Company (location) |
Product |
Description |
Indication |
Status |
Date |
Autoimmune | |||||
Atara Biotherapeutics Inc. (South San Francisco) |
ATA-188 |
T-cell immunotherapy |
Progressive or relapsing-remitting multiple |
Started a phase I study |
10/20/17 |
Atara Biotherapeutics Inc. (South San Francisco) |
ATA-190 |
Epstein-Barr virus (EBV) specific |
Multiple sclerosis (MS) |
Updated positive results for an ongoing phase I study showed six of 10 progressive MS patients experienced clinical improvements, which were first observed 2-14 weeks after the initial infusion; clinical improvements were reported in primary and secondary progressive MS patients from an established level of disability; reduction in fatigue was a consistent observation in responding patients; a correlation between clinical improvement and the reactivity of it against target EBV antigens (EBV reactivity) was also observed |
10/26/17 |
Enterome SA (Paris) |
EB-8018 |
Small molecule designed to block FimH |
Crohn's disease |
Completed the first phase I study; it was well-tolerated in healthy volunteers across a range of doses and showed a pharmacokinetic profile consistent with its design as a gut-restricted molecule that is soluble in the gut; it also exhibited minimal absorption into the blood, potentially limiting interactions with other drugs absorbed into the bloodstream |
10/30/17 |
Melinta Therapeutics Inc. (New Haven, Conn.) |
Radezolid |
Topical |
Mild to moderate acne vulgaris |
Phase I study was completed and results |
10/11/17 |
Prothena Corp. plc ( Dublin) |
PRX003 |
Monoclonal |
Psoriasis |
Results from a phase Ib multiple ascending dose trial testing 33 patients didn't produce meaningful clinical benefit as measured by responses in the Psoriasis Area and Severity Index 75; skin biopsies showed that it insufficiently reduced Th17 cell infiltration and other inflammatory markers; the company doesn't plan to move it into mid-stage development |
10/2/17 |
Cancer | |||||
Aduro Biotech Inc. (Berkeley, Calif.) |
JNJ-64041757 (ADU-214) |
Live, attenuated double-deleted Listeria |
Advanced or |
Initial data from a phase I dose-finding trial showed that of the nine patients treated with it (administered either 1x10^8 or 1x10^9 colony-forming units infused intravenously over one hour every 21 days), five experienced a best response of stable disease; biomarker data showed evidence of activation of innate immunity with transient cytokine increases in all patients as well as induction of mesothelin-specific T-cell immunity in a subset of patients |
10/18/17 |
Aprea Therapeutics AB (Stockholm) |
APR-246 |
Targets p53 tumor suppressor |
Platinum-resistant advanced and metastatic |
Enrolled the first patient in a phase Ib/II trial testing it in combination with cisplatin and 5-fluorouracil |
10/17/17 |
Atossa Genetics Inc. (Seattle) |
Endoxifen |
Active metabolite of tamoxifen |
Breast cancer |
Preliminary results from its phase I study showed all objectives being successfully met; there were no clinically significant safety signals and no clinically significant adverse events in participants receiving the compound |
10/26/17 |
Bergenbio ASA (Bergen, Norway) |
BGB324 |
Small molecule AXL kinase inhibitor |
Advanced |
Safety data from the phase Ib/II trial testing it in combination with either the MAP kinase inhibitors Mekinst plus Tafinlar or the immune checkpoint inhibitor Keytruda showed the recommended dose for it plus Mekinst and Tafinlar has been established and both sets of combinations were well tolerated |
10/19/17 |
Biohaven Pharmaceutical Holding Co. Ltd. (New Haven, Conn.) |
Trigriluzole |
Glutamate |
Inoperable, advanced or refractory cancers |
A phase 1 trial began enrollment and the first patient has started treatment |
10/31/17 |
Biolinerx Ltd. (Tel Aviv, Israel) |
BL-8040 |
CXCR4 antagonist |
Gastric cancer |
Partner Genentech Inc. (South San Francisco) started a phase Ib/II trial testing it in combination with atezolizumab |
10/19/17 |
Bluebird Bio Inc. (Cambridge, Mass.) |
bb21217 |
Chimeric antigen receptor T-cell |
Relapsed/ |
Treated the first patient in its phase I CRB-402 trial |
10/2/17 |
Bristol-Myers Squibb Co. (New York) |
Opdivo |
Nivolumab |
Previously |
An exploratory analysis of the effect of tumor mutation burden (TMB) on objective response rate (ORR) from the phase I/II CheckMate -032 showed patients with high TMB who received it plus Yervoy had an ORR of 46% while the ORR was 16% and 22% in patients with medium and low levels of TMB, respectively; for patients with high TMB who only received Opdivo, the ORR was 21%, compared to 7% and 5%, in patients with medium and low levels of TMB, respectively |
10/17/17 |
BTG plc (London) |
BTG-002814 |
Vandetanib-eluting radiopaque beads |
Primary liver |
Started the first phase I trial |
10/10/17 |
Calithera BioSciences Inc. (South San Francisco) and Incyte Corp. (Wilmington, Del.) |
INCB-01158 |
Arginase inhibitor |
Advanced non-small-cell lung cancer, colorectal cancer and other solid tumors |
The first patient was treated in an expansion cohort of a phase I trial testing it in combination with Keytruda |
10/20/17 |
Cantargia AB (Stockholm) |
CAN-04 |
Antibody treatment targeting IL1RAP |
Non-small-cell lung cancer, |
The first patient in the phase I/IIa CANFOUR trial has received three cycles of treatment with it, and two additional patients have received therapy with it; no serious adverse events have been recorded |
10/16/17 |
Cellectar Biosciences Inc (Madison, Wis.) |
CLR-131 |
Phospholipid |
Relapsed or refractory |
The fifth cohort of its phase I dose escalation safety trial will use two 15.625 mCI/m2 doses one week apart with the combined dose equaling 31.25 mCi/m2 – the same dose that resulted in a partial response in patients who participated in cohort 4 |
10/25/17 |
Celsion Corp. (Lawrenceville, N.J.) |
GEN-1 |
Interleukin-12 |
Stage III and IV ovarian cancer |
Final clinical and translational research data from its OVATION phase Ib dose-escalating trial showed that the intraperitoneal treatment in conjunction with neoadjuvant chemotherapy resulted in dose-dependent increases in IL-12 and interferon-gamma levels that were predominantly in the peritoneal fluid compartment with little to no changes observed in the patients' systemic circulation; results from the first 14 patients showed that two demonstrated a complete response, 10 demonstrated a partial response and two demonstrated stable disease, translating to a 100% disease control rate and an 86% objective response rate |
10/4/17 |
Celyad SA (Mont-Saint-Guibert, Belgium) |
CYAD-01 |
CAR T NKG2D |
Hematologic |
Early results from the first dose level in the hematological arm of its THINK trial, in which a dose level of 3x108 T cells were administered without any prior conditioning chemotherapy to a cohort of three patients with hematologic cancer – two with acute myeloid leukemia (AML) and one with multiple myeloma – showed one AML patient achieved a morphologic leukemia-free state after administration with the drug |
10/4/17 |
Checkpoint Therapeutics Inc. and TG Therapeutics Inc. (both in New York) |
CK-301 |
Anti-PD-L1 monoclonal antibody |
Recurrent or |
Dosed the first patient in a phase I trial |
10/6/17 |
Critical Outcome Technologies Inc. (London, Ontario) |
COTI-2 |
p53-dependent mechanism of action |
Head and neck squamous cell carcinoma (HNSCC) |
The first patient was dosed in the HNSCC arm of its phase I trial at a dosage level of 1 mg/kg |
10/12/17 |
Cstone Pharmaceuticals Co. Ltd. (Suzhou, China) |
CS-1001 |
Anti-PD-L1 |
Cancer |
Dosed the first patient in the phase I trial |
10/23/17 |
Curevac AG (Tübingen, Germany) |
RNAdjuvant |
Immunomodulator |
Tumors |
Initiated a phase I study |
10/30/17 |
Dynavax Technologies Corp. (Berkeley, Calif) |
DV-281 |
Inhaled Toll-like receptor 9 agonist |
Non-small-cell lung cancer, |
Dosed the first patient in a phase Ib dose-escalation trial |
10/20/17 |
Epizyme Inc. (Cambridge, Mass.) |
Tazemetostat |
Oral EZH2 inhibitor |
Relapsed or refractory INI1-negative |
Data from the dose escalation portion of its ongoing phase I trial in pediatric patients showed it was generally well-tolerated at all explored doses and that adverse events (AEs) were mostly mild to moderate; it showed anti-tumor activity across a range of INI1-negative cancers, including complete responses in patients with epithelioid sarcoma (n=1), chordoma (n=1) and atypical teratoid rhabdoid tumor (n=1) and a partial response in chordoma (n=1) |
10/30/17 |
Esanex Inc. (Indianapolis) |
SNX-5422 |
Hsp90 inhibitor |
Chronic lymphocytic leukemia with residual disease |
Dosed the first patient in the open-label trial testing it with Imbruvica |
10/20/17 |
Halozyme Therapeutics Inc. (San Diego) |
PEGPH20 |
Pegylated |
Locally advanced or metastatic cholangiocarcinoma and |
Started a phase Ib/II open-label, randomized trial, HALO-110-101 |
10/17/17 |
Halozyme Therapeutics Inc. (San Diego) |
PEGPH20 |
Pegylated |
Metastatic gastric or gastroesophageal junction |
The first patient was dosed in a phase Ib/II trial testing it with Tecentriq |
10/25/17 |
Hutchison China Meditech Ltd. (Shanghai) and Astrazeneca plc (London) |
Savolitinib |
Selective c-MET inhibitor |
EGFR mutation-positive |
Data from two phase Ib/II trials showed the addition of it, at 600 mg once a day, to osimertinib (80 mg, once daily) or gefitinib (250 mg, once daily), demonstrated preliminary anti-tumor activity in both trials |
10/19/17 |
Immunomet Therapeutics Inc. (Houston) |
IM-156 |
Oxidative phosphorylation, or OXPHOS, inhibitor |
Solid tumors |
Started a phase I trial |
10/4/17 |
Innate Pharma SA (Marseille, France) |
IPH-410 |
Anti-KIR3DL2 humanized |
Relapsed/ |
Final results of the dose-escalation part of the ongoing phase I study confirmed the safety profile and promising activity of it in the elderly and heavily pretreated patient population (n=25); the objective response rate (ORR) in the 20 patients with Sézary syndrome was 50%; the ORR lasting at least four months was 40%, the disease control rate was 90%, the median duration of response was 9.9 months and the median progression-free survival was 10.8 months; the recommended phase II dose was identified at 750 mg |
10/17/17 |
Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.) |
INO-5401 and INO-9012 |
T-cell activating immunotherapy encoding multiple antigens and immune activator encoding IL-12 |
Advanced |
Started a phase Ib/II immuno-oncology trial to evaluate them in combination with Tecentriq |
10/17/17 |
Loxo Oncology Inc. (Stamford, Conn.) |
Larotrectinib |
TRK inhibitor |
Tumors |
Top-line results from the independent review committee assessment of data combined from a phase I adult trial, the phase II NAVIGATE trial, and the phase I/II pediatric SCOUT trial showed the overall response rate, as measured by RECIST v1.1., was 75% and 80% |
10/19/17 |
Mateon Therapeutics Inc. (South San Francisco) |
OXi-4503 |
Vascular disrupting agent |
Elapsed/ |
Data from the fifth dose cohort of its phase Ib dose-ranging study of it in combination with cytarabine showed one of the patients showing disease remission discontinued the study due to an unrelated adverse event; the other patient continued to receive treatment with an additional two cycles of it and remains in complete remission with a cytogenetic complete response; it continues to show a favorable safety profile |
10/31/17 |
Minneamrita (Tampa, Fla.) |
Minnelide |
Compound derived from the thunder God vine (Tripterygium wilfordii) |
Advanced cancers |
The first patient has received treatment in a phase I trial |
10/25/17 |
Miragen Therapeutics Inc. (Boulder, Colo.) |
MRG-106 |
MicroRNA-155 inhibitor |
Mycosis cutaneous |
Interim results from the first part of a phase I study, which treated patients with 75-mg injections directly into a specific lesion, showed a decrease in lesion size for all injected lesions; data from the second part, a multiple dose-escalation design to evaluate 300-mg, 600-mg or 900-mg subcutaneous or intravenous administrations, showed that 22 of 23 patients (96%) treated systemically showed improvement in total skin disease as measured by the maximal change in each patient's modified Severity Weighted Assessment Tool (mSWAT) score; the therapy was generally well-tolerated |
10/16/17 |
Molecular Partners AG (Zurich) |
MP-0274 |
Protein therapeutic |
HER2-positive cancer |
The first patient was dosed in the phase I study |
10/13/17 |
Mundipharma EDO GmbH (Basel, Switzerland) |
Tinostamus-tine |
Alkylating |
Solid tumors |
Started a U.S. phase I/II study to determine the safety, tolerability, maximum dose and optimal dosing schedule in patients who have progressed after at least one line of therapy and no other standard therapy with proven clinical benefit is available |
10/11/17 |
Nantkwest Inc. (Culver City, Calif.) |
haNK cell therapy |
Natural killer cells |
Metastatic or locally advanced solid tumors |
Advanced into the clinic with a phase I study |
10/3/17 |
Oric Pharmaceuticals Inc. (South San Francisco) |
ORIC-101 |
Small-molecule inhibitor of the |
Tumors |
The first subject in a phase Ia study has been dosed |
10/30/17 |
Pharmamar SA (Madrid) |
Zepsyre |
Inhibitor of RNA polymerase II |
Relapsed small-cell lung cancer |
Data from a phase I/II trial showed treatment of patients with it and doxorubicin produced a progression-free survival (PFS) of 5.3 months, better than historical data of topotecan that produces PFS between 3.1 and 3.5 months; platinum-sensitive patients fared better with a PFS of up to 6.2 months compared to historical data of 3.25 to 4.3 months; the objective response rate for all patients taking the combination was 37%, compared to historical data of topotecan in relapsed disease ranging between 17% and 24% |
10/19/17 |
Pharmamar SA (Madrid, Spain) |
PM-1183 |
Lurbinectedin |
Relapsed small-cell lung cancer |
Data from a phase I/II trial combining it with doxorubicin showed that patients reached a progression-free survival (PFS) – 5.3 months – that compares favorably with historical data of topotecan as a single agent (the PFS varies between 3.1 months and 3.5 months); the objective response rate of 37% observed in patients from the combination compares to historical data of topotecan in relapsed disease of between 17% and 24%; in platinum-sensitive patients, the PFS observed in patients treated with the combination increased up to 6.2 months; with topotecan, historical data in those patients saw a PFS ranging from 3.25 months to 4.3 months |
10/11/17 |
Pieris Pharmaceuticals Inc. (Boston) |
PRS-343 |
Bispecific antibody-Anticalin fusion protein |
Advanced or |
The first patient was dosed in its phase I trial |
10/3/17 |
Radius Health Inc. (Waltham, Mass.) |
RAD140 |
Nonsteroidal selective androgen receptor modulator |
Hormone |
Enrolled the first patient in its phase I trial testing approximately 40 patients |
10/2/17 |
Sun Biopharma Inc. (Minneapolis) |
SBP-101 |
Polyamine compound |
Previously |
Completed patient enrollment in the phase Ia dose-escalation safety study; the data safety monitoring board recommended a safe and well-tolerated dose level to be used for further clinical development |
10/5/17 |
Targovax ASA (Oslo, Norway) |
TG-01/GM-CSF |
Antigen-specific cancer |
Resected |
Data from the second cohort of a phase I/II trial of patients with KRAS mutations, in combination with gemcitabine as adjuvant therapy, showed that one year after surgery, all 13 patients were alive and 11 of the 13 generated an immune response |
10/13/17 |
Tocagen, Inc. (San Diego) |
Toca-511 and Toca FC |
Vocimagene |
Brain cancer |
More than a quarter of brain cancer patients participating in a phase I gene therapy trial survived at least three years after treatment; they demonstrated a survival benefit across all high-grade glioma (HGG) patients, not just those with specific genetic mutations; in time, some partial responses also converted into complete responses |
10/30/17 |
Tracon Pharmaceuticals Inc. (San Diego) |
TRC-105 |
Carotuximab |
Stage IV |
Initial data from nine patients in a phase Ib study of it in combination with paclitaxel/carboplatin and Avastin showed partial responses by RECIST 1.1 occurred in three of eight (37%) evaluable patients, including one patient who had an 81% reduction in tumor volume; dose escalation from 8 mg/kg to 10 mg/kg proceeded without dose-limiting toxicity |
10/17/17 |
Transgene SA (Strasbourg, France) |
TG-6002 |
Oncolytic virus |
Recurrent |
The first patient has been treated in the first-in-human Oncovirac trial |
10/27/17 |
Vaccinex Inc. (Rochester, N.Y.) |
VX15-2503 |
Anti-semaphorin 4D IgG4 monoclonal antibody (MAb) |
Advanced |
The first patient was dosed in a phase Ib/II study |
10/27/17 |
X4 Pharmaceuticals Inc. (Cambridge, Mass.) |
X4P-001-IO |
CXCR4 inhibitor |
Advanced clear cell renal cell |
Phase I part of the phase I/II study of it combined with VEGF inhibitor Inlyta showed an objective response rate (ORR) of 29% (4/14), including one patient with a confirmed complete response; the combo achieved a disease control rate of 93% (13/14); about a third of patients entering the study had received just one prior line of therapy, with the majority having tried two |
10/31/17 |
Xbiotech Inc. (Austin, Texas) |
Onivyde |
Irinotecan liposome injection |
Advanced |
Enrolled the first patient into a phase I single arm study |
10/19/17 |
Ziopharm Oncology Inc. (Boston) |
Ad-RTS-hIL-12 |
Gene therapy designed to control the expression of human interleukin 12 |
Pediatric brain tumors |
The first patient was dosed in a phase I study |
10/17/17 |
Cardiovascular | |||||
Argenx NV (Breda, the Netherlands) |
ARGX-113 |
Fc-portion of an antibody designed using ABDEG engineering technology (subcutaneously administered ) |
Myasthenia gravis |
The first subject has been dosed in a phase I trial |
10/31/17 |
Global Blood Therapeutics Inc. (South San Francisco) |
GBT-440 |
Hemoglobin |
Sickle cell disease |
Data from the phase I/II case study of a 21-year-old woman with HbSC disease and a past medical history of vaso-occlusive crisis who received it orally once daily for two months at a dose of 600 mg and subsequently for four months at a dose of 900 mg, showed her baseline hemoglobin increased by 2.2 g/dl, from 10 g/dL to 12.2 g/dL (within the normal range) at six months; her unconjugated bilirubin returned to normal, decreasing by 57%, and her baseline reticulocyte count decreased by 17% |
10/12/17 |
Central nervous system | |||||
Abide Therapeutics Inc. (San Diego) |
ABX-1431 |
Monoacylglycerol lipase inhibitor |
Neuromyelitis optica and related neuroinflammatory disorders associated with central pain |
Dosed the first patient in its phase Ib trial |
10/11/17 |
Asterias Biotherapeutics Inc. (Fremont, Calif.) |
AST-OPC1 |
Oligodendrocyte progenitor |
Cervical spinal cord injury |
New 12-month data from the first efficacy cohort in an ongoing phase I/IIa SCiStar study showed 67% (four of six) of cohort 2 (AIS-A injuries administered 10 million AST-OPC1 cells) subjects have recovered two or more motor levels on at least one side through 12 months, which is more than double the rates of recovery seen in both matched historical controls and published data in a similar population |
10/3/17 |
Biscayne Neurothera-peutics Inc. (Miami) |
BIS-001ER |
Huperzine A |
Epilepsy |
Results from a phase Ib trial showed it met pre-defined endpoints, including dosing frequency, serum drug exposure and safety |
10/20/17 |
Intra-Cellular Therapies Inc. (New York) |
ITI-214 |
Selective PDE1 inhibitor |
Parkinson's |
Started enrollment in the phase I/II trial |
10/4/17 |
Ionis Pharmaceuticals Inc. (Carlsbad, Calif.) |
IONIS-MAPTRx |
Antisense drug selectively reducing the production of microtubule-associated protein tau |
Mild Alzheimer's disease |
Started a phase I/IIa trial |
10/16/17 |
Pain Therapeutics Inc. (Austin, Texas) |
PTI-125 |
Oral, small molecule |
Alzheimer's |
Completed a phase I trial and had favorable pharmacokinetics that justify further drug development |
10/25/17 |
Titan Pharmaceuticals Inc. (South San Francisco) |
Ropinirole implant |
Dopamine agonist |
Idiopathic Parkinson's |
The first patient was treated in a phase I/II trial |
10/12/17 |
Voyager Therapeutics Inc. (Cambridge, Mass.) |
VY-AADC01 |
Gene therapy |
Advanced Parkinson's |
Results from the ongoing phase Ib trial demonstrated durable, dose-dependent and time-dependent improvements across multiple measures of patients' motor function after a one-time administration of it, and with meaningfully lower doses of oral levodopa |
10/18/17 |
Xenon Pharmaceuticals Inc. (Burnaby, British Columbia) |
XEN-1101 |
Orally administered Kv7 potassium channel opener |
Epilepsy |
Started a phase I trial and the first subject was dosed |
10/18/17 |
Gastrointestinal | |||||
Seres Therapeutics Inc. (Cambridge, Mass.) |
SER-287 |
Preparation of highly purified bacterial spores |
Mild to moderate ulcerative colitis |
Top-line results from a phase Ib placebo-controlled induction study in 58 patients who were failing current therapies demonstrated that it resulted in a benefit in clinical remission rates, and an improvement in mucosal appearance by endoscopy; when administered daily, it showed a 40% clinical remission vs. 21.4% for the weekly administered cohort (vancomycin preceded SER-287 for both), and endoscopic improvements were higher with daily vs. weekly dosing (40% vs. 28.6%) |
10/3/17 |
Infection | |||||
3-V Biosciences Inc (Menlo Park, Calif.) |
TVB-2640 |
Fatty acid synthase inhibitor |
Nonalcoholic fatty liver disease and nonalcoholic steatohepatitis (NASH) |
Data from a phase I trial showed that it directly inhibits hepatic lipogenesis in healthy males who have characteristics of metabolic syndrome |
10/17/17 |
Alopexx Vaccine LLC (Concord, Mass.) |
AV0328 |
Broad-spectrum anti-microbial |
Infection |
Completed a phase I trial in 16 healthy volunteers; at the two highest doses, it increased antibody titers against PNAG; it induced robust bactericidal killing of N. gonorrhea, including antibiotic-resistant strains, and N. meningitidis (serogroups A, B, C, W, Y), as well as opsonization of S. pneumoniae, multi-drug resistant Klebsiella pneumonia, colistin and multi-drug resistant strains of E. coli and S. aureus, including MRSA clinical isolates |
10/26/17 |
Amplyx Pharmaceuticals Inc. (San Diego) |
APX-001 |
Oral and IV broad-spectrum antifungal |
Fungal infections |
Results from two phase I studies showed that all doses were safe and well-tolerated, with most adverse events mild, transitory and resolving without intervention; target exposures of it for efficacy against Candida and |
10/10/17 |
Arsanis Inc. (Waltham, Mass.) |
ASN-100 |
IV human IgG1 monoclonal |
Pneumonia |
Pharmacokinetic data from a phase I trial showed that significant lung concentrations of it were detected in lung epithelial lining fluid (ELF) of healthy volunteers within one day and out to 30 days after dosing |
10/10/17 |
Contravir Pharmaceuticals Inc. (Edison, N.J.) |
TXL |
Tenofovir exalidex |
Chronic hepatitis B infection |
Dosed the first patient in its phase I trial |
10/20/17 |
Entasis Therapeutics Inc. (Waltham, Mass.) |
ETX-2514 |
Broad-spectrum and potent inhibitor of class A, C, and D beta-lactamases |
Carbapenem-resistant |
Results from its four-part, phase I study in healthy subjects showed that it, either alone or in combination with sulbactam (SUL) and/or imipenem/cilastatin (IMP), was generally well-tolerated; the general safety profile was unchanged when co-administered, as a single dose, with SUL, with IMP, and with SUL and IMP |
10/10/17 |
Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.) |
GLS-5700 |
DNA-based vaccine |
Zika virus |
Results from the phase I trial testing showed it induced binding antibodies in all 40 participants after a three-dose vaccination regimen and in 95% of participants after two doses of vaccine |
10/6/17 |
Spero Therapeutics Inc. (Cambridge, Mass) |
SPR-994 |
Oral carbapenem antibiotic |
Healthy subjects |
Started a phase I safety, tolerability, and |
10/23/17 |
Spero Therapeutics Inc. (Cambridge, Mass.) |
SPR-741 |
Multi-drug resistant anti-bacterial therapy |
Infection |
Data from its phase I, single ascending-dose and multiple ascending-dose trial showed that it was well-tolerated in single doses of up to 800 mg and at doses up to 600 mg every eight hours for 14 days |
10/4/17 |
Vaxart Inc. (South San Francisco) |
VXA-G1.1-NN |
Norovirus tablet vaccine |
Norovirus |
Data from a phase Ib trial showed that the group that received the highest of four doses, 100% of subjects had a significant increase in IgA and IgG antibody secreting cells, and 90% of subjects had a two-fold or greater increase of norovirus blocking antibody titers in serum 28 days after dosing |
10/31/17 |
Miscellaneous | |||||
Abeona Therapeutics Inc. (New York) |
ABO-102 |
Intravenous associated virus (AAV) gene therapy |
Sanfilippo |
One-year data from cohort 1 of its ongoing phase I/II trial showed the three patients in cohort 1 experienced a 69.3% reduction in cerebral spinal fluid heparan sulfate; the patients had normalization of liver volumes of 80 %age points; MRI data showed evidence of stabilization of area of deep brain architecture in the thalamus and putamen; patients also experienced a stabilization of cognitive assessments as measured by Leiter-R non-verbal IQ and Vineland scales |
10/9/17 |
Abeona Therapeutics Inc. (New York) |
ABO-102 |
Intravenous |
Sanfilippo |
Two patients were enrolled in the third cohort of its phase I/II trial testing a dose of 3 x 1013 vg/kg; the first patient, who has reached 30 days post injection, saw a 66.7% reduction in CSF heparan sulfate and a 92.3% reduction in urinary heparan sulfate: the liver volume of that patient was reduced by 76%. |
10/12/17 |
Acura Pharmaceuticals Inc. (Palatine, Ill.) |
LTX-03 |
Hydrocodone |
Healthy subjects |
Top-line results from clinical study AP-LTX-300 demonstrated rapid release of it from the |
10/10/17 |
Avexis Inc. (Chicago) |
AVXS-101 |
Gene therapy |
Spinal muscular atrophy type 1 |
Data from its phase I trial showed, as of the Aug. 7 cutoff, all patients were alive and not on extended use of ventilation support at 20 months of age; nine of the 12 patients could sit unassisted for 30 seconds or more, while 11 of the 12 achieved sustained CHOP-INTEND scores of more than 40 for a mean of 18.8 months |
10/4/17 |
Biotime Inc. (Alameda, Calif.) |
Premvia |
Carrier for stromal vascular fraction cells |
Age-related |
The first patient was treated in an investigator-led trial |
10/12/17 |
Capricor Therapeutics Inc. (Los Angeles) |
CAP-1002 |
Allogeneic |
Duchenne |
Results from the first six months of follow-up data from the randomized 12-month phase I/II Hope trial showed teens and young men in the advanced stages of DMD experienced |
10/5/17 |
Cara Therapeutics Inc. (Stamford, Conn.) |
CR845 |
Peripheral kappa opioid receptor |
Stage III-V chronic kidney disease |
Dosed the first patient in its phase I |
10/26/17 |
Cellect Biotechnology Ltd. (Tel Aviv, Israel) |
Apograft |
Isolates stem cells through apoptosis |
Graft vs. host |
Results from a study of 20 patients testing it on stem cells derived from fat tissue showed it increased the number and activity of the fat-derived stem cells from samples obtained through liposuction |
10/26/17 |
Crinetics Pharmaceuticals Inc. (San Diego) |
CRN-00808 |
Nonpeptide |
Acromegaly |
Initiated a double-blind, randomized, placebo-controlled, single- and multiple-dose phase I study |
10/27/17 |
Enanta Pharmaceuticals Inc (Watertown, Mass.) |
EDP-305 |
FXR agonist |
Non-alcoholic fatty liver disease |
Pharmacokinetic data from a phase Ia/b trial supports once daily oral dosing of it; it increased FGF19 levels and reduced C4 levels as expected for a drug activating FXR |
10/24/17 |
Greenovation Biotech GmbH (Freiburg, Germany) |
Moss-aGal |
Recombinant form of human alpha-galactosidase |
Fabry disease |
Completed its phase I study; the single dose was well-tolerated |
10/18/17 |
Sound Pharmaceuticals Inc. (Seattle) |
SPI-1005 |
Mimics and induces glutathione |
Meniere's disease (MD) |
Top-line results from its phase Ib trial showed it demonstrated excellent safety, tolerability and improvements in both auditory and vestibular symptoms that define MD; treated subjects (55%) showed clinically relevant improvements in low frequency hearing vs. placebo-treated subjects (10%), which was significant (p <0.05); treatment also resulted in improvements in word recognition, tinnitus loudness and vertigo severity vs. placebo |
10/4/17 |
Respiratory | |||||
Aobiome LLC (Cambridge, Mass.) |
Ammonia |
Prophylaxis therapy |
Seasonal allergic rhinitis |
Started enrollment in the phase Ib/IIa trial |
10/4/17 |
Asit Biotech SA (Brussels, Belgium) |
gp-ASIT+ |
Allergy |
Grass pollen |
Phase I data showed that five consecutive injections were safe and well-tolerated, and the treatment was associated with the induction of grass pollen-specific antibodies capable of blocking the allergic reactions responsible for grass pollen rhinitis in humans |
10/13/17 |
Asit Biotech SA (Brussels, Belgium) |
hdm-ASIT+ |
Uses the ASIT |
House dust mite rhinitis |
Results of a follow-up study showed, out of the 36 initially randomized patients (9 placebo and 27 treated), in the phase I/II trial, 5 placebo and 14 treated patients underwent one complementary medical visit 8 months after the end of the treatment; the study did not demonstrate a long-term improvement of parameters |
10/25/17 |
Global Blood Therapeutics Inc. (South San Francisco) |
GBT-440 |
Hemoglobin |
Idiopathic |
Discontinued its program based on results from three proof-of-concept studies including a phase I study in healthy volunteers called Basecamp and two phase IIa studies in patients with IPF called GBT440-006 and Zephyr |
10/24/17 |
Synspira LLC (Cambridge, Mass.) |
SNSP-113 |
Modified |
Cystic fibrosis |
Started a multicenter phase Ia study |
10/19/17 |
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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. |