Company |
Product |
Description |
Indication |
Status |
Date |
CANCER | |||||
Agenus Inc. (Lexington, Mass.) and Glaxosmithkline plc (London) |
MAGE-A3 |
Cancer immunotherapeutic formulated with QS-21 Stimulon adjuvant |
Melanoma |
Phase III trial missed its primary endpoint, failing to significantly extend disease-free survival in patients compared to placebo |
9/6/13 |
Ariad Pharmaceuticals Inc. (Cambridge, Mass.) |
Iclusig |
Ponatinib |
Chronic myeloid leukemia |
Reached enrollment of 50% in a Phase III trial |
9/5/13 |
Arqule Inc. (Woburn, Mass.) and Daiichi Sankyo Co. Ltd. (Tokyo) |
Tivotanib |
MET inhibitor |
Hepatocellular cancer |
Will implement a lower dose in the Phase III trial following an observation of a higher incidence of neutropenia |
9/4/13 |
Celgene International Sarl (subsidiary of Celgene Corp.; Summit, N.J.) |
Pomalyst/Imnovid |
Pomalidomide |
Multiple myeloma |
Phase III data of Pomalyst/Imnovid plus low-dose dexamethasone showed it met its primary endpoint demonstrating improvement in progression-free survival compared with high-dose dexamethasone |
9/6/13 |
Celsion Corp. (Ames, Iowa) |
Thermodox |
Encapsulated form of doxorubicin |
Hepatocellular carcinoma |
Combined results showed a 61% increase in overall survival, but in a patient subgroup that received Thermodox plus radiofrequency ablation longer than 45 minutes and less than 90 minutes, overall survival jumped by 71% compared to a control arm |
9/17/13 |
Erytech Pharma SA (Lyon, France) |
Graspa |
Red blood cell-encapsulated L-asparaginase |
Acute lymphoblastic leukemia |
Completed enrollment in its pivotal Phase III trial |
9/11/13 |
Exelixis Inc. (South San Francisco) |
Cabozantinib |
A multikinase inhibitor |
Advanced hepatocellular carcinoma |
Started a Phase III trial; the primary endpoint is overall survival |
9/11/13 |
Exelixis Inc. (South San Francisco) |
Cabozantinib |
A multikinase inhibitor |
Metastatic castration-resistant prostate cancer |
Reached its enrollment target of 960 patients in a Phase III pivotal trial |
9/27/13 |
Newlink Genetics Corp. (Ames, Iowa) |
Algenpan-tucel-L |
A Hyperacute immunotherapy candidate |
Pancreatic cancer |
Met its accrual goal of 722 subjects in the Phase III trial |
9/18/13 |
Northwest Biotherapeutics Inc. (Bethesda, Md.) |
DCVax-L |
A dendritic cell-based vaccine |
Glioblastoma multiforme |
German regulators authorized the company’s Phase III trial |
9/18/13 |
Novarx Corp. (San Diego) |
Lucanix |
Belagenpuma-tucel-L |
Non-small-cell lung cancer |
Phase III data showed the primary endpoint of improving overall survival in 532 patients was not met |
9/26/13 |
Oncothyreon Inc. (Seattle) and Merck KGaA (Darmstadt, Germany) |
Tecemotide |
Formerly Stimuvax |
Non-small-cell lung cancer |
Will start a new Phase III trial, despite last year’s miss in which a subgroup showed a boost of just over 10 months in overall survival |
9/26/13 |
Pharmamar SA (Madrid, Spain) |
Yondelis |
Trabectidin |
Epithelial malignant pleural mesothelioma |
Phase II trial began |
9/26/13 |
Regado Biosciences Inc. (Basking Ridge, N.J.) |
REG1 |
Antithrombotic |
Patients undergoing percutaneous coronary intervention |
Enrolled the first patient in a Phase III trial |
9/18/13 |
Verastem Inc. (Cambridge, Mass.) |
VS-6063 |
Defactinib; cancer stem cell inhibitor |
Malignant pleural mesothelioma |
Started a Phase III trial |
9/11/13 |
Xbiotech Inc. (Austin, Texas) |
Xilonix |
Blocks inflammation involved in tumor growth and metastasis |
Colorectal cancer |
Plans to conduct a pivotal Phase III trial |
9/19/13 |
CARDIOVASCULAR | |||||
Relypsa Inc. (Redwood City, Calif.) |
Patiromer |
High-capacity non-absorbed oral potassium binder |
Hyperkalemia |
Phase III data showed statistically significant reduction of 1.01 mEq/L, and 76% of subjects had a serum potassium in the target range that was statistically significant |
9/11/13 |
CENTRAL NERVOUS SYSTEM | |||||
Iroko Pharmaceuticals LLC (Philadelphia) |
Diclofenac |
Nonsteroidal, anti-inflammatory drug, submicron diclofenac |
Acute pain following bunionectomy surgery |
Reduced the use of opioid rescue medication in a Phase III trial |
9/6/13 |
Kempharm Inc. (North Liberty, Iowa) |
KP201 |
Benzhydrocodone hydrochloride and acetaminophen |
Pain |
Pivotal data confirmed it was metabolized completely and no systemic prodrug exposure was observed; it was safe and well tolerated |
9/26/13 |
Pacira Pharmaceuticals Inc. (Parsippany, N.J.) |
Exparel |
Bupivacaine liposome injectable suspension |
Postsurgical pain management |
A study in 200 patients showed better pain control, improved knee flexion and a shorter hospital stay than patients who received a femoral nerve block with local anesthetic |
9/23/13 |
Relmada Therapeutics Inc. (New York) |
Levocap ER |
A once-a-day extended-release dosage form of opioid levorphanol in a tamper-resistant delivery system |
Pain |
Phase III study showed good bioavailability for the ER formulations; there were no serious adverse events |
9/11/13 |
DIABETES | |||||
Furiex Pharmaceuticals Inc. (Morrisville, N.C.) and Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) |
Alogliptin |
Dipeptidyl peptidase-4 inhibitor |
Type II diabetes |
Top-line data showed it met the primary endpoint of noninferiority compared to placebo in addition to standard of care with no increase in cardiovascular risk |
9/4/13 |
GASTROINTESTINAL | |||||
Sucampo AG (subsidiary of Sucampo Pharmaceuticals Inc.; Bethesda, Md.) |
Amitiza |
Lubiprostone |
Chronic idiopathic constipation |
Data demonstrated efficacy and tolerability, showing it improved constipation and related symptoms, compared to other therapies |
9/16/13 |
INFECTION | |||||
Achaogen Inc. (South San Francisco) |
Plazomicin |
Aminoglycoside antibiotic |
Multidrug-resistant gram-negative bacterial infections |
Received an SPA from the FDA for a Phase III trial |
9/24/13 |
Actelion Ltd. (Allschwil, Switzerland) |
Cadazolid |
250 mg orally twice daily for 10 days |
Clostridium difficile-associated diarrhea |
Will start a Phase III trial |
9/11/13 |
Astellas Pharma Inc. (Tokyo) |
Mycamine |
Micafungin |
Fungal infections in liver transplant |
Was found to be non-inferior to standard of care |
9/10/13 |
Chimerix Inc. (Durham, N.C.) |
CMX001 |
Oral nucleotide analogue lipid-conjugate brincidofovir |
Cytomegalo-virus |
Started dosing in its Phase III trial |
9/10/13 |
Paxvax Inc. (Menlo Park, Calif.) |
PXVX0200 |
Vaccine |
Cholera |
Began a Phase III trial |
9/9/13 |
Tetraphase Pharmaceuticals Inc. (Watertown, Mass.) |
Eravacycline |
Broad-spectrum oral and intravenous antibiotic |
Complicated intra-abdominal infections |
Dosed the first patient in its Phase III study |
9/4/13 |
INFLAMMATORY | |||||
Nutrition Science Partners Ltd. (joint venture between Hutchison China Meditech Ltd. and Nestle Health Science SA; Lutry, Switzerland) |
HMPL-004 |
Botanical drug |
Inflammatory bowel disease |
Launched a Phase III trial |
9/12/13 |
MISCELLANEOUS | |||||
Acacia Pharma Ltd. (Cambridge, UK) |
APD421 |
Comprises an intravenous formulation of marketed dopamine D2/D3 antagonist amisulpride |
To prevent postoperative nausea and vomiting |
Started two pivotal Phase III studies |
9/18/13 |
Astellas Pharma Inc. (Tokyo) |
Advagraf |
Prolonged-release capsules; tacrolimus therapy |
Liver transplant |
Data showed significantly better renal function and a lower incidence of acute organ rejection when compared with standard dose Advagraf therapy |
9/10/13 |
Auris Medical AG (Basel, Switzerland) |
AM-101 |
An N-methyl-d-aspartate (NMDA) receptor antagonist |
Tinnitus |
Started a Phase III trial; Phase II data showed the difference between active and placebo groups was significant on subjective tinnitus loudness, annoyance, sleep difficulties and impact and severity of the condition |
9/10/13 |
BHR Pharma LLC (Herndon, Va.) |
BHR-100 |
Intravenous progesterone formulation |
Severe traumatic brain injury |
Randomized the 1,180th and final patient in its Phase III trial |
9/4/13 |
Cempra Inc. (Chapel Hill, N.C.) |
Solithromycin |
A first-in-class fluoroketolide antibiotic candidate |
Mild to severe chronic liver disease |
Demonstrated safety and tolerability in patients; it is in Phase III |
9/6/13 |
Hyperion Therapeutics Inc. (South San Francisco) |
Ravicti |
Glycerol phenylbutyrate oral liquid |
Urea cycle disorders |
Pooled data showed mean monthly ammonia levels ranging from 17.2 umol/L and 24.8 umol/L during 12 months of treatment, remaining below the upper limit of normal |
9/4/13 |
Ophthotech Corp. (New York) |
Fovista |
An anti-platelet-derived growth factor |
Newly diagnosed neovascular age-related macular degeneration |
Initiated enrollment in a pivotal Phase III trial |
9/3/13 |
Prosensa Holding NV (Leiden, the Netherlands) |
Drisapersen |
Exon-skipping antisense drug |
Duchenne muscular dystrophy |
Phase III data showed it failed to meet the primary endpoint |
9/23/13 |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Androxal |
Enclomiphene |
Secondary hypogonadism |
Pivotal and safety data showed it met the efficacy endpoint and that both co-primary endpoints were met with respect to T normalization and sperm function |
9/18/13 |
Rockwell Medical Inc. (Wixom, Mich.) |
SFP |
Iron-delivery drug |
Iron deficiency in chronic kidney disease |
Top-line Phase III data showed it met the primary endpoint, demonstrating a statistically significant mean change in hemoglobin from baseline to end of treatment; it also met key secondary endpoints |
9/5/13 |
Therapeuticsmd Inc. (Boca Raton, Fla.) |
TX 12-001-HR |
Bioidentical combination 17ss-estradiol and progesterone drug candidate |
Menopause |
Dosed the first patient in the Replenish Phase III trial |
9/6/13 |
Veloxis Pharmaceuticals A/S (Horsholm, Denmark) |
Envarsus |
Once-daily LCP-Tacro |
Kidney transplant |
Phase III data demonstrated comparable efficacy and safety compared to twice-daily Prograf |
9/5/13 |
Ventrus Biosciences Inc. (New York) |
VEN 307 |
Diltiazem hydrochloride 2% cream |
Anal fissures |
VEN 307 was safe and well tolerated, and irritation and sensitization was similar to that seen with both placebo and saline, but significantly better than that seen with sodium lauryl sulfate |
9/12/13 |
|
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. SPA = Special Protocol Assessment. | |||||