Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Aurinia Pharmaceuticals Inc. (Victoria, British Columbia)

Voclosporin

A calcineurin inhibitor

Lupus nephritis

Enrolled the first patient in its phase IIb trial, called AURA-LV

6/27/14

Receptos Inc. (San Diego)

RPC1063

A sphingosine-1-phosphate (S1P) receptor modulator

Relapsing multiple sclerosis

Phase II data showed that patients on RPC1063 turned up a statistically significant reduction in gadolinium-enhancing lesions of 86% at both doses compared to patients on placebo; secondary endpoints measuring effects on other MRI parameters were also positive and statistically significant

6/1/14

Xenoport Inc. (Santa Clara, Calif.)

XP23829

Next-generation fumaric acid candidate

Moderate to severe chronic plaque-type psoriasis

Initiated a phase II trial to assess efficacy, safety and tolerability

6/24/14

CANCER

Alchemia Ltd. (Brisbane, Australia)

HA-Irinotecan

HyACT drug

Metastatic colorectal cancer

The first patient was enrolled in the phase II CHIME trial of HA-Irinotecan and Erbitux (cetuximab, Eli Lilly and Co.)

6/23/14

Ambit Biosciences Corp. (San Diego)

AC220

Quizartinib; an FLT3 inhibitor

FLT3-ITD-positive relapsed or refractory acute myeloid leukemia

Final phase II data showed a complete response rate of 47% and the rate of hematopoietic stem cell transplantation after quizartinib treatment was 34%; the median overall survival was 20.9 weeks for patients initially treated at 30 mg/day and 25.4 weeks for patients at 60 mg/day

6/13/14

Basilea Pharmaceutica Ltd. (Basel, Switzerland)

BAL101553

An oral small-molecule microtubule targeting agent with a dual action

Advanced or recurrent solid tumors

Started a phase IIa study in adult patients who have failed standard therapy or for whom no effective standard therapy is available

6/27/14

Can-Fite Biopharma Ltd. (Petach Tikva, Israel)

CF102

Second-line treatment

Advanced liver cancer

FDA has agreed with the study protocol for its phase II trial, and granted CF102 orphan drug designation in this indication

6/3/14

Clovis Oncology Inc. (Boulder, Colo.)

Rucaparib

An oral, small-molecule PARP inhibitor

Pancreatic cancer

Started with the dosing of the first patient in an open-label phase II study

6/17/14

CTI Biopharma Corp. (Seattle)

Tosedostat

A selective inhibitor of aminopeptidases

Acute myeloid leukemia or high-risk myelodysplastic syndrome

Started an international cooperative group phase II trial

6/13/14

Cyclacel Pharmaceuticals Inc. (Berkeley Heights, N.J.)

Sapacitabine

An oral nucleoside analogue

Myelodysplastic syndromes

The company will conduct a phase IIb randomized, controlled trial of a regimen of sapacitabine alternating with low dose chemotherapy versus an active control of low dose chemotherapy

6/3/14

Halozyme Therapeutics Inc. (San Diego)

PEGPH20

Based on the firm's recombinant human hyaluronidase enzyme

Pancreatic cancer

FDA removed the clinical hold on patient enrollment and dosing of PEGPH20 in the ongoing phase II study and permitted the study to resume under a revised protocol; the clinical hold was placed on the trial earlier this year after the company reported an unexpected imbalance in thromboembolic events between patients in the control group and those receiving a regimen containing PEGPH20 in combination with Abraxane (nab-paclitaxel, Celgene Corp. and gemcitabine)

6/6/14

Hutchison Medipharma Ltd. (Hong Kong)

Fruquintinib

Small molecule designed to selectively inhibit vascular endothelial growth factor receptors

Non-small-cell lung cancer

Initiated a phase II trial

6/6/14

Karyopharm Therapeutics Inc. (Natick, Mass.)

KPT-330

Oral selective inhibitor of nuclear export, or SINE, compound selinexor

Metastatic hormone-refractory prostate cancer

Initiated a phase II trial that will enroll approximately 50 patients with metastatic HRPC following treatment with at least one recently approved agent, including enzalutamide (Xtandi, Medivation Inc./Astellas Pharma Inc.), abiraterone (Zytiga, Johnson & Johnson) or radium 223 (Xofigo, Bayer AG)

6/10/14

Karyopharm Therapeutics Inc. (Natick, Mass.)

Selinexor (KPT-330)

XPO1 inhibitor

Acute myeloid leukemia (AML)

Initiated a phase II study in patients 60 years of age or older with relapsed or refractory AML who are ineligible for intensive chemotherapy and/or transplantation

6/25/14

Madison Vaccines Inc. (Madison, Wis.)

MVI-816 (pTVG-HP)

Plasmid DNA vaccine that targets prostatic acid phosphatase

Prostate cancer

Started enrollment in the expansion of a phase II trial for nonmetastatic prostate cancer patients who have rising levels of prostate-specific antigen after their primary treatment – either surgery or radiation – but before patients require androgen deprivation therapy

6/24/14

MEI Pharma Inc. (San Diego)

Pracinostat

Oral histone deacetylase (HDAC) inhibitor

Acute myeloid leukemia

Preliminary response data from the first stage of the two-stage, open-label phase II study showed that two patients achieved complete responses (CRs) and one achieved a CR with incomplete blood count recovery, each following one or two treatment cycles; the data met the pre-specified rate required to advance to the second stage of the trial

6/11/14

Newlink Genetics Corp. (Ames, Iowa)

Dorgenmel-tucel-L

Hyperacute immunotherapy

Melanoma

Phase II study of the immunotherapy in combination with pegylated interferon-alpha 2b demonstrated that the combination was capable of inducing complete and durable clinical responses with tumor regression and immune activation

6/2/14

Prima Biomed Ltd. (Sydney, Australia)

Cvac

Autologous dendritic cell-based product

Epithelial ovarian cancer

Reported phase II data showing that some patients treated with Cvac showed a decline or stabilization in levels of CA125

6/24/14

Quest Pharmatech Inc. (Edmonton, Alberta)

Oregovomab

Immunotherapy product candidate

Ovarian cancer

Patient enrollment has been completed in the company's phase IIb trial

6/10/14

Taiho Pharmaceutical Co. Ltd. (Tokyo) and Pharmamar SA (Madrid, Spain)

Yondelis

Antitumor agent

Malignant soft-tissue sarcoma

A phase II trial testing Yondelis in Japan showed that it significantly improved progression-free survival (PFS) in comparison to best standard of care (BSC), with median PFS of 5.6 months for the treatment arm vs. 0.9 months for the BSC arm (p < 0.0001)

6/5/14

Telormedix SA (Bioggio, Switzerland)

Vesimune

TMX-101; a toll-like receptor 7 agonist

Skin malignancies

Four of 10 evaluable patients with carcinoma in situ (CIS) of the bladder exhibited a response to Vesimune in a phase II trial that recruited 12 patients in all

6/2/14

Tolero Pharmaceuticals Inc. (Salt Lake City)

Alvocidib

Flavopiridol; a small-molecule inhibitor of cyclin-dependent kinases

Acute myeloid leukemia

Positive interim results from an ongoing, randomized phase II trial alvocidib, in combination with cytarabine plus mitoxantrone, showed complete response rates of 70% vs. 46% with the standard of care (p=0.003)

6/9/14

CARDIOVASCULAR

Ablynx NV (Ghent, Belgium)

Caplacizumab

A bivalent antibody fragment that neutralizes von Willebrand Factor

Thrombotic thrombocyto-penic purpura

Phase II data were positive; the company has initiated dosing in a phase I bioequivalence study of a new lyophilized formulation and is laying plans for a phase III

6/18/14

Cerenis Therapeutics SA (Toulouse, France)

CER-001

An engineered human apoA-I-containing pre-beta HDL mimetic

Familial primary hypoalpha-lipoproteinemia and homozygous familial hypercholest-erolemia

Two of its phase II studies, SAMBA and MODE (Modifying Orphan Disease Evaluation), with CER-001 met their primary clinical endpoints

6/3/14

D-Pharm Ltd. (Rehovot, Israel)

THR-18

Cardiovascular treatment

Acute ischemic stroke

A dose-finding phase II study of THR-18 confirmed the tolerability of the drug at a dose of 0.54 mg/kg when administered together with tissue plasminogen activator

6/3/14

Ischemix Inc. (Maynard, Mass.)

CMX-2043

Chemically modified, naturally occurring molecule

To prevent contrast-induced acute kidney injury in acute coronary syndrome patients undergoing coronary angiography

Enrolled the first patient in a phase IIa trial

6/27/14

Portola Pharmaceuticals Inc. (South San Francisco)

Andexanet alfa

A factor Xa inhibitor antidote

Venous thrombo-embolism prevention

Its phase II proof-of-concept study in healthy volunteers demonstrated that andexanet alfa immediately reversed the anticoagulation activity of enoxaparin, a low molecular weight heparin and standard of care in venous thromboembolism prevention; andexanet alfa was well tolerated, with no serious adverse events reported

6/12/14

CENTRAL NERVOUS SYSTEM

Affiris AG (Vienna, Austria)

AD02

Peptide-based therapeutic vaccine

Alzheimer's disease

Phase II data found that the substance it used as placebo, now dubbed AD04, appeared to halt disease progression through a poorly understood effect unconnected with AD02

6/5/14

Alder Biopharmaceuticals Inc. (Bothell, Wash.)

ALD403

A genetically engineered monoclonal antibody targeting CGRP

To prevent frequent episodic migraine

A randomized, double-blind, placebo-controlled, proof-of-concept trial showed that ALD403 met the primary endpoint, significantly reducing mean migraine days per month vs. placebo

6/27/14

Alkermes plc (Dublin)

ALKS 3831

Broad-spectrum oral antipsychotic

Schizophrenia

Started a second phase II study

6/3/14

Brainstorm Cell Therapeutics Inc. (New York and Petach Tikvah, Israel)

Nurown

Autologous mesenchymal stem cells secreting neurotrophic factors

Amyotrophic lateral sclerosis

The first patient has been enrolled in its phase II ALS trial

6/9/14

Convergence Pharmaceuticals Ltd. (Cambridge, UK)

CNV1014802

Sodium channel blocker

Severe facial pain trigeminal neuralgia

Phase II data showed a 60% reduction in the number of paroxysms suffered vs. a 12% reduction in the placebo arm; pain severity dropped by 55% in the treatment arm against 18% in placebo, by the end of the study

6/17/14

Cytokinetics Inc. (South San Francisco)

Tirasemtiv

Part of its skeletal muscle contractility program

Amyotrophic lateral sclerosis

Data showed that tirasemtiv may have a durable impact on slow vital capacity maintained through four weeks after the last dose of a 12-week course of treatment; the analysis adds further information to earlier results from BENEFIT-ALS, the company's phase IIb trial

6/3/14

Essentialis Inc. (Carlsbad, Calif.)

Diazoxide choline

Controlled-release tablets

Prader-Willi syndrome

Dosed the first patient in study PC025, which will enroll 12 PWS patients between 10 and 20 years old

6/30/14

Jazz Pharmaceuticals plc (Dublin)

JZP-110

Formerly ADX-NO5

Excessive daytime sleepiness in narcolepsy

Data from a phase IIb study showed that all primary and secondary endpoints were met and patients treated with JZP-110 experienced statistically significant improvements in objective and subjective symptoms of EDS

6/3/14

DIABETES

Ico Therapeutics Inc. (Vancouver, British Columbia)

Ico-007

A second-generation antisense drug targeting c-Raf kinase

Diabetic macular edema

Top-line results for the eight-month visual acuity (VA) primary endpoint for patients enrolled in its phase II IDEAL study found negative mean changes in VA measures in all four enrolled patient groups at months four and eight; findings showed that losses in VA were greater at higher dosage levels

6/10/14

Microbiome Therapeutics LLC (Broomfield, Colo.)

NM504

Microbiome modulator

Prediabetes and type 2 diabetes

Results from a randomized, placebo-controlled trial showed that the drug can improve blood glucose control and other metabolic parameters, and also decreased cholesterol levels and markers of chronic inflammation in the treated patients

6/24/14

Noxxon Pharma AG (Berlin)

NOX-E36

Emapticap pegol

Diabetic nephropathy

A phase IIa trial showed "strong trends" for reduction of protein in the urine as measured by the albumin to creatinine ratio, as well as for HbA1c levels in blood; the reductions were statistically significant in a subgroup of 49 patients that Noxxon contended to be most relevant for future studies in the indication

6/3/14

GASTROINTESTINAL

Galapagos NV (Mechelen, Belgium)

GLPG974

Free fatty acid receptor 2 inhibitor

Ulcerative colitis

Failed to demonstrate clinical benefit; company is terminating development

6/18/14

VBL Therapeutics Ltd. (Tel Aviv, Israel)

VB-201

Lecinoxoid compound

Psoriasis and ulcerative colitis

Completed enrollment of two phase II studies

6/23/14

INFECTION

Agenus Inc. (Lexington, Mass.)

Herpv

A synthetic vaccine

Genital herpes simplex virus-2

Phase II data showed it produced an immune response to the HSV antigens in the majority of patients after a series of vaccinations and a booster dose at six months

6/27/14

Merlion Pharmaceuticals Pte. Ltd. (Singapore)

Finafloxacin

A next-generation fast-acting fluoroquinolone antibiotic

Complicated urinary tract infections

Completed the planned recruitment in its phase II trial

6/19/14

INFLAMMATORY

Amgen Inc. (Thousand Oaks, Calif.) and Astrazeneca plc (London)

Brodalumab

A drug that targets the interleukin-17 receptor

Psoriatic arthritis

Phase II data from the 168-patient study showed that brodalumab significantly improved signs and clinical symptoms associated with psoriatic arthritis, including tender and swollen joints, at 12 weeks, as measured by a 20% improvement in the American College of Rheumatology (ACR20) response criteria

6/13/14

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

Rejoin

Adipose-derived mesenchymal precursor cell-based therapy

Knee osteoarthritis

Completed patient enrollment of 53 patients for its phase IIb trial

6/27/14

Flexion Therapeutics Inc. (Burlington, Mass.)

FX006

A non-opioid, sustained-release intra-articular formulation of TCA

Osteoarthritis of the knee

Top-line results from a phase IIa synovial fluid pharmacokinetic study demonstrated that a single intra-articular injection can provide therapeutic concentrations of drug in joint fluid for at least 12 weeks

6/19/14

MISCELLANEOUS

Aerie Pharmaceuticals Inc. (Bedminster, N.J.)

Roclatan

Eye drop consisting of Rhopressa and Xalatan

Glaucoma

Hit the primary endpoint in a phase IIb trial by showing statistically significant superiority over each of its components

6/26/14

Chemocentryx Inc. (Mountain View, Calif.)

CCX168

An orally administered inhibitor

Renal disease

Phase II data show that patients receiving CCX168 showed improvements in the Birmingham Vasculitis Activity Score (BVAS), an overall disease activity index; BVAS response at 12 weeks was higher in patients on CCX168 than those patients receiving the standard of care

6/3/14

Geron Corp. (Menlo Park, Calif.)

Imetelstat

A lipid-conjugated oligonucleotide inhibitor of human telomerase

Myelofibrosis

FDA removed the partial clinical hold on the investigator-sponsored trial that was placed in March due to a safety signal of hepatotoxicity; it remains on full clinical hold in essential thrombocythemia or polycythemia vera and in multiple myeloma

6/13/14

La Jolla Pharmaceutical Co. (San Diego)

GCS-100

Galectin-3 inhibitor

Chronic kidney disease

Completed enrollment of GCS-100-CS-4003, its phase II extension study

6/6/14

Lee's Pharmaceuticals Ltd. (Hong Kong)

RGN-259

Thymosin beta 4-based, preservative-free eye drops

Moderate to severe dry eye syndrome

Gained CFDA approval to commence phase II trials

6/25/14

Nora Therapeutics Inc. (Palo Alto, Calif.)

NT100

A biologic agent similar to a naturally occurring protein in the female reproductive tract

Miscarriage

Started its phase II RESPONSE research study in pregnant women with a history of unexplained recurrent pregnancy loss

6/27/14

Ohr Pharmaceutical Inc. (New York)

Squalamine

Eye drops

Wet age-related macular degeneration

Interim results from its phase II trial showed mean change in visual acuity at the end of study visit for the interim analysis group was +10.4 letters with Squalamine eye drops plus Lucentis as needed vs. +6.3 letters in the placebo eye drops plus Lucentis as needed arm, a 65% additional relative benefit (p = 0.18); however, an interim analysis showed there were no significant differences in the frequency of the Lucentis as needed injections, which was the primary endpoint of the study

6/25/14

Opko Health Inc. (Miami)

Lagova

Long-acting human growth hormone

Pediatric growth hormone deficiency (GHD)

Interim six-month results from a phase II study showed that height velocity achieved with each of the three different doses administered in the study ranged from 12.25 cm to almost 15.5 cm, with every child enrolled in the study experiencing a significant improvement in height velocity

6/27/14

Protalix Biotherapeutics Inc. (Carmiel, Israel)

PRX-112

Oral glucocere-brosidase, a plant cell-expressed form of an active glycosylated glucocerebrosidase enzyme naturally encapsulated within carrot cells

Gaucher disease

The first patient was enrolled in the phase IIa trial

6/19/14

Quark Pharmaceuticals Inc. (Fremont, Calif.)

QPI-1007

Synthetic siRNA designed to inhibit expression of Caspase 2

Primary angle closure glaucoma

The first patient was dosed in a double-masked, randomized sham-controlled phase IIa study

6/26/14

Umecrine Mood AB (Stockholm)

UC1010

A GABA-A-modulating steroid antagonist

Premenstrual dysphoric disorder

Data from an exploratory phase II study showed the primary endpoint was not met

6/13/14

Versartis Inc. (Redwood City, Calif.)

VRS-317

Long-acting human growth hormone

Growth hormone deficiency (GHD)

Phase IIa data from its six-month trial found that repeat dosing with VRS-317, given at weekly, semi-monthly and monthly intervals over a six-month period, achieved annualized six-month height velocity (the study's primary endpoint) comparable to the annual height velocity for similar GHD children given a dose of daily growth hormone that is the highest approved dose on the label of Norditropin and Genotropin

6/24/14

Xenetic Biosciences Inc. (Lexington, Mass.)

Erepoxen

Intravenous dose

Anemia in chronic kidney disease

The Serum Institute of India dosed the first patient in the second cohort of a phase IIa trial

6/12/14

RESPIRATORY

Asmacure Ltd. (Quebec City)

ASM-024

Dry powder for inhalation formulation

Moderate asthma

Phase I and IIa showed that ASM-024 at therapeutic doses below 20 mg was generally safe and well tolerated and support further exploration of an effect of the ASM-024 DPI alone or as an adjunctive treatment with standard of care in patients with moderate and severe asthma

6/19/14

Bellerophon Therapeutics LLC (Hampton, N.J.)

Inopulse

Selective short-acting pulmonary vasodilator designed to deliver brief, controlled doses of inhaled nitric oxide

Pulmonary hypertension associated with chronic obstructive pulmonary disease

Completed enrollment of its 159-patient phase II trial

6/30/14

Curis Inc. (Lexington, Mass.) said Roche AG (Basel, Switzerland)

Erivedge

Vismodegib

Idiopathic pulmonary fibrosis

Filed an investigational NDA to initiate a phase II trial to test oral doses of the drug at 150 mg over 52 weeks

6/24/14

Novabiotics Ltd. (Aberdeen, UK)

Lynovex

Cysteamine; dual mucolytic antibacterial drug

Cystic fibrosis

Started an open-label, single-arm phase IIa trial

6/26/14

Revive Therapeutics Ltd. (Toronto)

REV-00

A repurposed version of antidepressant tianeptine

To prevent opioid-induced respiratory depression in patients with sleep apnea

Results from the second half of its phase IIa proof-of-concept study showed that the drug might treat and/or prevent opioid-induced respiratory depression in patients with sleep apnea in a postoperative setting, without affecting analgesia; the trial also showed that REV-001 was safe and well tolerated at the 50-mg dose

6/30/14

Vertex Pharmaceuticals Inc. (Boston)

Kalydeco

Ivacaftor

Cystic fibrosis

Results of the two-part proof-of-concept study of ivacaftor in 24 people showed a statistically significant improvement in mean absolute lung function (FEV1) after treatment vs. placebo; results met the primary endpoint as evaluated using a Bayesian Hierarchical Model (BHM) analysis, which was supported by analysis using a Mixed Model for Repeated Measures

6/5/14


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.