Company |
Product |
Description |
Indication |
Status |
Date |
CANCER | |||||
Access Pharmaceuticals Inc. (New York) |
Mugard |
Muco-adhesive polymer treatment |
Biopsy-proven oral lichen planus |
Results from a pilot study of 20 patients showed statistically significant improvement in ulcerations and pain |
4/16/14 |
Affimed Therapeutics AG (Heidelberg, Germany) |
AFM13 |
Monotherapy |
Advanced relapsing/refractory Hodgkin lymphoma |
Phase I data confirmed safety in 28 patients |
4/11/14 |
Aprea AB (Stockholm) |
APR-246 |
A compound aimed at restoring normal function to tumor suppressor protein p53 |
Relapsed platinum-sensitive high-grade serous ovarian cancer |
Dosing started in its phase I/II trial of APR-246; it will enroll about 180 patients in a two-part design |
4/17/14 |
Arno Therapeutics Inc. (Flemington, N.J.) |
Onapristone |
An oral, anti-progestin hormone blocker |
Advanced castration-resistant prostate cancer |
Enrolled the first patient in a phase I/II trial |
4/8/14 |
Celldex Therapeutics Inc. (Hampton, N.J.) |
CDX-1401 |
Vaccine consisting of a fully human monoclonal antibody with specificity for the dendritic cell receptor DEC-205 linked to the NY-ESO-1 tumor antigen |
Solid tumors |
Final phase I data demonstrated robust antibody and T-cell responses and evidence of clinical benefit in patients with very advanced cancers and suggest that CDX-1401 may predispose patients to better outcomes on subsequent therapy with checkpoint inhibitors |
4/17/14 |
Cleveland Biolabs Inc. (Buffalo, N.Y.) |
CBL0102 |
Quinacrine; an orally administered small molecule |
Advanced cancers |
Phase I trial characterized the drug's safety, profiled its pharmacokinetics, and assessed it for preliminary evidence of antitumor activity in the liver |
4/17/14 |
Deciphera Pharmaceuticals LLC (Lawrence, Kan.) |
LY3009120 |
Pan-RAF inhibitor |
Cancer |
Started a phase I trial |
4/8/14 |
Development Center for Biotechnology (Taiwan) |
DCBCI0901 |
mTOR inhibitor |
Cancer |
Received approval from the FDA and the TFDA to begin phase I trials |
4/25/14 |
Dicerna Pharmaceuticals Inc. (Watertown, Mass.) |
DCR-MYC |
A synthetic double-stranded RNA in a stable lipid particle |
Solid tumors, multiple myeloma or lymphoma |
Started a phase I trial |
4/18/14 |
Helix Biopharma Corp. (Aurora, Ontario) |
L-DOS47 |
A targeted antibody derived from naturally occurring plant enzyme urease |
Stage IV recurrent or metastatic nonsquamous non-small-cell lung cancer |
Received FDA approval to start a phase I trial |
4/23/14 |
Immatics Biotechnologies GmbH (Tuebingen, Germany) |
IMA950 |
Cancer vaccine |
Adult brain tumor |
A phase I trial met its primary endpoints of safety and immunogenicity |
4/24/14 |
Innate Pharma SA (Marseille, France) |
Iirilumab |
Anti-KIR; checkpoint inhibitor |
Solid tumors |
Began the cohort expansion portion of an open label phase I trial |
4/1/14 |
Karyopharm Therapeutics Inc. (Natick, Mass.) |
KPT-330 |
Selinexor |
Leukemia |
Started a phase I trial of selinexor in children with high-risk malignancies resistant to standard chemotherapy |
4/22/14 |
Peregrine Pharmaceuticals Inc. (Tustin, Calif.) |
Bavituximab |
Immunotherapy |
Advanced melanoma |
Opened enrollment of a trial of bavituximab in combination with Yervoy (ipilimumab, Bristol-Myers Squibb Co.) |
4/24/14 |
Precision Biologics Inc. (Dallas) |
NEO-102 |
A reformulation of the monoclonal antibody NPC-1C |
Refractory metastatic pancreatic and colorectal cancer |
Phase I/IIa data showed preliminary signs of activity based on stabilization of disease |
4/11/14 |
Sorrento Therapeutics Inc. (San Diego) |
RTX |
Resiniferatoxin |
Intractable cancer pain |
Phase I/II data from six patients showed clinically meaningful improvement in quality of life, achieving, on average, a 20% reduction in their pain intensity |
4/7/14 |
Tocagen Inc. (San Diego) |
Toca 511 and Toca FC |
Vocimagene amiretrorepvec and flucytosine; gene therapy candidate |
Recurrent high-grade glioma |
Interim data of patients treated with the combination showed it to be safe and well tolerated with minimal treatment-related toxicity |
4/11/14 |
Xbiotech Inc. (Austin, Texas) |
Xilonix |
The firm's noncytotoxic antitumor therapy |
Advanced cancer |
Results from a phase I/II study showed an excellent safety profile, while overall, patients' constitutional symptoms improved |
4/18/14 |
CARDIOVASCULAR | |||||
Capricor Therapeutics Inc. (Los Angeles) |
CAP-1002 |
An allogeneic cardiospere-derived stem cell |
Myocardial infarction |
Phase I/II data showed it met its primary endpoint of safety |
4/1/14 |
Capstone Therapeutics Inc. (Tempe, Ariz.) |
AEM-28 |
An ApoE mimetic peptide |
Low-density lipoprotein/non-high-density lipoprotein cholesterol reduction |
Dosing was started in the phase Ia trial |
4/9/14 |
Cytori Therapeutics Inc. (San Diego) |
Cytori Cell Therapy |
Employs a mixed population of adipose derived regenerative cells extracted from a patient's own fat tissue using Cytori's Celution system |
Chronic ischemic heart failure |
Data from the PRECISE trial phase I/IIa feasibility trial demonstrated statistically significant differences in cardiac functional capacity between treated and placebo groups |
4/15/14 |
CENTRAL NERVOUS SYSTEM | |||||
Cynapsus Therapeutics Inc. (Toronto) |
APL-130277 |
A single 25-mg sublingual strip dose of apomorphine |
Parkinson's disease |
Interim data from its pilot study indicated that a higher load of drug on the strip does result in a higher amount of drug entering the bloodstream |
4/3/14 |
Glenmark Pharmaceuticals SA (Mumbai, India; subsidiary of Glenmark Pharmaceuticals Ltd.) |
GBR 900 |
Monoclonal antibody that targets TrkA |
Chronic pain |
Is entering human trials |
4/30/14 |
Prothena Corp. plc (Dublin) |
PRX002 |
Monoclonal antibody targeting alpha-synuclein |
Parkinson's disease |
Started a phase I trial |
4/10/14 |
Vernalis plc (Winnersh, UK) |
V81444 |
An A2A antagonist |
Parkinson's disease, attention deficit hyperactivity disorder and other disorders |
Results from a phase Ib/II proof-of-concept study showed it achieved a statistically significant improvement in the number of correct scores in PERMP-P measure (p = 0.019) compared to placebo |
4/25/14 |
DIABETES | |||||
Aspireo Pharmaceuticals Ltd. (Tel Aviv, Israel) |
DG3173 |
Somatroprim; a somatostatin analogue |
Diabetes |
Final phase Ib data demonstrated that while octreotide significantly inhibited the secretion of insulin and glucagon in humans and showed a significant and sustained increase of plasma glucose levels, the company's DG3173 had much less of an effect on insulin and glucagon release |
4/23/14 |
Halozyme Therapeutics Inc. (San Diego) |
Hylenex |
Recombinant and a new formulation |
Type 1 diabetes |
The primary endpoint of non-inferiority of A1C levels at six months was met for the CONSISTENT 1 trial |
4/1/14 |
Sernova Corp. (London, Ontario) |
Cell Pouch |
Contains insulin-producing islets |
Unstable type 1 diabetes |
Interim results from a phase I/II study demonstrated that in the first group of patients the implanted Cell Pouch is showing longer-term safety and biocompatibility |
4/23/14 |
INFECTION | |||||
Galectin Therapeutics Inc. (Norcross, Ga.) |
GR-MD-02 |
Targets galectin-3 |
Non-alcoholic steatohepatitis with advanced fibrosis |
The first patient in cohort 2 of its phase I trial was successfully dosed with 4 mg/kg, which is double the dose given in cohort 1; all eight patients have received their first infusion in cohort 2 |
4/18/14; 4/24/14 |
Hepatera Ltd. (Moscow) |
Myrcludex B |
Inhibits the entry mechanism of HBV and HDV |
Chronic hepatitis B virus and hepatitis delta virus |
Initiated a trial investigating it for the effects of Myrcludex in combination with pegylated interferon and the use of the entry inhibitor as pre-treatment before interferon therapy is initiated |
4/3/14 |
INFLAMMATORY | |||||
Ablynx NV (Ghent, Belgium) |
ALX-0061 |
Anti-IL-6R nanobody |
Inflammatory diseases |
Started dosing in healthy volunteers in a phase I trial evaluating a subcutaneous formulation |
4/23/14 |
MISCELLANEOUS | |||||
Agios Pharmaceuticals Inc. (Cambridge, Mass.) |
AG-348 |
A small molecule activator of PKR |
Pyruvate kinase deficiency |
Started a phase I trial |
4/18/14 |
Allena Pharmaceuticals Inc. (Newton, Mass.) |
ALLN-177 |
An orally administered recombinant oxalate degrading enzyme |
Hyperoxaluria and kidney stones |
Phase I data demonstrated a statistically significant difference in the reduction of urinary oxalate levels in 30 healthy subjects when the treatment was compared with placebo (p = 0.0002) |
4/30/14 |
Critical Pharmaceuticals Ltd. (Nottingham, UK) |
Teriparatide |
Nano-enabled intranasal teriparatide product |
Osteoporosis |
Started a phase I trial in healthy postmenopausal women |
4/16/14 |
Galectin Therapeutics Inc. (Norcross, Ga.) |
GR-MD-02 |
Galectin inhibitor |
Advanced fibrosis |
The first phase I cohort showed a therapeutic effect on fibrosis, inflammation and cellular injury |
4/2/14 |
Kamada Ltd. (Ness Ziona, Israel) |
Glassia |
Liquefied human alpha-1 antitrypsin |
Graft-vs.-host disease |
Company is starting a small phase I/II proof-of-concept study |
4/4/14 |
Lpath Inc. (San Diego) |
Lpathomab |
An antibody to lysophosphatidic acid |
Brain injury |
Started a study of brain injury induced by blast overpressure |
4/24/14 |
Nitto Denko Corp. (Osaka, Japan) |
RNAi-based drug |
Consists of an siRNA that inhibits the cause of fibrosis, as well as a targeted lipid nanoparticle delivery system |
Fibrosis in the liver and other organs |
Dosing of healthy volunteers in a phase I study has completed |
4/23/14 |
Oxford Biomedica plc (Oxford, UK) |
Retinostat |
A lentiviral vector-based treatment |
Neovascular wet age-related macular degeneration |
Completed the planned recruitment of 21 patients into its phase I trial |
4/8/14 |
Prothena Corp. plc (Dublin) |
NEOD001 |
A monoclonal antibody |
AL amyloidosis and persistent organ dysfunction |
Interim data from an ongoing phase I study showed that NEOD001 administered intravenously once per month was generally safe and well tolerated at the doses studied |
4/28/14 |
Questcor Pharmaceuticals Inc. (Anaheim Hills, Calif.) |
H.P. Acthar Gel |
Repository corticotropin |
Nephrotic syndrome |
Clinical results showed it could be a potentially useful therapy for inducing remission of proteinuria |
4/22/14 |
Revance Therapeutics Inc. (Newark, Calif.) |
RT002 |
Injectable botulinum toxin type A |
Moderate to severe glabellar (frown) lines |
Phase I/II data showed that the trial met its primary efficacy and safety endpoints |
4/23/14 |
Stemcells Inc. (Newark, Calif.) |
HuCNS-SC |
Human neural stem cells |
Spinal cord injury |
Completed enrollment of the phase I/II trial |
4/18/14 |
RESPIRATORY | |||||
Xenetic Biosciences Inc. (Lexington, Mass.) |
Pulmoxen |
A modified form of recombinant human Dnase I |
Cystic fibrosis |
Positive results from its phase I trial in Russia showed that two doses of 2,500 IU and 5,000 IU were found to be safe and well tolerated |
4/8/14 |
|
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. | |||||
