Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Akari Therapeutics plc (London)

Coversin

A second-generation complement inhibitor that acts on complement component-C5

Autoimmune diseases

UK Medicines & Healthcare products Regulatory Agency cleared the firm to conduct a phase Ib multiple ascending-dose trial

2/1/16

JHL Biotech Inc.

JHL1101

Biosimilar rituximab (Mabthera; Roche AG)

Rheumatoid arthritis

Received approval to start clinical trials in the Greater China region

2/18/16

Resolve Therapeutics LLC (Seattle)

RSLV-132

A novel Fc fusion protein consisting of human RNase attached to the Fc portion of human IgG 

System lupus erythematosus

Data from a multicenter, double-blind, placebo-controlled multiple ascending dose study of RSLV-132 in 32 subjects demonstrated safety and tolerability

2/11/16

Rock Creek Pharmaceuticals Inc. (Sarasota, Fla.)

Anatabine citrate

A small-molecule cholinergic agonist

Mild to moderate psoriasis

Received an interim report of the pharmacodynamic results of its phase I trial, showing that it produced significant reductions in STAT3 (signal transducer and activator of transcription 3), a key marker of inflammation, in volunteers

2/4/16

CANCER

Ablynx NV (Ghent, Belgium) and Boehringer Ingelheim GmbH (Ingelheim, Germany)

Nanobody

Half-life extended bispecific anti-VEGF/Ang2 nanobody

Advanced solid tumors

Administered the first dose in a phase I dose-escalation study

2/1/16

ADC Therapeutics SA (Lausanne, Switzerland)

ADCT-301

Antibody drug conjugate

Acute myeloid leukemia

The first patient was dosed in a phase I study

2/10/16

Argen-x NV (Breda, the Netherlands)

ARGX-111

MET-targeting human monoclonal "Simple Antibody"

Cancer

Opened three clinical trial sites in South Korea for the recruitment of MET-amplified cancer patients for the phase I safety expansion cohort

2/19/16

Astrazeneca plc (London) and Medimmune

Durvalumab and tremelimumab

Combination treatment of the anti-PD-L1 immune checkpoint inhibitor and the anti-CTLA-4 antibody

Locally advanced or metastatic non-small-cell lung cancer

Phase Ib data showed antitumor activity with a confirmed objective response rate of 23%

2/9/16

Biothera Pharmaceuticals Inc. (Eagan, Minn.)

Imprime PGG

A systemically administered beta glucan Pathogen Associated Molecular Patterning molecule

Cancer

Results from two phase I studies showed that both single doses and repeated doses of Imprime PGG demonstrated a favorable safety profile and were well tolerated; a total of 36 healthy volunteers were enrolled

2/26/16

Bluebird Bio Inc. (Cambridge, Mass.)

bb2121

A chimeric antigen receptor T-cell therapy targeting B-cell maturation antigen

Relapsed or refractory multiple myeloma

Treated the first patient in a phase I study

2/18/16

Cellceutix Corp. (Beverly, Mass.)

Kevetrin

A small molecule believed to induce activation of p53

Advanced solid tumors

Completed its phase I trial; phase I data showed that regardless of tumor types, 27 of the 40 patients (67.5%) had increases in p21 expression relative to pre-treatment levels; for those with the greatest increases at seven or 24 hours, the mean percent increase at 24 hours was 24.5%

2/17/16; 2/23/16

Celldex Therapeutics Inc. (Hampton, N.J.)

CDX-301

Recombinant human Flt3 ligand

Allogeneic hematopoietic stem cell transplantation

New data on CDX-301 from an open-label pilot study testing the hematopoietic cytokine alone and in combination with Mozobil (plerixafor, Sanofi SA) in sibling-matched donors who have certain hematologic malignancies showed that it was well tolerated and effective at mobilizing hematopoietic stem cells in healthy donors when given as a single agent for five days

2/23/16

Celsion Corp. (Lawrenceville, N.J.)

GEN-1

DNA-based immunotherapy

Ovarian cancer

Completed enrollment in the first cohort of patients in its phase Ib dose escalation trial, called Ovation

2/16/16

Celyad SA (Mont-Saint-Guibert, France)

NKR-2 T-cell therapy

Uses T cells with NKG2D receptor

Acute myeloid leukemia or multiple myeloma

Completed the 21-day safety follow-up of the last patient enrolled in the second dose level in the phase I/IIa study; no safety issues were reported

2/26/16

Critical Outcome Technologies Inc. (London, Ontario)

COTI-2

Has a p53-dependent mechanism

Gynecological cancers

The first patient in the phase I trial was dosed

2/17/16

Ignyta Inc. (San Diego)

Taladegib

Oral Hedgehog/Smoothened inhibitor

Platinum-resistant, recurrent ovarian cancer or recurrent, advanced solid tumors

Cancer Research UK initiated a phase I/Ib trial testing a combination of paclitaxel with taladegib

2/5/16

Immune Design Corp. (Seattle)

CMB305

A prime-boost approach in which LV305 is dosed sequentially with G305 to induce a synergistic anti-tumor cytotoxic T lymphocyte response

Cancers expressing the NY-ESO-1 tumor antigen

Data showed it was safe, without dose-limiting toxicities, and a significant subset of treated patients had NY-ESO-1 specific CD8 T cell responses that were generated or increased after therapy

2/10/16

Immune Design Corp. (Seattle)

G100

An intratumoral TLR4 agonist

Cancer

Phase I data showed the compound significantly modified the tumor microenvironment and maintained clinical benefit

2/10/16

Innate Pharma SA (Marseille, France)

Monalizumab

Checkpoint inhibitors; an anti-NKG2A antibody

Cancer

Started a phase I combination trial of monalizumab and PD-L1 candidate durvalumab (MEDI4736; Astrazeneca plc)

2/9/16

Mabvax Therapeutics Holdings Inc. (San Diego)

89Zr-Humab-5B1

New generation PET scan cancer imaging agent

Pancreatic cancer

Received notice from the FDA authorizing the start of a phase I trial

2/2/16

Oncbiomune Pharmaceuticals Inc. (Baton Rouge, La.)

Proscavax

Therapeutic vaccine

Prostate cancer

Phase I data showed it was safe and suggested initial indications of efficacy

2/24/16

Oncomed Pharmaceuticals Inc. (Redwood City, Calif.)

OMP-59R5

Tarextumab; anti-Notch2/3

Small-cell lung cancer

Updated survival data from a phase Ib trial showed that median progression-free survival was 4.4 months and the median overall survival was 10.3 months

2/23/16

Seattle Genetics Inc. (Bothell, Wash.)

SGN-CD19B

An antibody-drug conjugate targeted to CD19 using the firm's newest ADC technology

Relapsed or refractory patients with two subtypes of B-cell non-Hodgkin lymphoma

Started a phase I trial in diffuse large B-cell lymphoma and grade 3 follicular lymphoma

2/26/16

Soricimed Biopharma Inc. (Toronto)

SOR-C13

Binds to TRPV6, a calcium channel that is highly elevated in carcinomas

Advanced solid tumor cancers

Top-line phase I data showed the drug was generally safe and well tolerated by subjects and appeared to demonstrate potential activity, with 54% of subjects achieving stable disease

2/24/16

Strategia Therapeutics Inc. (Boston) and Fujifilm Pharmaceuticals U.S.A. Inc.

FF-10502

Antimetabolite agent; inhibits DNA synthesis

Advanced solid tumor cancers and lymphoma

Started a U.S.-based phase I trial

2/22/16

Trillium Therapeutics Inc. (Toronto)

TTI-621

A checkpoint inhibitor; an antibody-like fusion protein designed to block CD47

Relapsed or refractory hematologic malignancies

Started dosing in a phase I trial

2/4/16

Ziopharm Oncology Inc. (Boston)

CAR-T-cell therapy

Second generation non-viral CD19-specific chimeric antigen receptor modified T-cell therapy

Advance lymphoid malignancies

The first patient has been enrolled in a phase I study

2/10/16

Ziopharm Oncology Inc. (Boston)

Ad-RTS-hIL-12+veledimex

Viral gene therapy

Recurrent or progressive glioblastoma or grade III malignant glioma

The first patient was dosed at the succeeding dose level in the phase I study

2/25/16

CARDIOVASCULAR

Biocardia Inc. (San Carlos, Calif.)

Bone marrow cell therapy

Heart-muscle directed

Acute myocardial infarction

Nine people who experienced AMI participating in a phase I study showed statistically significant and clinically meaningful improvements at 12 month follow-up in New York Heart Association class, improvements in B-type natriuretic peptide levels, and improvements in left ventricular ejection fraction

2/23/16

Edge Therapeutics Inc. (Los Angeles)

EG-1962

A polymeric nimodipine microparticle

Aneurysmal subarachnoid hemorrhage

Phase I/II NEWTON data demonstrated a 3.5-day reduction in intensive care unit length of stay compared to oral nimodipine (median EG-1962 LOS was 13.5 days, median oral nimodipine LOS was 17 days)

2/22/16

Merganser Biotech Inc. (King of Prussia, Pa.)

M012

A hepcidin mimetic peptide

Rare hematological and iron overload diseases

Started dosing in the first clinical trial of M012 to treat diseases such as beta thalassemia and certain subtypes of myelodysplasia

2/25/16

CENTRAL NERVOUS SYSTEM

Aequus Pharmaceuticals Inc. (Vancouver, British Columbia)

AQS1301

A once-weekly transdermal form of aripiprazole

Central nervous system disorders

Results from an initial bioavailability study of lead program AQS1301 suggested that sustained, seven-day delivery of therapeutic doses of aripiprazole is possible with the current formulation; the formulation also was well tolerated

2/5/16

Biohaven Pharmaceutical Holding Co. Ltd. (British Virgin Islands)

BHV-0223

A glutamate modulating agent

Amyotrophic lateral sclerosis

Reported results from a phase I study confirming that the pharmacokinetic, safety and tolerability data with sublingual BHV-0223 support advancement of clinical development; data also showed that lower doses of BHV-0223 appear to have similar exposure profile to the current standard of care

2/25/16

Corium International Inc. (Menlo Park, Calif.)

Corplex

Once-weekly transdermal patch delivering the acetylcholinesterase inhibitor donepezil

Alzheimer's disease

Phase I data after a single application of the once-weekly Corplex patch, plasma concentrations approached steady state at the seventh day, with the average plasma concentration on that day via Corplex higher than the comparator, oral Aricept 5 mg, and about 70% of the expected plasma concentration of oral Aricept 10 mg

2/5/16

Heptares Therapeutics Inc. (subsidiary of Sosei Group Corp.; Tokyo)

HTL9936

The first selective muscarinic M1 receptor agonist

Dementia and schizophrenia

Positive phase Ib data exhibited robust and statistically significant changes in brain electrical activity measured using multiple electroencephalography biomarkers relevant to cognition

2/11/16

Kempharm Inc. (Coralville, Iowa)

KP201/APAP

An immediate-release, abuse-deterrent combination of KP201, a prodrug of hydrocodone, and acetaminophen

Pain

Data showed it was associated with lower exposure to hydrocodone at the eight- and 12-tablet doses compared to Norco (hydrocodone bitartrate/APAP), while exposure at the low-dose (four tablets) was bioequivalent; about 25% and 34% of subjects demonstrated a 20% or greater reduction in Cmax and AUC0-1 measures of exposure to hydrocodone, respectively, within the first hour at the 12-tablet dose of KP201/APAP as compared to an equivalent dose of Norco

2/22/16

Lpath Inc. (San Diego)

Lpathomab

A humanized monoclonal antibody targeting lysophosphatidic acid

Conditions of neuronal injury

Completed dosing in a phase Ia, double-blind, placebo-controlled, single ascending-dose study, showing the drug was well tolerated at all doses, with no serious adverse events or dose-limiting toxicities

2/2/16

Minerva Neurosciences Inc. (Waltham, Mass.)

MIN-202

JNJ-42847922; a selective orexin-2 receptor antagonist

Insomnia disorder and major depressive disorder

Top-line phase I data in patients in Japan showed that single-dose morning administration of MIN-202 was well tolerated at all three dose levels tested – 5 mg, 20 mg and 40 mg – and the observed plasma pharmacokinetic features were comparable to those seen in previous studies carried out in healthy non-Asian participants

2/2/16

Oryzon Genomics SA (Barcelona, Spain)

ORY-2001

A highly selective dual LSD1-MAOB inhibitor

Alzheimer's disease

Gained approval of its clinical trial application from the Spanish Drug Agency to initiate a phase I study

2/2/16

DIABETES

Adocia SA (Lyon, France) and Eli Lilly and Co. (Indianapolis)

Biochaperone Lispro

An ultra-rapid formulation of insulin Lispro

Diabetes

Started a phase Ib trial in 15 healthy Japanese subjects

2/2/16

Redhill Biopharma Ltd. (Tel Aviv, Israel)

Bekinda

RHB-102; an extended-release, once-daily oral pill formulation of the antiemetic drug ondansetron

Diarrhea-predominant irritable bowel syndrome

Successfully completed a first-in-human pharmacokinetic study at a dosage of 12 mg, which will be used in the phase II study

2/12/16

INFECTION

Benitec Biopharma Ltd. (Sydney)

TT-034

ddRNAi therapeutic

Hepatitis C virus

Is terminating further development of the phase I/IIa drug due to increasing competition, "limited and diminishing partnering interest" and a long road to market

2/29/16

Biota Pharmaceuticals Inc. (Atlanta)

BTA585

An oral respiratory syncytial virus fusion inhibitor

Respiratory syncytial virus

Data from the phase I multiple ascending-dose trial indicated BTA585 was generally well tolerated at all dose levels; there were no serious adverse events, and no drug-related, clinically significant adverse changes in ECGs or clinical laboratory values were observed

2/29/16

Iliad Biotechnologies LLC (New York)

BPZE1

A next-generation pertussis vaccine

Whooping cough

Completed enrollment of three randomized groups of study subjects in a phase Ib vaccine trial studying two higher doses and volumes of BZPZE1 as a follow-up to a prior phase I trial

2/22/16

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

MAT2501

Encochleated amikacin

Nontuberculous mycobacterium infections

FDA has cleared its investigational new drug application for a phase I trial

2/2/16

Seres Therapeutics Inc. (Cambridge, Mass.)

SER-109

An oral capsule; lead Ecobiotic microbiome therapeutic

Recurrent Clostridium difficile infection

Phase Ib/II data demonstrated that 87% of patients met the predefined endpoint of preventing recurrent CDI within eight weeks following administration of SER-109; 97% of patients achieved clinical cure during the eight-week period after SER-109 dosing, as defined by the absence of CDI requiring antibiotic treatment

2/10/16

Visterra Inc. (Cambridge, Mass.)

VIS410

Monoclonal antibody

Influenza A

Phase I data demonstrated that VIS410 was generally safe and well tolerated and that there were no serious adverse events or discontinuations that were drug related

2/29/16

MISCELLANEOUS

Achillion Pharmaceuticals Inc. (New Haven, Conn.)

ACH-4471

Orally administered small molecule; inhibits complement factor D

Paroxysmal nocturnal hemoglobinuria

Started a phase I study

2/12/16

Aeolus Pharmaceuticals Inc. (Mission Viejo, Calif.)

AEOL 10150

Investigational drug

Lung – acute radiation exposure

FDA removed a clinical hold, allowing the company to start its phase I study

2/23/16

Capricor Therapeutics Inc. (Los Angeles)

CAP-1002

Allogeneic, cardiosphere-derived stem cell therapy

Duchenne muscular dystrophy-related cardiomyopathy

Treated the first patient in the phase I/II HOPE-Duchenne trial

2/23/16

Curavac Inc. (Wilmington, Del.)

CV-MG01

An immunotherapy

Myasthenia gravis

Initiated patient enrollment in a phase Ib study

2/10/16

Lipocine Inc. (Salt Lake City)

LPCN 1107

Oral hydroxyprogesterone caproate

To prevent preterm birth

Top-line results from its multidose, pharmacokinetic, dose-finding study showed that average steady state HPC levels were comparable or higher for all three doses than for injectable HPC

2/17/16

Palatin Technologies Inc. (Cranbury, N.J.)

Bremelanotide

Subcutaneous, on-demand treatment

Female sexual dysfunction in premenopausal women with hypoactive sexual desire disorder

Data from a bremelanotide and alcohol use interaction phase I study concluded that the two can be safely co-administered and are generally well tolerated with no reports of drug-related serious adverse events

2/29/16

Prothena Corp. plc (Dublin)

NEOD001

A humanized monoclonal antibody that specifically targets the circulating soluble amyloid and deposited insoluble amyloid

Amyloid light-chain amyloidosis

Interim phase I/II data showed that monthly infusions were safe and well tolerated and that patients showed improvements in functional biomarkers predictive of improvement of disease and survival

2/10/16

Seattle Genetics Inc. (Bothell, Wash.)

SGN-CD33A

Vadastuximab talirine; an antibody-drug conjugate targeted to CD33

Myelodysplastic syndrome

Started a 130-patient, phase I/II trial

2/23/16

RESPIRATORY

Catalyst Pharmaceuticals Inc. (Coral Gables, Fla.)

Firdapse

Amifampridine phosphate

MuSK-antibody positive myasthenia gravis

An investigator-sponsored trial was initiated to evaluate the safety, tolerability and efficacy of Firdapse for symptomatic relief

2/9/16

DBV Technologies SA (Bagneux, France)

Viaskin Peanut

Peanut allergy therapy

Peanut allergy

Phase Ib data showed it was observed to have a favorable safety and tolerability profile; high adherence to treatment, which was documented by an overall 96% compliance rate and a 4% dropout, was also observed; Viaskin Peanut has fast track designation and breakthrough therapy designation in the U.S.

2/29/16

Pulmatrix Inc. (Lexington, Mass.)

PUR0200

An ISPERSE (Inhaled Small Particles Easily Respirable and Emitted) engineered dry powder form of a marketed long acting muscarinic antagonist bronchodilator

Chronic obstructive pulmonary disease

Enrollment is under way in the company's European pilot bioequivalence study

2/2/16

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.