Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Anacor Pharmaceuticals Inc. (Palo Alto, Calif.) |
Crisaborole |
Topical ointment 2% (formerly AN2728), a nonsteroidal anti-inflammatory phosphodeisterase-4 inhibitor |
Mild to moderate atopical dermatitis |
Top-line results from the long-term safety study showed it was well tolerated and demonstrated a safety profile consistent with that seen in the positive phase III pivotal studies reported in July |
11/2/15 |
Five Prime Therapeutics Inc. (South San Francisco) |
FPA008 |
Anti-CSF1R antibody that blocks the binding of CSF1 and IL-34 ligands to CSF1R |
Rheumatoid arthritis |
Preliminary data from part 3 of Five Prime's ongoing phase I trial showed it was well tolerated up to 6 mg/kg x 2 doses |
11/12/15 |
Pfenex Inc. (San Diego) |
PF530 |
A biosimilar candidate of Betaseron (interferon beta-1b, Bayer AG) |
Multiple sclerosis |
Phase I trial enrolled 12 subjects; data showed no statistically significant differences in PK and PD between the groups administered PF530 vs. the reference product |
11/16/15 |
Qu Biologics Inc. (Vancouver, British Columbia) |
QBECO SSI |
Derived from components of inactivated E. coli |
Crohn's disease |
Completed enrollment of the QBECO-01 phase I/II trial |
11/25/15 |
Vitae Pharmaceuticals Inc. (Fort Washington, Pa.) |
VTP-43742 |
Orally active RORgammat inhibitor |
Moderate to severe psoriasis |
Top-line phase I data showed it was safe and generally well tolerated at all dose levels assessed |
11/20/15 |
Xencor Inc. (Monrovia, Calif.) |
XmAb5871 |
Fc-engineered antibody |
Rheumatoid arthritis |
Phase Ib/IIa data demonstrated it was generally well tolerated and showed trends in improvement in RA disease activity by multiple disease activity measures and across multiple dose groups |
11/10/15 |
CANCER | |||||
Adaptimmune Therapeutics plc (Oxford, U.K.) |
Affinity enhanced T-cell therapy |
Targets the NY-ESO-1 cancer antigen |
Non-small-cell lung cancer (NSCLC) |
Started a phase I/II trial of its affinity-enhanced T-cell therapy targeting the NY-ESO-1 cancer antigen in patients with stage IIIb or stage IV NSCLC |
11/25/15 |
Advaxis Inc. (Princeton, N.J.) |
ADXS-HPV |
Axalimogene filolisbac |
Persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix |
Phase I data showed it may be safely administered with prophylactic antibiotics up to 1 x 1010 CFU, a 10-fold increase |
11/10/15 |
Agios Pharmaceuticals Inc. (Cambridge, Mass.) |
AG-120 |
Oral selective inhibitor of mutant isocitrate dehydrogenase-1 |
Advanced solid tumors |
First data from the dose-escalation portion of the ongoing phase I study showed no dose-limiting toxicities |
11/10/15 |
Aptose Biosciences Inc. (San Diego) |
APTO-253 |
Small molecule that induces a gene that expresses the Krüppel-like factor 4 (KLF4) master transcription factor |
Hematologic cancers |
FDA placed a clinical hold on the phase Ib trial, following the company's voluntary suspension of dosing due to an operational difficulty with an intravenous infusion pump |
11/23/15 |
Beigene Co. Ltd. (Beijing) |
BGB-290 |
Poly-ADP ribose polymerase inhibitor |
Solid tumors |
A first-in-human dose-escalation trial enrolled 29 patients that were treated; at the 80 mg twice daily, the maximum dose has not been reached, and dose escalation is ongoing; responses were seen even at the lowest dose tested; of 15 ovarian cancer patients, seven responded to the drug, with one complete and six partial responses |
11/10/15 |
Calithera Biosciences Inc. (South San Francisco) |
CB-839 |
Selective, orally bioavailable glutaminase inhibitor |
Renal cell carcinoma |
Phase I data support earlier findings of the clinical activity, tolerability and unique mechanism of action, as well as show one partial response, and stable disease across a variety of tumor types |
11/10/15 |
Cellceutix Corp. (Beverly, Mass.) |
Kevetrin |
Small molecule |
Solid tumors |
Completed enrollment in the phase I study |
11/12/15 |
Cerulean Pharma Inc. (Cambridge, Mass.) |
CRLX101 |
Nanoparticle-based, camptothecin-loaded |
Advanced gastric cancers |
Pre- and post-tumor treatment biopsies show the presence of CRLX101 and its anticancer payload in tumors and an almost exclusive absence of both from surrounding normal tissue; inhibition of the molecular targets of CRLX101 was demonstrated in post-treatment biopsies |
11/10/15 |
Chemocentryx Inc. (Mountain View, Calif.) |
CCX872 |
A CCR2 inhibitor |
Pancreatic cancer |
Phase Ib data showed that, after a single dose, across four patients in part A of the study, the pharmacokinetic profile was favorable and pharmacodynamic assays demonstrated excellent receptor coverage after 12 hours |
11/5/15 |
Cortice Biosciences Inc. (New York) |
TPI287 |
A microtubule stabilizing agent |
Recurrent glioblastoma |
Phase I/II data showed that of 11 evaluable patients, one achieved a complete response and three achieved stable disease; median progression-free survival is 1.4 months, and median overall survival is 4.7 months |
11/23/15 |
Essa Pharma Inc. (Houston) |
EPI-506 |
Intended to block a new target on the androgen receptor, the N-terminal domain |
Metastatic castration-resistant prostate cancer |
The first patient was enrolled in its phase I/II study |
11/30/15 |
Five Prime Therapeutics Inc. (South San Francisco) |
FPA144 |
An isoform-selective antibody |
Gastric cancer |
The dose-escalation part of the ongoing phase I trial has been completed and dose expansion has begun at a selected dose in new cohorts of patients whose tumors overexpress FGFR2b |
11/23/15 |
Galena Biopharma Inc. (San Ramon, Calif.) |
GALE-302 |
E39 peptide |
Ovarian and breast cancer |
Data from a recent phase Ib trial showed it achieved a statistically significant increase in local reactions and delayed type hypersensitivity when delivering GALE-302 after E39 (GALE-301) in the primary vaccine series (p<0.001) |
11/10/15 |
Genocea Biosciences Inc. (Cambridge, Mass.) |
ATLAS |
Rapid antigen identification screening system |
Cancer |
Findings from a pilot study involving a retrospective analysis of 10 checkpoint inhibitor-treated patients' T-cell responses to 23 known tumor-associated antigens support the potential of ATLAS, which successfully identified the cancer antigens to which either (or both) CD4+ or CD8+ T cells became activated |
11/5/15 |
Halozyme Therapeutics Inc. (San Diego) and Janssen Biotech Inc. (unit of Johnson & Johnson; New Brunswick, N.J.) |
Daratumumab |
Subcutaneously delivered with Halozyme's Enhanze technology |
Multiple myeloma |
Dosed the first patient in a phase Ib trial |
11/5/15 |
Heat Biologics Inc. (Durham, N.C.) |
HS-410 |
Vesigenurtacel-L |
High-risk, non-muscle-invasive bladder cancer |
Phase I results of HS-410 after standard-of-care Bacillus Calmette-Guerin showed the drug had a positive safety profile and was well tolerated with no patients discontinuing the trial due to adverse events |
11/9/15 |
Helix Biopharma Corp. (Aurora, Ontario) |
L-DOS47 |
An immunoconjugate-based drug |
Cancer |
Opened patient screening for the 14th dose level cohort of its ongoing phase I/II study in Poland; during the 13th cohort, one patient experienced a serious adverse event that was a dose-limiting toxicity and was withdrawn from further dosing and later recovered from the event |
11/5/15 |
Ignyta Inc. (San Diego) |
RXDX-105 |
Orally available, small molecule multikinase inhibitor |
Cancer |
Interim results from the company's ongoing phase I trial showed it was generally well tolerated |
11/10/15 |
Immunogen Inc. (Waltham, Mass.) |
Mirvetuximab soravtansine |
Folate receptor alpha-targeting antibody-drug conjugate candidate |
Platinum-resistant ovarian cancer |
Phase I data in 20 efficacy-evaluable patients identified an association between the amount of FRα present on patient cancer cells and response to treatment with the drug; it found nine of 10 patients with high levels of FRα had an objective response on treatment |
11/10/15 |
Immuodulon Therapeutics Ltd. (London) |
IMM-101 |
A bacterially derived systemic immunomodulator administered intradermally |
Stage IV metastatic melanoma |
The latest five-year survival results showed that of the 10 patients who entered the open-label, long-term follow-up study after the original phase I study in 2010, six are alive and still being treated |
11/20/15 |
Incyte Corp. (Wilmington, Del.) |
Epacadostat |
Selective indoleamine 2,3-dioxygenase inhibitor |
Advanced cancers |
Results from an ongoing proof-of-concept phase I/II study of epacadostat in combination with anti-PD-1 drug Keytruda (pembrolizumab, Merck & Co. Inc.) indicated a 79% disease control rate in evaluable patients |
11/4/15 |
Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.) |
INO-3112 |
DNA-based immunotherapy |
Head and neck cancer associated with human papillomavirus types 16 and 18 |
Interim data from a phase I/IIa study showed that it generated specific T-cell responses and was well tolerated |
11/6/15 |
Loxo Oncology Inc. (Stamford, Conn.) |
LOXO-101 |
A selective inhibitor of tropomyosin receptor kinase signaling molecules |
Solid tumors |
Phase I data showed all three evaluable patients achieved an objective response at the first response assessment |
11/9/15 |
Mirna Therapeutics Inc. (Austin, Texas) |
MRX34 |
MiR-34 replacement therapy |
Solid tumors |
Interim results from its ongoing phase I trial has a safety profile manageable with standard interventions or tests used by oncologists, and demonstrates the therapeutic potential |
11/10/15 |
Molecular Partners AG (Zurich-Schlieren, Switzerland) |
MP0250 |
Multi-Darpin-blocking vascular endothelial growth factor and hepatocyte growth factor |
Solid tumors |
Preliminary phase I data showed that MP0250 was well tolerated in 15 patients with solid tumors, with favorable pharmacokinetics and sustained exposure over multiple applications |
11/9/15 |
Newlink Genetics Corp. (Ames, Iowa) |
Indoximod |
Indoleamine-(2,3)-dioxygenase pathway inhibitor |
Glioblastoma |
Phase Ib data showed that indoximod in combination with temozolomide demonstrated preliminary efficacy, including a six-month progression-free survival rate of 25% |
11/24/15 |
Oncoceutics Inc. (Philadelphia) |
ONC-201 |
Small molecule with an active angular structure |
Solid tumors |
Phase I data showed the study advanced through all five intended dose levels, 125 mg through 625 mg, with only one patient per dose level, and identified a recommended phase II dose |
11/3/15 |
Oncoceutics Inc. (Philadelphia) |
ONC201 |
Small-molecule drug |
Multiple myeloma |
Began enrolling patients with various solid tumors in a dose-escalation trial |
11/30/15 |
Oncolytics Biotech Inc. (Calgary, Alberta) |
Reolysin |
Reovirus variant |
Relapsed or refractory multiple myeloma |
Initiated a phase Ib study of Reolysin in combination with standard doses of bortezomib (Velcade, Millennium: The Takeda Oncology Co.) and dexamethasone |
11/19/15 |
Onxeo SA (Paris) |
Belinostat |
Pan-histone deacetylase inhibitor |
Peripheral T-cell lymphoma |
Phase I data of belinostat in combination with CHOP chemotherapy regimen demonstrated it to be well-tolerated with an objective response rate of 89% based on 18 evaluable patients (16/18), with the vast majority, 72%, achieving a complete response (13/18) and 17% achieving a partial response (3/18) |
11/10/15 |
Oxigene Inc. (South San Francisco) |
CA4P |
Combretastatin A4 Prodrug |
Advanced recurrent ovarian cancer |
Initial phase Ib/II data of CA4P, in combination with Votrient (pazopanib, Glaxosmithkline plc), showing that, of nine patients, two showed partial responses, five had stable disease and two had progressive disease |
11/9/15 |
Polaris Group (San Diego) |
ADI-PEG 20 |
Pegylated arginine deiminase |
Malignant plural mesothelioma and nonsquamous non-small-cell lung carcinoma |
Phase I preliminary results showed no dose-limiting toxicity observed in the nine patients enrolled; seven patients had partial responses and two patients had stable disease as best response, for a 100% disease control rate |
11/12/15 |
Seattle Genetics Inc. (Bothell, Wash.) |
SGN-CD33A |
Vadastuximab talirine |
Relapsed or refractory acute myeloid leukemia |
Started a phase I/II trial |
11/24/15 |
Targovax AS (Lysaker, Norway) |
ONCOS-102 |
Adenovirus-based cancer immunotherapy |
Advanced tumors |
Immune biomarker data from a phase I study suggested that local immunotherapy with ONCOS-102 has the potential to activate immunologically silent tumors and reduce local immune suppression |
11/6/15 |
Tocagen Inc. (San Diego) |
Toca 511 in combination with Toca Fc |
Injectable RRV and extended-release version of 5-FC |
Glioma and other cancers |
Phase I and preclinical data demonstrated a favorable safety profile and extended overall survival in patients |
11/24/15 |
Tracon Pharmaceuticals Inc. (San Diego) |
TRC105 |
A monoclonal antibody to endoglin |
Advanced or metastatic renal cell carcinoma |
Phase Ib data of TRC105 with Inlyta (axitinib, Pfizer Inc.) showed an overall response rate in 29% of those receiving the combination treatment, with 59% showing stable disease, for an overall disease control rate of 88% |
11/9/15 |
Virttu Biologics Ltd. (Glasgow, UK) |
Seprehvir |
Oncolytic immunotherapeutic |
Mesothelioma |
Ongoing phase I/IIa clinical trial data identified a robust Th1 cytokine response and the appearance of antitumor IgG responses in most treated patients, confirming Seprehvir's potential for immunological efficacy |
11/6/15 |
Ziopharm Oncology Inc. (Boston) |
Veledimex |
Ad-RTS-hIL-12+ orally administered veledimex; a viral gene therapy candidate for the controlled expression of IL-12 |
Recurrent or progressive glioblastoma or grade III malignant glioma |
Results from an ongoing phase I dose-escalation study showed IL-12 was detectable in peripheral blood along with downstream interferon-gamma, indicating it crossed the blood-brain barrier |
11/20/15 |
CARDIOVASCULAR | |||||
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) and The Medicines Co. (Parsippany, N.J.) |
ALN-PCSsc |
Subcutaneous administration |
High cholesterol |
Results from the ongoing phase I trial showed that subcutaneous administration resulted in an up to 83% lowering of LDL-C, with an up to 64 ± 5 percent mean maximum reduction |
11/12/15 |
Capricor Therapeutics Inc. (Los Angeles) |
CAP-1002 |
Allogeneic, cardiosphere-derived cell therapy |
Advanced heart failure |
Multi-vessel intracoronary infusion of CAP-1002 in subjects with dilated cardiomyopathy was shown to be safe in this study with no major adverse cardiac events reported at one month or at six months post-infusion; the six-month data demonstrated encouraging and congruent preliminary efficacy signals in multiple parameters |
11/10/15 |
Ironwood Pharmaceuticals Inc. (Cambridge, Mass.) |
IW-1973 and IW-1701 |
Soluble guanylate cyclase stimulators |
Cardiovascular diseases, fibrotic diseases, muscular dystrophy and other disorders |
Started a phase Ib study of IW-1973 and a phase Ia study of IW-1701 |
11/4/15 |
CENTRAL NERVOUS SYSTEM | |||||
Aequus Pharmaceuticals Inc. (Vancouver, British Columbia) |
AQS1301 |
Once-weekly transdermal patch |
Bipolar disorder, schizophrenia, major depressive disorder and irritability associated with autistic disorder |
Will initiate a phase I trial following receipt of a no objection letter from Health Canada |
11/17/15 |
Alzheon Inc. (Framingham, Mass.) |
ALZ-801 |
A prodrug of tramiprosate |
Alzheimer's disease |
Started two studies, a phase Ib multiple dose-ranging trial and a phase Ib tablet bioequivalence study |
11/9/15 |
Neurocrine Biosciences Inc. (San Diego) |
NBI-640756 |
A vesicular monoamine transporter 2 inhibitor |
Essential tremor |
Started a sequential dose-escalation phase I safety and pharmacokinetics study exploring NBI-640756 in up to 32 healthy volunteers |
11/5/15 |
Ocera Therapeutics Inc. (Palo Alto, Calif.) |
OCR-002 |
Ornithine phenylacetate |
Neurocognitive symptoms of acute hepatic encephalopathy |
Phase I study showed the oral formulation in healthy subjects exhibited encouraging extended-release properties, demonstrated a desirable pharmacokinetic profile and was well tolerated |
11/18/15 |
Otonomy Inc. (San Diego) |
OTO-311 |
A sustained-exposure formulation of the N-Methyl-D-Aspartate receptor antagonist, gacyclidine |
Tinnitus |
Enrolled the first subjects in a phase I safety trial |
11/17/15 |
Portage Biotech Inc. (Toronto) |
BHV-0223 |
A glutamate modulating agent |
Mood and anxiety disorders |
Preliminary results showed a phase I trial met its study objectives and supports advancing it into later stages of development |
11/23/15 |
Rhythm Pharmaceuticals Inc. (Boston) |
RM-493 |
Setmelanotide; melanocortin-4 receptor |
MC4R heterozygous deficiency obesity |
Phase Ib trial showed that it reduced weight after four weeks of treatment, with good tolerability; it demonstrated strong trends for placebo-subtracted weight loss ( 2.62 kg; p=0.088), waist circumference ( 5.1 cm; p = 0.188) and daily caloric intake ( 351 kCal/day; p=not significant), without clinically important effects on heart rate or blood pressure |
11/9/15 |
Turing Pharmaceuticals AG (unit of Turing Pharmaceuticals LLC; New York) |
TUR-004 |
Oral formulation |
Epileptic encephalopathies |
The first subjects were dosed in the single ascending dose study |
11/17/15 |
Zosano Pharma Corp. (Fremont, Calif.) |
ZP-Triptan |
Zolmitriptan-coated microneedle patch |
Migraine |
Appears to be well tolerated and produced no safety issues during a phase I study |
11/4/15 |
DIABETES | |||||
Adocia SAS (Lyon, France) |
Biochaperone Combo |
A formulation combining insulin glargine and insulin lispro |
Type 2 diabetes |
Top-line phase Ib data showed it was significantly superior to Humalog Mix75/25 and exhibited no difference compared to the separate injections of Lantus and Humalog |
11/30/15 |
GASTROINTESTINAL | |||||
Hutchison China Meditech Ltd. (Hong Kong) |
HMPL 523 |
A selective small-molecule inhibitor targeting spleen tyrosine kinase |
Hypertension and severe diarrhea |
Completed its phase I study in healthy volunteers in Australia; no material off-target toxicities were observed and it exhibited a linear pharmacokinetic profile and a dose-dependent suppression of B-cell activation |
11/2/15 |
INFECTION | |||||
Alios Biopharma Inc. (South San Francisco) |
ALS-008176 |
Cytidine nucleoside analogue |
Respiratory syncytial virus |
In a randomized, double-blind study, 62 healthy volunteers were inoculated with RSV and randomized to ALS-008176 or placebo, in which the treatment resulted in a greater reduction (73% to 88% under the curve) of viral load and faster viral clearance compared to placebo |
11/20/15 |
Arsanis Inc. (Waltham, Mass.) |
ASN100 |
Combination of two monoclonal antibodies |
Staphylococcus aureus |
The first cohort was initiated in the phase I trial |
11/24/15 |
Cidara Therapeutics Inc. (San Diego) |
CD101 |
Intravenous therapy |
Candidemia |
Proved well tolerated systemically and at infusion sites across entire dose ranges during a phase I single ascending-dose study in healthy volunteers |
11/18/15 |
Nitto Denko Corp. (Osaka, Japan) |
ND-L02-s0201 |
Targeted siRNA lipid nanoparticle |
Non-alcoholic steatohepatitis and hepatitis C virus liver fibrosis |
Phase Ib/II studies showed it was well tolerated, with histological improvement of fibrosis observed |
11/23/15 |
Scynexis Inc. (Jersey City, N.J.) |
SCY-078 |
Oral glucan synthase inhibitor |
Invasive and life-threatening fungal infections |
Started a phase I trial |
11/13/15 |
Transgene SA (Paris) |
TG1050 |
Immunotherapy candidate |
Chronic hepatitis B virus infection |
The first patient was dosed in a phase I/Ib study |
11/5/15 |
INFLAMMATORY | |||||
Complexa Inc. (Pittsburgh) |
CXA-10 |
Endogenous nitro-fatty acid modulator of Nrf2 and NF-κB |
Inflammation and fibrosis |
Completed an extensive phase I development program |
11/13/15 |
Galapagos NV (Mechelen, Belgium) |
GLPG1972 |
First-in-class candidate |
Osteoarthritis |
Began dosing in a phase I study |
11/23/15 |
MISCELLANEOUS | |||||
Allergan plc (Dublin) |
Bimatoprost |
Sustained-release |
Glaucoma |
Six-month interim results from a 24-month phase I/II trial of 75 patients showed that a single dose lowered intraocular pressure in 92% of patients at four months and 71% at six months while demonstrating a favorable efficacy and safety profile |
11/17/15 |
Arqule Inc. (Burlington, Mass.) |
ARQ 092 |
Orally available, small molecule multikinase inhibitor |
Proteus syndrome |
Enrolled the first patient in the first clinical trial |
11/18/15 |
Biolinerx Ltd. (Tel Aviv, Israel) |
BL-8040 |
Synthetic peptide that functions as a high-affinity antagonist of CXCR4 |
Bone marrow failure conditions hypoplastic myelodysplastic syndrome (hMDS) and aplastic anemia (AA) |
Started a phase I/II trial |
11/24/15 |
Bluebird Bio Inc. (Cambridge, Mass.) |
Lentiglobin BB305 |
Gene therapy |
Beta-thalassemia |
Company reported that transfusion independence was achieved in patients with beta-thalassemia major with non-beta-zero genotypes followed for at least six months in the Northstar and HGB-205 studies |
11/6/15 |
Eyegate Pharmaceuticals Inc. (Waltham, Mass.) |
EGP-437 |
A combination of reformulated corticosteroid dexamethasone phosphate delivered using the company's Eyegate II delivery system |
Macular edema |
Interim data from an ongoing phase Ib/IIa pilot trial showed positive responses in some of the patients, with pseudophakic eyes; the therapy also showed no serious treatment-emergent adverse effects, including no increase in ocular pressure even at three times the iontophoretic dose |
11/6/15 |
Ix Biopharma Ltd. (Singapore) |
Pheonix |
Sildenafil |
Erectile dysfunction |
Completed a pilot bioavailability study comparing the pharmacokinetics of the sildenafil in Pheonix with that in Viagra (Pfizer Inc.), demonstrating Pheonix has a similar rate and extent of drug absorption as a Viagra tablet |
11/30/15 |
Kadmon Corp. LLC (New York) |
Tesevatinib |
Oral tyrosine kinase inhibitor |
Autosomal dominant polycystic kidney disease |
Phase Ib/IIa data demonstrated the tolerability of tesevatinib at 50 mg, 100 mg and 150 mg QD, with rash occurring in the 150-mg QD dose cohort; the phase IIa portion evaluated tesevatinib 150 mg administered twice or three times weekly and found that the tolerability of the intermittent dosing schedules was improved over 150-mg QD, but rash still occurred |
11/3/15 |
Miragen Therapeutics Inc. (Boulder, Colo.) |
MRG-201 |
Synthetic microRNA mimic (promiR) to microRNA-29b |
Cutaneous scleroderma |
Initiated a phase I study |
11/13/15 |
Novabay Pharmaceuticals Inc. (Emeryville, Calif.) |
NVC-422 |
Antimicrobial compound auriclosene |
To reduce urinary catheter blockage and encrustation |
Completed a 34-patient study evaluating it in patients with long-term indwelling urinary catheters |
11/5/15 |
Panoptica Inc. (Bernardsville, N.J.) |
PAN-90806 |
Topical, anti-VEGF eye drop |
Neovascular age-related macular degeneration |
Preliminary results from the monotherapy arm of a phase I/II study showed signals of safety and anti-VEGF biological activity |
11/13/15 |
Prometic Life Sciences Inc. (Laval, Quebec) |
Plasma-derived plasminogen |
Replacement therapy |
Plasminogen-deficiency |
Phase I therapy was used to treat an infant in critical condition in an intensive care unit at the Altona Children's Hospital in Hamburg, Germany; the administration of plasma was not successful in raising his plasminogen to an effective level, so the company's plasminogen was provided and within a few days, physicians observed reduction of the lesions |
11/5/15 |
Retrotope Inc. (Los Altos, Calif.) |
RT001 |
Designed as a stabilized fatty-acid aimed at shutting down the degradation of lipids in mitochondrial and cellular membranes |
Friedreich's ataxia |
Opened the second site for the ongoing, 28-day phase I/II study, testing ascending doses of orally dosed RT001 |
11/23/15 |
Rxi Pharmaceuticals Corp. (Marlborough, Mass.) |
RXI-109 |
An sd-rxRNA |
Retinal scarring |
Initiated a phase I/II trial |
11/6/15 |
RESPIRATORY | |||||
Concert Pharmaceuticals Inc. (Lexington, Mass.) |
CTP-656 |
Deuterium-modified ivacaftor |
Cystic fibrosis |
Began a phase I multiple ascending-dose trial |
11/13/15 |
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. For more information about individual companies and/or products, see Thomson Reuters Cortellis. |