Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Arena Pharmaceuticals Inc. (San Diego) |
APD334 |
Oral candidate designed to target sphingosine 1-phosphate subtype receptor |
Autoimmune diseases |
Started dosing in a phase Ib trial |
6/4/14 |
Protalex Inc. (Florham Park, N.J.) |
PRTX-100 |
Incorporates a highly purified form of Staphyloccal protein A |
Active rheumatoid arthritis |
Phase Ib data of PRTX-100 in combination with methotrexate or leflunomide indicated that the drug was generally safe and well tolerated; data from the unblinded analysis of 41 patients also revealed positive effects of treatment on certain measures of disease activity, though those effects did not reach statistical significance |
6/4/14 |
CANCER | |||||
Advanced Cancer Therapeutics Inc. (Louisville, Ky.) |
PFK-158 |
Small-molecule candidate that inactivates a cancer metabolism target by inhibiting 6-phosphofructo-2-kinase/fructose-2, 6-biphosphatase 3 |
Solid tumors |
Started phase I testing |
6/5/14 |
Bio-Path Holdings Inc. (Houston) |
BP-100-1.01 |
Liposomal Grb-2 |
Blood cancers |
Company is resuming its phase I trial now that sufficient drug quantities are in place to complete cohort six of the trial |
6/20/14 |
Curis Inc. (Lexington, Mass.) |
CUDC-427 |
An oral small molecule |
Advanced and/or refractory solid tumors or lymphomas |
Re-initiated dosing in the phase I single-agent trial |
6/6/14 |
Deciphera Pharmaceuticals LLC (Cambridge, Mass.) |
DCC-2701 |
MET/TIE2/VEGFR2/TRK inhibitor altiratinib |
Solid tumors |
Started a phase I trial |
6/2/14 |
Genspera Inc. (San Antonio) |
G-202 |
Activated by the prostate-specific membrane antigen enzyme |
Hepatocellular carcinoma |
Interim results from a phase Ib and ongoing phase II study in patients who had previously progressed on or who were intolerant of Nexavar showed that 80% had stable disease at two months and 50% exhibited stable disease at four months |
6/26/14 |
Glycomimetics Inc. (Gaithersburg, Md.) |
GMI-1271 |
An E-selectin antagonist |
Acute myeloid leukemia |
The first healthy volunteer was dosed in its phase I study designed to test the safety, tolerability and pharmacokinetics of GMI-1271 |
6/20/14 |
Heat Biologics Inc. (Durham, N.C.) |
HS-410 |
Vesigenpuma-tucel-L |
Bladder cancer |
Dose escalation committee has approved the expansion of the first cohort to full enrollment in its ongoing phase I/II study |
6/2/14 |
Immune Design Corp. (Seattle) |
LV305 |
An immune-oncology agent from the firm's Dcvex lentiviral vector platform |
Locally advanced, relapsed or metastatic breast cancer, melanoma, non-small-cell lung cancer, ovarian cancer or sarcoma |
Treated the first patient in a phase I trial that will enroll up to 36 patients |
6/6/14 |
Inovio Pharmaceuticals Inc. (Blue Bell, Pa.) |
INO-3112 |
Immunotherapy; a combination of Inovio's lead immunotherapy, VGX-3100, and its immune activator expressing interleukin-12 |
Human papillomavirus-associated head and neck cancer |
Started a phase I/IIa trial |
6/11/14 |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) and Astrazeneca plc (London) |
ISIS-ARRx |
An antisense drug that inhibits the production of the androgen receptor |
Various cancers |
Initiated a phase I study of ISIS-ARRx (AZD5312); the open-label, dose-escalation study will test the drug in patients with advanced solid tumors, including metastatic castrate-resistant prostate, breast, bladder and ovarian cancers, where the AR pathway is potentially a contributing factor |
6/11/14 |
Kadmon Corp. LLC (New York) |
KD019 |
Orally bioavailable small-molecule kinase inhibitor of Src, HER2, EGFR and VEGFR2/KDR |
HER2-positive breast cancer metastasized to the brain |
Started a phase Ib/IIa study |
6/5/14 |
Macrogenics Inc. (Rockville, Md.) |
MGD006 |
A humanized Dual-Affinity Re-Targeting bispecific antibody-based molecule |
Relapsed or refractory acute myeloid leukemia |
The first patient was dosed in a phase I study |
6/20/14 |
Mirna Therapeutics Inc. (Austin, Texas) |
MRX34 |
A microRNA mimic |
Cancer |
Enrolled the first patient in the hematological malignancy cohort of its ongoing phase I trial |
6/11/14 |
Northwest Biotherapeutics Inc. (Bethesda, Md.) |
Dcvax-Direct |
Personalized immune therapy |
Inoperable solid tumors |
In an ongoing phase I/II trial, all nine patients who have received four of the six planned injections are showing tumor cell death, tumor shrinkage, substantial immune cell accumulation in their tumors and/or stabilization of their advanced cancers |
6/12/14 |
Oncomed Pharmaceuticals Inc. (Redwood City, Calif.) |
Vantictumab and Fzd8-Fc |
Monoclonal antibodies targeting the Wnt pathway |
Cancer |
Company halted six phase Ib trials testing two Bayer AG-partnered drugs due to "on-target mild-to-moderate" bone-related adverse events |
6/16/14 |
CARDIOVASCULAR | |||||
Bluebird Bio Inc. (Cambridge, Mass.) |
Lentiglobin BB305 |
Gene therapy |
Beta-thalassemia major |
Phase I/II data from a study in Paris showed that at 4.5 months following autologous transplant, one patient had total hemoglobin of 10.1 g/dL, of which 6.6 g/dL was therapeutic betaAT87Q-globin 9, and at two months the second post-transplant patient showed total hemoglobin of 11.6 g/dL, of which 4.2 g/dL was betaAT87Q-globin; Lentiglobin was well tolerated |
6/17/14 |
Capstone Therapeutics Inc. (Tempe, Ariz.) |
AEM-28 |
Apo E mimetic peptide |
Refractory hypercholes-terolemia |
Started dosing for the phase Ib/IIa trial in refractory hypercholesterolemic subjects who are already on optimal cholesterol lowering therapy but are unable to reach target cholesterol levels |
6/12/14 |
Edge Therapeutics Inc. (Berkeley Heights, N.J.) |
EG-1962 |
Polymeric microparticle form of the calcium channel blocker nimodipine |
Aneurysmal subarachnoid hemorrhage |
Is advancing into the second cohort of a phase I/II trial after a data safety monitoring committee recommended increasing the dose to 200 mg, since no unexpected drug-related serious adverse events showed up with the 100-mg dose in the first cohort |
6/6/14 |
Regado Biosciences Inc. (Basking Ridge, N.J.) |
REG2 anticoagulation system |
Two-component system consisting of a subcutaneous depot formulation of pegnivacogin and anivamersen |
Venous thrombosis indications such as DVT and VTE prophylaxis |
Reported results from a phase I trial demonstrating the dose- and concentration-dependent attenuation of thrombin generation and subsequent restoration of thrombin following reversal associated with REG2 |
6/24/14 |
CENTRAL NERVOUS SYSTEM | |||||
Cara Therapeutics Inc. (Shelton, Conn.) |
CR845 |
Peripherally selective kappa opioid agonist |
Acute and chronic pain |
Dosed the first subjects in a phase Ia/Ib trial |
6/19/14 |
Coronado Biosciences Inc. (Burlington, Mass.) |
CNDO-201 |
Trichuris suis ova; oral therapy |
Autism spectrum disorder |
Data from a pilot study showed it failed to reach statistical significance, though benefits of TSO over placebo were observed in several subscales, including the Montefiore-Einstein Rigidity Scale, ABC Irritability, RBS-R Sameness and RBS-R Restricted Behavior |
6/27/14 |
Elite Pharmaceuticals Inc. (Northvale, N.J.) |
ELI-200 |
An abuse deterrent product |
Pain |
The first subject was dosed in the treatment phase of a human abuse liability clinical study for ELI-200 |
6/10/14 |
Enteris Biopharma Inc. (Boonton, N.J.) and Cara Therapeutics Inc. (Shelton, Conn.) |
CR845 |
Peripherally selective kappa opioid agonist; tablet formulation |
Acute and chronic pain |
Dosed the first subjects in a further phase Ia/Ib trial of a tablet formulation |
6/23/14 |
Sage Therapeutics Inc. (Cambridge, Mass.) |
SAGE-547 |
An allosteric modulator of GABAA receptors |
Super-refractory status epilepticus |
Preliminary data from its ongoing open-label phase I/II trial showing that the first three patients met both the primary endpoint of safety and tolerability and the key secondary endpoint of being successfully weaned off their anesthetic agents while SAGE-547 was being administered |
6/6/14 |
Trevena Inc. (King of Prussia, Pa.) |
TRV734 |
Mu-opioid receptor biased ligand |
Acute and chronic pain |
Results from a phase I trial demonstrated that it is pharmacologically active at a range of safe and well-tolerated doses |
6/24/14 |
Trevena Inc. (King of Prussia, Pa.) |
TRV130 |
G protein-biased ligand at the mu-opioid receptor |
Acute pain |
Phase Ib data suggest that it may reduce pain more effectively, act more quickly and produce less severe gastrointestinal side effects, reducing the risk of respiratory depression compared to existing opioids, improving the margin of safety for dosing |
6/25/14 |
DIABETES | |||||
Prometic Life Sciences Inc. (Laval, Quebec) |
PBI-4050 |
Oral anti-fibrotic drug |
Diabetes |
Completed a phase I trial in 40 healthy volunteers and found the oral anti-fibrotic drug to be safe and well tolerated, without any serious adverse events reported in any of the five cohorts tested |
6/19/14 |
Rhythm Pharmaceuticals Inc. (Boston) |
RM-493 |
Melanocortin 4 receptor (MC4R) agonist |
Obesity |
Results from a crossover study in 12 obese individuals suggested that RM-493 increased resting energy expenditure significantly |
6/24/14 |
Targacept Inc. (Winston-Salem, N.C.) |
TC-6499 |
Small molecule that modulates the activity of the alpha3beta4 and other neuronal nicotinic receptors |
Diabetic gastroparesis |
Started a phase I/II exploratory study |
6/26/14 |
INFECTION | |||||
Achillion Pharmaceuticals Inc. (New Haven, Conn.) |
ACH-3422 |
A uridine-analogue nucleotide polyermase inhibitor |
Genotype 1 chronic hepatitis C virus infection |
Started dosing for seven days in a phase I trial |
6/11/14 |
Basilea Pharmaceutica Ltd. (Basel, Switzerland) |
BAL30072 |
Gram-negative antibiotic |
Infections |
Started a phase I study |
6/13/14 |
Calimmune Inc. (San Diego) |
Cal-1 |
Gene-based stem cell therapy |
HIV |
The data safety monitoring board for a phase I/II trial confirmed no serious adverse events or dangerous side effects in the first group of patients, indicating that the firm can begin treating a second cohort |
6/26/14 |
Debiopharm Group (Lausanne, Switzerland) |
Debio 1450 |
An anti-infective agent selectively active against a large number of Staphylococcus species |
Methicillin-resistant S. aureus and vancomycin-intermediate S. aureus |
Launched a phase I study |
6/20/14 |
Dilaforette AB (Stockholm) |
Sevuparin |
Malaria treatment |
Malaria |
The drug was found to be safe and well tolerated as adjunct treatment in adults with uncomplicated falciparum malaria but did not achieve statistical significance in the primary endpoint |
6/2/14 |
Genocea Biosciences Inc. (Cambridge, Mass.) |
GEN-004 |
Vaccine |
To prevent infections from all serotypes of pneumococcus, or Streptococcus pneumoniae |
Results from a phase I study met its safety, tolerability and immunogenicity goals, including measurable increases in the blood of T helper 17 (TH17) cells; serum IgG titers increased in a dose-dependent manner to each of the antigens included in GEN-004, and measurable increases in peripheral TH17 responses were seen among subjects receiving the highest dose (100 mcg) with adjuvant |
6/24/14 |
Themis Bioscience GmbH (Vienna) |
Vaccine |
Prophylactic vaccine candidate |
Chikungunya fever |
Induced a significant neutralizing immune response and was confirmed as safe, based on interim results from a phase I study |
6/13/14 |
INFLAMMATORY | |||||
Cellular Biomedicine Group Inc. (Palo Alto, Calif.) |
Rejoin |
Human adipose-derived mesenchymal precursor cell therapy |
Knee osteoarthritis |
Six-month follow-up data analysis of its phase I/IIa trial showed an increase in cartilage volume of whole joint as early as three months after therapy, as measured via MRI Quantitative Assessment, and showed reduction of bone marrow lesions in some patients |
6/20/14 |
MISCELLANEOUS | |||||
Agios Pharmaceuticals Inc. (Cambridge, Mass.) |
AG-348 |
An orally available activator of pyruvate kinase-R |
Pyruvate kinase deficiency |
A multiple ascending dose phase I trial in healthy volunteers has started |
6/10/14 |
Bone Therapeutics SA (Gosselies, Belgium) |
Allob |
Allogeneic osteoblastic (bone-forming) cell therapy |
Delayed union fractures |
The first patient was dosed in a phase I/IIa trial |
6/26/14 |
Isis Pharmaceuticals Inc. (Carlsbad, Calif.) |
ISIS-DMPKRx |
Designed to improve the underlying genetic defect that causes DM1 |
Myotonic dystrophy type 1 |
Initiated a phase I study |
6/10/14 |
Ligand Pharmaceuticals Inc. (San Diego) |
Lasofoxifene |
Selective estrogen receptor modulator |
To increase circulating testosterone and gonadotropin levels |
Data from the first-in-human study, performed by Pfizer Inc., showed a single dose of lasofoxifene to increase circulating testosterone and gonadotropin levels in healthy young men; single oral doses of lasofoxifene of 1 mg, 3 mg, 30 mg and 100 mg all increased levels of testosterone, with the 30-mg and 100-mg doses raising levels by more than 80% |
6/24/14 |
Medgenics Inc. (Wayne, Pa.) |
MDGN-201 |
Epodure |
End-stage renal disease or chronic kidney disease |
The first patient was enrolled in its phase I/II study, with the aim of validating the potential of the firm's Biopump platform using a second-generation vector |
6/6/14 |
Stemcells Inc. (Newark, Calif.) |
HUCNS-SC |
Human neural stem cells |
Dry age-related macular degeneration |
Closed enrollment in its phase I/II trial with interim data showing a 70% reduction in the rate of geographic atrophy (GA) vs. the control eye and a 65% reduction in the rate of GA vs. the natural history of the disease, following a single dose |
6/23/14 |
Synageva Biopharma Corp. (Lexington, Mass.) |
SBC-102 |
Sebelipase alfa |
Lysosomal acid lipase deficiency |
Data showed that sebelipase alfa continues to improve disease-related abnormalities at two years in an ongoing phase I/II trial |
6/4/14 |
Ultragenyx Pharmaceutical Inc. (Novato, Calif.) |
KRN23 (US023) |
Anti-FGF23 antibody |
X-linked hypophos-phatemia |
Data from a phase I/II study demonstrated that blocking excess FGF23 increases urinary phosphorus reabsorption, serum phosphorus levels and 1,25 dihydroxy vitamin D levels, and repeat doses over four months led to an increase in serum phosphorus in 100% of patients, with about 89% reaching the low end of the normal range |
6/26/14 |
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. |