Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Adheron Therapeutics (San Francisco) |
SDP051 |
Lead Cad-11 inhibitor; humanized monoclonal antibody to Cadherin-11 |
Rheumatoid arthritis |
Phase I data showed it was safe and well tolerated, with no evidence of significant side effects |
1/9/14 |
Redhill Biopharma Ltd. (Tel-Aviv, Israel) |
RHB-104 |
An antibiotic |
Crohn's disease |
Phase I data showed it had higher bioavailability when given with food and there were no serious adverse events reported |
1/7/14 |
CANCER | |||||
Advaxis Inc. (Princeton, N.J.) |
ADXS-HPV |
Immunotherapy |
Human papillomavirus-associated head and neck cancer |
First patient was dosed in a phase I/II study |
1/22/14 |
Apceth GmbH (Munich, Germany) |
Mesenchymal stromal cells |
Genetically modified mesenchymal stromal cells harvested from a patient's own bone marrow |
Advanced adenocarcinoma of the gastrointestinal tract |
Phase I/II trial began to enroll patients |
1/24/14 |
Argen-X BV (Breda, the Netherlands) |
ARGX-110 |
Monoclonal antibody |
CD70-positive cancer |
Advanced it into the safety and efficacy expansion part of its phase Ib study |
1/14/14 |
Arno Therapeutics Inc. (Flemington, N.J.) |
Onapristone |
An oral, anti-progestin hormone blocker |
Progesterone receptor-positive tumors |
Enrolled the first patient in a phase I study |
1/22/14 |
Biolinerx Ltd. (Jerusalem) |
BL-8040 |
Selective antagonist of chemokine receptor CXCR4 |
Acute myeloid leukemia and other hematological disorders |
Filed the necessary regulatory submissions to commence a phase I trial |
1/14/14 |
Epizyme Inc. (Cambridge, Mass.) |
EPZ-5676 |
DOT1L histone methyltransferase inhibitor |
Acute leukemia |
Phase I data showed it hit a proof-of-concept milestone, achieving objective responses |
1/8/14 |
E-Therapeutics plc (Oxford, UK) |
ETS2101 |
Synthetic cannibinoid |
Brain cancer |
Phase I data showed that five dose-escalation steps have been completed without any drug-related serious adverse events; Patient recruitment into phase I trials of ETS2101 in brain cancer and in solid tumors was halted temporarily due to a drug supply issue |
1/8/14; 1/27/14 |
Immune Design Inc. (Seattle) |
ID-G100 |
Agent that includes Toll-like receptor-4 agonist glycopyranosyl lipid A and stems from the company's GLASS discovery program |
Merkel cell carcinoma |
The first patient was treated in a phase I trial |
1/28/14 |
Infinity Pharmaceuticals Inc. (Cambridge, Mass.) |
IPI-145 |
Oral inhibitor of phosphoinositide-3-kinade-delta and P13K-gamma |
Relapsed/refractory T-cell lymphoma |
Phase I data showed that IPI-145 was clinically active with an overall response rate of 38%, including one complete response and nine partial responses |
1/27/14 |
Karyopharm Therapeutics Inc. (Natick, Mass.) |
Selinexor |
An oral selective inhibitor of nuclear export, or SINE, compound |
Metastatic colorectal cancer |
Phase I data showed that one patient had a partial response by standard RECIST criteria and had remained on study for eight months; 11 patients had stable disease, 10 of those for eight weeks or longer, with four of those 10 patients (11 percent) demonstrating stable disease over 25 weeks |
1/23/14 |
Kolltan Pharmaceuticals Inc. (New Haven, Conn.) |
KTN3379 |
Monoclonal antibody designed to block the activity of ErbB3 |
Cancer |
Treated the first cancer patient as part of a phase I study evaluating its safety, preliminary evidence of antitumor activity and pharmacokinetics |
1/22/14 |
Merrimack Pharmaceuticals Inc. (Cambridge, Mass.) |
MM-398 |
A stable nanoliposomal irinotecan designed to exploit leaky tumor vasculature for enhanced drug delivery to tumors |
Pediatric solid tumors |
Enrolled the first patient in a phase I dose-escalating trial of MM-398 in conjunction with cyclophosphamide |
1/24/14 |
Nektar Therapeutics Inc. (San Francisco) |
NKTR-102 |
Etirinotecan pegol; a long-acting topoisomerase 1 inhibitor |
Advanced cancer |
Phase I data of NKTR-102 in combination with 5-fluorouracil showed the compound was safely combined with 5-FU/LV and showed signs of clinical benefit, including both objective responses and tumor marker reductions |
1/22/14 |
Oncomed Pharmaceuticals Inc. (Redwood City, Calif.) |
OMP-54F28 |
A fusion protein based on a truncated form of the Frizzled8 receptor |
Pancreatic cancer |
Started a multicenter phase Ib trial of OMP-54F28 (Fzd8-Fc) with nab-paclitaxel (Abraxane, Celgene Corp.) and gemcitabine |
1/14/14 |
Oncomed Pharmaceuticals Inc. (San Francisco) and Celgene Corp. (Summit, N.J.) |
Demcizumab |
Humanized monoclonal antibody that inhibits delta-like ligand 4 in the Notch signaling pathway |
First-line stage IV pancreatic cancer |
Phase Ib data of demcizumab combined with gemcitabine and Abraxane showed it was generally well tolerated, with fatigue, hypertension, nausea and vomiting the most common drug-related toxicities; three of six evaluable patients had partial responses and two had stable disease |
1/21/14 |
Oncomed Pharmaceuticals Inc. (San Francisco) and Glaxosmithkline plc (London) |
OMP-59R5 |
A fully human monoclonal antibody that targets the Notch2 and Notch3 receptors |
Advanced pancreatic cancer |
Phase Ib data showed it was well tolerated when used with standard of care, with no dose-limiting toxicities and typical adverse events; six of 13 patients treated with OMP-59R5, gemcitabine and Abraxane achieved stable disease |
1/21/14 |
Rexahn Pharmaceuticals Inc. (Rockville, Md.) |
RX-3117 |
A next-generation cancer cell-specific drug |
Solid tumors |
Started a phase Ib trial to study the safety and efficacy of RX-3117 in cancer patients |
1/9/14 |
Senesco Technologies Inc. (Bridgewater, N.J.) |
SNS01-T |
A gene regulatory approach to cancer therapy that is designed to selectively trigger apoptosis in B-cell cancers |
Multiple myeloma and non-Hodgkin's B-cell lymphoma |
Dosed SNS01-T in the first patient in the fourth cohort of its phase Ib/IIa study |
1/10/14 |
Starpharma Holdings Ltd. (Melbourne, Australia) |
DEP-Docetaxel |
Dendrimer-enhanced docetaxel chemotherapeutic product |
Solid tumors |
Received the approvals to start a phase I trial with 30 patients |
1/24/14 |
Vaxil Biotherapeutics Ltd. (Ness Ziona, Israel) |
Immucin |
Therapeutic vaccine |
Metastatic breast cancer |
Recruited the first patient for a study, VAXIL-010, that will include up to 20 patients with metastatic breast cancer displaying cancer marker MUC1, who will receive 12 injections of Immucin in combination with hormone therapy |
1/29/14 |
CARDIOVASCULAR | |||||
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
ALN-AT3 |
A subcutaneously administered RNAi therapeutic targeting antithrombin |
Hemophilia and rare bleeding disorders |
Started a phase I trial |
1/23/14 |
Catalyst Pharmaceutical Partners Inc. (Coral Gables, Fla.) and Biomarin Pharmaceutical Inc. (Novato, Calif.) |
Firdapse |
Amifampridine phosphate |
Lambert-Eaton myasthenic syndrome |
The first formal human cardiac safety study met its pre-specified endpoint, demonstrating no effect on heart rate or cardiac depolarization when administered at and above therapeutic levels |
1/9/14 |
Neuralstem Inc. (Rockville, Md.) |
NSI-566 |
Spinal cord-derived neural stem cells |
Motor deficits from ischemic stroke |
The first patient was treated in the phase I/II stem cell trial at Bayi Brain Hospital in Beijing, China; it is the first trial in which Neuralstem’s spinal cord-derived stem cells are being transplanted directly into a patient’s brain for placement near the stroke lesion |
1/14/14 |
Resverlogix Corp. (Calgary, Alberta |
RVX-208 |
A BET inhibitor |
Atherosclerosis |
Major adverse cardiac event (MACE) data from two trials were combined, demonstrating that treatment with RVX-208 led to a significant reduction in MACE; RVX-208-treated-patients had less cumulative events of 6.74% vs. 15.09% (p = 0.02) in the placebo-treated group |
1/16/14 |
CENTRAL NERVOUS SYSTEM | |||||
Collegium Pharmaceuticals Inc. (Canton, Mass.) |
Oxycodone Deterx |
Extended-release, abuse deterrent oxycodone microsphere-in-a-capsule product |
Pain |
Top-line data from a human abuse potential trial showed a significantly lower peak “drug liking” when compared with intranasal crushed immediate-release oxycodone (p < 0.0001) |
1/16/14 |
Cortice Biosciences Inc. (New York) |
TPI 287 |
An abeotaxane; a taxoid that binds to and stabilizes microtubules |
Mild to moderate Alzheimer's disease |
Enrollment began in a phase I trial |
1/22/14 |
Cynapsus Therapeutics Inc. (Toronto) |
APL-130277 |
Sublingual thin film strip formulation of apomorphine |
Parkinson's disease |
Reported positive top-line data from a healthy volunteer pilot crossover trial comparing it to a commercially available injectable formulation of apomorphine |
1/14/14 |
Cynapsus Therapeutics Inc. (Toronto) |
APL-130277 |
A sublingual thin film strip formulation of apomorphine |
Parkinson's disease |
Top-line data from a pilot study comparing APL-130277 to a commercially available injectable formulation of apomorphine indicated that APL-130277 may have advantages over an injectable product by reducing the frequency and intensity of side effects including nausea and vomiting vs. those commonly reported for a subcutaneous injectable formulation |
1/15/14 |
Oxford Biomedica plc (Oxford, UK) |
Prosavin |
A lentiviral, vector-based therapy |
Advanced Parkinson's disease |
Phase I/II data showed a favorable safety profile and a statistically significant improvement in motor function relative to baseline at six and 12 months post-treatment |
1/13/14 |
Stemcells Inc. (Newark, Calif.) |
HuCNS-SC |
Human neural stem cells |
Chronic spinal cord injury |
A team at the University of Calgary successfully transplanted its first subject in the phase I/II trial |
1/13/14 |
INFECTION | |||||
Enanta Pharmaceuticals Inc. (Watertown, Mass.) |
EDP-788 |
Bicyclolide that targets the bacterial ribosome |
Methicillin-resistant Staphylococcus aureus infections |
Started a phase I study |
1/14/14 |
Geovax Labs Inc. (Atlanta) |
GOVX-B11 |
Vaccine |
HIV |
Preliminary top-line results from a phase I trial investigating the ability of the vaccine to control HIV infections in patients who initiated antiretroviral drug treatment within 18 months of seroconversion showed enhanced CD8-positive T cells were elicited in eight of nine patients and enhanced CD4-positive T cell and antibody responses in four of nine participants, and there was a trend toward enhanced viral control in individuals with the best CD4-positive and CD8-positive T-cell responses |
1/14/14 |
Medivector Inc. (Boston) |
Favipiravir |
Inhibits the enzyme viral RNA polymerase that functions to make copies of the viral RNA segments and to synthesize mRNAs that produce the proteins that will form new viruses |
Uncomplicated influenza |
Enrolled the first patient in the U.S. in one of two favipiravir phase III studies; the trial is expected to enroll more than 1,000 patients in participating clinics and practices in North, Central and South America |
1/21/14 |
Vaxart Inc. (San Francisco) |
Vaccine |
Oral H1N1 seasonal influenza vaccine candidate |
Influenza |
Vaxart reported positive safety and immunogenicity data from a phase Ib dose-ranging study, inducing antihemagglutinin antibody responses in 88% of subjects |
1/10/14 |
MISCELLANEOUS | |||||
Aerie Pharmaceuticals Inc. (Bedminster, N.J.) |
AR-13324 |
Eye drops |
Glaucoma |
Top-line phase I data demonstrated very low systemic exposure to the drug, with blood levels at or below the limit of detection of 0.1 ng/mL at all time points; after eight days a once-daily administration reduced the average diurnal IOP to approximately 11 mmHg, representing a decrease of approximately 5 mmHg, or more than 30% |
1/10/14 |
Galectin Therapeutics Inc. (Norcross, Ga.) |
GR-MD-02 |
Targets galectin-3 |
Fatty liver disease (NASH) with advanced fibrosis |
Patient enrollment was completed in the first cohort of a phase I trial |
1/14/14 |
Oxthera AB (Stockholm) |
Oxabact |
An oral product composed of highly concentrated freeze-dried live bacteria, administered in capsules |
Primary hyperoxaluria |
Initiated a clinical study to be conducted at seven sites in three countries and will include a total of 24 patients |
1/9/14 |
Pluristem Therapeutics Inc. (Haifa, Israel) |
PLX-PAD |
Placental eXpanded cells |
Muscle injury |
Top-line results from its phase I/II trial showed the cells were safe and that statistical significance was reached (p = 0.0067) for the primary endpoint, defined as the change in maximal voluntary isometric contraction force of the gluteal muscle at six months after total hip replacement |
1/22/14 |
PTC Therapeutics Inc. (South Plainfield, N.J.) |
Unnamed compound |
Oral compound |
Spinal muscular atrophy |
Entered the first stage of clinical development |
1/23/14 |
Therapeuticsmd Inc. (Boca Raton, Fla.) |
TX 12-004-HR |
A rapidly acting vaginal preparation capsule |
Vulvar vaginal atrophy in postmenopausal women |
Final pharmacokinetic results from two studies showed substantially lower systemic estradiol exposure of TX 12-004-HR compared to Vagifem |
1/29/14 |
RESPIRATORY | |||||
Faron Pharmaceuticals Ltd. (Turku, Finland) |
FPCLI001 |
Treatment for respiratory distress syndrome |
Respiratory distress syndrome |
Data showed it reduced the odds of all-cause mortality by more than 80% |
1/16/14 |
Notes BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment. 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. |