Company |
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. |