Company (location) |
Product |
Description |
Indication |
Status |
Date |
Cancer | |||||
Affimed NV (Heidelberg, Germany) |
AFM-13 |
Tetravalent, bispecific NK cell engager |
Hodgkin lymphoma (HL) and CD30-positive lymphoma |
Data from two separate studies showed that it was well-tolerated and showed promising therapeutic efficacy both in combination with the anti-PD-1 antibody Keytruda in HL and as monotherapy in CD30-positive lymphoma; the combo trial showed an objective response rate (ORR) of 89%, including complete metabolic responses in 44% and partial metabolic responses in 44% of patients with one patient experiencing stable disease (SD); the monotherapy trial demonstrated that it could be safely administered, showed therapeutic activity as a single agent with an ORR of 66% (2/3), and one CR, one PR and one SD were observed |
2/2/18 |
Aravive Biologics Inc. (Houston) |
AVB-S6-500 |
GAS6 inhibitor |
Cancer |
Started a phase I trial in 40 healthy volunteers |
2/16/18 |
Bayer AG (Leverkusen, Germany) and Loxo Oncology Inc. (Stamford, Conn.) |
Larotrectinib |
Directly targets TRK |
Tumors with neurotrophic tyrosine receptor kinase (NTRK) gene fusions |
Data from its phase I adult trial, the phase II Navigate trial and the phase I/II pediatric Scout trial showed it demonstrated an overall response rate of 75% by central assessment and 80% by investigator assessment; at the time of analysis, 86% of responding patients remained on it or underwent surgery with curative intent; the median duration of response and progression-free survival had not been reached |
2/23/18 |
Beigene Ltd. (Cambridge, Mass.) |
Tislelizumab |
Anti-PD-1 antibody |
Urothelial carcinoma (UC) |
Phase I data suggest that it was generally well-tolerated and exhibited objective responses; 15 patients were evaluable; one patient had a confirmed complete response (CR), four achieved a confirmed partial response (PR), and three achieved stable disease (SD); nine evaluable patients had PD-L1 status determined; there was one CR, two PRs and one SD among six PD-L1-high patients, and one PR among three PD-L1-low or negative patients |
2/12/18 |
Briacell Therapeutics Corp. (Berkeley, Calif.) |
Bria-IMT |
Releases granulocyte-macrophage colony-stimulating factor |
Breast cancer |
Phase I/IIa data showed the regimen was well-tolerated with few side effects in the six patients who received inoculations; imaging studies on the first fully evaluable patient in the trial showed a mixed response, including "a clear response in multiple bilateral pulmonary nodules," although there was evidence of progressive disease at other sites |
2/1/18 |
Calithera Biosciences Inc. (South San Francisco) |
CB-839 |
Inhibitor of glutaminase |
Renal cell carcinoma (RCC) |
Phase I results of it in combination with Cabometyx produced a 40% overall response rate and 100% disease control in 12 evaluable patients with advanced clear cell RCC, and two additional patients with papillary RCC had disease control as well; updated results of it in combination with Afinitor showed disease control in 92% of the 24 evaluable RCC patients, including one patient with a partial response and 21 patients with stable disease, and median progression-free survival of 5.8 months |
2/7/18 |
Cancer Research UK (London) and Bicycle Therapeutics Ltd. (Cambridge, U.K.) |
BT-1718 |
Targets membrane type 1 matrix metalloproteinase |
Advanced solid tumors |
The first patient was dosed in their phase I/IIa trial |
2/14/18 |
Cytrx Corp. (Los Angeles) and Nantcell Inc. (Culver City, Calif., subsidiary of Nantworks LLC) |
Aldoxorubicin |
Cytotoxic delivering doxorubicin |
Advanced squamous cell carcinoma of either head and neck or non-small-cell lung cancer |
Dosed the first patient in the phase Ib portion of a phase Ib/II trial |
2/6/18 |
Daiichi Sankyo Co. Ltd. (Tokyo) |
U3-1402 |
HER3-targeting antibody-drug conjugate |
Metastatic or unresectable EGFR-mutated non-small-cell lung cancer |
The first patient was dosed in a phase I study |
2/7/18 |
Daiichi Sankyo Co. Ltd. (Tokyo) |
DS-1062 |
Trophoblast cell-surface antigen 2 (TROP2)-targeting antibody-drug conjugate |
Unresectable advanced non-small-cell lung cancer |
The first patient was dosed in a phase I study |
2/23/18 |
Exelixis Inc. (South San Francisco) |
Cabometyx |
Cabozantinib |
Refractory genitourinary tumors |
Expansion cohorts in a phase I trial of it in combination with either Opdivo or Opdivo plus Yervoy showed: 19 patients with metastatic urothelial carcinoma (mUC) were evaluable for response with a median follow-up of 15.7 months, with an overall response rate (ORR) of 42% (two complete responses [CRs] and six partial responses [PRs]) and disease control rate of 84%; seven of eight (88%) mUC patients with an objective response had not progressed at the time of the data cut-off; median progression-free survival in that patient population was 12.8 months, and the overall survival rate at 12 months was 77%; in the overall study, the ORR in 64 evaluable patients was 36% (three CRs and 20 PRs), with a median duration of response of 24 months |
2/8/18 |
Fate Therapeutics Inc. (San Diego) |
FATE-NK100 |
Allogeneic natural killer cell cancer immunotherapy |
Advanced solid tumors |
Treated the first patient in the phase I DIMENSION trial |
2/21/18 |
Gamida Cell Ltd. (Jerusalem) |
Nicord |
Universal bone marrow transplant solution |
High-risk hematologic malignancies |
A phase I/II trial met its primary endpoint; participants experienced rapid and durable neutrophil engraftment at a median of 11 days, representing an improvement over historical standard cord blood transplantation as well as compared to a database of patients from the Center for International Blood and Marrow Transplant Research; it demonstrated an acceptable safety profile, with moderate/severe chronic graft-vs.-host disease in 9.8% of patients at one year following transplantation |
2/21/18 |
Immunicum AB (Gothenburg, Sweden) |
Ilixadencel |
Dendritic cell therapy |
Hepatocellular carcinoma |
A phase I/II case study of a patient with bile duct cancer who was treated with it intratumorally after transarterial chemo-embolization treatment with doxorubicin showed delayed progression of the tumor until month six; the patient then received gemcitabine plus cisplatin and lived for 41 months, substantially longer than historical controls |
2/6/18 |
Immutep Ltd. (Sydney) |
LAG-3Ig or IMP-321 |
Eftilagimod alpha |
Unresectable or metastatic melanoma |
In the TACTI-mel (Two ACTive Immunotherapies in melanoma) phase I trial, the database safety monitoring board confirmed it is safe and well-tolerated in combination with Keytruda at doses up to 30 mg per subcutaneous injection |
2/12/18 |
Intensity Therapeutics Inc. (Westport, Conn.) |
INT-230-6 |
Amphiphilic cell penetration enhancer molecule with cisplatin and vinblastine |
Ovarian, thyroid, head and neck or skin cancers |
Data from cohort A of its phase I/II trial showed there were no dose-limiting toxicities; treatment was started in cohort B1 |
2/28/18 |
Kancera AB (Stockholm, Sweden) |
KAND-567 |
Targets CX3CR1 |
Lymphoma and cardiovascular disease |
Data from a phase I trial testing it in healthy volunteers showed it was safe and well-tolerated at up to 500 mg twice daily for seven days, with plasma levels five to 10 times higher than the calculated effective level for a therapeutic effect in humans |
2/21/18 |
Mabvax Therapeutics Holdings Inc. (San Diego) |
MVT-5873 |
Monoclonal antibody that targets an epitope on CA19-9 called sialyl Lewis A |
Pancreatic cancer |
Interim phase I data from the expanded cohort testing it in combination with first-line chemotherapy showed all six patients had measurable tumor reductions, including four partial responses and two patients with stable disease |
2/13/18 |
MEI Pharma Inc. (San Diego) |
ME-344 |
Novel mitochondrial inhibitor |
HER2-negative breast cancer |
Interim data from the first 20 patients in a study testing it in combination with Avastin justifies completing enrollment of the expected 40 total patients |
2/14/18 |
Nantkwest Inc. (Culver City, Calif.) |
HER2.taNK |
Natural killer cell-based immuno-oncology therapy |
HER2-positive glioblastoma |
Treated the first patient in a phase I trial |
2/14/18 |
Oasmia Pharmaceutical AB (Uppsala, Sweden) |
Docecal |
Nanoparticle formulation of docetaxel |
Metastatic breast cancer |
Completed treatment of 228 patients in a pharmacokinetic crossover clinical phase I study and a randomized clinical study comparing it to Taxotere |
2/21/18 |
Oncoceutics Inc. (Philadelphia) |
ONC-201 |
Small-molecule DRD2 antagonist |
Brain tumors containing the histone H3 K27M mutation |
The first patient was treated in a trial of pediatric patients |
2/1/18 |
Oncopep Inc. (Boston) |
PVX-410 |
Multipeptide therapeutic cancer vaccine |
Metastatic triple-negative breast cancer, human leukocyte antigen A2-positive |
Started a phase Ib trial |
2/23/18 |
Orano Med (Courbevoie, France) and Radiomedix Inc. (Houston) |
Alphamedix |
Radiolabeled SSTR-targeting therapeutic |
Somatostatin receptor-positive neuroendocrine tumors |
Dosed the first three patients in the phase I trial |
2/21/18 |
Panacea Pharmaceuticals Inc. (Gaithersburg, Md.) |
PAN-301-1 |
Nanoparticle immunotherapy vaccine |
Persistent prostate cancer |
Completed enrollment of 12 patients in a phase I trial; it has been well-tolerated with a highly favorable safety profile across all dose levels with no drug-related adverse events or dose-limiting toxicities observed; all subjects experienced dose-dependent HAAH-specific immune responses, including antibody levels in immunoassays and B-cell and T-cell responses, and a majority of subjects treated beyond three months achieved a reduction in prostate-specific antigen doubling rate and are receiving continued doses |
2/2/18 |
Redx Pharma Ltd. (Alderley Park, U.K.) |
RXC-004 |
Porcupine inhibitor |
Cancer |
Started a phase I/IIa study |
2/7/18 |
Scancell Holdings plc (Nottingham, U.K.) |
SCIB1 |
Cancer vaccine |
Melanoma |
Phase I/II data showed that of the 20 stage III/IV patients, 18 were alive as of the February 2018 cutoff; six of the 16 resected patients who received 2-4 mg doses had recurrence of their disease with two deaths; of the 14 surviving patients, all have lived for at least five years |
2/13/18 |
Sosei Group Corp. (Tokyo) and Astrazeneca plc (Cambridge U.K.) |
AZD-4635 |
Selective adenosine A2A receptor antagonist |
Advanced solid tumors |
Dosed the first patient in the phase Ib portion of a trial testing alone and in combination with durvalumab |
2/9/18 |
Targovax ASA (Oslo, Norway) |
ONCOS-102 |
Virus-based oncolytic immunotherapy |
Mesothelioma |
Completed the safety lead-in cohort and preliminary immune activation data in the phase I/II trial |
2/8/18 |
TG Therapeutics Inc. (New York) |
TGR-1202 |
Umbralisib |
Relapsed or refractory B-cell malignancies |
Phase I data showed that in patients with relapsed or refractory chronic lymphocytic leukemia, it produced an 85% objective response rate (ORR), including 50% per International Workshop on Chronic Lymphocytic Leukemia criteria and 35% partial response with lymphocytosis; in patients with relapsed or refractory follicular lymphoma, it produced a 53% ORR |
2/22/18 |
Trovagene Inc. (San Diego) |
PCM-075 |
Oral PLK1-selective adenosine triphosphate competitive inhibitor |
Acute myeloid leukemia |
The first patient completed the first cycle of dosing in combination with low-dose cytarabine in its phase Ib/II trial |
2/7/18 |
Xencor Inc. (Monrovia, Calif.) |
Xmab-18087 |
Bispecific antibody targeting SSTR2 and CD3 |
Neuroendocrine tumors and gastrointestinal stromal tumors |
Treated the first patient in a phase I trial |
2/23/18 |
Cardiovascular | |||||
Berlin Cures Holding AG (Zug, Switzerland) |
BC-007 |
DNA aptamer-based compound |
Dilated cardiomyopathy |
Phase I data showed that a single intravenous dose is able to eliminate autoantibodies targeting the beta-1 adrenoceptor completely and sustainably; it was well-tolerated and did not provoke any clinically relevant side effects |
2/28/18 |
Longeveron LLC (Miami |
MSC |
Mesenchymal stem cell therapy |
Hypoplastic left heart syndrome |
The first infant received treatment |
2/28/18 |
Myokardia Inc. (South San Francisco) |
MYK-491 |
Allosteric activator of myosin |
Dilated cardiomyopathy |
Dosing has begun for a phase Ib trial |
2/15/18 |
Dermatologic | |||||
Galapagos NV (Mechelen, Belgium) and Morphosys AG (Planegg, Germany) |
MOR-106 |
IL-17C antibody |
Atopic dermatitis (AD) |
In the phase I study, it showed first signs of activity and durable responses and was generally well-tolerated; at the highest dose level (10 mg/kg body weight), in 83% of patients an improvement of at least 50% in signs and extent of AD, as measured by EASI-50, was recorded at week four; onset of activity occurred within two to four weeks, depending on the dose administered |
2/21/18 |
Mallinckrodt plc (Staines-upon-Thames, U.K.) |
Expressgraft C9T1 |
Skin tissue |
Diabetic foot ulcers |
Enrolled the first patient in a phase I study |
2/15/18 |
Sienna Biopharma-ceuticals Inc. (Westlake Village, Calif.) |
SNA-125 |
Inhibits janus kinase 3 (JAK3) and tropomyosin receptor kinase A (TrkA) |
Psoriasis and associated pruritus |
The first patient was dosed in a phase I/II study |
2/15/18 |
Endocrine/Metabolic | |||||
Abeona Therapeutics Inc. (Cleveland) |
ABO-101 |
Adeno-associated viral-based gene therapy |
Mucopoly-saccharidosis IIIB |
Preliminary 30-day safety and biopotency signals from the first patient dosed in its phase I/II trial showed it was well-tolerated with no treatment-related adverse events or serious adverse events through 30 days of follow-up; significant heparan sulfate reductions were observed in cerebral spinal fluid (50%), urine (69%), plasma (60%) and urinary total glycosaminoglycan (67%); normalized NAGLU enzyme activity was observed |
2/8/18 |
Abeona Therapeutics Inc. (Cleveland) |
ABO-102 |
Adeno-associated viral-based gene therapy |
Mucopoly-saccharidosis IIIA |
The FDA allowed it to lower the enrollment age in the phase I/II trial to include subjects as young as 6 months |
2/12/18 |
Amicus Therapeutics Inc. (Cranbury, N.J.) |
ATB-200/AT-2221 |
Enzyme replacement therapy (ERT) |
Pompe disease |
Phase I/II data showed that of the eight ERT-switch patients, all eight increased their six-minute walk test at month 12 with a mean increase of 57.4 meters; both ERT-naïve patients treated for 12 months increased their six-minute walk test by a median of 86.8 meters; biomarkers of muscle damage (creatine kinase) and disease substrate (urine hexose tetrasaccharide) improved at month 12 |
2/9/18 |
Applied Therapeutics Inc. (New York) |
AT-001 |
Oral small molecule |
Diabetic complications |
Initiated a phase I trial |
2/13/18 |
Audentes Therapeutics Inc. (San Francisco) |
AT-342 |
AAV8 vector containing a functional copy of the UGT1A1 gene |
Crigler-Najjar syndrome |
Initiated dosing in VALENS, a phase I/II trial |
2/13/18 |
Biomarin Pharmaceutical Inc (San Rafael, Calif.) |
BMN-250 |
Enzyme replacement therapy |
Sanfilippo B syndrome |
Interim phase I/II data showed all three treated part one patients experienced decreases in liver size into the normal range for age |
2/8/18 |
Genethon (Evry, France) |
Adeno-associated viral vector |
Gene therapy |
Crigler-Najjar syndrome |
Started a European phase I/II trial |
2/13/18 |
Sangamo Therapeutics Inc. (Richmond, Calif.) |
SB-913 |
Intravenously delivered zinc finger nuclease (ZFN) therapeutic |
Hunter syndrome |
Safety data from the phase I/II CHAMPIONS study showed the first patient treated appeared to be tolerating the infusion well after the first six weeks, and liver function tests remained within normal limits; a second patient was treated in the study in mid-January |
2/8/18 |
Saniona AB (Copenhagen) |
Tesomet |
Tesofensine and metoprolol |
Type 2 diabetes |
Completed recruitment in its phase I pharmacokinetic study |
2/8/18 |
Sanofi SA (Paris) |
Avalglucosidase alfa |
NeoGAA |
Late-onset Pompe disease |
Safety data from the phase I/II NEO1 trial and the NEO-EXT extension trial showed that of the patients who completed NEO1, 19 of 24 patients entered the NEO-EXT study and 18 are still enrolled; the one discontinuation occurred for personal reasons; treatment-emergent adverse events were mostly mild across both studies |
2/9/18 |
Valbiotis SAS, (La Rochelle, France) |
VAL-070 |
Dietary supplement containing active plant extracts |
Mild to moderate dyslipidemia |
Completed recruitment for a phase I/II study |
2/28/18 |
Gastrointestinal | |||||
Palatin Technologies Inc. (Cranbury, N.J.) |
PL-8177 |
Selective melanocortin receptor 1 agonist peptide |
Ulcerative colitis and inflammatory bowel diseases |
Dosed the first healthy subject in its phase I trial |
2/6/18 |
Genitourinary/Sexual Health | |||||
Contravir Pharmaceuticals Inc. (Edison, N.J.) |
Txl |
Delivers high intrahepatic concentrations of tenofovir |
Severe renal impairment |
Phase I showed it was safe and well-tolerated in eight healthy subjects and eight patients; the blood concentrations of tenofovir in the renally impaired patients treated with it was similar to exposure levels after treatment with Viread, suggesting dosing adjustments won't be required |
2/7/18 |
Hematologic | |||||
Acticor Biotech SAS (Paris) |
ACT-017 |
Humanized monoclonal antibody fragment directed against a platelet glycoprotein (GPVI) |
Thrombosis |
Completed a phase I trial in healthy volunteers; the primary endpoint of safety and tolerability showed no serious adverse events reported at any doses tested; the maximum tolerated dose was not reached, though the complete inhibition of collagen-induced platelet aggregation was observed, meaning that an acceptable phase II recommended dose was obtained |
2/6/18 |
Catalyst Biosciences Inc. (South San Francisco) |
CB-2679d/ISU-304 |
Subcutaneous (SQ) prophylactic |
Hemophilia B |
Phase I/II data showed a continuous linear increase in factor IX (FIX) activity levels following daily dosing for six days; it increased FIX activity levels in five patients who were each dosed daily with 140 IU/kg SQ, from very low levels after washout of prior therapy to a median FIX activity level of 16% (range 11.5% to 18%); the observed increase in FIX activity levels after the daily dosing was linear |
2/12/18 |
Gamida Cell Ltd. (Jerusalem) |
Nicord |
Universal bone marrow transplant solution |
Sickle cell disease |
Results from a phase I/II sickle cell disease study showed rapid engraftment observed in all 13 patients at a median of seven days, with 11 of 13 patients (84.6%) alive at a median follow-up of 22 months |
2/21/18 |
Immune | |||||
Bellicum Pharmaceuticals, Inc. (Houston) |
BPX-501 |
Rivogenlecleucel |
Graft vs. host disease after hematopoietic stem cell transplants |
The FDA stopped three U.S.-based trials after three cases of encephalopathy possibly related to the experimental therapy cropped up; a European trial is unaffected |
2/1/18 |
Bellicum Pharmaceuticals, Inc. (Houston) |
BPX-501 |
Rivogenlecleucel |
Graft vs. host disease after hematopoietic stem cell transplants |
Received word from the FDA on what will be required to lift a clinical hold on U.S. studies; the company said it plans to revise U.S. study protocols, including the addition of more comprehensive monitoring and management of neurotoxicity; it will also revise the study's investigator brochure and informed consent documents to communicate the changes |
2/26/18 |
Cynata Therapeutics Ltd. (Melbourne, Australia) |
CYP-001 |
Cymerus mesenchymal stem cell product |
Steroid-resistant graft-vs. host disease (GvHD) |
Data from the first cohort of eight patients in its phase I trial showed that the overall response rate by day 100 was 100%, with all patients showing an improvement in the severity of GvHD by at least one grade compared to baseline; half of the patients had a complete response by day 100 and 87.5% were alive on day 100 |
2/28/18 |
Herantis Pharma plc (Espoo, Finland) |
Lymfactin |
Gene therapy expressing VEGF-C |
Breast cancer-associated lymphedema |
Completed enrollment in a phase I trial testing safety and tolerability |
2/9/18 |
Therachon AG (Basel, Switzerland) |
TA-46 |
Soluble recombinant human fibroblast growth factor receptor ligand trap |
Achondroplasia |
The first subject in its phase I trial was dosed |
2/15/18 |
Infection | |||||
Arch Biopartners Inc. (Toronto) |
AB-569 |
Ethylenediamine-tetraacetic acid and sodium nitrite |
Antibiotic-resistant bacterial infections |
Began to recruit and enroll healthy volunteers in a phase I trial |
2/14/18 |
Emergent Biosolutions Inc. (Gaithersburg, Md.) and Valneva SE (Lyon, France) |
VLA-1601 |
Vaccine |
Zika virus |
Started a phase I trial in healthy adults |
2/28/18 |
Eurocine Vaccines AB (Solna, Sweden) |
Immunose Flu |
Quadrivalent nasal influenza vaccine |
Influenza |
Started a phase I/II trial in adults, ages 50 to 75 |
2/8/18 |
Hemispherx Biopharma Inc. (Banff, Alberta) |
Ampligen |
Rintatolimod |
Influenza |
A phase I/II trial testing it following treatment with Flumist in 12 healthy volunteers showed the combination produced secretory IgA antibody responses of at least fourfold over baseline against at least one of the vaccine's homologous vaccine strains in 92% of the subjects; IgA antibodies against highly pathogenic avian influenza strains H5N1, H7N9 and H7N3 were observed |
2/1/18 |
Immuron Ltd. (Melbourne, Australia) |
IMM-529 |
Polyclonal antibody combination |
Clostridium difficile infection |
Enrolled the first patients in a phase I/II trial |
2/1/18 |
Vyera Pharmaceuticals LLC (New York) |
TUR-006 |
Dihydrofolate reductase inhibitor |
Toxoplasmosis |
Started a phase I single ascending-dose study |
2/12/18 |
Wuxi Biologics (Shanghai) and Tychan Pte Ltd. (Singapore) |
Tyzivumab |
Zika antibody |
Zika virus |
Dosed the first patient in a phase Ia trial in Singapore |
2/12/18 |
Inflammatory | |||||
Corbus Pharmaceuticals Holdings Inc. (Norwood, Mass.) |
Lenabasum |
Selective cannabinoid receptor type 2 (CB2) agonist |
Systemic sclerosis, cystic fibrosis, dermatomyositis and systemic lupus erythematosus |
Study data showed treatment with 5 mg or 20 mg twice daily inhibited the accumulation of neutrophils at the infected site; it also enhanced clearance of the injected bacteria |
2/16/18 |
Musculoskeletal | |||||
Spinalcyte LLC (Houston) |
Cybrocell |
Allogeneic human dermal fibroblast product |
Degenerative disc disease |
Phase I/II data showed approximately 70% of patients treated with it reported therapeutic improvement, including increase in regeneration, disc height, gene expression of structural genes such as collagen type I and collagen type II and the contents of structural proteins such as proteoglycan, which in turn generate the disc nucleus that provides cushioning for the spine |
2/14/18 |
Neurology/Psychiatric | |||||
Axsome Therapeutics Inc. (New York) |
AXS-09 |
Esbupropion and dextromethorphan |
CNS disorders |
Phase I data showed that it substantially increased dextromethorphan plasma concentrations, the primary endpoint, into a potentially therapeutic range with repeated dosing (p<0.0001 day one vs. day eight); it was well-tolerated with no serious adverse events reported |
2/27/18 |
Gensight Biologics SA (Paris) |
GS-010 |
rAAV2/2-ND4 gene therapy |
Leber hereditary optic neuropathy (LHON) |
Phase I/II data showed it safe and well-tolerated 96 weeks after treatment |
2/21/18 |
Impel Neuropharma Inc. (Seattle) |
INP-104 |
Intranasal version of dihydroergotamine dosed via Precision Olfactory Deliver device |
Acute migraine |
Phase I data showed a statistically significant improvement in bioavailability (Cmax and AUC) of it over a currently approved dihydroergotamine mesylate nasal spray; that bioavailability was comparable to DHE 45 (dihydroergotamine mesylate) intravenous injection after 20 minutes of exposure |
2/8/18 |
Sage Therapeutics Inc. (Cambridge, Mass.) |
SAGE-217 |
Positive allosteric modulator targeting synaptic and extrasynaptic GABA receptors |
Sleep disorders |
A phase I/II study of healthy adult volunteers showed it significantly improved sleep efficiency (SE), the primary endpoint of the trial, to a median of 85% (30 mg; p<0.0001) and 88% (45 mg; p<0.0001), respectively, compared with a median SE of 73% for placebo; it also demonstrated statistically significant improvements in total sleep time as well as sleep maintenance; it was generally well-tolerated |
2/1/18 |
Xenon Pharmaceuticals Inc. (Burnaby, British Columbia) |
XEN-901 |
Oral, small-molecule Nav1.6 sodium channel inhibitor |
Epilepsy |
Started a phase I trial |
2/21/18 |
Ocular | |||||
Regenxbio Inc. (Rockville, Md.) |
RGX-314 |
Includes the NAV AAV8 vector encoding an antibody fragment which inhibits VEGF |
Wet age-related macular degeneration |
Completed dosing of the third cohort of six patients in a phase I trial |
2/12/18 |
Santen Inc. (Emeryville, Calif.) |
DE-122 |
Carotuximab |
Refractory wet age-related macular degeneration |
Phase I/II data showed it was well tolerated with no serious adverse events reported; there were signs of its bioactivity as measured by mean change in central retinal subfield thickness based on the spectral domain optical coherence tomography |
2/13/18 |
Other/Miscellaneous | |||||
Adverum Biotechnologies Inc (Menlo Park, Calif.) |
ADVM-043 |
Gene therapy |
Alpha-1 antitrypsin deficiency |
Completed dosing and evaluation of patients (n=2) in the first cohort of the ADVANCE phase I/II trial |
2/27/18 |
Respiratory | |||||
Pulmatrix Inc. (Lexington, Mass.) |
Pulmazole |
Itraconazole |
Allergic bronchopulmonary aspergillosis in patients with asthma |
Treated the first subject in a phase I/Ib trial |
2/13/18 |
Notes The date indicated refers to the BioWorld issue in which the news item can be found. For more information about individual companies and/or products, see Cortellis. |