenorphine

Chronic moderate to moderately severe pain and opioid dependence

Filed a clinical trial application with Health Canada to conduct a phase I pharmacokinetic study, which is in development to treat both chronic moderate to moderately severe pain and opioid dependence

2/25/15

Trevena Inc. (King of Prussia, Pa.)

TRV734

Oral candidate

Moderate-to-severe acute and chronic pain

Data from a multiple ascending dose phase I study showed that pharmacokinetics, safety, tolerability and central nervous system activity were consistent with a previous phase I study

2/10/15

DIABETES

Kamada Ltd. (Ness Ziona, Israel)

Glassia

Intravenous alpha1-proteinase inhibitor therapy

Type 1 diabetes

Additional data from its ongoing extension study of the open-label phase I/II trial showed that mean peak C-peptide levels, reflecting endogenous insulin production and corresponding beta cell activity, were 0.40 pmol/ml, with 60% of these patients exhibiting a level ≥ 0.2 pmol/ml – considered a clinically meaningful trough level corresponding to fewer future serious diabetes complications

2/3/15

INFECTION

Dipexium Pharmaceuticals Inc. (New York)

Locilex

Pexiganan cream 0.8%; topical antibiotic peptide

Bacterial infections

Initial results of DPX-120, a phase I skin sensitization trial, demonstrated low potential for inducing contact sensitization; in total, only three out of 203 evaluable subjects were characterized as sensitized

2/18/15

Novavax Inc. (Gaithersburg, Md.)

Ebola vaccine

Ebola virus glycoprotein recombinant nanoparticle vaccine candidate adjuvanted with Matrix-M

Ebola virus

Launched enrollment in a phase I trial of the vaccine in healthy adults

2/13/15

Regulus Therapeutics Inc. (La Jolla, Calif.)

RG-101

Galnac-conjugated anti-miR targeting microRNA-122

Hepatitis C virus infection

Treatment with a single subcutaneous dose of 4 mg/kg of RG-101 as monotherapy resulted in significant and sustained viral load reductions in all patients, including difficult to treat genotypes, various liver fibrosis status and those who have experienced viral relapse after a prior IFN-containing regimen

2/10/15

Sangamo Biosciences Inc. (Richmond, Calif.)

SB-728-T

Zinc finger DNA-binding protein

HIV/AIDS

Phase I/II study demonstrated a dose-related increase in both total CD4 T-cell and ZFP transcription factor-modified CD4 T-cell engraftment in response to Cytoxan preconditioning up to 1 g/m2; all subjects underwent a treatment interruption and were taken off antiretroviral drugs at 16 weeks post-infusion

2/27/15

The Global Alliance for TB Drug Development (New York)

TBA-354

From the nitromidazole class

Tuberculosis

Started the first human trial of TBA-354

2/19/15

MISCELLANEOUS

Alnylam Pharmaceuticals Inc. (Cambridge, Mass.)

ALN-CC5

A subcutaneously administered RNAi therapeutic targeting complement component C5

Complement-mediated diseases

Started a phase I/II trial

2/3/15

Apellis Pharmaceuticals Inc. (Crestwood, Ky.)

APL-2

A next-generation inhibitor of the class of compstatin derivatives

Age-related macular degeneration

Began a phase I trial

2/11/15

Arrowhead Research Corp. (Pasadena, Calif.)

ARC-AAT

RNAi candidate

Liver disease associated with rare genetic disorder alpha-1 antitrypsin deficiency

Started dosing in a phase I trial; the study will be conducted in two parts, with part A enrolling healthy volunteers and part B recruiting patients with AATD

2/23/15

Cell Cure Neurosciences Ltd. (Jerusalem)

Opregen

A cell therapy

Dry age-related macular degeneration

Started a phase I/IIa study

2/19/15

Dilafor AB (Stockholm)

Tafoxiparin

Labor induction candidate

For labor induction

Completed a safety and pharmacokinetic multidosing study showing it was safe and well tolerated, with a predictive pharmacokinetic profile in both subcutaneous and intravenous administration

2/26/15

Flex Pharma Inc. (Boston)

Unnamed treatment

Designed to stop the firing of neurons by topically stimulating transient receptor potential ion channels in the gastrointestinal tract

Leg cramps

Findings based on three randomized, blinded, placebo-controlled crossover trials in 37 healthy volunteers showed that leg cramps are caused by excessive firing of alpha-motor neurons in the spinal cord that control muscle contraction; each study showed statistically significant results indicating that the company's treatment reduced electrically induced muscle cramps within 15 minutes, with the effect lasting six to eight hours, compared to subjects who received a vehicle control

2/20/15

Obseva SA (Geneva)

OBE001

Orally active oxytocin receptor antagonist

Preterm labor

A phase I study of OBE001 in 12 healthy women showed no adverse interactions with the corticosteroid betamethasone

2/27/15

Pluristem Therapeutics Inc. (Haifa, Israel)

PLX-PAD

Placental expanded cells

Muscle injury after total hip arthroplasty

Reported an additional finding from its phase I/II trial showing that six months after surgery the magnitude of improvement in muscle force of the contralateral (non-operated) gluteal muscle was approximately 40 times larger in patients treated with 150M PLX-PAD cells than in those who received placebo, and the difference was statistically significant (19.4 vs. 0.5 Nm, p = 0.0114)

2/3/15

Protalix Biotherapeutics Inc. (Carmiel, Israel)

PRX-102

A recombinant chemically modified version of human alpha-Galactosidase-A enzyme

Fabry disease

Phase I/II data showed that PRX-102 has a significantly longer circulatory half-life vs. currently marketed enzyme replacement therapies and that treatment with 0.2 mg/kg for six months demonstrated meaningful improvement or stability in main Fabry disease parameters, including stable cardiac and kidney function

2/13/15

Reveragen Biopharma (Silver Spring, Md.)

VBP15

Dissociative steroidal drug

Duchenne muscular dystrophy

Started a phase I trial

2/19/15

Stealth Biotherapeutics Inc. (Boston)

Bendavia

Targets the inner mitochondrial membrane

Mitochondrial myopathy

Started a phase I/II trial

2/18/15

Strategic Science and Technologies LLC (Cambridge, Mass.)

SST-6006

Topical sildenafil candidate

Female sexual arousal disorder

Initiated a phase I trial

2/10/15

RESPIRATORY

Adamis Pharmaceuticals Corp. (San Diego)

APC-1000

Generic version of inhaled oral steroid Qvar (beclomethasone dipropionate HFA; Teva Pharmaceuticals Co. Ltd.)

Asthma

A phase I, open-label, randomized, single-dose, four-way crossover pharmacokinetic study led it to predict that APC-1000 "will be as safe and efficacious as Qvar" ahead of the conclusion of an ongoing phase III noninferiority study

2/25/15

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.