Company (location)

Product

Description

Indication

Status

Date

Autoimmune

Atara Biotherapeutics Inc. (South San Francisco)

ATA-188

T-cell immunotherapy

Progressive or relapsing-remitting multiple
sclerosis (MS)

Started a phase I study

10/20/17

Atara Biotherapeutics Inc. (South San Francisco)

ATA-190

Epstein-Barr virus (EBV) specific
T-cell immunotherapy

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
next-generation oxazolidinone

Mild to moderate acne vulgaris

Phase I study was completed and results
demonstrated that it was well-tolerated with minimal systemic absorption

10/11/17

Prothena Corp. plc ( Dublin)

PRX003

Monoclonal
antibody targeting CD146 that is expressed on
T helper 17 cells (Th17)

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
monocytogenes-based
immunotherapy

Advanced or
metastatic
non-small-cell lung cancer

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
esophageal or gastroesophageal junction cancers

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
melanoma

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
modulating
compound

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
therapy targeting B-cell maturation antigen

Relapsed/
refractory
multiple
myeloma

Treated the first patient in its phase I CRB-402 trial

10/2/17

Bristol-Myers Squibb Co. (New York)

Opdivo

Nivolumab

Previously
treated small-cell lung cancer

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
cancer and
metastatic colorectal cancer

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,
pancreatic
cancer, colorectal cancer or triple-negative breast 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
ether-drug
conjugate

Relapsed or refractory
multiple
myeloma

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
gene-mediated immunotherapy

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
therapy

Hematologic
cancer

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
metastatic
cancers

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
monoclonal
antibody

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,
pancreatic
cancer, colorectal cancer or triple-negative breast 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
molecularly defined solid tumor

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
hyaluronidase

Locally advanced or metastatic cholangiocarcinoma and
gallbladder
adenocarcinoma

Started a phase Ib/II open-label, randomized trial, HALO-110-101

10/17/17

Halozyme Therapeutics Inc. (San Diego)

PEGPH20

Pegylated
hyaluronidase

Metastatic gastric or gastroesophageal junction
cancer

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
non-small-cell lung cancer

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
cytotoxicity-
inducing antibody

Relapsed/
refractory
cutaneous T-cell lymphoma

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
bladder cancer

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
containing
tropomyosin receptor kinase fusions

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/
refractory acute myeloid leukemia or myelodysplastic syndromes

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
fungoides (MF) form of

cutaneous
T-cell lymphoma

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
deacetylase-i
nhibiting molecule

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
glucocorticoid receptor

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
metastatic HER2-positive solid tumors

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
receptor positive breast cancer

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
treated locally advanced or
metastatic
pancreatic ductal adenocarcinoma

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
immunotherapy

Resected
adenocarcinoma of the pancreas

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
amiretrorepvec and 5-fluorouracil

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
nonsquamous-cell lung cancer

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
glioblastoma

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
non-small-cell lung cancer

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
carcinoma

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
pancreatic
adenocarcinoma and cachexia

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
modifier

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
population derived from human
embryonic stem cells

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
disease

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
disease

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
disease

The first patient was treated in a phase I/II trial

10/12/17

Voyager Therapeutics Inc. (Cambridge, Mass.)

VY-AADC01

Gene therapy
candidate

Advanced Parkinson's
disease

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
vaccine

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 
Aspergillus were exceeded; the average plasma half-life was about 2.5 days and the oral formulation showed greater than 90% bioavailability

10/10/17

Arsanis Inc. (Waltham, Mass.)

ASN-100

IV human IgG1 monoclonal
antibody

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 
Acinetobacter
baumannii 
infections

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
pharmacokinetics study

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
injection of adeno-

associated virus (AAV) gene therapy

Sanfilippo
syndrome type A (mucopolysaccharidosis type IIIA)

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
injection of adeno-associated virus (AAV) gene therapy

Sanfilippo
syndrome type A (mucopolysaccharidosis type IIIA)

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
bitartrate and
acetaminophen

Healthy subjects

Top-line results from clinical study AP-LTX-300 demonstrated rapid release of it from the
microparticle formulation, providing expected therapeutic drug levels in the bloodstream for a single dose

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
volume loss in the face

The first patient was treated in an investigator-led trial

10/12/17

Capricor Therapeutics Inc. (Los Angeles)

CAP-1002

Allogeneic
cardiosphere-derived cells

Duchenne
muscular
dystrophy (DMD)

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
meaningful improvements in cardiac and upper limb function after a single dose

10/5/17

Cara Therapeutics Inc. (Stamford, Conn.)

CR845

Peripheral kappa opioid receptor
agonist

Stage III-V chronic kidney disease

Dosed the first patient in its phase I
pharmacokinetic and safety trial

10/26/17

Cellect Biotechnology Ltd. (Tel Aviv, Israel)

Apograft

Isolates stem cells through apoptosis

Graft vs. host
disease

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
somatostatin
agonist

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
peroxidase

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
oxidizing
bacteria

Prophylaxis therapy

Seasonal allergic rhinitis

Started enrollment in the phase Ib/IIa trial

10/4/17

Asit Biotech SA (Brussels, Belgium)

gp-ASIT+

Allergy
immunotherapy

Grass pollen
rhinitis

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
technology platform

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
modifier

Idiopathic
pulmonary
fibrosis (IPF)

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
polysaccharide
molecule

Cystic fibrosis

Started a multicenter phase Ia study

10/19/17


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.

 

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