Company (Location) | Product | Description | Indication | Status (Date)# |
AUTOIMMUNE | ||||
Pfizer Inc. (New York) |
Tofacitinib |
Oral JAK inhibitor |
Rheumatoid arthritis |
Phase III data showed it met all primary efficacy endpoints (9/9) |
CANCER | ||||
Access Pharmaceuticals Inc. (Dallas) |
MuGard |
Mucoadhesive wound liquid |
Oral mucositis |
It was effective in preventing, delaying and managing radiotherapy- and chemotherapy-induced oral mucositis, and reduced the need for opiate pain medicine and tube feeding (9/29) |
Boehringer Ingelheim Pharmaceuticals Inc. (Ridgefield, Conn.) |
Afatinib |
An EGFR/HER2 inhibitor |
HER2-positive breast cancer |
Started two global Phase III trials (9/7) |
Exelixis Inc. (South San Francisco) |
Cabozantinib |
A dual inhibitor of MET and VEGFR |
Medullary thyroid cancer |
The pre-specified number of progression-free survival events required for unblinding data in its pivotal Phase III trial has been reached (9/8) |
Galena Biopharma Inc. (Portland, Ore.) |
NeuVax |
An E75 peptide derived from HER2 combined with the immune adjuvant granulocyte macrophage colony-stimulating factor |
Breast cancer |
Contracted with Aptiv Solutions to manage its Phase III trial to be conducted under an SPA (9/30) |
OncoGenex Inc. (Bothell, Wash.) |
Custirsen |
Clusterin inhibitor |
Cancer pain |
Amended the SPA to expand the inclusion criteria for the 300-patient Phase II trial for more than 12 weeks in men with metastatic castrate-resistant prostate cancer (9/20) |
RXi Pharmaceuticals Corp. (Worcester, Mass.) |
NeuVax |
Cancer vaccine |
HER2 positive breast cancer |
The FDA lifted the partial clinical hold and the company is on schedule to begin Phase III in early 2012 (9/13) |
Topotarget A/S (Copenhagen, Denmark) |
Belinostat |
An HDAC inhibitor |
Peripheral T-cell lymphoma |
Completed enrollment for the pivotal trial of belinostat for monotherapy treatment (9/27) |
CARDIOVASCULAR | ||||
Karo Bio AB (Huddinge, Sweden) |
Eprotirome |
Liver-selective thyroid hormone receptor agonist |
Heterozygous familial hyper-cholesterolemia |
Received approval to start a Phase III study (9/20) |
CENTRAL NERVOUS SYSTEM | ||||
AB Science SA (Paris) |
Masitinib |
Protein kinase inhibitor |
Primary progressive multiple sclerosis or relapse-free secondary multiple sclerosis |
Recruited the first patient in a Phase III study (9/7) |
BioDelivery Sciences International Inc. (Raleigh, N.C.) |
Buprenorphine |
Opioid analgesic delivered with BEMA |
Chronic pain |
Phase III data in a mixed opioid-naive and opioid experienced population failed to reach its primary endpoint of overall difference in pain intensity compared to placebo (9/30) |
Eisai Europe Ltd. (Hatfield, UK; a division of Eisai Co. Ltd.; Tokyo) |
Zonegran |
Zonisamide |
Partial-onset seizures in epilepsy |
Phase III data showed it was more effective than placebo and was well tolerated (9/1) |
Durect Corp. (Cupertino, Calif.) |
Posidur |
Uses Durect's technology to deliver bupivacaine |
Postoperative pain relief |
Dosed the last patient in a Phase III trial (9/7) |
Trophos SA (Marseille, France) |
Olesoxime |
Dosed with 330-mg oral capsules once a day |
Amyotrophic lateral sclerosis |
The last of 500 patients has now completed the pivotal Phase III efficacy study of olesoxime (9/23) |
DIABETES | ||||
Amylin Pharmaceuticals Inc. (San Diego), Eli Lilly and Co. (Indianapolis) and Alkermes Inc. (Waltham, Mass.) |
Bydureon |
Exenatide extended-release for injectable suspension |
Diabetes |
New analyses demonstrated patients treated with Bydureon saw improvements in select cardiovascular risk factors compared to patients who received commonsly prescribed diabetes treatments (9/13) |
Generex Biotechnology Corp. (Worcester, Mass.) |
Generex Oral-lyn |
A Buccal spray insulin |
Diabetes |
A trial in subjects with impaired glucose tolerance showed a significant reduction of HbA1c compared to the control group (9/19) |
Sanofi SA (Paris) |
Lyxumia |
Lixisenatide; a once-daily GLP-1 receptor agonist |
Type II diabetes |
Achieved its primary efficacy endpoint of significant HbA1c reduction vs. placebo in patients uncontrolled on metformin in the company's GetGoal-F1 trial (9/13) |
Vivus Inc. (Mountain View, Calif.) |
Avanafil |
A phosphodiesterase-5 inhibitor |
Erectile dysfunction (ED) in diabetics |
Phase III data showed male diabetics receiving the drug had a significant improvement in ED (9/14) |
Vivus Inc. (Mountain View, Calif.) |
Qnexa |
Phentermine/topiramate |
Diabetes |
Patients treated for more than two years with Qnexa had reductions in progression to Type II diabetes and improvements in glycemic status, and had a greater percent weight loss with Qnexa compared to placebo in the diabetic and metabolic syndrome subgroups (9/19) |
INFECTION | ||||
Cubist Pharmaceuticals Inc. (Lexington, Mass.) |
CB-183,315 |
Oral novel lipopeptide |
Clostridium difficile-associated diarrhea |
Company plans to move into pivotal testing in the first half of 2012 (9/30) |
Durata Therapeutics Inc. (Morristown, N.J.) |
Dalbavancin |
A long-acting intravenous lipoglycopeptide |
Acute bacterial skin and skin structure infections |
Started enrolling patients in a second pivotal Phase III study (9/30) |
Gilead Sciences Inc. (Foster City, Calif.) |
HIV Quad regimen |
Elvitegravir, cobicistat, emtricitabine and tenofovir disoproxil fumarate |
HIV |
The second pivotal Phase III trial met the expectations by demonstrating noninferiority, though not superiority, at week 48 compared to ritonavir-boosted atazanavir plus Truvada (9/21) |
Medivir AB (Huddinge, Sweden) and Tibotec Pharmaceuticals (Cork, Ireland) |
TMC435 |
Protease inhibitor |
Hepatitis C virus |
Enrollment is completed in three Phase III studies (9/1) |
Trius Therapeutics Inc. (San Diego) |
Tedizolid phosphate |
TR-701; a second generation oxazolidinone |
Acute bacterial skin infection |
Completed enrollment in the first of two Phase III trials (9/19) |
Vertex Pharmaceuticals Inc. (Cambridge, Mass.) |
Incivek |
Telaprevir |
Hepatitis C virus |
Phase III data showed that 65% of patients responded to Incivek combination treatment (9/16) |
MISCELLANEOUS | ||||
Ampio Pharmaceuticals Inc. (Greenwood Village, Colo.) |
Zertane |
Oral drug |
Premature ejaculation |
Phase III data showed there were no treatment-related serious adverse events and few adverse events during the 12-week extesion study, and there were no reports of dependency or tolerability issues (9/27) |
BHR Pharma LLC (Herndon, Va.) |
BHR-100 |
Intravenous progesterone |
Traumatic brain injury |
Enrolled 200 patients in the Phase III trial (9/8) |
Biopartners GmbH (Baar, Switzerland) and LG Life Sciences Ltd. (Seoul, South Korea) |
LB03002 |
Injectable therapy |
Growth hormone deficiency |
Phase III data showed it maintained expected growth rates for patients over 24 months, increasing IGF-1 levels toward normal (9/28) |
Keryx Biopharmaceuticals Inc. (New York) |
Zerenex |
Ferric iron-based phosphate binder |
Hyperphos-phatemia |
Completed enrollment in the long-term study component of its Phase III registration program (9/20) |
Protalix BioTherapeutics Inc. (Carmiel, Israel) |
Taliglucerase alfa |
A plant-cell expressed form of glucocerebrosidase |
Gaucher disease |
Phase III data showed at doses of 60 U/kg or 30 U/kg it reduced mean spleen volume by 38% and 26.9%, respectively (9/9) |
Santarus Inc. (San Diego) |
Budesonide MMX |
A locally acting corticosteroid developed in a tablet formulation using Cosmo's MMX multi-matrix system technology |
Ulcerative colitis |
Analysis of top-line safety data from a 12-month extended-use study in patients treated daily with either budesonide MMX 6 mg or placebo showed a similar frequency of treatment-related adverse events for both groups and no clinically meaningful differences in the numbers of patients with abnormal bone mineral density scans (9/21) |
SARcode Bioscience Inc. (Brisbane, Calif.) |
SAR 1118 |
A first-in-class molecule that inhibits T-cell inflammation |
Dry eye |
Enrolled the first patient in its pivotal Phase III trial (9/15) |
Soligenix Inc. (Princeton, N.J.) |
orBec |
Oral beclomethasone dipropionate |
Acute gastrointestinal graft-vs.-host disease |
Halted its confirmatory Phase III trial after an independent data safety monitoring board concluded the study was "highly unlikely" to meet its predetermined efficacy endpoint; no safety concerns were raised (9/16) |
Tarsa Therapeutics Inc. (Philadelphia) |
Ostora |
An oral recombinant salmon calcitonin tablet |
Postmenopausal osteoporosis |
Phase III data showed that the drug achieved all of its efficacy endpoints and that the safety profile did not differ substantially from nasal calcitonin or placebo (9/20) |
Trophos SA (Marseille, France) |
Olesoxime |
Promotes survival of neurons and other cells under stress conditions through interactions with the mitochondrial permeability transition pore |
Spinal muscular atrophy |
Completed enrollment of more than 160 patients in its pivotal trial (9/9) |
Veloxis Pharmaceuticals A/S (Horsholm, Denmark) |
LCP-Tacro |
Once-daily treatment |
Kidney transplant |
Phase III data showed it is noninferior in efficacy compared to twice-daily standard tacrolimus (9/7) |
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. |